Pope Elena, Krafchik Bernice R, Macarthur Colin, Stempak Diana, Stephens Derek, Weinstein Miriam, Ho Nhung, Baruchel Sylvain
University of Toronto, Section of Dermatology, Hospital for Sick Children, 555 University Ave, Toronto, Ontario, Canada M5G 1X8.
Pediatrics. 2007 Jun;119(6):e1239-47. doi: 10.1542/peds.2006-2962. Epub 2007 May 7.
Oral systemic corticosteroids are the mainstay of treatment for problematic hemangiomas; however, current information is based on anecdotal experience and retrospective studies. We aimed to determine whether systemic steroids are efficacious in proliferating hemangioma and to compare the efficacy and safety of 2 corticosteroid treatment modalities.
Twenty patients with problematic hemangiomas of infancy were randomly assigned to either daily oral prednisolone or monthly intravenous pulses of methylprednisolone. Their clinical outcomes (improvement using a visual analog score) and adverse events were compared at 3 months from baseline and 1 year of age. Data on possible surrogate markers of angiogenesis were available for the first 3 months.
At 3 months, orally treated patients had a median visual analog score of 70 compared with 12 in the intravenous group. This response pattern was similar at the patients' first birthday: 50.0 vs -1.5. Additional treatment beyond 3 months was needed for 65% of the patients (7 in the intravenous and 6 in the oral group). Six of 8 patients with impaired vision at enrollment had an improved function at 1 year (4 patients in the intravenous group and 3 patients in the oral group). Of the 4 surrogate markers of angiogenesis measured (plasma basic fibroblast growth factor, vascular endothelial growth factor, vascular cellular adhesion molecule 1, endoglin, and urine basic fibroblast growth factor), the only 2 that decreased over time were vascular cellular adhesion molecule 1 and endoglin. Patients in the oral group had a higher rate of adverse effects, such as hypertension (18.6% vs 13.1%), abnormal cortisol (78% vs 60%), and growth retardation.
Systemic corticosteroids are efficacious in stopping the proliferation of hemangiomas. The oral corticosteroids offered more clinical and biological benefit than the pulse steroids with higher risk of adverse effects.
口服全身性皮质类固醇是治疗有问题的血管瘤的主要方法；然而，目前的信息基于轶事经验和回顾性研究。我们旨在确定全身性类固醇对增殖性血管瘤是否有效，并比较两种皮质类固醇治疗方式的疗效和安全性。
20例患有婴儿期有问题血管瘤的患者被随机分配至每日口服泼尼松龙或每月静脉注射甲泼尼龙脉冲治疗。在基线后3个月和1岁时比较他们的临床结局（使用视觉模拟评分进行改善情况评估）和不良事件。在前3个月可获得血管生成可能替代标志物的数据。
3个月时，口服治疗的患者视觉模拟评分中位数为70，而静脉注射组为12。在患者1岁生日时这种反应模式相似：50.0对-1.5。65%的患者（静脉注射组7例，口服组6例）在3个月后需要额外治疗。8例入组时视力受损的患者中有6例在1岁时功能得到改善（静脉注射组4例，口服组3例）。在所测量的4种血管生成替代标志物（血浆碱性成纤维细胞生长因子、血管内皮生长因子、血管细胞黏附分子1、内皮糖蛋白和尿碱性成纤维细胞生长因子）中，仅随时间下降的2种是血管细胞黏附分子1和内皮糖蛋白。口服组患者不良反应发生率较高，如高血压（18.6%对13.1%）、皮质醇异常（78%对60%）和生长发育迟缓。
全身性皮质类固醇对阻止血管瘤增殖有效。口服皮质类固醇比脉冲类固醇提供了更多的临床和生物学益处，但不良反应风险更高。
