PD02 A Literature Review Of Treatment Schemes And Effects For Beta Thalassemia In China

Abstract

Introduction

The incidence of beta thalassemia varies greatly in different regions of China. Blood transfusion combined with iron chelation and hematopoietic stem cell transplantation (HSCT) is the main treatment for beta thalassemia in China. This study aimed to reveal the specific treatment schemes used for patients with beta thalassemia and to evaluate their effects.

Methods

A search strategy was developed to identify articles published between 1 January 2010 and 30 August 2021 in the following literature databases: PubMed, Embase, the China National Knowledge Infrastructure, Wanfang Data, and the Chinese BioMedical Literature Database.

Results

The most used iron chelation schemes in China for patients with beta thalassemia included deferoxamine (DFO) monotherapy, deferiprone (DFP) monotherapy, deferasirox (DFX) monotherapy, and combinations of DFP and DFO. Most studies reported that combinations of DFP, DFO, and DFX monotherapy performed better than DFO or DFP monotherapy alone in reducing the blood, heart, and liver iron load. However, the adverse effects of iron chelation affected patient compliance with treatment to a certain extent. Stem cells for patients receiving HSCT in China were mainly donated by human leukocyte antigen (HLA)-matched siblings or unrelated individuals. The five-year overall survival rate after HSCT ranged from 83 to 90 percent, while the five-year beta thalassemia-free survival rate ranged from 65 to 87 percent. Graft-versus-host disease and infection were the most common serious complications experienced by transplant recipients.

Conclusions

For patients in China with beta thalassemia, the most effective iron chelation treatment schemes were combinations of DFP, DFO, and DFX monotherapy. HSCT from HLA-matched siblings or unrelated donors resulted in a significant improvement in the cure rate for beta thalassemia. However, patients still need safer and more effective innovative treatments, and further evidence on existing treatments needs to be generated from larger scale studies in the Chinese population.