A complicated disease: what can be done to manage thalassemia major more effectively?

Abstract

Patients with thalassemia major suffer from many complications, but in the last two decades their lives have improved both in length and quality. We report herein the most common complications and the recent advances that have changed the course of this disease. Also, we report in detail some of the new therapeutic strategies already introduced in practice and briefly some that are still being developed.

Keywords:

• bisphosphonates
• gene therapy
• HbF reactivation
• iron chelation
• iron overload
• JAK2
• magnetic resonance imaging
• osteoporosis
• sotatercept
• stem cell transplantation