Development and validation of a pediatric severity index for sickle cell patients

Overview & Synopsis

Introduction. There is no instrument to measure severity of sickle cell disease (SCD) in pediatric patients that is generally accepted. The aim of this study was to develop and validate a severity index for SCD in children. Methods. We developed an index consisting of 12 items and tested its validity of the index using data from 92 children. We tested whether different scores were obtained for patients classified by severity both subjectively and objectively by a partially validated existing index. Furthermore, we tested whether the index could differentiate patients classified according to genotype or the number of alpha-gene deletions and evaluated whether the score on the index was correlated with the average number and days of hospitalizations/year, age and a risk of death score. Results. We explored the effect of three different weighting systems (score A, B and C) to summarize these items. All weightings demonstrated a significant difference between the scores of mild, moderate and severely affected patients, as classified by a subjective rating or with an existing index (p