Prediction of adverse outcomes in children with sickle cell disease

Background: The ability to identify infants with sickle cell anemia who are likely to have severe complications later in life would permit accurate pr ognostication and tailoring of therapy to match disease-related risks and f acilitate planning of clinical trials. We attempted to define the features of such babies by following the clinical course of 392 children with sickle cell disease from infancy to about the age of 10 years. Methods: We analyzed the records of 392 infants who received the diagnosis of homozygous sickle cell anemia or sickle cell-beta(0)-thalassemia before the age of six months and for whom comprehensive clinical and laboratory da ta were recorded prospectively; data were available for a mean (+/-SD) of 1 0.0+/-4.8 years. Results obtained before the age of two years were evaluate d to determine whether they predicted the outcome later in life. Results: Of the 392 infants in the cohort, 70 (18 percent) subsequently had an adverse outcome, defined as death (18 patients [26 percent]), stroke (2 5 [36 percent]), frequent pain (17 [24 percent]), or recurrent acute chest syndrome (10 [14 percent]). Using multivariate analysis, we found three sta tistically significant predictors of an adverse outcome: an episode of dact ylitis (defined as pain and tenderness in the hands or feet) before the age of one year (relative risk of an adverse outcome, 2.55; 95 percent confide nce interval, 1.39 to 4.67), a hemoglobin level of less than 7 g per decili ter (relative risk, 2.47; 95 percent confidence interval, 1.14 to 5.33), an d leukocytosis in the absence of infection (relative risk, 1.80; 95 percent confidence interval, 1.05 to 3.09). Conclusions: Three easily identifiable manifestations of sickle cell diseas e that may appear in the first two years of life (dactylitis, severe anemia , and leukocytosis) can help to predict the possibility of severe sickle ce ll disease later in life. (N Engl J Med 2000;342:83-9.) (C) 2000, Massachus etts Medical Society.