Unrelated donor bone marrow transplantation for correction of lethal congenital immunodeficiencies

The majority of children with lethal congenital immunodeficiencies lack histocompatible related bone marrow donors. T-cell depleted haploidentical parental bone marrow transplantation has been used successfully in selected patients with severe combined immunodeficiency (SCID), but it has not benefited most patients with other immunodeficiencies when it has been tried. For these reasons, we undertook a pilot study using closely matched unrelated donors for bone marrow transplantation of children with life-threatening primary immunodeficiencies. Unrelated donor searches were performed for 24 patients and one or more suitable donors were identified for 21 patients. Unrelated donor bone marrow transplantation (URD BMT) has been performed in 18 patients with various diagnoses: SCID (8), Wiskott-Aldrich syndrome (WAS) (2), Chediak-Higashi syndrome (CHS) (2), combined immunodeficiencies (3), Ataxia Telangiectasis (AT) (2), and one patient with combined immunodeficiency and large granular lymphocytosis (1). The overall actuarial survival rate is 59% with excellent results observed for infants with SCID and children with WAS and CHS.