ArticleChild's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery2014
Rapamycin has a beneficial effect on controlling epilepsy in children with tuberous sclerosis complex: results of 7 children from a cohort of 86.
Article in Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 3 of them syntheses that pooled it.
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Who cites it
23 citing papers in PubMed, 3 syntheses or guidelines pooled it, 41 citations in OpenAlex.
- Rapamycin and rapalogs for tuberous sclerosis complex.The Cochrane database of systematic reviews · 2023Pooled it
- Rapamycin and rapalogs for tuberous sclerosis complex.The Cochrane database of systematic reviews · 2016Pooled it
- Rapamycin and its analogues (rapalogs) for Tuberous Sclerosis Complex-associated tumors: a systematic review on non-randomized studies using meta-analysis.Orphanet journal of rare diseases · 2015Pooled it
- Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- The Efficacy and Safety of Rapamycin in Children with Tuberous Sclerosis: A Cross-sectional Study.Iranian journal of child neurology · 2023Article
- Monitoring and Managing Patients with Tuberous Sclerosis Complex: Current State of Knowledge.Journal of multidisciplinary healthcare · 2022Review
- Antiepileptic Effect and Safety Profile of Rapamycin in Pediatric Patients With Tuberous Sclerosis Complex.Frontiers in neurology · 2022Article
- Chronic mTORC1 inhibition rescues behavioral and biochemical deficits resulting from neuronal Depdc5 loss in mice.Human molecular genetics · 2019Article
- [Chinese expert consensus on surgical treatment of tuberous sclerosis complex-related epilepsy].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2019Article
- Akt Inhibitor Perifosine Prevents Epileptogenesis in a Rat Model of Temporal Lobe Epilepsy.Neuroscience bulletin · 2018Article
- Epilepsy in patients with autism: links, risks and treatment challenges.Neuropsychiatric disease and treatment · 2018Review
- Rapamycin therapy for neonatal tuberous sclerosis complex with cardiac rhabdomyomas: A case report and review.Experimental and therapeutic medicine · 2017Article
- Proteomic Analysis After Status Epilepticus Identifies UCHL1 as Protective Against Hippocampal Injury.Neurochemical research · 2017Article
- mTOR inhibitors in the pharmacologic management of tuberous sclerosis complex and their potential role in other rare neurodevelopmental disorders.Orphanet journal of rare diseases · 2017Review
- Lamotrigine decreases MRP8 and IL-7 in rat models of intractable epilepsy secondary to focal cortical dysplasia.Experimental and therapeutic medicine · 2016Article
- Convulsive seizures from experimental focal cortical dysplasia occur independently of cell misplacement.Nature communications · 2016Article
- Four-week rapamycin treatment improves muscular dystrophy in a fukutin-deficient mouse model of dystroglycanopathy.Skeletal muscle · 2016Article
- mTORC1 Prevents Preosteoblast Differentiation through the Notch Signaling Pathway.PLoS genetics · 2015Article
- Tuberous Sclerosis: A New Frontier in Targeted Treatment of Autism.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2015Review
- Tuberous sclerosis complex; single center experience.Turk pediatri arsivi · 2015Article
Corrections and comments
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Authors and funding
9 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeTuberous sclerosis complex (TSC) is a genetic disorder characterized by the formation of hamartomas in various organ systems. We would like share our experience from 86 patients and the results of rapamycin treatment in seven children with TSC.
methodsEighty-six children with TSC were enrolled into this retrospective study. The clinical features of seven children treated with oral rapamycin were presented in detail.
resultsThe most common complaint of administration was convulsion in 77 children (89.5%). Hypopigmented skin lesions, adenoma sebaceum, resistant epilepsy, intracardiac mass, renal angiomyolipomas, and West syndrome were detected (n = 83, 96.5%; n = 47, 54.7%; n = 36, 41.9%; n = 27, 31.4%; n = 18, 20.9%; and n = 13, 15.1%, respectively). Subependymal nodules were the most frequent finding in cranial imaging followed by cortical tubers and subependymal giant cell astrocytomas (n = 75, 87.2%; n = 71, 82.6%; and n = 8, 9.3%, respectively). Of the seven patients treated with rapamycin, the lesions of six children with facial adenoma sebaceum showed regression in various degrees. The frequency of convulsions decreased in five patients with resistant epilepsy within the first 6 months of the treatment, and complete control of convulsion for all patients was achieved in the second 6 months.
conclusionThis is the first study that showed that rapamycin is an effective agent for controlling epilepsy without any significant side effect in children with TSC. Rapamycin seems to be effective after 6 months of therapy, and we recommend tapering the dosage after successful management of epilepsy.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.