학술논문

Validation of prognostic scoring and assessment of clinical benefit for patients with bone sarcomas enrolled in phase I clinical trials
Document Type
article
Source
Oncotarget. 7(39)
Subject
Biomedical and Clinical Sciences
Clinical Sciences
Oncology and Carcinogenesis
Cancer
Clinical Trials and Supportive Activities
Clinical Research
Rare Diseases
Adolescent
Adult
Aged
Bone Neoplasms
Child
Chondrosarcoma
Clinical Decision-Making
Clinical Trials
Phase I as Topic
Decision Support Techniques
Female
Humans
Kaplan-Meier Estimate
Male
Middle Aged
Osteosarcoma
Patient Selection
Predictive Value of Tests
Proportional Hazards Models
Reproducibility of Results
Retrospective Studies
Risk Assessment
Risk Factors
Sarcoma
Ewing
Time Factors
Treatment Outcome
Young Adult
bone sarcoma
phase 1 trials
prognosis scores
Ewing sarcoma
osteosarcoma
Oncology and carcinogenesis
Language
Abstract
BackgroundWe sought to validate the Royal Marsden Hospital (RMH) and MD Anderson Cancer Center (MDACC) prognostic scoring systems for the selection of bone sarcoma patients for phase I clinical trials and to identify additional risk factors related to survival.Patients and methodsWe retrospectively reviewed the baseline characteristics and outcomes of 92 bone sarcoma patients who were referred to MDACC's Phase I Clinical Trials Program.ResultsNinety-two patients with Ewing sarcoma (N = 47), osteosarcoma (N = 22), chondrosarcoma (N = 16), and other tumors (N = 7) were evaluated; 78 were enrolled in at least 1 of 43 different phase I trials. The median overall survival (OS) was 8.8 months (95% confidence interval [CI] = 6.8-13.7 months). Independent factors that predicted shorter survival were male sex, >2 metastatic sites, >3 previous therapies, hemoglobin level 200 x103/L, creatinine level ≥1.3 mg/dL, and lactate dehydrogenase level >ULN. Patients with good RMH scores (0-1) had longer OS than patients with poor RMH scores (2-3) (HR = 5.8, 95% CI = 2.9-11.0; P < 0.0001), as did patients with low MDACC scores (0-1) as compared to patients with higher MDACC scores (2-4) (HR = 3.2, 95% CI = 1.9-5.6; P < 0.0001).ConclusionThe RMH prognostic score can be used to predict the OS of bone cancer patients referred for phase I trials. The MDACC score added no value to the RMH score and therefore does not have a role in assessment of patients with bone tumors. Patients with advanced bone sarcomas should be considered for phase I trials.