Vismodegib
證據等級: L4 | 預測適應症: 10 個
## 藥師評估報告
Vismodegib：從基底細胞癌到著色性乾皮症
一句話總結
Vismodegib 原本用於治療轉移性基底細胞癌。 TxGNN 模型預測它可能對**著色性乾皮症 (Xeroderma Pigmentosum)** 有效， 目前有 **5 篇文獻**支持這個方向。
快速總覽
| 項目 | 內容 |
|---|---|
| 原適應症 | 治療轉移性基底細胞癌，或不適合接受手術或放射線治療之局部晚期基底細胞癌成人病患。 |
| 預測新適應症 | basal cell carcinoma、medulloblastoma with extensive nodularity、sebaceous adenocarcinoma、sweat gland cancer、xeroderma pigmentosum、skin carcinoma、annular epidermolytic ichthyosis、epidermolysis bullosa simplex with mottled pigmentation、prostate cancer/brain cancer susceptibility、Brenner tumor |
| TxGNN 預測分數 | 99.91% |
| 證據等級 | L4 |
| 台灣上市 | ✓ 已上市 |
| 許可證數 | 2 張 |
| 建議決策 | Research Question |
預測適應症詳細分析
1. medulloblastoma with extensive nodularity L5 99.93% 主要分析
為什麼這個預測合理？
目前缺乏詳細的作用機轉資料。根據已知資訊，Vismodegib 是 Hedgehog 信號通路抑制劑，
其成分在基底細胞癌中的療效已被證實，機轉上可能適用於著色性乾皮症患者的基底細胞癌。
</details>2. xeroderma pigmentosum L4 99.91%
相關文獻（5 篇）
| PMID | 年份 | 類型 | 期刊 | 主要發現 |
|---|---|---|---|---|
| 36921168 | 2023 | Article | Revista paulista de pediatria | Xeroderma pigmentosum: case report. |
| 35283513 | 2021 | Article | Indian journal of dermatology | Current Therapeutic Strategies of Xeroderma Pigmentosum. |
| 28297142 | 2017 | Article | Pediatric dermatology | Vismodegib Therapy for Basal Cell Carcinoma in an 8-Year-Old Chinese Boy with Xe... |
| 30178564 | 2018 | Article | Pediatric dermatology | Use of vismodegib for the treatment of multiple basal cell carcinomas in a patie... |
| 33901791 | 2021 | Article | European journal of cancer (Ox | Combination of targeted therapy and immune checkpoint blocker in a patient with ... |
3. annular epidermolytic ichthyosis L5 99.87%
目前尚無針對此適應症的專門臨床研究。此為 TxGNN 模型預測結果，需進一步驗證。
4. epidermolysis bullosa simplex with mottled pigmentation L5 99.86%
目前尚無針對此適應症的專門臨床研究。此為 TxGNN 模型預測結果，需進一步驗證。
5. prostate cancer/brain cancer susceptibility L5 99.85%
目前尚無針對此適應症的專門臨床研究。此為 TxGNN 模型預測結果，需進一步驗證。
6. Brenner tumor L5 99.83%
目前尚無針對此適應症的專門臨床研究。此為 TxGNN 模型預測結果，需進一步驗證。
7. cutaneous adenocystic carcinoma L5 99.83%
目前尚無針對此適應症的專門臨床研究。此為 TxGNN 模型預測結果，需進一步驗證。
8. prostate leiomyoma L5 99.83%
目前尚無針對此適應症的專門臨床研究。此為 TxGNN 模型預測結果，需進一步驗證。
9. skin cancer L2 99.82%
臨床試驗（23 項）
| 試驗編號 | 階段 | 狀態 | 人數 | 主要發現 |
|---|---|---|---|---|
| NCT03035188 | PHASE2 | COMPLETED | 40 | A Phase II, Single-armed, Multicenter Trial of Neoadjuvant Vismodegib in Patient... |
| NCT01815840 | PHASE2 | COMPLETED | 229 | A Randomized, Double-blinded, Regimen-controlled, Phase II, Multicenter Study to... |
| NCT02690948 | PHASE1, PHASE2 | COMPLETED | 16 | A Phase 1b Open Label Study of Pembrolizumab for Unresectable or Metastatic Basa... |
| NCT03878524 | PHASE1 | TERMINATED | 2 | Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART)... |
| NCT01108094 | PHASE2 | COMPLETED | 29 | Pilot Biomarker Trial to Evaluate the Efficacy of Itraconazole in Patients With ... |
| NCT01631331 | EARLY_PHASE1 | COMPLETED | 15 | A Pilot Study to Investigate the Off Label Use of Vismodegib as an Adjuvant to S... |
| NCT01367665 | PHASE2 | COMPLETED | 1232 | A Single Arm, Open-label, Phase II, Multicentre Study, to Assess the Safety of V... |
| NCT05463757 | N/A | RECRUITING | 80 | Registration of Oral Hedgehog Inhibitors Vismodegib and Sonidegib in the Treatme... |
| NCT01700049 | PHASE2 | COMPLETED | 28 | ML28485:Phase 2B Single-site,Open-label,Nonrandomized Study Evaluating Efficacy ... |
| NCT01543581 | PHASE2 | COMPLETED | 3 | Placebo-controlled, Double Blind Study to Assess Efficacy and Safety of Oral Vis... |
| NCT02956889 | PHASE2 | TERMINATED | 14 | A Single Arm, Phase II, Multicenter Study To Assess The Efficacy And Safety Of V... |
| NCT05651828 | EARLY_PHASE1 | RECRUITING | 34 | Adaptive Therapy of Vismodegib in Advanced Basal Cell Carcinoma |
| NCT02694224 | PHASE2 | UNKNOWN | 40 | A Randomized Phase II Pilot Study to Evaluate Safety and Efficacy of the Additio... |
| NCT05238831 | EARLY_PHASE1 | WITHDRAWN | 0 | Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART)... |
| NCT04591431 | PHASE2 | UNKNOWN | 400 | The Rome Trial From Histology to Target: the Road to Personalize Target Therapy ... |
| NCT01835626 | PHASE2 | COMPLETED | 24 | A Phase II Study of Radiation Therapy and Vismodegib, for the Treatment of Local... |
| NCT01071564 | PHASE1 | TERMINATED | 13 | A Phase I Study of the Hedgehog Smoothened Antagonist GDC-0449 (NSC # 747691) Pl... |
| NCT00607724 | PHASE1 | COMPLETED | 68 | An Open-Label, Phase I Study of Systemic Hedgehog Pathway Antagonist, GDC-0449, ... |
| NCT02366312 | PHASE2 | COMPLETED | 2 | A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients... |
| NCT02465060 | PHASE2 | ACTIVE_NOT_RECRUITING | 6452 | Molecular Analysis for Therapy Choice (MATCH) |
| NCT06357988 | PHASE2 | ACTIVE_NOT_RECRUITING | 35 | MATCH Treatment Subprotocol T: GDC-0449 (Vismodegib) in Patients With Tumors (Ex... |
| NCT01898598 | PHASE2 | TERMINATED | 18 | A Randomized, Double-Blind, Placebo-Controlled, Phase II Study to Assess the Eff... |
| NCT02337517 | PHASE2 | TERMINATED | 6 | Pilot Study for the Treatment of Steroid-Refractory Sclerodermatous Chronic Graf... |
相關文獻（20 篇）
| PMID | 年份 | 類型 | 期刊 | 主要發現 |
|---|---|---|---|---|
| 37604067 | 2023 | Article | European journal of cancer (Ox | European consensus-based interdisciplinary guideline for diagnosis and treatment... |
| 22670903 | 2012 | Article | The New England journal of med | Efficacy and safety of vismodegib in advanced basal-cell carcinoma. |
| 31990414 | 2020 | Article | Journal of the European Academ | Sonidegib and vismodegib in the treatment of patients with locally advanced basa... |
| 26950094 | 2016 | Article | Nature genetics | Genomic analysis identifies new drivers and progression pathways in skin basal c... |
| 27436804 | 2016 | Article | Actas dermo-sifiliograficas | Resistance of Nonmelanoma Skin Cancer to Nonsurgical Treatments. Part II: Photod... |
| 32759706 | 2020 | Article | International journal of molec | Basal Cell Carcinoma: A Comprehensive Review. |
| 26833519 | 2016 | Article | Clinical medicine (London, Eng | Non-melanoma skin cancer. |
| 31288208 | 2019 | Article | European journal of cancer (Ox | Diagnosis and treatment of basal cell carcinoma: European consensus-based interd... |
| 38067165 | 2023 | Article | Cells | Basosquamous Carcinoma: Comprehensive Clinical and Histopathological Aspects, No... |
| 34000246 | 2021 | Article | The Lancet. Oncology | Cemiplimab in locally advanced basal cell carcinoma after hedgehog inhibitor the... |
| 29331385 | 2018 | Article | Journal of the American Academ | Guidelines of care for the management of basal cell carcinoma. |
| 31466584 | 2019 | Article | Dermatologic clinics | Nonsurgical Treatments for Nonmelanoma Skin Cancer. |
| 24756807 | 2014 | Article | Recent results in cancer resea | Vismodegib. |
| 30477683 | 2018 | Article | Annales de dermatologie et de | [Not Available]. |
| 35687691 | 2022 | Article | Science advances | Single-cell analysis of human basal cell carcinoma reveals novel regulators of t... |
| 27436800 | 2016 | Article | Actas dermo-sifiliograficas | Resistance of Nonmelanoma Skin Cancer to Nonsurgical Treatments. Part I: Topical... |
| 35697404 | 2022 | Article | Actas dermo-sifiliograficas | Vismodegib and Sonidegib in Locally Advanced and Metastatic Basal Cell Carcinoma... |
| 24857041 | 2014 | Article | Expert opinion on drug discove | Discovery and preclinical development of vismodegib. |
| 26566923 | 2016 | Article | International journal of derma | Oral therapy for nonmelanoma skin cancer in patients with advanced disease and l... |
| 30045105 | 2019 | Article | Dermatologic surgery : officia | Emerging Nonsurgical Therapies for Locally Advanced and Metastatic Nonmelanoma S... |
10. benign neoplasm of sweat gland L5 99.82%
目前尚無針對此適應症的專門臨床研究。此為 TxGNN 模型預測結果，需進一步驗證。
資料來源：DDInter 2.0（原文內容請參閱該網站）
**Musculoskeletal Diseases** 🟡 Moderate
- 注意事項：Musculoskeletal adverse reactions, which may be accompanied by serum creatine kinase elevations have been reported with the use of drugs that inhibit ...
## 結論與下一步
**決策：Research Question**
**理由：**
目前有一些文獻支持 Vismodegib 在著色性乾皮症患者中的潛在應用，但缺乏臨床試驗的直接證據。
**若要推進需要：**
- 進一步的臨床試驗來評估其療效和安全性
- 詳細的藥物作用機轉資料（MOA）
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本報告僅供學術研究參考，不構成醫療建議。藥物使用請遵循醫師指示，切勿自行調整用藥。任何老藥新用決策需經過完整的臨床驗證與法規審查。
最後審核：2026-02-20 | 審核者：TwTxGNN Research Team
本報告僅供學術研究參考，不構成醫療建議。藥物使用請遵循醫師指示，切勿自行調整用藥。任何老藥新用決策需經過完整的臨床驗證與法規審查。
最後審核：2026-02-20 | 審核者：TwTxGNN Research Team
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