Editorial Comment to Outcomes of everolimus treatment for renal angiomyolipoma associated with tuberous sclerosis complex: A single institution experience in Japan

Fujita, Kazutoshi; Nonomura, Norio

doi:10.1111/iju.13200

Cited by 1 publication

(2 citation statements)

References 5 publications

(10 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…As a result, to maintain the inhibitory effects on tumor growth, chronic – perhaps life‐long – treatment with mTORC1 inhibitors is indispensable. In fact, many studies report the regrowth of tumors after discontinuing mTORC1 treatment . Long‐term treatment can induce side‐effects, such as interstitial pneumonia, compromised, prolonged wound healing, aphtha and so on.…”

Section: Therapeutic Problems Of Mtorc1 Inhibitorsmentioning

confidence: 99%

See 1 more Smart Citation

Tuberous sclerosis complex: Recent advances in manifestations and therapy

et al. 2017

Self Cite

View full text Add to dashboard Cite

Tuberous sclerosis complex is an autosomal dominant inherited disorder characterized by generalized involvement and variable manifestations with a birth incidence of 1:6000. In a quarter of a century, significant progress in tuberous sclerosis complex has been made. Two responsible genes, TSC1 and TSC2, which encode hamartin and tuberin, respectively, were discovered in the 1990s, and their functions were elucidated in the 2000s. Hamartin-Tuberin complex is involved in the phosphoinositide 3-kinase-protein kinase B-mammalian target of rapamycin signal transduction pathway, and suppresses mammalian target of rapamycin complex 1 activity, which is a center for various functions. Constitutive activation of mammalian target of rapamycin complex 1 causes variable manifestations in tuberous sclerosis complex. Recently, genetic tests were launched to diagnose tuberous sclerosis complex, and mammalian target of rapamycin complex 1 inhibitors are being used to treat tuberous sclerosis complex patients. As a result of these advances, new diagnostic criteria have been established and an indispensable new treatment method; that is, "a cross-sectional medical examination system," a system to involve many experts for tuberous sclerosis complex diagnosis and treatments, was also created. Simultaneously, the frequency of genetic tests and advances in diagnostic technology have resulted in new views on symptoms. The numbers of tuberous sclerosis complex patients without neural symptoms are increasing, and for these patients, renal manifestations and pulmonary lymphangioleiomyomatosis have become important manifestations. New concepts of tuberous sclerosis complex-associated neuropsychiatric disorders or perivascular epithelioid cell tumors are being created. The present review contains a summary of recent advances, significant manifestations and therapy in tuberous sclerosis complex.

show abstract

Section: Therapeutic Problems Of Mtorc1 Inhibitorsmentioning

confidence: 99%

“…In fact, many studies report the regrowth of tumors after discontinuing mTORC1 treatment. 109,110 Long-term treatment can induce side-effects, such as interstitial pneumonia, compromised, prolonged wound healing, aphtha and so on.…”

Section: Therapeutic Problems Of Mtorc1 Inhibitorsmentioning

confidence: 99%