Personalized, pharmacokinetic-guided prophylaxis using simoctocog alfa may lead to higher rates of zero bleeds compared with extended half-life recombinant factor VIII (FVIII) concentrates among patients with hemophilia A, according to a study published in the European Journal of Haematology. 

Although FVIII prophylaxis has reduced the disease burden associated with hemophilia, recurrent bleeding episodes still occur among some users. Simoctocog alfa is a fourth-generation, recombinant B-domain deleted FVIII concentrate derived from a human cell line. Studies have indicated that a significant proportion of patients with hemophilia achieve zero spontaneous bleeds while on the medication. 

Because matching-adjusted indirect comparison is recognized as a sturdy method for comparing outcomes from different studies, even in the absence of a common comparator, the authors of the study sought to compare the efficacy of simoctocog alfa with 3 extended half-life recombinant FVIII concentrates—efmoroctocog alfa, damoctocog alfa pegol, and rurioctocog alfa pegol. 

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The research team identified 3 pivotal studies with to perform their analysis: the A-LONG study that assesses the merits of efmoroctocog alfa, the PROTECT VIII study that investigates damoctocog alfa pegol, and the PROPEL study that uses rurioctocog alfa pegol. The researchers obtained aggregate patient data from these 3 comparator studies and summarized them. For assessing simocotog alfa, the NuPreviq study was selected. 

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The researchers reported that the percentage of individuals with hemophilia with zero bleeds was higher when prescribed simoctocog alfa compared with efmoroctocog alfa (75.3% vs 45.3%) and damoctocog alfa pegol (76.8% vs 38.2%). As for rurioctocog alfa pegol, simoctocog alfa outperformed it in the 1% to 3% arm (78.4% vs 42.1%), but there was no statistically significant difference in the 8% to 12% arm. 

“Results of these indirect treatment comparisons suggest that pharmacokinetic-guided personalized treatment with simoctocog alfa in patients with severe hemophilia A can lead to decreased annualized bleeding rates and a higher number of patients experiencing zero bleeds in comparison to other extended half-life FVIII concentrates,” the authors concluded. 


Kessler CM, Corrales-Medina FF, Mannucci PM, Jiménez-Yuste V, Tarantino MD. Clinical efficacy of simoctocog alfa versus extended half-life recombinant FVIII concentrates in hemophilia A patients undergoing personalized prophylaxis using a matching-adjusted indirect comparison methodEur J Haematol. Published online August 16, 2023. doi:10.1111/ejh.14073