Real-world effectiveness of topical ruxolitinib in non-segmental vitiligo: repigmentation, mental health, and quality-of-life outcomes in an 860-patient multicentre study
Angelo V Marzano, Alessandra Narcisi, Gianluca Tavoletti, Luisa C Rossi, Maurizio Romagnuolo, Matteo Bianco, Paolo Amerio, Edvige Morea, Eugenio Provenzano, Franco Rongioletti, Stefania Guida, Lorenzo M Pinto, Alessandra D’Amore, Maria-Concetta Fargnoli, Alessia Pacifico, Paolo Iacovelli, Pietro Rubegni, Emanuele Trovato, Marco Romanelli, Alessandra Michelucci, Giuseppe Micali, Maria L Musumeci, Aldo L Di Marco, Maddalena Napolitano, Luigi Coronella, Caterina Foti, Alessandro Borghi, Lucrezia Pacetti, Serena Giacalone, Cristina B Spigariolo, Gianluca Nazzaro, Simone Ribero, Luca Mastorino, Anna Belloni Fortina, Alvise Sernicola, Oriana Simonetti, Marco Matteucci, Eustachio Nettis, Luca Stingeni, Giovanni Pellacani, Annunziata Dattola, Iris Zalaudek, Raffaele D Caposiena Caro, Emiliano Antiga, Maria E Baffa, Maria Esposito, Manfredo Bruni, Paolo Sena, Pamela Vezzoli, Lorenzo Rocca, Emanuele Cozzani, Giulia Gasparini, Niccolò Capurro, Paola Savoia, Piergiacomo Calzavara-Pinton, Mariachiara Arisi, Tomasini Carlo, Andrea Michelerio, Stefania Barruscotti, Mauro Picardo, Alessia Paganelli, Piergiorgio Malagoli, Alessandro Pileri, Roberta Giuffrida, Santo R Mercuri, Vittoria G Bianchi, Giuseppe Argenziano, Elisabetta Fulgione, Giampiero Girolomoni, Martina Maurelli, Antonio Costanzo, Alessandra Cartocci, Ketty Peris, Silvia Alberti-ViolettiAbstract
Background
Vitiligo affects 0·5–2% of the global population and carries a substantial psychosocial burden. Topical ruxolitinib 1·5% cream is approved for non-segmental vitiligo based on phase-3 trial data, but real-world evidence across heterogeneous populations remains limited.
Objectives
To evaluate the real-world effectiveness, safety, and patient-reported outcomes of topical ruxolitinib across 34 Italian dermatology centres, and to identify possible clinical predictors of treatment response.
Methods
Multicentre retrospective cohort study (July 2024–October 2025). Patients (≥12 years) with non-segmental vitiligo received ruxolitinib 1·5% cream twice daily. Primary outcomes were F-VASI75 and F-VASI90 at 24 and 52 weeks. Secondary outcomes were VNS, DLQI, VitiQoL, GAD-7, and PHQ-9.
Results
860 patients (mean age 47 years; 58% female; disease duration 13·5 years; 84% previously treated) completed 24-week assessment; 229 (26·6%) had reached 52 weeks at the time of data lock. F-VASI75 was achieved by 18·5% at 24 weeks, increasing to 38·4% at 52 weeks; F-VASI90 rose from 5·7% to 25·8%. In multivariable logistic regression, summer treatment initiation (adjusted OR 6·7, 95% CI 2·7–16·8) and Koebner phenomenon (adjusted OR 1·6, 95% CI 1·04–2·66) were independent predictors of F-VASI75 at 24 weeks. At baseline, 37% of patients had moderate-to-severe anxiety (GAD-7 ≥10); this fell to 11% at 24 weeks and 2% at 52 weeks. VitiQoL, DLQI, and PHQ-9 improved at all timepoints. Adverse events were mild in 98·9%; treatment discontinuation rate was 1·5%.
Conclusions
In a large real-world population considerably more heterogeneous than registration trial cohorts, topical ruxolitinib showed progressive repigmentation through 52 weeks with excellent tolerability. Summer initiation and Koebner phenomenon independently predicted early response, findings that may inform patient counselling and the design of prospective studies. The parallel sustained reduction in anxiety and depression argues for standardised mental health assessment as a routine component of vitiligo care.