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).

Autor:	Gottenberg JE; Rheumatology, Hôpitaux universitaires de Strasbourg, Strasbourg, France jacques-eric.gottenberg@chru-strasbourg.fr.; Rheumatology, East and South-West Reference Center, Strasbourg, France., Chaudier A; Rheumatology, Hôpitaux universitaires de Strasbourg, Strasbourg, France.; Rheumatology, East and South-West Reference Center, Strasbourg, France., Allenbach Y; Department of Internal Medicine and Clinical Immunology, Hospital University Department: Inflammation, Immunopathology and Biotherapy (DHU i2B), University Hospital Pitié Salpêtrière, Paris, France.; Internal Medicine, Ile-De-France Reference Center, Paris, France., Mekinian A; Service de Médecine Interne, DHUi2B, Hospital Saint-Antoine, Paris, France., Amoura Z; Internal Medicine, University Hospital Pitié Salpêtrière, Paris, France.; Internal Medicine, Lupus and SAPL Reference Center, Paris, France., Cacoub P; Internal Medicine, Ile-De-France Reference Center, Paris, France.; Service de médecine interne et immunologie clinique, Hopital Pitie-Salpetriere, Paris, France., Cornec D; Rhumatologie, Cavale Blanche Hospital, Brest, France.; Rheumatology, North and North-West Reference Center, Brest, France., Hachulla E; Internal Medicine, Lille University School of Medicine, Lille, France.; Internal Medicine, North and North-West Reference Center, Lille, France., Quartier P; Pediatric Immuno-Hematology and Rheumatology Unit, Hopital universitaire Necker-Enfants malades, Paris, France.; Paediatric, RAISE Reference Center, Paris, France., Melki I; Laboratory of Neurogenetics and Neuroinflammation, Imagine Institute, Paris, France.; General Paediatrics, Infectious Diseases and Internal Medicine, Hopital Universitaire Robert Debre, Paris, France., Richez C; Service de Rhumatologie, CHU Bordeaux GH Pellegrin, Bordeaux, France.; Rheumatology, East and South-West Reference Center, Bordeaux, France., Seror R; Rheumatology, Hôpital Bicêtre, Le Kremlin-Bicetre, France.; Rheumatology, Ile-De-France Reference Center, Le Kremlin-Bicetre, France., Terrier B; Internal Medicine, Ile-De-France Reference Center, Paris, France.; Internal Medicine, Hospital Cochin, Paris, France., Devauchelle-Pensec V; Rhumatologie, Cavale Blanche Hospital, Brest, France.; Rheumatology, North and North-West Reference Center, Brest, France., Henry J; Rheumatology, Hôpital Bicêtre, Le Kremlin-Bicetre, France.; Rheumatology, Ile-De-France Reference Center, Le Kremlin-Bicetre, France., Gatfosse M; Médecine Interne, Hôpital Saint-Antoine, Paris, France., Bouillet L; Internal Medicine, University Hospital Centre Grenoble Alpes, Grenoble, France., Gaigneux E; Rheumatology, Centre Hospitalier Départemental Vendée, La Roche-sur-Yon, France., Andre V; Rheumatology, Centre Hospitalier Départemental Vendée, La Roche-sur-Yon, France., Baulier G; Internal Medicine, Centre Hospitalier de Périgueux, Perigueux, France., Saunier A; Internal Medicine, Centre Hospitalier de Périgueux, Perigueux, France., Desmurs M; Rheumatology, Hospital Emile Muller, Mulhouse, France., Poulet A; Internal Medicine, Saint Joseph Hospital, Marseille, France., Ete M; Saint Joseph Hospital, Marseille, France., Bienvenu B; Internal Medicine, Saint Joseph Hospital, Marseille, France., Truchetet ME; Rheumatology, East and South-West Reference Center, Bordeaux, France.; Rheumatology, University Hospital Centre Bordeaux, Bordeaux, France., Michaud M; Internal Medicine, Hopital Joseph Ducuing, Toulouse, France., Larroche C; Service de médecine interne, Hopital Avicenne, Bobigny, France., Dellal A; Rheumatology, Groupe Hospitalier Intercommunal Le Raincy-Montfermeil, Montfermeil, France., Leurs A; Internal Medicine, Centre Hospitalier de Dunkerque, Dunkerque, France., Ottaviani S; Rheumatology, Hospital Bichat-Claude-Bernard, Paris, France., Nielly H; Internal Medicine, Begin Armed Forces Training Hospital, Paris, France., Vial G; Department of Internal Medicine and Clinical Immunology, CHU de Bordeaux, Bordeaux, France., Jaussaud R; Barbois Hospital, Vandoeuvre-les-Nancy, France., Rouvière B; Internal Medicine, Cavale Blanche Hospital, Brest, France., Jeandel PY; Service de Médecine Interne, University Hospital Centre Nice, Nice, France., Guffroy A; Rheumatology, East and South-West Reference Center, Strasbourg, France.; Internal Medicine, Hôpitaux universitaires de Strasbourg, Strasbourg, France., Korganow AS; Rheumatology, East and South-West Reference Center, Strasbourg, France.; Internal Medicine, Hôpitaux universitaires de Strasbourg, Strasbourg, France., Jouvray M; Internal Medicine, Begin Armed Forces Training Hospital, Paris, France., Meyer A; Rheumatology, Hôpitaux universitaires de Strasbourg, Strasbourg, France.; Rheumatology, East and South-West Reference Center, Strasbourg, France., Chatelus E; Rheumatology, Hôpitaux universitaires de Strasbourg, Strasbourg, France.; Rheumatology, East and South-West Reference Center, Strasbourg, France., Sordet C; Rheumatology, Hôpitaux universitaires de Strasbourg, Strasbourg, France.; Rheumatology, East and South-West Reference Center, Strasbourg, France., Felten R; Rheumatology, Hôpitaux universitaires de Strasbourg, Strasbourg, France.; Rheumatology, East and South-West Reference Center, Strasbourg, France., Sibilia J; Rheumatology, Hôpitaux universitaires de Strasbourg, Strasbourg, France.; Rheumatology, East and South-West Reference Center, Strasbourg, France., Litim-Ahmed-Yahia S; Rheumatology, Hôpitaux universitaires de Strasbourg, Strasbourg, France., Kleinmann JF; Rheumatology, Hôpitaux universitaires de Strasbourg, Strasbourg, France.; Rheumatology, East and South-West Reference Center, Strasbourg, France., Mariette X; Rheumatology, Hôpital Bicêtre, Le Kremlin-Bicetre, France.; Rheumatology, Ile-De-France Reference Center, Le Kremlin-Bicetre, France.
Jazyk:	angličtina
Zdroj:	RMD open [RMD Open] 2022 Nov; Vol. 8 (2).
DOI:	10.1136/rmdopen-2022-002324
Abstrakt:	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. Competing Interests: Competing interests: XM received consulting fees from BMS, Novartis, Sanofi, Pfizer, UCB, Janssen, GSK, Galapagos and BMS and is RMD Open associate editor. JE-G received grants from BMS, Lilly, Pfizer (payments made to University of Strasbourg Foundation; no access to TATA data) and consulting fees from BMS, Galapagos, Abbvie, Janssen, Novartis, Pfizer, Sanofi, Gilead, Roche, Chugai and Lilly. (© Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)
Databáze:	MEDLINE
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