Health Impact and Burden of Illness in Immune Thrombocytopenia: A Literature Review on Clinical, Humanistic, and Economic Burden

OBJECTIVES: Immune-thrombocytopenia (ITP) is a rare autoimmune-platelet disorder that meets the criteria for an orphan disease, with a prevalence of 1-5 cases per 10,000 population. This review aimed to summarize the evidence on clinical, humanistic, and economic burden among ITP patients.

METHODS: Embase^® and Medline^® databases, and key conferences of interest were searched from January-2013 to October-2023. Studies reporting quality-of-life (QoL), clinical symptoms, healthcare resource-utilizations (HCRU), and costs were included.

RESULTS: A total of 67 studies were included (clinical data:65; economic data:25; humanistic data:12). The most frequently reported clinical symptoms and conditions among ITP patients were fatigue (43%-94%), bleeding (14%-83%), and treatment-related thrombosis (21.4%-27%). Patients aged 60-89 years versus those aged 18-60 years had a significantly higher thrombosis rate (p=0.014), while fatigue rates were similar between groups (p=0.349). Women experienced higher symptom burden than men, with a numerically higher frequency of overall bleeding (63.3% vs 52.6%). The mortality rate among patients ranged from 0.8% to 9.9%. In comparison to the general population, ITP patients had lower QoL, with 23.5%-54% of patients complaining of fatigue being the most important driver for decreased QoL. Twenty-two studies assessed HCRU, majorly focusing on hospitalization (6%-54.8%), physician visits (48.7%-79.3), and intensive-care unit stays (7.7%-24%). Severe disease with bleeding events was associated with significantly higher HCRU than patients without bleeding (p=0.03). The overall per-patient cost of treating ITP patients was $21,290, with ambulatory encounters being the major cost driver ($12,978).

CONCLUSIONS: ITP patients face a significant clinical burden attributed to high rates of fatigue, bleeding, and thrombosis. This burden is further compounded by impaired QoL, increased HCRU, and overall treatment costs in these patients. Disease burden is higher in women and elderly patients. These findings provide valuable information for healthcare providers to reduce the disease burden of ITP.