Refractory Kasabach-Merritt phenomenon successfully treated with sirolimus, and a mini-review of the published work.
Kasabach-Merritt phenomenon (KMP) is a rare and life-threatening disease involving a vascular tumor combined with severe consumptive coagulopathy. We present for the first time a case of KMP with the vascular tumor involving two anatomical sites; the patient failed to respond to steroids and vincristine. Following sirolimus therapy at a dose of 0.8 mg/m(2) twice daily, the lesions shrank and the platelet count improved and remained normal 4 months after initial therapy. Current treatments for KMP are not particularly effective. Sirolimus at 0.8 mg/m(2) per dose, administrated twice daily, appears to be a safe and effective management option. It appears to be an interesting therapeutic option in refractory KMP, but the time to response is variable.
Department of Pediatric Surgery, Children's Hospital of Fudan University, Shanghai, China.
Zuopeng
Z
Wang
Kai
K
Li
Kuiran
K
Dong
Xianmin
X
Xiao
Shan
S
Zheng
D006801:Humans; D007223:Infant; D059885:Kasabach-Merritt Syndrome / Q000097:blood / Q000188:drug therapy* / Q000473:pathology; D008297:Male; D010976:Platelet Count; D020123:Sirolimus / Q000008:administration & dosage / Q000627:therapeutic use*; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors
C546842:MTOR protein, human; D058570:TOR Serine-Threonine Kinases; D020123:Sirolimus
Kasabach-Merritt phenomenon; case report; mini-review; sirolimus; treatment