Labopin Myriam, Ruggeri Annalisa, Gorin Norbert Claude, Gluckman Eliane, Blaise Didier, Mannone Lionel, Milpied Noel, Yakoub-Agha Ibrahim, Deconinck Eric, Michallet Mauricette, Fegueux Nathalie, Socié Gerard, Nguyen Stephanie, Cahn Jean Yves, de Revel Thierry, Garnier Federico, Faucher Catherine, Taright Namik, Kenzey Chantal, Volt Fernanda, Bertrand Dominique, Mohty Mohamad, Rocha Vanderson
Haematologica. 2014 Mar;99(3):535-40. doi: 10.3324/haematol.2013.092254. Epub 2013 Oct 18.
Double cord blood transplantation extends the use of cord blood to adults for whom a single unit is not available, but the procedure is limited by its cost. To evaluate outcomes and cost-effectiveness of double compared to single cord blood transplantation, we analyzed 134 transplants in adults with acute leukemia in first remission. Transplants were performed in France with reduced intensity or myeloablative conditioning regimens. Costs were estimated from donor search to 1 year after transplantation. A Markov decision analysis model was used to calculate quality-adjusted life-years and cost-effectiveness ratio within 4 years. The overall survival at 2 years after single and double cord blood transplants was 42% versus 62%, respectively (P=0.03), while the leukemia-free-survival was 33% versus 53%, respectively (P=0.03). The relapse rate was 21% after double transplants and 42% after a single transplant (P=0.006). No difference was observed for non-relapse mortality or chronic graft-versus-host-disease. The estimated costs up to 1 year after reduced intensity conditioning for single and double cord blood transplantation were € 165,253 and €191,827, respectively. The corresponding costs after myeloablative conditioning were € 192,566 and € 213,050, respectively. Compared to single transplants, double cord blood transplantation was associated with supplementary costs of € 21,302 and € 32,420 up to 4 years, but with increases in quality-adjusted life-years of 0.616 and 0.484, respectively, and incremental cost-effectiveness ratios of € 34,581 and €66,983 in the myeloablative and reduced intensity conditioning settings, respectively. Our results showed that for adults with acute leukemia in first complete remission in France, double cord transplantation is more cost-effective than single cord blood transplantation, with better outcomes, including quality-adjusted life-years.
双份脐血移植将脐血的使用扩展到无法获得单份脐血的成人患者,但该方法受成本限制。为评估双份脐血移植与单份脐血移植相比的疗效和成本效益,我们分析了134例首次缓解期的成人急性白血病患者的移植情况。这些移植在法国采用降低强度或清髓性预处理方案进行。成本从寻找供体到移植后1年进行估算。采用马尔可夫决策分析模型计算4年内的质量调整生命年和成本效益比。单份和双份脐血移植后2年的总生存率分别为42%和62%(P=0.03),而无白血病生存率分别为33%和53%(P=0.03)。双份移植后的复发率为21%,单份移植后为42%(P=0.006)。在非复发死亡率或慢性移植物抗宿主病方面未观察到差异。降低强度预处理后单份和双份脐血移植至1年的估计成本分别为165,253欧元和191,827欧元。清髓性预处理后的相应成本分别为192,566欧元和213,050欧元。与单份移植相比,双份脐血移植在4年内的额外成本分别为21,302欧元和32,420欧元,但质量调整生命年分别增加了0.616和0.484,在清髓性和降低强度预处理情况下的增量成本效益比分别为34,581欧元和66,983欧元。我们的结果表明,对于法国首次完全缓解期的成人急性白血病患者,双份脐血移植比单份脐血移植更具成本效益,且疗效更好,包括质量调整生命年。