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2009 ; 52
(5
): 609-15
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gab.com Text
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English Wikipedia
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
PMID19061213
show 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.