Prophylaxis use of clotting factor replacement products in people with non‐severe haemophilia: A review of the literature

People with non‐severe haemophilia appear to be under‐treated in many countries, and this may lead to joint damage and worsen quality of life.

[1]  M. Kruip,et al.  Joint status of patients with nonsevere hemophilia A , 2022, Journal of thrombosis and haemostasis : JTH.

[2]  J. Oldenburg,et al.  Emicizumab Prophylaxis in Persons with Mild or Moderate Hemophilia A: Results from the Interim Analysis of the HAVEN 6 Study , 2021, Blood.

[3]  C. Hermans,et al.  Optimising prophylaxis in haemophilia A: The ups and downs of treatment. , 2021, Blood reviews.

[4]  Michael Wang,et al.  Impact of haemophilia on patients with mild‐to‐moderate disease: Results from the P‐FiQ and B‐HERO‐S studies , 2021, Haemophilia : the official journal of the World Federation of Hemophilia.

[5]  D. Cooper,et al.  Identified unmet needs and proposed solutions in mild‐to‐moderate haemophilia: A summary of opinions from a roundtable of haemophilia experts , 2021, Haemophilia : the official journal of the World Federation of Hemophilia.

[6]  R. Sidonio,et al.  Women and girls with haemophilia and bleeding tendencies: Outcomes related to menstruation, pregnancy, surgery and other bleeding episodes from a retrospective chart review , 2020, Haemophilia : the official journal of the World Federation of Hemophilia.

[7]  A. Iorio,et al.  Non‐severe haemophilia: Is it benign? – Insights from the PROBE study , 2020, Haemophilia : the official journal of the World Federation of Hemophilia.

[8]  F. Leebeek,et al.  How I manage pregnancy in carriers of hemophilia and patients with von Willebrand disease. , 2020, Blood.

[9]  E. Santagostino,et al.  WFH Guidelines for the Management of Hemophilia, 3rd edition , 2020, Haemophilia : the official journal of the World Federation of Hemophilia.

[10]  J. Astermark,et al.  Joint health and treatment modalities in Nordic patients with moderate haemophilia A and B – The MoHem study , 2020, Haemophilia : the official journal of the World Federation of Hemophilia.

[11]  K. Nogami,et al.  Emicizumab Improves Ex Vivo Clotting Function in Patients with Mild/Moderate Hemophilia A , 2020, Thrombosis and Haemostasis.

[12]  C. Königs,et al.  Long‐term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A , 2020, Haemophilia : the official journal of the World Federation of Hemophilia.

[13]  Aaas News,et al.  Book Reviews , 1893, Buffalo Medical and Surgical Journal.

[14]  Jeffrey S. Stonebraker,et al.  Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males , 2019, Annals of Internal Medicine.

[15]  F. Peyvandi,et al.  Burden of mild haemophilia A: Systematic literature review , 2019, Haemophilia : the official journal of the World Federation of Hemophilia.

[16]  Katherine Fennedy,et al.  Moderate , 2019, Definitions.

[17]  S. Polinder,et al.  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 , 2019, BMJ Open.

[18]  E. Berntorp.  Moderate haemophilia in focus , 2019, Haemophilia : the official journal of the World Federation of Hemophilia.

[19]  S. Pérez-Alenda,et al.  Bayesian pharmacokinetic-guided prophylaxis with recombinant factor VIII in severe or moderate haemophilia A. , 2019, Thrombosis research.

[20]  M. Scott,et al.  Treatment regimens and outcomes in severe and moderate haemophilia A in the UK: The THUNDER study , 2018, Haemophilia : the official journal of the World Federation of Hemophilia.

[21]  F. Peyvandi,et al.  Risk factors for inhibitor development in severe hemophilia a. , 2018, Thrombosis research.

[22]  Adrienne Lee,et al.  Desmopressin in non‐severe haemophilia A: Test‐response and clinical outcomes in a single Canadian centre review , 2018, Haemophilia : the official journal of the World Federation of Hemophilia.

[23]  N. Key,et al.  DDAVP trial in discrepant non‐severe haemophilia A patients , 2018, Haemophilia : the official journal of the World Federation of Hemophilia.

[24]  S. Parikh,et al.  Australian multicentre study of current real‐world prophylaxis practice in severe and moderate haemophilia A and B , 2018, Haemophilia : the official journal of the World Federation of Hemophilia.

[25]  S. Cessie,et al.  Intensity of factor VIII treatment and the development of inhibitors in non‐severe hemophilia A patients: results of the INSIGHT case–control study , 2017, Journal of thrombosis and haemostasis : JTH.

[26]  M. Nijziel,et al.  Mortality caused by intracranial bleeding in non‐severe hemophilia A patients , 2017, Journal of thrombosis and haemostasis : JTH.

[27]  David B Clark,et al.  Impact of mild to severe hemophilia on engagement in recreational activities by US men, women, and children with hemophilia B: The Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B‐HERO‐S) study , 2017, European journal of haematology.

[28]  K. Khair,et al.  Treatment burden, haemostatic strategies and real world inhibitor screening practice in non‐severe haemophilia A , 2017, British journal of haematology.

[29]  J. Ackley.  Evidence-based nursing care guidelines , 2016 .

[30]  L. Kitanovski,et al.  Low-dose continuous infusion of factor VIII in patients with haemophilia A. , 2016, Blood transfusion = Trasfusione del sangue.

[31]  M. Schemper,et al.  FVIII-binding IgG modulates FVIII half-life in patients with severe and moderate hemophilia A without inhibitors. , 2016, Blood.

[32]  B. Riske,et al.  Men with severe hemophilia in the United States: birth cohort analysis of a large national database. , 2016, Blood.

[33]  T. Buckner,et al.  Management of adult non‐severe haemophilia A patients with inhibitors: a practice‐pattern survey , 2015, Haemophilia : the official journal of the World Federation of Hemophilia.

[34]  A. Iorio,et al.  Inhibitor development in non-severe haemophilia across Europe. , 2015, Thrombosis and haemostasis.

[35]  J. Bom,et al.  Inhibitor development and mortality in non‐severe hemophilia A , 2015, Journal of thrombosis and haemostasis : JTH.

[36]  J. Eikenboom,et al.  Primary postpartum haemorrhage in women with von Willebrand disease or carriership of haemophilia despite specialised care: a retrospective survey , 2015, Haemophilia : the official journal of the World Federation of Hemophilia.

[37]  D. Moss,et al.  A large‐scale computational study of inhibitor risk in non‐severe haemophilia A , 2015, British journal of haematology.

[38]  J. Mangel,et al.  Desmopressin responsiveness at a capped dose of 15 &mgr;g in type 1 von Willebrand disease and mild hemophilia A , 2014, Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis.

[39]  A. Lienhart,et al.  Diagnosis and management challenges in patients with mild haemophilia A and discrepant FVIII measurements , 2014, Haemophilia : the official journal of the World Federation of Hemophilia.

[40]  A. Ganguly,et al.  Severe and moderate haemophilia A and B in US females , 2014, Haemophilia : the official journal of the World Federation of Hemophilia.

[41]  D. Grobbee,et al.  Outcome in moderate haemophilia. , 2014, Blood transfusion = Trasfusione del sangue.

[42]  M. Franchini,et al.  Clinical Efficacy and Determinants of Response to Treatment with Desmopressin in Mild Hemophilia A , 2013, Seminars in Thrombosis & Hemostasis.

[43]  J. Astermark,et al.  GENERAL INTRODUCTION AND OUTLINE OF THE THESIS , 2012 .

[44]  B. Konkle,et al.  Factor VIII mutation and desmopressin‐responsiveness in 62 patients with mild haemophilia A , 2013, Haemophilia : the official journal of the World Federation of Hemophilia.

[45]  F. Leebeek,et al.  Response to desmopressin is strongly dependent on F8 gene mutation type in mild and moderate haemophilia A , 2013, Thrombosis and Haemostasis.

[46]  X. Zhang,et al.  Short‐term low‐dose secondary prophylaxis for severe/moderate haemophilia A children is beneficial to reduce bleed and improve daily activity, but there are obstacle in its execution: a multi‐centre pilot study in China , 2013, Haemophilia : the official journal of the World Federation of Hemophilia.

[47]  T. Nichols,et al.  Phase II prospective open-label trial of recombinant interleukin-11 in desmopressin-unresponsive von Willebrand disease and mild or moderate haemophilia A , 2012, Thrombosis and Haemostasis.

[48]  C. Kempton,et al.  Eradication of factor viii inhibitors in patients with mild and moderate hemophilia A , 2012, American journal of hematology.

[49]  E. Berntorp,et al.  Quality of life in adult patients with haemophilia – a single centre experience from Sweden , 2012, Haemophilia : the official journal of the World Federation of Hemophilia.

[50]  F. Leebeek,et al.  Inhibitor incidence after intensive FVIII replacement for surgery in mild and moderate haemophilia A: a prospective national study in the Netherlands , 2012, British journal of haematology.

[51]  K. Fischer,et al.  Risk of inhibitor development in mild haemophilia A increases with age , 2012, Haemophilia : the official journal of the World Federation of Hemophilia.

[52]  M. Carcao,et al.  DDAVP responsiveness in children with mild or moderate haemophilia A correlates with age, endogenous FVIII:C level and with haemophilic genotype , 2012, Haemophilia : the official journal of the World Federation of Hemophilia.

[53]  R. Liesner,et al.  Guideline on the management of haemophilia in the fetus and neonate * , 2011, British journal of haematology.

[54]  C. Manno,et al.  Associations between intracranial haemorrhage and prescribed prophylaxis in a large cohort of haemophilia patients in the United States , 2011, British journal of haematology.

[55]  C. Kempton,et al.  In non‐severe hemophilia A the risk of inhibitor after intensive factor treatment is greater in older patients: a case–control study , 2010, Journal of thrombosis and haemostasis : JTH.

[56]  M. Morfini,et al.  Mortality and causes of death in Italian persons with haemophilia, 1990–2007 , 2010, Haemophilia : the official journal of the World Federation of Hemophilia.

[57]  P. Mannucci,et al.  Molecular and phenotypic determinants of the response to desmopressin in adult patients with mild hemophilia A , 2009, Journal of thrombosis and haemostasis : JTH.

[58]  F. Rosendaal,et al.  Clinical outcome of moderate haemophilia compared with severe and mild haemophilia , 2009, Haemophilia : the official journal of the World Federation of Hemophilia.

[59]  K. Lindvall,et al.  Compliance with treatment and understanding of own disease in patients with severe and moderate haemophilia , 2006, Haemophilia : the official journal of the World Federation of Hemophilia.

[60]  V. Blanchette,et al.  DDAVP challenge tests in boys with mild/moderate haemophilia A * , 2002, British journal of haematology.

[61]  J. Gill,et al.  Evaluation of high concentration intranasal and intravenous desmopressin in pediatric patients with mild hemophilia A or mild-to-moderate type 1 von Willebrand disease. , 2002, The Journal of pediatrics.

[62]  C. Leissinger,et al.  High‐dose DDAVP intranasal spray (Stimate®) for the prevention and treatment of bleeding in patients with mild haemophilia A, mild or moderate type 1 von Willebrand disease and symptomatic carriers of haemophilia A , 2001, Haemophilia : the official journal of the World Federation of Hemophilia.

[63]  F. Rosendaal,et al.  Definitions in Hemophilia , 2001, Thrombosis and Haemostasis.

[64]  H. Hemker,et al.  The Effect of DDAVP Infusion on Thrombin Generation in Platelet-rich Plasma of von Willebrand Type 1 and in Mild Haemophilia A Patients , 2000, Thrombosis and Haemostasis.

[65]  E F Furstman,et al.  In Focus , 2016, Journal - Southern California Dental Association.

[66]  Masoud Parsania,et al.  THE PATIENTS , 1982, California state journal of medicine.