Hemophilia Inhibitor
| Select | Protocol | ID |
|---|---|---|
|
#910101 |
|
|
#911602 |
|
|
#911601 |
|
|
#910401 |
|
|
#910402 |
|
|
#910501 |
|
|
#910502 |
|
|
#910701 |
|
|
#911301 |
|
|
#911402 |
|
|
#911401 |
|
|
#911502 |
|
|
#911501 |
|
|
#910201 |
|
|
#910202 |
|
|
#910301 |
|
|
#910302 |
|
|
#911701 |
|
|
#910802 |
|
|
#910801 |
|
|
#910904 |
|
|
#910903 |
|
|
#911104 |
|
|
#911103 |
|
|
#910902 |
|
|
#911102 |
|
|
#910901 |
|
|
#911101 |
|
|
#911004 |
|
|
#911204 |
|
|
#911003 |
|
|
#911203 |
|
|
#911002 |
|
|
#911202 |
|
|
#911001 |
|
|
#911201 |
|
|
#910602 |
|
|
#910601 |
Release Date:
March 19, 2019
Scope of Collection
| Element Considered | Final Measures in Toolkit |
|---|---|
| Inhibitors | Factor IX Activity in Plasma |
| Factor VIII Activity in Plasma | |
| Quantitative Measure of Factor IX Inhibitor Activity | |
| Quantitative Measure of Factor VIII Inhibitor Activity | |
| Quantitative Measure of Factor VIII Inhibitor Activity in the Presence of Emicizumab | |
| Bleeding | Counts of Acute Bleeds in Hemophilia |
| Outcome measures to evaluate effectiveness of therapy | Response to Factor IX Infusion - Individual Pharmacokinetic Study |
| Response to Factor IX Infusion - Population-based Pharmacokinetic Study | |
| Response to Factor VIII Infusion - Individual Pharmacokinetic Study | |
| Response to Factor VIII Infusion - Population-based Pharmacokinetic Study | |
| Response to Factor VIII Infusion in the Presence of Emicizumab - Individual Pharmacokinetic Study | |
| Response to Factor VIII Infusion in the Presence of Emicizumab - Population-based Pharmacokinetic Study | |
| Patient reported outcomes | School and Work Attendance |
| Disease-specific Health-related Quality of Life in Hemophilia | |
| Physical activity and function | Hemophilia-specific Functional Activity |
| Joint health Score | |
| Adherence to treatment | Patient Recall of Clotting Factor Administration |
| Utilization of health care |
Working Group Roster:
WG Members:
|
Barbara Konkle, MD, Co-Chair Bloodworks Northwest Seattle, WA |
Steven Pipe, MD, Co-Chair University of Michigan Ann Arbor, MI |
|
Tyler Buckner, MD University of Colorado Aurora, CO |
Donna DiMichele, MD National Heart, Lung, and Blood Institute Bethesda, MD |
|
Diane Nugent, MD Case Western Reserve University Cleveland, OH |
Crystal Watson, BSW American Thrombosis & Hemostasis Rochester, NY |
|
Ellen M. Werner, PhD, MA National Heart, Lung, and Blood Institute (NHLBI) Blood Diseases Branch Bethesda, MD |
Hemophilia Inhibitor Research Panel Liaison, WG Manager and WG Supervisor:
|
Lauren Wood, MS, WG Manager RTI International Research Triangle Park, NC |
Wayne Huggins, PhD, WG Supervisor RTI International Research Triangle Park, NC |
Supplemental Information includes protocols that the Working Group or Expert Review Panel considered useful and highly relevant to the domain but do not meet the criteria for inclusion in the Toolkit.
There is no supplemental information for this collection.
Additional Information:
There is no additional information for this collection.