The Journal of Haemophilia Practice

The J. of Haemoph Pract ONL

<abstract><title style='display:none'>Abstract</title> In recent decades, few disorders have benefited more from scientific research than haemophilia. Those living with the condition have witnessed improved standards of care resulting in dramatic improvements in quality of life. The Katharine Dormandy Haemophilia Centre and Thrombosis Unit has made a proud contribution to this improvement in care.</abstract>

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<article-title>Celebrating 50 years of haemophilia care at the Royal Free Hospital</article-title>

Celebrating 50 years of haemophilia care at the Royal Free Hospital

{"article-title":"Celebrating 50 years of haemophilia care at the Royal Free Hospital"}

<abstract><title style='display:none'>Abstract</title> Use of recombinant clotting factor now represents the standard of care in haemophilia across the developed world. But the recombinant era would not have occurred without the isolation from plasma of factor VIII in sufficient quantities to allow characterisation and cloning. Much of this development work occurred at the Royal Free Hospital.</abstract>

<article-title>The road to cloning of factor VIII and the recombinant era</article-title>

The road to cloning of factor VIII and the recombinant era

{"article-title":"The road to cloning of factor VIII and the recombinant era"}

<abstract><title style='display:none'>Abstract</title> The HIV and hepatitis C epidemics of the 1980s represent the darkest days in the history of modern haemophilia care. The haemophilia centre at the Royal Free Hospital was at the forefront of research into the natural history of both diseases. This work led directly to the widespread use of recombinant products, as well as the establishment of combined haemophilia clinics with hepatologists and HIV physicians.</abstract>

<article-title>Blood borne infections and haemophilia: the worst of times</article-title>

Blood borne infections and haemophilia: the worst of times

{"article-title":"Blood borne infections and haemophilia: the worst of times"}

<abstract><title style='display:none'>Abstract</title> Advances in the understanding of haemophilia require effective collaboration, both at the national and international levels. Such collaborations are currently playing a major role in elucidating the natural history of acquired haemophilia, and also in clarifying the issue of product-related inhibitor development in previously untreated patients with haemophilia.</abstract>

<article-title>Diagnosis and management of inhibitors: the need for professional collaboration</article-title>

Diagnosis and management of inhibitors: the need for professional collaboration

{"article-title":"Diagnosis and management of inhibitors: the need for professional collaboration"}

<abstract><title style='display:none'>Abstract</title> The development of registries through international collaboration has facilitated better understanding of the rare bleeding disorders. Such work has shown that rare bleeding disorders are heterogeneous and need to be studied singularly, and that heterozygous patients may bleed. There is a need to understand the minimum plasma coagulant activity level to prevent spontaneous bleeding. Moreover, due to the low prevalence of rare bleeding disorders, the management of this patient population remains a challenge. Data collection on clinical history, efficacy and side effects of treatment needs to be harmonised.</abstract>

<article-title>International databases open the door to improved care for rare bleeding disorders</article-title>

International databases open the door to improved care for rare bleeding disorders

<abstract><title style='display:none'>Abstract</title> The Katharine Dormandy haemophilia centre has played an enormous role over many years in providing outreach programmes to centres in developing countries. Doing so offers the potential to improve patient care by enhancing understanding of the need for accurate diagnosis and management.</abstract>

<article-title>Supporting outreach programmes for haemophilia diagnostics</article-title>

Supporting outreach programmes for haemophilia diagnostics

{"article-title":"Supporting outreach programmes for haemophilia diagnostics"}

<abstract><title style='display:none'>Abstract</title> Modern haemophilia care based on good diagnosis and effective prophylaxis has allowed boys born with haemophilia to grow up leading essentially normal lives. Nevertheless, there remain challenges notably those posed by inhibitors and patient expectations. There is now a significant cohort of men in their 30s, 40s and 50s who have been looked after extremely well but many of whom now have significant ankle arthropathy because they played football, even when advised not to do so at a time when prophylaxis was limited or started late. The imminent era of longer-acting clotting factors and gene therapy will also impact on future patient expectations. Meeting and managing these challenges will be much enhanced by the development and maintenance of good relationships between the patient and the haemophilia team.</abstract>

<article-title>The modern challenges of haemophilia care in childhood and adolescence</article-title>

The modern challenges of haemophilia care in childhood and adolescence

{"article-title":"The modern challenges of haemophilia care in childhood and adolescence"}

<abstract><title style='display:none'>Abstract</title> The multidisciplinary team approach to providing comprehensive care is the cornerstone of the management of musculoskeletal issues in haemophilia. At the Royal Free the team takes a holistic approach to musculoskeletal disease management that is not just about assessment of a painful knee or having an operation. It recognises that patients sit on a spectrum of musculoskeletal disease severity that is dependent on age and the type of treatments available and the issues that may have been experienced with those treatments. The patient’s own goals sit at the heart of the team’s approach.</abstract>

<article-title>Musculoskeletal care in haemophilia: the importance of the joint team approach</article-title>

Musculoskeletal care in haemophilia: the importance of the joint team approach

{"article-title":"Musculoskeletal care in haemophilia: the importance of the joint team approach"}

<abstract><title style='display:none'>Abstract</title> The issue of women with bleeding disorders was first reported by Professor von Willebrand in 1926, but it is only from around the mid 1990s that the issue has been fully recognised. Much of this is due to the vision and hard work of Professor Christine Lee and colleagues at the Katharine Dormandy Haemophilia Centre. This work has led to better diagnosis, better quality care and improved quality of life for women with bleeding disorders.</abstract>

<article-title>Gynaecological and obstetric care for women with inherited bleeding disorders</article-title>

Gynaecological and obstetric care for women with inherited bleeding disorders

{"article-title":"Gynaecological and obstetric care for women with inherited bleeding disorders"}

<abstract><title style='display:none'>Abstract</title> Staff at the Katharine Dormandy Haemophilia Centre pioneered a systemic family therapy model for haemophilia, in which reviews combined medical care and family counselling. That approach has now been extended to specialised joint clinics such as in orthopaedics, women’s and genetic counselling. This multidisciplinary team approach enables specialist clinicians to focus on what they do best while the family therapy team manages the psychological, practical and family issues, and supports patients to make difficult decisions regarding their care.</abstract>

<article-title>An approach that puts the family at the centre of haemophilia care</article-title>

An approach that puts the family at the centre of haemophilia care

{"article-title":"An approach that puts the family at the centre of haemophilia care"}

<abstract><title style='display:none'>Abstract</title> Patients with haemophilia have benefited hugely from the advances in basic science achieved over the past 50 years. The introduction of clotting factor concentrates opened the door to home treatment and the subsequent availability of plasma and then recombinant products heralded the prophylaxis era. As clinicians and patients begin to manage the introduction of longer-acting factor concentrates, there are also many novel therapies and approaches in development, many of which offer the potential to transform the lives for individuals with inherited bleeding disorders and their families.</abstract>

<article-title>Into the future with novel emerging therapies for haemophilia</article-title>

Into the future with novel emerging therapies for haemophilia

{"article-title":"Into the future with novel emerging therapies for haemophilia"}

<abstract><title style='display:none'>Abstract</title> AAV-mediated gene therapy has the potential to be paradigm shifting as a treatment for haemophilia. A single administration of AAV vector can result in safe and consistent long-term expression of transgene (&gt;5 years), reduction in spontaneous bleeding episodes, reduction in clotting factor usage and an improvement in quality of life. There is huge commercial interest in this approach, with the expectation that an AAV gene therapy product for haemophilia B will be licensed by 2020.</abstract>

<article-title>Gene therapy for haemophilia: a very modern success story</article-title>

Gene therapy for haemophilia: a very modern success story

{"article-title":"Gene therapy for haemophilia: a very modern success story"}

<abstract><title style='display:none'>Abstract</title> The need to prepare children and young people with haemophilia for long-term care in the adult sector has long been accepted and is reflected in many national health care policies. Ideally, transition should be an individualised age- and development-appropriate process, through which each young person is empowered to self-manage, with support from their families and multidisciplinary team (MDT). It is widely recognised that young people who are not engaged in self-management frequently become non-adherent to therapy. For those with haemophilia, this can have a life-long impact on joint health and quality of life. In practice, anecdotal reports from individuals suggest that the transition process is often poorly planned, leaving them feeling lost and unsupported by the healthcare professionals who, until this point, have been core members of their support network. Rarely are the views of young people or their families sought regarding the outcome of their transition process. For those with less severe disease or rarer bleeding disorders, and for girls at the menarche, transition is often not addressed at all. This literature review forms the first part of a project that seeks to identify the outcome measures that can be used to drive a nurse-led initiative improvement in transition for young people with bleeding disorders, through changes in both working practices among health professionals and in the underpinning system, which enables them to put such changes into practice.</abstract>

<article-title>Literature Review: Transition From Paediatric to Adult Services in Haemophilia</article-title>

Literature Review: Transition From Paediatric to Adult Services in Haemophilia

{"article-title":"Literature Review: Transition From Paediatric to Adult Services in Haemophilia"}

The Journal of Haemophilia Practice is covered by the following services: <UL> <LI>Baidu Scholar</LI> <LI>Cabell's Whitelist </LI> <LI>CNKI Scholar (China National Knowledge Infrastructure)</LI> <LI>CNPIEC - cnpLINKer</LI> <LI>Dimensions</LI> <LI>EBSCO Discovery Service</LI> <LI>Google Scholar</LI> <LI>J-Gate</LI> <LI>JournalTOCs</LI> <LI>KESLI-NDSL (Korean National Discovery for Science Leaders)</LI> <LI>MyScienceWork</LI> <LI>Naver Academic</LI> <LI>Naviga (Softweco)</LI> <LI>Primo Central (ExLibris)</LI> <LI>QOAM (Quality Open Access Market)</LI> <LI>ReadCube</LI> <LI>Semantic Scholar</LI> <LI>Summon (ProQuest)</LI> <LI>TDNet</LI> <LI>WorldCat (OCLC)</LI></UL>

The Journal of Haemophilia Practice ist in den folgenden Services indiziert: <UL> <LI>Baidu Scholar</LI> <LI>Cabell's Whitelist </LI> <LI>CNKI Scholar (China National Knowledge Infrastructure)</LI> <LI>CNPIEC - cnpLINKer</LI> <LI>Dimensions</LI> <LI>EBSCO Discovery Service</LI> <LI>Google Scholar</LI> <LI>J-Gate</LI> <LI>JournalTOCs</LI> <LI>KESLI-NDSL (Korean National Discovery for Science Leaders)</LI> <LI>MyScienceWork</LI> <LI>Naver Academic</LI> <LI>Naviga (Softweco)</LI> <LI>Primo Central (ExLibris)</LI> <LI>QOAM (Quality Open Access Market)</LI> <LI>ReadCube</LI> <LI>Semantic Scholar</LI> <LI>Summon (ProQuest)</LI> <LI>TDNet</LI> <LI>WorldCat (OCLC)</LI></UL>

Editor-in-Chief Dr Kate Khair, Great Ormond Street Hospital, London, UK Managing Editor Mike Holland, Haemnet, UK <A href="mailto:mike@haemnet.com">mike@haemnet.com</A> Editorial Advisory Board Tami Barazani-Brutman, Sheba Medical Center at Sheba, Tel Hashomer Hospital, Israel Paul Batty, Queen Mary University of London, UK, and Queen’s University, Ontario, Canada Greig Blamey, Health Sciences Center, University of Alberta, Canada Erica Crilly, British Columbia Children's Hospital, Vancouver, Canada Janine Furmedge, The Royal Children’s Hospital Haemophilia Centre, Melbourne, Australia Rich Gorman, Brighton and Sussex Medical School, UK Chris Guelcher, Children’s National Hospital, Washington, USA Chris Harrington, Royal Free Hospital, London, UK Cathy Harrison, Sheffield Haemophilia &amp; Thrombosis Centre, Royal Hallamshire Hospital, Sheffield, UK Dan Hart, The Royal London Hospital and Barts Health NHS Trust, London, UK Adam Jones, University of Sunderland, UK Radek Kaczmarek, Indiana University School of Medicine, USA Sebastien Lobet, Cliniques Universitaires Saint-Luc, Brussels, Belgium Paul McLaughlin, Royal Free Hospital, London, UK Greta Mulders, Erasmus University Medical Center, Rotterdam, The Netherlands James Munn, University of Michigan Hemophilia and Coagulation Disorders Program, Michigan, USA Declan Noone, European Haemophilia Association, Brussels Jamie O'Hara, University of Chester, Cheshire, UK Debra Pollard, Royal Free Hospital, London, UK BJ Ramsay, Wellington Haemophilia Centre, Wellington, New Zealand Dakota J. Rosenfelt, PharmD, Missouri, US Sylvia von Mackensen, Institute of Medical Psychology at the University Medical Centre, Hamburg, Germany Linda Myrin Westesson, Sahlgrenska University Hospital, Gothenburg, Sweden Michelle Witkop, National Hemophilia Foundation, USA Technical Editor Kathryn Jenner, Haemnet, UK Publisher De Gruyter Poland Bogumiła Zuga 32A Str. 01-811 Warsaw, Poland T: +48 22 701 50 15

<DIV align=left> Manuscripts should be submitted via the journal’s Scholar One Manuscripts site: <A href="http://mc04.manuscriptcentral.com/jhaempract">mc04.manuscriptcentral.com/jhaempract</A>. You will need to log in. If you are using Scholar One for the first time, use the ‘Create an Account’ link. One you have logged in, select the ‘Author’ tab. You should then follow the on-screen instructions – the system will guide you through the process of manuscript submission. Alongside the manuscript, accompanying figures and tables, and general information about the manuscript, you will be asked to provide: <UL> <LI>The names of suggested reviewers (optional) </LI> <LI>A cover letter signed on behalf of all co-authors by the corresponding author </LI> <LI>An Open Access License agreement, completed by the corresponding author on behalf of all co-authors (available via the ‘Instructions and Forms’ tab on the journal’s Scholar One Manuscripts site: <A href="http://mc04.manuscriptcentral.com/jhaempract">mc04.manuscriptcentral.com/jhaempract</A>) </LI> <LI>Details of any conflicts of interest </LI> <LI>Confirmation that informed consent has been obtained where appropriate. </LI></UL> After submitting your manuscript, you will receive a confirmation email. You can check on the status of your manuscript at any time by logging in to your Scholar One account. The journal will notify you by email when a decision has been made on your manuscript. Manuscript types The Journal of Haemophilia Practice accepts <UL> <LI>Original research </LI> <LI>Case reports </LI> <LI>Case series </LI> <LI>Reviews in any field of bleeding disorders of relevance to current clinical practice </LI> <LI>Clinical Updates that describe current advances in any clinical field related to bleeding disorders </LI> <LI>Editorials that address a particular topic of current interest. </LI></UL> Please note that case reports should meet the standards outlined in the <A href="https://www.care-statement.org/">CARE Case Report Guidelines</A>. Authors submitting case reports are advised to consult the <A href="https://www.care-statement.org/checklist">CARE Checklist</A>. Manuscript format Wordcount Although we do not specify wordcounts for articles, we recommend the following: <UL> <LI>For shorter papers, e.g. case reports and small cohort studies: 1,000–3,000 words </LI> <LI>For reviews, larger studies and qualitative studies, which require explanation of methodologies: 3,000–7,000 words. </LI></UL> Title The title should be short and informative. For case reports, the words ‘case report’ should be included in the title. Authors Full author names and affiliation should be given. An ORCID iD for each author should be provided where available. Abstract For original articles, a short abstract should be provided using the following headings: Background or Introduction, Aims, Methods, Results, Conclusion. Keywords Submissions should include 3–6 keywords for indexing purposes. Reference style Journal titles should be abbreviated according to the style of Index Medicus and spelled out in full if not listed in Index Medicus. These can be looked up in the <A href="https://www.ncbi.nlm.nih.gov/nlmcatalog/journals">NLM Catalog</A>. The DOI (digital object identifier) associated with journal articles should be provided where available. Where there are six or more authors associated with a reference, the first three should be listed, followed by ‘et al.’ Datasets should be cited using the format outlined by the <A href="https://www.force11.org/datacitationprinciples">Joint Declaration of Data Citation Principles</A>: Authors; Year; Dataset title; Data repository/archive; Version (if relevant); Persistent identifier, e.g. DOI (if available). Reference style examples: <UL> <LI>Reference to an article: </LI></UL> Khair K, Klukowska A, Myrin Westesson L, et al. The burden of bleeds and other clinical determinants on caregivers of children with haemophilia (the BBC Study). Haemophilia 2019; 25(3): 416-423. doi: 10.1111/hae.13736. <UL> <LI>Reference to a book: </LI></UL> Jones P. Living with Haemophilia. 2002. Oxford: Oxford University Press. <UL> <LI>Reference to a chapter in a book: </LI></UL> Escobar MA, Roberts HR. Less common congenital disorders of hemostasis. In: Kitchens CS, ed. Consultative Hemostasis and Thrombosis, 3rd edn. 2013. Philadelphia, PA: W.B. Saunders Company. <UL> <LI>Reference to a webpage/online resource: </LI></UL> European Medicines Agency (EMA). European Public Assessment Report (EPAR) NovoSeven Summary for the Public. 2009. Available from http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/000074/human_med_000936.jsp&amp;mid=WC0b01ac058001d124 (accessed 9 September 2013). Figures and tables Please provide tables and figures at the end of the manuscript or as a separate file. Informed consent People who are reported in studies have a right to privacy that should not be violated without informed consent. Identifying information, including names, initials, dates of birth or hospital numbers, should not be published in written descriptions, photographs or pedigrees unless the information is essential for scientific purposes and the person (or parent or guardian) gives written informed consent for publication. Informed consent for this purpose requires that an identifiable person be shown the manuscript to be submitted for publication. Authors should disclose to these participants whether any potential identifiable material might be available via the internet or in print after publication. Each implicated individual’s consent should be written and archived with the authors. If the patient is a minor or lacks capacity to consent, the written informed consent of a parent or guardian must be given. If the person in the case study is deceased, written consent must be sought from a relative. Without consent, a case report may be considered for publication if the patient is sufficiently anonymised according to <A href="http://www.icmje.org/recommendations/">ICMJE guidelines</A>. Nonessential identifying details should always be omitted. Informed consent should be obtained if there is any doubt that anonymity can be maintained. For example, masking the eye region in photographs of patients is inadequate protection of anonymity. Consent must be mentioned in the manuscript. We reserve the right to request copies of consent documentation. The Editor will make the final determination of what constitutes personally identifiable information on a case-by-case basis. Open Access Statement The journal is an Open Access journal that allows a free unlimited access to all its contents without any restrictions upon publication to all users. <A href="https://sciendo-parsed-data-feed.s3.eu-central-1.amazonaws.com/JHP/Open_Access_License.pdf">Open Access License</A> </DIV>

The Journal of Haemophilia Practice is an international, open-access, peer-reviewed journal that accepts papers from all members of the multidisciplinary bleeding disorders care team. Any manuscript that is relevant to current haemophilia practice will be considered for publication by our editorial review team. To this end, we positively welcome: <UL> <UL> </UL> <LI>Original research </LI> <LI>Case reports </LI> <LI>Case Series </LI> <LI>Reviews in any field of bleeding disorders that are of relevance to current clinical practice </LI> <LI>Clinical Updates that describe current advances in any clinical field related to bleeding disorders </LI> <LI>Editorials that address a particular topic of current interest. </LI> <UL> </UL></UL> All manuscripts submitted to The Journal of Haemophilia Practice will first be subject to a technical review, including a quality check of all the files submitted, including tables, figures and references. This will include a plagiarism check (see below). Manuscripts will then be reviewed by the Editor-in-Chief, who will decide whether or not to proceed to peer-review by members of our editorial board and invited experts. The identity of the reviewers will not be disclosed to the authors. The review procedure will result in one of three decisions: <UL> <UL> </UL> <LI>Accept </LI> <LI>Accept subject to revision </LI> <LI>Reject </LI> <UL> </UL></UL> The editorial decision will be communicated to the authors as soon as the review process has been finalized. In case of revisions, the revised article will be sent to the reviewers who will decide on a new recommendation for revision, acceptance or rejection. The estimated time from the submission to first decision is approximately 4-6 weeks, and from the final revision to acceptance is approximately 2 weeks. Prior to publication, the corresponding author will receive a proof of their article in order to confirm the accuracy of the text or suggest modifications. “The Journal of Haemophilia Practice” is a double-blind peer reviewed journal. The journal has no article processing charges (APCs) nor article submission charges. Archiving Sciendo archives the contents of this journal in <A href="https://www.portico.org/">Portico</A> - digital long-term preservation service of scholarly books, journals and collections. Plagiarism Policy The editorial board is participating in a growing community of <A href="https://www.crossref.org/services/similarity-check/">Similarity Check System's</A> users in order to ensure that the content published is original and trustworthy. Similarity Check is a medium that allows for comprehensive manuscripts screening, aimed to eliminate plagiarism and provide a high standard and quality peer-review process.

Editions

Magazine et Edition

The Journal of Haemophilia Practice

Chercher

Volume 2 (2015): Edition 2 (July 2015)

Détails du journal et du numéro

Chercher

Articles

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