Clinical burden of propionic acidemia in the United States: a claims-based study by age stratum.

ABSTRACT:

Background

Patients with propionic acidemia (PA) may face recurrent metabolic decompensation events (MDEs) and multisystemic complications. This study compared characteristics and clinical outcomes of patients with PA and matched non-PA controls by age stratum.

Methods

Patients with PA from the US IQVIA PharMetrics Plus claims database (10/2015‒6/2022) had their follow-up time partitioned into age strata (0‒2, 3‒6, 7‒12, 13‒17, ≥ 18 years) and were matched 1:1 to randomly selected non-PA controls within each stratum. MDEs were identified as hospitalizations with claims for hyperammonemia and/or metabolic acidosis. Hospitalizations with claims for PA signs and symptoms were evaluated.

Results

Among 191 patients with PA and 230 matched non-PA controls (median follow-up:

SUBMITTER: Banerjee G 

PROVIDER: S-EPMC12265150 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature