title: The GOLD ReGISTry: a Global, Prospective, Observational Registry Collecting Longitudinal Data on Patients with Advanced and Localised Gastrointestinal Stromal Tumours.
abstract: BACKGROUND
  Gastrointestinal stromal tumours (GISTs) are the most common gastrointestinal sarcomas. This global, prospective registry followed patients with advanced or localised GIST (2007-2011).
  
  
  METHODS
  Current and evolving diagnostics, treatments and outcome measures in patients with GIST were assessed. Eligible patients were diagnosed with advanced or localised GIST within 15months of registry entry. No treatment plan was prescribed, and no visit schedule was mandated. Treating physicians recorded patient information, including tumour response, diagnostic methods, medications, surgeries performed, mutation status and adverse events leading to dose/medication changes. Survival outcomes were estimated using the Kaplan-Meier method. Other data were analysed using descriptive statistics.
  
  
  RESULTS
  The registry included 1663 patients (advanced GIST, n=1095; localised GIST, n=537). Medications (e.g. tyrosine kinase inhibitor use and dosing), disease progression or recurrence and physician assessment of response to treatment in registry patients were consistent with controlled trials and prevailing clinical recommendations. In advanced GIST, estimated 30-month progression-free survival (PFS) (59.8%) and overall survival (OS) (82.7%) were higher than results from previously reported trials (≈40% and ≈70%, respectively). Consistent with treatment guidelines, the most common initial treatments were imatinib for advanced GIST, and complete surgical resection for localised GIST. Computed tomography scans were the most common imaging technique used at diagnosis and follow-up. Mutation analysis was performed at diagnosis in only 15.3% and 14.5% of patients with advanced and localised GIST, respectively.
  
  
  CONCLUSIONS
  In this real-world GIST registry, patients with advanced GIST were treated with imatinib and patients with localised GIST received surgical resection, in accordance with prevailing clinical recommendations.
authors.affiliation: PUCRS School of Medicine, Porto Alegre, Brazil. Electronic address: chbe@via-rs.net.
authors.forename: Carlos H
authors.initials: CH
authors.lastname: Barrios
authors.affiliation: Mount Sinai Hospital, Toronto, ON, Canada. Electronic address: martin.blackstein@utoronto.ca.
authors.forename: Martin E
authors.initials: ME
authors.lastname: Blackstein
authors.affiliation: Universite Claude Bernard, Lyon, France. Electronic address: jean-yves.blay@lyon.unicancer.fr.
authors.forename: Jean-Yves
authors.initials: JY
authors.lastname: Blay
authors.affiliation: Istituto Nazionale Tumori, Milan, Italy. Electronic address: paolo.casali@istitutotumori.mi.it.
authors.forename: Paolo G
authors.initials: PG
authors.lastname: Casali
authors.affiliation: Alexander Fleming Institute, Department of Medical Oncology, Buenos Aires, Argentina. Electronic address: matiemi@yahoo.com.
authors.forename: Matias
authors.initials: M
authors.lastname: Chacon
authors.affiliation: Peking University Cancer Hospital, Beijing, China. Electronic address: zlgujin@126.com.
authors.forename: Jin
authors.initials: J
authors.lastname: Gu
authors.affiliation: University of Ulsan College of Medicine, Seoul, South Korea. Electronic address: ykkang@amc.seoul.kr.
authors.forename: Yoon-Koo
authors.initials: YK
authors.lastname: Kang
authors.affiliation: National Cancer Center Hospital East, 6-5-1 Kashiwanoha, Kashiwa, Chiba 277-8577, Japan. Electronic address: tnishida@east.ncc.go.jp.
authors.forename: Toshirou
authors.initials: T
authors.lastname: Nishida
authors.affiliation: Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA. Electronic address: das.purkayastha@novartis.com.
authors.forename: Das
authors.initials: D
authors.lastname: Purkayastha
authors.affiliation: Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA. Electronic address: richard.woodman@novartis.com.
authors.forename: Richard C
authors.initials: RC
authors.lastname: Woodman
authors.affiliation: HELIOS Klinikum Berlin-Buch, Berlin, Germany. Electronic address: peter.reichardt@helios-kliniken.de.
authors.forename: Peter
authors.initials: P
authors.lastname: Reichardt
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001208:Asia / Q000453:epidemiology; D014408:Biomarkers, Tumor / Q000235:genetics; D002170:Canada / Q000453:epidemiology; D004252:DNA Mutational Analysis; D013505:Digestive System Surgical Procedures; D018450:Disease Progression; D005060:Europe / Q000453:epidemiology; D005260:Female; D046152:Gastrointestinal Stromal Tumors / Q000235:genetics / Q000401:mortality / Q000473:pathology / Q000628:therapy; D006801:Humans; D000068877:Imatinib Mesylate / Q000627:therapeutic use; D053208:Kaplan-Meier Estimate; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D009154:Mutation; D009364:Neoplasm Recurrence, Local; D010818:Practice Patterns, Physicians'; D011237:Predictive Value of Tests; D011446:Prospective Studies; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use; D012042:Registries; D012307:Risk Factors; D013020:South America / Q000453:epidemiology; D013997:Time Factors; D014057:Tomography, X-Ray Computed; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D014408:Biomarkers, Tumor; D047428:Protein Kinase Inhibitors; D000068877:Imatinib Mesylate
keywords: Adjuvant; Advanced; CD117 (c-KIT)-positive gastrointestinal stromal tumour (GIST); Global cancer registry; Imatinib; Localised; Long-term outcomes; Mutational analysis; Patient management; Tyrosine kinase inhibitor