The impact of donor type on outcomes and cost of allogeneic hematopoietic cell transplant for pediatric leukemia: a merged CIBMTR and PHIS analysis: Pediatric acute leukemia transplant risks and utilization.

2020

2020 May 25

1523-6536

<p><strong>IMPORTANCE: </strong>AlloHCT may be associated with significant morbidity and mortality that result in increased healthcare utilization. To date, no multi-center comparative cost analyses have been performed specifically evaluating alloHCT in children with acute leukemia.</p>

<p><strong>OBJECTIVES: </strong>To describe the relationship between survival and healthcare utilization while investigating the hypothesis that matched sibling donor (MSD) alloHCT has significantly lower inpatient healthcare utilization compared to unrelated donor (URD) and that among URD, umbilical cord blood transplants (UCB) will have higher initial but lower long-term utilization.</p>

<p><strong>DESIGN: </strong>Retrospective cohort study Setting: Clinical and transplant 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.</p>

<p><strong>PARTICIPANTS: </strong>The merged dataset contained U.S. patients age 1-21 years who received alloHCT for acute leukemia from 2004-2011 with comprehensive CIBMTR data at a PHIS hospital.</p>

<p><strong>EXPOSURE: </strong>AlloHCT analyzed by donor type with specific analysis of utilization and costs using PHIS claims data.</p>

<p><strong>MAIN OUTCOME: </strong>The primary outcomes of overall survival (OS), leukemia free survival (LFS), and inpatient costs were evaluated using Kaplan-Meier curves, Cox, and Poisson models.</p>

<p><strong>RESULTS: </strong>632 patients were identified in both CIBMTR and PHIS. 5-year LFS was 60% for MSD, 47% for well-matched matched unrelated donor bone marrow (MUD), 48% for mismatched unrelated donor, and 45% for UCB (p=0.09). Total adjusted costs were significantly lower for MSD versus MUD by day 100 (adjusted cost ratio (ACR) 0.73, CI 0.62-0.86, p&lt;0.001), and higher for UCB versus MUD (ACR 1.27, CI 1.11-1.45, p&lt;0.001). By 2yrs, total adjusted costs remained significantly lower for MSD when compared to MUD (ACR 0.67, CI 0.56-0.81, p&lt;0.001) and higher for UCB compared to MUD (ACR 1.25, 95% CI 1.02-1.52, p=0.0280).</p>

<p><strong>CONCLUSIONS: </strong>UCB and MUD alloHCT provide similar survival outcomes; however, MUD alloHCT has a significant advantage in cost by day 100 and 2 years. Ongoing research is needed to determine if the cost difference among URD alloHCT remains significant with a larger sample size and/or beyond the 2 years following alloHCT.</p>

10.1016/j.bbmt.2020.05.016

Biol. Blood Marrow Transplant.

32464284

Clinical risks and healthcare utilization of haematopoietic cell transplantation for sickle cell disease in the U.S. using merged databases.

2017

1823-1832

2017 Nov

1592-8721

<p>Allogeneic hematopoietic cell transplant advances for sickle cell disease improve outcomes, but limited healthcare utilization analysis exists. We hypothesized that early transplant (age &lt; 10 years) improves outcomes and decreases healthcare utilization compared to late. We performed a retrospective study of US children transplanted for sickle cell during 2000-2013 using two large databases. Univariate and Cox models estimated associations between demographics, sickle cell severity, and transplant-related variables on mortality and chronic graft versus-host-disease; while, Wilcoxon, Kruskal-Wallis, or linear trend tests estimated against healthcare utilization. 161 patients with 90% 2-year overall survival (95% confidence interval [CI] 85-95%) displayed significantly higher mortality late versus early (hazard ratio (HR) 21, 95% CI 2.8-160.8, p=0.003) and unrelated compared to matched sibling donor (HR 5.9, 95% CI 1.7-20.2, p=0.005) and chronic graft versus host disease late (HR 1.9, 95% CI 1.0-3.5, p=0.034) and unrelated (HR 2.5, 95%CI 1.2-5.4; p=0.017). 176 merged patients had $467,747 median total adjusted transplant cost per patient (range: $344,029-$799,219). Matched sibling donor and low severity had the lowest healthcare utilization compared to other donor and severity types (p&lt;0.001 and p=0.022, respectively); late transplant demonstrated no association (p=0.775). Patients with 2-yr pre- and post-transplant data (N=41) showed significant admission (p&lt;0.001), length of stay (p&lt;0.001), and cost (p=0.008) reductions. Superior early transplant outcomes require prospective studies in young children without severe disease and an available matched sibling to provide conclusive evidence for this approach. Reduced post-transplant healthcare utilization inpatient care indicates that transplant may provide a sustained decrease in healthcare costs over time.</p>

10.3324/haematol.2017.169581

Haematologica

28818869