Clinical characteristics of pediatric allogeneic hematopoietic stem cell transplantation-associated thrombotic microangiopathy (TA-TMA): a retrospective single-center analysis

• Linlin Luo ^[1] ; Hao Xiong ^[2] ; Zhi Chen ^[2] ; Li Yang ^[2] ; Ming Sun ^[2] ; Wenjie Lu ^[2] ; Fang Tao ^[2] ; Zhuo Wang ^[2] ; Jianxin Li ^[2] ; Zuofeng Li ^[3] ; Sujie Tang ^[1]
  1. [1] Medical College of Jianghan University, Wuhan, 430056, Hubei Province, China
  2. [2] Wuhan Children’s Hospital (Wuhan Maternal and Child Healthcare Hospital), Wuhan, 430016, Hubei Province, China
  3. [3] Medical College of Wuhan University of Science and Technology, Wuhan, 430065, Hubei Province, China

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• Localización: Clinical & translational oncology, ISSN 1699-048X, Vol. 25, Nº. 8 (August), 2023, págs. 2451-2461
• Idioma: inglés
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• Resumen

  • Objectives To investigate the clinical features of thrombotic microangiopathy associated with allogeneic hematopoietic stem cell transplantation in children.

    Methods A retrospective analysis of continuous clinical data from HSCT received in the Department of Hematology and Oncology of Wuhan Children's Hospital from August 1, 2016 to December 31, 2021.

    Results During this period, 209 patients received allo-HSCT in our department, 20 (9.6%) of whom developed TA-TMA. TA-TMA was diagnosed at a median of 94 (7–289) days post-HSCT. Eleven (55%) patients had early TA-TMA within 100 days post-HSCT, while the other 9 (45%) patients had TA-TMA thereafter. The most common symptom of TA-TMA was ecchymosis (55%), while the main signs were refractory hypertension (90%) and multi-cavity effusion (35%). Five (25%) patients had central nervous system symptoms (convulsions and lethargy). All 20 patients had progressive thrombocytopenia, with 16 patients receiving transfusion of platelets that was ineffective. Ruptured red blood cells were visible in only two patients with peripheral blood smears. Cyclosporine A or Tacrolimus (CNI) dose was reduced once TA-TMA was diagnosed. Nineteen cases were treated with low-molecular-weight heparin, 17 patients received plasma exchange, and 12 patients were treated with rituximab. TA-TMA-related mortality percentage in this study was 45% (9/20).

    Conclusion Platelet decline and/or ineffective transfusion post-HSCT should be considered an early indicator of TA-TMA in pediatric patients. TA-TMA in pediatric patients may occur without evidence of peripheral blood schistocytes. Aggressive treatment is required once diagnosis is confirmed, but the long-term prognosis is poor.