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Deprecated: Implicit conversion from float 265.2 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Pediatr+Blood+Cancer 2009 ; 52 (5): 609-15 Nephropedia Template TP
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A PILOT STUDY OF HYDROXYUREA TO PREVENT CHRONIC ORGAN DAMAGE IN YOUNG CHILDREN WITH SICKLE CELL ANEMIA #MMPMID19061213
Thornburg CD; Dixon N; Burgett S; Mortier NA; Schultz WH; Zimmerman SA; Bonner M; Hardy KK; Calatroni A; Ware RE
Pediatr Blood Cancer 2009[May]; 52 (5): 609-15 PMID19061213show ga
Background: Hydroxyurea improves laboratory parameters and prevents acute clinical complications of sickle cell anemia (SCA) in children and adults, but its effects on organ function remain incompletely defined. Methods: To assess the safety and efficacy of hydroxyurea in young children with SCA and to prospectively assess kidney and brain function, 14 young children (mean age 35 months) received hydroxyurea at a mean maximum tolerated dose (MTD) of 28 mg/kg/day. Results: After a mean of 25 months, expected laboratory effects included significant increases in hemoglobin, MCV and %HbF along with significant decreases in reticulocytes, absolute neutrophil count, and bilirubin. There was no significant increase in glomerular filtration rate by DTPA clearance or Schwartz estimate. Mean transcranial Doppler (TCD) velocity changes were ?25.6 cm/sec (p<0.01) and ?26.8 cm/sec (p <0.05) in the right and left MCA vessels, respectively. At study exit, no child had conditional or abnormal TCD values, and none developed brain ischemic lesions or vasculopathy progression by MRI/MRA. Growth and neurocognitive scores were preserved and Impact-on-Family scores improved. Conclusions: These pilot data indicate hydroxyurea at MTD is well-tolerated by both children and families, and may prevent chronic organ damage in young children with SCA.