scholarly journals The impact of extended half‐life factor concentrates on patient reported health outcome measures in persons with hemophilia A and hemophilia B

2021 ◽  
Vol 5 (7) ◽  
Author(s):  
Haowei (Linda) Sun ◽  
Ming Yang ◽  
Man‐Chiu Poon ◽  
Adrienne Lee ◽  
K. Sue Robinson ◽  
...  
Keyword(s):  
Health Outcome ◽  
Outcome Measures ◽  
Half Life ◽  
Hemophilia A ◽  
Hemophilia B ◽  
Patient Reported ◽  
Reported Health ◽  
The Impact ◽  
Life Factor

scholarly journals Analysis of US Claims Real World Data of Hemophilia a & B Patients: Units Dispensed & Expenditures Associated with Utilization of and Switching from Standard to Extended Half-Life Factor Products

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 3508-3508
Author(s):  
Bartholomew J. Tortella ◽  
Amit Chhabra ◽  
José Alvir ◽  
Emily R. Rubinstein ◽  
Lisa J. Young ◽  
...  
Keyword(s):  
Real World ◽  
Half Life ◽  
Hemophilia A ◽  
Real World Data ◽  
World Data ◽  
Treatment Scheme ◽  
Before And After ◽  
Male Patients ◽  
The Impact ◽  
Life Factor

Abstract Background: Both standard half-life (SHL) and extended half-life (EHL) factor replacement products are used to treat patients with hemophilia A (hem-A) and B (hem-B). A comparison of international unit (IU) utilization and expenditures ($USD) was made between patients on SHL and EHL factor replacement products to compare utilization and to determine the impact of switching from an SHL to an EHL product. Methods: De-identified claims data from both the Truven Health MarketScan® Research (Truven) and Optum® Clinformatics® (Optum) US claims databases included male patients with hem-A (Truven from Aug 2014-Apr 2018 and Optum from Aug 2014-Dec 2017) and hem-B (Truven from Jun 2014-Apr 2018 and Optum from Jul 2014-Dec 2017) who had data for at least 3 months of product dispensation. The SHL and EHL groups were compared. A separate "switch" analysis using the Truven database examined expenditures and IUs before and after switching from nonacog-α (SHL) to FIX-Fc (EHL). Descriptive statistics were used and medians reported for expenditures and IUs to accommodate for the skewness of data distribution. Results: Hem-A: The analysis included 896 SHL and 202 EHL patients, including those who switched from an SHL to an EHL product. Quarterly expenditures and IUs dispensed were analyzed. Both the Optum and Truven databases (see Table) demonstrated higher IU dispensation (Optum: 35%, Truven: 50% higher) and expenditures (Optum: 87%, Truven: 117%) associated with EHL products versus SHL products. In the switch analysis, 35 patients switched from nonacog-α to FIX-Fc; median IUs rose from 61,228 to 75,914 [24%] and median expenditures rose from $78,945 to $155,203 [97%]. Hem-B: The analysis included 50 FIX-Fc, 13 FIX-Alb, and 132 nonacog-α patients. Both databases (see Table) demonstrated higher median expenditures for the EHL products compared with nonacog-α (Optum: 179% for FIX-Fc, 189% for FIX-Alb; Truven: 234% for FIX-Fc, 245% for FIX-Alb). The IU results varied in Optum: FIX-Fc was 23% higher than nonacog-α, but FIX-Alb was 9 % lower than nonacog-α. Similarly, in Truven, the IU results varied: IUs were mixed for the SHL product compared with the EHL products: 62% higher for FIX-Fc than nonacog-α, and approximately the same for FIX-Alb and nonacog-α. In the switch analysis, 16 patients switched from nonacog-α to FIX-Fc; median IUs rose from 62,857 (nonacog-α) to 69,816 (FIX-Fc) [11%], while expenditures rose from $75,064 (nonacog-a) to $210,482 (FIX-Fc) [180%]. Conclusions: This analysis in more than 1000 patients, unadjusted for severity of disease or treatment scheme, demonstrated in hem-A that higher IU utilization and expenditures were associated with EHL use compared with SHL use, and in hem-B, that higher expenditures were seen with EHL products, while IU dispensation in some cases decreased, remained the same, or increased. In patients switching from the SHL to an EHL product, higher IU dispensation and expenditures were seen. These real-world data may challenge assumptions regarding typical factor usage and expenditures associated with EHL products in patients with hem-A and hem-B. Additional analyses with adjustment for treatment scheme and disease severity are needed. Table. Table. Disclosures Tortella: Pfizer Inc.: Employment. Chhabra:Pfizer Inc.: Employment. Alvir:Pfizer Inc.: Employment. Rubinstein:Pfizer Inc.: Employment. Young:Pfizer Ltd.: Employment. Fogarty:Pfizer Inc.: Employment.

scholarly journals Creativity in Public Involvement: Promoting Meaningful Collaboration in Health Outcome Measures Research

2020 ◽  
Author(s):  
Katherine Broomfield ◽  
Claire Criag ◽  
Sarah Smith ◽  
Georgina Jones ◽  
Simon Judge ◽  
...  
Keyword(s):  
Health Outcome ◽  
Outcome Measures ◽  
Public Involvement ◽  
Development Project ◽  
Research Project ◽  
Creative Methods ◽  
Patient Reported ◽  
Communication Difficulties ◽  
Group Members ◽  
The Impact

Abstract Background: The role of public involvement (PI) in healthcare research is growing in importance and it is imperative that researchers continuously reflect on how to promote the inclusion of patients and service users in the design and delivery of research. PI offers a mechanism for end-users to be involved planning, executing, and reporting research that develops health outcome measures. Some patient groups, including people who have communication difficulties, may struggle to engage in the methods traditionally employed to promote PI engagement such as questionnaires and focus groups. Methods: This article describes a longitudinal case-study of a PI group, consisting of people who have communication difficulties, for a patient-reported outcome development project. Creative methods, informed by the participatory design principles of enacting, seeing and doing, were introduced stepwise into seven PI meetings. Data from video and visual minutes were used to evaluate the impact of the methods, following each group. Feedback, in the form of verbal and visual outputs taken directly from group meeting minutes, along with vignettes evidenced the impact of the methods on the project and group members.Results: Creative methods enabled the PI group members to meaningfully contribute in meetings, to interact dynamically and to engage with the aims and processes of the research project. Their involvement facilitated the development of accessible recruitment materials, informed data analysis and supported the dissemination of project outputs. Employing creative methods also enabled both PI group members and the academic team to reflect on their own roles within the research project and the impact that involvement in the PI group has had on their personal development and perspectives on research.Conclusion: The impact of using creative methods in PI for this PROM development project improved collaboration and understanding between PI members and the academic team. The meaningful engagement of people who have communication difficulties in PI generated a more accessible project in terms of both process and impact. Creativity has applicability beyond people whose communication is non-verbal; it should be harnessed by research teams to identify and breakdown barriers to involvement to develop outcome tools that reflect the diversity of our populations.

scholarly journals Real World Factor Dispensation and Expenditures in us Patients with Hemophilia B: Standard Half-Life vs. Extended Half-Life Factor IX Replacement Products

2018 ◽  
Vol 21 ◽  
pp. S111
Author(s):  
A Chhabra ◽  
D Spurden ◽  
BJ Tortella ◽  
PF Fogarty ◽  
A Pleil ◽  
...  
Keyword(s):  
Real World ◽  
Half Life ◽  
Factor Ix ◽  
Hemophilia B ◽  
Life Factor

scholarly journals In silico evaluation of limited sampling strategies for individualized dosing of extended half-life factor IX concentrates in hemophilia B patients

2021 ◽  
Author(s):  
T. Preijers ◽  
M. W. F. van Spengler ◽  
K. Meijer ◽  
K. Fijnvandraat ◽  
K. Fischer ◽  
...  
Keyword(s):  
In Silico ◽  
Half Life ◽  
Factor Ix ◽  
Hemophilia B ◽  
Weekly Dose ◽  
Sampling Strategies ◽  
Time Profiles ◽  
Limited Sampling ◽  
Individualized Dosing ◽  
Life Factor

Abstract Purpose Hemophilia B is a bleeding disorder, caused by a factor IX (FIX) deficiency. Recently, FIX concentrates with extended half-life (EHL) have become available. Prophylactic dosing of EHL-FIX concentrates can be optimized by assessment of individual pharmacokinetic (PK) parameters. To determine these parameters, limited sampling strategies (LSSs) may be applied. The study aims to establish adequate LSSs for estimating individual PK parameters of EHL-FIX concentrates using in silico evaluation. Methods Monte Carlo simulations were performed to obtain FIX activity versus time profiles using published population PK models for N9-GP (Refixia), rFIXFc (Alprolix), and rIX-FP (Idelvion). Fourteen LSSs, containing three or four samples taken within 8 days after administration, were formulated. Bayesian analysis was applied to obtain estimates for clearance (CL), half-life (t1/2), time to 1% (Time1%), and calculated weekly dose (Dose1%). Bias and precision of these estimates were assessed to determine which LSS was adequate. Results For all PK parameters of N9-GP, rFIXFc and rIX-FP bias was generally acceptable (range: −5% to 5%). For N9-GP, precision of all parameters for all LSSs was acceptable (< 25%). For rFIXFc, precision was acceptable for CL and Time1%, except for t1/2 (range: 27.1% to 44.7%) and Dose1% (range: 12% to 29.4%). For rIX-FP, all LSSs showed acceptable bias and precision, except for Dose1% using LSS with the last sample taken on day 3 (LSS 6 and 10). Conclusion Best performing LSSs were LSS with samples taken at days 1, 5, 7, and 8 (N9-GP and rFIXFc) and at days 1, 4, 6, and 8 (rIX-FP), respectively.

A new algorithm to build bridges between two patient-reported health outcome instruments: the MOS SF-36® and the VR-12 Health Survey

2018 ◽  
Vol 27 (8) ◽  
pp. 2195-2206 ◽  
Author(s):  
Alfredo Selim ◽  
William Rogers ◽  
Shirley Qian ◽  
James A. Rothendler ◽  
Erin E. Kent ◽  
...  
Keyword(s):  
Health Outcome ◽  
Health Survey ◽  
Sf 36 ◽  
Patient Reported ◽  
Reported Health

scholarly journals The Impact of Patient Interactive Systems on the Management of Pain in an Inpatient Hospital Setting: A Systematic Review

2019 ◽  
Vol 10 (04) ◽  
pp. 580-596 ◽  
Author(s):  
Raniah N. Aldekhyyel ◽  
Caitlin J. Bakker ◽  
Michael B. Pitt ◽  
Genevieve B. Melton
Keyword(s):  
Pain Management ◽  
Outcome Measures ◽  
User Satisfaction ◽  
Hospital Setting ◽  
Interactive Systems ◽  
Management Systems ◽  
Interactive System ◽  
Inpatient Setting ◽  
Patient Reported ◽  
The Impact

Background While some published literature exists on the use of interactive patient care systems, the effectiveness of these systems on the management of pain is unclear. To fill this gap in knowledge, we aimed to understand the impact and outcomes of pain management patient interactive systems in an inpatient setting. Methods A systematic literature review was conducted across seven databases, and results were independently screened by two researchers. To extract relevant data, critical appraisal forms were developed and each paper was examined by two experts. Information included patient interactive system category, patient population and number of participants/samples, experiment type, and specific outcome measures. Results Out of 58 full-text articles assessed for eligibility, 18 were eligible and included in the final qualitative synthesis. Overall, there were two main types of pain management interactive systems within the inpatient setting (standalone systems and integrated platform systems). While systems were diverse especially for integrated platforms, most reported systems were entertainment distraction systems. Reports examined a variety of outcome measures, including changes in patient-reported pain levels, patient engagement, user satisfaction, changes in clinical workflow, and changes in documentation. In the 13 systems measuring pain scores, 12 demonstrated a positive impact on pain level scores. Conclusion Pain management systems appear to be effective in lowering patient level scores, but research comparing the effectiveness and efficacy of one type of interactive system versus another in the management of pain is needed. While not conclusive, pain management systems integrated with other technology platforms show potentially promising effects with improving patient communication, education, and self-reporting.

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