Biological therapies need definitive randomized controlled clinical trials.

Adequate and well-controlled clinical trials remain critical tools for helping to bring benefit to patients in medical need.

Iron out for nontransfused thalassemia.

Individuals with ß-thalassemia intermedia and hemoglobinopathies of equivalent severity who are infrequently or never transfused can still develop serious complications of iron overload. Taher et al report the first 1-year, randomized, double-blind, placebo-controlled trial of oral iron chelation therapy with deferasirox in this population.

Transfusion and chelation practices in sickle cell disease: a regional perspective.

Although most common in tropical regions, population migration has meant that sickle cell disease is now one of the most prevalent genetic diseases worldwide. The issues and challenges faced by physicians and patients have been discussed by an international group of experts representing 4 key regions: the USA, Europe, Latin America, and the Middle East/Africa. Conclusive evidence to support the use of transfusion therapy for the prevention of stroke has resulted in key changes to patient management in all regions, and increasing numbers of patients are benefiting from this management approach. However, it is apparent that transfusion therapy is still under-utilized, largely due to concerns over iron overload, alloimmunization, limited blood supplies, and, sometimes, due to parental refusal. Once transfused, assessment and management of body iron levels can be poor, particularly in patients who are intermittently transfused. Compliance with chelation therapy regimens is a significant challenge, but new therapeutic options are likely to overcome some of the current barriers. Key requirements in all regions were considered to be the following: to provide greater physician, patient, and family education; to ensure effective transition from pediatric to adult care; and to establish national guidelines in order to ensure best practice is consistently applied.