β-
Thalassemia major (β-TM) patients require life-long
blood transfusions, resulting in
iron overload with multi-organ morbidity and mortality. Evidence from small randomized controlled trials (RCTs) published to date for
deferiprone (
DFP) monotherapy or in combination with
deferoxamine (DFO) is unclear. We summarized evidence on the efficacy of
DFP monotherapy compared to DFO, and
DFP-DFO combination
therapy compared to
DFP or DFO monotherapy in chronically transfused β-TM. We searched four electronic databases and examined the grey literature. Two authors independently assessed trial quality and extracted data. We calculated the relative risk for dichotomous outcomes and mean difference (MD) for continuous outcomes. We identified 15 RCTs (1003 participants) that met the inclusion criteria.
Deferiprone was more efficacious than DFO in improving cardiac ejection fraction [MD 2.88, 95% CI (95% confidence interval) 1.12 to 4.64, p = 0.001) and endocrine dysfunction (MD 0.09, 95% CI 0.08 to 0.10, p < 0.00001). The
DFP-DFO combination
therapy was more efficacious than
DFP or DFO monotherapy in improving cardiac ejection fraction (MD 5.67, 95% CI 1.32 to 10.02, p = 0.008). There was no significant difference in all other outcomes examined. Meta-analysis on changes in myocardial
iron content was not possible due to differences in data presentation. The quality of evidence for all outcomes was low. There is currently insufficient evidence to show that
DFP is superior to DFO in the treatment of
iron overload. The use of
DFP must be weighed against the potential side-effects, patient compliance and preference. Large RCTs with clinically relevant outcomes are required.