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Haemophilia: the Official Journal of the World Federation of Hemophilia

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https://read.qxmd.com/read/31066174/recombinant-factor-viii-products-and-inhibitor-development-in-previously-untreated-patients-with-severe-haemophilia-a-combined-analysis-of-three-studies
#1
Peter Volkers, Kay-Martin Hanschmann, Thierry Calvez, Hervé Chambost, Peter W Collins, Virginie Demiguel, Daniel P Hart, Charles R M Hay, Jenny Goudemand, Rolf Ljung, Ben P Palmer, Elena Santagostino, Ella M van Hardeveld, Marijke van den Berg, Brigitte Keller-Stanislawski
INTRODUCTION: Standard treatment of congenital haemophilia A is based on replacement therapy with coagulation factor VIII (FVIII) products. A major complication of FVIII therapy is the occurrence of IgG alloantibodies (inhibitors) that neutralize FVIII activity. AIM: The aim of the analysis was estimating the risk of high-titre inhibitor associated with the second-generation full-length product compared to third-generation full-length product and other recombinant FVIII (rFVIII)...
May 7, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31056808/uk-vs-us-physician-decision-making-in-the-treatment-of-haemophilia
#2
Christopher C Lamb, Adrian Wolfberg, Kalle Lyytinen
INTRODUCTION: Patient-physician shared decision-making (SDM) has become increasingly seen as having a positive effect on management of chronic diseases. However, little is known of the factors that encourage SDM or how effective it may be at improving health outcomes or how cost-effective it is. AIM: To investigate the uses and applications of patient physician-SDM in the management of haemophilia and the influence of healthcare systems in the United States and the United Kingdom...
May 5, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31050134/model-based-evaluation-of-low-dose-factor-viii-prophylaxis-in-haemophilia-a
#3
Ari Brekkan, Johanna Degerman, Siv Jönsson
INTRODUCTION: The optimal treatment modality for haemophilia A is lifelong prophylaxis which is expensive and may not be implementable everywhere where factor VIII (FVIII) availability is limited. A less costly alternative to prophylaxis is low-dose prophylaxis (LDP) which was compared to conventional prophylaxis in this model-based simulation study. AIM: To explore whether LDP is motivated where standard prophylaxis is not implementable, including evaluating LDP efficacy compared to high-dose prophylaxis and investigating the potential economic benefit of individualized dosing...
May 2, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31050121/intravenous-immunoglobulin-treatment-in-a-type-3-von-willebrand-disease-patient-with-alloantibodies-and-a-life-threatening-gastrointestinal-bleed
#4
LETTER
Vuokko Nummi, Elina Lehtinen, Anne Mäkipernaa, Timea Szanto, Riitta Lassila
No abstract text is available yet for this article.
May 2, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31050100/the-factor-viii-c-vwf-ag-ratio-as-a-useful-tool-to-predict-relapse-in-patients-with-acquired-haemophilia-a-a-retrospective-cohort-study
#5
Marc Trossaert, Julie Graveleau, Marie-Françoise Thiercelin-Legrand, Marianne Sigaud, Felipe Guerrero, Antoine Neel, Marc Fouassier, Laurent Sailler, Dominique Chauveau, Catherine Ternisien, Antoine Huart, Benjamin Gillet, Mohamed Hamidou, Marie C Bene, Sophie Voisin
INTRODUCTION: Acquired haemophilia A (AHA) is a rare autoimmune bleeding disorder caused by the presence of autoantibodies against coagulation factor VIII (FVIII). The mortality rate remains high. International recommendations define complete remission as undetectable inhibitor (<0.6 Bethesda Units [BU]) and normal FVIII activity (FVIII:C) that persists after immunosuppressive therapy stopped. For patients achieving remission, the risk of relapse reaches 20%. The risk factors for this relapse are not well known...
May 2, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31045306/ischaemic-events-are-rare-and-the-prevalence-of-hypertension-is-not-high-in-japanese-adults-with-haemophilia-first-multicentre-study-in-asia
#6
Azusa Nagao, Nobuaki Suzuki, Hideyuki Takedani, Naoya Yamasaki, Yushi Chikasawa, Akihiro Sawada, Takeshi Kanematsu, Masanori Nojima, Satoshi Higasa, Kagehiro Amano, Katsuyuki Fukutake, Teruhisa Fujii, Tadashi Matsushita, Takashi Suzuki
INTRODUCTION: With the increasing life expectancy of patients with haemophilia (PWH), the number of PWH with age-related comorbidities, such as ischaemic events, is increasing. AIM: We conducted this multicentre observational study to identify the risk factors for major ischaemic events in PWH. METHODS: This study was the first multicentre observational study, conducted with the participation of five haemophilia treatment centres in Japan, conducted in ≥30-year-old adult PWH...
May 2, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31044491/comparison-of-the-efficacy-and-safety-of-12-month-low-dose-factor-viii-tertiary-prophylaxis-vs-on-demand-treatment-in-severe-haemophilia-a-children
#7
Novie A Chozie, Fitri Primacakti, Djajadiman Gatot, Rahajuningsih D Setiabudhy, Angela B M Tulaar, Marcel Prasetyo
INTRODUCTION: Prophylaxis has commonly become standard treatment for severe haemophilia patients. The World Federation of Hemophilia (WFH) recommends low-dose prophylaxis in countries with resource constraints. OBJECTIVE: To determine efficacy and safety of low-dose factor VIII (FVIII) tertiary prophylaxis compared to on-demand treatment in severe haemophilia A children in Indonesia. METHODS: Eligible patients were randomly assigned to prophylaxis and on-demand groups...
May 2, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31044485/evaluation-of-an-automated-chromogenic-assay-for-factor-viii-clotting-activity-measurement-in-patients-affected-by-haemophilia-a
#8
Cristina Novembrino, Massimo Boscolo Anzoletti, Maria Elisa Mancuso, Sho Shinohara, Flora Peyvandi
INTRODUCTION: The original one-stage clotting assay is still the most widely used method to measure Factor VIII clotting activity (FVIII:C) in patients with haemophilia A (HA), although the use of chromogenic assays is increasing significantly. AIM: Evaluation of the analytical performance and diagnostic accuracy of BIOPHEN™ FVIII:C (HYPHEN BioMed, Neuville-sur-Oise, France) assay on Sysmex CS-2400 (Sysmex, Kobe, Japan) analyser. METHODS: Sixty patients with haemophilia A (HA; any severity) and 120 healthy Italian subjects were included...
May 2, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31044495/translation-validation-and-reliability-of-the-functional-capacity-questionnaire-haemophilia-activities-list-for-haemophilia-patients-in-brazil
#9
Ana A T Ramos, Álvaro L P Wolff, Claudia S Lorenzato, Luciano R L Pacheco, Marise B Zonta, Silvia Valderramas
INTRODUCTION: To date, none of the available assessment instruments to evaluate functional abilities for individuals with haemophilia has been translated and validated in Brazil. AIM: To translate, and test the construct validity, internal consistency and the reliability of the Haemophilia Activities List (HAL) for the Brazilian population with severe and moderate haemophilia (type A or B) and to investigate differences in the self-perception of functional abilities in patients adults with haemophilia classified according to the presence of joint bleeding and the performance of orthopaedic surgeries...
May 1, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31034126/barriers-and-challenges-faced-by-women-with-congenital-bleeding-disorders-in-europe-results-of-a-patient-survey-conducted-by-the-european-haemophilia-consortium
#10
Declan Noone, Naja Skouw-Rasmussen, Michelle Lavin, Karin P M van Galen, Rezan A Kadir
INTRODUCTION: Historically, issues faced by women with bleeding disorders (WBD) have been underestimated. While advances in genetic testing have resulted in improvements, significant challenges remain in the initial recognition of abnormal bleeding and referral of WBD. METHODS: The European Haemophilia Consortium (EHC) developed a questionnaire for WBD to provide insights into the barriers and challenges faced by WBD in Europe. RESULTS: In total, 709 WBD responded to the survey from 32 countries, predominantly from western European countries (94%)...
April 29, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31033112/the-changing-face-of-immune-tolerance-induction-in-haemophilia-a-with-the-advent-of-emicizumab
#11
Manuel Carcao, Carmen Escuriola-Ettingshausen, Elena Santagostino, Johannes Oldenburg, Ri Liesner, Beatrice Nolan, Angelika Bátorová, Saturnino Haya, Guy Young
INTRODUCTION: As a result of the new treatment paradigm that the haemophilia community will face with the availability of novel (non-factor) therapies, an updated consensus on ITI recommendations and inhibitor management strategies is needed. AIM: The Future of Immunotolerance Treatment (FIT) group was established to contemplate, determine and recommend the best management options for patients with haemophilia A and inhibitors. DISCUSSION AND CONCLUSIONS: Despite the considerable success of emicizumab in the management of inhibitor patients, the FIT group still sees the importance of eradicating inhibitors...
April 29, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31016855/health-related-quality-of-life-and-health-status-in-persons-with-haemophilia-a-with-inhibitors-a-prospective-multicentre-non-interventional-study-nis
#12
Johnny Mahlangu, Johannes Oldenburg, Michael U Callaghan, Midori Shima, Maria Elisa Mancuso, Peter Trask, Michael Recht, Claudia Garcia, Renchi Yang, Michaela Lehle, Harrison Macharia, Elina Asikanius, Gallia G Levy, Rebecca Kruse-Jarres, Sylvia von Mackensen
INTRODUCTION: Real-world data (RWD) on health-related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non-interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health-related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice. AIM: To report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS...
April 24, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/31016823/from-the-voices-of-people-with-haemophilia-a-and-their-caregivers-challenges-with-current-treatment-their-impact-on-quality-of-life-and-desired-improvements-in-future-therapies
#13
Ryan E Wiley, Charles P Khoury, Adrian W K Snihur, Marni Williams, David Page, Nicole Graham, Lori Laudenbach, Cindy Milne-Wren, Jayson M Stoffman
INTRODUCTION: Haemophilia A is a chronic disease requiring frequent intravenous infusions of recombinant factor VIII. Previous studies have shown that challenges associated with current treatments may have significant impacts on quality of life (QoL) that are as important as the health outcomes conferred by the therapy. Emerging therapeutic innovations offer the potential to mitigate treatment-related challenges, and it is therefore important to develop a better understanding of patient and caregiver experiences with existing haemophilia A treatments in order to characterize the full value of new treatments...
April 23, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/30994259/standard-and-water-rehabilitation-an-analysis-of-over-14-years-experience-in-patients-with-haemophilia-or-other-clotting-factor-disorders-after-orthopaedic-surgery
#14
E V Passeri, Marco Martinelli, Vincenzo Gatteri, Stefania Pivetti, Chiara Passeri, Luisa Cigolini, Sonia Chiari, Andrea Zenorini, Paolo Gaffurini, Stefano Bernardi, Ilaria Poli, Luciano Bissolotti
INTRODUCTION: In people with haemophilia (PWH) with severe arthropathy, total joint replacement (TJR) can be undertaken if conservative management fails. Post-operative rehabilitation treatment is an important part of the comprehensive management of patients undergoing TJR. AIM: To compare post-operative standard rehabilitation (SR) and SR plus water rehabilitation (WR) in PWH undergoing TJR. METHODS: PWH who were admitted to our centre between June 2003 and December 2016 for rehabilitation after TJR were included in the study...
April 17, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/30994257/missense-changes-in-the-catalytic-domain-of-coagulation-factor-x-account-for-minimal-function-preventing-a-perinatal-lethal-condition
#15
Mattia Ferrarese, Marcello Baroni, Patrizia Della Valle, Ivana Spiga, Antonella Poloniato, Armando D'Angelo, Mirko Pinotti, Francesco Bernardi, Alessio Branchini
INTRODUCTION: Inherited deficiencies in the coagulation pathway provide diversified models to investigate the molecular bases of perinatal lethality associated with null-like variants. Differently from X-linked haemophilias, homozygous/doubly heterozygous null variants in the rare autosomally inherited deficiency of factor X (FX) might be incompatible with perinatal survival. AIM: To provide experimental evidence about the null/close-to-null FX function. METHODS: The residual secreted (ELISA) and functional (thrombin generation assays) protein levels associated with the novel nonsense (c...
April 17, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/30994256/prophylactic-protocol-for-dental-care-in-ligneous-gingivitis-due-to-severe-plasminogen-deficiency-case-report-and-review-of-literature
#16
Maria T Sartori, Stefano Sivolella, Irene Di Pasquale, Graziella Saggiorato, Alessandro Perini, Francesca Boscaro, Fabrizio Fabris
INTRODUCTION: Severe plasminogen (PLG) deficiency causes ligneous conjunctivitis, a rare disease characterized by the growth of fibrin-rich pseudomembranes on mucosal surfaces; gums involvement leads to ligneous gingivitis (LG). Specific therapy for LG is not available yet. We report a prophylactic treatment with enoxaparin and fresh frozen plasma (FFP) for invasive dental procedures in a patient with LG, and a review of literature on LG treatment. METHODS: A 43-year-old female with LG was studied...
April 17, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/30994252/commentary-on-the-paper-hindfoot-malalignment-in-adults-with-haemophilic-ankle-arthropathy-the-importance-of-early-detection-and-orthotic-treatment-authored-by-de-la-corte-rodriguez-rodriguez-merchan-alvarez-roman-martin-salces-jimenez-yuste
#17
Alexej Barg, Cesar de Cesar Netto
No abstract text is available yet for this article.
April 17, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/30994251/coping-strategies-in-young-and-adult-haemophilia-patients-a-tool-for-the-adaptation-to-the-disease
#18
Ana Torres-Ortuño, Rubén Cuesta-Barriuso, Joaquín Nieto-Munuera, Pilar Galindo-Piñana, José Antonio López-Pina
INTRODUCTION: Chronic diseases, after diagnosis, involve changes that have to favour coping with the new situation. The resources used will help control, manage and adapt to the disease. The psychological aspects may be influencing how the individual faces the situation. AIM: To assess whether perceptions or beliefs and illness behaviour influence the choice of coping strategies for young and adult patients with haemophilia. METHODS: Multicenter cross-sectional descriptive study...
April 17, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/30994246/assessment-of-kinect-v2-for-elbow-range-of-motion-estimation-in-people-with-haemophilia-using-an-angle-correction-model
#19
Fernando Mateo, Juan J Carrasco, Marta Aguilar-Rodríguez, Emilio Soria-Olivas, Santiago Bonanad, Felipe Querol, Sofía Pérez-Alenda
INTRODUCTION: The joint range of motion (ROM) is an important clinical parameter used to assess the loss of functionality resulting from joint bleedings in people with haemophilia. These episodes require a close follow-up and, to decrease patients' hospital dependence, telemedicine tools are needed. Therefore, this study is aimed to analyse the validity of the Microsoft Kinect V2 sensor with corrected angle measurement to be used in the monitoring of elbow ROM in people with haemophilia...
April 17, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/30994216/periprocedural-management-of-von-willebrand-disease-an-institutional-experience
#20
LETTER
Craig D Seaman, Margaret V Ragni
No abstract text is available yet for this article.
April 17, 2019: Haemophilia: the Official Journal of the World Federation of Hemophilia
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