Literature DB >> 34008055

Everolimus as a possible prenatal treatment of in utero diagnosed subependymal lesions in tuberous sclerosis complex: a case report.

Sergio Cavalheiro1,2, Marcos Devanir Silva da Costa3,4, Rosana Richtmann5.   

Abstract

INTRODUCTION: The association between cardiac rhabdomyoma and intraventricular tumors and/or subcortical nodules is characteristic of tuberous sclerosis complex (TSC). Patients with TSC may have refractory seizures, autistic behavior, and cognitive decline. CASE REPORT: The patient received the fetal diagnosis of TSC at the age of 19 weeks of gestations, where presented at prenatal ultrasound cardiac and brain tumors. Fetal MRI showed a lesion in the right and left lateral ventricles near the foramen of Monro associated with subependymal lesions along the entire ependyma of the lateral ventricles and several subcortical tubercles, and the fetal Doppler echocardiogram revealed three cardiac lesions. The fetus underwent intrauterine treatment with everolimus and presented regression and subsequent stabilization of the cardiac and brain lesions; additionally, the patient did not develop seizures or autism and presented good neuropsychomotor development.
CONCLUSION: It is the first evidence that mTOR inhibitors may help to prevent neurological complications associated with TSC.
© 2021. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.

Entities:  

Keywords:  Fetal brain tumor; Fetal chemotherapy; Tuberous sclerosis complex

Mesh:

Substances:

Year:  2021        PMID: 34008055     DOI: 10.1007/s00381-021-05218-4

Source DB:  PubMed          Journal:  Childs Nerv Syst        ISSN: 0256-7040            Impact factor:   1.475


  10 in total

Review 1.  Prenatal detection of cerebral lesions in a fetus with tuberous sclerosis.

Authors:  M Sgro; T Barozzino; A Toi; J Johnson; M Sermer; D Chitayat
Journal:  Ultrasound Obstet Gynecol       Date:  1999-11       Impact factor: 7.299

2.  Neuropsychiatric aspects of tuberous sclerosis.

Authors:  P Curatolo; R Cusmai; F Cortesi; C Chiron; I Jambaque; O Dulac
Journal:  Ann N Y Acad Sci       Date:  1991       Impact factor: 5.691

Review 3.  The Role of mTOR Inhibitors in the Treatment of Patients with Tuberous Sclerosis Complex: Evidence-based and Expert Opinions.

Authors:  Paolo Curatolo; Marit Bjørnvold; Patricia E Dill; José Carlos Ferreira; Martha Feucht; Christoph Hertzberg; Anna Jansen; Sergiusz Jóźwiak; J Christopher Kingswood; Katarzyna Kotulska; Alfons Macaya; Romina Moavero; Rima Nabbout; Bernard A Zonnenberg
Journal:  Drugs       Date:  2016-04       Impact factor: 9.546

Review 4.  Everolimus in pregnancy: Case report and literature review.

Authors:  Mie Yamamura; Takashi Kojima; Masayuki Koyama; Ataru Sazawa; Takahiro Yamada; Hisanori Minakami
Journal:  J Obstet Gynaecol Res       Date:  2017-05-29       Impact factor: 1.730

5.  Antiepileptic treatment before the onset of seizures reduces epilepsy severity and risk of mental retardation in infants with tuberous sclerosis complex.

Authors:  Sergiusz Jóźwiak; Katarzyna Kotulska; Dorota Domańska-Pakieła; Barbara Lojszczyk; Małgorzata Syczewska; Dariusz Chmielewski; Dorota Dunin-Wąsowicz; Tomasz Kmieć; Joanna Szymkiewicz-Dangel; Maria Kornacka; Wanda Kawalec; Dariusz Kuczyński; Julita Borkowska; Katarzyna Tomaszek; Elżbieta Jurkiewicz; Maria Respondek-Liberska
Journal:  Eur J Paediatr Neurol       Date:  2011-04-19       Impact factor: 3.140

6.  Adjunctive everolimus therapy for treatment-resistant focal-onset seizures associated with tuberous sclerosis (EXIST-3): a phase 3, randomised, double-blind, placebo-controlled study.

Authors:  Jacqueline A French; John A Lawson; Zuhal Yapici; Hiroko Ikeda; Tilman Polster; Rima Nabbout; Paolo Curatolo; Petrus J de Vries; Dennis J Dlugos; Noah Berkowitz; Maurizio Voi; Severine Peyrard; Diana Pelov; David N Franz
Journal:  Lancet       Date:  2016-09-06       Impact factor: 79.321

7.  Fetal rhabdomyoma: prenatal diagnosis, clinical outcome, and incidence of associated tuberous sclerosis complex.

Authors:  Rima S Bader; David Chitayat; Edmond Kelly; Greg Ryan; Jeffrey F Smallhorn; Ants Toi; Lisa K Hornberger
Journal:  J Pediatr       Date:  2003-11       Impact factor: 4.406

8.  Severe pneumonia by Mycoplasma as an adverse event of everolimus therapy in patients with tuberous sclerosis complex.

Authors:  J Carlos Flores-González; Ana Estalella-Mendoza; Alfonso María Lechuga-Sancho; Sebastian Quintero-Otero; Fernando Rubio-Quiñones; Arturo Hernández-González; Mónica Saldaña-Valderas
Journal:  Eur J Paediatr Neurol       Date:  2016-05-14       Impact factor: 3.140

9.  Maternal Sirolimus Therapy for Fetal Cardiac Rhabdomyomas.

Authors:  Benjamin T Barnes; David Procaccini; Jude Crino; Karin Blakemore; Priya Sekar; Katelynn G Sagaser; Angie C Jelin; Lasya Gaur
Journal:  N Engl J Med       Date:  2018-05-10       Impact factor: 91.245

10.  Sirolimus therapy for fetal cardiac rhabdomyoma in a pregnant woman with tuberous sclerosis.

Authors:  Hyea Park; Chi Son Chang; Suk-Joo Choi; Soo-Young Oh; Cheong-Rae Roh
Journal:  Obstet Gynecol Sci       Date:  2019-06-21
  10 in total
  1 in total

Review 1.  Prenatal MR Imaging Phenotype of Fetuses with Tuberous Sclerosis: An Institutional Case Series and Literature Review.

Authors:  S K Goergen; M C Fahey
Journal:  AJNR Am J Neuroradiol       Date:  2022-03-24       Impact factor: 3.825

  1 in total

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