Source:

Karkoska
K
,
Todd
K
,
Niss
O
, et al
.
Implementation of near-universal hydroxyurea uptake among children with sickle cell anemia: A single-center experience [published online ahead of print March 19, 2021]
.
Pediatr Blood Cancer
. doi:
https://doi.org/10.1002/pbc.29008

Investigators from Cincinnati Children’s Hospital Medical Center (CCHMC), Ohio, conducted a retrospective cohort study to assess the impact of national guidelines recommending hydroxyurea for all infants with sickle cell anemia (SCA) at 9 months of age on hydroxyurea treatment practices. Patients with SCA aged ≤22 years and followed for at least 1 year in the Comprehensive Sickle Cell Center (CSCC) at CCHMC from 2010–2019 were eligible. Participants were identified through CSCC’s patient registry. Patients who receive care at CSCC typically are identified by newborn screening and have their first clinical visit by 2 months of age. The CSCC uses a multidisciplinary approach to patient care that involves a team of hematologists, nurse managers, psychologists, social workers, and pain management experts who meet weekly to review upcoming patients. During the study period, the CCSC participated in 2 national trials assessing pharmacokinetics-guided, hydroxyurea-dosing strategies in patients 6 months to 21 years old, enrolling in 2015 and 2019.

The primary exposure was time period, defined as before (2010–2013) or after (2014–2019) the publication of the 2014 National Heart, Lung, and Blood Institute (NHLBI) guidelines recommending hydroxyurea to all infants with SCA at 9 months. The primary outcome was hydroxyurea treatment history, as determined by medical chart review. Secondary outcomes included age of initiation of hydroxyurea and health care utilization (eg, hospital admissions), categorized as either SCA-related (eg, vaso-occlusive episodes, splenic sequestration, acute chest syndrome, SCA-related chronic pain, priapism) or non-SCA related. Investigators subsequently compared hydroxyurea treatment history among participants by time period as well as SCA-related hospital admissions across the total study period.

There were 273 participants included in the retrospective analysis. There were significantly more participants treated with hydroxyurea during 2014–2019 than 2010–2013 (P <0.01), with the age of initiation of hydroxyurea among participants also being significantly lower in 2014–2019 than in 2010–2013 (median of 2 vs 6 years, respectively; P <0.001). Of those who received hydroxyurea from 2014–2019, 80% were enrolled through CCSC’s participation in a hydroxyurea-dosing strategy national trial. The total number of SCA-related admissions across the overall study period declined from 67 admissions/100 patient-years in 2010 to 39 admissions/100 patient-years in 2019. (P <0.01).

The investigators conclude that the frequency of hydroxyurea prescription to children with SCA increased significantly at their institution after publication of national clinical guidelines.

Dr Hogan has disclosed no financial relationship relevant to this commentary. This commentary contains a discussion of an unapproved/investigative use of a commercial product/device.

Daily oral hydroxyurea reduces leukocytosis and endothelial adhesion molecule expression and raises hemoglobin F levels in children with SCA, resulting in fewer vaso-occlusive crises, episodes of acute chest syndrome, and hospitalizations. The 2014 NHLBI evidence-based guidelines for SCA management have helped standardize care by urging health professionals to educate and offer hydroxyurea...

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