F Proft1,2, M Fleck3, C Fiehn4, H Schulze-Koops1, M Witt1, T Dörner5, J C Henes6. 1. Sektion Rheumatologie und Klinische Immunologie, Medizinische Klinik IV, Klinikum der Universität München, München, Deutschland. 2. Abteilung für Rheumatologie, Medizinische Klinik für Gastroenterologie, Infektiologie und Rheumatologie, Campus Benjamin Franklin, Charité Universitätsmedizin Berlin, Berlin, Deutschland. 3. Asklepios Klinikum Bad Abbach, Bad Abbach, Deutschland. 4. Praxis für Rheumatologie und klinische Immunologie, Baden-Baden, Deutschland. 5. Abteilung für Rheumatologie, Medizinische Klinik. Rheumatologie und klinische Immunologie, Charité Universitätsmedizin Berlin, Berlin, Deutschland. 6. Abteilung für Onkologie, Hämatologie, klinische Immunologie, Rheumatologie, Pulmologie, Medizinische Klinik II der Universität Tübingen, Otfried-Mueller-Str. 10, 72076, Tübingen, Deutschland. Joerg.henes@med.uni-tuebingen.de.
Abstract
OBJECTIVE: To evaluate the safety and efficacy of therapy with biologics in patients with autoinflammatory diseases (AIF) or macrophage activating syndrome (MAS) in a real-life setting in Germany. METHODS: The German Register of Autoimmune Diseases 2 (GRAID2) is a retrospective, non-interventional, multicenter registry collecting data from all patients with inflammatory rheumatic diseases refractory to conventional therapy and treated with initial off-label biologics between August 2006 and December 2013. Patients with MAS could be included without prior treatment with a biologic agent. RESULTS: Data from 26 patients with AIF and 5 with MAS were collected. Of the AIF patients 13 (50%) were diagnosed with adult onset Still's disease (AOSD), 6 (23%) with familial Mediterranean fever (FMF), 4 (15.4%) with tumor necrosis factor-associated periodic syndrome (TRAPS), 1 (3.8%) patient with cryopyrin-associated periodic syndrome (CAPS) and 2 (8%) with undifferentiated fever syndromes. The 5 MAS patients suffered from rheumatoid arthritis (RA) with chronic myeloid leukemia, systemic lupus erythematosus and in 2 cases AOSD. In 1 patient a chronic neurological disease was documented without further differentiaton. All patients with TRAPS were primarily treated with etanercept and all CAPS patients with canakinumab. The AOSD and FMF patients were treated with anakinra as the first line off-label biologic in 6 out of 13 and 5 out of 6 cases, respectively. The MAS patients responded very well or well to therapy in 40% and 60% had a moderate response. There were no non-responders. Within the group of AIF patients the physicians documented a very effective or effective treatment in 38.5%, a moderate response in 30.8% and no response in 30.7%. The tolerance was very good in 5 out of 5 of the MAS and in 92% of the AIF patients. CONCLUSION: The data of this retrospective register provide indications for an effective and safe treatment with off-label biologic medication in patients with AIF and MAS in daily practice.
OBJECTIVE: To evaluate the safety and efficacy of therapy with biologics in patients with autoinflammatory diseases (AIF) or macrophage activating syndrome (MAS) in a real-life setting in Germany. METHODS: The German Register of Autoimmune Diseases 2 (GRAID2) is a retrospective, non-interventional, multicenter registry collecting data from all patients with inflammatory rheumatic diseases refractory to conventional therapy and treated with initial off-label biologics between August 2006 and December 2013. Patients with MAS could be included without prior treatment with a biologic agent. RESULTS: Data from 26 patients with AIF and 5 with MAS were collected. Of the AIF patients 13 (50%) were diagnosed with adult onset Still's disease (AOSD), 6 (23%) with familial Mediterranean fever (FMF), 4 (15.4%) with tumornecrosis factor-associated periodic syndrome (TRAPS), 1 (3.8%) patient with cryopyrin-associated periodic syndrome (CAPS) and 2 (8%) with undifferentiated fever syndromes. The 5 MASpatients suffered from rheumatoid arthritis (RA) with chronic myeloid leukemia, systemic lupus erythematosus and in 2 cases AOSD. In 1 patient a chronic neurological disease was documented without further differentiaton. All patients with TRAPS were primarily treated with etanercept and all CAPS patients with canakinumab. The AOSD and FMFpatients were treated with anakinra as the first line off-label biologic in 6 out of 13 and 5 out of 6 cases, respectively. The MASpatients responded very well or well to therapy in 40% and 60% had a moderate response. There were no non-responders. Within the group of AIF patients the physicians documented a very effective or effective treatment in 38.5%, a moderate response in 30.8% and no response in 30.7%. The tolerance was very good in 5 out of 5 of the MAS and in 92% of the AIF patients. CONCLUSION: The data of this retrospective register provide indications for an effective and safe treatment with off-label biologic medication in patients with AIF and MAS in daily practice.
Authors: Helen J Lachmann; Isabelle Kone-Paut; Jasmin B Kuemmerle-Deschner; Kieron S Leslie; Eric Hachulla; Pierre Quartier; Xavier Gitton; Albert Widmer; Neha Patel; Philip N Hawkins Journal: N Engl J Med Date: 2009-06-04 Impact factor: 91.245
Authors: Alice Corsia; Sophie Georgin-Lavialle; Véronique Hentgen; Eric Hachulla; Gilles Grateau; Albert Faye; Pierre Quartier; Linda Rossi-Semerano; Isabelle Koné-Paut Journal: Orphanet J Rare Dis Date: 2017-03-16 Impact factor: 4.123
Authors: F Proft; H Schulze-Koops; M Grunke; E Schrezenmeier; F Halleck; J Henes; L Unger; E Schmidt; C Fiehn; A Jacobi; C Iking-Konert; C Kneitz; R E Schmidt; B Bannert; R E Voll; R Fischer-Betz; I Kötter; H P Tony; J Holle; M Aringer; A Erler; F Behrens; G R Burmester; T Dörner Journal: Z Rheumatol Date: 2018-02 Impact factor: 1.372