Immune-mediated thrombotic thrombocytopenic purpura landscaping in Gulf countries: a real-world evidence study (ATHENA Study)

Background: Immune -mediated thrombotic thrombocytopenic purpura (iTTP) is a prevalent lifethreatening disorder, but there is a lack of clear data on the nature and local burden of this disease in Gulf countries. So, in this study, we aimed to assess the local burden of iTTP and understand its management pattern in the Arabian Gulf region. Methods: In this multinational, multicenter, retrospective study, data were collected from patients' records in the included countries (Saudi Arabia, United Arab Emirates, Kuwait, Qatar, and Oman). Patients with a diagnosis of iTTP in the past 36 months who met the eligibility criteria were included. Data on time to platelet normalization, organ damage markers, healthcare resource utilization data, and iTTP-related death were collected. Results: Sixty patients were included [mean age at diagnosis of 39.3 years, standard deviation (SD): 16.5 years]. Platelet normalization was achieved after a median [range] of 6 [3-32] days in the overall population and 4 [2-10] days in patients who received caplacizumab (n=7). In addition, lactate dehydrogenase, troponin, and creatinine levels improved post -treatment. To confirm the diagnosis of iTTP, ADAMTS13 and antiADAMTS13 antibody levels were tested in 56.7% and 46.7% of patients, respectively. Six patients died (none of whom received caplacizumab); four deaths were related to iTTP. Conclusions: Patients with iTTP who received caplacizumab showed shorter times to platelet normalization and higher survival rates versus those who did not receive caplacizumab. iTTP imposes a significant burden on patients and regional healthcare systems, so the availability of diagnostic tests and treatment options are critical to improve treatment outcomes and patients' survival.