Real-world first-line treatment of patients with BRAFV600E-mutant metastatic colorectal cancer: the CAPSTAN CRC study.

BACKGROUND: BRAFV600E mutations occur in 8%-12% of metastatic colorectal cancer (mCRC) cases and are associated with poor survival. European guidelines recommend combination (doublet or triplet) chemotherapy plus bevacizumab in first line. However, an unmet need remains for more effective treatments for these patients. PATIENTS AND METHODS: CAPSTAN CRC is a European, retrospective, multicenter, observational study evaluating real-world treatment practices for patients with BRAFV600E-mutant mCRC treated between 1 January 2016 and 31 January 2020. The primary objective was to describe first-line treatment patterns. Secondary objectives included describing baseline demographics, mutational testing procedures, treatment effectiveness, and safety. RESULTS: In total, 255 patients (median age 66.0 years; 58.4% female) with BRAFV600E-mutant unresectable mCRC from seven countries were included. Most had right-sided tumors (52.5%) and presented with synchronous disease at diagnosis (66.4%). Chemotherapy plus targeted therapy (68.7%) was preferred at first line over chemotherapy alone (31.3%). The main first-line treatments were FOLFOX plus bevacizumab (27.1%) and FOLFOXIRI (folinic acid, 5-fluorouracil, oxaliplatin, irinotecan) with/without bevacizumab (27.1%/19.2%). Median duration of first-line treatment was 4.9 months. Overall, 52.5% received second-line treatment. Across all first-line regimens, progression-free survival (PFS) and overall survival were 6.0 [95% confidence interval (CI) 5.3-6.7] months and 12.9 (95% CI 11.6-14.1) months, respectively. Triplet plus targeted therapy was associated with more adverse events (75.0%) compared with triplet chemotherapy alone (50.0%) and doublet chemotherapy alone (36.1%). Multivariate analysis identified low body mass index and presence of three or more metastatic sites as significant prognostic factors for PFS. CONCLUSIONS: This study is, to date, the largest real-world analysis of patients with BRAFV600E-mutant mCRC, providing valuable insights into routine first-line treatment practices for these patients. The data highlight the intrinsic aggressiveness of this disease subgroup, confirming results from previous real-world studies and clinical trials, and stressing the urgent need for more effective treatment options in this setting.

Validation of the Patient-Oriented SCORing for Atopic Dermatitis tool for black skin.

BACKGROUND: SCORing for Atopic Dermatitis (SCORAD) is a tool developed by the European Task Force on Atopic Dermatitis (AD) which is used by physicians to assess AD severity during consultations with their patients. Patient-Oriented SCORAD (PO-SCORAD) is a self-assessment tool for use by patients which has been validated in a study performed in European countries. However, there is currently no adapted tool for evaluating AD severity in black skin. OBJECTIVE: To evaluate the performance of the version of the PO-SCORAD specifically adapted for black skin patients (children and adults) with AD. METHODS: In this multicenter, cross-sectional and non-interventional study, children and adults with AD were recruited during regular consultations. This international study was performed in seven sub-Saharan countries (Benin, Burkina Faso, Cameroon, Ivory Coast, Gabon, Mali and Senegal). During the consultation, AD severity was assessed by the physician using SCORAD score and by the patients or parents using PO-SCORAD. RESULTS: One hundred and thirteen patients were included, 72 children and 41 adults, mainly females (61.6%). SCORAD assessed by physicians and PO-SCORAD assessed by patients/parents were well correlated (r = 0.66, P < 0.0001). Correlation coefficients for SCORAD and PO-SCORAD subscale scores were also good, except for symptom intensity criteria. CONCLUSION: Altogether, these data indicate that PO-SCORAD for black skin correlates well with SCORAD and is therefore a valuable tool, which requires no specific level of education, for use by black skin patients with AD.

Psychometric properties of a questionnaire (HEMO-FISS-QoL) to evaluate the burden associated with haemorrhoidal disease and anal fissures.

AIM: Current questionnaires designed to evaluate the burden of haemorrhoidal disease ignore symptoms such as bleeding, pain and itching. A specific questionnaire is needed to evaluate the global impact of anal disorders on patients' daily lives. METHOD: We developed a questionnaire (HEMO-FISS-QoL) to assess the symptom burden of anal disorders and administered it to 256 patients (mean age 46.2 years; men 60.4%) with haemorrhoidal disease (67.2%), anal fissure (29.3%) or both (3.5%). Psychometric properties were evaluated by testing the acceptability, construct validity and reliability of the questionnaire. Principal components and multi-trait analyses were used to identify dimensions and to assess construct validity. Backward Cronbach alpha curves and a graded response model were used to reduce the number of items and modalities. External validity was evaluated against SF-12 and the Psychological Global Well-Being Index (PGWBI) using Spearman's correlation coefficient. RESULTS: Principal component analysis defined four dimensions: physical disorders, psychology, defaecation and sexuality. The number of questions was reduced from 38 to 23. The HEMO-FISS-QoL scores correlated well with those of the SF-12 and PGWBI (P < 0.001). Cronbach's coefficients (all > 0.7) reflected good internal reliability of the different dimensions. The total score increased with the severity of the anal disorders and with their consequences (days off work and personal spending related to the disease). CONCLUSION: The HEMO-FISS-QoL questionnaire reliably evaluates the global impact of haemorrhoids and anal fissures on patients' daily lives. This simple tool may prove useful for treatment evaluation in clinical trials and daily practice.