The complement cascade as a target against SARS-CoV-2-induced pneumonia
Published on: 14th February, 2023
Fox, et al. [1] reported on the relevant cardiopulmonary findings in a series of autopsies of patients deceased from SARS-CoV-2 infection. In particular, regarding the histologic examination of the lungs, they observed bilateral diffuse alveolar damage with a lymphocytic infiltrate, thickened alveolar capillaries, fibrin thrombi within the capillaries and small vessels, and entrapment of neutrophils, without any significant neutrophilic, infiltrate within airways or the interstitium.
Coronary artery anomalies, myocardial bridging associated with fistula to pulmonary artery trunk. Case reports
Published on: 31st March, 2023
Between coronary artery anomalies, myocardial bridging means an epicardial coronary artery, mostly left anterior descending artery (LAD), running through an intramyocardial “tunnel” (usually in the middle segment), leading during systolic contraction, flow reduction, through the vessel. When this anomaly is associated with a coronary fistula, which “steals” more from the bloodstream, the symptoms are more pronounced, and the management complex is surgical in particular. Despite the presence from birth remains asymptomatic and it becomes clinically manifest later in the third to fourth decade of life, with a diverse palette of symptoms; angina, arrhythmias, and acute myocardial infarction up to sudden death. Diagnosis and particular management, medical, interventional, and surgical should avoid major cardiac complications and sudden death. We present two adult patients, with coronary artery bridging, one case associated with coronary artery fistula, LAD to pulmonary artery trunk, very symptomatic with surgical management, and the second only myocardial bridging controlled with medication and supervision.
Haemodynamic, Biochemical and Respiratory Implications of total Bronchoalveolar Lavage in Pulmonary Alveolar Proteinosis
Published on: 17th June, 2023
Introduction: Total bronchoalveolar lavage (BAL) continues to be the treatment of choice for alveolar proteinosis (AP), facilitating the removal of lipoprotein material. The purpose of this article is to evaluate the impact of haemodynamic, biochemical, and respiratory parameters, as well as the complications and evolution of patients undergoing this procedure. Methods: Retrospective, observational, and descriptive study of BAL. The technique was performed in the Intensive Care Unit. Blood gases, blood pressure, central venous pressure, body temperature, and fluid balance were analyzed. Results: Including eight patients, thirty-eight BAL were performed from 2008 to 2021. The mean instillation of saline at each session was 13.464 ± 4.002 ml per lung. No significant changes were observed before and after BAL in heart rate and blood pressure. Mean central venous pressure increased by 2.59 cm H20. The pO2 initial was 126 mmHg with a final mean of 69.7 mmHg, with statistical significance. The pCO2, HCO3 and pH parameters remained stable. Complications were observed during fifteen of the thirty-eight BAL (nine with arterial hypotension, three with glottic oedema, one acute pulmonary oedema, one pneumothorax, and one cardiorespiratory arrest). In terms of evolution, one case had a clinical-radiological resolution, one case of exits, one required lung transplantation, and the remaining five remained stable. Conclusion: This study demonstrates that the procedure, is well tolerated haemodynamically and that the biochemical alterations to which the patient is subjected are not compromised. With few complications and good results in delaying the progression of AP.
Lung Abnormalities in Liver Cirrhosis
Published on: 12th July, 2023
This article aims to provide what lung disorders can be caused by liver cirrhosis and also explain the pathophysiology of each etiologies. Regardless of preexisting lung illness, patients suffering from liver cirrhosis, especially decompensated liver cirrhosis can develop distinct pulmonary complications. Liver cirrhosis patients should be assessed for hepatopulmonary syndrome (HPS), portopulmonary hypertension (PoPH), hepatic hydrothorax (HH), and spontaneous bacterial empyema (SBEM) which are the most clinically significant pulmonary consequences, in particular when dyspnea develops in conjunction with hepatic cirrhosis. These entities differ in terms of pathophysiology, clinical characteristics, diagnosis, and suitable treatment options. This emphasizes the need for a specific diagnostic algorithm in liver cirrhosis patients presenting with dyspnea or other pulmonary symptoms. These pulmonary complications might be rare in patients with liver cirrhosis and portal hypertension but these complications might carry significant morbidity and mortality risks and, therefore, strong clinical suspicion is required to make an early accurate diagnosis. There are several medical therapies available for each condition in multiple studies but most of the treatments and procedures don’t have a significant benefit or have short-lived benefits. The only treatment that changes the clinical prognosis of decompensated cirrhosis effectively in the long term is liver transplantation. However, liver transplantation also needs careful consideration as in some cases it might increase the risk of morbidity and mortality.
Sudden Cardiac Death in a Neonate Due to Bilateral Absence of Coronary Artery Ostium
Published on: 24th July, 2023
Introduction: Congenital heart disease is a leading cause of neonatal mortality linked to birth defects. Despite the widespread availability of prenatal screenings, detection rates remain low. Accurate early detection of these lesions is pivotal to reducing neonatal morbidity and mortality.Methods: In this case, we present a neonate who experienced sudden cardiac death due to a rare, undiagnosed congenital cardiac anomaly - the bilateral absence of coronary artery ostium. Discussion: This case highlights the importance of prenatal detection of congenital cardiac anomalies. While fetal echocardiography is frequently utilized, it only identifies CHD in 36-50% of cases. This is attributed to inadequate imaging procedures, varied operator skills, and regional discrepancies. Early detection of severe CHD is essential for specialized treatment, thereby mitigating neonatal health risks and improving survival rates.Conclusion: Prenatal detection of CHD, especially coronary anomalies, continues to pose significant challenges. There is a pressing need to establish and enforce standardized protocols for fetal echocardiography aimed at these anomalies. To enhance care and improve outcomes, a joint effort between academic institutions and community centers is encouraged.Learning Objectives: • Congenital coronary artery anomalies are a significant cause of sudden cardiac death in children.• The absence of a coronary artery ostium is known to be associated with other congenital heart diseases, particularly pulmonary atresia with an intact ventricular septum. However, isolated coronary disease has also been reported in this case.• Prenatal echocardiography is a valuable tool for diagnosing congenital heart disease. However, certain limitations may be encountered when diagnosing coronary artery anomalies.
latrogenic Pneumothorax Caused by Thoracic Puncture Secondary to Anti-aging Medicine
Published on: 27th July, 2023
Introduction: Pneumothorax is defined as air in the pleural cavity, the iatrogenic cause happens after invasive procedures as thoracic needle aspiration, central line placement, pacemaker placement, etc. Nevertheless, it is possible to be found in non-medical procedures, such as alternative medicine, more specifically acupuncture. There are medications used against aging that FDA has warned to be used only in its first objective, an example is procaine hydrochloride.Case: A 63 y/o female, Mexico City resident, with anxiety, came to a follow-up appointment for an aging cohort, where CT, PFT, and laboratories are made for pulmonary healthy subjects to investigate normal lung aging and how other environmental factors can affect the lungs; at physical examination, we did not find any pleuropulmonary syndrome, but in the CT a left Iatrogenic pneumothorax was found; we re-asked for antecedents and she described that 15 days before the date she had 2 punctures in the middle clavicular line to apply procaine hydrochloride as an anti-aging treatment.Discussion: There is diverse evidence of adverse effects associated with acupuncture, and even if pneumothorax has a small percentage of incidence, in some cases it can be severe and can even cause fatalities.Conclusion: The pneumothorax in this case was iatrogenic due to the thoracic puncture for an anti-aging treatment, with a total resolution with conservative treatment, however we as health professionals have the responsibility to inform our patients that initiatives of administration ways can cause important complications.
The Predictive Value of Diaphragm Thickness Fraction on Postoperative Pulmonary Complications after Digestive Cancer Curative Surgery
Published on: 14th August, 2023
Background: Postoperative Pulmonary Complications (PPCs) escalate mortality, hospitalization, and costs. This study aimed to predict PPCs after curative digestive cancer surgery using thickness fraction (TFdi) determined by ultrasonography. Methods: A prospective study was conducted over a period of 9 months. Diaphragmatic ultrasound was performed pre-surgery and repeated postoperatively (within 24 hours of ICU admission, then day 3). Right and left hemidiaphragm thickness at end-expiration (TEE) and peak-inspiration (TPI) were measured using ultrasonography. The maximal diaphragm thickening fraction during inspiration (TFdi,max) was calculated: TFdi,max = (TPI–TEE)/TEE. Patients were classified into No-PPCs and PPCs groups. Results: 159 patients participated, 55 (34.6%) developed PPCs. ICU stay was longer in PPCs patients with more deaths. TFdi,max decreased postoperatively and remained lower in PPCs patients [44.83% ± 11.07 vs. 31.54% ± 8.45; p < 0.001]. The receiver operating characteristic curve yielded an area under the curve of 0.83 [95% IC: 0.754 – 0.887]. TFdi,max < 37% had 72.7% sensitivity (95% IC: 59.0% – 83.8%) and 80.8% specificity (95% IC: 71.8% – 87.8%), Positive and negative Likelihood Ratios were 3.7 (95% IC: 2.4 – 5.7) and 0.3 (95% IC:0.2 – 0.5), respectively. In multiple logistic regression, preoperative risk factors for PPCs included TFdi,max < 37% [OR: 7.10; 95% CI: 1.71 – 18.60; p < 0.001] and supramesocolic surgery [OR: 9.94; 95% CI: 3.62 – 27.29; p < 0.001]. Epidural administration was protective [OR: 0.21; 95% CI: 0.052 – 0.87; p = 0.031]. Conclusion: A low preoperative TFdi,max identifies high-risk PPCs patients after digestive cancer surgery, aiding targeted preventive strategies like inspiratory muscle preoperative training.
Cardiac Tamponade as the Cause of Pulmonary Edema: Case Report
Published on: 17th August, 2023
Introduction: Cardiac tamponade is an emergency syndrome that requires fast diagnosis and treatment; otherwise patient follows obstructive shock and cardiac arrest. Case report: A 70-year-old female was brought to the emergency department with hypoxemia. She had a history of progressive dyspnea over the past three weeks. Past medical history includes smoking. On physical examination: tachypnea, hypoxemia (SaO2 89%), jugular venous distention, arterial pressure 220/100 mmHg, heart rate rhythmic of 82 bpm. On pulmonary auscultation: diffuse and bilateral crackles. Lung ultrasound showed a bilateral B line and the echocardiogram demonstrated a pericardial effusion with signs of tamponade. A pericardiocentesis evacuated 620 ml of hemorrhagic fluid and the patient was transferred to the intensive care unit, hemodynamically stable, with SaO2 95%. At the ICU the echocardiogram, showed resolution of the cardiac tamponade and a tumor adhered to the lateral wall of the left ventricle. Chest CT demonstrated: a left lung tumor, infiltrating the pericardial sac. A pericardium biopsy demonstrated undifferentiated carcinoma. Discussion: Cardiac tamponade diagnosis requires a high level of suspicion. Respiratory failure, chest pain, and shock, observed in cardiac tamponade, are also present in different diseases. The most common finding of cardiac tamponade is dyspnea (78% of cases). Our patient had dyspnea due to pulmonary edema, secondary to left ventricle diastolic dysfunction caused by the tamponade. A bedside echocardiogram made the diagnosis of cardiac tamponade and guided the effective pericardiocentesis. Conclusion: Cardiac tamponade must be suspected in all cases of acute dyspnea. Echocardiogram is the method of choice for the diagnosis and for guiding the pericardiocentesis.
Hazardous Effects of Malaria for Patients Residing in Low-probability Malaria Areas: A Case Report and Critiques of the Literature for Cerebral Malaria
Published on: 13th September, 2023
Despite decades of global and country commitments towards eradicating malaria, malaria remains the most hazardous parasitic disease and the most common cause of fever for humans, especially in tropical countries. Plasmodium falciparum causes 90% of malaria cases. Coma [Cerebral Malaria (CM)], acidosis, hypoglycemia, severe anemia, renal dysfunction, and pulmonary edema are the most common complications of malaria caused by Plasmodium falciparum and the most common cause of death related to malaria. People from less prevalent malaria areas are at high risk of developing these complications. A 16-year-old male from a low malaria transmission area was diagnosed with CM. Prior to developing CM, he was treated with Coartem. CM is a medical emergency and one of the forms of severe malaria. CM has high mortality and morbidity rates. Yet, international health-related agencies, funders, and policy-makers are unfamiliar with it. The continuous occurrence of CM validates the considerable need for global investment in malaria control and elimination programs. Early administration of Artesunate to all patients suspected of having severe malaria would reduce global malaria-related mortality and morbidity. Simple tests, such as the determination of malaria parasitic density either with thin or thick blood smears, may influence the proper management of all severe malaria cases. However, in clinical practice, the determination of malaria parasitic density is not routinely done. Further commitments are needed to ensure routine determination of malaria parasitic density for all suspected severe malaria cases. Moreover, further commitments are needed to guarantee the proper management of CM because it is a major cause of reversible encephalopathy in tropical countries.
Fetal Ductal Constriction due to Maternal Intake of Metamizole
Published on: 4th October, 2023
The prevalence of intra-uterine dysfunction of ductus arteriosus is unknown and the clinical consequences are poorly understood. We report a case of prenatal diagnosis of premature closure of the ductus arteriosus due to maternal intake of metamizole during pregnancy. Fetal echocardiography at 37 weeks of gestation revealed a right ventricular hypertrophy and suspected stenosis of the tricuspid valve. A cesarean section led to an excellent neonatal outcome. The aim of this report is to show echocardiographic abnormalities and outcomes of this rare phenomenom.
Stable COPD Picture through Exhaled Breath Condensate, Questionnaires and Tests. A Proof of Concept Study
Published on: 5th December, 2023
Background: Changes in lung structures persist in stable Chronic Obstructive Pulmonary Disease (COPD), but their correlation with the clinical picture remains unclear. The purpose of this study was to assess the stable COPD picture via the relationship between exhaled breath condensate (EBC) particle concentration and the Saint George Respiratory Questionnaire (SGRQ), COPD Assessment Test (CAT), and six-minute walking test (6 MWT). Methods: 12 stable COPD and 12 healthy subjects participated in the study. The EBC was collected with Rtube and analyzed using the Accusizer FxNano. Particle concentration was measured and correlated with the findings of the tools used to assess the health status and functional profile of COPD. The results’ analysis was performed with the Spearman’s test and the Mann-Whitney U - test.Results: The COPD group presented a worse picture of health status and functional profile compared to the healthy group. Correlations were observed between components of the SGRQ and CAT. The two groups presented similar levels of EBC particle concentrations, but the number of small particles was higher in COPD subjects. A correlation of the EBC particle concentration with the activity and total score of the SGRQ was only observed in the healthy group. Conclusion: The total particle number was similar in the COPD and healthy groups. A few correlations between the EBC particles and tools used were also observed. The use of EBC particle concentration to monitor COPD status cannot be claimed with confidence because of the small sample size. Further research is necessary, particularly in large-scale groups.
A Perplexing Case of Genito-urinary Tuberculosis in an 83-Year-Old Woman with Increased Levels of Tumor Markers: A Case Report
Published on: 5th December, 2023
Background: Tuberculosis (TB) is a significant global health problem, and extrapulmonary TB can present with no specific clinical or radiographic findings. Genito-urinary TB is often associated with elevated tumor markers and can be misdiagnosed as ovarian/fallopian tube carcinomas, especially in elderly female patients, as genitourinary TB commonly affects women of reproductive age.Objective: We present a rare case of genito-urinary TB in an elderly female patient who was initially misdiagnosed with ovarian cancer with metastasis.Case presentation: An 83-year-old woman with a medical history of diabetes and hypertension presented with complaints of abdominal distension. Diagnostic imaging revealed lesions in the ovaries and omentum and tumor markers were elevated, leading to a suspicion of ovarian cancer with metastases to the omentum. The patient underwent a diagnostic laparotomy and surgical removal of ovaries, fallopian tubes, and the lesion of the greater omentum. However, no malignancy was found during the morphological evaluation. Further histopathological examination confirmed the final diagnosis of genito-urinary tuberculosis, and the patient received anti-TB drugs. The postoperative period was uneventful, and tumor marker levels decreased.Conclusion: As the clinical presentation of genito-urinary TB can mimic ovarian cancer, a histopathological examination should be performed for differential diagnosis, thereby reducing the possibility of inaccurate treatment. This case report highlights the importance of considering genito-urinary TB as a differential diagnosis in elderly female patients presenting with elevated tumor markers, abdominal distension, and suspected genital malignancy. It is crucial to carefully evaluate these cases and explore the possibility of genital TB as an alternative diagnosis, given the overlapping clinical presentation. This highlights the importance of a comprehensive diagnostic approach that includes considering TB in addition to malignancy, even in the context of elderly female patients.
Update on the Clinical Applications of Mesenchymal Stem Cells
Published on: 21st December, 2023
Mesenchymal stem cells are heterogenous adult multipotent stromal cells that can be isolated from various sources including bone marrow, peripheral blood, umbilical cord blood, dental pulp, and adipose tissue. They have certain regenerative, anti-inflammatory, immunomodulatory, immunosuppressive, antimicrobial, and other properties that enable them to have several therapeutic and clinical applications including treatment of various autoimmune disorders; role in hematopoietic stem cell transplantation and regenerative medicine; treatment of skin, pulmonary and cardiovascular disorders; treatment of neurological and eye diseases; as well as treatment of various infections and their complications. Different factors including donor age, biological source, route of administration, and signaling pathways have an impact on the functions and consequently the clinical applications of mesenchymal stromal cells. The products of mesenchymal stem cells such as extracellular vesicles and exosomes reproduce the biological effects and most of the therapeutic actions of the parent stem cells. Genetic engineering and the use of specific mesenchymal stromal cell products have improved their clinical efficacy and decreased their adverse effects. However, despite the recent progress in the use of mesenchymal stem cells, the clinical application of these cells in the treatment of several diseases still faces real challenges that need to be resolved. The current status of mesenchymal stem cells and the controversies related to their clinical utilization in various disease conditions will be thoroughly discussed in this review.
Delayed Diagnosis of Early-onset Sarcoidosis: A Case Report and Literature Review
Published on: 18th January, 2024
Background: Early-onset sarcoidosis is a rare systemic inflammatory granulomatous disease, distinguished by onset before the age of 4 and notably lacking pulmonary involvement. Unfortunately, the condition often shows clinical features similar to juvenile idiopathic arthritis, resulting in the misdiagnosis of numerous patients. This case report delves into the challenges associated with the delayed diagnosis of early-onset sarcoidosis, with a particular focus on the diagnostic methods employed to address this delayed recognition.Case presentation: A 15-year-old girl presented with a history of recurrent fever since infancy, accompanied by rash, arthritis, and joint deformity. Previously misdiagnosed with juvenile idiopathic arthritis, she underwent management with steroids and methotrexate, yielding no improvement. The diagnosis of early-onset sarcoidosis was made during adolescence based on serial examinations, comprehensive laboratory and radiological evaluations, and subsequent histopathology findings. Presently, the patient is receiving treatment with low-dose steroids and biologic therapy (Tocilizumab) and experiencing no disease progression.Conclusion: This case report underscores the importance of considering early-onset sarcoidosis in the differential diagnosis of pediatric patients exhibiting persistent arthritis from an early age. Early detection and treatment are crucial in averting complications and enhancing the overall quality of life.
Effect of Azithromycin on Lung Function and Pulmonary Exacerbations in Children with Post-infectious Bronchiolitis Obliterans. A Double-blind, Placebo-controlled Trial
Published on: 28th February, 2024
Introduction: Acute lower respiratory infection (ALRI) of viral etiology is a frequent consultation in pediatrics. Post-infectious bronchiolitis obliterans (PIBO) is a rare and potentially severe disorder following ALRI, characterized by partial or complete obstruction of the small airways by inflammatory tissue. There is evidence that macrolides reduce morbidity and mortality in diffuse panbronchiolitis, which may have similar inflammatory and obstructive components.We hypothesized that the effect of azithromycin (AZ) may improve lung function and reduce pulmonary exacerbations in PIBO.MethodsStudy design: A double-blind, randomized, placebo-controlled trial.Patients: We enrolled patients with PIBO followed-up at the Pulmonology department between 5 years to 18 years.Treatment regimen: The patients were randomized to receive active drug or placebo three times a week. Clinical evaluation: Clinical evaluation, pulse oximetry, lung function, and 6-min walk test were performed before and after study initiation and at 1, 3, and 6 months.CT scan and a quality of life questionnaire were performed at the beginning and the end of the Study.Results: 29 patients, 15 in G1 (10 males) and 14 in G2 (7 males) were included.There were no significant differences in FVC, FEV1, TLC, RV, or sGaw between the treatment group and controls. In addition, no significant differences were observed in exacerbations, quality of life questionnaire, or HRCT scan scores.Conclusion: No differences were observed between the groups. Further studies are necessary to allow us to find a better treatment, as azithromycin does not seem to be efficacious.
Why is Pain not Characteristic of Inflammation of the Lung Tissue?
Published on: 7th March, 2024
The article presents materials that make it possible to understand the reason for the absence of one of the classic signs of inflammatory processes in patients with acute pneumonia. The peculiarities of the functional significance of the lungs for the body are the reason that in the case of inflammation in the tissues of the organ, nature has provided for the presence of a more important adaptive mechanism instead of pain as a signal sign. Understanding the causes of the absence of pain in pneumonia in the initial period, which is most responsible for timely and effective care for these patients, allows us to look at the pathogenesis of the disease from a new point of view, which is of fundamental importance for the correction and selection of pathogenetic means of care.
Effect of TAK242 on MCP-1 and TGF-β in COPD Rats
Published on: 13th March, 2024
Objective: To investigate the mechanism of MCP-1 and TGF-β regulation by TAK242 in COPD rats. Methods: Thirty-six SD rats were randomly divided into normal, COPD control, and TAK242 groups. The normal group was freely fed, and the other groups used the method of fumigation plus lipopolysaccharide tracheal drip to establish an experimental animal model of COPD. After successful modeling, each experimental group received 0.9% NaCl solution and corresponding drugs by intraperitoneal injection for 7 d. After drug administration, lung function was examined; pathological changes in lung tissue were observed by light microscopy with hematoxylin-eosin staining; mRNA expression of MCP-1 and TGF-β was detected by q-PCR; and protein expression of MCP-1 and TGF-β in lung tissue was detected by Western blot and IHC, TGF-β protein expression in rat lung tissue. Results: Compared with the normal group, rats in the COPD control group showed signs and symptoms of COPD, decreased lung function, and increased expression of MCP-1 and TGF-β. The TAK242 group showed decreased expression of MCP-1 and TGF-β compared to the COPD control group. Conclusion: MCP-1, and TGF-β played a crucial role in the early stage of COPD fibrosis. TAK242 could ameliorate airway inflammation and inhibit the progression of COPD lung fibrosis in pre-existing rats in COPD model rats.
Longitudinal Risk Phenotyping in Incident Systemic Sclerosis-associated Pulmonary Arterial Hypertension (SSc-PAH): An Unsupervised Cluster Analysis of the PHAROS Registry
Published on: 15th March, 2024
Background: Systemic sclerosis-associated pulmonary arterial hypertension (SSc-PAH) is characterized by pulmonary arterial remodeling resulting in right ventricular failure and death if untreated. Despite therapeutic advances, there is survival variability within the SSc-PAH population. The aim of this study was to delineate high-risk subgroups of SSc-PAH using readily available clinical parameters.Methods: We analyzed data from the PHAROS database, a prospective observational registry of incident SSc-PAH patients. Latent class modeling was performed based on trends in 6MWD over time. We compared survival between the clusters regarding baseline clinical parameters and changes in these parameters over time.Results: We identified four unique groups within 103 patients meeting our inclusion criteria, based on trajectories of 6MWD. Patients in Cluster 4 exhibited a decline in 6MWD over time and had the worst prognosis with a median survival of 3 years. Patients in Cluster 3, with the lowest baseline 6MWD, were associated with lower median survival (5 years) when compared to Clusters 1 and 2 (> 9 and 7 years, respectively), despite an improvement in 6MWD over time. There were no meaningful changes in SF-36 and WHO functional class between the clusters, but BNP trended higher over time in the higher-risk clusters. Conclusion: We identified high-risk subsets of SSc-PAH characterized by significantly worse survival. Incident SSc-PAH patients with a decline in 6MWD over time or low baseline 6MWD had worse survival when compared to SSc-PAH patients who demonstrated relatively stable or mild reduction in 6MWD over time.
Macitentan in Adults with Sickle Cell Disease and Pulmonary Hypertension: A Proof-of-Concept Study
Published on: 22nd April, 2024
Pulmonary hypertension (PH) in sickle cell disease (SCD) is associated with a mortality rate of 37%. There is an upregulation of adhesion molecules which leads to the expression of endothelin-1, a potent vasoconstrictor. A prospective, descriptive study was done to determine the safety and efficacy of macitentan in patients with SCD and PH. Continuous variables were reported as mean ± SEM or percentage where appropriate. We screened 13 patients and recruited five. All five patients were adults. Data were analyzed as appropriate by student t - test. Statistical significance was assumed at p < 0.05. Baseline pulmonary hemodynamics obtained by right heart catheterization and systemic hemodynamics were (± SEM): mean pulmonary artery pressure (MPAP) 32 ± 8 mmHg, right atrial pressure (RAP) 9 ± 4 mmHg, pulmonary vascular resistance (PVR) 257 dynes-sec/cm5 and CI 3·7 ± 0.39 l/m2. Of all parameters, only PVR and 6-min walk distance changed significantly. For the group, MPAP decreased by 15.6%, PVR by 22.5% and RAP by 25.5%. The 6-minute walk distance increased over sixteen weeks except in Patient 4 who had a 3% decrease. The mean walk distance increased in the total distance, from 464 ± 158 meters to 477 ± 190 meters (p .123). In four patients, the adverse events were mild to moderate and did not lead to study drug discontinuation. Significant improvement in pulmonary hemodynamics and exercise capacity in patients with SCD-related pulmonary arterial hypertension. We found that macitentan was safe and well tolerated.
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