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New insights into haematopoietic stem cell transplantation for patients with haemoglobinopathies

Although improvements in conservative treatment have considerably ameliorated the prognosis of haemoglobinopathies, haematopoietic stem cell transplantation (HSCT) still remains the only curative treatment for patients with thalassemia and sickle cell disease (SCD). The majority of patients with thalassaemia given HSCT from a human leucocyte antigen (HLA)-identical sibling can be cured of their disease. Regarding patients with SCD, HSCT is reserved almost exclusively for children who have significant sickle-related morbidity. HSCT from an unrelated volunteer, carefully selected by high-resolution HLA-typing, can be an alternative for patients lacking a compatible family donor, as the results obtained using such donors are comparable with those obtained employing an HLA-identical sibling. Recently, it has been demonstrated that cord blood is as effective as, and possibly safer than, bone marrow for HSCT in paediatric patients. It is possible that, in the future, thalassaemia patients with poor health could benefit from HSCT employing nonmyeloablative, less toxic, conditioning regimens. More than 20 years have elapsed since the first successful allogeneic transplant of haematopoietic stem cells (HSCT), performed in Seattle on a young Italian patient with thalassaemia major (Thomas et al, 1982). Since then, hundreds of patients with this disease and with sickle cell disease (SCD) have been cured of their original disorder after having received HSCT, in most cases using a human leucocyte antigen (HLA)-identical sibling as the donor (Johnson et al, 1984; Vermylen et al, 1988, 1998; Giardini & Lucarelli, 1999). All these transplants have enabled the identification of the variables that influence post-transplant outcome, thus permitting more refined prognostic counselling (Lucarelli et al, 1993, 1996; Walters et al, 1996). HSCT still remains the only curative treatment for patients with haemoglobinopathies and thus it represents a most attractive therapeutic alternative, especially for those patients, and their families, who do not accept the prospective of a lifelong disease that requires continuous treatment with blood transfusion and drugs. However, HSCT is associated with a non-negligible risk of both transplantrelated mortality and morbidity and, especially in Western countries, only less than 25% of patients have a suitable compatible family donor. In view also of the relevant improvements achieved with conventional treatment, i.e. blood transfusion, inducers of c-chain synthesis and chelation therapy, it is worthwhile to discuss why patients with haemoglobinopathies should be offered an allograft, how the number of patients that can be treated with HSCT can be increased and how we can render transplantation safer and more effective.