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https://www.readbyqxmd.com/read/30066606/reaching-independence-through-forced-learning-learning-processes-and-illness-management-in-parents-of-children-affected-by-hemophilia
#1
Linda Myrin Westesson, Catarina Wallengren, Fariba Baghaei, Carina Sparud-Lundin
Hemophilia is a complex condition to manage, especially for parents to newly diagnosed children. This grounded theory study explores parents' learning processes and illness management in daily life during the first year after the start of their child's treatment. Using a longitudinal qualitative design, eight parents of four children were interviewed repeatedly during 12 to 14 months. The core category, reaching independence through forced learning, reflected the parents' learning process and their experiences of the challenges during the first year after start of treatment...
August 1, 2018: Qualitative Health Research
https://www.readbyqxmd.com/read/30049994/polyethylene-glycol-exposure-with-antihemophilic-factor-recombinant-pegylated-rurioctocog-alfa-pegol-and-other-therapies-indicated-for-the-pediatric-population-history-and-safety
#2
Reinhard Stidl, Michael Denne, Jimena Goldstine, Bill Kadish, Katherine I Korakas, Peter L Turecek
Polyethylene glycol (PEG) is an inert, water soluble polymer, used for decades in pharmaceuticals. Although PEG is considered safe, concerns persist about the potential adverse effects of long-term exposure to PEG-containing therapies, specifically in children, following the introduction of PEGylated recombinant factor products used for the treatment of hemophilia. Given the absence of long-term surveillance data, and to evaluate the potential risk, we estimated PEG exposure in the pediatric population receiving PEGylated therapies with pediatric indications administered intravenously or intramuscularly...
July 26, 2018: Pharmaceuticals
https://www.readbyqxmd.com/read/30013766/bay-81-8973-a-full-length-recombinant-factor-viii-for-the-treatment-of-hemophilia-a-product-review
#3
REVIEW
Johnny N Mahlangu, Sanjay P Ahuja, Jerzy Windyga, Nikki Church, Anita Shah, Lawrence Schwartz
BAY 81-8973 (Kovaltry® ) is an unmodified, full-length recombinant factor VIII (rFVIII) approved for the prevention and treatment of bleeding episodes in patients with hemophilia A. The amino acid sequence for BAY 81-8973 is identical to that of sucrose-formulated rFVIII (rFVIII-FS; Kogenate® FS/KOGENATE® , Bayer), but the two products differ in their manufacturing approaches. The manufacture of BAY 81-8973 includes several modifications and enhancements, such as the introduction of the gene for human heat shock protein 70, a molecular chaperone protein that facilitates folding of proteins; no addition of human- or animal-derived proteins in the cell culture, purification process, or final formulation; and use of a 20-nm filter to remove any potential aggregates and pathogens...
July 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/30004151/risk-factors-for-cardiovascular-disease-in-children-and-young-adults-with-haemophilia
#4
Jacqueline Limjoco, Courtney D Thornburg
INTRODUCTION: The origins of cardiovascular disease (CVD) begin in childhood. The primary objective of this cross-sectional cohort study was to determine the prevalence of cardiovascular risk factors in patients with congenital haemophilia A or B followed at Rady Children's Hospital San Diego Hemophilia and Thrombosis Treatment Center (HTC). We hypothesized that cardiovascular risk factors could be identified as part of a comprehensive clinic visit. MATERIALS AND METHODS: Standardized measurement of weight, height, waist circumference and blood pressure plus non-fasting glucose and lipid panel were performed...
July 13, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29848837/pharmacokinetic-studies-of-factor-viii-in-chinese-boys-with-severe-hemophilia-a-a-single-center-study
#5
Zhen-Ping Chen, Pei-Jing Li, Gang Li, Ling Tang, Ying-Zi Zhen, Xin-Yi Wu, Xiao-Ling Cheng, Koon Hung Luke, Victor S Blanchette, Man-Chiu Poon, Qiu-Lan Ding, Run-Hui Wu
Background: Although much attention has been paid to the pharmacokinetics (PKs) of different factor VIII (FVIII) concentrates in persons with hemophilia A (HA), limited information is available in young boys with severe HA. In this study, we aimed to assess the PK parameters of FVIII products in boys with severe HA in China. Methods: A total of 36 boys (plasma-derived [pd]-FVIII, n = 15; recombinant [r] FVIII, n = 21) were enrolled between January 2015 and May 2016 in Beijing Children's Hospital...
August 5, 2018: Chinese Medical Journal
https://www.readbyqxmd.com/read/29803274/evolving-complexity-in-hemophilia-management
#6
REVIEW
Stacy E Croteau
Rapid expansion of therapeutic options have increased the complexity of hemophilia care. Previously, on-demand therapy aimed to reduce morbidity and early mortality; however, now aggressive prophylaxis, particularly in children, encourages an active lifestyle. Accurate diagnosis, recognition of early threats to musculoskeletal health, and optimization of therapy are critical for both males and females affected by hemophilia. The diversity of emerging hemophilia therapies, from modified factor protein concentrates, to gene therapy, to nonfactor hemostatic strategies, provide an exciting opportunity to target unmet needs in the bleeding disorder community...
June 2018: Pediatric Clinics of North America
https://www.readbyqxmd.com/read/29735454/-prophylactic-treatment-with-low-and-intermediate-dose-factor-viii-in-children-with-severe-hemophilia-a-comprehensive-evaluation-of-joint-outcomes-and-correlation-analysis
#7
Jin-Mu Zhuang, Xue-Yan Sun, Xuan Zhou, Zhu-Qin Liu, Jing Sun
OBJECTIVE: To study the effect of low- and intermediate-dose factor VIII (FVIII) for prophylactic treatment of severe hemophilia A in children by comprehensively evaluating the outcomes of the joints. METHODS: Forty-seven children with severe hemophilia A (FVIII activity ≤2%) were enrolled in this study. Eighteen of the children received prophylactic treatment with low-dose FVIII (10 U/kg, 2-3 times a week), 20 received prophylactic treatment with intermediate-dose FVIII (15-30 U/kg, 3 times a week), and 9 received on-demand treatment with FVIII infusion when bleeding occurred according to the Chinese Expert Consensus on the Diagnosis and Treatment of Hemophilia...
April 20, 2018: Nan Fang Yi Ke da Xue Xue Bao, Journal of Southern Medical University
https://www.readbyqxmd.com/read/29723892/treatment-regimens-with-bypassing-agents-in-patients-with-hemophilia-a-and-inhibitors-a-survey-from-the-italian-association-of-hemophilia-centers-aice
#8
Antonio Coppola, Massimo Franchini, Giancarlo Castaman, Elena Santagostino, Cristina Santoro, Rita Carlotta Santoro, Massimo Morfini, Giovanni Di Minno, Angiola Rocino
The development of neutralizing antibodies (inhibitors) against infused factor VIII currently represents the main complication of replacement therapy in patients with severe hemophilia A. Inhibitors, indeed, particularly high-titer inhibitors (>5 BU/mL), greatly complicate the management of bleeding, exposing patients to an increased morbidity and mortality risk, thus representing a significant burden for physicians of Hemophilia Treatment Centers (HTCs). Although bypassing agents (i.e., activated prothrombin complex concentrate [APCC] and recombinant activated factor VII [rFVIIa]) are available for the treatment and prevention of bleeding in inhibitor patients, their efficacy, safety, and cost-benefit outcomes are poorly known in the long term and should be further improved...
May 3, 2018: Seminars in Thrombosis and Hemostasis
https://www.readbyqxmd.com/read/29695583/relevance-of-abusive-head-trauma-to-intracranial-hemorrhages-and-bleeding-disorders
#9
James D Anderst, Shannon L Carpenter, Rodney Presley, Molly Curtin Berkoff, Allison P Wheeler, Robert F Sidonio, J Michael Soucie
BACKGROUND: Bleeding disorders and abusive head trauma (AHT) are associated with intracranial hemorrhage (ICH), including subdural hemorrhage (SDH). Because both conditions often present in young children, the need to screen for bleeding disorders would be better informed by data that include trauma history and are specific to young children. The Universal Data Collection database contains information on ICH in subjects with bleeding disorders, including age and trauma history. Study objectives were to (1) characterize the prevalence and calculate the probabilities of any ICH, traumatic ICH, and nontraumatic ICH in children with congenital bleeding disorders; (2) characterize the prevalence of spontaneous SDH on the basis of bleeding disorder; and (3) identify cases of von Willebrand disease (vWD) that mimic AHT...
May 2018: Pediatrics
https://www.readbyqxmd.com/read/29537123/first-year-results-of-an-expanded-humanitarian-aid-programme-for-haemophilia-in-resource-constrained-countries
#10
G F Pierce, A Haffar, G Ampartzidis, F Peyvandi, S Diop, M El-Ekiaby, H M van den Berg
INTRODUCTION: The gaps in haemophilia treatment around the world are enormous; approximately 60% of an estimated 475 000 individuals are not identified. Of the 187 000 diagnosed, 30% (57 000) access clotting factor replacement therapy. Since 1996, humanitarian aid distributed by the World Federation of Hemophilia (WFH) has played a minor, yet vital role providing life-saving clotting factor to countries in emergency situations. Donated amounts have been small and sporadic, often salvaging short-dated products, providing little opportunity to leverage donations with governments...
March 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29505680/impact-of-hemophilia-b-on-quality-of-life-in-affected-men-women-and-caregivers-assessment-of-patient-reported-outcomes-in-the-b-hero-s-study
#11
Tyler W Buckner, Michelle Witkop, Christine Guelcher, Robert Sidonio, Craig M Kessler, David B Clark, Wendy Owens, Neil Frick, Neeraj N Iyer, David L Cooper
INTRODUCTION: Health-related quality of life (HRQoL) is impaired in patients with hemophilia; however, the impact in mild/moderate hemophilia B and affected women is not well characterized. OBJECTIVE: To evaluate factors that affect HRQoL in adults with hemophilia B and caregivers of affected children. METHODS: US adult patients and caregivers of affected children completed distinct ~1-hour online surveys including patient-reported outcome instruments...
June 2018: European Journal of Haematology
https://www.readbyqxmd.com/read/29485331/using-the-hemophilia-joint-health-score-for-assessment-of-children-reliability-of-the-spanish-version
#12
Cuesta-Barriuso R, Torres-Ortuño A, Pérez-Alenda S, Carrasco Juan J, Querol F, Nieto-Munuera J, López-Pina Ja
INTRODUCTION: Numerous measuring instruments for the evaluation of hemophilic arthropathy have been developed. One of the most used systems is the Hemophilia Joint Health Score (HJHS) given its sensitivity to clinical changes appearing in the joints because of recurrent hemarthrosis. OBJECTIVE: Assessing the interrater reliability, using the Spanish version of the HJHS (version 2.1) in children with hemophilia. DESIGN: Reliability study to assess the interrater reliability of the Spanish version of HJHS...
February 27, 2018: Physiotherapy Theory and Practice
https://www.readbyqxmd.com/read/29447219/optimization-of-prophylaxis-for-hemophilia-a
#13
Robert D Herbert, Carolyn R Broderick, Chris Barnes, Laurent Billot, Albert Zhou, Jane Latimer
BACKGROUND & AIMS: Prophylactic injections of factor VIII reduce the incidence of bleeds and slow the development of joint damage in people with hemophilia. The aim of this study was to identify optimal person-specific prophylaxis regimens for children with hemophilia A. METHODS: Analytic and numerical methods were used to identify prophylaxis regimens which maximize the time for which plasma factor VIII concentrations exceed a threshold, maximize the lowest plasma factor VIII concentrations, and minimize risk of bleeds...
2018: PloS One
https://www.readbyqxmd.com/read/29419797/infrared-thermography-as-a-non-invasive-tool-to-explore-differences-in-the-musculoskeletal-system-of-children-with-hemophilia-compared-to-an-age-matched-healthy-group
#14
Axel Seuser, Karin Kurnik, Anne-Katrin Mahlein
Recurrent joint bleeds and silent bleeds are the most common clinical feature in patients with hemophilia. Every bleed causes an immediate inflammatory response and is the leading cause of chronic crippling arthropathy. With the help of infrared thermography we wanted to detect early differences between a group of clinical non-symptomatic children with hemophilia (CWH) with no history of clinically detected joint bleeds and a healthy age-matched group of children. This could help to discover early inflammation and help implement early treatment and preventative strategies...
February 8, 2018: Sensors
https://www.readbyqxmd.com/read/29418040/male-gender-school-attendance-and-sports-participation-are-positively-associated-with-health-related-quality-of-life-in-children-and-adolescents-with-congenital-bleeding-disorders
#15
P F Limperg, M M H Joosten, K Fijnvandraat, M Peters, M A Grootenhuis, L Haverman
BACKGROUND: This study assesses health-related quality of life (HRQOL), and variables associated with HRQOL, in children and adolescents with haemophilia and congenital bleeding disorders (CBD) in the Netherlands. METHODS: Patients <18 years with CBD under treatment at the Hemophilia Comprehensive Care Center of the Academic Medical Center were included. Participants completed generic HRQOL questionnaires (TAPQOL 0-5 years; PedsQL 6-18 years). Differences and effect sizes in HRQOL compared to healthy peers, and between hemophilia severity groups, were tested using Mann Whitney U-tests...
May 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29356759/modified-primary-prophylaxis-in-previously-untreated-patients-with-severe-hemophilia-a-in-iran
#16
Mehran Karimi, Peyman Eshghi, Mohammad M Safarpour, Sezaneh Haghpanah, Aidin Meshksar, Zohreh Zahedi, Behnaz Habibpanah
BACKGROUND: Recently the low-dose tailoring method of primary prophylaxis has been introduced for previously untreated patients with hemophilia A. OBJECTIVE: To evaluate the efficacy and safety of low-dose tailoring method of primary prophylaxis in previously untreated patients with severe hemophilia A. MATERIALS AND METHODS: In this pre-post interventional study, 33 patients with severe hemophilia A who were previously untreated and affiliated to universities in the capital city and southern Iran were evaluated during 2014 to 2015...
April 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29240038/prevalence-of-hypertension-htn-and-cardiovascular-risk-factors-in-a-hospitalized-pediatric-hemophilia-population
#17
Warren Alperstein, Fernando F Corrales-Medina, Leonardo Tamariz, Ana M Palacio, Joanna A Davis
Improved life expectancy in hemophilia has led to a greater interest in age-related disorders. Hypertension (HTN) as well as cardiovascular disease have been increasingly reported in hemophilic adults but there is currently very limited data in the pediatric population. We conducted a cross-sectional study using data from the 2012 National Health Cost and Utilization Project database to determine the prevalence of HTN and associated cardiovascular risk factors in a hospitalized pediatric hemophilia population, between the ages of 0 to 21 years, in comparison with the general pediatric population...
April 2018: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/29141317/-study-of-gene-mutation-in-62-hemophilia-a-children
#18
Q Hu, A G Liu, L Q Zhang, A Zhang, Y Q Wang, S M Wang, Y J Lu, X Wang
Objective: To analyze the mutation type of FⅧ gene in children with hemophilia A and to explore the relationship among hemophilia gene mutation spectrum, gene mutation and clinical phenotype. Method: Sixty-two children with hemophilia A from Department of Pediatric Hematology, Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology between January 2015 and March 2017 were enrolled. All patients were male, aged from 4 months to 7 years and F Ⅷ activity ranged 0.2%-11.0%. Fifty cases had severe, 10 cases had moderate and 2 cases had mild hemophilia A...
November 2, 2017: Zhonghua Er Ke za Zhi. Chinese Journal of Pediatrics
https://www.readbyqxmd.com/read/29125443/neurosurgical-management-in-children-with-bleeding-diathesis-auditing-neurological-outcome
#19
Zaitun Zakaria, Chandrasekaran Kaliaperumal, Darach Crimmins, John Caird
OBJECTIVE The aim of this study was to assess the outcome of neurosurgical treatment in children with bleeding diathesis and also to evaluate the current management plan applied in the authors' service. METHODS The authors retrospectively analyzed all cases in which neurosurgical procedures were performed in pediatric patients presenting with intracranial hematoma due to an underlying bleeding tendency over a 5-year period at their institution. They evaluated the patients' neurological symptoms from the initial referral, hematological abnormalities, surgical treatment, neurological outcome, and scores on the Pediatric Glasgow Outcome Scale-Extended (GOS-E Peds) obtained 1 year after the last operation...
January 2018: Journal of Neurosurgery. Pediatrics
https://www.readbyqxmd.com/read/29050499/congenital-factor-deficiencies-in-children-a-report-of-a-single-center-experience
#20
Zafer Şalcıoğlu, Cengiz Bayram, Hülya Şen, Gizem Ersoy, Gönül Aydoğan, Arzu Akçay, Deniz Tuğcu, Ferhan Akıcı, Müge Gökçe, Metin Demirkaya, Ali Ayçiçek, Zafer Başlar
Congenital factor deficiencies (CFDs) refer to inherited deficiency of coagulation factors in the blood. A total of 481 patients with CFDs, who were diagnosed and followed at our Pediatric Hematology and Oncology Clinic between 1990 and 2015, were retrospectively evaluated. Of the 481 cases, 134 (27.8%) were hemophilia A, 38 (7.9%) were hemophilia B, 57 (11.8%) were von Willebrand disease (vWD), and 252 (52.3%) were rare bleeding disorders (RBDs). The median age of the patients at the time of diagnosis and at the time of the study was 4...
September 2018: Clinical and Applied Thrombosis/hemostasis
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