Jaime Rosenberg

Following the course of lung function in treatment-naïve patients, researchers found that lung function decline does not accelerate prior to mechanical ventilation.

The measure will allow for a more complete picture of treatment burden and how it affects things like health-related quality of life and adherence, explained researchers.

Right ventricular (RV) dysfunction has previously been linked to symptoms and mortality in pulmonary hypertension (PH), as well as in other conditions, and RV function plays an important role in disease risk and potentially in intensification of pulmonary arterial hypertension treatment.

The Chinese report details the case of a 6-year-old female patient with type 1 SMA who was successfully weaned off prolonged invasive ventilation after beginning treatment with nusinersen.

Prior to this study, explained researchers, data on the long-term course of osteoporosis, as evaluated through bone mineral density (BMD) in these patients, have been lacking.

Using a clustering algorithm, researchers were able to identify inflammatory and noninflammatory subsets of the disease, which may account for varying responses to certain treatments.

The researchers noted that their study is the first to assess the ability of noninvasive variables, both resting- and exercise-based, to identify pulmonary hypertension in interstitial lung disease.

A group of researchers detailed a case of acquired hemophilia A (AHA) following infection and again following vaccination, and provided additional insights through their systematic review of available data.

These new findings suggest that variability in factor VIII (FVIII) half-life in patients, though impacted by all 3 factors, is largely attributed to variability in the clearance of endogenous von Willebrand factor (VWF).

Researchers retrospectively analyzed data from patients who visited 2 myeloma centers between March and October 2020, before vaccines were widely available but during large-scale implementation of regular screening processes.

The researchers used Paraphase on more than 400 samples of spinal muscular atrophy (SMA) comprising 5 ethnicities.

Researchers of the multicenter study looked at the effect of nusinersen among 48 patients with spinal muscular atrophy (SMA) from 2 Italian hospitals, finding more active immune systems among the patients with SMA1, the most severe form of the disorder.

Idecabtagene vicleucel (ide-cel) reduced the risk of disease progression or death by 51% in patients with triple-class–exposed relapsed or refractory multiple myeloma (R/R MM).

The approach for cardiovascular disease (CVD) was based on expert opinion, personal experience, and adaptation of evidence from people who did not have hemophilia.

Study findings offer insight into how exercise intolerance varies among subgroups of pulmonary arterial hypertension associated with congenital heart disease (PAH-CHD), as previous data have characterized impaired exercise tolerance among PAH-CHD as a whole.

Having a diagnostic model that leverages artificial intelligence could fill a gap in the pulmonary hypertension (PH) diagnostic pathway, as there are currently just 2 validated screening algorithms for PH.

Researchers found that the anticoagulant effects of apixaban, betrixaban, edoxaban, and rivaroxaban were partially neutralized at an andexanet alfa dose of 50 mcg/mL and were completely neutralized at 100 mcg/mL.

The second half of 2022 brought the first gene therapies for hemophilia to the market in both the United States and abroad.

Investigators note that although the overall cost for implementing next-generation sequencing (NGS) testing would be more than implementing current polymerase chain reaction (PCR)–based testing, the assay may be more cost effective overall because it screens for multiple conditions.

The data showed that most doses of nusinersen were received on time while also underscoring the importance of being meticulous with the methodological approaches used with real-world databases for evaluating treatment patterns.

Throughout this study's 12-year period, the average annual percent change showed an uptrend for all groups in hospitalization for spinal cord compression (SCC), and this was higher for non-Hispanic Black patients vs Hispanic patients and non-Hispanic White patients.

Data show that patients who have factor VIII (FVIII) inhibitor titers of 20 or higher BU/mL took longer to achieve complete responses (CR) and had lower CR rates vs patients with lower titers.

The researchers underscored the importance of their findings for a wide group of patients with inherited bleeding disorders, as most colorectal cancer (CRC) screening programs across the globe use fecal immunochemical testing (FIT).

The subset of patients with psoriatic arthritis had the highest odds ratio of migraine.

Findings from the survey suggest that certain symptoms of depression could help identify patients vulnerable to both worsening psoriasis symptoms and disease-related quality of life (QoL).

The effects of physical activity and exercise have previously been reported, with data pointing to improvements in quality of life as well as such clinical markers as immune response in tumors

Data from 2 prospective studies showed correlations between impaired patient health-related quality of life (HRQOL) and disease severity, as well as correlations between the health of caregivers and their HRQOL and caregiver burden.

The long time to diagnosis is consistent with data pointing to pulmonary arterial hypertension (PAH) often being misdiagnosed as asthma, said the researchers, adding that the findings from their retrospective study provide valuable insight into where the diagnostic pathway lags.

With greater attention being paid to the heterogenous patient population, researchers questioned whether this has resulted in a more diverse group of patients included in Parkinson disease (PD) studies in the Netherlands.

Having models like these that produce more plausible results are crucial for decision-making in an era of targeted treatments that often come with immature data, argued researchers of their findings for minimal residual disease (MRD) in newly diagnosed multiple myeloma (NDMM).