Unmet needs of patients with cTTP
Prophylaxis is a recommended approach for some patients with congenital thrombotic thrombocytopenic purpura (cTTP):1,2
- For pregnant women with cTTP, the International Society on Thrombosis and Haemostasis (ISTH) recommends prophylactic treatment over no prophylactic treatment to reduce maternal and infant mortality and morbidity rates.1
- The British Society for Haematology (BSH) recommends that pregnant women with cTTP receive regular therapy prophylactically to prevent clinical thrombotic thrombocytopenic purpura (TTP) relapse.2
- The BSH recommends that ADAMTS13 prophylaxis should be considered for all patients with cTTP, with an individualized approach to dose and frequency according to symptoms, whether overt or non-overt.2
In a study reporting on prospective data on 87 patients from the Hereditary Thrombotic Thrombocytopenic Purpura (TTP) Registry of patients (NCT01257269), the annual incidence of acute events was 0.36 (95% confidence interval [CI], 0.29-0.44) with regular plasma treatment and 0.41 (95% CI, 0.30-0.56) without regular plasma treatment.3
Did you know?
A literature review, followed by expert haematologists interviews and telephone interviews of adults
with cTTP, revealed that the life of patients with cTTP, once a diagnosis is made (which, according
to analysis of a UK TTP registry, can sometimes take years4), is largely impacted by treatment and
travel burden, and emotional consequences of cTTP (e.g. anxiety and depression).5
Addressing the unmet needs of patients with cTTP requires a comprehensive approach that considers the challenges associated with the current standard of care. A multinational chart review study suggested that TTP patients care journey could be improved by novel, less burdensome therapies with demonstrated efficacy and safety profiles.6
Patient Stories
In this powerful video, Jill shares her journey of being diagnosed with cTTP as a child and the profound impact it has had on her life. From early struggles to finding strength, her story is one of resilience and hope.
Watch Jill's story:
This video features real patient(s) sharing their personal experience. Experience and view may change over time and from one person to another. This video is available to the general public for informational purposes only; it should not be used for diagnosing or treating a health condition or disease. It is not intended to substitute for consultation with a healthcare provider.
- Zheng XL, et al. J Thromb Haemost. 2020;18(10):2496-2502.
- Scully M, et al. Br J Haematol. 2023;203(4),546 563.
- Tarasco E, et al. Blood. 2021;137(25):3563 3575.
- Alwan F, et al. Blood. 2019;133(15):1644-1651.
- Oladapo AO, et al. Patient. 2019;12(5):503-512.
- Coppo P, et al. Blood. 2023;142(Suppl 1):4006.
ADAMTS13, A disintegrin and metalloproteinase with thrombospondin motifs 13
BSH, British Society for Haematology
CI, Confidence interval
cTTP, Congenital thrombotic thrombocytopenic purpura
ISTH, International Society on Thrombosis and Haemostasis
SmPC, Summary of product characteristics
TTP, Thrombotic thrombocytopenic purpura
UK, United Kingdom
