Tolerance and efficacy of targeted therapies prescribed for off-label indications in refractory systemic autoimmune diseases: data of the first 100 patients enrolled in the TATA registry (TArgeted Therapy in Autoimmune Diseases). Issue 2 (1st November 2022)

• Record Type: Journal Article
• Authors: Gottenberg, Jacques-Eric; Chaudier, Aurore; Allenbach, Yves; Mekinian, Arsène; Amoura, Zahir; Cacoub, Patrice; Cornec, Divi; Hachulla, Eric; Quartier, Pierre; Melki, Isabelle; Richez, Christophe; Seror, Raphaele; Terrier, Benjamin; Devauchelle-Pensec, Valérie; Henry, Julien; Gatfosse, Marc; Bouillet, Laurence; Gaigneux, Emeline; Andre, Vincent; Baulier, Gildas; Saunier, Aurélie; Desmurs, Marie; Poulet, Antoine; Ete, Mathieu; Bienvenu, Boris; Truchetet, Marie-Elise; Michaud, Martin; Larroche, Claire; Dellal, Azeddine; Leurs, Amélie; Ottaviani, Sebastien; Nielly, Hubert; Vial, Guillaume; Jaussaud, Roland; Rouvière, Bénedicte; Jeandel, Pierre-Yves; Guffroy, Aurelien; Korganow, Anne-Sophie; Jouvray, Mathieu; Meyer, Alain; Chatelus, Emmanuel; Sordet, Christelle; Felten, Renaud; Sibilia, Jean; Litim-Ahmed-Yahia, Samira; Kleinmann, Jean-Francois; Mariette, Xavier;
• Is Part Of: RMD open. Volume 8:Issue 2(2022)
• Publication Date: 2022-11-01
• Subjects: Autoimmune Diseases -- Autoimmunity -- Biological Therapy; Musculoskeletal system -- Diseases -- Periodicals; Rheumatism -- Periodicals; 616.7005
• DOI: 10.1136/rmdopen-2022-002324
• Languages: English
• ISSNs: 2056-5933

Abstract

Abstract : Objectives: To assess the tolerance and efficacy of targeted therapies prescribed off-label in refractory low-prevalence autoimmune and inflammatory systemic diseases. Methods: The TATA registry (TArgeted Therapy in Autoimmune Diseases) is a prospective, observational, national and independent cohort follow-up. The inclusion criteria in the registry are as follows: age >18 years; low-prevalence autoimmune and inflammatory systemic disease treated with off-label drugs started after 1 January 2019. Results: Hundred (100) patients (79 women) were enrolled. The median age was 52.5 years (95% CI 49 to 56) and the median disease duration before enrolment was 5 years (3 to 7). The targeted therapies at enrolment were as follows: Janus kinase/signal transducers and activators of transcription inhibitors (44%), anti-interleukin (IL)-6R (22%), anti-IL-12/23, anti-IL-23 and anti-IL-17 (9%), anti-B cell activating factor of the tumour necrosis factor family (5%), abatacept (5%), other targeted treatments (9%) and combination of targeted treatments (6%). 73% of patients were receiving corticosteroid therapy at enrolment (median dose 10 mg/day). The current median follow-up time is 9 months (8 to 10). Safety: 11 serious infections (incidence rate of 14.8/100 patient-years) and 1 cancer (1.3 cancers/100 patient-years) were observed. Two patients died from severe COVID-19 (2.7 deaths/100 patient-years). Efficacy: the targeted treatment was considered effective by the clinician in 56% of patients and allowed, in responders, a median reduction of oral corticosteroids of 15 (9 to 21) mg/day, below 7.5 mg/day in 76% of patients, while 28% discontinued. Conclusion: These initial results of the TATA registry confirm the diversity of targeted treatments prescribed off-label in refractory autoimmune diseases and their corticosteroid-sparing effect when effective. Tolerance was acceptable in these refractory patients with a long history of treatment with immunosuppressive drugs.