{
  "abstract": "Objectives To assess the patient characteristics and treatment patterns, effectiveness, safety and patient-reported outcomes (PROs) of sarilumab in routine clinical practice, used as monotherapy or in combination with conventional synthetic disease-modifying antirheumatic drugs (DMARDs) in patients with rheumatoid arthritis (RA) in Japan.Methods Prospective Sarilumab (Prefilled Syringe/Pen) Observational Study–Japan (PROFILE-J) was a 52-week, prospective, non-interventional, observational study conducted across Japan. Adult patients with RA initiating sarilumab were enrolled. Effectiveness endpoints included Clinical Disease Activity Index (CDAI) scores, disease activity and PROs. Safety endpoints included treatment-emergent adverse events (TEAEs) and other clinically relevant safety outcomes.Results Among 1998 patients (monotherapy, n=567; combination, n=1431), the mean age was 66.6 years, with 63.2% aged ≥65 years and 67.9% naïve to biologic DMARDs. Sarilumab retention at week 52 was 67.5%. Effectiveness analysis population included 1715 patients (monotherapy, n=461; combination, n=1254). Baseline mean CDAI scores (21.2) decreased to low disease activity range (9.7) at week 12; mean changes from baseline were −13.6 and −13.1 at week 24 and –14.2 and −14.9 at week 52 for monotherapy and combination therapy respectively. Other measures, including PROs, showed similar improvements in both groups. In the safety population, TEAEs occurred in 44.6% of patients, with a slightly lower rate observed in monotherapy (40.2%) than in combination therapy (46.3%).Conclusions This study described real-world treatment patterns of sarilumab, highlighting frequent initiation in biologic DMARD-naïve patients, particularly among older adults. Sarilumab demonstrated sustained effectiveness and a consistent safety profile with no new safety signals, supporting its use as monotherapy or combination therapy.Trial registration number jRCT1071190016.",
  "authors": [
    {
      "affiliations": [
        "Department of Molecular Targeted Therapeutics, University of Occupational and Environmental Health, Kitakyushu, Japan"
      ],
      "name": "Yoshiya Tanaka"
    },
    {
      "affiliations": [
        "The First Department of Internal Medicine, University of Occupational and Environmental Health, Kitakyushu, Japan"
      ],
      "name": "Kentaro Hanami"
    },
    {
      "affiliations": [
        "The First Department of Internal Medicine, University of Occupational and Environmental Health, Kitakyushu, Japan"
      ],
      "name": "Yusuke Miyazaki"
    },
    {
      "affiliations": [
        "Yokohama Rheumatology Clinic, Yokohama, Japan"
      ],
      "name": "Yoshiaki Ishigatsubo"
    },
    {
      "affiliations": [
        "Department of Allergy and Clinical Immunology, Graduate School of Medicine, Chiba University, Chiba, Japan"
      ],
      "name": "Taro Iwamoto"
    },
    {
      "affiliations": [
        "Rheumatology, Ushio Clinic, Osaka, Japan"
      ],
      "name": "Kazuyasu Ushio"
    },
    {
      "affiliations": [
        "Division of Rheumatology, Department of Medicine, National Hospital Organisation Tokyo Medical Center, Meguro, Japan"
      ],
      "name": "Yutaka Okano"
    },
    {
      "affiliations": [
        "Rheumatology, Onobori Clinic, Tsukuba, Japan"
      ],
      "name": "Makoto Onobori"
    },
    {
      "affiliations": [
        "Department of Rheumatology and Allergology, St Marianna University School of Medicine, Kawasaki, Japan"
      ],
      "name": "Kimito Kawahata"
    },
    {
      "affiliations": [
        "Department of Rheumatology and Clinical Immunology, Kobe University Hospital, Kobe, Japan"
      ],
      "name": "Jun Saegusa"
    },
    {
      "affiliations": [
        "Orthopedic Surgery, Showa Inan General Hospital, Komagane, Japan"
      ],
      "name": "Yukio Nakamura"
    },
    {
      "affiliations": [
        "Rheumatic Disease Center, Mabi Memorial Hospital, Kurashiki, Japan"
      ],
      "name": "Masamitsu Natsumeda"
    },
    {
      "affiliations": [
        "Department of Hematology and Rheumatology, Kindai University Faculty of Medicine Graduate School of Medical Sciences, Osakasayama, Japan"
      ],
      "name": "Yuji Nozaki"
    },
    {
      "affiliations": [
        "Department of Rheumatology, Seirei Hamamatsu General Hospital, Hamamatsu, Japan"
      ],
      "name": "Toshiaki Miyamoto"
    },
    {
      "affiliations": [
        "Rheumatology, Hiroshima Rheumatology Clinic, Hiroshima, Japan"
      ],
      "name": "Yuji Yamanishi"
    },
    {
      "affiliations": [
        "Aichi Developmental Disability Center, Kasugai, Japan"
      ],
      "name": "Naoki Ishiguro"
    },
    {
      "affiliations": [
        "Department of Respiratory Medicine and Clinical Immunology, Osaka University Graduate School of Medicine, Suita, Japan"
      ],
      "name": "Atsushi Kumanogoh"
    },
    {
      "affiliations": [
        "Saitama Medical University, Iruma, Japan",
        "Keio University School of Medicine, Shinjuku, Japan"
      ],
      "name": "Tsutomu Takeuchi"
    },
    {
      "affiliations": [
        "National Hospital Organisation Tokyo National Hospital, Kiyose, Japan"
      ],
      "name": "Shigeto Tohma"
    },
    {
      "affiliations": [
        "Department of Environmental Epidemiology, University of Occupational and Environmental Health, Kitakyushu, Japan"
      ],
      "name": "Yoshihisa Fujino"
    },
    {
      "affiliations": [
        "Division of Rheumatology, Faculty of Medicine, Toho University, Meguro, Japan"
      ],
      "name": "Hideto Kameda"
    }
  ],
  "title": "Real-world effectiveness, safety and patient-reported outcomes of sarilumab in patients with rheumatoid arthritis: a 52-week, prospective, open-label, non-interventional, observational, nationwide, large-cohort study in Japan (PROFILE-J)",
  "uid": "8540b0b6-e732-5415-b854-79e17b2bdfe9"
}