# 2/2 Pomalidomide for Bleeding in Patients with Hereditary Hemorrhagic Telangiectasia (HHT) Bleeding Data Coordinating Center

> **NIH NIH U24** · RESEARCH TRIANGLE INSTITUTE · 2021 · $394,666

## Abstract

Project Summary
Hereditary hemorrhagic telangiectasia (HHT) is an inherited disorder characterized by diffuse arteriovenous
malformations (AVMs) and telangiectasia that affect many organs. The most common manifestations of HHT
are epistaxis and GI bleeding; however AVMs that involve the lungs, brain, or liver can also lead to devastating
cardiovascular complications including congestive heart failure, pulmonary hemorrhage, and hemorrhagic
stroke. Estimates suggest a prevalence of more than 100,000 cases of HHT in the United States. In addition to
bleeding and cardiovascular manifestations, the morbidity of HHT leads to a high incidence of anxiety and
depression that greatly affects social function. There is no broadly accepted, effective therapy for HHT.
Ablation of HHT lesions via interventional approaches is commonly used but provides only transient benefit.
Medical approaches have included anti-fibrinolytic therapy, estrogen, and octreotide, among others; however,
these are not effective in most patients. A recent study demonstrated that nasal sprays containing tranexamic
acid, estriol, or bevacizumab were no more effective than placebo in treating HHT-associated epistaxis. The
current study builds on results of several small studies that suggest efficacy of thalidomide in HHT; however,
thalidomide is not available for clinical trials in the United States. Thus, we propose to study pomalidomide, a
third-generation thalidomide analog with similar anti-angiogenic activity but less toxicity than thalidomide. A
small, industry-funded pilot study by the PI has demonstrated efficacy and safety of pomalidomide in HHT, and
FDA has issued an IND to expand these observations. This resubmission proposes a randomized, placebo-
controlled study developed with support from an NHLBI U34 Clinical Trial Planning Grant and U24 Clinical
Trials Development Resource for Hematologic Disorders by a team of HHT experts, a patient advocate, and
experienced clinical trial statisticians. The same team that designed the study will conduct it, using an sIRB at
the Cleveland Clinic. We propose to (1) determine the efficacy and safety of pomalidomide in patients with
HHT and epistaxis requiring periodic iron infusion or blood transfusion, (2) determine the effect of
pomalidomide on quality of life in patients with HHT using NIH PROMIS tools and an HHT-specific
questionnaire, and (3) create a biorepository of samples from study patients for future biomarker and
mechanistic studies. This study may validate pomalidomide as a new therapeutic option and change the
paradigm for treatment of HHT.

## Key facts

- **NIH application ID:** 10122980
- **Project number:** 5U24HL140090-03
- **Recipient organization:** RESEARCH TRIANGLE INSTITUTE
- **Principal Investigator:** Sonia M Thomas
- **Activity code:** U24 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $394,666
- **Award type:** 5
- **Project period:** 2019-03-06 → 2024-02-29

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10122980

## Citation

> US National Institutes of Health, RePORTER application 10122980, 2/2 Pomalidomide for Bleeding in Patients with Hereditary Hemorrhagic Telangiectasia (HHT) Bleeding Data Coordinating Center (5U24HL140090-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10122980. Licensed CC0.

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