(230) The economic burden of immune thrombocytopenia in the United States: insights from a systematic literature review and key evidence gaps

Background: : Immune thrombocytopenia (ITP) is a rare autoimmune disorder characterized by low platelet levels, leading to clinical presentations that range from asymptomatic to uncontrolled bleeding. Severe cases often require hospitalization and costly treatments, including infusions, transfusions, and splenectomy. Understanding the economic burden associated with ITP is critical to inform healthcare decision-making in the US.

Objective: : This systematic literature review aims to summarize data on the economic burden associated with ITP in the US and identify evidence gaps to inform future research.

Methods: : Systematic searches were conducted in MEDLINE and Embase on April 26, 2025. English language articles published since January 2015 were evaluated for inclusion. Supplementary searches of recent major conferences and grey literature were conducted. Studies were screened for relevance at 2 levels (title/abstract and full text) by 2 researchers independently using the same pre-specified PICOS criteria, with disagreements adjudicated by a third reviewer.

Results: : Overall, 737 publications were identified, and 58 unique studies met inclusion criteria and described economic outcomes among patients with ITP in the US. Several large claims-based analyses were included (average sample size of 3,688 patients with ITP).

Most studies (84%) reported healthcare resource utilization (HCRU) metrics, most commonly hospitalizations and length of stay, splenectomies, and infusion (IVIG, steroids) and transfusion (platelets, red blood cells) rates. The National Inpatient Sample was the most common data source (17 studies). Direct costs were reported in 25 studies. While costs associated with hospitalization were the most common (15 studies), costs associated with bleeding-related episodes, treatments, and adverse events were also reported. No studies reported indirect costs specific to patients with ITP in the US. Additionally, 7 economic models were identified, including cost-effectiveness analyses, cost-consequence models, and a cost-minimization model. The cost associated with severe bleeding events was consistently identified as a key driver. Other key parameters included costs associated with drug administration, treatment discontinuation, and drug wastage.

Conclusions: : Direct costs and HCRU associated with ITP in the US are well-characterized. Key direct cost drivers include hospitalizations, bleeding events, and treatments. Indirect costs, including work loss and caregiver burden, specific to US patients with ITP represent an important evidence gap.

Sponsorship:: Novartis