Ferriprox for MDS – pro
DERRIPROX® (deferiprone) is an iron chelator indicated for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. The approval in second line is reasonable both because the studies for approval were done in second line and because Ferroprox may be inferior to Exjade in first line (Cemak et al). This is not an innocuous drug; the most serious side effect seen in about two percent of patients treated with Ferriprox was the development of agranulocytosis, a serious and potentially life-threatening reduction in the number of granulocytes (a type of white blood cell that fights infection).
The recent BMJ guidelines says: “Iron chelation therapy cannot be routinely recommended for MDS patients with transfusional iron overload. Grade 1C
Consideration may be given to chelation therapy for patients with a very good prognosis, specifically patients with WHO refractory anaemia (RA), RA with ringed sideroblasts (RARS) and isolated del(5q). Triggers may include more than 20 units of red cells transfused, serum ferritin >1000 μg/l in patients for whom continuing red cell transfusion is predicted. Grade 2C
Patients treated with iron chelation therapy should ideally receive this treatment within clinical trials.
Desferrioxamine remains the therapy of choice with the longest record of safety and efficacy of all three agents available. Deferasirox is recommended for patients intolerant of desferrioxamine. Deferiprone could be considered in patients with normal baseline neutrophil counts. Grade 2C
Note: If approving for a different duration then the standard duration provided by the client noted above, please indicate why in a brief sentence or two.
Cermak, Jaroslav,2011, http://www.eventure-online.com/eventure/publicAbstractView.do?id=161926&congressId=4634
Cermak J, Jonasova A, Vondrakova J, Walterova L, Hochova I, Siskova M, Neuwirtova R.Efficacy and safety of administration of oral iron chelator deferiprone in patients with early myelodysplastic syndrome. Hemoglobin. 2011;35(3):217-27.
Ferriprox, Prescribing Information, 2013
Guidelines for the diagnosis and management of adult myelodysplastic syndromes,BMJ Volume 164, Issue 4
Pages: 467-616
February 2014
Correction(s) for this article
Sally B. Killick
Mhairi Mitchell,1 Steven D Gore, and Amer M Zeidan, Iron chelation therapy in myelodysplastic syndromes: where do we stand?Expert Rev Hematol. 2013 Aug; 6(4): 397–410.