The long-term burden of disease

Patients with congenital thrombotic thrombocytopenic purpura (cTTP) experience disease- and treatment-related complications and burdens that have a negative impact on their health-related quality of life (HRQoL).¹ In a study integrating inputs from a literature review, expert opinion and patient interviews, patients interviewed by telephone indicated that the most salient impacts of cTTP were the diminished ability to work/study, financial distress, feeling depressed, feeling anxious, and mood swings.² A multinational chart review study found that, besides the impact on the patient, managing patients with cTTP can exert a burden on healthcare systems, as many patients require multiple hospital and ICU stays.³
 

For patients needing ADAMTS13 supplementation, prophylactic fresh-frozen plasma (FFP) infusion can prove burdensome.⁴

• 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
• Second, FFP includes the ADAMTS13 protein as well as other proteins that can trigger allergic reactions and may carry a risk of pathogen transmission to patients. These allergic reactions can vary from hives to life-threatening anaphylactic shock, with a higher incidence observed in patients receiving prophylactic FFP compared to those treated with FFP on an on-demand basis.⁴ Registry data has shown that 58% of patients receiving prophylactic FFP infusions required premedication against allergic reactions before each infusion, including steroids, antihistamine agents, and anti-allergic medicines.⁴

Existing research underscores the profound impact of cTTP on various facets of patients lives. Efforts to develop more patientfriendly management approaches that minimise disruptions to daily life and reduce treatment-related burdens are essential.^1-3