Ensayos clínicos

Inclusion Criteria:

1. A clinical diagnosis of HHT as defined by the Curacao criteria

2. Age > 18 years

3. Platelet count ≥ 100,000/µl

4. WBC ≥ 2,500/µl

5. INR ≤ 1.4 and normal ± 2 sec activated partial thromboplastin time (aPTT) by local
laboratory criteria (except for patients on a stable dose of warfarin or direct oral
anticoagulants)

6. Epistaxis severity score ≥ 3 measured over the preceding three months, measured at the
screening visit

7. A requirement for anemia, as determined by local laboratory normal ranges, and/or
parenteral infusion of at least 250 mg of iron or transfusion of 1 unit of blood over
the 24 weeks preceding the screening visit

8. Females of childbearing potential (FCBP) must adhere to the scheduled pregnancy
testing (once very two weeks) as required in the POMALYST REMS program. FCBP must a
negative serum pregnancy test with a sensitivity of at least 50 mIU/mL within 10 - 14
days prior to and again within 24 hours of prescribing pomalidomide and must either
commit to continued abstinence from heterosexual intercourse or begin TWO acceptable
methods of birth control, one highly effective method and one additional effective
method AT THE SAME TIME, at least 28 days before she starts taking pomalidomide. Men
must agree to use a latex condom during sexual contact with a FCBP even if they have
had a vasectomy.

9. Ability to understand and sign informed consent

10. All study participants must agree to be registered into the FDA mandated POMALYST REMS
program, and be willing and able to comply with the requirements of the POMALYST REMS
program

Exclusion Criteria:

1. Women currently breast feeding

2. Renal insufficiency, serum creatinine > 2.0 mg/dl

3. Hepatic insufficiency, bilirubin > 2.0 (or >4.0 in the setting of a prior clinical or
genetic diagnosis of Gilbert's syndrome) or transaminases > 3.0x normal

4. Prior treatment with thalidomide or other imid drugs within previous 6 months

5. Prior treatment with bevacizumab (systemic or nasal) within previous 6 weeks

6. Prior treatment with pazopanib within previous 6 weeks

7. The use of octreotide or estrogens within the previous month

8. History of prior unprovoked thromboembolism confirmed by venous ultrasound or other
imaging modalities

9. Peripheral neuropathy, confirmed by neurologic consultation

10. Known underlying hypoproliferative anemia (i.e. myelodysplasia, aplastic anemia)

11. Currently enrolled in other interventional trials

12. Known hypersensitivity to thalidomide or lenalidomide.

13. The development of erythema nodosum if characterized by a desquamating rash while
taking thalidomide or similar drugs.

14. Known SMAD-4 mutation, unless there has been a colonoscopy with normal (negative)
results, or in which the patient has had no more than 5 small (in the opinion of the
gastroenterologist) colonic polyps completely removed within the preceding 18 months

15. Anything that in the investigator's opinion is likely to interfere with completion of
the study

Eligibility last updated 6/1/22. Questions regarding updates should be directed to the study team contact.