Variability of Prognostic Results Based on Biological Parameters in Sickle Cell Disease Cohort Studies in Children: What Should Clinicians Know?

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...

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Main Authors: Julie Sommet, Enora Le Roux, Bérengère Koehl, Zinedine Haouari, Damir Mohamed, André Baruchel, Malika Benkerrou, Corinne Alberti
Format: Article
Language:English
Published: MDPI AG 2021-02-01
Series:Children
Subjects:
Online Access:https://www.mdpi.com/2227-9067/8/2/143
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author Julie Sommet
Enora Le Roux
Bérengère Koehl
Zinedine Haouari
Damir Mohamed
André Baruchel
Malika Benkerrou
Corinne Alberti
author_facet Julie Sommet
Enora Le Roux
Bérengère Koehl
Zinedine Haouari
Damir Mohamed
André Baruchel
Malika Benkerrou
Corinne Alberti
author_sort Julie Sommet
collection DOAJ
description 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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spelling doaj.art-9e0bebd775f449e794c8130ee8abafe12023-12-11T17:00:34ZengMDPI AGChildren2227-90672021-02-018214310.3390/children8020143Variability of Prognostic Results Based on Biological Parameters in Sickle Cell Disease Cohort Studies in Children: What Should Clinicians Know?Julie Sommet0Enora Le Roux1Bérengère Koehl2Zinedine Haouari3Damir Mohamed4André Baruchel5Malika Benkerrou6Corinne Alberti7AP-HP, Hôpital Robert-Debré, Unité d’épidémiologie clinique, Inserm, CIC 1426, 48 bd Serurier, F-75019 Paris, FranceAP-HP, Hôpital Robert-Debré, Unité d’épidémiologie clinique, Inserm, CIC 1426, 48 bd Serurier, F-75019 Paris, FranceAP-HP, Hôpital Robert-Debré, Centre de Référence de la Drépanocytose, Service d’Hématologie, 48 bd Serurier, F-75019 Paris, FranceAP-HP, Hôpital Robert-Debré, Centre de Référence de la Drépanocytose, Service d’Hématologie, 48 bd Serurier, F-75019 Paris, FranceAP-HP, Hôpital Robert-Debré, Unité d’Epidémiologie Clinique, 48 bd Serurier, F-75019 Paris, FranceAP-HP, Hôpital Robert-Debré, Service d’Hématologie, 48 bd Serurier, F-75019 Paris, FranceUniversité de Paris, UMR 1123, ECEVE, 10 av de Verdun, F-75010 Paris, FranceAP-HP, Hôpital Robert-Debré, Unité d’épidémiologie clinique, Inserm, CIC 1426, 48 bd Serurier, F-75019 Paris, FranceBackground: 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.https://www.mdpi.com/2227-9067/8/2/143sickle cell diseasecohortbiological parameter
spellingShingle Julie Sommet
Enora Le Roux
Bérengère Koehl
Zinedine Haouari
Damir Mohamed
André Baruchel
Malika Benkerrou
Corinne Alberti
Variability of Prognostic Results Based on Biological Parameters in Sickle Cell Disease Cohort Studies in Children: What Should Clinicians Know?
Children
sickle cell disease
cohort
biological parameter
title Variability of Prognostic Results Based on Biological Parameters in Sickle Cell Disease Cohort Studies in Children: What Should Clinicians Know?
title_full Variability of Prognostic Results Based on Biological Parameters in Sickle Cell Disease Cohort Studies in Children: What Should Clinicians Know?
title_fullStr Variability of Prognostic Results Based on Biological Parameters in Sickle Cell Disease Cohort Studies in Children: What Should Clinicians Know?
title_full_unstemmed Variability of Prognostic Results Based on Biological Parameters in Sickle Cell Disease Cohort Studies in Children: What Should Clinicians Know?
title_short Variability of Prognostic Results Based on Biological Parameters in Sickle Cell Disease Cohort Studies in Children: What Should Clinicians Know?
title_sort variability of prognostic results based on biological parameters in sickle cell disease cohort studies in children what should clinicians know
topic sickle cell disease
cohort
biological parameter
url https://www.mdpi.com/2227-9067/8/2/143
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