Quality of Care of Children With Sickle Cell Disease (SCD) Screened at Birth in France

NCT03119922 | Completed

• 1 other identifier: NI 12001
• Study Type: observational
• Target: 1,750
• Locations: 0 countries
• Sites: N/A

Brief Summary

The main objective of this study is to evaluate at the national level (France)

• the early healthcare practices for children with sickle cell disease screened at birth,
• the adequacy of theses practices with the national recommendations,
• their variability over time and according the characteristics of treatment centers. Will be studied in particular the diffusion of the latest preventive measures (practice of trans-cranial Doppler and pneumococcal conjugate vaccine) and their link with the residual risks of death, stroke and invasive pneumococcal infections. The study includes all patients born in France between 01/01/2006 and 31/12/2010. Events are recorded and analysed only during the first 5 years of life.

Conditions

New-borns Screening; Sickle Cell Disease

Keywords

major sickle cell disease (SCD); children; pneumococcal preventive measures

Outcome Measures

Primary Outcomes (3)

• Survival at 3 years of age

  between 1/01/2014 and 31/12/2015

• Probability of survival at 5 years of age

  between 1/01/2014 and 31/12/2015

• Causes of death

  between 1/01/2014 and 31/12/2015

Secondary Outcomes (5)

• Use of pneumococcal preventive measures: proportion of children who received a full pneumococcal vaccine program (4Prevenar + 1 P23) at 3 and 5 years of age

  between 1/01/2014 and 31/12/2015

• National diffusion of Trans-cranial Doppler (TCD) : proportion of children who underwent a least one TCD at the age of 2 years and the proportion of children annually monitored

  between 1/01/2014 and 31/12/2015

• Proportion of children lost to follow-up at birth, at 3 and 5 years of age and reasons for the lack of monitoring

  between 1/01/2014 and 31/12/2015

• Poportion of patient with current residual risk of stroke and invasive bacterial infections especially pneumococcal infection at 3 years of age

  between 1/01/2014 and 31/12/2015

• Number of patient with current residual risk of stroke and invasive bacterial infections especially pneumococcal infection at 5 years of age (probability)

  between 1/01/2014 and 31/12/2015

Study Arms (1)

SCD french new born

New-borns diagnosed by NBS from 01/01/2006 to 31/12/2010 (AFDPHE data, France)

Other: No intervention

Interventions

No intervention OTHER

SCD french new born

Eligibility Criteria

• Age: 3 Years - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

The project is national and aims exhaustiveness of participation of all children diagnosed with major sickle cell syndrome through newborn screening in the period of interest. The choice 2006 - 2010 is justified by the fact that during this recent period, new prevention tools (TCD, Prevenar) were fully available. The upper limit set on 31/12/2010 led for all children to a minimum follow of 3 years,

You may qualify if:

• Children born in France (metropolitan France and DOM/TOM) between 01/01/2006 and 31/12/2010
• Identified with a major sickle cell syndrome through national newborn screening .
• All types of major sickle cell disease are concerned: SS, S-Beta0 or +, SC, other (S-O-Arab, S-D Punjab)

You may not qualify if:

• Refusal to participate in the study expressed by the holders of parental authority

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• CNAMTS: collaborator
• AFDPHE: collaborator
• National reference center for thalassemia: collaborator
• National laboratories in charge of the SCD new born screening: collaborator
• Reference and competence centers for RBC disorders: collaborator

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 10, 2017
• First Posted: April 19, 2017
• Study Start: March 1, 2014
• Primary Completion: December 1, 2015
• Study Completion: March 1, 2016
• Last Updated: April 19, 2017

Record last verified: 2016-12