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Unmet needs of cTTP patients

Recommendations for prophylactic treatment of cTTP patients:

  • 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.
  • The British Society for Haematology (BSH) also recommends that pregnant women with cTTP receive regular therapy prophylactically to prevent clinical TTP relapse.
  • 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.
     
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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

 

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Did you know?

A literature review, followed by expert haematologists interviews and telephone interviews with cTTP adults, revealed that the life of patients with cTTP, once a diagnosis is made, is largely impacted by treatment and travel burden, and emotional consequences of cTTP (e.g., anxiety and depression).5

 

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.6


 

References and acronyms

  1. Scully M, et al. N Engl J Med. 2024;390(17):1584-1596.
  2. Scully M, et al. Br J Haematol. 2023;203(4):546-563.
  3. Zheng XL, et al. J Thromb Haemost. 2020;18(10):2496 2502.
  4. Tarasco E, et al. Blood. 2021;137(25):3563 3575.
  5. Oladapo AO, et al. Patient. 2019;12(5):503 512.
  6. Coppo P, et al. Blood. 2023;142(Suppl 1):4006.

 

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

 

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What causes cTTP?

Congenital thrombotic thrombocytopenic purpura (cTTP) is caused by autosomal recessive mutations in the ADAMTS13 gene.1,2

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