ASH 2023 Highlight 3

We take pleasure in presenting the third daily highlight from the ASH 2023 event, featuring Evelien Krumb, a PhD candidate and medical trainee affiliated with Cliniques Universitaires Saint-Luc in Brussels. She delivered an exhaustive synopsis of three presentations concentrating on the management of bleeding disorders across the entirety of an individual’s lifespan.

The initial presentation, conducted by Dr Steven Pipe, delved into the primary analysis of the Haven 7 study, scrutinising the utilisation of emicizumab in infants aged ≤12 months and diagnosed with severe haemophilia A (HA) and devoid of FVIII inhibitors. Challenges associated with the administration of FVIII in paediatric patients with HA have impeded the global consensus advocating early bleeding prophylaxis. The availability of emicizumab presents a potential alleviation of the treatment-related burdens. The Haven 7 study enlisted 55 children, with over half categorised as minimally treated patients. Emicizumab demonstrated remarkably low annualised bleed rates (ABRs), with traumatic bleeding occurrences noted in those who experienced bleeding episodes and an absence of spontaneous treated bleeds. Treatment-related adverse events, confined to injection-site reactions, were reported by nine participants. Thirty serious adverse events were documented, none of which were attributed to the treatment. The deduction drawn from these findings is that emicizumab is both safe and efficacious for application in children aged ≤12 months, with a slated seven-year follow-up expected to yield valuable long-term data.

Subsequently, Dr Flora Peivandi presented pharmacokinetic (PK) data emanating from the XTEND-Kids study, evaluating efanesoctocog alfa in children below 12 years afflicted with severe HA. Efanesoctocog alfa represents a novel category of FVIII replacement therapy meticulously engineered to surmount the FVIII half-life ceiling imposed by von Willebrand factor. The study enrolled a total of 74 participants, with 37 included in the pharmacokinetic (PK) subgroup (n=19, <6 years; n=18, ≥6 –<12 years). In both age cohorts, mean FVIII activity levels were consistently maintained throughout the study duration, and all children achieved levels considered normal or near normal for FVIII. Notably, no significant difference was observed in the mean half-life and clearance between the two age groups. Additionally, the mean half-life and clearance were comparable between children with blood group O and those with blood group non-O.

Lastly, Dr. Lynn Malec expounded on the outcomes of exit interviews conducted with caregivers of previously treated patients with HA, drawn from the XTEND-Kids study. The objective of these interviews was to gain insights into caregivers’ perspectives regarding the impact of HA and its treatment, as well as their experiences with efanesoctocog alfa prophylaxis. All children, with the exception of one, were undergoing regular prophylaxis before the study, receiving treatment with a conventional FVIII replacement product. Almost half of the caregivers reported noticeable impacts on their children’s day-to-day life and mood attributable to HA before the commencement of the study. Efanesoctocog alfa prophylaxis demonstrated notable enhancements in both the daily lives and functionality of the children and their caregivers. Significantly, all caregivers expressed a preference for efanesoctocog alfa over their child’s previous treatment. The unanimous preference for efanesoctocog alfa among caregivers is particularly intriguing. These findings underscore the potential role of efanesoctocog alfa prophylaxis in children with HA, affirming its positive impact on both patients’ and caregivers’ experiences.

References:

Pipe S. ASH 2023. #505

Peyvandi F. ASH 2023. #506

Malec L. ASH 2023. #507

With the educational support of: