Ferriprox for sickle cell – pro

The data to support deferoxamine and a similar drug deferiprone use in other disorders associated with iron overload than thalassemia is limited. Deferoxamine appears capable of increasing iron excretion in sickle cell patients.50 Intermittent IV DFO infusions were effective in decreasing liver iron concentrations in two small cohorts of sickle cell anemia patients. More recent trials have assessed the efficacy of deferiprone in young children. El Alfy and colleagues reviewed its use in 100 children (mean age 5.1 ± 2.4 years) with thalassemia or sickle cell disease.68 At the end of the 6 month treatment period there was a significant drop in mean serum ferritin levels from 2532 ± 1463 ng/L to 2176 ± 1144 ug/L (p<0.05). Similar results were reported in another trial of pediatric patients (median age 10.6 years).69 Mean serum ferritin levels decreased from a mean of 4,677 ± 1,130 ng/L at baseline to 3,363 ± 1,149 ng/L following a median treatment duration of 11.4 months (range 1.4-39.6 months).

There is more evidence for other chelators. A Cochrane review addressed the use Exjade, deferasirox, for the treatment of iron overload. In assessing its use in sickle cell anemia patients the reviewers concluded that deferasirox was as effective as deferoxamine in reducing iron overload.

 

Meerpohl JJ, Antes G, Rücker G. Deferasirox for managing Transfusional Iron
Overload in People with Sickle Cell Disease. Cochrane Database Syst Rev. 2010;4:CD007477.

Cohen A, Schwartz E. Excretion of Iron in Response to Deferoxamine in Sickle
Cell Anemia. J Pediatr. 1978;92:659-62.

Kalpatthi R, Peters B, Holloman D, et al. Safety and Efficacy of High Dose
Intravenous Desferoxamine for Reduction of Iron Overload Due to Chronic
Transfusion in Sickle Cell Patients. Blood. 2008;112:abst 1430.

Silliman CC, Peterson VM, Mellman DL, et al. Iron Chelation by Deferoxamine
in Sickle Cell Patients with Severe Transfusion-induced Hemsiderosis: A
Randomized, Double-blind Study of the Dose-response Relationship. J Lab Clin
Med. 1993;122:48-54.

Ferriprox,Prescribing Information, 2016

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