Effective treatment of understanding medical research: A Synthesis of Findings from 16 Studies

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Original Abstract of the Article

Key Research Findings

Tuberous sclerosis complex (TSC) is an autosomal dominant disorder caused by the constitutive activation of the mammalian target of rapamycin (mTOR) pathway. 7 Most patients with TSC experience disfigurement caused by skin lesions involving facial angiofibromas. 7 Facial angiofibromas are the most prevalent cutaneous manifestation, affecting 80% of patients. 14 Many patients have been left untreated due to a lack of therapeutic options that are less invasive than surgery or laser treatment. 7 TSC is characterized by benign tumors in multiple organs, including the brain, kidneys, skin, lungs, and heart. 1 Epilepsy develops in 70 to 90% of children with TSC and is often resistant to medication. 11 TSC-associated neuropsychiatric disorders (TAND) occur in the majority of those with TSC, and they have a significant impact on patients and their families. 4

Treatment Summary

Treatment for TSC includes oral mTOR inhibitors such as sirolimus and everolimus, topical treatments like topical sirolimus for facial angiofibromas, surgery, and add-on cannabidiol (CBD) treatment for drug-resistant seizures. 7 5 8 12 13

Benefits and Risks

Benefit Summary

mTOR inhibitors may be effective in treating TSC-associated lesions. 6 Topical sirolimus has proven to be an effective and safe therapy for facial angiofibromas. 5 8 Surgery may be the intervention most likely to achieve long-term seizure freedom for drug-resistant TSC-associated epilepsy. 13

Risk Summary

mTOR inhibitors may cause side effects such as reduced renal function. 9 Systemic treatment with mTOR inhibitors like sirolimus causes adverse effects. 5 TSC is a multisystem genetic disorder and can cause a variety of symptoms. 3

Comparison of Studies

Similarities

Many studies suggest that mTOR inhibitors are promising therapeutic agents for the treatment of TSC-associated lesions. 6 3 10 2 16 Topical sirolimus has proven to be an effective treatment for facial angiofibromas. 5 8

Differences

Studies focus on evaluating the effectiveness and safety of different treatments. 7 1 6 11 12 13 14 3 4 15 5 9 10 2 8 16

Consistency and Contradictions of Findings

These studies provide consistent evidence suggesting that mTOR inhibitors are promising therapeutic agents for the treatment of TSC-associated lesions. 6 3 10 2 16

Implications for Real-World Application

Treatment for TSC needs to be individualized based on the symptoms and needs of each patient. 13 Treatments such as mTOR inhibitors have potential side effects, and the risks and benefits need to be carefully evaluated. 9 5

Limitations of Current Research

Many of these studies are relatively small, and larger studies may be needed. 7 5 8

Future Research Directions

Longitudinal studies are needed to evaluate the long-term effectiveness and safety of mTOR inhibitors. 3 10 Research is needed to develop new therapies for the treatment of TSC. 4

Conclusion

TSC is a multisystem genetic disorder that significantly impacts patients and their families. 3 4 These studies suggest that mTOR inhibitors are promising therapeutic agents for the treatment of TSC-associated lesions. 6 3 10 2 16 However, these therapies have potential side effects, and the risks and benefits need to be carefully evaluated. 9 5 Further research is needed to develop better therapies for the treatment of TSC. 4

Treatment List

Sirolimus, Everolimus, Cannabidiol, Surgery

Literature analysis of 16 papers
Positive Content
16
Neutral Content
0
Negative Content
0
Article Type
9
1
6
4
16
1.

The effect of everolimus on renal angiomyolipoma in patients with tuberous sclerosis complex being treated for subependymal giant cell astrocytoma: subgroup results from the randomized, placebo-controlled, Phase 3 trial EXIST-1.

Author: KingswoodJ Chris, JozwiakSergiusz, BelousovaElena D, FrostMichael D, KupermanRachel A, BebinE Martina, KorfBruce R, FlaminiJ Robert, KohrmanMichael H, SparaganaSteven P, WuJoyce Y, BrechenmacherThomas, SteinKaren, BerkowitzNoah, BisslerJohn J, FranzDavid N

Everolimus
Angiomyolipoma
Tuberous sclerosis complex

This study explores the use of everolimus, a medication that blocks a specific cellular pathway (mTOR inhibitor), in treating a type of tumor (angiomyolipoma) in patients with tuberous sclerosis complex (TSC). The study specifically focuses on patients with TSC who also have a type of brain tumor (subependymal giant cell astrocytoma, SEGA).

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

2.

Efficacy and safety of sirolimus for renal angiomyolipoma in patients with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis: a systematic review.

Author: PengZhu-feng, YangLu, WangTing-ting, HanPing, LiuZhen-hua, WeiQiang

Shrink angiomyolipoma
Hyperlipidemia
Regrowth of angiomyolipoma
Respiratory infection
Skin lesions
Stomatitis

A systematic review of 94 patients with renal angiomyolipoma treated with sirolimus found that renal angiomyolipoma shrank during sirolimus therapy but tended to regrow after therapy was stopped. Sirolimus was generally effective and safe for treating renal angiomyolipoma in patients with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis. The most common side effects were stomatitis, respiratory infection, skin lesions, and hyperlipidemia.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

3.

Systemic effects of treatment with mTOR inhibitors in tuberous sclerosis complex: a comprehensive review.

Author: SadowskiK, KotulskaK, SchwartzR A, JóźwiakS

mTOR inhibitors for TSC

Tuberous sclerosis complex (TSC) is a genetic disorder that causes non-cancerous tumors in different parts of the body. Medications called mTOR inhibitors have been shown to be effective for treating specific tumors associated with TSC. This review examines research on whether these medications might have a broader effect on the disease itself, potentially improving outcomes for people with TSC.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

4.

The use of everolimus in the treatment of neurocognitive problems in tuberous sclerosis (TRON): study protocol for a randomised controlled trial.

Author: RandellElizabeth, McNamaraRachel, DaviesD Mark, Owen-JonesEleri, KirbyNigel, AngelLianna, DrewCheney, Cannings-JohnRebecca, SmalleyMichelle, SaxenaAnurag, McDermottEmer, StockwellLaura, de VriesPetrus J, HoodKerry, SampsonJulian R

TAND treatment

Tuberous sclerosis complex (TSC) is a rare genetic disorder affecting 1 in 6,000 people, causing tumors in various organs and neurological problems. Most people with TSC experience TSC-associated neuropsychiatric disorders (TAND) that significantly impact their daily lives, education, work, and social interactions. Improved treatments for TAND could reduce the burden on patients, families, and the healthcare system.

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

5.

Efficacy and Safety of Topical Sirolimus Therapy for Facial Angiofibromas in the Tuberous Sclerosis Complex : A Randomized Clinical Trial.

Author: Wataya-KanedaMari, NakamuraAyumi, TanakaMari, HayashiMisa, MatsumotoShoji, YamamotoKoji, KatayamaIchiro

Tumor size reduction
Skin dryness
Skin irritation
Tuberous sclerosis complex

Topical sirolimus gel is safe and effective for facial angiofibromas in tuberous sclerosis complex (TSC). The optimal concentration of sirolimus was 0.2%.

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

6.

Effect of everolimus on skin lesions in patients treated for subependymal giant cell astrocytoma and renal angiomyolipoma: final 4-year results from the randomized EXIST-1 and EXIST-2 studies.

Author: FranzD N, BuddeK, KingswoodJ C, BelousovaE, SparaganaS, de VriesP J, BerkowitzN, RidolfiA, BisslerJ J

Everolimus efficacy
TSC treatment

Tuberous sclerosis complex (TSC) is a genetic disorder that causes tumors to grow in various organs, including the brain, kidneys, heart, and skin. Skin lesions are very common in TSC. Everolimus, a medication, is thought to be effective for treating these lesions because it targets the underlying cause of the disease.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

7.

Sirolimus Gel Treatment vs Placebo for Facial Angiofibromas in Patients With Tuberous Sclerosis Complex: A Randomized Clinical Trial.

Author: Wataya-KanedaMari, OhnoYuuki, FujitaYasuyuki, YokozekiHiroo, NiizekiHironori, OgaiMasaaki, FukaiKazuyoshi, NagaiHiroshi, YoshidaYuichi, HamadaIzumi, HioTaihei, ShimizuKenji, MurotaHiroyuki

Skin lesion treatment
TSC treatment

Tuberous sclerosis complex (TSC) is a genetic disorder that causes tumors to grow in various organs, including the skin. Many people with TSC have facial growths (angiofibromas) that can be disfiguring. Because effective treatments that are less invasive than surgery or laser therapy have been lacking, many people have gone untreated.

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

8.

Efficacy and Safety of Topical Rapamycin in Patients With Facial Angiofibromas Secondary to Tuberous Sclerosis Complex: The TREATMENT Randomized Clinical Trial.

Author: KoenigMary Kay, BellCynthia S, HebertAdelaide A, RobersonJoan, SamuelsJoshua A, SlopisJohn M, TatePatti, NorthrupHope,

Angiofibroma improvement
Safe treatment
Application site discomfort

Topical rapamycin was found to be effective and safe for the treatment of TSC-related facial angiofibromas. The preferred dose was 1% once daily.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, U.S. Gov't, Non-P.H.S.Research funding provided by US government agencies other than the Public Health Service.

Language : English

9.

Effect of everolimus on renal function in patients with tuberous sclerosis complex: evidence from EXIST-1 and EXIST-2.

Author: BisslerJohn J, BuddeKlemens, SauterMatthias, FranzDavid N, ZonnenbergBernard A, FrostMichael D, BelousovaElena, BerkowitzNoah, RidolfiAntonia, Christopher KingswoodJ

Preserves renal function
Reduces angiomyolipoma volume
Proteinuria

Everolimus treatment does not appear to be nephrotoxic in patients with renal angiomyolipoma or subependymal giant cell astrocytoma associated with tuberous sclerosis complex.

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

10.

Efficacy and safety of mTOR inhibitors (rapamycin and its analogues) for tuberous sclerosis complex: a meta-analysis.

Author: LiMin, ZhouYing, ChenChaoyang, YangTing, ZhouShuang, ChenShuqing, WuYe, CuiYimin

Reduced seizure frequency
Reduced tumor volume
Stomatitis

mTOR inhibitors effectively reduced tumor volume and seizure frequency in patients with tuberous sclerosis complex (TSC). While they are a good therapeutic choice, they may increase the risk of stomatitis, but not upper respiratory tract infections or nasopharyngitis.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

11.

Prevention of Epilepsy in Infants with Tuberous Sclerosis Complex in the EPISTOP Trial.

Author: KotulskaKatarzyna, KwiatkowskiDavid J, CuratoloPaolo, WeschkeBernhard, RineyKate, JansenFloor, FeuchtMartha, KrsekPavel, NabboutRima, JansenAnna C, WojdanKonrad, SijkoKamil, Głowacka-WalasJagoda, BorkowskaJulita, SadowskiKrzysztof, Domańska-PakiełaDorota, MoaveroRomina, HertzbergChristoph, HulshofHanna, SchollTheresa, BenovaBarbora, AronicaEleonora, de RidderJessie, LagaeLieven, JóźwiakSergiusz,

Reduced epilepsy severity

Epilepsy develops in a majority of children with tuberous sclerosis complex (TSC) and is often resistant to medication. The EPISTOP clinical trial aimed to compare preventive versus conventional antiepileptic treatment in infants with TSC.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

12.

Add-on Cannabidiol Treatment for Drug-Resistant Seizures in Tuberous Sclerosis Complex: A Placebo-Controlled Randomized Clinical Trial.

Author: ThieleElizabeth A, BebinE Martina, BhathalHari, JansenFloor E, KotulskaKatarzyna, LawsonJohn A, O'CallaghanFinbar J, WongMichael, SahebkarFarhad, CheckettsDaniel, KnappertzVolker,

TSC seizure reduction
Diarrhea
Liver transaminase elevation
Somnolence

This double-blind, placebo-controlled trial of 224 patients with tuberous sclerosis complex (TSC) and medication-resistant epilepsy evaluated the efficacy and safety of cannabidiol (CBD) at doses of 25 mg/kg/day (CBD25) and 50 mg/kg/day (CBD50) compared to placebo. Both CBD25 and CBD50 significantly reduced TSC-associated seizures compared to placebo. The most common side effects were diarrhea and somnolence, which occurred more frequently in the CBD groups. CBD25 had a better safety profile than CBD50.

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

14.

Topical rapamycin in the treatment of facial angiofibromas in tuberous sclerosis: a systematic review based on evidence.

Author: Cortell FusterClara, Martínez GómezMaría Amparo, Cercós LletiAna Cristina, Climente MartíMónica

Effective treatment
Safe treatment

Topical rapamycin is effective and safe for the treatment of facial angiofibromas in tuberous sclerosis. Multiple studies demonstrated its efficacy, with the exception of 5 patients in one study. Long-term studies are needed to establish an evidence-based therapeutic protocol.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

15.

The role of neurosurgery in the management of tuberous sclerosis complex-associated epilepsy: a systematic review.

Author: NieJeffrey Z, KarrasConstantine L, TrybulaS Joy, TexakalidisPavlos, AldenTord D

TSC-associated epilepsy treatment

Tuberous sclerosis complex (TSC) is a genetic disorder that causes brain lesions, often leading to seizures. While medication is the first treatment, over half of people with TSC don't respond well and need surgery. This review will examine research on surgical treatments for seizures in people with TSC, focusing on the outcomes.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

16.

Everolimus as a therapeutic option in refractory epilepsy in children with tuberous sclerosis: a systematic review.

Author: MenezesCatarina Ester Gomes, SantosDébora Lopes Dos, NeryErick Santos, SerpaEvelin Duarte, MoraisLécio Aragão Souza, DutraLucas Santana, Portela FilhoMarcos Baruch, GoesJulieta Sobreira

TS treatment

Tuberous sclerosis (TS) is a genetic disorder that can cause seizures, which are often difficult to control. Everolimus, a medication that has been shown to be effective for other aspects of TS, may also be helpful for treating seizures that don't respond to standard treatment in people with TS.

Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

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