An open-label study to evaluate biomarkers and safety in systemic sclerosis patients treated with paquinimod
| Autor: | Helena Eriksson, Birgitta Sparre, Roger Hesselstrand, Marie Törngren, Gabriela Riemekasten, Dirk M. Wuttge, Jörg H W Distler, Fredrik Andersson, Helén Carlsson Nyhlén, Helén Tuvesson, Oliver Distler |
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| Přispěvatelé: | University of Zurich, Hesselstrand, Roger |
| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
0301 basic medicine
medicine.medical_specialty Chemokine 2745 Rheumatology 610 Medicine & health Diseases of the musculoskeletal system CCL2 Gastroenterology 03 medical and health sciences 0302 clinical medicine Quality of life Interferon Internal medicine medicine Humans ddc:610 Adverse effect 030203 arthritis & rheumatology 2403 Immunology Scleroderma Systemic biology integumentary system business.industry Skin fibrosis 10051 Rheumatology Clinic and Institute of Physical Medicine medicine.disease Rheumatology Clinical trial 030104 developmental biology Treatment Outcome RC925-935 biology.protein 2723 Immunology and Allergy Quality of Life Quinolines Systemic sclerosis Paquinimod business Progressive disease Biomarkers medicine.drug Research Article |
| Zdroj: | Arthritis Research & Therapy Arthritis Research & Therapy, Vol 23, Iss 1, Pp 1-12 (2021) |
| ISSN: | 1478-6362 1478-6354 |
| Popis: | Objectives To evaluate the changes in disease-related biomarkers and safety of paquinimod, an oral immunomodulatory compound, in patients with systemic sclerosis (SSc). Methods In this open-label, single-arm, multicenter study, SSc patients with a rapidly progressive disease received paquinimod for 8 weeks. Blood and skin biopsies were collected at baseline, during treatment, and at follow-up for the analyses of type I interferon (IFN) activity, chemokine (C-C motif) ligand 2 (CCL2), and the number of myofibroblasts. The safety of paquinimod was evaluated throughout the study. Results Nine SSc patients were enrolled and completed the study treatment with paquinimod at 3 mg/day for 8 weeks. After the treatment, a reduction of type I IFN activity in the plasma from one patient with elevated baseline IFN activity was recorded. A trend towards reduced IFN activity in the skin after treatment was also observed in patients. The serum level of CCL2 was reduced in 7 of 9 patients after paquinimod treatment. There was a median reduction of 10% of the number of myofibroblasts in skin biopsies at week 8 compared to baseline. No change in modified Rodnan skin score and quality of life was detected in the study. Reported adverse events (AEs) were mild to moderate and expected with the most common being arthralgia (n = 3) and headache (n = 3), and C-reactive protein (CRP) increase. Conclusions Analysis of biomarkers before and after treatment suggest reduced type I IFN activity and reduced number of myofibroblasts in lesional skin. Paquinimod was overall well tolerated with mild to moderate and expected AEs. Trial registration ClinicalTrials.gov, NCT01487551. Registered on 7 September 2011 |
| Databáze: | OpenAIRE |
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