Tocilizumab in refractory anti-synthetase syndrome-associated interstitial lung disease:a phenotype-stratified cohort study
- 1Department of Rheumatology; Beijing Key Laboratory of Precision Diagnosis, Treatment and Innovative Translation in Inflammatory Myopathies, China-Japan Friendship Hospital, No.2 Yinghua East Road, Chaoyang District, Beijing, 100029 China
Abstract
Background: Anti-synthetase syndrome-associated ILD (ASyS-ILD) spans a spectrum from active inflammation to established fibrosis; IL-6 pathway involvement supports a rationale for tocilizumab, yet phenotype-specific efficacy data are lacking.
Methods: Thirty-two patients with refractory ASyS-ILD received intravenous tocilizumab (8 mg/kg every 4 weeks) for ≥ 3 months. Patients were stratified into three phenotypes: glucocorticoid-dependent (n = 8, unable to taper prednisone below 15 mg/day without relapse), glucocorticoid-resistant (n = 18, failure to achieve remission despite ≥ 3 months of induction therapy), and progressive pulmonary fibrosis (PPF, n = 6, fulfilling ≥ 2 symptom/physiological/radiological progression criteria within 12 months on stable immunosuppression without active inflammation). Changes in lung function, high-resolution computed tomography (HRCT) scores and glucocorticoid dosage were analyzed using generalised estimating equations and paired tests.
Results: Improvements in forced vital capacity (FVC)% predicted, diffusing capacity for carbon monoxide (DLCO)% predicted, and HRCT scores were observed at 3 months and sustained at 6 months. Among the glucocorticoid-dependent group, 7/8 patients (87.5%) tapered prednisone to ≤ 10 mg/day. Sixteen of 18 glucocorticoid-resistant patients (88.9%) achieved treatment response. In the PPF subgroup, aggregate lung function did not improve from baseline; however, 5/6 patients (83.3%) showed FVC trajectories shifting from decline toward stabilisation or improvement (mean slope difference, + 0.81% predicted/month; 95% CI, -0.11 to 1.73; p = .073). No severe adverse events or treatment-related deaths occurred.
Conclusions: In this retrospective study, tocilizumab was associated with glucocorticoid tapering and reduced disease activity in glucocorticoid-dependent and -resistant patients. In the PPF subgroup, lung function did not change significantly on between-timepoint analysis, and a slope-based analysis suggested possible attenuation of decline that did not reach statistical significance. These hypothesis-generating findings provide a phenotype-based rationale for prospective controlled trials.
Supplementary Information: The online version contains supplementary material available at https://doi.org/10.1186/s12931-026-03839-4.
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BibTeX
@article{yang2026tocilizumab,
title = {{Tocilizumab in refractory anti-synthetase syndrome-associated interstitial lung disease:a phenotype-stratified cohort study}},
author = {Hanbo Yang and Yongpeng Ge and Sizhao Li and WenLi Li and Linrong He and Fang Chen and Wei Jiang and Xiaoqin Luo and Xiaoming Shu and Qinglin Peng and Guochun Wang and Xin Lu},
journal = {Respiratory Research},
year = {2026},
month = aug,
doi = {10.1186/s12931-026-03839-4},
url = {https://doi.org/10.1186/s12931-026-03839-4},
note = {CC BY-NC-ND 4.0},
}
RIS
TY - JOUR
TI - Tocilizumab in refractory anti-synthetase syndrome-associated interstitial lung disease:a phenotype-stratified cohort study
AU - Hanbo Yang
AU - Yongpeng Ge
AU - Sizhao Li
AU - WenLi Li
AU - Linrong He
AU - Fang Chen
AU - Wei Jiang
AU - Xiaoqin Luo
AU - Xiaoming Shu
AU - Qinglin Peng
AU - Guochun Wang
AU - Xin Lu
PY - 2026
DA - 2026/08/03
JO - Respiratory Research
DO - 10.1186/s12931-026-03839-4
UR - https://doi.org/10.1186/s12931-026-03839-4
AB - Background: Anti-synthetase syndrome-associated ILD (ASyS-ILD) spans a spectrum from active inflammation to established fibrosis; IL-6 pathway involvement supports a rationale for tocilizumab, yet phenotype-specific efficacy data are lacking. Methods: Thirty-two patients with refractory ASyS-ILD received intravenous tocilizumab (8 mg/kg every 4 weeks) for ≥ 3 months. Patients were stratified into three phenotypes: glucocorticoid-dependent (n = 8, unable to taper prednisone below 15 mg/day without relapse), glucocorticoid-resistant (n = 18, failure to achieve remission despite ≥ 3 months of induction therapy), and progressive pulmonary fibrosis (PPF, n = 6, fulfilling ≥ 2 symptom/physiological/radiological progression criteria within 12 months on stable immunosuppression without active inflammation). Changes in lung function, high-resolution computed tomography (HRCT) scores and glucocorticoid dosage were analyzed using generalised estimating equations and paired tests. Results: Improvements in forced vital capacity (FVC)% predicted, diffusing capacity for carbon monoxide (DLCO)% predicted, and HRCT scores were observed at 3 months and sustained at 6 months. Among the glucocorticoid-dependent group, 7/8 patients (87.5%) tapered prednisone to ≤ 10 mg/day. Sixteen of 18 glucocorticoid-resistant patients (88.9%) achieved treatment response. In the PPF subgroup, aggregate lung function did not improve from baseline; however, 5/6 patients (83.3%) showed FVC trajectories shifting from decline toward stabilisation or improvement (mean slope difference, + 0.81% predicted/month; 95% CI, -0.11 to 1.73; p = .073). No severe adverse events or treatment-related deaths occurred. Conclusions: In this retrospective study, tocilizumab was associated with glucocorticoid tapering and reduced disease activity in glucocorticoid-dependent and -resistant patients. In the PPF subgroup, lung function did not change significantly on between-timepoint analysis, and a slope-based analysis suggested possible attenuation of decline that did not reach statistical significance. These hypothesis-generating findings provide a phenotype-based rationale for prospective controlled trials. Supplementary Information: The online version contains supplementary material available at https://doi.org/10.1186/s12931-026-03839-4.
SN - 1465-9921
SN - 1465-993X
ER -