Program DHC-Virtual
20 - 21 January 2021
Clinical Abstracts session 2
Aplastic Anaemia in the Netherlands
20 January
10:24 10:36
L. Bogers

Treatment with ATGAM leads to enduring haematological responses and 3-year survival probability of 84% in adult acquired aplastic anaemia patients in the Netherlands

Lisette Bogers (1), Liesbeth de Wreede (2), Marco de Groot (3), Saskia Langemeijer (4), Harry Koene (5), Ellen Meijer (6), Erfan Nur (6), Marc Raaijmakers (7), Reinier Raymakers (8), Tjeerd Snijders (9), Jennifer Tjon (1), Stijn Halkes (1)
(1) LUMC, Hematology, Leiden, (2) LUMC, Biomedical Data Sciences, Leiden, (3) UMCG, Hematology, Groningen, (4) Radboud UMC, Hematology, Nijmegen, (5) Antonius Ziekenhuis, Hematology, Nieuwegein, (6) Amsterdam-UMC, Hematology, Amsterdam, (7) Erasmus MC, Hematology, Rotterdam, (8) UMCU, Hematology, Utrecht, (9) Medisch Spectrum Twente, Hematology, Enschede
No potential conflicts of interest

Acquired aplastic anaemia(AA) is characterized by bone marrow failure. First-line treatment is either allogeneic stem cell transplantation(alloSCT) or intensive immunosuppressive therapy(IST) consisting of horse-derived Anti-Thymocyte globulin(ATGAM) and ciclosporin. Patients who are refractory 6 months after first-line treatment with IST can be treated with second-line alloSCT, rabbit-derived ATG(Thymoglobulin) or Eltrombopag, a Thrombopoietin-mimetic. In 2013, a national registry was started in which seven university hospitals and two large non-academic hospitals collect data on all consecutive adult AA patients, that received ATGAM and ciclosporin as first-line treatment. We present the outcomes of 103 patients from this registry.


Data on adults with AA who received ISTas first-line treatment was collected in the LUMC, A-UMC, UMCG, UMCU, RadboudUMC, Erasmus MC, Medisch spectrum Twente and Antonius Ziekenhuis Nieuwegein. The data included baseline-characteristics and follow-up data at 3, 6, 9 and 12 months. After 12 months, follow-up data was collected yearly. Response was defined as transfusion independency and neutrophil count >0.5 x 109/L. Overall survival(OS) was evaluated with the Kaplan-Meier method.


In October 2020, 103 patients treated with first-line IST consisting of ATGAM and ciclosporin were registered. Median age at start of treatment was 56 years (18-83). Median follow-up time was 30 months. OS probability at 36 months was 84% (CI 76-91%). Three patients died within six months after start of treatment and for 5 patients follow up time was less than 6 months. Of 95 evaluable patients, response rate at 6 months was 61% (CI 51-70%). From 37 non responding(NR) patients at 6 months, 11 continued ciclosporin without additional treatment of whom 9 became transfusion independent up to 16 months after ATGAM treatment. 26 NR patients received second-line treatment with alloSCT (n=7), Thymoglobulin (n=6), Eltrombopag (n=12) or Danazol (n=1) and 17 of them responded. Eight of the 9 patients without response to second-line treatment died. Among the 58 patients with initial response at 6 months, AA relapsed in 15. Three relapsing patients were treated with supportive care and in 3 patients ciclosporin was reintroduced (2 responses). Nine patients received second-line treatment (3 alloSCT, 6 eltrombopag) of whom 4 had a response and 4 of the NR patients died.


First-line treatment with ATGAM and ciclosporin in adult AA patients leads to a 6 month response rate of 61%. The majority of patients with refractory or relapsing disease do respond to second-line treatment). However, 12 of the 14 patients without a response to second-line therapy died as a result of bone marrow failure or treatment related toxicity. Future recommendations should aim to improve the outcome of second-line treatment in AA patients.