27 October 2020
Evaluating thalassemia treatments
Published online 2 January 2010
Although new treatments to control thalassaemia intermedia (TI) have been developed, data on the efficacy of these measures remain sparse. Researchers from Lebanon, Egypt, the United Arab Emirates, Oman, Iran and Italy, however, have conducted the largest overview yet of patients with TI and the effects of treatment on disease-related complications in six different centers.
The results highlight concern about the often-used splenectomy approach. Researchers found higher rates of most complications in splenectomized patients compared with those who did not undergo the procedure. This was attributed to higher platelet counts and increased thrombin, leading to aggregation, and often increasing rates of thrombosis and pulmonary hypertension (PHT). A higher incidence of iron overload in splenectomized patients also suggests that the spleen might play a role in scavenging excess iron.
Researchers suggest that those patients considered for splenectomy might benefit from earlier intervention with transfusion therapy to reduce complications. Although this might increase iron accumulation, iron chelation can be used to counter that. Earlier intervention was further emphasized because iron overload and other complications increased with age. The research also echoes other studies on sickle-cell disease in which a beneficial role for hydroxyurea was observed.
The study calls for further clinical trials to evaluate therapies and put in place clear guidelines for handling TI.
- Taher, A. T. et al. Overview on Practices in Thalassemia Intermedia Management Aiming for Lowering Complication-rates Across a Region of Endemicity: the OPTIMAL CARE study. Blood 23 Dec 2009 doi: 10.1182/blood-2009-09-243154