Development of a core outcome set for cutaneous squamous cell carcinoma trials: identification of core domains and outcomes.

BACKGROUND: The lack of uniformity in the outcomes reported in clinical studies of the treatment of cutaneous squamous cell carcinoma (cSCC) complicates efforts to compare treatment effectiveness across trials. OBJECTIVES: To develop a core outcome set (COS), a minimum set of agreed-upon outcomes to be measured in all clinical trials of a given disease or outcome, for the treatment of cSCC. METHODS: One hundred and nine outcomes were identified via a systematic literature review and interviews with 28 stakeholders. After consolidation of this long list, 55 candidate outcomes were rated by 19 physician and 10 patient stakeholders, in two rounds of Delphi exercises. Outcomes scored 'critically important' (score of 7, 8 or 9) by ≥ 70% of patients and ≥ 70% of physicians were provisionally included. At the consensus meeting, after discussion and voting of 44 international experts and patients, the provisional list was reduced to a final core set, for which consensus was achieved among all meeting participants. RESULTS: A core set of seven outcomes was finalized at the consensus meeting: (i) serious or persistent adverse events, (ii) patient-reported quality of life, (iii) complete response, (iv) partial response, (v) recurrence-free survival, (vi) progression-free survival and (vii) disease-specific survival. CONCLUSIONS: In order to increase the comparability of results across trials and to reduce selective reporting bias, cSCC researchers should consider reporting these core outcomes. Further work needs to be performed to identify the measures that should be reported for each of these outcomes.

Systemic treatment of locally advanced nonmetastatic cutaneous squamous cell carcinoma: a review of the literature.

BACKGROUND: There is a small subset of locally advanced nonmetastatic cutaneous squamous cell carcinoma (cSCC) for which local therapy is not curative or feasible, making systemic therapy a possible treatment option. OBJECTIVES: To calculate overall response rates (ORR), median time to response (TTR) and median duration of response (DOR) of locally advanced nonmetastatic cSCC to systemic therapy [targeted agents, biological response modifiers (BRM) and chemotherapy]. PATIENTS AND METHODS: Medline and PubMed were searched for reports of nonmetastatic locally advanced cSCC treated with systemic therapy from 1970 to 2011. No limits were placed on study design. ORR, TTR and DOR were calculated for systemic therapy overall and for each treatment category. RESULTS: Twenty-eight observational studies yielded 119 patients for analysis. The ORR for systemic therapy was 72% (TTR 9 weeks, DOR 42 weeks). Targeted therapy and BRM achieved ORR of 100% (TTR 12 weeks, DOR 20 weeks) and 86% (TTR 10 weeks, DOR 20 weeks), respectively, and oral chemotherapy, intravenous chemotherapy and intra-arterial chemotherapy achieved ORR of 20% (TTR 10 weeks, DOR 24 weeks), 68% (TTR 3 weeks, DOR 44 weeks) and 100% (TTR 15 weeks, DOR 112 weeks), respectively. A limitation of this study was that no controlled data were identified and sample sizes were small. CONCLUSIONS: Systemic treatment leads to objective responses in locally advanced cutaneous SCC that are not amenable to local cure.