Revisiting acute chest syndrome (ACS) associated with sickle cell disease (SCD) vaso-occlusive pain episodes (VOE): Insight from a prospective, multicenter Phase-3 randomized controlled trial
Abstract
Background: ACS occurs in up to 20% of hospitalized patients with SCD-VOE, often prolonging and complicating hospital stay. Despite its clinical significance, variations exist in the management and outcomes of ACS across institutions.
Objective: Determine prevalence of ACS in children and young adults hospitalized for SCD-VOE and describe emergency department (ED) presentation, clinical course and practice variation across institutions.
Methods: Cross-sectional analysis of data collected from a PECARN-endorsed multicenter, double-blinded, randomized, placebo-controlled phase-3 trial of intravenous arginine therapy in hospitalized patients with SCD-VOE aged 3-21 (NCT04839354) at 10 pediatric EDs across the US. ACS defined by radiology-interpreted chest radiograph (CXR) positive for new infiltrate and clinical team diagnosis. ACS severity was defined a priori as mild (no oxygen (O2) use or RBC transfusion), moderate (O2 use or transfusion), or severe (bilevel positive airway pressure (BiPAP) use, intubation, or pediatric intensive care unit (PICU) transfer).
Results: 271 patients enrolled (median age 15[11,18] years, 51% male; 74% HbSS/Sb°; 76% on Hydroxyurea (HU). ACS occurred in 20% (n=54; median age 13[9,16] years; 76% male; 89% HbSS/Sb°; 80% on HU); 18 diagnosed in the ED, and 36 diagnosed during their hospitalization. 72% of patients with inpatient-diagnosed ACS had a negative CXR in the ED with a mean time to diagnosis of 2.4±1.6 days. Patients with ACS at any time were…
How strong is the evidence?
Evidence Hub reads this as a randomized trial, done in people. Its evidence level is 2 of 5 (High · randomized trial). Randomized trials of a reasonable size are the strongest single-study design. Reported size: 271 participants or records.
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