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Inhibitor treatment using unactivated prothrombin complex concentrates: the Pennsylvania experience--1978-1982
Insights
Prothrombin complex concentrates (PCC) show safety and efficacy in treating bleeding episodes in patients with Factor VIII inhibitors. However, high treatment costs and usage for recurrent hemarthroses are significant concerns.
Area of Science:
- Hematology
- Pharmacology
Background:
- Factor VIII inhibitors complicate hemophilia treatment.
- Prothrombin complex concentrates (PCC) are an alternative therapy.
Purpose of the Study:
- To evaluate the safety and efficacy of unactivated prothrombin complex concentrates (PCC) in patients with Factor VIII inhibitors.
Main Methods:
- Retrospective analysis of 48 patients with Factor VIII inhibitors treated with PCC between 1978-1982.
- Data collected on bleeding episodes, PCC usage, adverse events, and hospitalization rates.
Main Results:
- PCC demonstrated safety with minimal severe allergic reactions or thrombotic complications.
- Efficacy suggested by successful treatment of hemarthroses and muscle hemorrhages.
- Hospitalization rates comparable to non-inhibitor patients, though less effective than AHF therapy.
- High annual PCC usage and cost noted, particularly for recurrent bleeding.
Conclusions:
- Unactivated PCC is a safe and reasonably effective treatment for bleeding episodes in patients with Factor VIII inhibitors.
- High cost and intensive use for recurrent hemarthroses warrant further investigation and management strategies.
Abstract:
Data were analyzed on 48 patients with Factor VIII:C inhibitors, 31 of whom were treated with more than 6.7 million units of unactivated prothrombin complex concentrates (PCC) during the years 1978 through 1982 for more than 1,000 bleeding episodes. Twelve of the patients who were on home therapy used 3.1 million units, or 47% of the total PCC usage. Only one patient had a severe life-threatening allergic reaction to PCC, and no thrombotic complications were observed, supporting the concept that these products are safe when used in doses of 50-75 u/Kg. Fifty percent of hemarthroses and 65% of muscle hemorrhages were treated with no more than two infusions per episode, suggesting that PCC was effective at least half of the time. Comparison of the number of days hospitalized per year for inhibitor and non-inhibitor patients also indicated that PCC was effective, although not as effective as AHF therapy in non-inhibitor patients. The life styles of PCC treated patients were comparable to what might be expected for non-inhibitor patients with severe disease, and no deaths were attributed to PCC treatment failure during the four year period. Of major concern, however, was the cost of therapy. In 1981-82, the average use of PCC for inhibitor patients was 106,000 units per treated patient per year, compared to 40,000 AHF units for all Factor VIII deficient treated patients per year. Much of this usage was for recurrent hemarthroses in a small number of patients requiring eight or more infusions per episode.