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Background: Vismodegib has been used for the treatment of locally advanced basal cell carcinoma (laBCC) and metastatic BCC (mBCC) since 2011. Most efficacy and safety data are provided by clinical trials. This study evaluates the effectiveness of vismodegib for the treatment of laBCC, mBCC and basal cell nevus syndrome (BCNS) patients, and the tumour characteristics associated with a higher probability of achieving a complete response in the Netherlands., Methods: A retrospective cohort study that included all patients ≥18 years with histologically proven basal cell carcinoma that received ≥1 dose of vismodegib between July 2011 and September 2019 in the Netherlands., Results: In total, 48 laBCC, 11 mBCC and 19 BCNS patients were included. Median progression-free survival was 10.3 months (95% confidence interval (CI), 7.5-22.6) for laBCC, 11.7 (95% CI, 5.2-17.5) for mBCC and 19.1 (95% CI, 7.4-20.2) for BCNS. Larger laBCCs were associated with a lower probability of complete response (hazard ratio (HR) 0.77 per increase in cm, p = 0.02). Of all BCNS patients, 63% received ≥2 treatment sequences with vismodegib; all achieved partial responses., Conclusions: Half of the aBCC patients progress within 1 year after the start of vismodegib treatment. More research is needed to investigate other treatment strategies after vismodegib progression and to evaluate long-term effects of repetitive vismodegib treatment.

With the continuously rising incidence and changing populations of patients with basal cell carcinoma, evidence about the different treatment modalities is mandatory. Randomized clinical trials, such as the surgery versus imiquimod for nodular superficial basal cell carcinoma trial, can provide this evidence. Patients can then be informed about all aspects of alternative treatment options so that conscious, shared decisions can be made., (Copyright © 2016 The Authors. Published by Elsevier Inc. All rights reserved.)

The development of the hedgehog pathway inhibitor vismodegib provides a new treatment option for metastasised and locally advanced basal cell carcinoma in which surgical excision or radiotherapy is contraindicated. Only a fraction of patients with basal cell carcinoma are eligible for this therapy, but it is effective in the majority of those who do receive vismodegib. However, development of tumour resistance is quite common and adverse events frequently lead to discontinuation of therapy. Intermittent treatment or combination therapy could reduce the occurrence of tumour resistance and diminish toxicity. We present three patients who were successfully treated with vismodegib: a 73-year-old man with locally advanced basal cell carcinoma, an 82-year-old man with basal cell carcinoma that had metastasised to the lungs, and a 42-year-old man with Gorlin syndrome.

Background: Noninvasive treatments are frequently used in treatment of superficial basal cell carcinoma (sBCC) because of better cosmetic results, lower costs, and less burden on health care services when compared with surgical excision. However, probability of treatment failure is higher after noninvasive therapies and may depend on histologic tumor characteristics., Objective: We sought to investigate whether tumor thickness and adnexal extension are determinants of treatment failure in sBCC treated with topical methylaminolevulinate-photodynamic therapy, imiquimod, or 5-fluorouracil., Methods: Data were derived from a randomized controlled trial on the effectiveness of methylaminolevulinate photodynamic therapy, imiquimod, and 5-fluorouracil for treatment of sBCC (ISRCTN79701845). For tumors with treatment failure (n = 112) and a randomly selected control group of tumors without treatment failure (n = 224) data on tumor thickness and adnexal extension were retrospectively collected. Treatment failure was defined as a clinically and histologically persistent or recurrent tumor within 1-year posttreatment., Results: Tumor thickness of included patients ranged from 0.2 to 1.0 mm. Tumor thickness and adnexal extension of sBCC were not significantly associated with treatment failure of methylaminolevulinate photodynamic therapy, imiquimod, or 5-fluorouracil., Limitations: Follow-up period of 1 year is a limitation., Conclusion: There seems to be no need to determine tumor thickness or adnexal extension in sBCC before treatment., (Copyright © 2015 American Academy of Dermatology, Inc. Published by Elsevier Inc. All rights reserved.)

Background: A recent noninferiority randomized controlled trial (RCT) indicated that imiquimod can be considered as superior to methylaminolevulinate photodynamic therapy (MAL-PDT) in the treatment of superficial basal cell carcinoma (sBCC). Knowledge of treatment effectiveness in subgroups of patients is of great value in clinical practice to select the most effective treatment for an individual patient with sBCC., Objectives: To explore whether the relative treatment effect of MAL-PDT and imiquimod is consistent across subgroups defined by patient and tumour characteristics., Methods: Data were derived from a single-blinded, noninferiority, multicentre RCT comparing MAL-PDT, topical imiquimod and fluorouracil (ISRCTN79701845). Treatment success was defined as free of tumour recurrence at 12-month follow-up. Subgroup analyses were performed for subgroups defined by sex, age, tumour location and tumour size., Results: Two hundred and two patients received MAL-PDT and 198 received imiquimod. The superiority of imiquimod vs. MAL-PDT was observed in subgroups of females, sBCC on the trunk and large tumours with risk differences in favour of imiquimod of 18·4% [95% confidence interval (CI) 7·8-29·0%], 21·0% (95% CI 10·9-31·1%) and 18·9% (95% CI 7·1-30·7%), respectively. Higher probability of treatment success for imiquimod vs. MAL-PDT was consistently found in all other subgroups with the exception of sBCC localized on the lower extremities in older patients. In the latter subgroup, the risk difference at the expense of imiquimod was -57·3% (95% CI -81·7% to -32·9%)., Conclusions: Imiquimod remains the first-choice treatment for sBCC in terms of effectiveness. In older patients with sBCC on the lower extremities MAL-PDT might be preferred. Results should be interpreted carefully as subgroup analyses were exploratory and not driven by prior hypotheses., (© 2014 British Association of Dermatologists.)

Background: Generally basal cell carcinoma can be simply and curatively treated. However, large and long-standing tumours can be locally very destructive and in rare cases even metastasize. Hedgehog pathway inhibitors such as vismodegib constitute a new and promising therapy for metastatic or locally advanced basal cell carcinoma., Case Description: We describe a patient with metastasized basal cell carcinoma and basal cell nevus syndrome who, in the context of a study, was successfully treated with vismodegib. The main undesirable effects in our patient were muscle cramps, loss of taste, nausea and hair loss., Conclusion: Basal cell carcinoma is potentially a locally destructive skin tumour that only very rarely metastasizes. Hedgehog pathway inhibitors such as vismodegib can be administered in a selected group of patients with basal cell carcinoma.

Basal cell carcinoma is the most common type of skin cancer and its incidence is still rising. In recent years, new treatment modalities have been developed and existing modalities refined. The aim of this article is to give a histology-based overview of the available evidence-based research. The literature was searched for randomized controlled trials from which the efficacy of investigated treatments was obtained. Where possible, treatment modalities were evaluated specifically. Selection criteria were histological subtype, primary or recurrent basal cell carcinoma and tumour localization. Although surgery remains the preferred treatment for most basal cell carcinomas, patient and tumour characteristics should be taken into account when choosing the most suitable treatment.

A 53-year-old woman with blistering on the hands was diagnosed with porphyria cutanea tarda (PCT). At that time, she was treated with interferon for liver metastasis of a pancreas tumor since 7 years. Although apparently rare, PCT has been described as possible cutaneous manifestation both of hepatic tumors and interferon treatment. Further, PCT can also be exacerbated by hepatitis C virus infection, alcohol, estrogens, HFE gene mutations predisposing for hereditary hemochromatosis, hexachlorobenzene, and 2,3,7,8-tetrachlorodibenzo-p-dioxin. Therefore, it is important to keep in mind that the manifestation of PCT may be associated with distinct precipitating factors, which are briefly discussed here.

Background A recent noninferiority randomized trial showed that in terms of clinical effectiveness imiquimod was superior and topical fluorouracil noninferior to methylaminolaevulinate photodynamic therapy (MAL-PDT) for treatment of superficial basal-cell carcinoma ( sBCC). Although it was expected that MAL-PDT would be more costly than either cream, a full cost-effectiveness analysis is necessary to determine the balance between effectiveness and costs. Objective To determine whether imiquimod or topical fluorouracil are cost-effective treatments for sBCC compared with MAL-PDT. Methods An economic evaluation was performed from a healthcare perspective. Data on resource use and costs were collected alongside the randomized clinical trial. The incremental cost-effectiveness ratio was expressed as the incremental costs per additional patient free of tumour recurrence. Results At 12 months follow-up, the total mean costs for MAL- PDT were €680, for imiquimod cream €526 and for topical fluorouracil cream €388. Both imiquimod and topical fluorouracil were cost-effective treatments compared with MAL- PDT. Comparing costs and effectiveness of both creams led to a incremental investment of €4451 to achieve an additional patient free of tumour recurrence. The acceptability curve showed that, for a threshold value of €4451, the probability of imiquimod being more cost-effective than topical fluorouracil was 50%. Conclusion Based on the 12 months follow-up results, imiquimod and topical fluorouracil cream are more cost-effective than MAL-PDT for treatment of sBCC. Hence, substituting MAL-PDT with either imiquimod or topical fluorouracil results in cost savings; these savings will be larger for topical fluorouracil. Long-term follow-up effectiveness data are necessary to confirm the cost-effectiveness of imiquimod vs. topical 5-fluorouracil cream. [ABSTRACT FROM AUTHOR]