Lifelong Care Research Articles

How to best take care of young patients with glaucoma

AbstractPaediatric glaucoma is a potentially sight‐threatening disease and is considered the second leading cause of treatable childhood blindness. Paediatric glaucoma is a clinical entity including a wide range of conditions: primary congenital glaucoma, glaucoma secondary to ocular (e.g., aniridia, Peter's anomaly), or systemic disease (e.g., Sturge Weber) and glaucoma secondary to acquired condition (pseudophakic, traumatic, uveitic glaucoma). The treatment algorithm of childhood glaucoma is a step‐by‐step approach, often starting with surgery, as in primary congenital glaucoma cases. Medical therapy is also crucial in the management of paediatric glaucoma.Here we discuss the approach to a child with glaucoma, from the diagnosis to the medical and surgical treatment. Efficacy and side effects of each approach will be discussed.In the talk it is emphasized that paediatric glaucoma is one of the most challenging ocular disorder, requiring a step‐by‐step approach, several examinations, often multiple surgeries, and lifelong care and that a strong alliance between eye care staff and family is of paramount importance in this challenging but also rewarding journey.

EPH88 Determinants of Caregiving Burden Among Caregivers of Adult Patients With Beta-Thalassemia Major in China

β-thalassemia major is an autosomal recessive genetic disease which causes death and disability. The quality of life of adult patients with β-thalassemia major is significantly lower than that of children with β-thalassemia major due to the serious complications. Therefore, the adult patients with β-thalassemia major make lifelong care necessary. Moreover, the caregivers of adult patients bear not only the physical pressures of caring, but also the emotional and economic ones. Thus, this study examined the predictors of caregiver burden in adult patients with β-thalassemia major in China.

Modernization of a Large Spinal Cord Injuries and Disorders Registry: The Veterans Administration Experience.

Since the 1990s, Veterans Health Administration (VHA) has maintained a registry of Veterans with Spinal Cord Injuries and Disorders (SCI/Ds) to guide clinical care, policy, and research. Historically, methods for collecting and recording data for the VHA SCI/D Registry (VSR) have required significant time, cost, and staffing to maintain, were susceptible to missing data, and caused delays in aggregation and reporting. Each subsequent data collection method was aimed at improving these issues over the last several decades. This paper describes the development and validation of a case-finding and data-capture algorithm that uses primary clinical data, including diagnoses and utilization across 9 million VHA electronic medical records, to create a comprehensive registry of living and deceased Veterans seen for SCI/D services since 2012. A multi-step process was used to develop and validate a computer algorithm to create a comprehensive registry of Veterans with SCI/D whose records are maintained in the enterprise wide VHA Corporate Data Warehouse. Chart reviews and validity checks were used to validate the accuracy of cases that were identified using the new algorithm. An initial cohort of 28,202 living and deceased Veterans with SCI/D who were enrolled in VHA care from 10/1/2012 through 9/30/2017 was validated. Tables, reports, and charts using VSR data were developed to provide operational tools to study, predict, and improve targeted management and care for Veterans with SCI/Ds. The modernized VSR includes data on diagnoses, qualifying fiscal year, recent utilization, demographics, injury, and impairment for 38,022 Veterans as of 11/2/2022. This establishes the VSR as one of the largest ongoing longitudinal SCI/D datasets in North America and provides operational reports for VHA population health management and evidence-based rehabilitation. The VSR also comprises one of the only registries for individuals with non-traumatic SCI/Ds and holds potential to advance research and treatment for multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), and other motor neuron disorders with spinal cord involvement. Selected trends in VSR data indicate possible differences in the future lifelong care needs of Veterans with SCI/Ds. Future collaborative research using the VSR offers opportunities to contribute to knowledge and improve health care for people living with SCI/Ds.

The availability of essential medicines for cardiovascular diseases at healthcare facilities in low- and middle-income countries: The case of Bangladesh.

Long-term, often lifelong care for cardiovascular disease (CVD) patients requires consistent use of medicine; hence, the availability of essential medicine for CVD (EM-CVD) is vital for treatment, quality of life, and survival. We aimed to assess the availability of EM-CVD and explore healthcare facility (HCF) characteristics associated with the availability of those medicines in Bangladesh. This study utilized publicly available cross-sectional data from the 2014 and 2017 waves of the Bangladesh Health Facilities Survey (BHFS). The analysis included 204 facilities (84 from the 2014 BHFS and 120 from the 2017 BHFS) that provide CVD diagnosis and treatment services. The outcome variable "EM-CVD availability" was calculated as a counting score of the following tracer medicines: angiotensin-converting enzyme (ACE) inhibitors (enalapril), thiazide, beta-blockers (atenolol), calcium channel blockers (amlodipine and nifedipine), aspirin, and simvastatin/atorvastatin. A multivariable Poisson regression model was used to identify the HCF characteristics associated with EM-CVD availability. The number of Bangladeshi HCFs that provide CVD screening and treatment services increased just a little between 2014 and 2017 (from 5.4% to 7.9%). Since 2014, there has been an increase in the availability of calcium channel blockers (from 37.5% to 38.5%), aspirin (from 25.3% to 27.9%), and simvastatin/atorvastatin (from 8.0% to 30.7%), whereas there has been a decrease in the availability of ACE inhibitors (enalapril) (from 12.5% to 6.5%), thiazide (from 15.7% to 11.1%), and beta-blockers (from 42.5% to 32.5%). The likelihood of EM-CVD being available was higher among private and urban facilities than among public and rural facilities. Furthermore, facilities that had 24-hour staff coverage and performed quality assurance activities had a higher chance of having EM-CVD available than those that did not have 24-hour staff coverage and did not undertake quality assurance activities. Government authorities should think about a wide range of policy implications, such as putting more emphasis on public and rural facilities, making sure staff is available 24 hours a day, and performing quality assurance activities at facilities to make EM-CVD more available.

Chronic Non-communicable Diseases, Pain, and Coping Strategies: An Overview

Around the world, chronic diseases have impacted the health and quality of life of many individuals. These chronic non-communicable diseases (NCDs), such as cancer, diabetes, cardiovascular disorders, and lung disease, are expensive to treat, frequently require lifelong care, and worsen drastically if treatment is postponed or of poor quality. This phenomenon will place pressure on healthcare systems to adjust to meet these evolving demands by creating efficient preventive strategies. One of the key epidemiological trends of the current times is the rise of chronic and degenerative diseases. This study will provide an overview of chronic diseases and helps to provide a more detailed image of pain and coping strategies. This requires adapting health systems and health policies and a shift from disease-centered to people-centered approaches and population health measures.

PSUN160 Transfusion Related Endocrinopathies and Thalassemia (TREAT) Study

Abstract Thalassemia (Thal), the most common form of inherited anemia worldwide, may require regular blood transfusions and life-long medical care. The introduction of oral chelators and imaging methods to monitor iron overload transformed Thal from a rapidly fatal disease of childhood to a chronic illness compatible with prolonged life expectancy. Regular blood transfusions resulting in tissue iron overload and oxidative stress may lead to liver disease and polyendocrinopathy. In this observational descriptive study, we described the patient characteristics and prevalence of endocrinopathies (growth failure, hypothyroidism, hypogonadism, low bone mass, and impaired glucose intolerance) in a contemporary group of transfusion dependent Thal patients cared for our tertiary care center since 2000. A total of 253 transfusion dependent Thal patients were identified. Of those patients, 51.8% were female; 22% (n=56) were adopted, mainly from China. The following ethnicities were represented: Chinese 19.9%, Indian 12.6%, Laotian 7.8%, Asian 7.8%, Italian 7.8%, Vietnamese 3.9%, Afgani 3.4%, Pakistani 3.4%, Thai 2.9%, Greek 2.4%, Cambodian 2.4%, Filipino, Caucasian, Iranian 1.9% each. Majority of the diagnoses were Beta Thal Major (69.9%), followed by E Beta 0 Thal (14.6%). The median age at first visit was 10.6 years (IQR1-IQR3: 2.2-25), last visit was 27 years (IQR1-IQR3: 12.1–39.4) with 33.5% of the patients ≤18 years of age. Median follow-up time was 10 years. Median final adult height (height z-score) for females was 159.5 cm (-0.53) (IQR1-IQR3: 152.4,160.3cm; height z-score: -1.68, -0.47) and for males 167.5cm (-1.3) (IQR1-IQR3: 161.5, 171cm; height z-score: -2.13, -0.81). Only, 16.5% (n=14) of children were on growth hormone treatment due to low-normal growth hormone surrogates or peak growth hormone <10ng/ml in growth hormone stimulation test. Hypothyroidism was diagnosed in 9.9% (n=25) of the patients and 5 patients had central hypothyroidism. Hypogonadism was diagnosed in 32.4% of adult female (n=24), and 44.6% of adult male (n=29) patients who were subsequently prescribed sex steroid replacement. Low bone mass in those with z-scores ≤2.0 was noted in 22.9% (n=43) female and 26% (n=49) males. The mean bone mineral density z-score for spine was -2.1±1.1 for females and -2.5±1.3 for males. Either impaired glucose tolerance or insulin-dependent DM was observed in 29% of the patients (n=54). Endocrinopathies are common and continue to be a significant morbidity in transfusion dependent Thal patients despite improved chelation agent availability and tissue iron monitoring. Future long-term longitudinal and intervention studies elucidating and mitigating potential effects of iron overload on developing children's developing endocrine organs are needed. Presentation: Sunday, June 12, 2022 12:30 p.m. - 2:30 p.m.

Short version of the S2k guideline on drug therapy of neurogenic lower urinary tract dysfunction (NLUTD)

In Germany about one million patients suffer from neurogenic lower urinary tract dysfunction (NLUTD). If left untreated, various forms of NLUTD can lead to secondary damage of the lower and upper urinary tract. Thus, the guideline was developed for the drug therapy of patients with NLUTD, who frequently require lifelong care and aftercare. The guideline was developed in aconsensus process with several meetings and online reviews, and final recommendations were decided on in online consensus meetings. Ballots were sent to elected officials of the contributing professional societies. Level of consensus was given for each coordinated recommendation ( https://www.awmf.org/leitlinien/detail/ll/043-053.html ). RESULTS/MOST IMPORTANT RECOMMENDATIONS: (Video)urodynamic classification of the NLUTD should be conducted before the use of antimuscarinic drugs (84.2%). Approved oral antimuscarinics should be used as first choice. Contraindications must be respected (100%). If oral treatment is ineffective or in the case of adverse drug reaction (ADRs) alternatively instillation of oxybutynin solution intravesically (83%) or onabotulinumneurotoxine (OBoNT) injection should be offered (89.5%). In case of failure or ADRs of antimuscarinics, β3 sympathomimetic mirabegron can be used to treat neurogenic detrusor overactivity (NDO) (off-label use) (100%). In case of paraplegia below C8 or multiple sclerosis with an expanded disability status scale (EDSS) of ≤ 6.5, OBoNT injection can be offered as an alternative (89.5%). Drug therapy for NDO should be started early in newborns/young children (84.2%). Conservative, nondrug therapy should be considered in frail elderly (94.7%). No parasympathomimetic therapy should be used to treat neurogenic detrusor underactivity (94.7%). Precise knowledge of the neurological underlying disease/sequence of trauma and the exact classification of the NLUTD are required for development of individualized therapy.

Altered Extracellular Matrix as an Alternative Risk Factor for Epileptogenicity in Brain Tumors.

Seizures are one of the most common symptoms of brain tumors. The incidence of seizures differs among brain tumor type, grade, location and size, but paediatric-type diffuse low-grade gliomas/glioneuronal tumors are often highly epileptogenic. The extracellular matrix (ECM) is known to play a role in epileptogenesis and tumorigenesis because it is involved in the (re)modelling of neuronal connections and cell-cell signaling. In this review, we discuss the epileptogenicity of brain tumors with a focus on tumor type, location, genetics and the role of the extracellular matrix. In addition to functional problems, epileptogenic tumors can lead to increased morbidity and mortality, stigmatization and life-long care. The health advantages can be major if the epileptogenic properties of brain tumors are better understood. Surgical resection is the most common treatment of epilepsy-associated tumors, but post-surgery seizure-freedom is not always achieved. Therefore, we also discuss potential novel therapies aiming to restore ECM function.

EXCESS TIME TO ADULT CONGENITAL HEART DISEASE CARE

Over 90% of children with congenital heart disease (CHD) reach adulthood. Many of these individuals require lifelong cardiology care. Loss to follow-up predisposes patients to late recognition of cardiac complications. However, whether or not a young adult attends an ACHD clinic is a crude outcome variable. Rather, the time between the final pediatric visit and the first ACHD visit, over and above what was recommended by the referring pediatric cardiologist, is a variable that captures not only whether a patient was seen in an ACHD clinic but also the time delay, if any, in arriving there.

Paediatrician's guide to post-operative care for biventricular CHD: a review.

Paediatricians play an integral role in the lifelong care of children with CHD, many of whom will undergo cardiac surgery. There is a paucity of literature for the paediatrician regarding the post-operative care of such patients. The aim of this manuscript is to summarise essential principles and pertinent lesion-specific context for the care of patients who have undergone surgery or intervention resulting in a biventricular circulation. Familiarity with common issues following cardiac surgery or intervention, as well as key details regarding specific lesions and surgeries, will aid the paediatrician in providing optimal care for these patients.

Exploring the Economic Aspects of β-Thalassemia in Jordan in 2019

Thalassemia are inherited hematological disorders considered among the most common genetic disorders worldwide, occurring more frequently in the Mediterranean Region. The WHO estimates that Beta-thalassemia affects 2.9% of the world’s population. In Jordan, the carrier prevalence rate of thalassemia is from 2-4%. Patients with thalassemia need a lifelong care, devastating their quality of life and imposing overwhelming psychological and financial burden on patients and their families. The Jordanian Ministry of Health (MOH) is the sole facility responsible for treating these patients from the pre-marital program until required medications regardless of their nationality. This study aimed to estimate the economic burden of thalassemia in Jordan in 2019. All 680 thalassemia patients admitted to thalassemia centers in Jordan and coming to out-patients’ clinics from July 1st to Aug 31st, 2019 are included. Data were collected using a pre-developed questionnaire from the electronic medical records. The economic burden was estimated from MOH perspective and societal perspective. The average annual cost was estimated to be 2,674 JOD for a single thalassemia Jordanian insured patient and 4,627 JOD for un-insured, while the non-Jordanian patient’ annual cost was estimated 4,751 JOD if insured and 6,651 JOD if un-insured. The total economic burden of thalassemia in Jordan in 2019 was estimated to be 2,148,741 JOD. Of this amount, 1,393,329 JOD was for Jordanians and 755,412 JOD for non-Jordanians. In conclusion, this high burden of thalassemia in Jordan requires adopting new controlling policies; pre-marriage counseling, education and raising awareness should be encouraged.

Status of adult outpatients with congenital heart disease in Japan: The Japanese Network of Cardiovascular Departments for Adult Congenital Heart Disease Registry

BackgroundThe Japanese Network of Cardiovascular Departments for Adult Congenital Heart Disease (JNCVD-ACHD) was founded in 2011 for the lifelong care of adult patients with congenital heart disease (ACHD patients). This network maintains the first Japanese ACHD registry. Methods and resultsFrom 2011 to 2019, the JNCVD-ACHD registered 54 institutions providing specialized care for ACHD patients in 32 of the 47 prefectures in Japan. The registry collected data on the disease profile for 24,048 patients from 50 institutions and the patient characteristics for 9743 patients from 24 institutions. The most common ACHDs were atrial septal defect (20.5 %), ventricular septal defect (20.5 %), tetralogy of Fallot (12.9 %), and univentricular heart (UVH)/single ventricle (SV; 6.6 %). ACHD patients without biventricular repair accounted for 37.0 % of the population. Also examined were the serious anatomical and/or pathophysiological disorders such as pulmonary arterial hypertension (3.0 %) including Eisenmenger syndrome (1.2 %), systemic right ventricle under biventricular circulation (sRV-2VC; 2.8 %), and Fontan physiology (6.0 %). The sRV-2VC cases comprised congenitally corrected transposition of the great arteries without anatomical repair (61.9 %) and transposition of the great arteries with atrial switching surgery (38.1 %). The primary etiology (86.4 %) for Fontan physiology was UVH/SV. In addition, developmental/chromosomal/genetic disorders were heterotaxy syndromes (asplenia, 0.9 %; polysplenia, 0.7 %), trisomy 21 (4.0 %), 22q11.2 deletion (0.9 %), Turner syndrome (0.2 %), and Marfan syndrome (1.1 %). ConclusionsAlthough the specific management of ACHD has systematically progressed in Japan, this approach is still evolving. For ideal ACHD care, the prospective goals for the JNCVD-ACHD are to create local networks and provide a resource for multicenter clinical trials to support evidence-based practice.

Tuberous sclerosis complex

Tuberous sclerosis complex (TSC) is aphakomatosis and is atumor predisposition syndrome. As agenetic multisystem disease, patients present with a broad range of changes in the brain, heart, skin, kidneys, and lungs. Which imaging modalities are required to monitor TSC patients according to current international recommendations? Common findings in TSC are cortical tubers, subependymal nodules, and giant cell astrocytomas in the central nervous system (CNS), rhabdomyomas in the heart, and cysts and angiomyolipomas in the kidneys. Magnetic resonance imaging (MRI) of the brain and kidneys and abdominal ultrasound are the imaging modalities of choice, due to the very good soft tissue contrast and lack of X‑ray radiation. Using standard and functional MRI sequences in a multimodal approach, the type, malignancy, size, and morphology of changes in TSC can be reliably determined. Abdominal ultrasound using high-resolution transducers can be used to rapidly and reliably detect even the smallest changes in the kidneys. Regular follow-up of patients with TSC using MRI and ultrasound is necessary for early detection of complications, for planning individualized therapy, and for optimal lifelong care.

Development of a Self-management and Peer-Mentoring Intervention to Improve Transition Readiness Among Young Adult Survivors of Pediatric Cancer: Formative Qualitative Research Study.

BackgroundChildhood cancer survivors require lifelong risk-based follow-up care. It should be noted that less than one-third of adult survivors of childhood cancer report any survivor-focused care, and fewer than 1 in 5 obtain risk-based follow-up care. It is thought that this may be due to inadequate transition readiness, including low levels of knowledge, skills, motivation, and resources to make the transition to independent self-management of follow-up care. Interventions that focus specifically on improving the transition from parent-managed to self-managed care are needed. Theory and prior research suggest that targeting self-management skills and using peer mentoring may be innovative strategies to improve transition readiness.ObjectiveThis study aims to identify the content of a self-management intervention to improve transition readiness among adolescent and young adult (AYA) survivors.MethodsIntervention development occurred in 3 stages: formative research with AYA survivors to identify barriers and facilitators to obtaining risk-based survivorship care, content development using feedback from multiple stakeholders (AYA survivors, parents, and providers), and content refinement (usability testing) of the initial proposed educational modules for the program. Content analysis, guided by the social-ecological model of AYA readiness for transition, was used to identify themes and develop and refine the content for the intervention.ResultsA total of 19 AYA survivors participated in the formative research stage, and 10 AYA survivors, parents, and health care providers participated in the content development and refinement stages. The major barrier and facilitator themes identified included knowledge of cancer history and risks; relationships with health care providers; relationships with family members involved in care; emotions about health, follow-up care, and transfer of care; and lifestyle behaviors and life transitions. These themes were translated into 5 self-management modules: understanding treatment history and the survivorship care plan, managing health care logistics and insurance, communicating with health care providers and family members involved in care, dealing with emotions, and staying healthy in the context of life transitions. Feedback from the key stakeholders indicated that the content was relevant but should include participative elements (videos and tailored feedback) to make the intervention more engaging. The AYA survivors were receptive to the idea of working with a peer mentor and expressed a preference for using SMS text messaging, telephone calls, or videoconference to communicate with their mentor.ConclusionsIncorporating AYA survivors, parents, and providers in the design was essential to developing the content of a self-management and peer-mentoring intervention. AYA survivors confirmed the important targets for the intervention and facilitated design decisions in line with our target users’ preferences. The next step will be to conduct a single-arm trial to determine the feasibility and acceptability of the proposed intervention among AYA survivors of childhood cancer.

2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia.

Beta-thalassemia and particularly its transfusion-dependent form (TDT) is a demanding clinical condition, requiring life-long care and follow-up, ideally in specialized centers and by multidisciplinary teams of experts. Despite the significant progress in TDT diagnosis and treatment over the past decades that has dramatically improved patients’ prognosis, its management remains challenging. On one hand, diagnostic and therapeutic advances are not equally applied to all patients across the world, particularly in several high-prevalence eastern regions. On the other, healthcare systems in low-prevalence western countries that have recently received large numbers of migrant thalassemia patients, were not ready to address patients’ special needs. Thalassaemia International Federation (TIF), a global patient-driven umbrella federation with 232 member-associations in 62 countries, strives for equal access to quality care for all patients suffering from thalassemia or other hemoglobinopathies in every part of the world by promoting education, research, awareness, and advocacy. One of TIF’s main actions is the development and dissemination of clinical practice guidelines for the management of these patients. In 2021, the fourth edition of TIF’s guidelines for the management of TDT was published. The full text provides detailed information on the management of TDT patients and the clinical presentation, pathophysiology, diagnostic approach, and treatment of disease complications or other clinical entities that may occur in these patients, while also covering relevant psychosocial and organizational issues. The present document is a summary of the 2021 TIF guidelines for TDT that focuses mainly on clinical practice issues and recommendations.

What Counts for the Old and Oldest Old?-An Analysis of Patient Criteria for Choosing a Dentist-Part I: Awareness and Selection Criteria, Infrastructure, and Dental Office Equipment.

Utilization of a dentist is influenced by many factors. The aim of this study is to present the factors relating to how patients become aware of a dentist, according to which criteria they select the dentist, and which factors in the infrastructure, equipment of dental offices, and human interactions are important for patients. A telephone survey with 466 participants (female 59.9%) in three age groups (ag 1: 35–50 years, ag 2: 70–84 years, ag 3: >85 years) in three German cities was conducted. Data were analyzed with respect to age, gender, and place of residence. Hardly any differences in the selection of the dentist and the selection criteria applied were found between the sexes, the age groups, or the places of residence. Recommendation seems to be the major aspect regarding how patients become aware of or select their dentist (n = 278, 65.6%), while modern technologies, e.g., the internet, play a subordinate role (n = 31, 7.3%). The unimportance of modern technologies increases significantly with the increase in age. As age increases, factors such as infrastructure (e.g., elevator available (ANOVA p < 0.001; Bonferoni correction: significant differences between ag 1 and ag 2 p < 0.001, ag 1 and ag 3 p < 0.001, and ag 2 and ag 3 p = 0.009); accessibility by wheelchair (ANOVA p < 0.001; Bonferoni correction: significant differences between ag 1 and ag 2 p = 0.006; and ag 1 and ag 3 p < 0.001); etc.) and dental office equipment become significantly important and influence the choice of dentist, while the importance of good parking facilities significantly decreased with age (ANOVA p = 0.003; Bonferoni correction: significant differences between ag 1 and ag 3 p = 0.004, and ag 2 and ag 3 p = 0.023). With increasing age, e.g., the importance of a television in the waiting room (ANOVA p = 0.012; Bonferoni correction: significant differences between ag 1 and ag 3 p = 0.014; and ag 2 and ag 3 p = 0.011), a modern waiting room (ANOVA p < 0.001; Bonferoni correction: significant differences between ag 1 and ag 3 p < 0.001; and ag 2 and ag 3 p < 0.001) or the possibility to visualize the oral situation on a screen decreases significantly (ANOVA p < 0.001; Bonferoni correction: significant differences between ag 1 and ag 2 p < 0.001; ag 1 and ag 3 p < 0.001, and ag 2 and ag 3 p < 0.001). If dentists want to welcome and treat older people, they should adapt the accessibility, infrastructure and equipment of their practice to the needs of older people in order to be able to guarantee continuous lifelong dental care regardless of the need for assistance or care.

Preventing type 1 diabetes development and preserving beta-cell function.

Type 1 diabetes (T1D) is the most common chronic disease of childhood presenting a significant burden, both in terms of day-to-day medical management and lifelong care. Studies aligned with diverse strategies to prevent or modify the course of T1D are reviewed. The diagnosis of T1D precedes the classic clinical presentation when insulin dependence develops. With an increased understanding of the pathophysiology of the autoimmune process leading to T1D, treatment strategies to prevent the development of autoimmunity and/or modify the immune response have been trialed in persons at risk for developing the disease. Interventions prior to insulin dependence or very early after clinical diagnosis show some promise both in preventing disease onset and prolonging beta-cell insulin production. Significant progress has been made in the treatment of T1D. However, suboptimal glycemic control remains a challenge impacting overall health and quality of life for patients with this chronic disease. Although physicians and basic sciences investigators continue to pursue the prevention of the autoimmune process, the advent of disease-modifying agents is a promising strategy. Further studies are needed to ensure that insulin preservation can be achieved longer term.

Social management of patients affected by a bone growth problem

Children and adolescents with a bone growth disorder need lifelong medical care. The establishment of a social support system allows them to benefit from the necessary assistance for the evolution of their pathology, from birth to their entry into school and then into professional life.

Models of lifelong care for children and adolescents with chronic conditions in low-income and middle-income countries: a scoping review

Globally, non-communicable diseases (NCDs) or chronic conditions account for one-third of disability-adjusted life-years among children and adolescents under the age of 20. Health systems must adapt to respond to the...

CO113 Clinical Outcomes of Patients with Hemophilia Receiving Different Treatment Regimens in China: Based on Patient-Reported Data

Hemophilia is a rare disease hereditary bleeding disorder which needed continuous treatment and life-long care. Regular prophylaxis treatment (RPT) is recommended as a standard treatment regimen, while intermittent prophylaxis treatment (IPT) and on-demand treatment (ODT) is commonly utilized in reality under resource constrain. In China, approximately 39,000 patients live with hemophilia. However, little is known about patients’ treatment model in real life and related clinical outcomes. The aim of this study was to analyze patient-reported clinical outcomes with three hemophilia treatment regimens in real world in China.