What are the clinical consequences of cTTP?
The clinical presentation ranges from life-threatening, acute, overt TTP events to milder TTP manifestations, including thrombocytopenia, haemolytic anaemia, abdominal pain, headaches, and neurological symptoms.1 End-organ damage due to recurrent overt and non-overt TTP events, such as stroke, chronic kidney disease, or cardiac involvement, can develop.1
As well as acute events, ongoing subacute manifestations of cTTP can also occur despite the absence of typical clinical and laboratory findings.1,2 Symptoms of subacute cTTP can include lethargy, headaches, migraines and abdominal pain. It is important for clinicians to be aware of these manifestations as they may indicate underlying disease.3 Additionally, over time, cTTP can result in long-term neurological, cardiovascular, or renal complications.1 Retrospective data has shown that patients with TTP have a higher mortality rate as compared to the general population.4 This suggests that TTP should be viewed as a chronic disease with acute events.5,6
In a study integrating inputs from a literature review, expert opinions, and patient interviews, patients interviewed by telephone indicated that the most salient impacts of cTTP were the diminished ability to work/study, financial distress, feeling depressed, feeling anxious, and mood swings.7
In practice, the signs and symptoms associated with cTTP are highly complex and can vary widely between patients.1,8 They can range from asymptomatic disease, to a single episode and chronic-relapsing forms, and multiorgan failure.1,8 An increased awareness of the clinical manifestations of cTTP, including patient-reported outcomes, is important in order to provide adequate short-term and long-term treatment.8
- Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.
- Scully M. Blood. 2021;137(25):3469-3471.
- Alwan F, et al. Blood. 2019;133(15):1644-1651.
- Sukumar S, et al. Blood. 2020;136(Suppl 1):22-23. Abstract 377.
- Sakai K, Matsumoto M. J Clin Med. 2023;12(10):3365.
- Nuñez Zuno JA, Khaddour K. Thrombotic Thrombocytopenic Purpura Evaluation and Management. [Updated 2023 Sep 27]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan. Accessed February 2025. Available at: https://www.ncbi.nlm.nih.gov/books/NBK470585/
- Oladapo AO, et al. Patient. 2019;12(5):503-512.
- Oladapo A, et al. J Patient Rep Outcomes. 2023;7(1):68.
cTTP, Congenital thrombotic thrombocytopenic purpura
SmPC, Summary of product characteristics
TTP, Thrombotic thrombocytopenic purpura