Comparison of efficacy and safety of corticosteroid and vincristine in treating kaposiform hemangioendothelioma and tufted angioma: A multicenter prospective randomized controlled clinical trial

Kaposiform haemangioendothelioma (KHE) and tufted angioma (TA) are rare vascular tumors that can cause life-threatening Kasabach-Merritt phenomenon. No evidence-based treatment strategies have yet been established, and its management is still a challenge. The purpose of this multicenter prospective randomized controlled study was to evaluate and compare the efficacy of corticosteroid and vincristine (VCR) in the treatment of KHE and TA. All patients with KHE/TA who met the diagnostic criteria were consecutively recruited. The patients were randomized into a methylprednisolone (MP) group and a VCR group. The primary outcome was the single main parameter effective rate and overall effective rate of corticosteroid and VCR over 1 month after treatment. The single main parameters included platelets, fibrinogen, tumor size, texture, and appearance. From May 2016 to April 2018, a total of 59 patients completed the clinical trial, including 29 in the MP group and 30 in the VCR group. The results showed that VCR was superior to corticosteroid in the improvement of platelet (80.0% vs 44.0%, P = 0.019) and tumor texture (68.9% vs 30.8%, P = 0.007). Although the efficacy of VCR on fibrinogen (23.3% vs 20.7%, P = 1.000), tumor size (23.3% vs 13.8%, P = 0.273), and appearance (65.5% vs 46.2%, P = 0.120) was higher than that of corticosteroid, there was no significant difference (P > 0.05). Meanwhile, the overall effective rate of VCR was higher than that of corticosteroid (56.7% vs 31.0%), but the difference was also not statistically significant (P = 0.067). In conclusion, the therapeutic effect of VCR was significantly better than that of corticosteroid with regard to treating thrombocytopenia and tumor texture. We recommend that VCR could be an option for first-line treatment in KHE/TA patients.