Pomalidomide for the Treatment of Bleeding in HHT

Open to people ages 18 years and up

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 or PTT per local laboratory designation) 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 hemoglobin assessment and 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

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 oral 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

   • • Use of these treatments is not permitted during study participation.