Symptomatic improvement using everolimus in infants with cardiac rhabdomyoma.
Ann Pediatr Cardiol
; 12(1): 45-48, 2019.
Article
en En
| MEDLINE
| ID: mdl-30745769
ABSTRACT
BACKGROUND:
Cardiac rhabdomyoma (CR) often shows spontaneous regression and needs close follow-up. These tumors may be associated with tuberous sclerosis complex (TSC), caused by the disinhibition of m-TOR protein.OBJECTIVE:
The aim of the study is to observe the efficacy of everolimus in infants with significant CR. MATERIALS ANDMETHODS:
This is a single-center prospective observational study including infants with significant CR causing either clinical symptoms or obstruction to the blood flow. Everolimus was administered at a dose of 4.5 mg/M2/wk till the symptomatic improvement. Liver and renal function tests were monitored during treatment.RESULTS:
There were six cases of suspected CR included in the study. Median age and weight of patients were 5 days (range 1-90 days) and 3.2 kg (range 2.2-4.5 kg), respectively. One patient was excluded after surgical excision biopsy during concomitant closure of associated large perimembranous ventricular septal defect confirmed it as a fibroma. The remaining all five cases showed regression of tumor during mean follow-up of 6.1 ± 5.1 months. One child developed varicella infection necessitating temporary discontinuation of medicine during follow-up. One case had sudden death at 4 months of age.CONCLUSION:
Everolimus appears to be useful in selected cases of symptomatic CR. Multicentric studies are needed to determine its safety and efficacy in larger population.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Tipo de estudio:
Clinical_trials
/
Diagnostic_studies
/
Observational_studies
Idioma:
En
Revista:
Ann Pediatr Cardiol
Año:
2019
Tipo del documento:
Article
País de afiliación:
India