Ferriprox for sickle cell anemia

M Levin, MD — Wed, 17 Jul 2013 18:30:31 +0000

FERRIPROX® (deferiprone) is an iron chelator indicated for the treatment of patients with iron overload form transfusions due to thalassemia syndromes when current chelation therapy, most commonly Exjade, 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 bone marrow failure to produce white cells, a serious and potentially life-threatening complications. 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.

For Professional version see here

Transfusing Young Anemic Women: A Careful Assessment is Required

M Levin, MD — Wed, 20 Jun 2012 17:53:14 +0000

Anemia is common in young women and is usually due to iron deficiency. If anemia has a slower, subacute onset, younger patients without significant medical problems can tolerate low hemoglobin levels. Often times, however, in addition to being anemic for some time, young women may have recently bled. In this situation there is less time for the body to become used to the low red cell levels and a moderate anemia may cause symptoms, such as, shortness of breath, rapid heart beating, difficulty in exerting oneself, or profound tiredness. This is means that a careful and complete clinical assessment is required before a blood transfusion is offered and that transfusions should not be given for based on low hemoglobin levels alone. Blood transfusions come with some risks. Transfusions can rarely cause allergic reactions and transmit infections and several studies have shown that patients who receive transfusions, on average, stay in the hospital longer, have higher hospital bills, and are generally more ill than patients who did not receive a transfusion. Some studies have even shown a greater likelihood of death in extensively transfused patients. Since each unit infuses over at least four hours, and time is needed for observation between and after the two units often an admission for transfusions can be appropriate, or they can be given on the outpatient basis or in the emergency room.

Read the Professional version here.

Cancer Treatment Today » Transfusion

Ferriprox for sickle cell anemia

Transfusing Young Anemic Women: A Careful Assessment is Required