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Completed

NCT Number: NCT07772050

IV Bevacizumab Treatment of Patients With Hereditary Hemorrhagic Telangiectasia (HHT) or Rendu-Osler (RO) Disease: A Retrospective Analysis of French Cohort Data From 2009 to 2024

Rendu-Osler disease (RO), also known as Hereditary Haemorrhagic Telangiectasia (HHT), is an autosomal dominant genetic disorder. Its pathophysiology is associated with an imbalance in angiogenic balance. The resulting epistaxis and digestive bleeding can be severe, requiring repeated transfusions in 5% of patients. In addition, some patients with HHT may present with liver damage, leading to high-flow heart failure.

Vascular Endothelial Growth Factor (VEGF) plays a role in the pathophysiology of Rendu-Osler disease, leading to the use of a recombinant anti-VEGF monoclonal antibody, bevacizumab. Studies conducted and promoted by HCL have demonstrated the efficacy and safety of bevacizumab in patients with HHT.

In 2014, bevacizumab was granted orphan drug designation for this disease. The 2020 international guidelines for the diagnosis and management of HHT suggest the use of systemic antiangiogenic agents in cases of epistaxis resistant to other treatments. The use of bevacizumab has been widely published in uncontrolled studies, demonstrating its efficacy in reducing epistaxis, improving anaemia, reducing transfusion dependence and improving quality of life.

In France, bevacizumab is currently prescribed outside the scope of its marketing authorization. Treatment indications are validated within the French network of expert centres and the FAVAmulti rare disease network. In other countries, access to this treatment varies widely according to local regulations, and is currently being evaluated in the European VASCERN network.

The aim of this study is to describe the use of bevacizumab in patients treated for HHT in France.

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Key information

Conditions

Hereditary Hemorrhagic Telangiectasia (HHT) Anemia Cardiac Output, High Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Heart Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Hemostatic Disorders Pathologic Processes Pathological Conditions, Signs and Symptoms Rendu Osler Disease Signs and Symptoms Telangiectasia, Hereditary Hemorrhagic Telangiectasis Vascular Diseases Vascular Malformations

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Hôpital Femme Mère Enfant

Bron, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • - Adult over 18 years of age
  • Rendu-Osler disease treated with bevacizumab prescribed for this condition
  • Treated and followed in France

Exclusion criteria

  • - Patient opposition to data use

Treatment and study plan

Real World data study.

Other

The bevacizumab population is defined as patients who received at least one bevacizumab injection. Descriptive analyses will be performed using demographic, clinical and genetic data

Primary outcomes

  1. The cumulated number of RBC (Red Blood Cell) units transfused

    Time frame: Efficacy outcomes were assessed across three consecutive 6-month periods: Period P1 (baseline), 6 months prior to the first infusion of bevacizumab ; Period P2, first 6 months following the initial induction infusion ; Period P3, the 6 months immediatel

    The number of RBC units transfused (date and number of units)

Secondary outcomes

  1. Hemoglobin values (g/L)

    Time frame: Efficacy outcomes were assessed across three consecutive 6-month periods: Period P1 (baseline), 6 months prior to the first infusion of bevacizumab ; Period P2, first 6 months following the initial induction infusion ; Period P3, the 6 months immediatel

    Hemoglobin values were collected continually, the average of all available hemoglobin values were calculated for each period, anemia was defined as an hemoglobin mean level less than 120 g/dl regardless of gender

Other outcomes

  1. The cumulated amount of iron administrated (mg)

    Time frame: Efficacy outcomes were assessed across three consecutive 6-month periods: Period P1 (baseline), 6 months prior to the first infusion of bevacizumab ; Period P2, first 6 months following the initial induction infusion ; Period P3, the 6 months immediatel

    The cumulated amount of iron administrated (mg)

  2. The HSS (Hematologic Support Score) was calculated for each period

    Time frame: Efficacy outcomes were assessed across three consecutive 6-month periods: Period P1 (baseline), 6 months prior to the first infusion of bevacizumab ; Period P2, first 6 months following the initial induction infusion ; Period P3, the 6 months immediatel

    (Additional information about the outcome measure, if needed for clarification. Limit: 600 characters) HSS is a validated composite metric integrating transfusion burden and iron therapy; HSS =(Units of RBC transfused) + (mg elemental Fe/250)

  3. In patients in group 2 (see section 'groups and interventions'), Cardiac index was measured by echocardiography

    Time frame: Baseline and 24 weeks afer Day 1 (the first bevacizumab infusion)

    (Additional information about the outcome measure, if needed for clarification. Limit: 600 characters) A cardiac index of 2.5-3.9L/min/m² was considered normal , irrespective of gender

  4. To assess the safety profile

    Time frame: From Day 1 (the first bevacizumab infusion) through treatment completion

    A predefined list of common adverse events was available in the database with the option to add newly observed adverse events. Any adverse event is discussed to assess its imputability to bevacizumab and to determine the appropriate management strategy (treatment discontinuation, continuation, or dose adjustment). Survival status was collected systematically.

Sponsors and collaborators

Lead sponsor

Hospices Civils de Lyon

Other

Registry information

Official study title

IV Bevacizumab Treatment of Patients With Hereditary Hemorrhagic Telangiectasia or Rendu-Osler Disease: A Retrospective Analysis of French Cohort Data From 2009 to 2024

Acronym: CoBé

Important dates

Study start
2009
Primary completion
2024
Study completion
2025
First posted
Aug 19, 2026
Registry last updated
Aug 19, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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