Quality of life in thalassemia patients after unrelated hematopoietic stem cell transplantation

Thalassemia major is a chronic, progressive hemoglobin disorder requiring life-long transfusion and iron chelation therapy for survival. Extensive ongoing medical care in this pathology may have a considerable impact on the physical, psychosocial well-being and quality of life (QoL) of patients and their families. Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only cure available for thalassemia. Unfortunately, only a substantial minority of patients have an HLA compatible donor within the family. More than 70 percent of patients need to search for an unrelated compatible donor in voluntary donor registries worldwide. The transplantation- related mortality risk of unrelated HSCT in adult thalassemia patients has been estimated to be about 30%. For this reason, patients without a family donor are faced with the difficult choice between accepting the high risks of radical treatment or putting aside their hopes of recovery and continuing regular transfusion and iron chelation therapy. This complex decision-making process raises several social, psychological and ethical problems. The central ethical issue of HSCT in a non-malignant disease can be framed in terms of a conflict between two fundamental principles of medical ethics: non-maleficence and beneficence; the question arises as to who will be responsible for the decision and what criteria can be used to strike a fair balance of benefit and burden. The QoL in patients surviving HSCT is not very different from that observed in conventionally treated patients. This makes it important for the physician to direct care at the whole person rather than just ofthe disease. Another important ethical principle is the respect of patient autonomy.