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Journals Haemophilia : the Official Jou...

Haemophilia : the Official Journal of the World Federation of Hemophilia

https://read.qxmd.com/read/37766495/preclinical-development-and-characterization-of-a-human-plasma-derived-high-purity-factor-x-concentrate-for-therapeutic-use
#1
JOURNAL ARTICLE
Joanne Lloyd, Peter Feldman
INTRODUCTION: Hereditary factor X deficiency is a rare bleeding disorder, with limited treatment options. This paper describes the approach to pre-clinical development and characterization of a high-purity plasma-derived factor X concentrate, to achieve orphan drug marketing authorization for the treatment of hereditary factor X deficiency. METHODS: A chromatographic process was developed, to purify factor X from human plasma for fractionation. The product was characterized using in vitro, in vivo and ex vivo tests for potency, purity, thrombogenicity, immunogenicity, toxicity and stability...
September 27, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37766492/the-in-vitro-effect-of-anticoagulant-agents-on-coagulation-and-fibrinolysis-in-the-presence-of-emicizumab-in-the-plasmas-from-patients-with-haemophilia-a
#2
JOURNAL ARTICLE
Tomoko Onishi, Suguru Harada, Hanako Shimo, Yoshihito Tashiro, Tetsuhiro Soeda, Keiji Nogami
INTRODUCTION: Emicizumab is used as hemostatic prophylaxis for patients with hemophilia A (PwHA), irrespective of the presence of inhibitors. Although bacterial infection can lead to a procoagulant state, there is limited information on coagulation and fibrinolysis potentials in emicizumab-treated PwHA and on the use of anticoagulants in such cases. AIM: We examined whether anticoagulants affect the coagulation and fibrinolysis potentials in plasma from PwHA spiked with emicizumab...
September 27, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37766484/a-national-assessment-of-standards-of-care-for-inherited-bleeding-disorders-in-canada
#3
JOURNAL ARTICLE
David Page, Sarah Crymble, Lawrence Jardine, JoAnn Nilson, Kathy Mulder, Natasha Pardy, Bojan Pirnat, Milena Pirnat, Wendy Quinn, Karen Sims, Marie-Hélène Thompson, Pam Wilton
AIM: The Canadian Integrated and Comprehensive Care Standards for Inherited Bleeding Disorders were adopted in June 2020. In early 2023, a self-assessment of each of the 26 Canadian inherited bleeding disorder treatment centre's (IBDTCs) capacity to meet the Standards was conducted. The goal was to validate the standards by assessing appropriateness and adherence. As a result, centres can compare their own practices and capacity against those of all centres, identify barriers to adherence, identify opportunities for remedial actions and use the results locally as evidence for needed resources...
September 27, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37758651/targeting-higher-factor-viii-levels-for-prophylaxis-in-haemophilia-a-a-narrative-review
#4
REVIEW
Lynn Malec, Davide Matino
INTRODUCTION: The standard of care in severe haemophilia A is prophylaxis, which has historically aimed for a factor VIII (FVIII) trough level of ≥1%. However, despite prophylactic treatment, people with haemophilia remain at risk of bleeds that have physical and quality of life implications, and that impact everyday life. AIM: The aim of this review was to evaluate evidence supporting the relationship between targeting higher FVIII activity levels with prophylaxis and improved outcomes in people with haemophilia A...
September 27, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37758646/the-isth-bat-score-and-outcomes-after-endometrial-ablation-in-women-with-heavy-menstrual-bleeding
#5
JOURNAL ARTICLE
Heleen P Eising, Marieke C Punt, Tjard Schermer, Jaklien C Leemans, Marlies Y Bongers
BACKGROUND: The International Society on Thrombosis and Haemostasis bleeding assessment tool (ISTH-BAT), is used during the diagnostic workup of bleeding disorders. Data on ISTH-BAT scores in women with heavy menstrual bleeding (HMB) undergoing endometrial ablation (EA) could be essential in optimizing HMB counselling. OBJECTIVE: To investigate the postsurgical incidence of amenorrhea, dysmenorrhea, quality of life, re-intervention after EA, and ISTH-BAT score. METHODS: This study included women who have undergone EA because of HMB...
September 27, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37751318/interferences-by-factor-viii-and-lupus-anticoagulant-in-the-modified-one-stage-assay-for-emicizumab
#6
LETTER
Capucine Habay, Claire Auditeau, Adeline Blandinières, Nûn K Bentounes, Cécile Lavenu-Bombled, Annie Harroche, Cecile Bally, Laurent Frenzel, Delphine Borgel, Dominique Lasne
No abstract text is available yet for this article.
September 25, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37747421/delivery-of-gene-therapy-in-haemophilia-treatment-centres-in-the-united-states-practical-aspects-of-preparedness-and-implementation
#7
REVIEW
Steven Pipe, Kayla Douglas, Nina Hwang, Guy Young, Priya Patel, Patrick Fogarty
INTRODUCTION: Haemophilia treatment centres (HTCs) and healthcare providers (HCPs) will need to adapt to a new treatment paradigm with the emergence of adeno-associated virus (AAV)-based gene therapy for the treatment of haemophilia in adults. AIM: This review examines the upcoming patient and institutional journeys, along with practical aspects of preparedness for clinical delivery of gene therapy by HTCs. METHODS: Based on our clinical experience and examination of published literature, we explored the parallel journeys for patients and treatment centres to navigate before, during, and after administration of gene therapy...
September 25, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37729476/guidance-for-the-antithrombotic-management-of-persons-with-haemophilia-and-cardiovascular-disease
#8
JOURNAL ARTICLE
Pier Mannuccio Mannucci, Pasquale Agosti, Maddalena Lettino
No abstract text is available yet for this article.
September 20, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37729473/how-does-joint-impairment-affect-the-functional-capacity-of-the-lower-limb-in-early-haemophilia-related-arthropathy
#9
JOURNAL ARTICLE
Carlos Cruz-Montecinos, Maria Eulalia Landro, Guillermo Cambiaggi, Horacio Caviglia, Carla Daffunchio
INTRODUCTION: The impact of joint damage on functional capacity in patients with mild haemophilia (PwMH) has yet to be well studied. The primary aim of this study was to investigate the effect of joint impairment on the functional capacity of the lower limb in PwMH. The secondary aim was to identify physical predictors of lower limb functional capacity. METHOD: Forty-nine PwMH were evaluated. Dynamic balance was assessed using Time Up and Go (TUG). Thirty-second sit-to-stand (30-STS) and 60-second-STS (60-STS) were used to assess muscle power and endurance, respectively...
September 20, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37729471/does-the-method-of-telehealth-delivery-affect-the-physiotherapy-management-of-adults-with-bleeding-disorders-a-comparison-of-audioconferencing-and-videoconferencing
#10
JOURNAL ARTICLE
Scott Russell, Simon Whitehart, Jane Mason, Peter Window
AIM: To determine whether the method of telehealth delivery (audioconferencing or videoconferencing) affects the physiotherapy management of adults with inherited bleeding disorders. METHODS: A cross-sectional observational study was utilised involving 40 physiotherapy consultations (23 initial consultations and 17 follow-up consultations) of adults (>18) with a diagnosed bleeding disorder. Each consultation involved an initial audioconferencing component followed immediately by a separate videoconferencing component...
September 20, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37729439/cases-of-less-than-expected-fviii-activity-in-previously-treated-patients-during-post-marketing-surveillance-of-n8-gp
#11
JOURNAL ARTICLE
Johannes Oldenburg, Gary Benson, Pratima Chowdary, Susan Halimeh, Tadashi Matsushita, Anne Nørland, Mohd Nawi Wahid, Laszlo Nemes
INTRODUCTION: Turoctocog alfa pegol (N8-GP) is a glycoPEGylated, extended half-life (EHL), human recombinant factor VIII (FVIII) approved for the treatment and prevention of bleeding episodes in patients with haemophilia A. Since its launch in August 2019, > 800 patients have been treated worldwide. AIM: To present data from identified post-marketing cases of less-than-expected FVIII activity in previously treated patients (PTPs) without inhibitors after switching to N8-GP...
September 20, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37729437/mobilization-under-anaesthesia-for-correction-of-knee-flexion-deformity-in-haemophilia
#12
JOURNAL ARTICLE
Prince Thakkar, Judy Ann John, Raji Thomas, Merlyn Tilak, Madhavi Kandagaddala, Pradeep Poonnoose, Nambiathayil Abubacker Fouzia, Aby Abrahm, Alok Srivastava
INTRODUCTION: Haemophilia patients not treated with primary or secondary prophylaxis, often present with recurrent haemarthrosis. Knee is the most frequently involved joint leading to disabling knee flexion deformity (KFD). Here, we present a retrospective study of our experience on the role of mobilization under general anaesthesia in the correction of KFD. AIM: To study the effectiveness of mobilization under anaesthesia (MUA) for correction of knee flexion deformity (KFD) in persons with haemophilia (PWH)...
September 20, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37718627/initial-von-willebrand-factor-antigen-values-in-adolescent-females-predict-future-values
#13
JOURNAL ARTICLE
Clay T Cohen, Mark Zobeck, Jacquelyn M Powers
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)...
September 18, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37718593/bridging-the-gap-survey-highlights-challenges-and-solutions-in-outreach-and-identification-of-people-with-inherited-bleeding-disorders
#14
JOURNAL ARTICLE
Juliana Hagembe, Alain Baumann, Maria E Santaella, Thomas Sannie, Catherine Lambert, Anne Louise Cruickshank, Carlos Gaitan-Fitch, Ekawat Suwantaroj, Cesar Garrido, Salome Mekhuzla
INTRODUCTION: Inherited bleeding disorders (IBD) are genetic conditions that affect blood clotting, leading to complications such as prolonged or spontaneous bleeding into muscles or joints. Early identification and treatment are crucial to prevent complications and improve outcomes. However, effective patient outreach and identification programs for IBD face significant challenges globally. AIM: This study aimed to identify successful patient outreach initiatives for IBD, barriers encountered during implementation, and approaches used to overcome them...
September 17, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37718591/evaluation-of-the-care-pathway-in-the-context-of-the-dispensing-of-emicizumab-hemlibra-in-community-and-hospital-pharmacies-in-france-a-patient-satisfaction-survey
#15
JOURNAL ARTICLE
Valérie Chamouard, Julie Freyssenge, Gaetan Duport, Fabienne Volot, Rémi Varin, Nicolas Giraud, Yesim Dargaud, Laurie Fraticelli
INTRODUCTION: Since June 2021 in France, patients with haemophilia A with anti-factor VIII inhibitors and patients with severe haemophilia A without anti-factor VIII inhibitors, and treated with emicizumab (Hemlibra), have to choose the dispensing circuit community or hospital pharmacy. AIM: To evaluate satisfaction of patients whether they choose dispensation from a community pharmacy or retained dispensation from the hospital pharmacy, to understand the main motivation for choosing the community or the hospital pharmacy...
September 17, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37718575/treatment-related-outcome-from-patient-report-outcome-pro-of-children-with-severe-and-moderate-hemophilia-a-in-china-an-analysis-report-of-registration-data-from-patient-organization-hemophilia-home-care-center
#16
JOURNAL ARTICLE
Tao Guan, Di Ai, Nan Zhao, Guoqing Liu, Wanru Yao, Zhenping Chen, Zhenping Li, Yaohan Zhou, Runhui Wu
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...
September 17, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37718571/racial-and-ethnic-differences-in-reported-haemophilia-death-rates-in-the-united-states
#17
REVIEW
Stacey A Fedewa, Amanda B Payne, Duc Tran, Lorraine Cafuir, Ana Antun, Christine L Kempton
INTRODUCTION: People with haemophilia's life expectancies have improved over time. Whether progress has been experienced equitably is unknown. AIM: To examine recorded haemophilia death (rHD) rates according to race and ethnicity in the United States (US). METHODS: In this cohort study, rHDs were examined with US National Vital Statistics' 1999-2020 Multiple Cause-of-Death data. rHD was defined as having a haemophilia A (D66) or B (D67) ICD-10 code in the death certificate (underlying or multiple causes of death)...
September 17, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37707428/characterization-of-a-recombinant-factor-ix-molecule-fused-to-coagulation-factor-xiii-b-subunit
#18
JOURNAL ARTICLE
Stephanie Desage, Alexandre Leuci, Nathalie Enjolras, Lori A Holle, Sneha Singh, Xavier Delavenne, Alisa S Wolberg, Arijit Biswas, Yesim Dargaud
INTRODUCTION AND AIM: Severe haemophilia B (HB) is characterized by spontaneous bleeding episodes, mostly into joints. Recurrent bleeds lead to progressive joint destruction called haemophilic arthropathy. The current concept of prophylaxis aims at maintaining the FIX level >3-5 IU/dL, which is effective at reducing the incidence of haemophilic arthropathy. Extended half-life FIX molecules make it easier to achieve these target trough levels compared to standard FIX concentrates...
September 14, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37707369/development-of-viii-inhibitors-beyond-50-exposure-days-to-rfviii-during-prophylactic-emicizumab-therapy
#19
LETTER
Yuichi Mitani, Mamoru Honda, Yoshitaka Mizushima, Makiko Mori, Kohei Fukuoka, Koichi Oshima, Yuki Arakawa, Katsuaki Taira, Yutaka Tanami, Katsuyoshi Koh
No abstract text is available yet for this article.
September 14, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://read.qxmd.com/read/37694835/how-good-does-chatgpt-answer-frequently-asked-questions-about-haemophilia
#20
LETTER
Caroline Vandewyngaert, Michael Iarossi, Cedric Hermans
No abstract text is available yet for this article.
September 11, 2023: Haemophilia: the Official Journal of the World Federation of Hemophilia
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