Abstract
IntroductionSince 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.
Highlights
Since the 1970s outcome of aplastic anemia (AA) patients has improved significantly due to the introduction of immunosuppressive therapy (IST) and allogeneic hematopoietic transplantation (HCT)
Aplastic anemia (AA) is a rare hematological disease characterized by bone marrow failure causing pancytopenia with
Median age at diagnosis was 24 years (1-80 years), 18 years (1-66 years) in patients treated by hematopoietic cell transplantation (HCT) compared to 27 years (IQR 1-80 years) in the IST group
Summary
Median follow up of surviving patients was 17 years (range: 1-41 years). Median age at diagnosis was 24 years (1-80 years), 18 years (1-66 years) in patients treated by HCT compared to 27 years (IQR 1-80 years) in the IST group. We defined current event free survival (cEFS) as the probability of being alive and in > partial remission even after several lines of treatment at the time of last treatment; e.g. a patient with IST, relapsing after. Disease-related complications Bleeding and infection were common (42.1% in the HCT and 56.2 % the IST group, respectively 89.5% and 90.7%, p=0.088 and 0.751), but its occurrence did not differ significantly between the two treatment groups (Table 3). In long-term survivors death was mainly due to malignancy (42.8%) in the HCT group, whereas in post-IST long-term surviving patients the majority of death were caused by typical chronic conditions in the elderly such as heart disease
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