Sommet Julie, Roux Enora Le, Koehl Bérengère, Haouari Zinedine, Mohamed Damir, Baruchel André, Benkerrou Malika, Alberti Corinne
AP-HP, Hôpital Robert-Debré, Unité d'épidémiologie clinique, Inserm, CIC 1426, 48 bd Serurier, F-75019 Paris, France.
Université de Paris, UMR 1123, ECEVE, 10 av de Verdun, F-75010 Paris, France.
Children (Basel). 2021 Feb 13;8(2):143. doi: 10.3390/children8020143.
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.
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).
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.
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.
许多儿科研究使用不同的方法收集或分析生物学参数（BP），描述了生物学参数与镰状细胞病（SCD）严重程度之间的关联。本文评估了用于收集BP以及对其进行后续统计分析的方法，以及这些方法如何影响SCD儿童队列研究的预后结果。
首先，我们确定已发表的SCD队列研究中使用的收集和统计方法。其次，将这些方法应用于我们的375名SCD儿童队列，以评估BP与脑血管病变（CV）的关联。
在16项队列研究中，在随访期间收集BP一次或多次。统计分析中确定的方法有：（1）每位患者一个基线值；（2）最后已知值；（3）所有值的平均值；（4）分两阶段对所有值进行建模。将这四种不同的统计方法应用于我们的队列，BP与CV之间关联的结果和解释因所用方法而异。
BP的预后价值取决于所选的统计分析方法。队列研究中应考虑对预后因素进行适当的统计分析，这应能得出有价值且可重复的结论。
