Aims:
Immunotherapy has revolutionised the management of advanced cutaneous squamous cell carcinoma (CSCC)1-5. However, the stringent inclusion criteria of clinical trials results in key populations with advanced CSCC being excluded in the key registrational studies. This includes the elderly, the immunocompromised, those with autoimmune disease and organ transplant recipients. This has generated interest in reviewing real-world populations treated with immunotherapy via access schemes, however, to date many of these reports have been limited by small patient numbers6-9. To our knowledge this is the largest real-world report of advanced CSCC patients treated with immunotherapy.
Methods:
This was a multi-centre national retrospective review performed across 15 Australian institutions of patients with advanced CSCC who received immunotherapy via an access program. The primary endpoint was the best overall response rate (ORR) as per standardised assessment criteria using the hierarchy of Response Evaluation Criteria in Solid Tumors 1.1, modified World Health Organisation clinical response criteria or Positron Emission Tomography Response Criteria 1.0. We assessed toxicity as per Common Terminology Criteria for Adverse Events version 5 and correlated baseline clinico-pathological features with both overall (OS) and progression free survival (PFS).
Results:
286 patients were analysed. Median age was 75.2 years (range 39.3-97.5); 81% were male, 31% immunocompromised, 9% had an autoimmune disease and 21% were ECOG 2+. ORR was 63% with 28% complete responses, 35% partial responses, 22% stable disease and 16% with progressive disease. Median follow-up was 12 months. The 12-month OS and PFS were 78% (95%CI 72-83) and 65% (95%CI 58-70) respectively. In multivariate analysis poorer ECOG and immunocompromised status were associated with worse OS and PFS. 19% of patients reported grade 2+ immune-related adverse events.
Conclusions:
This study demonstrates that immunotherapy is effective and well-tolerated in a real-world cohort of advanced CSCC patients. Comparable efficacy was seen to what has been demonstrated in clinical trials with similar toxicity.