PMID- 35279879 OWN - NLM STAT- MEDLINE DCOM- 20220719 LR - 20221015 IS - 1468-3083 (Electronic) IS - 0926-9959 (Print) IS - 0926-9959 (Linking) VI - 36 IP - 8 DP - 2022 Aug TI - Analysis of efficacy and safety of vismodegib therapy in patients with advanced basal cell carcinoma - real world multicenter cohort study. PG - 1219-1228 LID - 10.1111/jdv.18070 [doi] AB - BACKGROUND: Basal cell carcinoma (BCC) is the most frequent non-melanoma skin cancer. The basis of treatment is surgical resection. The treatment of locally advanced and metastatic disease is currently based on sonidegb or vismodegib, small molecule inhibitors of the hedgehog signalling pathway. OBJECTIVES: The study aimed to retrospectively analyse the efficacy and safety of treatment with vismodegib in 108 patients with locally advanced or metastatic disease treated from August 1st, 2017 to December 31st, 2020. The primary objective was to evaluate the objective response rate (ORR), overall survival (OS) and progression-free survival rates. The secondary aims of the study were the disease control rate, the incidence of adverse events (AEs) and the estimation of the factors that potentially impact the treatment outcome and patient survival. METHODS: Patients treated in national drug programme were enrolled into this retrospective cohort study. Evaluation of the treatment efficacy was performed according to CT/MRI scans and by the response evaluation criteria in solid tumours (RECIST) 1.1. The safety evaluation was performed according to the Common Terminology Criteria for Adverse Events v. 5.0 (CTCAE) classification and severity assessment. RESULTS: The median duration of treatment was 14 months (range 1-94 months). The median progression-free survival reached 30.5 months (95% CI; 24.8-36.3), and the progression-free survival rate after 6, 12 and 24-months were 92%, 78% and 61%, respectively. The median overall survival was 41.5 months (95% CI; 31.6-51.3), and the overall survival rate after 1, 2 and 3 years accordingly 86%, 73% and 60%. The univariant and multivariant analysis indicated that the female gender is an independent positive prognostic factor of progression-free survival. CONCLUSIONS: The response to treatment is the prognostic factor for response maintenance and better overall survival. The therapy was well tolerated with the safety profile consistent in general with known from previous studies. CI - (c) 2022 The Authors. Journal of the European Academy of Dermatology and Venereology published by John Wiley & Sons Ltd on behalf of European Academy of Dermatology and Venereology. FAU - Slowinska, M AU - Slowinska M AD - Department of Dermatology, Military Institute of Medicine, Warsaw, Poland. FAU - Dudzisz-Sledz, M AU - Dudzisz-Sledz M AD - Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland. FAU - Sobczuk, P AU - Sobczuk P AD - Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland. AD - Department of Experimental and Clinical Physiology, Laboratory of Centre for Preclinical Research, Medical University of Warsaw, Warsaw, Poland. FAU - Lasinska, I AU - Lasinska I AUID- ORCID: 0000-0001-5426-2725 AD - Department of Medical and Experimental Oncology, Heliodor Swiecicki Clinical Hospital, Poznan University of Medical Sciences, Poznan, Poland. AD - Department of Nursing, Institute of Medical Sciences, Collegium Medicum, University of Zielona Gora, Zielona Gora, Poland. FAU - Pietruszka, A AU - Pietruszka A AD - 1st Radiation and Clinical Oncology Department, Maria Sklodowska-Curie National Research Institute of Oncology, Gliwice Branch, Gliwice, Poland. FAU - Cybulska-Stopa, B AU - Cybulska-Stopa B AD - Clinical Oncology Department, Maria Sklodowska-Curie National Research Institute of Oncology, Cracow Branch, Cracow, Poland. FAU - Kowalczyk, A AU - Kowalczyk A AD - Department of Oncology and Radiotherapy, Medical University of Gdansk, Gdansk, Poland. FAU - Switaj, T AU - Switaj T AD - Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland. FAU - Czarnecka, I AU - Czarnecka I AD - Department of Dermatology, Military Institute of Medicine, Warsaw, Poland. FAU - Kosela-Paterczyk, H AU - Kosela-Paterczyk H AD - Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland. FAU - Rogala, P AU - Rogala P AD - Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland. FAU - Paluchowska, E AU - Paluchowska E AD - Department of Dermatology, Military Institute of Medicine, Warsaw, Poland. FAU - Skladowski, K AU - Skladowski K AD - 1st Radiation and Clinical Oncology Department, Maria Sklodowska-Curie National Research Institute of Oncology, Gliwice Branch, Gliwice, Poland. FAU - Mackiewicz, J AU - Mackiewicz J AD - Department of Medical and Experimental Oncology, Heliodor Swiecicki Clinical Hospital, Poznan University of Medical Sciences, Poznan, Poland. AD - Department of Diagnostics and Cancer Immunology, Greater Poland Cancer Centre, Poznan, Poland. FAU - Rutkowski, P AU - Rutkowski P AD - Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland. FAU - Owczarek, W AU - Owczarek W AD - Department of Dermatology, Military Institute of Medicine, Warsaw, Poland. LA - eng PT - Journal Article PT - Multicenter Study DEP - 20220405 PL - England TA - J Eur Acad Dermatol Venereol JT - Journal of the European Academy of Dermatology and Venereology : JEADV JID - 9216037 RN - 0 (Anilides) RN - 0 (Antineoplastic Agents) RN - 0 (Hedgehog Proteins) RN - 0 (HhAntag691) RN - 0 (Pyridines) SB - IM MH - Anilides/adverse effects MH - *Antineoplastic Agents/adverse effects MH - *Carcinoma, Basal Cell/drug therapy/pathology MH - Female MH - Hedgehog Proteins/metabolism MH - Humans MH - Pyridines MH - Retrospective Studies MH - *Skin Neoplasms/pathology PMC - PMC9541446 EDAT- 2022/03/14 06:00 MHDA- 2022/07/20 06:00 PMCR- 2022/10/07 CRDT- 2022/03/13 20:33 PHST- 2021/08/10 00:00 [received] PHST- 2022/02/09 00:00 [accepted] PHST- 2022/03/14 06:00 [pubmed] PHST- 2022/07/20 06:00 [medline] PHST- 2022/03/13 20:33 [entrez] PHST- 2022/10/07 00:00 [pmc-release] AID - JDV18070 [pii] AID - 10.1111/jdv.18070 [doi] PST - ppublish SO - J Eur Acad Dermatol Venereol. 2022 Aug;36(8):1219-1228. doi: 10.1111/jdv.18070. Epub 2022 Apr 5.