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BACKGROUND: The evolution in pulmonary arterial hypertension (PAH) management has been summarised in three iterations of the European Society of Cardiology/European Respiratory Society (ESC/ERS) guidelines. No study has assessed whether changes in management, as reflected in the changing guidelines, has translated to improved long-term survival in PAH. METHODS: We performed a mixed retrospective/prospective analysis of treatment-naïve, incident PAH patients (n=392) diagnosed at three major centres in Canada from 2009 to 2021. Patients were divided into two groups based on their diagnosis date and in accordance with the 2009 and 2015 ESC/ERS guideline iterations. Overall survival was assessed based on date of diagnosis and initial treatment strategy (i.e. monotherapy versus combination therapy). RESULTS: There was a shift towards more aggressive upfront management with combination therapy in Canada after the publication of the 2015 ESC/ERS guidelines (10.4% and 30.8% in patients from 2009 to 2015 and 36.0% and 57.4% in patients diagnosed after 2015 for baseline and 2-year follow-up, respectively). A key factor associated with combination therapy after 2015 was higher pulmonary vascular resistance (p=0.009). The 1-, 3- and 5-year survival rates in Canada were 89.2%, 75.6% and 56.0%, respectively. Despite changes in management, there was no improvement in long-term survival before and after publication of the 2015 ESC/ERS guidelines (p=0.53). CONCLUSIONS: There was an increase in the use of initial and sequential combination therapy in Canada after publication of the 2015 ESC/ERS guidelines, which was not associated with improved long-term survival. These data highlight the continued difficulties of managing this aggressive pulmonary disease in an era without a cure.
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Cardiologia , Hipertensão Arterial Pulmonar , Hipertensão Pulmonar Primária Familiar/terapia , Humanos , Estudos Retrospectivos , Taxa de SobrevidaRESUMO
BACKGROUND: Combined post- and precapillary pulmonary hypertension (CpcPH) portends poor outcomes in pulmonary hypertension related to left heart disease (PH-LHD). While recent evidence does not support the use of targeted pulmonary arterial hypertension (PAH) therapy in PH-LHD, there is a lack of clinical data on their use in CpcPH. We evaluated the outcomes in patients with CpcPH treated with PAH therapies. METHODS: Retrospectively, 50 patients meeting hemodynamic criteria of CpcPH and started on PAH-targeted drugs were identified. Fifty age- and gender-matched PAH patients were chosen as controls. We evaluated the change in 6-minute walk distance, World Health Organization functional class (FC), tricuspid annular plane systolic excursion, BNP or NT-proBNP, and pulmonary artery systolic pressure at 3, 6, 12, and 24 months of follow-up. RESULTS: After adjusting for age and gender, there was no improvement in World Health Organization FC in CpcPH over 2 years (odds ratio of change to FC I/II 1.01, 95% CI: 0.98-1.04). There was no significant improvement in 6-minute walk distance (ß coefficient 0.21, 95% CI: -0.98 to 1.4), reduction in BNP/NT-proBNP (ß coefficient -12.16, 95% CI: -30.68 to 6.37), increase in tricuspid annular plane systolic excursion (ß coefficient 0.074, 95% CI: 0.010-0.139), or decrease in pulmonary artery systolic pressure (0.996, 95% CI: 0.991-1.011) in CpcPH with therapy. There was higher mortality in CpcPH compared to PAH on treatment (24% vs 4%, Pâ¯=â¯.003). CONCLUSIONS: There were no improvements in symptoms, exercise capacity, or echocardiographic parameters with PAH-targeted therapy in CpcPH. Further studies into potential treatments benefiting this population are needed.
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Anti-Hipertensivos/uso terapêutico , Hemodinâmica/fisiologia , Hipertensão Pulmonar/tratamento farmacológico , Ecocardiografia , Feminino , Seguimentos , Humanos , Hipertensão Pulmonar/diagnóstico , Hipertensão Pulmonar/fisiopatologia , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Resultado do TratamentoRESUMO
INTRODUCTION: Tracheobronchomegaly (Mounier-Kuhn Syndrome) is a rare disease characterized by tracheal enlargement and associated loss of elastic fibers in the trachea and main bronchi. MATERIALS: MEDLINE, Index Medicus, and other databases were searched with pre-defined criteria to identify cases of tracheobronchomegaly (TBM). Two new cases of TBM were also identified from the Provincial Medical Genetics Program of British Columbia. RESULTS: We identified 166 publications describing 365 occurrences of TBM. We observed that affected individuals could be grouped into subgroups according to clinical features. Type 1A (105 individuals) consists of infants who developed TBM after having undergone fetoscopic tracheal occlusion, and Type 1B patients (24 individuals) are infants and children who developed TBM after prolonged intubation. Type 2 individuals developed TBM following recurrent pulmonary infections (2A) (14 individuals) or pulmonary fibrosis (2B) (10 individuals). Type 3 represents TBM with evidence of extra-pulmonary elastolysis (18 individuals), and Type 4 denotes the development of TBM with no clear predisposing factors (196 individuals). Both of our patients had TBM and evidence of extra-pulmonary elastolysis. As well, one patient had a mildly dilated aortic root, which is a previously unreported co-occurrence. CONCLUSION: We introduce a novel classification scheme, which may sort patients into etiologically distinct groups, furthering our understanding of its pathogenesis and potentially, prevention or therapy. We also hypothesize that TBM and generalized elastolysis may have etiological commonalities, suggesting a need for further study.
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Traqueobroncomegalia/classificação , Traqueobroncomegalia/etiologia , Cútis Laxa/complicações , Fetoscopia/efeitos adversos , Humanos , Lactente , Intubação Intratraqueal/efeitos adversos , Masculino , Pessoa de Meia-Idade , Fibrose Pulmonar/complicações , Infecções Respiratórias/complicaçõesRESUMO
Pulmonary hypertension (PH) is a rare condition associated with significant morbidity and mortality. The priorities for future research in PH according to patients, caregivers, and clinicians have not been established. We performed a James Lind Alliance priority setting partnership in Canada. An initial survey of 249 respondents (76% patients and/or caregivers, 12.5% clinicians) generated 1588 questions, which were combined into 187 summary questions. An evidence check identified 352 systematic reviews and guidelines which were mapped to the summary questions, which determined that 157 summary questions were unanswered by existing research. An interim prioritisation survey (240 respondents, 66% patients and/or caregivers, 18% clinicians) asked respondents to choose which of the 157 summary questions were among the 30-40 most important. In a final workshop patients, caregivers, and clinicians discussed and ranked the top 25 questions from the interim survey to identify the Top 10 PH research priorities. These results will inform researchers and funding bodies about patient, caregiver, and clinician priorities for future research on PH.
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Pesquisa Biomédica , Hipertensão Pulmonar , Humanos , Hipertensão Pulmonar/terapia , Prioridades em Saúde , Cuidadores , Inquéritos e QuestionáriosRESUMO
Background: Risk stratification is fundamental in the management of pulmonary arterial hypertension (PAH). Pulmonary artery pulsatility index (PAPi), defined as pulmonary arterial pulse pressure divided by right atrial pressure (RAP), is a hemodynamic index shown to predict acute right ventricular (RV) dysfunction in several settings. Our objective was to test the prognostic utility of PAPi in a diverse multicentre cohort of patients with PAH. Methods: A multicentre retrospective cohort study of consecutive adult patients with a new diagnosis of PAH on right heart catheterization between January 2016 and December 2020 was undertaken across 4 major centres in Canada. Hemodynamic data, clinical data, and outcomes were collected. The association of PAPi and other hemodynamic variables with mortality was assessed by receiver-operating characteristic curves and Cox proportional hazards modeling. Results: We identified 590 patients with a mean age of 61.4 ± 15.5 years, with 66.3% being female. A low PAPi (defined as < 5.3) was associated with higher mortality at 1 year: 10.2% vs 5.2% (P = 0.02). In a multivariable model including age, sex, body mass index, and functional class, a low PAPi was associated with mortality at 1 year (area under the curveof 0.64 (95% confidence interval 0.55-0.74). However, high RAP (> 8 mm Hg) was similarly predictive of mortality, with an area under the curve of 0.65. Conclusion: PAPi was associated with mortality in a large incident PAH cohort. However, the discriminative value of PAPi was not higher than that of RAP alone.
Contexte: La stratification des risques est fondamentale dans la prise en charge de l'hypertension artérielle pulmonaire (HTAP). L'indice de pulsatilité des artères pulmonaires (iPAP), défini comme la pression différentielle dans les artères pulmonaires divisée par la pression auriculaire droite (PAD), est un indice hémodynamique qui s'est révélé prédictif d'une dysfonction ventriculaire droite (VD) aiguë dans plusieurs situations. Notre objectif était d'évaluer l'utilité pronostique de l'iPAP dans une cohorte multicentrique diversifiée de patients atteints d'HTAP. Méthodologie: Une étude de cohorte multicentrique rétrospective de patients adultes consécutifs atteints d'une HTAP nouvellement diagnostiquée par cathétérisme cardiaque droit entre janvier 2016 et décembre 2020 a été effectuée dans quatre grands centres au Canada. Les données hémodynamiques, les données cliniques et les résultats ont été recueillis. La corrélation de l'iPAP et d'autres va-riables hémodynamiques avec la mortalité a été évaluée par les courbes caractéristiques opérationnelles du receveur et des modèles à risques proportionnels de Cox. Résultats: Nous avons recensé 590 patients dont l'âge moyen était de 61,4 ± 15,5 ans; la proportion de femmes était de 66,3 %. Un faible iPAP (défini comme une valeur < 5,3) a été associé à une hausse de la mortalité à 1 an : 10,2 % contre 5,2 % (p= 0,02). Dans un modèle multivarié comprenant l'âge, le sexe, l'indice de masse corporelle et la classe fonctionnelle, un faible iPAP a été associé à la mortalité à 1 an (aire sous la courbe de 0,64 [intervalle de confiance à 95 %; de 0,55 à 0,74]). Cependant, une PAD élevée (> 8 mmHg) a aussi été un facteur prédictif de mortalité, l'aire sous la courbe étant de 0,65. Conclusions: L'iPAP a été associé à la mortalité dans une vaste cohorte de patients atteints d'une HTAP. Toutefois, la valeur discriminante de l'iPAP n'a pas été supérieure à celle de la PAD seule.
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BACKGROUND: Initial combination therapy with an endothelin receptor antagonist (ERA) and riociguat in pulmonary arterial hypertension (PAH) has limited supporting data. METHODS: We performed a prospective, single-arm, open-label trial of riociguat, and ambrisentan for incident PAH patients in functional class III. The primary endpoint was pulmonary vascular resistance (PVR) at 4-months. RESULTS: Twenty patients (59 ± 13 years old, 85% female) enrolled and 1 died before their 4-month follow-up. Fifteen patients completed a 4-month and 13 completed the 12-month follow-up. At 4-months PVR decreased 54% with an absolute change of -5.8 Wood units (95% CI -4.0; -7.5, p < 0.001). Other hemodynamic variables and risk scores also improved. Six patients discontinued riociguat and 8 discontinued ambrisentan, with 5 (25%) discontinuing both. CONCLUSIONS: These results do not support the routine use of riociguat plus ambrisentan in initial regimens. Future studies are needed to compare this strategy with phosphodiesterase-5 inhibitors and an ERA with respect to tolerability and long-term outcomes.
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Hipertensão Pulmonar , Hipertensão Arterial Pulmonar , Idoso , Anti-Hipertensivos/uso terapêutico , Antagonistas dos Receptores de Endotelina/uso terapêutico , Hipertensão Pulmonar Primária Familiar/tratamento farmacológico , Feminino , Humanos , Hipertensão Pulmonar/diagnóstico , Hipertensão Pulmonar/tratamento farmacológico , Masculino , Pessoa de Meia-Idade , Fenilpropionatos , Estudos Prospectivos , Pirazóis , Piridazinas , Pirimidinas , Resultado do TratamentoRESUMO
Atrial septal defects are one of the most frequently diagnosed congenital heart defects in adulthood. The presence of concurrent moderate or severe pulmonary arterial hypertension without Eisenmenger syndrome at the time of diagnosis can make for a challenging clinical scenario. There is continually evolving literature to determine the ideal approach to this subset of patients. Here we aim to review the clinical presentation, history, medical therapy, and closure options for atrial septal defects-pulmonary arterial hypertension with predominant left-to-right shunting, in the absence of Eisenmenger syndrome.
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Comunicação Interatrial , Hipertensão Arterial Pulmonar , Adulto , Comunicação Interatrial/complicações , Comunicação Interatrial/terapia , Humanos , Hipertensão Arterial Pulmonar/complicaçõesRESUMO
Chronic lung allograft dysfunction (CLAD) is the most common cause of mortality in lung transplant recipients after the 1st year of transplantation. CLAD has traditionally been classified into two distinct obstructive and restrictive forms: bronchiolitis obliterans syndrome and restrictive allograft syndrome. However, CLAD may manifest with a spectrum of imaging and pathologic findings and a combination of obstructive and restrictive physiologic abnormalities. Although the initial CT manifestations of CLAD may be nonspecific, the progression of findings at follow-up should signal the possibility of CLAD and may be present on imaging studies prior to the development of functional abnormalities of the lung allograft. This review encompasses the evolution of CT findings in CLAD, with emphasis on the underlying pathogenesis and pathologic condition, to enhance understanding of imaging findings. The purpose of this article is to familiarize the radiologist with the initial and follow-up CT findings of the obstructive, restrictive, and mixed forms of CLAD, for which early diagnosis and treatment may result in improved survival. Supplemental material is available for this article. © RSNA, 2021.
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BACKGROUND: Centralized care models are often used for rare diseases like pulmonary hypertension (PH). It is unknown how living in a rural or remote area influences outcomes. METHODS: We identified all patients from our PH database who carried a diagnosis of WHO Group 1 or WHO Group 4 PH. Using Canadian postal code data, patients were classified as living in a rural area; or a small, medium or large community size. The commute time from patient residence to our clinic was determined using mapping software. We compared baseline catheterization data according to community size and commute time. At follow up, we evaluated the association between community size and commute time with prognostic parameters of functional class, walk distance and echocardiography. RESULTS: Of the 342 patients identified, 72(21%) patients lived in rural areas, while 26(8%), 49(14%) and 195(57%) resided in small, medium and large population centres, respectively. The commute time was <1 h for 160(47%), 1-3 h for 62(18%), and >3 h for 120(35%). There was no association seen for any catheterization parameter by either community size or commute time. At last follow up, there was no association between any prognostic parameter and community size or commute time. CONCLUSIONS: We found no association between community size or commute time with severity of illness at diagnosis, or markers of prognosis at follow up. This suggests that patients who reside in rural or remote environments are not experiencing deficiencies in care compared to urban patients.
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Hipertensão Pulmonar , Canadá , Humanos , Hipertensão Pulmonar/diagnóstico por imagem , Hipertensão Pulmonar/epidemiologia , Qualidade da Assistência à Saúde , População Rural , CaminhadaRESUMO
BACKGROUND: Previous meta-analyses of treatments for pulmonary arterial hypertension (PAH) have not shown mortality benefit from any individual class of medication. METHODS: MEDLINE, EMBASE, and the Cochrane Central Register of Controlled Trials were searched from inception through November 2009 for randomized trials that evaluated any pharmacotherapy in the treatment of PAH. Reference lists from included articles and recent review articles were also searched. Analysis included randomized placebo controlled trials of at least eight weeks duration and studies comparing intravenous medication to an unblinded control group. RESULTS: 1541 unique studies were identified and twenty-four articles with 3758 patients were included in the meta-analysis. Studies were reviewed and data extracted regarding study characteristics and outcomes. Data was pooled for three classes of medication: prostanoids, endothelin-receptor antagonists (ERAs), and phosphodiesterase type 5 (PDE5) inhibitors. Pooled relative risks (RRs) and 95% confidence intervals (CIs) were calculated for mortality, 6-minute walk distance, dyspnea scores, hemodynamic parameters, and adverse effects. Mortality in the control arms was a combined 4.2% over the mean study length of 14.9 weeks. There was significant mortality benefit with prostanoid treatment (RR 0.49, CI 0.29 to 0.82), particularly comparing intravenous agents to control (RR 0.30, CI 0.14 to 0.63). Mortality benefit was not observed for ERAs (RR 0.58, CI 0.21 to 1.60) or PDE5 inhibitors (RR 0.30, CI 0.08 to 1.08). All three classes of medication improved other clinical and hemodynamic endpoints. Adverse effects that were increased in treatment arms include jaw pain, diarrhea, peripheral edema, headache, and nausea in prostanoids; and visual disturbance, dyspepsia, flushing, headache, and limb pain in PDE5 inhibitors. No adverse events were significantly associated with ERA treatment. CONCLUSIONS: Treatment of PAH with prostanoids reduces mortality and improves multiple other clinical and hemodynamic outcomes. ERAs and PDE5 inhibitors improve clinical and hemodynamic outcomes, but have no proven effect on mortality. The long-term effects of all PAH treatment requires further study.
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Anti-Hipertensivos/uso terapêutico , Antagonistas dos Receptores de Endotelina , Hipertensão Pulmonar/tratamento farmacológico , Hipertensão Pulmonar/mortalidade , Inibidores da Fosfodiesterase 5 , Prostaglandinas/uso terapêutico , Sistema de Registros , Humanos , Prevalência , Ensaios Clínicos Controlados Aleatórios como Assunto/estatística & dados numéricos , Análise de Sobrevida , Taxa de Sobrevida , Resultado do TratamentoRESUMO
BACKGROUND: Several studies have been published in the literature on the diagnostic accuracy of NT-pro-BNP for pleural effusions from heart failure in the last decade. The purpose of our study was to perform a systematic review and meta-analysis on the diagnostic accuracy of pleural fluid NT-pro-BNP for pleural effusions of cardiac origin. METHODS: MEDLINE, EMBASE, PapersFirst, and the Cochrane collaboration and the Cochrane Register of controlled trials were searched. All searches were inclusive as of March 2010. Studies were only included if the absolute number of true-positive, false-negative, true-negative, and false-positive observations were available, and the "reference standards" were described clearly. Two investigators independently reviewed articles and extracted data. Quality was assessed with the Quality Assessment for Diagnostic Accuracy Studies (QUADAS). The bivariate model for diagnostic meta-analysis was used to obtain a pooled sensitivity and a pooled specificity. RESULTS: Ten studies (total number of patients 1120) were included in the meta-analysis. The average pleural fluid NT-pro-BNP level in effusions of cardiac origin was 6140 pg/mL. The pooled sensitivity and specificity of all studies combined was 94% (95% CI: 90-97) and 94% (95% CI: 89-97) respectively. The pooled positive likelihood ratio was 15.2 (95% CI: 8.1-28.7) and the pooled negative likelihood ratio was 0.06 (95% CI: 0.03-0.11). The area under the ROC curve was 0.98 (95% CI: 0.96-0.99) and the diagnostic odds ratio was 246 (95% CI: 81-745). CONCLUSIONS: Pleural fluid NT-pro-BNP is a very useful biomarker with high diagnostic accuracy for distinguishing pleural effusions of cardiac origin.
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Insuficiência Cardíaca/complicações , Peptídeo Natriurético Encefálico/metabolismo , Fragmentos de Peptídeos/metabolismo , Derrame Pleural/etiologia , Derrame Pleural/metabolismo , Biomarcadores/metabolismo , Humanos , Cavidade Pleural/metabolismo , Derrame Pleural/diagnóstico , Sensibilidade e EspecificidadeRESUMO
BACKGROUND: The extent to which saline loading (SL) during cardiac catheterization influences clinical practice in pulmonary hypertension (PH) is unknown. We surveyed a national cohort of PH specialists to determine how SL affected diagnosis and management. METHODS: Relevant clinical and hemodynamic data pre-SL for patients with a baseline pulmonary arterial wedge pressure (PAWP) ≤15â¯mmâ¯Hg were presented as surveys to 7 PH specialists. The specialists were asked to classify patients according to the WHO classification scheme, rate their confidence, and state their treatment plans. Hemodynamic data following 500â¯mL of SL was then presented, and specialists answered the same questions. A positive fluid challenge (PFC) was defined as PAWP >18â¯mmâ¯Hg after SL. RESULTS: Seven specialists evaluated 48 cases, for a total of 336 surveys. SL influenced PH classification with 19.6% of cases reclassified as having a component of Group 2 PH. SL increased confidence in PH classification (mean difference 0.25; 95% CI 0.15-0.35). With a PFC, physicians were more likely to classify patients as PH due to left heart disease (OR 6.1; 95% CI 2.8-13.1), extend time to follow-up (OR 3.2; 95% CI 1.6-6.7), and discharge patients from PH clinic (OR 5.0; 95% CI 1.9-13.1). SL changed the treatment plan in 6.5% of cases, mostly with a PFC causing reconsideration in treatment initiation. CONCLUSION: The addition of SL to hemodynamic assessment of PH can impact physicians' classification and management decisions. However, decisions are not solely based on the SL results, but rather the entirety of the clinical data available.
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Cardiopatias , Hipertensão Pulmonar , Cateterismo Cardíaco , Hemodinâmica , Humanos , Hipertensão Pulmonar/diagnóstico , Hipertensão Pulmonar/epidemiologia , Hipertensão Pulmonar/terapia , Pressão Propulsora PulmonarRESUMO
BACKGROUND: Asthma is associated with enormous healthcare expenditures that include both direct and indirect costs. It is also associated with the loss of future potential earnings related to both morbidity and mortality. The objective of the study is to determine the burden of disease costs associated with asthma. METHODS: We performed a systematic search of MEDLINE, EMBASE, CINAHL, CDSR, OHE-HEED, and Web of Science Databases between 1966 and 2008. RESULTS: Sixty-eight studies met the inclusion criteria. Hospitalization and medications were found to be the most important cost driver of direct costs. Work and school loss accounted for the greatest percentage of indirect costs. The cost of asthma was correlated with comorbidities, age, and disease severity. CONCLUSION: Despite the availability of effective preventive therapy, costs associated with asthma are increasing. Strategies including education of patients and physicians, and regular follow-up are required to reduce the economic burden of asthma.
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Asma/economia , Asma/mortalidade , Gastos em Saúde/estatística & dados numéricos , Antiasmáticos/economia , Antiasmáticos/uso terapêutico , Asma/tratamento farmacológico , Comorbidade , Hospitalização/economia , Hospitalização/estatística & dados numéricos , HumanosRESUMO
Fluid challenge during right heart catheterization has been used for unmasking pulmonary hypertension (PH) related to left-sided heart disease. We evaluated the clinical and hemodynamic factors affecting the response to fluid challenge and investigated the role of fluid challenge in the classification and management of PH patients. We reviewed the charts of 67 patients who underwent fluid challenge with a baseline pulmonary arterial wedge pressure (PAWP) of ≤ 18 mmHg. A positive fluid challenge (PFC) was defined as an increase in PAWP to > 18 mmHg after 500 mL saline infusion. Clinical characteristics and echocardiographic and hemodynamic parameters were compared between PFC and negative fluid challenge (NFC). PFC was associated with female sex, increased BMI, and hypertension. A greater rise in PAWP was observed in PFC (6.8 ± 2.3 vs. 3.8 ± 2.7 mmHg, P = 0.001). A larger increase in PAWP correlated with a lower transpulmonary gradient (r = -0.42, P < 0.001), diastolic pulmonary gradient (r = -0.42, P < 0.001), and pulmonary vascular resistance (r = -0.38, P < 0.001). We found 100% of the patients with PFC were classified as WHO group 2 PH compared to 49% of the NFC patients ( P < 0.001). Fewer patients with PFC were started on advanced PH therapies and more were discharged from PH clinic. A PFC and the magnitude of PAWP increase after saline loading are associated with parameters related to left heart disease. In our population, fluid challenge appeared to influence the classification of PH and whether patients are started on therapy or discharged from clinic.
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BACKGROUND: Gaucher disease type 1 (GD1) is a lysosomal storage disease rarely resulting in end stage pulmonary hypertension (PH) and interstitial lung disease. There have only been two previous case reports of patients with GD1 receiving lung transplants. CASE PRESENTATION: We report a case of successful bilateral sequential lung transplantation in a patient with end-stage GD1-related PH. Prior to transplant, the patient was on enzyme replacement therapy with imiglucerase and pulmonary vasodilator therapy with bosentan, sildenafil and epoprostenol. The patient had pre-transplant comorbidities of prior splenectomy and osteopenia. She underwent bilateral sequential lung transplantation with basiliximab, methylprednisolone and mycophenolate mofetil induction. Her explanted lungs demonstrated severe pulmonary arterial hypertensive changes, but no Gaucher cells. She was maintained on MMF, tacrolimus, prednisone, imiglucerase and warfarin post-transplant. Her post-transplant course was complicated by hemorrhagic shock, prolonged support with extracorporeal membrane oxygenation, and acute renal failure requiring dialysis. Despite these complications, the patient was discharged and is doing well nine months post-transplantation. CONCLUSIONS: This is one of only three reported cases of lung transplantation in patients with GD1. Each case has involved previously splenectomised, female patients with GD1. This is the first to report transplantation in a patient with severe PH and no pulmonary parenchymal disease. As evidenced in our patient, long term treatment with imiglucerase may eliminate the Gaucher cells in the lungs. The PH in these patients is most consistent with pulmonary arterial hypertension, raising the question of whether this should be reclassified as WHO Group 1 PH.
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BACKGROUND: The accurate measurement of cardiac output (CO) is required in patients with pulmonary hypertension (PH).While both the thermodilution (TDCO) and indirect Fick (IFCO) methods are commonly used, there is little data comparing them in patients with PH. METHODS: We performed a retrospective analysis of patients evaluated at our center. All patients who had right heart catheterization (RHC) within 3â¯months of an echocardiogram, and CO assessment by both TDCO and IFCO methods were included. Bland-Altman analysis was used to assess agreement between the two methods. We further evaluated their agreement in each sex, and within tertiles of age, BMI and TR severity. We investigated the correlation between each method of CO and objective parameters of right ventricular function on echocardiography. RESULTS: In a cohort of 168 patients, the correlation between IFCO and TDCO was modest at (râ¯=â¯0.61). On average, values for CO were lower with IFCO than with TDCO, by 0.62â¯L/min (95% CI -0.82, -0.40). This difference was greater for females: 0.86â¯L/min (95% CI -1.08, -0.63) and in the highest tertile of BMI: 0.97â¯L/min (95% CI -1.4, -0.55). Moderate and severe TR did not in general result in lower TDCO values. Echocardiographic parameters of right ventricular function were correlated more strongly with TDCO than with IFCO. CONCLUSION: In PH patients, IFCO was substantially lower than TDCO on average, suggesting that these two techniques cannot be used interchangeably. TDCO correlated more strongly with echocardiographic measures of RV function, suggesting that it may be preferred over IFCO.
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Velocidade do Fluxo Sanguíneo/fisiologia , Cateterismo Cardíaco/métodos , Débito Cardíaco/fisiologia , Hipertensão Pulmonar/fisiopatologia , Hipertensão Pulmonar/terapia , Idoso , Cateterismo Cardíaco/tendências , Feminino , Humanos , Hipertensão Pulmonar/diagnóstico , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Termodiluição/métodos , Termodiluição/tendênciasRESUMO
BACKGROUND: Angiosarcoma involving the lung can represent either primary or metastatic malignancy. Due to the rarity of this condition, knowledge surrounding the natural history and clinical presentation is scarce. The aim of this scoping review is to summarize the existing literature on pulmonary angiosarcoma, particularly as it pertains to the clinical presentation and ancillary tests used for diagnosis in addition to histopathology. METHODS: We will conduct a systematic search using Ovid MEDLINE and EMBASE electronic databases. Two investigators will independently screen identified titles and abstracts to select articles reporting on pulmonary angiosarcoma. The data will be summarized in a narrative fashion and organized according to aspects of epidemiology, risk factors, clinical presentation, diagnostic methods, and treatment. DISCUSSION: Scoping reviews are increasingly used to synthesize the evidence on a particular topic, to identify gaps in the literature, and to determine if future systematic reviews are feasible. In order to improve the care of patients with angiosarcoma, earlier recognition and diagnosis is required. This review will be valuable for highlighting the range of clinical presentations and the role of imaging and other diagnostic tools in the diagnosis of metastatic and primary pulmonary angiosarcoma. SYSTEMATIC REVIEW REGISTRATION: PROSPERO registration: CRD42017059052.
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Hemangiossarcoma/patologia , Neoplasias Pulmonares/diagnóstico , Humanos , Pulmão , Neoplasias Pulmonares/patologia , Revisões Sistemáticas como AssuntoRESUMO
The determination of LV filling pressure is integral to the diagnosis of pulmonary arterial hypertension (PAH). The American Society of Echocardiography (ASE) has devised algorithms for their estimation. We aimed to test these algorithms in a population referred for suspected PAH. In our retrospective study, we evaluated the accuracy of the ASE Algorithms compared to right heart catheterization done within three months, in patients seen during 2006-2014. All echocardiograms were classified as showing normal, elevated or indeterminate filling pressures. Those with indeterminate pressures were excluded. We evaluated the diagnostic properties of this algorithm to predict a pulmonary artery wedge pressure (PAWP) and left ventricular end diastolic pressure (LVEDP) >15 mmHg. A total of 94 patients were included. The ASE algorithms yielded indeterminate results in 50 (53.2%) patients. This occurred more commonly in older patients and patients with cardiovascular comorbidities. The algorithm had a high sensitivity for predicting an elevated PAWP at 89.5% (95% confidence interval [CI] = 66.9-98.7) and an elevated LVEDP at 100% (95% CI = 76.8-100). The algorithm had a negative predictive value of 81.8% and 100% for predicting an elevated PAWP (95% CI = 52.4-94.8) and LVEDP, respectively, but a poor positive predictive value. The ASE algorithm for predicting LV filling pressures often cannot be applied in populations with suspected PAH. When they are interpretable, they have a high negative predictive value for elevated PAWP and LVEDP. We recommend caution when using these algorithms in populations with suspected PAH.
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BACKGROUND: In recent years, a significant number of costly oral therapies have become available for the treatment of pulmonary arterial hypertension (PAH). Funding decisions for these therapies requires weighing up their effectiveness and costs. OBJECTIVE: The aim of this study was to assess the cost effectiveness of monotherapy with oral PAH-specific therapies versus supportive care as initial therapy for patients with functional class (FC) II and III PAH in Canada. METHODS: A cost-utility analysis, from the perspective of a healthcare system and based on a Markov model, was designed to estimate the costs and quality-adjusted life-years (QALYs) associated with bosentan, ambrisentan, riociguat, tadalafil, sildenafil and supportive care for PAH in treatment-naïve patients. Separate analyses were conducted for cohorts of patients commencing therapy at FC II and III PAH. Transition probabilities, based on the relative risk of improving and worsening in FC with treatment versus placebo, were derived from a recent network meta-analysis. Utility values and costs were obtained from published data and clinical expert opinion. Extensive sensitivity analyses were conducted. RESULTS: Analysis suggests that sildenafil is the most cost-effective therapy for PAH in patients with FC II or III. Sildenafil was both the least costly and most effective therapy, thereby dominating all other treatments. Tadalafil was also less costly and more effective than supportive care in FC II and III; however, sildenafil was dominant over tadalafil. Even given the uncertainty within the clinical inputs, the probabilistic sensitivity analysis showed that apart from sildenafil and tadalafil, the other PAH therapies had negligible probability of being the most cost effective. CONCLUSION: The results show that initiation of therapy with sildenafil is likely the most cost-effective strategy in PAH patients with either FC II or III disease.
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Hipertensão Pulmonar/tratamento farmacológico , Administração Oral , Análise Custo-Benefício , Humanos , Cadeias de Markov , Anos de Vida Ajustados por Qualidade de Vida , Citrato de Sildenafila/economia , Citrato de Sildenafila/uso terapêutico , Tadalafila/economia , Tadalafila/uso terapêuticoRESUMO
Although left atrial function has been extensively studied in patients with heart failure, the determinants and clinical correlates of impaired right atrial (RA) function have been poorly studied. We investigated measures of RA function in pulmonary arterial hypertension (PAH). We identified all treatment-naive patients with World Health Organization category 1 PAH seen at our center during 2000-2011 who had right heart catheterization and 6-minute walk test (6MWT) within 1 month of initial echocardiographic examination. Atrial size was measured using the monoplane area-length method, and atrial function was quantified using total, passive, and active RA emptying fractions (RAEFs). We compared measures of RAEF with known prognostic clinical, echocardiographic, and hemodynamic parameters. For the subset of patients with follow-up echocardiographic examination/6MWT within 6-18 months, we investigated the change in RAEF. In an exploratory analysis, we investigated the association between RAEF and mortality. Our population consisted of 39 patients with treatment-naive (incident) PAH, 30 of whom had follow-up testing. The mean total, passive, and active RAEFs were 24.4% ± 15.1%, 8.5% ± 6.9%, and 17.6% ± 13.9%, respectively. Total and active RAEFs correlated with tricuspid annular plane systolic excursion (P = 0.004 and P = 0.005) and cardiac output (P = 0.02 and P = 0.01). The change in active RAEF correlated with change in 6-minute walk distance (P = 0.02). In our Cox regression analysis, low active and total RAEF were associated with mortality, with hazard ratios of 5.6 (95% confidence interval [CI], 1.2-26.2; P = 0.03) and 4.2 (95% CI, 1.1-15.5; P = 0.03), respectively. Passive RAEF was poorly reproducible and not associated with outcome. Measures of RAEF appear to have prognostic importance in PAH and warrant further study.