Dataset Information

Real-Life Effectiveness of Vismodegib in Patients with Metastatic and Advanced Basal Cell Carcinoma: Characterization of Adverse Events and Assessment of Health-Related Quality of Life using the Dermatology Life Quality Index (DLQI) Test.

ABSTRACT:

Introduction

Non-melanoma skin cancer (NMSC) is the most common type of human tumor, with an estimated five million new cases each year. NMSC has been described as having a major impact on the health-related quality of life of the patient. Vismodegib is a hedgehog pathway inhibitor therapy for patients who are affected by locally advanced basal cell carcinoma (laBCC) and metastatic basal cell carcinoma and are ineligible for surgery and/or radiotherapy. The objective of this study was to assess treatment-emergent adverse events reported by patients with advanced BCC who were undergoing hedgehog pathway inhibitor therapy with vismodegib, and to quantify their health-related quality of life using the Dermatology Life Quality Index (DLQI) questionnaire.

Methods

Patients with advanced and/or multiple basal cell carcinomas treated with vismodegib at the Non-Melanoma Skin Cancer Unit of the University of Naples Federico II (Italy) were consecutively enrolled. Each patient was evaluated every month until the end of the treatment cycle to assess adverse events related to the drug and the patient's quality of life.

Results

48 patients (35 males and 13 females) with advanced BCC were included in the study. Muscle spasms, alopecia, and dysgeusia were the most frequently reported adverse events. 41 patients completed the DLQI questionnaire at the baseline visit and after 6 months of treatment. The average reported DLQI score decreased from a mean value of 5.7 at the baseline visit to 0.4 after 6 months of treatment.

Conclusion

This is the first study to demonstrate a significant change in patient health-related quality of life from baseline to 6 months after hedgehog pathway inhibitor therapy initiation using the DLQI test. Interestingly, patients with BCC in visible areas such as the face or neck presented an overall DLQI score that was higher than that of patients with BCC located on the trunk and legs at the baseline visit, but the DLQI scores of these two groups were almost the same after 6 months of vismodegib therapy.

SUBMITTER: Villani A

PROVIDER: S-EPMC6704212 | biostudies-literature |

REPOSITORIES: biostudies-literature

ACCESS DATA

Similar Datasets

Project description:IntroductionSonidegib is approved to treat locally advanced basal cell carcinoma (laBCC) in the USA, EU, Switzerland, and Australia and metastatic basal cell carcinoma (mBCC) in Switzerland and Australia in patients not amenable to surgery or radiotherapy. Vismodegib is approved to treat patients with mBCC, recurrent laBCC, or those not candidates for surgery or radiation. There is no head-to-head trial comparing Hedgehog inhibitors. We describe time to onset and severity of adverse events (AEs) in two studies reporting cumulative AE incidence every treatment cycle: the sonidegib phase 2 BOLT study and the expanded-access, open-label vismodegib study.MethodsThis analysis included patients with histologically confirmed laBCC or mBCC from BOLT who received sonidegib 200 mg once daily (QD) and patients from the vismodegib study who received vismodegib 150 mg QD. Cumulative occurrence of AEs and median time to AE onset were calculated on 30-day cycles for sonidegib and 28-day cycles for vismodegib. AEs were graded for severity using the Common Terminology Criteria for Adverse Events. Only common (at least 15% incidence) AEs were analyzed in this study.ResultsOver 18 treatment cycles, the most common all-grade AEs for sonidegib and vismodegib were muscle spasm (54.4% vs 70.6%; P = 0.0236), alopecia (49.4% vs 58.0%; no significant difference [NS]), and dysgeusia (43.0% vs 70.6%; P = 0.0003); incidences of diarrhea, nausea, fatigue, and weight decrease were 31.6% vs 25.2% (NS), 39.2% vs 19.3% (P = 0.0032), 32.9% vs 19.3% (P = 0.0429), and 30.4% vs 16.0% (P = 0.0217), respectively. Sonidegib-treated patients had more delayed median time to onset for all AEs than vismodegib-treated patients, except fatigue and weight decrease (NS). Most AEs reported were grade ≤ 2.ConclusionThis post hoc analysis suggests lower overall incidence and slower onset of certain AEs in patients treated with sonidegib compared with vismodegib. In the absence of head-to-head comparisons, the relevance of these findings needs further studies to provide conclusive evidence.

Project description:INTRODUCTION:The Patient-Oriented Eczema Measure (POEM) assesses patient-reported frequency of atopic dermatitis (AD) symptoms, while the Children's Dermatology Life Quality Index (CDLQI) measures the impact of skin disease on health-related quality of life (HRQoL) in children. There is currently no threshold for clinically meaningful within-person change in POEM or CDLQI scores in adolescents. Here we empirically derive within-person thresholds of meaningful within-person change in POEM and CDLQI scores in adolescents with moderate-to-severe AD. METHODS:Data were used from a phase 3, randomized, double-blind, placebo-controlled trial of dupilumab in adolescents (aged ≥ 12 to < 18 years) with moderate-to-severe AD. Anchor-based methods were employed using the mean change in POEM and CDLQI scores from baseline to week 16 linked with a 1-point improvement in Patient Global Assessment of Disease (PGAD), a score of "a little better" on the Patient Global Assessment of Treatment effect (PGAT), a 50-74% improvement from baseline in the Eczema Area and Severity Index (EASI-50-74), and a 1-point improvement in Investigator's Global Assessment (IGA) score. RESULTS:A mean change of - 7.8 and - 5.6 in the POEM score was associated with PGAD and PGAT anchors, respectively. EASI-50-74 was associated with a mean change in POEM score of - 8.2, while the IGA anchor was associated with a mean change of - 7.9 in POEM score. The mean changes in CDLQI score associated with PGAD and PGAT anchors were - 6.4 and - 6.6, respectively, while CDLQI mean scores changed by - 8.3 and - 8.0 for the EASI and IGA anchors, respectively. CONCLUSION:In adolescents (aged ≥ 12 to < 18 years) with moderate-to-severe AD, a within-person change of 6-8 points in POEM and CDLQI scores, independently, can be considered a reasonable responder threshold for clinically meaningful change in each of the two scales, respectively. TRIAL REGISTRATION:ClinicalTrials.gov Identifier: NCT03054428. FUNDING:Sanofi and Regeneron Pharmaceuticals, Inc.

Project description:BackgroundNonphysicians are expanding practice into specialty medicine. There are limited studies on patient and physician perspectives as well as safety outcomes regarding the nonphysician practice of cosmetic procedures.ObjectiveTo identify the patient (consumer) and physician perspective on preferences, adverse events, and outcomes following cosmetic dermatology procedures performed by physicians and nonphysicians.Materials and methodsInternet-based surveys were administered to consumers of cosmetic procedures and physician members of the American Society for Dermatologic Surgery. Descriptive statistics and graphical methods were used to assess responses. Comparisons between groups were based on contingency chi-square analyses and Fisher exact tests.ResultsTwo thousand one hundred sixteen commenced the patient survey with 401 having had a cosmetic procedure performed. Fifty adverse events were reported. A higher number of burns and discoloration occurred in the nonphysician-treated group and took place more often in a spa setting. Individuals seeing nonphysicians cited motivating factors such as level of licensure (type) of nonphysician, a referral from a friend, price, and the location of the practitioner. Improper technique by the nonphysician was cited most as a reason for the adverse event. Both groups agree that more regulation should be placed on who can perform cosmetic procedures. Recall bias associated with survey data.ConclusionPatients treated by nonphysicians experienced more burns and discoloration compared with physicians, and they are encountering these nonphysicians outside a traditional medical office, which are important from a patient safety and regulatory standpoint. Motivating factors for patients seeking cosmetic procedures may also factor into the choice of provider.Key pointsBoth patients and physicians think more regulation should be in place on who can perform cosmetic procedures. More adverse events such as burns and discolorations occurred with patients seeing nonphysicians compared with those seeing physicians. In addition, for those seeing nonphysicians, a majority of these encounters took place in spa settings. Patient safety is of utmost concern when it comes to elective cosmetic medical procedures. More adverse events and encounters occurring outside traditional medical settings when nonphysicians performed these procedures call into question the required training and oversight needed for such procedures.