Eight years of experience with vismodegib for advanced and multiple basal cell carcinoma patients in the Netherlands: a retrospective cohort study

Verkouteren, Babette J A; Wakkee, Marlies; Reyners, An; Nelemans, Patty J.; Aarts, Maureen J.B.; Rácz, Emőke; Terra, Jorrit B.; Devriese, Lot A.; Alers, Robert‐Jan; Doorn, Remco van; Bekkenk, Marcel W.; Reinders, Marie G H C; Mosterd, Klara

doi:10.1038/s41416-020-01220-w

Cited by 12 publications

(17 citation statements)

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“…Dose reduction or an intermittent schedule is commonly prescribed in daily practice to reduce the intensity of vismodegibinduced AEs and maintain patients on treatment, not compromising the efficacy outcomes. [4][5][6][7][8] However, if CR is the treatment goal, as shown in our study, changes in vismodegib dosing reduce the likelihood of achieving a CR with 70.8% of patients with dosing changes obtaining NCR. Intermittent dosing was indeed recently reported as a cause favouring non-response to vismodegib.…”

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confidence: 61%

“…Muscle cramps most frequently required dosage modifications in patients. 6 In conclusion, a reduced probability of achieving CR to vismodegib is associated with changes in dosing and occurrence of TRAEs, mainly muscle cramps, in addition to the high-risk histologic subtype. If CR is the goal of treatment in clinical practice, Baseline NLR Median (range) 1.9 (0.9-6.5) 2.1 (0.3-5.4) 0.625…”

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confidence: 88%

“…C. Pellegrini, 1 A. Piccerillo, 2,3 G. Spallone, 4 T. Rocco, 1 A. Ventura, 1 S. Necozione, 5 L. Bianchi, 4 K. Peris, 2,3 A. Cortellini 6,7…”

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“…Editor, Rates of Neisseria gonorrhoea including antimicrobial-resistant gonorrhoea are increasing particularly in men who have sex with men (MSM) [1][2][3][4] As a consequence of contact tracing, increasing numbers of patients are presenting as sexual contacts of gonorrhoea. [5][6][7][8] Understanding the clinical characteristics of MSM who present as contacts of gonorrhoea, particularly those who are diagnosed with gonorrhoea, will inform future strategies for gonorrhoea control. We aimed to explore the characteristics of MSM presenting as sexual contacts of gonorrhoea from our large urban sexual health clinic.…”

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confidence: 99%

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Clinical determinants of complete response to vismodegib in locally advanced basal cell carcinoma: a multicentre experience

Fargnoli

Pellegrini

Piccerillo

et al. 2021

Acad Dermatol Venereol

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Of the enrolled subjects, 71% were women and 29% were men. Based on POEM 18% had mild, 50% moderate and 22% severe AD. Based on the EASI score 42% were classified as mild, 27% as moderate and 7% as severe. Three per cent had clear skin, and 20% had almost clear skin based on EASI score. (Table 1).Thornton et al. 3 showed that the cost per retained participant in 24 studies recruited through Facebook was between $1.36 and $110. The costs in our study were in the higher end probably due to the geographic limitations and two in-clinic visits.Most of the recruited subjects had mild AD. Recruitment at hospital (outpatient clinics and wards) results in participants with moderate-to-severe disease severity. Online recruitment could be a worthy supplement to traditional recruitment to allow recruitment of participants from the entire disease severity scale.In conclusion, online patient recruitment using Facebook appears to be an effective method to recruit subjects with AD, both from a time and a cost perspective. The majority of recruited subjects were young females with mild AD.

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confidence: 61%

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confidence: 88%

“…C. Pellegrini, 1 A. Piccerillo, 2,3 G. Spallone, 4 T. Rocco, 1 A. Ventura, 1 S. Necozione, 5 L. Bianchi, 4 K. Peris, 2,3 A. Cortellini 6,7…”

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mentioning

confidence: 99%

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Clinical determinants of complete response to vismodegib in locally advanced basal cell carcinoma: a multicentre experience

Fargnoli

Pellegrini

Piccerillo

et al. 2021

Acad Dermatol Venereol

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“…Radiotherapy is relatively contraindicated in patients with BCNS, owing to the increased risk of BCCs in the irradiated area 1,38 . Hh pathway inhibitors (HPIs), such as vismodegib and sonidegib, may be indicated in advanced BCC 1,2 and are very effective in the treatment of multiple BCNS‐associated BCCs (Tables S2 and S3; see Supporting Information) 39–43 . These treatments are not a lifelong option because of side‐effects, and BCCs will recur after treatment discontinuation (Table S4; see Supporting Information) 39–46 .…”

Section: Summary Of Recommendationsmentioning

confidence: 99%

A guideline for the clinical management of basal cell naevus syndrome (Gorlin–Goltz syndrome)*

et al. 2021

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The overall objective of this guideline is to provide up-to-date, evidence-based recommendations for the diagnosis and surveillance of all symptoms in children and adults with either basal cell naevus syndrome (BCNS), a clinical suspicion of BCNS, or a parent with BCNS. In the last two groups, the guidelines should be followed until the diagnosis of BCNS can be rejected with certainty. The guideline aims to:• Update and expand on the previous guidelines by an appraisal of all relevant literature from January 2011 up to January 2021• Address important, practical, clinical questions relating to the primary guideline objective• Provide guideline recommendations • Discuss potential developments and future directions The guideline is presented as a detailed review with highlighted recommendations for practical use in the clinic by dermatologists and other healthcare professionals, including general practitioners, clinical geneticists, paediatricians, ophthalmologists, craniomaxillofacial surgeons, neurologists, cardiologists and psychologists. ExclusionsThe guideline does not cover therapeutic recommendations for (nondermatological) symptoms, as the guideline mainly focuses on screening and follow-up of symptoms. Therapeutic recommendations for basal cell carcinomas (BCCs) in general have been published in international BCC guidelines. 1,2 Stakeholder involvement and peer reviewThe guideline was developed at the Maastricht University Medical Centre (MUMC+), the Dutch BCNS expert centre accredited

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Real-World Treatment Patterns and Outcomes Among Patients with Basal Cell Carcinoma Following First-Line Hedgehog Inhibitor Discontinuation

Cowey

Chen

Aguilar

et al. 2022

Dermatol Ther (Heidelb)

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Introduction Until recently, patients discontinuing first-line (1L) hedgehog inhibitors (HHIs) for basal cell carcinoma (BCC) had few subsequent treatment options. The objective of this study was to describe the treatment journey and prognosis of patients discontinuing 1L HHI for BCC. Methods This was a retrospective cohort study of patients with BCC who discontinued 1L HHI treatment in The US Oncology Network between 1 January 2012 and 1 January 2019 (with follow-up until 1 May 2020). Two cohorts were identified: patients who initiated a second-line (2L) treatment (2L initiators), and patients with 1L progression or toxicity without pathology-confirmed complete response who did not initiate 2L treatment (2L non-initiators). Patient demographics, treatment characteristics, and outcomes are reported for each cohort. Results Among 115 patients with BCC who received 1L HHI treatment, 63.5% ( n = 73/115) discontinued 1L HHIs. Of those, 50.7% ( n = 37/73) discontinued because of documented toxicity or progression, without evidence of a complete response. We identified 4 patients who initiated 2L systemic treatment (median age 68.7 years, 100.0% female) and 15 patients who were eligible for the 2L non-initiator cohort (median age 80.2 years, 20.0% female). Median 1L HHI duration was 6.8 months (range 1.9–20.6 months) for the 2L non-initiator cohort and 8.6 months (range 6.8–42.2 months) for 2L initiators. At the end of follow-up, among 2L non-initiators (median follow-up duration 9.7 months), 40.0% were lost to follow-up, 33.3% had died, 20.0% continued observation, and 6.7% transitioned to an academic medical center or hospital; among 2L initiators (median follow-up duration 6.3 months), 50.0% were lost to follow-up, 25.0% had died, and 25.0% continued observation. Conclusions Following 1L HHI discontinuation, lack of standardized care and suboptimal outcomes were observed, including limited receipt of 2L treatment. Further studies are necessary to evaluate the impact of newer BCC treatment options.

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Eight years of experience with vismodegib for advanced and multiple basal cell carcinoma patients in the Netherlands: a retrospective cohort study

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Cited by 12 publications

References 14 publications

Clinical determinants of complete response to vismodegib in locally advanced basal cell carcinoma: a multicentre experience

Clinical determinants of complete response to vismodegib in locally advanced basal cell carcinoma: a multicentre experience

A guideline for the clinical management of basal cell naevus syndrome (Gorlin–Goltz syndrome)*

Real-World Treatment Patterns and Outcomes Among Patients with Basal Cell Carcinoma Following First-Line Hedgehog Inhibitor Discontinuation

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