Treatment of Advanced Basal Cell Carcinoma with Hedgehog Pathway Inhibitors: A Multidisciplinary Expert Meeting

Giorgi, Vincenzo De; Scarfì, Federica; Trane, Luciana; Silvestri, Flavia; Venturi, Federico; Zuccaro, Biancamaria; Spinelli, Giuseppe; Scoccianti, Silvia; Rosa, Francesco De; Dika, Emi; Longo, Caterina

doi:10.3390/cancers13225706

Cited by 9 publications

(6 citation statements)

References 32 publications

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“…The antagonistic effect on Hedgehog signalling is a major treatment opportunity for patients with locally advanced, metastatic BCCs as well as patients with NBCCS who have a high risk of developing numerous BCCs throughout their lives 7–9,13 . Vismodegib and Sonidegibare both indicated for this peculiar subset of patients.…”

Section: Discussionmentioning

confidence: 99%

“…Although most BCCs are surgically treated with total resection, some of the lesions may proceed to a locally advanced or metastatic stage. Systemic treatment with a hedgehog inhibitors (HHIs) such as Vismodegib or Sonidegib is indicated in this population 7–9 . The chemical structures of Vismodegib and Sonidegib are different as they bind different residues of the same functional pocket, but they both suppress hedgehog signalling through selective inhibition of smoothened.…”

Section: Introductionmentioning

confidence: 99%

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Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome)

Venturi

Trane²,

Silvestri

et al. 2023

Skin Health and Disease

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Nevoid basal cell carcinoma syndrome (NBCCS, Gorlin syndrome) is a rare genetic condition characterized by the early development of numerous cutaneous basal cell carcinomas (BCCs). Although most BCCs are surgically treated with total resection, some of the lesions may proceed to a locally advanced or metastatic stage. Systemic treatment with a hedgehog inhibitors (HHIs) such as Vismodegib or Sonidegib is indicated in this population. We report cases of two patients with confirmed diagnoses of NBCCS. Both patients had undergone multiple surgical excisions and had been treated with oral Vismodegib 150 mg/day for a locally advanced tumour. They both discontinued the therapy due to its specific adverse effects (AEs) and are now being treated with oral Sonidegib, which has had better tolerability and a complete response. The aims of this report was to demonstrate the efficacy of HHI treatment with Sonidegib in patients with NCBBS who had previously treated with Vismodegib but discontinued it because of its specific AEs. Our experience in two patients shows that Sonidegib can be considered in Gorlin patients intolerant but responding to Vismodegib.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome)

Venturi

Trane²,

Silvestri

et al. 2023

Skin Health and Disease

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“…In contrast, therapy with HPI allows one to look at the target therapy from a neoadjuvant point of view in cases where the response is particularly good and fast and in patients with a high risk of withdrawal. 12,13 In this context, attention to the quality of life of patients via the proposed scores can play an important role and can act as a sentinel for withdrawal. Here, the presence of a caregivers is fundamental to improve the situation.…”

Section: Discussionmentioning

confidence: 99%

The importance of caregiving in patients with advanced basal cell carcinoma in treatment with hedgehog-pathway inhibitors: an observational prospective study

Trane¹,

Silvestri

Venturi

et al. 2022

Preprint

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Background Oral target therapy with hedgehog-pathway inhibitors has revolutionized the standard of care for patients with advanced basal cell carcinoma. These patients are frail and elderly patients with various comorbidities and on pharmacological polytherapy. This scenario requires the clinician to manage the adverse events that can have a significant impact on therapeutic adherence. Objectives We evaluated the impact of caregivers in the treatment of patients with advanced BCC under treatment in terms of continuation of therapy over time. Methods All patients included in this observational prospective study have histologically confirmed metastatic or locally advanced basal cell carcinoma and were treated with hedgehog-pathway inhibitors from January 2016 to December 2021 at the Department of Dermatology at the University of Florence, Italy. The collected patient data included age, sex, BCC site and extension, number of cycles, dose, duration and tolerability of the therapy, marital status (single, divorced, married/live-in, widow/widower), and information such as living with someone, and the presence of any caregivers. Results Of the 34 patients included, 33 had a LaBCC and 1 metastatic BCC. There were 11 females (32.4%) and 23 males (67.6%). Among Basal Cell Carcinoma patients treated with hedgehog-pathway inhibitors, patients who were married or lived with a care-giver could better tolerate the therapy relative to single patients who live alone. Indeed, married/live-in patients and/or those with an adequate caregiver experienced greater therapeutic adherence and tolerated adverse events better. Conclusions Given the greater therapeutic adherence of married/live-in patients whose caregiver is the partner, it is essential to consider patient’s marital status. It is advisable to involve the caregiver upon enrollment. There should be a training discussion on the various possible adverse events and the best way to mitigate them. Success in therapy is linked not only to an informed patient but also to a trained caregiver.

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“…Identifying biomarkers of resistance or response would be useful to target HPIs to patients most likely to benefit. 40 …”

Section: Case Reportsmentioning

confidence: 99%

Experience with sonidegib in patients with advanced basal cell carcinoma: case reports

Puig¹,

Serra‐Guillén²,

Pérez‐Pastor³

et al. 2022

DIC

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Sonidegib is a Hedgehog signalling pathway inhibitor approved for use in patients with advanced basal cell carcinoma (BCC) not eligible for surgery or radiotherapy. This report describes clinical experience with sonidegib in two patients with locally advanced BCC (one with a tumour adjacent to the right eye and the other with a tumour associated with the left ear) and in one patient with Gorlin syndrome. Two of the patients had recurrent and intractable tumours. Treatment with sonidegib 200 mg/day led to remission in both patients with locally advanced BCC within 7 months and to a reduction in the size and number of lesions after 4 months in the patient with Gorlin syndrome. Adverse effects reported in these patients were cramps, alopecia, ageusia and weight loss, all of which were mild and consistent with the known toxicity profile for sonidegib. Sonidegib has an important role to play in the effective treatment of challenging cases of advanced BCC. In parallel, a need remains to improve management protocols for patients with advanced BCC, particularly through earlier intervention and a multidisciplinary team approach.

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Treatment of Advanced Basal Cell Carcinoma with Hedgehog Pathway Inhibitors: A Multidisciplinary Expert Meeting

Cited by 9 publications

References 32 publications

Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome)

Tolerance of sonidegib after intolerance of vismodegib—Experience in two patients with nevoid basal cell carcinoma syndrome (Gorlin syndrome)

The importance of caregiving in patients with advanced basal cell carcinoma in treatment with hedgehog-pathway inhibitors: an observational prospective study

Experience with sonidegib in patients with advanced basal cell carcinoma: case reports

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