Trial reportJournal of the European Academy of Dermatology and Venereology : JEADV2018
Long-term efficacy and safety of sonidegib in patients with locally advanced and metastatic basal cell carcinoma: 30-month analysis of the randomized phase 2 BOLT study.
Trial report in Journal of the European Academy of Dermatology and Venereology : JEADV, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01327053 (Phase II, Randomized Double-blind Study of Efficacy and Safety of Two Dose Levels of LDE225 in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma), which is not on this map. Cited by 81 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Phase II, Randomized Double-blind Study of Efficacy and Safety of Two Dose Levels of LDE225 in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma
Who cites it
81 citing papers in PubMed, 3 syntheses or guidelines pooled it, 190 citations in OpenAlex.
- S2k guideline basal cell carcinoma of the skin (update 2023).Journal der Deutschen Dermatologischen Gesellschaft = Journal of the German Society of Dermatology : JDDG · 2024Guideline
- Swiss Recommendations for Cutaneous Basal Cell Carcinoma.Dermatology (Basel, Switzerland) · 2023Guideline
- A Practical Guide for the Follow-Up of Patients with Advanced Basal Cell Carcinoma During Treatment with Hedgehog Pathway Inhibitors.The oncologist · 2019Guideline
- A Phase II Trial of Perioperative Oral Itraconazole for the Management of Low-Risk Basal Cell Carcinoma.Experimental dermatology · 2026Trial
- Trial
- Long-term efficacy and safety of sonidegib in patients with advanced basal cell carcinoma: 42-month analysis of the phase II randomized, double-blind BOLT study.The British journal of dermatology · 2020Trial
- Glasdegib in combination with cytarabine and daunorubicin in patients with AML or high-risk MDS: Phase 2 study results.American journal of hematology · 2018Trial
- Cell differentiation-related signaling pathways: biological functions, diseases and therapeutic targets.Signal transduction and targeted therapy · 2026Review
- Article
- Targeted therapies in adolescent and young adult patients with central nervous system tumors.Neuro-oncology advances · 2026Review
- Real-World Treatment Patterns of Patients with Basal Cell Carcinoma Using Sonidegib and Vismodegib: Discontinuation Rates and Clinical Conditions During Treatment.Dermatology and therapy · 2026Article
- Clinical Outcomes of Sonidegib in Vismodegib-Exposed Locally Advanced Basal Cell Carcinoma: Insights From a Multicenter Descriptive Study.Journal of skin cancer · 2026Article
- Secondary resistance to sonidegib is rare in locally advanced basal cell carcinoma.Journal of the European Academy of Dermatology and Venereology : JEADV · 2025Article
- Immunohistochemical expression of smoothened in periocular basal cell, squamous cell and sebaceous carcinomas.Scientific reports · 2025Article
- Double-sided niche regulation in skin stem cell and cancer: mechanisms and clinical applications.Molecular cancer · 2025Review
- Survey of the impact of BOLT-trial data on oncologists' and dermatologists' decision-making in treating patients with locally advanced basal cell carcinoma.Dermatology reports · 2025Article
- Review
- Therapeutic Advances in Advanced Basal Cell Carcinoma.Cancers · 2024Review
- Evaluation of the Tolerability of Hedgehog Pathway Inhibitors in the Treatment of Advanced Basal Cell Carcinoma: A Narrative Review of Treatment Strategies.American journal of clinical dermatology · 2024Review
- Images in Immunotherapy and Precision Oncology: Advanced Basal Cell Carcinoma.Journal of immunotherapy and precision oncology · 2024Article
21 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
20 authors at 17 institutions in 8 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPatients with locally advanced basal cell carcinoma (laBCC) or metastatic BCC (mBCC), two difficult-to-treat populations, have had limited treatment options. Sonidegib, a hedgehog pathway inhibitor (HPI), was approved in laBCC based on results from the BOLT trial.
objectiveTo evaluate long-term efficacy and safety of sonidegib in laBCC and mBCC in the BOLT 18- and 30-month analyses.
methodsBOLT (NCT01327053, ClinicalTrials.gov), a double-blind phase 2 study, enrolled patients from July 2011 until January 2013. Eligible HPI-treatment-naïve patients with laBCC not amenable to curative surgery/radiotherapy or mBCC were randomized 1 : 2 to sonidegib 200 mg (laBCC, n = 66; mBCC, n = 13) or 800 mg (laBCC, n = 128; mBCC, n = 23). Tumour response was assessed per central and investigator review.
resultsWith 30 months of follow-up, among patients treated with sonidegib 200 mg (approved dose), objective response rates were 56.1% (central) and 71.2% (investigator) in laBCC and 7.7% (central) and 23.1% (investigator) in mBCC. Tumour responses were durable as follows: median duration of response was 26.1 months (central) and 15.7 months (investigator) in laBCC and 24.0 months (central) and 18.1 months (investigator) in mBCC. Five patients with laBCC and three with mBCC in the 200-mg arm died. Median overall survival was not reached in either population; 2-year overall survival rates were 93.2% (laBCC) and 69.3% (mBCC). In laBCC, efficacy was similar regardless of aggressive or non-aggressive histology. Sonidegib 200 mg continued to have a better safety profile than 800 mg, with lower rates of grade 3/4 adverse events (43.0% vs. 64.0%) and adverse events leading to discontinuation (30.4% vs. 40.0%).
conclusionSonidegib continued to demonstrate long-term efficacy and safety in these populations. These data support the use of sonidegib 200 mg per local treatment guidelines.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.