Cargando…

Design of a prospective observational study on the effectiveness and real-world usage of recombinant factor VIII Fc (rFVIIIFc) compared with conventional products in haemophilia A: the A-SURE study

INTRODUCTION: Haemophilia A is a rare bleeding disorder caused by coagulation factor VIII (FVIII) deficiency. This is treated with factor VIII, conventionally using products with a half-life of 8–12 hours typically administered every 2–3 days. Recombinant FVIII Fc (rFVIIIFc) represents a new generat...

Descripción completa

Detalles Bibliográficos
Autores principales:	Oldenburg, Johannes, Hay, Charles R M, Jiménez-Yuste, Víctor, Peyvandi, Flora, Schved, Jean-François, Szamosi, Johan, Winding, Bent, Lethagen, Stefan
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BMJ Publishing Group 2019
Materias:	Haematology (Incl Blood Transfusion)
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6549707/ https://www.ncbi.nlm.nih.gov/pubmed/31152037 http://dx.doi.org/10.1136/bmjopen-2018-028012

Ejemplares similares

Desmopressin treatment combined with clotting factor VIII concentrates in patients with non-severe haemophilia A: protocol for a multicentre single-armed trial, the DAVID study
por: Schütte, Lisette M, et al.
Publicado: (2019)

AKATSUKI study: a prospective, multicentre, phase IV study evaluating the safety of emicizumab under and immediately after immune tolerance induction therapy in persons with congenital haemophilia A with factor VIII inhibitors
por: Matsushita, Tadashi, et al.
Publicado: (2022)

AOZORA: long-term safety and joint health in paediatric persons with haemophilia A without factor VIII inhibitors receiving emicizumab – protocol for a multicentre, open-label, phase IV clinical study
por: Shima, Midori, et al.
Publicado: (2022)

Study protocol for assessment of the coagulation potential of concomitantly used factor VIII concentrates in patients with haemophilia A with emicizumab prophylaxis (CAGUYAMA Study): a multicentre open-label non-randomised clinical trial
por: Takeyama, Masahiro, et al.
Publicado: (2023)

Design of the HEM-POWR study: a prospective, observational study of real-world treatment with damoctocog alfa pegol in patients with haemophilia A
por: Sanabria, Martin, et al.
Publicado: (2021)

Twelve-month prevalence of haemarthrosis and joint disease using the Haemophilia Joint Health score: evaluation of the UK National Haemophilia Database and Haemtrack patient reported data: an observational study
por: Wilkins, Richard A, et al.
Publicado: (2022)

Health-related quality of life of patients with haemophilia: a cross-sectional survey in the Northeast of China
por: Niu, Jinyu, et al.
Publicado: (2022)

Bone health in symptomatic carriers of haemophilia A: a protocol for a multicentre prospective matched-cohort study
por: Tang, Grace H, et al.
Publicado: (2019)

PHILEOS (haemoPHILia and ostEoporOSis) Study: protocol of a multicentre prospective case–control study
por: Tardy‐Poncet, Brigitte, et al.
Publicado: (2021)

Exploration of the impact of gene therapy on the lives of people with haemophilia and their families: a protocol for the mixed-methods exigency study
por: Fletcher, Simon, et al.
Publicado: (2022)

Protocol for a feasibility randomised controlled trial of a musculoskeletal exercise intervention versus usual care for children with haemophilia
por: Hashem, Ferhana, et al.
Publicado: (2019)

Pharmacokinetics and complementary evaluation system-based guidance on prophylaxis of paediatric patients with haemophilia A in China with Kovaltry: protocol of the LEAP study
por: Kun, Huang, et al.
Publicado: (2021)

Assessment of global coagulation function under treatment with emicizumab concomitantly with bypassing agents in haemophilia A with inhibitor (UNEBI Study): multicentre open-label non-randomised clinical trial
por: Ogiwara, Kenichi, et al.
Publicado: (2022)

Determinants of adherence and consequences of the transition from adolescence to adulthood among young people with severe haemophilia (TRANSHEMO): study protocol for a multicentric French national observational cross-sectional study
por: Resseguier, Noémie, et al.
Publicado: (2018)

DosEmi study protocol: a phase IV, multicentre, open-label, crossover study to evaluate non-inferiority of pharmacokinetic-guided reduced dosing compared with conventional dosing of emicizumab in people with haemophilia A
por: Donners, Anouk, et al.
Publicado: (2023)

Effectiveness of two psychological interventions for pain management, emotional regulation and promotion of quality of life among adult Portuguese men with haemophilia (PSY-HaEMOPEQ): study protocol for a single-centre prospective randomised controlled trial
por: Pinto, Patrícia Ribeiro, et al.
Publicado: (2017)

Risk Factors for Alloimmunisation after red blood Cell Transfusions (R-FACT): a case cohort study
por: Zalpuri, Saurabh, et al.
Publicado: (2012)

Patient self-assessment of hospital pain, mood and health-related quality of life in adults with sickle cell disease
por: Anie, Kofi A, et al.
Publicado: (2012)

Assessment of whole body MRI and sestamibi technetium-99m bone marrow scan in prediction of multiple myeloma disease progression and outcome: a prospective comparative study
por: Khalafallah, Alhossain A, et al.
Publicado: (2013)

Stem cell transplantation of matched sibling donors compared with immunosuppressive therapy for acquired severe aplastic anaemia: a Cochrane systematic review
por: Peinemann, Frank, et al.
Publicado: (2014)

Current practice of venous thromboembolism prevention in acute trusts: a qualitative study
por: McFarland, L, et al.
Publicado: (2014)

An audit of anticoagulant management to assess anticoagulant control using decision support software
por: Harper, Paul, et al.
Publicado: (2014)

Randomised controlled trial of weekly chloroquine to re-establish normal erythron iron flux and haemoglobin recovery in postmalarial anaemia
por: Cox, Sharon E, et al.
Publicado: (2013)

Intracranial haemorrhage in thrombocytopenic haematology patients—a nested case–control study: the InCiTe study protocol
por: Estcourt, Lise J, et al.
Publicado: (2014)

HFE gene mutation and oxidative damage biomarkers in patients with myelodysplastic syndromes and its relation to transfusional iron overload: an observational cross-sectional study
por: De Souza, Geane Felix, et al.
Publicado: (2015)

A study protocol for a randomised controlled trial evaluating clinical effects of platelet transfusion products: the Pathogen Reduction Evaluation and Predictive Analytical Rating Score (PREPAReS) trial
por: Ypma, Paula F, et al.
Publicado: (2016)

Validation of the multivariable In-hospital Mortality for PulmonAry embolism using Claims daTa (IMPACT) prediction rule within an all-payer inpatient administrative claims database
por: Coleman, Craig I, et al.
Publicado: (2015)

Characteristics of platelet indices, neutrophil-to-lymphocyte ratio and erythrocyte sedimentation rate compared with C reactive protein in patients with cerebral infarction: a retrospective analysis of comparing haematological parameters and C reactive protein
por: Lee, Jong-Han, et al.
Publicado: (2014)

Use of heparins in patients with cancer: individual participant data meta-analysis of randomised trials study protocol
por: Schünemann, Holger J, et al.
Publicado: (2016)

Protocol for updating a systematic review of randomised controlled trials on the prophylactic use of intravenous immunoglobulin for patients undergoing haematopoietic stem cell transplantation
por: Cowan, Juthaporn, et al.
Publicado: (2015)

Clinical effects of blood donor characteristics in transfusion recipients: protocol of a framework to study the blood donor–recipient continuum
por: Chassé, Michaël, et al.
Publicado: (2015)

Is self-monitoring an effective option for people receiving long-term vitamin K antagonist therapy? A systematic review and economic evaluation
por: Sharma, Pawana, et al.
Publicado: (2015)

Mutation screening for thalassaemia in the Jino ethnic minority population of Yunnan Province, Southwest China
por: Wang, Shiyun, et al.
Publicado: (2015)

Health-related quality of life after pulmonary embolism: a cross-sectional study
por: Tavoly, Mazdak, et al.
Publicado: (2016)

Gender disparities in red blood cell transfusion in elective surgery: a post hoc multicentre cohort study
por: Gombotz, Hans, et al.
Publicado: (2016)

Aneurysmal SubArachnoid Hemorrhage—Red Blood Cell Transfusion And Outcome (SAHaRA): a pilot randomised controlled trial protocol
por: English, Shane W, et al.
Publicado: (2016)

Rationale and design of platelet transfusions in haematopoietic stem cell transplantation: the PATH pilot study
por: Tay, Jason, et al.
Publicado: (2016)

Third-generation CD28/4-1BB chimeric antigen receptor T cells for chemotherapy relapsed or refractory acute lymphoblastic leukaemia: a non-randomised, open-label phase I trial protocol
por: Tang, Xiao-Yi, et al.
Publicado: (2016)

Antithrombotic stewardship: a multidisciplinary team approach towards improving antithrombotic therapy outcomes during and after hospitalisation: a study protocol
por: Dreijer, Albert R, et al.
Publicado: (2016)

Is it feasible to conduct a randomised controlled trial of pretransplant exercise (prehabilitation) for patients with multiple myeloma awaiting autologous haematopoietic stem cell transplantation? Protocol for the PREeMPT study
por: Keen, Carol, et al.
Publicado: (2018)

Cannot write session to /tmp/vufind_sessions/sess_iv6phc5q4cmp44hhc2tghtdidv