Phase II basket trial of dual anti-CTLA-4 and anti-PD-1 blockade in rare tumors

1. Treatment of desmoid tumours with dual anti-CTLA-4 and anti-PD-1 blockade with ipilimumab and nivolumab resulted in an overall response rate (ORR) of 18.8% and a clinical benefit rate (CBR) of 62.5%.

Evidence Rating Level: 1 (Excellent)

Desmoid tumours are an extremely rare type of neoplasm originating from fibroblasts. The current standard of care involve active surveillance with surgery being reserved for specific cases. Systemic therapy involving various agents including chemotherapy and tyrosine kinase inhibitors is indicated for cases of unresectable disease. This phase II trial therefore sought to investigate the efficacy of dual anti-CTLA-4 and anti-PD-1 immune checkpoint inhibition on desmoid tumours. 16 patients (median age [range] = 37 [20-82] years) from various centres in the United States were included in the study on the basis of having a histologically confirmed desmoid tumour with no other available treatment options that could improve overall survival (OS) or contraindications to such treatment options. The ORR was 18.8% (3/16) with an overall CBR of 62.5% (10/16). Three patients had a partial response (PR) with seven patients showing evidence of tumour shrinkage. The median progression-free survival (PFS) was 19.4 months (95% CI 8.3 months-not reached). Overall, treatment with ipilimumab and nivolumab in patients with desmoid tumours resulted in an ORR of 18.8% and a CBR of 62.5%, representing a possible additional treatment option for individuals living with desmoid tumours. 

Click to read the study in BMJ

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