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Ipilimumab, Opdivo Provides Durable Response in Melanoma Brain Metastases


A study recently found that an immunotherapy combination treatment of ipilimumab (Yervoy) and nivolumab (Opdivo) shrank melanoma that had spread to the brain in more than half of participants.

An immunotherapy combination treatment of ipilimumab (Yervoy) and nivolumab (Opdivo) shrank melanoma that had spread to the brain in more than half of participants, according to a recent study. Results from the trial conducted by researchers at The University of Texas MD Anderson Cancer Center were published in the New England Journal of Medicine.

The open-label, multicenter, phase 2 trial enrolled 94 patients in the single-arm study. At a minimum follow-up of 9 months and a median of 14 months, the rate of clinical benefit was 57% (95% CI, 47 to 68). Specifically, 24 (26%) patients had a complete response, 28 (30%) had a partial response, and 2 (2%) had stable disease. The rate of extracranial clinical benefit was 56% (95% CI, 46 to 67).

“The absence of progression for that long with brain metastases is huge. Historically, the overall 1-year survival rate for patients with brain metastases is less than 20%, with the immunotherapy combination in this study, it’s 82%,” Hussein Tawbi, MD, PhD, associate professor of Melanoma Medical Oncology at MD Anderson, said in a statement.

In terms of adverse effects, 34 patients (36.2%) had some type of central nervous side effect, the most common of which was a headache experienced by 21 participants. Seven of the 34 patients experiencing adverse effects had more serious grade 3 or 4 toxicities—3 headaches, 2 patients experienced brain swelling, 1 patient had a brain hemorrhage, and 1 experienced syncope.

In total, 52 patients (55%) had a grade 3 or 4 side effect, with 19 (20%) having to leave the trial early. The safety profile of the combination treatment was similar to that reported in patients with melanoma who do not have brain metastases, although 1 patient did die from immune-related myocarditis.

Overall, nivolumab combined with ipilimumab had clinically meaningful intracranial efficacy, as well as extracranial activity in patients with melanoma who had untreated brain metastases.

“This practice-changing study proved that you can start with immunotherapy first with these patients, tackling both brain and extracranial disease at the same time,” said Tawbi. “And it opens up new opportunities for development of systemic therapies for metastatic melanoma.”


Tawbi H, Forsyth P, Algazi A, et al. Combined nivolumab and ipilimumab in melanoma metastatic to the brain. N Engl J Med. 2018; 379: 722-730. nejm.org/doi/full/10.1056/NEJMoa1805453. Published August 23, 2018. Accessed August 30, 2018.

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