Efficacy and safety of sirolimus in the treatment of refractory kaposiform hemangioendothelioma
Author(s): Li Kai, Wang Zuopeng, Yao Wei, Dong Kuiran, Xiao Xianmin
Pages: 886-889
Year: 2014 Issue: 12
Journal: Chinese Journal of Pediatric Surgery
Keyword: Hemangioendothelioma; Sirolimus; Validation studies;
Abstract: Objective To reported the efficacy and safety of sirolimus in the treatment of refractory Kaposiform Hemangioendothelioma (KHE).Methods Retrospective evaluations were conducted for a cohort of 7 patients with refractory KHE receiving sirolimus for compassionate use after failed multiple therapies from January 2010 to November 2013.There were 4 boys and 3 girls.And extremities,trunk and faciocervical region were involved.Initial dose was 0.8 mg/m2 twice daily at approximately 12-hour intervals.Subsequent dosing adjustments were made to maintain a target level of 10-15 ng/ml.And prophylactic trimoxazole was started for potential risk of immunosuppression.Results The mean age was (3.2 ± 1.7) months.There were significant improvements in clinical status,including tumor shrinkage,platelet increment and coagulation function improvement.The side effects were mild and tolerable.Steroids and vincristine were quickly withdrawn.Time to initial response was (6.5 ± 3.4) days.Average stabilization time of platelet was (16.0 ± 7.6) days.Average time for sirolimus as single-agent therapy was (1.7 ± 0.4) months.Average duration of sirolimus treatment was (4.8 ± 4.9) months.Sirolimus treatment continued in all cases except for drug withdrawal because of BCG vaccination failure in 1 case.There was no symptomatic relapse.Conclusions Sirolimus appears to be efficacious and safe for patients with refractory KHE.
Pages: 886-889
Year: 2014 Issue: 12
Journal: Chinese Journal of Pediatric Surgery
Keyword: Hemangioendothelioma; Sirolimus; Validation studies;
Abstract: Objective To reported the efficacy and safety of sirolimus in the treatment of refractory Kaposiform Hemangioendothelioma (KHE).Methods Retrospective evaluations were conducted for a cohort of 7 patients with refractory KHE receiving sirolimus for compassionate use after failed multiple therapies from January 2010 to November 2013.There were 4 boys and 3 girls.And extremities,trunk and faciocervical region were involved.Initial dose was 0.8 mg/m2 twice daily at approximately 12-hour intervals.Subsequent dosing adjustments were made to maintain a target level of 10-15 ng/ml.And prophylactic trimoxazole was started for potential risk of immunosuppression.Results The mean age was (3.2 ± 1.7) months.There were significant improvements in clinical status,including tumor shrinkage,platelet increment and coagulation function improvement.The side effects were mild and tolerable.Steroids and vincristine were quickly withdrawn.Time to initial response was (6.5 ± 3.4) days.Average stabilization time of platelet was (16.0 ± 7.6) days.Average time for sirolimus as single-agent therapy was (1.7 ± 0.4) months.Average duration of sirolimus treatment was (4.8 ± 4.9) months.Sirolimus treatment continued in all cases except for drug withdrawal because of BCG vaccination failure in 1 case.There was no symptomatic relapse.Conclusions Sirolimus appears to be efficacious and safe for patients with refractory KHE.
Related Articles