Pediatric transplant–associated thrombotic microangiopathy health care utilization and implications of eculizumab therapy

• Type of Material: Article
• Authors: Christopher Dandoy, Cincinnati Children’s Medical Center; Michelle Long Schoettler, Emory University; Leslie Lehmann, Harvard Medical School; Pei-Chi Kao, Harvard Medical School; Nan Chen, Harvard Medical School; Sonata Jodele, Cincinnati Children’s Medical Center; Satheesh Chonat, Emory University; Kirsten Marie Williams, Emory University; Wendy B. London, Harvard Medical School; Christine Duncan, Harvard Medical School
• Language: English
• Date: 2024-03-12
• Publisher: The American Society of Hematology
• Publication Version: Final Published Version
• Copyright Statement: © 2024 by The American Society of Hematology
• License: Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International
• Final Published Version (URL): https://doi.org/10.1182%2Fbloodadvances.2023011078
• Title of Journal or Parent Work: Blodd Advances
• Volume: 8
• Issue: 5
• Start Page: 1220
• End Page: 1233
• Grant/Funding Information: The authors are grateful for the funding that supported this work ([M.L.S.] National Institutes of Health LRP and National Institutes of Health K12 National Cancer Institute).
• Keywords: transplant-associated thrombotic microangiopathy (TA-TMA); inpatient costs; criteria; health care use (HCU); Pediatric Health Information System; hospitalization; hematopoietic cell transplant (HCT)
• Research Categories: Health Sciences, Medicine and Surgery; Biology, Cell

Abstract

The health care use (HCU) burden of transplant-associated thrombotic microangiopathy (TA-TMA) and its treatments are unknown. The objective of this study was to investigate inpatient costs associated with meeting criteria for TA-TMA in the first year after hematopoietic cell transplant (HCT). This institutional review board–approved retrospective multicenter study included serial children who underwent HCT from 1 January 2015 to 1 July 2019. A standardized unit cost (adjusted for geographic location, differences in cost of living, and inflation) for inpatient hospitalization was extracted from the Pediatric Health Information System data and linked to clinical data. Both total cost and cost per day from 15 days before stem cell infusion to 1-year after HCT were calculated. Among allogeneic (allo) transplant recipients, after adjusting for severe grade 3/4 acute graft-versus-host disease (GVHD), infections, and HLA mismatch, costs were not different in TA-TMA (n = 137) vs no TA-TMA (n = 238). Severe GVHD was significantly associated with increased costs. Among allo high-risk (HR) TMA-TMA, unadjusted costs were significantly higher in the eculizumab-treated cohort (n = 19) than in the supportive care group (n = 36). However, after adjusting for gastrointestinal bleeding that occurred disproportionately in the eculizumab (n = 6) vs supportive care (n = 0) cohort, eculizumab treatment was not associated with increased total costs. More studies are needed to determine the etiology of increased HCU costs in those with HR-TA-TMA and predict those more likely to benefit from eculizumab, reducing HCU and improving outcomes.