OBJECTIVES: Congenital Thrombotic Thrombocytopenic Purpura (cTTP) is an ultra-rare, genetic, life-threatening blood clotting condition associated with serious medical complications. This study aimed to describe the healthcare resource use and associated costs related to cTTP patient care in France.

METHODS: A retrospective, real-world study using data from the French National hospital database (January 1^st, 2015 to December 31^st, 2020) was conducted. The study population included hospitalized patients identified using an algorithm which included the presence of at least one ICD-10 diagnosis code M31.1 “Thrombotic microangiopathy” and regular plasma transfusions. Descriptive statistics (medians, means, Q1-Q3) on patient characteristics, hospital stays and associated costs were performed within the overall population and by subpopulations (children, pregnant women and other non-pregnant adults).

RESULTS: Overall, 28 cTTP patients were identified (median age 26 years, 61% female), being 32.1% pregnant women, 25.0% children, and 42.9% other adults. Most cTTP hospitalizations (91%) were outpatient, and 57% of patients had at least one emergency room admission. cTTP patients had a median monthly number of 0.5 (0.2-1.5) hospitalizations for a median monthly cumulative duration of 2.6 (0.7-4.1) days, which costed a median of €1,536 (€720-€3,483) per month. Median time between two plasma transfusions was 2-3 weeks (1.3-3.9 weeks). Regarding the three subpopulations, the number of hospitalizations seemed to be lower in pregnant women; and duration of cTTP hospitalizations and costs related to cTTP hospitalizations seemed to be higher in other non-pregnant adults.

CONCLUSIONS: This first real-world study conducted using French hospital claims database highlights the heterogeneity among cTTP patients in three distinct subpopulations (children, pregnant women and other adults) receiving plasma regularly. At patient level, disease management and its related costs are significant and should continue to be assessed in real-life settings as new cTTP therapies become available to patients.