Development and validation of the Child Hemophilia Treatment Experience Measure: A new observer-reported outcome measure.

Introduction: The Child Hemophilia Treatment Experience Measure (Child Hemo-TEM) was developed to capture the treatment burden experience of children with haemophilia (CwH).

Aim: Describe the development of this novel haemophilia-specific measure.

Methods: Interviews were conducted with clinical experts, CwH and CwH's caregivers.

Measuring treatment impacts on symptoms in adults with hypoparathyroidism: findings from the PaTHway trial.

Background: Hypoparathyroidism is a rare endocrine disease frequently associated with serious physical and cognitive symptoms. This study's purpose was to understand the impacts of the phase 3 PaTHway clinical trial treatment, TransCon PTH, on patients' overall, physical, and cognitive hypoparathyroidism signs/symptoms and what patients consider meaningful improvement.

Methods: Individual telephone exit interviews were conducted with patients who recently completed the PaTHway trial blinded period.

Opening the Bandgap of Metallic Half-Heuslers via the Introduction of d-d Orbital Interactions.

Half-Heusler compounds with semiconducting behavior have been developed as high-performance thermoelectric materials for power generation. Many half-Heusler compounds also exhibit metallic behavior without a bandgap and thus inferior thermoelectric performance. Here, taking metallic half-Heusler MgNiSb as an example, a bandgap opening strategy is proposed by introducing the d-d orbital interactions, which enables the opening of the bandgap and the improvement of the thermoelectric performance.

Weekly Somapacitan in GH Deficiency: 4-Year Efficacy, Safety, and Treatment/Disease Burden Results From REAL 3.

Context: Growth hormone deficiency (GHD) in children is currently treated with daily injections of GH, which can be burdensome for patients and their parents/guardians. Somapacitan is a GH derivative in development for once-weekly treatment of GHD.

Objective: This work aimed to assess the efficacy and safety of somapacitan, and associated disease/treatment burden, after 4 years of treatment and 1 year after switching to somapacitan from daily GH.

Understanding treatment burden in hemophilia: development and validation of the Hemophilia Treatment Experience Measure (Hemo-TEM).

Background: To capture the broad range of treatment burden issues experienced by adolescent and adult people with hemophilia (PWH), the Hemophilia Treatment Experience Measure (Hemo-TEM) was developed. We describe the development of this new hemophilia-specific patient-reported outcome (PRO) measure including concept elicitation, cognitive debriefing, and psychometric validation.

Results: Concept elicitation interviews were conducted with 5 clinical experts and 30 adult PWH in the United States (US).

Content validation of the SF-36v2® Health Survey Acute for use in hypoparathyroidism.

Purpose: The purpose of this study was to conduct cognitive debriefing (CD) interviews with adults diagnosed with chronic hypoparathyroidism (HP) to assess the content validity of the SF-36v2® Health Survey Acute (SF-36v2) measure in this population.

Methods: CD interviews were conducted with adults with HP in the United States (US). Interviews were conducted by a trained moderator using a semi-structured interview guide, employing a think-aloud method in conjunction with verbal probing.

Psychometric Validation of the Growth Hormone Deficiency-Child Treatment Burden Measure (GHD-CTB) and the Growth Hormone Deficiency-Parent Treatment Burden Measure (GHD-PTB).

Purpose: The aim was to evaluate the measurement properties of the Growth Hormone Deficiency-Child Treatment Burden Measure-Child (GHD-CTB-Child), a patient-reported outcome (PRO) for children aged 9 to < 13 years; the Growth Hormone Deficiency-Child Treatment Burden Measure-Observer (GHD-CTB-Observer), an observer-reported outcome (ObsRO) version completed by parents/guardians of children with growth hormone deficiency (GHD) aged 4 to < 9 years; and the Growth Hormone Deficiency-Parent Treatment Burden Measure (GHD-PTB), a PRO that assesses the treatment burden of parents/guardians living with children with GHD aged 4 to < 13 years.

Methods: A non-interventional, multi-center, clinic-based study across 30 private practice and large institutional sites in the United States and the United Kingdom was conducted. The sample consisted of 145 pre-pubertal children aged 9 to < 13 years at enrollment with a physician confirmed GHD diagnosis as well as 98 parents/guardians of pre-pubertal younger children aged 4 to < 9 years at enrollment with a physician confirmed GHD diagnosis.

Evaluation of Real-world Data for Breast-MRI BI-RADS IV Lesions.

Background/aim: Magnetic resonance imaging (MRI) is an important diagnostic tool in the detection of breast cancer. The Breast Center of the municipal Hospital Holweide, Cologne, annually cares for and treats patients with changes in the breast. A special problem is posed by Breast Imaging-Reporting and Data System (BI-RADS) 4 lesions.

From the Lab to the Field: Translating Applications of Near-Infrared Light from Laboratory to the Field to Improve Honeybee Mitochondrial Function and Hive Health.

Bee populations are under threat from diverse sources from climate change to insecticide use. These culminate in physiological stress undermining mitochondrial function. In laboratory environments, mitochondrial stress can be ameliorated by long wavelength light that protects insects individually against stress.

Improving adherence to guidelines for spine pain care: what tools could support primary care clinicians in conforming to guidelines?

Background: Spine pain is one of the most common conditions seen in primary care and is often treated with ineffective, aggressive interventions, such as prescription pain medications, imagery and referrals to surgery. Aggressive treatments are associated with negative side effects and high costs while conservative care has lower risks and costs and equivalent or better outcomes. Despite multiple well-publicised treatment guidelines and educational efforts recommending conservative care, primary care clinicians (PCCs) widely continue to prescribe aggressive, low-value care for spine pain.

Development and Validation of the Diabetes Pen Experience Measure: A New Patient-Reported Outcome Measure.

Background: Satisfaction with insulin-delivery devices has been shown to improve treatment adherence, translating into better glycemic control. The Diabetes Pen Experience Measure (DPEM) is a new patient-reported outcome measure to evaluate patients' experience when using an injection device.

Methods: The DPEM was developed using literature review and concept elicitation interviews with clinical experts and patients.

Effective GH Replacement With Once-weekly Somapacitan vs Daily GH in Children with GHD: 3-year Results From REAL 3.

Context: Current GH therapy requires daily injections, which can be burdensome. Somapacitan is a long-acting GH derivative in development for treatment of GH deficiency (GHD).

Objective: Evaluate the efficacy, safety, and tolerability of once-weekly somapacitan after 3 years of treatment.

Functioning and well-being in older children and adolescents with achondroplasia: A qualitative study.

The study aimed to explore how having achondroplasia affects older children and adolescents' day-to-day functioning and well-being. Individual/focus group interviews were conducted with older children/adolescents between the ages of 9 to <18 years and diagnosed with achondroplasia to elicit key concepts. An adapted grounded theory approach informed the qualitative analysis of interview data.

Psychometric validation of the Hypoparathyroidism Patient Experience Scales (HPES).

Background: Hypoparathyroidism (HP) is a rare endocrine disorder characterized by absent or inappropriately low levels of circulating parathyroid hormone with associated significant physical and cognitive symptoms. This study evaluated the psychometric properties of the Hypoparathyroidism Patient Experience Scales (HPES), which were developed as disease-specific, patient-reported outcome (PRO) measures to assess the symptoms and impacts associated with HP in adults.

Methods: Data from a non-interventional, observational study (N = 300) and a Phase 2 clinical trial (N = 59) were used in the psychometric evaluation.

A qualitative study of the impacts of having an infant or young child with achondroplasia on parent well-being.

Background: Currently, there is limited research on how having a child diagnosed with achondroplasia affects parents' lives. The purpose of the study was to investigate the experiences of parents of infants and young children less than two years of age with achondroplasia.

Methods: Concept elicitation interviews were conducted with parents of children less than 2 years of age with achondroplasia in the United States and Spain.

PaTH Forward: A Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial of TransCon PTH in Adult Hypoparathyroidism.

Context: Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH). TransCon PTH is an investigational long-acting prodrug of PTH(1-34) for the treatment of hypoparathyroidism.

Objective: This work aimed to investigate the safety, tolerability, and efficacy of daily TransCon PTH in adults with hypoparathyroidism.

Psychometric Validation of the Growth Hormone Deficiency-Child Impact Measure (GHD-CIM).

Objective: The aim of this study was to perform psychometric testing of the Growth Hormone Deficiency-Child Impact Measure (GHD-CIM): a patient-reported outcome (PRO) for children with GHD aged 9 to < 13 years and an observer-reported outcome (ObsRO) for parents/guardians of children who are unable to answer for themselves.

Methods: A non-interventional, multicenter, clinic-based study was conducted in 30 private-practice and large institutional sites in the US and the UK. Psychometric analyses were conducted following an a priori validation statistical analysis plan.

Assessing physical symptoms, daily functioning, and well-being in children with achondroplasia.

This study's purpose was to provide qualitative evidence to support the development of two observer-reported outcome measures assessing the physical symptoms/complications of achondroplasia in children and impacts on children's quality of life. Individual/focus group concept elicitation interviews were conducted with parents of children aged 2 to <12 years with achondroplasia and experts. Qualitative analysis of transcripts, based on an adapted grounded theory approach, informed item generation and measure development.

Living with hypoparathyroidism: development of the Hypoparathyroidism Patient Experience Scale-Impact (HPES-Impact).

Purpose: Hypoparathyroidism (HP) is a rare endocrine disorder characterized by absent or inappropriately low levels of circulating parathyroid hormone (PTH). Research indicates that HP patients on conventional therapy may have a reduced quality of life. The study's purpose was to develop a new disease-specific measure of the impacts of hypoparathyroidism on functioning and well-being and provide evidence for its content validity based on rigorous qualitative research methodologies for patient-reported outcomes development.

Assessing the impacts of having a child with achondroplasia on parent well-being.

Purpose: This study's purpose was to develop a better understanding of the experiences of parents of children with achondroplasia and to provide qualitative evidence to support the development of a patient-reported outcome (PRO) measure of parent impacts.

Methods: Concept elicitation (CE) individual/focus group interviews were conducted with parents of children aged 2 to < 12 years with achondroplasia in the United States and Spain. The qualitative analysis informed the PRO measure development.

Understanding the burden of illness of excessive daytime sleepiness associated with obstructive sleep apnea: a qualitative study.

Background: Obstructive sleep apnea (OSA) is associated with excessive daytime sleepiness (EDS), which may go undiagnosed and can significantly impair a patient's health-related quality of life (HRQOL). This qualitative research examined timing and reasons patients sought medical care for their EDS and OSA symptoms, and the impact of EDS on HRQOL.

Methods: Focus groups were conducted in 3 US cities with 42 participants currently experiencing EDS with OSA.

Once-Weekly Somapacitan vs Daily GH in Children With GH Deficiency: Results From a Randomized Phase 2 Trial.

Context: Daily growth hormone (GH) injections can be burdensome for patients and carers. Somapacitan is a long-acting, reversible albumin-binding GH derivative in development for once-weekly administration in patients with growth hormone deficiency (GHD).

Objective: The objective of this study is to evaluate the efficacy, safety, and tolerability of once-weekly somapacitan vs once-daily GH.

Assessing the Patient Experience of Hypoparathyroidism Symptoms: Development of the Hypoparathyroidism Patient Experience Scale-Symptom (HPES-Symptom).

Background And Objective: Hypoparathyroidism is a rare endocrine disorder characterized by absent or inappropriately low levels of circulating parathyroid hormone. Patients with hypoparathyroidism receiving standard-of-care therapy report debilitating physical and cognitive symptoms, which may indicate a reduced health-related quality of life. The purpose of this study was to develop a new disease-specific measure of the signs and symptoms of hypoparathyroidism, the Hypoparathyroidism Patient Experience Scale-Symptom (HPES-Symptom), and provide evidence for the content validity of items in the measure based on rigorous qualitative research methodologies for patient-reported outcome development.