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2.
Am J Respir Crit Care Med ; 201(8): e26-e51, 2020 Apr 15.
Artigo em Inglês | MEDLINE | ID: mdl-32293205

RESUMO

Background: The diagnosis of sarcoidosis is not standardized but is based on three major criteria: a compatible clinical presentation, finding nonnecrotizing granulomatous inflammation in one or more tissue samples, and the exclusion of alternative causes of granulomatous disease. There are no universally accepted measures to determine if each diagnostic criterion has been satisfied; therefore, the diagnosis of sarcoidosis is never fully secure.Methods: Systematic reviews and, when appropriate, meta-analyses were performed to summarize the best available evidence. The evidence was appraised using the Grading of Recommendations, Assessment, Development, and Evaluation approach and then discussed by a multidisciplinary panel. Recommendations for or against various diagnostic tests were formulated and graded after the expert panel weighed desirable and undesirable consequences, certainty of estimates, feasibility, and acceptability.Results: The clinical presentation, histopathology, and exclusion of alternative diagnoses were summarized. On the basis of the available evidence, the expert committee made 1 strong recommendation for baseline serum calcium testing, 13 conditional recommendations, and 1 best practice statement. All evidence was very low quality.Conclusions: The panel used systematic reviews of the evidence to inform clinical recommendations in favor of or against various diagnostic tests in patients with suspected or known sarcoidosis. The evidence and recommendations should be revisited as new evidence becomes available.

3.
Am Heart J ; 223: 106-109, 2020 May.
Artigo em Inglês | MEDLINE | ID: mdl-32240829

RESUMO

BACKGROUND: The diagnosis of cardiac sarcoidosis (CS) is challenging. Because of the current limitations of endomyocardial biopsy as a reference standard, physicians rely on advanced cardiac imaging, multidisciplinary evaluation, and diagnostic criteria to diagnose CS. AIMS: To compare the 3 main available diagnostic criteria in patients clinically judged to have CS. METHODS: We prospectively included patients clinically judged to have CS by a multidisciplinary sarcoidosis team from November 2016 to October 2017. We included only incident cases (diagnosis of CS within 1 year of inclusion). We applied retrospectively the following diagnostic criteria: the World Association of Sarcoidosis and Other Granulomatous Diseases (WASOG), the Heart Rhythm Society (HRS), and the Japanese Circulation Society (JCS) 2016 criteria. RESULTS: We identified 69 patients. Diagnostic criteria classified patients as follows: WASOG as highly probable (1.4%), probable (52.2%), possible (0%), some criteria (40.6%), and no criteria (5.8%); HRS as histological diagnosis (1.4%), probable (52.2%), some criteria (40.6%), and no criteria (5.8%); JCS as histological diagnosis (1.4%), clinical diagnosis (58%), some criteria (39.1%), and no criteria (1.4%). Concordance was high between WASOG and HRS (κ = 1) but low between JCS and the others (κ = 0.326). CONCLUSIONS: A high proportion of patients clinically judged to have CS are unable to be classified according to the 3 main diagnostic criteria. There is low concordance between JCS criteria and the other 2 criteria (WASOG and HRS).

5.
Eur Respir J ; 55(5)2020 May.
Artigo em Inglês | MEDLINE | ID: mdl-32139456

RESUMO

INTRODUCTION: Sarcoidosis-associated pulmonary hypertension (SAPH) is associated with reduced survival in single-centre studies. The international Registry for SAPH (ReSAPH) with long-term follow-up was established to enrich our knowledge of this complication of sarcoidosis. This analysis aims to elucidate factors associated with reduced transplant-free survival in SAPH patients. METHODS: ReSAPH contains prospectively collected outcomes of SAPH patients since the time of registry enrolment. Information analysed includes right heart catheterisation data, pulmonary function testing, chest radiography, Scadding stage and 6-min walk distance (6MWD), among others. Cox regression models were used to identify independent predictors of transplant-free survival. RESULTS: Data from 215 patients followed for a mean±sd 2.5±1.9 years were available for analysis. In the 159 precapillary patients, the Kaplan-Meier-adjusted 1-, 3- and 5-year transplant-free survival was 89.2%, 71.7% and 62.0%, respectively. Kaplan-Meier-adjusted 1-, 3- and 5-year transplant-free survival in the incident group was 83.5%, 70.3% and 58.3%, respectively, and in the prevalent group was 94.7%, 72.2% and 66.3%, respectively. Patients with reduced diffusing capacity of the lung for carbon monoxide (D LCO) (<35% predicted) and 6MWD <300 m in the precapillary cohort had significantly worse transplant-free survival. Reduced 6MWD and preserved forced expiratory volume (FEV1)/forced vital capacity (FVC) ratio were identified as independent risk factors for reduced transplant-free survival in the precapillary cohort. CONCLUSION: Reduced D LCO (<35% pred) and 6MWD (<300 m) at the time of registry enrolment were associated with reduced transplant-free survival in the overall precapillary cohort. Preserved FEV1/FVC ratio was identified as an independent risk factor for worsened outcomes.

6.
Am Heart J ; 220: 246-252, 2020 02.
Artigo em Inglês | MEDLINE | ID: mdl-31911261

RESUMO

Approximately 5% of patients with sarcoidosis have clinically manifest cardiac involvement. Clinical features of Cardiac Sarcoidosis are dependent on the location, extent, and activity of the disease. First line therapy is usually with prednisone and this is recommended based on clinician experience, expert opinion and small observational cohorts. There are no published clinical trials in cardiac sarcoidosis and multiple experts in the field have called for randomized clinical trials to answer important patient care questions. Corticosteroid are associated with multiple adverse effects including hypertension, diabetes, weight gain, osteoporosis, and increased risk of infections. In contrast Methotrexate is generally well tolerated and is increasingly used in other forms of sarcoidosis. OBJECTIVES: The Cardiac Sarcoidosis Multi-Center Randomized Controlled Trial (CHASM CS-RCT; NCT03593759) is a multicenter randomized controlled trial designed to evaluate the optimal initial treatment strategy for patients with active cardiac sarcoidosis. We hypothesize that (1) a low dose prednisone/methotrexate combination will have non-inferior efficacy to standard dose prednisone and that (2) the low dose prednisone/ methotrexate combination will result in significantly better quality of life than standard dose prednisone, as a result of reduced burden of side effects. METHODS/DESIGN: Eligible study subjects will have active clinically manifest cardiac sarcoidosis presenting with one or more of the following clinical findings: advanced conduction system disease, significant sinus node dysfunction, non-sustained or sustained ventricular arrhythmia, left ventricular dysfunction or right ventricular dysfunction. Subjects will be randomized in a 1:1 ratio to prednisone 0.5 mg/kg/day for 6 months (maximum dose 30 mg daily) OR to prednisone 20 mg daily for 1 month, then 10 mg daily for 1 month, then 5 mg daily for one month then stop AND methotrexate 15-20 mg once weekly for 6 months. The primary endpoint is summed perfusion rest score on 6-month PET (blinded core-lab review). The summed perfusion rest score is measure of myocardial fibrosis/scar. The design is non-inferiority with a sample size of 97 per group. DISCUSSION: Given the multiorgan system potential adverse side effects of prednisone, proving noninferiority of an alternate regimen would be sufficient to make the alternative compare favorably to standard dose steroids. This is the first ever clinical trial in cardiac sarcoidosis and thus in addition to the listed goals of the trial, we will also establish a multi-center, multinational cardiac sarcoidosis clinical trials network. Such a collaborative infrastructure will enable a new era of high quality data to guide physicians when treating cardiac sarcoidosis patients.


Assuntos
Cardiomiopatias/tratamento farmacológico , Glucocorticoides/administração & dosagem , Metotrexato/administração & dosagem , Prednisona/administração & dosagem , Ensaios Clínicos Controlados Aleatórios como Assunto , Sarcoidose/tratamento farmacológico , Cardiomiopatias/complicações , Esquema de Medicação , Quimioterapia Combinada , Estudos de Equivalência como Asunto , Glucocorticoides/efeitos adversos , Humanos , Estudos Multicêntricos como Assunto , Prednisona/efeitos adversos , Estudos Prospectivos , Qualidade de Vida , Projetos de Pesquisa , Sarcoidose/complicações
7.
Chest ; 157(5): 1188-1198, 2020 May.
Artigo em Inglês | MEDLINE | ID: mdl-31954102

RESUMO

BACKGROUND: Limited data are available on the association between clinically measured disease severity markers and quality of life (QOL) in idiopathic pulmonary fibrosis (IPF). The study examined the associations between objective disease severity metrics and QOL in a contemporary IPF population. METHODS: This study evaluated baseline data from patients enrolled in the multicenter, US-based Idiopathic Pulmonary Fibrosis Prospective Outcomes Registry between June 2014 and July 2018. Disease severity metrics included FVC % predicted, diffusing capacity for carbon monoxide (Dlco) % predicted, supplemental oxygen use with activity, supplemental oxygen use at rest, and two summary scores (the Gender-Age-Lung Physiology index, based on gender, age, and % predicted values for Dlco and FVC; and the Composite Physiologic Index, based on % predicted values for Dlco, FVC, and FEV1). Multivariable adjusted regression models were used to examine cross-sectional associations between each severity measure and St. George's Respiratory Questionnaire (SGRQ) total score. RESULTS: Among 829 patients with complete SGRQ data, the median (interquartile range) SGRQ score at enrollment was 40 (26-53), with higher scores indicating worse QOL. Modest SGRQ impairments were observed with increasing Gender-Age-Lung Physiology score (2.9 [1.8-4.0] per 1-point increase] and with increasing Composite Physiologic Index scores (3.0 [2.4-3.6] per 5-point increase). Substantial SGRQ impairments were observed for oxygen use with activity (15.6 [12.9-18.2]), oxygen use at rest (16.2 [13.0-19.4]), and decreasing Dlco (5.0 [4.0-6.1] per 10% decrease in % predicted). CONCLUSIONS: Objective measures of disease severity, including severity scores, physiologic parameters, and supplemental oxygen use, are associated with worse QOL in patients with IPF. TRIAL REGISTRY: ClinicalTrials.gov; No.: NCT01915511; URL: www.clinicaltrials.gov.

8.
Pharmacotherapy ; 40(1): 33-39, 2020 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-31705703

RESUMO

INTRODUCTION: Positive hemodynamic response to vasopressin after 6 hours of infusion was independently associated with lower mortality in a previous retrospective study of patients with septic shock. However, factors previously associated with higher plasma vasopressin concentration were not associated with response, and the relationship between plasma vasopressin concentration and hemodynamic response has not been evaluated. OBJECTIVES: This cross-sectional study compared plasma vasopressin concentrations in hemodynamic responders and nonresponders to vasopressin in patients with septic shock to evaluate plasma vasopressin concentration as a therapeutic target for hemodynamic response to vasopressin. METHODS: Adult patients with septic shock were included if they were treated with fixed-dose vasopressin as an adjunct to catecholamines for at least 3 hours. Patients were assigned to groups based on vasopressin response. RESULTS: Ten hemodynamic responders to vasopressin and eight nonresponders were included. Blood samples for plasma vasopressin concentration were collected 3-6 hours after vasopressin initiation. Baseline characteristics were similar between groups. No difference was detected in plasma vasopressin concentrations between hemodynamic responders and nonresponders (median 88.6 pg/ml [interquartile range (IQR) 84.4-107.5 pg/ml] vs 89.9 pg/ml [IQR 67.5-157.4 pg/ml], p=0.79, respectively). We also did not detect a difference between groups after correcting for vasopressin dose; median vasopressin plasma concentration per 0.01 units/minute of vasopressin infusion for responders was 25.9 pg/ml (IQR 21.8-31.8 pg/ml) versus 29.5 pg/ml (IQR 23.0-57.5 pg/ml, p=0.48) for nonresponders. No difference in clinical outcomes was detected between groups. The findings were robust to multiple sensitivity analyses. CONCLUSIONS: This study does not support the use of plasma vasopressin concentrations as a therapeutic target to predict hemodynamic response to exogenous vasopressin in septic shock.

10.
Expert Rev Respir Med ; 14(3): 285-298, 2020 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-31868547

RESUMO

Introduction: Care of patients with sarcoidosis requires familiarity with its natural history as well as of various immunosuppressants employed in its treatment. We would like to share our approach to management based on our experience and understanding of the relevant literature.Areas covered: Asymptomatic patients with pulmonary sarcoidosis ought to be managed conservatively. Systemic sarcoidosis with burdensome symptoms usually responds to corticosteroids, but one needs to consider the risk of long-term steroid toxicity as well as relapse. Rapidly tapering steroids can decrease cumulative exposure without compromising efficacy. Steroid-sparing anti-sarcoidosis (SSAS) agents take longer to act and are associated with unique but mostly reversible toxicities. Used judiciously and with careful monitoring, they effectively suppress granulomatous inflammation. Patients intolerant of or failing to improve with a particular drug can be switched to another, and occasionally combination therapy with two SSAS agents might prove effective. A small proportion of patients are refractory, but often achieve control and sometimes remission with stepping up to biologic therapy.Expert opinion: Adopting a strategy of early SSAS therapy ought to effectively control sarcoidosis and avoid harm from prolonged corticosteroid dosing.

11.
Artigo em Inglês | MEDLINE | ID: mdl-31825646

RESUMO

RATIONALE: Socioeconomic factors are associated with worse disease severity at presentation in sarcoidosis, but the relative importance of socioeconomic variables on morbidity and disease burden has not been fully elucidated. OBJECTIVES: To determine the association between income and sarcoidosis outcomes after controlling for socioeconomic and disease-related factors. METHODS: Using the Sarcoidosis Advanced Registry for Cures (SARC) database, we analyzed data from 2318 United States sarcoidosis patients to determine the effect of income and other variables on outcomes. We divided comorbidities arising after diagnosis into those likely related to steroid use and those likely related to sarcoidosis. We assessed the development of health-related, functional and socioeconomic outcomes following the diagnosis of sarcoidosis. MEASUREMENTS AND MAIN RESULTS: In multivariate analysis, low income patients had significantly higher rates of new sarcoidosis related comorbidities [<$35,000 OR 2.4 (1.7-3.3), $35,000-$85,000 OR 1.4 (1.1-1.9), >$85,000 (REF)], new steroid related comorbidities [<$35,000 OR 1.3 (0.9-2.0), $35,000-$85,000 OR 1.5 (1.1-2.1), >$85,000 (REF)], had lower health-related quality of life as assessed by the Sarcoidosis Health Questionnaire (p<0.001) and experienced more impact on family finances [<$35,000 OR 7.9 (4.9-12.7), $35,000-$85,000 OR 2.7 (1.9-3.9), >$85,000 (REF)]. The use of supplemental oxygen, need for assistive devices, and job loss were more common in lower income patients. Development of comorbidities after diagnosis of sarcoidosis occurred in 63% of patients and were strong independent predictors of poor outcomes. In random forest modeling, income was consistently a leading predictor of outcome. CONCLUSIONS: These results suggest the burden from sarcoidosis preferentially impacts the economically disadvantaged.

12.
ERJ Open Res ; 5(4)2019 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-31687368

RESUMO

Our study presents findings on a previously developed standard set of clinical outcome data for pulmonary sarcoidosis patients. We aimed to assess whether changes in outcome varied between the different centres and to evaluate the feasibility of collecting the standard set retrospectively. This retrospective observational comparative benchmark study included six interstitial lung disease expert centres based in the Netherlands, Belgium, the UK and the USA. The standard set of outcome measures included 1) mortality, 2) changes in pulmonary function (forced vital capacity (FVC), forced expiratory volume in 1 s, diffusing capacity of the lung for carbon monoxide), 3) soluble interleukin-2 receptor (sIL-2R) change, 4) weight changes, 5) quality-of-life (QoL) measures, 6) osteoporosis and 7) clinical outcome status (COS). Data collection was considered feasible if the data were collected in ≥80% of all patients. 509 patients were included in the retrospective cohort. In total six patients died, with a mean survival of 38±23.4 months after the diagnosis. Centres varied in mean baseline FVC, ranging from 110 (95% CI 92-124)% predicted to 99 (95% CI 97-123)% pred. Mean baseline body mass index (BMI) of patients in the different centres varied between 27 (95% CI 23.6-29.4) kg·m-2 and 31.8 (95% CI 28.1-35.6) kg·m-2. 310 (60.9%) patients were still on systemic therapy 2 years after the diagnosis. It was feasible to measure mortality, changes in pulmonary function, weight changes and COS. It is not (yet) feasible to retrospectively collect sIL-2R, osteoporosis and QoL data internationally. This study shows that data collection for the standard set of outcome measures for pulmonary sarcoidosis was feasible for four out of seven outcome measures. Trends in pulmonary function and BMI were similar for different hospitals when comparing different practices.

13.
BMJ ; 367: l5553, 2019 Oct 22.
Artigo em Inglês | MEDLINE | ID: mdl-31641045

RESUMO

Sarcoidosis is a highly variable granulomatous multisystem syndrome. It affects individuals in the prime years of life; both the frequency and severity of sarcoidosis are greater in economically disadvantaged populations. The diagnosis, assessment, and management of pulmonary sarcoidosis have evolved as new technologies and therapies have been adopted. Transbronchial needle aspiration guided by endobronchial ultrasound has replaced mediastinoscopy in many centers. Advanced imaging modalities, such as fluorodeoxyglucose positron emission tomography scanning, and the widespread availability of magnetic resonance imaging have led to more sensitive assessment of organ involvement and disease activity. Although several new insights about the pathogenesis of sarcoidosis exist, no new therapies have been specifically developed for use in the disease. The current or proposed use of immunosuppressive medications for sarcoidosis has been extrapolated from other disease states; various novel pathways are currently under investigation as therapeutic targets. Coupled with the growing recognition of corticosteroid toxicities for managing sarcoidosis, the use of corticosteroid sparing anti-sarcoidosis medications is likely to increase. Besides treatment of granulomatous inflammation, recognition and management of the non-granulomatous complications of pulmonary sarcoidosis are needed for optimal outcomes in patients with advanced disease.


Assuntos
Glucocorticoides/uso terapêutico , Hipertensão Pulmonar/prevenção & controle , Imunossupressores/uso terapêutico , Fibrose Pulmonar/prevenção & controle , Sarcoidose Pulmonar/diagnóstico , Biomarcadores/sangue , Broncoscopia/métodos , Aspiração por Agulha Fina Guiada por Ultrassom Endoscópico/métodos , Fluordesoxiglucose F18/administração & dosagem , Humanos , Hipertensão Pulmonar/etiologia , Hipertensão Pulmonar/mortalidade , Incidência , Pulmão/diagnóstico por imagem , Pulmão/patologia , Tomografia por Emissão de Pósitrons/métodos , Prognóstico , Fibrose Pulmonar/etiologia , Fibrose Pulmonar/mortalidade , Sarcoidose Pulmonar/complicações , Sarcoidose Pulmonar/tratamento farmacológico , Sarcoidose Pulmonar/epidemiologia , Resultado do Tratamento
14.
Pulm Pharmacol Ther ; 59: 101839, 2019 12.
Artigo em Inglês | MEDLINE | ID: mdl-31518649

RESUMO

PURPOSE: Although safety and tolerability of approved antifibrotics has been reported extensively, little is known about their effects on weight. We analyzed predictors of weight change after one year of uninterrupted antifibrotic therapy in patients followed at our institution's interstitial lung disease clinic. METHODS/RESULTS: We identified 80 patients on antifibrotic therapy (44 pirfenidone/36 nintedanib) with at least one year of follow-up and no therapy interruptions. Thirty-five patients (44%) lost more than 5% of their baseline body weight, and 11 (19%) lost more than 10%. A higher proportion of patients on nintedanib experienced a clinically significant weight loss (>5%) versus pirfenidone (61% vs 30%, p = 0.005). Univariate and multivariate analyses identified nintedanib therapy and a higher composite physiologic index (CPI) as predictors of weight loss. CONCLUSIONS: Weight loss is common among IPF patients on antifibrotic therapy. Nintedanib therapy and more advanced disease were identified as predictors of weight loss in this population.

15.
Am J Cardiol ; 124(10): 1630-1635, 2019 Nov 15.
Artigo em Inglês | MEDLINE | ID: mdl-31500815

RESUMO

Cardiac sarcoidosis (CS) is frequently difficult to treat. Infliximab (IFX) is useful for extracardiac sarcoidosis, but its use in CS has been limited due to concerns about cardiotoxicity and an FDA blackbox warning about use in heart failure. We reviewed 36 consecutive patients treated with infliximab for CS refractory to standard therapies. IFX was initiated for patients with refractory dysrhythmias, moderate to severe cardiomyopathy, and evidence of persistent F-18 fluorodeoxyglucose uptake on positron emission tomography scan, despite standard therapies. We compared the prednisone dose, ejection fraction (EF), and dysrhythmias before and after IFX therapy. The prednisone-equivalent steroid dose decreased from a median of 20 mg at initiation of infliximab to 7.5 at 6 months and 5 mg at 12 months postinitiation of infliximab (p <0.001). In the 25 patients with serial EF measurements, no statistically significant difference was detected in EF (41% at baseline, 42% at 6 months). Of the 16 patients with serial dysrhythmia data, there was a trend toward reduction of percent of patients with ventricular tachycardia (VT), from 32% at baseline, to 22% at 6 months and 19% at 12 months (p = 0.07). Adverse events were common, occurring in 6 of 36 patients, with 3 of 36 patients stopping infliximab for a prolonged period. In responder analysis, 24 patients improved in at least 1 of 3 outcome categories. In conclusion, infliximab may be useful for refractory cardiac sarcoidosis.

16.
BMJ Case Rep ; 12(8)2019 Aug 21.
Artigo em Inglês | MEDLINE | ID: mdl-31439559

RESUMO

Acute fibrinous organising pneumonia is distinct from the classic diffuse alveolar damage, organising pneumonia and eosinophilic pneumonia. A 52-year-old woman presented with fever, productive cough, night sweats and left-sided pleuritic chest pain for a week. Physical examination was significant only for decreased breath sounds in the left infraclavicular area laterally. Imaging studies revealed a peripheral thick-walled left upper lobe cavitary lesion, left lower lobe consolidation and an enlarged subcarinal lymph node. She was treated with doxycycline for 10 days without improvement. Pertinent laboratory tests, microbiologic workup and fibre-optic bronchoscopy were non-diagnostic and a CT-guided left upper lobe lung biopsy revealed acute fibrinous organising pneumonia. She was treated with azithromycin with complete resolution of symptoms. To our knowledge, this is the first reported case of acute fibrinous organising pneumonia presenting as a cavitary lung lesion and the first with treatment response to azithromycin.


Assuntos
Pneumonia em Organização Criptogênica/diagnóstico , Antibacterianos/uso terapêutico , Azitromicina/uso terapêutico , Broncoscopia , Tosse/etiologia , Pneumonia em Organização Criptogênica/complicações , Pneumonia em Organização Criptogênica/diagnóstico por imagem , Pneumonia em Organização Criptogênica/tratamento farmacológico , Diagnóstico Diferencial , Feminino , Febre/etiologia , Humanos , Pessoa de Meia-Idade , Tomografia Computadorizada por Raios X
17.
Ann Am Thorac Soc ; 16(11): 1341-1350, 2019 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-31322914

RESUMO

Increasing awareness of cardiac manifestations of sarcoidosis and the widespread availability of advanced imaging tests have led to a tidal wave of interest in a condition that was once considered rare. In this Focused Review, we explore important clinical questions that may confront specialists faced with possible cardiac involvement. In the absence of an ideal reference standard, three main sets of clinical criteria exist: the Japanese Ministry of Health and Welfare, the Heart Rhythm Society, and the World Association for Sarcoidosis and Other Granulomatous Disorders criteria. Once cardiac sarcoidosis is suspected, clinicians should be familiar with the prevalence of the disease in different clinical scenarios. Before obtaining advanced cardiac imaging, electrocardiogram, ambulatory electrocardiogram, echocardiogram, and B-type natriuretic peptide may be useful. The available therapies for cardiac sarcoidosis include immunosuppression, antiarrhythmic medications, heart failure medications, device therapy, ablation therapy, and heart transplantation. Contemporary data suggest that long-term survival in cardiac sarcoidosis is better than previously believed. There is no randomized controlled trial demonstrating benefits of screening, but screening is recommended based on observational data.

18.
Respir Res ; 20(1): 105, 2019 May 30.
Artigo em Inglês | MEDLINE | ID: mdl-31142314

RESUMO

BACKGROUND: Idiopathic pulmonary fibrosis (IPF) is a progressive disease with a variable clinical course and high mortality. We used data from a large national US registry of patients with IPF to investigate relationships between patient characteristics, including markers of disease severity, and mortality. METHODS: The analysis cohort comprised patients enrolled in the IPF-PRO Registry from its inception on 5 June 2014 to 26 October 2017. The primary criterion for inclusion in this registry is that patients must be diagnosed or confirmed with IPF at the enrolling centre within 6 months. Associations between patient characteristics and markers of disease severity at enrolment and mortality outcomes were investigated using univariable, multivariable and adjustment models. RESULTS: Among 662 patients enrolled, 111 patients died or had a lung transplant over a follow-up period of 30 months. The probability of being free of both events at month 30 was 50.6% (95% CI: 40.0, 60.2). When patient characteristics and markers of disease severity were jointly examined in a multivariable analysis, oxygen use at rest (hazard ratio [HR] 2.44 [95% CI: 1.45, 4.10]), lower forced vital capacity (FVC) % predicted (HR 1.28 [95% CI: 1.10, 1.49] per 10% decrease) and diffusion capacity for carbon monoxide (DLco) % predicted (HR 1.25 [95% CI: 1.04, 1.51] per 10% decrease) were significantly associated with increased risk of death or lung transplant. The risk of death or lung transplant increased with increasing age in patients ≥62 years old (HR 1.18 [95% CI: 0.99, 1.40] per 5-year increase), and decreased with increasing age in patients <62 years old (HR 0.60 [95% CI: 0.39, 0.92] per 5-year increase). CONCLUSIONS: In an observational US registry of patients with IPF, oxygen use at rest, lower FVC % predicted, and lower DLco % predicted were associated with risk of death or lung transplant. An audio podcast of the lead author discussing these data can be downloaded from: http://www.usscicomms.com/respiratory/snyder/IPF-PROsurvival1/ . TRIAL REGISTRATION: ClinicalTrials.gov number: NCT01915511 .


Assuntos
Fibrose Pulmonar Idiopática/mortalidade , Fibrose Pulmonar Idiopática/cirurgia , Transplante de Pulmão/mortalidade , Sistema de Registros , Idoso , Idoso de 80 Anos ou mais , Estudos de Coortes , Feminino , Seguimentos , Humanos , Fibrose Pulmonar Idiopática/diagnóstico , Transplante de Pulmão/tendências , Masculino , Pessoa de Meia-Idade , Mortalidade/tendências , Valor Preditivo dos Testes
19.
BMJ Open Respir Res ; 6(1): e000394, 2019.
Artigo em Inglês | MEDLINE | ID: mdl-30956806

RESUMO

Introduction: Routine and international comparison of clinical outcomes enabling identification of best practices for patients with pulmonary sarcoidosis is lacking. The aim of this study was to develop a standard set of outcome measures for pulmonary sarcoidosis, using the value-based healthcare principles. Methods: Six expert clinics for interstitial lung diseases in four countries participated in a consensus-driven RAND-modified Delphi study. A mixed-method approach was applied for the identification of an outcome measures set and initial conditions for patients with pulmonary sarcoidosis. The expert team consisted of multidisciplinary professionals (n=14) from Cleveland Clinic, Cincinnati MC, Erasmus MC, Leuven UZ, Royal Brompton and St. Antonius Hospital. During a ranking process, participants were instructed to rank variables on a scale from 1 to 10 based on whether it has (1) impact of the outcome on quality of life, (2) impact of quality of care on the outcome and (3) the number of patients negatively affected by the outcome. Results: An outcome measures set was defined consisting of seven outcome measures: mortality, pulmonary function, soluble interleukin-2 receptor change as an activity biomarker, weight gain, quality of life, osteoporosis and clinical outcome status. Discussion: Collecting outcomes in pulmonary sarcoidosis internationally and the use of a broadly accepted set can enable international comparison. Differences in outcomes can potentially be used as a starting point for quality improvement initiatives.

20.
Am J Respir Crit Care Med ; 200(2): 160-167, 2019 07 15.
Artigo em Inglês | MEDLINE | ID: mdl-31034241

RESUMO

Over the past decade, several large registries of patients with idiopathic pulmonary fibrosis (IPF) have been established. These registries are collecting a wealth of longitudinal data on thousands of patients with this rare disease. The data collected in these registries will be complementary to data collected in clinical trials because the patient populations studied in registries have a broader spectrum of disease severity and comorbidities and can be followed for a longer period of time. Maintaining the quality and completeness of registry databases presents administrative and resourcing challenges, but it is important to ensuring the robustness of the analyses. Data from patient registries have already helped improve understanding of the clinical characteristics of patients with IPF, the impact that the disease has on their quality of life and survival, and current practices in diagnosis and management. In the future, analyses of biospecimens linked to detailed patient profiles will provide the opportunity to identify biomarkers linked to disease progression, facilitating the development of precision medicine approaches for prognosis and therapy in patients with IPF.


Assuntos
Fibrose Pulmonar Idiopática , Sistema de Registros , Bancos de Espécimes Biológicos , Comorbidade , Progressão da Doença , Humanos , Estudos Observacionais como Assunto , Índice de Gravidade de Doença
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