摘要： 目的·探讨小儿卡波西型血管内皮瘤（Kaposi form hemangioendothelioma，KHE）的临床特点及治疗策略。方法·回顾2009年1月—2017年12月上海交通大学医学院附属新华医院收治的19例KHE患儿的临床资料，统计分析病例的一般特点、肿瘤性状、是否合并卡萨巴赫-梅里特（卡梅）现象（Kasabach-Merritt phenomenon，KMP）、治疗方法和转归等。比较出现KMP的患儿和未出现KMP的患儿在肿瘤是否累及表皮以及深部肌肉上的差异。结果·19例KHE患儿中男性13例（68.4%），女性6例（31.6%）。发病于1岁以内17例（89.5%），其中12例（63.2%）为出生时即发现。发生部位为头颈部6例，躯干4例，四肢9例；未见多部位发病者。累及表皮者13例（68.4%），表现为皮肤大面积不规则紫红色、质硬、皮温增高的包块；未累及表皮者6例（31.6%），表现为皮下质硬、活动度差的包块。7例（36.8%）患儿发病后伴有KMP。出现KMP患儿与未出现KMP患儿在肿瘤是否累及表皮上的差异无统计学意义（
P=0.617），而在肿瘤是否累及深部肌肉上的差异存在统计学意义（
P=0.017）。所有病例最终均经手术病理确诊（肿瘤完全切除8例、部分切除5例、活检6例），术中发现肿瘤均呈实质性，并呈侵袭性生长。术前使用普萘洛尔治疗者7例，均无明显效果；口服糖皮质激素治疗者10例，其中5例肿瘤缩小后手术切除；且单用糖皮质激素治疗的有效率优于单用普萘洛尔（
P=0.044）。糖皮质激素治疗效果不佳者中有2例联合长春新碱治疗后肿瘤最终缩小。术后随访2~5年，肿瘤完全切除者未见复发，未完全切除者术后予口服激素治疗也可实现带瘤生存。结论·KHE多婴儿期起病，肿瘤累及深部肌肉时更易出现KMP。临床表现典型者应尽早手术切除，不典型者可活检明确诊断。合并KMP或无手术条件者可尝试采用糖皮质激素治疗或联合长春新碱治疗。普萘洛尔治疗效果不佳，但可作为一种诊断性治疗的手段。
关键词: 卡波西型血管内皮瘤, 卡萨巴赫-梅里特（卡梅）现象, 糖皮质激素, 治疗 Abstract: Objective· To assess the clinical characteristics and treatment strategies of Kaposi form hemangioendothelioma (KHE) in children.
Methods· The retrospective study was carried out on 19 cases of children diagnosed with KHE from January 2009 to December 2017 in Xinhua Hospital, Shanghai Jiao Tong University School of Medicine.The general demographics, tumor characteristics, presence or absence of Kasabach-Merritt phenomenon (KMP), treatment and outcome were statistically analyzed. The difference between the children with KMP and without KMP were compared in whether the tumor involved the skin and the deep muscles.
Results· Of the 19 children with KHE, 13 (68.4%) were male and 6 (31.6%) were female; 17 children (89.5%) presented within the first year of life and 12 children (63.2%) were detected at birth; 6 cases occurred on head and neck, 4 cases in the trunk, 9 cases in the extremities, and none was multifocal.Seven cases (36.8%) were complicated with KMP after onset. There was no significant difference in cutaneous lesions (P=0.617), while there was a significant difference in deep muscles involvement (P=0.017) between the cases complicated with KMP and without KMP. All cases were diagnosed on the basis of histopathology and immunohistochemistry after operation, in which 8 were completely removed, 5 were partially excised, and 6 were performed biopsy. All the tumors were substantial and aggressive growth. Seven children were treated by propranolol before operation but no obvious effect was found. Ten children were given oral corticosteroids, and five of them were found tumor shrinking and underwent operation. The treatment of corticosteroids resulted in higher effect than that of propranolol (P=0.044). Two children who showed resistance to corticosteroids accepted a combination therapy of corticosteroids and vincristine, and the tumor shrank at last. During the follow-up of 2 to 5 years, the cases which performed complete resection were without recurrence, and those who accepted partial excision also survived with postoperative corticosteroids treatment.
Conclusion· Most KHE cases occur in infancy. The cases might be predisposed with KPM when the tumors involve the deep muscles. The children with typical clinical features should undergo operation as soon as possible. Besides, those with atypical characters should take biopsy to achieve definite diagnosis. The cases with KMP or with giant tumors that are impossible to be removed could be given corticosteroids treatment with or without vincristine. Although propranolol showed a poor curative effect, it could be used for a diagnostic treatment.
Key words: Kaposi form hemangioendothelioma (KHE), Kasabach-Merritt phenomenon (KMP), corticosteroid, treatment PDF全文下载地址:
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