Everolimus plus long-acting release pasireotide (pasireotide-LAR) has promising efficacy compared to a single agent for the treatment of radioiodine-refractory differentiated thyroid cancer and medullary thyroid carcinoma (MTC), according to the results of a randomized phase 2 clinical trial that was published in Cancers.

The aim of the trial was to assess the clinical efficacy and establish the most promising regimen and optimal combination sequence of the 2 treatments. A total of 42 patients, the median age of 65 years with progressive differentiated thyroid cancer or MTC were enrolled. Patients could either be untreated or treated with only 1 systemic agent.

Patients were divided into 3 groups. Those in the first group were treated with 10 mg of everolimus once a day. Those in the second group were given 60 mg of pasireotide-LAR as an intramuscular injection every 4 weeks. Finally, those in the third group received a combination of the 2 treatments. In case of disease progression, patients in the first or second groups switched to the combination therapy and continued with it until a further progression.

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The results showed that there was no objective response based on the Response Evaluation Criteria in Solid Tumors criteria in patients in any of the 3 groups. The 1-year progression-free survival was 8.3 months and 49.9% for patients treated with everolimus only. It was 1.8 months and 36.4% for those treated with pasireotide-LAR only, and finally, it was 8.1 months and 25% for those treated with the combination therapy.

The median and 1-year progression-free survival to the second progression were 26.3 months and 78.4% for patients in the first group, 17.5 months and 70% for patients in the second group, and 8.1 months and 25% for those in the third group.

The researcher reported that “the most frequent adverse events were anemia, stomatitis, fatigue, hyperglycemia, and hypercholesterolemia.”

“The delayed combination of everolimus and pasireotide-LAR following progression on single agent everolimus appeared intriguing as a combination strategy,” they concluded.


Bauman JE, Chen Z, Zhang C, et al. A multicenter randomized phase II study of single agent efficacy and optimal combination sequence of everolimus and pasireotide LAR in advanced thyroid cancer. Cancers. 2022;14(11):2639. doi:10.3390/cancers14112639

Pasireotide & everolimus in adult patients with radioiodine-refractory differentiated & medullary thyroid cancer. US National Library of Medicine. Updated February 17, 2021. Accessed June 17, 2022.