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Online applications designed to facilitate tailoring of hemophilia A prophylaxis supported a regimen change in nearly half of patients enrolled in a recent study, investigators report.
The development of individualized pharmacokinetic profiles using those online tools may have contributed to improved adherence and decreased bleeding, according to Azusa Nagao, MD, a researcher in the department of blood coagulation at Ogikubo Hospital, Tokyo, and colleagues.
Dr. Nagao and colleagues calculated individual pharmacokinetic profiles for the 39 patients using myPKFiT for those patients receiving antihemophilic factor (recombinant) and the Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo) for those using other factor concentrates.
Based on profiling results, changes to prophylaxis regimens were warranted for 20 of the 39 patients, Dr. Nagao and coresearchers reported in Thrombosis Research.
Those changes included a higher dosage of factor concentrate in eight patients, a shorter infusion interval in five patients, a change from a standard half-life product to an extended half-life product in five patients, and a reduction in prophylaxis dose for two patients.
Adherence stayed the same in patients who had a dose increase and improved for those who switched to a shorter infusion interval and for those who switched to an extended half-life product, according to the researchers.
Annualized bleeding rate significantly decreased for patients who switched to an extended half-life product (P less than .05), and there was a trend toward decrease in annualized joint bleeding rate in this group, the researchers reported.
The annualized joint bleeding rate did significantly decrease in patients without end-stage arthropathy (P less than .05), with no significant change in this outcome for those with end-stage arthropathy.
“The impact of adopting population pharmacokinetics-based treatment as measured by changes in treatment adherence and bleeding rate was modest, but the study power was limited by the characteristics of the study population,” the researchers wrote.
The myPKFiT and WAPPS-Hemo tools are designed to simplify the development of individual pharmacokinetic profiles to guide prophylaxis dosing in patients with hemophilia A, according to the researchers.
“Full [pharmacokinetic] analysis poses a big burden, especially on children and their families,” they wrote.
A full pharmacokinetic study requires a 72-hour washout period and sample collection at 10 time points over the course of about 48 hours, along with associated hospital admissions or outpatient clinic visits, they noted.
The myPKFiT application, cleared by the Food and Drug Administration for use in hemophilia A patients being treated with antihemophilic factor (recombinant), can generate an individual’s pharmacokinetic profile based on as few as two blood samples, according to a news release from Shire.
Nagao reported receiving a research grant from Shire unrelated to this study. One coauthor also reported disclosures related to Shire, Bayer, Pfizer, Novo Nordisk, CSL Behring, and others.
SOURCE: Nagao A et al. Thromb Res. 2019 Jan;173:79-84.
Online applications designed to facilitate tailoring of hemophilia A prophylaxis supported a regimen change in nearly half of patients enrolled in a recent study, investigators report.
The development of individualized pharmacokinetic profiles using those online tools may have contributed to improved adherence and decreased bleeding, according to Azusa Nagao, MD, a researcher in the department of blood coagulation at Ogikubo Hospital, Tokyo, and colleagues.
Dr. Nagao and colleagues calculated individual pharmacokinetic profiles for the 39 patients using myPKFiT for those patients receiving antihemophilic factor (recombinant) and the Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo) for those using other factor concentrates.
Based on profiling results, changes to prophylaxis regimens were warranted for 20 of the 39 patients, Dr. Nagao and coresearchers reported in Thrombosis Research.
Those changes included a higher dosage of factor concentrate in eight patients, a shorter infusion interval in five patients, a change from a standard half-life product to an extended half-life product in five patients, and a reduction in prophylaxis dose for two patients.
Adherence stayed the same in patients who had a dose increase and improved for those who switched to a shorter infusion interval and for those who switched to an extended half-life product, according to the researchers.
Annualized bleeding rate significantly decreased for patients who switched to an extended half-life product (P less than .05), and there was a trend toward decrease in annualized joint bleeding rate in this group, the researchers reported.
The annualized joint bleeding rate did significantly decrease in patients without end-stage arthropathy (P less than .05), with no significant change in this outcome for those with end-stage arthropathy.
“The impact of adopting population pharmacokinetics-based treatment as measured by changes in treatment adherence and bleeding rate was modest, but the study power was limited by the characteristics of the study population,” the researchers wrote.
The myPKFiT and WAPPS-Hemo tools are designed to simplify the development of individual pharmacokinetic profiles to guide prophylaxis dosing in patients with hemophilia A, according to the researchers.
“Full [pharmacokinetic] analysis poses a big burden, especially on children and their families,” they wrote.
A full pharmacokinetic study requires a 72-hour washout period and sample collection at 10 time points over the course of about 48 hours, along with associated hospital admissions or outpatient clinic visits, they noted.
The myPKFiT application, cleared by the Food and Drug Administration for use in hemophilia A patients being treated with antihemophilic factor (recombinant), can generate an individual’s pharmacokinetic profile based on as few as two blood samples, according to a news release from Shire.
Nagao reported receiving a research grant from Shire unrelated to this study. One coauthor also reported disclosures related to Shire, Bayer, Pfizer, Novo Nordisk, CSL Behring, and others.
SOURCE: Nagao A et al. Thromb Res. 2019 Jan;173:79-84.
Online applications designed to facilitate tailoring of hemophilia A prophylaxis supported a regimen change in nearly half of patients enrolled in a recent study, investigators report.
The development of individualized pharmacokinetic profiles using those online tools may have contributed to improved adherence and decreased bleeding, according to Azusa Nagao, MD, a researcher in the department of blood coagulation at Ogikubo Hospital, Tokyo, and colleagues.
Dr. Nagao and colleagues calculated individual pharmacokinetic profiles for the 39 patients using myPKFiT for those patients receiving antihemophilic factor (recombinant) and the Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo) for those using other factor concentrates.
Based on profiling results, changes to prophylaxis regimens were warranted for 20 of the 39 patients, Dr. Nagao and coresearchers reported in Thrombosis Research.
Those changes included a higher dosage of factor concentrate in eight patients, a shorter infusion interval in five patients, a change from a standard half-life product to an extended half-life product in five patients, and a reduction in prophylaxis dose for two patients.
Adherence stayed the same in patients who had a dose increase and improved for those who switched to a shorter infusion interval and for those who switched to an extended half-life product, according to the researchers.
Annualized bleeding rate significantly decreased for patients who switched to an extended half-life product (P less than .05), and there was a trend toward decrease in annualized joint bleeding rate in this group, the researchers reported.
The annualized joint bleeding rate did significantly decrease in patients without end-stage arthropathy (P less than .05), with no significant change in this outcome for those with end-stage arthropathy.
“The impact of adopting population pharmacokinetics-based treatment as measured by changes in treatment adherence and bleeding rate was modest, but the study power was limited by the characteristics of the study population,” the researchers wrote.
The myPKFiT and WAPPS-Hemo tools are designed to simplify the development of individual pharmacokinetic profiles to guide prophylaxis dosing in patients with hemophilia A, according to the researchers.
“Full [pharmacokinetic] analysis poses a big burden, especially on children and their families,” they wrote.
A full pharmacokinetic study requires a 72-hour washout period and sample collection at 10 time points over the course of about 48 hours, along with associated hospital admissions or outpatient clinic visits, they noted.
The myPKFiT application, cleared by the Food and Drug Administration for use in hemophilia A patients being treated with antihemophilic factor (recombinant), can generate an individual’s pharmacokinetic profile based on as few as two blood samples, according to a news release from Shire.
Nagao reported receiving a research grant from Shire unrelated to this study. One coauthor also reported disclosures related to Shire, Bayer, Pfizer, Novo Nordisk, CSL Behring, and others.
SOURCE: Nagao A et al. Thromb Res. 2019 Jan;173:79-84.
FROM THROMBOSIS RESEARCH
Key clinical point:
Major finding: Adherence stayed the same or improved in patients whose prophylaxis regimens were changed based on the tool results, while bleeding rates improved for specific patient subsets.
Study details: Prospective study including 39 patients receiving hemophilia A prophylaxis.
Disclosures: Study authors reported disclosures related to Shire, Bayer, Pfizer, Novo Nordisk, CSL Behring, and others.
Source: Nagao A et al. Thromb Res. 2019 Jan;173:79-84.