¹The Pediatric Blood and Marrow Transplant Program, Duke University Medical Center, Durham, NC, USA

Correspondence: Dr VK Prasad, Pediatric Blood and Marrow Transplant, Duke University Medical Center, Box 3350, 2400 Pratt Street, Suite 1400, Durham, NC 27710, USA. E-mail: vinod.prasad@duke.edu

Received 24 October 2007; Revised 14 November 2007; Accepted 14 November 2007; Published online 7 January 2008.

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) can prolong life and improve its quality in patients with inherited metabolic diseases. HSCT offers a permanent source of enzyme replacement therapy and also might mediate nonhematopoietic cell regeneration or repair. Unrelated cord blood is an exciting newer graft source for treatment of patients with these fatal disorders, providing increased access to donors and significant clinical efficacy, particularly when transplantation is performed in early stages. Pre-transplant performance status is highly predictive of overall survival.