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OBJECTIVE: Continuous positive airway pressure (CPAP) is the standard treatment for obstructive sleep apnea (OSA). Unsatisfactory adherence to CPAP is an important clinical issue to resolve. Cluster analysis is a powerful tool to distinguish subgroups in a multidimensional fashion. This study aimed to investigate the use of cluster analysis for predicting CPAP adherence using clinical polysomnographic (PSG) parameters and patient characteristics. PATIENTS/METHODS: Participants of this multicenter observational study were 1133 patients with OSA who were newly diagnosed and implemented CPAP. Ward's method of cluster analysis was applied to in-laboratory diagnostic PSG parameters and patient characteristics. CPAP adherence was assessed during 90- and 365-day periods after CPAP initiation in each cluster. We adopted the Centers for Medicare and Medicaid Services criterion for CPAP adherence, i.e., CPAP use ≥4 h per night for 70 % or more of the observation period. Logistic regression analysis was performed to stratify clusters according to CPAP adherence. RESULTS: Five clusters were identified through cluster analysis. Clustering was significantly associated with CPAP adherence at 90- and 365-day periods after CPAP initiation. Logistic regression revealed that the cluster with features including apnea predominant sleep-disordered breathing, high apnea-hypopnea index, and relatively older age demonstrated the highest CPAP adherence. CONCLUSION: Cluster analysis revealed hidden connections using patient characteristics and PSG parameters to successfully identify patients more likely to adhere to CPAP for 90 days and up to 365 days. When prescribing CPAP, it is possible to identify patients with OSA who are more likely to be non-adherent.
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Obstructive sleep apnea (OSA) causes excessive daytime sleepiness, impaired daytime functioning, and an increased risk of cardiovascular diseases. Continuous positive airway pressure (CPAP) is a highly effective therapy for moderate to severe OSA. Although CPAP adherence is commonly assessed using a 4-hthreshold, determining the optimal usage time based on clinical outcomes is crucial. While subjective sleepiness often improves with ≥4 h of CPAP usage, an extended duration (≥6 h) may be necessary to impact objective sleepiness. CPAP demonstrated a modest yet clinically meaningful dose-dependent effect on lowering blood pressure. For patients seeking antihypertensive benefits from CPAP therapy, the goal should extend beyond 4 h of use to maximize the therapeutic impact. Recognizing individual variations in sleep duration and responses to CPAP therapy is essential. The adoption of 'individualized goals for CPAP use,' outlining target times for specific outcomes, should also consider an individual's total sleep duration, including periods without CPAP. The impact of CPAP on clinical outcomes may vary, even with the same duration of CPAP use, depending on the period without CPAP use, particularly during the first or second half of sleep. Patients who remove or initiate CPAP midway or have a low CPAP usage frequency may require different forms of guidance. Tailoring patient education to address CPAP usage patterns may be necessary to enhanced satisfaction, self-efficacy, and adherence to therapy. Management of CPAP treatment should be personalized to meet individual needs and adapted based on specific response patterns for achieving treatment efficacy.
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Pressão Positiva Contínua nas Vias Aéreas , Cooperação do Paciente , Apneia Obstrutiva do Sono , Apneia Obstrutiva do Sono/terapia , Humanos , Fatores de Tempo , Educação de Pacientes como Assunto , Sono/fisiologia , Resultado do Tratamento , Pressão Sanguínea , Índice de Gravidade de DoençaRESUMO
There have been several reports of drug-induced lung injury caused by molecular-targeted agents. Additionally, medical history of interstitial lung disease and chest irradiation are established risk factors for the development and progression of drug-induced lung injury. Moreover, the presence of fibrosis on chest computed tomography before treatment is a predictive factor for the appearance of pneumonia induced by anticancer drugs. Accordingly, patients with a history of interstitial lung disease or pneumonitis were excluded from clinical trials of dabrafenib and trametinib combination therapy for patients with previously treated BRAF V600E-mutant metastatic non-small-cell lung cancer. This article presents a case of successful dabrafenib and trametinib combination therapy in a patient with BRAF V600E-mutant non-small-cell lung cancer who had a history of radiation pneumonitis and developed recurrence after conventional chemoradiotherapy.
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Background: Oxidative stress is an important mechanism for the development and progression of chronic obstructive pulmonary disease (COPD). It may also contribute to systemic manifestation in patients with COPD. Reactive oxygen species (ROS) including free radicals play a crucial role in oxidative stress in COPD. The aims of this study were to determine serum scavenging capacity profile against multiple free radicals and to evaluate its correlation with pathophysiology, exacerbations, and prognosis in patients with COPD. Methods: Serum scavenging capacity profile against multiple free radicals comprising hydroxyl radical (â¢OH), superoxide radical (O2 -â¢), alkoxy radical (ROâ¢), methyl radical (â¢CH3), alkylperoxyl radical (ROOâ¢), and singlet oxygen (1O2) was assessed using the multiple free-radical scavenging method in 37 patients with COPD (mean age, 71 years; mean forced expiratory volume in 1 s, 55.2% predicted). The severity of emphysema was evaluated by Goddard classification on chest computed tomography. Exacerbations were recorded prospectively for 1 year and the overall mortality was assessed 5 years after the initial assessment. Results: â¢OH scavenging capacity was significantly decreased (p < 0.05) and O2 -⢠and â¢CH3 scavenging capacity tended to decrease in patients with COPD compared to that in healthy controls. On the other hand, ROO⢠scavenging capacity tended to increase. In addition, RO⢠scavenging capacity was associated with severity of emphysema (p < 0.05) and exacerbation frequency (p < 0.02). There was a difference in the profile of the scavenging capacity between survived and deceased patients with COPD for 5 years after initial assessment. Conclusion: Characteristic profile of free radical scavenging capacity can provide insight into the pathophysiology and prognosis of patients with COPD.
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Enfisema , Doença Pulmonar Obstrutiva Crônica , Enfisema Pulmonar , Humanos , Idoso , Doença Pulmonar Obstrutiva Crônica/diagnóstico , Prognóstico , Volume Expiratório Forçado , Radicais Livres , Progressão da DoençaRESUMO
BACKGROUND AND OBJECTIVES: Osteoporosis is a common complication of chronic obstructive pulmonary dis-ease (COPD). It is impractical to measure bone mineral density (BMD) in all patients with COPD. This study aimed to investigate the relationship between Mini Nutritional Assessment Short-Form (MNA-SF), a simple nutritional status questionnaire, and osteoporosis, and to determine whether it can be used as a reliable screening tool for osteoporosis in patients with COPD. METHODS AND STUDY DESIGN: Thirty-seven patients with stable COPD were enrolled in this prospective cohort study. Patients with MNA-SF scores >11 were defined as well-nourished, and those with scores of ≤11 being at risk for malnutrition. Body composition, BMD, and undercarboxylated osteocalcin (ucOC), a bone metabolism marker, were measured using bioelectrical impedance, dual energy X-ray, and electrochemiluminescence immunoassay, respectively. RESULTS: Seventeen (45.9%) were classified as at risk for malnutrition, and 13 (35.1%) had osteoporosis. Patients at risk for malnutrition had significantly more osteoporosis and higher ucOC values than well-nourished patients (p=0.007, p=0.030, respectively). Patients with osteoporosis also had significantly lower body mass index (BMI) and fat-free mass index than those without osteoporosis (p= 0.007 and p=0.005, respectively), although FEV1 % pred was not significantly different. MNA-SF (cutoff value; 11) had better sensitivity to identify the presence of osteoporosis than BMI (cutoff value; 18.5 kg/m2) (sensitivity, 0.769; specificity, 0.708; sensitivity, 0.462; specificity, 0.875, respectively). CONCLUSIONS: MNA-SF was associated with osteoporosis and bone metabolism markers in patients with COPD. MNA-SF may be a useful screening tool for osteoporo-sis in patients with COPD.
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Desnutrição , Osteoporose , Doença Pulmonar Obstrutiva Crônica , Humanos , Idoso , Avaliação Nutricional , Estudos Prospectivos , Estado Nutricional , Desnutrição/diagnóstico , Desnutrição/etiologia , Doença Pulmonar Obstrutiva Crônica/complicações , Osteoporose/diagnóstico , Osteoporose/etiologia , Avaliação GeriátricaRESUMO
Background: Positive airway pressure (PAP) is a highly effective treatment for obstructive sleep apnea (OSA), but adherence limits its efficacy. In addition, coverage of PAP by CMS (Centers for Medicare & Medicaid Services) and other insurers in the United States depends on adherence. This leaves many beneficiaries without PAP, disproportionally impacting non-white and low socioeconomic position patients with OSA and exacerbating sleep health disparities. Methods: An inter-professional, multidisciplinary, international committee with various stakeholders was formed. Three working groups (the historical policy origins, impact of current policy, and international PAP coverage models) met and performed literature reviews and discussions. Using surveys and an iterative discussion-based consensus process, the policy statement recommendations were created. Results: In this position paper, we advocate for policy change to CMS PAP coverage requirements to reduce inequities and align with patient-centered goals. We specifically call for eradicating repeat polysomnography, eliminating the 4-hour rule, and focusing on patient-oriented outcomes such as improved sleepiness and sleep quality. Conclusions: Modifications to the current policies for PAP insurance coverage could improve health disparities.
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Síndromes da Apneia do Sono , Apneia Obstrutiva do Sono , Idoso , Humanos , Estados Unidos , Medicare , Apneia Obstrutiva do Sono/terapia , Sono , PolíticasRESUMO
A 28-year-old man with ankylosing spondylitis (AS) who was treated with a tumour necrosis factor-alpha (TNF-α) inhibitor, adalimumab, presented with newly detected multiple bilateral pulmonary nodules on chest computed tomography (CT). We suspected bacterial infection, including those caused by acid-fast bacilli, or adalimumab-related condition, such as sarcoidosis. After adalimumab cessation, no resolution of the pulmonary shadows was observed. Moreover, pulmonary cavitation appeared on chest CT at 7 weeks, prompting surgical lung biopsy. Acid-fast bacteria culture of the lung tissue showed negative results. Pathological examination suggested that confluent granulomas associated with sarcoidosis might have obstructed the blood vessels, causing necrosis and lung cavitation. Consequently, prednisolone was initiated, and these shadows were reduced. After administering anti-interleukin (IL)-17A antibody for treatment of AS and prednisolone withdrawal, these shadows were not exacerbated. TNF-α inhibitor-induced sarcoidosis could cause cavitary lesions due to vascular invasion of granulomas.
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Background: Among international cardiologists it is unclear whether equipoise exists regarding the benefit of diagnosing and managing obstructive sleep apnea (OSA) to improve atrial fibrillation (AF) outcomes and whether clinical practice and equipoise are linked. Methods: Between January 2019 and June 2020 we distributed a web-based 12-question survey regarding OSA and AF management to practicing cardiologists in 16 countries. Results: The United States, Japan, Sweden, and Turkey accounted for two-thirds of responses. 863 cardiologists responded; half were general cardiologists, a quarter electrophysiologists. Responses regarding treating OSA with CPAP to improve AF endpoints were mixed. 33% of respondents referred AF patients for OSA screening. OSA was diagnosed in 48% of referred patients and continuous positive airway pressure (CPAP) was prescribed for 59% of them. Nearly 70% of respondents believed randomized controlled trials (RCTs) of OSA treatment in AF patients were necessary and indicated willingness to contribute to such trials. Conclusions: There was no clinical equipoise among surveyed cardiologists; a majority expressed certainty that combined OSA and AF treatment is superior to AF treatment alone for improving AF outcomes. However, a minority of surveyed cardiologists referred AF patients for OSA testing, and while half of screened AF patients had OSA, CPAP was prescribed in little more than half of them, reflecting the view that better clinical trial evidence is needed to support this practice. Our results underscore the need for larger, multi-national prospective studies of OSA treatment and AF outcomes to inform more uniform society guideline recommendations.
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The prevalence of sleep disordered breathing (SDB) is reportedly very high. Among SDBs, the incidence of obstructive sleep apnea (OSA) is higher than previously believed, with patients having moderate-to-severe OSA accounting for approximately 20% of adult males and 10% of postmenopausal women not only in Western countries but also in Eastern countries, including Japan. Since 1998, when health insurance coverage became available, the number of patients using continuous positive airway pressure (CPAP) therapy for sleep apnea has increased sharply, with the number of patients about to exceed 500,000 in Japan. Although the "Guidelines for Diagnosis and Treatment of Sleep Apnea Syndrome (SAS) in Adults" was published in 2005, a new guideline was prepared in order to indicate the standard medical care based on the latest trends, as supervised by and in cooperation with the Japanese Respiratory Society and the "Survey and Research on Refractory Respiratory Diseases and Pulmonary Hypertension" Group, of Ministry of Health, Labor and Welfare and other related academic societies, including the Japanese Society of Sleep Research, in addition to referring to the previous guidelines. Because sleep apnea is an interdisciplinary field covering many areas, this guideline was prepared including 36 clinical questions (CQs). In the English version, therapies and managements for SAS, which were written from CQ16 to 36, were shown. The Japanese version was published in July 2020 and permitted as well as published as one of the Medical Information Network Distribution Service (Minds) clinical practice guidelines in Japan in July 2021.
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Síndromes da Apneia do Sono , Apneia Obstrutiva do Sono , Adulto , Pressão Positiva Contínua nas Vias Aéreas , Feminino , Humanos , Masculino , Prevalência , Síndromes da Apneia do Sono/diagnóstico , Síndromes da Apneia do Sono/epidemiologia , Síndromes da Apneia do Sono/terapia , Apneia Obstrutiva do Sono/diagnóstico , Apneia Obstrutiva do Sono/epidemiologia , Apneia Obstrutiva do Sono/terapia , Inquéritos e QuestionáriosRESUMO
BACKGROUND: Some chronic obstructive pulmonary disease (COPD) patients develop hypoxemia with disease progression, with some even requiring long-term oxygen therapy (LTOT). Lung function, especially diffusing capacity, and the annual decline in PaO2, are reported to be predictive factors of chronic respiratory failure. However, the association between lung morphometry evaluated using computed tomography (CT) images and LTOT initiation is unknown. METHODS: We retrospectively evaluated the relationship between clinical indices, including pulmonary function, body mass index (BMI), and CT parameters, at baseline and LTOT initiation in two prospective COPD cohorts. In the Nara Medical University cohort (n = 76), the low attenuation area (LAA) and its fractal dimension (fractal D) were adapted as the indices for parenchymal destruction in CT images. The association between these CT measurements and LTOT initiation was replicated in the Kyoto University cohort (n = 130). RESULTS: In the Nara Medical University cohort, lower BMI (hazard ratio [HR]:0.70, p = 0.006), lower % diffusing capacity (%DLCO) (HR: 0.92, p = 0.006), lower %DLCO/VA (HR, 0.90, p = 0.008), higher RV/TLC (HR, 1.26, p = 0.012), higher LAA% (HR: 1.18, p = 0.001), and lower fractal D (HR: 3.27 × 10-8, p < 0.001) were associated with LTOT initiation. Multivariate analysis in the Kyoto University cohort confirmed that lower %DLCO and lower fractal D were independently associated with LTOT initiation, whereas LAA% was not. CONCLUSION: Fractal D, which is the index for morphometric complexity of LAA in CT analysis, is predictive of LTOT initiation in COPD patients.
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Fractais , Doença Pulmonar Obstrutiva Crônica , Estudos de Coortes , Humanos , Oxigênio , Oxigenoterapia , Estudos Prospectivos , Doença Pulmonar Obstrutiva Crônica/diagnóstico por imagem , Doença Pulmonar Obstrutiva Crônica/terapia , Estudos Retrospectivos , Tomografia Computadorizada por Raios XRESUMO
The prevalence of sleep-disordered breathing (SDB) is reportedly very high. Among SDBs, the incidence of obstructive sleep apnea (OSA) is higher than previously believed, with patients having moderate-to-severe OSA accounting for approximately 20% of adult males and 10% of postmenopausal women not only in Western countries but also in Eastern countries, including Japan. Since 1998, when health insurance coverage became available, the number of patients using continuous positive airway pressure (CPAP) therapy for sleep apnea has increased sharply, with the number of patients about to exceed 500,000 in Japan. Although the "Guidelines for Diagnosis and Treatment of Sleep Apnea Syndrome (SAS) in Adults" was published in 2005, a new guideline was prepared to indicate the standard medical care based on the latest trends, as supervised by and in cooperation with the Japanese Respiratory Society and the "Survey and Research on Refractory Respiratory Diseases and Pulmonary Hypertension" Group, of Ministry of Health, Labor and Welfare and other related academic societies, including the Japanese Society of Sleep Research, in addition to referring to the previous guidelines. Since sleep apnea is an interdisciplinary field covering many areas, this guideline was prepared including 36 clinical questions (CQs). In the English version, therapies and managements for SAS, which were written from CQ16 to 36, were shown. The Japanese version was published in July 2020 and permitted as well as published as one of the Medical Information Network Distribution Service (Minds) clinical practice guidelines in Japan in July 2021.
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We describe a case of Trousseau's syndrome in a patient with lung carcinoma. A 69-year-old man presented with pleural effusion. Further evaluation revealed EGFR mutation-positive non-small cell carcinoma in the upper lobe with extensive lymph node, bone, and brain metastases. Administration of osimertinib, an EGFR tyrosine kinase inhibitor, resulted in partial tumor response, but caused osimertinib-induced pneumonitis 10 weeks later. Prednisolone restrained lung injury progression and was gradually tapered. However, he presented with impaired consciousness and right hemiplegia. Magnetic resonance imaging revealed a left middle cerebral artery M1 segment occlusion. D-dimer level was elevated to 19.5 µg/mL. In the absence of atherosclerotic or cardiogenic thrombi, these findings led to the diagnosis of Trousseau syndrome. Endovascular therapy, but not tissue plasminogen activator, improved his condition with no recurrences. These treatment strategies are crucial to restore function in patients with potentially disabling cerebral infarction due to Trousseau syndrome.
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PURPOSE: Chronic obstructive pulmonary disease (COPD) is a worldwide problem because of its high prevalence and mortality. However, there is no fundamental treatment to ameliorate their pathological change in COPD lung. Recently, adipose-derived mesenchymal stem cells (ADSCs) have attracted attention in the field of regenerative medicine to repair damaged organs. Moreover, their utility in treating respiratory diseases has been reported in some animal models. However, the detailed mechanism by which ADSCs improve chronic respiratory diseases, including COPD, remains to be elucidated. We examined whether human ADSCs (hADSCs) ameliorated elastase-induced emphysema and whether hADSCs differentiated into alveolar epithelial cells in a murine model of COPD. METHODS: Female SCID-beige mice (6 weeks old) were divided into the following four groups according to whether they received an intratracheal injection of phosphate-buffered saline or porcine pancreatic elastase, and whether they received an intravenous injection of saline or hADSCs 3 days after intratracheal injection; Control group, hADSC group, Elastase group, and Elastase-hADSC group. We evaluated the lung function, assessed histological changes, and compared gene expression between hADSCs isolated from the lung of Elastase-hADSC group and naïve hADSCs 28 days after saline or elastase administration. RESULTS: hADSCs improved the pathogenesis of COPD, including the mean linear intercept and forced expiratory volume, in an elastase-induced emphysema model in mice. Furthermore, hADSCs were observed in the lungs of elastase-treated mice at 25 days after administration. These cells expressed genes related to mesenchymal-epithelial transition and surface markers of alveolar epithelial cells, such as TTF-1, ß-catenin, and E-cadherin. CONCLUSION: hADSCs have the potential to improve the pathogenesis of COPD by differentiating into alveolar epithelial cells by mesenchymal-epithelial transition.
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Enfisema , Células-Tronco Mesenquimais , Doença Pulmonar Obstrutiva Crônica , Enfisema Pulmonar , Animais , Modelos Animais de Doenças , Feminino , Humanos , Camundongos , Camundongos Endogâmicos C57BL , Camundongos SCID , Elastase Pancreática , Doença Pulmonar Obstrutiva Crônica/induzido quimicamente , Doença Pulmonar Obstrutiva Crônica/terapia , Enfisema Pulmonar/induzido quimicamente , Enfisema Pulmonar/genética , Enfisema Pulmonar/terapia , SuínosRESUMO
BACKGROUND: Dyspnea is a common symptom in patients with COPD. It causes physical inactivity and impaired health-related quality of life. Although optimal breathing methods alleviate dyspnea, it is unclear whether breathing instability has a clinical impact on patients with COPD. This study aimed to investigate whether resting breathing instability during wakefulness was associated with dyspnea assessed by the modified Medical Research Council (mMRC) dsypnea scale and whether breathing instability can be a novel predictor of clinical outcomes. METHODS: Forty-four subjects with stable COPD were enrolled (mean age, 71.0 y). Resting breathing was monitored for 15 min by using respiratory inductance plethysmography. Breathing instability was evaluated with the coefficient of variation for breath-by-breath respiratory duration and tidal volume ([Formula: see text]) by using an artifact-free respiratory signal for 5 min. Pulmonary function testing and blood gas analysis were performed (mean FEV1 percent of predicted, 68.5%). Questionnaires with regard to dyspnea and health-related quality of life were also completed. Exacerbations were recorded prospectively for 1 year after the initial assessment. RESULTS: The coefficients of variation for [Formula: see text] were significantly higher in the subjects with an mMRC dyspnea scale score ≥ 2 versus those with an mMRC dyspnea scale score < 2 (26.4 ± 7.4% vs 20.3 ± 6.4%, P = .006) . The coefficients of variation for respiratory duration and VT were not associated with age, body mass index, and pulmonary function variables. In multivariate analysis, FEV1 percent of predicted and coefficient of variation for [Formula: see text] remained significant predictors for an mMRC dyspnea scale score ≥ 2 (P = .004 and P = .01, respectively). Coefficient of variation values were also correlated with several health-related quality of life domains. The exacerbation frequency was associated with the coefficient of variation for [Formula: see text]. CONCLUSIONS: Resting breathing pattern during wakefulness is a novel assessment tool for severity of dyspnea, which can be one of the predictors for exacerbation in patients with COPD.
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Doença Pulmonar Obstrutiva Crônica , Qualidade de Vida , Idoso , Dispneia/etiologia , Humanos , Doença Pulmonar Obstrutiva Crônica/complicações , Respiração , Índice de Gravidade de Doença , VigíliaRESUMO
Dupilumab, a human monoclonal antibody against interleukin-4 (IL-4) and IL-13, has been approved for treating severe asthma and eosinophilic chronic rhinosinusitis (ECRS). Patients with ECRS are often candidates for endoscopic sinus surgery (ESS). However, a considerable number of patients have recurrent ECRS. ECRS is an important factor influencing asthma control. Here, we present two cases of severe asthma and recurrent ECRS after ESS. Although they had been treated with inhaled corticosteroids and a long-acting ß2-agonist, they experienced frequent asthma exacerbations. Laboratory examinations revealed increased serum eosinophils and immunoglobulin E (IgE). Furthermore, the Asthma Control Test (ACT) score and forced expiratory volume in 1 sec (FEV1) were indicative of airway obstruction. After treatment with dupilumab, asthma, rhinosinusitis symptoms, and pulmonary function improved remarkably. Dupilumab therapy improved quality of life in these patients with severe asthma and ECRS.
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Nintedanib is a multi-target receptor tyrosine kinase inhibitor that reduces the decline in forced vital capacity (FVC) and prevents acute exacerbations in idiopathic pulmonary fibrosis (IPF), which is a risk factor for lung cancer. However, it remains unclear whether nintedanib is an effective treatment for lung cancer in patients with IPF. Here, we describe an 82-year-old man with non-small cell lung carcinoma complicated by IPF who was treated with nintedanib. High-resolution computed tomography (HRCT) showed a subpleural basal-predominant reticular shadow and traction bronchiectasis with a honeycomb pattern. His FVC decreased over time, and his 6-min walk test showed oxygen desaturation. Furthermore, an enlarged nodular lesion was detected after 6 months of referral. Biopsy confirmed non-small cell carcinoma. Because of the risk of acute exacerbation of IPF by chemotherapy, supportive care was selected. Nintedanib was started as treatment for the IPF. Nine months later, HRCT revealed partial remission without exacerbation of IPF. This case indicates the possibility of nintedanib monotherapy in suppressing lung cancer complicated by IPF. Patients with lung cancer complicated by IPF in whom treatment is effective remain unknown. Additional research is needed to identify effective therapy for lung cancer with IPF.
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Carcinoma Pulmonar de Células não Pequenas/complicações , Carcinoma Pulmonar de Células não Pequenas/tratamento farmacológico , Fibrose Pulmonar Idiopática/etiologia , Indóis/uso terapêutico , Neoplasias Pulmonares/complicações , Neoplasias Pulmonares/tratamento farmacológico , Idoso de 80 Anos ou mais , Humanos , Fibrose Pulmonar Idiopática/patologia , Indóis/farmacologia , MasculinoRESUMO
BACKGROUND: Pompe disease is an autosomal recessive disorder caused by deficiency of the acid α-glucosidase (GAA) enzyme. GAA deficiency induces progressive glycogen accumulation which leads to weakness of the respiratory muscle including the diaphragm. Pompe disease is one of the few myopathies, for which an established therapy is available. Thus, earlier detection of potential late-onset Pompe disease (LOPD) and earlier intervention would have a significant clinical impact. PURPOSE: Our hypothesis is that sleep problems including sleep disordered breathing (SDB) and clinical symptoms may indicate an early stage of LOPD since decreased respiratory muscle activity generally first presents during sleep. Thus, the aims of this prospective, multicenter observational cohort study in Japan (PSSAP-J) are to demonstrate a higher prevalence of LOPD in a sleep lab-based population (primary outcome), and to identify predictive factors for LOPD from findings in diagnostic polysomnography (PSG) and clinical symptoms (secondary outcomes). METHODS: The study design is a prospective multicenter observational cohort study. Consecutive patients who present to sleep labs due to suspected SDB for an overnight PSG will be enrolled. All patients will be measured for creatine kinase, GAA activity, and if necessary, genetic analysis of GAA. Furthermore, chest X-ray, pulmonary function test, and arterial blood gas analysis will be collected. Then, prevalence and specific findings of LOPD will be assessed. RESULT: Congenital myopathy shows a shift from slow-deep to rapid-shallow breathing during transition from wakefulness to sleep accompanying a symptom of waking with gasping (actual further results are pending). DISCUSSION: The distribution in respiratory physiology between during wakefulness and sleep specific to LOPD may provide insights into early-stage detection. CLINICAL TRIAL REGISTRATION NUMBER: UMIN000039191, UMIN Clinical Trials Registry ( http://www.umin.ac.jp/ctr ).
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Doença de Depósito de Glicogênio Tipo II/diagnóstico , Programas de Rastreamento , Síndromes da Apneia do Sono/epidemiologia , Idade de Início , Diagnóstico Precoce , Doença de Depósito de Glicogênio Tipo II/epidemiologia , Humanos , Japão/epidemiologia , Polissonografia , Estudos Prospectivos , Projetos de PesquisaRESUMO
Purpose: This review will trace the elements of neurostimulation for obstructive sleep apnea and details on its implementation, efficacy and safety, immediate clinical outcomes, and future prospects. Methods: The literature on upper airway neurostimulation was surveyed from July, 2013, to July 2019, with a focus on the components of devices, evidence for clinical utility, and adverse events. Results: Current technology is focused on the hypoglossal nerve stimulation (HNS). The most long-term experience is with the Inspire Medical System (Maple Grove, MN USA) which has both FDA and European regulatory approval. Given the inclusion criteria (BMI <35, ideally <32), AHI 15-65/h, and a favorable anterior-posterior velopharyngeal collapse pattern on DISE), across many centers ~65% of patients who are intolerant to primary therapy achieve clinical success (AHI <20/h with a reduction of <50% in AHI), and more have symptomatic relief. Adverse events are generally mild, often self-limited, with occasional need for uncomplicated surgical adjustments or replacement of the implantable generator. Three other devices are in various phases of development, each with a differences in nerve electrodes, implantable components, power sources, proprietary programming, and activation patterns. Conclusions: HNS is not considered a first-line treatment option. HNS therapy, however, should be considered as one alternative therapeutic option for patients meeting the inclusion criteria when more traditional therapeutic options have been considered.