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- Title
Prospective evaluation of the effect of deferasirox on hematologic response in transfusion-dependent patients with low-risk MDS and iron overload.
- Authors
Rose, Christian; Lenoir, Caroline; Gyan, Emmanuel; Hacini, Maya; Amé, Shanti; Corront, Bernadette; Beyne-Rauzy, Odile; Adiko, Didier; Loppinet, Elena; Ali-Ammar, Nadia; Laribi, Kamel; Wattel, Eric; Dreyfus, François; Roué, Claire S.; Cheze, Stephane
- Abstract
Objectives: To assess the reduction of transfusions rate in transfusion-dependent patients with low-risk myelodysplastic syndrome (MDS) with iron overload treated with deferasirox. Methods: Prospective observational study. Primary endpoint was reduction in transfusion requirements (RTR) at 3 months, (assessed on 8-week period). Secondary end-points were hematologic improvement according to International Working Group (IWG) 2006 criteria at 3, 6, and 12 months. Results: Fifty-seven patients were evaluable. After 3 months of chelation, no effect was seen on transfusion requirement (5.9 packed red blood cells (PRBC) vs 5.8 before chelation). According to the Kaplan-Meier analysis, the probability of RTR at 3, 6, and 12 months was assessed as 3.5%, 9.1%, and 18.7%, respectively. Median duration of RTR was 182 days. However, during the 12-month follow-up after deferasirox initiation, 17 patients (31.5%) achieved minor erythroid response [HI-E] according to IWG criteria, 10 of whom having achieved Hb improvement at month 12. Conclusion: After 3 months of treatment, deferasirox had no impact on transfusion requirement in regularly transfused patients with low-risk MDS. However, deferasirox could induce 31% of erythroid response during the 12-month follow-up period thus suggesting that iron chelation therapy with deferasirox may induce an effect on hematopoiesis in a subset of patients with MDS and iron overload.
- Subjects
DEFERASIROX; HEMATOLOGIC agents; MYELODYSPLASTIC syndromes; ERYTHROCYTES; CHELATION
- Publication
European Journal of Haematology, 2018, Vol 101, Issue 2, p165
- ISSN
0902-4441
- Publication type
Article
- DOI
10.1111/ejh.13088