HSCT is the only curative option to date for α-thalassaemia survivors. Patients with α-thalassaemia may require earlier intervention than those with β-thalassaemia as adverse effects of α-thalassaemia starts as early as in utero. As findings from studies conducted on patients with β-thalassaemia demonstrated that it is unnecessary to reach full donor chimerism to achieve transfusion independence, reduced toxicity conditioning can be utilized to minimize transplant-related morbidity and mortality. In order to reduce the likelihood of graft failure and enable the adoption of less toxic preparative regimens, the concept of pre-transplant immunosuppression can be similarly implemented in patients with α-thalassaemia. Recent advances and comparable outcomes seen when using human leukocyte antigen (HLA) matched and mismatched related and unrelated donor sources have also expanded the donor pool. The advent of newer targeted GVHD preventive therapy, which may be added to the conventional regimens, will be particularly useful for patient populations of diverse ethnic backgrounds. All these aspects should be taken into consideration while designing and performing transplants tailored for patients with α-thalassaemia.

From: Chapter 12, CURATIVE THERAPIES FOR α-THALASSAEMIA

Cover of Guidelines for the Management of α-Thalassaemia
Guidelines for the Management of α-Thalassaemia [Internet].
Amid A, Lal A, Coates TD, et al., editors.
Nicosia (Cyprus): Thalassaemia International Federation; 2023.
© Thalassaemia International Federation.

NCBI Bookshelf. A service of the National Library of Medicine, National Institutes of Health.