2.1 Study design, patients, and treatment
This study retrospectively analyzed 20 patients who underwent allogeneic HSCT and were confirmed as having TA-TMA in the Department of Hematology and Oncology, Wuhan Children’s Hospital from August 2016 to December 2021. TA-TMA was diagnosed according to the criteria proposed by the Hematopoietic Stem Cell Application Group, Chinese Society of Hematology, Chinese Medical Association3. The criteria for inclusion were: lactate dehydrogenase (LDH) levels increased to upper normal limit, proteinuria, hypertension, thrombocytopenia <50 × 109/L or a 50% decrease in platelet count, decreased hemoglobin, evidence of microangiopathy, and plasma sC5b-9 values above the upper limit of normal. If 3 of the 7 laboratory or clinical indicators were met, or if tissue biopsy indicators had evidence of microthrombosis, then a diagnosis of TA-TMA was confirmed.
Once diagnosis of TA-TMA was established, the dose of cyclosporine and tacrolimus (CNI) was immediately reduced. Plasma exchange, rituximab, low molecular weight heparin, calcium, defibrotide, eculizumab, and other supportive treatments were administered.