Gorham-Stout Disease Successfully Treated With Sirolimus and Zoledronic Acid Therapy

Journal of Pediatric Hematology/oncology
Stuart L CramerJoseph G Pressey

Abstract

Gorham-Stout disease is a life-threatening disorder often manifested by lymphatic malformation and osteolysis. Unfortunately, available therapies are not uniformly effective and often carry substantial morbidity. We report an 18-year-old male with Gorham-Stout disease manifested by lytic rib lesions and an intractable pleural effusion that responded dramatically to the combination of the mammalian target of rapamycin (mTOR) inhibitor sirolimus and the aminobisphosphonate zoledronic acid after failing interferon therapy. This tolerable therapeutic combination has demonstrated synergism in preclinical cancer models and merits further study in vascular anomalies.

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Citations

Jul 21, 2017·Pediatric Blood & Cancer·Denise M AdamsCameron C Trenor
Oct 11, 2017·Journal of Neuro-ophthalmology : the Official Journal of the North American Neuro-Ophthalmology Society·Jason H Peragallo, Bruno P Soares
Aug 25, 2017·Journal of Neuro-ophthalmology : the Official Journal of the North American Neuro-Ophthalmology Society·Inna G StrohNeil R Miller
Mar 6, 2019·The Korean Journal of Thoracic and Cardiovascular Surgery·Sungbin ChoSehoon Choi
Mar 20, 2020·Development·Marco MonroyMichael T Dellinger
Oct 4, 2018·Der Orthopäde·Ozge Gulsum IlleezIlknur Aktas
Mar 9, 2019·Laryngoscope Investigative Otolaryngology·Reema PadiaJonathan Perkins
Mar 28, 2018·Pediatric Dermatology·Veronika DvorakovaAlan D Irvine
Jan 2, 2020·BMC Musculoskeletal Disorders·Chuanxi ZhengShiquan Zhang
Mar 20, 2021·Journal of Vascular Surgery. Venous and Lymphatic Disorders·Marlies Geeurickx, Veerle Labarque
Jun 14, 2019·International Journal of Pediatric Otorhinolaryngology·François SimonVincent Couloigner
Apr 13, 2021·Pediatric Blood & Cancer·Kiersten W Ricci, Ionela Iacobas

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Methods Mentioned

BETA
biopsy

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