Haploidentical HSCT with post-transplant CY for primary immunodeficiencies and inherited disorders in children

Allogenic hematopoietic stem cell transplantation (HSCT) is a treatment option for children with various malignant or non-malignant diseases, including selected primary immunodeficiencies (PIDs) and inherited disorders. A graft from a healthy, HLA-genoidentical, matched sibling donor is the best option but is available in fewer than 25% of cases. Less than 70% of the remaining patients will have a suitable matched unrelated donor, a proportion that is even lower for patients belonging to ethnic groups poorly represented in donor registries [1].
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research