Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8453280 | Leukemia Research | 2018 | 8 Pages |
Abstract
Immunosuppressive treatment is a disease-modifying therapy for lower-risk myelodysplastic syndromes (MDS). However, IST is relatively rarely used and long-term outcomes of patients are seldom reported. We retrospectively studied outcomes of 20 patients with lower-risk non del 5q MDS with transfusion dependency, with horse or rabbit antithymocyte globulinâ¯Â±â¯ciclosporine A, and frontline eltrombopag in two of them. IPSS-R was low, intermediate and high in 30%, 55% and 10% of the patients, respectively. Fifty-five percent of the patients had hypocellular bone marrow (BM). Baseline mutations were detected in 31.5% of the patients and were more frequent in patients with normo/hypercellular MDS than in patients with hypocellular MDS. Transfusion independence rate for both red blood cells (RBC) and platelets was achieved in 45% of patients. RBC transfusion duration â¤6 months, B-cell counts >0.2â¯G/L and, marginally, BM blasts â¤2% were associated with higher transfusion independence rate. Age and cellularity did not influence the response rate. Median transfusion independence duration was 53 months. Cumulative incidence of progression to a more aggressive myeloid disease was 0 in patients without baseline mutations and 33% in patients with baseline mutations (Pâ¯=â¯.008). Median progression-free and overall survival after treatment onset and median overall survival after loss of transfusion independence were 45.5 months, 68 months and not reached, respectively. In conclusion, antithymocyte globulinâ¯Â±â¯ciclosporine A results in durable responses in MDS, irrespective of age, in patients with lower-risk disease without B-cell lymphopenia and treated early in the course of the disease.
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Authors
C. Kelaidi, T. Braun, R. Arana, A. Marceau-Renaut, G. Lazarian, J. Soret, S. Cereja, R. Letestu, V. Eclache, D. Lusina, F. Baran-Marszak, L. Ades, C. Preudhomme, A. Martin, P. Fenaux, C. Gardin,