The Impact of Donor Type on Outcomes and Cost of Allogeneic Hematopoietic Cell Transplantation for Pediatric Leukemia: A Merged Center for International Blood and Marrow Transplant Research and Pediatric Health Information System Analysis

Staci D. Arnold, Ruta Brazauskas, Naya He, Yimei Li, Matt Hall, Yoshiko Atsuta, Jignesh Dalal, Theresa Hahn, Nandita Khera, Carmem Bonfim, Shahrukh Hashmi, Susan Parsons, William A. Wood, Amir Steinberg, César O. Freytes, Christopher E. Dandoy, David I. Marks, Hillard M. Lazarus, Hisham Abdel-Azim, Menachem BitanMiguel Angel Diaz, Richard F. Olsson, Usama Gergis, Adriana Seber, Baldeep Wirk, C. Fred LeMaistre, Celalettin Ustun, Christine Duncan, David Rizzieri, David Szwajcer, Franca Fagioli, Haydar Frangoul, Jennifer M. Knight, Rammurti T. Kamble, Paulette Mehta, Raquel Schears, Prakash Satwani, Michael A. Pulsipher, Richard Aplenc, Wael Saber

Research output: Contribution to journalArticle

Abstract

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.

Original languageEnglish (US)
Pages (from-to)1747-1756
Number of pages10
JournalBiology of Blood and Marrow Transplantation
Volume26
Issue number9
DOIs
StatePublished - Sep 2020

All Science Journal Classification (ASJC) codes

  • Hematology
  • Transplantation

Fingerprint Dive into the research topics of 'The Impact of Donor Type on Outcomes and Cost of Allogeneic Hematopoietic Cell Transplantation for Pediatric Leukemia: A Merged Center for International Blood and Marrow Transplant Research and Pediatric Health Information System Analysis'. Together they form a unique fingerprint.

  • Cite this

    Arnold, S. D., Brazauskas, R., He, N., Li, Y., Hall, M., Atsuta, Y., Dalal, J., Hahn, T., Khera, N., Bonfim, C., Hashmi, S., Parsons, S., Wood, W. A., Steinberg, A., Freytes, C. O., Dandoy, C. E., Marks, D. I., Lazarus, H. M., Abdel-Azim, H., ... Saber, W. (2020). The Impact of Donor Type on Outcomes and Cost of Allogeneic Hematopoietic Cell Transplantation for Pediatric Leukemia: A Merged Center for International Blood and Marrow Transplant Research and Pediatric Health Information System Analysis. Biology of Blood and Marrow Transplantation, 26(9), 1747-1756. https://doi.org/10.1016/j.bbmt.2020.05.016