Prognosis

Patient registry data collected between 2000 and 2011 in Sweden reported 5-year survival of around 61% in all patients with aplastic anaemia (AA).[13]

In patients with non severe AA, 5-year survival was approximately 81% in a study using data collected from 1998 to 2018 in the US.[102]​ Some spontaneously remit; others may live many years without progression of disease.

The prognosis of patients with severe or very severe AA has markedly improved over the last 30 years. The 5-year survival with immunosuppressive therapy, using horse antithymocyte globulin (ATG) plus ciclosporin, is around 75% to 80%, but lower with rabbit ATG.[13][84]​​ However, survival rate may be age-dependent. In one study, younger patients (aged 19 to 39 years) treated with immunosuppressive therapy had a significantly higher 5-year survival than older patients (aged ≥60 years) treated with immunosuppressive therapy (90% vs. 52%, respectively).[13] ​A study using data from patients with severe AA in the US reported that among patients who survived at least one year after stem cell transplant (SCT), or immunosuppressive treatment, 5-year survival from 2011 to 2018 was comparable to the US population.[103]

With SCT, young patients with a matched related donor have a probability of long-term survival >80%; for older patients the prognosis is not quite as good. Outcomes after matched unrelated donor SCT have improved significantly, such that overall survival is similar to matched related donor SCT when using ATG-based conditioning regimens, although with a higher risk of graft-versus-host disease (GVHD).[104] In contrast, the use of alemtuzumab-based conditioning is associated with a lower risk of GVHD in the setting of unrelated donor SCT.[105]​ One meta-analysis of studies published between 2015 and 2019 found that 5-year survival was greater with upfront alternative donor transplantation (matched unrelated donor or HLA haploidentical donor) compared with both salvage alternative donor transplantation and immunosuppressive therapy.[106]

The impact of best supportive care is extremely important for patients in all stages of the disease. Developments in supportive care have led to a reduction in infection-related deaths and thereby improved the survival of refractory patients.[107]

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