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Haemophilia: the Official Journal of the World Federation of Hemophilia

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https://www.readbyqxmd.com/read/29781145/core-outcome-set-for-gene-therapy-in-haemophilia-results-of-the-corehem-multistakeholder-project
#1
A Iorio, M W Skinner, E Clearfield, D Messner, G F Pierce, M Witkop, S Tunis
BACKGROUND: Gene therapy trial results show potential to cure haemophilia A and haemophilia B. Securing broad access to a cure for a lifelong chronic disease is anticipated to face barriers at the individual and healthcare system levels, which can be partly mitigated by harmonized planning of clinical research studies. The aim of the coreHEM project was to determine the set of outcome measures required to evaluate efficacy, safety, comparative effectiveness and value of gene therapy for haemophilia...
May 20, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29741299/methodologies-for-data-collection-in-congenital-haemophilia-with-inhibitors-chwi-critical-assessment-of-the-literature-and-lessons-learned-from-recombinant-factor-viia
#2
REVIEW
C M Kessler, S Benchikh El Fegoun, A Worster
AIMS: To systematically review the effectiveness of on-demand treatment with recombinant coagulation factor VIIa (rFVIIa) in congenital haemophilia with inhibitors and, if feasible, perform a meta-analysis of the data. MATERIALS AND METHODS: Publications from Embase® , MEDLINE® , MEDLINE® In-Process and the Cochrane Central Register of Controlled Trials were searched. Selected publications were reviewed for inclusion by two independent expert reviewers. Discrepancies were reconciled by a third independent reviewer...
May 9, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29732708/pharmacokinetic-modelling-and-validation-of-the-half-life-extension-needed-to-reduce-the-burden-of-infusions-compared-with-standard-factor-viii
#3
C Hermans, J Mahlangu, J Booth, H Schütz, E Santagostino, G Young, H-Y Lee, K N Steinitz-Trost, V Blanchette, E Berntorp
INTRODUCTION: Currently, no universally accepted definition of extended half-life (EHL) recombinant FVIII (rFVIII) exists. Identifying the minimum half-life extension ratio required for a reduction in dosing frequency compared with standard rFVIII could enable a more practical approach to decisions around prophylaxis with EHL rFVIII. AIM: To identify the half-life extension ratio required to decrease rFVIII dosing frequency by at least 1 day while maintaining the proportion of patients with plasma rFVIII levels above 1 IU/dL and without increasing the total weekly dose...
May 6, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29732654/diagnostic-delay-in-acquired-haemophilia-analysis-of-causes-and-consequences-in-a-20-year-spanish-cohort
#4
LETTER
J Pardos-Gea, N Fernández-Díaz, R Parra, V Cortina, C Altisent
No abstract text is available yet for this article.
May 6, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29732648/the-multiple-benefits-of-sport-in-haemophilia
#5
A Farrugia, A Gringeri, S von Mackensen
No abstract text is available yet for this article.
May 6, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29732646/ddavp-trial-in-discrepant-non-severe-haemophilia-a-patients
#6
LETTER
H C Okoye, B I Nielsen, K Lee, Y L Abajas, N S Key, M A Rollins-Raval
No abstract text is available yet for this article.
May 6, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29726070/a-comparative-analysis-of-heterogeneity-in-commercially-available-recombinant-factor-viii-products
#7
D Baunsgaard, A D Nielsen, P F Nielsen, A Henriksen, A K Kristensen, H W Bagger, M Ezban
INTRODUCTION: Advances in analytical technologies enable investigation of possible correlations between molecular structure, aggregation and subvisible particle content. Regulatory agencies place increasing attention on potential risks associated with protein aggregates in the micron range in biological therapeutics. AIM: Assess the heterogeneity, high-molecular-weight protein (HMWP) species, subvisible particle content and posttranslational modifications in six commercially available recombinant FVIII (rFVIII) products...
May 3, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29707881/prophylactic-treatment-of-bleeding-episodes-in-children-12-years-with-moderate-to-severe-hereditary-factor-x-deficiency-fxd-efficacy-and-safety-of-a-high-purity-plasma-derived-factor-x-pdfx-concentrate
#8
R Liesner, C Akanezi, M Norton, J Payne
BACKGROUND: Hereditary factor X (FX) deficiency (FXD) affects 1:500 000-1:1 000 000 people worldwide. A novel, high-purity plasma-derived FX concentrate (pdFX) is available in the United States and European Union as replacement therapy for FXD, but data are scarce on pdFX use in children <12 years. AIM: This prospective, open-label phase 3 study assessed the safety, efficacy and pharmacokinetics of pdFX in children <12 years with moderate/severe FXD. METHODS: Subjects aged <12 years with basal plasma FX activity (FX:C) <5 IU/dL received pdFX as prophylactic and on-demand treatment, with doses adjusted to maintain FX:C > 5 IU/dL...
April 30, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29707874/total-elbow-arthroplasty-in-haemophilia
#9
REVIEW
T M Dale, J M Saucedo, E C Rodriguez-Merchan
INTRODUCTION: Advanced haemophilic arthropathy of the elbow can be a devastating manifestation of severe haemophilia with significant implications for activities of daily living. AIM: To summarize the currently available data on total elbow arthroplasty (TEA) for haemophilic arthropathy of the elbow, to draw comparisons to TEA for other indications, and to outline some considerations regarding TEA in this unique population. METHODS: A narrative review of the literature has been performed...
April 30, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29707861/perioperative-replacement-therapy-in-haemophilia-b-an-appeal-to-b-more-precise
#10
H C A M Hazendonk, T Preijers, R Liesner, P Chowdary, D Hart, D Keeling, M H E Driessens, B A P Laros-van Gorkom, F J M van der Meer, K Meijer, K Fijnvandraat, F W G Leebeek, R A A Mathôt, P W Collins, M H Cnossen
INTRODUCTION: Haemophilia B is caused by a deficiency of coagulation factor IX (FIX) and characterized by bleeding in muscles and joints. In the perioperative setting, patients are treated with FIX replacement therapy to secure haemostasis. Targeting of specified FIX levels is challenging and requires frequent monitoring and adjustment of therapy. AIM: To evaluate perioperative management in haemophilia B, including monitoring of FIX infusions and observed FIX levels, whereby predictors of low and high FIX levels were assessed...
April 29, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29673013/postural-control-during-a-transition-task-in-haemophilic-children-adolescents-and-young-adults-with-haemophilic-ankle-arthropathy
#11
K Deschamps, F Staes, M Eerdekens, K Peerlinck, C Hermans, J Vandesande, S Lobet
BACKGROUND: The aim of this study was to determine whether young haemophilic boys with and without MRI-based signs of ankle arthropathy demonstrate reduced balance ability during a transition task with eyes open and eyes closed. METHODS: Thirty-four haemophilic bodies and 28 typically developing boys aged 6-20 years participated to this study. Structural integrity of the tarsal foot joints of all haemophilic boys was assessed with MRI. All participants performed a standard transition task from double-leg stance to single-leg stance with eyes open and eyes closed...
April 19, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29665224/type-3-von-willebrand-disease-mistaken-for-moderate-haemophilia-a-a-lesson-still-to-be-learned
#12
LETTER
E Galletta, V Daidone, E Zanon, S Casonato
No abstract text is available yet for this article.
April 17, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29665194/replacement-therapy-during-surgery-in-von-willebrand-disease-needs-personalization
#13
E Berntorp
No abstract text is available yet for this article.
April 17, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29665188/the-interaction-of-factor-v-and-tissue-factor-pathway-inhibitor-in-a-myeloma-patient-with-acquired-factor-v-deficiency
#14
LETTER
E De Maertelaere, E Castoldi, I Van Haute, D Deeren, K M Devreese
No abstract text is available yet for this article.
April 17, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29665146/frequencies-of-intron-1-and-22-inversions-of-factor-viii-gene-a-first-report-in-afghan-patients-with-severe-haemophilia-a
#15
LETTER
S H Mousavi, S A Mesbah-Namin, N Rezaie, S Zeinali
No abstract text is available yet for this article.
April 17, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29665141/screening-of-haemophilia-carriers-in-moderate-and-severe-haemophilia-a-and-b-prevalence-and-determinants
#16
LETTER
W Bernard, C Lambert, S Henrard, C Hermans
No abstract text is available yet for this article.
April 17, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29665138/haemophilia-commentary-the-utility-of-bats
#17
M Sholzberg
No abstract text is available yet for this article.
April 17, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29656540/management-of-pregnancy-complications-in-type-2n-von-willebrand-disease-associated-to-a-novel-mutation
#18
LETTER
Y Brennan, E J Favaloro, J Curtin, J Curnow
No abstract text is available yet for this article.
April 14, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29656491/recurrent-f8-and-f9-gene-variants-result-from-a-founder-effect-in-two-large-french-haemophilia-cohorts
#19
F Lassalle, O Marmontel, C Zawadzki, M Fretigny, P Bouvagnet, C Vinciguerra
INTRODUCTION: Haemophilia A (HA) and haemophilia B (HB) are X-linked recessive diseases, caused by a large number of pathogenic variants in the F8 and F9 genes. With the exception of introns 22 and 1 inversions which are frequent in severe HA cases, about 2000 unique variants in F8 and 1000 in F9 have been described in databases and their recurrence remains limited. AIM AND METHODS: During routine analysis, we identified two recurrent missense variants, the F8 gene c...
April 14, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29635852/intracranial-haemorrhage-in-children-with-inherited-bleeding-disorders-in-the-uk-2003-2015-a-national-cohort-study
#20
E A Chalmers, J Alamelu, P W Collins, M Mathias, J Payne, M Richards, O Tunstall, M Williams, B Palmer, A Mumford
INTRODUCTION: Intracranial haemorrhage in children with inherited bleeding disorders is a potentially life-threatening complication and presents a significant therapeutic challenge. AIM: To define the characteristics, management and outcomes of intracranial haemorrhage presenting in UK children ≤16 years of age with inherited bleeding disorders from 2003 to 2015. METHOD: Retrospective analysis of children treated at UK haemophilia centres...
April 10, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
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