MOTHIF II : THERAPEUTIC MANAGEMENT AND USE OF CLOTTING FACTORS IN HEMOPHILIA A & B IN FRANCE II (MOTHIF II)

Retrospective and Descriptive Analysis of the Regimens of Treatment With FVIII, FIX and By-passing Agents for Hemophiliacs A or B With or Without Inhibitors, From the Existing Registry BERHLINGO, Before and After the Launch of New Entended Half-life Clotting Factors in Seven Haemophilia Treatment Centers in France

MOTHIF II is a non-interventional, multicenter, retrospective, observational data collection in seven French Haemophilia Treatment Centers of the BERHLINGO network. In the context of the arrival of new extended half-life products, the MOTHIF II study aims to describe the changes in therapeutic management of patients with hemophilia A & B, following the provision of FVIII and FIX extended half-life factors in France; it will also permit to carry out a budget impact analysis to quantify the economic significance of this new era.

Study Overview

• Status: Completed
• Conditions: Hemophilia
• Intervention / Treatment: Other: No Interventional study

Detailed Description

Treatment regimens for haemophiliacs patients treated with clotting factors will be analysed globally for the patients of the two periods, by subgroup:

• type of hemophilia: A or B
• severity: severe, moderate, mild
• history of inhibitors

and then for each subgroup:

• by regimen: 1) Prophylaxis 2) On demand 3) ITI
• by type of clotting factors: FVIII or FIX or by-passing agents, plasma-derived or recombinant, standard or extended half-life clotting factors, INN

Will also be included in the analyse:

• demographic data: age (years)
• clinical data: weight (kgs) if available
• treatment data: regimen, consumptions (number of UI of clotting factors consumed on the period) and costs of clotting factors (€).

• Study Type: Observational
• Enrollment (Actual): 2072

Contacts and Locations

Study Locations

• France
  • Angers, France, 49933
    • Angers University Hospital
  • Brest, France, 29609
    • Brest University Hospital
  • Caen, France, 14033
    • CAEN University Hospital
  • Le Mans, France, 72037
    • Le Mans University Hospital
  • Nantes, France, 44093
    • Nantes University Hospital
  • Rennes, France, 35033
    • Rennes University Hospital
  • Tours, France, 37044
    • Tours University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Estimation of the population of the study: 2 000 patients will be enrolled by seven centers in France (treated or not).

Settings : 7 Hemophilia Traitement Center of Western France : Angers, Brest, Caen, Le Mans, Nantes, Rennes and Tours

Description

Inclusion Criteria:

• All patients hemophiliac A or B, with or without inhibitors, included in the Nhemo (7 centres) and the Behrlingo database, treated with factor 8 or factor 9 products or by-passing agents
• All Hemophilia A or B patients of any severity without inhibitors, included in the NHEMO database and the BEHRLINGO database, without any treatment by clotting factors
• All Hemophilia A or B patients having given consent to be included in the study (2 times 12 months), during their usual follow-up (consultation…). For the other patients, a derogation from the obligation to inform the patients will be made to the CNIL.

Exclusion Criteria:

• patients under guardianship

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
A comparison of the consumptions of CF before and after the laucnh of EHL CF is an assessment of the the impact of EHL CF on the treatment of haemophiliacs patients	Deletion of the measure of % of switch	24 months (1st July 2015- 30th June 2016 & 1st July 2017-30th June 2018 (before and after the launch of extended half-life CF in France))

Collaborators and Investigators

• Sponsor: Nantes University Hospital

Study record dates

Study Major Dates

• Study Start (ACTUAL): November 13, 2019
• Primary Completion (ACTUAL): December 31, 2019
• Study Completion (ACTUAL): December 31, 2019

Study Registration Dates

• First Submitted: March 22, 2018
• First Submitted That Met QC Criteria: July 13, 2018
• First Posted (ACTUAL): July 24, 2018

Study Record Updates

• Last Update Posted (ACTUAL): May 18, 2020
• Last Update Submitted That Met QC Criteria: May 15, 2020
• Last Verified: May 1, 2020

More Information

Terms related to this study

• Keywords: Hemophilia B; Hemophilia A; Research database; Clotting Factors; Regimen of Treatment; Impact Budget Analysis
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: RC17_0288

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No