Unrelated cord blood transplantation for thalassaemia: a single-institution experience of 35 patients

Abstract

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.

A novel conditioning regimen improves outcomes in β-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation

“…Compared with these aforementioned studies, our follow-up time was shorter and the age of our patients was younger. Our results were also comparable to the outcomes of Jaing et al 22 for unrelated cord blood transplants for patients with TM: of the 40 unrelated cord blood transplants that were studied, the 5-year OS and TFS was 88.3% Ϯ 6.7% and 73.9% Ϯ 7.4%, respectively, and the cumulative incidence of TRM at 2 years was 11.7% Ϯ 6.7%.…”

Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel

“…These remarkably good results are likely attributable to the high number of total nucleated cells/kg infused (7.8x10 7 /kg). 44 Combining data from 3 different registries, Ruggeri et al found the outcome of UCBT in TM to be much less favorable; in 35 TM patients, an OS of 62% with a DFS of only 21% was reported. The cumulative incidence of graft failure was 52%.…”

Development and Recent Progresses of Gene Therapy for β-Thalassemia

“…10,11 The development of new techniques to improve the management of graft versus-host disease (GVHD), and to perform HSCT from unrelated donors or cord blood stem cells may expand the pool of potential donors in the near future; currently they remain largely investigational. [12][13][14] Because of these significant disadvantages, and shortage of suitable donors, HSCT is limited to less than one third of the patients; for the vast majority of thalassemic subjects lacking an HLA-matched donor, clinical improvement or even a final cure might be achieved by gene therapy approaches.…”