Now showing items 1-20 of 185

    • Burkitt Lymphoma/Leukaemia in Children & Young Adolescents.

      O'Rourke, E; Malone, A; O'Marcaigh, A; Storey, L; Betts, D; McDermott, M; Smith, O P (2020-01-16)
    • Adolescents Presenting with Mental Health Crises.

      Gadancheva, V; Barry, H; McNicholas, F (2019-12-16)
      Aims The aim of the study is to examine emergency mental health (MH) presentations and assessments of adolescents (16-18) presenting to a Dublin adult emergency department (ED). Methods Clinical data was collected on all patients over a 12 month period. Results Seventy seven adolescents presented, the majority out of hours (n=58, 75%) and with either self-harm (n=34, 44%) or suicidal ideation (n=28, 36%), where females significantly outnumbered males (89% vs 66%; p=.028). Other presentations included low mood, anxiety and behavioural problems, and following assessment from an adult mental health service team member, 55% (n=39) were given an Axis I psychiatric diagnosis. Almost all adolescents were discharged following assessment (n=68, 96%), the majority being referred on to Child and Adolescent Mental Health Service (CAMHS) (55, 78%). Conclusion Crisis presentations to EDs often occur in adolescents with co-existing psychiatric disorders, and require skilled and therapeutic assessment. Knowledge of appropriate services for onward referral is essential, and highlight the importance of a close collaborative between adult EDs and CAMHS.
    • Cyanotic Congenital Heart Disease Modes of Presentation and Prenatal Detection.

      Lynch, Á; Ng, L; Lawlor, P; Lavelle, M; Gardner, F; Breatnach, C; McMahon, C J; Franklin, O (2019-12-16)
      Prenatal detection of structural congenital heart disease (CHD) optimises cardiovascular stability pre-operatively and post-operative outcomes. We compared prenatal detection rates of critical CHD in units offering universal fetal anomaly scans with those offering imaging to selected women. One hundred and thirteen infants met inclusion criteria. The overall pre-natal detection rate for critical CHD was 57% of liveborn infants. It was 71% (57/80) in hospitals who offered a universal anomaly scan and 29% (9/31) in centres offering a limited service. Postnatal diagnosis was associated with PICU admission (p=0.016) and preoperative mechanical ventilation (p=0.001). One-year mortality was 10 fold higher in the postnatally diagnosed group 15% vs 1.55% (p=0.0066). There is a significant disparity between centres offering universal anomaly versus selective screening. Prenatal detection confers advantage in terms of pre-operative stability and one year survival. Failure to deliver an equitable service exposes infants with CHD to avoidable risk. Abreviations CHD, congenital heart disease; PICU, paediatric intensive care unit; NWIHP, National Women and Infants Health Programme; NCHC, CHIC, National Children’s Heart Centre at Children’s Health Ireland at Crumlin; TAPVD, total anomalous pulmonary venous drainage, HLHD, Hypoplastic left heart disease, HRHD, Hypoplastic right heart disease, LVOTO, left ventricular outflow tract obstruction.
    • Risk Factors for Respiratory Syncytial Virus Bronchiolitis Admissions.

      Meenaghan, S; Breatnach, C; Smith, H (2020-01-16)
      Aim Determine the seasonal incidence of hospital Respiratory Syncytial Virus (RSV) bronchiolitis and explore the variables associated with admission to ward versus the Paediatric Intensive Care Unit (PICU). Method Retrospective case-control study. Children, aged ≤2 years, between November and March, over a 3 year period with a positive RSV nasopharyngeal aspirate test. Results A total of 557 children were included; 19% (n=106) required PICU admission. Children admitted to the PICU were younger in age, median (IQR) 6.93 (3.96, 11.89) weeks compared to children who remained on the wards 11.00 (5.86, 24.14) weeks. Being underweight at the point of admission (adjusted odds ratio 3.15, 95% 1.46, 6.70, p=0.003) was associated with a PICU admission. Conclusion Number of RSV bronchiolitis hospitalisations are increasing each year. Age, weight and the use of HFNC were independent predictors for PICU admission.
    • Duplication of referral, a tsunami of paper: how much does it cost the Irish health services?

      Walsh, Nicola; Malone, Lisa; Lynch, Sally Ann (2022-01-10)
      Background: Prolonged waiting lists increase costs as medical problems may become more expensive to fix. There are also hidden financial costs. Irish Clinical Genetic services have long out-patient waiting times. We noticed duplicate referrals (patients on the waiting list) being re-referred because the patient still had not been seen. These re-referrals waste consultant and administrative time, pose a clinical risk by distracting clinician time, and are costly to our health service. Methods: We prospectively collected duplicate referral data over a 3-month period (1 October 2020-31 January 2021) in order to estimate costs. We costed (1) referring consultant and administrative time; (2) stationary, postage, and storage cost; and (3) receiving consultant and administrative time processing these referrals. Results: We noted 82/986 (8%) referrals to our service over the trial period were duplicate. The mean length of time between first and duplicate referral was 306 days. In 35/82 (42.68%), a duplicate referral had already been received (e.g. 3rd or more referral for same patient). In total, we received 132 re-referral letters for 82 patients. Duplicate referrals changed triage outcome in 7/82 (8.54%) cases. Conclusion: National Treatment Purchase Fund data suggests that 271,560 patients are waiting > 12 months for both in- and out-patient public appointments on 1 January 2021. Assuming duplicate referrals are occurring across the Irish health system with equal frequency after 12 months of waiting (8% of total appointments), then we estimate a conservative cost of 757,392 € per quarter to the health service and an annual cost to the HSE of 3,029,568 €.
    • COVID-19-related occupational stress in staff in an acute paediatric teaching hospital in Ireland.

      Murray, Johanna; Adamis, Dimitrios; mcnicholas, fiona
      Background: The COVID-19 pandemic has resulted in major strains for healthcare staff. Objectives: This study aims to assess prevalence of occupational burnout (BO) during COVID-19 in staff working in an acute paediatric hospital setting. Participants: One hundred and thirty-three staff, out of 1900 eligible staff (9.6% response rate), completed an online or paper and pencil survey. Methods: The Copenhagen Burnout Inventory was used as the main outcome measure. Additional questions examined the impact of COVID-19 and restrictions on work setting and personal health. Results: The majority of respondents reported moderate or higher levels of BO for personal (n=93; 70%) and work domains (n=83; 62%). Rates of patient-related BO were lower (n=18; 13%). Higher rates of BO were found in staff with self-rated COVID-19 adverse effects on physical (n=50, 38%) and mental health (n=88, 66%) (F (2, 13.019)=16.019, p<0.001). The majority of staff had no stress reduction training at any stage in their career, either professional (60%), on the job (62%) or postpandemic (59%) work. Although most (82%) were aware of occupational health supports, few (30%) reported an intention to access these if needed; 65% (n=86) of the respondents seriously considered changing jobs in the last 6-12 months. Conclusion: High level of occupational stress among hospital staff during COVID-19, in the absence of stress reduction training is a risk factor for BO. Interventions, acceptable to the employee, are urgently needed given the likelihood of additional work demands as COVID-19 continues.
    • Exploring carer burden amongst those caring for a child or adolescent with an eating disorder during COVID-19.

      Maunder, Kristen; mcnicholas, fiona (2021-10-03)
      Background: Carer burden amongst carers of youth with an eating disorder is substantial and if not addressed can lead to negative outcomes for the patient, carer and family. The Coronavirus Disease 2019 (COVID-19) pandemic has made caring for youth with an ED even more onerous and preliminary research is beginning to emerge demonstrating the profound negative impact the pandemic is having upon individuals with EDs and their carers. MAIN: In this review, we briefly summarize what is known about carer burden in families where a young person has an ED, consider the additional impact consequent to COVID-19 and highlight the need for interventions aimed at alleviating this. Pre-COVID-19 research identifies high levels of psychological and physical strain amongst those caring for a child with an ED. Themes are beginning to emerge as to why COVID-19 may further exacerbate carer burden: (1) reduced access to ED services; (2) increased physical vulnerability and exacerbation of psychiatric co-morbidity amongst youth with EDs; (3) increased practical demands placed on carers; and (4) social isolation and decreased social support. Conclusion: The COVID-19 pandemic poses a specific threat to the mental health of youth with EDs and their carers. Given the salient role families play in caring for youth with an ED, attending to carer burden is imperative. Supporting carers through all phases of their child's ED journey by offering adaptive and flexible supportive services which accommodate time constraints, geographic barriers and possible COVID-19 spread is essential.
    • COVID-19 related occupational stress in teachers in Ireland.

      Minihan, Elisha; Adamis, Dimitrios; Dunleavy, Michele; Martin, Angela; Gavin, Blanaid; mcnicholas, fiona (2021-12-21)
      Moderate or high levels of personal burnout was reported by 82% (n = 202) of the sample and 79% (n = 193) reported work burnout. COVID-19 related adverse effects were reported by teachers on physical (43%) and mental health (67%), with deterioration in eating (34%), sleeping (70%) and alcohol use (33%). 100 (42%) participants felt unable to keep safe at work. Low levels of job satisfaction were present (66%), negatively correlating with burnout scores (rs=-0.405, p<.01). 142 (58%) teachers had seriously considered changing jobs in the previous 6-12 months.
    • A model for occupational stress amongst paediatric and adult critical care staff during COVID-19 pandemic.

      Feeley, T; Ffrench-O'Carroll, R; Tan, M H; Magner, C; L'Estrange, K; O'Rathallaigh, E; Whelan, S; Lyons, B; O'Connor, E (2021-02-25)
      Purpose: The coronavirus 2019 pandemic has placed all intensive care unit (ICU) staff at increased risk of psychological distress. To date, measurement of this distress has largely been by means of validated assessment tools. We believe that qualitative data may provide a richer view of staff experiences during this pandemic. Methods: We conducted a cross-sectional, observational study using online and written questionnaires to all ICU staff which consisted of validated tools to measure psychological distress (quantitative findings) and open-ended questions with free-text boxes (qualitative findings). Here, we report our qualitative findings. We asked four questions to explore causes of stress, need for supports and barriers to accessing supports. A conventional content analysis was undertaken. Results: In total, 269 of the 408 respondents (65.9%) gave at least one response to a free-text question. Seven overarching themes were found, which contribute to our proposed model for occupational stress amongst critical care staff. The work environment played an important role in influencing the perceived psychological impact on healthcare workers. Extra-organisational factors, which we termed the "home-work interface" and uncertainty about the future, manifested as anticipatory anxiety, had a proportionally larger influence on worker well-being than would be expected in non-pandemic conditions. Conclusion: Our findings have important implications for appropriate allocation of resources and ensuring well-being of the ICU multidisciplinary team for this and future pandemics. Keywords: Anticipatory anxiety; COVID-19; Critical care staff; Home-work interface; Occupational stress; Pandemic; Work intensification.
    • Clinical experience with the AKT1 inhibitor miransertib in two children with PIK3CA-related overgrowth syndrome.

      Forde, Karina; Resta, Nicoletta; Ranieri, Carlotta; Rea, David; Kubassova, Olga; Hinton, Mark; Andrews, Katrina A; Semple, Robert; Irvine, Alan D; Dvorakova, Veronika (2021-02-27)
      Background: PIK3CA-related overgrowth spectrum (PROS) refers to a group of rare disorders, caused by somatic activating mutations in PIK3CA, resulting in abnormal PI3K-AKT-mTOR pathway signalling. Significant associated morbidity is frequently observed, and approved treatments are lacking. Miransertib (ARQ 092) is a novel, orally available, selective pan-AKT inhibitor with proven in vitro efficacy. Following recent results of the use of AKT inhibitors in Proteus syndrome (PS) and AKT-mutant cancers, we investigated its therapeutic use in two patients with severe PROS who had exhausted conventional treatment methods. Results: Two patients, one with CLOVES variant (P1) and one with facial infiltrating lipomatosis and hemimegalencephaly (P2), were commenced on miransertib treatment on a compassionate use basis. In patient one, intra-abdominal and paraspinal overgrowth had resulted in respiratory compromise, obstructive uropathy, dysfunctional seating and lying postures, and chronic pain. In patient two, hemifacial overgrowth and hemimegalencephaly had caused difficulties with articulation and oral function, and refractory epilepsy. Miransertib treatment was continued for a median duration of 22 months (range 22-28). In patient one, alleviation of respiratory compromise was observed and functionally, seating and lying postures improved. Serial volumetric MRI analysis revealed 15% reduction in calculated volumes of fatty overgrowth between treatment commencement and end. In patient two, reduction in seizure burden and improved parent-reported quality of life measures were reported. Treatment was discontinued in both patients due to lack of sustained response, and poor compliance in year two of treatment (P2). No significant toxicities were reported. Conclusion: We report the first paediatric case series of the use of miransertib in two children with PROS. Objective clinical response was observed in patient one, and improvement in key qualitative outcomes was reported in patient two. Treatment was well tolerated with no significant toxicities reported. This case series highlights the potential therapeutic utility of miransertib in selected paediatric patients with severe PROS, and further demonstrates the potential for re-purposing targeted therapies for the treatment of rare diseases. An open label, Phase 1/2 study of miransertib in children with PROS and PS is underway to more accurately assess the efficacy of miransertib in the treatment of PROS disorder (NCT03094832).
    • Opportunities to Target T Cell Trafficking in Pediatric Inflammatory Bowel Disease.

      Giannoudaki, Eirini; Gargan, Siobhan; Hussey, Seamus; Long, Aideen; Walsh, Patrick T (2021-03-18)
      T cell subsets are considered central orchestrators of inflammation and homeostasis in the intestine and are established targets for the treatment of inflammatory bowel disease. While approaches aimed at the neutralization of T cell effector cytokines have provided significant benefits for pediatric and adult patients, more recent strategies aimed at inhibiting the infiltration of pathogenic T cell subsets have also emerged. In this review, we describe current knowledge surrounding the function of T cell subsets in pediatric inflammatory bowel disease and outline approaches aimed at targeting T cell trafficking to the intestine which may represent a new treatment option for pediatric inflammatory bowel disease.
    • Medical education and training within congenital cardiology: current global status and future directions in a post COVID-19 world.

      McMahon, Colin J; Tretter, Justin T; Redington, Andrew N; Bu'Lock, Frances; Zühlke, Liesl; Heying, Ruth; Mattos, Sandra; Krishna Kumar, R; Jacobs, Jeffrey P; Windram, Jonathan D (2021-04-12)
      Despite enormous strides in our field with respect to patient care, there has been surprisingly limited dialogue on how to train and educate the next generation of congenital cardiologists. This paper reviews the current status of training and evolving developments in medical education pertinent to congenital cardiology. The adoption of competency-based medical education has been lauded as a robust framework for contemporary medical education over the last two decades. However, inconsistencies in frameworks across different jurisdictions remain, and bridging gaps between competency frameworks and clinical practice has proved challenging. Entrustable professional activities have been proposed as a solution, but integration of such activities into busy clinical cardiology practices will present its own challenges. Consequently, this pivot towards a more structured approach to medical education necessitates the widespread availability of appropriately trained medical educationalists, a development that will better inform curriculum development, instructional design, and assessment. Differentiation between superficial and deep learning, the vital role of rich formative feedback and coaching, should guide our trainees to become self-regulated learners, capable of critical reasoning yet retaining an awareness of uncertainty and ambiguity. Furthermore, disruptive innovations such as "technology enhanced learning" may be leveraged to improve education, especially for trainees from low- and middle-income countries. Each of these initiatives will require resources, widespread advocacy and raised awareness, and publication of supporting data, and so it is especially gratifying that Cardiology in the Young has fostered a progressive approach, agreeing to publish one or two articles in each journal issue in this domain.
    • Smartphone Usage Among Doctors in the Clinical Setting in Two Culturally Distinct Countries: Cross-sectional Comparative Study.

      Nair, Anjali Ajay; Afroz, Samreen; Ahmed, Bushra Urooj; Ahmed, Uzma Urooj; Foo, Chi Chung; Zaidan, Hind; Corbally, Martin (2021-05-10)
      Background: Smartphones and mobile applications have seen a surge in popularity in recent years, a pattern that has also been reflected in the health care system. Despite increased reliance among clinicians however, limited research has been conducted on the uptake and impact of smartphone usage in medical practice, especially outside the Western world. Objective: This study aimed to identify the usage of smartphones and medical apps by doctors in the clinical setting in 2 culturally distinct countries: King Hamad University Hospital (KHUH), Bahrain and Queen Mary Hospital (QMH), Hong Kong. Methods: A cross-sectional, comparative study was conducted where doctors in both hospitals were asked to take part in a 15-item online survey. The questions were categorized into the following groups: demographics of the study population, ownership and main use of smartphones, number and names of medical apps currently owned, rating usage of smartphones for medical purposes, time spent on a smartphone related to clinical use, clinical reliance on smartphones, and views on further integration of smartphones. The results were then tabulated and analyzed using SPSS Statistics 25 for Mac (IBM Corp Inc, Armonk, NY). Results: A total of 200 doctors were surveyed, with a total of 99.0% (99/100) of the doctors owning a smartphone in both KHUH and QMH; 58% (57/99) and 55% (54/99) of the doctors from KHUH and QMH, respectively, identified communication as their main use of smartphones in the clinical setting (P=.004). Doctors from KHUH were likely to spend more time on medical apps than doctors from QMH (P=.002). According to the overall results of both hospitals, 48% (32/67) of the junior doctors claimed high reliance on smartphones, whereas only 32.3% (41/127) of the senior doctors said the same (P=.03). Of doctors in KHUH and QMH, 78.0% (78/100) and 69.0% (69/100), respectively, either strongly agreed or agreed that smartphones need to be integrated into the clinical setting. In terms of preferences for future apps, 48% (48/100) and 56% (56/100) of the doctors in KHUH and QMH, respectively, agreed that more medical applications need to be created in order to support smartphone use in the clinical setting. Conclusions: These results suggest a substantial acceptance of smartphones by doctors in the clinical setting. It also elicits the need to establish policies to officially integrate smartphone technology into health care in accordance with ethical guidelines. More emphasis should be placed on creating medical applications that aid health care professionals in attaining their information from accurate sources and also regulate a system to monitor the usage of mobile devices within hospitals to prevent a breach of patient privacy and confidentiality.
    • One and done? Outcomes from 3961 patients managed via a virtual fracture clinic pathway for paediatric fractures.

      Kennedy, Jim; Blackburn, Carol; Barrett, Michael; O'Toole, Patrick; Moore, David (2021-06-01)
      Purpose: The aim of this paper is to describe our experience with a virtual fracture management pathway in the setting of a paediatric trauma service. Methods: All patients referred to the virtual fracture clinic service from the Paediatric Emergency Department (PED) were prospectively collected. Outcome data of interest (patients discharged, referred for urgent operative treatment, referred back to emergency department for further evaluation, referred for face-to-face clinical assessment and all patients who re-presented on an unplanned basis for further management of the index injury) were compiled and collated. Cost analysis was performed using established costing for a virtual fracture clinic within the Irish Healthcare System. Results: There were a total of 3961 patients referred to the virtual fracture clinic from the PED. Of these, 70% (n = 2776) were discharged. In all, 26% (n = 1033) were referred to a face-to-face appointment. Of discharged patients, 7.5% (n = 207) required an unplanned face-to-face evaluation. A total of 0.1% (n = 3) subsequently required operative treatment relating to their index injury. Implementation of the virtual fracture clinic model generated calculated savings of €254 120. Conclusion: This prospective evaluation has demonstrated that a virtual fracture clinic pathway for minor paediatric trauma is safe, effective and brings significant cost savings.
    • Recombinant factor VIII Fc for the treatment of haemophilia A.

      Hermans, Cedric; Mancuso, Maria Elisa; Nolan, Beatrice; Pasi, K John (2021-03-31)
      Prophylaxis with factor VIII (FVIII) is the current therapeutic approach for people with haemophilia A. However, standard half-life (SHL) FVIII products must be injected frequently, imposing a substantial burden on the individual and making it difficult to tailor therapy according to patient need and lifestyle, which could impact adherence. Recombinant FVIII Fc fusion protein (rFVIIIFc; Elocta® , Sobi; Eloctate® , Sanofi) is a recombinant fusion protein that undergoes slower clearance from the body than SHL FVIII products. This pharmacokinetic property of rFVIIIFc allows prophylactic administration every 3-5 days, or once weekly in selected patients, with doses adjusted to patient needs and clinical outcomes. Higher FVIII levels can be achieved maintaining dosing frequency similar to that usually applied with SHL FVIII. This review provides a summary of recent data from the A-LONG, Kids A-LONG, ASPIRE and PUPs A-LONG studies and recently published real-world experience relevant to rFVIIIFc use in individualised regimens. The review also introduces ongoing studies of rFVIIIFc, including its use for induction of immune tolerance, and discusses some aspects to consider when switching patients to rFVIIIFc and managing ongoing treatment. In summary, rFVIIIFc is suitable for individualised prophylaxis regimens that can be tailored according to patient clinical needs and lifestyle.
    • Novel clinical and genetic insight into CXorf56-associated intellectual disability.

      Rocha, Maria Eugenia; Silveira, Tainá Regina Damaceno; Sasaki, Erina; Sás, Daíse Moreno; Lourenço, Charles Marques; Kandaswamy, Krishna K; Beetz, Christian; Rolfs, Arndt; Bauer, Peter; Reardon, Willie; et al. (2019-12-10)
      Intellectual disability (ID) is one of most frequent reasons for genetic consultation. The complex molecular anatomy of ID ranges from complete chromosomal imbalances to single nucleotide variant changes occurring de novo, with thousands of genes identified. This extreme genetic heterogeneity challenges the molecular diagnosis, which mostly requires a genomic approach. CXorf56 is largely uncharacterized and was recently proposed as a candidate ID gene based on findings in a single Dutch family. Here, we describe nine cases (six males and three females) from three unrelated families. Exome sequencing and combined database analyses, identified family-specific CXorf56 variants (NM_022101.3:c.498_503del, p.(Glu167_Glu168del) and c.303_304delCTinsACCC, p.(Phe101Leufs*20)) that segregated with the ID phenotype. These variants are presumably leading to loss-of-function, which is the proposed disease mechanism. Clinically, CXorf56-related disease is a slowly progressive neurological disorder. The phenotype is more severe in hemizygote males, but might also manifests in heterozygote females, which showed skewed X-inactivation patterns in blood. Male patients might present previously unreported neurological features such as epilepsy, abnormal gait, tremor, and clonus, which extends the clinical spectrum of the disorder. In conclusion, we confirm the causative role of variants in CXorf56 for an X-linked form of intellectual disability with additional neurological features. The gene should be considered for molecular diagnostics of patients with ID, specifically when family history is suggestive of X-linked inheritance. Further work is needed to understand the role of this gene in neurodevelopment and intellectual disability.
    • Protocol for a prospective, observational, longitudinal study in paediatric patients with moderate-to-severe atopic dermatitis (PEDISTAD): study objectives, design and methodology.

      Paller, Amy S; Guttman-Yassky, Emma; Irvine, Alan D; Baselga, Eulalia; de Bruin-Weller, Marjolein; Jayawardena, Shyamalie; Zhang, Annie; Mina-Osorio, Paola; Rizova, Elena; Ozturk, Zafer E (2020-03-24)
      Introduction: Atopic dermatitis (AD) is a chronic inflammatory skin disease often associated with atopic comorbidities and has significant impact on children and their families. There is a lack of robust and longitudinal long-term data on disease characteristics and typical clinical practice with currently available treatments in children with moderate-to-severe AD. Hence, an observational study is needed to evaluate AD characteristics and progression in paediatric patients with moderate-to-severe AD. Methods and analysis: Pediatric Study in Atopic Dermatitis (PEDISTAD) is a prospective, observational, longitudinal study in paediatric patients with moderate-to-severe AD who are currently receiving systemic or topical treatment and whose disease is not adequately controlled by topical prescription therapies or for whom those therapies are not medically advisable. 1300 children at 100-150 sites in approximately 20 countries worldwide will be enrolled and followed for 5 years. AD therapy is at the discretion of the investigator. Data collected will include: AD disease characteristics and comorbidities; current therapy for AD and initiation of new treatments/changes in current treatment; patient-reported/caregiver-reported outcomes; days missed from school/work for the patient/caregiver; healthcare professional visits; safety and biomarkers. Ethics and dissemination: This study is conducted in accordance with the principles established by the 18th World Medical Assembly and all subsequent amendments and the guidelines for Good Epidemiology Practice. Each individual country assures that ethics approval has been received and local regulatory requirements are met. Ethics approval has been obtained in all countries currently participating in PEDISTAD. Study data will be disseminated in manuscripts submitted to peer-reviewed medical journals as well as in abstracts submitted to congresses and in the resulting posters and presentations.
    • Heart University: a new online educational forum in paediatric and adult congenital cardiac care. The future of virtual learning in a post-pandemic world?

      Tretter, Justin T; Windram, Jonathan; Faulkner, Theresa; Hudgens, Michelle; Sendzikaite, Skaiste; Blom, Nico A; Hanseus, Katarina; Loomba, Rohit S; McMahon, Colin J; Zheleva, Bistra; et al. (2020-04-13)
      Online learning has become an increasingly expected and popular component for education of the modern-day adult learner, including the medical provider. In light of the recent coronavirus pandemic, there has never been more urgency to establish opportunities for supplemental online learning. Heart University aims to be "the go-to online resource" for e-learning in CHD and paediatric-acquired heart disease. It is a carefully curated open access library of paedagogical material for all providers of care to children and adults with CHD or children with acquired heart disease, whether a trainee or a practising provider. In this manuscript, we review the aims, development, current offerings and standing, and future goals of Heart University.
    • Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study.

      Nolan, Beatrice; Mahlangu, Johnny; Pabinger, Ingrid; Young, Guy; Konkle, Barbara A; Barnes, Chris; Nogami, Keiji; Santagostino, Elena; Pasi, K John; Khoo, Liane; et al. (2020-03-30)
      Introduction: The efficacy and safety of recombinant factor VIII Fc fusion protein (rFVIIIFc) as an extended half-life treatment for severe haemophilia A were demonstrated in the Phase 3 A-LONG and Kids A-LONG studies. Eligible subjects who completed A-LONG and Kids A-LONG could enrol in ASPIRE (NCT01454739), an open-label extension study. Aim: To report the long-term safety and efficacy of rFVIIIFc in subjects with severe haemophilia A who enrolled in ASPIRE. Methods: Previously treated subjects received one or more of the following regimens: individualized prophylaxis (IP), weekly prophylaxis, modified prophylaxis or episodic treatment. Subjects could switch treatment regimen at any time. The primary endpoint was inhibitor development. Results: A total of 150 subjects from A-LONG and 61 subjects from Kids A-LONG enrolled in ASPIRE. Most subjects received the IP regimen (A-LONG: n = 110; Kids A-LONG: n = 59). Median (range) treatment duration in ASPIRE for subjects from A-LONG and Kids A-LONG was 3.9 (0.1-5.3) years and 3.2 (0.3-3.9) years, respectively. No inhibitors were observed (0 per 1000 subject-years; 95% confidence interval, 0-5.2) and the overall rFVIIIFc safety profile was consistent with prior studies. For subjects on the IP regimen, annualized bleed rates (ABR) remained low (median overall ABR for adults and adolescents was <1.0) and extended-dosing intervals were maintained (median of 3.5 days) for the majority of subjects in ASPIRE. Conclusion: ASPIRE results, which include up to 5 years of follow-up data, confirm earlier reports on the consistent and well-characterized safety and efficacy of rFVIIIFc treatment for severe haemophilia A.