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Background <p>Hereditary angioedema (HAE) causes recurring swelling attacks, leading to substantial disease burden. This real-world, retrospective study aimed to evaluate HAE attack rates before and after berotralstat initiation stratified by patients’ baseline attack frequency.</p> Methods <p>Specialty Pharmacy data from Optime Care, Inc. (12/2020–01/2024), the sole berotralstat dispenser in the United States, were analyzed. Eligible patients had&#xa0; ≥ 2 berotralstat dispensings (first&#xa0;=&#xa0; index) and ≥ 1 self-assessment of attacks at baseline (90-days pre-index) and follow-up (first-to-last dispensing). Patients were classified by HAE type (based on laboratory measurements) and baseline attacks (≥ 5, 2–4, 1, and 0 attacks/month). Follow-up attack rates were compared with baseline using mean differences, confidence intervals, and<i> P</i>-values. Among those with 0 baseline attacks/month, proportions with 0 follow-up attacks/month were assessed.</p> Results <p>Of 390 eligible patients with HAE with C1 esterase inhibitor (C1INH) deficiency (HAE-C1INH) and 311 with HAE with normal C1INH (HAE-nC1INH), most were female (64.1% and 77.5%) with mean ages of 39.3 and 48.1 years, respectively. Mean attack rates decreased from 2.50 to 0.79 attacks/month (HAE-C1INH) and from 4.59 to 1.68 attacks/month (HAE-nC1INH) at 12-months of berotralstat treatment (both <i>P</i>&#xa0;&lt;&#xa0;0 .001), with sustained reductions at 18-months. Patients with ≥ 1 baseline attack/month experienced significantly lower attack rates after berotralstat initiation. Among patients with 0 baseline attacks/month, most also maintained 0 attacks/month in each follow-up interval (HAE-C1INH: 70–85%; HAE-nC1INH: 61–81%).</p> Conclusion <p>Berotralstat was associated with significant and sustained reductions in attack rates among patients with HAE, regardless of baseline attack rate. Patients with 0 baseline attacks/month maintained low attack rates following berotralstat initiation.</p>

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Attack rate reductions following berotralstat initiation among US patients with hereditary angioedema in the real-world

  • 期刊论文

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  • 发表日期：

    2026年1月25日

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  • Mark Davis-Lorton,
  • Raffi Tachdjian,
  • Lorena Lopez-Gonzalez,
  • Sean D. MacKnight,
  • Ramya Ramasubramanian,
  • François Laliberté,
  • Patrick Gillard,
  • Meri LiVecchi,
  • Sandra Nestler-Parr,
  • William R. Lumry

摘要

Background

Hereditary angioedema (HAE) causes recurring swelling attacks, leading to substantial disease burden. This real-world, retrospective study aimed to evaluate HAE attack rates before and after berotralstat initiation stratified by patients’ baseline attack frequency.

Methods

Specialty Pharmacy data from Optime Care, Inc. (12/2020–01/2024), the sole berotralstat dispenser in the United States, were analyzed. Eligible patients had  ≥ 2 berotralstat dispensings (first =  index) and ≥ 1 self-assessment of attacks at baseline (90-days pre-index) and follow-up (first-to-last dispensing). Patients were classified by HAE type (based on laboratory measurements) and baseline attacks (≥ 5, 2–4, 1, and 0 attacks/month). Follow-up attack rates were compared with baseline using mean differences, confidence intervals, and P-values. Among those with 0 baseline attacks/month, proportions with 0 follow-up attacks/month were assessed.

Results

Of 390 eligible patients with HAE with C1 esterase inhibitor (C1INH) deficiency (HAE-C1INH) and 311 with HAE with normal C1INH (HAE-nC1INH), most were female (64.1% and 77.5%) with mean ages of 39.3 and 48.1 years, respectively. Mean attack rates decreased from 2.50 to 0.79 attacks/month (HAE-C1INH) and from 4.59 to 1.68 attacks/month (HAE-nC1INH) at 12-months of berotralstat treatment (both P < 0 .001), with sustained reductions at 18-months. Patients with ≥ 1 baseline attack/month experienced significantly lower attack rates after berotralstat initiation. Among patients with 0 baseline attacks/month, most also maintained 0 attacks/month in each follow-up interval (HAE-C1INH: 70–85%; HAE-nC1INH: 61–81%).

Conclusion

Berotralstat was associated with significant and sustained reductions in attack rates among patients with HAE, regardless of baseline attack rate. Patients with 0 baseline attacks/month maintained low attack rates following berotralstat initiation.