To assess very long-term outcomes of children with severe aplastic anemia (SAA) and impact of histopathology and of different treatments over time.
We conducted a retrospective study of 57 consecutive patients with SAA during 1973-2019. According to period, treatment consisted of androgens, immunosuppressive treatment (IST) and hematopoietic cell transplantation (HCT) in 14, 31 and 13 patients, respectively. Histopathology immune profiles were studied on bone marrow (BM).
Response rate (RR) to androgens was 35%, with long-term survivorship in 4 of 5 responders. RR and 10-year overall survival (OS) after IST was 65% and 80%, respectively. RR was higher in girls (92% vs. 43% in boys, P=0.02). Mean baseline BM values of CD34+ and of B-lymphocytes in responders vs. non-responders were 1.3% vs. 0 (P=0.08) and 14.1% vs. 9.7% (P=0.07), respectively. After IST, BM cellularity gradually increased and cytotoxic T-lymphocytes decreased (time variation P=0.003 and 0.07, respectively). Outcome did not differ between patients with IST or frontline HCT. Ten-year OS improved over time, increasing from 35.3% to 77.1% and 77% during 1973-1985, 1986-2003 and 2004-2019, respectively.
Histopathology may refine response prediction to IST. The course of SAA in children, a previously fatal disease, was altered in recent times.

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