Tocilizumab in Refractory ASS-ILD

NCT07662265 | Completed DMC FDA Drug

• 1 other identifier: 2026-KY-238
• Study Type: observational
• Target: 111
• Locations: 1 country
• Sites: 1

Brief Summary

Refractory anti-synthetase syndrome-associated interstitial lung disease (ASyS-ILD) lacks targeted therapy. Interleukin-6 drives both inflammation and fibrosis. We evaluated the real-world efficacy and safety of tocilizumab, an IL-6 receptor antagonist. This single-center retrospective cohort study included patients with refractory ASyS-ILD treated between January 2020 and July 2025. Tocilizumab recipients were compared with those receiving standard of care (SOC) immunosuppressants. Overlap weighting based on propensity scores was used to balance 11 baseline variables. The primary outcome was improvement in symptoms and HRCT findings at ≥3 months. Secondary outcomes included changes in biomarkers and pulmonary function, progression-free survival (PFS), and adverse events. Cox regression identified independent predictors of symptom progression. Generalized additive models (GAM) explored nonlinear relationships, and XGBoost-SHAP machine learning assessed variable importance.

Conditions

Antisynthetase Syndrome; Tocilizumab

Keywords

ASyS-ILD; tocilizumab; treat

Outcome Measures

Primary Outcomes (1)

• Disease progression

  Disease progression is classified data, which included the number of patients with symptoms progression（Symptom progression was defined as worsening of dyspnea, cough or rash requiring escalation of therapy or hospitalization） or with the worsening of pattern demonstrated on CT scans (defined as an increase in the extent of reticulation, traction bronchiectasis, or honeycombing on follow-up HRCT ) or the number of the patients with FVC% decline \>10% or with DLCO% decline \>15%.

  From the start of tocilizumab treatment or second-line treatment to disease progression, the longest possible duration is until October 31, 2025.

Secondary Outcomes (8)

• serum test of CK

  From the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

• Serum test of ferritin

  From the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

• Serum test of IL6

  From the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

• Serum test of LDH

  From the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

• Serum test of CRP

  From the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

Study Arms (1)

Refractory anti-synthetase syndrome-associated interstitial lung disease

Inclusion criteria were: (1) age ≥18 years; (2) fulfillment of the 2017 EULAR/ACR classification criteria for ASyS; (3) HRCT-confirmed ILD; (4) refractory disease, defined as active disease despite prior treatment with glucocorticoids and at least one first-line immunosuppressant (including methotrexate, mycophenolate mofetil, azathioprine, cyclosporine, cyclophosphamide, baricitinib, or upadacitinib); and (5) availability of baseline and follow-up clinical data at ≥3 months. Exclusion criteria were: (1) pregnancy or lactation; (2) pre-existing psychiatric disorders that would preclude study participation; (3) prior lung transplantation; and (4) missing key outcome data.

Drug: Tociliuzumab

Interventions

Tociliuzumab DRUG

Tocilizumab, a humanized monoclonal antibody against the IL-6 receptor, is approved for the treatment of rheumatoid arthritis, juvenile idiopathic arthritis, and giant cell arteritis. In patients with rheumatoid arthritis-associated ILD, tocilizumab has been shown to reduce KL-6 levels, a biomarker of alveolar epithelial injury. However, evidence for tocilizumab in ASyS-ILD is limited to isolated case reports, and no systematic evaluation of its efficacy and safety in this specific population has been published.

Refractory anti-synthetase syndrome-associated interstitial lung disease

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

We reviewed electronic medical records of all adult patients diagnosed with ASyS-ILD at our institution between January 1, 2020, and July 30, 2025. Inclusion criteria were: (1) age ≥18 years; (2) fulfillment of the 2017 EULAR/ACR classification criteria for ASyS; (3) HRCT-confirmed ILD; (4) refractory disease, defined as active disease despite prior treatment with glucocorticoids and at least one first-line immunosuppressant (including methotrexate, mycophenolate mofetil, azathioprine, cyclosporine, cyclophosphamide, baricitinib, or upadacitinib); and (5) availability of baseline and follow-up clinical data at ≥3 months. Exclusion criteria were: (1) pregnancy or lactation; (2) pre-existing psychiatric disorders that would preclude study participation; (3) prior lung transplantation; (4) missing key outcome data; (5) Patients with Anti-MDA5 dermatomyositis. Patients who received tocilizumab (8 mg/kg intravenously every 4 weeks) were assigned to the tocilizumab group (N=41). Patients who

You may qualify if:

• age ≥18 years;
• fulfillment of the 2017 EULAR/ACR classification criteria for ASyS;
• HRCT-confirmed ILD;
• refractory disease, defined as active disease despite prior treatment with glucocorticoids and at least one first-line immunosuppressant (including methotrexate, mycophenolate mofetil, azathioprine, cyclosporine, cyclophosphamide, baricitinib, or upadacitinib);
• availability of baseline and follow-up clinical data at ≥3 months.

You may not qualify if:

• pregnancy or lactation;
• pre-existing psychiatric disorders that would preclude study participation;
• prior lung transplantation;
• missing key outcome data;
• Patients with Anti-MDA5 dermatomyositis.

Sponsors & Collaborators

• Hu Yinan: lead

Study Sites (1)

China-Japan Friendship Hospital

Beijing, Beijing Municipality, 100000, China

Location

MeSH Terms

Conditions

Antisynthetase syndrome

Interventions

tocilizumab

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Attending Physician

Study Record Dates

• First Submitted: June 3, 2026
• First Posted: June 23, 2026
• Study Start: January 1, 2022
• Primary Completion: October 31, 2025
• Study Completion: October 31, 2025
• Last Updated: June 23, 2026

Record last verified: 2026-06

Locations

CN (1)