Real-World Data on the Effectiveness and Safety of wilate for the Treatment of von Willebrand Disease

Abstract Background The efficacy and safety of wilate (human von Willebrand factor/coagulation factor VIII) in patients with von Willebrand disease (VWD) has been demonstrated in clinical trials. Here, we present real-world data on the use of wilate for the routine care of patients with VWD. Objectives The objectives of this observational, prospective, phase 4 study were to evaluate the safety, tolerability, and effectiveness of wilate in on-demand treatment of bleeding episodes (BEs), long-term prophylaxis, and surgical prophylaxis among patients with any type of VWD. Methods Patients were enrolled at 31 study centers in 11 countries and followed for up to 2 years. Safety endpoints included adverse drug reactions (ADRs) and drug tolerability. Effectiveness was assessed using annualized bleeding rates (ABRs) during prophylaxis and predefined criteria for the treatment of BEs and surgical prophylaxis. Results A total of 111 patients (76 [68%] female) including 41 (37%) children were treated with wilate. Twenty-five patients received prophylaxis, 29 on-demand treatment, and 62 surgical prophylaxis. Tolerability was rated by patients as “excellent” for 96.2% of 6,497 infusions. No unexpected ADRs or thrombotic events were reported. Median ABR during prophylaxis was 1.9. Effectiveness was assessed as “excellent” or “good” by patients and investigators for 100% of BEs treated on-demand, 98% (patient rating) and 99% (investigator rating) of breakthrough BEs, and 99% of surgical procedures (investigator rating). Conclusion wilate was safe, well tolerated, and effective for the prevention and treatment of bleeding in pediatric and adult VWD patients in a real-world setting.

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Real World Data Analysis of US Claims Database on Coagulation Factor IX Expenditures in Patients with Hemophilia B: Standard half-Life Vs. Extended Half-Life Products

Value in Health ◽

10.1016/j.jval.2018.04.1752 ◽

2018 ◽

Vol 21 ◽

pp. S262

Author(s):

D Spurden ◽

A Chhabra ◽

BJ Tortella ◽

PF Fogarty ◽

E Rubinstein ◽

...

Keyword(s):

Data Analysis ◽

Real World ◽

Half Life ◽

Coagulation Factor ◽

Factor Ix ◽

Hemophilia B ◽

Real World Data ◽

Claims Database ◽

World Data ◽

Coagulation Factor Ix

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Real-World Data Analytics Fit for Regulatory Decision-Making

American Journal of Law & Medicine ◽

10.1177/0098858818789429 ◽

2018 ◽

Vol 44 (2-3) ◽

pp. 197-217 ◽

Cited By ~ 7

Author(s):

Sebastian Schneeweiss ◽

Robert J. Glynn

Keyword(s):

Decision Making ◽

Real World ◽

Randomized Clinical Trials ◽

Routine Care ◽

Added Value ◽

Real World Data ◽

Healthcare Database ◽

World Data ◽

Medical Products ◽

Regulatory Decision

Healthcare database analyses (claims, electronic health records) have been identified by various regulatory initiatives, including the 21st Century Cures Act and Prescription Drug User Fee Act (“PDUFA”), as useful supplements to randomized clinical trials to generate evidence on the effectiveness, harm, and value of medical products in routine care. Specific applications include accelerated drug approval pathways and secondary indications for approved medical products. Such real-world data (“RWD”) analyses reflect how medical products impact health outside a highly controlled research environment. A constant stream of data from the routine operation of modern healthcare systems that can be analyzed in rapid cycles enables incremental evidence development for regulatory decision-making.Key evidentiary needs by regulators include 1) monitoring of medication performance in routine care, including the effectiveness, safety and value; 2) identifying new patient strata in which a drug may have added value or unacceptable harms; and 3) monitoring targeted utilization. Four broad requirements have been proposed to enable successful regulatory decision-making based on healthcare database analyses (collectively, “MVET”): Meaningful evidence that provides relevant and context-informed evidence sufficient for interpretation, drawing conclusions, and making decisions; valid evidence that meets scientific and technical quality standards to allow causal interpretations; expedited evidence that provides incremental evidence that is synchronized with the decision-making process; and transparent evidence that is audible, reproducible, robust, and ultimately trusted by decision-makers.Evidence generation systems that satisfy MVET requirements to a high degree will contribute to effective regulatory decision-making. Rapid-cycle analytics of healthcare databases is maturing at a time when regulatory overhaul increasingly demands such evidence. Governance, regulations, and data quality are catching up as the utility of this resource is demonstrated in multiple contexts.

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Abstract #946: Assessment of Acromegaly Patients with and Without Diabetes Treated with Lanreotide Depot: 2-Year Real World Data

Endocrine Practice ◽

10.1016/s1530-891x(20)45236-x ◽

2016 ◽

Vol 22 ◽

pp. 219

Author(s):

Roberto Salvatori ◽

Olga Gambetti ◽

Whitney Woodmansee ◽

David Cox ◽

Beloo Mirakhur ◽

...

Keyword(s):

Real World ◽

Real World Data ◽

World Data

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Evaluating a Macrocognition Model of Team Collaboration using Real-world Data from the Haiti Relief Effort

PsycEXTRA Dataset ◽

10.1037/e578902012-052 ◽

2011 ◽

Author(s):

Susan G. Hutchins

Keyword(s):

Real World ◽

Real World Data ◽

Team Collaboration ◽

World Data ◽

Relief Effort

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Author response for "Real‐world data on efficacy and safety of obinutuzumab plus chlorambucil, rituximab plus chlorambucil and rituximab plus bendamustine in the frontline treatment of chronic lymphocytic leukemia: The GO‐CLLEAR Study by the Czech CLL Study Group"

10.1002/hon.2744/v2/response1 ◽

2020 ◽

Author(s):

Anna Panovská ◽

Lucie NĚmcová ◽

Lucie Nekvindová ◽

Martin Špaček ◽

Martin Šimkovič ◽

...

Keyword(s):

Chronic Lymphocytic Leukemia ◽

Real World ◽

Lymphocytic Leukemia ◽

Author Response ◽

Study Group ◽

Efficacy And Safety ◽

Real World Data ◽

World Data ◽

Frontline Treatment

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Von Willebrand disease and Weibel-Palade bodies

Hämostaseologie ◽

10.1055/s-0037-1619048 ◽

2010 ◽

Vol 30 (03) ◽

pp. 150-155 ◽

Cited By ~ 9

Author(s):

J. W. Wang ◽

J. Eikenboom

Keyword(s):

Platelet Adhesion ◽

Coagulation Factor ◽

Von Willebrand Disease ◽

Inflammatory Factors ◽

Limited Data ◽

Storage Organelle ◽

And Function ◽

Von Willebrand ◽

Willebrand Factor

SummaryVon Willebrand factor (VWF) is a pivotal haemostatic protein mediating platelet adhesion to injured endothelium and carrying coagulation factor VIII (FVIII) in the circulation to protect it from premature clearance. Apart from the roles in haemostasis, VWF drives the formation of the endothelial cell specific Weibel-Palade bodies (WPBs), which serve as a regulated storage of VWF and other thrombotic and inflammatory factors. Defects in VWF could lead to the bleeding disorder von Willebrand disease (VWD).Extensive studies have shown that several mutations identified in VWD patients cause an intracellular retention of VWF. However, the effects of such mutations on the formation and function of its storage organelle are largely unknown. This review gives an overview on the role of VWF in WPB biogenesis and summarizes the limited data on the WPBs formed by VWD-causing mutant VWF.

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