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Prophylaxis is a recommended approach for some patients with congenital thrombotic thrombocytopenic purpura (cTTP): 1 1. Zheng XL, et al. J Thromb Haemost. 2020; 18(10):2696-2502. 2 2. Scully M, et al. Br J Haematol. 2023;203:546-563.
- 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 1. Zheng XL, et al. J Thromb Haemost. 2020; 18(10):2696-2502.
- The British Society for Haematology (BSH) and the Swedish TTP treatment recommendations also recommends that pregnant women with cTTP receive regular therapy prophylactically to prevent clinical TTP relapse. 2 2. Scully M, et al. Br J Haematol. 2023;203:546-563. 3 3. https://www.socialstyrelsen.se/kunskapsstod-och-regler/omraden/sallsynt…
- The BSH also 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 2. Scully M, et al. Br J Haematol. 2023;203:546-563.
In a study reporting on prospective data on 87 patients from the Hereditary TTP Registry of patients, the annual incidence of acute episodes 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. 4 4. Tarasco E, et al. Blood. 2021;137(25):3563–3575.
Did you know?
A literature review, followed by expert haematologistsÈ 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 Uo TTP registry, can sometimes take years
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5. Alwan F, et al. Blood. 2019;133(15):1644–1651.
), is largely impacted by treatment and travel burden, and emotional consequences of cTTP (e.g. anxiety and depression).
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6. Oladapo AO, et al. Patient. 2019;12(5):503–512.
Addressing the unmet needs of cTTP patients 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. 7 7. Coppo P, et al. Blood. 2023;142(Suppl 1):4006.
Acronyms
ADAMTS13, A disintegrin and metalloproteinase with a thrombospondin motifs 13
BSH, British Society for Haematology
CI, Confidence interval
cTTP, Congenital TTP
ISTH, International Society on Thrombosis and Haemostasis
SmPC, Summary of product characteristics
TTP, Thrombotic thrombocytopenic purpura
The long-term burden of disease
Uncover the life-long impact cTTP can have on patients.
