Pediatric Oncology Branch, National Cancer Institute, Center for Cancer Research, National Institutes of Health, Bethesda, MD 20892, USA.
Biol Blood Marrow Transplant. 2013 Apr;19(4):632-9. doi: 10.1016/j.bbmt.2013.01.013. Epub 2013 Jan 20.
Between 2004 and 2010, 189 adult patients were enrolled on the National Cancer Institute's cross-sectional chronic graft-versus-host disease (cGVHD) natural history study. Patients were evaluated by multiple disease scales and outcome measures, including the 2005 National Institutes of Health (NIH) Consensus Project cGVHD severity scores. The purpose of this study was to assess the validity of the NIH scoring variables as determinants of disease severity in severely affected patients in efforts to standardize clinician evaluation and staging of cGVHD. Out of 189 patients enrolled, 125 met the criteria for severe cGVHD on the NIH global score, 62 of whom had moderate disease, with a median of 4 (range, 1-8) involved organs. Clinician-assigned average NIH organ score and the corresponding organ scores assigned by subspecialists were highly correlated (r = 0.64). NIH global severity scores showed significant associations with nearly all functional and quality of life outcome measures, including the Lee Symptom Scale, Short Form-36 Physical Component Scale, 2-minute walk, grip strength, range of motion, and Human Activity Profile. Joint/fascia, skin, and lung involvement affected function and quality of life most significantly and showed the greatest correlation with outcome measures. The final Cox model with factors jointly predictive for survival included the time from cGVHD diagnosis (>49 versus ≤49 months, hazard ratio [HR] = 0.23; P = .0011), absolute eosinophil count at the time of NIH evaluation (0-0.5 versus >0.5 cells/μL, HR = 3.95; P = .0006), and NIH lung score (3 versus 0-2, HR = 11.02; P < .0001). These results demonstrate that NIH organs and global severity scores are reliable measures of cGVHD disease burden. The strong association with subspecialist evaluation suggests that NIH organ and global severity scores are appropriate for clinical and research assessments, and may serve as a surrogate for more complex subspecialist examinations. In this population of severely affected patients, NIH lung score is the strongest predictor of poor overall survival, both alone and after adjustment for other important factors.
在 2004 年至 2010 年期间，189 名成年患者参与了美国国立癌症研究所的横断面慢性移植物抗宿主病（cGVHD）自然史研究。患者通过多种疾病量表和结果测量进行评估，包括 2005 年美国国立卫生研究院（NIH）共识项目 cGVHD 严重程度评分。本研究的目的是评估 NIH 评分变量作为严重受影响患者疾病严重程度的决定因素的有效性，以努力实现 cGVHD 临床评估和分期的标准化。在纳入的 189 名患者中，有 125 名患者根据 NIH 总体评分符合严重 cGVHD 的标准，其中 62 名患者患有中度疾病，中位数为 4（范围 1-8）个受累器官。临床医生分配的平均 NIH 器官评分与专科医生分配的相应器官评分高度相关（r=0.64）。NIH 总体严重程度评分与几乎所有功能和生活质量结局测量均有显著关联，包括 Lee 症状量表、健康调查简表 36 项躯体成分量表、2 分钟步行测试、握力、关节活动度和人体活动概况。关节/筋膜、皮肤和肺部受累对功能和生活质量的影响最大，与结局测量的相关性最强。对具有共同预测生存能力的因素进行的最终 Cox 模型包括从 cGVHD 诊断到 NIH 评估的时间（>49 个月与≤49 个月，危险比[HR]为 0.23；P=0.0011）、NIH 评估时绝对嗜酸性粒细胞计数（0-0.5 与>0.5 细胞/μL，HR 为 3.95；P=0.0006）和 NIH 肺部评分（3 与 0-2，HR 为 11.02；P<.0001）。这些结果表明，NIH 器官和总体严重程度评分是 cGVHD 疾病负担的可靠测量指标。与专科医生评估的强关联表明，NIH 器官和总体严重程度评分适用于临床和研究评估，并且可以作为更复杂的专科医生检查的替代指标。在这组严重受影响的患者中，NIH 肺部评分是总生存率不良的最强预测因素，无论是单独评估还是在调整其他重要因素后评估均如此。
