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AXATILIMAB-CSFR INJ,SOLN

Clinical Criteria Summary

Document 831: MON Axatilimab NIKTIMVO Monograph March 2026

Indication & Disease State

  • Treatment of chronic graft-versus-host disease (cGVHD)
  • Refractory or recurrent cGVHD with active signs/symptoms per NIH consensus criteria

Patient Eligibility & Demographics

  • Adults/pediatric patients weighing at least 40kg
  • Karnofsky score > 60
  • Adequate organ function

Prior Therapy Requirements

  • Failure of at least 2 prior lines of therapy
  • s/p > 2 lines of therapy (median 4 lines in trial population)
  • Prior therapies may include ibrutinib, ruxolitinib, or belumosudil

Disease Severity & Grading Criteria

  • Chronic Graft NIH Consensus Criteria for organ scoring (Score 0-3: least to most impactful versus Host Disease Grading classified by symptoms, affected organ systems and extent of disease involvement)
  • Mild: < 2 affected organs; no clinically significant functional dysfunction
  • Moderate: > 3 organs with no dysfunction or > 1 organ with dysfunction but no major disability
  • Severe: Major disability

Dosing & Administration Parameters

  • 0.3 mg/kg (max 35 mg) IV over 30 min every 3 weeks
  • IV formulation required

Document 838: Axatilimab NIKTIMVO Criteria March 2026

Exclusion Criteria

  • Weight less than 40 kg
  • Known pregnancy
  • Lactating

Inclusion Criteria

  • Care provided by a VA or VA Community Care hematology provider
  • Eastern Cooperative Oncology Group Performance Status 0 - 2
  • Chronic graft-versus-host disease progression after at least 2 prior lines of systemic therapy [that included ibrutinib, ruxolitinib and/or belumosudil, unless contraindicated]

Reproductive & Pregnancy Management

  • For females who can become pregnant: Pregnancy must be excluded prior to receiving axatilimab.
  • For females who can become pregnant: Counseling provided on potential risks vs benefits of treatment and the use of effective contraception during therapy and for 30 days after stopping treatment.

Source Documents