title: Personalized Therapy for Generalized Lymphatic Anomaly/Gorham-Stout Disease With a Combination of Sunitinib and Taxol.
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.
authors.affiliation: *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.
authors.forename: Jochen
authors.initials: J
authors.lastname: Rössler
authors.forename: Ulrich
authors.initials: U
authors.lastname: Saueressig
authors.forename: Gian
authors.initials: G
authors.lastname: Kayser
authors.forename: Moritz
authors.initials: M
authors.lastname: von Winterfeld
authors.forename: Gianoula L
authors.initials: GL
authors.lastname: Klement
mesh_terms: D059250:Administration, Metronomic; D000293:Adolescent; D002648:Child; D059350:Chronic Pain / Q000209:etiology; D003131:Combined Modality Therapy; D004041:Dietary Fats / Q000008:administration & dosage; D004357:Drug Synergism; D004359:Drug Therapy, Combination; D017809:Fatal Outcome; D006801:Humans; D007211:Indoles / Q000008:administration & dosage / Q000627:therapeutic use*; D044148:Lymphatic Abnormalities / Q000150:complications / Q000000981:diagnostic imaging / Q000178:diet therapy / Q000188:drug therapy*; D008297:Male; D010015:Osteolysis, Essential / Q000150:complications / Q000000981:diagnostic imaging / Q000178:diet therapy / Q000188:drug therapy*; D017239:Paclitaxel / Q000008:administration & dosage / Q000627:therapeutic use*; D010384:Pelvic Bones / Q000000981:diagnostic imaging; D010996:Pleural Effusion / Q000209:etiology; D057285:Precision Medicine; D011758:Pyrroles / Q000008:administration & dosage / Q000627:therapeutic use*; D011859:Radiography; D012008:Recurrence; D012074:Remission Induction; D012121:Respiration, Artificial; D012131:Respiratory Insufficiency / Q000209:etiology / Q000628:therapy; D000077210:Sunitinib; D014280:Triglycerides / Q000008:administration & dosage
chemical_list: D004041:Dietary Fats; D007211:Indoles; D011758:Pyrroles; D014280:Triglycerides; D017239:Paclitaxel; D000077210:Sunitinib