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<p>Young children with Hemophilia A (CwHA) are at increased risk of arthropathy and inhibitor formation, thereby warranting prophylaxis at an early age. We studied efficacy and safety of emicizumab prophylaxis in CwHA (severe) without inhibitor. Retrospective analysis was performed in young CwHA (&lt;3 year of age at recruitment) who were started on emicizumab between Sep 2021 to Dec 2023. They were either treatment naïve or minimally treated (≤ 5 exposure days). Standard dose of emicizumab was used. Annualized bleeding rate (ABR) was calculated. Emicizumab trough levels were performed. Thirteen children with median age 16 month (6-35) at start of emicizumab were followed-up for median 24 month (14-46). The cumulative follow-up was 342 month. All children were symptomatic at recruitment with 4/13 CwHA having joint bleeds. Median all bleed ABR decreased from 1.7 to zero post-emicizumab. A total of four adverse events (all grade-1) in three children were noted. Mean emicizumab levels were 23.6±10.5 µg/ml (95% CI: 17.24-30.01). Emicizumab levels of 10 children (77%) were below therapeutic range. Hemophilia joint health score for all children&gt;4 yr (n=7) was zero. We found emicizumab prophylaxis safe and efficacious in young CwHA without inhibitors. Emicizumab levels lower than therapeutic-range was not associated with significant bleeds in our cohort.</p>

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Real World Data of Emicizumab Prophylaxis in Young Children with Severe Hemophilia A Without Inhibitors

  • 期刊论文

  • 发表日期：

    2026年4月29日

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  • Sanjeev Khera,
  • Renjith Mathew Verghese,
  • M. K. Safal Muhammed,
  • Preeti Tripathi,
  • Mridula Nair,
  • Rajiv Kumar,
  • Rajan Kapoor

摘要

Young children with Hemophilia A (CwHA) are at increased risk of arthropathy and inhibitor formation, thereby warranting prophylaxis at an early age. We studied efficacy and safety of emicizumab prophylaxis in CwHA (severe) without inhibitor. Retrospective analysis was performed in young CwHA (<3 year of age at recruitment) who were started on emicizumab between Sep 2021 to Dec 2023. They were either treatment naïve or minimally treated (≤ 5 exposure days). Standard dose of emicizumab was used. Annualized bleeding rate (ABR) was calculated. Emicizumab trough levels were performed. Thirteen children with median age 16 month (6-35) at start of emicizumab were followed-up for median 24 month (14-46). The cumulative follow-up was 342 month. All children were symptomatic at recruitment with 4/13 CwHA having joint bleeds. Median all bleed ABR decreased from 1.7 to zero post-emicizumab. A total of four adverse events (all grade-1) in three children were noted. Mean emicizumab levels were 23.6±10.5 µg/ml (95% CI: 17.24-30.01). Emicizumab levels of 10 children (77%) were below therapeutic range. Hemophilia joint health score for all children>4 yr (n=7) was zero. We found emicizumab prophylaxis safe and efficacious in young CwHA without inhibitors. Emicizumab levels lower than therapeutic-range was not associated with significant bleeds in our cohort.