USE OF DDAVP TO IMPROVE HEMOSTASIS IN GAUCHER'S DISEASE

本研究旨在确定高谢氏病患者

This study was undertaken to determine whether patients with Gaucher's disease and thrombocytopenia could safely undergo invasive procedures (i.e., liver biopsy) under the coverage of the drug Deamino-D-Arginine- Vasopressin (DDAVP) and thereby avoid exposure to blood products. The patients all received pre-procedure screening with CBC, Coagulation screen (PT, PTT, TT, Fibrinogen), LFT's, BUN & serum creatinine, and assessment of primary hemostasis by Simplate bleeding time. All patients who met the study parameters (platelet count of 50,000 but less than 100,000; bleeding time of 10 mins.) then received a standard infusion of DDAVP (0.3ug/kg IV over 30 mins.). A bleeding time was then repeated l hour after completion of the DDAVP infusion. All patients who demonstrated a shortening of bleeding time to less than or equal to 10 mins. then underwent a liver biopsy. Follow-up evaluation of von Willebrand factor and platelet function were subsequently obtained on all patients who received DDAVP to correlate with the response to DDAVP.