ORLANDO—It may be possible for hemophilia patients to receive less factor VIII (FVIII) without increasing their risk of bleeding, according to a study presented at the World Federation of Hemophilia 2016 World Congress.
The study1 showed that hemophilia A patients who received prophylactic FVIII from the home infusion provider Option Care received 6 fewer units of FVIII per week than patients receiving prophylactic FVIII from specialty pharmacies.
This translated to a cost savings of more than $20,000 per patient each year.
The home infusion patients also had a lower annual bleed rate (ABR) than what is typically observed with intensive prophylaxis protocols in hemophilia, according to previous studies.
“By working with prescribers to closely monitor bleeds and collaborate on clinically appropriate optimization of treatment dose, Option Care’s utilization of factor VIII is less than the average with excellent outcomes,” said Joan Couden, RN, national program director for Option Care’s Bleeding Disorders Program.
“Our findings show we can save payers, including Medicare, Medicaid, and managed care insurers, significant costs without negatively impacting annual bleed rates.”
For this study, Couden and her colleagues conducted a retrospective analysis using dispensing data records from Option Care spanning the period from July 2015 through December 2015. The team compared these data to aggregate specialty pharmacy records from November 2013 through March 2014, which were analyzed in a previous study.2
In both data sets, patients receiving any FVIII product for prophylactic therapy were included. Patients being treated episodically or for immune tolerance induction were excluded, as were patients with extremely abnormal weights (40% below the 5th percentile or 40% above the 95th percentile based on weight-for-age charts from the US Centers for Disease Control and Prevention).
The researchers calculated a patient’s weekly dose of FVIII by multiplying the prescribed infusion dose by the dose frequency and dividing the product by the patient’s weight. Patients with an overall mean weekly dose greater than 2 standard deviations from the mean were excluded.
There were 77 home infusion patients and 520 specialty pharmacy patients.
The home infusion patients had a mean FVIII dose of 102 units/kg/week, compared to a mean of 108 units/kg/week for the specialty pharmacy patients.
This difference translates to savings of $21,166 per patient per year among the home infusion patients.
Couden and her colleagues could not compare the ABR between the 2 data sets because the ABR was not measured in the specialty pharmacy patients. However, they said the ABR in the home infusion patients was favorable when compared to ABRs in published studies.
The mean ABR for the home infusion patients was 1.70. And, according to a recent review of research on hemophilia treatment strategies, mean ABRs range from 2 to 5 for intensive treatment protocols.3