Nivolumab, Ipilimumab Combo Demonstrates Clinical Benefit Among Adults With Pediatric Solid Tumors

The combination of nivolumab (Opdivo) and ipilimumab (Yervoy) demonstrated significant clinical benefit in patients with solid pediatric tumors that progressed in adulthood, a patient population with few treatment options, according to study findings presented at the 2019 American Society of Clinical Oncology Annual Meeting in Chicago, Illinois.

According to findings of the first cohort from the phase 2, single-arm study, during a median follow-up of 4.3 months, the combination yielded a 39.3% clinical benefit rate among 30 patients.

“Solid pediatric tumors that appear in adulthood are a heterogeneous group characterized by a low incidence, lack of therapeutic options, and reduced survival,” wrote the researchers.

While on the treatment, patients achieved a median overall survival of 6.8 months. All patients had locally advanced or metastatic pediatric malignancies that had progressed or were not candidates for traditional therapy.

“One case of metastatic esthesioneuroblastoma achieved a dramatic tumor response and represents the first patient with this extremely rare histology treated with immunotherapy,” noted the researchers.

In addition to the 1 patient who achieved a deep partial response during treatment, 10 had stable disease, 17 had progressive disease, and 2 patients died before radiologic evaluation.

The patients, who were enrolled across 15 centers of the Spanish Group for Rare Cancer, included 4 with medulloblastoma, 4 with neuroblastoma, and 3 with Ewing family tumors. The majority (90%) of patients had received prior systemic therapy, with partial response representing the best response, which occurred in 37% of these patients. The median amount of previous treatment lines was 3.

The treatment regimen consisted of nivolumab 3 mg/kg once every 2 weeks plus ipilimumab 1 mg/kg once every 6 weeks for 6 months or until disease progression or discontinuation due to treatment toxicity, for a maximum of 24 months. Among the 30-person cohort, 6 patients have been treated for at least 6 months, with 1 discontinuing due to adverse events.

A total of 12 (40%) patients experienced adverse events of any grade and 6 (20%) experienced an adverse event possibly related to the treatment combination.

Reference:

Mieglo X, Diaz-Beveridge R, Sepulveda J, et al. Interim analysis of a phase II study of nivolumab combined with ipilimumab in patients with pediatric solid tumors in adulthood (GETHI021). Presented at: Presented at: 2019 American Society of Clinical Oncology Annual Meeting; May 31-June 4, 2019; Chicago, IL. Abstract: 2613.