CLINICAL AND ECONOMIC BURDEN OF HEMOLYTIC ANEMIA IN US PATIENTS WITH SICKLE CELL DISEASE: A REAL-WORLD ANALYSIS
Author(s)
Jungyoon Moon, MS1, Vanessa Shih, MS, PharmD1, Susanna Libby, BSc2, Emma Chatterton, MRes2, Rolandas Urbstonaitis, Pharm D1, Amber Yates, MD1.
1Agios Pharmaceuticals, Inc., Cambridge, MA, USA, 2Adelphi Real World, Bollington, United Kingdom.
1Agios Pharmaceuticals, Inc., Cambridge, MA, USA, 2Adelphi Real World, Bollington, United Kingdom.
OBJECTIVES: To characterize the relationship between anemia levels and work-related outcomes, vaso-occlusive crises (VOCs), and healthcare resource utilization (HCRU) in US patients with sickle cell disease (SCD).
METHODS: The Adelphi SCD Disease Specific Programme™ is a real-world, cross-sectional survey of patients and physicians (conducted: August 2024-April 2025). This analysis included physician-reported data for US patients aged ≥17 years whose anemia level (defined by hemoglobin [Hb] <8.0 g/dL, 8.0-9.9 g/dL, and 10.0-11.9 g/dL) was within the same Hb group at both survey initiation and 12 months prior. Descriptive analyses assessed differences in work-related outcomes, VOCs, and HCRU by anemia level.
RESULTS: Data included 168 patients from 45 physicians. Mean (standard deviation [SD]) age was 30.9 (11.7) years; 60% were male. Anemia level was Hb <8.0g/dL in 10% of patients, 8.0-9.9g/dL in 49%, and 10.0-11.9g/dL in 41%. Overall, 44% of patients were reported to be in full-time employment; 15%, 51%, and 42% in patients with Hb <8.0 g/dL, 8.0-9.9 g/dL, and 10.0-11.9 g/dL, respectively. Unemployment rates were reported as 31%, 9%, and 12%, respectively. Of patients in full/part-time work, or students (n=65), mean (SD) number of sick days/month was 2.6 (1.3), 2.0 (1.6), and 1.3 (1.5) across Hb levels, respectively. In the prior 12 months, ≥1 SCD-related hospitalization was reported in 100%, 50%, and 35% of patients with Hb <8.0 g/dL, 8.0-9.9 g/dL, and 10.0-11.9 g/dL. Mean (SD) number of VOCs was 1.6 (1.5) overall, and 3.3 (1.4), 1.6 (1.1), and 1.1 (1.6) across Hb levels, respectively. Mean (SD) number of VOC hospitalizations was 2.9 (1.4), 1.6 (0.8), and 1.3 (0.6), respectively.
CONCLUSIONS: Patients with SCD in the US experience high disease burden, including adverse work-related outcomes, hospitalizations, and frequent VOCs. This burden is observed across all anemia levels but is greatest in patients with Hb <8.0g/dL.
METHODS: The Adelphi SCD Disease Specific Programme™ is a real-world, cross-sectional survey of patients and physicians (conducted: August 2024-April 2025). This analysis included physician-reported data for US patients aged ≥17 years whose anemia level (defined by hemoglobin [Hb] <8.0 g/dL, 8.0-9.9 g/dL, and 10.0-11.9 g/dL) was within the same Hb group at both survey initiation and 12 months prior. Descriptive analyses assessed differences in work-related outcomes, VOCs, and HCRU by anemia level.
RESULTS: Data included 168 patients from 45 physicians. Mean (standard deviation [SD]) age was 30.9 (11.7) years; 60% were male. Anemia level was Hb <8.0g/dL in 10% of patients, 8.0-9.9g/dL in 49%, and 10.0-11.9g/dL in 41%. Overall, 44% of patients were reported to be in full-time employment; 15%, 51%, and 42% in patients with Hb <8.0 g/dL, 8.0-9.9 g/dL, and 10.0-11.9 g/dL, respectively. Unemployment rates were reported as 31%, 9%, and 12%, respectively. Of patients in full/part-time work, or students (n=65), mean (SD) number of sick days/month was 2.6 (1.3), 2.0 (1.6), and 1.3 (1.5) across Hb levels, respectively. In the prior 12 months, ≥1 SCD-related hospitalization was reported in 100%, 50%, and 35% of patients with Hb <8.0 g/dL, 8.0-9.9 g/dL, and 10.0-11.9 g/dL. Mean (SD) number of VOCs was 1.6 (1.5) overall, and 3.3 (1.4), 1.6 (1.1), and 1.1 (1.6) across Hb levels, respectively. Mean (SD) number of VOC hospitalizations was 2.9 (1.4), 1.6 (0.8), and 1.3 (0.6), respectively.
CONCLUSIONS: Patients with SCD in the US experience high disease burden, including adverse work-related outcomes, hospitalizations, and frequent VOCs. This burden is observed across all anemia levels but is greatest in patients with Hb <8.0g/dL.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO137
Topic
Clinical Outcomes, Patient-Centered Research
Topic Subcategory
Clinician Reported Outcomes
Disease
Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)