Conditional survival and standardized mortality ratios of patients with severe aplastic anemia surviving at least one year after hematopoietic cell transplantation or immunosuppressive therapy

Immunosuppressive treatment (IST) and hematopoietic cell transplant (HCT) are standard therapies for severe aplastic anemia (SAA). We report on conditional survival and standardized mortality ratios (SMR), which compares the mortality risk with the general population adjusted for age, gender, and race/ethnicity, in patients with SAA alive for at least 12 months after treatment with IST or HCT between 2000 and 2018. Two treatment periods were defined a priori (2000-2010, 2011-2018), given changes to treatment regimens and differences in length of follow-up. The SMRs of patients treated during the period 2000-2010 and survived 1 year were 3.50 (95% confidence interval [CI]: 2.62-4.58), 4.12 (95% CI 3.20-5.21), and 8.62 (95% CI 6.88-10.67) after IST, matched related donor HCT, and alternative donor HCT, respectively. For the period 2011-2018, the corresponding SMRs were 2.89 (95% CI 1.54-4.94), 3.12 (95% CI 1.90-4.82), and 4.75 (95% CI 3.45-6.38), respectively. For IST patients, their mortality risk decreased over time, and became comparable to the general population by 5 years. For patients who underwent HCT during 2000-2010 and 2011-2018, their mortality risk became comparable to the general population after 10 years and after 5 years, respectively. Thus, 1-year survivors after IST or HCT can expect their longevity beyond 5-years to be comparable to that of the US population.