A Report of Transplantation of 224 Patients with Beta Thalassemia Major in Tehran, 1991– 2004.

Introduction: Beta Thalassemia Major is one of the most common single gene disorders and the most common type of hemoglobinopathies in Iran, with more than 20,000 patients. North and South parts of Iran have the highest prevalence, although it exists all over the country. In Iran, the first thalassemic patient was transplanted in our center, in 1991.

Methods and patients: Till now (August 2004) 224 Beta Thalassemia Major Patients have been transplanted in this center. Age range =2–17 years, M/F=123/101, Class I=99, class II=81 and class III= 44. 124 patients (55.4%) received Bone Marrow, 94 (42%) Peripheral Blood stem cell and 6 (2.7%) Cord Blood for transplantation. Their conditioning regimen in class I and II included Busulfan 3.5mg/kg for 4days, Cyclophosphamide (CY) 50mg/kg for 4 days and their GVHD prophylaxis regimen was Cyclosporine (CYX) 3mg/kg (IV from day −2 till day +5) and 12.5mg/kg (PO after then) and in class III, included Busulfan 4mg/kg for 4days and CY 40mg/kg for 4days and their GVHD prophylaxis regimen was CYX plus MTX 10mg/m² on day +1 and 6mg/m2 on days +3 and +6.

Results:188 (84%) patients are alive and 36(16.1%) deceased.188 (84%) patients have passed 100 days after transplantation.168 (75%) patients had developed clinical acute GVHD after transplantation (grade II–IV= 57.1%). 67(35.6%) patients developed chronic GVHD (44 limited, 23 extensive). Three to ten year overall survival was 81% and disease free survival was 70 %.

Conclusion: Blood and marrow transplantation (based on other documented studies and our experience) is the treatment of choice for class I& II and is indicated in class III thalassemic patients.