scholarly journals Ongoing clinical trials and treatment options for patients with systemic sclerosis–associated interstitial lung disease

Rheumatology ◽  
2018 ◽  
Vol 58 (4) ◽  
pp. 567-579 ◽  
Author(s):  
Dinesh Khanna ◽  
Donald P Tashkin ◽  
Christopher P Denton ◽  
Martin W Lubell ◽  
Cristina Vazquez-Mateo ◽  
...  
Keyword(s):  
Interstitial Lung Disease ◽  
Lung Disease ◽  
Treatment Guidelines ◽  
Treatment Options ◽  
Multiple Organ ◽  
Organ Systems ◽  
The Uk ◽  
Disease Modifying Treatment ◽  
Substantial Morbidity

Abstract SSc is a rare CTD that affects multiple organ systems, resulting in substantial morbidity and mortality. Evidence of interstitial lung disease (ILD) is seen in ∼80% of patients with SSc. Currently there is no approved disease-modifying treatment for ILD and few effective treatment options are available. CYC is included in treatment guidelines, but it has limited efficacy and is associated with toxicity. MMF is becoming the most commonly used medication in clinical practice in North America and the UK, but its use is not universal. Newer agents targeting the pathogenic mechanisms underlying SSc-ILD, including fibrotic and inflammatory pathways, lymphocytes, cell–cell and cell–extracellular membrane interactions, hold promise for better treatment outcomes, including improved lung function, patient-related outcomes and quality of life. Here we review ongoing trials of established and novel agents that are currently recruiting patients with SSc-ILD.

scholarly journals Cyclophosphamide Versus Obinutuzumab for the Treatment of Anti-MDA5 Positive Inflammatory Myopathy with Interstitial Lung Disease: A Study Protocol and Literature Review

2019 ◽  
Vol 19 (02) ◽  
pp. 53-58
Author(s):  
Ho So ◽  
Chi Chiu Mok
Keyword(s):  
Interstitial Lung Disease ◽  
Lung Disease ◽  
Primary Outcome ◽  
Treatment Guidelines ◽  
Controlled Trial ◽  
Treatment Options ◽  
Inflammatory Myopathy ◽  
Idiopathic Inflammatory Myopathy ◽  
High Dose ◽  
Multicenter Randomized Controlled Trial

In patients with idiopathic inflammatory myopathy, the presence of the melanoma differentiation-associated gene 5 (MDA5) antibody carries an extremely poor prognosis as a result of the associated interstitial lung disease (ILD) that is often rapidly progressive and refractory to therapies. Management of anti-MDA5 associated ILD is a challenging task as there is a paucity of clinical data and treatment guidelines in the literature. We hereby describe a proposed protocol for a multicenter randomized controlled trial to compare the efficacy of intravenous cyclophosphamide and obinutuzumab in combination with high-dose glucocorticoids and tacrolimus in terms of mortality at six months (primary outcome). The epidemiology, pathogenesis and treatment options of anti-MDA5 associated ILD are briefly reviewed.

scholarly journals Effective Treatment of Rheumatoid Arthritis-Associated Interstitial Lung Disease by B-Cell Targeted Therapy with Rituximab

2012 ◽  
Vol 2012 ◽  
pp. 1-3 ◽  
Author(s):  
Wolfgang Hartung ◽  
Judith Maier ◽  
Michael Pfeifer ◽  
Martin Fleck
Keyword(s):  
Rheumatoid Arthritis ◽  
Interstitial Lung Disease ◽  
Lung Disease ◽  
B Cell ◽  
Treatment Options ◽  
Joint Inflammation ◽  
Dmard Therapy ◽  
Sustained Improvement ◽  
Minute Walk

Rheumatoid arthritis- (RA-) associated interstitial lung disease (RA-ILD) is the extra-articular complication with most adverse impact on the quality of life and survival in RA patients. However, treatment options are limited and controlled studies are lacking. Here, we present the case of a 66-year-old patient suffering from severe RA-ILD, which has been successfully treated with Rituximab (RTX). After failure of conventional DMARD therapy, our patient showed sustained improvement of clinical pulmonary parameters as well as joint inflammation following B-cell depletion with RTX. The six-minute-walk test improved from 380 meters to 536 meters and the forced vital capacity from 2.49 liters to 3.49. The disease activity score could be reduced from 7.7 to 2.8. Therefore, RTX might be considered as an alternative treatment for RA-ILD in patients not responding to conventional DMARD therapy.

Renal Manifestations of Tuberous Sclerosis Complex

2020 ◽  
Vol 7 (3) ◽  
pp. 5-19
Author(s):  
Nikhil Nair ◽  
Ronith Chakraborty ◽  
Zubin Mahajan ◽  
Aditya Sharma ◽  
Sidarth Sethi ◽  
...  
Keyword(s):  
Tuberous Sclerosis ◽  
Tuberous Sclerosis Complex ◽  
Gene Disruption ◽  
Treatment Guidelines ◽  
Renal Cysts ◽  
Skin Lesions ◽  
Multiple Organ ◽  
Tsc2 Gene ◽  
Genetic Condition ◽  
Organ Systems

Tuberous sclerosis complex (TSC) is a genetic condition caused by a mutation in either the TSC1 or TSC2 gene. Disruption of either of these genes leads to impaired production of hamartin or tuberin proteins, leading to the manifestation of skin lesions, tumors and seizures. TSC can manifests in multiple organ systems with the cutaneous and renal systems being the most commonly affected. These manifestations can secondarily lead to the development of hypertension, chronic kidney disease, and neurocognitive declines. The renal pathologies most commonly seen in TSC are angiomyolipoma, renal cysts and less commonly, oncocytomas. In this review, we highlight the current understanding on the renal manifestations of TSC along with current diagnosis and treatment guidelines.

Lung disease manifestations in Down Syndrome

2021 ◽  
Author(s):  
Soula Danopoulos ◽  
Gail H. Deutsch ◽  
Claire Dumortier ◽  
Thomas Jay Mariani ◽  
Denise Al Alam
Keyword(s):  
Down Syndrome ◽  
Lung Disease ◽  
Chromosomal Abnormalities ◽  
Respiratory Illness ◽  
Multiple Organ ◽  
Upper Airways ◽  
Live Births ◽  
Organ Systems ◽  
Entire Lung ◽  
Lower Airways

Down Syndrome (DS) is one of the most prevalent chromosomal abnormalities world-wide, affecting 1 in 700 live births. Although multiple organ systems are affected by the chromosomal defects, respiratory failure and lung disease are the leading causes of morbidity and mortality observed in DS. Manifestations of DS in the respiratory system encompass the entire lung starting from the nasopharynx, trachea/upper airways to the lower airways and alveolar spaces, as well as vascular and lymphatic defects. Most of our knowledge on respiratory illness in persons with DS arises from pediatric studies, however many of these disorders present early in infancy supporting developmental mechanisms. In this review we will focus on the different lung phenotypes in DS, as well as the genetic and molecular pathways that may be contributing to these complications during development.

scholarly journals Determinants of Health-Related Quality of Life Decline in Interstitial Lung Disease

2020 ◽  
Author(s):  
Phillen Nozibuyiso Maqhuzu ◽  
Boglárka Lilla Szentes ◽  
Michael Kreuter ◽  
Thomas Bahmer ◽  
Nicolas Kahn ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Interstitial Lung Disease ◽  
Lung Disease ◽  
Psychological Impact ◽  
Sociodemographic Factors ◽  
Health Related Quality ◽  
Lower Baseline ◽  
Related Quality ◽  
Health Related

Abstract Background: Health-related quality of life (HRQL) in interstitial lung disease (ILD) patients is impaired. We aimed to identify baseline predictors for HRQL decline within a 12-month observation period. Methods: We analyzed 194 ILD patients from two German ILD-centers in the observational HILDA study. We employed the disease-specific King’s Brief Interstitial Lung Disease questionnaire (K-BILD) with the subdomains ‘psychological impact’, ‘chest symptoms’ and ‘breathlessness and activities’, and the generic EQ-5D Visual Analog Scale (VAS). We evaluated how many patients experienced a clinically meaningful decline in HRQL. Subsequently, we investigated medical and sociodemographic factors as potential predictors of HRQL deterioration. Results: Within the study population (34.0% male, Ø age 61.7) mean HRQL scores hardly changed between baseline and follow up (K-BILD: 52.8 vs. 52.5 | VAS: 60.0 vs. 57.3). On the intra-individual level, 30.4% (n = 59) experienced a clinically relevant deterioration in K-BILD total score and 35.4% (n = 68) in VAS. Lower baseline forced vital capacity % predicted determined HRQL decline in K-BILD total score (ß– coefficient -0.02, p = 0.007), VAS (ß–coefficient -0.03, p < 0.0001), and in the subdomain ‘psychological impact’ (ß– coefficient -0.02, p = 0.014). Lower baseline diffusing capacity of carbon monoxide % predicted determined deterioration in ‘breathlessness and activities’ (ß– coefficient -0.04, p = 0.003) and ‘chest symptoms’ (ß– coefficient -0.04, p = 0.002). Additionally, increasing age predicted decline in ‘psychological impact’ (ß–coefficient 0.06, p < 0.007). Conclusion: A third of ILD patients experience a clinically relevant HRQL deterioration within 12 months, which is mainly predicted by lung function baseline value.

scholarly journals Patient-reported quality of life in fibrotic interstitial lung disease: novel assessments of self-management ability and affect

2021 ◽  
Vol 7 (1) ◽  
pp. 00011-2021
Author(s):  
Teng Moua ◽  
Aahd Kubbara ◽  
Paul Novotny ◽  
Jennifer L. Ridgeway ◽  
Andrew H. Limper ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Interstitial Lung Disease ◽  
Lung Disease ◽  
Self Management ◽  
Management Ability ◽  
Patient Reported

scholarly journals Biomarkers in systemic sclerosis-associated interstitial lung disease: review of the literature

Rheumatology ◽  
2019 ◽  
Vol 58 (9) ◽  
pp. 1534-1546 ◽  
Author(s):  
Olivier Bonhomme ◽  
Béatrice André ◽  
Fanny Gester ◽  
Dominique de Seny ◽  
Catherine Moermans ◽  
...  
Keyword(s):  
Interstitial Lung Disease ◽  
Lung Disease ◽  
Wide Spectrum ◽  
Unknown Origin ◽  
Multiple Organ ◽  
Correct Identification ◽  
Review Of The Literature ◽  
Clinical Value ◽  
Diagnosis And Prognosis ◽  
Risk Of Progression

Abstract SSc is a rare disease of unknown origin associated with multiple organ involvement. One of the major complications that drives the mortality of SSc patients is interstitial lung disease. The course of SSc-interstitial lung disease progression has a wide spectrum. Since the treatment is based on aggressive immunosuppression it should not be given to stable or non-progressing disease. The correct identification of disease with high risk of progression remains a challenge for early therapeutic intervention, and biomarkers remain urgently needed. In fact, eight categories of biomarkers have been identified and classified according to the different biological pathways involved. The purpose of this article is to describe the main biomarkers thought to be of interest with clinical value in the diagnosis and prognosis of SSc-interstitial lung disease.

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