TY - JOUR
T1 - The Impact of Donor Type on Outcomes and Cost of Allogeneic Hematopoietic Cell Transplantation for Pediatric Leukemia
T2 - A Merged Center for International Blood and Marrow Transplant Research and Pediatric Health Information System Analysis
AU - Arnold, Staci D.
AU - Brazauskas, Ruta
AU - He, Naya
AU - Li, Yimei
AU - Hall, Matt
AU - Atsuta, Yoshiko
AU - Dalal, Jignesh
AU - Hahn, Theresa
AU - Khera, Nandita
AU - Bonfim, Carmem
AU - Hashmi, Shahrukh
AU - Parsons, Susan
AU - Wood, William A.
AU - Steinberg, Amir
AU - Freytes, César O.
AU - Dandoy, Christopher E.
AU - Marks, David I.
AU - Lazarus, Hillard M.
AU - Abdel-Azim, Hisham
AU - Bitan, Menachem
AU - Diaz, Miguel Angel
AU - Olsson, Richard F.
AU - Gergis, Usama
AU - Seber, Adriana
AU - Wirk, Baldeep
AU - LeMaistre, C. Fred
AU - Ustun, Celalettin
AU - Duncan, Christine
AU - Rizzieri, David
AU - Szwajcer, David
AU - Fagioli, Franca
AU - Frangoul, Haydar
AU - Knight, Jennifer M.
AU - Kamble, Rammurti T.
AU - Mehta, Paulette
AU - Schears, Raquel
AU - Satwani, Prakash
AU - Pulsipher, Michael A.
AU - Aplenc, Richard
AU - Saber, Wael
N1 - Publisher Copyright:
© 2020 American Society for Transplantation and Cellular Therapy
PY - 2020/9
Y1 - 2020/9
N2 - 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.
AB - 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.
KW - Acute leukemia
KW - Bone marrow transplant
KW - Cost
KW - Donor type
KW - Healthcare utilization
KW - Hematopoietic cell transplant
KW - Outcomes
KW - Pediatric
KW - Stem cell transplant
KW - Transplant
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UR - http://www.scopus.com/inward/citedby.url?scp=85087396573&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2020.05.016
DO - 10.1016/j.bbmt.2020.05.016
M3 - Article
C2 - 32464284
AN - SCOPUS:85087396573
SN - 1083-8791
VL - 26
SP - 1747
EP - 1756
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 9
ER -