Effect of everolimus on skin lesions in patients treated for subependymal giant cell astrocytoma and renal angiomyolipoma: final 4-year results...
Franz D. N., Budde K., Kingswood J. C., Belousova E., Sparagana S., de V. P. J., Berkowitz N., Ridolfi A., Bissler J. J.
Journal of the European Academy of Dermatology and Venereology
Vol.32, Issue10, P. 1796-1803
Опубликовано: 2018
Тип ресурса: Статья
Аннотация:
Background: Tuberous sclerosis complex (TSC) is a genetic disorder associated with tumour growth in various organs, including the brain, kidneys, heart and skin. Cutaneous lesions are prevalent manifestations of TSC, occurring in up to 90[%] of patients. Oral mammalian target of rapamycin inhibitors, such as everolimus, is believed to be effective for treatment of TSC-associated lesions because they act on the underlying disease pathophysiology. Objective: We evaluated the long-term effect of oral everolimus on TSC-associated skin lesions as a secondary objective in the phase III studies EXIST-1 (NCT00789828) and EXIST-2 (NCT00790400) after approximately 4 years of treatment. Materials and methods: Everolimus was dosed 4.5 mg/m2/day (titrated to trough 5–15 ng/mL) in patients with TSC-associated subependymal giant cell astrocytoma in EXIST-1, and 10 mg/day initially in adult patients with TSC- or sporadic lymphangioleiomyomatosis–associated renal angiomyolipoma in EXIST-2. Following posi
Ключевые слова:
everolimus; placebo; antineoplastic agent; everolimus; adolescent; adult; amenorrhea; angiomyolipoma; Article; child; controlled study; convulsion; disease association; disease course; disease severity; double blind procedure; drug dose titration; drug efficacy; drug safety; drug tolerability; female; follow up; human; hypercholesterolemia; kidney tumor; major clinical study; male; mouth ulcer; phase 3 clinical trial; pneumonia; preschool child; priority journal; proteinuria; randomized controlled trial; renal angiomyolipoma; renal angiomyolipoma; rhinopharyngitis; school child; side effect; stomatitis; subependymal giant cell astrocytoma; treatment response; tuberous sclerosis; young adult; angiomyolipoma; astrocytoma; central nervous system tumor; complication; infant; kidney tumor; middle aged; skin tumor; tuberous sclerosis; Adolescent; Adult; Angiomyolipoma; Antineoplastic Agents; Astrocytoma; Central Nervous System Neoplasms; Child; Child, Preschool; Everolimus; Female; Humans; I
Язык текста: Английский
ISSN: 1468-3083
Franz D. N.
Budde K.
Kingswood J. C.
Belousova E.
Sparagana S.
de V. P. J. Vries P.J.
Berkowitz N.
Ridolfi A.
Bissler J. J.
Франз Д. Н.
Будде К.
Кингсwоод Й. C.
Белоусова Е.
Спарагана С.
де В. П. Й. Вриес П.Й.
Беркоwитз Н.
Ридолфи А.
Бисслер Й. Й.
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
Effect of everolimus on skin lesions in patients treated for subependymal giant cell astrocytoma and renal angiomyolipoma: final 4-year results...
Текст визуальный непосредственный
Journal of the European Academy of Dermatology and Venereology
John Wiley & Sons, Inc.
Vol.32, Issue10 P. 1796-1803
2018
Статья
everolimus placebo antineoplastic agent everolimus adolescent adult amenorrhea angiomyolipoma Article child controlled study convulsion disease association disease course disease severity double blind procedure drug dose titration drug efficacy drug safety drug tolerability female follow up human hypercholesterolemia kidney tumor major clinical study male mouth ulcer phase 3 clinical trial pneumonia preschool child priority journal proteinuria randomized controlled trial renal angiomyolipoma renal angiomyolipoma rhinopharyngitis school child side effect stomatitis subependymal giant cell astrocytoma treatment response tuberous sclerosis young adult angiomyolipoma astrocytoma central nervous system tumor complication infant kidney tumor middle aged skin tumor tuberous sclerosis Adolescent Adult Angiomyolipoma Antineoplastic Agents Astrocytoma Central Nervous System Neoplasms Child Child, Preschool Everolimus Female Humans
Background: Tuberous sclerosis complex (TSC) is a genetic disorder associated with tumour growth in various organs, including the brain, kidneys, heart and skin. Cutaneous lesions are prevalent manifestations of TSC, occurring in up to 90[%] of patients. Oral mammalian target of rapamycin inhibitors, such as everolimus, is believed to be effective for treatment of TSC-associated lesions because they act on the underlying disease pathophysiology. Objective: We evaluated the long-term effect of oral everolimus on TSC-associated skin lesions as a secondary objective in the phase III studies EXIST-1 (NCT00789828) and EXIST-2 (NCT00790400) after approximately 4 years of treatment. Materials and methods: Everolimus was dosed 4.5 mg/m2/day (titrated to trough 5–15 ng/mL) in patients with TSC-associated subependymal giant cell astrocytoma in EXIST-1, and 10 mg/day initially in adult patients with TSC- or sporadic lymphangioleiomyomatosis–associated renal angiomyolipoma in EXIST-2. Following posi