[Rapamycin in the treatment of cardiac rhabdomyoma associated with tuberous sclerosis complex].
Zhonghua Er Ke Za Zhi
; 54(6): 424-7, 2016 Jun 02.
Article
em Zh
| MEDLINE
| ID: mdl-27256228
ABSTRACT
OBJECTIVE:
To assess the efficacy and safety of mammalian target of rapamycin (mTOR) inhibitor rapamycin in treatment of children with cardiac rhabdomyoma, associated with tuberous sclerosis complex (TSC).METHOD:
The clinical data of children with cardiac rhabdomyomas, who had received a diagnosis of TSC previously, were collected between September 2011 and November 2015 from Pediatric Department of the People's Liberation Army General Hospital.Patients in line with the inclusion criteria received long-term treatment with sirolimus.The starting doses of sirolimus was 1 mg/ (m(2)·d), and the plasma concentration was maintained at 5-10 µg/L.The size and number of cardiac rhabdomyomas were analyzed after treatment with rapamycin, and the efficacy and safety were assessed. The Wilcoxon test was used to analyze data.RESULT:
All the 51 children met the inclusion and exclusion criteria, including 30 males and 21 females.The median age for rapamycin treatment was 15.0 months (7.0-35.0 months). Tumors disappeared in 26 (51%) children, decreased by more than 50%(including 50%) in 15 (29%) children, decreased by less than 50% in 5 (12%) children, and had no change or progressed in 4 (8%) children.The number of tumors decreased by 77(72%). The median maximum diameter of tumor was 8.7 (5.9-11.3) mm before treatment, 0.0 (0.0-4.0) mm after treatment, and the median decrease of tumor size were 6.7 (3.9-10.0) mm (Z=-8.817, P<0.01). The median disappearance time was 3.26 (2.92-5.37) months.Among different age groups, after treatment by rapamycin, the rate of tumor's disappearance was 50% (12/24) in 0-1 years group.Tumors disappeared in 10 of 16 patients in >1-3 years group and in 4 of 11 patients in >3 years group.The rate of tumor's disappearance was the highest after 3 months of treatment as compared with 6 and 12 months of treatment.Ten children had adverse event that was related to rapamycin.Canker sore was reported in one child and dyslipidemia was reported in 9 children.CONCLUSION:
Rapamycin is efficacious and well tolerate in treatment of cardiac rhabdomyomas associate with TSC, and lead to a reduction in tumor size and number, in addition, significantly shorten the duration of cardiac rhabdomyoma.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Rabdomioma
/
Esclerose Tuberosa
/
Sirolimo
/
Neoplasias Cardíacas
Tipo de estudo:
Risk_factors_studies
Limite:
Child, preschool
/
Female
/
Humans
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Infant
/
Male
Idioma:
Zh
Revista:
Zhonghua Er Ke Za Zhi
Ano de publicação:
2016
Tipo de documento:
Article
País de afiliação:
China