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Evaluation of Watchful Waiting and Tumor Behavior in Patients With Basal Cell CarcinomaAn Observational Cohort Study of 280 Basal Cell Carcinomas in 89 Patients

Educational Objective
To describe the tumor behavior of basal cell carcinomas managed with watchful waiting.
1 Credit CME
Key Points

Question  What are reasons for watchful waiting in basal cell carcinoma (BCC), and what is the tumor behavior?

Findings  In this cohort study of 280 BCCs in 89 patients in the Netherlands, patient-related factors (eg, limited life expectancy or frailty) were the most important reasons to choose watchful waiting. The minority of tumors increased in size (46.8%); of those, infiltrative/micronodular BCCs were estimated at a tumor increase of 4.46 mm in 1 year, whereas nodular, superficial, or clinical BCCs were estimated at 1.06-mm growth in 1 year.

Meaning  Watchful waiting might be an appropriate approach in patients with BCC with a limited life expectancy and asymptomatic nodular or superficial BCCs.

Abstract

Importance  Few studies have examined watchful waiting (WW) in patients with basal cell carcinoma (BCC), although this approach might be suitable in patients who might not live long enough to benefit from treatment.

Objective  To evaluate reasons for WW and to document the natural course of BCC in patients who chose WW and reasons to initiate later treatment.

Design, Setting, and Participants  An observational cohort study was performed at a single institution between January 2018 and November 2020 studying patients with 1 or more untreated BCC for 3 months or longer.

Exposures  Watchful waiting was chosen by patients and proxies regardless of this study.

Main Outcome and Measures  The reasons for WW and treatment were extracted from patient files and were categorized for analyses. Linear mixed models were used to estimate tumor growth and identify covariates associated with tumor growth.

Results  Watchful waiting was chosen for 280 BCCs in 89 patients (47 men [53%] and 42 women [47%]), with a median (interquartile range [IQR]) follow-up of 9 (4-15) months. The median (IQR) age of the included patients was 83 (73-88) years. Patient-related factors or preferences (ie, prioritizations of comorbidities, severe frailty, or limited life expectancy) were reasons to initiate WW in 74 (83%) patients, followed by tumor-related factors (n = 49; 55%). Treatment-related and circumstantial reasons were important for 35% and 46% of the patients, respectively. The minority of tumors increased in size (47%). Tumor growth was associated with BCC subtype (odds ratio, 3.35; 95% CI, 1.47-7.96; P = .005), but not with initial tumor size and location. The estimated tumor diameter increase was 4.46 mm (80% prediction interval, 1.42 to 7.46 mm) in 1 year for BCCs containing at least an infiltrative/micronodular component and 1.06 mm (80% prediction interval, −1.79 to 4.28 mm) for the remaining BCCs (only nodular/superficial component/clinical diagnosis). Most common reasons to initiate treatment were tumor burden or potential tumor burden, resolved reason(s) for WW, and reevaluation of patient-related factors.

Conclusions and Relevance  In this cohort study of patients with BCC, WW was an appropriate approach in several patients, especially those with asymptomatic nodular or superficial BCCs and a limited life expectancy. Patients should be followed up regularly to determine whether a WW approach is still suitable and whether patients still prefer WW and to reconsider consequences of treatment and refraining from treatment.

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CME Disclosure Statement: Unless noted, all individuals in control of content reported no relevant financial relationships. If applicable, all relevant financial relationships have been mitigated.

Article Information

Accepted for Publication: July 4, 2021.

Published Online: September 8, 2021. doi:10.1001/jamadermatol.2021.3020

Corresponding Author: Marieke E. C. van Winden, MD, MSc, Department of Dermatology, Radboud University Medical Center, PO Box 9101, 6500 HB Nijmegen, the Netherlands (marieke.vanwinden@radboudumc.nl).

Author Contributions: Drs van Winden and Bronkhorst had full access to all of the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis.

Concept and design: van Winden, Hetterschijt, van de Kerkhof, de Jong, Lubeek.

Acquisition, analysis, or interpretation of data: All authors.

Drafting of the manuscript: van Winden, Bronkhorst, van de Kerkhof, Lubeek.

Critical revision of the manuscript for important intellectual content: Hetterschijt, Bronkhorst, van de Kerkhof, de Jong, Lubeek.

Statistical analysis: van Winden, Bronkhorst.

Obtained funding: van de Kerkhof, Lubeek.

Administrative, technical, or material support: van Winden, Hetterschijt, de Jong.

Supervision: van de Kerkhof, de Jong, Lubeek.

Conflict of Interest Disclosures: Dr de Jong reported receiving research grants from AbbVie, Novartis, Janssen Pharmaceuticals, Leo Pharma, and UCB for research on psoriasis and serving as a consultant and/or paid speaker for and/or participated in research sponsored by companies that manufacture drugs used for the treatment of psoriasis, including AbbVie, Almirall, Janssen Pharmaceuticals, Novartis, Lilly, Celgene, Leo Pharma, Sanofi, and UCB outside the submitted work; all funding goes to the independent research fund of the Department of Dermatology of Radboud University Medical Center, Nijmegen, the Netherlands. Dr Lubeek reported serving as a consultant/paid speaker for Sun Pharma and Sanofi Genzyme outside the submitted work; all financial compensations were paid to the independent research fund of the Department of Dermatology of the Radboud University Medical Center, Nijmegen, the Netherlands. No other disclosures were reported.

Additional Contributions: We thank the patients or their family members for granting permission to publish this information. We also thank all physicians who included patients for this study or who participated in the pilot study.

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