{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,9,11]],"date-time":"2025-09-11T17:21:57Z","timestamp":1757611317889,"version":"3.44.0"},"reference-count":12,"publisher":"BMJ","issue":"2","license":[{"start":{"date-parts":[[2022,11,1]],"date-time":"2022-11-01T00:00:00Z","timestamp":1667260800000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["RMD Open"],"accepted":{"date-parts":[[2022,7,19]]},"published-print":{"date-parts":[[2022,11]]},"abstract":"<jats:sec>\n                  <jats:title>Objectives<\/jats:title>\n                  <jats:p>To assess the tolerance and efficacy of targeted therapies prescribed off-label in refractory low-prevalence autoimmune and inflammatory systemic diseases.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>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 &gt;18 years; low-prevalence autoimmune and inflammatory systemic disease treated with off-label drugs started after 1 January 2019.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>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\u2009mg\/day). The current median follow-up time is 9 months (8 to 10).<\/jats:p>\n                  <jats:p>\n                     <jats:italic>Safety:<\/jats:italic> 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).<\/jats:p>\n                  <jats:p>\n                     <jats:italic>Efficacy:<\/jats:italic> 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.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusion<\/jats:title>\n                  <jats:p>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.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1136\/rmdopen-2022-002324","type":"journal-article","created":{"date-parts":[[2022,11,1]],"date-time":"2022-11-01T11:04:37Z","timestamp":1667300677000},"page":"e002324","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":4,"title":["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)"],"prefix":"10.1136","volume":"8","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9469-946X","authenticated-orcid":false,"given":"Jacques-Eric","family":"Gottenberg","sequence":"first","affiliation":[{"name":"Rheumatology, H\u00f4pitaux universitaires de Strasbourg, Strasbourg, France"},{"name":"Rheumatology, 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