Unrelated donor cord blood transplantation for non-malignant disorders in children and adolescents.

Abstract

This study analyzes the data reported to the Korean Cord Blood Registry between

1994 and 2008, involving children and adolescents with non-malignant diseases.

Sixty-five patients were evaluated in this study: SAA (n = 24), iBMFS, (n = 16),

and primary immune deficiency/inherited metabolic disorder (n = 25). The CI of

neutrophil recovery was 73.3% on day 42. By day 100, the CI of acute grade II-IV

graft-versus-host disease was 32.3%. At a median follow-up of 71 months, five-yr

OS was 50.7%. The survival rate (37.5%) and CI of neutrophil engraftment (37.5%)

were lowest in patients with iBMFS. Deaths were mainly due to infection,

pulmonary complications, and hemorrhage. In a multivariate analysis, the presence

of >3.91 x 10(5) /kg of infused CD34 + cells was the only factor consistently

identified as significantly associated with neutrophil engraftment (p = 0.04) and

OS (p = 0.03). UCBT using optimal cell doses appears to be a feasible therapy for

non-malignant diseases in children and adolescents for whom there is no

appropriate HLA-matched related donor. Strategies to reduce transplant-related

toxicities would improve the outcomes of UCBT in non-malignant diseases.