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Observational Study
. 2022 Nov;8(2):e002324.
doi: 10.1136/rmdopen-2022-002324.

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)

Jacques-Eric Gottenberg  1   2 , Aurore Chaudier  3   2 , Yves Allenbach  4   5 , Arsène Mekinian  6 , Zahir Amoura  7   8 , Patrice Cacoub  5   9 , Divi Cornec  10   11 , Eric Hachulla  12   13 , Pierre Quartier  14   15 , Isabelle Melki  16   17 , Christophe Richez  18   19 , Raphaele Seror  20   21 , Benjamin Terrier  5   22 , Valérie Devauchelle-Pensec  10   11 , Julien Henry  20   21 , Marc Gatfosse  23 , Laurence Bouillet  24 , Emeline Gaigneux  25 , Vincent Andre  25 , Gildas Baulier  26 , Aurélie Saunier  26 , Marie Desmurs  27 , Antoine Poulet  28 , Mathieu Ete  29 , Boris Bienvenu  28 , Marie-Elise Truchetet  19   30 , Martin Michaud  31 , Claire Larroche  32 , Azeddine Dellal  33 , Amélie Leurs  34 , Sebastien Ottaviani  35 , Hubert Nielly  36 , Guillaume Vial  37 , Roland Jaussaud  38 , Bénedicte Rouvière  39 , Pierre-Yves Jeandel  40 , Aurelien Guffroy  2   41 , Anne-Sophie Korganow  2   41 , Mathieu Jouvray  36 , Alain Meyer  3   2 , Emmanuel Chatelus  3   2 , Christelle Sordet  3   2 , Renaud Felten  3   2 , Jean Sibilia  3   2 , Samira Litim-Ahmed-Yahia  3 , Jean-Francois Kleinmann  3   2 , Xavier Mariette  20   21
Affiliations
Observational Study

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)

Jacques-Eric Gottenberg et al. RMD Open. 2022 Nov.

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.

Keywords: Autoimmune Diseases; Autoimmunity; Biological Therapy.

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Conflict of interest statement

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.

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