Quality of Life in Patients With Congenital Afibrinogenemia

NCT03484065 | Completed

• 1 other identifier: 2016-00447
• Study Type: observational
• Target: 250
• Locations: 20 countries
• Sites: 25

Brief Summary

The aim of this observational study is to evaluate the quality of life in patients with congenital afibrinogenemia using the Haemo-QoL SF for kids and the Haem-A-QoL for adult patients.

Conditions

Afibrinogenemia, Congenital

Keywords

Congenital fibrinogen disorders

Outcome Measures

Primary Outcomes (1)

• The influence of the afibrinogenemia on the patients' quality of life assessed by the Haemo-QoL SF questionnaire (for children) and the Haem-A-QoL questionnaire (for adult)

  The quality of life questionnaire includes item assessing: * Physical health * Feeling * View * Family * Friends * Others * Sport and school * Treatment * Perceived support * Dealing * Future * Relationship

  At inclusion

Secondary Outcomes (1)

• Impact of the afibrinogenemic patient's clinical phenotype on the patients' quality of life

  At Inclusion

Study Arms (1)

Afibrinogenemia

Other: Questionnaire quality of life

Interventions

Questionnaire quality of life OTHER

Afibrinogenemia

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients suffering from congenital afibrinogenemia

You may qualify if:

• Congenital afibrinogenemia confirmed by biology (absence of circulating fibrinogen) and genotype

You may not qualify if:

• Lack of participant's consent
• Patient unable to understand the questionnaire

Sponsors & Collaborators

• University Hospital, Geneva: lead

Study Sites (28)

Children's Hospital of Orange Count

Orange, California, 92868, United States

Location

Cohen Children's Medical Center

New Hyde Park, New York, 11040, United States

Location

Béni Messous

Algiers, Algeria

Location

University of Calgary

Calgary, Canada

Location

Cairo University Pediatric Hospital

Cairo, Egypt

Location

Chru Lille

Lille, France

Location

Universitats Kilinikum Frankfurt

Frankfurt, Germany

Location

Dr von Haumer Children's Hospital

Munich, Germany

Location

St John Medical College Hospital

Bangalore, India

Location

All India Institute of Medical Sciences

New Delhi, India

Location

Sapienza Università di Roma

Roma, Italy

Location

University School of Medicine

Hamamatsu, Japan

Location

Kuwait University

Kuwait City, Kuwait

Location

Hotel Dieu-de-France

Beirut, Lebanon

Location

St George Hospital

Beirut, Lebanon

Location

Hopital d'Enfants de Rabat

Rabat, Morocco

Location

Radboud University Medical Centre

Nijmegen, Netherlands

Location

National Institute Of Blood Disease and Bone Marrow Transplantation

Karachi, Pakistan

Location

Institute of Hematology and Transfusion Medicine

Warsaw, Poland

Location

University Clinical Center

Belgrade, Serbia

Location

National Centre of Hemostasis and Thrombosis

Martin, Slovakia

Location

Hospital Universitari i Politecnic La Fe

Valencia, Spain

Location

Inselspital

Bern, Switzerland

Location

University Hospitals of Geneva

Geneva, Switzerland

Location

Hopital d'Enfants Bechir Hamza

Tunis, Tunisia

Location

Uludag University

Bursa, Turkey (Türkiye)

Location

Cerrahpasa Faculty of Medicine

Istanbul, Turkey (Türkiye)

Location

Erciyes University

Kayseri, Turkey (Türkiye)

Location

Related Publications (1)

• Casini A, von Mackensen S, Santoro C, Djambas Khayat C, Belhani M, Ross C, Dorgalaleh A, Naz A, Unal E, Abdelwahab M, Lozeron ED, Trillot N, Susen S, Peyvandi F, de Moerloose P; QualyAfib Study Group. Clinical phenotype, fibrinogen supplementation, and health-related quality of life in patients with afibrinogenemia. Blood. 2021 Jun 3;137(22):3127-3136. doi: 10.1182/blood.2020009472.

  PMID: 33512441 DERIVED

MeSH Terms

Conditions

Afibrinogenemia

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Alessandro Casini, MD

  University Hospitals of Geneva

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: March 14, 2018
• First Posted: March 30, 2018
• Study Start: June 1, 2016
• Primary Completion: July 1, 2019
• Study Completion: June 1, 2020
• Last Updated: November 12, 2020

Record last verified: 2020-11

Data Sharing

• IPD Sharing: Will not share

Locations

KU (1); CA (1); NL (1); SE (1); US (2); PL (1); TU (1); FR (1); AL (1); SL (1); LE (2); ES (1); EG (1); CH (2); IT (1); JP (1); DE (2); PA (1); IN (2); MO (1)