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Objectives: We aimed to characterise baseline disease and treatment burden in a large population with haemophilia A/B, both with (HAwI/HBwI) and without (HA/HB) inhibitors. Methods: The prospective, non‐interventional explorer6 study included patients ≥12 years old with severe HA, severe/moderate HB or HAwI/HBwI of any severity, treated according to local standard of care (excluding previous/current exposure to concizumab or emicizumab). Baseline characteristics and historical clinical data were collected and patient‐reported outcomes, including treatment burden, were assessed. Results: The explorer6 study enrolled 231 patients with haemophilia (84 HAwI/HBwI) from 33 countries. At baseline, patients with HA/HB treated with prophylaxis had the lowest median annualised bleeding rates (ABRs; 2.0), irrespective of haemophilia type; of these patients, 27.5% (HA) and 31.4% (HB) had target joints. Patients with HAwI/HBwI treated episodically reported the highest treatment burden. Of these patients, 28.5% (HAwI) and 25.1% (HBwI) performed sports activities in the month before screening. Conclusion: Despite receiving routine clinical care, historical and baseline information from patients enrolled in explorer6 showed that patients with HA/HB treated episodically and patients with HAwI/HBwI had higher ABRs, higher treatment burden and participated in sports less than those with HA/HB treated with prophylaxis. Emerging treatments could be beneficial in addressing these unmet medical needs. [ABSTRACT FROM AUTHOR]

Purpose: Adherence to growth hormone treatment is known to affect growth outcomes. Both device preference and ease of use have been shown to affect treatment adherence. In this study, we assessed device preference and ease of use with two long-acting growth hormones, somapacitan (Sogroya®, Novo Nordisk A/S) and somatrogon (Ngenla®, Pfizer). Patients and Methods: In a randomized, crossover study conducted between September 20 and November 2, 2023, we recruited 33 adolescents with a growth-related disorder, and 37 caregivers, at six locations in the United States. Each participant was trained in the use of both devices and asked to perform a simulated injection. Device training time, preparation and injection time, and injection completeness were recorded. Participants also completed the Device Handling and Preference Questionnaire (DHPAQ) to indicate their device preference and ease of use opinions. Following conclusion of the "standard" visit, 10 adolescents and 10 caregivers were randomly selected to participate in a sub-study to validate the relevance, comprehensiveness, and comprehension of the DHPAQ. Results: The majority of participants (84.3%; 95% confidence interval [CI]: 74;92) preferred the somapacitan device to the somatrogon device (p <  0.0001). Almost all (98.6%; 95% CI: 92;100) participants answered that the somapacitan device was easy or very easy to use, while three-quarters (74.3%; 95% CI: 62;84) answered the same for the somatrogon device. Average training and injection times were lower for the somapacitan device than for the somatrogon device. Also, more patients successfully completed the injection with the somapacitan device than with the somatrogon device (97.1% vs 92.9%). Cognitive debriefing interviews indicated the DHPAQ was relevant, comprehensive, and fully comprehended. Conclusion: The somapacitan device was preferred to the somatrogon device by a majority of participants. More participants considered the somapacitan device to be easy or very easy to use than the somatrogon device. Plain Language Summary: • Children who grow slower in height than their peers often need growth hormone replacement treatment to reach normal height. This treatment involves daily or weekly injections for several years. Injecting treatment regularly is important for it to work properly, but the schedule can be difficult to adhere to. • From previous research, we know injection devices can influence adherence to treatment. Researchers in this study aimed to compare two different pen injectors (once-weekly medicines called somapacitan and somatrogon) to see which one patients and caregivers prefer and find easier to use. • This USA-based study included 33 adolescents with growth problems who injected themselves, and 37 caregivers of younger patients. During one visit, they were trained in how to use each device, observed giving a practice injection, and asked to fill in a questionnaire with their experience. Half of the participants did this with the somapacitan pen injector first and the somatrogon one second, while the other half followed the opposite order. • The researchers found that 84.3% of participants preferred the somapacitan pen‑injector, while 12.9% preferred the somatrogon pen-injector, and this difference was statistically significant. Almost all (98.6%) participants found the somapacitan pen‑injector easy or very easy to use, while three-quarters (74.3%) answered the same for somatrogon. On average, the participants learned to use the somapacitan device faster and more correctly than the somatrogon device. • In conclusion, given the device preference and ease of use, patients may be more likely to adhere to treatment with somapacitan compared with those receiving somatrogon. [ABSTRACT FROM AUTHOR]

Objective of this preference and handling study was to understand patient and caregiver pen-injector handling experiences, and device preferences compared to their existing devices. In this study, most participants found it easy to learn to use the pen-injector. 98% of participants reported that the pen-injector was easy or very easy to use compared with their current device. 88% of participants reported a preference for the concizumab pen-injector over their current device. This shows that the pen-injector can easily and effectively be used to inject concizumab, providing more control and independence in patients' treatment while reducing the burden caused by intravenous modes of administration. [ABSTRACT FROM AUTHOR]

Background: Truncated tau appears to be specifically related to disease pathology and recent studies have shown the presence and elevation of several truncated tau species in Cerebrospinal fluid (CSF) of subjects with Alzheimer’s disease (AD); however, the relevance of truncated Tau measurements in blood is still being studied. Objective: The aim of the current study was to assess the longitudinal associations between baseline levels of two novel blood biomarker candidates measuring truncated tau, Tau-A and Tau-C, and the risk of incident dementia and AD in elderly women. Methods: Using solid phase competitive ELISA, two tau fragments were detected in serum of 5,309 women from the Prospective Epidemiological Risk Factor study. The study was an observational, prospective study of Danish postmenopausal women. Subjects were followed with registry-linkage for up to 15 years (median follow-up time 13.7 years). Cox regression was used to assess the utility of the biomarker candidates in relation to dementia and AD. Results: High levels of Tau-A and Tau-C (above the median) in blood were associated with lower risk of dementia and AD (Tau-A: Dementia HR[95% CI] = 0.85[0.70–1.04]; AD 0.71[0.52–0.98] and Tau-C: Dementia 0.84[0.70–1.00]; AD 0.78[0.60–1.03]). Tau-C gave a very modest increase in the AUC in a 5-year prediction horizon as compared to a reference model with age and education, while a combination of the two did not improve their predictive capacity. Conclusions: Measurement of tau in serum is feasible. The serological tau turnover profile may be related to the diagnosis and development of dementia and AD. The exact processing and profile in serum in relation to cognitive disorders remains to be further assessed to provide simple non-invasive tests to identify subjects with progressive cognitive disorders. [ABSTRACT FROM AUTHOR]

Background: An altered tumor microenvironment is one of the earliest signs of cancer and an important driver of the disease. We have seen previously that biomarkers reflecting tumor microenvironment modifications, such as matrix metalloproteinase (MMP)-degraded type 1 collagen (C1M), MMP-degraded type IV collagen (C4M), and citrullinated and MMP-degraded vimentin (VICM), were higher in the serum of cancer patients than in healthy controls. However, it is not known if these biomarkers could predict an increased risk of cancer. The aim of this study was to investigate whether C1M, C4M, and VICM were elevated prior to diagnosis of solid cancers in a large prospective study. Methods: Between 1999 and 2001, 5,855 postmenopausal Danish women ages 48 to 89 years enrolled in the Prospective Epidemiologic Risk Factor study. Baseline demographics and serum were collected at the time of registration. Follow up cancer diagnoses were obtained from the Danish Cancer Registry in 2014. Serum C1M, C4M, and VICM levels were measured by competitive ELISAs. Results: A total of 881 women were diagnosed with solid cancers after baseline. C1M, C4M, and VICM levels were significantly elevated in women diagnosed less than 1 year after baseline. C1M and VICM, but not C4M, were independent predictors of increased risk of cancer. Conclusion: C1M, C4M, and VICM are elevated prior to cancer diagnosis. C1M and VICM are both independent predictors of increased cancer risk. Impact: C1M and VICM are predictors for increased risk of cancer. [ABSTRACT FROM AUTHOR]

Introduction Aim Methods Results Conclusions The Child Hemophilia Treatment Experience Measure (Child Hemo‐TEM) was developed to capture the treatment burden experience of children with haemophilia (CwH).Describe the development of this novel haemophilia‐specific measure.Interviews were conducted with clinical experts, CwH and CwH's caregivers. Interviews were analysed according to adapted grounded theory principles. Based on the analysis, a preliminary measure was developed and debriefed. Psychometric analyses were performed according to an a priori analysis plan using data collected in a cross‐sectional web survey and a final measure was generated.Interviews with four clinical experts, 25 CwH ages 8 to <12 years, and 25 caregivers of CwH <12 years were conducted. Concepts endorsed by ≥10% of CwH and caregivers were: adherence, ease of use, emotional impacts, physical impacts, treatment concerns, and interference with daily life. Cognitive debriefing assessments were conducted to ensure participant understanding and item relevance. Caregivers found the measure to be understandable, comprehensive, and relevant. However, several issues with CwH completing the measure were identified and it was decided to only develop an observer‐reported outcome version. Data for psychometric validation was collected in a web survey (N = 187). Item reduction dropped 12 items. Factor analysis generated a single, 7‐item, internally consistent (α = .855) factor, which consisted of items covering all relevant a priori concepts. The majority of a priori convergent and all known groups validity hypotheses were confirmed.The study findings provide evidence that the Child Hemo‐TEM is a brief, well‐designed, and valid and reliable measure of haemophilia treatment burden. [ABSTRACT FROM AUTHOR]