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Abstract：
OBJECTIVE: The GODDESS© tool was developed to assess Desmoid Tumor/Aggressive Fibromatosis (DT/AF) symptom severity and impact on patients\' lives. This study evaluated GODDESS©\'s cross-sectional and longitudinal measurement properties.
METHODS: The Phase 3, randomized placebo-controlled, DeFi study (NCT03785964) of nirogacestat in DT/AF was used to assess GODDESS©\'s reliability, construct validity, responsiveness, and estimate of meaningful change thresholds (MCTs). Other patient-reported outcome (PRO) measures included Patient Global Impression of Severity (PGIS) in DT/AF symptoms, EORTC QLQ-C30, Brief Pain Inventory Short Form, and PROMIS Physical Function short-form 10a v2.0 plus 3 items.
RESULTS: DeFi participants (N = 142) had a median age of 34 years (range: 18-76) and were mostly female (64.8%), with extra-abdominal (76.8%) or intra-abdominal tumors (23.2%). The GODDESS© symptom/impact scales showed internal consistency at baseline, cycles 4 and 7 (Cronbach\'s α > 0.70) and test-retest reliability (intra-class correlation coefficient > 0.85). GODDESS© scales correlated moderately to highly with PRO measures capturing similar content and differentiated among PGIS and Eastern Cooperative Oncology Group groups. GODDESS© scales detected improvement over time. For the total symptom score, a 1.30-point decrease was estimated as the within-person MCT and a 1.00-point decrease as the between-group MCT. For the physical functioning impact score, estimated within- and between-group MCTs were 0.60-point and 0.50-point decreases, respectively. Few participants exhibited symptom worsening.
CONCLUSIONS: GODDESS© was found to be reliable, valid, responsive, and interpretable as a clinical trial endpoint in the pooled sample of DT/AF patients. Estimated MCTs can be used to define responders and assess group-level differences in future, unblinded, efficacy analyses.
UNASSIGNED: NCT03785964; December 24, 2018.
METHODS: The Phase 3, randomized placebo-controlled, DeFi study (NCT03785964) of nirogacestat in DT/AF was used to assess GODDESS©\'s reliability, construct validity, responsiveness, and estimate of meaningful change thresholds (MCTs). Other patient-reported outcome (PRO) measures included Patient Global Impression of Severity (PGIS) in DT/AF symptoms, EORTC QLQ-C30, Brief Pain Inventory Short Form, and PROMIS Physical Function short-form 10a v2.0 plus 3 items.
RESULTS: DeFi participants (N = 142) had a median age of 34 years (range: 18-76) and were mostly female (64.8%), with extra-abdominal (76.8%) or intra-abdominal tumors (23.2%). The GODDESS© symptom/impact scales showed internal consistency at baseline, cycles 4 and 7 (Cronbach\'s α > 0.70) and test-retest reliability (intra-class correlation coefficient > 0.85). GODDESS© scales correlated moderately to highly with PRO measures capturing similar content and differentiated among PGIS and Eastern Cooperative Oncology Group groups. GODDESS© scales detected improvement over time. For the total symptom score, a 1.30-point decrease was estimated as the within-person MCT and a 1.00-point decrease as the between-group MCT. For the physical functioning impact score, estimated within- and between-group MCTs were 0.60-point and 0.50-point decreases, respectively. Few participants exhibited symptom worsening.
CONCLUSIONS: GODDESS© was found to be reliable, valid, responsive, and interpretable as a clinical trial endpoint in the pooled sample of DT/AF patients. Estimated MCTs can be used to define responders and assess group-level differences in future, unblinded, efficacy analyses.
UNASSIGNED: NCT03785964; December 24, 2018.
摘要：
目的：开发GODDESS©工具来评估纤维瘤/侵袭性纤维瘤病(DT/AF)症状的严重程度和对患者生活的影响。这项研究评估了GODDESS©的横截面和纵向测量特性。
方法：第三阶段，随机安慰剂对照，使用DT/AF中nirogacestat的DeFi研究（NCT03785964）来评估GODDESS©的可靠性，构造效度，响应性、响应性并估计有意义的变化阈值(MCT)。其他患者报告结果（PRO）指标包括DT/AF症状中的患者严重程度总体印象（PGIS），EORTCQLQ-C30，简短疼痛清单，和PROMIS物理功能缩写10av2.0加3个项目。
结果：DeFi参与者（N=142）的中位年龄为34岁（范围：18-76岁），大多数为女性（64.8％），腹外（76.8％）或腹内肿瘤（23.2％）。GODDESS©症状/影响量表在基线时显示出内部一致性，第4和第7周期(Cronbach'sα>0.70)和重测信度(类内相关系数>0.85)。GODDESS©量表与PRO指标的相关性中等到高度，这些指标捕获了相似的内容，并在PGIS和东部合作肿瘤学小组之间有所区分。GODDESS©量表检测到随着时间的推移而有所改善。对于总症状评分，估计人内MCT下降1.30点,组间MCT下降1.00点.对于身体功能影响评分，组内和组间MCT的估计值分别为0.60点和0.50点下降，分别。很少有参与者表现出症状恶化。
结论：发现GODDESS©是可靠的，有效,响应，在合并的DT/AF患者样本中可解释为临床试验终点。估计的MCT可用于定义响应者并评估未来的群体水平差异，未失明,功效分析。
■NCT03785964；2018年12月24日。
方法：第三阶段，随机安慰剂对照，使用DT/AF中nirogacestat的DeFi研究（NCT03785964）来评估GODDESS©的可靠性，构造效度，响应性、响应性并估计有意义的变化阈值(MCT)。其他患者报告结果（PRO）指标包括DT/AF症状中的患者严重程度总体印象（PGIS），EORTCQLQ-C30，简短疼痛清单，和PROMIS物理功能缩写10av2.0加3个项目。
结果：DeFi参与者（N=142）的中位年龄为34岁（范围：18-76岁），大多数为女性（64.8％），腹外（76.8％）或腹内肿瘤（23.2％）。GODDESS©症状/影响量表在基线时显示出内部一致性，第4和第7周期(Cronbach'sα>0.70)和重测信度(类内相关系数>0.85)。GODDESS©量表与PRO指标的相关性中等到高度，这些指标捕获了相似的内容，并在PGIS和东部合作肿瘤学小组之间有所区分。GODDESS©量表检测到随着时间的推移而有所改善。对于总症状评分，估计人内MCT下降1.30点,组间MCT下降1.00点.对于身体功能影响评分，组内和组间MCT的估计值分别为0.60点和0.50点下降，分别。很少有参与者表现出症状恶化。
结论：发现GODDESS©是可靠的，有效,响应，在合并的DT/AF患者样本中可解释为临床试验终点。估计的MCT可用于定义响应者并评估未来的群体水平差异，未失明,功效分析。
■NCT03785964；2018年12月24日。
