CLINICAL AND ECONOMIC BURDEN IN PEDIATRIC PATIENTS WITH SICKLE CELL DISEASE WITH RECURRENT VASO-OCCLUSIVE CRISES IN THE UNITED STATES
Author(s)
JIAHE li, MPH1, Arie Barlev, MSc, PharmD1, Siddharth Jain, MS, PharmD1, Kristin Evans, PhD2, Michelle Jerry, MS2, Duncan Brown, BSc, PhD1;
1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2Merative, Ann Arbor, MI, USA
1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2Merative, Ann Arbor, MI, USA
OBJECTIVES: Sickle cell disease (SCD) is a genetic blood disorder that causes red blood cells to become sickle-shaped, leading to vaso-occlusive crises (VOCs), hemolysis, and end-organ damage. Burden of SCD in pediatric patients with recurrent VOCs has not been well detailed. This study investigated clinical and economic outcomes for pediatric patients aged 5-11 years with SCD with recurrent VOCs in the United States (US).
METHODS: This retrospective analysis used US MarketScan Commercial, Medicare, and Multi-State Medicaid Databases to identify patients with ≥1 inpatient or ≥2 outpatient claims for SCD between January 1, 2014-December 31, 2024. SCD with recurrent VOCs was defined as ≥2 diagnosed VOCs per year in any two consecutive years after and including the first qualifying SCD claim. The date of the second VOC in the second year was the index date. Patients were required to have >24-months continuous enrollment pre-index and >12-months post-index, and be between 5-11 years at the index date, with no evidence of hematopoietic stem cell transplant or sickle cell trait. Baseline demographics, chronic complications, health care resource utilization and total costs (2024 USD) during follow up were summarized.
RESULTS: 665 pediatric patients with SCD with recurrent VOCs were identified. Mean patient age was 8.1 (SD: 2.0) years and 47.1% were female; 10.2% of patients were in the commercial database and 89.8% in the Medicaid database. Mean follow-up time was 4.3 (SD: 2.3) years. The most common chronic complications were chronic lung disease (62.0%) and cardiovascular complications (33.2%). Patients had a mean of 1.5 (SD: 1.6) inpatient admissions and 2.5 (SD: 2.4) emergency room visits per patient per year (PPPY). Mean total healthcare costs were $36,904 (SD: $63,791) PPPY.
CONCLUSIONS: From a young age, SCD with recurrent VOCs imposes significant clinical and economic burden, highlighting the need for therapies that reduce healthcare burden and improve pediatric patient outcomes.
METHODS: This retrospective analysis used US MarketScan Commercial, Medicare, and Multi-State Medicaid Databases to identify patients with ≥1 inpatient or ≥2 outpatient claims for SCD between January 1, 2014-December 31, 2024. SCD with recurrent VOCs was defined as ≥2 diagnosed VOCs per year in any two consecutive years after and including the first qualifying SCD claim. The date of the second VOC in the second year was the index date. Patients were required to have >24-months continuous enrollment pre-index and >12-months post-index, and be between 5-11 years at the index date, with no evidence of hematopoietic stem cell transplant or sickle cell trait. Baseline demographics, chronic complications, health care resource utilization and total costs (2024 USD) during follow up were summarized.
RESULTS: 665 pediatric patients with SCD with recurrent VOCs were identified. Mean patient age was 8.1 (SD: 2.0) years and 47.1% were female; 10.2% of patients were in the commercial database and 89.8% in the Medicaid database. Mean follow-up time was 4.3 (SD: 2.3) years. The most common chronic complications were chronic lung disease (62.0%) and cardiovascular complications (33.2%). Patients had a mean of 1.5 (SD: 1.6) inpatient admissions and 2.5 (SD: 2.4) emergency room visits per patient per year (PPPY). Mean total healthcare costs were $36,904 (SD: $63,791) PPPY.
CONCLUSIONS: From a young age, SCD with recurrent VOCs imposes significant clinical and economic burden, highlighting the need for therapies that reduce healthcare burden and improve pediatric patient outcomes.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE401
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)