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Haemophilia: the Official Journal of the World Federation of Hemophilia

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https://www.readbyqxmd.com/read/30408848/concizumab-restores-thrombin-generation-potential-in-patients-with-haemophilia-pharmacokinetic-pharmacodynamic-modelling-results-of-concizumab-phase-1-1b-data
#1
Hermann Eichler, Pantep Angchaisuksiri, Kaan Kavakli, Paul Knoebl, Jerzy Windyga, Victor Jiménez-Yuste, Philip Harder Delff, Pratima Chowdary
INTRODUCTION: Concizumab enhances thrombin generation (TG) potential in haemophilia patients by inhibiting tissue factor pathway inhibitor (TFPI). In EXPLORER3 (phase 1b), a dose-dependent pharmacokinetic/pharmacodynamic (PK/PD) relationship was confirmed between concizumab dose, free TFPI and TG potential. AIM: Determine the association between concizumab exposure, PD markers (free TFPI; peak TG) and bleeding episodes to establish the minimum concizumab concentration for achieving sufficient efficacy...
November 8, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30408840/severe-acquired-von-willebrand-syndrome-secondary-to-systemic-lupus-erythematosus
#2
Francesca Stufano, Luciano Baronciani, Eugenia Biguzzi, Giovanna Cozzi, Paola Colpani, Marta Chisini, Flora Peyvandi
No abstract text is available yet for this article.
November 8, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30408835/treatment-regimens-and-outcomes-in-severe-and-moderate-haemophilia-a-in-the-uk-the-thunder-study
#3
Martin J Scott, Hua Xiang, Daniel P Hart, Benedict Palmer, Peter W Collins, David Stephensen, Camelia S Sima, Charles R M Hay
INTRODUCTION: The THUNDER study provides an analysis of treatment patterns and outcomes in UK patients with severe or moderate haemophilia A (SHA/MHA) in 2015. METHODS: Patients with SHA or MHA registered with the UK National Haemophilia Database (NHD) were segregated by severity, inhibitor status and age. Haemophilia joint health score (HJHS) was derived from NHD records and treatment regimen and annualized bleed/joint-bleed rate (ABR/AJBR) from Haemtrack (HT) in HT-compliant patients...
November 8, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30402994/long-term-safety-and-efficacy-of-turoctocog-alfa-in-prophylaxis-and-treatment-of-bleeding-episodes-in-severe-haemophilia-a-final-results-from-the-guardian-2-extension-trial
#4
Steven R Lentz, Dragana Janic, Kaan Kavakli, Predrag Miljic, Johannes Oldenburg, Margareth C Ozelo, Elena Santagostino, Takashi Suzuki, Silva Zupancic Šalek, Lars Korsholm, Irina Matytsina, Andreas Tiede
INTRODUCTION: Turoctocog alfa is a recombinant factor VIII (FVIII) molecule, approved for treatment and prophylaxis of bleeding in patients with haemophilia A. In the guardian 1 (adolescents/adults) and guardian 3 (children) phase 3 trials, turoctocog alfa demonstrated a favourable efficacy and safety profile. Guardian 1 or 3 completers could enrol in the guardian 2 extension. Final guardian 2 results are reported here. AIM: Investigate long-term safety and efficacy of turoctocog alfa administered for prophylaxis and treatment of bleeds...
November 6, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30394622/the-prevalence-and-burden-of-hand-and-wrist-bleeds-in-von-willebrand-disease
#5
LETTER
Désirée van Deukeren, Eveline P Mauser-Bunschoten, Roger E G Schutgens, Jeroen Eikenboom, Karina Meijer, Karin Fijnvandraat, Britta A P Laros-van Gorkom, Marjon Cnossen, Joke de Meris, Johanna G van der Bom, Frank W G Leebeek, Karin P M van Galen
No abstract text is available yet for this article.
November 5, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30394617/continuous-infusion-of-simoctocog-alfa-in-haemophilia-a-patients-undergoing-surgeries
#6
Pål Andre Holme, Geir E Tjønnfjord
INTRODUCTION: There are two major principles for coagulation factor replacement in the clinical management of surgical procedures in patients with haemophilia, repetitive bolus injections every 6-12 hours or administration of coagulation factor concentrates by continuous infusion. AIM: The aim was to investigate the efficacy of simoctocog alfa (human-cl rhFVIII) delivered by continuous infusion for bleeding prophylaxis during surgery in patients with haemophilia A...
November 5, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30375147/assessment-of-tensile-mechanical-properties-of-the-achilles-tendon-in-adult-patients-with-haemophilic-arthropathy-reproducibility-study
#7
Carlos Cruz-Montecinos, Sofía Pérez-Alenda, Felipe Contreras-Sepúlveda, Felipe Querol, Mauricio Cerda, Huub Maas
No abstract text is available yet for this article.
October 30, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30345592/thromboelastography-and-thrombin-generation-assay-in-inherited-afibrinogenemia
#8
Guy Aaron Young, Roxana Carmona, Viridiana Cano Garcia
Fibrinogen is a glycoprotein with a crucial role in blood coagulation. Upon enzymatic cleavage by thrombin, fibrinogen is converted from its soluble form to insoluble fibrin which is key structural protein of a clot. It also participates in platelet aggregation by binding to GPIIb/IIIa. Genetic alterations can lead to either complete or partial, quantitative or qualitative defects of fibrinogen. Inherited afibrinogenemia is a rare bleeding disorder with autosomal recessive inheritance due to a complete absence of fibrinogen...
October 21, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30311330/improving-access-to-haemophilia-therapies-beyond-humanitarian-aid
#9
LETTER
Albert Farrugia, Giancarlo Liumbruno, Gabriele Calizzani, Fabio Candura, Samantha Profili, Josephine Cassar
No abstract text is available yet for this article.
October 12, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30299569/use-of-two-complementary-new-molecular-techniques-next-generation-sequencing-and-droplet-digital-pcr-for-diagnosis-of-an-f8-gene-deletion-and-subsequent-carrier-analysis-in-a-family-with-haemophilia-a-a-case-report
#10
LETTER
Priyanka Gangodkar, Shatakshi Ranade, Siddharth Anand, Ashwini Bapat, Kavita Khatod, Parth Shah, Meenal Agarwal, Nikhil Phadke
No abstract text is available yet for this article.
October 9, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30295404/investigating-the-relationship-between-the-hjhs-and-hal-in-routine-clinical-practice-a-retrospective-review
#11
Paul McLaughlin, Richard Morris, Pratima Chowdary
INTRODUCTION: Comprehensive musculoskeletal assessment for monitoring joint health in haemophilia includes both physical assessment with Haemophilia Joint Health Score (HJHS) and assessment of self-reported function by Haemophilia Activities List (HAL). METHODS: Correlation between physical assessment and joint function was undertaken between HJHS and HAL in patients with SHA and SHB who had both assessments at the same visit over a one-year period. RESULTS: Data from 120 patients (96-SHA/24 = SHB) with a median age 33 years (range 19-73) were included...
October 8, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30295389/bleeding-events-and-safety-outcomes-in-persons-with-haemophilia-a-with-inhibitors-a-prospective-multi-centre-non-interventional-study
#12
Johnny Mahlangu, Johannes Oldenburg, Michael U Callaghan, Midori Shima, Elena Santagostino, Maggie Moore, Michael Recht, Claudia Garcia, Renchi Yang, Michaela Lehle, Harrison Macharia, Elina Asikanius, Gallia G Levy, Rebecca Kruse-Jarres
INTRODUCTION: Prospectively collected, real-world data on bleeds, haemophilic treatment and safety outcomes in persons with haemophilia A (PwHA) with factor VIII (FVIII) inhibitors are limited. A prospective, global, multi-centre, non-interventional study (NIS; NCT02476942) collected detailed real-world data in PwHA treated per local routine clinical practice. AIM: To characterize bleeding rates, haemophilic treatment practices, prophylaxis adherence and adverse events (AEs) in adult/adolescent PwHA with inhibitors in the NIS...
October 8, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30273987/usefulness-of-bone-microarchitectural-and-geometric-dxa-derived-parameters-in-haemophilic-patients
#13
Fabio Massimo Ulivieri, Giulia Antonella Angela Rebagliati, Luca Petruccio Piodi, Luigi Piero Solimeno, Gianluigi Pasta, Elena Boccalandro, Maria Rosaria Fasulo, Maria Elisa Mancuso, Elena Santagostino
INTRODUCTION: Haemophilia is a recessive X-linked inherited bleeding disorder, whose typical symptom is spontaneous intra-articular haemorrhage leading to joint damage, which can be quantified by the Haemophilia Joint Health Score (HJHS). Arthropathy and other characteristics of haemophilic patients may reduce bone mineral density (BMD), increasing the risk for fragility fractures, which also may occur due to bone quality impairment. AIM: To evaluate bone quantity by BMD and bone quality by Trabecular Bone Score (TBS), bone strain (BS) and hip structural analysis (HSA) in a haemophilic population, and to relate these parameters to general and specific risk factors for osteoporosis and to HJHS...
October 1, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30259602/association-between-abo-blood-group-and-bleeding-phenotype-in-patients-with-mild-rare-bleeding-disorders
#14
Luca Spiezia, Elena Campello, Giacomo Turatti, Chiara Simion, Mariangela Fadin, Sabrina Gavasso, Graziella Saggiorato, Francesca Sartorello, Patrizia Zerbinati, Paolo Simioni
No abstract text is available yet for this article.
September 27, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30248217/once-weekly-prophylaxis-with-40-iu-kg-nonacog-beta-pegol-n9-gp-achieves-trough-levels-of-15-in-patients-with-haemophilia-b-pooled-data-from-the-paradigm%C3%A2-trials
#15
Johannes Oldenburg, Manuel Carcao, Steven R Lentz, Johnny Mahlangu, Maria Elisa Mancuso, Tadashi Matsushita, Claude Négrier, Wan Hui Ong Clausen, Silke Ehrenforth, Guy Young
INTRODUCTION: Prophylaxis with replacement factor IX (FIX) reduces bleeding frequency and improves quality of life in haemophilia B patients. With prophylaxis, the likelihood of bleeding is lowered with increasing trough levels. New products with extended half-life (EHL) can maintain high factor activity levels over prolonged periods, compared with standard FIX products. AIM: To evaluate the safety, efficacy and pharmacokinetics of the new recombinant FIX EHL product, nonacog beta pegol (N9-GP), using pooled data, with a focus on-but not limited to-prophylaxis at 40 IU/kg...
September 24, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30248215/impact-of-intermediate-dose-prophylaxis-on-progression-of-haemarthropathy-in-patients-with-severe-haemophilia-a-a-10-year-single-centre-experience-in-korea
#16
Ju Y Kim, Da J Lee, Tong J Chun, Chur W You
AIM: To determine the impact of 10-year intermediate-dose prophylaxis on haemarthropathy progression in patients with severe haemophilia A (SHA). METHODS: Prophylactic treatment with intermediate dose was given maximally for 10 years to 42 patients with SHA in a haemophilia treatment centre in Korea. Patients were divided into three groups based on prophylactic treatment started age: 1-10 (group A'), 11-20 (group B'), and ≥21 (group C'). Average annual increase of Pettersson score (P-score) was compared between the treatment groups...
September 24, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30207633/utility-of-a-screening-tool-for-haemostatic-defects-in-a-multicentre-cohort-of-adolescents-with-heavy-menstrual-bleeding
#17
Ayesha Zia, Joseph Stanek, Myra Christian-Rancy, Sanjay P Ahuja, Stephanie Savelli, Sarah H O'Brien
INTRODUCTION: Heavy menstrual bleeding (HMB) may be expected for many adolescents after menarche. Accurate assessment of HMB, a key component in the diagnosis of a haemostatic defect (HD), is a well-recognized challenge. AIM: Our objective was to determine the diagnostic accuracy of an HMB-specific screening tool for HDs in adolescents with HMB, presenting to a secondary care setting. METHODS: Adolescents with HMB were evaluated for a HD at 4 US centres...
September 12, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30175490/orthotopic-liver-transplantation-for-haemophilia-a-may-not-always-lead-to-a-phenotypic-cure-of-haemophilia-a-a-case-report
#18
LETTER
Yuji Suzuki, Keisuke Kakisaka, Tomoko Matsumoto, Keiji Nogami, Hirokatsu Katagiri, Takeshi Takahara, Yasuhiro Takikawa
No abstract text is available yet for this article.
September 3, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30175488/identification-of-a-homozygous-missense-mutation-p-cys379gly-in-the-d1-domain-of-von-willebrand-factor-propeptide-in-a-family-with-type-2a-iic-von-willebrand-disease
#19
LETTER
Toshio Shigekiyo, Kengo Udaka, Etsuko Sekimoto, Hironobu Shibata, Shuji Ozaki, Yukio Higuchi, Masanori Matsumoto
No abstract text is available yet for this article.
September 3, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/30144219/challenges-in-diagnosis-and-management-of-acquired-factor-xiii-fxiii-inhibitors
#20
LETTER
Joan D Beckman, Raj S Kasthuri, Alisa S Wolberg, Alice D Ma
No abstract text is available yet for this article.
August 24, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
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