Update on Thalassemia Treatment in Taiwan, Including Bone Marrow Transplantation, Chelation Therapy, and Cardiomyopathy Treatment Effects

Abstract

Over the past few decades, Taiwan has seen striking improvements in the life expectancy of its 400 registered β-thalassemia major (β-TM) patients due mainly to adequate transfusion regimens and effective iron chelation therapy. Since 1995, Taiwanese citizens have enjoyed universal health care through National Health Insurance (NIH), receiving comprehensive treatment at minimal cost. In 1984, a national program for thalassemia prevention, control, and hematopoietic stem cell transplantation (HSCT) was initiated. Recent data show 1- and 2-year event-free survival rates of 85 and 78%, respectively. Chelation agents like deferoxamine (DFO), deferiprone (L1) and deferasirox (DFRA) are available in Taiwan, and therapy is tailored to individuals based on drug availability and tissue distribution of iron load. Intensive chelation regimens combining L1 and DFO are recommended in patients with cardiac complications, while DFRA has been found to be effective in reducing serum ferritin, with acceptable side effects. Here, we report advances in thalassemia treatment in Taiwan and suggest treatment guidelines.

Keywords:

• Thalassemia
• Iron chelator
• Cardiomyopathy
• Hematopoietic stem cell transplantation (HSCT)
• Taiwan
• Guideline

Declaration of Interest: The authors report no conflicts of interest. The authors alone are responsible for the content and writing of this article.