Ferriprox for sickle cell – pro

M Levin, MD — Wed, 17 Jul 2013 18:25:17 +0000

FERRIPROX® (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 therapy is being approved under the FDA’s accelerated approval program, designed to provide patients with earlier access to promising new drugs followed by further studies to confirm the drug’s clinical benefit. The accelerated approval program allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on an endpoint that is reasonably likely to predict a clinical benefit to patients, or on an effect on a clinical endpoint other than survival or irreversible morbidity (illness).

ApoPharma has agreed to several post-marketing requirement and commitments. One commitment includes further study of the use of Ferriprox in patients with sickle cell disease who have transfusional iron overload. One such study is: Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease, NCT01835496.

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At this time, there is not sufficient literature support for the use of Ferriptox in sickle cell disease.

Cermak, Jaroslav,2011, http://www.eventure-online.com/eventure/publicAbstractView.do?id=161926&congressId=4634

Ferriprox, Prescribing Information, 2013

Testing for Alpha-thalassemia

M Levin, MD — Sun, 21 Apr 2013 12:31:49 +0000

The human alpha gene is found on the short arm of chromosome 16 and there are two hemoglobin alpha chain genes, HBA1 and HBA2. Almost all alpha thalassemias result from a loss of one or more of the alpha genes; the more genes dropped, the more severe is the disease. Unlike Beta-thalssemia, which is found around the Mediterranean, alpha-thalassemias is more geographically widespread and can be found in the descendents of people from Africa and Asia, as well as the Mediterranean. . Molecular genetic testing of HBA1 and HBA2 detects loss of genes in about 90% and point mutations inside a gene in about 10% of affected individuals.

Testing is indicated for:
•Patients with symptoms of alpha-thalassemia or anemia with smaller read cell size.
•Family members of an affected patient who are at risk to be carriers of alpha-thalassemia.
•Carrier screening for individuals of Asian, African, and Mediterranean background.

Individuals with beta thalassssemia trait rarely have anemia and another coexistent gemoglobin mutation should be looked for, if anemia is present. Workup consists of laboratory evaluation, especially CBC, hemoglobin electrophoresis, and genetic testing.

For Professional version see here

Cancer Treatment Today » thalassemia

Ferriprox for sickle cell – pro

Testing for Alpha-thalassemia