Key clinical trials of systemic antiangiogenic therapies for the management of HHT
| Drug . | Trial . | Type . | n . | Status or major findings . |
|---|---|---|---|---|
| Intravenous bevacizumab | ||||
| NCT04404881 | Single-arm trial | 33 | Recruiting | |
| Dupuis-Girod19 | RCT | 24 | ↔ Transfusion and ↑Hgb, QOL | |
| Al-Samkari20 | Retrospective | 238 | ↓ESS, transfusion and ↑Hgb | |
| Vazquez27 | Retrospective | 20 | ↑Hgb and ↓transfusion | |
| Al-Samkari28 | Retrospective | 13 | ↓Transfusion, epistaxis and ↑Hgb | |
| Guilhem29 | Retrospective | 46 | Clinical improvement in 74% | |
| Iyer30 | Retrospective | 34 | ↓ESS, transfusion | |
| Dupuis-Girod21 | Single-arm trial | 25 | ↓Cardiac index, epistaxis, and ↑QOL | |
| Pazopanib | ||||
| NCT03850964 | RCT | 70 | Recruiting | |
| Parambil24 | Retrospective | 13 | ↓Transfusion, ESS and ↑Hgb | |
| Faughnan31 | Single-arm trial | 7 | ↓Epistaxis and ↑Hgb, QOL | |
| Pomalidomide | ||||
| Al-Samkari23 | RCT | 144 | ↓ESS and ↑QOL | |
| Thalidomide | ||||
| Invernizzi32 | Single-arm trial | 31 | ↓ESS, transfusion and ↑Hgb | |
| Tacrolimus | ||||
| NCT04646356 | Single-arm trial | 30 | Active not recruiting | |
| Alvarez-Hernandez26 | Single-arm trial | 11 | ↓ESS and ↑Hgb | |
| Nintedanib | ||||
| NCT04976036 | RCT | 48 | Recruiting | |
| NCT03954782 | RCT | 61 | Completed | |
| VAD044 | ||||
| NCT05406362 | RCT | 80 | Recruiting | |
| Sirolimus | ||||
| NCT05269849 | Single-arm trial | 10 | Recruiting |
| Drug . | Trial . | Type . | n . | Status or major findings . |
|---|---|---|---|---|
| Intravenous bevacizumab | ||||
| NCT04404881 | Single-arm trial | 33 | Recruiting | |
| Dupuis-Girod19 | RCT | 24 | ↔ Transfusion and ↑Hgb, QOL | |
| Al-Samkari20 | Retrospective | 238 | ↓ESS, transfusion and ↑Hgb | |
| Vazquez27 | Retrospective | 20 | ↑Hgb and ↓transfusion | |
| Al-Samkari28 | Retrospective | 13 | ↓Transfusion, epistaxis and ↑Hgb | |
| Guilhem29 | Retrospective | 46 | Clinical improvement in 74% | |
| Iyer30 | Retrospective | 34 | ↓ESS, transfusion | |
| Dupuis-Girod21 | Single-arm trial | 25 | ↓Cardiac index, epistaxis, and ↑QOL | |
| Pazopanib | ||||
| NCT03850964 | RCT | 70 | Recruiting | |
| Parambil24 | Retrospective | 13 | ↓Transfusion, ESS and ↑Hgb | |
| Faughnan31 | Single-arm trial | 7 | ↓Epistaxis and ↑Hgb, QOL | |
| Pomalidomide | ||||
| Al-Samkari23 | RCT | 144 | ↓ESS and ↑QOL | |
| Thalidomide | ||||
| Invernizzi32 | Single-arm trial | 31 | ↓ESS, transfusion and ↑Hgb | |
| Tacrolimus | ||||
| NCT04646356 | Single-arm trial | 30 | Active not recruiting | |
| Alvarez-Hernandez26 | Single-arm trial | 11 | ↓ESS and ↑Hgb | |
| Nintedanib | ||||
| NCT04976036 | RCT | 48 | Recruiting | |
| NCT03954782 | RCT | 61 | Completed | |
| VAD044 | ||||
| NCT05406362 | RCT | 80 | Recruiting | |
| Sirolimus | ||||
| NCT05269849 | Single-arm trial | 10 | Recruiting |
ESS, epistaxis severity score; Hgb, hemoglobin, transfusion, red blood cell transfusion; QOL, quality of life; RCT, placebo-controlled randomized controlled trial.