Healthcare Resource U tilization and Healthcare Costs After Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy in the United States: The ENDURE Study
Author(s)
Michelle Schoettler, MD1, Andrew Messali, PharmD, PhD2, Danielle Hyman, PhD3, Kirti Batra, MBA4, Qiana Amos, PhD, MPH4, Christine Higham, MD5.
1Emory University, Atlanta, GA, USA, 2Director, Global Health Economics and Outcomes Research, Alexion Pharmaceuticals, Boston, MA, USA, 3Alexion Pharmaceuticals, Boston, MA, USA, 4Optum, Eden Prairie, MN, USA, 5UCSF Benioff Children’s Hospitals, San Francisco, CA, USA.
1Emory University, Atlanta, GA, USA, 2Director, Global Health Economics and Outcomes Research, Alexion Pharmaceuticals, Boston, MA, USA, 3Alexion Pharmaceuticals, Boston, MA, USA, 4Optum, Eden Prairie, MN, USA, 5UCSF Benioff Children’s Hospitals, San Francisco, CA, USA.
OBJECTIVES: Thrombotic microangiopathy is a potential devastating complication occurring in ~10-20% of hematopoietic stem cell transplants (HSCT-TMA); survival is poor and economic outcomes associated with this complication are poorly described. The objective of this analysis was to describe healthcare resource utilization (HCRU) and costs among HSCT recipients with and without HSCT-TMA.
METHODS: The ENDURE study is a retrospective analysis of Komodo’s Healthcare Map, a large, multi-payer, US-based claims database. Adult and pediatric HSCT recipients were identified using procedure codes (CPT, HCPCS, and ICD-10). HSCT recipients without a TMA code (controls) within 12 months following HSCT were matched 10:1 to patients with HSCT-TMA (cases). The index date was either the date of TMA (cases) or a date chosen based on equivalent time post-HSCT (controls). The follow-up period began with the index date and ended at death or disenrollment. All patients were required to have at least 12 months of pre-index and 1 month of post-index continuous enrollment to avoid survivor bias. All data are reported separately for adults and pediatrics.
RESULTS: Between January 2016 and March 2024, 1,732 adult and 325 pediatric patients with HSCT-TMA met inclusion criteria, which were matched to 18,691 adult and 2,335 pediatric HSCT recipients. Median time from HSCT to TMA was 38 days for adults and 44 days for pediatrics. Rates of all-cause hospitalizations among pediatric and adult patients with HSCT-TMA were 44% and 173% higher than controls, respectively (p<0.05). The length of these hospitalizations was over 4 days among patients with HSCT-TMA, compared to 2 days among controls (p<0.001). Rates of all-cause ER visits among pediatric and adult patients with HSCT-TMA were 91% and 109% higher than controls, respectively (p<0.001). HSCT-TMA was associated with higher costs in every setting.
CONCLUSIONS: HSCT-TMA is associated with significantly higher HCRU. Effective treatment has the potential to save lives and healthcare resources.
METHODS: The ENDURE study is a retrospective analysis of Komodo’s Healthcare Map, a large, multi-payer, US-based claims database. Adult and pediatric HSCT recipients were identified using procedure codes (CPT, HCPCS, and ICD-10). HSCT recipients without a TMA code (controls) within 12 months following HSCT were matched 10:1 to patients with HSCT-TMA (cases). The index date was either the date of TMA (cases) or a date chosen based on equivalent time post-HSCT (controls). The follow-up period began with the index date and ended at death or disenrollment. All patients were required to have at least 12 months of pre-index and 1 month of post-index continuous enrollment to avoid survivor bias. All data are reported separately for adults and pediatrics.
RESULTS: Between January 2016 and March 2024, 1,732 adult and 325 pediatric patients with HSCT-TMA met inclusion criteria, which were matched to 18,691 adult and 2,335 pediatric HSCT recipients. Median time from HSCT to TMA was 38 days for adults and 44 days for pediatrics. Rates of all-cause hospitalizations among pediatric and adult patients with HSCT-TMA were 44% and 173% higher than controls, respectively (p<0.05). The length of these hospitalizations was over 4 days among patients with HSCT-TMA, compared to 2 days among controls (p<0.001). Rates of all-cause ER visits among pediatric and adult patients with HSCT-TMA were 91% and 109% higher than controls, respectively (p<0.001). HSCT-TMA was associated with higher costs in every setting.
CONCLUSIONS: HSCT-TMA is associated with significantly higher HCRU. Effective treatment has the potential to save lives and healthcare resources.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE113
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Rare & Orphan Diseases