关键词: 卡-梅综合症, 西罗莫司, 纤维蛋白原, 肿瘤消退, 停药

Abstract: Objective To explore the feasibility of sirolimus treatment for Kasabach Merritt Syndrome (KMS) in young children. Methods From the beginning of 2013 to the end of 2016, 15 patients diagnosed as KMS were retrospectively analyzed. All the patients received prednisolone therapy combined with supportive treatment like platelet infusion. Thirteen patients lacking positive response were treated with sirolimus. Results Among those 13 cases, 1 patient unfortunately terminated the treatment because of severe infection, other 12 patients experienced rapid recovery of hemogram. Tumor volume also decreased dramatically in 11 cases, the other one had unsatisfactory responding with an almost controlled hemogram, but unchanged tumor volume. Nine cases had withdrawn the medication, no recurrence reported in the following observation. Compared with the patients received both sirolimus and propranolol, patients received sirolimus alone manifested no significant differences in hemogram recovery, DIC remission and tumor involution ( P>0.05). Serous fibrinogen (FIB) responding to the medication during early stage of the pathology can predict the prognosis of tumor volume clearance afterwards (Pearson correlation coefficient=-0.629, P<0.05). Conclusion Sirolimus together with prednisolone should be valued as the first line treatment of KMS. The drug has effective restoring of coagulopathy so as its reversing impact on the haemangioma proliferation. More importantly, the effect is constant, so that a withdrawal after stabilization turns out to be secure and feasible.

Key words: Kasabach Merritt Syndrome, Sirolimus, Fibrinogen, Tumor involution, Withdrawl