Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is currently successfully used in the treatment of many non-malignant hematopoietic disorders, including acquired and inherited bone marrow failure. HSCT from a HLA-identical sibling donor (MSD) is the treatment of choice, better results are achieved using bone marrow graft.
Conditioning regimens and graft-versus-host disease prophylaxis are different in dependence on the underlying disease, age and clinical condition of the patient, type of donor and stem cell graft. Transplantation from MSD is the first-line treatment for children and young adults with SAA.
Patients with hemoglobinopathies and inherited bone marrow failure syndromes with available MSD should be referred for transplantation as soon as possible, before the development of serious complications and iron overload-related tissue damage. Indications for HSCT from matched unrelated donor in the treatment of non-malignant hematopoietic disorders are expanding in dependence on very good results in the last two decades.
The aim of successfully HSCT is not only to increase the percentage of surviving patients but also to improve the quality of life with the minimum of early complications and late effects.