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Prognosis Remains Unclear After Early Sickle Cell Symptoms


DALLAS -- Serious symptoms of sickle cell disease in childhood don't imply a greater risk of premature stroke or death for patients as they grow up, according to researchers here.

DALLAS, Jan. 30 -- Serious symptoms of sickle cell disease in childhood don't imply a greater risk of premature stroke or death for patients as they grow up, according to researchers here.

The findings, from a long-term retrospective study of more than 200 children with two forms of sickle cell disease, leave physicians without useful prognostic guidelines, said Charles Quinn, M.D., of the University of Texas Southwestern Medical Center.

Until there's a deeper understanding of the disease course, Dr. Quinn and colleagues reported in the January issue of Blood, intensive disease-modifying therapy in young children shouldn't be undertaken on the basis of a few episodes of early pain or swelling of the fingers.

Indeed, they said, the only early vaso-occlusive symptom that appeared to have predictive value was acute chest syndrome (ACS). This was defined as acute pulmonary illness in a patient with sickle cell disease, characterized by a new radiographic pulmonary infiltrate and some combination of fever, hypoxemia, thoracic pain, and signs and symptoms of respiratory illness.

Children who have early episodes of ACS, the researcher found, are likely to have more of them as they grow up than do other children with sickle cell disease.

Two other common early symptoms - dactylitis, or painful swelling of the fingers, and non-dactylitis pain - did not predict any future complication, the researchers said.

The findings make it difficult to counsel parents of babies with sickle cell disease about what they can expect, Dr Quinn said.

"We can't give them very much in the way of specifics, exactly what this child will likely go through, or what to expect from the disease in the future," he said.

The researchers analyzed medical records of 244 children with sickle cell disease, members of the Dallas Newborn Cohort, who had been treated in hospital for one of the three early symptoms within the first year of life.

Children in the study had either sickle cell anemia or sickle-?0-thalassemia. Median length of follow-up was 12.1 years.

The researchers studied the associations between the three early symptoms and later adverse events, including death and stroke, use of disease-modifying therapy, and hospitalizations for pain events and ACS.

Only ACS was a significant predictor in multivariate analysis - children with early ACS were twice as likely as other children to have later ACS episodes. The odds ratio was 2.23, with a 95% confidence interval from 1.52 to 3.29, which was statistically significant at P

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