Given the limitations of standard therapies and the lack of robust long-term data, this study aimed to evaluate the real-world efficacy and safety of rituximab in patients with neuropsychiatric systemic lupus erythematosus (NPSLE) treated at a single center.

We conducted a retrospective analysis of NPSLE patients treated with rituximab at Peking Union Medical College Hospital (PUMCH) from 2015 to 2025. Demographic characteristics, clinical manifestations, laboratory findings, Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K), SLEDAI with NP items removed, SLICC/ACR damage index (SDI), treatments, and clinical outcomes were systematically evaluated at baseline and followed up over 36 months. Changes in NP events from onset to follow-up were evaluated at each visit using a physician-reported 7-point Likert scale (1=patient demise, 2=much worse, 3=worse, 4=no change, 5=improved, 6=much improved, 7=resolved).

A total of 32 patients were included. The majority were female (90.6%), with a mean age of 31.41 ± 8.75 years old. The mean follow-up duration was 19.91 ± 13.69 months. 71.9% of patients presented with new-onset NPSLE, while 28.1% had recurrent NPSLE. 87.5% of patients had central nervous system (CNS) involvement, with the most frequent subtypes being cerebrovascular disease (34.4%), acute confusional state (15.6%), and myelopathy (15.6%). Peripheral nervous system involvement was present in 25.0% of patients. Previous treatments include glucocorticoids (84.4%), hydroxychloroquine (62.5%), cyclophosphamide (25.0%), and mycophenolate mofetil (34.4%). The average dose of rituximab at baseline was 1265.63 ± 666.53 mg. Following rituximab treatment, B cell depletion was achieved and maintained. In parallel, a significant and sustained improvement was observed in clinical outcome scores (baseline: 2.31 ± 0.47; month 36: 5.58 ± 1.08, p < 0.001), alongside significant reductions in both SLEDAI-2K (baseline: 15.59 ± 7.55; month 36: 2.27 ± 2.97, p < 0.0001) and SLEDAI with NP items removed (baseline: 8.09 ± 5.41; month 36: 0.82 ± 0.98, p < 0.0001) at all follow-up time points (p<0.001). Organ remission rates achieved ≥50% for all the involved systems. Significant improvements were also observed in key laboratory parameters, including elevated platelet counts, hemoglobin levels, increased complement levels (C3 and C4), alongside reduced anti-dsDNA antibody titers and erythrocyte sedimentation rate (ESR) (p<0.05). The maximum daily glucocorticoid dose (prednisone-equivalent) significantly decreased from 703.75 ± 428.30 mg at baseline—reflecting pulse or high-dose therapy—to 6.59 ± 7.18 mg at 36 months (p < 0.001). No significant adverse effects attributable to rituximab were observed.  

In this real-world cohort with long-term follow-up, rituximab was associated with sustained clinical improvement and an acceptable safety profile in patients with NPSLE.