<p>Discussion remains concerning the safety and tolerability of anti-thymocyte globulin (ATG)-based immunosuppressive therapy (IST) in older patients with aplastic anaemia (AA). Using data of 127 consecutive patients from the Dutch adult AA registry, we evaluated long-term treatment success of standard ATG-based IST as first-line treatment with a multi-state model. Only one death was potentially associated with ATG. Overall survival at 5 years was 79%. We defined Transplantation-, Treatment- and Disease-Free Survival (TT-DFS) as the ultimate success of this treatment. This means that a patient is alive, is currently transfusion-independent without having received an allogeneic stem cell transplantation, has not developed AML or MDS, and has stopped all medication for AA. The probability of TT-DFS was 42% at 5 years after start of IST. In patients younger than 40 years (<i>n</i> = 36) and in patients aged 60 or above (<i>n</i> = 53), this was 58% and 34%, respectively. Older age, more severe AA and absence of a PNH-clone of ≥ 1% all reduced the likelihood of reaching TT-DFS. These analyses on unselected nationwide data indicate that ATG-based IST is effective and safe also in older patients. They suggest that age, AA severity and presence of a PNH-clone should be taken into account when considering this treatment in older patients.</p>

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Anti-thymocyte globulin-based treatment frequently leads to enduring treatment success in both old and young adult patients with aplastic anaemia: a real-world analysis from the Dutch aplastic anaemia registry

  • C. J. M. Halkes,
  • E. A. S. Koster,
  • E. J. M. Bogers,
  • F. C. J. I. Heubel-Moenen,
  • L. G. M. Daenen,
  • S. K. Klein,
  • S. M. C. Langemeijer,
  • E. Nur,
  • M. H. G. Raaijmakers,
  • T. J. F. Snijders,
  • J. M. L. Tjon,
  • L. C. de Wreede

摘要

Discussion remains concerning the safety and tolerability of anti-thymocyte globulin (ATG)-based immunosuppressive therapy (IST) in older patients with aplastic anaemia (AA). Using data of 127 consecutive patients from the Dutch adult AA registry, we evaluated long-term treatment success of standard ATG-based IST as first-line treatment with a multi-state model. Only one death was potentially associated with ATG. Overall survival at 5 years was 79%. We defined Transplantation-, Treatment- and Disease-Free Survival (TT-DFS) as the ultimate success of this treatment. This means that a patient is alive, is currently transfusion-independent without having received an allogeneic stem cell transplantation, has not developed AML or MDS, and has stopped all medication for AA. The probability of TT-DFS was 42% at 5 years after start of IST. In patients younger than 40 years (n = 36) and in patients aged 60 or above (n = 53), this was 58% and 34%, respectively. Older age, more severe AA and absence of a PNH-clone of ≥ 1% all reduced the likelihood of reaching TT-DFS. These analyses on unselected nationwide data indicate that ATG-based IST is effective and safe also in older patients. They suggest that age, AA severity and presence of a PNH-clone should be taken into account when considering this treatment in older patients.