Biologic disease-modifying antirheumatic drugs in the treatment of major monogenic autoinflammatory diseases: literature review and clinical observation

2021 ◽  
Vol 15 (6) ◽  
pp. 95-100
Author(s):  
S. O. Salugina ◽  
E. S. Fedorov
Keyword(s):  
High Efficiency ◽  
Interleukin 1 ◽  
Autoinflammatory Diseases ◽  
Disease Modifying Antirheumatic Drugs ◽  
Antirheumatic Drugs ◽  
Disease Modifying ◽  
Factor Α ◽  
Genetically Determined ◽  
Late Stages

Autoinflammatory diseases (AIDs) are a heterogeneous group of rare genetically determined conditions, the main manifestations of which are episodes of fever in combination with other signs of systemic inflammation: skin rashes, musculoskeletal and neurological disorders, damage to the organs of vision, hearing, etc., as well as acute phase markers and the absence of autoantibodies. The use of biological therapy, especially inhibitors of interleukin 1 (iIL1), in most common monogenic AIDs (mAID) – FMF, TRAPS, HIDS/MKD, CAPS – has shown its high efficiency and led to significant progress in the treatment of these patients. Currently, iIL1 are the first-line drugs for mAIDs therapy, primarily CAPS. In the case of their ineffectiveness or intolerance in certain situations, other biologic disease-modifying antirheumatic drugs can also be used – inhibitors of tumor necrosis factor α and iIL6, but this issue needs further investigation. The article describes a patient with mAID, in whom the diagnosis was made more than 40 years after the onset; administration of targeted therapy even in the late stages of the disease led to a significant improvement in many symptoms and quality of life. 

scholarly journals Biological disease-modifying antirheumatic drugs in the main monogenic autoinflammatory diseases treatment. Experience of application in rheumatological practice

2021 ◽  
Vol 15 (4) ◽  
pp. 24-30
Author(s):  
S. O. Salugina ◽  
E. S. Fedorov ◽  
M. I. Kaleda
Keyword(s):  
Tumor Necrosis Factor ◽  
Tumor Necrosis ◽  
Periodic Fever ◽  
Interleukin 1 ◽  
Autoinflammatory Diseases ◽  
Response To Treatment ◽  
Disease Modifying Antirheumatic Drugs ◽  
Antirheumatic Drugs ◽  
Disease Modifying ◽  
Necrosis Factor

Objective: to assess the frequency of prescription, efficacy and tolerability of biological disease-modifying antirheumatic drugs (bDMARDs) therapy in patients with major monogenic autoinflammatory diseases (mAID) according to the Federal Rheumatology Center clinical practice. Patients and methods. From 2008 to 2020 years, 158 patients with mAID were included in the study, 53 of whom were prescribed bDMARDs: 12 patients had Familial Mediterranean Fever (FMF); 26 – Cryopyrin-Associated Periodic Syndromes (CAPS), including 21 patients with MuckleWells Syndrome (MWS) and 5 – with Chronic Infantile Onset Neurologic Cutaneous Articular / Neonatal Onset Multisystem Inflammatory Disease (CINCA/NOMID), 12 patients had Tumor necrosis factor (TNF) receptor-Associated Periodic Fever Syndrome (TRAPS) and 3 – Hyper-Immunoglobulinemia D-syndrome (HIDS/MKD). Among all these patients 25 were male and 28 female, aged 1.5 to 44 years, 45 were children (under 18) and 8 adults. Interleukin 1 inhibitors (iIL1) were prescribed in accordance with the following scheme: canakinumab – subcutaneously 2–5 mg/kg or 150 mg per injection, every 4–8 weeks; anakinra – subcutaneously 1–5 mg/kg or 100 mg/day, daily. Etanercept (ETC) was injected subcutaneously 0.4–0.8 mg/kg 1–2 times a week, and adalimumab (ADA) was injected subcutaneously 20–40 mg once every 2 weeks. Tocilizumab (TCZ) was administered intravenously, 8–12 mg/kg once every 2–4 weeks. The duration of the disease at the time of treatment initiation ranged from 1 to 44 years. The duration of bDMARDs therapy in patients with mAID ranged from 1 month to 12 years.Results and discussion. From 158 patients with mAID, in 53 (33.5%) bDMARDs were administered. They were used more often in patients with CAPS (56.6%), and less often – in TRAPS (26.4%), FMF (28.3%) and HIDS/MKD (5.7%). iIL1 were the most frequently prescribed bDMARDs (90.6%): canakinumab (in 38 patients) and anakinra (in 10), they were mainly used in patients with CAPS, in 2/3 of patients with TRAPS, HIDS/MKD and colchicine-resistant FMF. During the first days of iIL1 treatment, all patients with mAID showed a statistically significant clinical improvement: normalization of general condition, emotional recovery, relief of fever, disappearance of rash, decrease in the severity of lymphadenopathy and hepatosplenomegaly, relief or significant positive dynamics of eye symptoms, subjective improvement in hearing and audiogram (with dynamic control in patients with CAPS), decrease in the level of acute phase markers (in all cases). In 7 patients with CAPS, who received anakinra, after a positive response was achieved, switching to canakinumab was performed, which maintained the full effectiveness of therapy. TCZ (in 7 patients) and inhibitors of tumor necrosis factor α (iTNFα) – ADA (in 3) and ETC (in 4), – were used less frequently. iTNFα were more often prescribed to FMF patients with a complete response to treatment. Tolerability of bDMARD therapy was satisfactory in all patients. Conclusion. Currently, iIL1 are the first line of therapy among biological agents for mAID, especially in patients with CAPS. If they are ineffective or intolerant in certain situations, alternative bDMARDs (iTNFα and IL6 inhibitors) can also be used, but this issue needs further study.

Quality of life of patients with Sjogren’s disease with ongoing therapy with diseasemodifying antirheumatic drugs

2020 ◽  
pp. 33-38
Author(s):  
E. Yu. Gan ◽  
L. P. Evstigneeva
Keyword(s):  
Quality Of Life ◽  
Short Form ◽  
Life Quality ◽  
Well Being ◽  
Disease Modifying Antirheumatic Drugs ◽  
Antirheumatic Drugs ◽  
Sf 36 ◽  
Disease Modifying ◽  
Sjögren’S Disease

Purpose of the study. Assessing the association between the life quality of patients with Sjogren’s Disease and ongoing therapy with various disease-modifying antirheumatic drugs.Material and methods. The study was conducted on the basis of the regional rheumatology center of the consultative diagnostic clinic of the Sverdlovsk Regional Clinical Hospital No. 1. This work is based on the results of a simultaneous study of 74 patients with primary Sjogren’s Disease (SD), distributed in three comparison groups receiving various disease-modifying antirheumatic drugs chlorambucil, methotrexate and hydroxychloroquine. The diagnosis of SD was carried out according to European-American criteria AECGC (2002) [18]. In order to analyze the quality of life of patients with SD, the 36-Item Short Form Health Survey (SF‑36) was used. Statistical data processing was carried out using Statistica 7.0 program.Results. Assessment of the quality of life of patients with SD, which is an integrative criterion of human health and well-being, revealed the absence of statistically significant differences (p > 0.05) on eight scales and two health components of the SF‑36 questionnaire in the analyzed groups that differ in the treatment of disease-modifying antirheumatic drugs chlorambucil, methotrexate and hydroxychloroquine.Conclusions. The obtained data indicate an equivalent quality of life in SD patients treated with different disease-modifying antirheumatic drugs methotrexate, chlorambucil and hydroxychloroquine, and therefore hydroxychloroquine can be considered as an alternative basic therapy in patients with SD with certain limitations and contraindications methotrexate and chlorambucil.

Therapiekonzepte zur Behandlung von Patienten mit nicht infektiöser Uveitis: biologische Disease Modifying Antirheumatic Drugs

2018 ◽  
Vol 235 (05) ◽  
pp. 553-561
Author(s):  
Karoline Walscheid ◽  
Uwe Pleyer ◽  
Arnd Heiligenhaus
Keyword(s):  
Interleukin 1 ◽  
Disease Modifying Antirheumatic Drugs ◽  
Antirheumatic Drugs ◽  
Off Label ◽  
Tnf Α ◽  
Disease Modifying ◽  
Anti Cd20

ZusammenfassungBiologika stellen eine hochwirksame Therapieoption für verschiedene nicht infektiöse Uveitisformen dar. Einziges zugelassenes Biologikum ist der TNF-α-Inhibitor Adalimumab, alle anderen Präparate müssen im Rahmen einer Off-Label-Therapie gegeben werden. Die Indikation zur Therapieinitiierung mit einem Biologikum besteht, wenn die Erkrankung nicht ausreichend anspricht auf eine Behandlung mit systemischen Steroiden und/oder csDMARDs (konventionell-synthetischen disease modifying antirheumatic drugs) oder diese aufgrund von unerwünschten Wirkungen nicht gegeben werden können. Derzeit in der klinischen Anwendung befindliche biologische DMARD-Präparate wirken über zytokinspezifische Mechanismen (TNF-α-Inhibition, Interferone, Hemmung der Signaltransduktion von Interleukin-1 [IL-1], IL-6 und IL-17) sowie Hemmung der T-Zell-Kostimulation (CTLA-4-Fusionsprotein), oder B-Zell-Depletion (Anti-CD20). Alle Präparate müssen parenteral verabreicht werden. Die Einleitung einer Biologikatherapie sollte nach interdisziplinärer Abstimmung und Ausschluss von Kontraindikationen erfolgen. Ein regelmäßiges klinisches und laborchemisches Monitoring unter der Therapie ist erforderlich.

Simultaneous Response in Several Domains in Patients with Psoriatic Disease Treated with Etanercept as Monotherapy or in Combination with Conventional Synthetic Disease-modifying Antirheumatic Drugs

2018 ◽  
Vol 45 (6) ◽  
pp. 802-810 ◽  
Author(s):  
Frank Behrens ◽  
Lothar Meier ◽  
Jörg C. Prinz ◽  
Jürgen Jobst ◽  
Ralph Lippe ◽  
...  
Keyword(s):  
Combination Therapy ◽  
Disease Activity ◽  
Short Form ◽  
Medical Outcomes ◽  
Disease Modifying Antirheumatic Drugs ◽  
Antirheumatic Drugs ◽  
Treatment Targets ◽  
Link Type ◽  
Disease Modifying

Objective.To evaluate patients with psoriatic arthritis (PsA) receiving etanercept (ETN) monotherapy or ETN plus conventional synthetic disease-modifying antirheumatic drugs (csDMARD) to determine the proportion achieving a clinically meaningful response in arthritis, psoriasis, and quality of life simultaneously.Methods.A prospective, multicenter, 52-week observational study in patients with active PsA evaluated treatment with ETN in clinical practice (ClinicalTrials.gov:NCT00293722). This analysis assessed simultaneous achievement of 3 treatment targets: low disease activity (LDA) based on 28-joint count Disease Activity Score (DAS28); body surface area (BSA) involvement ≤ 3%; and a score > 45 on the Medical Outcomes Study Short Form-12 (SF-12) physical component summary.Results.Of 579 patients, 380 received ETN monotherapy and 199 received combination ETN plus csDMARD. At 52 weeks, data for all 3 disease domains were available for 251 patients receiving monotherapy and 151 receiving combination therapy. In the monotherapy and combination therapy groups, 61 (24.3%) and 37 (24.5%) patients, respectively, achieved all 3 treatment targets simultaneously. A significantly greater proportion of patients receiving monotherapy versus combination therapy achieved SF-12 > 45 (43.0% vs 31.8%; p < 0.05) and DAS28 LDA (72.5% vs 62.3%; p < 0.05). Conversely, BSA ≤ 3% was reached by a significantly greater proportion receiving combination therapy (75.5% vs 56.6%; p < 0.001). However, baseline BSA involvement was higher for the monotherapy group.Conclusion.While nearly half the patients achieved arthritis and psoriasis treatment targets simultaneously and one-fourth reached all 3 treatment targets, combining ETN and csDMARD did not substantially improve clinical response compared with ETN monotherapy in this real-world PsA patient population.

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