BREEZE Optional Extension Phase: Long-term safety and efficacy of treprostinil dry powder inhaler (Tyvaso DPI) in pulmonary arterial hypertension.

Introduction: Pulmonary arterial hypertension (PAH) is a rare and progressive disease associated with significant morbidity and mortality. Prostacyclins, including treprostinil, are a mainstay of PAH treatment, particularly in patients with intermediate to high risk of death. Following the approval of treprostinil inhalation solution for PAH, treprostinil dry powder inhaler (DPI) was developed as a small, portable, low-maintenance device to improve patient experience.

Unilateral Absence of Pulmonary Artery Complicated With Severe Pulmonary Hypertension: Favorable Response to Parenteral Therapy.

Background: Unilateral absence of pulmonary artery (UAPA) is a rare disorder that can present as an isolated lesion or in association with other congenital heart defects. An important complication of UAPA is the development of pulmonary hypertension (PH).

Case Summary: We discuss an adult patient with severe PH in the setting of congenital UAPA and highlight the hemodynamic response to combination therapy that included parenteral treprostinil.

A structured approach for identifying disease analogs for pulmonary arterial hypertension.

Drug manufacturers often use disease analogs to describe diseases-particularly rare diseases-to payers, policymakers, and stakeholders. However, these comparisons are typically anecdotal. We propose a 4-step, systematic approach to identify disease analogs based on prespecified metrics and apply it to a rare disease, pulmonary arterial hypertension (PAH), as a case study.

The Potential Impact of Sotatercept on Long-Term Disability in Commercially Insured Patients with Pulmonary Arterial Hypertension in the United States: An Exercise in Actuarial Modeling.

Pulmonary arterial hypertension (PAH) is a progressive disease commonly leading to functional impairment that can impact the ability to work. In the US, disabled workers often qualify for employer-sponsored long-term disability (LTD) benefits and Social Security Disability Insurance (SSDI). We used actuarial techniques to model scenarios of disability costs associated with PAH, with and without treatment with sotatercept, a first-in-class therapy that slowed PAH disease progression in clinical trials.

A real-world comparative effectiveness analysis of macitentan versus ambrisentan and bosentan on hospitalizations and healthcare resource utilization in patients with pulmonary arterial hypertension.

Background: Few studies have evaluated macitentan alongside other endothelin receptor antagonists (ERAs) in patients with pulmonary arterial hypertension (PAH). This retrospective, observational, real-world, comparative effectiveness analysis assessed outcomes in PAH with macitentan versus other ERAs.

Methods: Adults (≥18 years) were included from the de-identified Optum Clinformatics Data Mart database (January 2014-December 2023).

Descriptive Analysis of Oxygen Dependency and Hemodynamic Changes Following Pulmonary Thromboendarterectomy in Chronic Thromboembolic Pulmonary Hypertension Patients.

Background Chronic thromboembolic pulmonary hypertension (CTEPH) is characterized by persistent clot burden and secondary vascular remodeling. Pulmonary thromboendarterectomy (PTE) remains the preferred curative treatment for CTEPH. This study presents a descriptive analysis of surgical outcomes in patients undergoing PTE for CTEPH at a tertiary referral hospital.

Characteristics of sleep disordered breathing in children with achondroplasia.

Purpose: We aimed to evaluate the characteristics of sleep disordered breathing (SDB) in a cohort of pediatric patients with achondroplasia and to describe magnetic resonance imaging (MRI) findings and management strategies.

Methods: A single center retrospective study that included a cohort of pediatric achondroplasia patients and age, gender, and AHI-matched normally developed controls. Medical records and PSGs were reviewed for both cohorts and additionally MRI findings and SDB management strategies were evaluated in the achondroplasia cohort.

Treatment With Oral or Inhaled Treprostinil in Patients With Pulmonary Arterial Hypertension and Cardiovascular Comorbidities.

Background: An increasing number of patients with pulmonary arterial hypertension (PAH) have cardiovascular comorbidities. However, the effects of comorbidities on responses to PAH treatment are not well understood.

Research Question: Do cardiovascular comorbidities in patients with PAH influence the efficacy and tolerability of inhaled or oral treprostinil?

Study Design And Methods: All patients from phase 3 studies Clinical Investigation Into Inhaled Treprostinil Sodium in Patients With Severe Pulmonary Arterial Hypertension (TRIUMPH) (N = 235) and Phase III Clinical Worsening Study of UT-15C in Subjects With PAH Receiving Background Oral Monotherapy (FREEDOM-EV) (N  =  690) were included in this post hoc analysis and were classified as having 0, ≥ 1, or ≥ 2 cardiovascular comorbidities of interest based on patient medical history.

Obstructive sleep apnea in 12 to 24 months old toddlers referred for sleep study in a tertiary care center.

Purpose: To assess OSA prevalence, comorbidities, and the influence of sleep stages and body positions on respiratory events distribution in toddlers aged 12-24 months.

Methods: A single center retrospective study that included toddlers aged 12-24 months old who underwent overnight PSG. OSA severity was categorized by obstructive apnea-hypopnea index (OAHI) as mild (1-4.

Economic Burden Associated with Pulmonary Arterial Hypertension in the United States.

Background: Pulmonary arterial hypertension (PAH) is a progressive disease characterized by elevated pressure in the pulmonary arteries, commonly resulting in right heart failure. PAH is associated with a high economic burden throughout the duration of the disease.

Methods: This retrospective cohort study of the Milliman Contributor Health Source Data, the Medicare 100% Research Identifiable Files, and the Merative Marketscan Commercial dataset between 2018 and 2020 identified adult patients with prevalent PAH based on the earliest qualifying diagnosis date or medication date ('index date') between January 1, 2019 and November 30, 2020.

Adenotonsillectomy outcomes in children with down syndrome and obstructive sleep apnea: a single center study.

Purpose: This study aimed to evaluate polysomnographic (PSG) outcomes of tonsillectomy and adenoidectomy (T&A) in children with Down Syndrome (DS) and OSA, and the difference in PSG outcomes of T&A between children with DS and age- and gender-matched normally developing (non-DS) children.

Methods: This was a single center retrospective study that included children with DS and OSA who underwent T&A and had pre-operative and post-operative PSG. The baseline and the differences of pre- and post-operative PSG variables were compared with those of an age- and gender-matched group of non-DS children.

Pulmonary artery vasodilators for treatment of pulmonary hypertension complicating fibrosing mediastinitis.

Fibrosing mediastinitis (FM) is a heterogeneous disease characterized by sclerosing fibrosis of mediastinal structures. Pulmonary hypertension (PH) may complicate the course of the disease and can contribute significantly to the morbidity of FM. Due to the rarity and complexity of the disease, evidence-based guidelines are not currently available, and the optimal treatment approach is unknown.

Inhaled treprostinil in patients with pulmonary hypertension associated with interstitial lung disease with less severe haemodynamics: a post hoc analysis of the INCREASE study.

Background: Inhaled treprostinil (iTre) is the only treatment approved for pulmonary hypertension due to interstitial lung disease (PH-ILD) to improve exercise capacity. This post hoc analysis evaluated clinical worsening and PH-ILD exacerbations from the 16-week INCREASE study and change in 6-minute walking distance (6MWD) in the INCREASE open-label extension (OLE) in patients with less severe haemodynamics.

Methods: Patients were stratified by baseline pulmonary vascular resistance (PVR) of <4 Wood units (WU) versus ≥4 WU and <5 WU versus ≥5 WU.

Polysomnographic outcomes of revision adenoidectomy in children with obstructive sleep apnea and recurrent/residual adenoidal hypertrophy.

Introduction: Recurrent/residual adenoidal hypertrophy after adenotonsillectomy in children can result in obstructive sleep apnea (OSA). We aimed to assess the polysomnographic (PSG) outcomes of revision adenoidectomy in children with recurrent/residual adenoidal hypertrophy and OSA.

Methods: This was a single-center retrospective study that included children with sleep studies that confirmed OSA and known history of adenotonsillectomy who were diagnosed with adenoidal hypertrophy and subsequently underwent revision adenoidectomy.

A hypothesis: Potential contributions of metals to the pathogenesis of pulmonary artery hypertension.

Pulmonary artery hypertension (PAH) is characterized by vasoconstriction and vascular remodeling resulting in both increased pulmonary vascular resistance (PVR) and pulmonary artery pressure (PAP). The chronic and high-pressure stress experienced by endothelial cells can give rise to inflammation, oxidative stress, and infiltration by immune cells. However, there is no clearly defined mechanism for PAH and available treatment options only provide limited symptomatic relief.

GPCRs and fibroblast heterogeneity in fibroblast-associated diseases.

G protein-coupled receptors (GPCRs) are the largest and most diverse class of signaling receptors. GPCRs regulate many functions in the human body and have earned the title of "most targeted receptors". About one-third of the commercially available drugs for various diseases target the GPCRs.

Metallomics in pulmonary arterial hypertension patients.

Pulmonary arterial hypertension (PAH) prevalence is increasing worldwide, and the prognosis is poor with 5-year survival < 50% in high risk patients. The relationship between metal exposure/essential metal dyshomeostasis and PAH/right ventricular dysfunction is less investigated. The aim of this study is to investigate vegetable consumptions and metal levels between PAH patients and controls.

Role of neck radiography in assessing recurrent/residual adenoid hypertrophy in children with OSA and history of adenotonsillectomy: a sleep physician perspective.

Study Objectives: Recurrent/residual adenoid hypertrophy after adenotonsillectomy in children can result in obstructive sleep apnea (OSA). We aimed to assess the role of soft tissue neck X-ray (STN-XR) in evaluating recurrent/residual adenoid tissue hypertrophy.

Methods: This was a single-center retrospective study that included children with sleep study-confirmed OSA and a known history of adenotonsillectomy who underwent STN-XR to evaluate for recurrent/residual adenoid tissue hypertrophy.