MATCHING-ADJUSTED INDIRECT COMPARISON OF ETRANACOGENE DEZAPARVOVEC VERSUS MARSTACIMAB FOR THE TREATMENT OF SEVERE HEMOPHILIA B
Author(s)
Robert Klamroth, MD1, Michael Recht, MD2, Songkai Yan, MS3, Douglass Drelich, MD3, Emily Prentiss, MS4, Sarah Organ, MS5, Harry Odia, MS5, Hongseok Kim, MS3, Becky Hooper, MS5, Amrita Debnath, MS5, Radovan Tomic, MS6, Jan Astermark, MD7.
1Department for Internal Medicine, Haemophilia Treatment Centre, Vivantes Klinikum im Friedrichshain, Berlin, Germany, 2Yale School of Medicine, New Haven, CT, USA, 3CSL Behring, King of Prussia, PA, USA, 4EVERSANA, Chicago, IL, USA, 5EVERSANA, Victoria, BC, Canada, 6CSL Behring, Milan, Italy, 7Department of Translational Medicine, Lund University; Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö, Sweden.
1Department for Internal Medicine, Haemophilia Treatment Centre, Vivantes Klinikum im Friedrichshain, Berlin, Germany, 2Yale School of Medicine, New Haven, CT, USA, 3CSL Behring, King of Prussia, PA, USA, 4EVERSANA, Chicago, IL, USA, 5EVERSANA, Victoria, BC, Canada, 6CSL Behring, Milan, Italy, 7Department of Translational Medicine, Lund University; Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö, Sweden.
OBJECTIVES: To compare the efficacy of a single dose of etranacogene dezaparvovec (EDZ) versus routine prophylaxis with marstacimab in the treatment of adults with severe hemophilia B (HB), using matching-adjusted indirect comparisons (MAIC).
METHODS: Unanchored MAICs compared EDZ individual patient data (IPD) from HOPE-B trial and publicly available summary-level data (SLD) from BASIS trial for subjects with severe HB without inhibitors who received prior factor IX (FIX) prophylaxis and received routine prophylaxis with marstacimab. Due to lack of HB subgroup data in the primary publication of BASIS, publicly available SLD was obtained from the German Gemeinsamer Bundesausschuss (G-BA) dossier, French Haute Autorité de Santé (HAS) decision document, and conference materials. Patients from HOPE-B were matched to BASIS by excluding those who would not be eligible for or enrolled in BASIS. Then, HOPE-B patients were weighted to align with BASIS on age, BMI, and geographic region. The following outcomes were assessed: mean annualized bleeding rate (ABR), mean annualized spontaneous bleeding rate (AsBR), mean annualized joint bleeding rate (AjBR), proportion of patients without bleeds, and annualized FIX consumption. Bleeding outcomes were based on treated bleeds.
RESULTS: After adjustment for above factors, EDZ was associated with statistically significantly lower mean ABR using G-BA (rate ratio [RR]: 0.06 [95% confidence interval (CI): 0.01, 0.43]) and HAS data (RR: 0.06 [95% CI: 0.06, 0.42]), AsBR using G-BA data (RR: 0.06 [95% CI: 0.01, 0.56]), AjBR using G-BA data (RR: 0.06 [95% CI: 0.01, 0.54]), along with statistically significantly higher proportion of patients without bleeds using G-BA (odds ratio [OR]: 8.67 [95% CI: 1.36, 55.41]) and HAS data (OR: 10.84 [95% CI: 1.67, 70.26]), and statistically significantly lower mean FIX consumption using conference materials (difference: -285.75 IU/kg/year [95% CI: -442.99, -128.52]).
CONCLUSIONS: MAICs suggest favorable efficacy for EDZ versus prophylaxis with marstacimab based on available data.
METHODS: Unanchored MAICs compared EDZ individual patient data (IPD) from HOPE-B trial and publicly available summary-level data (SLD) from BASIS trial for subjects with severe HB without inhibitors who received prior factor IX (FIX) prophylaxis and received routine prophylaxis with marstacimab. Due to lack of HB subgroup data in the primary publication of BASIS, publicly available SLD was obtained from the German Gemeinsamer Bundesausschuss (G-BA) dossier, French Haute Autorité de Santé (HAS) decision document, and conference materials. Patients from HOPE-B were matched to BASIS by excluding those who would not be eligible for or enrolled in BASIS. Then, HOPE-B patients were weighted to align with BASIS on age, BMI, and geographic region. The following outcomes were assessed: mean annualized bleeding rate (ABR), mean annualized spontaneous bleeding rate (AsBR), mean annualized joint bleeding rate (AjBR), proportion of patients without bleeds, and annualized FIX consumption. Bleeding outcomes were based on treated bleeds.
RESULTS: After adjustment for above factors, EDZ was associated with statistically significantly lower mean ABR using G-BA (rate ratio [RR]: 0.06 [95% confidence interval (CI): 0.01, 0.43]) and HAS data (RR: 0.06 [95% CI: 0.06, 0.42]), AsBR using G-BA data (RR: 0.06 [95% CI: 0.01, 0.56]), AjBR using G-BA data (RR: 0.06 [95% CI: 0.01, 0.54]), along with statistically significantly higher proportion of patients without bleeds using G-BA (odds ratio [OR]: 8.67 [95% CI: 1.36, 55.41]) and HAS data (OR: 10.84 [95% CI: 1.67, 70.26]), and statistically significantly lower mean FIX consumption using conference materials (difference: -285.75 IU/kg/year [95% CI: -442.99, -128.52]).
CONCLUSIONS: MAICs suggest favorable efficacy for EDZ versus prophylaxis with marstacimab based on available data.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
P27
Topic
Clinical Outcomes
Topic Subcategory
Comparative Effectiveness or Efficacy
Disease
Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)