Avelumab Plus Methotrexate for Gestational Trophoblastic Tumors: The TROPHAMET Phase 1/2 Nonrandomized Clinical Trial.
Avelumab plus methotrexate achieved high rates of hCG normalization in low-risk gestational trophoblastic tumors.
Avelumab Plus Methotrexate for Gestational Trophoblastic Tumors: The TROPHAMET Phase 1/2 Nonrandomized Clinical Trial.
To evaluate the safety and efficacy of avelumab combined with methotrexate as first-line therapy in patients with low-risk GTT.
DESIGN, SETTING, AND PARTICIPANTS: TROPHAMET (Avelumab and Methotrexate in Low-Risk Gestational Trophoblastic Neoplasias as First-Line Treatment) was a multicenter, phase 1/2 nonrandomized clinical trial of patients with low-risk GTT (FIGO score ≤6) treated at academic referral centers.
Of 27 female patients treated (median [range] age, 35 [20-50] years), 26 were assessable for efficacy.
Eight patients (31%) had FIGO scores of 1 to 2; 8 patients (31%) had scores of 3 to 4; and 10 patients (38%) had scores of 5 to 6.
The treatment regimen demonstrated acceptable safety: 1 DLT occurred (grade 3 sepsis on central venous catheter); immune- and treatment-related adverse events of grade 2 or higher occurred in 6 patients (22%), all of which fully resolved except for 1 case of grade 2 dysthyroidism; and there were no grade 4 or higher events.
In this nonrandomized clinical trial, avelumab combined with methotrexate demonstrated a manageable safety profile and high efficacy in low-risk GTT in more than 95% of patients, with durable responses and preserved fertility.
This combination may represent a promising first-line strategy warranting evaluation in comparative studies, particularly for patients at higher risk of chemoresistance.