scholarly journals Tocilizumab Treatment of Patients with Systemic Sclerosis: Clinical Data

2017 ◽  
Vol 2 (2_suppl) ◽  
pp. S29-S35 ◽  
Author(s):  
Dinesh Khanna ◽  
Angelika Jahreis ◽  
Daniel E. Furst
Keyword(s):  
Systemic Sclerosis ◽  
Treatment Options ◽  
Current Treatment ◽  
Phase 2 Trial ◽  
Immune Mediated ◽  
Interleukin 6 Receptor ◽  
Organ Specific ◽  
Pharmacologic Management ◽  
Disease Modifying ◽  
Disease Modifying Treatment

Systemic sclerosis (SSc) is an immune-mediated disease characterized by progressive, and often severe, inflammation and fibrosis of the skin and internal organs, such as the lungs, heart, kidneys, and gastrointestinal tract. There is an unmet medical need to treat patients with SSc given the high SSc-related mortality rate. Furthermore, disease-modifying therapies are lacking, and current treatment options focus on the management of individual organ-specific complications associated with the disease. Immunosuppressive therapies are the mainstay of pharmacologic management of major SSc-associated complications, such as skin and lung manifestations, but their overall effectiveness is limited and toxicity issues are common. Advances in understanding of the pathological processes involved in the immunologic and fibrotic mechanisms of SSc have led to clinical research focusing on targeted immunotherapies, in an effort to develop much needed disease-modifying treatment options. The interleukin-6 receptor-alpha antagonist tocilizumab has demonstrated promising efficacy in a phase 2 trial and is being investigated in a phase 3 trial. This article provides an overview of current pharmacologic treatment options for the management of SSc-related complications and discusses tocilizumab for the treatment of SSc in clinical trials.

Selected natural and synthetic agents effective against Parkinson’s disease with diverse mechanisms

2021 ◽  
Vol 21 ◽  
Author(s):  
Hayrettin Ozan Gulcan
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Treatment Options ◽  
Treatment Strategies ◽  
Current Treatment ◽  
Dopamine Metabolism ◽  
Dopaminergic Agonists ◽  
Alternative Drug ◽  
Synthetic Agents ◽  
Disease Modifying

: Similar to other neurodegenerative diseases, Parkinson’s disease (PD) has been extensively investigated with respect to its neuropathological background and possible treatment options. Since the symptomatic outcomes are generally related to dopamine deficiency, the current treatment strategies towards PD mainly employ dopaminergic agonists as well as the compounds acting on dopamine metabolism. These drugs do not provide disease modifying properties; therefore alternative drug discovery studies focus on targets involved in the progressive neurodegenerative character of PD. This study has aimed to present the pathophysiology of PD concomitant to the representation of drugs and promising molecules displaying activity against the validated and non-validated targets of PD.

scholarly journals Charcot-Marie-Tooth: From Molecules to Therapy

2019 ◽  
Vol 20 (14) ◽  
pp. 3419 ◽  
Author(s):  
Jonathan Morena ◽  
Anirudh Gupta ◽  
J. Chad Hoyle
Keyword(s):  
Diagnostic Yield ◽  
Treatment Options ◽  
Research Attention ◽  
Charcot Marie Tooth ◽  
Inherited Neuropathy ◽  
Clinical Background ◽  
Disease Modifying ◽  
Therapeutic Research ◽  
Next Generation Sequencing Ngs ◽  
Disease Modifying Treatment

Charcot-Marie-Tooth (CMT) is the most prevalent category of inherited neuropathy. The most common inheritance pattern is autosomal dominant, though there also are X-linked and autosomal recessive subtypes. In addition to a variety of inheritance patterns, there are a myriad of genes associated with CMT, reflecting the heterogeneity of this disorder. Next generation sequencing (NGS) has expanded and simplified the diagnostic yield of genes/molecules underlying and/or associated with CMT, which is of paramount importance in providing a substrate for current and future targeted disease-modifying treatment options. Considerable research attention for disease-modifying therapy has been geared towards the most commonly encountered genetic mutations (PMP22, GJB1, MPZ, and MFN2). In this review, we highlight the clinical background, molecular understanding, and therapeutic investigations of these CMT subtypes, while also discussing therapeutic research pertinent to the remaining less common CMT subtypes.

scholarly journals SUCCESSFUL OUTCOME IN A CASE OF CRITICAL DIGITAL ISCHEMIA. CASE REPORT AND LITERATURE ANALYSIS

2016 ◽  
Vol 25 (3) ◽  
pp. 163-166
Author(s):  
Cristina Pomirleanu ◽  
◽  
Georgiana Strugariu ◽  
Andreea Axinte ◽  
Iulian Resmerita ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Treatment Options ◽  
Microvascular Disease ◽  
Current Treatment ◽  
Tissue Loss ◽  
Successful Outcome ◽  
Dramatic Improvement ◽  
Recent Onset ◽  
Digital Ischemia ◽  
Complex Approach

Critical digital ischemia (CDI) remains a distinct, serious complication of peripheral vasculopathy in systemic sclerosis, macrovascular as well as microvascular disease, abnormal vascular remodeling, vasoconstriction-vasodilation imbalance and fibrosis being key underlying factors. Successful short- as well as long-term outcomes may arise from early diagnosis, broad assessment and prompt therapeutic intervention. However, current treatment options are far from ideal, indicating the need for a complex approach focused on pathobiologic issues. We report the case of a middle age postmenopausal woman with a recent onset of CDI and subsequent gangrene, evolving in a very early PM-Scl positive systemic sclerosis. Dramatic improvement with complete resolution of CDI as well as healing of digital gangrene with minimal tissue loss was rapidly achieved under vasodilating therapy, after two cycles of intravenous prostanoids.

scholarly journals Observational Study of Treatment Outcome in Early Diffuse Cutaneous Systemic Sclerosis

2009 ◽  
Vol 37 (1) ◽  
pp. 116-124 ◽  
Author(s):  
ARIANE L. HERRICK ◽  
MARK LUNT ◽  
NINA WHIDBY ◽  
HOLLY ENNIS ◽  
ALAN SILMAN ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Randomized Clinical Trials ◽  
Relative Effectiveness ◽  
Outcome Data ◽  
Rate Of Change ◽  
Inverse Probability ◽  
Diffuse Cutaneous Systemic Sclerosis ◽  
Disease Modifying ◽  
Disease Modifying Treatment ◽  
Over Time

Objective.Randomized clinical trials in early diffuse cutaneous systemic sclerosis (dcSSc) are challenging. We used an observational approach to estimate the relative effectiveness of different current treatment approaches, capturing entry and outcome data in a standardized way.Methods.Patients with dcSSc within 3 years of the onset of skin thickening were included. Standardized entry and followup data were collected in relation to the first disease-modifying treatment at baseline and 4–6 weeks, then 3, 6, 12, 18, 24, 30, and 36 months. The 5 different protocols were (1) intravenous cyclophosphamide followed by mycophenolate mofetil (MMF); (2) antithymocyte globulin followed by MMF; (3) MMF alone; (4) no disease-modifying treatment; (5) other immunosuppressant treatment. The primary outcome measure was the modified Rodnan skin score (mRSS). Inverse probability of treatment weights were used to allow for differing patient characteristics between groups.Results.The study included 147 patients from 12 centers. Numbers of patients starting on Protocols 1 to 5 were 29, 25, 61, 19, and 13, respectively. mRSS decreased over time from 24 (IQ 19–32) at baseline to 15.5 (IQ 9–24.5) at 3 years. Although there were differences in the magnitude of the change for different protocols, there were no significant differences between protocols in the rate of change of mRSS over time (p = 0.43). When inverse probability weights were applied, the results remained nonsignificant (p = 0.41).Conclusion.Using this observational approach, there were no obvious differences in outcome between groups after allowing as far as possible for baseline differences in treatment allocations.

scholarly journals Successful use of Netakimab in the treatment of psoriasis accompanied by the psoriatic onychodystrophy

2020 ◽  
pp. 64-70
Author(s):  
N. N. Potekaev ◽  
O. V. Zhukova ◽  
S. I. Artemyeva
Keyword(s):  
Plaque Psoriasis ◽  
Treatment Options ◽  
Nail Plate ◽  
Current Treatment ◽  
Efficacy And Safety ◽  
High Efficacy ◽  
Results Of Treatment ◽  
Interleukin 17A ◽  
Immune Mediated ◽  
Wide Range

Psoriasis is a chronic immune-mediated disease that is accompanied by a significant number of comorbid pathologies. Damage to the nail plates (psoriatic onychodystrophy) is widespread among patients with psoriasis and is associated with significant functional as well as psychosocial impairments. Despite the fact that nails constitute a small percentage of the surface of the human body, the damage to this particular area can lead to a deterioration in the quality of life and irreversible disability. In addition, studies have shown that nail psoriasis is indicative of a more severe course of the disease and it can also be associated with psoriatic arthritis or it can be a predictor of its development. Current treatment options for psoriasis accompanied by the nail plates damage include many topical and systemic methods, however, patients often report dissatisfaction with the results of treatment due to low efficacy or many side effects. Achieving higher efficiency is possible with the use of biologic therapy. Currently, a wide range of biologics have been developed that modulate key elements in the immunopathogenesis of psoriasis.The pathogenesis of psoriasis is a multifactorial process, however, it is the IL23 / Th17 signaling pathway that is key in this process. Interleukin-17A is the principal effector of this pathway and overexpression of IL-17A leads to epidermal hyperplasia and an excessive inflammatory response seen in psoriasis. Therefore, interleukin-17A is a promising therapeutic target.Considering the critical pathogenetic role as well as the high efficacy and safety of IL-17A inhibitors, the study of their effect on the psoriatic onychodystrophy manifestations is of great clinical importance.Netakimab is the first Russian original IL-17 inhibitor which is a promising modern agent for the treatment of moderate-to-severe plaque psoriasis. The obtained real clinical data indicate the high efficacy and safety of the use of Netakimab in patients with both plaque psoriasis and «severe» psoriasis in difficult to treat localizations, such as damage of the nail plate.

scholarly journals Recent Treatments of Interstitial Lung Disease with Systemic Sclerosis

2015 ◽  
Vol 9s1 ◽  
pp. CCRPM.S23315 ◽  
Author(s):  
Hidekata Yasuoka
Keyword(s):  
Systemic Sclerosis ◽  
Interstitial Lung Disease ◽  
Lung Disease ◽  
Treatment Options ◽  
Immune Dysfunction ◽  
Pulmonary Involvement ◽  
Current Treatment ◽  
Microvascular Injury ◽  
Selection Of Patients ◽  
Selection Of

Systemic sclerosis (SSc) is a disorder characterized by immune dysfunction, microvascular injury, and fibrosis. Organ involvement in patients with SSc is variable; however, pulmonary involvement occurs in up to 90% of patients with SSc. Interstitial lung disease (ILD) is a major cause of mortality and, thus, a major determinant in the prognosis of patients with SSc. This review summarizes current findings about the characteristics of ILD in patients with SSc, selection of patients with SSc-ILD who are candidates for the treatment, and current treatment options.

Immune-mediated ophthalmoparesis with anti-GD1a antibodies

2021 ◽  
Vol 14 (11) ◽  
pp. e244273
Author(s):  
Norma McKean ◽  
Charmaine Chircop
Keyword(s):  
Neurological Examination ◽  
Intravenous Immunoglobulins ◽  
Immune Mediated ◽  
Post Infusion ◽  
History Of ◽  
Disease Modifying ◽  
Ct Brain ◽  
Disease Modifying Treatment ◽  
Mr Brain

A young woman presented to neurology with a 1 month history of progressive diplopia on lateral gaze and a 1 week history of headaches. On examination she was found to have complex ophthalmoparesis with binocular horizontal diplopia, failure of abduction bilaterally and limited upgaze with convergence-retraction nystagmus. The rest of the neurological examination was normal. She was admitted for investigations: blood, CT brain, MR brain and lumbar puncture results were normal. Anti-GD1a antibodies were strongly positive; anti-GM1, anti-GM2 and anti-GD1b were also positive. On follow-up 3 weeks later, the complex ophthalmoplegia persisted. It was decided to treat with intravenous immunoglobulins (IVIgs) with good response but recurrence at 2 weeks post infusion. She was treated with 4 weekly IVIg courses and remains responsive and controlled over 1 year since presentation but becomes symptomatic in the week running up to each dose; thus, disease modifying treatment is currently being considered.

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