Pomalidomide may become the first approved therapy for hereditary haemorrhagic telangiectasia

In the first adequately powered randomised clinical trial on hereditary haemorrhagic telangiectasia (HHT), pomalidomide reduced epistaxis and improved quality of life (QoL) compared with a placebo.

Currently, there are no FDA-approved therapies for the treatment of HHT and no adequately powered randomised controlled trials testing systemic therapies have been conducted in this population. To tackle this problem, the phase 2 PATH-HHT trial (NCT03910244) randomised 144 participants with HHT 2:1 to pomalidomide, 4 mg/day, or placebo. After 24 weeks of treatment, Prof. Keith McCrae (Cleveland Clinic, OH, USA) and colleagues analysed the change in the epistaxis severity score from baseline [1].

After 24 weeks of treatment, the drop in epistaxis severity score was significantly larger in participants treated with pomalidomide than placebo controls (difference -0.95; 95% CI -1.58 to -0.32; P=0.003; see Figure). HHT-QoL mean score changes from baseline favoured the experimental arm over the placebo arm (difference -1.4; 95% CI -2.6 to -0.3; P=0.015).

Figure: Epistaxis severity score change over time from baseline [1]



PT, post-treatment.

Neutropenia was more frequently observed in participants on pomalidomide (45% vs 10%; P<0.001) However, these cases were manageable by dose reductions, which did not appear to affect the efficacy of the treatment. Rash (36%) and constipation/diarrhoea (60%) were other common side effects in the pomalidomide arm. A tremor was observed in 8 participants in the experimental arm. “We will further investigate whether these cases were related to treatment,” stressed Prof. McCrae.

Overall, in the first adequately powered randomised clinical trial in HHT, pomalidomide was associated with decreased epistaxis severity scores and improvements in quality of life.

1. McCrae K, et al. PATH-HHT, a double-blind, randomized, placebo-controlled trial in hereditary hemorrhagic telangiectasia demonstrates that pomalidomide reduces epistaxis and improves quality of life. Abstract LBA-3, 65^th ASH Annual Meeting, 9–12 December 2023, San Diego, CA, USA.

 

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Posted on 7 February, 20247 February, 2024