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Axatilimab for cGvHD: Results from the phase II AGAVE-201 trial
By Ella Dixon
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CSF-1 is a key mediator of inflammation and fibrosis; therefore, inhibiting CSF-1 can reduce the symptoms of cGvHD. Axatilimab is an IgG4 monoclonal antibody that inhibits CSF-1 signaling. The phase II multinational, randomized AGAVE-201 trial evaluated three doses of axatilimab in 241 patients aged ≥2 years with recurrent/refractory cGvHD:
The primary endpoint was overall response in the first six treatment cycles. The key secondary endpoint was patient-reported decrease in cGvHD symptom burden, with safety also assessed.
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The primary endpoint was met across all dose groups, with response rates of 74% (95% CI, 63–83), 67% (95% CI, 55–77), and 50% (95% CI, 39–61) for the 0.3 mg, 1 mg, and 3 mg groups, respectively. |
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A clinically meaningful reduction in cGvHD symptoms, as measured by >5 point reduction in the modified Lee Symptom Scale, was observed in 60%, 69%, and 41% of patients in the 0.3 mg, 1 mg, and 3 mg groups, respectively. |
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The most common adverse events included dose-dependent transient laboratory abnormalities (≤62%), associated with CSF1 inhibition, and periorbital edema (3–29%). Higher doses led to more frequent discontinuations due to adverse events (6% for 0.3 mg, 22% for 1 mg, and 18% for 3 mg doses). |
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Axatilimab therefore shows promise as a new therapeutic option for cGvHD, particularly for patients refractory to prior lines of therapy, in addition to improving symptom control and reducing steroid dependence. |
Abbreviations: cGvHD, chronic graft-versus-host disease; CSF-1, colony stimulating factor-1; CI, confidence interval.
- Wolff D, Cutler C, Lee SJ, et al. Axatilimab in recurrent or refractory chronic graft-versus-host disease. N Engl J Med. 2024;391(11):1002-1014. DOI: 10.1056/NEJMoa2401537
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