Tuberous sclerosis medical therapy: Difference between revisions
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==Medical Therapy== | ==Medical Therapy== | ||
=== Epilepsy === | |||
* Anti-[[seizure]] medications can be prescribed to control [[seizures]]. The preferred medication is [[vigabatrin]]<ref name="pmid19557123">{{cite journal |author=Tsao CY |title=Current trends in the treatment of infantile spasms |journal=Neuropsychiatr Dis Treat |volume=5 |issue= |pages=289–99 |year=2009 |pmid=19557123 |pmc=2695218 |doi= |url=http://www.dovepress.com/articles.php?article_id=3150}}</ref>, but other [[anticonvulsants]], [[clobazam]], [[Ketogenic diet|ketogenic]] diet, [[vagal nerve stimulation]] or surgical resection of CNS lesion responsible for the [[epilepsy]].<ref name=":0">Henske, Elizabeth P., et al. "Tuberous sclerosis complex." ''Nature reviews Disease primers'' 2.1 (2016): 1-18.</ref> Treatment before the onset of [[epilepsy]] and other neuropsychiatric manifestations may be beneficial, but trials testing this approach are still ongoing (EPISTOP trial). [[mTOR]] inhibitors may present with some anti-epileptogenic properties.<ref name=":0" /> | |||
* [[ | |||
* [[ | === Lymphangioleiomyomatosis === | ||
* [[Sirolimus]] should be used in women presenting with TSC-associated [[lymphangioleiomyomatosis]] or sporadic LAM with <70% of the predicted normal FEV1. [[Everolimus]] has also been show to improve these patients' condition.<ref name=":0" /> | |||
=== Angiomyolipoma === | |||
* Treatment is done in a pre-emptive manner to prevent bleeding, reduce tumor size or slow its growth. Percutaneous [[Therapeutic embolization|embolization]] (which may present with postembolization syndrome), systemic [[everolimus]] (preferred method) and nephron-sparing surgical resection are possible treatments.<ref name=":0" /> | |||
=== Subependymal Giant Cell Astrocytoma === | |||
* Surgical resection is the most performed treatment, but may complicate with incomplete resection, hemorrhage, infection and cerebrospinal fluid obstruction.<ref name=":0" /> Due to these complications, treatment with [[mTOR]] inhibitors have been recommended, being well tolerated and presenting with side effects that decreases over time.<ref name=":0" /> | |||
=== Tuberous Sclerosis Complex-associated Neuropsychiatric Disorder === | |||
* There are no specific interventions, patients must be followed closely by a psychiatrist. Trials exploring the use of [[mTOR]] inhibitors for this manifestation are ongoing.<ref name=":0" /> | |||
=== Skin Lesions === | |||
For TSC-associated skin lesions, the following treatments may be performed: | |||
* [[Sunscreen|Sun protection]]; | |||
* Ablation with pulse laser dye or [[CO2]]; | |||
* Surgery | |||
* "Camouflage make-up" | |||
* Topical mTOR inhibitors. | |||
=== mTOR Inhibitors side effect: === | |||
mTOR inhibition therapy may cause [[stomatitis]], [[Diabetes mellitus|diabetes]], [[hyperlipidemia]], wound-healing complications, infertility and delayed sexual maturation.<ref name=":0" /> | |||
==References== | ==References== | ||
Revision as of 23:13, 19 June 2020
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]
Medical Therapy
Epilepsy
- Anti-seizure medications can be prescribed to control seizures. The preferred medication is vigabatrin[1], but other anticonvulsants, clobazam, ketogenic diet, vagal nerve stimulation or surgical resection of CNS lesion responsible for the epilepsy.[2] Treatment before the onset of epilepsy and other neuropsychiatric manifestations may be beneficial, but trials testing this approach are still ongoing (EPISTOP trial). mTOR inhibitors may present with some anti-epileptogenic properties.[2]
Lymphangioleiomyomatosis
- Sirolimus should be used in women presenting with TSC-associated lymphangioleiomyomatosis or sporadic LAM with <70% of the predicted normal FEV1. Everolimus has also been show to improve these patients' condition.[2]
Angiomyolipoma
- Treatment is done in a pre-emptive manner to prevent bleeding, reduce tumor size or slow its growth. Percutaneous embolization (which may present with postembolization syndrome), systemic everolimus (preferred method) and nephron-sparing surgical resection are possible treatments.[2]
Subependymal Giant Cell Astrocytoma
- Surgical resection is the most performed treatment, but may complicate with incomplete resection, hemorrhage, infection and cerebrospinal fluid obstruction.[2] Due to these complications, treatment with mTOR inhibitors have been recommended, being well tolerated and presenting with side effects that decreases over time.[2]
Tuberous Sclerosis Complex-associated Neuropsychiatric Disorder
- There are no specific interventions, patients must be followed closely by a psychiatrist. Trials exploring the use of mTOR inhibitors for this manifestation are ongoing.[2]
Skin Lesions
For TSC-associated skin lesions, the following treatments may be performed:
- Sun protection;
- Ablation with pulse laser dye or CO2;
- Surgery
- "Camouflage make-up"
- Topical mTOR inhibitors.
mTOR Inhibitors side effect:
mTOR inhibition therapy may cause stomatitis, diabetes, hyperlipidemia, wound-healing complications, infertility and delayed sexual maturation.[2]