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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
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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
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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
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Journal Article
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
2026
Overview
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.
Publisher
Springer Berlin Heidelberg,Springer Nature B.V,Springer
Subject
/ Adult
/ Aged
/ Anemia
/ Anemia, Aplastic - drug therapy
/ Anemia, Aplastic - epidemiology
/ Anemia, Aplastic - mortality
/ Antilymphocyte Serum - administration & dosage
/ Antilymphocyte Serum - adverse effects
/ Antilymphocyte Serum - therapeutic use
/ Disease
/ Female
/ Humans
/ Immune suppressive treatment
/ Immunosuppressive Agents - therapeutic use
/ Male
/ Medicine
/ Oncology
/ Patients
/ Success
/ Toxicity
