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Guidelines Mng Hemophilia

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Published by the World Federation of Hemophilia World Federation of Hemophilia, 2005 The WFH encourages redistribution of its publications for educational purposes by not-for-prot 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 Hemophilias Web site at Additional copies are also available from the WFH at: World Federation of Hemophilia 1425 Ren Lvesque Boulevard West, Suite 1010 Montral, Qubec H3G 1T7 CANADA Tel.: (514) 875-7944 Fax: (514) 875-8916 E-mail: [email protected] Internet: The World Federation of Hemophilia does not endorse particular treatment products or manufacturers; any reference to a product name is not an endorsement by the WFH. The World Federation of Hemophilia does not engage in the practice of medicine and under no circumstances recommends particular treatment for specic individuals. Dose schedules and other treatment regimes 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.


Introduction General Management of HemophiliaWhat is Hemophilia? Diagnosis of Hemophilia Bleeding Manifestations in HemophiliaSites of Bleeding Incidence of Different Sites of Bleeding Incidence of Bleeding into Different Joints

1 33 3 4 4 4 4 5 5 5 6 7 7 7 7 8 8 9 9 10 10 11 11 11 12 12 12 13 13 13 14 14 14 15 16 16

Chronic Complications of Hemophilia Carriers Comprehensive CareComprehensive Care Team Functions of Comprehensive Care Program The Family

Management of HemophiliaPrinciples of Care Management of Bleeding Adjunctive Management Home Therapy Prophylaxis Surgery Inhibitors Management of Bleeding Allergic Reactions in Hemophilia B Patients with Inhibitors Immune Tolerance Induction Patients Switching to New Concentrates

Genetic Counselling/Prenatal Diagnosis Delivery of Infants with Known or Suspected Hemophilia Vaccination Psychosocial IssuesDaily Living For the Person with Hemophilia For the Family For the Community

Dental Care Monitoring Outcome of Treatment Sports and Hemophilia

Guidelines for the Management of Hemophilia

Laboratory DiagnosisIntroduction Knowledge and Expertise in Coagulation Laboratory TestingPrinciples of Diagnosis Technical Aspects Preparation of the Patient Prior to Taking a Blood Sample Collecting the Sample End-Point Detection Screening Tests Correction (Mixing) Studies Factor Assay Trained Personnel

1717 17 17 17 17 17 18 19 19 19 20 20 20 21 21 22 22

Use of the Correct Equipment and ReagentsEquipment Reagents

Quality AssuranceExternal Quality Assessment Scheme (EQAS) Reference Materials and Standards

Musculoskeletal Complications of HemophiliaIntroduction Acute Hemarthrosis Aspiration Muscle Hematomas Chronic Hemarthrosis Chronic Synovitis Synovectomy Chronic Hemophilic Arthropathy Pseudotumours Fractures

2323 23 24 25 25 25 26 26 27 28

Selection of Clotting Factor Concentrates and Other DrugsClotting Factor Concentrates Choice of Products for Replacement Therapy Purity Viral Inactivation/Elimination Plasma-Derived/Recombinant Products

2929 30 30 31 31

Guidelines for the Management of Hemophilia

Cryoprecipitate Fresh Frozen Plasma and Cryo-Poor Plasma Other Pharmacological Options Desmopressin (DDAVP) Tranexamic Acid Aminocaproic Acid

31 32 32 32 34 35

Treatment of Bleeding in HemophiliaBasic Principles of Treatment Treatment of Hemophilia A (FVIII Deciency)FVIII Concentrates Cryoprecipitate/Fresh Frozen Plasma Desmopressin (DDAVP)

3737 37 37 38 38 38 38 39 40 40 40 40 40 41 41 41 41 42 42 42 42 43 43 43 43 44 44 44

Treatment of Hemophilia B (FIX Deciency)FIX Concentrates Fresh Frozen Plasma Antibrinolytic Agents

Treatment of Specic HemorrhagesJoint Hemorrhage Muscle Hemorrhage Iliopsoas Hemorrhage Central Nervous System Hemorrhage/Head Trauma Throat and Neck Hemorrhage Acute Gastrointestinal Hemorrhage Acute Hemorrhage in the Abdomen Ophthalmic Trauma or Hemorrhage Renal Hemorrhage Oral Hemorrhage Epistaxis Soft Tissue Hemorrhage Lacerations and Abrasions

Other Management IssuesDental Care Surgery Minor Invasive Procedures Allergic Reactions to Factor Replacement Products

Tables: Plasma Factor Level and Duration of Administration Bibliography

45 47

Guidelines for the Management of Hemophilia

INTRODUCTIONAlthough effective therapy has been available for hemophilia for at least 30 years, many issues remain unresolved regarding the management of this condition, particularly with reference to doses and duration of factor replacement therapy for different types of bleeding, immune tolerance induction, and surgical prophylaxis. Many countries that are starting to establish care for hemophilia do not have standard protocols to ensure the proper management of hemophilia. As the World Federation of Hemophilia (WFH) has expanded its various programs to improve care for people with hemophilia worldwide, more and more requests have been made for standard guidelines that are appropriate for countries where economic resources are limited, to ensure a basic level of care. As there were no guidelines for the management of hemophilia with a universal outlook, the WFH established a working group in 2003 to develop such guidelines. This group, consisting of Paul Giangrande, Man Chiu Poon, Mary Chua, Angus McCraw, Jerome Wiedel, and Alok Srivastava (Chair), has worked to put together this document with the exemplary editorial help of Elizabeth Myles at WFH headquarters. In the absence of good evidence for current protocols in practice in the management of hemophilia, the information included here is mainly adapted from published consensus guidelines from different centres or countries, including Hemophilia of Georgia (USA), the Association of Hemophilia Clinic Directors of Canada, the National Hemophilia Foundation (USA), Italian Association of Hemophilia Centres, Hemophilia Federation, India, and the South African Hemophilia Foundation. Directors of the International Hemophilia Training Centres of the WFH as well as all the centres involved in the WFH Twinning Program were consulted. The current version of these guidelines incorporates the suggestions from many of these physicians, for which we are extremely grateful and welcome further comments. As and if new evidence is generated on different aspects of the management of hemophilia, these guidelines will be updated and revised as necessary. The purpose of these guidelines is to provide protocols for treatment to centres that are beginning to establish care for people with hemophilia. They may also help standardize care at the more established ones. The WFH hopes that as well as helping those less familiar with the management of this condition, this publication will be a small step towards harmonizing care of people with hemophilia in the world until evidence-based practice is possible. Alok Srivastava, MD WFH Treatment Guidelines Working Group

Guidelines for the Management of Hemophilia



GENERAL MANAGEMENT OF HEMOPHILIAWhat is Hemophilia? Hemophilia is an X-linked congenital bleeding disorder with a frequency of about one in 10,000 births. Hemophilia is caused by a deciency of coagulation factor VIII (FVIII) (hemophilia A) or factor IX (FIX) (hemophilia B) related to mutations of the clotting factor gene. The number of affected persons worldwide is estimated to be about 400,000. Hemophilia A is more common than hemophilia B, representing 80-85% of the total. The life expectancy of persons born with hemophilia, who have access to adequate treatment, should approach normal with currently available treatment.

Diagnosis of HemophiliaAccurate diagnosis is important and essential for effective management. Hemophilia should be suspected in patients presenting with a history of: Easy bruising in early childhood; Spontaneous bleeding (particularly into the joints and soft tissue); and Excessive bleeding following trauma or surgery. While the history of bleeding is usually lifelong, some severe hemophilic children may not have bleeding symptoms until after the age of one or later when they begin walking and exploring their world. Patients with mild hemophilia may not have excessive bleeding unless they experience trauma or surgery. A family history of bleeding is commonly obtained. Hemophilia generally affects males on the maternal side. However, both FVIII and FIX genes are prone to new mutations, and as many as 1/3 of all patients may not have a family history of these disorders. Screening tests will show a prolonged activated partial thromboplastin time (aPTT) in severe and moderate cases but may not show prolongation in mild hemophilia. A denitive diagnosis depends on factor assay to demonstrate deciency of FVIII or FIX. The severity of bleeding manifestations in hemophilia is generally correlated with the clotting factor level as shown in the following table.

Guidelines for the Management of Hemophilia


SeveritySevere Moderate Mild

Clotting factor level % activity (IU/ml)1% (< 0.01) 1%-5% (0.01-0.05) 5%-40% (0.05-0.40)

Bleeding episodesSpontaneous bleeding, predominantly in joints and muscles Occasional spontaneous bleeding. Severe bleeding with trauma, surgery Severe bleeding with major trauma or surgery

Bleeding Manifestations in HemophiliaS

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