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McCrae, K., Swaidani, S., Samour, M., Silver, B., Parambil, J., & Thomas, S. (2019). Pomalidomide in HHT: Results of a pilot study. Angiogenesis, 22(4), 624. https://doi.org/10.1007/s10456-019-09686-w
Oral Communications SESSION 2: ENG/ALK1 SIGNALING Poster Presentations: Drug Treatments of HHT
Objective: A broader therapeutic portfolio is needed for HHT. Based on studies demonstrating efficacy of thalidomide, we examined the efficacy and safety of pomalidomide in a single-arm pilot study.
Methods: Eligible patients had 1) GI bleeding requiring transfusion of C 4 units PRBC or 4 iron infusions, or 2) epistaxis with epistaxis severity score (ESS) C 4 requiring C 2 units PRBC or 500 mg intravenous iron, each within the preceding 4 months. The primary endpoint was a 50% reduction in parenteral iron or transfusion. The secondary endpoint was to define the effect of pomalidomide on the ESS. Treatment was initiated with 1 mg, increasing to 5 mg of pomalidomide/daily, which was continued for 4 months then tapered.
Results: Nine patients provided consent. One was not treated due to an intervening cardiac event. Two developed a drug-related AE (rash) within 3 weeks of treatment and were removed, thus we report on 6 patients. Two patients were on study for only 5 months and removed for non-drug-related AEs; both had primarily GI bleeding. One of these met the primary endpoint while the other did not; however, this patient significantly reduced the ESS. The remaining four patients completed the study and all met the primary endpoint and demonstrated significant reductions in the ESS. Decreased levels of plasma MMPs and/or HB-EGF were observed in responders.
Conclusions: This pilot study demonstrates safety and suggests efficacy of pomalidomide in HHT, and provides support for a randomized, placebo-controlled study that will open August, 2019. *This study was supported by Celgene, Summit NJ.