Socchi Floriane, Bigorre Michele, Normandin Marion, Captier Guillaume, Bessis Didier, Mondain Michel, Blanchet Catherine, Akkari Mohamed, Amedro Pascal, Gavotto Arthur
Department of Paediatric and Congenital Cardiology, M3C Regional Reference Centre, Clinical Investigation Centre, Montpellier University Hospital, Montpellier, France.
Department of Paediatric Plastic Surgery, Montpellier University Hospital, Montpellier, France.
Br J Clin Pharmacol. 2021 Apr;87(4):1970-1980. doi: 10.1111/bcp.14593. Epub 2020 Nov 16.
Infantile haemangioma (IH) is the most common benign tumour in children. Since 2014, propranolol has become the first-choice therapy and currently Hemangiol is the only approved drug for complicated haemangioma. This post-marketing study reports the use of Hemangiol for IH in paediatric practice.
From January 2014 to November 2018, 94 children (median age 4 [0; 21] months; 75% female) treated with Hemangiol for proliferative IH were enrolled in the study. The systematic paediatric cardiology consultation never contraindicated beta-blockers. Two Hemangiol initiation protocols were used: a conventional ambulatory 3-week titration phase protocol (n = 76, 80.9%), and a rapid initiation protocol with a 48-hour dose escalation in conventional hospitalization for severe proliferative or ulcerated IH (n = 18, 19.1%). In both protocols, the haemodynamic tolerance was good. The mean maintenance dose of Hemangiol was 2.7 ± 0.8 mg/kg/day, with a median treatment duration of 7 [1.5; 19] months. Adverse events (AEs) have been found in 25 (26,6%) patients, including 8 (8.5%) patients with serious AEs (uncontrolled bronchial hyperreactivity, n = 5; serious hypoglycaemia, n = 3). Some patients had one or more AEs, a total of 24 nonserious AEs was reported in 19 patients (sleep disturbances, n = 9; respiratory disorders, n = 5; digestive disorders, n = 6). No cardiac adverse event was reported.
This post-marketing surveillance drug study supports the good tolerance of Hemangiol in children with IH. A rapid initiation protocol is of interest when treatment is urgent. The pretherapeutic paediatric cardiology consultation should not be systematic but only indicated for specific patients. CLINICALTRIALS.GOV: NCT04105517.
婴儿血管瘤（IH）是儿童最常见的良性肿瘤。自2014年以来，普萘洛尔已成为首选治疗药物，目前Hemangiol是唯一获批用于复杂性血管瘤的药物。这项上市后研究报告了Hemangiol在儿科实践中用于婴儿血管瘤的情况。
2014年1月至2018年11月，94例接受Hemangiol治疗增殖期婴儿血管瘤的儿童（中位年龄4[0；21]个月；75%为女性）纳入本研究。系统性儿科心脏病学会诊从未对β受体阻滞剂提出禁忌。采用了两种Hemangiol起始方案：传统门诊3周滴定阶段方案（n = 76，80.9%），以及针对严重增殖期或溃疡型婴儿血管瘤在传统住院治疗中48小时剂量递增的快速起始方案（n = 18，19.1%）。在两种方案中，血流动力学耐受性均良好。Hemangiol的平均维持剂量为2.7±0.8mg/kg/天，中位治疗持续时间为7[1.5；19]个月。25例（26.6%）患者出现不良事件（AE），其中8例（8.5%）患者出现严重不良事件（未控制的支气管高反应性，n = 5；严重低血糖，n = 3）。一些患者出现一种或多种不良事件，19例患者共报告24例非严重不良事件（睡眠障碍，n = 9；呼吸系统疾病，n = 5；消化系统疾病，n = 6）。未报告心脏不良事件。
这项上市后监测药物研究支持Hemangiol在婴儿血管瘤儿童中具有良好的耐受性。在治疗紧急时，快速起始方案很有意义。治疗前的儿科心脏病学会诊不应是常规的，而仅适用于特定患者。临床试验注册库：NCT04105517。
