The majority of multicentric Castleman disease (MCD) patients in China are idiopathic MCD (iMCD) with systemic manifestations. The evaluation of symptomatic treatment response in iMCD currently relies on clinicians' assessments of patients' symptoms rather than patient-reported outcomes (PROs), which may be influenced by subjective judgments from physicians. PRO results in iMCD patients are rarely mentioned in current treatment response evaluations.

This study aims to comprehensively describe changes in symptom burden, quality of life, psychological status, and social function in iMCD patients before and after treatment using PROs, and analyze their relationship with treatment responses.

Patients with iMCD diagnosed and treated at Peking Union Medical College Hospital were included. PRO survey results (MCD-SS, SF-36, PHQ-9, and WPAI:GH) were collected from patients before and after treatment, along with clinical data, symptom, biochemical, and lymph node evaluation results. Paired analysis was used to observe changes in PRO results before and after treatment, which were then compared with treatment responses (biochemcial and symptomatic responses) evaluated according to Castleman Disease Collaborative Network (CDCN) criteria.

A total of 19 iMCD patients were included. At the baseline, the MCD-SS reported a median of 11 MCD-related clinical symptoms, with a baseline median total score of 3.30 and a median fatigue dimension score of 5.50. After treatment, both the total MCD-SS score and fatigue score decreased significantly (P: 0.022~0.049). In terms of overall health status, the median “vitality” dimension score of the SF-36 increased significantly 8~12 months after treatment (55.0 vs. 75.0, P = 0.0055), and 78.9% of patients self-reported their health status as “slightly better” or “much better” compared to baseline. The PHQ-9 survey indicated a median baseline score of 8.0, with 13 patients (68.4%) exhibiting depressive symptoms and 7 patients (36.8%) potentially having moderate-to-severe depression. After treatment, the proportion of moderate/severe depressive symptoms decreased to 15.8%. Regarding social function, the WPAI:GH survey showed that 47.7% of iMCD patients were unemployed before treatment, decreasing to 31.6% 4~6 months after treatment. PRO results were consistent with symptomatic and biochemical responses evaluated with CDCN criteria. For iMCD patients with clinical fatigue grades of G0, G1, and G2, the median MCD - SS fatigue scores were 2.0, 5.5, and 7.5 points respectively (P < 0.005). In patients who achieved complete symptomatic or biochemical remission, the depression - related scores, fatigue scores, and overall symptom scores decreased significantly (P: 0.0083~0.035).

Our study concluded that PROs, as measurable indicators of patient self-report, demonstrate similarity to objective treatment response evaluations and can serve as a supplementary component in response assessment.

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2025
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