Therapy for pediatric aplastic anemia: 7 years experience at the Puerto Rico Pediatric Oncology Program

Nine pediatric patients with aplastic or hypoplastic anemia were seen at the Pediatric Hematology-Oncology service of the University of Puerto Rico, from 1980 to first trimester of 1987. Included were eitht males and one female ranging from twenty months to sixteen 8/12 years (mean 13 years). Hematomas and fever were the most common complaints. The interval since the first symptoms and the diagnosis varied from one day to one week. The peripheral cell count was abnormal in all patients; pancytopenia being the most common finding. Five of seven who received antithymocyte globulin (ATG-AM), showed improvement in their peripheral count. The median time interval from the end of ATG-AM and response was two months (range 2 weeks to 15 months)

Response of patients with hemophilia A and von Willebrand disease to desmopressin (DDAVP)

Factor VIII (FVIII) response to desmopressin (deamino-8-D-arginine vasopressin, abbreviated DDAVP) was studied in patients with mild Hemophilia A and von Willebrand Disease (vWD) who attend our Hemophilia Clinic. Thirty eight children and 9 adults had their F VIII components assayed 60 minutes after intravenous (IV) administration of DDAVP, 0.35 ug/kg. Among 27 hemophiliacs, F VIII coagulant activity (F VIII: C) increased from a mean of 16.8 to 59.2 u/dL; with an average 3.2-fold increase. In 20 vWD patients, the mean F VIII:C and von Willebrand Factor increased from a mean of 50.1 to 136%; and from 22.6 to 75.6%; with an average 3.0 and 5.7-fold increase, respectively. The overall F VIII:C response was good or excellent in 81.5% of the hemophiliaacs, and in 89.5% of the vWD patients tested. No significant side effects were observed. This study has demonstrated that IV DDAVP can cause an increase of F VIII:C and vWF to hemostatic levels, and thus it may be useful for the control of bleeding episodes in most of the patients tested in our clinic