Low-cost matched sibling bone marrow transplant for standard-risk thalassemia in a limited-resource setting

Thalassemias are the most common inherited genetic disorder in India and a major public health burden with bone marrow transplant (BMT) considered the only established curative therapy. We describe outcomes for patients (n = 71) with standard-risk thalassemia (liver size < 2 cm and age <15 years), receiving BMT in 6 low-cost start up BMT centers in the Indian sub-continent from August-2013 to July-2016. Patients received HLA-matched sibling donor unmanipulated BMT. Conditioning was with busulfan (14 mg/kg oral total over days −10 to −7, serum levels not measured), cyclophosphamide (200 mg/kg total over days −5 to −2) and anti-thymocyte globulin (Genzyme 4 mg/kg or Fresenius 16 mg/kg over days −12 to −10). Kaplan Meier survival analysis revealed thalassemia free survival (TFS) of 83% with overall survival (OS) of 93% with a median follow up of 17.5 months (IQR 13.4-22.6). Twelve percent of the patients rejected, 12% had severe GVHD (7% acute grade 3-4 GVHD and 4% had moderate/severe chronic GVHD). All 3 patients with acute GVHD Grade 3 are off immunosuppression, and those with chronic GVHD are well with Lansky score >80 at the last follow up. 5 patients (7%) died, mortality related to transplant. Enough data existed for 2 centers in India (36/71 transplants) to analyze overall costs from admission up to one-year post-BMT which revealed a median cost of Rs 7,30,445 ($11519) [Range Rs 4,52,821-10,32,842 ($ 7079-16147)]. In conclusion, children with thalassemia in resource limited settings can achieve good outcomes with BMT at a reasonable cost.