PMID- 24526257 OWN - NLM STAT- MEDLINE DCOM- 20140605 LR - 20211021 IS - 2168-619X (Electronic) IS - 2168-6181 (Linking) VI - 140 IP - 4 DP - 2014 Apr TI - Propranolol vs prednisolone for symptomatic proliferating infantile hemangiomas: a randomized clinical trial. PG - 323-30 LID - 10.1001/jamaoto.2013.6723 [doi] AB - IMPORTANCE: While propranolol is touted as superior to prednisolone for treating infantile hemangiomas (IH), a randomized clinical trial (RCT) comparing the outcome and tolerability of these medications for symptomatic, proliferating IH has not been reported. OBJECTIVES: To determine if oral propranolol is more efficacious and better tolerated than prednisolone in treating symptomatic, proliferating IH and to determine the feasibility of conducting a multi-institutional, RCT comparing efficacy and tolerability of both medications. DESIGN, SETTING, AND PARTICIPANTS: Phase 2, investigator-blinded, multi-institutional RCT conducted in 3 academic vascular anomalies clinics on 19 of 44 eligible infants aged between 2 weeks and 6 months. All participating patients had symptomatic proliferating IH treated between September 1, 2010, and August 1, 2012. INTERVENTIONS: Treatment with oral propranolol vs prednisolone (2.0 mg/kg/d) until halted owing to toxic effects or clinical response. MAIN OUTCOMES AND MEASURES: Primary outcome was change in IH size after 4 months of therapy. Secondary outcomes were response rate and frequency and severity of adverse events (AEs). RESULTS: The primary outcome showed no difference in lesion size or affected skin area after 4 months of therapy: 41% and 1.32 mm2 for prednisolone vs 64% and 0.55 mm2 for propranolol (P = .12 for lesion size, and P = .56 for affected skin area). Longitudinal analyses showed a faster response in total lesion outer dimension with prednisolone (P = .03), but this advantage over time was not noted when central clearing and outer dimension were included in the analysis (P = .91). The overall frequency of AEs was similar (44 for prednisolone vs 32 for propranolol) (P = .84), but prednisolone-treated participants had more grade 3 severe AEs (11 vs 1) (P = .01), particularly growth retardation resulting in size and weight below the fifth percentile. Early study withdrawal owing to AEs occurred in 6 (75%) of 8 patients in the prednisolone group but 0 of 11 propranolol-treated participants. The mean duration of therapy was shorter for prednisolone (141 vs 265 days), reflecting the higher rate of early withdrawals. CONCLUSIONS AND RELEVANCE: Both medications show similar efficacy for reducing the area of symptomatic, proliferating IH. Although prednisolone showed a faster response rate, propranolol was better tolerated with significantly fewer severe AEs. Propranolol should be the first line of therapy for symptomatic IH unless contraindicated or unless future studies demonstrate severe AEs from propranolol. Recruiting participants for a phase 3 RCT would be difficult owing to safety profiles measured here and emerging trends favoring propranolol. TRIAL REGISTRATION clinicaltrials.gov Identifier: NCT00967226. FAU - Bauman, Nancy M AU - Bauman NM AD - Department of Otolaryngology, Children's National Medical Center, Washington, DC. FAU - McCarter, Robert J AU - McCarter RJ AD - Department of Biostatistics, Children's National Medical Center, Washington, DC. FAU - Guzzetta, Philip C AU - Guzzetta PC AD - Department of General Surgery, Children's National Medical Center, Washington, DC. FAU - Shin, Jennifer J AU - Shin JJ AD - Department of Otolaryngology, Harvard Medical School, Boston, Massachusetts. FAU - Oh, Albert K AU - Oh AK AD - Department of Plastic Surgery, Children's National Medical Center, Washington, DC. FAU - Preciado, Diego A AU - Preciado DA AD - Department of Otolaryngology, Children's National Medical Center, Washington, DC. FAU - He, Jianping AU - He J AD - Department of Biostatistics, Children's National Medical Center, Washington, DC. FAU - Greene, Elizabeth Anne AU - Greene EA AD - Department of Cardiology, Children's National Medical Center, Washington, DC. FAU - Puttgen, Katherine B AU - Puttgen KB AD - Department of Dermatology, Johns Hopkins School of Medicine, Baltimore, Maryland. LA - eng SI - ClinicalTrials.gov/NCT00967226 GR - R21 HD062959/HD/NICHD NIH HHS/United States GR - UL1RR031988/RR/NCRR NIH HHS/United States GR - R21HD062959-02/HD/NICHD NIH HHS/United States PT - Clinical Trial, Phase II PT - Comparative Study PT - Journal Article PT - Multicenter Study PT - Randomized Controlled Trial PT - Research Support, N.I.H., Extramural PL - United States TA - JAMA Otolaryngol Head Neck Surg JT - JAMA otolaryngology-- head & neck surgery JID - 101589542 RN - 0 (Antineoplastic Agents, Hormonal) RN - 0 (Vasodilator Agents) RN - 9PHQ9Y1OLM (Prednisolone) RN - 9Y8NXQ24VQ (Propranolol) SB - IM MH - Administration, Oral MH - Antineoplastic Agents, Hormonal/administration & dosage/*therapeutic use MH - Female MH - Hemangioma/*drug therapy MH - Humans MH - Infant MH - Infant, Newborn MH - Male MH - Prednisolone/administration & dosage/*therapeutic use MH - Propranolol/administration & dosage/*therapeutic use MH - Treatment Outcome MH - United States MH - Vasodilator Agents/administration & dosage/*therapeutic use EDAT- 2014/02/15 06:00 MHDA- 2014/06/06 06:00 CRDT- 2014/02/15 06:00 PHST- 2014/02/15 06:00 [entrez] PHST- 2014/02/15 06:00 [pubmed] PHST- 2014/06/06 06:00 [medline] AID - 1827974 [pii] AID - 10.1001/jamaoto.2013.6723 [doi] PST - ppublish SO - JAMA Otolaryngol Head Neck Surg. 2014 Apr;140(4):323-30. doi: 10.1001/jamaoto.2013.6723.