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haemophilia review

K Holstein, A Batorova, M Carvalho, K Fijnvandraat, P Holme, K Kavakli, T Lambert, A Rocino, V Jiménez-Yuste, J Astermark
INTRODUCTION: Inhibitor development in people with haemophilia is a serious complication that may require intensive and costly interventions. The goal of inhibitor management should be permanent inhibitor eradication through immune tolerance induction (ITI), but well-designed studies are lacking and the management of patients is therefore defined by the experience and views of the clinician. OBJECTIVES: To explore the current clinical practice and outcome of ITI therapy in Europe and how this may have changed over the last decade, as well as to provide consensus recommendations to guide clinicians in their clinical practice...
October 15, 2016: Thrombosis Research
K Fischer, P Poonnoose, A L Dunn, P Babyn, M J Manco-Johnson, J A David, J van der Net, B Feldman, K Berger, M Carcao, P de Kleijn, M Silva, P Hilliard, A Doria, A Srivastava, V Blanchette
INTRODUCTION: The implementation of early long-term, regular clotting factor concentrate (CFC) replacement therapy ('prophylaxis') has made it possible to offer boys with haemophilia a near normal life. Many different regimens have reported favourable results, but the optimum treatment regimens have not been established and the cost of prophylaxis is very high. Both for optimizing treatment and reimbursement issues, there is a need to provide objective evidence of both short- and long-term results and benefits of prophylactic regimens...
September 15, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
P de Kleijn, E P Mauser-Bunschoten, K Fischer, C Smit, H Holtslag, C Veenhof
INTRODUCTION: Musculoskeletal impact of haemophilia justifies physiotherapy throughout life. Recently the Dutch Health Care Institute constrained their 'list of chronic conditions', and withdrew financial coverage of physiotherapy for elderly persons with haemophilia (PWH). This decision was based on lack of scientific evidence and not being in accordance with 'state of science and practice'. METHODS: In general, evidence regarding physiotherapy is limited, and especially in rare diseases like haemophilia...
September 6, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
Gillian M Keating
BAY 81-8973 (octocog alfa; Kovaltry(®)) is an unmodified, full-length, recombinant factor VIII (FVIII) concentrate with the same amino acid sequence as Kogenate(®) FS, but produced with innovative manufacturing technologies. This narrative review discusses the clinical efficacy and tolerability of BAY 81-8973 in haemophilia A, as well as summarizing its pharmacological properties. Results of the LEOPOLD I, LEOPOLD II and LEOPOLD Kids trials demonstrated that routine prophylaxis with intravenous BAY 81-8973 was associated with a low annualized bleeding rate (ABR) in previously treated adult and paediatric patients with severe haemophilia A...
October 2016: BioDrugs: Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy
Anna See, Siti Radhziah Sudirman, Xin Yong Huang
BACKGROUND: Acquired haemophilia A is caused by the development of an autoantibody to factor VIII in a person with previously normal haemostasis. The most common clinical presentation in hereditary haemophilia is intra-articular bleeding. In contrast, acquired haemophilia more commonly presents with skin, soft tissue and mucosal haemorrhages. METHODS: We present a case of a patient with idiopathic acquired haemophilia A, whose initial presentation was that of spontaneous submental and submandibular haematoma which rapidly progressed to involve multiple sites in the airway, ultimately necessitating an emergent tracheotomy...
August 30, 2016: European Archives of Oto-rhino-laryngology
K M Lövgren, H Søndergaard, S Skov, B Wiinberg
In haemophilia A (HA) management, antidrug antibodies, or inhibitors, are a serious complication that renders factor VIII (FVIII) replacement therapy ineffective, increases morbidity and reduces quality of life for affected patients. Inhibitor development aetiology is multifactorial and covers both genetic and therapy related risk factors. Many therapy-related risk factors have proven difficult to confirm due to several confounding factors and the small study populations available. However, clinical studies indicate that e...
September 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
S M J Mortazavi, B Haghpanah, M M Ebrahiminasab, T Baghdadi, G Toogeh
INTRODUCTION: Haemophilic arthropathy (HA) is a debilitating complication of haemophilia which leads to TKA in severe cases. AIM: We conducted a prospective study of the outcome of TKA in our haemophilia cohort to define the outcomes in this population and increase the cost effectiveness of the procedure in our developing country. METHODS: We reviewed patients with haemophilia who underwent TKA between April 2010 and April 2014. Patients with at least 6 months of follow-up were included...
August 26, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
M D Tarantino, A Cuker, B Hardesty, J C Roberts, M Sholzberg
INTRODUCTION: A recombinant porcine factor VIII B-domain-deleted product (rpFVIII; OBIZUR, Baxalta Incorporated, Deerfield, IL 60015, USA) was recently approved for treatment of bleeding episodes in adults with acquired haemophilia A (AHA) in the United States. To date, no clinical experience outside the registration study has been reported. AIM: To describe early clinical experience using rpFVIII for AHA. METHODS: A retrospective chart review of seven patients with AHA treated with rpFVIII at four institutions from November 2014 to October 2015...
August 10, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
Grainne Colgan, Joseph F Baker, N Donlon, N Hogan, T McCarthy
BACKGROUND: Undergoing a major surgical intervention such as total hip arthroplasty (THA) with an underlying clotting disorder like haemophilia poses its own unique challenges. Despite the advances in factor replacement and medical management, the potential for excessive and uncontrolled haemorrhage still exists. The aim of this study was to quantify blood loss, peri-operative transfusion requirements and risk of haematoma formation in a cohort of patients with haemophilia undergoing THA...
December 2016: Journal of Orthopaedics
James E Frampton
Efmoroctocog alfa (Elocta(®), Eloctate(®), Eloctate™), a first-in-class recombinant factor VIII-Fc fusion protein (rFVIIIFc), has an extended half-life compared with conventional factor VIII (FVIII) preparations, including recombinant FVIII (rFVIII) products. It is approved for the treatment and prophylaxis of bleeding in patients with haemophilia A in multiple countries worldwide. Data accumulated from pivotal phase III studies (A-LONG in adults and adolescents aged ≥12 years; Kids A-LONG in children aged <12 years) and their ongoing extension study (ASPIRE) have demonstrated the long-term effectiveness of efmoroctocog alfa for the treatment of acute bleeding episodes, perioperative management and routine prophylaxis in previously treated males with severe haemophilia A...
September 2016: Drugs
Veronika Waldboth, Christine Patch, Romy Mahrer-Imhof, Alison Metcalfe
INTRODUCTION: The transition into adulthood is a developmental stage within the life cycle. A chronic childhood condition can disrupt this transition and create major challenges for both the young person and his or her family. Little is known about families' experiences when living with a rare genetic disease. Therefore, the purpose of this literature review was to understand experiences of families living with a chronic childhood disease during transition into adulthood by integrating evidence...
October 2016: International Journal of Nursing Studies
D M Adcock, J Mammen, S C Nair, S A de Lima Montalvão
Selected quality issues pertinent to the determination of accurate results in the haemostasis laboratory are discussed. Specifically, the implementation of a successful external quality-assessment scheme is described, including its impact on result accuracy as well as the programme's unique challenges and opportunities. Errors in the preanalytical phase of laboratory testing represent the greatest source for reporting incorrect test results. Some of the most common preanalytical errors are described including those that necessitate sample rejection...
July 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
M L Witkop, K Peerlinck, B A Luxon
Clinical care of patients with haemophilia (PWH) has progressed rapidly over the past decade. Current therapy has allowed patients with haemophilia to live longer and many patients are now experiencing the co-morbidities of the general population. In this review article, we focus on three common diseases states that affect PWH: chronic pain, obesity and hepatitis C. Pain has been a co-morbidity for many years and PWH often have unusual needs for chronic pain relief compared to the general population. Obesity is not only increasing in the general population but also in patients with hereditary bleeding disorders...
July 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
G Batsuli, S L Meeks, R W Herzog, S Lacroix-Desmazes
INTRODUCTION: Haemophilia A is an X-linked bleeding disorder characterized by a deficiency of coagulation protein factor VIII (FVIII). A challenging complication of therapeutic FVIII infusions is the formation of neutralizing alloantibodies against the FVIII protein defined as inhibitors. The development of FVIII inhibitors drastically alters the quality of life of the patients and is associated with tremendous increases in morbidity as well as treatment costs. AIM: Current clinical immune tolerance induction protocols to reverse inhibitors are lengthy, costly and not effective in all patients...
July 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
E Berntorp
INTRODUCTION: Looking into the future is difficult and sometimes hazardous. A reliable look into haemophilia treatment in 2030 should be based on history and contemporary progress as well as dilemmas. Today, the issue of inhibitors overshadows the entire haemophilia community together with lack of treatment for large parts of the world's persons with haemophilia. AIMS: The aim of this paper was to provide a perspective on haemophilia treatment in 2030 and its provenance...
July 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
M R Cahill, B T Colvin
Haematologists are long standing proponents of evidence based practice-well exemplified among professionals who care for patients with haemophilia. The rapidly expanding range of therapeutic products and the numerous accompanying clinical trials are swiftly interpreted and translated into clinical practice. This translation is formalised by frequently updated quidelines issued by the United Kingdom Haemophilia Centre Directors' Organisation (UKHCDO) and relevant to all doctors involved in the care of patients with haemophilia...
1997: Hematology (Amsterdam, Netherlands)
C H T Yeung, N Santesso, D Zeraatkar, A Wang, M Pai, M Sholzberg, H J Schünemann, A Iorio
BACKGROUND: Integrated care models have been adopted for individuals with chronic conditions and for persons with rare diseases, such as haemophilia. OBJECTIVE: To summarize the evidence from reviews for the effects of integrated multidisciplinary care for chronic conditions in adults and to provide an example of using this evidence to make recommendations for haemophilia care. SEARCH METHODS: We searched MEDLINE, EMBASE, CINAHL and Cochrane Database of Systematic Reviews up to January 2016, and reviewed reference lists of retrieved papers...
July 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
C H T Yeung, N Santesso, M Pai, C Kessler, N S Key, M Makris, T Navarro-Ruan, J M Soucie, H J Schünemann, A Iorio
BACKGROUND: Haemophilia care is commonly provided via multidisciplinary specialized management. To date, there has been no systematic assessment of the impact of haemophilia care delivery models on patient-important outcomes. OBJECTIVE: To conduct a systematic review of published studies assessing the effects of the integrated care model for persons with haemophilia (PWH). SEARCH METHODS: We searched MEDLINE, EMBASE and CINAHL up to April 22, 2015, contacted experts in the field, and reviewed reference lists...
July 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
M Pai, N Santesso, C H T Yeung, S J Lane, H J Schünemann, A Iorio
BACKGROUND: Rigorous and transparent methods are necessary to develop clinically relevant and evidence-based practice guidelines. We describe the development of the National Hemophilia Foundation-McMaster Guideline on Care Models for Haemophilia Management, which addresses best practices in haemophilia care delivery. METHODS: We assembled a Panel of persons with haemophilia (PWH), parents of PWH, clinical experts and guideline methodologists. Conflicts of interest were disclosed and managed throughout...
July 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
M Pai, N S Key, M Skinner, R Curtis, M Feinstein, C Kessler, S J Lane, M Makris, E Riker, N Santesso, J M Soucie, C H T Yeung, A Iorio, H J Schünemann
This guideline was developed to identify evidence-based best practices in haemophilia care delivery, and discuss the range of care providers and services that are most important to optimize outcomes for persons with haemophilia (PWH) across the United States. The guideline was developed following specific methods described in detail in this supplement and based on the GRADE (Grading of Recommendations, Assessment, Development and Evaluation approach). Direct evidence from published literature and the haemophilia community, as well as indirect evidence from other chronic diseases, were reviewed, synthesized and applied to create evidence-based recommendations...
July 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
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