Thalidomide produces transfusion independence in long-standing refractory anemias of patients with myelodysplastic syndromes

Thalidomide was administered to 83 patients with myelodysplastic syndrome (MDS), starting at 100 mg by mouth daily and increasing to 400 mg as tolerated. Thirty-two patients stopped therapy before 12 weeks (minimum period for response evaluation), and 51 completed 12 weeks of therapy. International...

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Bibliographic Details
Published in:Blood 2001-08, Vol.98 (4), p.958-965
Main Authors: Raza, Azra, Meyer, Peter, Dutt, Diya, Zorat, Francesca, Lisak, Laurie, Nascimben, Fabiana, du Randt, Morne, Kaspar, Christopher, Goldberg, Cathryn, Loew, Jerome, Dar, Saleem, Gezer, Sefer, Venugopal, Parameswaran, Zeldis, Jerome
Format: Article
Language:English
Subjects:
Aged
Anemia - blood
Anemia - drug therapy
Anemia - etiology
Biological and medical sciences
Blood Cell Count
Blood Transfusion
Blood. Blood coagulation. Reticuloendothelial system
Bone Marrow - drug effects
Bone Marrow - pathology
Female
Hematopoiesis - drug effects
Hemoglobins - metabolism
Humans
Male
Maximum Tolerated Dose
Medical sciences
Myelodysplastic Syndromes - blood
Myelodysplastic Syndromes - complications
Myelodysplastic Syndromes - drug therapy
Pharmacology. Drug treatments
Pilot Projects
Thalidomide - pharmacology
Thalidomide - toxicity
Treatment Outcome
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Summary:Thalidomide was administered to 83 patients with myelodysplastic syndrome (MDS), starting at 100 mg by mouth daily and increasing to 400 mg as tolerated. Thirty-two patients stopped therapy before 12 weeks (minimum period for response evaluation), and 51 completed 12 weeks of therapy. International Working Group response criteria for MDS were used to evaluate responses. Intent-to-treat (ITT) analysis classified all off-study patients as nonresponders. Off-study patients belonged to a higher risk category (P = .002) and had a higher percentage of blasts in their pretherapy bone marrow than patients who completed 12 weeks of therapy (P = .003). No cytogenetic or complete responses were seen, but 16 patients showed hematologic improvement, with 10 previously transfusion-dependent patients becoming transfusion independent. Responders had lower pretherapy blasts (P = .016), a lower duration of pretherapy platelet transfusions (P = .013), and higher pretherapy platelets (P = .003). Among responders, 9 had refractory anemia (RA); 5 had RA with ringed sideroblasts; and 2 had RA with excess blasts. By ITT analysis, 19% of patients (16 of 83) responded, and when only evaluable patients were analyzed, 31% (16 of 51) responded. It was concluded that thalidomide, as a single agent, is effective in improving cytopenias of some MDS patients, especially those who present without excess blasts.
ISSN:0006-4971
1528-0020
DOI:10.1182/blood.V98.4.958