hemophilia children

BACKGROUND: Emicizumab is a bispecific antibody that bridges activated factor (F)IX and FX, mimicking the function of missing activated FVIII and thus improving hemostasis in people with hemophilia A. The efficacy and safety of emicizumab were demonstrated in 4 phase III clinical trials (HAVEN 1-4). OBJECTIVES: Here, we describe pharmacokinetics (PKs), pharmacodynamics (PDs), and exploratory safety biomarkers in HAVEN 1 to 4. METHODS: Participants received emicizumab at a loading dose of 3 mg/kg weekly for 4 weeks, followed by maintenance doses of 1...

INTRODUCTION: Measuring health-related quality of life (HRQoL) in haemophilia patients provides a comprehensive patient's functional and the impact of the disease and its treatment. AIM: This study aimed to assess the HRQoL of haemophilia children and related factors. METHODS: We conducted a cross-sectional study from 6 April 2019 to 29 April 2019. We recruited children aged 8-16 years old who were members of the Yogyakarta Province Branch, Indonesian Haemophilia Society...

BACKGROUND: In China, there were over 65,000 hemophilia patients according to estimations requiring overall description. However, former Chinese studies related to hemophilia were based on data of relatively small sample size from single-center or selected multi-centers in specific areas. OBJECTIVE: This study is aimed to provide an overview of patients with hemophilia in China and basic information for optimized hemophilia care and policy decisions in the future...

INTRODUCTION: An evolving haemophilia treatment landscape provides new possibilities for previously unattainable lifestyles. AIM: We sought to understand how people with haemophilia (PwH) and their caregivers value the potential benefits of novel prophylactic treatments. We conducted a discrete-choice experiment (DCE) to quantify preferences for features of haemophilia treatments among adults and caregivers of children with haemophilia. A best-worst scaling (BWS) exercise measured the perceived burden of treatment administration features...

BACKGROUD: A huge amount of data about psychosocial issues of people with haemophilia (PwH) are available; however, these materials are fragmentary and largely outdated, failing to reflect the impact of current treatment strategies. AIM: Describing the influence of illness on psychosocial aspects of adult PwH (≥18 years) and caregivers of children with haemophilia (CPwH) without inhibitors, in Italy. METHODS: Surveys (for adult PwH, CPwH and haemophilia specialists) were developed by a multidisciplinary working group and conducted from November 2019 to June 2020...

INTRODUCTION: Prompt, appropriate coagulation factor replacement according to injury and bleeding severity in persons with haemophilia is required to prevent acute and long-term complications. AIMS: Increase proportion of persons with haemophilia A (HA) and B (HB) treated appropriately for an acute injury and bleeding episode at a tertiary children's emergency department (ED) from 65% to 85% and sustain for one year. Secondary aim: increase time interval between patient ED encounters with out-of-range factor dosing...

BACKGROUND: There are great expectations for the potential role of gene therapy in the treatment of hemophilia. At the same time, developments in the field of hemophilia gene therapy have always raised ethical issues. It remains unknown how these ethical issues are perceived by stakeholders, particularly regarding the most recent developments in the field. OBJECTIVES: To obtain insight into stakeholders' morally reasoned opinions on gene therapy for hemophilia. METHODS: We conducted qualitative research with Dutch people with hemophilia ( n  = 13), parents of children with hemophilia ( n  = 5), physicians ( n  = 4), nurses ( n  = 3), a regulator ( n  = 1), and a representative from a pharmaceutical company ( n  = 1)...

BACKGROUND: Clinical trials have shown success in bleed prevention with emicizumab, but real-world data on the effectiveness of emicizumab in preventing serious bleeds in the pediatric population are lacking. OBJECTIVES: To report real-world data on the effectiveness of Emicizumab in pediatric persons with hemophilia A. METHODS: We completed a retrospective chart review of 37 pediatric male patients aged ≤18 years on emicizumab prophylaxis for a median duration of 30...

INTRODUCTION: Physical therapists (PTs) are essential providers within the Hemophilia Treatment Centers (HTCs) team caring for persons with inherited blood disorders (PWBD). OBJECTIVE: Little is known regarding PTs understanding and concordance of MASAC PT Recommendations (MASAC#238), educational resources used to maintain competency and support for a mentorship programme. METHODS: PTs at federally funded HTCs were eligible to participate in a descriptive non-validated study exploring: (i) demographics, (ii) educational background, (iii) experience in evaluation and treatment of PWBD, (iv) practice patterns indicative of concordance with MASAC#238 and (v) opinion regarding PT mentorship...

In recent years, the diagnosis and treatment of hemophilic children in China has significantly improved. However, oral health conditions, which affect quality of life, haven't received attention in this population. To explore the oral health status and oral hygiene of children and adolescents with hemophilia in the Children's Hemophilia Comprehensive Care Center of China. Dental and oral hygiene examinations were performed in children and adolescents with hemophilia who visited Beijing Children's Hospital. DMFT/dmft (decayed, missing, filled teeth in permanent and primary teeth) was assessed according to World Health Organization (WHO) criteria...

BACKGROUND: Hemophilic arthropathy frequently results in musculoskeletal adjustments that impair functional capacity. AIM: We investigated the effects of kinesiologic taping on patients with hemophilic arthropathy in this study. MATERIALS AND METHODS: Twenty-six patients were enrolled. Thirteen patients formed the Kinesiologic taping group (KT) and received the interventional protocol consisting of three kinesiologic taping sessions during three weeks; and 13 patients formed the control group (CG) and made three weeks of home exercises...

Maternal hematological disorders during pregnancy may pose a risk to the neurological development of newborns. To investigate the association between maternal hematological disorders during pregnancy and neurological outcomes in newborns, this mixed cohort study was conducted on 200 pregnant women diagnosed with hematological disorders during pregnancy. Some cases have been identified in the past who have completed the pregnancy in full, as well as cases in pregnancy. Currently, the children of all mothers have been followed up to evaluate the neurological outcomes of the children at the age of three months...

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INTRODUCTION: Recent recommendations of core outcome sets for haemophilia highlight the need for including measures of performance-based physical health and physical function sustainability. To date, there is no consensus on what outcomes might be of value to clinicians and patients. AIM: To identify instruments of performance-based physical function to monitor musculoskeletal health in people with haemophilia that are practical in the clinical setting. METHODS: Utilising components from the Activities and Participation Category of the WHO International Classification of Functioning (WHO-ICF), a consensus-based, decision analysis approach was used to: identify activities people with haemophilia have most difficulty performing; identify quantitative performance-based measures of identified activities via a scoping review; and obtain views on acceptability of the tests utilising a DELPHI approach...

BACKGROUND: Recurrent joint bleeds are a major cause of morbidity in severe hemophilia. Prophylaxis with efmoroctocog alfa (a recombinant factor VIII Fc fusion protein, [rFVIIIFc]) has demonstrated benefits beyond bleed control, including joint health maintenance. OBJECTIVES: To assess long-term efficacy and safety of rFVIIIFc prophylaxis in severe hemophilia A in phase 3 pivotal (A-LONG/Kids A-LONG) and extension (ASPIRE) studies. METHODS: Longitudinal analysis included pooled data from A-LONG/Kids A-LONG and ASPIRE...

No abstract text is available yet for this article.

INTRODUCTION: Diagnosing von Willebrand Disease (VWD) in adolescent females is challenging as menstruation and physiologic stress elevate von Willebrand factor (VWF) laboratory values. AIM: To develop a VWF prediction model for adolescent females based on initial VWF results. METHODS: We identified female patients aged 9 to 21 years with any VWF laboratory test over a 5-year period (2017-2021) at any Texas Children's Hospital facility. Patient demographics, VWF testing, haemoglobin concentration, serum ferritin and site of clinical testing were collected (initial and subsequent laboratory evaluations)...

OBJECTIVES: To assess current treatment-related outcomes for children with severe and moderate haemophilia A (cHA) in China. METHODS: This cross-section Patient Report Outcome (PRO) report collected PRO data of severe and moderate cHAs registered in the 'Hemophilia Home Care Center' database (https://web.bjxueyou.cn) between January 2021 and November 2022. Data included records of bleeding, activities, and concentrates consumption. All patients had a confirmed diagnosis of moderate or severe haemophilia A (FVIII: C ≤ 5%) and were < 18 years old...

OBJECTIVE: To explore the feasibility, safety and cost effectiveness of the use of peripherally inserted central catheter (PICC) in children with hemophilia A and inhibitors who underwent ITI treatment. METHOD: This retrospective cohort study evaluated the effect of PICC placement and ITI on bleeding rates, costs, and parents' satisfaction before and within 6 months after PICC placement in children with hemophilia A and inhibitors. RESULTS: A total of 20 children with hemophilia A and high-titer inhibitors were included, with a success rate for PICC placement of 100%, at a cost of ¥6730...

AIM: An open-label phase 2/3 study of plasminogen, human-tvmh administered intravenously in paediatric and adult subjects with type 1 plasminogen deficiency was conducted. Interim data was previously reported. The final data on 15 subjects who completed the study up to a maximum of 124 weeks are reported here. METHODS: The primary objectives were to evaluate efficacy of plasminogen replacement therapy on clinically evident or visible lesions during 48 weeks of dosing and to achieve an increase in trough plasminogen activity levels by at least an absolute 10% above baseline during 12 weeks of treatment...