OUTCOMES OF IMMUNOSUPPRESSIVE THERAPY OVER THREE DECADES IN A BRAZILIAN COHORT OF PATIENTS WITH APLASTIC ANAEMIA

@article{pereira2026outcomes,
  title = {{OUTCOMES OF IMMUNOSUPPRESSIVE THERAPY OVER THREE DECADES IN A BRAZILIAN COHORT OF PATIENTS WITH APLASTIC ANAEMIA}},
  author = {Thales Dalessandro Meneguin PEREIRA and Vanderson ROCHA and Balduino BIASOLI and Valeria BUCCHERI and Elvira Deolinda Rodrigues Pereira VELLOSO},
  journal = {Hematology, Transfusion and Cell Therapy},
  year = {2026},
  month = oct,
  doi = {10.1016/j.htct.2026.106582},
  url = {https://doi.org/10.1016/j.htct.2026.106582},
  note = {CC BY},
}

TY  - JOUR
TI  - OUTCOMES OF IMMUNOSUPPRESSIVE THERAPY OVER THREE DECADES IN A BRAZILIAN COHORT OF PATIENTS WITH APLASTIC ANAEMIA
AU  - Thales Dalessandro Meneguin PEREIRA
AU  - Vanderson ROCHA
AU  - Balduino BIASOLI
AU  - Valeria BUCCHERI
AU  - Elvira Deolinda Rodrigues Pereira VELLOSO
PY  - 2026
DA  - 2026/10/23
JO  - Hematology, Transfusion and Cell Therapy
DO  - 10.1016/j.htct.2026.106582
UR  - https://doi.org/10.1016/j.htct.2026.106582
AB  - Introduction: Aplastic anaemia is a rare disease, characterized by cytopenias and hypocellular bone marrow. Haematopoietic stem cell transplantation (HSCT) is the treatment modality associated with the best outcomes. Another therapy option is immunosuppression, most commonly composed of antithymocyte globulin (ATG, in most cases rabbit-derived), plus ciclosporin (CSA) with or without eltrombopag. Objectives: To evaluate the outcomes of immunosuppressive treatment (IST) in a single-centre cohort of adult patients with aplastic anaemia over three decades at a tertiary referral centre, thereby contributing data on how the disease is managed in Brazil. Materials and Methods: Data from 300 patients were reviewed over the past three decades, including characteristics at diagnosis, treatment, response and survival. Results were presented with 95% confidence intervals (95% CI), with p<0.05 considered statistically significant. Results: Disease incidence was 10 cases/year, with 96% of cases originating from the state of São Paulo; median age at diagnosis was 26 years (max. 84 years). Congenital bone marrow failure syndromes accounted for 13% (n=40) of cases. Around 70% of patients required treatment at diagnosis. A positive PNH clone at diagnosis was found in 36% (n = 73) of patients investigated (median clone size 11%); 7% presented with thrombosis. Regarding treatment, 40 patients underwent HSCT. Eighty-three patients received IST with ATG and CSA, with eltrombopag added to IST in 10 cases. Due to lack of response, 6 patients were retreated with a further course of IST. Thirty-one patients received CSA and eltrombopag. Alemtuzumab was used only as salvage treatment, in 17 cases. Overall response rates (ORR) at 6 months were: 85.7% for HSCT, 91.8% for ATG, 90% for CSA + eltrombopag, and 62.5% for alemtuzumab. Overall survival (OS) among patients treated with ATG-containing regimens remained stable throughout follow-up (87.6% at 2-years; median follow-up 6.6-years). There was no significant difference in OS by decade of treatment, treatment line (first-line vs salvage) or disease severity. However, patients under 40-years of age had better OS (92.2% vs. 76%). The alemtuzumab-treated group had a 2-year OS of 72.2%. On univariate analysis, factors associated with a higher risk of death after ATG were haemoglobin (HR = 0.76, 95% CI 0.58–0.98), neutrophil count at diagnosis (HR=0.42, 95% CI 0.19–0.85) and decade of diagnosis (HR = 1.09, 95% CI 1.02–1.17); platelets, age, time from diagnosis to treatment, PNH clone and aplasia severity were not statistically significant. On multivariate analysis, the only variable that retained significance was neutrophil count (HR = 0.57, 95% CI 0.38–0.87, p = 0.0086). The cumulative incidence of relapse was 18.8% (95% CI 10.2–29.4) and of clonal evolution (MDS/AML) was 4.5% (95% CI 1.2–11.4) at 5 years (both with death as a competing event), with no significant difference between IST groups in either case. Discussion and conclusion: In this single-centre Brazilian cohort with 30 years of follow-up, response rates to IST were comparable to those reported in the literature using horse ATG, with stable overall survival across decades and a relapse incidence comparable to that previously reported. Alemtuzumab proved to be a viable salvage option, albeit with inferior response rates. These findings help elucidate how aplastic anaemia is managed in Brazil, clarifying the real availability and equity of these therapeutic options beyond reference centres.
SN  - 2531-1379
SN  - 2531-1387
ER  -