Caplacizumab
證據等級: L5 | 預測適應症: 10 個
## 藥師評估報告
Caplacizumab：從後天性TTP到血小板原發釋放障礙
一句話總結
Caplacizumab 原為治療後天性血栓性血小板低下紫斑症(aTTP)的抗von Willebrand因子奈米抗體，TxGNN 預測其可能對血小板原發釋放障礙(primary release disorder of platelets)有治療潛力。
快速總覽
| 項目 | 內容 | |------|------| | 原適應症 | 後天性血栓性血小板低下紫斑症 (aTTP)，合併血漿置換術及免疫抑制劑使用 | | 預測新適應症 | primary release disorder of platelets、pseudo-von Willebrand disease、Glanzmann thrombasthenia、Scott syndrome、thrombotic thrombocytopenic purpura、bleeding diathesis due to a collagen receptor defect、hemorrhagic disorder due to a constitutional thrombocytopenia、fetal and neonatal alloimmune thrombocytopenia、hemophilia、platelet-type bleeding disorder | | TxGNN 預測分數 | 99.9998% | | 證據等級 | L5 (僅預測) | | 台灣上市 | 已上市 | | 許可證數 | 1張 (多個包裝規格) | | 建議決策 | Hold |
預測適應症詳細分析
1. primary release disorder of platelets L5 100.00% 主要分析
為什麼這個預測合理？
Caplacizumab 是一種人源化雙價奈米抗體(Nanobody)：
- 作用機轉：結合 von Willebrand 因子(vWF)的A1區域，阻斷血小板與vWF的交互作用
- 抗血栓形成：防止血小板在vWF多聚體上的異常聚集
- 高預測分數：TxGNN 給予極高分數(99.9998%)，排名第8
然而，需謹慎看待此預測：
- 血小板原發釋放障礙是血小板功能缺陷疾病，涉及血小板釋放反應異常
- Caplacizumab 作用於 vWF-血小板軸，而非直接影響血小板釋放機制
- 理論上 caplacizumab 可能加重而非改善血小板功能障礙
臨床試驗
目前無針對此特定適應症的臨床試驗登記。
2. pseudo-von Willebrand disease L5 100.00%
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。
3. Glanzmann thrombasthenia L5 100.00%
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. Scott syndrome L5 100.00%
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. thrombotic thrombocytopenic purpura L1 100.00%
臨床試驗（14 項）
| 試驗編號 | 階段 | 狀態 | 人數 | 主要發現 |
|---|---|---|---|---|
| NCT04720261 | PHASE2 | UNKNOWN | 125 | Efficacy of a Personalized Caplacizumab Regimen Based on ADAMTS13 Activity Monit... |
| NCT06291025 | NA | NOT_YET_RECRUITING | 131 | Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Witho... |
| NCT04985318 | N/A | RECRUITING | 350 | Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of Acq... |
| NCT05785468 | N/A | UNKNOWN | 1 | A Retrospective, Observational Study on the Response to Caplacizumab Treatment i... |
| NCT05468320 | PHASE3 | COMPLETED | 51 | An Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety... |
| NCT07205861 | N/A | RECRUITING | 1200 | Auto-immune Thrombotic Thrombocytopenic Purpura : Retrospective Epidemiological ... |
| NCT02553317 | PHASE3 | COMPLETED | 145 | A Phase III Double-blind, Randomized, Parallel Group, Multicenter Placebo-contro... |
| NCT05262881 | N/A | UNKNOWN | 50 | A Retrospective, Observational Study on the Response to Caplacizumab Treatment i... |
| NCT05263193 | N/A | COMPLETED | 4 | Retrospective Data Collection of Pediatric Patients With Immune-mediated Thrombo... |
| NCT06376786 | N/A | RECRUITING | 132 | Italian iTTP Registry (a Prospective Observational Study) |
| NCT01151423 | PHASE2 | COMPLETED | 75 | A Phase II, Single-blind, Randomized, Placebo-controlled Trial to Study the Effi... |
| NCT04074187 | PHASE2, PHASE3 | COMPLETED | 21 | An Open-label Multicenter Trial to Study the Efficacy and Safety of Caplacizumab... |
| NCT02878603 | PHASE3 | COMPLETED | 104 | Prospective Follow-up Study for Patients Who Completed Study ALX0681-C301 (HERCU... |
| NCT05876221 | N/A | COMPLETED | 223 | Platelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thromb... |
相關文獻（20 篇）
| PMID | 年份 | 類型 | 期刊 | 主要發現 |
|---|---|---|---|---|
| 30625070 | 2019 | Article | The New England journal of med | Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. |
| 28416507 | 2017 | Article | Blood | Thrombotic thrombocytopenic purpura. |
| 26863353 | 2016 | Article | The New England journal of med | Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura. |
| 32914526 | 2020 | Article | Journal of thrombosis and haem | ISTH guidelines for treatment of thrombotic thrombocytopenic purpura. |
| 32914582 | 2020 | Article | Journal of thrombosis and haem | ISTH guidelines for the diagnosis of thrombotic thrombocytopenic purpura. |
| 40388146 | 2025 | Article | JAMA | Immune Thrombotic Thrombocytopenic Purpura: A Review. |
| 33540569 | 2021 | Article | Journal of clinical medicine | Thrombotic Thrombocytopenic Purpura: Pathophysiology, Diagnosis, and Management. |
| 37689812 | 2023 | Article | International journal of hemat | Diagnostic and treatment guidelines for thrombotic thrombocytopenic purpura (TTP... |
| 38958481 | 2024 | Article | Blood | Thrombotic thrombocytopenic purpura: 100 years of research on Moschcowitz syndro... |
| 38838300 | 2024 | Article | Blood | Management of immune thrombotic thrombocytopenic purpura without therapeutic pla... |
| 40608084 | 2025 | Article | Intensive care medicine | Thrombotic thrombocytopenic purpura: early diagnosis and effective treatment in ... |
| 34266669 | 2022 | Article | Medicina clinica | Recommendations for the diagnosis and treatment of patients with thrombotic thro... |
| 36053773 | 2023 | Article | Blood advances | Adding caplacizumab to standard of care in thrombotic thrombocytopenic purpura: ... |
| 40533296 | 2025 | Article | Journal of thrombosis and haem | 2025 focused update of the 2020 ISTH guidelines for management of thrombotic thr... |
| 37045600 | 2023 | Article | Expert review of hematology | Efficacy and safety of caplacizumab in the treatment of thrombotic thrombocytope... |
| 40235949 | 2025 | Article | EClinicalMedicine | Caplacizumab use in immune-mediated thrombotic thrombocytopenic purpura: an inte... |
| 38874905 | 2024 | Article | Blood coagulation & fibrinolys | Efficacy and relative safety of caplacizumab in immune-mediated thrombotic throm... |
| 37476982 | 2023 | Article | International journal of techn | Effectiveness and safety of caplacizumab in acquired thrombotic thrombocytopenic... |
| 31576256 | 2019 | Article | Cureus | Clinical Efficacy and Safety Profile of Caplacizumab for Acquired Thrombotic Thr... |
| 37169697 | 2023 | Article | Transfusion and apheresis scie | Real-world data of the use and experience of caplacizumab for the treatment of a... |
6. bleeding diathesis due to a collagen receptor defect L5 100.00%
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. hemorrhagic disorder due to a constitutional thrombocytopenia L5 100.00%
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. fetal and neonatal alloimmune thrombocytopenia L5 99.99%
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. hemophilia L4 99.99%
相關文獻（20 篇）
| PMID | 年份 | 類型 | 期刊 | 主要發現 |
|---|---|---|---|---|
| 30625070 | 2019 | Article | The New England journal of med | Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. |
| 26863353 | 2016 | Article | The New England journal of med | Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura. |
| 32914526 | 2020 | Article | Journal of thrombosis and haem | ISTH guidelines for treatment of thrombotic thrombocytopenic purpura. |
| 32914582 | 2020 | Article | Journal of thrombosis and haem | ISTH guidelines for the diagnosis of thrombotic thrombocytopenic purpura. |
| 40235949 | 2025 | Article | EClinicalMedicine | Caplacizumab use in immune-mediated thrombotic thrombocytopenic purpura: an inte... |
| 40533296 | 2025 | Article | Journal of thrombosis and haem | 2025 focused update of the 2020 ISTH guidelines for management of thrombotic thr... |
| 33529333 | 2021 | Article | Blood | Redefining outcomes in immune TTP: an international working group consensus repo... |
| 36138517 | 2022 | Article | Journal of thrombosis and haem | Long-term follow-up of patients treated with caplacizumab and safety and efficac... |
| 33881463 | 2021 | Article | Blood advances | Caplacizumab prevents refractoriness and mortality in acquired thrombotic thromb... |
| 32634237 | 2020 | Article | Blood advances | ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patie... |
| 31691462 | 2020 | Article | Journal of thrombosis and haem | Efficacy and safety of open-label caplacizumab in patients with exacerbations of... |
| 39221451 | 2024 | Article | Research and practice in throm | Caplacizumab improves clinical outcomes and is well tolerated across clinically ... |
| 28445600 | 2017 | Article | Journal of thrombosis and haem | Caplacizumab reduces the frequency of major thromboembolic events, exacerbations... |
| 26688483 | 2015 | Article | The Lancet. Haematology | ISTH 2015 Congress. |
| 36696206 | 2023 | Article | Journal of thrombosis and haem | Impact of first-line use of caplacizumab on treatment outcomes in immune thrombo... |
| 38592185 | 2024 | Article | Journal of clinical medicine | Real-World Data Analysis of Patients Affected by Immune-Mediated Thrombotic Thro... |
| 39549837 | 2025 | Article | Journal of thrombosis and haem | Bortezomib for rituximab-refractory immune-mediated thrombotic thrombocytopenic ... |
| 32634236 | 2020 | Article | Blood advances | Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic... |
| 36813118 | 2023 | Article | Journal of thrombosis and haem | Autoantibodies enhance ADAMTS-13 clearance in patients with immune thrombotic th... |
| 40915571 | 2025 | Article | Journal of thrombosis and haem | Evaluation of different platelet-dependent von Willebrand factor activity assays... |
10. platelet-type bleeding disorder L2 99.99%
臨床試驗（3 項）
| 試驗編號 | 階段 | 狀態 | 人數 | 主要發現 |
|---|---|---|---|---|
| NCT05468320 | PHASE3 | COMPLETED | 51 | An Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety... |
| NCT07205861 | N/A | RECRUITING | 1200 | Auto-immune Thrombotic Thrombocytopenic Purpura : Retrospective Epidemiological ... |
| NCT06376786 | N/A | RECRUITING | 132 | Italian iTTP Registry (a Prospective Observational Study) |
台灣上市資訊
| 許可證字號 | 商品名 | 劑型 | 許可證持有者 | |-----------|--------|------|-------------| | 衛部菌疫輸字第001243號 | 可利康凍晶注射劑10毫克 (Cablivi) | 注射劑 | 賽諾菲股份有限公司 |
核准適應症：
- 適用於合併血漿置換術及免疫抑制劑，治療後天性血栓性血小板低下紫斑症(aTTP)
- 適用族群：成人及12歲以上且體重40公斤以上的青少年
安全性考量
常見副作用
- 出血事件(鼻出血、牙齦出血最常見)
- 頭痛
- 蕁麻疹
嚴重警語
- 出血風險增加
- 使用期間應避免手術及侵入性處置
- 需監測出血徵象
藥物交互作用(主要)
| 交互作用藥物 | 嚴重程度 | |-------------|---------| | 阿斯匹靈 | 重度 | | 抗凝血劑(apixaban, rivaroxaban等) | 重度 | | 血小板抑制劑 | 重度 | | Fondaparinux | 重度 | | NSAIDs | 中度 |
結論與下一步
證據等級：L5 (僅 TxGNN 預測，無臨床或藥理學支持)
建議：Hold (暫不建議)
- 血小板原發釋放障礙與 caplacizumab 的作用機轉無明顯關聯
- 抑制 vWF-血小板交互作用理論上可能惡化而非改善血小板釋放功能
- 極高的 TxGNN 分數可能反映兩者在血小板相關疾病知識圖譜中的位置接近，而非真正的治療關聯
下一步建議：
- 不建議進行臨床探索
- 若臨床遇到 aTTP 合併血小板功能障礙的病例，可收集相關數據以釐清
- 建議專注於 caplacizumab 在其他血栓性微血管病變(如非典型HUS)的潛在應用研究
特別注意：此預測可能為 TxGNN 演算法的偽陽性結果，臨床上不應依據此預測進行治療決策。
相關藥物報告
- Potassium Iodide - 證據等級 L5
- Amorolfine - 證據等級 L5
- Clobetasone - 證據等級 L5
- Cerliponase Alfa - 證據等級 L5
- Travoprost - 證據等級 L5
用 AI 幫我深入分析
想進一步了解這個藥物的老藥新用潛力嗎？點擊下方按鈕，直接開啟 AI 助手進行分析。
Claude：點擊後會複製提問並開啟新視窗，請按 Ctrl+V（Mac: Cmd+V）貼上
ChatGPT：點擊後直接開啟並自動帶入提問
引用本報告
如需引用本報告，請使用以下格式：
APA 格式：
TwTxGNN. (2026). Caplacizumab老藥新用驗證報告. https://twtxgnn.yao.care/drugs/caplacizumab/
BibTeX 格式：
@misc{twtxgnn_caplacizumab,
title = {Caplacizumab老藥新用驗證報告},
author = {TwTxGNN Team},
year = {2026},
url = {https://twtxgnn.yao.care/drugs/caplacizumab/}
}
免責聲明
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
歡迎分享您對本報告的看法、補充資訊或提出問題。討論內容會同步到 GitHub Discussions。 需要 GitHub 帳號才能留言。