Arnold Staci D, Brazauskas Ruta, He Naya, Li Yimei, Hall Matt, Atsuta Yoshiko, Dalal Jignesh, Hahn Theresa, Khera Nandita, Bonfim Carmem, Hashmi Shahrukh, Parsons Susan, Wood William A, Steinberg Amir, Freytes César O, Dandoy Christopher E, Marks David I, Lazarus Hillard M, Abdel-Azim Hisham, Bitan Menachem, Diaz Miguel Angel, Olsson Richard F, Gergis Usama, Seber Adriana, Wirk Baldeep, LeMaistre C Fred, Ustun Celalettin, Duncan Christine, Rizzieri David, Szwajcer David, Fagioli Franca, Frangoul Haydar, Knight Jennifer M, Kamble Rammurti T, Mehta Paulette, Schears Raquel, Satwani Prakash, Pulsipher Michael A, Aplenc Richard, Saber Wael
Aflac Cancer and Blood Disorder Center Children's Healthcare of Atlanta Emory University, Atlanta, Georgia.
Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin; Division of Biostatistics, Institute for Health and Equity, Medical College of Wisconsin, Milwaukee, Wisconsin.
Biol Blood Marrow Transplant. 2020 Sep;26(9):1747-1756. doi: 10.1016/j.bbmt.2020.05.016. Epub 2020 May 25.
Allogeneic hematopoietic stem cell transplantation (alloHCT) may be associated with significant morbidity and mortality, resulting in increased healthcare utilization (HCU). To date, no multicenter comparative cost analyses have specifically evaluated alloHCT in children with acute leukemia. In this retrospective cohort study, we examined the relationship between survival and HCU while investigating the hypothesis that matched sibling donor (MSD) alloHCT has significantly lower inpatient HCU with unrelated donor (URD) alloHCT, and that among URDs, umbilical cord blood (UCB) alloHCT will have higher initial utilization but lower long-term utilization. Clinical and transplantation outcomes data from the Center for International Blood and Marrow Transplant Research (CIBMTR) were merged with inpatient cost data from the Pediatric Health Information System (PHIS) database using a probabilistic merge methodology. The merged dataset comprised US patients age 1 to 21 years who underwent alloHCT for acute leukemia between 2004 and 2011 with comprehensive CIBMTR data at a PHIS hospital. AlloHCT was analyzed by donor type, with specific analysis of utilization and costs using PHIS claims data. The primary outcomes of overall survival (OS), leukemia-free survival (LFS), and inpatient costs were evaluated using Kaplan-Meier curves and Cox and Poisson models. A total of 632 patients were identified in both the CIBMTR and PHIS data. The 5-year LFS was 60% for MSD alloHCT, 47% for well-matched matched unrelated donor bone marrow (MUD) alloHCT, 48% for mismatched unrelated donor alloHCT, and 45% for UCB alloHCT (P = .09). Total adjusted costs were significantly lower for MSD alloHCT versus MUD alloHCT by day 100 (adjusted cost ratio [ACR], .73; 95% confidence interval [CI], .62 to .86; P < .001), and higher for UCB alloHCT versus MUD alloHCT (ACR, 1.27; 95% CI, 1.11 to 1.45; P < .001). By 2 years, total adjusted costs remained significantly lower for MSD alloHCT compared with MUD alloHCT (ACR, .67; 95% CI, .56 to .81; P < .001) and higher for UCB alloHCT compared with MUD alloHCT (ACR, 1.25; 95% CI, 1.02 to 1.52; P = .0280). Our data show that UCB and MUD alloHCT provide similar survival outcomes; however, MUD alloHCT has a significant advantage in cost by day 100 and 2 years. More research is needed to determine whether the cost difference among URD alloHCT approaches remains significant with a larger sample size and/or beyond 2 years post-alloHCT.
异基因造血干细胞移植（alloHCT）可能会导致显著的发病率和死亡率，从而增加医疗资源的利用（HCU）。迄今为止，尚无多中心比较成本分析专门评估急性白血病患儿的alloHCT。在这项回顾性队列研究中，我们在调查以下假设的同时，研究了生存与HCU之间的关系：与无关供体（URD）alloHCT相比，匹配的同胞供体（MSD）alloHCT的住院HCU显著更低；在URD中，脐带血（UCB）alloHCT的初始利用率更高，但长期利用率更低。使用概率合并方法，将国际血液和骨髓移植研究中心（CIBMTR）的临床和移植结果数据与儿科健康信息系统（PHIS）数据库中的住院成本数据进行合并。合并后的数据集包括2004年至2011年间在PHIS医院接受急性白血病alloHCT且具有全面CIBMTR数据的1至21岁美国患者。根据供体类型对alloHCT进行分析，并使用PHIS索赔数据对利用率和成本进行具体分析。使用Kaplan-Meier曲线以及Cox和Poisson模型评估总生存（OS）、无白血病生存（LFS）和住院成本等主要结局。在CIBMTR和PHIS数据中总共识别出632例患者。MSD alloHCT的5年LFS为60%，配型良好的无关供体骨髓（MUD）alloHCT为47%，配型不合的无关供体alloHCT为48%，UCB alloHCT为45%（P = 0.09）。到第100天时，MSD alloHCT的总调整成本显著低于MUD alloHCT（调整成本比[ACR]，0.73；95%置信区间[CI]，0.62至0.86；P < 0.001），而UCB alloHCT高于MUD alloHCT（ACR，1.27；95% CI，1.11至1.45；P < 0.001）。到2年时，MSD alloHCT的总调整成本仍显著低于MUD alloHCT（ACR，0.67；95% CI，0.56至0.81；P < 0.001），UCB alloHCT高于MUD alloHCT（ACR，1.25；95% CI，1.02至1.52；P = 0.0280）。我们的数据表明，UCB和MUD alloHCT提供相似的生存结局；然而，MUD alloHCT在第100天和2年时在成本方面具有显著优势。需要更多研究来确定，在样本量更大和/或alloHCT后超过2年的情况下，URD alloHCT方法之间的成本差异是否仍然显著。
