Faculty Collaboration Database

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. Biol Blood Marrow Transplant 2020 Sep;26(9):1747-1756

Date

05/29/2020

Pubmed ID

32464284

Pubmed Central ID

PMC7518194

DOI

10.1016/j.bbmt.2020.05.016

Scopus ID

2-s2.0-85087396573 (requires institutional sign-in at Scopus site)   6 Citations

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.

Author List

Arnold SD, Brazauskas R, He N, Li Y, Hall M, Atsuta Y, Dalal J, Hahn T, Khera N, Bonfim C, Hashmi S, Parsons S, Wood WA, Steinberg A, Freytes CO, Dandoy CE, Marks DI, Lazarus HM, Abdel-Azim H, Bitan M, Diaz MA, Olsson RF, Gergis U, Seber A, Wirk B, LeMaistre CF, Ustun C, Duncan C, Rizzieri D, Szwajcer D, Fagioli F, Frangoul H, Knight JM, Kamble RT, Mehta P, Schears R, Satwani P, Pulsipher MA, Aplenc R, Saber W

Authors

Ruta Brazauskas PhD Associate Professor in the Institute for Health and Equity department at Medical College of Wisconsin
Jennifer M. Knight MD, MS Associate Professor in the Psychiatry and Behavioral Medicine department at Medical College of Wisconsin
Wael Saber MD, MS Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adolescent
Adult
Child
Child, Preschool
Health Information Systems
Hematopoietic Stem Cell Transplantation
Humans
Infant
Leukemia, Myeloid, Acute
Retrospective Studies
Unrelated Donors
Young Adult