Biostate 71,500 36,500 108,000 7 855 * The total in this table combines the values for patients with mild, moderate and severe conditions. The severity of a patient’s condition is not always known at initial presentation. This table includes product issues to patients with unknown/not specified treatment regimens. All products listed above are in IUs unless stated. **IUs sums all the products except NovoSeven 24 - IU HMA HMB Haemophilia A Haemophilia B Severity Regimen type Total IUs Number of patients Total IUs Number of patients Adult - Mild On demand 3,535,050 182 1,453,000 45 Prophylaxis 560,000 6 72,000 <5 Adult - Moderate On demand 5,780,000 68 2,310,500 41 Prophylaxis 6,928,500 26 3,824,000 13 Tolerisation 120,000 <5 Adult - Severe On demand 19,227,250 102 4,598,000 24 Prophylaxis 69,014,325 241 6,863,000 26 Tolerisation 1,421,000 <5 447,000 <5 Adult - Total 106,586,125 <635 19,567,500 <159 Paediatric - Mild On demand 339,250 43 130,750 11 Prophylaxis 639,250 6 189,500 <5 Paediatric - Moderate On demand 392,750 35 397,500 9 Prophylaxis 4,218,000 25 996,750 7 Paediatric - Severe On demand 410,493 24 85,750 6 Prophylaxis 39,586,750 224 4,574,750 33 Tolerisation 3,448,250 14 Paediatric - Total 49,034,743 371 6,375,000 <71 * Values in this table exclude products issued to patients with unknown severity classification or treatment regimen, so they will vary from those figures shown previously. **Patients may receive more than one regimen type and may therefore be counted multiple times.
Appendix A Characteristics of Rare Clotting Factor Deficiencies 26 - Missing Factor Incidence* Inheritance Severity of Bleeding Treatment Factor I Afibrinogenemia Hypofibrinogenemia Dysfibrinogenemia 5 in 10 million not available 1 in 1 million Autosomal recessive Recessive or dominant Recessive or dominant Usually mild, except in afibrinogenemia •Fibrinogen conc. (Not funded in Australia) •Cryoprecipitate •Fresh frozen plasma Factor II 1 in 2 million Autosomal recessive Moderate to severe when factor levels are low; usually mild •Prothrombin complex conc. •Fresh frozen plasma Factor V 1 in 1 million Autosomal recessive Moderate to severe when factor levels are low; usually mild •Fresh frozen plasma Combined Factor V and Factor VIII 1 in 1 million† Autosomal recessive‡ Usually mild •Fresh frozen plasma •Factor VIII conc. •Desmopressin Factor VII 1 in 500,000 Autosomal recessive Severe when factor levels are low •Recombinant Factor VIIa conc. •Factor VII conc. •Fresh frozen plasma Factor X 1 in 1 million Autosomal recessive Moderate to severe when factor levels are low •Prothrombin complex conc. •Fresh frozen plasma Combined deficiency of vitamin Kdependent clotting factors not available Autosomal recessive Usually mild, but a few families have reported very low levels and more severe symptoms •Vitamin K •Prothrombin complex conc. •Fresh frozen plasma Factor XI 1 in 100,000 Recessive or dominant Mild to moderate when factor levels are low •Factor XI concentrate •Antifibrinolytic drugs •Fibrin glue •Fresh frozen plasma Factor XIII 1 in 3 million Autosomal recessive Moderate to severe when factor levels are low •Factor XIII conc. •Cryoprecipitate •Fresh frozen plasma Note: Australian Prothrombin Complex Concentrate is not used for FVII deficiency * Estimates only † 1 in 100,000 in some populations, including Israel, Iran, and Italy ‡ Very rarely, Factor VIII deficiency can be inherited separately from only one parent