Since its use was first reported in the New England Journal in 2008, propranolol has become the preferred treatment for infantile haemangiomas. However, there is no consensus as to the optimum dose or duration of treatment and there is a paucity of formal trial data. The authors performed an international multi-centre double-blind placebo-controlled randomised trial. Using an ‘adaptive’ design, the efficacy of one or 3mg propranolol/kg body weight for either three or six months was compared. Out of these four possible groups one (the most efficacious) was then compared against placebo. To assess success, standard photos were taken at 24 weeks and submitted to a central panel of independent blinded investigators. Complete resolution or near-resolution at 24 weeks was classed as success whilst use of any other treatment or withdrawal from treatment was classed as failure. This was a large study – 456 infants were randomised – thus making it more than 10x larger than the largest earlier study. The interim analysis found that the highest dose and longest duration of treatment was best, and when this was compared to placebo the results were clear cut in favour of treatment: 60% vs. 4% resolution (p<0.001). Interestingly the length of treatment was important as there was little resolution after only three months treatment with either 1mg or 3mg, whilst 49% achieved resolution after six months treatment even with 1mg/kg strength (p<0.001). There were two main drawbacks with this study design: a) the most common dose used in clinical practice 2mg/kg/day was not tested but rather a higher and lower dose; b) as the design contained a placebo arm it was unethical to include severe haemangiomas and so these were excluded from the study. Though propranolol is an efficacious and safe treatment it can cause bronchospasm, bradycardia (usually within an hour of administration), hypotension and hypoglycaemia (and should therefore be given after a feed). Of the 400 or so patients receiving active treatment however, only one patient had a serious adverse event (bradycardia in the context of enterocolitis) but a single event did not reach statistical significance.
A randomised, controlled trial of oral propranolol in infantile haemangioma
Reviewed by Jonathan C P Roos
A randomized, controlled trial of oral propranolol in infantile hemangioma.
CONTRIBUTOR
Jonathan CP Roos
Harvard, Cambridge & Moorfields-trained Consultant Oculoplastic Surgeon and academic based in London at www.FaceRestoration.com. Publishes in the world’s leading medical journals and lectures internationally on aesthetics, eyelid diseases and thyroid eyes.
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