Ignite Creation Date:
2024-05-06 @ 12:54 PM
Last Modification Date:
2024-10-26 @ 1:05 PM
Study NCT ID:
NCT03875924
Status:
COMPLETED
Last Update Posted:
2020-05-19
First Post:
2019-03-13
Brief Title:
REAL-LIFE DATA OF CONSTITUTIONAL VON WILLEBRAND DISEASE IN WESTERN FRANCE HOPSCOTcH-WILL
Sponsor:
Nantes University Hospital
Organization:
Nantes University Hospital
Study Overview
Official Title:
Multicenter Retrospective Study From 5 Hemostasis Treatment Centers in Western France Severe Hemorrhagic Treated Occurrences Patterns and Global Substitutive COagulation Factors THerapy in the Inherited Von WILLebrand Disease
Status:
COMPLETED
Status Verified Date:
2020-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Von Willebrand disease VWD is the most common constitutional bleeding disorder in the world caused by missing or defective von Willebrand factor VWF In France VWD affects approximatively 7000 patients
There are many types of VWD The severest forms are characterized by the occurrence of extremely serious bleedings requiring in-stays with clotting factors CF treatments in specialized hospital units andor an ambulatory substitutive therapy both of them are highly expensive
In France Hemostasis Treatment Centers HTC have the opportunity to record these kinds of data in a database called NHEMO Net-Hemostasis care database for constitutional bleeding disorders Further ahead the data can be coded dumped into and extracted from the research database BERHLINGO and analyzed
The HOPSCOTcH-WILL study will be a retrospective non-interventional multicenter national cohort study will provide an overview of the real-life management of patients with VWD in western France requiring a substitutive treatment with VWF as well as a description of the characteristics of their hemorrhagic events
Model Observationnal real world evidence study Time Horizon 2015-2018 HTC France Western University Hospitals BERHLINGO network Nantes University Hospital promotion Angers University Hospital Brest University Hospital Le Mans Regional Hospital Rennes University Hospital
There are many types of VWD The severest forms are characterized by the occurrence of extremely serious bleedings requiring in-stays with clotting factors CF treatments in specialized hospital units andor an ambulatory substitutive therapy both of them are highly expensive
In France Hemostasis Treatment Centers HTC have the opportunity to record these kinds of data in a database called NHEMO Net-Hemostasis care database for constitutional bleeding disorders Further ahead the data can be coded dumped into and extracted from the research database BERHLINGO and analyzed
The HOPSCOTcH-WILL study will be a retrospective non-interventional multicenter national cohort study will provide an overview of the real-life management of patients with VWD in western France requiring a substitutive treatment with VWF as well as a description of the characteristics of their hemorrhagic events
Model Observationnal real world evidence study Time Horizon 2015-2018 HTC France Western University Hospitals BERHLINGO network Nantes University Hospital promotion Angers University Hospital Brest University Hospital Le Mans Regional Hospital Rennes University Hospital
Detailed Description:
von Willebrand Disease vWD represents the most common autosomal inherited bleeding disorders with a prevalence up to 1 nevertheless the clinical relevant patients represent only 001 of the population
vWD is caused by a deficiency andor abnormality of von Willebrand factor VWF inhibiting a satisfactory hemostasis vWD is usually classified into three main types according to quantitative Types 1 and 3 or qualitative Types 2A 2B 2M 2N deficiencies Type 3 patients have the highest bleeding risk type 2 patients are intermediate and type 1 patients have the lowest bleeding risk Beyond the typology VWF levels and therefore the hemorrhagic exposition are also notably influenced by physiological age exercise pregnancy gene mutations or pathological inflammation and cancer variables Furthermore blood group plays a major role in plasma VWF levels in fact they are 25-35 lower in type-0 individuals than in non-0 As a result the clinical and biological identification of vWD can be extremely challenging and may lead to a delay in the diagnosis
In vWD the clinical manifestations are essentially mucosal bleeding symptoms Epistaxis menorrhagia easy bruising gingival bleeding are flagrant manifestations of the disease and despite the mostly small somatic impact in the majority of patients they may significantly affect the quality of life Nevertheless severe affected patients may face upper consequences gastrointestinal GI bleeding may be particularly frequent and difficult to manage and they are as well at high risk to develop major and life-threatening bleeds after surgical procedures even after minor ones such as tooth extraction
To guarantee the best medical care management VWD patients are followed in specialized rare disease centers both for the ambulatory follow-up and the hospitalizations outpatient aftercare These hospital structures are the only ones authorized to prescribe and dispense in- outpatients substitutive treatments
Indeed the optimal effective treatment of the disorder is the replacement of VWF by using VWF concentrate obtained by fractionation of human plasma in emergency recombinant or plasma-derived factor VIII FVIII can be added to the VWF In case of history of inhibitors high dose of FVIII or by-passing agents can also be used The current treatment for vWD involves intravenous injections of VWF that are either given on demand in response to a bleeding event or as a prophylactic therapy that is administered 2 to 4 times a week Numerous studies have shown the efficacy of prophylactic therapy in severe patients but the investigators lack data about surgery procedures and perioperative management Moreover regardless of the small amounts of patients those treatments remain quite costly and may represent an economic burden for the Assurance Health System
Due to the future availability of the first recombinant VWF the investigators need a better understanding of the current French practices The lack of literature on the topic is undeniably blatant and the few relevant papers concerned specific pd-VWF concentrates no RWD publication has been identified for the specific French cohort The heterogeneity of VWD concentrates brands and VWD types make the development of personalized care quite difficult in this context and more information is needed especially for the peri-operative treatment The economic evaluations usually focus on prophylaxis treatments or a specific concentrate neglecting a significant percentage of patients
The objective of our 4-years retrospective study HOPSCOTcH-WILL is to provide an accurate and detailed account of current vWD therapeutic management by collecting real-life data on hemorrhagic treated events in 5 Western French HTC Angers Brest Le Mans Nantes Rennes The data will be retrospectively recorded during 48 months from 2015 to 2018 for patients followed in the 5 HTC involved in the study A focus will be done on GI episodes and surgeries which are the most at risk hemorrhagic events in vWD
HOPSCOTcH-WILL could be considered as reliable photography of Western French HTC at a particular time Therefore and especially with the launch of a new recombinant VWF it is particularly important to get a clear state-of-the-art and to assess the economic impact for the French Health System
vWD is caused by a deficiency andor abnormality of von Willebrand factor VWF inhibiting a satisfactory hemostasis vWD is usually classified into three main types according to quantitative Types 1 and 3 or qualitative Types 2A 2B 2M 2N deficiencies Type 3 patients have the highest bleeding risk type 2 patients are intermediate and type 1 patients have the lowest bleeding risk Beyond the typology VWF levels and therefore the hemorrhagic exposition are also notably influenced by physiological age exercise pregnancy gene mutations or pathological inflammation and cancer variables Furthermore blood group plays a major role in plasma VWF levels in fact they are 25-35 lower in type-0 individuals than in non-0 As a result the clinical and biological identification of vWD can be extremely challenging and may lead to a delay in the diagnosis
In vWD the clinical manifestations are essentially mucosal bleeding symptoms Epistaxis menorrhagia easy bruising gingival bleeding are flagrant manifestations of the disease and despite the mostly small somatic impact in the majority of patients they may significantly affect the quality of life Nevertheless severe affected patients may face upper consequences gastrointestinal GI bleeding may be particularly frequent and difficult to manage and they are as well at high risk to develop major and life-threatening bleeds after surgical procedures even after minor ones such as tooth extraction
To guarantee the best medical care management VWD patients are followed in specialized rare disease centers both for the ambulatory follow-up and the hospitalizations outpatient aftercare These hospital structures are the only ones authorized to prescribe and dispense in- outpatients substitutive treatments
Indeed the optimal effective treatment of the disorder is the replacement of VWF by using VWF concentrate obtained by fractionation of human plasma in emergency recombinant or plasma-derived factor VIII FVIII can be added to the VWF In case of history of inhibitors high dose of FVIII or by-passing agents can also be used The current treatment for vWD involves intravenous injections of VWF that are either given on demand in response to a bleeding event or as a prophylactic therapy that is administered 2 to 4 times a week Numerous studies have shown the efficacy of prophylactic therapy in severe patients but the investigators lack data about surgery procedures and perioperative management Moreover regardless of the small amounts of patients those treatments remain quite costly and may represent an economic burden for the Assurance Health System
Due to the future availability of the first recombinant VWF the investigators need a better understanding of the current French practices The lack of literature on the topic is undeniably blatant and the few relevant papers concerned specific pd-VWF concentrates no RWD publication has been identified for the specific French cohort The heterogeneity of VWD concentrates brands and VWD types make the development of personalized care quite difficult in this context and more information is needed especially for the peri-operative treatment The economic evaluations usually focus on prophylaxis treatments or a specific concentrate neglecting a significant percentage of patients
The objective of our 4-years retrospective study HOPSCOTcH-WILL is to provide an accurate and detailed account of current vWD therapeutic management by collecting real-life data on hemorrhagic treated events in 5 Western French HTC Angers Brest Le Mans Nantes Rennes The data will be retrospectively recorded during 48 months from 2015 to 2018 for patients followed in the 5 HTC involved in the study A focus will be done on GI episodes and surgeries which are the most at risk hemorrhagic events in vWD
HOPSCOTcH-WILL could be considered as reliable photography of Western French HTC at a particular time Therefore and especially with the launch of a new recombinant VWF it is particularly important to get a clear state-of-the-art and to assess the economic impact for the French Health System
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None