Our study was designed to prospectively determine whether or not unrelated cord blood transplantation (CBT) can produce outcomes comparable to related donor transplantation for children with b-thalassaemia. In 35 patients, 40 transplants were performed between October 2003 and September 2009. HLA matching at enrolment was 6/6 (n ¼ 8), 5/6 (n ¼ 16), 4/6 (n ¼ 27), or 3/6 (n ¼ 1) by low-resolution HLA-A, -B, and high-resolution DRB1. These patients received non-manipulated grafts without ex vivo expansion or T-cell depletion. The median number of nucleated and CD34 þ cells infused was 7.8 Â 10 7 /kg (range, 2.8-14.7 Â 10 7 /kg) and 4.0 Â 10 5 /kg (range, 1.7-19.9 Â 10 5 /kg), respectively. The 5-year OS and thalassaemia-free survival after the first transplant were 88.3 and 73.9%, respectively. The cumulative incidence of TRM at 2 years was 11.7%. Fourteen patients developed chronic skin GVHD. Thirty patients were alive and transfusion-independent with a Lansky performance score X80% achieved between 6 and 76 months post transplant (median, 36 months). These data compare acceptably with the survival rates of related-donor BMT for thalassaemia and suggest that patients without an available HLAcompatible sibling but who have well-matched unrelated donors should also be considered for CBT.

The finding that children after transplant may achieve quality of life similar to their healthy peers is important information for parents to consider as they consider treatment options. For those sick children who cannot regularly attend school, their emotional and social functioning should be closely monitored.

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