Artificial intelligence in paediatric endocrinology: conflict or cooperation.

Artificial intelligence (AI) in medicine is transforming healthcare by automating system tasks, assisting in diagnostics, predicting patient outcomes and personalising patient care, founded on the ability to analyse vast datasets. In paediatric endocrinology, AI has been developed for diabetes, for insulin dose adjustment, detection of hypoglycaemia and retinopathy screening; bone age assessment and thyroid nodule screening; the identification of growth disorders; the diagnosis of precocious puberty; and the use of facial recognition algorithms in conditions such as Cushing syndrome, acromegaly, congenital adrenal hyperplasia and Turner syndrome. AI can also predict those most at risk from childhood obesity by stratifying future interventions to modify lifestyle. AI will facilitate personalised healthcare by integrating data from 'omics' analysis, lifestyle tracking, medical history, laboratory and imaging, therapy response and treatment adherence from multiple sources. As data acquisition and processing becomes fundamental, data privacy and protecting children's health data is crucial. Minimising algorithmic bias generated by AI analysis for rare conditions seen in paediatric endocrinology is an important determinant of AI validity in clinical practice. AI cannot create the patient-doctor relationship or assess the wider holistic determinants of care. Children have individual needs and vulnerabilities and are considered in the context of family relationships and dynamics. Importantly, whilst AI provides value through augmenting efficiency and accuracy, it must not be used to replace clinical skills.

Key features of puberty onset and progression can help distinguish self-limited delayed puberty from congenital hypogonadotrophic hypogonadism.

Introduction: Delayed puberty (DP) is a frequent concern for adolescents. The most common underlying aetiology is self-limited DP (SLDP). However, this can be difficult to differentiate from the more severe condition congenital hypogonadotrophic hypogonadism (HH), especially on first presentation of an adolescent patient with DP. This study sought to elucidate phenotypic differences between the two diagnoses, in order to optimise patient management and pubertal development. Methods: This was a study of a UK DP cohort managed 2015-2023, identified through the NIHR clinical research network. Patients were followed longitudinally until adulthood, with a definite diagnosis made: SLDP if they had spontaneously completed puberty by age 18 years; HH if they had not commenced (complete, cHH), or had commenced but not completed puberty (partial, pHH), by this stage. Phenotypic data pertaining to auxology, Tanner staging, biochemistry, bone age and hormonal treatment at presentation and during puberty were retrospectively analysed. Results: 78 patients were included. 52 (66.7%) patients had SLDP and 26 (33.3%) patients had HH, comprising 17 (65.4%) pHH and 9 (34.6%) cHH patients. Probands were predominantly male (90.4%). Male SLDP patients presented with significantly lower height and weight standard deviation scores than HH patients (height p=0.004, weight p=0.021). 15.4% of SLDP compared to 38.5% of HH patients had classical associated features of HH (micropenis, cryptorchidism, anosmia, etc. p=0.023). 73.1% of patients with SLDP and 43.3% with HH had a family history of DP (p=0.007). Mean first recorded luteinizing hormone (LH) and inhibin B were lower in male patients with HH, particularly in cHH patients, but not discriminatory. There were no significant differences identified in blood concentrations of FSH, testosterone or AMH at presentation, or in bone age delay. Discussion: Key clinical markers of auxology, associated signs including micropenis, and serum inhibin B may help distinguish between SLDP and HH in patients presenting with pubertal delay, and can be incorporated into clinical assessment to improve diagnostic accuracy for adolescents. However, the distinction between HH, particularly partial HH, and SLDP remains problematic. Further research into an integrated framework or scoring system would be useful in aiding clinician decision-making and optimization of treatment. .

Evaluating the Usefulness and Ease of Use of a Next-Generation-Connected Drug Delivery Device for Growth Hormone Therapy: Qualitative Study of Health Care Professionals' Perceptions.

BACKGROUND: Digital solutions targeting children's health have become an increasingly important element in the provision of integrated health care. For the treatment of growth hormone deficiency (GHD), a unique connected device is available to facilitate the delivery of recombinant human growth hormone (r-hGH) by automating the daily injection process and collecting injection data such that accurate adherence information is available to health care professionals (HCPs), caregivers, and patients. The adoption of such digital solutions requires a good understanding of the perspectives of HCPs as key stakeholders because they leverage data collection and prescribe these solutions to their patients. OBJECTIVE: This study aimed to evaluate the third generation of the easypod device (EP3) for the delivery of r-hGH treatment from the HCP perspective, with a focus on perceived usefulness and ease of use. METHODS: A qualitative study was conducted, based on a participatory workshop conducted in Zaragoza, Spain, with 10 HCPs experienced in the management of pediatric GHD from 7 reference hospitals in Spain. Several activities were designed to promote discussion among participants about predefined topics based on the Technology Acceptance Model and the Unified Theory of Acceptance and Use of Technology to provide their perceptions about the new device. RESULTS: Participants reported 2 key advantages of EP3 over previous easypod generations: the touch screen interface and the real-time data transmission functionality. All participants (10/10, 100%) agreed that the new device should be part of a digital health ecosystem that provides complementary functionalities including data analysis. CONCLUSIONS: This study explored the perceived value of the EP3 autoinjector device for the treatment of GHD by HCPs. HCPs rated the new capabilities of the device as having substantial improvements and concluded that it was highly recommendable for clinical practice. EP3 will enhance decision-making and allow for more personalized care of patients receiving r-hGH.

Supportive use of digital technologies during transition to adult healthcare for young people with long-term conditions, focusing on Type 1 diabetes mellitus: A scoping review.

Type 1 diabetes mellitus (T1DM) is the second most common chronic or long-term condition (LTC) affecting young people (YP); when transitioning from paediatric to adult healthcare, young people with LTCs such as T1DM are expected to self-manage medication, diet and clinical appointments. This scoping review aimed to analyse research examining ways digital health technologies were used to support YP with LTCs during transition from paediatric to adult healthcare and to establish YP's needs, experiences and challenges when transitioning. We aimed to identify knowledge gaps and inform development of a novel chatbot with components such as avatars and linked videos to help YP with T1DM gain self-management confidence and competence during transition. Nineteen studies identified through searching five electronic databases were included in this review. A combination of digital health technologies was used to support transition of YP with LTCs to adult healthcare. Barriers to successful transition were reported and YP described the importance of social relationships and transition readiness and expressed the need for individualised interventions that acknowledge social factors such as work and college. No supportive chatbots with components to help YP with T1DM were identified. This contribution will inform future development and evaluation of such a chatbot.

Accelerating digital health literacy for the treatment of growth disorders: The impact of a massive open online course.

Background: Growth hormone deficiency (GHD) is a rare disorder characterized by inadequate secretion of growth hormone (GH) from the anterior pituitary gland. One of the challenges in optimizing GH therapy is improving adherence. Using digital interventions may overcome barriers to optimum treatment delivery. Massive open online courses (MOOCs), first introduced in 2008, are courses made available over the internet without charge to a large number of people. Here, we describe a MOOC aiming to improve digital health literacy among healthcare professionals managing patients with GHD. Based on pre- and post-course assessments, we evaluate the improvement in participants' knowledge upon completion of the MOOC. Methods: The MOOC entitled 'Telemedicine: Tools to Support Growth Disorders in a Post-COVID Era' was launched in 2021. It was designed to cover 4 weeks of online learning with an expected commitment of 2 h per week, and with two courses running per year. Learners' knowledge was assessed using pre- and post-course surveys via the FutureLearn platform. Results: Out of 219 learners enrolled in the MOOC, 31 completed both the pre- and post-course assessments. Of the evaluated learners, 74% showed improved scores in the post-course assessment, resulting in a mean score increase of 21.3%. No learner achieved 100% in the pre-course assessment, compared with 12 learners (40%) who achieved 100% in the post-course assessment. The highest score increase comparing the pre- and the post-course assessments was 40%, observed in 16% of learners. There was a statistically significant improvement in post-course assessment scores from 58.1 ± 18.9% to 72.6 ± 22.4% reflecting an improvement of 14.5% (p < 0.0005) compared to the pre-course assessment. Conclusion: This "first-of-its-kind" MOOC can improve digital health literacy in the management of growth disorders. This is a crucial step toward improving the digital capability and confidence of healthcare providers and users, and to prepare them for the technological innovations in the field of growth disorders and growth hormone therapy, with the aim of improving patient care and experience. MOOCs provide an innovative, scalable and ubiquitous solution to train large numbers of healthcare professionals in limited resource settings.

Playing your pain away: designing a virtual reality physical therapy for children with upper limb motor impairment.

Children with upper limb motor impairment often undergo repetitive therapeutic physiotherapy sessions to minimize functional disabilities of the affected area. Even though therapeutic processes can improve functional outcomes and minimize persistent disabilities, patients often neglect to participate fully in physical therapies due to the associated procedural pain. Over recent decades, there has been a growing interest in designing non-pharmacological interventions which aim to minimize pain during physical therapies and improve functional outcomes. Via two interrelated studies, we explored the use of virtual reality (VR) as a tool to provide therapeutic physiotherapy for child patients in an out-patient hospital department. We found that VR is an effective solution for children with upper limb motor impairment undergoing painful therapeutic process within a hospital environment. VR can improve functional disabilities, alleviate perceived pain, reduce the perceived difficulty of rehabilitation exercises, increase exercise duration and produce positive emotions towards the therapy. Trial registration number and date of registration Protocol ID NCT03998995. Release Date: June 25, 2019.

Home-based immersive virtual reality physical rehabilitation in paediatric patients for upper limb motor impairment: a feasibility study.

Upper limb motor impairment (ULMI) rehabilitation is a long-term, demanding and challenging process to recover motor functionality. Children and adolescents may be limited in daily life activities due to reduced functions such as decreased joint movement or muscle weakness. Home-based therapy with Immersive Virtual Reality can offer greater accessibility, delivery and early rehabilitation to significantly optimise functional outcomes and quality of life. This feasibility study aimed to explore the perceptions and impacts of an immersive and interactive VR scenario suitable for ULMI rehabilitation for children at home. It was analysed using mixed methods (quantitative and qualitative) and from a multidirectional perspective (patients, clinicians and family members). Amongst the main results, it was found that IVR for ULMI home rehabilitation (1) is easy to learn and acceptable; (2) improves motor function; (3) reduces the difficulty in the reproduction of therapeutic movements; (4) is motivating and enjoyable and (5) improves quality of life. This study is the first study on the use of IVR applied to home rehabilitation of ULMI in children. These results suggested that similar outcomes may be possible with self-directed IVR home rehabilitation compared to face to face conventional rehabilitation, which can be costly to both the patient and the healthcare system, decreasing the length of stay at the hospital and treatment duration. It has also presented an innovative solution to the Covid-19 emergency where children could not receive their clinic therapy. Further research is recommended to understand better the mechanisms involved in physiotherapeutic recovery and how IVR rehabilitation helps to improve conventional treatments. Trial Registration Protocol ID NCT05272436. Release Date: 9th March 2022.

Precision diagnostics in children.

Medical practice is transforming from a reactive to a pro-active and preventive discipline that is underpinned by precision medicine. The advances in technologies in such fields as genomics, proteomics, metabolomics, transcriptomics and artificial intelligence have resulted in a paradigm shift in our understanding of specific diseases in childhood, greatly enhanced by our ability to combine data from changes within cells to the impact of environmental and population changes. Diseases in children have been reclassified as we understand more about their genomic origin and their evolution. Genomic discoveries, additional 'omics' data and advances such as optical genome mapping have driven rapid improvements in the precision and speed of diagnoses of diseases in children and are now being incorporated into newborn screening, have improved targeted therapies in childhood and have supported the development of predictive biomarkers to assess therapeutic impact and determine prognosis in congenital and acquired diseases of childhood. New medical device technologies are facilitating data capture at a population level to support higher diagnostic accuracy and tailored therapies in children according to predicted population outcome, and digital ecosystems now tailor therapies and provide support for their specific needs. By capturing biological and environmental data as early as possible in childhood, we can understand factors that predict disease or maintain health and track changes across a more extensive longitudinal path. Data from multiple health and external sources over long-time periods starting from birth or even in the in utero environment will provide further clarity about how to sustain health and prevent or predict disease. In this respect, we will not only use data to diagnose disease, but precision diagnostics will aid the 'diagnosis of good health'. The principle of 'start early and change more' will thus underpin the value of applying a personalised medicine approach early in life.

Meaningful involvement of children and young people in health technology development.

The anatomical, physiological, and developmental changes which arise as children mature through childhood and adolescence support the need to develop new health technologies that meet the specific requirements of children and young people (CYP). Failing to involve CYP during the development of technology increases the risk that the outcome falls short of their expectations and needs, leading to rejection of novel interventions. Through participation in health technology development, CYP and their families can provide context, insight, personal experience and tacit knowledge to ensure that the end-product is usable, acceptable, and can be integrated into its intended environment. A nuanced, balanced understanding of the methods that can be used to facilitate participation will support researchers in choosing an effective approach to involving CYP in health technology development. Methodological approaches include patient and public involvement and engagement, co-design, and experienced based co-design. These methods can be used in isolation or in combination, to facilitate meaningful involvement of CYP and encourage the development of impactful solutions, in consideration of the context, stakeholders, and objectives of the project. We provide the rationale and justification for involving CYP in health technology design and development, an explanation of the methods supporting meaningful involvement, and case studies exemplifying real world application of these methods with positive outputs.

Overcoming challenges to develop technology for child health.

Millions of children and young people (CYP) in the UK are affected by chronic or rare health conditions. Rapid advances in science and technology have resulted in CYP with chronic and rare conditions now surviving well into adulthood. New technologies have the potential to improve short- and long-term health outcomes for CYP with health conditions, prevent adult onset disease and complications, and reduce the burden on health services. There is thus a need for targeted investment and appropriate governance in child health technology development to address the specific needs of this population; health technology must be versatile to meet the social, anatomical, cognitive, psychological, and physiological changes inherent to childhood development. Despite the growing demand for health technology for a sizeable global population, industry still wrongly perceives the market size is relatively small, and health technology development is often localised and fragmented with limited scope for spread and adoption. These challenges can be overcome by validating and prioritising unmet needs, involving CYP and their families throughout the innovation pathway, facilitating effective partnerships with key stakeholders, and utilising national and international infrastructure and networks. This paper outlines five innovations supported by NIHR Children and Young People MedTech Co-operative that illustrate how common challenges in child health technology development can be overcome. It is essential that we continue to address such challenges and invest in the health and wellbeing of CYP.

Managing interorganisational collaborations to develop medical technologies: the contribution of interpersonal relationships.

The development of medical technologies that effectively meet clinical and patient needs increasingly relies upon collaborative working between clinicians, businesses and universities. While this "open" innovation process may provide access to additional resources, knowledge, and expertise the process is not frictionless. At the personal level, individuals may have different ways of working and incentives and at the organisational level, partners may have their own cultures and processes. Thus, interorganisational collaboration is not necessarily a panacea, but has advantages and disadvantages. The challenges are somewhat heightened in the MedTech sector where collaborative working cuts across established professional boundaries, brings together diverse knowledge from an array of disciplines, and often disrupts existing medical practice. Given these factors, this article presents a review of the extant management literature examining the complexities within multi-party collaboration and ways to drive these partnerships forwards. The article emphasises the critical value of interpersonal relationships within collaborations and offers means of strengthening them.

Treatment of Acquired Hypothalamic Obesity: Now and the Future.

The hypothalamus is the centre of neuroendocrine regulation of energy homeostasis and appetite. Maldevelopment of, or damage to, the key hypothalamic nuclei disrupts the coordinated balance between energy intake and expenditure leading, to rapid and excessive weight gain. Hypothalamic obesity is compounded by a disruption of the hypothalamic-pituitary axis, sleep disruption, visual compromise, and neurological and vascular sequalae. Amongst suprasellar tumors, craniopharyngioma is the most common cause of acquired hypothalamic obesity, either directly or following surgical or radiotherapeutic intervention. At present, therapy is limited to strategies to manage obesity but with a modest and variable impact. Current approaches include optimizing pituitary hormone replacement, calorie restriction, increased energy expenditure through physical activity, behavioral interventions, pharmacotherapy and bariatric surgery. Current pharmacotherapeutic approaches include stimulants that increase energy consumption, anti-diabetic agents, hypothalamic-pituitary substitution therapy, octreotide, and methionine aminopeptidase 2 (MetAP2) inhibitors. Some pharmacological studies of hypothalamic obesity report weight loss or stabilization but reported intervention periods are short, and others report no effect. The impact of bariatric surgery on weight loss in hypothalamic obesity again is variable. Novel or combined approaches to manage hypothalamic obesity are thus required to achieve credible and sustained weight loss. Identifying etiological factors contributing hypothalamic obesity may lead to multi-faceted interventions targeting hyperphagia, insulin resistance, decreased energy expenditure, sleep disturbance, hypopituitarism and psychosocial morbidity. Placebo-controlled trials using current single, or combination therapies are required to determine the impact of therapeutic agents. A well-defined approach to defining the location of hypothalamic damage may support the use of future targeted therapies. Intranasal oxytocin is currently being investigated as an anorexogenic agent. Novel agents including those targeting pro-opimelanocortin-C and AgRP/NPY expressing neurons and the MC4 receptor may result in better outcomes. This article discusses the current challenges in the management of hypothalamic obesity in children and young people and future therapeutic approaches to increasing weight loss and quality of life in these patients.

Long-Term Efficacy of T3 Analogue Triac in Children and Adults With MCT8 Deficiency: A Real-Life Retrospective Cohort Study.

CONTEXT: Patients with mutations in thyroid hormone transporter MCT8 have developmental delay and chronic thyrotoxicosis associated with being underweight and having cardiovascular dysfunction. OBJECTIVE: Our previous trial showed improvement of key clinical and biochemical features during 1-year treatment with the T3 analogue Triac, but long-term follow-up data are needed. METHODS: In this real-life retrospective cohort study, we investigated the efficacy of Triac in MCT8-deficient patients in 33 sites. The primary endpoint was change in serum T3 concentrations from baseline to last available measurement. Secondary endpoints were changes in other thyroid parameters, anthropometric parameters, heart rate, and biochemical markers of thyroid hormone action. RESULTS: From October 15, 2014 to January 1, 2021, 67 patients (median baseline age 4.6 years; range, 0.5-66) were treated up to 6 years (median 2.2 years; range, 0.2-6.2). Mean T3 concentrations decreased from 4.58 (SD 1.11) to 1.66 (0.69) nmol/L (mean decrease 2.92 nmol/L; 95% CI, 2.61-3.23; P < 0.0001; target 1.4-2.5 nmol/L). Body-weight-for-age exceeded that of untreated historical controls (mean difference 0.72 SD; 95% CI, 0.36-1.09; P = 0.0002). Heart-rate-for-age decreased (mean difference 0.64 SD; 95% CI, 0.29-0.98; P = 0.0005). SHBG concentrations decreased from 245 (99) to 209 (92) nmol/L (mean decrease 36 nmol/L; 95% CI, 16-57; P = 0.0008). Mean creatinine concentrations increased from 32 (11) to 39 (13) µmol/L (mean increase 7 µmol/L; 95% CI, 6-9; P < 0.0001). Mean creatine kinase concentrations did not significantly change. No drug-related severe adverse events were reported. CONCLUSIONS: Key features were sustainably alleviated in patients with MCT8 deficiency across all ages, highlighting the real-life potential of Triac for MCT8 deficiency.

Adjuvant Rituximab-Exploratory Trial in Young People With Graves Disease.

CONTEXT: Remission rates in young people with Graves hyperthyroidism are less than 25% after 2 years of thionamide antithyroid drug (ATD). OBJECTIVE: We explored whether rituximab (RTX), a B-lymphocyte-depleting agent, would increase remission rates when administered with a short course of ATD. METHODS: This was an open-label, multicenter, single-arm, phase 2 trial in young people (ages, 12-20 years) with Graves hyperthyroidism. An A'Hern design was used to distinguish an encouraging remission rate (40%) from an unacceptable rate (20%). Participants presenting with Graves hyperthyroidism received 500 mg RTX and 12 months of ATD titrated according to thyroid function. ATDs were stopped after 12 months and primary outcome assessed at 24 months. Participants had relapsed at 24 months if thyrotropin was suppressed and free 3,5,3'-triiodothyronine was raised; they had received ATD between months 12 and 24; or they had thyroid surgery/radioiodine. RESULTS: A total of 27 participants were recruited and completed the trial with no serious side effects linked to treatment. Daily carbimazole dose at 12 months was less than 5 mg in 21 of 27 participants. Thirteen of 27 participants were in remission at 24 months (48%, 90% one-sided CI, 35%-100%); this exceeded the critical value (9) for the A'Hern design and provided evidence of a promising remission rate. B-lymphocyte count at 28 weeks, expressed as a percentage of baseline, was related to likelihood of remission. CONCLUSION: Adjuvant RTX, administered with a 12-month course of ATD, may increase the likelihood of remission in young people with Graves hyperthyroidism. A randomized trial of adjuvant RTX in young people with Graves hyperthyroidism is warranted.

Medical Device Development for Children and Young People-Reviewing the Challenges and Opportunities.

Development of specific medical devices (MDs) is required to meet the healthcare needs of children and young people (CYP). In this context, MD development should address changes in growth and psychosocial maturation, physiology, and pathophysiology, and avoid inappropriate repurposing of adult technologies. Underpinning the development of MD for CYP is the need to ensure MD safety and effectiveness through pediatric MD-specific regulations. Contrary to current perceptions of limited market potential, the global pediatric healthcare market is expected to generate around USD 15,984 million by 2025. There are 1.8 billion young people in the world today; 40% of the global population is under 24, creating significant future healthcare market opportunities. This review highlights a number of technology areas that have led to successful pediatric MD, including 3D printing, advanced materials, drug delivery, and diagnostic imaging. To ensure the targeted development of MD for CYP, collaboration across multiple professional disciplines is required, facilitated by a platform to foster collaboration and drive innovation. The European Pediatric Translational Research Infrastructure (EPTRI) will be established as the European platform to support collaboration, including the life sciences industrial sector, to identify unmet needs in child health and support the development, adoption, and commercialization of pediatric MDs.

Impact of Digital Educational Interventions to Support Parents Caring for Acutely Ill Children at Home and Factors That Affect Their Use: Protocol for a Systematic Review.

BACKGROUND: Urgent and emergency care health services are overburdened, and the use of these services by acutely ill infants and children is increasing. A large proportion of these visits could be sufficiently addressed by other health care professionals. Uncertainty about the severity of a child's symptoms is one of many factors that play a role in parents' decisions to take their children to emergency services, demonstrating the need for improved support for health literacy. Digital interventions are a potential tool to improve parents' knowledge, confidence, and self-efficacy at managing acute childhood illness. However, existing systematic reviews related to this topic need to be updated and expanded to provide a contemporary review of the impact, usability, and limitations of these solutions. OBJECTIVE: The purpose of this systematic review protocol is to present the method for an evaluation of the impact, usability, and limitations of different types of digital educational interventions to support parents caring for acutely ill children at home. METHODS: The review will be structured using the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Protocols (PRISMA-P) and Population, Intervention, Comparator, and Outcome (PICO) frameworks. Five databases will be systematically searched for studies published in English during and after 2014: Medline, EMBASE, CINAHL, APA PsycNet, and Web of Science. Two reviewers will independently screen references' titles and abstracts, select studies for inclusion based on the eligibility criteria, and extract the data into a standardized form. Any disagreements will be discussed and resolved by a third reviewer if necessary. Risk of bias of all studies will be assessed using the Mixed-Methods Appraisal Tool (MMAT), and a descriptive analysis will be used to evaluate the outcomes reported. RESULTS: The systematic review will commence during 2021. CONCLUSIONS: This systematic review will summarize the impact, usability, and limitations of digital interventions for parents with acutely ill children. It will provide an overview of the field; identify reported impacts on health and behavioral outcomes as well as parental knowledge, satisfaction, and decision making; and identify the factors that affect use to help inform the development of more effective and sustainable interventions. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): PRR1-10.2196/27504.

Immersive virtual reality in children with upper limb injuries: Findings from a feasibility study.

PURPOSE: Children who sustain Upper Limb Injuries (ULIs), including fractures and burns, may undergo intensive rehabilitation. The discomfort of therapy can reduce their compliance, limit their range of motion (ROM) and lead to chronic pain. Virtual Reality (VR) interventions have been found to reduce anticipated and procedural pain.This feasibility study aimed to explore perceptions and impacts of a custom-made, fully immersive Head-Mounted Display VR (HMD-VR) experience within a United Kingdom (UK) National Health Service (NHS) outpatient rehabilitation service for children with ULIs. METHODS: Ten children aged 9-16 in one UK Children's hospital trialled HMD-VR during one rehabilitation session. They, their parents (n = 10), and hospital physiotherapy staff (n = 2) were interviewed about their perceptions of pain, difficulty, enjoyability, therapeutic impacts, benefits, and limitations. Children rated the sessions on enjoyability, difficulty, and pain compared to usual rehabilitation exercises. Physiotherapists were asked to provide range of motion readings. RESULTS: Inductive thematic analysis of interview data generated three themes, 'Escape through Engagement'; 'Enhanced Movement'; and 'Adaptability and Practicality'. Children rated the session as more enjoyable, less difficult and painful than their usual rehabilitation exercises. Findings suggested that HMD-VR was an engaging, enjoyable experience that distracted children from the pain and boredom of therapy. Also, it seemed to enhance the movement they achieved. Participants perceived it was useful for rehabilitation and adaptable to individual needs and other patient groups. Suggestions were made to increase adaptability and build in practical safeguards. CONCLUSION: Findings from this small-scale feasibility study suggested HMD-VR was perceived as usable, acceptable, and effective with potential for further development. Future work could include larger scale trials.

An eHealth Framework for Managing Pediatric Growth Disorders and Growth Hormone Therapy.

BACKGROUND: The use of technology to support health and health care has grown rapidly in the last decade across all ages and medical specialties. Newly developed eHealth tools are being implemented in long-term management of growth failure in children, a low prevalence pediatric endocrine disorder. OBJECTIVE: Our objective was to create a framework that can guide future implementation and research on the use of eHealth tools to support patients with growth disorders who require growth hormone therapy. METHODS: A total of 12 pediatric endocrinologists with experience in eHealth, from a wide geographical distribution, participated in a series of online discussions. We summarized the discussions of 3 workshops, conducted during 2020, on the use of eHealth in the management of growth disorders, which were structured to provide insights on existing challenges, opportunities, and solutions for the implementation of eHealth tools across the patient journey, from referral to the end of pediatric therapy. RESULTS: A total of 815 responses were collected from 2 questionnaire-based activities covering referral and diagnosis of growth disorders, and subsequent growth hormone therapy stages of the patient pathway, relating to physicians, nurses, and patients, parents, or caregivers. We mapped the feedback from those discussions into a framework that we developed as a guide to integration of eHealth tools across the patient journey. Responses focused on improved clinical management, such as growth monitoring and automation of referral for early detection of growth disorders, which could trigger rapid evaluation and diagnosis. Patient support included the use of eHealth for enhanced patient and caregiver communication, better access to educational opportunities, and enhanced medical and psychological support during growth hormone therapy management. Given the potential availability of patient data from connected devices, artificial intelligence can be used to predict adherence and personalize patient support. Providing evidence to demonstrate the value and utility of eHealth tools will ensure that these tools are widely accepted, trusted, and used in clinical practice, but implementation issues (eg, adaptation to specific clinical settings) must be addressed. CONCLUSIONS: The use of eHealth in growth hormone therapy has major potential to improve the management of growth disorders along the patient journey. Combining objective clinical information and patient adherence data is vital in supporting decision-making and the development of new eHealth tools. Involvement of clinicians and patients in the process of integrating such technologies into clinical practice is essential for implementation and developing evidence that eHealth tools can provide value across the patient pathway.