Project to Update the Study of Congenital Haemophilia in Spain (PUCHS)

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.

Study Overview

• Status: Completed
• Conditions: Congenital Bleeding Disorder; Haemophilia A; Haemophilia B
• Intervention / Treatment: Other: No treatment given

• Study Type: Observational
• Enrollment (Actual): 1157

Contacts and Locations

Study Locations

• Spain
  • Madrid, Spain, 28033

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• 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.

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Retrospective collection of data	Other: No treatment given; Patients will not be treated with any specific product. Data collection only.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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)	Assessed at study visit (day 1)
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	Assessed at study visit (day 1)

Secondary Outcome Measures

Outcome Measure	Time Frame
Mean current age at the time of visit to the HTC in 2013	Assessed at study visit (day 1)
Mean age at diagnosis	Assessed at study visit (day 1)
Proportion (%) of patients with/without genetic screening performed	Assessed at study visit (day 1)
Proportion (%) of patients who needed support of a reference Haemophilia Healthcare Centre in 2013	Assessed at study visit (day 1)
Proportion (%) of patients who received plasmatic or recombinant treatment	Assessed at study visit (day 1)
Proportion (%) of patients who received on demand or prophylaxis treatment	Assessed at study visit (day 1)
Mean number of bleeding episodes	Assessed at study visit (day 1)
Mean number of joint bleeds	Assessed at study visit (day 1)
Proportion (%) of patients with established arthropathy	Assessed at study visit (day 1)
Proportion (%) of patients with infectious complications	Assessed at study visit (day 1)
Proportion (%) of patients with HIV (Human immunodeficiency virus)	Assessed at study visit (day 1)
Proportion (%) of patients with presence of inhibitors	Assessed at study visit (day 1)
Proportion (%) of patients with immune tolerance induction treatment performed	Assessed at study visit (day 1)
Total number of surgery interventions performed	ThAssessed at study visit (day 1)
Proportion (%) of patients with quality of life questionnaire (QoLQ) performed in 2013	Assessed at study visit (day 1)
Mean number of days of absenteeism to school/university in children and adolescents in 2013	Assessed at study visit (day 1)
Mean number of days of absenteeism from work in 2013	Assessed at study visit (day 1)
Proportion (%) of patients with treatment administered at home	Assessed at study visit (day 1)

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S

Publications and helpful links

Helpful Links

• Clinical Trials at Novo Nordisk

Study record dates

Study Major Dates

• Study Start: March 1, 2014
• Primary Completion (Actual): December 1, 2014
• Study Completion (Actual): December 1, 2014

Study Registration Dates

• First Submitted: October 8, 2013
• First Submitted That Met QC Criteria: October 8, 2013
• First Posted (Estimate): October 10, 2013

Study Record Updates

• Last Update Posted (Estimate): November 30, 2015
• Last Update Submitted That Met QC Criteria: November 27, 2015
• Last Verified: November 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Hemostatic Disorders; Hemophilia A; Hemophilia B; Blood Coagulation Disorders
• Other Study ID Numbers: HAEM-4076; U1111-1136-7052 (Other Identifier: WHO); NOV-HEM-2013-01 (Other Identifier: Novo Nordisk)