Efficacy and safety of a step-down regimen of low dosage of glucocorticoids combined with early administration of synthetic or biologic immunosuppressants in anti-synthetase syndrome: A pilot study

Semin Arthritis Rheum. 2024 Dec:69:152560. doi: 10.1016/j.semarthrit.2024.152560. Epub 2024 Sep 24.

Abstract

Introduction: Anti-synthetase syndrome (ASS) is a rare autoimmune disease characterized by the presence of anti-aminoacyl-transfer-RNA synthetase antibodies (ARS) and the involvement of muscles, skin, joints, and lungs. Despite increasing interest and evidence, optimal clinical management remains unclear due to a lack of randomized control trials. This study aims to evaluate the efficacy and safety of a treatment regimen involving early co-administration of glucocorticoids and immunosuppressants, with rapid prednisone tapering.

Materials and methods: We prospectively enrolled patients referred to our multidisciplinary "Myositis Clinic" with a diagnosis of ASS. Clinical, serological, instrumental and medications data were collected at baseline and at 6 and 12 months follow-up. According to treatment protocol, patients were treated with traditional synthetic immunosuppressants or rituximab (RTX) depending on clinical manifestations. Prednisone (PDN) was gradually tapered and eventually discontinued within 6 or 12 months.

Results: A total of twenty-seven subjects were enrolled: arthritis, myositis and ILD were assessed in 9, 16 and 18 patients, respectively, and all of them had an active disease. RTX was administered after methotrexate (MTX) in 4 cases of refractory joint involvement and co-administration of a second immunosuppressant was necessary in 2 patients. When muscle involvement was present, first-line therapy was MTX, followed by mycophenolate mofetil (MMF) or RTX, which allowed to achieve low disease activity or remission, respectively. Eight ILD-patients were treated with MMF and switched to RTX in 5 cases of inefficacy, but all patients were in clinical remission at the end of follow-up. At 12 months, 12 patients discontinued PDN.

Conclusions: This study is the first to prospectively report on the efficacy and safety of a stepwise, steroid-sparing treatment ASS encompassing various domains. MTX, as well as other synthetic immunosuppressants, showed limited efficacy in ASS-related arthritis, while RTX emerged as a promising option. This study recommends early RTX use in case of arthritis, suggesting it as a pivotal treatment for ILD too, and raises questions regarding maintenance therapy and treatment-free remission.

Keywords: Anti-synthethase syndrome; Arthritis; Glucocorticoids; Interstitial lung disease; Myositis; Rituximab.

MeSH terms

  • Adult
  • Aged
  • Drug Tapering
  • Drug Therapy, Combination*
  • Female
  • Glucocorticoids* / administration & dosage
  • Glucocorticoids* / therapeutic use
  • Humans
  • Immunosuppressive Agents* / administration & dosage
  • Immunosuppressive Agents* / therapeutic use
  • Male
  • Methotrexate / administration & dosage
  • Methotrexate / therapeutic use
  • Middle Aged
  • Myositis* / drug therapy
  • Pilot Projects
  • Prednisone / administration & dosage
  • Prednisone / therapeutic use
  • Prospective Studies
  • Rituximab / administration & dosage
  • Rituximab / adverse effects
  • Rituximab / therapeutic use
  • Treatment Outcome

Substances

  • Immunosuppressive Agents
  • Glucocorticoids
  • Rituximab
  • Prednisone
  • Methotrexate

Supplementary concepts

  • Antisynthetase syndrome