Very long-term follow-up of aplastic anemia treated with immunosuppressive therapy or allogeneic hematopoietic cell transplantation

• DOI: 10.1007/s00277-020-04271-4
• Publication Date: 2020-09-18T23:07:44Z
• Authors: Beatrice Drexler; Felicitas Zurbriggen; Tamara Diesch; Romaine Viollier; Joerg P. Halter; Dominik Heim; Andreas Holbro; Laura Infanti; Andreas Buser; Sabine Gerull; Michael Medinger; André Tichelli; Jakob R. Passweg
• Keywords: Adult; Aged, 80 and over; Immunosuppression Therapy; Male; Adolescent; Hematopoietic Stem Cell Transplantation; Anemia, Aplastic; Infant; Recovery of Function; Middle Aged; Allografts; Hematopoiesis; 3. Good health; 03 medical and health sciences; 0302 clinical medicine; Child, Preschool; Humans; Original Article; Female; Child; Aged; Follow-Up Studies; Retrospective Studies

AbstractIntroductionSince the 1970s outcome of aplastic anemia (AA) patients has improved significantly due to the introduction of immunosuppressive therapy (IST) and allogeneic hematopoietic transplantation (HCT). However, patients may suffer from persistent disease, relapse, clonal evolution, graft-versus-host disease and other late effects. Here, we analyse very long-term outcome of all AA patients at our institution comparing not only survival, but also response status and complications.MethodsPatient charts of all 302 AA patients treated between 1973 and 2017 at the University Hospital Basel, Switzerland, were retrospectively analysed.ResultsFirst line treatment was IST in 226 (75%) and HCT in 76 (25%) patients. Overall survival at 30 years was similar in patients treated initially by HCT and IST (44% (±14%), and 40% (± 9%) respectively, with better results in more recent years. Partial and no response occurred more frequently after IST, relapse incidence after IST was 24 %, whereas non-engraftment and graft failure was documented in 15 patients (19 %) after HCT. Clonal evolution to myelodysplastic syndrome / acute myeloid leukemia was 16 % at 25 years in IST patients, 1.3 % in HCT patients, iron overload (18 versus 4 %, p = 0.002) and cardiovascular events (11 versus 1 %, p=0.011) occured significantly more often in IST than HCT treated patients. The majority of long-term survivors, 96% of those alive at 25 years, were in complete remission at last follow up, irrespective of the initial treatment modality.ConclusionVery long term survivors after AA are those with stable hematopoietic recovery.