Hydroxycarbamide treatment and brain MRI/MRA findings in children with sickle cell anaemia.
Silent cerebral infarction (SCI) is the most common neurological abnormality among children with sickle cell anaemia (SCA). The effect of hydroxycarbamide (also termed hydroxyurea) on the development and progression of SCI is unclear. We evaluated brain magnetic resonance imaging/angiography (MRI/MRA) in children with SCA receiving long-term hydroxycarbamide therapy. Fifty participants (median 9·4 years, range 1·1-17·3) enrolled in the Hydroxyurea Study of Long-Term Effects (HUSTLE; NCT00305175) underwent brain MRI/MRA and laboratory evaluations before hydroxycarbamide initiation and after 3 and 6 years of treatment to maximum tolerated dose. SCI and vascular stenosis were evaluated. At baseline, 3 and 6 years, SCI were present in 19/50 (38%), 20/49 (41%), and 7/17 (41%), respectively. At 3 years, one child developed a SCI lesion, and another progressed (single lesion to multiple). Lower haemoglobin (Hb) (80 g/l vs. 86 g/l, P = 0·049), fetal Hb (5·0% vs. 10·4%, P < 0·001) and oxygen saturation (97% vs. 98%, P = 0·027) before hydroxycarbamide initiation were associated with SCI. No patients had vascular stenosis identified on MRA, transient ischaemic attack or stroke. Our data indicate that children receiving hydroxycarbamide over a 3- to 6-year period have a low rate of new or worsening cerebrovascular disease. Further studies are needed to confirm that hydroxycarbamide can prevent the onset and progression of SCI.
['Nottage|Kerri A|KA|', 'Ware|Russell E|RE|', 'Aygun|Banu|B|', 'Smeltzer|Matthew|M|', 'Kang|Guolian|G|', 'Moen|Joseph|J|', 'Wang|Winfred C|WC|', 'Hankins|Jane S|JS|http://orcid.org/0000-0003-4439-7321', 'Helton|Kathleen J|KJ|']
D000293:Adolescent D000755:Anemia, Sickle Cell D058070:Asymptomatic Diseases D020520:Brain Infarction D002648:Child D002675:Child, Preschool D005260:Female D005319:Fetal Hemoglobin D006801:Humans D006918:Hydroxyurea D007223:Infant D018810:Magnetic Resonance Angiography D008279:Magnetic Resonance Imaging D008297:Male D016896:Treatment Outcome
hydroxycarbamide brain MRI sickle cell anaemia silent cerebral infarct brain MRA