Journal of Cystic Fibrosis最新文献

英文中文

65 Thymic stromal lymphopoietin and IL-33 alarmin cytokine production by primary human cystic fibrosis lung macrophages is induced by infection with Pseudomonas aeruginosa and further increased by CFTR modulators 65 感染铜绿假单胞菌可诱导原代人囊性纤维化肺巨噬细胞产生胸腺基质淋巴细胞生成素和 IL-33 警报细胞因子，CFTR 调节剂可进一步增加其产生量

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis

Pub Date : 2024-09-01 DOI: 10.1016/S1569-1993(24)00908-1

D. Aridgides, D. Mellinger, L. Gwilt, A. Ashare

引用次数: 0

82 Unveiling the effect of elexacaftor/tezacaftor/ivacaftor therapy on Pseudomonas aeruginosa persistence and adaptation through RNA expression profiling 82 通过RNA表达谱分析揭示elexacaftor/tezacaftor/ivacaftor疗法对铜绿假单胞菌持久性和适应性的影响

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis

Pub Date : 2024-09-01 DOI: 10.1016/S1569-1993(24)00924-X

M. Letizia, L. Whiteley, F. Diggle, E. McKone, P. Singh, M. Whiteley

引用次数: 0

62 Decoding Pseudomonas aeruginosa adaptive response to elexacaftor/tezacaftor/ivacaftor: a multi-omics approach 62 解码铜绿假单胞菌对 elexacaftor/tezacaftor/ivacaftor 的适应性反应：一种多组学方法

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis

Pub Date : 2024-09-01 DOI: 10.1016/S1569-1993(24)00905-6

L. Veschetti, S. Piccolo, G. Vingiani, C. Paganin, E. Fiscarelli, A. Bragonzi, C. Cigana

引用次数: 0

7 Anti-Psl/PcrV bispecific monoclonal antibody potentiates in vitro and in vivo bacterial clearance of Pseudomonas aeruginosa from children with cystic fibrosis who failed tobramycin eradication therapy 7 抗 Psl/PcrV 双特异性单克隆抗体可增强对妥布霉素根除疗法失败的囊性纤维化儿童铜绿假单胞菌的体内外细菌清除能力

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis

Pub Date : 2024-09-01 DOI: 10.1016/S1569-1993(24)00850-6

S. Goldman, K. Kwong, A. Beauchamp, V. Waters, A. DiGiandomenico, D. Nguyen

引用次数: 0

Education, employment, and income among people living with cystic fibrosis across three decades – A matched cohort study using Danish health registries 三十年来囊性纤维化患者的教育、就业和收入情况--利用丹麦健康登记进行的匹配队列研究。

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis

Pub Date : 2024-09-01 DOI: 10.1016/j.jcf.2024.03.009

Background

Past and ongoing advancements in cystic fibrosis (CF) care warrant long-term analysis of the societal impact of the condition. This study aims to evaluate changes in key socioeconomic factors across three decades among people living with CF (pwCF), compared with both the general population and an early-onset chronic disease population.

Methods

This nationwide, registry-based, matched cohort study included all pwCF ≥ 18 years in Denmark in the years 1990, 2000, 2010, and 2018. Each person living with CF was matched to five individuals in the general population and five individuals living with type 1 diabetes or juvenile arthritis based on age, sex, and municipality.

Results

The Danish adult CF population increased nearly fourfold from 88 in 1990 to 331 in 2018, and mean age increased by ten years. The educational level of pwCF was similar to the two comparator cohorts, while pwCF were less often in employment and more often permanently outside the labor force. Personal and household income levels of the CF cohort were higher than those of the comparator cohorts.

Conclusions

The disadvantage in employment for pwCF remained, but, over time, the societal profiles of the one-year CF cohorts increasingly converged with those of the comparator cohorts, indicative of improved clinical management, extended life expectancy, and the supportive role of the Danish welfare system in reducing health inequalities. Further research should be done to evaluate the effects of the newly introduced modulator therapies on employment, considering the broader societal impact and impact on quality of life.

背景：囊性纤维化（CF）治疗领域过去和现在的进步需要对该疾病的社会影响进行长期分析。本研究旨在评估三十年来囊性纤维化患者（pwCF）与普通人群和早发慢性病人群相比在关键社会经济因素方面的变化：这项基于登记的全国性配对队列研究纳入了 1990 年、2000 年、2010 年和 2018 年丹麦所有年龄≥ 18 岁的 CF 患者。根据年龄、性别和市镇，每位CF患者都与五位普通人群和五位1型糖尿病或幼年关节炎患者匹配：丹麦成年 CF 患者从 1990 年的 88 人增加到 2018 年的 331 人，增长了近四倍，平均年龄增加了 10 岁。长者的受教育程度与两个参照组群相似，但长者的就业率较低，更多的长者长期没有工作。残疾人家庭组群的个人和家庭收入水平高于参照组群：但随着时间的推移，一年期 CF 群体的社会概况与参照群体的社会概况日益趋同，这表明临床管理得到改善、预期寿命延长以及丹麦福利制度在减少健康不平等方面发挥了支持作用。考虑到更广泛的社会影响和对生活质量的影响，应开展进一步研究，评估新引入的调节疗法对就业的影响。

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引用次数: 0

Elexacaftor/tezacaftor/ivacaftor improves nasal nitric oxide in patients with cystic fibrosis Elexacaftor/tezacaftor/ivacaftor 可改善囊性纤维化患者的鼻腔一氧化氮。

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis

Pub Date : 2024-09-01 DOI: 10.1016/j.jcf.2024.03.003

Background

In health, nitric oxide (NO) shows high concentrations in the upper airways, while nasal NO (nNO) is significantly lower in patients with sinonasal inflammation, such as people with cystic fibrosis (PwCF). In PwCF treated with elexacaftor/tezacaftor/ivacaftor (ETI; PwCF-ETI), clinical improvement of sinonasal symptoms and inflammation was observed. We therefore hypothesised that ETI may increase nNO in PwCF.

Methods

25 PwCF-ETI underwent nNO measurement at baseline and after 3 to 24 months of ETI treatment. NNO was measured using velum closure (VC) techniques in cooperative patients and tidal breathing (TB) for all patients. As controls, 7 CF patients not eligible for ETI (PwCF-non ETI) and 32 healthy controls (HC) were also repeatedly investigated.

Results

In PwCF-ETI, sinonasal symptoms, lung function parameters and sweat chloride levels improved from baseline to follow-up whereas there was no change in PwCF-non ETI and HC. NNO increased from a median (IQR) value at baseline to follow-up from 348.2 (274.4) ppb to 779.6 (364.7) ppb for VC (P < 0.001) and from 198.2 (107.0) ppb to 408.3 (236.1) ppb for TB (P < 0.001). At follow-up, PwCF-ETI reached nNO values in the normal range. In PwCF-non ETI as well as HC, nNO did not change between baseline and follow-up.

Conclusions

In PwCF-ETI, the nNO values significantly increased after several months of ETI treatment in comparison to baseline and reached values in the normal range. This suggests that nNO is a potential non-invasive biomarker to examine sinonasal inflammatory disease in PwCF and supports the observation of clinical improvement in these patients.

背景：一氧化氮（NO）在健康人的上呼吸道中浓度较高，而鼻腔NO（nNO）在囊性纤维化患者（PwCF）等鼻窦炎患者中则明显较低。在接受 elexacaftor/tezacaftor/ivacaftor （ETI；PwCF-ETI）治疗的 PwCF 患者中，我们观察到鼻窦症状和炎症得到了临床改善。因此，我们假设 ETI 可增加 PwCF 的 nNO。方法：25 名 PwCF-ETI 在基线和接受 ETI 治疗 3-24 个月后接受了 nNO 测量。在合作患者中使用 velum closure (VC) 技术测量 nNO，在所有患者中使用潮式呼吸 (TB) 技术测量 nNO。作为对照组，还对 7 名不符合 ETI 条件的 CF 患者（PwCF-non ETI）和 32 名健康对照组（HC）进行了反复调查：结果：PwCF-ETI 患者的鼻窦症状、肺功能参数和汗液氯化物水平从基线到随访期间均有所改善，而 PwCF-non ETI 和 HC 患者则没有变化。从基线到随访，VC 的 NNO 中位值（IQR）从 348.2 (274.4) ppb 增加到 779.6 (364.7) ppb（P < 0.001），TB 的 NNO 中位值（IQR）从 198.2 (107.0) ppb 增加到 408.3 (236.1) ppb（P < 0.001）。在随访中，PwCF-ETI 的 nNO 值达到正常范围。在非 ETI 的 PwCF 和 HC 中，nNO 在基线和随访期间没有变化：在接受 ETI 治疗数月后，PwCF-ETI 的 nNO 值与基线值相比明显升高，并达到正常值范围。这表明 nNO 是检查 PwCF 鼻窦炎性疾病的潜在非侵入性生物标志物，并支持对这些患者临床改善的观察。

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引用次数: 0

Widespread alterations in systemic immune profile are linked to lung function heterogeneity and airway microbes in cystic fibrosis 囊性纤维化患者全身免疫特征的广泛改变与肺功能异质性和气道微生物有关。

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis

Pub Date : 2024-09-01 DOI: 10.1016/j.jcf.2024.04.015

Background

Excessive inflammation and recurrent airway infections characterize people with cystic fibrosis (pwCF), a disease with highly heterogeneous clinical outcomes. How the overall immune response is affected in pwCF, its relationships with the lung microbiome, and the source of clinical heterogeneity have not been fully elucidated.

Methods

Peripheral blood and sputum samples were collected from 28 pwCF and an age-matched control group. Systemic immune cell subsets and surface markers were quantified using multiparameter flow cytometry. Lung microbiome composition was reconstructed using metatranscriptomics on sputum samples, and microbial taxa were correlated to circulating immune cells and surface markers expression.

Results

In pwCF, we found a specific systemic immune profile characterized by widespread hyperactivation and altered frequencies of several subsets. These included substantial changes in B-cell subsets, enrichment of CD35⁺/CD49d⁺ neutrophils, and reduction in dendritic cells. Activation markers and checkpoint molecule expression levels differed from healthy subjects. CTLA-4 expression was increased in Tregs and, together with impaired B-cell subsets, correlated with patients' lung function. Concentrations and frequencies of key immune cells and marker expression correlated with the relative abundance of commensal and pathogenic bacteria in the lungs.

Conclusion

The CF-specific immune signature, involving hyperactivation, immune dysregulation with alteration in Treg homeostasis, and impaired B-cell function, is a potential source of lung function heterogeneity. The activity of specific microbes contributes to disrupting the balance of the immune response. Our data provide a unique foundation for identifying novel markers and immunomodulatory targets to develop the future of cystic fibrosis treatment and management.

背景：过度炎症和反复气道感染是囊性纤维化患者（pwCF）的特征，这种疾病的临床结果具有高度异质性。囊性纤维化患者的整体免疫反应如何受到影响、其与肺部微生物组的关系以及临床异质性的来源尚未完全阐明：方法：收集了 28 例肺结核患者和年龄匹配的对照组的外周血和痰样本。采用多参数流式细胞术对全身免疫细胞亚群和表面标记物进行量化。使用元转录组学重建了痰样本的肺微生物组组成，并将微生物类群与循环免疫细胞和表面标志物的表达相关联：结果：在 pwCF 中，我们发现了一种特殊的全身免疫特征，其特点是广泛的过度激活和多个亚群频率的改变。这包括 B 细胞亚群的显著变化、CD35+/CD49d+ 中性粒细胞的富集以及树突状细胞的减少。活化标志物和检查点分子的表达水平与健康人不同。Tregs中CTLA-4的表达增加，与受损的B细胞亚群一起与患者的肺功能相关。关键免疫细胞的浓度和频率以及标记物的表达与肺部共生细菌和致病细菌的相对数量相关：结论：CF 特异性免疫特征包括过度激活、免疫失调（Treg 平衡改变）和 B 细胞功能受损，是肺功能异质性的潜在来源。特定微生物的活动会破坏免疫反应的平衡。我们的数据为确定新型标记物和免疫调节靶点奠定了独特的基础，从而为未来的囊性纤维化治疗和管理提供依据。

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引用次数: 0

Concentrations of dehydroepiandrosterone-sulphate (DHEA-S) in people with cystic fibrosis on and off elexacaftor-tezacaftor-ivacaftor 服用和停用 elexacaftor-tezacaftor-ivacaftor 的囊性纤维化患者体内硫酸脱氢表雄酮 (DHEA-S) 的浓度。

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis

Pub Date : 2024-09-01 DOI: 10.1016/j.jcf.2024.04.007

Background

Levels of sulfated Dehydroepiandrosterone (DHEA-S) are unknown in people with Cystic Fibrosis (pwCF). DHEA-S is reported to have an inverse association with inflammation and warrants evaluation in pwCF.

Methods

We compared differences in DHEA-S and other hormones between pwCF (n = 180) and without CF (n = 180) and DHEA-S association with percent predicted forced expiratory volume in one second (ppFEV1). We also evaluated DHEA-S levels in people with CF on elexacaftor-tezacaftor-ivacaftor (ETI) (n = 145).

Results

PwCF (not on ETI) had lower DHEA-S levels compared to healthy non-CF controls. DHEA-S levels in individuals with CF on ETI were similar to those without CF. Lower DHEA-S levels were associated with lower ppFEV1.

Conclusions

PwCF (not on ETI) have lower levels of DHEA-S than people without CF or people with CF on ETI. Additional studies are needed to investigate the impact of DHEA-S on the health of pwCF and mechanisms involved.

背景：囊性纤维化患者 (pwCF) 的硫酸化去氢表雄酮 (DHEA-S) 水平尚不清楚。据报道，DHEA-S 与炎症呈反向关系，因此有必要对囊性纤维化患者进行评估：我们比较了囊性纤维化患者（n = 180）和非囊性纤维化患者（n = 180）之间 DHEA-S 和其他激素的差异，以及 DHEA-S 与一秒内预测用力呼气容积百分比（ppFEV1）的关系。我们还评估了服用 elexacaftor-tezacaftor-ivacaftor (ETI) 的 CF 患者（n = 145）的 DHEA-S 水平：结果：与健康的非 CF 对照组相比，PwCF（未服用 ETI）患者的 DHEA-S 水平较低。服用 ETI 的 CF 患者的 DHEA-S 水平与非 CF 患者相似。较低的 DHEA-S 水平与较低的 ppFEV1 有关：结论：与非 CF 患者或服用 ETI 的 CF 患者相比，PwCF（未服用 ETI）患者的 DHEA-S 水平较低。需要进行更多的研究来探讨 DHEA-S 对慢性阻塞性肺疾病患者健康的影响及其机制。

引用次数: 0

Clinical outcomes of two infants with cystic fibrosis, including presence of the vas deferens, born to a woman with cystic fibrosis taking CFTR modulators during both pregnancies 一名囊性纤维化妇女在两次怀孕期间均服用了 CFTR 调节剂，她所生的两名囊性纤维化婴儿的临床结果（包括输精管的存在）。

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis

Pub Date : 2024-09-01 DOI: 10.1016/j.jcf.2024.06.003

引用次数: 0

Role for DPP4 inhibitor therapy in cystic fibrosis related diabetes: A single centre experience DPP4 抑制剂疗法在囊性纤维化相关糖尿病中的作用：单一中心的经验。

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis

Pub Date : 2024-09-01 DOI: 10.1016/j.jcf.2024.06.007

Introduction

Insulin remains the only recommended medical treatment for cystic fibrosis related diabetes (CFRD) Whilst there is an established role for orally bioavailable incretin mimetic agents such as the dipeptidyl peptidase-4 inhibitors (DPP4-I) in Type 2 diabetes mellitus, there exists little data on their utility in CFRD.

Aim

To examine the use of DPP4-I therapy in patients with CFRD at a single large adult cystic fibrosis center.

Method

People with CFRD prescribed a DPP4-I were identified from our specialist CFRD clinic and records were retrospectively examined for indication for therapy, tolerability and effectiveness. Analysis of continuous glucose monitoring data Libre 2 was done for these patients (CGM) pre and at least 3 months post therapy was performed.

Results

23 people with CF (PwCF) with a mean (SD) age of 35.0 ± 2.4 years were included in this analysis . In 21 patients DPP4-I was prescribed as a monotherapy and it was given in combination with insulin in 2 others. Indications for therapy included reactive hypoglycaemia (n = 10) post prandial hyperglycaemia (8), insulin avoidance (3), metformin intolerance (1) and unclear (1). Therapy was well tolerated with no discontinuations due to adverse effects. Significant improvements were noted in Time in Range- Pre vs Post: 78.0 [67.5 – 84.0] vs 89.0 [79.8 – 96.0]%, p = 0.005, Time above Range -Pre vs Post: 19.5 [12.5 – 30.8] vs 6.0 [2.5 – 16.5]%, p = 0.006 and glucose variability Pre versus Post: 28.3 [25.4 – 31.1] vs 26.9 [23.1 – 31.3], p = 0.021, Of the 10 subjects who initiated therapy for hypoglycaemia, 7 reported an improvement in symptoms. No significant difference was found in weight pre and post: 61.5 ± 15.0 kg vs 62.5 ± 15.3 kg, p = 0.326 or Hba1c pre vs post: 41.0 [36.0 – 53.3] mmol/mol versus 40.5 [36.8 – 47.3], p = 0.727.

Conclusion

DPP4-I is well tolerated in CFRD and can lead to an improved glycaemic control in these patients with significant improvement in validated CGM metrics

简介：虽然二肽基肽酶-4 抑制剂（DPP4-I）等口服生物增量素模拟药物在 2 型糖尿病中的作用已得到确认，但有关其在囊性纤维化相关糖尿病（CFRD）中的作用的数据却很少：方法：从我们的囊性纤维化病专科门诊中找出开具 DPP4-I 治疗处方的囊性纤维化病患者，并对其治疗指征、耐受性和有效性的记录进行回顾性检查。对这些患者治疗前和治疗后至少 3 个月的连续血糖监测数据 Libre 2 进行了分析。结果：23 名平均（标清）年龄为 35.0 ± 2.4 岁的 CF 患者（PwCF）被纳入本次分析。21 名患者接受了 DPP4-I 单药治疗，另有 2 名患者接受了 DPP4-I 与胰岛素联合治疗。治疗指征包括反应性低血糖（10 例）、餐后高血糖（8 例）、避免使用胰岛素（3 例）、二甲双胍不耐受（1 例）和不明确（1 例）。治疗耐受性良好，没有因不良反应而停药。治疗前与治疗后的在量程时间对比有明显改善：78.0 [67.5 - 84.0] vs 89.0 [79.8 - 96.0]%，p = 0.005；超过量程的时间 - 治疗前 vs 治疗后：19.5 [12.5 - 30.8] vs 6.0 [2.5 - 16.5]%，p = 0.006；血糖变异性 - 前 vs 后：28.3 [25.4 - 31.1] vs 26.9 [23.1 - 31.3]，p = 0.021，在 10 名开始接受低血糖治疗的受试者中，有 7 人报告症状有所改善。治疗前后体重无明显差异：61.5±15.0千克 vs 62.5±15.3 千克，p = 0.326 或 Hba1c 前后无明显差异：41.0 [36.0 - 53.3] mmol/mol 对 40.5 [36.8 - 47.3]，p = 0.727：DPP4-I在CFRD患者中耐受性良好，可改善这些患者的血糖控制，并显著改善有效的CGM指标。

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