Guidelines for the management of
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- Bu səhifədəki naviqasiya:
- Dr. Alok Srivastava (Chair)
- Dr. Nigel S. Key
- Dr. Christopher A. Ludlam
- Kathy Mulder
- Dr. Alison Street
- Summary and introduction
- 2. Special management issues
- 3. Laboratory diagnosis
- 5. Treatment of specific hemorrhages
- 6. Complications of hemophilia
- 7. Plasma factor level and duration of administration
- Acute bleeds should be treated as quickly as
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GUIDELINES FOR THE MANAGEMENT OF HEMOPHILIA 2 nd edition These guidelines were originally published by Blackwell Publishing in Haemophilia; Epub 6 JUL 2012. DOI: 10.1111/j.1365-2516.2012.02909.x. They are reprinted with their permission. © Blackwell Publishing Ltd., 2012 The WFH encourages redistribution of its publications for educational purposes by not-for-profit hemophilia organizations. For permission to reproduce or translate this document, please contact the Communications Department at the address below. This publication is accessible from the World Federation of Hemophilia’s website at www.wfh.org. Additional copies are also available from the WFH at: World Federation of Hemophilia 1425, boul. René-Lévesque O., bureau 1010 Montréal, Québec H3G 1T7 Canada Tel.: (514) 875-7944 Fax: (514) 875-8916 E-mail: wfh@wfh.org www.wfh.org GUIDELINES FOR THE MANAGEMENT OF HEMOPHILIA 2 nd
Prepared by the Treatment Guidelines Working Group, on behalf of the World Federation of Hemophilia (WFH) Dr. Alok Srivastava (Chair) Department of Hematology, Christian Medical College, Vellore, Tamil Nadu, India
Department of Oral Surgery, The Royal Infirmary, Glasgow, Scotland
Van Creveldkliniek and Department of Hematology, University Medical Center Utrecht, Utrecht, the Netherlands Dr. Nigel S. Key Department of Medicine, University of North Carolina, Chapel Hill, NC, U.S.A.
Sheffield Haemophilia and Thrombosis Centre, Royal Hallamshire Hospital, Sheffield, UK
Department of Orthopaedics and Traumatology, Fundación Santa Fe University Hospital Fundación Cosme y Damián and Universidad de los Andes and Universidad del Rosario, Bogotá, Colombia
Comprehensive Care Haemophilia and Thrombosis Centre, Royal Infirmary, Edinburgh, U.K. Dr. Johnny N. Mahlangu Haemophiia Comprehensive Care Centre, Johannesburg Hospital and Department of Molecular Medicine and Haematology, Faculty of Health Sciences, National Health Laboratory Services and University of the Witwatersrand, Johannesburg, South Africa
Bleeding Disorders Clinic, Health Sciences Center Winnipeg, Canada
Departments of Medicine, Pediatrics and Oncology, and Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, University of Calgary, Foothills Hospital and Calgary Health Region, Alberta, Canada Dr. Alison Street Department of Haematology, Alfred Hospital Melbourne, Australia
Acknowledgements A professional agency was engaged to assist with the literature search and to grade the evidence. In addition, given the fact that many recommendations are based on expert opinion, a draft version of these guidelines was circulated to many others involved in hemophilia care outside of the writing group. The authors are grateful to those who provided detailed comments. Finally, we would like to acknowledge the extraordinary effort from WFH staff, Jennifer Laliberté, and also Elizabeth Myles, in completing this work. Disclaimer The World Federation of Hemophilia (WFH) does not endorse particular treatment products or manu- facturers; any reference to a product name is not an endorsement by the WFH. The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. Dose schedules and other treatment regimens are continually revised and new side-effects recognized. These guidelines are intended to help develop basic standards of care for the management of hemophilia and do not replace the advice of a medical advisor and/or product insert information. Any treatment must be designed according to the needs of the individual and the resources available. Summary and introduction ................................. 6
................................................. 7 1.1 What is hemophilia? ............................... 7 Bleeding manifestations ........................... 7 1.2 Principles of care ..................................... 8 1.3 Comprehensive care ............................... 9 Comprehensive care team ........................ 9 Functions of a comprehensive care program ................................................. 10 1.4 Fitness and physical activity ................. 11 1.5 Adjunctive management ...................... 12 1.6 Prophylactic factor replacement therapy .. 12 Administration and dosing schedules ..... 13 1.7 Home therapy ...................................... 13 1.8 Monitoring health status and outcome 14 1.9 Pain management ................................ 15 Pain caused by venous access ................. 15 Pain caused by joint or muscle bleeding .. 15 Post-operative pain ................................ 15 Pain due to chronic hemophilic arthropathy ............................................ 15 1.10 Surgery and invasive procedures .......... 16 1.11 Dental care and management .............. 17 References ..................................................... 18
.......................... 21 2.1 Carriers ................................................. 21 2.2 Genetic testing/counselling and prenatal diagnosis ................................ 22 2.3 Delivery of infants with known or suspected hemophilia .......................... 22 2.4 Vaccinations .......................................... 23 2.5 Psychosocial issues ............................... 23 2.6 Sexuality ............................................... 23 2.7 Ageing hemophilia patients ................. 24 Osteoporosis ........................................... 24 Obesity ................................................... 24 Hypertension ......................................... 24 Diabetes mellitus (DM) .......................... 24 Hypercholesterolemia ............................. 25 Cardiovascular disease ......................... 25 Psychosocial impact .............................. 25 2.8 Von Willebrand disease and rare bleeding disorders ................................. 25 References ...................................................... 26
................................... 29 3.1 Knowledge and expertise in coagulation laboratory testing ............. 29 Principles of diagnosis ........................... 29 Technical aspects ................................... 29 Trained personnel .................................. 32 3.2 Use of the correct equipment and reagents ................................................. 32 Equipment ............................................ 32 Reagents ................................................ 33 3.3 Quality assurance ................................. 34 Internal quality control (IQC) ............... 34 External quality assessment (EQA) ....... 34 References ...................................................... 34 CONTENTS 4. Hemostatic agents ........................................ 37 4.1 Clotting factor concentrates ................. 37 Product selection .................................... 37 FVIII concentrates ................................. 38 FIX concentrates ................................... 39 4.2 Other plasma products ......................... 40 Fresh frozen plasma (FFP) ..................... 40 Cryoprecipitate ...................................... 41 4.3 Other pharmacological options ........... 41 Desmopressin (DDAVP) ........................ 41 Tranexamic acid .................................... 42 Epsilon aminocaproic acid ..................... 43 References ...................................................... 43
............. 47 5.1 Joint hemorrhage (hemarthrosis) ......... 47 Arthrocentesis ........................................ 48 5.2 Muscle hemorrhage ............................. 49 Iliopsoas hemorrhage ............................ 50 5.3 Central nervous system hemorrhage/head trauma .................... 50 5.4 Throat and neck hemorrhage ............... 51 5.5 Acute gastrointestinal (GI) hemorrhage ........................................... 51 5.6 Acute abdominal hemorrhage ............. 51 5.7 Ophthalmic hemorrhage ...................... 51 5.8 Renal hemorrhage ................................. 52 5.9 Oral hemorrhage ................................... 52 5.10 Epistaxis ............................................... 52 5.11 Soft tissue hemorrhage ......................... 53 5.12 Lacerations and abrasions ..................... 53 References ...................................................... 53
..................... 55 6.1 Musculoskeletal complications ............. 55 Synovitis ................................................ 55 Chronic hemophilic arthropathy ............ 56 Principles of physiotherapy/physical medicine in hemophilia .......................... 57 Pseudotumours ...................................... 58 Fractures ................................................ 58 Principles of orthopedic surgery in hemophilia ............................................. 58 6.2 Inhibitors ............................................... 59 Management of bleeding ....................... 60 Allergic reactions in patients with hemophilia B .......................................... 61 Immune tolerance induction ................. 61 Patients switching to new concentrates .. 61 6.3 Transfusion-transmitted and other infection-related complications ........... 61 Principles of management of HIV infection in hemophilia .......................... 62 Principles of management of HCV infection in hemophilia .......................... 62 Principles of management of HBV infection in hemophilia .......................... 62 Principles of management of bacterial infection in hemophilia .......................... 63 References ...................................................... 63
administration .............................................. 69 7.1 Choice of factor replacement therapy protocols ............................................... 69 References ...................................................... 73
Oxford Centre for Evidence-Based Medicine, 2011 Levels of Evidence ....... 74
TABLES AND FIGURES Table 1-1: Relationship of bleeding severity to clotting factor level .......................................................... 8 Table 1-2: Sites of bleeding in hemophilia ................................................................................................. 8 Table 1-3: Approximate frequency of bleeding at different sites ............................................................... 8 Table 1-4: Definitions of factor replacement therapy protocols ............................................................... 12 Table 1-5: Strategies for pain management in patients with hemophilia ................................................. 15 Table 1-6: Definition of adequacy of hemostasis for surgical procedures ............................................... 16 Table 3-1: Interpretation of screening tests .............................................................................................. 31 Table 5-1: Definition of response to treatment of acute hemarthrosis ................................................... 48 Table 7-1: Suggested plasma factor peak level and duration of administration (when there is no significant resource constraint) .................................................................. 71 Table 7-2: Plasma factor peak level and duration of administration (when there is significant resource constraint) ....................................................................... 72 Figure 7-1: Strategies for clotting factor replacement at different ages and impact on outcomes ............ 69
GUIDELINES FOR THE MANAGEMENT OF HEMOPHILIA 6 The first edition of these guidelines, published in 2005 by the WFH, served its purpose of being a useful document for those looking for basic infor- mation on the comprehensive management of hemophilia. The need for revision has arisen for several reasons. The most significant of these was to incorporate the best existing evidence on which recommendations were based. There is recent high quality data from randomized controlled trials estab- lishing the efficacy and superiority of prophylactic factor replacement over episodic treatment – though the optimal dose and schedule for prophylaxis continue to be subjects of further research. There is also greater recognition of the need for better assessment of outcomes of hemophilia care using newly developed, validated, disease-specific clini- metric instruments. This revised version addresses these issues in addition to updating all sections. These guidelines contain several recommenda- tions regarding the clinical management of people with hemophilia (practice statements, in bold). All such statements are supported by the best available evidence in the literature, which were graded as per the 2011 Oxford Centre for Evidence-Based Medicine (see Appendix I). Where possible, references for recommendations that fell outside the selection for practice statements were also included. These refer- ences have not been graded. A question often raised when developing a guide- line document such as this is its universal applicability given the diversity of health services and economic systems around the world. Our strongly held view is that the principles of management of hemophilia are the same all over the world. The differences are mainly in the doses of clotting factor concentrates (CFC) used to treat or prevent bleeding, given that the costs of replacement products comprise the major expense of hemophilia care programs. Recognizing this reality, these guidelines continue to include a dual set of dose recommendations for CFC replacement therapy. These are based on published literature and practices in major centres around the world. It should be appreciated, however, that the lower doses recom- mended may not achieve the best results possible and should serve as the starting point for care to be initi- ated in resource-limited situations, with the aim of gradually moving towards more optimal doses, based on data and greater availability of CFC. One of the reasons for the wide acceptance of the first edition of these guidelines was its easy reading format. While enhancing the content and scope of the document, we have ensured that the format has remained the same. We hope that it will continue to be useful to those initiating and maintaining hemophilia care programs. Furthermore, the extensive review of the literature and the wide consensus on which practice statements have been made may encourage practice harmonization around the world. More importantly, in areas where practice recommendations lack adequate evidence, we hope that this document will stimulate appropriate studies. Introduction Summary
Hemophilia is a rare disorder that is complex to diagnose and to manage. These evidence-based guidelines offer practical recommendations on the diagnosis and general management of hemo- philia, as well as the management of complications including musculo skeletal issues, inhibitors, and transfusion-transmitted infections. By compiling these guidelines, the World Federation of Hemophilia (WFH) aims to assist healthcare providers seeking to initiate and/or maintain hemophilia care programs, encourage practice harmonization around the world and, where recommendations lack adequate evidence, stimulate appropriate studies.
7 1.1 What is hemophilia? 1. Hemophilia is an X-linked congenital bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII) (in hemophilia A) or factor IX (FIX) (in hemophilia B). The deficiency is the result of mutations of the respective clotting factor genes. 2. Hemophilia has an estimated frequency of approximately one in 10,000 births. 3. Estimations based on the WFH’s annual global surveys indicate that the number of people with hemophilia in the world is approximately 400,000 [1]. 4. Hemophilia A is more common than hemophilia B, representing 80-85% of the total hemophilia population. 5. Hemophilia generally affects males on the maternal side. However, both F8 and F9 genes are prone to new mutations, and as many as 1/3 of all cases are the result of spontaneous muta- tion where there is no prior family history. 6. Accurate diagnosis of hemophilia is essential to inform appropriate management. Hemophilia should be suspected in patients presenting with a history of: ■ easy bruising in early childhood ■ “spontaneous” bleeding (bleeding for no apparent/known reason), particularly into the joints, muscles, and soft tissues ■ excessive bleeding following trauma or surgery 7. A family history of bleeding is obtained in about two-thirds of all patients. 8. A definitive diagnosis depends on factor assay to demonstrate deficiency of FVIII or FIX. Bleeding manifestations 1. The characteristic phenotype in hemophilia is the bleeding tendency. 2. While the history of bleeding is usually life-long, some children with severe hemophilia may not have bleeding symptoms until later when they begin walking or running. 3. Patients with mild hemophilia may not bleed excessively until they experience trauma or surgery.
4. The severity of bleeding in hemophilia is gener- ally correlated with the clotting factor level, as shown in Table 1-1. 5. Most bleeding occurs internally, into the joints or muscles (see Table 1-2 and Table 1-3). 6. Some bleeds can be life-threatening and require immediate treatment (see Section 5). 1 GENERAL CARE AND MANAGEMENT OF HEMOPHILIA GUIDELINES FOR THE MANAGEMENT OF HEMOPHILIA 8 1.2 Principles of care 1. The primary aim of care is to prevent and treat bleeding with the deficient clotting factor. 2. Whenever possible, specific factor deficiency should be treated with specific factor concentrate. 3. People with hemophilia are best managed in a comprehensive care setting (see ‘Comprehensive care’, on page 9). 4. Acute bleeds should be treated as quickly as Yüklə 0,65 Mb. Dostları ilə paylaş:
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