• Xandra W van den Tweel, Johanna H van der Lee, Harriët Heijboer, Marjolein Peters, and Karin Fijnvandraat.
• Department of Pediatric Hematology, Emma Children's Hospital, Academic Medical Center, Amsterdam, The Netherlands. x.w.vandentweel@amc.nl
• Am. J. Hematol. 2010 Oct 1; 85 (10): 746-51.

AbstractThere 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. 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 α-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. 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 < 0.01). The index clearly differentiated patients by genotype (P < 0.01) or α-gene deletions (P < 0.01). The correlation with hospitalization was moderate. Age and the risk of death score were weakly associated with the pediatric severity index for SCD. This is the first pediatric SCD severity index that was developed and validated using modern clinimetric methodology. The validity and reliability of this index should be further evaluated in a prospective study including a larger cohort, preferably diagnosed at birth.© 2010 Wiley-Liss, Inc.

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