AJMC Contributor

The data come from the observational Cost of Hemophilia in Europe: A Socioeconomic Survey II (CHESS II) study of 288 Spanish patients with hemophilia A and B, which showed certain differences between disease subtypes but overall similar trends in disease impact.

An abstract presented at the 2024 American Society of Clinical Oncology annual meeting showed that patients treated with zanubrutinib or acalabrutinib had more favorable safety and efficacy outcomes.

Researchers say their findings will help inform treatment protocols for this patient population, as questions remain about optimal duration of these treatments following first-line treatment with pyridostigmine.

The proof-of-concept study showed that antisense oligonucleotides developed by the researchers successfully restored cellular development and brain cell function in patients with the disease characterized by multiorgan dysfunction.

Myasthenic crisis has been estimated to occur in up to 30% of patients with myasthenia gravis after thymectomy.

A retrospective study of over 100 pediatric and adolescent patients showed that providing virtual training for insulin pump use was both safe and effective.

Prospective data from patients attending a rural practice in West Virginia between 2016 and 2023 show that a quality improvement program that followed guideline recommendations and was tailored to specific patient needs reduced preventable bleeds and lowered costs.

Survey data from over 200 patients showed that treatment discontinuation was lower for those taking mycophenolate or methotrexate than for those taking azathioprine for their myasthenia gravis (MG).

In addition to her advanced age and obesity status, the patient had a history of pulmonary embolism managed with an inferior vena cava filter, increasing her risk of thrombosis.

Among over 60,000 nursing home residents who initiated antihypertensive medication, rates of excess fractures due to falls per 100 person-years were as high as 5 among certain patient groups, such as those with dementia and high blood pressure (BP).

Data from the study showed that exa-cel prevented vaso-occlusive crises in all but 1 of the 30 evaluable patients for at least 12 months.

Over a median follow-up time of nearly 3 years, three-fourths (72.8%) of patients with myasthenia gravis (MG) were hospitalized and half visited the accident and emergency department.

Self-reported data from adult and pediatric males with hemophilia A show that burden of the disease persists regardless of severity, highlighting a need for improved prophylactic treatment.

The randomized trial found that reducing sitting time over a 6-month period was association with reduced systolic blood pressure (BP) among a group of older patients with obesity and high rates of hypertension.

In their preclinical model, the researchers found efficacy both in vitro and in vivo by using CRISPR-Cas9 to mimic porphyria and combining the technology with light therapy.

The LIFE-T1D model, demonstrated an ability to estimate the lifetime risk of heart disease among several groups of people with type 1 diabetes (T1D).

Among a small group of patients with severe hemophilia, researchers found that getting in more than 10,000 steps each day was linked to significant improvements in perceived physical health and quality of life (QOL).

Researchers explain that these insights have important implications, if validated in further research, as challenges with early detection of dilated cardiomyopathy complicate myasthenia gravis (MG)-prognosis.

Analyzing trends from online conversations among patients with myasthenia gravis (MG), researchers found that issues with current treatments were mentioned in nearly 24% of conversations.

Researchers found that in vitro clot formulation and fibrinolysis assays were able to determine the hemostatic effects of different replacement agents, as well as identify distinct pharmacodynamic responses across the different mechanisms of action.

Findings from this new study demonstrate the safety and efficacy of rIX-FP among a group of treatment-naive patients often characterized by young age and particular risk for severe bleeding.

The researchers found that popularity of videos did not necessarily correlate with the quality of information provided in the video.

These insights, explain the researchers of the study, complement observational studies, which have previously indicated a causal relationship between the 2 diseases.

A better understanding of biomarkers in ocular graft-vs-host disease (oGVHD) can help drive a more comprehensive understanding of the disease and improve patient outcomes, say researchers.

The researchers note that their findings can help inform adjustments for future research among individuals living with myasthenia gravis (MG).

This finding on continuous glucose monitoring (CGM) remained even after the researchers adjusted for glycemic control according to HbA1C levels.

These findings, together with real-world data, could help inform risk classification of myasthenic crisis beyond known risk factors, such as infection.

This secondary analysis follows initial results from a phase 3 trial, which suggested that vitamin D supplementation with ergocalciferol improved insulin sensitivity, subsequently slowing the rise of insulin requirements in patients who have type 1 diabetes (T1D).

Prevalence of hypertension remained 30% throughout the study period and was lower than expected, based on a lower threshold for hypertension endorsed by guidelines in 2017.

A group of researchers has compiled the latest advancements in preventing and treating graft-vs-host disease (GVHD).