Clinical trial NCT01959555: Project to Update the Study of Congenital Haemophilia in Spain
Official title: Project to Update the Study of Congenital Haemophilia in Spain
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Congenital Bleeding Disorder
Haemophilia A
Haemophilia B
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2013-10-10
Enrollment: 1157
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients in active follow-up in HTCs (Haemophilia Treatment Centres) will be included in this study. A patient is considered as active if a visit to the HTC is performed in the period of January 2013 to December 2013.

Brief summary: This study is conducted in Europe. The aim of the study is to update the knowledge about the prevalence and severity of congenital haemophilia (A and B) in Spain.


Eligibility criteria: Inclusion Criteria:

* Informed consent obtained before any study-related activities. (Study-related activities are any procedure related to recording of data according to the protocol)
* Male patients of any age diagnosed with congenital haemophilia A or B who visited their HTC at least once during the period of January 2013 to December 2013

Exclusion Criteria:

* Previous participation in this study
* Female patients
* Acquired haemophilia

Primary outcomes:
measure: Prevalence of congenital haemophilia A and B in Spain in 2013, described as proportion (%) of patients in the total population (extracted from the last available census of the Spanish population for the regions considered for the study)
timeFrame: Assessed at study visit (day 1)
measure: Severity (mild, moderate or severe) of congenital haemophilia A and B in Spain during 2013, described as proportion (%) per category of the total number of patients with haemophilia A or B
timeFrame: Assessed at study visit (day 1)
Secondary outcomes:
measure: Mean current age at the time of visit to the HTC in 2013
timeFrame: Assessed at study visit (day 1)
measure: Mean age at diagnosis
timeFrame: Assessed at study visit (day 1)
measure: Proportion (%) of patients with/without genetic screening performed
timeFrame: Assessed at study visit (day 1)
measure: Proportion (%) of patients who needed support of a reference Haemophilia Healthcare Centre in 2013
timeFrame: Assessed at study visit (day 1)
measure: Proportion (%) of patients who received plasmatic or recombinant treatment
timeFrame: Assessed at study visit (day 1)
measure: Proportion (%) of patients who received on demand or prophylaxis treatment
timeFrame: Assessed at study visit (day 1)
measure: Mean number of bleeding episodes
timeFrame: Assessed at study visit (day 1)
measure: Mean number of joint bleeds
timeFrame: Assessed at study visit (day 1)
measure: Proportion (%) of patients with established arthropathy
timeFrame: Assessed at study visit (day 1)
measure: Proportion (%) of patients with infectious complications
timeFrame: Assessed at study visit (day 1)
measure: Proportion (%) of patients with HIV (Human immunodeficiency virus)
timeFrame: Assessed at study visit (day 1)
measure: Proportion (%) of patients with presence of inhibitors
timeFrame: Assessed at study visit (day 1)
measure: Proportion (%) of patients with immune tolerance induction treatment performed
timeFrame: Assessed at study visit (day 1)
measure: Total number of surgery interventions performed
timeFrame: ThAssessed at study visit (day 1)
measure: Proportion (%) of patients with quality of life questionnaire (QoLQ) performed in 2013
timeFrame: Assessed at study visit (day 1)
measure: Mean number of days of absenteeism to school/university in children and adolescents in 2013
timeFrame: Assessed at study visit (day 1)
measure: Mean number of days of absenteeism from work in 2013
timeFrame: Assessed at study visit (day 1)
measure: Proportion (%) of patients with treatment administered at home
timeFrame: Assessed at study visit (day 1)
Other outcomes:
Locations:
city: Madrid
country: Spain
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References: