Preliminary Experience of the Pakistani Network for the Cure and Prevention of Thalassaemia Supported by the Cure2Children Foundation.

Abstract

4331 Since May 2008, the Cure2Children Foundation has supported both financially and professionally a network of centers in Pakistan for stem cell transplantation applied to the cure of thalassemia major, a very prevalent disease with more than 60.000 registered cases in that country. The methodology employed consists of matched-related standard bone marrow transplantation after myeloablative chemotherapy as currently employed at the Mediaterranean Institute of Hematology for younger patients with transfusion-dependent thalassermia (thiotepa 10 mg/kg, busulfan 14 mg/kg and cyclophosphamide 200 mg/kg) followed by GVHD/rejection prophylaxis with prednisone/methotrexate/cyclosporin. Management standards for central venous access, severe pancytopenia, immunosuppression, hospital infection control, and other relevant issues have been addressed by local training as well as with web-based data management and videoconferencing. A total of 15 transplants have been performed to date, 7 at an established center (National Institute of Blood Diseases, Karachi), and 8 at newly developed services (5 at the Pakistan Institute of Medical Sciences, Islamabad, and 3 at Shifa International Hospital, Islamabad). Patient characteristics Median age 3.4 years (range 0.9 to 6.2), 7 males and 8 females. Results At a median follow up of 135 days (range 17-336), actuarial thalassemia-free survival is 79% and overall survival 93%. So far two patients had a graft failure and are alive and well after autologous reconstitution. One patient died for an intracranial bleed, one developed grade 3 acute GVHD (which responded promptly to steroid therapy), 4 had subclinical CMV activation and one developed pulmonary tuberculosis (currently on treatment and doing well). No case of chronic GVHD has been observed so far. Conclusions These preliminary results are comparable to those obtained in western centers, detailed updated clinical and cost analysis will be presented. We believe that this ongoing experience might contribute to extend access to allogeneic transplantation for the cure of low-risk thalassemia children in limited resource settings. Disclosures: No relevant conflicts of interest to declare.