Nevoid basal cell carcinoma syndrome (NBCCS), also known as Gorlin syndrome (GS), is a genetic disorder characterized by the development of multiple cutaneous BCCs due to mutations in the hedgehog signaling pathway. The use of hedgehog pathway inhibitors—vismodegib and sonidegib—has emerged as a promising therapeutic strategy for managing BCCs in individuals with GS. In a retrospective study conducted between March 2012 and January 2024, a cohort of 16 Gorlin syndrome patients who received treatment with either sonidegib or vismodegib were analyzed. The primary objectives of the study were to evaluate the efficacy, safety profile, and duration of response to oral hedgehog inhibitors in this patient population. The study assessed various parameters, including the number of new BCCs that developed before and after treatment initiation, the duration and sustainability of treatment responses, as well as the incidence of adverse effects associated with hedgehog inhibitor therapy. The findings of the study revealed that sustained treatment with hedgehog inhibitors could effectively suppress the progression of both new and existing BCCs. Furthermore, the results indicated that sonidegib exhibited superior efficacy and safety compared to vismodegib in the treatment of BCCs in individuals with GS. Notably, adjustments to the administration schedule of sonidegib were found to improve tolerability without compromising therapeutic efficacy, potentially leading to prolonged durations of treatment response and disease control.

Alopecia areata (AA) is a chronic autoimmune disorder causing nonscarring hair loss. Conventional AA treatments show limited efficacy, highlighting the need for more effective therapeutic options. Ritlecitinib, an oral Janus kinase 3/TEC family kinase inhibitor, demonstrated efficacy and safety in patients aged ≥ 12 years, with data available up to 48 weeks. However, real-life investigations into its effectiveness and safety are limited. This study sought to address this knowledge gap by investigating treatment outcomes among patients with AA undergoing ritlecitinib in a real-life setting. We retrospectively reviewed charts across three Italian tertiary referral centres. Compared with the ALLEGRO trial, a higher rate of Severity of Alopecia Tool score 20 responses has been observed.

Background: Prurigo nodularis (PN) is a debilitating skin condition. When inadequate disease control is achieved or other systemic therapies are contraindicated, Janus kinase inhibitors (JAKis) may be considered, although real-world evidence remains limited. Objectives: To investigate clinical findings and treatment outcomes among patients diagnosed with PN and treated with JAKis in a real-world setting. Methods: Retrospective cohort study across 23 Italian tertiary referral hospitals. Patients with PN were eligible if aged ≥ 18 years and had received a JAKi with a minimum follow-up of 12 weeks. The primary outcome was defined as the proportion of patients achieving a reduction of ≥ 4 points from baseline in the Peak Pruritus Numerical Rating Scale score. Key secondary outcomes included the rates of patients reaching a significant reduction in Investigator Global Assessment of Prurigo Nodularis Stage (IGA PN-S) and Investigator Global Assessment of Prurigo Nodularis Activity (IGA PN-A) scores. Results: Of the total, 71 patients met the inclusion criteria. At week 16, the proportion of patients achieving the primary outcome was 94% for upadacitinib [n = 52/55; 95% confidence interval (CI) 87–100%], 83% for abrocitinib (n = 10/12; 95% CI 52–98%) and 100% for baricitinib (n = 4/4; 95% CI 40–100%), with results sustained at week 24. An IGA PN-A score of 0/1 was achieved in 90% of patients treated with upadacitinib and abrocitinib and in 50% of those on baricitinib by week 24. Improvements were observed across all other secondary outcomes assessed, with no safety concerns reported. Conclusions: This study suggests that JAKis can achieve clinically meaningful outcomes in PN irrespective of atopic background, supporting their use across diverse patient profiles. Further research is warranted to validate these observations and explore their long-term effects.