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A pilot study of hydroxyurea to prevent chronic organ damage in young children with sickle cell anemia
Author(s) -
Thornburg Courtney D.,
Dixon Natalia,
Burgett Shelly,
Mortier Nicole A.,
Schultz William H.,
Zimmerman Sherri A.,
Bonner Melanie,
Hardy Kristina K.,
Calatroni Agustin,
Ware Russell E.
Publication year - 2009
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.21738
Subject(s) - medicine , neurocognitive , transcranial doppler , renal function , sickle cell anemia , anemia , hemoglobin , gastroenterology , pediatrics , cognition , disease , psychiatry
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. Pediatr Blood Cancer 2009;52:609–615. © 2008 Wiley‐Liss, Inc.