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1. Fizazi K, Prow DM, Do KA, Wang X, Finn L, Kim J, Daliani D, Papandreou CN, Tu SM, Millikan RE, Pagliaro LC, Logothetis CJ, Amato RJ: Alternating dose-dense chemotherapy in patients with high volume disseminated non-seminomatous germ cell tumours. Br J Cancer; 2002 May 20;86(10):1555-60
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  • [Title] Alternating dose-dense chemotherapy in patients with high volume disseminated non-seminomatous germ cell tumours.
  • The aim of this phase II study was to assess the efficacy and toxicity of a dose-dense alternating chemotherapy regimen in this subset of patients.
  • Forty-two patients (72.4%) had a complete response to therapy.
  • With a median follow-up time of 31 months, the 3-year progression-free survival rate was 71% (95% confidence interval, 60 to 84%) and the 3-year overall survival rate was 73% (95% confidence interval: 62 to 86%).
  • Early side effects included mainly grade 4 haematologic toxicity (neutropaenia in 79% of patients, thrombocytopaenia in 69%, anaemia in 22%), grade 4 stomatitis (19%), and four early deaths (7% of patients), at least partially related to toxicity.
  • Because outcomes with this regimen compare favourably with outcome after standard therapy, dose-dense chemotherapy should be further investigated in this subset of patients.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Germinoma / drug therapy. Seminoma / drug therapy. Testicular Neoplasms / drug therapy
  • [MeSH-minor] Adolescent. Adult. Anemia, Refractory, with Excess of Blasts / chemically induced. Biomarkers, Tumor / blood. Bleomycin / administration & dosage. Bleomycin / adverse effects. Cisplatin / administration & dosage. Cisplatin / adverse effects. Cyclophosphamide / administration & dosage. Cyclophosphamide / adverse effects. Dactinomycin / administration & dosage. Dactinomycin / adverse effects. Disease-Free Survival. Doxorubicin / administration & dosage. Doxorubicin / adverse effects. Etoposide / administration & dosage. Etoposide / adverse effects. Gastrointestinal Diseases / chemically induced. Hematologic Diseases / chemically induced. Humans. Lung Neoplasms / drug therapy. Lung Neoplasms / secondary. Male. Methotrexate / administration & dosage. Methotrexate / adverse effects. Middle Aged. Peripheral Nervous System Diseases / chemically induced. Prognosis. Prospective Studies. Remission Induction. Survival Analysis. Survival Rate. Treatment Outcome. Vincristine / administration & dosage. Vincristine / adverse effects

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  • [Copyright] comCopyright 2002 Cancer Research UK
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  • (PMID = 12085204.001).
  • [ISSN] 0007-0920
  • [Journal-full-title] British journal of cancer
  • [ISO-abbreviation] Br. J. Cancer
  • [Language] eng
  • [Publication-type] Clinical Trial; Clinical Trial, Phase II; Journal Article
  • [Publication-country] Scotland
  • [Chemical-registry-number] 0 / Biomarkers, Tumor; 11056-06-7 / Bleomycin; 1CC1JFE158 / Dactinomycin; 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide; Q20Q21Q62J / Cisplatin; YL5FZ2Y5U1 / Methotrexate; BOP-CISCA-POMB-ACE regimen
  • [Other-IDs] NLM/ PMC2746595
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2. Fenaux P, Gattermann N, Seymour JF, Hellström-Lindberg E, Mufti GJ, Duehrsen U, Gore SD, Ramos F, Beyne-Rauzy O, List A, McKenzie D, Backstrom J, Beach CL: Prolonged survival with improved tolerability in higher-risk myelodysplastic syndromes: azacitidine compared with low dose ara-C. Br J Haematol; 2010 Apr;149(2):244-9
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  • In the phase III AZA-001 trial, low-dose cytarabine (LDara-C), the most widely used low-dose chemotherapy in patients with higher-risk myelodysplastic syndrome (MDS) who are ineligible for intensive treatment, was found to be associated with poorer survival compared with azacitidine.
  • This analysis further compared the efficacy and the toxicity of these two drug regimens.
  • Azacitidine prolonged overall survival versus LDara-C in patients with poor cytogenetic risk, presence of -7/del(7q), and French-American-British subtypes refractory anaemia with excess blasts (RAEB) and RAEB in transformation.
  • When analyzed per patient year of drug exposure, azacitidine treatment was associated with fewer grade 3-4 cytopenias and shorter hospitalisation time than LDara-C in these higher-risk MDS patients.
  • [MeSH-major] Antimetabolites, Antineoplastic / therapeutic use. Azacitidine / therapeutic use. Cytarabine / therapeutic use. Myelodysplastic Syndromes / drug therapy
  • [MeSH-minor] Adult. Aged. Aged, 80 and over. Drug Administration Schedule. Erythrocyte Transfusion. Female. Humans. Male. Middle Aged. Survival Analysis. Treatment Outcome

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  • [ErratumIn] Br J Haematol. 2010 Jun;149(6):919
  • (PMID = 20136825.001).
  • [ISSN] 1365-2141
  • [Journal-full-title] British journal of haematology
  • [ISO-abbreviation] Br. J. Haematol.
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / K24 CA111717
  • [Publication-type] Clinical Trial, Phase III; Comparative Study; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Antimetabolites, Antineoplastic; 04079A1RDZ / Cytarabine; M801H13NRU / Azacitidine
  • [Other-IDs] NLM/ NIHMS490238; NLM/ PMC4000023
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3. Raza A, Mehdi M, Mumtaz M, Ali F, Lascher S, Galili N: Combination of 5-azacytidine and thalidomide for the treatment of myelodysplastic syndromes and acute myeloid leukemia. Cancer; 2008 Oct 1;113(7):1596-604
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  • [Title] Combination of 5-azacytidine and thalidomide for the treatment of myelodysplastic syndromes and acute myeloid leukemia.
  • BACKGROUND: The treatment of myelodysplastic syndromes (MDS) remains a challenge to the clinician despite recent advances.
  • Many patients will either not respond or will have only limited and/or brief responses to single-agent therapy.
  • Six patients had refractory anemia (RA), 2 patients had RA with ringed sideroblasts, 10 patients had RA with excess blasts (RAEB), 1 patient had RAEB in transformation, 4 patients had chronic myelomonocytic leukemia, 1 patient had chronic idiopathic myelofibrosis, and 16 patients had AML.
  • It was noteworthy that 9 of 14 patients with AML had a history of prior MDS, 2 of 9 patients achieved a CR, 4 of 9 patients had HI (HI-E and bilineage HI), and 1 patient had stable disease and was continuing treatment.
  • CONCLUSIONS: The current findings indicated that a combination of low-dose AZA and thalidomide was well tolerated and was effective therapy for the treatment of patients with MDS and AML arising from prior MDS.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Azacitidine / administration & dosage. Leukemia, Myeloid, Acute / drug therapy. Myelodysplastic Syndromes / drug therapy. Thalidomide / administration & dosage
  • [MeSH-minor] Bone Marrow Cells / pathology. Drug Administration Schedule. Humans. Oligonucleotide Array Sequence Analysis. Pilot Projects. Survival Analysis. Treatment Outcome


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4. Germing U, Strupp C, Kuendgen A, Aivado M, Giagounidis A, Hildebrandt B, Aul C, Haas R, Gattermann N: Refractory anaemia with excess of blasts (RAEB): analysis of reclassification according to the WHO proposals. Br J Haematol; 2006 Jan;132(2):162-7

  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Refractory anaemia with excess of blasts (RAEB): analysis of reclassification according to the WHO proposals.
  • The French-American-British (FAB) classification assigns patients with myelodysplastic syndromes to the category of refractory anaemia with excess blasts (RAEB) if they have a medullary blast count of 5-20%, and/or a peripheral blast count of 2-5%.
  • The new World Health Organization (WHO) classification subdivides RAEB into RAEB I with a medullary blast count < or =10% and a peripheral blast count < or =5% and RAEB II with >10% medullary and/or >5% peripheral blasts.
  • RAEB II is also diagnosed if Auer rods are present.
  • In 558 patients, we analysed these subtypes of RAEB in terms of haematological characteristics, karyotype anomalies and prognosis.
  • RAEB I was diagnosed in 256 and RAEB II in 302 patients.
  • In the RAEB II group, 22% of patients had >5% peripheral blasts or the presence of Auer rods.
  • The median survival was 16 months for RAEB I as compared with 9 months for RAEB II.
  • No significant differences were identified between the RAEB subtypes with respect to clinical, morphological, haematological and cytogenetic parameters.
  • The survival data support the WHO reclassification of RAEB based on peripheral and medullary blast counts and Auer rods.
  • The WHO classification is useful for diagnosis and provides risk stratification, supported by cytogenetic data for clinical decision making, identifying those RAEB patients with an unfavourable prognosis who should be offered chemotherapy or stem cell transplantation.
  • [MeSH-major] Anemia, Refractory, with Excess of Blasts / classification

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  • [ErratumIn] Br J Haematol. 2006 Jul;134(2):247
  • (PMID = 16398650.001).
  • [ISSN] 0007-1048
  • [Journal-full-title] British journal of haematology
  • [ISO-abbreviation] Br. J. Haematol.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] England
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5. Raza A, Qawi H, Lisak L, Andric T, Dar S, Andrews C, Venugopal P, Gezer S, Gregory S, Loew J, Robin E, Rifkin S, Hsu WT, Huang RW: Patients with myelodysplastic syndromes benefit from palliative therapy with amifostine, pentoxifylline, and ciprofloxacin with or without dexamethasone. Blood; 2000 Mar 1;95(5):1580-7
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  • [Title] Patients with myelodysplastic syndromes benefit from palliative therapy with amifostine, pentoxifylline, and ciprofloxacin with or without dexamethasone.
  • Thirty-five patients with myelodysplastic syndrome (MDS) were registered on protocol MDS 96-02 and were receiving continuous therapy with pentoxifylline 800 mg 3 times a day and ciprofloxacin 500 mg twice a day by mouth; dexamethasone was added to the regimen for the partial responders and the nonresponders after 12 weeks at a dose of 4 mg by mouth every morning for 4 weeks.
  • Amifostine was administered intravenously 3 times a week at 3 dose levels (200 mg/M(2), 300 mg/M(2), and 400 mg/M(2)) to cohorts of 10 patients each.
  • Therapy has been continued for 1 year in responders.
  • Twenty-nine have completed at least 12 weeks of therapy and are available for response evaluation.
  • Of the 21 men and 8 women (median age, 67 years), 20 had refractory anemia (RA), 3 had RA with ringed sideroblasts (RARS), 5 had RA with excess blasts (RAEB), and 1 had chronic myelomonocytic leukemia (CMMoL).
  • Interestingly, the responses were frequently slow to appear, and continued improvement in counts was seen up to 12 months of therapy and beyond.
  • This study supports the feasibility of treating patients with MDS with the unique approach of cytoprotection and anticytokine therapies as well as the principle that prolonged commitment to treatment is desirable when noncytotoxic agents are administered. (Blood.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Myelodysplastic Syndromes / drug therapy. Palliative Care
  • [MeSH-minor] Adult. Aged. Aged, 80 and over. Amifostine / administration & dosage. Amifostine / adverse effects. Anorexia / chemically induced. Blood Cell Count / drug effects. Ciprofloxacin / administration & dosage. Ciprofloxacin / adverse effects. Dexamethasone / administration & dosage. Dexamethasone / adverse effects. Female. Gastrointestinal Diseases / chemically induced. Hematologic Diseases / chemically induced. Humans. Hypotension / chemically induced. Male. Middle Aged. Pentoxifylline / administration & dosage. Pentoxifylline / adverse effects. Treatment Outcome


6. Yin CC, Medeiros LJ, Bueso-Ramos CE: Recent advances in the diagnosis and classification of myeloid neoplasms--comments on the 2008 WHO classification. Int J Lab Hematol; 2010 Oct;32(5):461-76
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  • [Title] Recent advances in the diagnosis and classification of myeloid neoplasms--comments on the 2008 WHO classification.
  • Two new entities - refractory cytopenia with unilineage dysplasia and refractory cytopenia of childhood have been added to the group of myelodysplastic syndromes (MDS), and 'refractory anemia with excess blasts-1' has been redefined to emphasize the prognostic significance of increased blasts in the peripheral blood.
  • A list of cytogenetic abnormalities has been introduced as presumptive evidence of MDS in cases with refractory cytopenia but without morphologic evidence of dysplasia.
  • The term 'therapy-related myeloid neoplasms' is used to cover the spectrum of disorders previously known as t-AML, t-MDS, or t-MDS/MPN occurring as complications of cytotoxic chemotherapy and/or radiation therapy.
  • [MeSH-minor] Anemia, Refractory, with Excess of Blasts / classification. Humans. Leukemia, Myeloid, Acute / classification. Leukemia, Myeloid, Acute / diagnosis. Protein-Tyrosine Kinases / genetics

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  • (PMID = 20626469.001).
  • [ISSN] 1751-553X
  • [Journal-full-title] International journal of laboratory hematology
  • [ISO-abbreviation] Int J Lab Hematol
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / P30 CA016672
  • [Publication-type] Journal Article; Review
  • [Publication-country] England
  • [Chemical-registry-number] EC 2.7.10.1 / Protein-Tyrosine Kinases
  • [Other-IDs] NLM/ NIHMS691453; NLM/ PMC4452117
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7. Ten Oever J, Kuijper PH, Kuijpers AL, Dercksen MW, Vreugdenhil G: Complete remission of MDS RAEB following immunosuppressive treatment in a patient with Sweet's syndrome. Neth J Med; 2009 Sep;67(8):347-50
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  • [Title] Complete remission of MDS RAEB following immunosuppressive treatment in a patient with Sweet's syndrome.
  • We report on a patient with myelodysplastic syndrome (MDS), classified as refractory anaemia with excess of blasts-2, and histiocytoid Sweet's syndrome.
  • Inhibition of T-cell mediated myelosuppression by corticosteroids or a proapoptotic effect of doxycycline may have attributed as well.
  • [MeSH-major] Adrenal Cortex Hormones / therapeutic use. Anemia, Refractory, with Excess of Blasts / drug therapy. Anti-Bacterial Agents / therapeutic use. Doxycycline / therapeutic use. Immunosuppressive Agents / therapeutic use. Sweet Syndrome / complications
  • [MeSH-minor] Aged. Fatal Outcome. Humans. Killer Cells, Natural. Male. Recurrence. Remission Induction. Treatment Outcome

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  • (PMID = 19767665.001).
  • [ISSN] 1872-9061
  • [Journal-full-title] The Netherlands journal of medicine
  • [ISO-abbreviation] Neth J Med
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] Netherlands
  • [Chemical-registry-number] 0 / Adrenal Cortex Hormones; 0 / Anti-Bacterial Agents; 0 / Immunosuppressive Agents; N12000U13O / Doxycycline
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8. Terpos E, Mougiou A, Kouraklis A, Chatzivassili A, Michalis E, Giannakoulas N, Manioudaki E, Lazaridou A, Bakaloudi V, Protopappa M, Liapi D, Grouzi E, Parharidou A, Symeonidis A, Kokkini G, Laoutaris NP, Vaipoulos G, Anagnostopoulos NI, Christakis JI, Meletis J, Bourantas KL, Zoumbos NC, Yataganas X, Viniou NA, Greek MDS Study Group: Prolonged administration of erythropoietin increases erythroid response rate in myelodysplastic syndromes: a phase II trial in 281 patients. Br J Haematol; 2002 Jul;118(1):174-80
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  • Treatment with recombinant human erythropoietin (rHuEpo) improves anaemia in approximately 20% of patients with myelodysplastic syndromes (MDS).
  • We investigated the potential advantage of a prolonged administration of rHuEpo to achieve higher erythroid response rates (RR) in 281 MDS patients: 118 with refractory anaemia (RA), 77 with refractory anaemia and ringed sideroblasts (RARS), 59 with refractory anaemia with excess of blasts and blast count < 10% (RAEB-I), and 27 with RAEB and blast count between 11-20% (RAEB-II).
  • Response to treatment was evaluated after 12 and 26 weeks of therapy.
  • The overall RR was 45.1%; the RR for RA, RARS, RAEB-I and RAEB-II were 48.3%, 58.4%, 33.8% and 13% respectively.
  • A significant increase in RR was observed at week 26 in RA, RARS and RAEB-I patients, as the response probability increased with treatment duration.
  • [MeSH-major] Erythropoietin / therapeutic use. Myelodysplastic Syndromes / drug therapy
  • [MeSH-minor] Aged. Aged, 80 and over. Anemia, Refractory / drug therapy. Anemia, Refractory, with Excess of Blasts / drug therapy. Anemia, Sideroblastic / drug therapy. Female. Humans. Male. Middle Aged. Prospective Studies. Treatment Outcome


9. Lu Y, Wu T, Cao XY, Wang JB, Yin YM, Lu DP: [Effects of allogeneic hematopoietic stem cell transplantation on 60 patients with myelodysplastic syndrome]. Zhonghua Nei Ke Za Zhi; 2010 Mar;49(3):200-3
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  • DFS rates in RA/RAS/5q-, RCMD, RAEB-I/RAEB-II and acute myelocytic leukemia (AML) subgroups were 84.6%, 80.0%, 81.0%, 56.2%, respectively (P > 0.05).
  • DFS rates for percentages of blasts in bone marrow pre-transplant were 87.0%, 65.5%, 75.0% in < 5% blasts, 5% - 20% blasts, > 20% blasts subgroups, respectively (P > 0.05).
  • CONCLUSIONS: Since favorable clinical outcomes have been seen in all kinds of MDS by allo-HSCT, HSCT should be the first-line therapy for MDS.
  • No significant differences are found based on different stem cell donors and the percentages of bone marrow blasts pre-HSCT, unrelated or haploidentical donors should be important alternatives if there is no identical sibling available.
  • Chemotherapy before transplantation is not necessary except overt acute leukemia.
  • [MeSH-major] Hematopoietic Stem Cell Transplantation. Myelodysplastic Syndromes / therapy
  • [MeSH-minor] Adolescent. Adult. Disease-Free Survival. Female. Humans. Male. Middle Aged. Treatment Outcome. Young Adult


10. Potru R, Ahn J, Fung H, Cohen SM: A case of myelodysplastic syndrome in a liver transplant patient. Transplant Proc; 2009 Nov;41(9):3947-8
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  • In 2007, the patient developed weakness, malaise, and shortness of breath.
  • Bone marrow biopsy showed MDS, with refractory anemia and excess blasts-1.
  • The patient underwent chemotherapy and reduction in immunosuppression without a clinical response.
  • Our experience suggested that MDS, although rare, should be considered in the differential diagnosis of pancytopenia after OLT.
  • Once diagnosed, immunosuppression reduction, chemotherapy, and even stem cell transplantation may be the appropriate treatment in selected candidates.
  • [MeSH-major] Liver Cirrhosis / surgery. Liver Transplantation / adverse effects. Myelodysplastic Syndromes / diagnosis
  • [MeSH-minor] Anemia / drug therapy. Antiviral Agents / therapeutic use. Erythropoietin / therapeutic use. Hepatitis C / complications. Hepatitis C / drug therapy. Hepatitis C / surgery. Humans. Interferon-alpha / therapeutic use. Male. Middle Aged. Polyethylene Glycols / therapeutic use. Recombinant Proteins. Recurrence. Reoperation


11. Raj K, Ho A, Creamer JD, du Vivier AW, Salisbury JR, Mufti GJ: Complete response of deep neutrophilic dermatosis associated with myelodysplastic syndrome to 5-azacytidine. Br J Dermatol; 2007 May;156(5):1039-41
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  • We describe a patient in whom a deep neutrophilic dermatosis preceded evolution of disease from refractory anaemia to RAEB (refractory anaemia with excess blasts) and resolved completely on treating the disease with 5-azacytidine.
  • We suggest that 5-azacytidine should be considered in the treatment of immune mediated cutaneous manifestations of MDS.
  • [MeSH-major] Antimetabolites, Antineoplastic / therapeutic use. Azacitidine / therapeutic use. Skin Diseases / drug therapy
  • [MeSH-minor] Aged. Anemia, Refractory, with Excess of Blasts / complications. Head. Humans. Male. Neck. Remission Induction. Shoulder

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  • (PMID = 17408390.001).
  • [ISSN] 0007-0963
  • [Journal-full-title] The British journal of dermatology
  • [ISO-abbreviation] Br. J. Dermatol.
  • [Language] eng
  • [Grant] United Kingdom / Medical Research Council / / G84/6222
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Antimetabolites, Antineoplastic; M801H13NRU / Azacitidine
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12. Kuendgen A, Knipp S, Fox F, Strupp C, Hildebrandt B, Steidl C, Germing U, Haas R, Gattermann N: Results of a phase 2 study of valproic acid alone or in combination with all-trans retinoic acid in 75 patients with myelodysplastic syndrome and relapsed or refractory acute myeloid leukemia. Ann Hematol; 2005 Dec;84 Suppl 1:61-6
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  • [Title] Results of a phase 2 study of valproic acid alone or in combination with all-trans retinoic acid in 75 patients with myelodysplastic syndrome and relapsed or refractory acute myeloid leukemia.
  • Valproic acid (VPA) inhibits histone deacetylase activity and induces differentiation of acute myeloid leukemia (AML) blasts in vitro.
  • Median treatment duration was 4 months for VPA and 2 months for ATRA.
  • Response rates were strongly dependent on disease type according to WHO classification.
  • We found a response rate of 52% in MDS patients with a normal blast count (refractory sideroblastic anemia, refractory cytopenia with multilineage dysplasia, and refractory sideroblastic cytopenia with multilineage dysplasia).
  • The response rate was 6% in refractory anemia with excess blasts (I + II), 16% in AML, and 0% in chronic myelomonocytic leukemia.
  • For patients with high-risk MDS, VPA may be combined with chemotherapy or demethylating drugs.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Histone Deacetylase Inhibitors. Leukemia, Myeloid / drug therapy. Myelodysplastic Syndromes / drug therapy. Valproic Acid / therapeutic use
  • [MeSH-minor] Acute Disease. Adult. Aged. Aged, 80 and over. Cell Differentiation / drug effects. Female. Histone Deacetylases / drug effects. Humans. Kaplan-Meier Estimate. Male. Middle Aged. Remission Induction. Treatment Outcome. Tretinoin / administration & dosage


13. Ravoet C, Mineur P, Robin V, Debusscher L, Bosly A, André M, El Housni H, Soree A, Bron D, Martiat P: Farnesyl transferase inhibitor (lonafarnib) in patients with myelodysplastic syndrome or secondary acute myeloid leukaemia: a phase II study. Ann Hematol; 2008 Nov;87(11):881-5
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  • Lonafarnib was administered orally at a dose of 200 mg twice daily for three courses of 4 weeks (separated by 1 to 4 weeks without treatment).
  • Sixteen patients were included: FAB/RAEB (n = 10), RAEB-T (n = 2), sAML (n = 2) and chronic myelomonocytic leukaemia (CMML; n = 2); WHO/RAEB-1 (n = 4), RAEB-2 (n = 5), AML (n = 5), CMML (n = 2).
  • One patient died of infection, and the treatment was stopped in one other who developed atrial fibrillation.
  • [MeSH-major] Farnesyltranstransferase / antagonists & inhibitors. Leukemia, Myeloid, Acute / drug therapy. Myelodysplastic Syndromes / drug therapy. Piperidines / adverse effects. Pyridines / adverse effects


14. Kelaidi C, Eclache V, Fenaux P: The role of lenalidomide in the management of myelodysplasia with del 5q. Br J Haematol; 2008 Feb;140(3):267-78
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  • Defined by isolated del 5q and no excess of marrow blasts, the '5q- syndrome' is a specific type of myelodysplastic syndrome (MDS) with particular characteristics, including severe anaemia, frequent thrombocytosis, typical dysmegakaryopoiesis and favourable outcome.
  • Until the advent of lenalidomide, repeated red blood cell (RBC) transfusions were generally the only treatment for 5q- syndrome, which was resistant to other therapeutic approaches.
  • Lenalidomide can lead to RBC transfusion independence in at least two-thirds of cases of 5q- syndrome, and about one half of those responses are maintained after 2 years of treatment.
  • Grade 3 or 4 neutropenia and thrombocytopenia, especially during the first 6-8 weeks of treatment, are the major side effects of lenalidomide, justifying close clinical and blood counts monitoring.
  • [MeSH-major] Antineoplastic Agents / therapeutic use. Chromosome Deletion. Chromosomes, Human, Pair 5. Myelodysplastic Syndromes / drug therapy. Myelodysplastic Syndromes / genetics. Thalidomide / analogs & derivatives
  • [MeSH-minor] Anemia, Refractory / drug therapy. Anemia, Refractory / genetics. Cytogenetics. Humans. Prognosis. Treatment Outcome


15. Wallvik J, Stenke L, Bernell P, Nordahl G, Hippe E, Hast R: Serum erythropoietin (EPO) levels correlate with survival and independently predict response to EPO treatment in patients with myelodysplastic syndromes. Eur J Haematol; 2002 Mar;68(3):180-5
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  • [Title] Serum erythropoietin (EPO) levels correlate with survival and independently predict response to EPO treatment in patients with myelodysplastic syndromes.
  • Treatment with recombinant erythropoietin (EPO) can alleviate anaemia in patients with myelodysplastic syndromes (MDS).
  • The present study, based on a long-term follow-up of 68 MDS patients (26RA, 16 RAS, 26 RAEB) treated with EPO alone, pinpoints pre-treatment variables associated with response induction, response duration and overall survival.
  • Using univariate logistic regression models, responders displayed significantly lower baseline serum EPO levels (S-EPO), more often normal bone marrow blast cell content (RA/RAS vs. RAEB), normal cytogenetics and no need for erythrocyte transfusion.
  • The median overall survival time from start of EPO treatment was 26 months, significantly longer for responders than for non-responders (49 vs. 18 months, P=0.018).
  • The international prognostic scoring system (IPSS) for MDS predicted survival (P=0.003) and progression to acute leukemia (P<0.001) but not response to EPO treatment.
  • Our data facilitate the optimal selection of MDS patients suitable for EPO treatment and pinpoint S-EPO as a powerful predictor of response and overall survival in MDS.
  • [MeSH-major] Erythropoietin / blood. Erythropoietin / therapeutic use. Myelodysplastic Syndromes / blood. Myelodysplastic Syndromes / drug therapy
  • [MeSH-minor] Adult. Aged. Aged, 80 and over. Anemia, Refractory / blood. Anemia, Refractory / drug therapy. Anemia, Refractory, with Excess of Blasts / blood. Anemia, Refractory, with Excess of Blasts / drug therapy. Bone Marrow Cells / pathology. Erythrocyte Transfusion. Female. Hemoglobins / analysis. Humans. Logistic Models. Male. Middle Aged. Prognosis. Recombinant Proteins. Survival Rate


16. Pawson R, Potter MN, Theocharous P, Lawler M, Garg M, Yin JA, Rezvani K, Craddock C, Rassam S, Prentice HG: Treatment of relapse after allogeneic bone marrow transplantation with reduced intensity conditioning (FLAG +/- Ida) and second allogeneic stem cell transplant. Br J Haematol; 2001 Dec;115(3):622-9
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  • [Title] Treatment of relapse after allogeneic bone marrow transplantation with reduced intensity conditioning (FLAG +/- Ida) and second allogeneic stem cell transplant.
  • Fludarabine-containing 'non-myeloablative' chemotherapy followed by further allo SCT may offer more rapid and effective disease control.
  • We report 14 patients with relapse after allo SCT for acute leukaemia [seven acute myeloid leukaemia (AML), five acute lymphoblastic leukaemia (ALL)] or refractory anaemia with excess blasts in transformation (RAEB-t, n = 2) treated with fludarabine, high-dose cytosine arabinoside (ara-C) and granulocyte colony-simulating factor (G-CSF) with (n = 10) or without (n = 2) idarubicin (FLAG +/- Ida) or DaunoXome (FLAG-X) (n = 2) and second allo SCT from the original donor.
  • Transplants were well tolerated with no treatment-related deaths.
  • FLAG +/- Ida and second allo SCT is a safe and useful approach and may be more effective than DLI in the treatment of acute leukaemias relapsing after conventional allo SCT.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / administration & dosage. Bone Marrow Transplantation. Cytarabine / administration & dosage. Granulocyte Colony-Stimulating Factor / administration & dosage. Idarubicin / administration & dosage. Leukemia / therapy. Transplantation Conditioning / methods. Vidarabine / administration & dosage. Vidarabine / analogs & derivatives
  • [MeSH-minor] Acute Disease. Adult. Anemia, Refractory, with Excess of Blasts / therapy. Child. Child, Preschool. Disease-Free Survival. Female. Filgrastim. Graft vs Host Disease / immunology. Graft vs Host Disease / prevention & control. Humans. Leukemia, Myeloid / therapy. Male. Middle Aged. Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy. Recombinant Proteins. Recurrence. Reoperation. Retrospective Studies. Survival Rate. Transplantation, Homologous

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  • (PMID = 11736947.001).
  • [ISSN] 0007-1048
  • [Journal-full-title] British journal of haematology
  • [ISO-abbreviation] Br. J. Haematol.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Recombinant Proteins; 04079A1RDZ / Cytarabine; 143011-72-7 / Granulocyte Colony-Stimulating Factor; FA2DM6879K / Vidarabine; PVI5M0M1GW / Filgrastim; ZRP63D75JW / Idarubicin; Ida-FLAG protocol
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17. Invernizzi R, Pecci A, Travaglino E, Gobbi PG, Malabarba L, Ramajoli I, Ascari E: Clinical and biological effects of treatment with amifostine in myelodysplastic syndromes. Br J Haematol; 2002 Jul;118(1):246-50
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  • [Title] Clinical and biological effects of treatment with amifostine in myelodysplastic syndromes.
  • /three times a week for three consecutive weeks).
  • Significant reduction of the marrow blasts was observed in one case of refractory anaemia with excess of blasts.
  • [MeSH-major] Amifostine / therapeutic use. Antioxidants / therapeutic use. Myelodysplastic Syndromes / drug therapy
  • [MeSH-minor] Aged. Apoptosis / drug effects. Bone Marrow Cells / drug effects. Bone Marrow Cells / pathology. Female. Follow-Up Studies. Humans. Male. Middle Aged. Neutrophils / pathology. Platelet Count. Reticulocytes / pathology. Treatment Outcome


18. Seipelt G, Germing U, Koschmieder S, Böhme A, Aul C, Hoelzer D: Secondary acute myeloid leukaemia with monosomy 7 in identical adult twins. Br J Haematol; 2002 Feb;116(2):338-40

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  • In the first twin, induction therapy resulted in complete remission (CR).
  • The patient died of sepsis 11 months after diagnosis.
  • The other twin presented with leucopenia and thrombocytopenia and refractory anaemia (RA) was diagnosed.
  • Twenty-eight months following diagnosis the patient progressed to RA with excess blasts in transformation and induction chemotherapy was initiated without achieving CR.
  • [MeSH-minor] Acute Disease. Anemia, Refractory / complications. Anemia, Refractory / genetics. Anemia, Refractory, with Excess of Blasts / complications. Anemia, Refractory, with Excess of Blasts / genetics. Hematopoietic Stem Cell Transplantation. Humans. Male. Middle Aged. Remission Induction. Transplantation, Homologous. Twins, Monozygotic

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  • (PMID = 11841435.001).
  • [ISSN] 0007-1048
  • [Journal-full-title] British journal of haematology
  • [ISO-abbreviation] Br. J. Haematol.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] England
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19. Li ZL, Gong M, Xu SH, Huang FZ, Chen YR, Ma YG: [Cyclosporine A based therapy for myelodysplastic syndrome]. Zhongguo Shi Yan Xue Ye Xue Za Zhi; 2005 Oct;13(5):867-70
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  • [Title] [Cyclosporine A based therapy for myelodysplastic syndrome].
  • To determine the efficacy and tolerance to cyclosporine A (CsA) based therapy in patients with myelodysplastic syndrome (MDS), 16 patients with MDS consisting of 10 refractory anemia (RA) and 6 refractory anemia with accessory blasts less than 10% (RAEB-1) were analyzed.
  • Treatment responses were classified according to the International Working Group (IWG) criteria as complete remission (CR), partial remission (PR), hematological improvement (HI) and no response (NR).
  • Response rates shown in neutrophil lineage, platelet and erythroid lineage were 83.3%, 66.7% and 60%, respectively; their shortest time required to obtain some hematologic improvement after initiation of CsA therapy was 2 weeks, 1 month and 1 month, respectively.
  • Of 13 patients being transfusion-dependent before treatment, 3 patients did not need transfusion any more and 5 showed the reduced transfusion requirements after CsA therapy.
  • Of 6 patients with RAEB, 1 patient had no response and died of RAEB-t and 5 patients had transient responses.
  • The total response rate decreased to 50% in the patients with CsA therapy lasting more than 3 months at the end of following-up.
  • In conclusion, the usefulness of CsA based therapy for MDS-RA and RAEB-1 with any marrow cellularity is useful, the CsA dose of 3-5 mg/(kg.d) is safe and efficacious.

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  • (PMID = 16277860.001).
  • [ISSN] 1009-2137
  • [Journal-full-title] Zhongguo shi yan xue ye xue za zhi
  • [ISO-abbreviation] Zhongguo Shi Yan Xue Ye Xue Za Zhi
  • [Language] CHI
  • [Publication-type] English Abstract; Journal Article
  • [Publication-country] China
  • [Chemical-registry-number] 0 / Androgens; 0 / Immunosuppressive Agents; 0 / Recombinant Proteins; 11096-26-7 / Erythropoietin; 83HN0GTJ6D / Cyclosporine; FXC9231JVH / Calcitriol
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20. Platzbecker U, Haase M, Herbst R, Hänel A, Voigtmann K, Thiede CH, Mohr B, Schleyer E, Leopold T, Orth M, Hänel M, Ehninger G, Bornhäuser M: Activity of sirolimus in patients with myelodysplastic syndrome--results of a pilot study. Br J Haematol; 2005 Mar;128(5):625-30
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  • [Title] Activity of sirolimus in patients with myelodysplastic syndrome--results of a pilot study.
  • Increasing data suggest that sirolimus might affect these pathways positively, thus being of possible therapeutic benefit in patients with this disease.
  • Three patients [1 x refractory anaemia with excess blasts (RAEB)-2, 1 x RAEB-1, 1 x refractory cytopenia with multilineage dysplasia] showed either a major (1 x platelet, 1 x neutrophil) or a minor (1 x erythroid, 2 x platelet) haematological response according to International Working Group criteria.
  • [MeSH-major] Immunosuppressive Agents / administration & dosage. Myelodysplastic Syndromes / drug therapy. Sirolimus / administration & dosage
  • [MeSH-minor] Administration, Oral. Aged. Aged, 80 and over. Anemia, Refractory, with Excess of Blasts / blood. Anemia, Refractory, with Excess of Blasts / drug therapy. Erythrocyte Count. Female. Humans. Leukocyte Count. Male. Middle Aged. Pilot Projects. Platelet Count. Treatment Outcome


21. Webb DK, Passmore SJ, Hann IM, Harrison G, Wheatley K, Chessells JM: Results of treatment of children with refractory anaemia with excess blasts (RAEB) and RAEB in transformation (RAEBt) in Great Britain 1990-99. Br J Haematol; 2002 Apr;117(1):33-9
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  • [Title] Results of treatment of children with refractory anaemia with excess blasts (RAEB) and RAEB in transformation (RAEBt) in Great Britain 1990-99.
  • Between 1990 and 1999, 36 children with refractory anaemia with excess blasts (RAEB) and RAEB in transformation (RAEBt), not associated with Down's syndrome, were diagnosed in Britain.
  • A total of 31 children received intensive chemotherapy, six of whom proceeded to a bone marrow allograft in first remission, whereas two received an autograft.
  • Of the 23 given chemotherapy only, four died of toxicity, 10 relapsed and nine are alive in first remission.
  • Two children received an allograft without prior chemotherapy but died of toxicity.
  • The overall survival was 51% at 5 years, and was superior in children with RAEBt (63%) compared with RAEB (28%, P = 0.03).
  • Monosomy 7 was the most common abnormality (33% of cases).
  • Allowing for cytogenetics, outcomes of therapy appear similar to those for de novo acute myeloid leukaemia (AML), and it is appropriate for children with RAEB/RAEBt to be registered in AML trials.
  • [MeSH-major] Anemia, Refractory, with Excess of Blasts / therapy. Bone Marrow Transplantation
  • [MeSH-minor] Acute Disease. Adolescent. Child. Child, Preschool. Chromosomes, Human, Pair 7. Female. Humans. Infant. Leukemia, Myeloid / mortality. Leukemia, Myeloid / therapy. Male. Monosomy. Patient Selection. Randomized Controlled Trials as Topic. Survival Rate. Transplantation, Homologous. Treatment Outcome

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  • (PMID = 11918530.001).
  • [ISSN] 0007-1048
  • [Journal-full-title] British journal of haematology
  • [ISO-abbreviation] Br. J. Haematol.
  • [Language] eng
  • [Publication-type] Journal Article; Review
  • [Publication-country] England
  • [Number-of-references] 30
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22. Pitini V, Arrigo C, Sauta MG, Altavilla G: Myelodysplastic syndrome appearing during imatinib mesylate therapy in a patient with GIST. Leuk Res; 2009 Sep;33(9):e143-4
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  • [Title] Myelodysplastic syndrome appearing during imatinib mesylate therapy in a patient with GIST.
  • The introduction of imatinib has been a major advance in the treatment of gastrointestinal stromal tumor (GIST).
  • This may be an important issue because some patients (pts) with chronic myelogenous leukemia (CML) develop, during imatinib treatment, chromosomal abnormalities in philadelphia chromosome (Ph) negative cells with evolution to myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML), furthermore a nonrandom association between GIST and myeloid leukemia has been recently reported.
  • We report here a case of refractory cytopenia with mutilineage dysplasia (RAEB-1) with monosomy 7 which rapidly transformed into AML in a patient with GIST during imatinib treatment.
  • [MeSH-major] Antineoplastic Agents / therapeutic use. Gastrointestinal Stromal Tumors / drug therapy. Myelodysplastic Syndromes / chemically induced. Piperazines / therapeutic use. Pyrimidines / therapeutic use


23. Hur M, Lee KM, Cho HC, Park YI, Kim SH, Chang YW, Kim YR, Cho HI: Protein 4.1 deficiency and deletion of chromosome 20q are associated with acquired elliptocytosis in myelodysplastic syndrome. Clin Lab Haematol; 2004 Feb;26(1):69-72
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  • A 66-year-old male presented with peripheral pancytopenia, and was diagnosed with MDS [refractory anaemia (RA)].
  • After 18 months, he had progressed to RA with excess blasts in transformation.
  • Marked elliptocytosis, persistent for more than 17 months, decreased strikingly after chemotherapy with idarubicin and Ara-C.
  • [MeSH-minor] Aged. Anemia, Refractory / etiology. Anemia, Refractory / genetics. Antibiotics, Antineoplastic / therapeutic use. Cytarabine / therapeutic use. Elliptocytosis, Hereditary / etiology. Elliptocytosis, Hereditary / genetics. Elliptocytosis, Hereditary / pathology. Humans. Idarubicin / therapeutic use. Immunosuppressive Agents / therapeutic use. Male. Pancytopenia / etiology. Pancytopenia / genetics

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  • (PMID = 14738441.001).
  • [ISSN] 0141-9854
  • [Journal-full-title] Clinical and laboratory haematology
  • [ISO-abbreviation] Clin Lab Haematol
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Antibiotics, Antineoplastic; 0 / Cytoskeletal Proteins; 0 / Immunosuppressive Agents; 0 / Membrane Proteins; 0 / erythrocyte membrane band 4.1 protein; 04079A1RDZ / Cytarabine; ZRP63D75JW / Idarubicin
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24. Millard TP, Aitchison R, Wilkinson JD: Aleukaemic leukaemia cutis presenting as a benign-appearing eruption. Clin Exp Dermatol; 2003 Mar;28(2):148-50
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  • A 68-year-old Caucasian male presented with a 5-week history of a widespread pruritic papular eruption.
  • Full blood count revealed pancytopaenia but no blasts.
  • Bone marrow aspirate showed reduced red cell precursors and 10% blasts, consistent with myelodysplastic syndrome (refractory anaemia with excess blasts).
  • No chemotherapy was administered.

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  • (PMID = 12653700.001).
  • [ISSN] 0307-6938
  • [Journal-full-title] Clinical and experimental dermatology
  • [ISO-abbreviation] Clin. Exp. Dermatol.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] England
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25. Oertel J, Oertel B, Dörken B: Detection of small numbers of cells characteristic for haematological disorders in peripheral blood (the deep diff). Clin Lab Haematol; 2002 Apr;24(2):73-80
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  • [Title] Detection of small numbers of cells characteristic for haematological disorders in peripheral blood (the deep diff).
  • This approach (the 'deep diff') enabled the detection of small numbers of diagnostically significant cells in a majority of patients.
  • We found ringed sideroblasts in 5/7 patients with sideroblastic anaemia and megaloblasts in 9/10 patients with megaloblastic anaemia.
  • Increased counts of leukaemic cells were found in 12/13 patients with acute leukaemia with < 3% blasts in the conventional white cell differential.
  • Increased blasts were also observed in six patients with refractory anaemia with excess of blasts (RAEB).
  • Decreased blasts were found in five patients with aplastic anaemia and in nine patients with bone marrow aplasia after intensive chemotherapy.
  • We conclude that the 'deep diff', augmented by immunocytochemistry, may be useful in the diagnosis of haematological disorders.
  • [MeSH-minor] Anemia / blood. Anemia, Refractory, with Excess of Blasts / blood. Centrifugation. Coloring Agents. Eosine Yellowish-(YS). Humans. Leukemia / blood. Lymphoma, Non-Hodgkin / blood. Methylene Blue. Neoplastic Cells, Circulating. Retrospective Studies. Staining and Labeling / methods. Waldenstrom Macroglobulinemia / blood

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  • (PMID = 11985551.001).
  • [ISSN] 0141-9854
  • [Journal-full-title] Clinical and laboratory haematology
  • [ISO-abbreviation] Clin Lab Haematol
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Coloring Agents; 0 / May-Grunwald Giemsa; T42P99266K / Methylene Blue; TDQ283MPCW / Eosine Yellowish-(YS)
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26. Kondo H, Kasahara Y, Mori A: Remission induction of refractory anaemia with excess blasts in transformation by sole treatment with granulocyte colony-stimulating factor with persistent chromosomal abnormality. Acta Haematol; 2002;107(3):177-81
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  • [Title] Remission induction of refractory anaemia with excess blasts in transformation by sole treatment with granulocyte colony-stimulating factor with persistent chromosomal abnormality.
  • We report a patient with myelodysplastic syndrome (MDS), refractory anaemia with excess blasts in transformation, in whom complete remission (CR) was achieved with the administration of granulocyte colony-stimulating factor (G-CSF).
  • The 76-year-old patient was admitted to our hospital with a fever and a productive cough; a diagnosis of pneumonia was thus made.
  • Following treatment with antibiotics, the patient's condition improved, and MDS was diagnosed from peripheral blood and bone marrow examinations after the patient recovered from the infection.
  • The patient achieved a sustained haematological CR that was confirmed by morphological and flow cytometric examination after treatment with G-CSF alone, although chromosomal abnormalities persisted.
  • According to the literature, in almost all patients with acute myeloid leukaemia or MDS who were reported to achieve CR by G-CSF, the course was associated with infection, although our case did not have this complication during the course of G-CSF therapy.
  • We suggest that patients with G-CSF alone without infection can achieve CR and that this may be related to a differentiation effect of G-CSF based on persistent chromosomal abnormality in this case.
  • [MeSH-major] Anemia, Refractory, with Excess of Blasts / drug therapy. Anemia, Refractory, with Excess of Blasts / genetics. Chromosome Aberrations. Granulocyte Colony-Stimulating Factor / therapeutic use. Remission Induction

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  • [Copyright] Copyright 2002 S. Karger AG, Basel
  • (PMID = 11978940.001).
  • [ISSN] 0001-5792
  • [Journal-full-title] Acta haematologica
  • [ISO-abbreviation] Acta Haematol.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] Switzerland
  • [Chemical-registry-number] 143011-72-7 / Granulocyte Colony-Stimulating Factor; EC 3.1.3.48 / Antigens, CD45
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27. Dixit A, Chatterjee T, Mishra P, Choudhary DR, Mahapatra M, Saxena R, Choudhry VP: Cyclosporin A in myelodysplastic syndrome: a preliminary report. Ann Hematol; 2005 Sep;84(9):565-8
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  • Therapeutic approaches are not well established in patients with myelodysplastic syndrome (MDS).
  • We evaluated response to cyclosporin A (CyA) in 19 cases with MDS who were enrolled for the study [13 refractory anemia (RA), 5 refractory anemia with excess of blasts (RAEB), and 1 refractory anemia with ringed sideroblasts (RARS)].
  • Four cases of RA showed minor response and two cases of RA did not respond to CyA therapy.
  • A minor response was also seen in one RAEB and one RARS case, while one RAEB case that initially showed a major response relapsed on therapy.
  • The first effect of therapy was evident after a mean period of 2.5 months.
  • One case developed renal failure on therapy and later died of septicemia.
  • CyA could be an effective mode of therapy in patients with MDS especially those having RA.
  • [MeSH-major] Cyclosporine / administration & dosage. Myelodysplastic Syndromes / drug therapy
  • [MeSH-minor] Adult. Aged. Anemia, Refractory / drug therapy. Anemia, Refractory, with Excess of Blasts / drug therapy. Anemia, Sideroblastic / drug therapy. Bone Marrow / pathology. Female. Humans. Leukocyte Count. Male. Middle Aged. Platelet Count. Renal Insufficiency / chemically induced. Treatment Outcome


28. Jackson G, Taylor P, Smith GM, Marcus R, Smith A, Chu P, Littlewood TJ, Duncombe A, Hutchinson M, Mehta AB, Johnson SA, Carey P, MacKie MJ, Ganly PS, Turner GE, Deane M, Schey S, Brookes J, Tollerfield SM, Wilson MP: A multicentre, open, non-comparative phase II study of a combination of fludarabine phosphate, cytarabine and granulocyte colony-stimulating factor in relapsed and refractory acute myeloid leukaemia and de novo refractory anaemia with excess of blasts in transformation. Br J Haematol; 2001 Jan;112(1):127-37
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  • [Title] A multicentre, open, non-comparative phase II study of a combination of fludarabine phosphate, cytarabine and granulocyte colony-stimulating factor in relapsed and refractory acute myeloid leukaemia and de novo refractory anaemia with excess of blasts in transformation.
  • The primary objective of this study was to determine the complete remission (CR) rate achieved with the FLAG (fludarabine phosphate, cytarabine and granulocyte colony-stimulating factor) regimen in patients with relapsed or refractory acute myeloid leukaemia (AML) or de novo refractory anaemia with excess of blasts in transformation (RAEB-t).
  • Induction treatment consisted of between one and two courses of FLAG.
  • Patients achieving CR received between one and two courses of consolidation treatment.
  • CR rates were: 17 out of 21 (81%) in late relapse AML (Group 1), 13 out of 44 (30%) in early relapse/refractory AML (Group 2), and 10 out of 18 (56%) in de novo RAEB-t (Group 3).
  • Median survival times were 1.4 years, 3 months and 1.6 years in Groups 1, 2 and 3 respectively.
  • The FLAG regimen offers a very effective alternative treatment for CR induction in poor prognosis adult patients with either relapsed or refractory AML or de novo RAEB-t.
  • FLAG delivers high-dose treatment without increasing overall toxicity, an approach which is of particular value in older patients, who constitute the majority in these diseases.
  • It is therefore an important advance in developing new treatment options for these patients.
  • [MeSH-major] Anemia, Refractory, with Excess of Blasts / drug therapy. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Leukemia, Myeloid / drug therapy

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  • (PMID = 11167793.001).
  • [ISSN] 0007-1048
  • [Journal-full-title] British journal of haematology
  • [ISO-abbreviation] Br. J. Haematol.
  • [Language] eng
  • [Publication-type] Clinical Trial; Clinical Trial, Phase II; Journal Article; Multicenter Study
  • [Publication-country] England
  • [Chemical-registry-number] 04079A1RDZ / Cytarabine; 143011-72-7 / Granulocyte Colony-Stimulating Factor; FA2DM6879K / Vidarabine; FLAG protocol
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29. Poulain S, Lepelley P, Preudhomme C, Cambier N, Cornillon J, Wattel E, Cosson A, Fenaux P: Expression of the multidrug resistance-associated protein in myelodysplastic syndromes. Br J Haematol; 2000 Sep;110(3):591-8
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  • In the myelodysplastic syndromes (MDS), P-glycoprotein (P-gp) expression is clinically associated with drug resistance, whereas the clinical significance of multidrug resistance-associated protein (MRP1) is uncertain.
  • Bone marrow from 56 patients with MDS, including six with refractory anaemia (RA)/RA with ringed sideroblasts (RARS), 23 cases of RA with excess blasts/in transformation (RAEB/T), four patients with chronic myelomonocytic leukaemia (CMML) and 23 cases of MDS having progressed to acute myeloid leukaemia (MDS-AML), were studied.
  • Ten of the 26 patients treated with intensive chemotherapy achieved complete remission including six out of 16 MRP1+ and four out of ten MRP1- cases (P = NS).
  • [MeSH-minor] ATP-Binding Cassette, Sub-Family B, Member 1 / analysis. Adult. Animals. Antigens, CD34 / analysis. Cell Line. Chi-Square Distribution. Chromosome Aberrations / metabolism. Chromosome Disorders. Flow Cytometry. Humans. Immunohistochemistry / methods. Leukemia, Myeloid / drug therapy. Leukemia, Myeloid / metabolism. Middle Aged. Multidrug Resistance-Associated Proteins. Neoplasm Proteins / analysis. Treatment Outcome. Vault Ribonucleoprotein Particles

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  • (PMID = 10997969.001).
  • [ISSN] 0007-1048
  • [Journal-full-title] British journal of haematology
  • [ISO-abbreviation] Br. J. Haematol.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Chemical-registry-number] 0 / ATP-Binding Cassette Transporters; 0 / ATP-Binding Cassette, Sub-Family B, Member 1; 0 / Antigens, CD34; 0 / Multidrug Resistance-Associated Proteins; 0 / Neoplasm Proteins; 0 / Vault Ribonucleoprotein Particles; 0 / major vault protein
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30. Raj K, John A, Ho A, Chronis C, Khan S, Samuel J, Pomplun S, Thomas NS, Mufti GJ: CDKN2B methylation status and isolated chromosome 7 abnormalities predict responses to treatment with 5-azacytidine. Leukemia; 2007 Sep;21(9):1937-44
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  • [Title] CDKN2B methylation status and isolated chromosome 7 abnormalities predict responses to treatment with 5-azacytidine.
  • All six had less than 5% bone marrow (BM) blasts at the time of haematological improvements (HI) (2 had pre-existing refractory anaemia (RA), 4 had refractory anaemia with excess blasts (RAEB)).
  • A further patient with RAEB had blast reduction to less than 5% without HI.
  • [MeSH-major] Antimetabolites, Antineoplastic / administration & dosage. Azacitidine / administration & dosage. Chromosome Aberrations. Chromosomes, Human, Pair 7. Cyclin-Dependent Kinase Inhibitor p15 / genetics. Myelodysplastic Syndromes / drug therapy. Myelodysplastic Syndromes / genetics
  • [MeSH-minor] Adolescent. Adult. Aged. Aged, 80 and over. Apoptosis / drug effects. Bone Marrow Cells / pathology. DNA Methylation / drug effects. Female. Genetic Markers. Humans. Injections, Subcutaneous. Male. Middle Aged. Predictive Value of Tests. Promoter Regions, Genetic / physiology. Survival Rate. Treatment Outcome

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  • (PMID = 17611569.001).
  • [ISSN] 0887-6924
  • [Journal-full-title] Leukemia
  • [ISO-abbreviation] Leukemia
  • [Language] eng
  • [Grant] United Kingdom / Medical Research Council / / G84/6222
  • [Publication-type] Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Antimetabolites, Antineoplastic; 0 / CDKN2B protein, human; 0 / Cyclin-Dependent Kinase Inhibitor p15; 0 / Genetic Markers; M801H13NRU / Azacitidine
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31. Ferrero D, Darbesio A, Giai V, Genuardi M, Dellacasa CM, Sorasio R, Bertini M, Boccadoro M: Efficacy of a combination of human recombinant erythropoietin + 13-cis-retinoic acid and dihydroxylated vitamin D3 to improve moderate to severe anaemia in low/intermediate risk myelodysplastic syndromes. Br J Haematol; 2009 Feb;144(3):342-9
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  • [Title] Efficacy of a combination of human recombinant erythropoietin + 13-cis-retinoic acid and dihydroxylated vitamin D3 to improve moderate to severe anaemia in low/intermediate risk myelodysplastic syndromes.
  • The efficacy of human recombinant erythropoietin (rEPO) in myelodysplastic syndromes (MDS) has generally been best in untransfused patients with 'refractory anaemia' according to the World Health Organization (WHO).
  • We treated 63 MDS patients [excluding refractory anaemia with excess blasts, type 2 (RAEB2)] with a previously tested combination of 13-cis-retinoic acid and dihydroxylated vitamin D3 +/- 6-thioguanine in addition to rEPO.
  • Most patients were categorized as refractory cytopenia with multilineage dysplasia and RAEB1, with intermediate 1 International Prognostic Scoring System (IPSS) score; all had Hb <95 g/l, and 70% required regular erythrocyte transfusions.
  • Treatment was well tolerated, and erythroid response rate according to new International Working Group criteria was 60%: 50% in RAEB1 and 64% in non-RAEB patients (P = 0.383).
  • Median survival reached 14 months for RAEB1 and 55 months for non-RAEB patients, with a significant difference in the latter between responders and non-responders (median 82 months vs. 44 months; P = 0.036).
  • Our combined therapy, independent of rEPO dosage, achieved in patients with unfavourable response predictors, a rate of anaemia improvement comparable to the best obtained in lower risk patients by high-dose rEPO.
  • [MeSH-major] Anemia / drug therapy. Calcitriol / therapeutic use. Erythropoietin / therapeutic use. Isotretinoin / therapeutic use. Myelodysplastic Syndromes / drug therapy. Vitamins / therapeutic use
  • [MeSH-minor] Adult. Aged. Aged, 80 and over. Drug Therapy, Combination. Female. Humans. Kaplan-Meier Estimate. Male. Middle Aged. Recombinant Proteins. Risk. Survival Rate. Treatment Outcome


32. Strupp C, Knipp S, Hartmann J, Gattermann N, Haas R, Germing U: A pilot study of bendamustine in elderly patients with high-risk MDS and AML. Leuk Lymphoma; 2007 Jun;48(6):1161-6
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  • We examined the efficacy of bendamustine in 15 pretreated patients (12 men, 3 women, median age 69 years) with acute myeloid leukaemia (AML) or myelodysplastic syndromes (MDS) 3 AML, 5 sAML, 5 CMML II, 1 RAEB II.
  • Nine of 15 patients had no side effects of the treatment, six patients suffered from vomiting and epigastric pain as adverse effects of bendamustine.
  • In summary, treatment with bendamustine in patients with high-risk MDS or sAML with leukocytosis can result in a significant reduction of leukocytes, but fails to achieve hematological responses or improvement of transfusions dependency.
  • [MeSH-major] Leukemia, Myeloid / drug therapy. Myelodysplastic Syndromes / drug therapy. Nitrogen Mustard Compounds / therapeutic use
  • [MeSH-minor] Acute Disease. Aged. Antineoplastic Agents / adverse effects. Antineoplastic Agents / therapeutic use. Bendamustine Hydrochloride. Drug Evaluation. Female. Humans. Male. Pilot Projects

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  • [CommentIn] Leuk Lymphoma. 2007 Jun;48(6):1064-6 [17577766.001]
  • (PMID = 17577779.001).
  • [ISSN] 1042-8194
  • [Journal-full-title] Leukemia & lymphoma
  • [ISO-abbreviation] Leuk. Lymphoma
  • [Language] eng
  • [Publication-type] Evaluation Studies; Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / Nitrogen Mustard Compounds; 981Y8SX18M / Bendamustine Hydrochloride
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33. Perez A, Kennedy C, Standen G, Oxley J: A case of monocytic leukaemia cutis in a patient with myelodysplastic syndrome transforming to acute myeloid leukaemia. Clin Exp Dermatol; 2004 Sep;29(5):497-8
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  • A 49-year-old woman presented with a 1-year-history of a widespread eruption which proved to be due to leukaemia cutis.
  • Subsequently, she developed pancytopaenia and a bone marrow biopsy revealed refractory anaemia with excess blasts in transformation (RAEB-T) with a high monoblastic component.
  • Prompt diagnosis in this situation may identify a group of high-risk patients with myelodysplastic syndrome for whom chemotherapy and allogenic bone marrow transplantation, rather than the more conventional approach of supportive treatment, could be a more appropriate management strategy.
  • [MeSH-major] Anemia, Refractory, with Excess of Blasts / pathology. Leukemia / pathology. Leukemia, Myeloid, Acute / pathology


34. Maciejewski JP, Risitano AM, Sloand EM, Wisch L, Geller N, Barrett JA, Young NS: A pilot study of the recombinant soluble human tumour necrosis factor receptor (p75)-Fc fusion protein in patients with myelodysplastic syndrome. Br J Haematol; 2002 Apr;117(1):119-26
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  • The drug was well tolerated and 15 patients were evaluable.
  • Progression to refractory anaemia with excess blasts in transformation (RAEBt) or leukaemia was observed in three patients.
  • Although anti-TNF therapy with Enbrel was well tolerated at the dosages used in MDS, its efficacy as a single agent appears low.
  • [MeSH-major] Immunoglobulin G / therapeutic use. Myelodysplastic Syndromes / drug therapy. Receptors, Tumor Necrosis Factor / therapeutic use. Tumor Necrosis Factor-alpha / antagonists & inhibitors
  • [MeSH-minor] Adult. Aged. Anemia, Refractory / drug therapy. Anemia, Refractory / therapy. Anemia, Refractory, with Excess of Blasts / drug therapy. Anemia, Refractory, with Excess of Blasts / therapy. Blood Transfusion. Bone Marrow Cells / immunology. Colony-Forming Units Assay. Etanercept. Humans. Middle Aged. Pilot Projects. Treatment Failure

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  • [ErratumIn] Br J Haematol 2002 Jun;117(4):1002. Ristiano Antonio M [corrected to Risitano Antonio M]
  • (PMID = 11918541.001).
  • [ISSN] 0007-1048
  • [Journal-full-title] British journal of haematology
  • [ISO-abbreviation] Br. J. Haematol.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Immunoglobulin G; 0 / Receptors, Tumor Necrosis Factor; 0 / Tumor Necrosis Factor-alpha; OP401G7OJC / Etanercept
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35. Gubinelli E, Cocuroccia B, Fazio M, Annessi G, Girolomoni G: Papular neutrophilic dermatosis and erythema elevatum diutinum following erythropoietin therapy in a patient with myelodysplastic syndrome. Acta Derm Venereol; 2003;83(5):358-61
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  • [Title] Papular neutrophilic dermatosis and erythema elevatum diutinum following erythropoietin therapy in a patient with myelodysplastic syndrome.
  • A 65-year-old man with refractory anaemia with an excess of blasts developed an erythematous papular eruption symmetrically distributed on the legs and trunk 3 months after initiation of erythropoietin therapy.
  • The lesions showed a dense neutrophilic infiltrate in the absence of leucocytoclastic vasculitis, and did not fit the criteria of a well-defined neutrophilic dermatosis.
  • Concomitant with the rapid resolution of these skin lesions following erythropoietin discontinuation, typical lesions of erythema elevatum diutinum arose on the extensor surface of the fingers, knees and elbows, which responded to a brief course of dapsone treatment.
  • Although typical and atypical neutrophilic dermatoses have been reported in patients with haematological disorders, they have also been associated with the use of drugs, in particular granulocyte colony-stimulating factor.
  • [MeSH-major] Colony-Stimulating Factors / adverse effects. Erythema / chemically induced. Erythropoietin / adverse effects. Myelodysplastic Syndromes / drug therapy. Neutrophil Infiltration / drug effects

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  • (PMID = 14609104.001).
  • [ISSN] 0001-5555
  • [Journal-full-title] Acta dermato-venereologica
  • [ISO-abbreviation] Acta Derm. Venereol.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] Norway
  • [Chemical-registry-number] 0 / Colony-Stimulating Factors; 11096-26-7 / Erythropoietin
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36. Mantovani L, Lentini G, Hentschel B, Wickramanayake PD, Loeffler M, Diehl V, Tesch H: Treatment of anaemia in myelodysplastic syndromes with prolonged administration of recombinant human granulocyte colony-stimulating factor and erythropoietin. Br J Haematol; 2000 May;109(2):367-75
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  • [Title] Treatment of anaemia in myelodysplastic syndromes with prolonged administration of recombinant human granulocyte colony-stimulating factor and erythropoietin.
  • Treatment with recombinant human erythropoietin (rhEPO) improves anaemia in approximately 20% of the patients with myelodysplastic syndromes (MDS).
  • Recent reports suggest that a combination treatment with rhEPO plus recombinant human granulocyte colony-stimulating factor (rhG-CSF) given for up to 18 weeks may result in a higher erythroid response rate than with rhEPO alone.
  • We investigated the potential advantage of an even more prolonged schedule of combined rhG-CSF and rhEPO treatment to obtain and maintain stable responses.
  • In a phase II study, 33 patients with MDS [17 with refractory anaemia (RA), eight with RA with ringed sideroblasts (RARS), eight with RA with excess blasts (RAEB) with bone marrow blast counts less than 20%] were scheduled to receive at least 36 weeks of combined therapy with rhG-CSF and rhEPO.
  • Seventeen of 28 evaluable patients demonstrated an erythroid response [61%; 95% confidence interval (CI) 41-78] after 12 weeks of treatment.
  • Seven of these responses developed between week 12 and week 36, whereas two initially responding patients became refractory.
  • The cytokine therapy was generally well tolerated.
  • After 1 year and 2 years of continuous combined treatment, 50% of the initially included patients showed a continuing response.
  • Our results suggest that a prolonged combination treatment with rhG-CSF and rhEPO is highly effective in achieving a stable and long-lasting erythroid response in many patients with MDS and low blast count.
  • [MeSH-major] Anemia / drug therapy. Erythropoietin / administration & dosage. Granulocyte Colony-Stimulating Factor / administration & dosage. Myelodysplastic Syndromes / drug therapy
  • [MeSH-minor] Adult. Aged. Aged, 80 and over. Disease-Free Survival. Drug Administration Schedule. Drug Therapy, Combination. Erythrocyte Count. Female. Humans. Male. Middle Aged. Recombinant Proteins. Time Factors. Treatment Outcome


37. Zhou FL, Zhang WG, Cao XM, Chen YX, He AL, Liu J, Zhao WH, Ma XR, Chen G: [Retrospective observation of curative effects on MDS refractory anemia with combination of all-trans retinoic acid, 1, 25-dihydroxyvitamin D3 and androgen]. Zhongguo Shi Yan Xue Ye Xue Za Zhi; 2005 Oct;13(5):861-6
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  • [Title] [Retrospective observation of curative effects on MDS refractory anemia with combination of all-trans retinoic acid, 1, 25-dihydroxyvitamin D3 and androgen].
  • This study was aimed to examine whether a combination of all-trans retinoic acid (ATRA), 1, 25-dihydroxyvitamin D(3) and androgen possesses the therapeutic value for the MDS-refractory anemia (MDS-RA), and to analyze the mechanisms in detail.
  • The remaining 33 cases (group B) were provided with vitamin supplementation, chalybeate drugs, and one or two of the combination.
  • The further treatment for 16 out of 62 patients (25.81%), 13 failures (10 deaths, 2 RAEB and 1 RAEB-T) and 3 transformations (M(2), M(3), M(5)) with a median survival interval of 26.25 months, were observed and interrupted for some reasons.
  • Furthermore, the disease progression was observed in 12 out of 33 patients (36.36%) with a median survival interval of 16 months, 9 failures (including 6 deaths, 2 RAEB and 1 RAEB-T) and 3 transformations (M(2), M(3), M(4)).
  • The following requirements, if were met, would be significant for prognosis: the combination regiment, no transformation, children, no complication, female, 90-120 g/L of hemoglobin concentration, normal cellular bone marrow and uni-cytopenias (P < 0.05).
  • Moreover, Cox regression showed that therapy, transformation and age are all the independent factors (P < 0.05).
  • It is concluded that the combination of above mentioned 3 drugs may be effective and safe treatment for the patients with MDS-RA.
  • Its relevant mechanisms can be involved in the combination, that elicits a wide range of pharmacological effects, such as differentiation, anti-tumor-promotion, anti-apoptosis, anti-angiogenesis, anti-cachexia and immunoregulation.


38. Cesaro S, Strugo L, Alaggio R, Cecchetto G, Rigobello L, Pillon M, Cusinato R, Zanesco L: Voriconazole for invasive aspergillosis in oncohematological patients: a single-center pediatric experience. Support Care Cancer; 2003 Nov;11(11):722-7
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  • Voriconazole is a new triazole active orally and parenterally that recently proved effective in the treatment of invasive aspergillosis and in empirical antifungal therapy for persistently febrile neutropenic patients.
  • We report our experience with voriconazole in seven children with invasive aspergillosis, i.e., four girls and three boys with a median age of 5 (range 2-13) years affected by acute lymphoblastic leukemia (3), acute myeloid leukemia (2), refractory anemia with excess of blasts (1), and severe aplastic anemia (1).
  • First-line therapy in all patients was liposomal amphotericin B (AmBisome) administered at a dosage of 3-5 mg/kg day.
  • The voriconazole treatment was well tolerated.
  • Three patients are alive and well 6, 5, and 4 months after the diagnosis of aspergillosis.
  • Voriconazole appears to be an effective salvage treatment for invasive aspergillosis in pediatric patients, with good responses in patients who recover from neutropenia or are not relapsing.
  • [MeSH-major] Antifungal Agents / administration & dosage. Aspergillosis / drug therapy. Aspergillosis / etiology. Hematologic Neoplasms / therapy. Pyrimidines / administration & dosage. Triazoles / administration & dosage
  • [MeSH-minor] Adolescent. Amphotericin B / administration & dosage. Anemia, Aplastic / drug therapy. Anemia, Refractory / drug therapy. Antineoplastic Agents / adverse effects. Bone Marrow Transplantation / adverse effects. Child. Child, Preschool. Dose-Response Relationship, Drug. Drug Administration Schedule. Female. Humans. Leukemia, Myelomonocytic, Acute / drug therapy. Male. Neutropenia / chemically induced. Neutropenia / complications. Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy. Time Factors. Treatment Outcome. Voriconazole

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  • (PMID = 13680324.001).
  • [ISSN] 0941-4355
  • [Journal-full-title] Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer
  • [ISO-abbreviation] Support Care Cancer
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Germany
  • [Chemical-registry-number] 0 / Antifungal Agents; 0 / Antineoplastic Agents; 0 / Pyrimidines; 0 / Triazoles; 7XU7A7DROE / Amphotericin B; JFU09I87TR / Voriconazole
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39. Ribrag V, Suzan F, Ravoet C, Feremans W, Guerci A, Dreyfus F, Damaj G, Vantelon JM, Bourhis JH, Fenaux P: Phase II trial of CPT-11 in myelodysplastic syndromes with excess of marrow blasts. Leukemia; 2003 Feb;17(2):319-22
NCI CPTC Antibody Characterization Program. NCI CPTC Antibody Characterization Program .

  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Phase II trial of CPT-11 in myelodysplastic syndromes with excess of marrow blasts.
  • We conducted a phase II trial of CPT-11 in 26 patients with high-risk MDS (RAEB 1: n = 4; RAEB 2: n = 9; MDS having progressed to AML: n = 10; CMML: n = 3) who could not receive anthracycline/cytarabine intensive chemotherapy.
  • Induction therapy consisted of four courses of CPT-11 given intravenously at 200 mg/m(2) every 2 weeks.
  • Patient characteristics were: median age, 71 (range 51-77); sex, (M/F), 21/5, median % marrow blasts cells, 13.5 (range 7-52).
  • Six patients stopped treatment after only one or two courses of CPT-11 due to severe infection (n = 2), progressive disease (n = 3), acute lysis syndrome with renal failure (n = 1).
  • Thus CPT-11 has an interesting activity in MDS with excess of blasts; toxicity is easily managed and most patients can be treated in the out-clinic setting.
  • [MeSH-major] Anemia, Refractory, with Excess of Blasts / drug therapy. Antineoplastic Agents, Phytogenic / therapeutic use. Bone Marrow / pathology. Camptothecin / analogs & derivatives. Camptothecin / therapeutic use. Myelodysplastic Syndromes / drug therapy. Myelodysplastic Syndromes / pathology
  • [MeSH-minor] Aged. Blast Crisis. Disease Progression. Female. Humans. Infusions, Intravenous. Leukemia, Myeloid, Acute. Male. Middle Aged. Survival Analysis. Time Factors

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  • (PMID = 12592329.001).
  • [ISSN] 0887-6924
  • [Journal-full-title] Leukemia
  • [ISO-abbreviation] Leukemia
  • [Language] eng
  • [Publication-type] Clinical Trial; Clinical Trial, Phase II; Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Antineoplastic Agents, Phytogenic; 7673326042 / irinotecan; XT3Z54Z28A / Camptothecin
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40. Hofmann WK, Kell WJ, Fenaux P, Castaigne S, Ganser A, Chomienne C, Burnett R, Kowal C, Hoelzer D, Burnett AK: Oral 9-cis retinoic acid (Alitretinoin) in the treatment of myelodysplastic syndromes: results from a pilot study. Leukemia; 2000 Sep;14(9):1583-8
The Lens. Cited by Patents in .

  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Oral 9-cis retinoic acid (Alitretinoin) in the treatment of myelodysplastic syndromes: results from a pilot study.
  • The MDS subtypes were distributed as follows: 14 refractory anaemia (RA), four refractory anaemia with ringed sideroblasts (RARS), and 12 refractory anaemia with excess blasts (RAEB).
  • None of these had previously received treatment for MDS other than supportive therapy.
  • 9CRA (Alitretinoin capsules, kindly provided by Allergan-Ligand Retinoid Therapeutics) was given daily at 60 mg/m2 p.o. for 1 week, followed by an intra-patient escalation to 100 mg/m2 during the second week, up to a maximum of 140 mg/m2.
  • The planned treatment duration was 48 weeks.
  • Four (16%), two with RA, two with RAEB, had minor responses resulting in decreased transfusion requirements or increased neutrophils.
  • In conclusion, although modest responses were noted in this study, the treatment tolerability was suboptimal.
  • [MeSH-major] Antineoplastic Agents / therapeutic use. Myelodysplastic Syndromes / drug therapy. Tretinoin / therapeutic use
  • [MeSH-minor] Administration, Oral. Adult. Aged. Aged, 80 and over. Blood Transfusion. Female. Hematopoiesis. Humans. Male. Middle Aged. Pilot Projects. Treatment Outcome


41. Yamada T, Tsurumi H, Kasahara S, Hara T, Sawada M, Moriwaki H: Immunosuppressive therapy for myelodysplastic syndrome: efficacy of methylprednisolone pulse therapy with or without cyclosporin A. J Cancer Res Clin Oncol; 2003 Aug;129(8):485-91
NCI CPTC Antibody Characterization Program. NCI CPTC Antibody Characterization Program .

  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Immunosuppressive therapy for myelodysplastic syndrome: efficacy of methylprednisolone pulse therapy with or without cyclosporin A.
  • We investigated whether immunosuppressive therapy using methylprednisolone (mPSL) with or without cyclosporin A (CsA) could benefit patients with myelodysplastic syndrome (MDS).
  • Eligibility criteria for this study were a clinical diagnosis of MDS with less than 5% blast in peripheral blood, less than 10% blast in bone marrow and advanced cytopenia.
  • Among 73 patients with MDS, 18 eligible and consecutive patients (8 men and 10 women), aged 48 to 87 years (median: 66.5 years) were assigned to receive mPSL pulse therapy (1,000 mg daily for 3 consecutive days, followed by tapering oral prednisolone; n= 12) or mPSL pulse with CsA therapy (4 to 5 mg/kg administered twice daily; n= 6).
  • Six of 18 patients (33.3%; 3 of 10 patients with RA, 2 of 6 patients with RAEB, 1 of 2 patients with CMMoL) responded to immunosuppressive therapy.
  • One of 6 patients with hypocellular bone marrow and 5 of 12 patients with normocellular or hypercellular marrow responded to immunosuppressive therapy.
  • No patient with myelofibrosis responded to the therapy.
  • It is possible that immunosuppressive therapy might be effective for a certain subset of patients with MDS.
  • [MeSH-major] Cyclosporine / administration & dosage. Immunosuppressive Agents / therapeutic use. Methylprednisolone / administration & dosage. Myelodysplastic Syndromes / drug therapy
  • [MeSH-minor] Aged. Aged, 80 and over. Drug Therapy, Combination. Female. Hemoglobins / drug effects. Humans. Male. Middle Aged. Pulse Therapy, Drug. Survival Analysis. Treatment Outcome

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  • (PMID = 12856174.001).
  • [ISSN] 0171-5216
  • [Journal-full-title] Journal of cancer research and clinical oncology
  • [ISO-abbreviation] J. Cancer Res. Clin. Oncol.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Germany
  • [Chemical-registry-number] 0 / Hemoglobins; 0 / Immunosuppressive Agents; 83HN0GTJ6D / Cyclosporine; X4W7ZR7023 / Methylprednisolone
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42. Xiao Z, Liu L, Xu Z, Qin T, Zhang Y, Zhang T: Low-dose melphalan in myelodysplastic syndromes: an effective treatment for elderly RAEB-I or II patients? Leuk Lymphoma; 2010 Mar;51(3):549-51
Hazardous Substances Data Bank. MELPHALAN .

  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Low-dose melphalan in myelodysplastic syndromes: an effective treatment for elderly RAEB-I or II patients?
  • [MeSH-major] Anemia, Refractory, with Excess of Blasts / drug therapy. Melphalan / therapeutic use. Myelodysplastic Syndromes / drug therapy
  • [MeSH-minor] Adolescent. Adult. Age Factors. Aged. Cytogenetics. Female. Humans. Karyotyping. Male. Middle Aged. Remission Induction. Treatment Outcome

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  • (PMID = 20038270.001).
  • [ISSN] 1029-2403
  • [Journal-full-title] Leukemia & lymphoma
  • [ISO-abbreviation] Leuk. Lymphoma
  • [Language] eng
  • [Publication-type] Letter
  • [Publication-country] England
  • [Chemical-registry-number] Q41OR9510P / Melphalan
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43. Bowen DT, Hellstrom-Lindberg E: Best supportive care for the anaemia of myelodysplasia: inclusion of recombinant erythropoietin therapy? Leuk Res; 2001 Jan;25(1):19-21

  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Best supportive care for the anaemia of myelodysplasia: inclusion of recombinant erythropoietin therapy?
  • [MeSH-major] Anemia, Refractory, with Excess of Blasts / drug therapy. Erythropoietin / therapeutic use
  • [MeSH-minor] Anemia, Refractory. Erythrocyte Transfusion / economics. Humans. Recombinant Proteins

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  • [CommentOn] Leuk Res. 2001 Jan;25(1):13-18 [11137555.001]
  • (PMID = 11137556.001).
  • [ISSN] 0145-2126
  • [Journal-full-title] Leukemia research
  • [ISO-abbreviation] Leuk. Res.
  • [Language] eng
  • [Publication-type] Comment; Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Recombinant Proteins; 11096-26-7 / Erythropoietin
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