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Hemophilia A

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https://www.readbyqxmd.com/read/28750425/emerging-therapeutic-strategies-in-the-treatment-of-hemophilia-a
#1
Vincent Muczynski, Olivier D Christophe, Cécile V Denis, Peter J Lenting
No abstract text is available yet for this article.
July 27, 2017: Seminars in Thrombosis and Hemostasis
https://www.readbyqxmd.com/read/28738402/biodistribution-of-liver-derived-mesenchymal-stem-cells-after-peripheral-injection-in-a-hemophilia-a-patient
#2
Etienne M Sokal, Catherine Anne Lombard, Véronique Roelants, Mustapha Najimi, Sharat Varma, Camillo Sargiacomo, Joachim Ravau, Giuseppe Mazza, François Jamar, Julia Versavau, Vanessa Jacobs, Marc Jacquemin, Stéphane Eeckhoudt, Catherine Lambert, Xavier Stéphenne, Françoise Smets, Cédric Hermans
BACKGROUND: With the exception of liver transplantation, there is no cure for hemophilia, which is currently managed by preemptive replacement therapy. Liver-derived stem cells are in clinical development for inborn and acquired liver diseases and could represent a curative treatment for hemophilia A. The liver is a major factor VIII (FVIII) synthesis site, and mesenchymal stem cells have been shown to control joint bleeding in animal models of hemophilia. Adult-derived human liver stem cells (ADHLSCs) have mesenchymal characteristics and have been shown able to engraft in and repopulate both animal and human livers...
August 2017: Transplantation
https://www.readbyqxmd.com/read/28737602/treatment-of-congenital-thrombotic-thrombocytopenia-purpura-a-new-paradigm
#3
Louis M Aledort, Tammuella C Singleton, Paula J Ulsh
Congenital thrombotic thrombocytopenia purpura (cTTP) is a very rare disorder worldwide. Standard treatment of recognized cases has been to administer fresh frozen plasma as the source of ADAMTS13, to replenish the absent ADAMTS13 enzyme. An alternative source, a plasma-derived factor VIII concentrate used for hemophilia A, and found to contain this enzyme, was reported to be effective in 1 patient in the United States. We now report details on a US cohort of 8 cTTP patients who have been successfully treated for varying periods with a marketed antihemophilic factor concentrate Koate-DVI...
July 21, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28731873/health-related-quality-of-life-in-iranian-adult-men-with-severe-hemophilia
#4
Mohammad Faranoush, Ehsan Shahverdi, Raheb Qorbani, Mostafa Moghaddam
: Health-related quality of life (HRQOL) assessment should be part of the regular clinical assessment of persons with hemophilia. This study assessed quality of life of severe hemophilia patients (Type A) in Tehran comprehensive hemophilia care center. This cross-sectional study was done in 2016 and 84 men aged over 20 years with severe hemophilia (Type A) were assessed. All patients have been treated over 10 years at the comprehensive hemophilia care center, Iran. The HRQOL assessment includes the A36 Hemophilia-QOL questionnaire, sociodemographical and clinical characteristics...
July 20, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/28731872/bleeding-risk-assessment-in-hemophilia-a-carriers-from-dakar-senegal
#5
Moussa Seck, Blaise F Faye, Abibatou Sall, Diariétou Sy, Sokhna A Touré, Nata Dieng, Youssou B Guéye, Macoura Gadji, Awa O Touré, Cathérine Costa, Dominique Lasne, Chantal Rothschild, Saliou Diop
: Hemophilia A carriers have an abnormal X chromosome with a molecular abnormality of FVIII gene. These carriers, long considered to be free of bleeding risk, could have the same symptoms as mild hemophiliacs. This study aim to assess bleeding risk of hemophilia A carriers monitored at the Clinical Hematology of Dakar. This is a prospective study of a period of 6 months including 22 hemophilia A carriers aged between 8 and 48 years. Hemophilia carriers were recruited using the genealogical tree of hemophiliacs followed in the service...
July 20, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/28728770/don-t-push-your-luck-educational-family-board-not-bored-game-for-school-age-children-living-with-chronic-conditions
#6
Andrea Kennedy, Lisa Semple, Kerri Alderson, Vanessa Bouskill, Janice Karasevich, Brenda Riske, Sheri van Gunst
PURPOSE: Children who are living with chronic conditions may be supported in self-care through enjoyable active learning and family social processes. This research focused on development and evaluation of "Don't Push Your Luck!", an educational board game designed to inspire family discussion about chronic conditions, and help affected children learn about self-care choices and consequences. DESIGN AND METHODS: Mixed-method research was conducted with families from one outpatient Cystic Fibrosis Clinic and four Hemophilia Treatment Centres in Canada and United States (N=72)...
July 2017: Journal of Pediatric Nursing
https://www.readbyqxmd.com/read/28727494/efficacy-of-standard-prophylaxis-versus-on-demand-treatment-with-bayer-s-sucrose-formulated-recombinant-fviii-rfviii-fs-in-chinese-children-with-severe-hemophilia-a
#7
Yongqiang Zhao, Juan Xiao, Renchi Yang, Runhui Wu, Yu Hu, Horst Beckmann, Junde Wu, Qingsong Hou, Jing Sun
In China, care of patients with severe hemophilia primarily involves insufficient dosing of on-demand treatment and secondary low-dose prophylaxis (10 IU/kg 2× /wk). We sought to evaluate 3× /wk, standard-dose prophylaxis with sucrose-formulated recombinant factor VIII (rFVIII-FS; Bayer) compared with on-demand treatment in Chinese children with severe hemophilia A. Children and adolescents aged 2-16 years with severe hemophilia A, no inhibitors, and no prophylaxis for >6 consecutive months before study entry were eligible for this 24-week, interventional, sequential-treatment study...
July 20, 2017: Pediatric Hematology and Oncology
https://www.readbyqxmd.com/read/28725275/ash-meeting-2016-developments-in-hemostaseology
#8
REVIEW
Clemens Feistritzer, Birgit Mosheimer
During the annual meeting of the American Society of Hematology (ASH) in San Diego/California, novel developments in the field of hemostaseology were presented. Alternative treatment strategies besides factor replacement were discussed for patients with hemophilia. One of the highlights of the meeting in this year's plenary session was the presentation of successful adeno-associated virus mediated gene transfer in patients with hemophilia B leading to sustained elevation of factor IX:C (FIX:c). Other alternative treatment approaches in patients with hemophilia A may include bispecific antibodies mimicking factor VIIIa (FVIIIa) activity or disrupting anticoagulant proteins...
2017: Memo
https://www.readbyqxmd.com/read/28724345/simultaneous-bilateral-total-knee-arthroplasty-in-hemophilia-is-it-recommended
#9
E Carlos Rodriguez-Merchan
Simultaneous bilateral total knee arthroplasty (TKA) is a potentially cost-saving manner of caring for patients with hemophilia who have bilateral symptomatic hemophilic knee arthropathy. Because factor VIII is used only once in bilateral TKA, it is performed in hemophilia to reduce costs. Areas covered: This article reviews the potential role of simultaneous bilateral TKA in patients with hemophilia. Expert commentary: Although two recent reports have mentioned the benefits of simultaneous bilateral TKA in patients with hemophilia, the data are still scant and preliminary...
July 20, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/28716211/to-serve-and-protect-the-modulatory-role-of-von-willebrand-factor-on-factor-viii-immunogenicity
#10
REVIEW
Robin B Hartholt, Alice S van Velzen, Ivan Peyron, Anja Ten Brinke, Karin Fijnvandraat, Jan Voorberg
Hemophilia A is a bleeding disorder characterized by the absence or dysfunction of blood coagulation factor VIII (FVIII). Patients are treated with regular infusions of FVIII concentrate. In response to treatment, approximately 30% of patients with severe hemophilia A develop inhibitory antibodies targeting FVIII. Both patient and treatment related risk factors for inhibitor development have been described. Multiple studies comparing the immunogenicity of recombinant and plasma-derived FVIII have yielded conflicting results...
July 4, 2017: Blood Reviews
https://www.readbyqxmd.com/read/28705864/cannav%C3%A3-a-valsecchi-c-garagiola-i-et-al-nonneutralizing-antibodies-against-factor-viii-and-risk-of-inhibitor-development-in-severe-hemophilia-a-blood-2017-129-10-1245-1250
#11
https://www.readbyqxmd.com/read/28696534/a-fusion-protein-of-interleukin-4-and-interleukin-10-protects-against-blood-induced-cartilage-damage-in-vitro-and-in-vivo
#12
Lize F D van Vulpen, Jelena Popov-Celeketic, Monique E R van Meegeren, Katja Coeleveld, Jacob M van Laar, C Erik Hack, Roger E G Schutgens, Simon C Mastbergen, Floris P J G Lafeber
BACKGROUND: Joint damage still causes significant morbidity in hemophilia. It results from synovial inflammation and direct cartilage-degenerating properties of blood components. Interleukin (IL)-4 and IL-10 have been shown to protect cartilage from blood-induced damage. Recently an IL4-10 fusion protein has been developed to combine the function of IL-4 and IL-10 and increase their bioavailability. OBJECTIVES: In this study it is evaluated whether this IL4-10 fusion protein protects against blood-induced joint damage...
July 11, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28695182/adolescent-total-knee-arthroplasty
#13
John R Martin, Alan K Sutak, Todd A Milbrandt, Valerie A Martin, Robert T Trousdale
BACKGROUND: Management of adolescent patients with end-stage arthritis is challenging. Nonoperative treatments may be ineffective and total knee arthroplasty (TKA) is rarely performed. Currently, minimal long-term data are available on the outcomes in this patient population. Our goal was to describe TKA for patients with end-stage arthritis who were aged 20 years and younger. METHODS: The Joint Registry at our institution was used to identify 19 patients (29 TKAs) aged 20 years and younger that underwent a primary TKA...
June 2017: Arthroplasty Today
https://www.readbyqxmd.com/read/28692842/pk-driven-prophylaxis-versus-standard-prophylaxis-when-a-tailored-treatment-may-be-a-real-and-achievable-cost-saving-approach-in-children-with-severe-hemophilia-a
#14
Samantha Pasca, Marta Milan, Lucia Sarolo, Ezio Zanon
BACKGROUND: Prophylaxis is the gold standard for the treatment of children with severe hemophilia. In the last years a new approach to prophylaxis based on annual bleeding rate (ABR), pharmacokinetics (PK) and lifestyle of each patient has begun to be adopted in hemophilia treatment. AIM: Aim of our observational retrospective study was to evaluate whether in a group of children with severe hemophilia A (HA) a tailored approach may be used to replace standard therapy, reducing costs...
July 5, 2017: Thrombosis Research
https://www.readbyqxmd.com/read/28692575/rendering-factor-xa-zymogen-like-as-a-therapeutic-strategy-to-treat-bleeding
#15
Nabil K Thalji, Rodney M Camire
PURPOSE OF REVIEW: New therapies are needed to control bleeding in a range of clinical conditions. This review will discuss the biochemical properties of zymogen-like factor Xa, its preclinical assessment in different model systems, and future development prospects. RECENT FINDINGS: Underlying many procoagulant therapeutic approaches is the rapid generation of thrombin to promote robust clot formation. Clinically tested prohemostatic agents (e.g., factor VIIa) can provide effective hemostasis to mitigate bleeding in hemophilia and other clinical situations...
July 7, 2017: Current Opinion in Hematology
https://www.readbyqxmd.com/read/28691885/targeting-of-antithrombin-in-hemophilia-a-or-b-with-rnai-therapy
#16
K John Pasi, Savita Rangarajan, Pencho Georgiev, Tim Mant, Michael D Creagh, Toshko Lissitchkov, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Liana Gercheva-Kyuchukova, Vasily Mamonov, Margarita Timofeeva, Chang-Heok Soh, Pushkal Garg, Akshay Vaishnaw, Akin Akinc, Benny Sørensen, Margaret V Ragni
Background Current hemophilia treatment involves frequent intravenous infusions of clotting factors, which is associated with variable hemostatic protection, a high treatment burden, and a risk of the development of inhibitory alloantibodies. Fitusiran, an investigational RNA interference (RNAi) therapy that targets antithrombin (encoded by SERPINC1), is in development to address these and other limitations. Methods In this phase 1 dose-escalation study, we enrolled 4 healthy volunteers and 25 participants with moderate or severe hemophilia A or B who did not have inhibitory alloantibodies...
July 10, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/28691557/emicizumab-prophylaxis-in-hemophilia-a-with-inhibitors
#17
Johannes Oldenburg, Johnny N Mahlangu, Benjamin Kim, Christophe Schmitt, Michael U Callaghan, Guy Young, Elena Santagostino, Rebecca Kruse-Jarres, Claude Negrier, Craig Kessler, Nancy Valente, Elina Asikanius, Gallia G Levy, Jerzy Windyga, Midori Shima
Background Emicizumab (ACE910) bridges activated factor IX and factor X to restore the function of activated factor VIII, which is deficient in persons with hemophilia A. This phase 3, multicenter trial assessed once-weekly subcutaneous emicizumab prophylaxis in persons with hemophilia A with factor VIII inhibitors. Methods We enrolled participants who were 12 years of age or older. Those who had previously received episodic treatment with bypassing agents were randomly assigned in a 2:1 ratio to emicizumab prophylaxis (group A) or no prophylaxis (group B)...
July 10, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/28691218/safety-of-a-pasteurized-plasma-derived-factor-viii-and-von-willebrand-factor-concentrate-analysis-of-33-years-of-pharmacovigilance-data
#18
Peter Kouides, Kathrin Wawra-Hehenberger, Anna Sajan, Henry Mead, Toby Simon
BACKGROUND: Haemate-P/Humate-P (Humate-P) is a pasteurized human plasma-derived concentrate containing both Factor VIII and von Willebrand factor for treatment of hemophilia A and von Willebrand disease (VWD). STUDY DESIGN AND METHODS: We analyzed the safety of Humate-P based on more than 33 years of postmarketing pharmacovigilance data, representing an estimated exposure of approximately 25,000 patient-years. The analysis comprises reports of potential adverse drug reactions (ADRs) from all sources, reported as part of routine pharmacovigilance at CSL Behring...
July 10, 2017: Transfusion
https://www.readbyqxmd.com/read/28689155/combination-of-hemostatic-therapies-for-treatment-of-patients-with-hemophilia-a-and-inhibitors
#19
Tami Livnat, Ivan Budnik, Sarina Levy-Mendelovich, Einat Avishai, Mudi Misgav, Assaf Arie Barg, Aharon Lubetsky, Tami Brutman-Barazani, Gili Kenet
BACKGROUND: Therapy application and monitoring of patients with hemophilia A (HA) and inhibitors are challenging. In the current study, combined FVIII - bypass therapy was implemented for a cohort of severe HA patients with inhibitors. METHODS: Plasma of 15 HA patients with inhibitors was spiked ex vivo with FVIII, rFVIIa, FEIBA and their combinations and thrombin generation (TG) was studied. Some patients who experienced hemarthroses or required minor surgeries were treated by a combined concomitant administration of FVIII+FEIBA as IV bolus doses...
June 27, 2017: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/28688133/in-silico-evaluation-of-limited-blood-sampling-strategies-for-individualized-recombinant-factor-ix-prophylaxis-in-hemophilia-b-patients
#20
T Preijers, H C A M Hazendonk, K Fijnvandraat, F W G Leebeek, M H Cnossen, R A A Mathôt
BACKGROUND: Patients with severe hemophilia B regularly administer prophylactic intravenous doses of clotting factor IX (FIX) concentrate to maintain a trough level of at least 0.01 IUmL(-1) in order to prevent joint bleeds. Assessment of individual pharmacokinetic (PK) parameters allows individualization of the recombinant FIX (rFIX) dose. AIM: This in silico study aims to evaluate the predictive performance of limited sampling strategies (LSSs) with 1 to 3 samples to estimate individual PK parameters of rFIX...
July 8, 2017: Journal of Thrombosis and Haemostasis: JTH
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