Pub Date : 2025-01-01Epub Date: 2024-10-23DOI: 10.1002/ppul.27326
Catherine Enochs, Amy G Filbrun, Michelle Hjelm, Julie Lehrmann, Lisa Mullen, Rebeca Packard, Jessica Roach, Anna K Saulitis, Samya Z Nasr
Background: The cystic fibrosis (CF) Responsibility. Independence. Self-care. Education. (R.I.S.E.) program was developed to provide assessment and education, supporting transition readiness for people with cystic fibrosis (pwCF). Lack of integration within electronic health records (EHR) was a barrier to implementation of CF R.I.S.E. University of Michigan was able to integrate CF R.I.S.E. into EHR.
Aim: To improve implementation and EHR documentation of CF R.I.S.E. module completion by pwCF across two (CF) programs from baseline (10.5%) to 75% per month in 6 months (January through June 2023).
Methods: Two CF programs utilized quality improvement (QI) methods and tools and ad hoc support by a CF Learning Network (QI) specialist. Eligibility included pwCF ≥16 years old seen in CF clinics who accepted CF R.I.S.E.
Participation: Beginning January 2, 2023, programs met in biweekly, virtual meetings to discuss implementation. Deidentified data were collected monthly tracking modules completed by pwCF and number of team members engaging with CF R.I.S.E. and documenting in EHR. Data timelines were baseline (November-December 2022), project period (January-June 2023), and post-project (July-December 2023).
Results: Completion rates increased from baseline (10.5%) to 48% (range 33% to 81%) through December 2023. During the project, an average 7.7 team members completed an average 19.2 modules per month. Post-project, an average 8 team members completed an average 16.5 modules per month.
Conclusions: This collaboration demonstrated how utilization of EHR allowed for successful CF R.I.S.E. improvement at both programs. Shared software utilization and QI initiatives may be a way to facilitate timely dissemination of best practices through learning health systems.
{"title":"Improving implementation and team communication by integrating a cystic fibrosis transition readiness (CF R.I.S.E.) program into electronic health records.","authors":"Catherine Enochs, Amy G Filbrun, Michelle Hjelm, Julie Lehrmann, Lisa Mullen, Rebeca Packard, Jessica Roach, Anna K Saulitis, Samya Z Nasr","doi":"10.1002/ppul.27326","DOIUrl":"10.1002/ppul.27326","url":null,"abstract":"<p><strong>Background: </strong>The cystic fibrosis (CF) Responsibility. Independence. Self-care. Education. (R.I.S.E.) program was developed to provide assessment and education, supporting transition readiness for people with cystic fibrosis (pwCF). Lack of integration within electronic health records (EHR) was a barrier to implementation of CF R.I.S.E. University of Michigan was able to integrate CF R.I.S.E. into EHR.</p><p><strong>Aim: </strong>To improve implementation and EHR documentation of CF R.I.S.E. module completion by pwCF across two (CF) programs from baseline (10.5%) to 75% per month in 6 months (January through June 2023).</p><p><strong>Methods: </strong>Two CF programs utilized quality improvement (QI) methods and tools and ad hoc support by a CF Learning Network (QI) specialist. Eligibility included pwCF ≥16 years old seen in CF clinics who accepted CF R.I.S.E.</p><p><strong>Participation: </strong>Beginning January 2, 2023, programs met in biweekly, virtual meetings to discuss implementation. Deidentified data were collected monthly tracking modules completed by pwCF and number of team members engaging with CF R.I.S.E. and documenting in EHR. Data timelines were baseline (November-December 2022), project period (January-June 2023), and post-project (July-December 2023).</p><p><strong>Results: </strong>Completion rates increased from baseline (10.5%) to 48% (range 33% to 81%) through December 2023. During the project, an average 7.7 team members completed an average 19.2 modules per month. Post-project, an average 8 team members completed an average 16.5 modules per month.</p><p><strong>Conclusions: </strong>This collaboration demonstrated how utilization of EHR allowed for successful CF R.I.S.E. improvement at both programs. Shared software utilization and QI initiatives may be a way to facilitate timely dissemination of best practices through learning health systems.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":" ","pages":"e27326"},"PeriodicalIF":2.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11733705/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142505722","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01Epub Date: 2024-11-06DOI: 10.1002/ppul.27382
Lena Papadakis, Tayler Stander, Jacqueline Mombourquette, Christopher J Richards, Lael M Yonker, Brenden Lawton, Margot Hardcastle, Julia Zweifach, Leonard Sicilian, Lindsay Bringhurst, Isabel P Neuringer
Background: The benefits of Elexecaftor-Tezacaftor-Ivacaftor (ETI) therapy on the health and wellbeing of people with CF (pwCF) are well documented. Since approval, however, a growing number of potential side effects have emerged in reports from clinical practice. With current understanding of ETI tolerability limited to data from clinical trials, the prevalence of side effects and their impact on care decision making remains poorly categorized.
Methods: A 10-question survey was developed and distributed to patients 18 years or older who were treated at the Massachusetts General Hospital CF centers. Reports of side effects were measured across 12 distinct categories, and dose adjustments and discontinuation due to side effects were collected. If a patient reported no side effects, they did not have to complete the entire survey.
Results: Among 92 respondents initiated on ETI, 51 respondents (55.4%) reported potential side effects and 41 (44.5%) respondents reported no adverse events. The most commonly reported side effects were mental health, changes in appearance, and gastrointestinal complaints, which were reported by 22.8%, 30.4%, and 21.7% of patients, respectively. Eighteen (19.6%) respondents modified their dosing in response to side effects, and six discontinued treatment permanently (6.52%) due to persistent side effects.
Conclusions: Responses demonstrated marked heterogeneity, with most respondents reporting at least one side effect following initiation. Dose modification was commonly utilized to mitigate adverse effects, however few respondents had to discontinue treatment. These findings demonstrate the importance of monitoring for potential drug-related side effects of ETI in clinical settings.
背景:Elexecaftor-Tezacaftor-Ivacaftor(ETI)疗法对 CF 患者(pwCF)的健康和福祉的益处有据可查。然而,自获得批准以来,临床实践报告中出现了越来越多的潜在副作用。由于目前对 ETI 耐受性的了解仅限于临床试验数据,因此副作用的发生率及其对护理决策的影响仍未得到很好的分类:方法:我们编制了一份包含 10 个问题的调查问卷,并分发给在麻省总医院 CF 中心接受治疗的 18 岁及以上患者。对 12 个不同类别的副作用报告进行了测量,并收集了因副作用导致的剂量调整和停药情况。如果患者报告没有副作用,则无需完成整个调查:在 92 名开始使用 ETI 的受访者中,51 名受访者(55.4%)报告了潜在的副作用,41 名受访者(44.5%)报告未出现不良事件。最常报告的副作用是精神健康、外观变化和胃肠道不适,分别有 22.8%、30.4% 和 21.7% 的患者报告了这些副作用。18名受访者(19.6%)因副作用而改变了用药剂量,6名受访者(6.52%)因持续副作用而永久停止治疗:结论:受访者的回答具有明显的异质性,大多数受访者在开始治疗后都报告了至少一种副作用。为减轻不良反应,通常会调整剂量,但很少有受访者不得不中断治疗。这些发现表明,在临床环境中监测 ETI 潜在的药物相关副作用非常重要。
{"title":"Heterogeneity in Reported Side Effects Following Initiation of Elexacaftor-Tezacaftor-Ivacaftor: Experiences at a Quaternary CF Care Center.","authors":"Lena Papadakis, Tayler Stander, Jacqueline Mombourquette, Christopher J Richards, Lael M Yonker, Brenden Lawton, Margot Hardcastle, Julia Zweifach, Leonard Sicilian, Lindsay Bringhurst, Isabel P Neuringer","doi":"10.1002/ppul.27382","DOIUrl":"10.1002/ppul.27382","url":null,"abstract":"<p><strong>Background: </strong>The benefits of Elexecaftor-Tezacaftor-Ivacaftor (ETI) therapy on the health and wellbeing of people with CF (pwCF) are well documented. Since approval, however, a growing number of potential side effects have emerged in reports from clinical practice. With current understanding of ETI tolerability limited to data from clinical trials, the prevalence of side effects and their impact on care decision making remains poorly categorized.</p><p><strong>Methods: </strong>A 10-question survey was developed and distributed to patients 18 years or older who were treated at the Massachusetts General Hospital CF centers. Reports of side effects were measured across 12 distinct categories, and dose adjustments and discontinuation due to side effects were collected. If a patient reported no side effects, they did not have to complete the entire survey.</p><p><strong>Results: </strong>Among 92 respondents initiated on ETI, 51 respondents (55.4%) reported potential side effects and 41 (44.5%) respondents reported no adverse events. The most commonly reported side effects were mental health, changes in appearance, and gastrointestinal complaints, which were reported by 22.8%, 30.4%, and 21.7% of patients, respectively. Eighteen (19.6%) respondents modified their dosing in response to side effects, and six discontinued treatment permanently (6.52%) due to persistent side effects.</p><p><strong>Conclusions: </strong>Responses demonstrated marked heterogeneity, with most respondents reporting at least one side effect following initiation. Dose modification was commonly utilized to mitigate adverse effects, however few respondents had to discontinue treatment. These findings demonstrate the importance of monitoring for potential drug-related side effects of ETI in clinical settings.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":" ","pages":"e27382"},"PeriodicalIF":2.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583970","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01Epub Date: 2024-11-08DOI: 10.1002/ppul.27395
Pratyaksha Rana, Saurabh Deshpande, Megha M Sheth, Amit Mishra, Hit B Jivani, T M Harishkar, Dinesh Patel
{"title":"Large Distal Aorto-Pulmonary Tunnel: Hitherto Unreported Type of Aortopulmonary Connection.","authors":"Pratyaksha Rana, Saurabh Deshpande, Megha M Sheth, Amit Mishra, Hit B Jivani, T M Harishkar, Dinesh Patel","doi":"10.1002/ppul.27395","DOIUrl":"10.1002/ppul.27395","url":null,"abstract":"","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":" ","pages":"e27395"},"PeriodicalIF":2.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142605736","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01Epub Date: 2024-09-25DOI: 10.1002/ppul.27289
Phuc Huu Phan, Hanh My Thi Tran, Canh Ngoc Hoang, Thang Van Nguyen, Bin Huey Quek, Jan Hau Lee
Introduction: This study aimed to describe the epidemiology and etiologies of critical respiratory diseases of ex-premature infants (EPIs) admitted to the Pediatric Intensive Care unit (PICU).
Methods: Infants ≤2 years old with acute respiratory illnesses admitted to PICU of Vietnam National Children's Hospital from November 2019 to April 2021 were enrolled and followed up to hospital discharge. We compared respiratory pathogens, outcomes, and PICU resources utilized between EPIs and term infants. Among EPIs, we described clinical characteristics and evaluated the association between associated factors and mortality.
Results: Among 1183 patients, aged ≤2 years were admitted for critical respiratory illnesses, 202 (17.1%) were EPIs. Respiratory viruses were detected in 53.5% and 38.2% among EPIs and term infants, respectively. Compared to term infants, a higher proportion of EPIs required mechanical ventilation (MV) (85.6 vs. 66.5%, p < .005) and vasopressor support (37.6 vs. 10.7%%, p < .005). EPIs had a higher median PICU length of stay (11.0 [IQR: 7; 22] vs. 6.0 days [IQR: 3; 11], p = .09), hospital length of stay (21.5 [IQR: 13; 40] vs. 10.0 days [IQR: 5; 18], p < .005) and case fatality rate (31.3% vs. 22.6%) compared to term infants. Among EPIs, PIM-3 score (adjusted odds ratio [aOR]: 1.51; 95% confidence interval [CI]: 1.30-1.75) and PELOD-2 score at admission (aOR: 1.41; 95% CI: 1.08-1.85) were associated with mortality.
Conclusions: EPIs with critical respiratory illnesses constituted a significant population in the PICU, required more PICU support, and had worse clinical outcomes compared to term infants.
{"title":"The epidemiology of critical respiratory diseases in ex-premature infants in Vietnam: A prospective single-center study.","authors":"Phuc Huu Phan, Hanh My Thi Tran, Canh Ngoc Hoang, Thang Van Nguyen, Bin Huey Quek, Jan Hau Lee","doi":"10.1002/ppul.27289","DOIUrl":"10.1002/ppul.27289","url":null,"abstract":"<p><strong>Introduction: </strong>This study aimed to describe the epidemiology and etiologies of critical respiratory diseases of ex-premature infants (EPIs) admitted to the Pediatric Intensive Care unit (PICU).</p><p><strong>Methods: </strong>Infants ≤2 years old with acute respiratory illnesses admitted to PICU of Vietnam National Children's Hospital from November 2019 to April 2021 were enrolled and followed up to hospital discharge. We compared respiratory pathogens, outcomes, and PICU resources utilized between EPIs and term infants. Among EPIs, we described clinical characteristics and evaluated the association between associated factors and mortality.</p><p><strong>Results: </strong>Among 1183 patients, aged ≤2 years were admitted for critical respiratory illnesses, 202 (17.1%) were EPIs. Respiratory viruses were detected in 53.5% and 38.2% among EPIs and term infants, respectively. Compared to term infants, a higher proportion of EPIs required mechanical ventilation (MV) (85.6 vs. 66.5%, p < .005) and vasopressor support (37.6 vs. 10.7%%, p < .005). EPIs had a higher median PICU length of stay (11.0 [IQR: 7; 22] vs. 6.0 days [IQR: 3; 11], p = .09), hospital length of stay (21.5 [IQR: 13; 40] vs. 10.0 days [IQR: 5; 18], p < .005) and case fatality rate (31.3% vs. 22.6%) compared to term infants. Among EPIs, PIM-3 score (adjusted odds ratio [aOR]: 1.51; 95% confidence interval [CI]: 1.30-1.75) and PELOD-2 score at admission (aOR: 1.41; 95% CI: 1.08-1.85) were associated with mortality.</p><p><strong>Conclusions: </strong>EPIs with critical respiratory illnesses constituted a significant population in the PICU, required more PICU support, and had worse clinical outcomes compared to term infants.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":" ","pages":"e27289"},"PeriodicalIF":2.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142351783","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Background and aims: The long-term respiratory sequelae of COVID-19 infection in children remain poorly understood and may differ across countries. This study aims to investigate the respiratory sequelae, including residual respiratory symptoms and pulmonary function in Thai children. The secondary aim is to identify factors associated with the respiratory sequelae.
Materials and methods: This is an observational study involving 56 healthy children, aged between 7 and 18 years, who were diagnosed with COVID-19 infection from July 2021 to February 2023. Clinical data relating to COVID-19 infection and persistent symptoms after the infection were assessed after the infection up to 6 months. Spirometry was performed to assess pulmonary function.
Results: Post-COVID-19 symptoms were identified in 14 patients (25%), with fatigue, cough, and dyspnea being common symptoms (28%-35%). A significant correlation was found between post COVID-19 symptoms and pneumonia (OR = 6.00, 95%CI [1.54,23.33], p = .01). Abnormal pulmonary function was identified in 10 patients (17.8%) with obstructive impairment being the most common. However, there was no significant association between clinical factors and pulmonary function impairment.
Conclusion: Prolonged respiratory symptoms and abnormal pulmonary function following COVID-19 infection are not uncommon in children. The post-COVID-19 symptoms are possibly associated with COVID-19 pneumonia.
{"title":"Respiratory sequelae after COVID-19 infection in Thai healthy children.","authors":"Sirapoom Niamsanit, Wicharn Boonjindasup, Suchada Sritippayawan, Jitladda Deerojanawong, Nuanchan Prapphal, Chanthana Harnruthakorn, Jiratchaya Sophonphan, Watit Niyomkarn","doi":"10.1002/ppul.27329","DOIUrl":"10.1002/ppul.27329","url":null,"abstract":"<p><strong>Background and aims: </strong>The long-term respiratory sequelae of COVID-19 infection in children remain poorly understood and may differ across countries. This study aims to investigate the respiratory sequelae, including residual respiratory symptoms and pulmonary function in Thai children. The secondary aim is to identify factors associated with the respiratory sequelae.</p><p><strong>Materials and methods: </strong>This is an observational study involving 56 healthy children, aged between 7 and 18 years, who were diagnosed with COVID-19 infection from July 2021 to February 2023. Clinical data relating to COVID-19 infection and persistent symptoms after the infection were assessed after the infection up to 6 months. Spirometry was performed to assess pulmonary function.</p><p><strong>Results: </strong>Post-COVID-19 symptoms were identified in 14 patients (25%), with fatigue, cough, and dyspnea being common symptoms (28%-35%). A significant correlation was found between post COVID-19 symptoms and pneumonia (OR = 6.00, 95%CI [1.54,23.33], p = .01). Abnormal pulmonary function was identified in 10 patients (17.8%) with obstructive impairment being the most common. However, there was no significant association between clinical factors and pulmonary function impairment.</p><p><strong>Conclusion: </strong>Prolonged respiratory symptoms and abnormal pulmonary function following COVID-19 infection are not uncommon in children. The post-COVID-19 symptoms are possibly associated with COVID-19 pneumonia.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":" ","pages":"e27329"},"PeriodicalIF":2.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472202","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01Epub Date: 2024-10-25DOI: 10.1002/ppul.27295
Yen Hoang Do, Wim van Aalderen, Ellen Dellbrügger, Claude Grenzbach, Jonathan Grigg, Ulrike Grittner, Eric Haarman, Camilo José Hernandez Toro, Bulent Karadag, Siri Roßberg, Tina-Maria Weichert, Abigail Whitehouse, Antonio Pizzulli, Stephanie Dramburg, Paolo Maria Matricardi
<p><strong>Introduction: </strong>Wheezing is an important indicator of exacerbated respiratory symptoms in early childhood and must be monitored to regulate pharmacological therapy. However, parents' subjective perception of wheezing in their children is not always precise. We investigated the objective identification of children's wheezing by parents using a digital wheeze detector (WheezeScan<sup>TM</sup>, OMRON Healthcare Co. Ltd), its longitudinal usage patterns, and its relationship with SABA administration.</p><p><strong>Methods: </strong>We conducted a secondary nested analysis of data from the intervention arm of a multi-center randomized controlled trial completed in 2021-2022 in Berlin (Germany), London (United Kingdom), and Istanbul (Turkey). Children aged 4 to 84 months with doctor's diagnosed wheezing (GINA step 1 or 2) were included. Using an electronic diary (Wheeze-Monitor<sup>TM</sup>, TPS), parents monitored and recorded for 120 days at home the presence or absence of their child's wheezing, detected both, with WheezeScan<sup>TM</sup> ("objective" wheezing), and subjective ("perceived" wheezing). Parents also recorded the child's symptoms, medication intake, and family quality of life. Questionnaires regarding symptom control, quality of life, and parental self-efficacy were answered at baseline and after 90 and 120 days.</p><p><strong>Results: </strong>Eighty-one/87 families completed the intervention arm of the study. WheezeScan<sup>TM</sup> was on average used 0.7 (SD 0.6) times a day, with each patient reporting a positive, negative, or "error" outcome on average in 57%, 39%, and 5% of measurements, respectively. The use of WheezeScan<sup>TM</sup> declined slightly during the first 90 days of monitoring and steeply thereafter. Repeated usage of WheezeScan<sup>TM</sup> in the same day was more frequent after a "wheeze" (HR 1.5, 95% CI 1.37-1.65, p < 0.001) and an "error" (HR 2.01, 95% CI 1.70-2.38, p < 0.001) result, compared to a "no wheeze" outcome. The average per-patient daily agreement between "objective" and "perceived" wheezing/non-wheezing was 75% at the start of the monitoring period and only weakly persisted as time passed (Spearman's rho=0.09). The frequency of short-acting beta-2-agonists (SABA) administration was lower in days with closely interspaced consecutive device uses during which the patient's status was perceived as "never wheeze" (32/455, 7%) than in those perceived as "persistent wheeze" (53/119, 44%; OR 36.6, 95% CI [14.3, 94.1]).</p><p><strong>Conclusion: </strong>Daily use of a digital WheezeScan<sup>TM</sup> at home allows parents to detect their child's unperceived wheezing and discloses to caregivers the longitudinal patterns of a child's wheezing disorder. Digital monitoring of wheezing also highlights poor adherence to guidelines in SABA administration for wheezing children, with under-treatment being much more frequent than over-treatment. This pioneering study opens new perspectives for furt
{"title":"Objective detection of wheeze at home by parents through a digital device: usage patterns and relationship with SABA administration.","authors":"Yen Hoang Do, Wim van Aalderen, Ellen Dellbrügger, Claude Grenzbach, Jonathan Grigg, Ulrike Grittner, Eric Haarman, Camilo José Hernandez Toro, Bulent Karadag, Siri Roßberg, Tina-Maria Weichert, Abigail Whitehouse, Antonio Pizzulli, Stephanie Dramburg, Paolo Maria Matricardi","doi":"10.1002/ppul.27295","DOIUrl":"10.1002/ppul.27295","url":null,"abstract":"<p><strong>Introduction: </strong>Wheezing is an important indicator of exacerbated respiratory symptoms in early childhood and must be monitored to regulate pharmacological therapy. However, parents' subjective perception of wheezing in their children is not always precise. We investigated the objective identification of children's wheezing by parents using a digital wheeze detector (WheezeScan<sup>TM</sup>, OMRON Healthcare Co. Ltd), its longitudinal usage patterns, and its relationship with SABA administration.</p><p><strong>Methods: </strong>We conducted a secondary nested analysis of data from the intervention arm of a multi-center randomized controlled trial completed in 2021-2022 in Berlin (Germany), London (United Kingdom), and Istanbul (Turkey). Children aged 4 to 84 months with doctor's diagnosed wheezing (GINA step 1 or 2) were included. Using an electronic diary (Wheeze-Monitor<sup>TM</sup>, TPS), parents monitored and recorded for 120 days at home the presence or absence of their child's wheezing, detected both, with WheezeScan<sup>TM</sup> (\"objective\" wheezing), and subjective (\"perceived\" wheezing). Parents also recorded the child's symptoms, medication intake, and family quality of life. Questionnaires regarding symptom control, quality of life, and parental self-efficacy were answered at baseline and after 90 and 120 days.</p><p><strong>Results: </strong>Eighty-one/87 families completed the intervention arm of the study. WheezeScan<sup>TM</sup> was on average used 0.7 (SD 0.6) times a day, with each patient reporting a positive, negative, or \"error\" outcome on average in 57%, 39%, and 5% of measurements, respectively. The use of WheezeScan<sup>TM</sup> declined slightly during the first 90 days of monitoring and steeply thereafter. Repeated usage of WheezeScan<sup>TM</sup> in the same day was more frequent after a \"wheeze\" (HR 1.5, 95% CI 1.37-1.65, p < 0.001) and an \"error\" (HR 2.01, 95% CI 1.70-2.38, p < 0.001) result, compared to a \"no wheeze\" outcome. The average per-patient daily agreement between \"objective\" and \"perceived\" wheezing/non-wheezing was 75% at the start of the monitoring period and only weakly persisted as time passed (Spearman's rho=0.09). The frequency of short-acting beta-2-agonists (SABA) administration was lower in days with closely interspaced consecutive device uses during which the patient's status was perceived as \"never wheeze\" (32/455, 7%) than in those perceived as \"persistent wheeze\" (53/119, 44%; OR 36.6, 95% CI [14.3, 94.1]).</p><p><strong>Conclusion: </strong>Daily use of a digital WheezeScan<sup>TM</sup> at home allows parents to detect their child's unperceived wheezing and discloses to caregivers the longitudinal patterns of a child's wheezing disorder. Digital monitoring of wheezing also highlights poor adherence to guidelines in SABA administration for wheezing children, with under-treatment being much more frequent than over-treatment. This pioneering study opens new perspectives for furt","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":" ","pages":"e27295"},"PeriodicalIF":2.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11715141/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142505727","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
M Guedes, D Frankel, S A Baron, J Berbis, N Stremler-Le Bel, M Baravalle-Einaudi, P Roll, E Kaspi, J C Dubus
Introduction: Cellular characteristics of induced sputum (IS) are not investigated in cystic fibrosis (CF) patients.
Objectives: This pilot study, conducted on 17 expectorating CF adolescents, compared sputa obtained the same day, in a stable period, by autogenic drainage (expectorating sputum, ES) and 4 h later after inhaling hypertonic saline (IS).
Results: No difference was noted concerning weight, volume, and percentage of dead cells between the two collection methods. Sample quality (< 50% of dead cells and < 20% squamous cells) was higher in the case of IS than ES (94.1% vs. 58.8%, p = 0.03), with a doubled cell count (p = 0.01), a higher proportion of alveolar macrophages (p = 0.03), and a lower proportion of squamous cells (p = 0.004). The detection of germs increased by 44% in IS samples, possibly modifying therapeutic management in 17.6% of the patients.
Conclusion: IS improves the quality and the microbiological detection of the sample, even among CF patients who spontaneously expectorate.
{"title":"Induced Sputum: A Valuable Tool for Assessing Cellular and Microbiological Characteristics in Cystic Fibrosis Expectorating Teenagers.","authors":"M Guedes, D Frankel, S A Baron, J Berbis, N Stremler-Le Bel, M Baravalle-Einaudi, P Roll, E Kaspi, J C Dubus","doi":"10.1002/ppul.27488","DOIUrl":"https://doi.org/10.1002/ppul.27488","url":null,"abstract":"<p><strong>Introduction: </strong>Cellular characteristics of induced sputum (IS) are not investigated in cystic fibrosis (CF) patients.</p><p><strong>Objectives: </strong>This pilot study, conducted on 17 expectorating CF adolescents, compared sputa obtained the same day, in a stable period, by autogenic drainage (expectorating sputum, ES) and 4 h later after inhaling hypertonic saline (IS).</p><p><strong>Results: </strong>No difference was noted concerning weight, volume, and percentage of dead cells between the two collection methods. Sample quality (< 50% of dead cells and < 20% squamous cells) was higher in the case of IS than ES (94.1% vs. 58.8%, p = 0.03), with a doubled cell count (p = 0.01), a higher proportion of alveolar macrophages (p = 0.03), and a lower proportion of squamous cells (p = 0.004). The detection of germs increased by 44% in IS samples, possibly modifying therapeutic management in 17.6% of the patients.</p><p><strong>Conclusion: </strong>IS improves the quality and the microbiological detection of the sample, even among CF patients who spontaneously expectorate.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 1","pages":"e27488"},"PeriodicalIF":2.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009725","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Anaïs Le, Thomas Perrin, David Drummond, Philippe Reix, Brigitte Bader-Meunier, Gillian I Rice, Darragh Duffy, Marie-Louise Frémond, Alice Hadchouel
{"title":"Efficacy of Ruxolitinib in Severe Pediatric Intra-Alveolar Hemorrhages Unrelated to COPA Mutations.","authors":"Anaïs Le, Thomas Perrin, David Drummond, Philippe Reix, Brigitte Bader-Meunier, Gillian I Rice, Darragh Duffy, Marie-Louise Frémond, Alice Hadchouel","doi":"10.1002/ppul.27481","DOIUrl":"10.1002/ppul.27481","url":null,"abstract":"","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 1","pages":"e27481"},"PeriodicalIF":2.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11734376/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142984393","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01Epub Date: 2024-12-31DOI: 10.1002/ppul.27466
Carlos E Rodríguez-Martínez, Monica P Sossa-Briceño, Jefferson Antonio Buendia
Objectives: Since 2019 as-needed low-dose ICS-formoterol in a single inhaler has been recommended for treatment of mild asthma in children aged more than 12 years. Alternatively, the use of ICS-albuterol has been proposed in countries where ICS-formoterol is not available or affordable. The aim of the present study was to evaluate the cost-utility of as-needed ICS-albuterol in separate inhalers compared to ICS-formoterol in a single inhaler as reliever therapy in pediatric patients with mild asthma living in Colombia.
Methods: A Markov-type model was developed to estimate the costs and health outcomes of a simulated cohort of pediatric patients with mild asthma treated for 12 months. The effectiveness data and transition probabilities were obtained from relevant randomized clinical trials (RCTs). Cost data were obtained from official databases provided by the Colombian Ministry of Health. The main outcome was the variable "quality-adjusted life-years" (QALYs).
Results: The base-case analysis showed that compared with the use of as-needed ICS-albuterol in separate inhalers, the use of ICS-formoterol in a single in pediatric patients with mild asthma was associated with lower costs (US$475.51 vs. 735.33 average cost per patient) and the greatest gain in QALYs (0.9367 vs. 0.9352 QALYs on average per patient), thus leading to dominance.
Conclusions: Compared with the use of as-needed ICS-albuterol in separate inhalers, the use of ICS-formoterol in a single inhaler as reliever therapy is cost-effective in patients aged 12 years or more with mild asthma, because it showed a greater gain in QALYs at lower total treatment costs.
{"title":"As-Needed Inhaled Corticosteroid-Formoterol in a Single Inhaler Compared to Inhaled Corticosteroid-Albuterol in Separate Inhalers as Reliever Therapy in Mild Pediatric Asthma: A Cost-Utility Analysis.","authors":"Carlos E Rodríguez-Martínez, Monica P Sossa-Briceño, Jefferson Antonio Buendia","doi":"10.1002/ppul.27466","DOIUrl":"10.1002/ppul.27466","url":null,"abstract":"<p><strong>Objectives: </strong>Since 2019 as-needed low-dose ICS-formoterol in a single inhaler has been recommended for treatment of mild asthma in children aged more than 12 years. Alternatively, the use of ICS-albuterol has been proposed in countries where ICS-formoterol is not available or affordable. The aim of the present study was to evaluate the cost-utility of as-needed ICS-albuterol in separate inhalers compared to ICS-formoterol in a single inhaler as reliever therapy in pediatric patients with mild asthma living in Colombia.</p><p><strong>Methods: </strong>A Markov-type model was developed to estimate the costs and health outcomes of a simulated cohort of pediatric patients with mild asthma treated for 12 months. The effectiveness data and transition probabilities were obtained from relevant randomized clinical trials (RCTs). Cost data were obtained from official databases provided by the Colombian Ministry of Health. The main outcome was the variable \"quality-adjusted life-years\" (QALYs).</p><p><strong>Results: </strong>The base-case analysis showed that compared with the use of as-needed ICS-albuterol in separate inhalers, the use of ICS-formoterol in a single in pediatric patients with mild asthma was associated with lower costs (US$475.51 vs. 735.33 average cost per patient) and the greatest gain in QALYs (0.9367 vs. 0.9352 QALYs on average per patient), thus leading to dominance.</p><p><strong>Conclusions: </strong>Compared with the use of as-needed ICS-albuterol in separate inhalers, the use of ICS-formoterol in a single inhaler as reliever therapy is cost-effective in patients aged 12 years or more with mild asthma, because it showed a greater gain in QALYs at lower total treatment costs.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":" ","pages":"e27466"},"PeriodicalIF":2.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142909938","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}