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

• Published in: Haematologica (Roma) Vol. 108; no. 12; pp. 3298 - 3307
• Main Authors: Nakamura, Ryotaro, Patel, Bhavisha A, Kim, Soyoung, Wong, F Lennie, Armenian, Saro H, Groarke, Emma M, Keesler, Daniel A, Hebert, Kyle M, Heim, Michael, Eapen, Mary, Young, Neal S
• Format: Journal Article
• Language: English
• Published: Italy Ferrata Storti Foundation 01-12-2023
• Subjects: Anemia, Aplastic; Hematopoietic Stem Cell Transplantation - adverse effects; Humans; Immunosuppression Therapy; Immunosuppressive Agents - therapeutic use; Infant; Transplantation, Homologous
• ISSN: 0390-6078; 1592-8721; 1592-8721
• DOI: 10.3324/haematol.2023.282781

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 compare 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. Given changes to treatment regimens and differences in length of follow-up, two treatment periods were defined a priori: 2000-2010 and 2011-2018. The SMR of patients treated during the period 2000-2010 and who survived one 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 SMR 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 five years. For patients who underwent HCT during 2000-2010 and 2011-2018, their mortality risk became comparable to the general population after ten years and after five years, respectively. Thus, 1-year survivors after IST or HCT can expect their longevity beyond five years to be comparable to that of the general US population.