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Patients with cTTP experience disease- and treatment-related complications and burdens that have a negative impact on their HRQoL. ^{1 1. Oladapo A, et al. J Patient Rep Outcomes. 2023;7(1):68.} In a literature review, the most salient impacts of cTTP - among the patients interviewed by telephone - included diminished ability to work/study, financial distress, feeling depressed, feeling anxious, and mood swings. ^{2 2. Oladapo AO, et al. Patient. 2019;12(5):503–512.} A multinational chart review study found that, besides the impact on the patient, managing the manifestations of cTTP can also exert a burden on healthcare systems, as many patients require multiple hospital and ICU stays. ^{3 3. Coppo P, et al. Blood. 2023;142(Suppl 1):4006.}

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In patients requiring ADAMTS13 supplementation, prophylactic FFP infusion is often burdensome in multiple ways: ^{4 4. Sakai K et al. J Clin Med. 2023;12(10):3365.}

• First, FFP infusions are performed frequently (once every 1–3 weeks) and only in a hospital setting. Moreover, due to its high volume, FFP administration takes several hours of infusion, and care must be taken to avoid volume overload in patients with impaired cardiac or renal function. ^{4 4. Sakai K et al. J Clin Med. 2023;12(10):3365.}
• Second, FFP contains not only the ADAMTS13 protein but also other proteins that may cause allergic reactions and can potentially transmit pathogens to patients. Allergic reactions can range from hives to life-threatening anaphylactic shock and are more apparent in patients receiving prophylactic FFP compared to those receiving on-demand FFP. Registry data has shown that 58% of patients receiving prophylactic FFP infusion required premedication against allergic reactions before each FFP infusion, including steroids, antihistamine agents, and anti-allergic medicines. ^{4 4. Sakai K et al. J Clin Med. 2023;12(10):3365.}

Existing research underscores the profound impact of cTTP on various facets of patients' lives. Efforts to develop more patient-friendly management approaches that minimise disruptions to daily life and reduce treatment-related burdens is essential. ^{1 1. Alwan F, et al. Blood. 2019;133(15):1644–1651. 3 3. Coppo P, et al. Blood. 2023;142(Suppl 1):4006.}

Acronyms

ADAMTS13, A disintegrin and metalloproteinase with a thrombospondin motifs 13
cTTP, Congenital TTP
ICU, Intensive care unit
FFP, Fresh frozen plasma
HRQoL, Health-related QoL
SmPC, Summary of product characteristics
TTP, Thrombotic thrombocytopenic purpura