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Updated: Nov 15, 2025
A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Variability of Prognostic Results Based on Biological Parameters in Sickle Cell Disease Cohort Studies in Children:
Julie Sommet1,2,3, Enora Le Roux1,2,4, Bérengère Koehl5,6
1AP-HP, Hôpital Robert-Debré, Unité d'épidémiologie clinique, Inserm, CIC 1426, 48 bd Serurier, F-75019 Paris, France.
Insights
The method used to analyze blood pressure (BP) significantly impacts the prediction of sickle cell disease (SCD) severity in children. Different statistical approaches yield varying results for assessing BP
Area of Science:
- Pediatric Hematology
- Clinical Epidemiology
- Biostatistics
Background:
- Pediatric studies often explore the link between biological parameters (BP) and sickle cell disease (SCD) severity.
- Existing research uses diverse methods for collecting and analyzing BP data in children with SCD.
- This variability in methodology complicates the interpretation of prognostic results.
Purpose of the Study:
- To assess how different BP collection and statistical analysis methods affect prognostic outcomes in pediatric SCD cohort studies.
- To evaluate the association between BP and cerebral vasculopathy (CV) in a cohort of 375 children with SCD using various statistical approaches.
Main Methods:
- Systematic identification of BP collection and statistical analysis methods in published SCD cohort studies.
- Application of four distinct statistical methods (baseline, last known, mean, two-stage modeling) to a cohort of 375 children with SCD.
- Evaluation of the association between BP and cerebral vasculopathy (CV) using these varied methods.
Main Results:
- BP data were collected either once or multiple times during follow-up in 16 cohort studies.
- The four statistical methods applied to the study cohort produced different results and interpretations regarding the BP-CV association.
- The choice of statistical analysis method demonstrably influences the observed relationship between BP and CV.
Conclusions:
- The prognostic value of biological parameters (BP) in pediatric SCD is contingent upon the statistical analysis method employed.
- Standardizing or carefully considering statistical approaches is crucial for achieving valuable and reproducible conclusions in prognostic factor studies.
- This highlights the need for methodological rigor in pediatric SCD research to ensure reliable prognostic assessments.
Background:
Many pediatric studies describe the association between biological parameters (BP) and severity of sickle cell disease (SCD) using different methods to collect or to analyze BP. This article assesses the methods used for collection and subsequent statistical analysis of BP, and how these impact prognostic results in SCD children cohort studies.
Methods:
Firstly, we identified the collection and statistical methods used in published SCD cohort studies. Secondly, these methods were applied to our cohort of 375 SCD children, to evaluate the association of BP with cerebral vasculopathy (CV).
Results:
In 16 cohort studies, BP were collected either once or several times during follow-up. The identified methods in the statistical analysis were: (1) one baseline value per patient (2) last known value; (3) mean of all values; (4) modelling of all values in a two-stage approach. Applying these four different statistical methods to our cohort, the results and interpretation of the association between BP and CV were different depending on the method used.
Conclusion:
The BP prognostic value depends on the chosen statistical analysis method. Appropriate statistical analyses of prognostic factors in cohort studies should be considered and should enable valuable and reproducible conclusions.
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