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Favorable Outcomes from Allogeneic Hematopoietic Cell Transplantation in Thailand for Thalassemias and Hemoglobinopathies
Objectives: To assess outcomes of HCT for thalassemias and hemoglobinopathies in single medical center in Thailand.
Methods: Case series study for thalassemia and hemoglobinopathy patients undergoing HCT at Bangkok Hospital Medical Center from February 2009 thru October 2013.
Results: There were totally 14 patients. 10 cases were Thais, 1 was French-Thai, 1 was Bangladeshi, 1 was Lao, and 1 was Omani who had sickle cell disease (SCD). 12 cases were diagnosed as beta-thalassemia/hemoglobin E diseases, 1 as transfusion-dependent alpha-thalassemia, and 1 as SCD. Among 14 HCTs, 9 patients underwent bone marrow transplant (BMT), 4 patients underwent umbilical cord blood transplant (CBT), and 1 patient underwent combined cord blood and marrow transplantation. All 14 related donors were fully-HLA-matched, 4 had normal typing, 9 had thalassemia trait, and 1 had sickle cell trait. Male to female patients ratio were 11:3. Patients’ ages at transplant varied from 2 years to 15 years 11 months with median of 4 years 10 months. Patient’s body weight varied from 11.1 to 50 kilogram (median 17.2 kilogram). According to Pesaro classification, among 13 thalassemia patients there were 8 class I, and 5 class II patients. Busulfan, fludarabine, and rabbit ATG were mainly used as myeloablative conditioning regimen. Cyclosporine and short-course methotrexate were mainly used as graft-versus-host disease (GvHD) prophylaxis in BMT group, while cyclosporine alone was used in CBT group. CD34+ cell doses per kilogram body weight recipients were ranged from 5.6 to 34.7x10^6 (median 11.3x10^6) in BMT group (n=9), and from 1.6 to 3x10^5 (median 2.3x10^5) in CBT group (n=4). Complete donor engraftments were achieved in 11 patients. Mixed-chimerism states with donor predominance were present in 2 patients from BMT and 1 patient from CBT group. No patients experienced graft failure. Neutrophil recoveries were evident on days +10 to +23 (median day +14), and platelet recoveries were observed on days +19 to +64 (median day +40). 2 patients had mild veno-occlusive diseases and were later completely reversible. No patients developed acute or chronic GvHD. There were no mortalities. 1 patient had treatable pneumocystis pneumonia at 4 months post CBT. Median follow-up time for all patients was 2 years (1 month to 4 years 8 months). Overall (OS) and disease-free survival (DFS) were 100% and 100% for all patients (n=14). Based on risk class, the OS and DFS for class I thalassemia patients (n=9) were 100% and 100%, and class II patients (n=4) were 100% and 100%, respectively.
Conclusions: Our experiences in HCT for thalassemias and hemoglobinopathies were very favorable. Regular follow-up visits are encouraged to determine long term outcomes.