The Development and Preliminary Validation of the CTCL-PRO-20: a Novel Quality of Life Survey for Patients with Cutaneous T-cell Lymphoma

Cutaneous T-cell lymphoma (CTCL) is a rare disease with a high symptom burden, negatively impacting patients’ quality of life (QoL). There is no agreed-upon CTCL-specific metric to measure QoL in CTCL. Thus, we created a novel questionnaire to fill this gap. Eighty CTCLpatients participated in this cross-sectional survey study. An iterative process of principal components analysis (PCA) yielded a 5-component solution with 20 items; this patient-reported outcomes (PRO) questionnaire, the CTCL-PRO-20, had high internal-consistency reliability (Cronbach’s α=0.93). Components measured self-consciousness (α =.94), treatment burden (α=.92), lack of CTCL resources (α=.74), sleep/fatigue (α=.87), and skin concerns (α=.88). High Pearson correlations were observed between the CTCL-PRO-20 and QoL measures more relevant to patients’ experiences (e.g., MF/SS-CTCL QoL and Skindex-29); lower correlations were observed with the generic-QoL (SF-36) and single-focused (e.g., sleep-disturbance) measures. Patients with stable/no active disease (vs. progressive disease) reported significantly lower treatment-burden scores (better QoL). Patients who received systemic and topical treatments (vs. no treatment) reported significantly higher CTCL-PRO-20 scores (poorer QoL). This single-center study provides preliminary evidence of construct and convergent validity of the CTCL-PRO-20 and its components, warranting further validation in a well-powered study to examine its sensitivity to change after treatment intervention.