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.

For Lay version see here

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

Transfusing Young Anemic Women: A Careful Assessment is Required – pro

M Levin, MD — Wed, 20 Jun 2012 17:56:39 +0000

Anemia is common in young women and is usually due to iron deficiency. If anemia has a slower, subacute onset, younger patients without medical problems can tolerate low hemoglobin levels. Often times, however, in addition to subacute or chronic iron deficiency anemia young women can have anemia of acute blood loss. In this situation there is less time for the body to become habituated to the low red cell levels and a moderately low hemoglobin may coexist with acute symptoms of anemia. Blood transfusions may be required in such situations. This is means that a careful and complete clinical assessment is required before blood transfusion is offered and that transfusions should not be given based on low hemoglobin levels alone. Blood transfusions come with some risks 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 was appropriate, or they can be given in the emergency room or in an outpatient setting.
References:

Scott BH, Seifert FC, Grimson R. Blood transfusion is associated with increased resource utilisation, morbidity and mortality in cardiac surgery. Ann Card Anaesth. 2008 Jan-Jun;11(1):15-19.

Rose AH, Kotzé A, Doolan D, Norfolk DR, Bellamy MC. Massive transfusion–evaluation of current clinical practice and outcome in two large teaching hospital trusts in Northern England. Vox Sang. Th. 2008 Jan-Jun;11(1):15-19.

UPTODATE: Practical aspects of red blood cell transfusion in adults: Storage, processing, modifications, and infusionAuthor:Steven Kleinman, MDSection Editor:Lynne Uhl, MDDeputy Editor:Jennifer S Tirnauer, MD 2022

Giancarlo Liumbruno at al, Guidelines for the transfusion of red blood cells
Blood Transfus. 2009 January; 7(1): 49–64.

Framework for the Development of an out of Acute Hospital Blood Transfusion Service – http://www.transfusionguidelines.org.uk/docs/pdfs/bbt-01_sp_tx-framework-v3.pdf, 2007

Read the Layperson version here.

A Compendium of Transfusion Practice Guidelines, 4th ed. American Red Cross. Available at: https://www.redcrossblood.org/content/dam/redcrossblood/hospital-page-documents/334401_compendium_v04jan2021_bookmarkedworking_rwv01.pdf (Accessed on March 17, 2021).

Cancer Treatment Today » Transfusion

Ferriprox for sickle cell – pro

Transfusing Young Anemic Women: A Careful Assessment is Required – pro