Study in England finds congenital TTP comes with high cost burden
Doctor visits, hospitalizations contribute to high economic costs
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People with congenital thrombotic thrombocytopenic purpura (cTTP) face a substantial burden related to healthcare use and costs, particularly for inpatient admissions directly related to the disease, according to a real-world study in England.
The study demonstrated that almost 40% of patients experienced an acute TTP episode during follow-up periods that ranged up to 10 years, with more than 25% of requiring intensive care. The annual healthcare cost for a cTTP patient was £6,155 (about $8,313), compared with £858 (about $1,159) for participants without cTTP.
“These results provide valuable insights for healthcare decision makers and may support future healthcare planning, the development of clinical guidelines and policy initiatives aimed at improving the management of rare diseases such as cTTP,” the researchers wrote.
The study, “Clinical burden and healthcare resource use of congenital thrombotic thrombocytopenic purpura in England: A linked primary and secondary care data analysis,” was published in the British Journal of Haematology.
TTP is caused by a deficiency of the ADAMTS13 enzyme, and is characterized by the formation of small clots in blood vessels. cTTP is driven by mutations in the ADAMTS13 gene. TTP symptoms can arise suddenly, driven by low counts of platelets (cell fragments that are key to clotting), hemolytic anemia (low red blood cell levels caused by red blood cell destruction), and organ damage. Relapses occur when symptoms return after remission from a previous episode.
Quantifying treatment costs
Current guidelines recommend that people with TTP receive prophylactic (preventive) treatment to reduce the risk of relapses. Treatments include Adzynma (ADAMTS13, recombinant-krhn) or plasma infusions.
Researchers conducted a retrospective study using the Clinical Practice Research Datalink, which collects data from a network of general practitioners, to evaluate the clinical burden, healthcare resource use, and associated costs of treating cTTP patients in England.
The study used data from 36 patients with cTTP and 72 control participants without TTP. Patients had a mean age of 39.4, and most were women (86.1%). They were followed up for up to 10 years — a mean of 4.4 years in primary care and 5.7 years in secondary (specialized) care. Those with cTTP more commonly had previous organ damage (52.8% vs. 8.3%), particularly in the kidneys (25%) and brain (22.2%), than controls.
Fourteen patients (38.9%) experienced a total of 27 acute TTP episodes, resulting in a rate of 0.13 per person-year — meaning that on average, patients may experience 1.3 events over a 10-year period. These events lasted a mean of 13 days, and about a quarter involved a stay in the intensive care unit.
Patients with organ damage experienced a higher mean number of acute TTP events than those without (1.43 vs. 0.59). Managing acute events cost a median of £13,872 (approximately $18,731).
“The risk of this medical emergency remains high despite the availability of plasma-based therapies,” the researchers wrote.
About 20% of patients experienced at least one organ damage event during follow-up. The rate of organ damage events was 0.05 per person-year in the overall patient population, meaning an average of five cases in 100 people followed for one year.
cTTP patients used healthcare resources more than controls, particularly inpatient admissions (26.76 times higher), followed by outpatient appointments (4.01 times), emergency care visits (1.87 times), and primary care appointments (1.42 times higher). The proportion of healthcare related to TTP itself was 43.6% for inpatient admissions, 5.9% for primary care appointments, and 2.5% for outpatient appointments.
The average yearly cost of healthcare for a cTTP patient was mostly related to inpatient admissions (75.6%), followed by outpatient appointments (18.6%), primary care (4%), and emergency care attendances (1.8%).
“This study highlights the considerable clinical and economic burden of cTTP,” and “demonstrates the high clinical and economic burden of cTTP and provides evidence to inform healthcare planning,” the researchers wrote.
