The Role of mTOR Inhibitors in the Treatment of Patients with Tuberous Sclerosis Complex: Evidence-based and Expert Opinions.

Abstract:

:Tuberous sclerosis complex (TSC) is a genetic disorder arising from mutations in the TSC1 or TSC2 genes. The resulting over-activation of the mammalian target of rapamycin (mTOR) signalling pathway leaves patients with TSC susceptible to the growth of non-malignant tumours in multiple organs. Previously, surgery was the main therapeutic option for TSC. However, pharmacological therapy with mTOR inhibitors such as everolimus and sirolimus is now emerging as an alternate approach. Everolimus and sirolimus have already been shown to be effective in treating subependymal giant cell astrocytoma (SEGA) and renal angiomyolipoma (AML), and everolimus is currently being evaluated in treating TSC-related epilepsy. In November 2013 a group of European experts convened to discuss the current options and practical considerations for treating various manifestations of TSC. This article provides evidence-based recommendations for the treatment of SEGA, TSC-related epilepsy and renal AML, with a focus on where mTOR inhibitor therapy may be considered alongside other treatment options. Safety considerations regarding mTOR inhibitor therapy are also reviewed. With evidence of beneficial effects in neurological and non-neurological TSC manifestations, mTOR inhibitors may represent a systemic treatment for TSC.

journal_name

Drugs

journal_title

Drugs

authors

Curatolo P,Bjørnvold M,Dill PE,Ferreira JC,Feucht M,Hertzberg C,Jansen A,Jóźwiak S,Kingswood JC,Kotulska K,Macaya A,Moavero R,Nabbout R,Zonnenberg BA

doi

10.1007/s40265-016-0552-9

subject

Has Abstract

pub_date

2016-04-01 00:00:00

pages

551-65

issue

5

eissn

0012-6667

issn

1179-1950

pii

10.1007/s40265-016-0552-9

journal_volume

76

pub_type

杂志文章,评审

相关文献

DRUGS文献大全
  • The funny current: cellular basis for the control of heart rate.

    abstract::The 'funny' (pacemaker, I(f)) current, first described almost 30 years ago in sinoatrial node (SAN) myocytes, is a mixed sodium/potassium inward current, activated on hyperpolarisation in the diastolic range of voltages. 'Funny' (f) channels are activated by intracellular cyclic adenosine monophosphate (cAMP) concentr...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200767002-00003

    authors: DiFrancesco D,Borer JS

    更新日期:2007-01-01 00:00:00

  • Sirolimus use in recipients of expanded criteria donor kidneys.

    abstract::With changing donor characteristics and the growing shortage in organ supply, renal transplant practitioners have sought to optimize the use of expanded criteria donor (ECD) kidneys, which have poorer outcomes than standard criteria donor (SCD) kidneys. The outcomes may represent an acceptable trade-off if ECD transpl...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200868001-00006

    authors: House AA,Nguan CY,Luke PP

    更新日期:2008-01-01 00:00:00

  • Retention in care and medication adherence: current challenges to antiretroviral therapy success.

    abstract::Health behaviors such as retention in HIV medical care and adherence to antiretroviral therapy (ART) pose major challenges to reducing new HIV infections, addressing health disparities, and improving health outcomes. Andersen's Behavioral Model of Health Service Use provides a conceptual framework for understanding ho...

    journal_title:Drugs

    pub_type: 杂志文章

    doi:10.1007/s40265-015-0373-2

    authors: Holtzman CW,Brady KA,Yehia BR

    更新日期:2015-04-01 00:00:00

  • Glatiramer acetate: a review of its use in relapsing-remitting multiple sclerosis and in delaying the onset of clinically definite multiple sclerosis.

    abstract::Glatiramer acetate is a synthetic analogue of the multiple sclerosis (MS)-associated antigen, myelin basic protein. It is indicated in the EU, US and many other countries to reduce the frequency of relapses in patients with relapsing-remitting MS (RRMS), and for the treatment of patients who have experienced a well de...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/11204560-000000000-00000

    authors: Carter NJ,Keating GM

    更新日期:2010-08-20 00:00:00

  • Capecitabine.

    abstract::Capecitabine is an orally administered fluoropyrimidine carbamate used for the treatment of paclitaxel- or anthracycline-refractory breast cancer. Capecitabine is metabolised via a 3-step process to the active agent fluorouracil. The final step of this process occurs preferentially in malignant tissue. In patients wit...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-199958010-00006

    authors: Dooley M,Goa KL

    更新日期:1999-07-01 00:00:00

  • Modified Regulatory Pathways to Approve Generic Drugs in the US and a Systematic Review of Their Outcomes.

    abstract:BACKGROUND:Generic drugs are approved on the basis of pharmaceutical equivalence and bioequivalence. Some drug products have unique structural or functional attributes, necessitating modified approaches to bioequivalence determinations. OBJECTIVE:The aim of this systematic review was to identify studies that evaluated...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.1007/s40265-015-0382-1

    authors: Kesselheim AS,Polinski JM,Fulchino LA,Isaman DL,Gagne JJ

    更新日期:2015-04-01 00:00:00

  • [Properties and features of nabumetone].

    abstract::Nabumetone is a nonsteroidal anti-inflammatory drug (NSAID) of the 2,6-disubstituted naphthylalkanone class. It is a prodrug metabolised to an active metabolite, 6-methoxy-2-naphthylacetic acid (6-MNA), which preferentially inhibits cyclo-oxygenase-2 (COX-2) and has both anti-inflammatory and analgesic properties. The...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:

    authors: Davies NM,McLachlan AJ

    更新日期:2000-01-01 00:00:00

  • Cefdinir: a review of its use in the management of mild-to-moderate bacterial infections.

    abstract::Cefdinir (Omnicef) is an oral third-generation cephalosporin with good in vitro activity against many pathogens commonly causative in community-acquired infections. The drug provides good coverage against Haemophilus influenzae, Moraxella catarrhalis and penicillin-susceptible Streptococcus pneumoniae, the most common...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200464130-00004

    authors: Perry CM,Scott LJ

    更新日期:2004-01-01 00:00:00

  • Acute upper gastrointestinal bleeding.

    abstract::The treatment of acute upper gastrointestinal bleeding is becoming more dependent upon early diagnosis. For the majority of patients whose bleeding has stopped when they come to the physician's attention, treatment for peptic disorders remains the mainstay of therapy. For patients who present to the physician actively...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-198937010-00007

    authors: Schaffner JA

    更新日期:1989-01-01 00:00:00

  • A comparison of roxatidine acetate and ranitidine in gastric ulcer healing.

    abstract::A randomised multicentre double-blind study was conducted to compare the efficacy and safety of roxatidine acetate 75 mg twice daily and ranitidine 150 mg twice daily in 295 patients with endoscopically confirmed gastric ulcers. Substantial reductions in ulcer diameters and healing rates of 85.6 and 88.2% for roxatidi...

    journal_title:Drugs

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.2165/00003495-198800353-00021

    authors: Judmaier G

    更新日期:1988-01-01 00:00:00

  • Viral respiratory tract infections in transplant patients: epidemiology, recognition and management.

    abstract::Viral respiratory tract infections (RTIs) are common causes of mild illness in immunocompetent children and adults, with occasional significant morbidity or mortality in the very young, very old or infirm. However, recipients of solid organ transplants (SOT) or haematopoietic stem cell transplants (HSCT) are at marked...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200767100-00004

    authors: Lee I,Barton TD

    更新日期:2007-01-01 00:00:00

  • Comparative efficacy, safety and pharmacokinetics of verapamil SR vs verapamil IR in hypertensive patients.

    abstract::The antihypertensive effects of the regular immediate release formulation of verapamil (verapamil IR) and the newer sustained release formulation of verapamil (verapamil SR) were compared in Hispanic patients with untreated essential hypertension. Verapamil IR was given in 3 divided doses (80 or 160mg 3 times daily) a...

    journal_title:Drugs

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.2165/00003495-199200441-00002

    authors: Fuenmayor NT,Faggin BM,Cubeddu LX

    更新日期:1992-01-01 00:00:00

  • An overview of the long-term safety experience of nabumetone.

    abstract::Safety data have been gathered in US clinical trials of nabumetone on 1912 patients from August 1981 to May 1988. Dosing in the double-blind trials was 100 mg at bedtime, but in open-label trials patients could increase the dosage of nabumetone to 1500 or 2000 mg if required. Adverse experiences reported in the double...

    journal_title:Drugs

    pub_type: 临床试验,杂志文章

    doi:10.2165/00003495-199000405-00008

    authors: Willkens RF

    更新日期:1990-01-01 00:00:00

  • Management of cystitis in young women.

    abstract::The common clinical problem of recurrent symptomatic urinary tract infections (UTI) in young women may be inconvenient and distressing to both the patient and her family, particularly if the infections are not managed in a rational manner. A suggested approach is included in table 1. ...

    journal_title:Drugs

    pub_type: 杂志文章

    doi:10.2165/00003495-197713020-00003

    authors: Bailey RR

    更新日期:1977-02-01 00:00:00

  • Cryptococcal infections: changing epidemiology and implications for therapy.

    abstract::Although the incidence of HIV-associated cryptococcosis has decreased in developed countries since the introduction of antiretroviral therapy, this disease continues to cause significant morbidity and mortality in sub-Saharan Africa among patients with AIDS. Important strides have been made in an attempt to decrease t...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.1007/s40265-013-0037-z

    authors: La Hoz RM,Pappas PG

    更新日期:2013-05-01 00:00:00

  • Delamanid: a review of its use in patients with multidrug-resistant tuberculosis.

    abstract::Delamanid (Deltyba(®)), a nitroimidazo-oxazole derivative, is a new anti-tuberculosis (TB) drug which exhibits potent in vitro and in vivo antitubercular activity against drug-susceptible and -resistant strains of Mycobacterium tuberculosis. It is approved in several countries, including Japan and those of the EU, for...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.1007/s40265-014-0331-4

    authors: Blair HA,Scott LJ

    更新日期:2015-01-01 00:00:00

  • Everolimus.

    abstract::Everolimus is an immunosuppressant that blocks growth factor-mediated proliferation of haematopoietic and nonhaematopoietic cells. Oral everolimus 0.75 or 1.5mg twice daily significantly reduced the incidence of the primary composite endpoint, efficacy failure 6 months after transplantation, compared with azathioprine...

    journal_title:Drugs

    pub_type: 杂志文章

    doi:10.2165/00003495-200464080-00005

    authors: Chapman TM,Perry CM

    更新日期:2004-01-01 00:00:00

  • Correction to: Deprescribing Opioids in Chronic Non‑cancer Pain: Systematic Review of Randomised Trials.

    abstract::Page 9, Fig. 2, which originally appeared as. ...

    journal_title:Drugs

    pub_type: 已发布勘误

    doi:10.1007/s40265-020-01416-7

    authors: Mathieson S,Maher CG,Ferreira GE,Hamilton M,Jansen J,McLachlan AJ,Underwood M,Lin CC

    更新日期:2020-10-01 00:00:00

  • Valsartan/hydrochlorothiazide.

    abstract::Valsartan/hydrochlorothiazide (HCTZ) combines an angiotensin II AT1 receptor blocker with a thiazide diuretic to produce additive blood pressure reductions without major effects on heart rate. HCTZ did not significantly alter valsartan pharmacokinetics; during combination therapy, HCTZ pharmacokinetics differed from t...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-199957050-00008

    authors: Langtry HD,McClellan KJ

    更新日期:1999-05-01 00:00:00

  • Bevacizumab combination therapy: for the first-line treatment of advanced epithelial ovarian, fallopian tube or primary peritoneal cancer.

    abstract::Bevacizumab is a recombinant, humanized anti-vascular endothelial growth factor (VEGF) monoclonal antibody that neutralizes the biological activity of VEGF and inhibits tumour angiogenesis. In two pivotal, well designed, phase III, clinical trials (GOG-0218 and ICON7) in women with advanced epithelial ovarian, fallopi...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/11208940-000000000-00000

    authors: Dhillon S

    更新日期:2012-05-07 00:00:00

  • Treatment of chronic schizophrenia.

    abstract::The comprehensive treatment of schizophrenia requires the full resources of a clinical team that is able to offer treatment for the acute psychotic state in a hospital environment, and appropriate rehabilitation following resolution of the florid symptoms. Following a second or subsequent relapse, maintenance therapy ...

    journal_title:Drugs

    pub_type: 杂志文章

    doi:10.2165/00003495-197714040-00004

    authors: Johnson DA

    更新日期:1977-10-01 00:00:00

  • Current, new and future treatments in dyslipidaemia and atherosclerosis.

    abstract::The new therapeutic options available to clinicians treating dyslipidaemia in the last decade have enabled effective treatment for many patients. The development of the HMG-CoA reductase inhibitors (statins) have been a major advance in that they possess multiple pharmacological effects (pleiotropic effects) resulting...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200060010-00005

    authors: Chong PH,Bachenheimer BS

    更新日期:2000-07-01 00:00:00

  • Pemphigus vulgaris: an evidence-based treatment update.

    abstract:BACKGROUND:While a variety of intervention options have been described for pemphigus vulgaris, the optimal treatment strategy has not been established. OBJECTIVES:The objective of this systematic review is to assess the literature on the efficacy and safety of interventions for the treatment of pemphigus vulgaris. DA...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.1007/s40265-015-0353-6

    authors: Zhao CY,Murrell DF

    更新日期:2015-02-01 00:00:00

  • Practical treatment recommendations for the safe use of anaesthetics.

    abstract::General anaesthesia is the reversible depression of central nervous system function. There is still no agreement over what constitutes depth of anaesthesia, and the clinical anaesthetist must thus titrate drug input according to clinical signs (heart rate, blood pressure, somatic movement, autonomic responses). The po...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-199243010-00006

    authors: Sear JW

    更新日期:1992-01-01 00:00:00

  • The sore throat. When to investigate and when to prescribe.

    abstract::Sore throats are most commonly due to infections, many of which are viral and do not require specific treatment. Symptoms and signs of the common cold, influenza or croup, the occurrence of conjunctivitis in some adenoviral infections, generalised lymphadenopathy and splenomegaly in glandular fever or the presence of ...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-199040060-00007

    authors: Lang SD,Singh K

    更新日期:1990-12-01 00:00:00

  • Current status of dopamine agonists in Parkinson's disease management.

    abstract::The occurrence of late side effects of long term levodopa therapy (fluctuations in motor performance, abnormal movements, and symptoms unresponsive to dihydroxyphenylalanine) led to the search for novel anti-Parkinsonian drugs. Dopamine agonists are one of the newer families of anti-Parkinsonian agents, and they inclu...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-199346030-00005

    authors: Montastruc JL,Rascol O,Senard JM

    更新日期:1993-09-01 00:00:00

  • New oral anticoagulants for the treatment of venous thromboembolism: understanding differences and similarities.

    abstract::Venous thromboembolism (VTE) is a major cause of morbidity, mortality, and healthcare expenditure. In the United States, approximately 0.1 % of the population experiences an initial VTE event each year. Anticoagulation therapy is the cornerstone of acute VTE treatment and for prevention of recurrent VTE events. Conven...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.1007/s40265-014-0301-x

    authors: Dobesh PP,Fanikos J

    更新日期:2014-11-01 00:00:00

  • Targeting the PI3K/AKT/mTOR Pathway in Hormone-Positive Breast Cancer.

    abstract::Approximately 70% of invasive breast cancers have some degree of dependence on the estrogen hormone for cell proliferation and growth. These tumors have estrogen and/or progesterone receptors (ER/PR+), generally referred to as hormone receptor positive (HR+) tumors, as indicated by the presence of positive staining an...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.1007/s40265-020-01394-w

    authors: Nunnery SE,Mayer IA

    更新日期:2020-11-01 00:00:00

  • The polyol pathway. A historical review.

    abstract::Polyhydric alcohols (polyols) are widely distributed in nature, and the enzymes of the polyol pathway (aldose reductase and sorbitol dehydrogenase) are present in many mammalian tissues. The function of this pathway remains a mystery. A primary role for the pathway in the pathogenesis of 'sugar cataract' was provided ...

    journal_title:Drugs

    pub_type: 杂志文章

    doi:10.2165/00003495-198600322-00003

    authors: Clements RS Jr

    更新日期:1986-01-01 00:00:00

  • Managing cancer-related anorexia/cachexia.

    abstract::Cancer-related anorexia/cachexia (CAC) is a complex phenomenon in which metabolic abnormalities, proinflammatory cytokines produced by the host immune system, circulating tumour-derived catabolic factors, decreased food intake, and probably additional unknown factors, all play different roles. This review examines the...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200161040-00004

    authors: Mantovani G,Macciò A,Massa E,Madeddu C

    更新日期:2001-01-01 00:00:00