Among these, thrombocytopenia and acute kidney injury were consistent with the clinical syndrome of thrombotic microangiopathy (TMA), with each case including reduced complement factor C3 or complement factor C4 levels, reduced platelets, evidence of hemolysis and changes in serum creatinine and cystatin C. After medical management, including 1–2 doses of intravenous eculizumab (600 mg) and platelet transfusion (n = 1) or dialysis (n = 1), both TEAEs resolved within 15 days. The gene discussed is CST3; the disease is Thrombocytopenia.