What is it about?
This is an update of a previously reported case of thrombotic thrombocytopenic purpura (TTP) that was refractory to plasma exchange, steroids, vincristine, rituximab and N-acetylcysteine, but achieved remission after the institution of eculizumab. This case report suggested that eculizumab might be an effective treatment of TTP. However, upon further investigation, it was discovered that the patient had concurrent atypical hemolytic uremic syndrome (AHUS) due to autoantibodies of complement factor H. His persistent thrombocytopenia was due to AHUS rather than TTP. The patient achieved remission because eculizumab controlled his AHUS.
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Why is it important?
In a patient with TTP that is "refractory" to treatment, it is important to consider the existence of other causes of thrombocytopenia, hemolysis, or organ dysfunction.
Perspectives
In conventional syndrome-based approaches, TTP is only diagnosed in patients presenting with the diad of thrombocytopenia and microangiopathic hemolytic anemia (MAHA); the triad of thrombocytopenia, MAHA, and neurologic deficits; or the pentad of thrombocytopenia, MAHA, neurologic deficits, renal abnormalities and fever. In this scheme, it is impossible to distinguish TTP from AHUS, which is defined by the triad of thrombocytopenia, MAHA and renal function impairment. Only with pathogenesis based approach is it possible to clearly distinguish TTP from AHUS and other causes of the syndrome of thrombocytopenia and MAHA.
Dr Han Mou Tsai
Han Mou Tsai
Read the Original
This page is a summary of: Use of eculizumab in the treatment of a case of refractory, ADAMTS13-deficient thrombotic thrombocytopenic purpura: additional data and clinical follow-up, British Journal of Haematology, May 2013, Wiley,
DOI: 10.1111/bjh.12387.
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