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Challenges in Managing Acute Bleeding in Patients with Hemophilia

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Challenges in Managing Acute Bleeding in Patients with Hemophilia Challenges in Managing Acute Bleeding in Patients with Hemophilia Presentation Outline Hemophilia and Bleeding Disorders: 1. Epidemiology and genetics Diagnosis and Clinical Features 2. Clinical features of hemophilia Mark T. Reding, MD 3. Inhibitors in congenital hemophilia Director, Center for Bleeding and Clotting Disorders 4. Acquired hemophilia University of Minnesota Medical Center, Fairview Associate Professor University of Minnesota Medical School Minneapolis, MN Q1: How often do you manage acute What is Hemophilia? bleeding in patients with hemophilia? a. Quite often, I am associated with a • Congenital bleeding disorder hemophilia treatment center • Due to deficiency or absence of a b. Once a year coagulation cascade protein c. Every few years • Hemophilia A = factor VIII deficiency d. Never • Hemophilia B = factor IX deficiency • Others . Rare Bleeding Disorders Hemophilia A Hemophilia B Protein Prevalence Genetics Specific Rx Fibrinogen 1 : 1,000,000 AR Yes • Factor VIII deficiency • Factor IX deficiency Factor II 1 : 2,000,000 AR No • Classical hemophilia • Christmas disease Factor V 1 : 1,000,000 AR No • 1 in 5,000 to 10,000 • 1 in 30,000 male Factor VII 1 : 500, 000 AR Yes malbithle births bir ths Factor X 1 : 1,000,000 AR No • 80% of total cases • 20% of total cases Factor XI 1 : 1,000,000 AD Yes # • Spontaneous • Spontaneous Factor XIII 1 : 2,000,000 AR Yes mutations = 30% mutations = 20% AR = autosomal recessive, AD = autosomal dominant # Not available in U.S. • Account for 3 - 5% of all inherited coagulation disorders Clinical phenotypes are indistinguishable • Higher prevalence in areas of geographic or social isolation 1 Challenges in Managing Acute Bleeding in Patients with Hemophilia • Hemophilia affects Age Distribution of the U.S. all racial and Hemophilia Population socioeconomic groups equally • There are ~ 20,000 Age (yr) Number Percent hemophiliacs in the 2 – 19 8584 48% United States • More than 500,000 20 – 44 6418 36% hemophiliacs 45 – 64 2274 13% worldwide 65+ 524 3% Centers for Disease Control and Prevention. Summary report of UDC Activity (National). Report date May 30, 2011. Genetics of Hemophilia Genetics of Hemophilia • Genes for factors VIII and IX are located on the X chromosome • Females are carriers, males are affected Hig h rate o f spontaneous mutations • Unaware female carriers • New mutation in baby boy • ~30% have no family history of hemophilia Genetics of Hemophilia Diagnosis of Hemophilia + Family History No Family History • Identify carriers • Bleeding with birth trauma, circumcision, • Pre-conception immunizations counseling • Suspected child • Cord blood testing of abuse males • Joint bleeds and • Defer testing of hematomas start to females until sx or occur when learning considering pregnancy to walk 2 Challenges in Managing Acute Bleeding in Patients with Hemophilia Diagnosis of Hemophilia Clinical Features of Hemophilia Laboratory testing Severity of bleeding tendency depends on the factor level • Normal CBC Mild ( > 5% ) Moderate (1-5%) Severe ( < 1 %) • Normal platelet function • Bleed only • Bleed after • Frequent after severe injury, surgery spontaneous • Normal PT / INR injury, trauma, bleeding • May have or surgery • Prolonged aPTT occasional • Diagnosis • May not be spontaneous made in early • Measure factor VIII and IX levels diagnosed until bleeding childhood adulthood Clinical Features of Hemophilia: Joint bleed (hemarthrosis) The Clinical Problem of Joint Bleeding • Hemarthrosis, primarily involving the ankles, knees, and elbows, is the most common complication of hemophilia • 45% experience first joint bleed within first year of life • Median age at first joint bleed: 17 – 26 months • 90% have at least one joint bleed by 4 years of age • 90% of those with severe hemophilia have chronic degenerative changes involving at least 1 joint by age 25 • 40% report restricted physical activities due to arthropathy Lafeber et al. Haemophilia. 2008; 14(Suppl 4):3-9. Valentino et al. Semin Hematol. 2008; 45(Suppl 1):S50-S57. ©2009 Rush University Medical Center, used with permission. The Clinical Problem of Joint Bleeding Clinical Features of Hemophilia: Joint bleed (hemarthrosis) Blood in joint space • 26 yo with severe hemophilia A and fVIII Recurrent Inflammation inhibitor bleeding • Recurrent traumatic and spontaneous knee bleeds Acute and chronic synovitis • Left side surgically replaced • Hemophilic arthropathy is characterized by cartilage and bone • Note severe muscular destruction, bone remodeling, and progressive loss of function atrophy • Prophylactic administration of clotting factor concentrates is essential for preventing hemophilic arthropathy SK 3 Challenges in Managing Acute Bleeding in Patients with Hemophilia Clinical Features of Hemophilia: Clinical Features of Hemophilia: Joint bleed (hemarthrosis) Joint bleed (hemarthrosis) • 36 year old • 36 year old • Severe hemophilia A • Severe hemophilia A • Recurrent left knee bleeds • Recurrent left knee • Severe hemophilic bleeds arthropathy • Severe hemophilic arthropathy • Underwent total knee arthroplasty • Infected prosthesis had to be removed 3 months later TS TS Patient: LF, 22-yo male with severe hemophilia A March 2008 May 2010 June 2010 Severe hemophilia A, no inhibitor, morbidly obese RS Clinical Features of Hemophilia: Joint bleed (hemarthrosis) • Severe hemophilia A with inhibitor and • 36 year old, severe hemophilia A, followed by hemophilia advanced arthropathy treatment center since birth. No history of fVIII inhibitor. • Required right total hip arthroplasty SK • Target joint in childhood, no longer bleeds (or moves). NJ 4 Challenges in Managing Acute Bleeding in Patients with Hemophilia 44-yo male with severe hemophilia A, right elbow fracture after fall (July 2010) DK December 2010 – 4 months s/p arthroplasty, doing well Clinical Features of Hemophilia: Soft tissue bleeding February 2012 – Resumed truck driving and heavy lifting. Acquired hemophilia, non-traumatic elbow bleed Not doing so well. DT Clinical Features of Hemophilia: Clinical Features of Hemophilia: Soft tissue bleeding Deep muscle bleeds • 56 year old with severe hemophilia A and inhibitor • 20 year old with • Fell on icy sidewalk mild hemophilia A • Did no t trea t aggress ive ly • No trauma enough • Bled after light • Required transfusion of 6 jogging units RBCs GR 5 Challenges in Managing Acute Bleeding in Patients with Hemophilia Clinical Features of Hemophilia: Clinical Features of Hemophilia: Deep muscle bleeds Intracranial bleeds • 6 year old with severe hemophilia A • Bumped head on school playground equipment, did not appear to have any significant injury • Parents noted change in behavior later that • 52 year old with severe evening hemophilia B • Spontaneous bleed Clinical Features of Hemophilia: Inhibitors in Congenital Hemophilia Soft tissue bleeding • Some hemophilia patients “see” factor VIII or factor IX as a foreign protein • Infusion of factor concentrate to prevent or treat bleeding triggers an immune response • Antibodies (“inhibitors”) directed against factor VIII or factor IX neutralize the procoagulant effect and render standard treatment useless Severe hemophilia A with inhibitor, neck bleed provoked by coughing GR Inhibitors in Congenital Hemophilia Inhibitors in Congenital Hemophilia • Development of inhibitors is currently the most severe complication of factor replacement therapy Factor VIIIFactor VIII Factor IXFactor IX • Typically seen in those with severe hemophilia • Common (~25%) • Rare (< 5%) • Hemophilia A – inhibitors develop in ~25% • Well-studied and • Risk factors poorly characterized defined •Hemophilia B – inhibitors developp% in < 5% • Eradicated in ~ 70% • ITT often fails • No longer associated with increased mortality with ITT (immune • Allergic reactions, tolerance therapy) However . nephrotic syndrome Bleeding more difficult to control May develop following treatment with both Devastating joint disease and disability plasma derived and recombinant factor products Major clinical and economic challenges Similar bleeding patterns, diagnosis, and management 6 Challenges in Managing Acute Bleeding in Patients with Hemophilia Factors Influencing Inhibitor Development Clinical Recognition of Inhibitors in Hemophilia A • Usually develop in small children, after only a small number of factor exposures Patient Variables Treatment Variables • Disease severity • Number and pattern of • Change in bleeding pattern fVIII exposures • FVIII gene defect • Type of fVIII product • Poor response to treatment with factor • Ethnicity InhibitorInhibitor • Concurrent immune • FH of inhibitors system challenges • Allergic reactions often herald the • HLA type • Frequency of development of factor IX inhibitors • Individual immune monitoring response traits • May develop later in life in those with mild or moderate hemophilia A complex interaction of many variables leads to Often after intense factor exposure following inhibitor development in a particular individual surgery or trauma Gouw SC. Semin Thromb Hemost. 2009; 35:723-34. Measurement of Factor VIII Inhibitors: Treatment of Inhibitors Bethesda Assay • “Bypassing Agents” Reciprocal of - Prothrombin complex concentrates dilution at which - Recombinant factor VIIa 50% of normal FVIII activity is • Bypassing agents have unpredictable efficacy (50 – 90%) observed - More bleeding, more joint damage - SiikSurgery is risky • Immune Tolerance Therapy 1:1 1:2 1:5 1:10 1:20 1:50 11 - Expensive: ~ $1 million per patient P e r f o r m F V I I I a s s a
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