Deferasirox for managing iron overload in people with thalassaemia

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Abstract

Thalassaemia is a hereditary anaemia due to ineffective erythropoiesis. In particular, people with thalassaemia major develop secondary iron overload resulting from regular red blood cell transfusions. Iron chelation therapy is needed to prevent long-term complications.Both deferoxamine and deferiprone are effective; however, a review of the effectiveness and safety of the newer oral chelator deferasirox in people with thalassaemia is needed.

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Most cited references 83

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Beta-thalassemia.

Deborah Rund, Eliezer Rachmilewitz (2005)

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Global epidemiology of haemoglobin disorders and derived service indicators

Bernadette Modell, Matthew Darlison (2008)

To demonstrate a method for using genetic epidemiological data to assess the needs for equitable and cost-effective services for the treatment and prevention of haemoglobin disorders. We obtained data on demographics and prevalence of gene variants responsible for haemoglobin disorders from online databases, reference resources, and published articles. A global epidemiological database for haemoglobin disorders by country was established, including five practical service indicators to express the needs for care (indicator 1) and prevention (indicators 2-5). Haemoglobin disorders present a significant health problem in 71% of 229 countries, and these 71% of countries include 89% of all births worldwide. Over 330 000 affected infants are born annually (83% sickle cell disorders, 17% thalassaemias). Haemoglobin disorders account for about 3.4% of deaths in children less than 5 years of age. Globally, around 7% of pregnant women carry b or a zero thalassaemia, or haemoglobin S, C, D Punjab or E, and over 1% of couples are at risk. Carriers and at-risk couples should be informed of their risk and the options for reducing it. Screening for haemoglobin disorders should form part of basic health services in most countries.

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Survival in medically treated patients with homozygous beta-thalassemia.

Carolyn MacMillan, Magnus A McGee, David N Olivieri … (1994)

The prognosis of patients with homozygous beta-thalassemia (thalassemia major) has been improved by transfusion and iron-chelation therapy. We analyzed outcome and prognostic factors among patients receiving transfusions and chelation therapy who had reached the age at which iron-induced cardiac disease, the most common cause of death, usually occurs. Using the duration of life without the need for either inotropic or antiarrhythmic drugs as a measure of survival without cardiac disease, we studied 97 patients born before 1976 who were treated with regular transfusions and chelation therapy. We used Cox proportional-hazards analysis to assess the effect of prognostic factors and life-table analysis to estimate freedom from cardiac disease over time. Of the 97 patients, 59 (61 percent) had no cardiac disease; 36 (37 percent) had cardiac disease, and 18 of them had died. Univariate analysis demonstrated that factors affecting cardiac disease-free survival were age at the start of chelation therapy (P < 0.001), the natural log of the serum ferritin concentration before chelation therapy began (P = 0.01), the mean ferritin concentration (P < 0.001), and the proportion of ferritin measurements exceeding 2500 ng per milliliter (P < 0.001). With stepwise Cox modeling, only the proportion of ferritin measurements exceeding 2500 ng per milliliter affected cardiac disease-free survival (P < 0.001). Patients in whom less than 33 percent of the serum ferritin values exceeded 2500 ng per milliliter had estimated rates of survival without cardiac disease of 100 percent after 10 years of chelation therapy and 91 percent after 15 years. The prognosis for survival without cardiac disease is excellent for patients with thalassemia major who receive regular transfusions and whose serum ferritin concentrations remain below 2500 ng per milliliter with chelation therapy.

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Author and article information

Journal

Title: Cochrane Database of Systematic Reviews

Publisher: Wiley

ISSN (Electronic): 14651858

Publication date (Electronic): August 15 2017

Affiliations

[1 ]Medical Center - Univ. of Freiburg, Faculty of Medicine, Univ. of Freiburg; Cochrane Germany; Breisacher Straße 153 Freiburg Germany 79110

[2 ]Cologne Germany

[3 ]Faculty of Medicine and Medical Center - University of Freiburg; Institute for Medical Biometry and Statistics; Stefan-Meier-Str. 26 Freiburg Germany 79104

[4 ]University Hospital Frankfurt, Goethe University; Department of Obstetrics and Gynaecology; Frankfurt Germany

[5 ]Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg; Center for Medical Biometry and Medical Informatics; Stefan-Meier-Str. 26 Freiburg Germany 79104

[6 ]University Medical Center Freiburg; Pediatric Hematology & Oncology, Center for Pediatrics & Adolescent Medicine; Mathildenstrasse 1 Freiburg Germany 79106

[7 ]University Hospital Zurich and University of Zurich; Department of Neonatology; Frauenklinikstrasse 10 Zurich Switzerland