1. To establish the prevalence of PH in our population of children with SCD by standardized echocardiography; 2. To investigate the predictive value of alveolar and bronchial FeNO for PH in SCD patients. 3. To establish (genetic) risk factors for PH…
ID
Source
Brief title
Condition
- Haemoglobinopathies
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Correlations between echocardiographic results (tricuspidalis Regrurgitant Jet
Velocity Value; m/s) and FeNO at different expiratory flow velocities will be
tested, in order to evaluate the predictive value of exhaled NO in diagnosis of
PH.
Secondary outcome
Evaluation of possible (genetic) risk factors for pulmonatry hypertension in
SCD
Background summary
As life expectancy improves in patients with hemoglobinopathies, including
sickle cell disease (SCD), new chronic complications are emerging such as
pulmonary hypertension. Pulmonary hypertension (PH) is a serious complication
of SCD and is strongly associated with early mortality. Studies in adults
report prevalences of 30-34%. Recently, similar percentages have been reported
in children with SCD. Diagnostic methods to detect PH include: heart
catheterisation and echocardiography. As nitric oxide (NO) depletion due to the
chronic hemolysis in SCD is an important factor in the pathogenesis of PH in
SCD, we hypothesized that measurement of exhaled NO (fraction of exhaled nitric
oxide or FeNO), may play a complementary role in identifying patients at risk
for PH. Sullivan et al. recently reported lower FeNO levels in children with
SCD, but did not correlate this finding with PH.
Study objective
1. To establish the prevalence of PH in our population of children with SCD by
standardized echocardiography;
2. To investigate the predictive value of alveolar and bronchial FeNO for PH in
SCD patients.
3. To establish (genetic) risk factors for PH in SCD (severity of hemolysis,
level of fetal hemoglobin, level of methemoglobin, DNA analysis).
Study design
Observational pilot study (n = 20), if results prove the study feasible, a
large prospective study will be initiated in our total patient cohort (n= 150),
with an interim analysis after at least 50 patients. Spirometry and
measurements of exhaled NO will be performed in our study group as has been
extensively applicated in children in earlier studies (M.W.H. Pijnenburg;
Protocol ID/ number: MEC212.505/2002/92, MEC203.288/2001/164,
MEC194.549/2000/180).
Study burden and risks
In addition to the current annual diagnostic work-up (blood analysis,
echocardiography, ophthalmological evaluation, trancranial doppler evaluation),
spirometry and measurement of FeNO (duration of procedure: 30 minutes,
non-invasive, neglible risk for patient) will take place during a regular
follow-up visit to the outpatient clinic.
Dr. Molewaterplein 60
3015 GJ Rotterdam
Nederland
Dr. Molewaterplein 60
3015 GJ Rotterdam
Nederland
Listed location countries
Age
Inclusion criteria
Sickle cell patient, 12 to 18 years of age.
Exclusion criteria
Acutely or critically ill patients, recent bloodtransfusion (< 3 months prior), not able to understand Dutch adequately.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL23409.078.08 |