PDF(Pubmed)
Abstract：
UNASSIGNED: Kaposiform hemangioendothelioma (KHE) is a rare vascular tumor with a high risk of mortality. Few studies with large samples of KHE have been reported. KHE may develop into the Kasabach-Merritt phenomenon (KMP), which is characterized by thrombocytopenia and consumptive coagulopathy. The features of severe symptomatic anemia and life-threatening low platelets make the management of KHE associated with KMP challenging.
UNASSIGNED: The aim of this study was to examine the clinical characteristics of patients with KHE and discuss the treatment experience for different risk groups of KHE.
UNASSIGNED: Through a retrospective review of 70 patients diagnosed with KHE between 2017 and 2022 in our center, we classify lesions into three clinicopathological stages based on the tumor involving depth, and divided the severity of KHE into three levels by estimating clinicopathological stages and severity of thrombocytopenia. Treatments of different severity groups were estimated with sufficient data.
UNASSIGNED: In our cohort, 27% were neonates, and KHE lesion occurred at birth in 84% of patients. There was a slight male predominance (32 girls and 38 boys). Common clinical characteristics included associated coagulation disorder (100%), locally aggressive cutaneous blue-purple mass (89%), thrombocytopenia (78%), and local pain or joint dysfunction (20%). The lower extremities were the dominant location (35%), followed by the trunk (29%), the maxillofacial region and neck (24%), and the upper extremities (10%). Of the total cohort, 78% developed KMP; the median age at which thrombocytopenia occurred was 27.8 days. The median platelet count of patients who were associated with KMP was 24,000/µL in our cohort. Ninety-two percent of patients were given surgery treatment and 89% of these patients were given high-dose methylprednisolone (5-6 mg/kg daily) before surgery. In 55 patients with KMP, 36% were sensitive to high-dose corticosteroid therapy. Patients from the low-risk group (eight cases) underwent operation, all of whom recovered without recurrence after a maximum follow-up of 5 years. Out of 26 patients from the high-risk group, 25 underwent surgery treatment, with 1 case undergoing secondary surgery after recurrence and 1 case taking sirolimus. Out of 36 cases from the extremely high-risk group, 32 underwent surgery (including 2 cases who underwent external carotid artery ligation and catheterization), 3 of whom underwent secondary operation after recurrence, and the remaining 4 cases took medicine. The mean length of having sirolimus was 21 months; two cases stopped taking sirolimus due to severe pneumonia. Two cases died at 1 and 3 months after discharge.
UNASSIGNED: Our study describes the largest assessment of high-risk patients with KHE who have undergone an operation to date, with 5 years of follow-up to track recovery, which provides invaluable knowledge for the future treatment of patients with KHE and KMP from different risk groups: Early surgical intervention may be the most definitive treatment option for most patients with KHE; multimodality treatment is the best choice for the extremely high-risk group.
UNASSIGNED: The aim of this study was to examine the clinical characteristics of patients with KHE and discuss the treatment experience for different risk groups of KHE.
UNASSIGNED: Through a retrospective review of 70 patients diagnosed with KHE between 2017 and 2022 in our center, we classify lesions into three clinicopathological stages based on the tumor involving depth, and divided the severity of KHE into three levels by estimating clinicopathological stages and severity of thrombocytopenia. Treatments of different severity groups were estimated with sufficient data.
UNASSIGNED: In our cohort, 27% were neonates, and KHE lesion occurred at birth in 84% of patients. There was a slight male predominance (32 girls and 38 boys). Common clinical characteristics included associated coagulation disorder (100%), locally aggressive cutaneous blue-purple mass (89%), thrombocytopenia (78%), and local pain or joint dysfunction (20%). The lower extremities were the dominant location (35%), followed by the trunk (29%), the maxillofacial region and neck (24%), and the upper extremities (10%). Of the total cohort, 78% developed KMP; the median age at which thrombocytopenia occurred was 27.8 days. The median platelet count of patients who were associated with KMP was 24,000/µL in our cohort. Ninety-two percent of patients were given surgery treatment and 89% of these patients were given high-dose methylprednisolone (5-6 mg/kg daily) before surgery. In 55 patients with KMP, 36% were sensitive to high-dose corticosteroid therapy. Patients from the low-risk group (eight cases) underwent operation, all of whom recovered without recurrence after a maximum follow-up of 5 years. Out of 26 patients from the high-risk group, 25 underwent surgery treatment, with 1 case undergoing secondary surgery after recurrence and 1 case taking sirolimus. Out of 36 cases from the extremely high-risk group, 32 underwent surgery (including 2 cases who underwent external carotid artery ligation and catheterization), 3 of whom underwent secondary operation after recurrence, and the remaining 4 cases took medicine. The mean length of having sirolimus was 21 months; two cases stopped taking sirolimus due to severe pneumonia. Two cases died at 1 and 3 months after discharge.
UNASSIGNED: Our study describes the largest assessment of high-risk patients with KHE who have undergone an operation to date, with 5 years of follow-up to track recovery, which provides invaluable knowledge for the future treatment of patients with KHE and KMP from different risk groups: Early surgical intervention may be the most definitive treatment option for most patients with KHE; multimodality treatment is the best choice for the extremely high-risk group.
摘要：
■Kapososiform血管内皮瘤（KHE）是一种罕见的血管肿瘤，具有很高的死亡风险。很少有大量KHE样本的研究报道。KHE可能会发展为Kasabach-Merritt现象（KMP），其特征是血小板减少症和消耗性凝血病。严重的症状性贫血和危及生命的低血小板的特征使得与KMP相关的KHE的管理具有挑战性。
■本研究的目的是检查KHE患者的临床特征，并讨论不同KHE风险组的治疗经验。
■通过对我们中心2017年至2022年间诊断为KHE的70例患者的回顾性审查，我们根据肿瘤的累及深度将病变分为三个临床病理阶段，并通过估计临床病理分期和血小板减少的严重程度将KHE的严重程度分为三个水平。用足够的数据估计不同严重程度组的治疗。
■在我们的队列中，27%是新生儿，84％的患者在出生时发生KHE病变。男性占主导地位（32名女孩和38名男孩）。常见的临床特征包括相关的凝血障碍（100％），局部侵袭性皮肤蓝紫色肿块（89%），血小板减少症（78%），和局部疼痛或关节功能障碍（20％）。下肢占优势（35%），其次是后备箱(29%)，颌面部和颈部（24％），和上肢（10%）。在整个队列中，78%发展为KMP；发生血小板减少症的中位年龄为27.8天。在我们的队列中，与KMP相关的患者的血小板计数中位数为24,000/µL。92％的患者接受了手术治疗，其中89％的患者在手术前接受了大剂量甲基强的松龙（每天5-6mg/kg）。在55例KMP患者中，36%的患者对大剂量糖皮质激素治疗敏感。低危组（8例）患者接受了手术，所有患者在最长5年随访后均痊愈,无复发.在高危人群的26名患者中，25人接受了手术治疗，1例复发后二次手术，1例服用西罗莫司。在极高危人群的36例中，32例行手术（其中2例行颈外动脉结扎和导管插入术），其中3人复发后接受了二次手术，其余4例服药。西罗莫司的平均长度为21个月；2例因严重肺炎而停止服用西罗莫司。2例患者在出院后1个月和3个月死亡。
我们的研究描述了迄今为止接受手术的KHE高危患者的最大评估，经过5年的随访以追踪恢复情况，这为不同风险组的KHE和KMP患者的未来治疗提供了宝贵的知识：对于大多数KHE患者，早期手术干预可能是最确定的治疗选择；多模式治疗是极高危人群的最佳选择。
■本研究的目的是检查KHE患者的临床特征，并讨论不同KHE风险组的治疗经验。
■通过对我们中心2017年至2022年间诊断为KHE的70例患者的回顾性审查，我们根据肿瘤的累及深度将病变分为三个临床病理阶段，并通过估计临床病理分期和血小板减少的严重程度将KHE的严重程度分为三个水平。用足够的数据估计不同严重程度组的治疗。
■在我们的队列中，27%是新生儿，84％的患者在出生时发生KHE病变。男性占主导地位（32名女孩和38名男孩）。常见的临床特征包括相关的凝血障碍（100％），局部侵袭性皮肤蓝紫色肿块（89%），血小板减少症（78%），和局部疼痛或关节功能障碍（20％）。下肢占优势（35%），其次是后备箱(29%)，颌面部和颈部（24％），和上肢（10%）。在整个队列中，78%发展为KMP；发生血小板减少症的中位年龄为27.8天。在我们的队列中，与KMP相关的患者的血小板计数中位数为24,000/µL。92％的患者接受了手术治疗，其中89％的患者在手术前接受了大剂量甲基强的松龙（每天5-6mg/kg）。在55例KMP患者中，36%的患者对大剂量糖皮质激素治疗敏感。低危组（8例）患者接受了手术，所有患者在最长5年随访后均痊愈,无复发.在高危人群的26名患者中，25人接受了手术治疗，1例复发后二次手术，1例服用西罗莫司。在极高危人群的36例中，32例行手术（其中2例行颈外动脉结扎和导管插入术），其中3人复发后接受了二次手术，其余4例服药。西罗莫司的平均长度为21个月；2例因严重肺炎而停止服用西罗莫司。2例患者在出院后1个月和3个月死亡。
我们的研究描述了迄今为止接受手术的KHE高危患者的最大评估，经过5年的随访以追踪恢复情况，这为不同风险组的KHE和KMP患者的未来治疗提供了宝贵的知识：对于大多数KHE患者，早期手术干预可能是最确定的治疗选择；多模式治疗是极高危人群的最佳选择。
