Pulmonary Circulation最新文献

{"title":"Actigraphy methodology in the Kids Mod PAH trial: Physical activity as a functional endpoint in pediatric clinical trials","authors":"Catherine M. Avitabile, Usha S. Krishnan, Delphine Yung, Stephanie S. Handler, Nidhy Varghese, Angela Bates, Jeff Fineman, Rachel Sullivan, Grace Friere, Eric Austin, Mary P. Mullen, Carol Pereira, Eric J. Christensen, Gayane Yenokyan, Joseph M. Collaco, Steven H. Abman, Lew Romer, D. Dunbar Ivy, Erika B. Rosenzweig","doi":"10.1002/pul2.12339","DOIUrl":"https://doi.org/10.1002/pul2.12339","url":null,"abstract":"Pulmonary vasodilator treatment can improve hemodynamics, right ventricular function, symptoms, and survival in pediatric pulmonary hypertension (PH). However, clinical trial data are lacking due to many constraints. One major limitation is the lack of relevant trial endpoints reflective of hemodynamics or functional status in patients in whom standard exercise testing is impractical, unreliable, or not reproducible. The Kids Mod PAH trial (Mono- vs. Duo Therapy for Pediatric Pulmonary Arterial Hypertension) is an ongoing multicenter, Phase III, randomized, open-label, pragmatic trial to compare the safety and efficacy of first-line combination therapy (sildenafil and bosentan) to first-line monotherapy (sildenafil alone) in 100 pediatric patients with PH across North America. Investigators will measure participants’ physical activity with a research-grade, wrist-worn actigraphy device at multiple time points as an exploratory secondary outcome. Vector magnitude counts per minute and activity intensity will be compared between the treatment arms. By directly and noninvasively measuring physical activity in the ambulatory setting, we aim to identify a novel, simple, inexpensive, and highly reproducible approach for quantitative assessment of exercise tolerance in pediatric PH. These data will increase the field's understanding of the effect of pulmonary vasodilator treatment on daily activity – a quantitative measure of functional status and wellbeing in pediatric PH and a potential primary outcome for future clinical trials in children with cardiopulmonary disorders.","PeriodicalId":20927,"journal":{"name":"Pulmonary Circulation","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2024-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140075041","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An inverse causal relationship between serum 25-hydroxyvitamin D levels and pulmonary hypertension: A two-sample Mendelian randomization study.","authors":"Ce Chao, Min Wang, Kun Mei, Chao Ma, Yongxiang Qian, Xiaoying Zhang","doi":"10.1002/pul2.12350","DOIUrl":"10.1002/pul2.12350","url":null,"abstract":"<p><p>Observational studies have confirmed that 25-hydroxyvitamin D (25(OH)D) is associated with pulmonary hypertension (PH), but the causal association between each other is unclear. Therefore, Mendelian randomization (MR) method was performed to validate the causal association between PH and serum 25(OH)D levels. The summary data for 25(OH)D and PH were from the National Human Genome Research Institute-European Bioinformatics Institute. Catalog of human genome-wide association studies and FinnGen biobank consortium. MR analysis was utilized to explore the potential causal association between PH and 25(OH)D. To evaluate this association, inverse variance weighting was considered as the primary method. Cochran's Q test, MR-Egger intercept test, and \"leave-one-out\" sensitivity analyses were utilized to control the pleiotropy and heterogeneity in the study. Two-sample MR analysis revealed an inverse causal relationship between 25(OH)D and PH (odds ratio: 0.376, 95% confidence interval: 0.162-0.876, <i>p</i> = 2.334 × 10^-2). There was no significant heterogeneity and pleiotropy. The present study confirmed the inverse causal relationship between 25(OH)D and PH. This pathway may provide another treatment pathway in PH. Further studies to elucidate this pathway is indicated.</p>","PeriodicalId":20927,"journal":{"name":"Pulmonary Circulation","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2024-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10918714/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140060425","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pulmonary vascular pressure respiratory swings in COPD and ILD candidates for lung transplantation: Large but different","authors":"Juan C. Grignola, Alvaro Calabuig, Pedro Trujillo, Carles Bravo, Fernando Azpiroz, Manuel López Messeguer, Enric Domingo","doi":"10.1002/pul2.12348","DOIUrl":"https://doi.org/10.1002/pul2.12348","url":null,"abstract":"We analyzed the effect of respiratory swings on interpreting intravascular pulmonary vascular pressures (PVPs) in chronic obstructive pulmonary disease (COPD) and interstitial lung disease (ILD) candidates for lung transplantation (LTx) and the role of the alterations in pulmonary function tests on the dynamic respiratory variations. Twenty-eight consecutive patients were included. All patients underwent a complete hemodynamic study (right atrial, mean pulmonary arterial, and pulmonary arterial occlusion pressures [RAP, mPAP, and PAOP]-) and pulmonary function testing (force vital capacity [FVC], forced expiratory volume in the first second [FEV1], and residual volume [RV]). A subgroup of 10 patients underwent simultaneous esophageal pressure (PES). All hemodynamic parameters and PES were collected during apnea after an unforced expiration (ee) and during spontaneous breathing averaging five respiratory cycles (mrc). The respiratory swing (osc) was estimated as the difference between maximum–minimum values of pressures during the respiratory cycle. Intravascular RAPee, mPAPee, and PAOPee were higher than mrc values (<i>p</i> &lt; 0.05), leading to 11% of pulmonary hypertension (PH) misdiagnosis and 37% of postcapillary PH misclassification. PAOPosc of COPD was higher than ILD patients and RAPosc (<i>p</i> &lt; 0.05). Only PAOPosc correlated with FVC, FEV1, and RV (<i>p</i> &lt; 0.05). ILD PESmrc was lower than COPD (<i>p</i> &lt; 0.05), and it was associated with a significantly higher transmural than intravascular RAPmrc, mPAPmrc, and PAOPmrc. PESmrc was significantly correlated with FVC. Transmural mPAPmrc and PAOPmrc readings determined around 20% of reclassification of the patients compared to ee measurements. Candidates for LTx showed large respiratory swings in PVP, which were correlated with pulmonary function alterations. mrc PVP would be more closely approximated to the true transmural PVP leading to PH reclassification. Adjusting PVP for PES should be considered in COPD and ILD candidates of LTx with severe alterations in pulmonary functional tests and suspicion of a PESmrc far from 0. PES respiratory swings could be different in ILD to COPD patients.","PeriodicalId":20927,"journal":{"name":"Pulmonary Circulation","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2024-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140046702","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Simplified risk stratification based on cardiopulmonary exercise test: A Spanish two-center experience","authors":"Amaya Martínez-Meñaca, Alejandro Cruz-Utrilla, Víctor Manuel Mora-Cuesta, Raquel Luna-López, Teresa Segura-de la Cal, Ángela Flox-Camacho, Pilar Alonso-Lecue, Pilar Escribano-Subias, José Manuel Cifrián-Martínez","doi":"10.1002/pul2.12342","DOIUrl":"https://doi.org/10.1002/pul2.12342","url":null,"abstract":"A simplified 4-strata risk stratification approach based on three variables is widespread in pulmonary arterial hypertension (PAH) at follow-up. This study aimed to assess the impact of replacing the 6-min walk test (6MWT) with the peak 0₂ uptake evaluated by the cardiopulmonary exercise test (CPET) on risk stratification by this scale. We included 180 prevalent patients with PAH from two reference hospitals in Spain, followed up between 2006 and 2022. Patients were included if all the variables of interest were available within a 3-month period on the Spanish Registry of Pulmonary Arterial Hypertension (REHAP): functional class (FC); NT-proBNP; 6MWT; and CPET. The original 4-strata model (NT-proBNP, 6MWT, FC) identified most patients at low or intermediate-low risk (36.7% and 51.1%, respectively). Notably, the modified scale (NT-proBNP, CPET, FC) improved the identification of patients at intermediate-high risk up to 18.9%, and at high risk up to 1.1% in comparison with the previous 12.2% and 0.0% in the original scale. This new model increased the number of patients correctly classified into higher-risk strata (positive NRI of 0.06), as well as classified more patients without events in lower-risk strata (negative NRI of 0.04). The proposed score showed a slightly superior prognostic capacity compared with the original model (Harrel's C-index 0.717 vs. 0.709). Using O₂ uptake instead of distance walked in the 6MWT improves the identification of high-risk patients using the 4-strata scale. This change could have relevant prognostic implications and lead to changes in the specific treatment of PAH.","PeriodicalId":20927,"journal":{"name":"Pulmonary Circulation","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2024-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139978512","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Change in health-related quality of life at early follow-up in patients with pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension.","authors":"Bodil Ivarsson, Anders Johansson, Barbro Kjellström","doi":"10.1002/pul2.12349","DOIUrl":"10.1002/pul2.12349","url":null,"abstract":"<p><p>Symptoms associated with pulmonary arterial hypertension (PAH) or chronic thromboembolic pulmonary hypertension (CTEPH) impact patient's health-related quality of life (HRQoL). Studies on change and if a minimal clinically important difference (MCID) in HRQoL is reached within a year after diagnosis are lacking. The aim was to investigate the change in HRQoL as well as the proportion of patients that reached MCID at an early postdiagnosis visit. The study included adult patients from the Swedish PAH & CTEPH registry, diagnosed 2008-2021, with Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) at time of diagnosis and a follow-up. Data were analyzed as total population and dichotomized for sex, age (<65 vs. ≥65 years), time of diagnosis (≤2015 vs. >2015) and pulmonary hypertension (PH) subgroups. Data reported as median, interquartile range (IQR), and proportions (%). There were 151 patients (PAH = 119, CTEPH = 32) with an available CAMPHOR score at diagnosis and follow-up. CAMPHOR total sum was 31 (IQR: 21-43) and 25 (14-36); (<i>p</i> < 0.001) at diagnosis and follow-up, respectively. At follow-up, 56% had reached MCID in total sum, while for domains activity, symptoms, and QoL 27%, 33%, and 39% reached MCID, respectively. These results were independent of PH subgroup, diagnosis before or after 2015 and sex. Age below 65 years was related to improvements in activity and worsening of symptoms. In conclusion on a group level, improvements in CAMPHOR total sum as well as all domains were seen in the first year after diagnosis, however, only slightly more than half of the patients reached MCID for CAMPHOR total sum.</p>","PeriodicalId":20927,"journal":{"name":"Pulmonary Circulation","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2024-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10899573/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139990989","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Recognition, diagnosis, and operability assessment of chronic thromboembolic pulmonary hypertension (CTEPH): A global cross-sectional scientific survey (CLARITY).","authors":"Grzegorz Kopeć, Paul Forfia, Kohtaro Abe, Amélie Beaudet, Virginie Gressin, Mitja Jevnikar, Catherina Meijer, Yan Zhi Tan, Olga Moiseeva, Karen Sheares, Nika Skoro-Sajer, Mario Terra-Filho, Helen Whitford, Zhenguo Zhai, Gustavo A Heresi","doi":"10.1002/pul2.12330","DOIUrl":"10.1002/pul2.12330","url":null,"abstract":"<p><p>Early recognition and diagnosis of chronic thromboembolic pulmonary hypertension (CTEPH) is crucial for improving prognosis and reducing the disease burden. Established clinical practice guidelines describe interventions for the diagnosis and evaluation of CTEPH, yet limited insight remains into clinical practice variation and barriers to care. The CTEPH global cross-sectional scientific survey (CLARITY) was developed to gather insights into the current diagnosis, treatment, and management of CTEPH and to identify unmet medical needs. This paper focuses on the recognition and diagnosis of CTEPH and the referral and evaluation of these patients. The survey was offered to hospital-based medical specialists through Scientific Societies and other medical organizations, from September 2021 to May 2022. Response data from 353 physicians showed that self-reported awareness of CTEPH increased over the past 10 years among 96% of respondents. Clinical practices in acute pulmonary embolism (PE) follow-up and CTEPH diagnosis differed among respondents. While 50% of respondents working in a nonexpert center reported to refer patients to an expert pulmonary hypertension/CTEPH center when CTEPH is suspected, 51% of these physicians did not report referral of patients with a confirmed diagnosis for further evaluation. Up to 50% of respondents involved in the evaluation of referred patients have concluded a different operability status than that indicated by the referring center. This study indicates that early diagnosis and timely treatment of CTEPH is challenged by suboptimal acute PE follow-up and patient referral practices. Nonadherence to guideline recommendations may be impacted by various barriers to care, which were shown to vary by geographical region.</p>","PeriodicalId":20927,"journal":{"name":"Pulmonary Circulation","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2024-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10880430/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139932601","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The beneficial effects of balloon pulmonary angioplasty for patients with chronic thromboembolic pulmonary hypertension are accompanied by increased body mass index and improved nutritional status.","authors":"Shinya Fujii, Shinya Nagayoshi, Takashi Miyamoto, Kazuo Ogawa, Michihiro Yoshimura","doi":"10.1002/pul2.12347","DOIUrl":"10.1002/pul2.12347","url":null,"abstract":"<p><p>Although balloon pulmonary angioplasty (BPA) improves pulmonary hypertension and the prognosis of patients with chronic thromboembolic pulmonary hypertension (CTEPH), subsequent changes in body mass index (BMI), nutritional status, and appetite have not been fully investigated. This retrospective study aimed to clarify changes in BMI, nutritional status, and appetite after BPA. Fifty-two consecutive patients with CTEPH who underwent complete revascularization with BPA between July 2014 and July 2023 and were available for follow-up were evaluated. We compared the presence or absence of increased appetite, BMI change, and nutritional status before and after BPA treatment. BPA significantly improved the mean pulmonary artery pressure from 37.4 ± 8.7 mmHg to 18.7 ± 2.8 mmHg and the partial pressure of oxygen from 61.6 ± 9.3 mmHg to 82.8 ± 9.9 mmHg (both <i>p</i> < 0.001). Appetite was increased in 82.7% of the patients. BMI significantly increased from 23.9 ± 3.7 kg/m² to 24.9 ± 3.7 kg/m² (<i>p</i> < 0.001). As a nutritional indicator, the Geriatric Nutritional Risk Index increased from 105.3 ± 10.2 to 108.3 ± 8.3 (<i>p</i> < 0.001). The median Controlling Nutritional Status scores showed no significant change but the range was significantly improved from 0-9 to 0-3 (<i>p</i> = 0.006). In conclusion, patients with CTEPH show improved oxygenation and hemodynamics, increased BMI, and improved nutritional status following BPA. This sequence of changes may help improve patient prognosis.</p>","PeriodicalId":20927,"journal":{"name":"Pulmonary Circulation","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2024-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10873570/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139900365","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Understanding what drives genetic study participation: Perspectives of patients, carers, and relatives.","authors":"Emilia M Swietlik, Michaela Fay, Nicholas W Morrell","doi":"10.1002/pul2.12346","DOIUrl":"10.1002/pul2.12346","url":null,"abstract":"<p><p>Genetic research's growing importance in understanding pulmonary arterial hypertension (PAH) and developing effective treatments prompted the RAPID-PAH study. This study sought feedback from stakeholders who participated in two genomic studies to enhance genetic study delivery and clinical integration. Stakeholders from nine UK PH centres, representing various roles, ages, genders, and mutation statuses, took part in 53 semi-structured interviews and focus groups. Transcripts were thematically coded using inductive analysis. Clustering analysis was conducted to identify patient groups that shared attitudes. In this paper, we focus on patients', carers', and relatives' perspectives. The key interview themes revealed insights into participants' attitudes toward genetic research and testing more generally, expertise and knowledge of the disease itself, motivations and barriers to participating in genetic research, awareness of and interest in consent procedures and the use of personal and genetic data, as well as the process of communicating individual genetic results. Factors influencing genetic research participation included altruistic motives, personal diagnostic experiences, and family-related hopes. Clustering analysis produced distinct clusters based on the presence of barriers and motivators for research participation; however, hardly any patients shared identical sets of attitudes, emphasising the need for personalised approaches to recruitment. Most patients reported poor engagement with study-related materials. Patients who received individual genetic results expressed satisfaction with the process, whereas those who did not were disappointed with the lack of feedback. Reflecting on patient perspectives, we offer recommendations to improve the genetic study delivery process. Enhancing genetic research integration into clinical practice requires tailored engagement, clear communication, and support from healthcare stakeholders.</p>","PeriodicalId":20927,"journal":{"name":"Pulmonary Circulation","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2024-02-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10867872/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139741876","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pulmonary artery wave intensity analysis in pulmonary hypertension associated with heart failure and reduced left ventricular ejection fraction.","authors":"Ivan H W Yim, Kim H Parker, Nigel E Drury, Hoong Sern Lim","doi":"10.1002/pul2.12345","DOIUrl":"10.1002/pul2.12345","url":null,"abstract":"<p><p>Wave intensity analysis (WIA) uses simultaneous changes in pressure and flow velocity to determine wave energy, type, and timing of traveling waves in the circulation. In this study, we characterized wave propagation in the pulmonary artery in patients with pulmonary hypertension associated with left-sided heart disease (PHLHD) and the effects of dobutamine. During right heart catheterization, pressure and velocity data were acquired using a dual-tipped pressure and Doppler flow sensor wire (Combowire; Phillips Volcano), and processed offline using customized Matlab software (MathWorks). Patients with low cardiac output underwent dobutamine challenge. Twenty patients with PHLHD (all heart failure with reduced left ventricular ejection fraction) were studied. Right ventricular systole produced a forward compression wave (FCW), followed by a forward decompression wave (FDW) during diastole. Wave reflection manifesting as backward compression wave (BCW) following the FCW was observed in 14 patients. Compared to patients without BCW, patients with BCW had higher mean pulmonary artery pressure (28.7 ±  6.12 vs. 38.6 ± 6.5 mmHg, <i>p</i> = 0.005), and lower pulmonary arterial capacitance (PAC: 2.88 ± 1.75 vs. 1.73 ± 1.16, <i>p</i> = 0.002). Pulmonary vascular resistance was comparable. Mean pulmonary artery pressure of 34.5 mmHg (area under the curve [AUC]: 0.881) and PAC of 2.29 mL/mmHg (AUC: 0.833) predicted BCW. The magnitude of the FCW increased with dobutamine (<i>n</i> = 11) and correlated with pulmonary artery wedge pressure. Wave reflection in PHLHD is more likely at higher pulmonary artery pressures and lower PAC and the magnitude of reflected waves correlated with pulmonary artery wedge pressure. Dobutamine increased FCW but did not affect wave reflection.</p>","PeriodicalId":20927,"journal":{"name":"Pulmonary Circulation","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2024-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10859878/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139723885","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Younger age at initiation of subcutaneous treprostinil is associated with better response in pediatric Group 1 pulmonary arterial hypertension.","authors":"Justin J Kochanski, Jeffrey A Feinstein, Michelle Ogawa, Victor Ritter, Rachel K Hopper, Gregory T Adamson","doi":"10.1002/pul2.12328","DOIUrl":"10.1002/pul2.12328","url":null,"abstract":"<p><p>Children with severe Group 1 pulmonary arterial hypertension (PAH) have an unpredictable response to subcutaneous treprostinil (TRE) therapy, which may be influenced by age, disease severity, or other unknown variables at time of initiation. In this retrospective single-center cohort study, we hypothesized that younger age at TRE initiation, early hemodynamic response (a decrease in pulmonary vascular resistance by ≥30% at follow-up catheterization), and less severe baseline hemodynamics (Rp:Rs < 1.1) would each be associated with better clinical outcomes. In 40 pediatric patients with Group I PAH aged 17 days-18 years treated with subcutaneous TRE, younger age (cut-off of 6-years of age, AUC 0.824) at TRE initiation was associated with superior 5-year freedom from adverse events (94% vs. 39%, <i>p</i> = 0.002), better WHO functional class (I or II: 88% vs. 39% <i>p</i> = 0.003), and better echocardiographic indices of right ventricular function at most recent follow-up. Neither early hemodynamic response nor less severe baseline hemodynamics were associated with better outcomes. Patients who did not have a significant early hemodynamic response to TRE by first follow-up catheterization were unlikely to show subsequent improvement in PVRi (1/8, 13%). These findings may help clinicians counsel families and guide clinical decision making regarding the timing of advanced therapies.</p>","PeriodicalId":20927,"journal":{"name":"Pulmonary Circulation","volume":null,"pages":null},"PeriodicalIF":2.6,"publicationDate":"2024-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10860541/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139723887","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}