title: Clinical outcomes of patients with advanced gastrointestinal stromal tumors: safety and efficacy in a worldwide treatment-use trial of sunitinib.
abstract: The objectives of this study were to provide sunitinib to patients with gastrointestinal stromal tumor (GIST) who were otherwise unable to obtain it and to collect broad safety and efficacy data from a large population of patients with advanced GIST after imatinib failure. (ClinicalTrials.gov identifier NCT00094029).
  
  
  
  Imatinib-resistant/intolerant patients with advanced GIST received sunitinib on an initial dosing schedule of 50 mg daily in 6-week cycles (4 weeks on treatment, 2 weeks off treatment). Tumor assessment frequency was according to local practice, and response was assessed by investigators according to Response Evaluation Criteria in Solid Tumors version 1.0. Overall survival (OS) and safety were assessed regularly. Post hoc analyses evaluated different patterns of treatment management.
  
  
  
  At final data cutoff, 1124 patients comprised the intent-to-treat population, and 15% of these patients had a baseline Eastern Cooperative Oncology Group performance status ≥2. The median treatment duration was 7.0 months. The median time to tumor progression was 8.3 months (95% confidence interval [CI], 8.0-9.4 months), the median OS was 16.6 months (95% CI, 14.9-18.0 months), and 36% of patients were alive at the time of analysis. Patients for whom the initial dosing schedule was modified exhibited longer median OS (23.5 months) than those who were treated strictly according to the initial dosing schedule (11.1 months). The most common treatment-related grade 3 and 4 adverse events were hand-foot syndrome (11%), fatigue (9%), neutropenia (8%), hypertension (7%), and thrombocytopenia (6%). Treatment-related adverse events associated with cardiac function (eg, congestive heart failure and myocardial infarction) were reported at frequencies of ≤1% each.
  
  
  
  This treatment-use study confirms the long-term safety and efficacy of sunitinib in a large international population of patients with advanced GIST after imatinib failure.
authors: Department of Interdisciplinary Oncology, HELIOS Clinic Berlin-Buch, Berlin, Germany.
authors: Peter
authors: P
authors: Reichardt
authors: Yoon-Koo
authors: YK
authors: Kang
authors: Piotr
authors: P
authors: Rutkowski
authors: Jochen
authors: J
authors: Schuette
authors: Lee S
authors: LS
authors: Rosen
authors: Beatrice
authors: B
authors: Seddon
authors: Suayib
authors: S
authors: Yalcin
authors: Hans
authors: H
authors: Gelderblom
authors: Charles C
authors: CC
authors: Williams
authors: Elena
authors: E
authors: Fumagalli
authors: Guido
authors: G
authors: Biasco
authors: Herbert I
authors: HI
authors: Hurwitz
authors: Pamela E
authors: PE
authors: Kaiser
authors: Kolette
authors: K
authors: Fly
authors: Ewa
authors: E
authors: Matczak
authors: Liang
authors: L
authors: Chen
authors: Maria José
authors: MJ
authors: Lechuga
authors: George D
authors: GD
authors: Demetri
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D020533:Angiogenesis Inhibitors / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D003967:Diarrhea / Q000139:chemically induced; D018572:Disease-Free Survival; D005221:Fatigue / Q000139:chemically induced; D005260:Female; D005770:Gastrointestinal Neoplasms / Q000188:drug therapy* / Q000401:mortality; D046152:Gastrointestinal Stromal Tumors / Q000188:drug therapy* / Q000401:mortality; D006801:Humans; D007211:Indoles / Q000009:adverse effects / Q000627:therapeutic use*; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D016016:Proportional Hazards Models; D011758:Pyrroles / Q000009:adverse effects / Q000627:therapeutic use*; D012509:Sarcoma / Q000188:drug therapy / Q000401:mortality; D000077210:Sunitinib; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D020533:Angiogenesis Inhibitors; D007211:Indoles; D011758:Pyrroles; D000077210:Sunitinib
keywords: efficacy; gastrointestinal stromal tumor; long-term safety; sunitinib; treatment-use trial; worldwide
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