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Case Rep Pediatr. 2016;2016:8712962. doi: 10.1155/2016/8712962. Epub 2016 Jun 27.

Regression of Cardiac Rhabdomyomas in a Neonate after Everolimus Treatment.

Case reports in pediatrics

Helen Bornaun, Kazım Öztarhan, Tugba Erener-Ercan, Reyhan Dedeoğlu, Deniz Tugcu, Çiğdem Aydoğmuş, Merih Cetinkaya, Sultan Kavuncuoglu

Affiliations

  1. Department of Pediatric Cardiology, Kanuni Suleyman Teaching and Research Hospital, 34303 Istanbul, Turkey.
  2. Department of Neonatology, Kanuni Suleyman Teaching and Research Hospital, 34303 Istanbul, Turkey.
  3. Department of Pediatric Cardiology, Cerrahpasa Medical Faculty, Istanbul University, 34098 Istanbul, Turkey.
  4. Department of Pediatric Hematology-Oncology, Kanuni Suleyman Teaching and Research Hospital, 34303 Istanbul, Turkey.

PMID: 27429821 PMCID: PMC4939353 DOI: 10.1155/2016/8712962

Abstract

Cardiac rhabdomyoma often shows spontaneous regression and usually requires only close follow-up. However, patients with symptomatic inoperable rhabdomyomas may be candidates for everolimus treatment. Our patient had multiple inoperable cardiac rhabdomyomas causing serious left ventricle outflow-tract obstruction that showed a dramatic reduction in the size after everolimus therapy, a mammalian target of rapamycin (mTOR) inhibitor. After discontinuation of therapy, an increase in the diameter of masses occurred and everolimus was restarted. After 6 months of treatment, rhabdomyomas decreased in size and therapy was stopped. In conclusion, everolimus could be a possible novel therapy for neonates with clinically significant rhabdomyomas.

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