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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. Socialstyrelsen – Trombotisk trombocytopen purpura; https://www.socialstyrelsen.se/kunskapsstod-och-regler/omraden/sallsynta-halsotillstand/om-kunskapsdatabasen/sok-bland-sallsynta-halsotillstand/trombotisk-trombocytopen-purpura/}

✓ 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}

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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}

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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 ^{5 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). ^{6 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