Predictors of splenic function preservation in children with sickle cell anemia treated with hydroxyurea. (2nd June 2014)

• Record Type: Journal Article
• Authors: Nottage, Kerri A.; Ware, Russell E.; Winter, Bryan; Smeltzer, Matthew; Wang, Winfred C.; Hankins, Jane S.; Dertinger, Stephen D.; Shulkin, Barry; Aygun, Banu
• Is Part Of: European journal of haematology. Volume 93:Number 5(2014:Nov.)
• Page Start: 377
• Page End: 383
• Publication Date: 2014-06-02
• Subjects: Hematology -- Periodicals; Blood -- Diseases -- Periodicals; Blood -- Periodicals; 616.15005
• DOI: 10.1111/ejh.12361
• Languages: English
• ISSNs: 0902-4441

Abstract

<abstract abstract-type="main" id="ejh12361-abs-0001"> <title>Abstract</title> <sec id="ejh12361-sec-0001" sec-type="section"> <title>Background</title> <p>More than 90% of children with sickle cell anemia (SCA) lose splenic function by the age of 2 yrs. Splenic function may improve with hydroxyurea, but previous studies are conflicting. We prospectively evaluated the effect of hydroxyurea on splenic filtrative function.</p> </sec> <sec id="ejh12361-sec-0002" sec-type="section"> <title>Methods</title> <p>Children with SCA enrolled in the Hydroxyurea Study of Long‐Term Effects (HUSTLE‐NCT00305175) underwent clinical evaluations including Tc<sup>99</sup>m liver–spleen (LS) scans before hydroxyurea initiation and after 3 yrs of treatment to maximum tolerated dose (MTD). LS scans were classified as follows: no uptake, <10% uptake, decreased but ≥10% uptake, and normal.</p> </sec> <sec id="ejh12361-sec-0003" sec-type="section"> <title>Results</title> <p>Mean age (<italic>N</italic> = 40) was 9.1 yrs, range 2.3–17.0. After 3 yrs of treatment, 13 (33%) had uptake on LS scan. These 13 children were younger (median age 6.0 vs. 10.6 yrs, <italic>P</italic> = 0.008), had a higher HbF at baseline (mean 10.2% vs. 5.8%, <italic>P</italic> = 0.004) and after 3 yrs (22.9% vs. 13.9%, <italic>P</italic> < 0.001), achieved MTD more rapidly (median 288 vs. 358 d, <italic>P</italic> = 0.021), and were more likely to have baseline splenic uptake (<italic>P</italic> < 0.001).</p> </sec> <sec id="ejh12361-sec-0004" sec-type="section"> <title>Conclusions</title> <p>Hydroxyurea at MTD is associated with preserved or improved splenic filtrative function, with 33% demonstrating LS scan uptake after 3 yrs. Younger age, higher %HbF, and baseline splenic function are associated with a favorable outcome.</p> </sec> </abstract>