New strategies for haploidentical transplantation

Haploidentical transplantation in children opens the possibility to offer this treatment to every child with an otherwise incurable disease, such as some hematological or oncological malignancies, inborn or acquired bone marrow-failure syndromes, hemoglobinopathies, immunodeficiencies, or other genetic diseases. Although initial attempts at haploidentical transplantation were associated with a high transplant-related mortality, recent insights into the biology of haploidentical transplantation, the availability of effective in vivo large-scale graft-manipulation technology, and improved supportive care strategies have led to and are still leading to significantly better outcomes of haploidentical transplantation as compared with previous decades. In addition, expensive and time-consuming searches for matched unrelated donors (MUDs) as well as the expensive establishment and maintenance of cord blood banks are not necessary. Moreover, the worldwide donor registries comprise mainly donors of Caucasian origin and patients of non-Caucasian origin have a lower chance of finding a MUD. Therefore, haploidentical transplantation allows the treatment of children independently of their ethnic background in a timely fashion according to the status of their underlying disease.