Clinical trial NCT03484065: Quality of Life in Patients With Congenital Afibrinogenemia
Official title: Quality of Life in Patients With Congenital Afibrinogenemia
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Afibrinogenemia, Congenital
Keywords: Congenital fibrinogen disorders
Lead sponsor: University Hospital, Geneva
Sponsor class: OTHER
First posted: 2018-03-30
Enrollment: 250
Sex: ALL
Minimum age (years): 8
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients suffering from congenital afibrinogenemia

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.

Detailed description: In this observational study, children and adults suffering from congenital afibrinogenemia confirmed by biology will be enrolled. All patients will receive a questionnaire on quality of life in their own language during a routine visit and filled out by the patient at home. A general questionnaire will be filled out by the patient's physician.

Eligibility criteria: Inclusion Criteria:

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

Exclusion Criteria:

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

Primary outcomes:
measure: 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)
timeFrame: At inclusion
Secondary outcomes:
measure: Impact of the afibrinogenemic patient's clinical phenotype on the patients' quality of life
timeFrame: At Inclusion
Other outcomes:
Locations:
city: Orange
country: United States
facility: Children's Hospital of Orange Count
state: California
city: New Hyde Park
country: United States
facility: Cohen Children's Medical Center
state: New York
city: Algier
country: Algeria
facility: Béni Messous
city: Calgary
country: Canada
facility: University of Calgary
city: Cairo
country: Egypt
facility: Cairo University Pediatric Hospital
city: Lille
country: France
facility: Chru Lille
city: Frankfurt
country: Germany
facility: Universitats Kilinikum Frankfurt
city: Munich
country: Germany
facility: Dr von Haumer Children's Hospital
city: Bangalore
country: India
facility: St John Medical College Hospital
city: New Delhi
country: India
facility: All India Institute of Medical Sciences
city: Roma
country: Italy
facility: Sapienza Università di Roma
city: Hamamatsu
country: Japan
facility: University School of Medicine
city: Kuwait
country: Kuwait
facility: Kuwait University
city: Beirut
country: Lebanon
facility: Hotel Dieu-de-France
city: Beirut
country: Lebanon
facility: St George Hospital
city: Rabat
country: Morocco
facility: Hopital d'Enfants de Rabat
city: Nijmegen
country: Netherlands
facility: Radboud University Medical Centre
city: Karachi
country: Pakistan
facility: National Institute Of Blood Disease and Bone Marrow Transplantation
city: Warsaw
country: Poland
facility: Institute of Hematology and Transfusion Medicine
city: Belgrade
country: Serbia
facility: University Clinical Center
city: Martin
country: Slovakia
facility: National Centre of Hemostasis and Thrombosis
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe
city: Bern
country: Switzerland
facility: Inselspital
city: Geneva
country: Switzerland
facility: University Hospitals of Geneva
city: Tunis
country: Tunisia
facility: Hopital d'Enfants Bechir Hamza
city: Bursa
country: Turkey
facility: Uludag University
city: Istanbul
country: Turkey
facility: Cerrahpasa Faculty of Medicine
city: Kayseri
country: Turkey
facility: Erciyes University
Study officials:
affiliation: University Hospitals of Geneva
name: Alessandro Casini, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.