PMID- 26886375
OWN - NLM
STAT- MEDLINE
DCOM- 20160803
LR  - 20220408
IS  - 1536-3678 (Electronic)
IS  - 1077-4114 (Linking)
VI  - 38
IP  - 3
DP  - 2016 Apr
TI  - Gorham-Stout Disease Successfully Treated With Sirolimus and Zoledronic Acid 
      Therapy.
PG  - e129-32
LID - 10.1097/MPH.0000000000000514 [doi]
AB  - 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.
FAU - Cramer, Stuart L
AU  - Cramer SL
AD  - Departments of *Pediatrics, Division of Hematology and Oncology, Children's of 
      Alabama daggerPathology, University of Alabama at Birmingham, Birmingham, AL 
      double daggerDepartment of Radiology  section signCancer and Blood Disorders Institute, Cincinnati 
      Children's Hospital Medical Center, Cincinnati, OH.
FAU - Wei, Shi
AU  - Wei S
FAU - Merrow, Arnold C
AU  - Merrow AC
FAU - Pressey, Joseph G
AU  - Pressey JG
LA  - eng
PT  - Case Reports
PT  - Journal Article
PL  - United States
TA  - J Pediatr Hematol Oncol
JT  - Journal of pediatric hematology/oncology
JID - 9505928
RN  - 0 (Bone Density Conservation Agents)
RN  - 0 (Diphosphonates)
RN  - 0 (Imidazoles)
RN  - 0 (Immunosuppressive Agents)
RN  - 6XC1PAD3KF (Zoledronic Acid)
RN  - W36ZG6FT64 (Sirolimus)
SB  - IM
MH  - Adolescent
MH  - Bone Density Conservation Agents/administration & dosage
MH  - Diphosphonates/administration & dosage
MH  - Drug Therapy, Combination/*methods
MH  - Humans
MH  - Imidazoles/administration & dosage
MH  - Immunosuppressive Agents/administration & dosage
MH  - Male
MH  - Osteolysis, Essential/*drug therapy
MH  - Sirolimus/administration & dosage
MH  - Zoledronic Acid
EDAT- 2016/02/18 06:00
MHDA- 2016/08/04 06:00
CRDT- 2016/02/18 06:00
PHST- 2016/02/18 06:00 [entrez]
PHST- 2016/02/18 06:00 [pubmed]
PHST- 2016/08/04 06:00 [medline]
AID - 10.1097/MPH.0000000000000514 [doi]
PST - ppublish
SO  - J Pediatr Hematol Oncol. 2016 Apr;38(3):e129-32. doi: 
      10.1097/MPH.0000000000000514.