Therapeutic Advances in Respiratory Disease最新文献

SummaryWhat is this summary about?• The Asthma Impairment and Risk Questionnaire (AIRQ^®) has been designed and tested to measure patients' levels of asthma control in a healthcare setting.• Unlike other available questionnaires that only assess asthma symptoms that can be bothersome or limit a person's activities and quality of life (impairment-related symptoms), the AIRQ also includes questions related to risk of an asthma attack. This allows for a broader measurement of asthma control and a prediction of the chance of having future asthma attacks.• AIRQ scores are linked to a patient's own experience of their health and how it impacts their daily life (health-related quality of life).• The AIRQ may make it easier for patients and healthcare professionals to have shared decision-making discussions that can lead to better asthma care and asthma outcomes.• This document summarizes several published studies of the AIRQ in people with asthma.

Connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) is a progressive and high-risk subtype of PAH, with outcomes generally worse than those seen in idiopathic PAH. Early recognition and treatment are essential for improving survival, yet early-stage CTD-PAH remains challenging to identify, particularly for non-specialist clinicians. The 2022 ESC/ERS guidelines introduced several key updates that support an earlier diagnosis and more targeted management. These include a revised echocardiographic threshold for pulmonary hypertension probability (tricuspid regurgitation velocity >2.8 m/s), a lowered hemodynamic definition of PAH (mean pulmonary artery pressure >20 mmHg and pulmonary vascular resistance >2 WU), and a preference for annual screening using the DETECT algorithm in asymptomatic systemic sclerosis (SSc) patients. Additionally, novel therapeutic targets such as the activin/TGF-β pathway have been incorporated into updated treatment algorithms. Although CTD-PAH remains associated with worse outcomes than idiopathic PAH, recent advances in screening, risk assessment, and targeted therapies have begun to improve the trajectory of the disease. Early detection, personalized treatment, and comprehensive care are now key to transforming this high-risk condition into a more manageable one.

Background: Nontuberculous mycobacterial lung disease (NTM-LD) is a chronic infection of the lungs with a high symptom burden. NTM-LD treatment is typically long and complicated, which can impact quality of life and mental health. Increased support for psychological challenges is a priority for this population.

Objectives: We describe integrating psychological care into a multidisciplinary outpatient NTM program, patient characteristics, and results of patient-reported outcomes (PRO) screening (of depression, anxiety, fatigue, health-related quality of life, quality of life, and Top Problems).

Design: Retrospective observational cohort study design.

Methods: Processes and structure around psychology integration are described. Descriptive data obtained via retrospective chart review (IRB approved) are presented on patient sociodemographic factors, psychiatric medication and psychotherapy use, and results of PRO screenings with NTM-LD patients anticipated to start NTM antibiotic treatment or already on treatment. Relationships between variables were examined using nonparametric statistics.

Results: From 2020 to 2024, 175 patients with NTM-LD were screened. Patients were on average 65.7 ± 9.8 years old, female (74.2%), white (91.4%), and on Medicare (69.1%). On average, this group experienced a moderate degree of socioeconomic disadvantage; 94.9% of patients lived in areas with a mental health provider shortage, and 42.3% lived in medically underserved areas. Patients reported considerable rates of mild or higher depression (54.3%) and anxiety (32.0%). Many utilized psychiatric (52.6%) or pain (20.6%) medications, while engagement in psychotherapy was low (5.1%). Patients reported impacts on quality of life, fatigue, and health-related quality of life, and the most common Top Problems were: "Shortness of breath, Breathlessness, Getting winded," "Fatigue/Low energy," and "Cough/Choking."

Conclusion: A licensed psychologist was successfully integrated into the NTM program. The disparity between PRO results and psychotherapy engagement highlights a key opportunity for mental health interventions. Integrated psychological services may provide streamlined access to mental healthcare.

Background: Chronic cough (CC) has a negative effect on patients' quality of life and everyday activities. Emotional reactions are an important aspect of how patients deal with illness, and CC can lead to feelings of uncertainty, loss of control, helplessness, self-consciousness, embarrassment, worry, fear, frustration, irritability, and anger.

Objectives: To assess the emotions related to bouts of cough in patients with refractory or unexplained chronic cough (RCC/UCC), using a validated questionnaire.

Design: This was a multicenter, non-interventional study.

Methods: Patients with RCC/UCC were recruited from hospital outpatient clinics in Spain. The impact of RCC/UCC on patients' emotions was assessed using the self-reported Discrete Emotions Questionnaire (DEQ), which assesses eight distinct situationally induced state emotions, scored on a Likert scale from 1 to 7, with higher scores indicating that the emotion is experienced to a greater degree.

Results: The survey was completed by 190 patients (148 women, 42 men; mean age 58.0 years) of whom 120 had RCC and 70 had UCC (mean cough duration 6.3 years). Highest mean (SD) scores were seen for the anger subscale (3.6 (1.7)) and anxiety subscale (3.3 (1.6), followed by the disgust (2.6 (1.5)), fear (2.6 (1.7)), and sadness (2.5 (1.5)) subscales. The same pattern was seen in subgroups by gender and by diagnosis (RCC or UCC). There were no significant differences in mean scores for any subscale between patients with RCC and those with UCC. Cough severity and the presence of cough-related stress urinary incontinence (SUI) were independently associated with negative emotion subscale scores.

Conclusion: Among patients with RCC/UCC, bouts of cough triggered negative emotions such as anger, anxiety, disgust, fear, and sadness, as evaluated using a validated questionnaire. Results were similar for RCC and UCC. Increasing cough severity and the presence of SUI were independently associated with higher (worse) scores for negative emotions.

Background: Afatinib is indicated for patients with advanced-stage non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) mutations, including uncommon mutations. However, the differences in survival benefits between patients with different types of EGFR mutations remain unclear.

Objectives: This study aimed to compare the effectiveness of afatinib treatment in patients harboring the EGFR-G719X mutation with that in patients carrying other uncommon EGFR mutations.

Design: This was a retrospective study.

Methods: Ninety-two patients with locally advanced and metastatic NSCLC, of whom 49 patients with EGFR-G719X mutations that were both single and compound, and 43 patients harbored other uncommon EGFR mutations, who were treated with afatinib as first-line treatment. The patients were followed up and evaluated every 3 months or when there were symptoms of progressive disease. The endpoints were the objective response rate (ORR), time-to-treatment failure (TTF), and overall survival (OS).

Results: The average ages of patients with the EGFR-G719X and uncommon EGFR mutations were 62.7 years and 63.1 years, respectively. There were no significant differences in sex or smoking history between the two groups. In total, 28.6% of patients with the G719X mutation and 23.3% of patients with other mutations had brain metastases. The ORR of patients with the G719X mutation was 79.6%, which was 10% higher than that of patients with other EGFR mutations. Patients harboring the EGFR-G719X mutation had median TTF and median OS periods of 19.3 months and 31.4 months, respectively, which were significantly higher than those of patients carrying other mutations at 11.2 months. Subgroup analysis showed that TTF and OS benefits were observed in female patients, patients without brain metastasis, and patients with good performance status who harbored the G719X mutation.

Conclusion: Patients with the EGFR-G719X mutation achieve significantly better TTF and OS benefits than those with other uncommon EGFR mutations.

Background: Pulmonary segmentectomy is increasingly recognized as a viable alternative to lobectomy for early stage non-small-cell lung cancer (NSCLC), offering comparable oncological outcomes with potentially reduced morbidity. Identifying reliable predictors for postoperative complications and prolonged air leak (PAL) is crucial for optimizing patient selection. While multifactorial scoring systems exist, their complexity limits clinical utility and the predictive value of single factors, such as forced expiratory volume in 1s (FEV₁) and diffusing capacity for carbon monoxide (DL_CO), remains underexplored.

Objectives: This study aimed to evaluate the ability of preoperative FEV₁ and DL_CO to predict complications (Clavien-Dindo ⩾ 3a) and PAL in patients undergoing pulmonary segmentectomy.

Design: A retrospective, single-center study compared outcomes between patients undergoing segmentectomy (n = 33) and lobectomy (n = 126) for NSCLC.

Methods: Patient characteristics, complication rates, and PAL incidence were analyzed. Logistic regression and ROC curve analyses assessed the predictive accuracy of FEV₁ and DL_CO for complications and PAL.

Results: Baseline characteristics, including FEV₁ and DL_CO, were comparable between the segmentectomy and lobectomy groups (p > 0.05). FEV₁ was identified as a significant predictor of complications, with lower values associated with increased risk. DL_CO exhibited an even stronger predictive value for complications in the segmentectomy cohort, with an AUC of 0.924, indicating excellent predictive accuracy. In contrast, neither FEV₁ nor DL_CO demonstrated significant predictive value for PAL, which occurred in 30% of segmentectomy and 20% of lobectomy patients (p > 0.05).

Conclusion: Preoperative FEV₁ and DL_CO are valuable predictors of complications (Clavien-Dindo ⩾ 3a) in pulmonary segmentectomy, with DL_CO showing high predictive accuracy. However, their inability to reliably predict PAL highlights the need for multifactorial models to enhance risk assessment. Despite the limited sample size, our findings align with larger studies and reinforce the clinical utility of FEV₁ and DL_CO for preoperative risk stratification in segmentectomy patients.

Chronic obstructive pulmonary disease (COPD) refers to a group of lung diseases that are distinct in underlying aetiology but share a common disease course of persistent and progressive airflow restriction. People living with COPD, as well as the people who care for them, frequently have severe and unmet physical and psychosocial needs, including breathlessness, fatigue, cough, anxiety and depression. Early proactive palliative care is well placed to address these needs, yet it is frequently under-utilised in this group. This narrative review aimed to identify core components of palliative care and examine how existing models of care are implemented to better understand which models can best serve the needs of people with COPD. Symptom palliation, advance care planning, and support for caregivers emerged as the common components underpinning both generalist and specialist models of palliative care. Models of proactive palliative care were diverse in terms of where and how care was delivered as well as which health professionals were involved. Five key models of palliative care were identified: (1) multi-disciplinary integrated services, (2) nurse-led care, (3) hospice and residential aged care, (4) home-based care, and (5) telemonitoring and telehealth. Each model describes a diverse set of interventions and many of these share common elements, including the normalisation of palliative principles within routine care and the provision of diverse delivery settings to accommodate individual preferences and needs. Successful palliative care models must be practical, accessible and innovative to respond to individuals' complex and evolving needs, foster multi-disciplinary collaboration and input and optimally utilise local healthcare resources.

Idiopathic pulmonary fibrosis (IPF) is often regarded as the archetypal progressive fibrosing interstitial lung disease (ILD). The term "progressive pulmonary fibrosis" (PPF) generally describes progressive lung fibrosis in an individual with an ILD other than IPF. Both IPF and PPF are associated with loss of lung function, worsening dyspnea and quality of life, and premature death. Current treatments slow the decline in lung function but have side effects that may deter the initiation or continuation of treatment. There remains a high unmet need for additional therapies that can be used alone or in combination with current therapies to preserve lung function in patients with IPF and PPF. Phosphodiesterase-4 (PDE4) is an enzyme involved in the regulation of inflammatory processes. Pre-clinical studies have shown that preferential inhibition of PDE4B has anti-inflammatory and antifibrotic effects and a lower potential for gastrointestinal adverse events than pan-PDE4 inhibition. The preferential PDE4B inhibitor nerandomilast demonstrated efficacy in preserving lung function in a phase II trial in patients with IPF and is under investigation in phase III trials as a treatment for IPF and PPF.