The efficacy and safety of lyophilized cryoprecipitate in hemophilia A.

This prospective study of assessing the efficacy and safety of lyophilized cryoprecipitate (LC), which was heat-treated at 60 degrees C for 25 hours, was conducted in 23 patients with hemophilia A (severe 13, moderate 9, mild 1) at the International Hemophilia Training Center, Bangkok from 1997 to 1998. A total of 223 infusions of LC were given. The status of the patients could be classified into 4 groups: group I, non-bleeding (n = 13); group II, severe bleeding requiring hospitalization (n = 9); group III, appendectomy (n = 1) and group IV, early bleeding controlled by modified home treatment (n = 200). Pharmacokinetic studies were conducted in groups I and II. The mean in vivo half-life of factor VIII clotting activity (F VIII:C) was 12.6 hours and the mean in vivo incremental recovery at baseline was 2.1 per cent/unit/kg. The mean clearance was 3.22 ml/kg/h. There was no statistically significant difference in these parameters between groups I and II (p > 0.05). The hemostasis was successfully achieved and 1 to 2 small urticarial wheals were observed in only 2 infusions. In addition, 9 out of 23 patients received LC exclusively for 1 year. None of them developed inhibitor to F VIII:C nor did any contract additional transfusion-transmitted infection except one who developed anti-hepatitis C virus seroconversion after receiving 16 bottles of LC in 4 months. Therefore, the more efficient virus-inactivation in the preparation of LC should be established.

Home treatment for patients with congenital bleeding disorders in a developing country.

Ninety-six patients with congenital bleeding disorders were enrolled in a home treatment program from 1979 to 1997. The diagnosis included severe and moderate hemophilia A and B (n = 63), mild hemophilia A (n = 18), von Willebrand disease (vWD) (n = 12) and congenital factor VII deficiency (n = 3). The median age was 9 years and the median duration of follow-up was 4 3/12 years. The home treatment was modified 4 ways: (1) Using locally-prepared single units of fresh dry plasma in the majority of the patients while mild hemophilia A and vWD patients received 1-deamino 8D-arginine vasopressin. (2) Recruiting local health personnel as the primary care providers. (3) Teaching and training patients and parents intensively. (4) Maintaining an effective control system. The heartful effort of the health personnel was not in vain; the patients learned to take good care of themselves. Twenty patients and 20 parents or relatives were able to perform venipuncture properly and no adverse effect was observed. Since the hemorrhage was treated very early, the severity and sequelae of bleeding were decreased. The utilized blood components and days of hospitalization were reduced. Impressively, the absenteeism from work or school was minimized so that the patients could enjoy a near normal life in their family, school and society.