Pediatric hematology and oncology
December 13, 2025 · via Europe PMC
Trends in HU utilization and cerebrovascular outcomes before and after publication of the 2014 National Heart, Lung, and Blood Institute sickle cell disease guidelines.
Dain AS, Li Y, Master S, +5 more, Kwiatkowski JL.
AI summary — for doctors, generated from the abstract only
After the 2014 NHLBI sickle cell guidelines, hydroxyurea was started earlier and more often, and detected cerebrovascular disease fell, though reduced MRI screening complicates interpretation.
What was studied
Single-center retrospective study of 530 patients with SCD SS/Sβ0 comparing hydroxyurea prescribing and cerebrovascular disease outcomes before and after the 2014 guidelines.
Key findings
- Hydroxyurea prescriptions rose and age at initiation fell post-guideline
- CVD incidence 2.2/100 person-years post-guideline vs 4.4/100 person-years pre-guideline
- MRI screening decreased post-guideline, lowering CVD detection
Clinical relevance
This suggests early hydroxyurea may be associated with less cerebrovascular disease, but detection bias and confounding prevent firm conclusions.
Who should care
Limitations
Retrospective single-center before-after design; reduced MRI screening introduces detection bias.
Earlier hydroxyurea use may protect the brain in sickle cell disease, but better-controlled data are needed.
This is an AI-generated summary of the published abstract, not a clinical recommendation. It may not reflect the full text or subsequent updates — verify against the original source before acting on it.
Abstract
The 2014 National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend offering hydroxyurea to all patients with sickle cell disease SS/Sβ0 (SCD) aged ≥ 9 months. The relationship between early hydroxyurea initiation and the development of cerebrovascular disease (CVD) is unclear. This retrospective study describes trends in HU prescriptions and CVD outcomes in patients with SCD SS/Sβ0 followed at a single center before and after guideline publication. CVD was defined as the first of abnormal transcranial Doppler ultrasound, silent cerebral infarct, steno-occlusive vasculopathy, or arterial ischemic stroke. Among 530 patients with SCD, hydroxyurea prescriptions rose post-guideline, with lower age at initiation. CVD was lower post-guideline (2.2/100py) versus pre-guideline (4.4/100py). However, MRI screening also decreased post-guideline, leading to lower CVD detection. Results suggest decreased CVD, including silent cerebral infarcts, in the era of early hydroxyurea use; further analyses addressing age at hydroxyurea initiation and relevant confounders are needed to confirm whether earlier hydroxyurea initiation improves protection against CVD.
The 2014 National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend offering hydroxyurea to all patients with sickle cell disease SS/Sβ0 (SCD) aged ≥ 9 months. The relationship between early hydroxyurea initiation and the development of cerebrovascular disease (CVD) is unclear. This retrospective study describes trends in HU prescriptions and CVD outcomes in patients with SCD SS/Sβ0 followed at a single center before and after guideline publication. CVD was defined as the first of abnormal transcranial Doppler ultrasound, silent cerebral infarct, steno-occlusive vasculopathy, or arterial ischemic stroke. Among 530 patients with SCD, hydroxyurea prescriptions rose post-guideline, with lower age at initiation. CVD was lower post-guideline (2.2/100py) versus pre-guideline (4.4/100py). However, MRI screening also decreased post-guideline, leading to lower CVD detection. Results suggest decreased CVD, including silent cerebral infarcts, in the era of early hydroxyurea use; further analyses addressing age at hydroxyurea initiation and relevant confounders are needed to confirm whether earlier hydroxyurea initiation improves protection against CVD.
DOI
10.1080/08880018.2025.2579978
PMID
41388752
PMCID
PMC12928155
Research metadata (titles, abstracts, authors, publication dates) is sourced from Europe PMC, which aggregates content from PubMed/MEDLINE, PubMed Central, and other providers under the U.S. National Library of Medicine (NLM/NCBI). NLM does not claim copyright on abstracts; the original journal publishers or authors may hold rights to the full articles. AI-generated summaries are derived only from the abstract, never the full text, and are always labeled as AI-generated.
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