Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival

Hematopoietic stem cell transplantation in Omenn syndrome: a single-center experience

“…Myers et al 21 have recently shown that early HSCT in the neonatal period leads to superior thymic output and thus improved survival in infants with SCID. While early recognition is essential to maximize the chances of successful outcome, we have been able to obtain prolonged survival and sustained immune reconstitution even in patients transplanted as late as at 18 months of life.…”

Hematopoietic stem cell transplantation for primary immunodeficiency disease

“…4,6 In addition, transplants performed within the first month of life are associated with more rapid T-cell reconstitution, perhaps due to superior thymic capacity. 7 Impact of SCID phenotype T-B þ NKÀ SCID The most common form of SCID, accounting for approximately 50% of all cases, is due to a defect in the gene for the common gamma chain (gc) located on Xq13. Another defect, JAK3 deficiency, results in a similar T-B þ NKÀ phenotype (Table 2).…”

Unrelated cord blood transplantation for severe combined immunodeficiency and other primary immunodeficiencies

“…5,6 Similarly, pre-or postnatal diagnosis of SCID and the use of HCT in the neonatal period led to significantly better results: 95% survival in a series of 21 neonates. 12 Despite these advances, immunologic recovery in haploidentical transplants continues to be slower and less complete than with HLAidentical siblings and over 60% of survivors require permanent intravenous immunoglobulin (IVIg) treatment. 5,6,13 On the other hand, results of non-SCID immunodeficiencies, particularly WAS and other T-cell deficiencies, remain poor.…”