Background

The Advanced Systemic Mastocytosis Symptom Assessment Form (AdvSM-SAF) was developed to evaluate symptoms of advanced systemic mastocytosis (AdvSM). This study aimed to psychometrically evaluate AdvSM-SAF scores and provide score interpretation guidelines.

Methods

The 10-item AdvSM-SAF was administered daily (scored as a seven-day average) in EXPLORER, an open-label Phase 1 study in AdvSM. Score distribution, reliability, construct-related validity, sensitivity to change, and interpretation guidelines were evaluated for AdvSM-SAF items, gastrointestinal symptom score (GSS), skin symptom score (SSS), and total symptom score (TSS).

Results

Thirty-one patients contributed to the analyses. At Baseline, the GSS, SSS, and TSS had adequate internal consistency (α > 0.7) and test-retest reliability (intraclass correlation coefficients >0.7). AdvSM-SAF scores were moderately to strongly correlated with variables as expected, and distinguished among clinically distinct groups. Observed relationships between change scores in the AdvSM-SAF and other assessments reflect evidence that AdvSM-SAF scores change in concert with other assessments designed to measure similar constructs. The magnitude of AdvSM-SAF weekly TSS mean change scores based on different anchor groupings was as expected (improvement > stable > worsening). Candidate clinically meaningful between-group difference estimates (GSS = 2–4, SSS = 2–3, and TSS = 4–7 points) and within-person change estimates (GSS = 6–9, SSS = 1–4, TSS = 9–14) for AdvSM-SAF weekly scores were generated.

Conclusion

The AdvSM-SAF produced reliable, construct-valid, and sensitive scores when administered in the target patient population. These results, along with its strong development history and evidence of content validity, indicate that the AdvSM-SAF is fit for the purpose of measuring treatment benefit in individuals with AdvSM.

中文翻译：

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高级系统性肥大细胞增多症症状评估表 (AdvSM-SAF) 的心理测量学评估

背景

高级全身性肥大细胞增多症症状评估表 (AdvSM-SAF) 旨在评估晚期全身性肥大细胞增多症 (AdvSM) 的症状。本研究旨在从心理测量角度评估 AdvSM-SAF 分数并提供分数解释指南。

方法

EXPLORER 是一项 AdvSM 的开放标签 1 期研究，每天使用 10 项 AdvSM-SAF（评分为 7 天平均值）。对 AdvSM-SAF 项目、胃肠道症状评分 (GSS)、皮肤症状评分 (SSS) 和总症状评分 (TSS) 的评分分布、可靠性、结构相关的有效性、对变化的敏感性和解释指南进行了评估。

结果

31 名患者参与了分析。在基线时，GSS、SSS 和 TSS 具有足够的内部一致性（α > 0.7）和重测信度（组内相关系数 > 0.7）。AdvSM-SAF 评分与预期的变量中度至强相关，并在临床不同的组中进行区分。在 AdvSM-SAF 和其他评估中观察到的变化分数之间的关系反映了 AdvSM-SAF 分数与旨在衡量类似结构的其他评估一致变化的证据。基于不同锚定分组的 AdvSM-SAF 每周 TSS 平均变化得分的幅度如预期（改善 > 稳定 > 恶化）。候选具有临床意义的组间差异估计（GSS = 2-4、SSS = 2-3 和 TSS = 4-7 分）和人内变化估计（GSS = 6-9，

结论

AdvSM-SAF 在目标患者人群中使用时产生了可靠的、结构有效的和敏感的评分。这些结果，连同其强大的发展历史和内容有效性的证据，表明 AdvSM-SAF 适合用于衡量 AdvSM 患者的治疗益处。