Abstract
Transfusion dependency and iron overload are common among patients with myelodysplastic syndromes (MDS) treated with red blood cell (RBC) transfusions. Transfusion dependency is associated with leukemic progression and shorter survival. Guidelines recommend iron chelation therapy to manage iron overload, however little is known about the chelation patterns in daily clinical practice. The objective of this multicenter, retrospective, cross-sectional, observational study was to evaluate iron status and its management in transfusion-dependent MDS patients. A total of 193 patient records from 29 centers were eligible for inclusion. Median patient age was 76, and median age at diagnosis of MDS was 74. Patients had received an average of 13.4 ± 7.6 RBC units in the past 4 months; 44% had received more than 50 units since their MDS diagnosis. Medium serum ferritin was 1,550 μg/L. Ninety patients (46.6%) received iron chelation therapy with either deferoxamine (41%), deferasirox (36%), and deferoxamine followed by deferasirox (23%). There were no statistically significant differences between chelated and nonchelated patients in terms of International Prognostic Scoring System (IPSS), French-American-British (FAB), and/or World Health Organization (WHO) status, though chelated patients had received more RBC transfusions (p = 0.014). Iron chelation therapy may be underutilized in transfusion-dependent patients. Undertreatment can be reduced by complementing sound clinical judgment with the generally accepted guidelines of a serum ferritin level >1,000 μg/L and/or two or more RBC transfusions per month for the past year; considering patients on the basis of their IPSS, FAB, and/or WHO status; and individually tailored treatment regimens. Prospective randomized trials are necessary to establish causally the efficacy of iron chelation therapy in MDS.
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Acknowledgments
This study was sponsored by Novartis Pharma. The authors thank the other investigators in this study, as well as their staff: Marc André, Charleroi; Yves Beguin, Liège; Dominique Boulet, Mons; Dimitri Breems, Antwerpen; Randal D’Dondt, Oostende ; Robrecht De Bock, Wilrijk ; Dries Deeren, Roeselare ; Anne Deweweire, Baudour ; André Efira, Brussel; Walter Feremans, Bruxelles; Augustin Ferrant, Woluwe; Kurt Geldhof, Ieper; Jan Lemmens, Wilrijk; Lucien Noens, Gent; Marjan Petrick, Gent; Pascal Pierre, Arlon; Ann Van de Velde, Edegem; Koen Van Eygen, Kortrijk; Steven Van Steenweghen, Liège; Wim Wynendaele, Bonheiden; all in Belgium. The authors also thank Erin Arizmendi for her editorial, proofreading, and administrative assistance.
Disclosures of conflicts of interest
All authors completed the ICMJE uniform disclosure form for potential conflicts of interest. W. Pluymers is an employee of Novartis Pharma. Abraham and K. MacDonald are employees of Matrix45. By company policy, they are prohibited from owning equity in client organizations (except through mutual funds or other independent collective investment instruments) or contracting independently with client organizations. Matrix45 provides similar services to other biopharmaceutical companies on a nonexclusivity basis.
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Poster presentation at the 2009 Congress of the European Haematological Association, Berlin, Germany, June 4–7, 2009. Concurrent abstract published as: Delforge M, Selleslag D, Triffet A, et al. The use of iron chelation therapy for transfusion-dependent myelodysplastic syndrome patients: a cross-sectional study in Belgium. Haematologica, 2009;94: abstract 0806
Michel Delforge, Dominik Selleslag, and Christophe Ravoet, on behalf of the Belgian Hematology Society.
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Delforge, M., Selleslag, D., Triffet, A. et al. Iron status and treatment modalities in transfusion-dependent patients with myelodysplastic syndromes. Ann Hematol 90, 655–666 (2011). https://doi.org/10.1007/s00277-011-1164-9
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DOI: https://doi.org/10.1007/s00277-011-1164-9