Abstract
Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare and potentially
lethal disease characterized by fragmentary hemolysis, moderate-to-severe thrombocytopenia,
end-organ dysfunction, and severely reduced ADAMTS13 levels (< 10%). Survival in iTTP
has improved significantly since the introduction of plasma exchange as standard therapy
combined with immune suppression to address the underlying pathophysiology. A host
of challenges remain including prompt recognition of the disease, treatment of the
end-organ effects of the disease, improving the early mortality rate, significantly
reducing the relapse rate as well as addressing refractory disease. Discussed in this
narrative review of iTTP are the recent measures aimed at addressing these issues,
including improvements in clinical prediction models, postremission maintenance approaches
with early retreatment as well as the development of novel therapies.
Keywords
thrombocytopenic purpura - thrombotic microangiopathy - ADAMTS13 - plasma exchange
