Epidemiology, Treatment Patterns, Clinical Outcomes, and Disease Burden Among Patients with Immune-mediated Thrombotic Thrombocytopenic Purpura in the United States

Background: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a life-threatening thrombotic microangiopathy. Due to its rarity, epidemiology and real-world outcomes data are scarce.

Objectives: The aim was to assess epidemiology, treatment patterns, clinical outcomes, and disease burden in patients with iTTP in the United States.

Methods: This longitudinal, retrospective observational study of the Optum-Humedica database included patients with an iTTP diagnosis (≥1 documented ADAMTS13 activity less than 10% or one or more iTTP episodes) from January 2007 to December 2019.

Results: Of 666 patients with an iTTP diagnosis between October 2015 and December 2019, 302 (45%) had one or more iTTP episodes. The pooled annual incidence of documented iTTP during this period was 3.43/million, and the annual incidence of one or more iTTP episodes was 1.81/million. Patients with one or more iTTP episodes received a median of six therapeutic plasma exchange (TPE) sessions per episode; 86% received corticosteroids, and 59% received rituximab. Exacerbations occurred in 17% (52/302) and relapse in 11% (34/302); 34% (103/302) had one or more thromboembolic events. Mortality rates during the study period were 25% (167/666) among all patients with iTTP diagnosis, and 14% (41/302) among patients with one or more iTTP episodes. In the assessment of disease burden (January 2007 to September 2019), patients in the iTTP cohort ( = 514) presented with a mean of 14 comorbidities, compared with 3 in a matched non-iTTP cohort ( = 2570). In a cluster analysis, duration of iTTP episode and mortality rate were greater in older versus younger patients.

Conclusions: Despite treatment with TPE and immunosuppressants, patients with iTTP have high risk of morbidity and mortality, demonstrating the need for more effective therapies.