Memorial Sloan Kettering Cancer Center, New York, NY, USA.
SpringWorks Therapeutics, Inc., Stamford, CT, USA.
Qual Life Res. 2023 Oct;32(10):2861-2873. doi: 10.1007/s11136-023-03445-7. Epub 2023 Jun 22.
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.
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.
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.
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.
NCT03785964; December 24, 2018.
GODDESS 工具旨在评估韧带样纤维瘤病/侵袭性纤维瘤病（DT/AF）的症状严重程度及其对患者生活的影响。本研究评估了 GODDESS 的横断面和纵向测量特性。
采用 3 期、随机、安慰剂对照的 DeFi 研究（NCT03785964），对尼罗塞斯特治疗 DT/AF 的效果进行评估，以评估 GODDESS 的可靠性、结构有效性、反应性和有意义变化阈值（MCT）的估计值。其他患者报告的结果（PRO）测量指标包括 DT/AF 症状的患者整体印象严重程度（PGIS）、EORTC QLQ-C30、简短疼痛量表和 PROMIS 物理功能 10a v2.0 加 3 项。
DeFi 参与者（N=142）的中位年龄为 34 岁（范围：18-76），大多数为女性（64.8%），有腹外（76.8%）或腹内（23.2%）肿瘤。GODDESS 症状/影响量表在基线、第 4 周期和第 7 周期时显示出内部一致性（Cronbach's α>0.70）和测试-重测可靠性（组内相关系数>0.85）。GODDESS 量表与捕捉相似内容的 PRO 测量指标中度至高度相关，并区分了 PGIS 和东部肿瘤协作组（ECOG）的组别。GODDESS 量表检测到随时间的改善。对于总症状评分，估计 1.30 点的下降为个体内 MCT，1.00 点的下降为组间 MCT。对于身体功能影响评分，估计的个体内和组间 MCT 分别为 0.60 点和 0.50 点的下降。很少有参与者出现症状恶化。
在韧带样纤维瘤病/侵袭性纤维瘤病患者的汇总样本中，GODDESS 被发现具有可靠性、有效性、反应性和可解释性，可作为临床试验终点。估计的 MCT 可用于定义应答者，并在未来的、未设盲的、疗效分析中评估组间差异。
NCT03785964；2018 年 12 月 24 日。
