Department of Clinical Haematology, Haemato-Oncology & Bone Marrow (Stem cell), Transplantation, Christian Medical College, Ludhiana, India.
School of Public Health, Postgraduate Institute of Medical Education & Research, Chandigarh, India.
Biol Blood Marrow Transplant. 2018 Oct;24(10):2119-2126. doi: 10.1016/j.bbmt.2018.04.005. Epub 2018 Apr 16.
Hematopoietic stem cell transplantation (HSCT) is the only cure for thalassemia major (TM), which inflicts a significant 1-time cost. Hence, it is important to explore the cost effectiveness of HSCT versus lifelong regular transfusion-chelation (TC) therapy. This study was undertaken to estimate incremental cost per quality-adjusted life-year (QALY) gained with the intervention group HSCT, and the comparator group TC, in TM patients. A combination of decision tree and Markov model was used for analysis. A hospital database, supplemented with a review of published literature, was used to derive input parameters for the model. A lifetime study horizon was used and future costs and consequences were discounted at 3%. Results are presented using societal perspective. Incremental cost per QALY gained with use of HSCT as compared with TC was 64,096 (US$986) in case of matched related donor (MRD) and 1,67,657 (US$2579) in case of a matched unrelated donor transplantation. The probability of MRD transplant to be cost effective at the willingness to pay threshold of Indian per capita gross domestic product is 94%. HSCT is a long-term value for money intervention that is highly cost effective and its long-term clinical and economic benefits outweigh those of TC.
造血干细胞移植（HSCT）是重型地中海贫血（TM）的唯一治愈方法，需要一次性支付高昂的费用。因此，探索 HSCT 与终身定期输血-螯合（TC）治疗相比的成本效益非常重要。本研究旨在评估干预组 HSCT 和对照组 TC 治疗 TM 患者的增量成本效益比（每获得一个质量调整生命年[QALY]的增量成本）。采用决策树和马尔可夫模型相结合的方法进行分析。利用医院数据库，并结合文献复习，为模型输入参数。使用终生研究时间范围，未来成本和结果以 3%的贴现率贴现。结果从社会角度呈现。与 TC 相比，使用 HSCT 的增量成本效益比在匹配相关供者（MRD）的情况下为 64096 美元（986 美元），在匹配无关供者移植的情况下为 167657 美元（2579 美元）。MRD 移植在印度人均国内生产总值支付意愿阈值下具有成本效益的概率为 94%。HSCT 是一种长期具有成本效益的干预措施，其长期的临床和经济效益超过 TC。
