Final analysis of phase II results with cemiplimab in metastatic basal cell carcinoma after hedgehog pathway inhibitors.

BACKGROUND: Metastatic basal cell carcinoma (mBCC) is a rare condition with no effective second-line treatment options. Cemiplimab is an immune checkpoint inhibitor that blocks the binding of programmed cell death-1 (PD-1) to its ligands, programmed death-ligand 1 (PD-L1) and programmed death-ligand 2 (PD-L2). Here, we present the final analysis of cemiplimab in patients with mBCC after first-line hedgehog pathway inhibitor (HHI) treatment (NCT03132636). PATIENTS AND METHODS: In this open-label, single-arm, phase II study, adults with mBCC and Eastern Cooperative Oncology Group performance status ≤1, post-HHI treatment, received cemiplimab 350 mg intravenously every 3 weeks for ≤93 weeks or until disease progression or unacceptable toxicity. The primary endpoint was objective response rate (ORR) by independent central review (ICR). Duration of response (DOR) was a key secondary endpoint. Other secondary endpoints were ORR per investigator assessment, progression-free survival (PFS), overall survival (OS), complete response rate, safety, and tolerability. RESULTS: Fifty-four patients were enrolled: 70% were male and the median age of patients was 64 [interquartile range (IQR) 57.0-73.0] years. The median duration of follow-up was 8 months (IQR 4-21 months). The ORR per ICR was 22% [95% confidence interval (CI) 12% to 36%], with 2 complete responses and 10 partial responses. Among responders, the median time to response per ICR was 3 months (IQR 2-7 months). The estimated median DOR per ICR was not reached [95% CI 10 months-not evaluable (NE)]. The disease control rate was 63% (95% CI 49% to 76%) per ICR and 70% (95% CI 56% to 82%) per investigator assessment. The median PFS per ICR was 10 months (95% CI 4-16 months); the median OS was 50 months (95% CI 28 months-NE). The most common treatment-emergent adverse events were fatigue [23 (43%)] and diarrhoea [20 (37%)]. There were no treatment-related deaths. CONCLUSIONS: Cemiplimab demonstrated clinically meaningful antitumour activity, including durable responses, and an acceptable safety profile in patients with mBCC who had disease progression on or intolerance to HHI therapy.

Estimating Utility Values for Health States of Nigerian Individuals with Stroke or Epilepsy Using the SF-36: A Brief Report on the Results of a Cross-Sectional Survey.

Background. Stroke and epilepsy are the most common neurologic conditions affecting individuals. The Short Form Six-Dimension Health Index (SF-6D) is a preference-based measure of health developed to estimate utility values from the SF-36. This study estimated utility values for health states of Nigerian individuals with stroke or epilepsy using the SF-36. Methods. SF-36 responses from 125 and 69 individuals with stroke and persons with epilepsy, respectively, were transformed into health state utility values using the SF-6D algorithm. The Excel program developed by Brazier and colleagues was used to generate the SF-6D utility score estimated using a set of parametric preference weights. The health state utility values were determined using ordinal health state and standard gamble valuation techniques. Results. Mean (s) ages of the stroke and epilepsy participants were 63.1 (11) and 39.6 (16) y, respectively. The mean (s) utility scores for stroke and epilepsy were 0.52 (0.10) and 0.65 (0.1) for standard gamble and 0.48 (0.13) and 0.68 (0.11), respectively, using the ordinal health state paradigm. The mean (s) utility of stroke (female = 0.46 [0.15]; male = 0.50 [0.12]) and epilepsy (female = 0.65 [0.13], male = 0.69 [0.11]) participants were reported. The mean (s) annual episodes of seizure was 18.7 (39). Conclusions. To our knowledge, this is the first study to suggest that females with stroke and those with epilepsy considered their health to be poorer than that of their male counterparts. The significance of our findings is that they may be helpful for researchers, policy makers, and clinicians by providing input into economic evaluations to facilitate resource allocation for stroke survivors and people living with epilepsy to improve their health outcomes and reduce the huge burden associated with the conditions. Highlight: We estimated a health state utility value for stroke and epilepsy to aid researchers and public health policy makers in conducting health economic analysis and outcomes research.