Tocilizumab in the treatment of severe and refractory parenchymal neuro-Behçet's syndrome: case series and literature review

Jinjing Liu; Dong Yan; Zhimian Wang; Yunjiao Yang; Shangzhu Zhang; Di Wu; Lingyi Peng; Zhichun Liu; Wenjie Zheng

doi:10.1177/1759720X20971908

Tocilizumab in the treatment of severe and refractory parenchymal neuro-Behçet's syndrome: case series and literature review

Ther Adv Musculoskelet Dis. 2020 Nov 16:12:1759720X20971908. doi: 10.1177/1759720X20971908. eCollection 2020.

Authors

Jinjing Liu¹, Dong Yan², Zhimian Wang¹, Yunjiao Yang¹, Shangzhu Zhang¹, Di Wu¹, Lingyi Peng¹, Zhichun Liu², Wenjie Zheng³

Affiliations

¹ Department of Rheumatology and Clinical Immunology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Key Laboratory of Rheumatology and Clinical Rheumatology, Ministry of Education, National Clinical Research Center for Dermatologic and Immunologic Diseases, Beijing, China.
² Department of Rheumatology and Immunology, The Second Affiliated Hospital of Soochow University, Suzhou, China.
³ Department of Rheumatology and Clinical Immunology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Key Laboratory of Rheumatology and Clinical Rheumatology, Ministry of Education, National Clinical Research Center for Dermatologic and Immunologic Diseases, No. 1 Shuafuyuan, Dongcheng District, Beijing, 100730, China.

Abstract

Objectives: This study aimed to investigate the efficacy and safety of tocilizumab (TCZ) in severe and refractory parenchymal neuro-Behçet's syndrome (p-NBS).

Methods: We retrospectively analyzed five patients with p-NBS treated with TCZ in our center between 2013 and 2020, and six cases from literature research with the index terms "neuro-Behçet's syndrome" and "tocilizumab" on PubMed NCBI.

Results: A total of 11 patients with p-NBS were enrolled (5 males, 6 females), with a mean age of 34.5 ± 8.0 years at the onset. All the patients had parenchymal neurological lesions, six patients (54.5%) suffered from multiple lesions, and nine patients (81.8%) were disabled. Before TCZ administration, all the patients had failed conventional therapy, eight patients (72.7%) received two or more immunosuppressants, and five patients showed insufficient response or intolerance to other biologics. TCZ was administrated at 8 mg/kg every 4 weeks, with background glucocorticoids (GCs) and immunosuppressants. After a median follow-up of 13 (interquartile range, 3.5-23.5) months, all the patients achieved both clinical and radiological improvements, and the Behçet's Disease Current Activity Form score improved significantly (3 versus 0, median, p = 0.004), the Rankin score also decreased (4 versus 2, median, p = 0.005). Levels of interleukin-6 in the cerebrospinal fluid decreased significantly in five patients (533.4 ± 389.7 pg/ml versus 34.5 ± 27.1 pg/ml, p = 0.048), after a median of two (interquartile range, 1-4) times of TCZ infusions. Furthermore, the GC dosage (per os) reduced from 69.2 ± 16.9 mg/d to 16.4 ± 16.2 mg/d (p = 0.000), and immunosuppressants were tapered in number and dosage in seven (63.6%) and four (36.3%) patients, respectively. No serious adverse events or deaths were observed during follow-up.

Conclusions: TCZ is well tolerated and effective in severe and refractory p-NBS, with a favorable GC- and immunosuppressant-sparing effect. Cerebrospinal fluid interleukin-6 might be used to monitor the effects of TCZ in p-NBS.

Keywords: Behçet’s syndrome; neurological involvement; tocilizumab.