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

TitleLow-cost matched sibling bone marrow transplant for standard-risk thalassemia in a limited-resource setting
Publication TypeJournal Article
Year of Publication2017
AuthorsRamprakash, S, Agarwal, R, Dhanya, R, Marwah, P, Soni, R, Yaqub, N, Fatima, I, Zhara, T, Gooneratne, L, Williams, S, Khalid, S, Sen, S, Kanwar, V, Faulkner, L
Publication NamePediatric Hematology Oncology Journal
KeywordsC2C full text article, Cost, Cost analysis, Cost Control, Developing countries, Frugal innovation, Healthcare cost, HSCT, India, LMIC vs HIC, Thalassemia

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.