4^th International Conference on Thalassemia, Oct 31 – Nov 1 2009, Delhi, India

Preliminary experience of the Pakistani network for the cure and prevention of thalassaemia supported by the Cure2Children Foundation.

Lawrence B. Faulkner, Tahir Shamsi¹, Saqib Ansari¹, Tasneem Farzana¹, Sadaf Khalid, Luigi Clemente, Naila Yaqub², Tahira Zafar², Kamran Rashid³, Yasir Iqbal³, Pietro Sodani⁴, Cristiano Gallucci⁴, Buket Erer⁴, and Guido Lucarelli⁴.

Cure2Children Foundation, Florence, Italy; ¹National Institute of Blood Diseases, Karachi, Pakistan, ²Pakistan Institute of Medical Sciences, Islamabad, Pakistan; ³Shifa International Hospital, Islamabad, Pakistan; ⁴Mediterranean Institute of Hematology, Rome, Italy.

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 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). 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 14 transplants have been performed to date, 7 at an established center (National Institute of Blood Diseases, Karachi), and 7 at newly developed services (5 at the Pakistan Institute of Medical Sciences, Islamabad, and 2 at Shifa International Hospital, Islamabad).

Patient characteristics: Median age 3.4 years (range 0.9 to 6.2), 6 males and 8 females.

Results: At a median follow up of 108 days (range 2-308), actuarial thalassemia-free survival is 76% and overall survival 92%. 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 thalassemia in limited resource settings.

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