Personalized Therapy for Generalized Lymphatic Anomaly/Gorham-Stout Disease With a Combination of Sunitinib and Taxol.

Jochen Rössler, Ulrich Saueressig, Gian Kayser, Moritz von Winterfeld, Gianoula L Klement

Journal: Journal of pediatric hematology/oncology 2016;37(8):e481-5

PMID: 26458155

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Abstract

The recently revised ISSVA classification approved in Melbourne in April 2014 recognizes generalized lymphatic anomaly and lymphatic malformation in Gorham-Stout disease. The 2 entities can overlap in presentation, as both are characterized by destructive lymphatic vessel invasion of the axial skeleton and surrounding soft tissues. At least at present, no standard therapeutic options exist, and due to the rarity of the disease, no clinical trials are available. We present 2 patients, 1 with generalized lymphatic anomaly and 1 with lymphatic malformation in Gorham-Stout disease, with severe exacerbation during puberty. The first child presented in florid pulmonary failure and pleural effusion, the other with severe pain due to bone destruction of the pelvis and inability to walk. Both were treated using individualized protocols. The manuscript describes the rationale for choosing sunitinib in combination with low-dose (metronomic) taxol. Both patients experienced clinical and radiologic response without major toxicities, suggesting that patients with rare conditions may benefit from individualized, molecularly based therapies.

Address: *Division of Pediatric Hematology/Oncology †Division of Pediatric Radiology ‡Institute of General Pathology, University Hospital of Freiburg, Freiburg §Institute of Pathology, Charité University Medicine Berlin, Berlin, Germany ∥Pediatric Hematology Oncology ¶Newman Lakka Institute for Personalized Cancer Care, Floating Hospital for Children at Tufts Medical Center, Boston, MA.

DOI: 10.1097/MPH.0000000000000436

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MeSH Terms: Administration, Metronomic, Adolescent, Child, Chronic Pain, Combined Modality Therapy, Dietary Fats, Drug Synergism, Drug Therapy, Combination, Fatal Outcome, Humans, Indoles, Lymphatic Abnormalities, Male, Osteolysis, Essential, Paclitaxel, Pelvic Bones, Pleural Effusion, Precision Medicine, Pyrroles, Radiography, Recurrence, Remission Induction, Respiration, Artificial, Respiratory Insufficiency, Sunitinib, Triglycerides