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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.
Kingswood, J Chris; Jozwiak, Sergiusz; Belousova, Elena D; Frost, Michael D; Kuperman, Rachel A; Bebin, E Martina; Korf, Bruce R; Flamini, J Robert; Kohrman, Michael H; Sparagana, Steven P; Wu, Joyce Y; Brechenmacher, Thomas; Stein, Karen; Berkowitz, Noah; Bissler, John J; Franz, David N.
Afiliação
  • Kingswood JC; Sussex Renal Unit, Royal Sussex County Hospital, Brighton, UK The Trafford Department of Renal Medicine, Royal Sussex County Hospital, Brighton BN2 5BE, UK.
  • Jozwiak S; Department of Child Neurology, The Children's Memorial Health Institute, Warsaw, Poland.
  • Belousova ED; Moscow Research Institute of Pediatrics and Pediatric Surgery, Moscow, Russia.
  • Frost MD; Minnesota Epilepsy Group, St. Paul, MN, USA.
  • Kuperman RA; Department of Neurology, Children's Hospital & Research Center Oakland, Oakland, CA, USA.
  • Bebin EM; Department of Neurology, University of Alabama School of Medicine, Birmingham, AL, USA.
  • Korf BR; Department of Genetics, University of Alabama at Birmingham, Birmingham, AL, USA.
  • Flamini JR; Children's Healthcare of Atlanta, Atlanta, GA, USA.
  • Kohrman MH; University of Chicago Medicine Comer Children's Hospital, Chicago, IL, USA.
  • Sparagana SP; Texas Scottish Rite Hospital for Children, Dallas, TX, USA.
  • Wu JY; Division of Pediatric Neurology, Mattel Children's Hospital UCLA, Los Angeles, CA, USA.
  • Brechenmacher T; Novartis Pharmaceuticals S.A.S., Rueil-Malmaison, France.
  • Stein K; Novartis Pharmaceuticals Corporation, Florham Park, NJ, USA.
  • Berkowitz N; Novartis Pharmaceuticals Corporation, Florham Park, NJ, USA.
  • Bissler JJ; Present address: Division of Nephrology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA Present address: Division of Nephrology, Le Bonheur Children's Hospital and University of Tennessee College of Medicine, Memphis, TN, USA.
  • Franz DN; Department of Pediatrics and Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Nephrol Dial Transplant ; 29(6): 1203-10, 2014 Jun.
Article em En | MEDLINE | ID: mdl-24729041
BACKGROUND: Tuberous sclerosis complex (TSC) is characterized by benign tumours in multiple organs, including the brain, kidneys, skin, lungs and heart. Our objective was to evaluate everolimus, an mTOR inhibitor, in the treatment of angiomyolipoma in patients with subependymal giant cell astrocytoma (SEGA) associated with TSC. METHODS: EXamining everolimus In a Study of Tuberous Sclerosis Complex-1 (NCT00789828), a prospective, double-blind, randomized, placebo-controlled, Phase 3 study, examined everolimus in treating SEGA associated with TSC. Patients with serial SEGA growth from pre-baseline to baseline scans were randomly assigned (2:1) to receive 4.5 mg/m(2)/day everolimus (target blood trough: 5-15 ng/mL; n = 78) or placebo (n = 39). Angiomyolipoma response rates were analysed in patients (n = 44) with target baseline angiomyolipoma lesions (≥1 angiomyolipoma; longest diameter ≥1.0 cm). An angiomyolipoma response rate, defined as the proportion of patients with confirmed angiomyolipoma response, was assessed by kidney CT or MRI screening at baseline, at 12, 24 and 48 weeks and annually. RESULTS: Angiomyolipoma response rates were 53.3% (16/30) and 0% (0/14) for everolimus- and placebo-treated patients, respectively. Angiomyolipoma reductions ≥50% in the sum of volumes of all target lesions were seen only in everolimus-treated patients (56.5, 78.3 and 80.0%) compared with placebo-treated patients (0% at each time point) at Weeks 12, 24 and 48, respectively. Greater percentages of everolimus-treated patients had angiomyolipoma reductions ≥30% at these same time points (82.6, 100 and 100% versus 8.3, 18.2 and 16.7% for everolimus versus placebo, respectively). CONCLUSIONS: Everolimus showed efficacy in reducing angiomyolipoma lesion volume in patients with SEGA associated with TSC.The trial is registered with ClinicalTrials.gov, number NCT00789828; http://clinicaltrials.gov/ct2/show/NCT00789828?term=EXIST-1&rank=1.
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Texto completo: 1 Coleções: 01-internacional Contexto em Saúde: 1_ASSA2030 / 2_ODS3 Base de dados: MEDLINE Assunto principal: Astrocitoma / Esclerose Tuberosa / Angiomiolipoma / Sirolimo / Neoplasias Renais / Neoplasias Primárias Múltiplas / Antineoplásicos Tipo de estudo: Clinical_trials / Observational_studies / Risk_factors_studies Limite: Adult / Child, preschool / Female / Humans / Male Idioma: En Revista: Nephrol Dial Transplant Ano de publicação: 2014 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Contexto em Saúde: 1_ASSA2030 / 2_ODS3 Base de dados: MEDLINE Assunto principal: Astrocitoma / Esclerose Tuberosa / Angiomiolipoma / Sirolimo / Neoplasias Renais / Neoplasias Primárias Múltiplas / Antineoplásicos Tipo de estudo: Clinical_trials / Observational_studies / Risk_factors_studies Limite: Adult / Child, preschool / Female / Humans / Male Idioma: En Revista: Nephrol Dial Transplant Ano de publicação: 2014 Tipo de documento: Article