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Rarer Haemostatic Disorders Treatments see UKHCDO guidelines 2004 Haemophilia 10, 593-628

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Rarer Haemostatic Disorders Treatments see UKHCDO guidelines 2004 Haemophilia 10, 593-628

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    1. Rarer Haemostatic Disorders Treatments (see UKHCDO guidelines 2004 Haemophilia 10, 593-628) Paula Bolton-Maggs Consultant Haematologist Manchester Royal Infirmary, UK

    9. Rare coagulation disorders Autosomal recessive (?) inheritance More common in racial groups where cousin marriage frequent Factor XI common in Ashkenazy Jews Generally, lack of clear correlation between bleeding and level of factor, so more difficult to manage

    10. Severe deficiency – rare disorders

    11. In vivo properties (1:Rizza 1982, 2: Mannucci 2004)

    12. What products are manufactured? FV deficiency: no concentrate – use virally-treated FFP Octaplas – pooled from approx 1500 donations, SD treated, sourced from Austria, Germany and USA Methylene-blue treated single donor units Platelet disorders: DDAVP, fibrinolytic inhibitors, platelet transfusions and possibly rVIIa

    13. Fibrinogen Treatment products Unlicensed virally inactivated concentrates ZLB SNBTS LFB Half life 3-5 days Tranexamic acid Cryo is a good source but not virally inactivated

    14. Fibrinogen products

    15. Treatment for FII Beriplex PN (ZLB) contains II, VII, IX and X DEFIX HT (SNBTS) contains II, IX and X Prothromplex (Baxter) contains II, IX and X Prothromplex T (Baxter) similar but also contains factor VII Kaskadil (LFB) with VII

    16. Treatment Products FVII rVIIa is the treatment of choice although little data Factor IX concentrates containing FVII Factor VII concentrates (LFB and Baxter) (FFP) A level of 10-15 u/dl is probably sufficient for surgery (level of 2 u/dl shown to normalise the thrombin generation test but not tested in clinical situation)

    17. Products for FVII deficiency

    18. Factor X Treatment Products No factor X concentrate Intermediate purity Factor IX concentrates Increased risk of thrombosis (FFP) A level of 10-25 u/dl is probably sufficient for surgery People with levels >10 u/dl probably require no replacement therapy

    19. Products for FX (and FII) deficiency

    20. Therapeutic options in FXI deficiency Fresh frozen plasma (Virally treated) Factor XI concentrates (BPL, LFB) but thrombogenic Fibrinolytic inhibitors – very effective for dental extractions Fibrin glue – main experience in Israel DDAVP - unconvincing rVIIa – risk of thrombosis

    21. Products for FXI deficiency

    22. Factor XIII deficiency 6 patients managed from 19-35 years Prophylaxis – long half life 11-14 days Pasteurised plasma-derived concentrate available from ZLB (Fibrogammin-P) Give 1000 units every 5-6 weeks which raises level to 30-35 u/dl (NB assay problems) Platelets contain FXIII and in an emergency may be useful.

    23. SSC working group on “Rare Bleeding Disorders” (RBDs) In 2004, during the 50th SSC meeting organized by the ISTH and held in Venice, a SSC working group on "Rare Bleeding Disorders" (RBDs) was held within the framework of the FVIII/IX subcommittee

    27. Available treatments

    28. Problems with RBDs treatments Inability to get product and costs: - Central and South America, Asia and Middle East, Russia - India: sometimes products are past expiration date No availability of product: - Brazil and Vietnam: general lack of products - FXI reported by Arizona, Brazil, Belgium, London-UK, Switzerland centres - Denmark: sometimes stopped FIX-X product - Iran: Prothrombin complex Adverse effects: mild urticaria, reaction to plasma, thrombosis with FXI in the past, fear of blood borne viral infections Other: - Panama: products available only for insured patients

    31. Our work could represent the starting point for the establishment of an International Database under the supervision of an International Society such as WFH or ISTH In order to create an International collaboration on RBDD and to decide eventual clinical trials, it will be necessary to build up a STEERING COMMITTEE composed by experts in clinic, treatment and genotype-phenotype characterization coming from all over the world Next steps

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