New-borns ScreeningEVADREPNCT03119922
Quality of Care of Children With Sickle Cell Disease (SCD) Screened at Birth in France
An observational study of New-borns Screening and Sickle Cell Disease, run by Assistance Publique - Hôpitaux de Paris.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 5 years
- Conditions the study is for
- New-borns Screening, Sickle Cell Disease
- How many people
- 1,750 participants
- Where
- No sites listed yet
- Study dates
- Mar 2014to Mar 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 5 years
- All sexes
- No healthy volunteers
- Child
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,
Who may be able to join
- 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)
Who cannot join
- Refusal to participate in the study expressed by the holders of parental authority
About this study
Official title: National Project for Healthcare Assessment of SCD Children Diagnosed by Newborn Screening in France
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.
Read the detailed description
In agreement with the National Insurance Fund (CNAMTS) and the French Association for Screening and Prevention of Child Handicaps (AFDPHE), the French SCD newborn screening (NBS) program is not universal but "targeted" to children identified "at risk" because of the geographical origin of their parents (from a country where the prevalence of sickle cell trait is high: Sub-Saharan Africa, the Caribbean, North Africa, Mediterranean Basin). The number of children diagnosed at birth is increasing steadily each year and sickle cell disease is now in France the most common disease found among those screened in the neonatal period. The total number of new-borns diagnosed by NBS from 01/01/2006 to 31/12/2010 is 1800 (AFDPHE data).
In the absence of preventive care, a high mortality rate is observed during the first 5 years of life, the two main causes of death being invasive pneumococcal infection and splenic sequestration acute. Early care allows to largely preventing invasive pneumococcal disease (by combination of PeniV long-term treatment and pneumococcal vaccinations) and severe acute splenic sequestrations (by establishing a parent education program). Also, prevention of cerebral vascular disease, the most serious complications of sickle cell disease in young children, has been made possible the last 10-15 years by the systematic use of trans-cranial Doppler (TCD). In rich countries, the mortality rate of SCD children detected at birth, is now reduced to less than1-2%. The last French report analysed the results of national NBS program for the period 1995- 2000. Consequently these results did not take into account the most recent advances (use of pneumococcal conjugate vaccine and diffusion of TCD). It is now well demonstrated that these two preventive actions have further improved the morbidity and mortality of children with sickle cell disease.
Recently the French paediatric team from Creteil (France), reported the results of 217 children screened at birth between 1988 and 2007: this single-center study finds excellent results with a 1.9% probability of occurrence of stroke at the age of 18 years for SS and S-Beta0 children. These results should be compared with those obtained for the whole concerned population in France. In order to describe and analyse recent national results on residual morbidity and mortality, the investigators conducted this national retrospective study (EVADREP) studying clinical care during the 5 first years of life of SCD patients diagnosed at birth for the period 2006- 2010.
- Conditions
- New-borns Screening, Sickle Cell Disease
- Being tested
- No intervention
- Keywords
- major sickle cell disease (SCD), children, pneumococcal preventive measures
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
| Study group | Role | What they receive |
|---|---|---|
| SCD french new born | New-borns diagnosed by NBS from 01/01/2006 to 31/12/2010 (AFDPHE data, France) Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival at 3 years of age
Measured: between 1/01/2014 and 31/12/2015
Probability of survival at 5 years of age
Measured: between 1/01/2014 and 31/12/2015
Causes of death
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: between 1/01/2014 and 31/12/2015
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2017
- Study startMar 2014
- Primary completionDec 2015
- Study completionMar 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for New-borns ScreeningStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 1,750 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- CNAMTS, AFDPHE, National reference center for thalassemia, National laboratories in charge of the SCD new born screening, Reference and competence centers for RBC disorders
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03119922
- Sponsor's protocol ID
- NI 12001
- Acronym
- EVADREP
- Status last verified
- Dec 2016
- First submitted
- 10 Jan 2017
- Last updated
- 19 Apr 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- National Project for Healthcare Assessment of SCD Children Diagnosed by Newborn Screening in France
- ClinicalTrials.gov ID
- NCT03119922
- Acronym / sponsor ID
- EVADREP / NI 12001
- Conditions
- New-borns Screening, Sickle Cell Disease
- Keywords
- major sickle cell disease (SCD), children, pneumococcal preventive measures
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,750 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- CNAMTS, AFDPHE, National reference center for thalassemia, National laboratories in charge of the SCD new born screening, Reference and competence centers for RBC disorders
- Study start
- Mar 2014
- Primary completion
- Dec 2015 (actual)
- Study completion
- Mar 2016 (actual)
- First posted
- 19 Apr 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
In agreement with the National Insurance Fund (CNAMTS) and the French Association for Screening and Prevention of Child Handicaps (AFDPHE), the French SCD newborn screening (NBS) program is not universal but "targeted" to children identified "at risk" because of the geographical origin of their parents (from a country where the prevalence of sickle cell trait is high: Sub-Saharan Africa, the Caribbean, North Africa, Mediterranean Basin). The number of children diagnosed at birth is increasing steadily each year and sickle cell disease is now in France the most common disease found among those screened in the neonatal period. The total number of new-borns diagnosed by NBS from 01/01/2006 to 31/12/2010 is 1800 (AFDPHE data).
In the absence of preventive care, a high mortality rate is observed during the first 5 years of life, the two main causes of death being invasive pneumococcal infection and splenic sequestration acute. Early care allows to largely preventing invasive pneumococcal disease (by combination of PeniV long-term treatment and pneumococcal vaccinations) and severe acute splenic sequestrations (by establishing a parent education program). Also, prevention of cerebral vascular disease, the most serious complications of sickle cell disease in young children, has been made possible the last 10-15 years by the systematic use of trans-cranial Doppler (TCD). In rich countries, the mortality rate of SCD children detected at birth, is now reduced to less than1-2%. The last French report analysed the results of national NBS program for the period 1995- 2000. Consequently these results did not take into account the most recent advances (use of pneumococcal conjugate vaccine and diffusion of TCD). It is now well demonstrated that these two preventive actions have further improved the morbidity and mortality of children with sickle cell disease.
Recently the French paediatric team from Creteil (France), reported the results of 217 children screened at birth between 1988 and 2007: this single-center study finds excellent results with a 1.9% probability of occurrence of stroke at the age of 18 years for SS and S-Beta0 children. These results should be compared with those obtained for the whole concerned population in France. In order to describe and analyse recent national results on residual morbidity and mortality, the investigators conducted this national retrospective study (EVADREP) studying clinical care during the 5 first years of life of SCD patients diagnosed at birth for the period 2006- 2010.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SCD french new born | – | Other: No interventionNew-borns diagnosed by NBS from 01/01/2006 to 31/12/2010 (AFDPHE data, France) |
Who may qualify
- Ages
- 3 to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- 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,
Key inclusion criteria (3 listed)
- 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)
Key exclusion criteria (1 listed)
- Refusal to participate in the study expressed by the holders of parental authority
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No intervention | Other | SCD french new born |
Primary outcome
Survival at 3 years of age
Time frame: between 1/01/2014 and 31/12/2015
Probability of survival at 5 years of age
Time frame: between 1/01/2014 and 31/12/2015
Causes of death
Time frame: between 1/01/2014 and 31/12/2015
5 secondary outcomes
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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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)
Time frame: between 1/01/2014 and 31/12/2015
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Apr 2017.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of New-borns Screening and Sickle Cell Disease, run by Assistance Publique - Hôpitaux de Paris.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Survival at 3 years of age (time frame: between 1/01/2014 and 31/12/2015). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Apr 2017.