Scholarlink

<p>Axatilimab (axatilimab-csfr; Niktimvo<sup>®</sup>), an anti-colony stimulating factor 1 receptor (CSF1R) monoclonal antibody, is a useful addition to treatment options in the USA for chronic graft-versus-host disease (cGVHD) in patients weighing ≥&#xa0;40 kg who have failed ≥ two prior lines of systemic therapy. It acts through a novel mechanism, inhibiting macrophage activation and differentiation via CSF1R blockade. In the AGAVE-201 clinical trial, axatilimab 0.3 mg/kg once every 2 weeks improves patient condition and reduces patient-reported symptom severity. While no patients achieved overall complete responses, responses were observed in all affected organs, including organs with difficult-to-treat fibrotic manifestations. The tolerability profile of axatilimab at the approved dosage was generally manageable. Infections were the most common adverse events, and laboratory abnormalities caused by CSF1R blockade were transient and not associated with end-organ damage. Current evidence is limited by the small patient population and open-label phase 2 trial design; results from ongoing clinical trials of axatilimab in cGVHD are awaited with interest.</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Axatilimab (Niktimvo®) in chronic graft-versus-host disease: a profile of its use

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年4月10日

查看全文
  • Michael B. Brown,
  • Hannah A. Blair

摘要

Axatilimab (axatilimab-csfr; Niktimvo®), an anti-colony stimulating factor 1 receptor (CSF1R) monoclonal antibody, is a useful addition to treatment options in the USA for chronic graft-versus-host disease (cGVHD) in patients weighing ≥ 40 kg who have failed ≥ two prior lines of systemic therapy. It acts through a novel mechanism, inhibiting macrophage activation and differentiation via CSF1R blockade. In the AGAVE-201 clinical trial, axatilimab 0.3 mg/kg once every 2 weeks improves patient condition and reduces patient-reported symptom severity. While no patients achieved overall complete responses, responses were observed in all affected organs, including organs with difficult-to-treat fibrotic manifestations. The tolerability profile of axatilimab at the approved dosage was generally manageable. Infections were the most common adverse events, and laboratory abnormalities caused by CSF1R blockade were transient and not associated with end-organ damage. Current evidence is limited by the small patient population and open-label phase 2 trial design; results from ongoing clinical trials of axatilimab in cGVHD are awaited with interest.