Original Investigation

Avelumab Plus Methotrexate for Gestational Trophoblastic Tumors

JAMA Oncology 10.1001/jamaoncol.2026.1697

June 11, 2026 at 11:00 AM EDT

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Does adding the immune checkpoint inhibitor avelumab to first-line methotrexate improve cure rates in low-risk gestational trophoblastic tumors (GTT)?In this phase 1/2 nonrandomized clinical trial of 26 patients, the addition of avelumab to first-line methotrexate demonstrated a manageable safety profile and resulted in human chorionic gonadotropin normalization with treatment discontinuation and no relapse in 96.2% of patients after a median of 41 months of follow-up. Among patients interested in pregnancy, 93% subsequently conceived, suggesting preserved fertility.Avelumab plus methotrexate may represent a promising first-line option for low-risk GTT, with potential to reduce methotrexate resistance, particularly in patients with higher International Federation of Gynecology and Obstetrics scores.

Corresponding Author: Benoit You, MD, PhD, Centre Hospitalier Lyon-Sud, Centre d’Investigation de Thérapeutiques en Oncologie et Hématologie de Lyon, Service d’Oncologie Médicale, Chemin du Grand Revoyet, 69495 Pierre-Benite, Lyon, France (benoit.you@chu-lyon.fr).

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