title: Sirolimus for Vincristine-Resistant Kasabach-Merritt Phenomenon: Report of Eight Patients.
abstract: BACKGROUND
  The use of sirolimus for patients with multidrug-resistant Kasabach-Merritt phenomenon (KMP) has been reported in recent years. We present the experience of a single center in treating vincristine-resistant KMP using sirolimus alone.
  
  
  METHODS
  Children with vincristine-resistant KMP who were treated with oral sirolimus alone were eligible for inclusion in the study. We evaluated responses according to graded response criteria and acute toxicities according to the National Cancer Institute Common Toxicity Criteria.
  
  
  RESULTS
  Between March 2012 and October 2014, eight patients underwent sirolimus treatment. The response rate of hematologic parameters was 100% (8/8). Three tumors shrank enough to allow excision. The tumors were resected after hematologic parameters normalized. Of the five patients with unresectable vascular lesions, three had complete response, and two had partial response of their tumors at the completion of long-term (39.7 ± 24.4 wks) sirolimus treatment. Grade 3 or 4 adverse events were not documented during treatment or follow-up. No recurrence or progression of the disease was observed during follow-up.
  
  
  CONCLUSIONS
  In this small case series, we found sirolimus to be highly effective, with minimal side effects, for vincristine-resistant KMP. A larger study to compare sirolimus and vincristine for KMP is warranted.
authors.affiliation: Department of Pediatric Surgery, Second Affiliated Hospital, College of Medicine, Xi'an JiaoTong University, Xi'an, China.
authors.forename: Huaijie
authors.identifier: http://orcid.org/0000-0001-7259-3966
authors.initials: H
authors.lastname: Wang
authors.affiliation: Department of Pediatric Surgery, Second Affiliated Hospital, College of Medicine, Xi'an JiaoTong University, Xi'an, China.
authors.forename: Yitao
authors.initials: Y
authors.lastname: Duan
authors.affiliation: Department of Pediatric Surgery, Second Affiliated Hospital, College of Medicine, Xi'an JiaoTong University, Xi'an, China.
authors.forename: Ya
authors.initials: Y
authors.lastname: Gao
authors.affiliation: Department of Pediatric Surgery, Second Affiliated Hospital, College of Medicine, Xi'an JiaoTong University, Xi'an, China.
authors.forename: Xinkui
authors.initials: X
authors.lastname: Guo
mesh_terms: D001774:Blood Chemical Analysis; D004334:Drug Administration Schedule; D019008:Drug Resistance, Neoplasm; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D059885:Kasabach-Merritt Syndrome / Q000175:diagnosis / Q000188:drug therapy*; D008297:Male; D061214:Patient Safety; D012074:Remission Induction; D012189:Retrospective Studies; D012494:Sampling Studies; D020123:Sirolimus / Q000008:administration & dosage*; D012878:Skin Neoplasms / Q000175:diagnosis / Q000188:drug therapy*; D016896:Treatment Outcome; D014750:Vincristine / Q000627:therapeutic use
chemical_list: D014750:Vincristine; D020123:Sirolimus