Efficacy of first-line tocilizumab therapy in early polymyalgia rheumatica: a prospective longitudinal study

• Published in: Annals of the rheumatic diseases Vol. 75; no. 8; pp. 1506 - 1510
• Main Authors: Devauchelle-Pensec, Valérie, Berthelot, Jean Marie, Cornec, Divi, Renaudineau, Yves, Marhadour, Thierry, Jousse-Joulin, Sandrine, Querellou, Solène, Garrigues, Florent, De Bandt, Michel, Gouillou, Maelenn, Saraux, Alain
• Format: Journal Article
• Language: English
• Published: England BMJ Publishing Group 01.08.2016
• Series: Concise report
• Subjects: Aged; Antibodies, Monoclonal, Humanized - administration & dosage; Antibodies, Monoclonal, Humanized - adverse effects; Antibodies, Monoclonal, Humanized - therapeutic use; Antirheumatic Agents - administration & dosage; Antirheumatic Agents - adverse effects; Antirheumatic Agents - therapeutic use; Clinical and Epidemiological Research; Drug Administration Schedule; Drug Therapy, Combination; Female; Glucocorticoids - adverse effects; Glucocorticoids - therapeutic use; Human health and pathology; Humans; Infusions, Intravenous; Life Sciences; Longitudinal Studies; Male; Middle Aged; Polymyalgia Rheumatica - diagnostic imaging; Polymyalgia Rheumatica - drug therapy; Positron Emission Tomography Computed Tomography; Prednisone - adverse effects; Prednisone - therapeutic use; Prospective Studies; Rhumatology and musculoskeletal system; Severity of Illness Index; Treatment Outcome; DMARDs (biologic); Magnetic Resonance Imaging; Disease Activity; Polymyalgia Rheumatica; Treatment; tocilizumab; polymyalgia rheumatica
• ISSN: 0003-4967; 1468-2060
• DOI: 10.1136/annrheumdis-2015-208742

BackgroundGlucocorticoids are the cornerstone treatment of polymyalgia rheumatica (PMR) but induce adverse events.ObjectivesTo evaluate the efficacy and safety of first-line tocilizumab in PMR.MethodsIn a prospective open-label study (ClinicalTrials.gov: NCT01713842), 20 glucocorticoid-free patients fulfilling Chuang's PMR criteria, with symptom onset within the last 12 months and a PMR activity score (PMR-AS) >10, each received three tocilizumab infusions at 4-week intervals, without glucocorticoids, followed by oral prednisone from weeks 12 to 24 (0.15 mg/kg if PMR-AS ≤10 and 0.30 mg/kg otherwise). The primary end point was the proportion of patients with PMR-AS≤10 at week 12.ResultsBaseline median PMR-AS was 36.6 (IQR 30.4–43.8). At week 12, all patients had PMR-AS≤10 and received the low prednisone dosage. Median PMR-AS at weeks 12 and 24 was 4.5 (3.2–6.8) and 0.95 (IQR 0.4–2), respectively (p<0.001 vs baseline for both time points). No patient required rescue treatment. Positron emission tomography-CT showed significant improvements. The most common adverse events were transient neutropenia (n=3) and leucopenia (n=5); in one patient, the second tocilizumab infusion was omitted due to leucopenia.ConclusionsTocilizumab monotherapy is effective in recent-onset PMR. Randomised controlled trials are warranted.Trial registration numberNCT01713842.