Prophylaxis and therapy with factor VII concentrate (human) immuno, vapor heated in patients with congenital factor VII deficiency: a summary of case reports.

Hereditary factor VII deficiency is a rare autosomal recessive condition, usually associated with normal or reduced levels of a functionally defective molecule. The available means of treating this condition in North America presents serious health risks to the patient. Transfusion with fresh frozen plasma carries a risk of volume overload and a significant risk for viral transmission. Sustained prothrombin complex therapy is associated with a high risk for thrombogenic complications. This communication describes the use of Factor VII Concentrate (Human) Immuno, Vapor Heated--an intermediate purity factor VII concentrate from Immuno A.G.--for the treatment of 13 patients with factor VII deficiency. Treatment regimens described include those for long-term prophylaxis (three children), acute hemorrhages (two children, one adult), peripartum prophylaxis (one patient), and surgical coverage (two children, four adults). Prophylaxis and therapy were successful in all cases, the medication was well-tolerated, and there were no complications. In the three cases of long-term prophylaxis in children, doses of 10-50 IU/kg were given one to three times a week; one patient has undergone long-term prophylaxis for approximately 8 years, one patient for 1 year, and one patient for 1 1/2 years. Three cases in which Factor VII Concentrate was principally used for treatment of acute episodes of bleeding are described. One infant received Factor VII Concentrate on about 50 occasions for treatment of mucosal bleeding; a correction to 40-100% resulted in cessation of bleeding within 15 min in all cases. For treatment of an episode of intracranial bleeding, an 8-year-old boy received a dose of 37 IU/kg Factor VII Concentrate every 6 hr for peak factor VII levels of approximately 100% and troughs as low as 4% over the 11-day treatment period. A 37-year-old adult male with intracranial bleeding received alternating doses of 16 IU/kg and 8 IU/kg every 6 hr for 10 days with peak factor VII levels in the upper thirties (%). The peak favor VII level during surgical coverage with Factor VII Concentrate (neurosurgery, open reduction of ankle bones, dental surgery, pituitary adenoma surgery, closed liver biopsy) was approximately 100% in all cases, with trough levels ranging from 8 to 65% over treatment periods of 24 hr to 16 days using treatment intervals of 6-12 hr.

Prevalence of HTLV-III antibody among New Mexico residents with hemophilia.

Persons with hemophilia who have received therapy since 1978 are at risk for the acquired immunodeficiency syndrome. Plasma and serum specimens collected from 1980 to 1985 from 73 New Mexico residents with hemophilia were tested by enzyme immunoassay for antibody to human T-cell lymphotropic virus type III (HTLV-III), and positive results were confirmed by Western blot. Antibody to HTLV-III was first detected in New Mexico residents with hemophilia in 1981. Among 49 persons tested in 1984-1985, seropositivity was found in 35%. Of these, 17 of 32 (53%) commercial concentrate versus 0 of 17 cryoprecipitate users were seropositive (P = .002). Of the 7 with hemophilia B, 3 (43%) were seropositive versus 14 of 42 (33%) with hemophilia A. Of 17 Albuquerque residents with hemophilia, 2(12%) were seropositive as compared with 15 of 32 (47%) persons with hemophilia who resided outside the city (P = .02). Compared with patients with hemophilia outside of Albuquerque, those living in Albuquerque tended to have milder disease and to use cryoprecipitate rather than commercial concentrate. Less frequent treatment (mild disease) and use of cryoprecipitate were associated with a decreased risk of HTLV-III infection.

Phase I study of ICRF-187 in pediatric cancer patients and comparison of its pharmacokinetics in children and adults.

A phase I study of ICRF-187 as a 2-hour iv infusion daily for 3 days was conducted in 46 evaluable pediatric patients. The maximum tolerated dose was 3500 mg/m2/day X 3 based on changes in hepatic function and coagulation abnormalities encountered when larger dosages were administered. One patient with acute lymphocytic leukemia achieved a complete remission and four cleared the blood of lymphoblasts. No other objective responses were seen. Pharmacokinetic analysis showed that the children had a larger volume of distribution per kilogram of body weight in the central compartment and total body and a more rapid total-body clearance than adults. These parameters can explain only part of the increased tolerance of children to ICRF-187.

Evaluation of a skin test for chicken pox.

Results with a VZV skin test as a marker of past infection were compared with histories of chicken pox and specific antibody detected by ELISA in 100 individuals--25 of whom were pediatric patients with malignant diseases. A negative or uncertain history was not reliable, neither were the skin test results among the oncology patients. However, among the normal individuals, the skin test when compared with the ELISA had a sensitivity of 85%, a specificity of 100%, and a positive predictive value of 100%.

Carpal tunnel syndrome in hemophilia.

Carpal tunnel syndrome occurring as a complication of hemophilia is documented in the literature. Most reports, however, indicate that the condition can be relieved by splinting and replacement therapy. Two cases of carpal tunnel syndrome in hemophiliacs are presented. In one patient, replacement therapy was successful in relieving the condition. However, in the other patient, decompression of the carpal tunnel and internal neurolysis of the median nerve were carried out after replacement therapy failed. To our knowledge, this is the first time that intraneural bleeding in the nerve has been documented as a cause of peripheral neuropathy in hemophilia. Surgical release of the carpal canal together with the epineurectomy and internal neurolysis resulted in complete recovery.