Showing papers by "National Jewish Health published in 2015"

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

Abstract: A total of 1108 patients underwent randomization and received study drug. The mean baseline FEV 1 was 61% of the predicted value. In both studies, there were significant improvements in the primary end point in both lumacaftor–ivacaftor dose groups; the difference between active treatment and placebo with respect to the mean absolute improvement in the percentage of predicted FEV 1 ranged from 2.6 to 4.0 percentage points (P<0.001), which corresponded to a mean relative treatment difference of 4.3 to 6.7% (P<0.001). Pooled analyses showed that the rate of pulmonary exacerbations was 30 to 39% lower in the lumacaftor–ivacaftor groups than in the placebo group; the rate of events leading to hospitalization or the use of intravenous antibiotics was lower in the lumacaftor–ivacaftor groups as well. The incidence of adverse events was generally similar in the lumacaftor–ivacaftor and placebo groups. The rate of discontinuation due to an adverse event was 4.2% among patients who received lumacaftor–ivacaftor versus 1.6% among those who received placebo. CONCLUSIONS These data show that lumacaftor in combination with ivacaftor provided a benefit for patients with cystic fibrosis homozygous for the Phe508del CFTR mutation. (Funded by Vertex Pharmaceuticals and others; TRAFFIC and TRANSPORT ClinicalTrials.gov numbers, NCT01807923 and NCT01807949.)

Connective Tissue Disease-associated Interstitial Lung Diseases (CTD-ILD) — Report from OMERACT CTD-ILD Working Group

Abstract: Objective Interstitial lung disease (ILD) is common in connective tissue disease (CTD) and is the leading cause of mortality. Investigators have used certain outcome measures in randomized controlled trials (RCT) in CTD-ILD, but the lack of a systematically developed, CTD-specific index that captures all measures relevant and meaningful to patients with CTD-ILD has left a large and conspicuous gap in CTD-ILD research. Methods The CTD-ILD working group, under the aegis of the Outcome Measures in Rheumatology (OMERACT) initiative, has completed a consensus group exercise to reach harmony on core domains and items for inclusion in RCT in CTD-ILD. During the OMERACT 12 meeting, consensus was sought on domains and core items for inclusion in RCT. In addition, consensus was pursued on a definition of response in RCT. Consensus was defined as ≥ 75% agreement among the participants. Results OMERACT 12 participants endorsed the domains with minimal modifications. Clinically meaningful progression for CTD-ILD was proposed as ≥ 10% relative decline in forced vital capacity (FVC) or ≥ 5% to Conclusion There is consensus on domains for inclusion in RCT in CTD-ILD and on a definition of clinically meaningful progression. Data-driven approaches to validate these results in different cohorts and RCT are needed.

Benefits of Physical Training in Sarcoidosis.

Abstract: Background Sarcoidosis patients suffer from fatigue and exercise limitation. The aim of this study was to establish whether a physical training program improves these and other outcomes important to sarcoidosis patients. Methods From 11/2012 to 9/2014, 201 sarcoidosis patients were referred to the ild care expertise team, Ede, the Netherlands. In our center, all patients are routinely recommended to undergo testing at baseline to determine their physical functioning and encouraged to complete a 12-week, supervised physical training program. Ninety patients underwent baseline testing and returned for repeat testing at 3 months in the interim, 49 completed the training program (Group I) and 41 chose not to participate (Group II). Change over time (from baseline to 3 months) in fatigue, exercise capacity, and skeletal muscle strength were assessed between the two groups. Results At baseline, there were no between-group differences for fatigue, DLCO %, FVC %, or exercise capacity [assessed by percent predicted six-minute walk distance (6MWD %) and Steep Ramp Test (SRT)]. The 6MWD for Group I improved between baseline and 3 months, while the 6MWD remained the same in Group II (F = 72.2, p\0.001). Group I showed a significantly larger decrease of fatigue compared with Group II (F = 6.27, p = 0.014). Lung function tests did not change in either group. Conclusion A supervised physical training program improves exercise capacity and fatigue among sarcoidosis patients and should be included in their management regimen.

Impact of Age and Sex on Response to Asthma Therapy

Abstract: Rationale: Age and sex are associated with differences in asthma prevalence and morbidity.Objectives: To determine if age and sex associate with distinct phenotypes and a variable response to therapy in subjects with mild to moderate asthma.Methods: We used Asthma Clinical Research Network data to determine the impact of age and sex on phenotypes and treatment failures among subjects participating in 10 trials from 1993 to 2003.Measurements and Main Results: A total of 1,200 subjects were identified (median age, 30.4 yr; male, 520 [43.3%]; female, 680 [56.7%]) and analyzed. A higher proportion of subjects greater than or equal to 30 years old experienced treatment failures (17.3% vs. 10.3%; odds ratio [OR], 1.82; confidence interval [CI], 1.30–2.54; P < 0.001), and rates increased proportionally with increasing age older than 30 across the cohort (OR per yr, 1.02 [CI, 1.01–1.04]; OR per 5 yr, 1.13 [CI, 1.04–1.22]; P < 0.001). Lower lung function and longer duration of asthma were associated with a higher ...

Comparison of actigraphy immobility rules with polysomnographic sleep onset latency in children and adolescents

Abstract: Purpose While actigraphy has gained popularity in pediatric sleep research, questions remain about the validity of actigraphy as an estimate of sleep-wake patterns In particular, there is little consistency in the field in terms of scoring rules used to determine sleep onset latency The purpose of this study was to evaluate different criteria of immobility as a measure of sleep onset latency in children and adolescents

Motivating Patient Adherence to Allergic Rhinitis Treatments

Abstract: Patient nonadherence significantly burdens the treatment of allergic rhinitis (AR). Fewer than half of prescribed doses of intranasal corticosteroid medication are taken. The challenges for immunotherapies are even greater. While sustained treatment for 3 to 5 years is required for full benefit, most patients receiving immunotherapy, either subcutaneous or sublingual, stop treatment within the first year. Although research into interventions to improve AR adherence is lacking, lessons learned from adherence interventions in other chronic health conditions can be applied to AR. Two well-established, overriding models of care-the chronic care model and patient-centered care-can improve adherence. The patient-centered care model includes important lessons for allergy providers in their daily practice, including understanding and targeting modifiable barriers to adherence. Additionally, recent studies have begun to leverage health information and communication technologies to reach out to patients and promote adherence, extending patient-centered interventions initiated by providers during office visits.

Interstitial lung disease in undifferentiated forms of connective tissue disease.

Abstract: The intersection of interstitial lung disease (ILD) and connective tissue disease (CTD) is complex and commonly includes the scenario whereby ILD is identified in patients with pre-existing, well-characterized forms of CTD, is the presenting manifestation of a well-characterized form of CTD, or arises within the context of a poorly defined, “undifferentiated” form of CTD. Determining that an ILD is CTD associated is important because this knowledge often impacts management and prognosis. Identifying occult CTD in patients with an “idiopathic” ILD can be challenging and requires a comprehensive, often multidisciplinary, evaluation. There is much uncertainty and controversy surrounding undifferentiated forms of CTD-associated ILD and prospective studies are needed to provide a better understanding of the natural history of these cohorts, how to best manage them, and to determine whether they behave similar to definite forms of CTD-associated ILD.

Assessment and management of connective tissue disease-associated interstitial lung disease.

Abstract: The intersection of the connective tissue diseases (CTD) and the interstitial lung diseases (ILD) is complex. Although often considered as a single entity, “CTD-ILD” actually reflects a heterogeneous spectrum of diverse CTDs and a variety of patterns of interstitial pneumonia. The evaluation of patients with CTD that develop ILD, or the assessment for underlying CTD in those presenting with presumed “idiopathic” ILD can be challenging and these evaluations can be optimized by effective multidisciplinary collaboration. When a diagnosis of CTD-ILD is confirmed, careful and thorough assessments to determine extra- versus intra-thoracic disease activity, and degrees of impairment are needed. Pharmacologic intervention with immunosuppression is the mainstay of therapy for all forms of CTD-ILD, but should be reserved only for those that demonstrate clinically significant and/or progressive disease. The management of CTD-ILD is not yet evidence based and there is a desperate need for controlled trials across the spectrum of CTD-ILD. Non-pharmacologic management strategies and addressing comorbidities or aggravating factors should be part of a comprehensive treatment plan for individuals with CTD-ILD.

Behavioral couples therapy for smoking cessation: A pilot randomized clinical trial.

Abstract: Heather LaChance, Patricia A. Cioe, Erin Tooley, Suzanne M. Colby, Timothy J. O€Farrell, andChristopher W. KahlerOnline First Publication, February 2, 2015. http://dx.doi.org/10.1037/adb0000051CITATIONLaChance, H., Cioe, P. A., Tooley, E., Colby, S. M., O€Farrell, T. J., & Kahler, C. W. (2015,February 2). Behavioral Couples Therapy for Smoking Cessation: A Pilot Randomized ClinicalTrial. Psychology of Addictive Behaviors. Advance online publication.http://dx.doi.org/10.1037/adb0000051

Regulation of IgE Responses by γδ T Cells.

Abstract: Immunoglobulin E (IgE) antibodies play a crucial role in host defense against parasite infections. However, inappropriate IgE responses are also involved in the pathogenesis of allergic diseases. The generation of IgE antibodies is a tightly controlled process regulated by multiple transcription factors, cytokines, and immune cells including γδ T cells. Accumulating evidence demonstrates that γδ T cells play a critical role in regulating IgE responses; however, both IgE-enhancing and IgE-suppressive effects are suggested for these cells in different experimental systems. In this review, we examine the available evidence and discuss the role of γδ T cells in IgE regulation both in the context of antigen-induced immune responses and in the state of partial immunodeficiency.

Methods for detection of respiratory diseases

Abstract: Disclosed are methods of identifying, predicting and treating subjects at risk for exacerbation or the presence of a respiratory disease, by detecting expression levels of one or more proteins associated with the respiratory disease, wherein said one or more proteins is selected from the group of CCL24, IL2RA, APOA4, GC, IgA, LPA, KLK3 F, FAS, NRCAM, TNFRSFIOC, IL12B, IL23A, RAGE, CCL20, ICAM1, SERPINA7, CDH13 and CDH1, and wherein said respiratory disease may be chronic obstructive pulmonary disease or emphysema.

Development of ATAQ-LAM: a tool to assess quality of life in Lymphangioleiomyomatosis

Abstract: Background Lymphangioleiomyomatosis (LAM) is a progressive lung disease that impairs health-related quality of life (HRQL).

Methods for Categorizing and Treating Subjects at Risk for Pulmonary Exacerbation and Disease Progression

Abstract: The present invention is related to novel methods for categorizing and treating a population of subjects that are at risk for increased pulmonary exacerbation and disease progression.

Immunosuppressive Management of Cardiac Sarcoidosis

Abstract: Sarcoidosis is a multi-system granulomatous disorder of yet unknown etiology that predominantly involves the lungs in over 90 % of cases but can also involve any organ in the body. Cardiac sarcoidosis is detected clinically in about 5 % of patients with sarcoidosis but on autopsy in as many as 40 % suggesting that the majority of cases may be underdiagnosed. Complications of cardiac sarcoidosis can include ventricular dysfunction, conduction abnormalities, ventricular arrhythmias and sudden cardiac death. These may occur suddenly, without warning in a previously asymptomatic or undiagnosed patient. There are currently no guidelines on how to definitively manage cardiac sarcoidosis. The goal of immunosuppressive (IS) therapy in cardiac sarcoidosis is to reverse any ongoing active granulomatous myocarditis, potentially prevent progression of myocardial granulomatous inflammation to scar tissue, and reduce or prevent the development of serious or life threatening cardiac complications. Management of cardiac sarcoidosis requires the collaborative effort of a sarcoidosis expert, an electrophysiologist, and a general cardiologist.

Patient-Centered Care for Sarcoidosis

Abstract: Caring for patients with sarcoidosis is a challenge. As a relatively rare disorder of unknown etiology, with the potential to affect multiple organ systems in an unpredictable fashion, a patient-centered rather than disease centered approach is very important. Some of the keys to a patient-centered approach include health literacy, patient education, shared decision-making, patient-centered outcomes, and a multidisciplinary team approach which actively involves the patient. These components for a patient-centered approach will be discussed in this chapter.

Method for treating pulmonary exacerbation and disease progression in subjects having cystic fibrosis

Abstract: The present invention is related to a method for determining pulmonary disease progression severity in a subject having cystic fibrosis and treating the subject according to the severity. The method comprises obtaining a whole blood sample from the subject; detecting the mRNA expression level of each of the following genes: TLR2, ADAM9, PLXND1, CD163, CD36, CD64, CSPG2, IL32, HPSE, HCA112; determining the severity of the pulmonary disease progression based on the subject's combined mRNA expression level of the genes; and treating the subject.

ANCA-associated renal vasculitis and acute exacerbation of scleroderma-associated interstitial lung disease: A case report

Abstract: In this report, we describe a patient with the unusual concomitant presentation of an acute exacerbation of systemic sclerosis-associated interstitial lung disease with the development of anti-neutrophil cytoplasmic antibody-associated active renal vasculitis. Systemic sclerosis is a connective tissue disease characterized by systemic autoimmunity and organ fibrosis and vasculopathy. Interstitial lung disease is identified in the majority of patients with systemic sclerosis and may have varying degrees of severity. Patients with systemic sclerosis-associated interstitial lung disease rarely develop acute exacerbations of interstitial lung disease. An overlap of systemic vasculitis has been reported to occur in patients with systemic sclerosis – yet this too is a rare occurrence. This case is unique in that it demonstrates the concomitant presentation of these rare clinical scenarios and serves to highlight the importance of a comprehensive and multidiscciplinary approach to the evaluation of patients in general, and systemic autoimmune patients in particular.

Cases in Cardiac Sarcoidosis

Abstract: Cardiac sarcoidosis continues to be a very challenging diagnosis and can prove to be even more challenging to manage. In this case series, the authors will walk the reader through difficult cases in the management of real-life cases of sarcoidosis with cardiac involvement. Imaging results, catheterization and biopsy results, clinical scenarios, and pitfalls in the care of these patients will be reviewed through case examples with summaries of key points.