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Diagnosis and Management of Haemophilia

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Diagnosis and Management of Haemophilia CLINICAL REVIEW Diagnosis and management Follow the link from the online version of this article to obtain certi ed continuing medical education credits of haemophilia Karin Fijnvandraat,1 2 Marjon H Cnossen,3 Frank W G Leebeek,4 Marjolein Peters1 2 1Amsterdam Haemophilia Haemophilia, which means love (“philia”) of blood SOURCES AND SELECTION CRITERIA Treatment Centre, Academic (“haemo”), is associated with prolonged and excessive We searched Medline and the Cochrane Database of Systematic Medical Center, 1100 DD bleeding. It is a hereditary disorder of haemostasis that Amsterdam, Netherlands Reviews for “haemophilia” and “hemophilia”, and we consulted 2Department of Paediatric occurs in one in 5000 men (prevalence of 10 in 100 000 the TRIP database for national guidelines on the management Haematology, Emma Children’s people) and is caused by a deficiency of clotting factor VIII of haemophilia. Finally, we also used our personal reference Hospital, Academic Medical Center, (in haemophilia A) or factor IX (in haemophilia B) as a result libraries, giving preference to the highest level of evidence Amsterdam, Netherlands of defects in the F8 and F9 genes. Basic knowledge of the available. 3Department of Paediatric Haematology and Oncology, inheritance and management of haemophilia is essential for Erasmus Medical Center, Sophia a broad group of healthcare workers, because severe or even concentration is expressed in international units (IU); 1 Children’s Hospital, Rotterdam, life threatening bleeding can be prevented if the condition is IU is defined as the concentration of coagulation factor Netherlands 4Department of Haematology, adequately diagnosed and promptly treated. Furthermore, in 1 mL of normal pooled plasma. Healthy people have a Erasmus Medical Center, in women carriers who have an affected fetus, special pre- factor VIII or factor IX plasma concentration of 0.50-1.50 Rotterdam, Netherlands cautions are needed to prevent perinatal bleeding in both IU/mL. Factor VIII or factor IX concentrations can also Correspondence to: K Fijnvandraat the mother and the newborn baby. This review presents cur- be expressed as percentages of normal pooled plasma [email protected] rent recommendations for the diagnosis and management (defined as 100%), with normal levels between 50% and Cite this as: BMJ 2012;344:e2707 doi: 10.1136/bmj.e2707 of haemophilia, which are generally based on observational 150%. Patients with haemophilia are classified into three studies and case series because few randomised clinical severity groups: severe, moderate, and mild.4 Patients with t rials have been published in this relatively rare disease. severe haemophilia have no measurable factor VIII or fac- tor IX (<0.01 IU/mL or <1%) and may bleed spontaneously What causes haemophilia? without preceding trauma. In patients with moderate and When the body is injured, the haemostatic process is imme- mild haemophilia, the plasma factor VIII or factor IX con- diately initiated to protect the body’s integrity and to pre- centration is 0.02-0.05 IU/mL (or 2-5%) or 0.06-0.40 IU/ vent further bleeding. Activation of platelets at the site of mL (or 6-40%), respectively. Excessive bleeding usually injury is followed by sequential activation of clotting factors occurs after minor trauma, dental procedures, or surgery. and fibrin formation. Factor VIII and factor IX are essential The diagnosis of haemophilia can be established for enhancement of thrombin generation and propagation shortly after birth of an affected son when the mother is a of fibrin formation. Circulating factor VIII is bound to von known carrier. When this is not the case, the diagnosis is Willebrand factor (VWF) to protect it from proteolytic degra- established when bleeding symptoms occur, either spon- dation.1 Both factor VIII and factor IX are encoded by genes taneously or after mild trauma. Characteristic bleeding located on the long arm of the X chromosome. When the F8 symptoms are intracranial bleeding in full term infants or F9 gene sequence is disrupted, the synthesis of factor VIII after delivery, painful swelling of the joints caused by hae- or factor IX is reduced or absent, or a less functionally active marthroses, unexplained bruising when the baby starts to form is produced (http://hadb.org.uk/).2 3 crawl or walk, postoperative bleeding, extensive subcuta- neous bleeding after venepuncture, and muscle bleeding— How can patients with haemophilia be identified? either spontaneously or after intramuscular vaccination. The severity of bleeding symptoms in patients with hae- mophilia depends on the residual plasma concentration How is the diagnosis of haemophilia established? of the deficient coagulation factor. Coagulation factor Haemorrhagic manifestations that cannot be explained by trauma and prolonged or excessive bleeding after SUMMARY POINTS surgical or dental procedures require a thorough haemo- In patients with major (head) trauma or major spontaneous bleeding, always give coagulation static laboratory evaluation, including activated partial factor concentrates without delay, before diagnostic imaging or other interventions thromboplastin time and analysis of factor VIII and factor Prophylactic administration of factor VIII or factor IX concentrate is the standard of care for patients IX. Patients with haemophilia usually have a prolonged with severe haemophilia in countries where this is economically feasible activated partial thromboplastin time, although a normal Avoid drugs that affect haemostasis, such as platelet inhibitors and anticoagulants because they result cannot rule out a mild form of haemophilia. Before aggravate bleeding symptoms a definite diagnosis of mild haemophilia A is established, A safety amulet that makes patients with haemophilia easily recognisable may be life saving and is a defect in the binding of factor VIII and VWF, which can recommended for all patients also cause reduced concentrations of factor VIII, needs to Advise female relatives of patients with haemophilia to seek genetic counselling because they may be ruled out. This defect characterises type 2N or “Nor- be carriers mandy type” von Willebrand disease, which can also Female carriers may have reduced plasma concentrations of factor VIII or factor IX, similar to those affect female patients because unlike haemophilia it is seen in patients with mild haemophilia and may also have bleeding symptoms inherited in an autosomal pattern. The diagnosis of type 36 BMJ | 5 MAY 2012 | VOLUME 344 CLINICAL REVIEW bmj.com 2N von Willebrand disease can be established by special Whenever possible avoid drugs that impair haemosta- 5 Previous articles in this binding assays or by molecular genetic testing. Bleeding sis, such as platelet inhibitors and anticoagulant drugs, series in patients with type 2N von Willebrand disease is treated because these will aggravate bleeding symptoms. Ж The management by administration of VWF concentrate. of overactive bladder Use of prophylaxis to prevent joint damage Use of clotting factor concentrates to treat haemorrhages syndrome Repetitive joint bleeding may cause extensive damage to Bleeding in patients with haemophilia is treated by admin- the synovia by deposition of iron and subsequent chronic (BMJ 2012;344:e2365) istration of the deficient clotting factor. The first clotting inflammation. This causes pain, swelling, and instability of Cluster headache Ж factor concentrates were derived from plasma and became the joint, predisposing it to further bleeding and ultimate (BMJ 2012;344:e2407) available around 1970. Recombinant factor VIII and factor development of arthropathy: painful joint deformation Ж The management of IX concentrates were developed in the 1990s. As stated in a with limited function. ingrowing toenails recent UK guideline, these are currently the preferred treat- To prevent haemarthrosis, a recent UK guideline recom- (BMJ 2012;344:e2089) ment for children in the developed world, and for adult mended prophylactic administration of factor VIII or factor Ж Assessment and patients in some countries, such as the United Kingdom IX concentrate two to three times a week as the standard management of and Sweden.6 of care for patients with severe haemophilia A or B who vulval pain The dose and duration of clotting factor administration live in countries where this is economically feasible.8 (BMJ 2012;344:e1723) depends on the severity and type of bleeding. An impor- Patients with moderate or mild haemophilia are gener- Ж Diagnosis and tant treatment principle in case of major (head) trauma or ally treated “on demand”—only when bleeding occurs. A management of primary bleeding is: factor first. Coagulation factor concentrates well conducted randomised clinical trial in 65 boys with hyperparathyroidism should always be administered without delay before per- severe haemophilia A recently established the benefits (BMJ 2012;344:e1013) forming diagnostic imaging or other interventions. This of prophylaxis. The relative risk of joint damage detected strategy can be life saving because clotting factors are a by magnetic resonance imaging at the age of 6 years with prerequisite for the immediate arrest of haemorrhage in on demand treatment versus prophylaxis was 6.1 (95% patients with haemophilia. Unfortunately, this need is not confidence interval 1.5 to 24.4).9 This beneficial effect was always recognised, and preventable morbidity or death confirmed in another randomised trial of 45 children.10 A does still occur. well designed case-control study established
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