REAL-WORLD TREATMENT PATTERNS AMONG PEOPLE WITH MODERATE-TO-SEVERE HEMOPHILIA A AND B IN CHINA: A RETROSPECTIVE DATABASE STUDY
Author(s)
Asma HAMID, MD, MPH1, Enkhgerel Nasanbat, MPH1, Mingyue Gao, MPH2, Martina Furegato, MSc1, Amy AI, MPhil3, Xue Bai, MD3, Jiyoon Choi, PharmD, MBA4, Amy Chen, PhD5, Marion Afonso, MSc6.
1Oracle, Paris, France, 2Cerner Enviza, an Oracle company, Shanghai, China, 3Sanofi, Shanghai, China, 4Sanofi, Morristown, NJ, USA, 5Sanofi, Cambridge, MA, USA, 6Sanofi, Gentilly, France.
1Oracle, Paris, France, 2Cerner Enviza, an Oracle company, Shanghai, China, 3Sanofi, Shanghai, China, 4Sanofi, Morristown, NJ, USA, 5Sanofi, Cambridge, MA, USA, 6Sanofi, Gentilly, France.
OBJECTIVES: To assess treatment patterns among people with hemophilia (PwH) in China.
METHODS: Men (aged ≥12 years) with moderate-to-severe hemophilia A/B (HA/HB) in China were identified retrospectively between January 2021 and March 2023, using the National Population Health Data Center (NPHDC) Chronic Disease Prevention and Control database. Index was defined as date of first hemophilia prescription during identification period and follow-up period was from index until death or study end (March 2024). During follow-up, treatment regimens and persistence were reported yearly. Data were summarized using descriptive statistics. Kaplan-Meier analysis was used to estimate prophylactic treatment persistence.
RESULTS: At index, 602 people with HA (94.0%) and HB (6.0%) were included; mean (SD) age was 34.3 (15.9) years, 82.1% were adults, and 79.6% had severe hemophilia.
During the overall follow-up period, 77.7% and 36.7% PwH had ≥1 bleeding and joint bleeding episode, respectively; 87.4% PwH experienced ≥1 treatment regimen switch. Most PwH received on-demand treatment regimen (62.6%), followed by prophylactic treatment regimen (29.1%). All PwH on prophylactic regimen were treated with standard half-life recombinant factor; mean (SD) monthly dose and monthly frequency were 7,991.5 (4,457.0) IU and 1.7 (1.2), respectively. Approximately, 86.3% and 13.7% PwH on prophylactic regimen had ≥1 treatment discontinuation and treatment switch, respectively. The proportion of PwH receiving on-demand treatment regimen declined from 57.1% to 42.8%, while prophylaxis treatment regimen increased from 19.1% to 25.4%, from first to third year follow-up, respectively. Among 231 PwH initiating prophylaxis treatment regimen at least once during follow-up, persistence declined from 78.2% (95% CI: 72.1-83.2) to 15.0% (95% CI: 8.8-22.8).
CONCLUSIONS: In conclusion, majority of PwH in China experience bleeding episodes and receive on-demand treatments. The low overall rate of regular prophylaxis and frequent treatment discontinuations suggest ongoing challenges in hemophilia care and underscore the unmet need for optimized treatment strategies in China.
METHODS: Men (aged ≥12 years) with moderate-to-severe hemophilia A/B (HA/HB) in China were identified retrospectively between January 2021 and March 2023, using the National Population Health Data Center (NPHDC) Chronic Disease Prevention and Control database. Index was defined as date of first hemophilia prescription during identification period and follow-up period was from index until death or study end (March 2024). During follow-up, treatment regimens and persistence were reported yearly. Data were summarized using descriptive statistics. Kaplan-Meier analysis was used to estimate prophylactic treatment persistence.
RESULTS: At index, 602 people with HA (94.0%) and HB (6.0%) were included; mean (SD) age was 34.3 (15.9) years, 82.1% were adults, and 79.6% had severe hemophilia.
During the overall follow-up period, 77.7% and 36.7% PwH had ≥1 bleeding and joint bleeding episode, respectively; 87.4% PwH experienced ≥1 treatment regimen switch. Most PwH received on-demand treatment regimen (62.6%), followed by prophylactic treatment regimen (29.1%). All PwH on prophylactic regimen were treated with standard half-life recombinant factor; mean (SD) monthly dose and monthly frequency were 7,991.5 (4,457.0) IU and 1.7 (1.2), respectively. Approximately, 86.3% and 13.7% PwH on prophylactic regimen had ≥1 treatment discontinuation and treatment switch, respectively. The proportion of PwH receiving on-demand treatment regimen declined from 57.1% to 42.8%, while prophylaxis treatment regimen increased from 19.1% to 25.4%, from first to third year follow-up, respectively. Among 231 PwH initiating prophylaxis treatment regimen at least once during follow-up, persistence declined from 78.2% (95% CI: 72.1-83.2) to 15.0% (95% CI: 8.8-22.8).
CONCLUSIONS: In conclusion, majority of PwH in China experience bleeding episodes and receive on-demand treatments. The low overall rate of regular prophylaxis and frequent treatment discontinuations suggest ongoing challenges in hemophilia care and underscore the unmet need for optimized treatment strategies in China.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
RWD130
Topic
Real World Data & Information Systems
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)