CLINICAL, HUMANISTIC, AND HEALTHCARE RESOURCE UTILIZATION BURDEN OF SICKLE CELL DISEASE IN INDIA: INSIGHTS FROM THE ASCRIBE STUDY
Author(s)
Manisha R. Madkaikar, MD1, Tuphan Kanti Dolai, Doctor of Medicine2, Dheeraj Kataria, RPh3, Akshay Khandeparkar, Doctor of Medicine4, MUZAMMIL KHAN ALIYAR KHAN PATHAN, Doctor of Medicine4, Beth Poirrette, Master of Science5, Tulika Seth, Doctor of Medicine6.
1ICMR -National Institute for Research on Blood and Immune Disorders, Mumbai, India, 2Department of Hematology, NRS Medical College & Hospital, Kolkata, India, 3Novo Nordisk India Private Ltd, Bangalore, India, 4Novo Nordisk India Private Ltd, Bengaluru, India, 5Adelphi Real World, Bollington, United Kingdom, 6All India Institute of Medical Sciences, New Delhi, India.
1ICMR -National Institute for Research on Blood and Immune Disorders, Mumbai, India, 2Department of Hematology, NRS Medical College & Hospital, Kolkata, India, 3Novo Nordisk India Private Ltd, Bangalore, India, 4Novo Nordisk India Private Ltd, Bengaluru, India, 5Adelphi Real World, Bollington, United Kingdom, 6All India Institute of Medical Sciences, New Delhi, India.
OBJECTIVES: The Analysis of Sickle Cell disease Real-world Impact and BurdEn (ASCRIBE) study characterized clinical, humanistic, and healthcare resource utilization burden (HCRU) of sickle cell disease (SCD) in India.
METHODS: Data from the Adelphi SCD Disease Specific Programme™ were collected in India (November 2024-January 2025). In this cross-sectional survey with retrospective data collection, physicians managing ≥5 people with SCD (PwSCD)/month completed patient record forms on demographics, clinical characteristics, HCRU and treatment for 1-15 consecutive patients (aged ≥2-years, confirmed SCD). PwSCD (aged ≥18-years) and caregivers voluntarily completed forms including PROMIS-Pain Intensity, FACIT-Fatigue, and Burden Scale for Family Caregivers (BSFC). Descriptive statistics were used (mean±standard deviation for continuous variables; frequencies and percentages for categorical variables).
RESULTS: Overall, 32 physicians provided data for 265 PwSCD (mean age 21.4±12.1 years; 56% male; 75% HbSS/HbSβ0 genotype). Most PwSCD (n=260) were receiving treatment; primarily hydroxyurea (97%) and pain relief (85%). In the past year, 81% experienced ≥1 vaso-occlusive crisis (VOC) (mean VOCs 2.6±1.8). At survey, common symptoms included acute pain (72%), chronic pain (63%) and organ complications (38%). In the past year, 80% (n=211/264) were hospitalized (mean length of stay 7.1±5.5 nights; n=192). Among those with hospitalization (n=211), 91% were VOC-driven, 89% via emergency departments (available data n=210), and 17% required intensive care (available data n=210). Overall, 48% (n=126/262) received ≥1 blood transfusion (mean 3.3±3.0) in the past year. PwSCD (n=81) reported mean FACIT-Fatigue score of 26.6±8.5 (clinical threshold <34.0); mean PROMIS-Pain score was 59.5±8.3 (US population norms 50.0). Caregivers reported providing a mean of 55.3±37.8 hours of weekly care (n=73/77), and BSFC score of 15.1±3.7 (n=74/77; severe burden).
CONCLUSIONS: PwSCD in India face substantial clinical and humanistic burden with high VOC-related hospitalizations despite widespread hydroxyurea use. These findings may support local health technology assessment and underscore the need for optimized SCD management.
METHODS: Data from the Adelphi SCD Disease Specific Programme™ were collected in India (November 2024-January 2025). In this cross-sectional survey with retrospective data collection, physicians managing ≥5 people with SCD (PwSCD)/month completed patient record forms on demographics, clinical characteristics, HCRU and treatment for 1-15 consecutive patients (aged ≥2-years, confirmed SCD). PwSCD (aged ≥18-years) and caregivers voluntarily completed forms including PROMIS-Pain Intensity, FACIT-Fatigue, and Burden Scale for Family Caregivers (BSFC). Descriptive statistics were used (mean±standard deviation for continuous variables; frequencies and percentages for categorical variables).
RESULTS: Overall, 32 physicians provided data for 265 PwSCD (mean age 21.4±12.1 years; 56% male; 75% HbSS/HbSβ0 genotype). Most PwSCD (n=260) were receiving treatment; primarily hydroxyurea (97%) and pain relief (85%). In the past year, 81% experienced ≥1 vaso-occlusive crisis (VOC) (mean VOCs 2.6±1.8). At survey, common symptoms included acute pain (72%), chronic pain (63%) and organ complications (38%). In the past year, 80% (n=211/264) were hospitalized (mean length of stay 7.1±5.5 nights; n=192). Among those with hospitalization (n=211), 91% were VOC-driven, 89% via emergency departments (available data n=210), and 17% required intensive care (available data n=210). Overall, 48% (n=126/262) received ≥1 blood transfusion (mean 3.3±3.0) in the past year. PwSCD (n=81) reported mean FACIT-Fatigue score of 26.6±8.5 (clinical threshold <34.0); mean PROMIS-Pain score was 59.5±8.3 (US population norms 50.0). Caregivers reported providing a mean of 55.3±37.8 hours of weekly care (n=73/77), and BSFC score of 15.1±3.7 (n=74/77; severe burden).
CONCLUSIONS: PwSCD in India face substantial clinical and humanistic burden with high VOC-related hospitalizations despite widespread hydroxyurea use. These findings may support local health technology assessment and underscore the need for optimized SCD management.
Conference/Value in Health Info
2026-09, ISPOR Asia Pacific 2026, Bangkok, Thailand
Value in Health, Volume 55, Issue S1
Code
PCR33
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Rare & Orphan Diseases