Prospective evaluation of the effect of deferasirox on hematologic response in transfusion‐dependent patients with low‐risk MDS and iron overload
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, (...
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Published in | European journal of haematology Vol. 101; no. 2; pp. 165 - 173 |
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Main Authors | , , , , , , , , , , , , , , |
Format | Journal Article |
Language | English |
Published |
England
Wiley Subscription Services, Inc
01.08.2018
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Subjects | |
Online Access | Get full text |
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Summary: | 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 endpoints 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. |
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Bibliography: | Funding information The study was sponsored by Novartis Pharma SAS (Rueil‐Malmaison, France) and designed by the sponsor in close collaboration with the Study Scientific Committee. The data management and statistical analyses were conducted by ICTA Project Management (Fontaine‐lès‐Dijon, France). Authors had full access to the data and participated actively in interpreting data and critically reviewing the article with the assistance of a medical writer funded by the sponsor. ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 |
ISSN: | 0902-4441 1600-0609 |
DOI: | 10.1111/ejh.13088 |