Late Effects in Hematopoietic Cell Transplant Recipients with Acquired Severe Aplastic Anemia: A Report from the Late Effects Working Committee of the Center for International Blood and Marrow Transplant Research

摘要

With improvements in hematopoietic cell transplantation (HCT) outcomes for severe aplastic anemia (SAA), there is a growing population of SAA survivors following HCT. However, there is a paucity of information regarding late effects that occur after HCT in SAA survivors. This study describes the malignant and non-malignant late effects in survivors with SAA following HCT. A descriptive analysis was conducted of 1,718 patients post-HCT for acquired SAA between 1995–2006 reported to the CIBMTR. The prevalence and cumulative incidences of late effects are reported for 1-year HCT survivors with SAA. Of the HCT recipients, 1,176 (68.5%) and 542 (31.5%) patients underwent a matched sibling donor (MSD) or unrelated donor (URD) HCT, respectively. The median age at the time of HCT was 20 years. The median interval from diagnosis to transplant was 3 months for MSD HCT and 14 months for URD HCT. The median follow-up was 70 months and 67 months for MSD and URD HCT survivors, respectively. Overall survival at 1 year, 2 years, and 5 years for the entire cohort was 76% (95% confidence interval [CI]: 74–78), 73% (95% CI: 71–75), and 70% (95% CI: 68–72). Among 1-year survivors of MSD HCT, 6% had one late effect and 1% had multiple late effects. For 1-year survivors of URD HCT, 13% had one late effect and 2% had multiple late effects. Among survivors of MSD HCT, the cumulative incidences of developing late effects were all less than 3% and did not increase over time. In contrast, for recipients of URD HCT, the cumulative incidence of developing several late effects exceeded 3% by five years: gonadal dysfunction 10.5% (95% CI: 7.3–14.3), growth disturbance 7.2% (95% CI: 4.4–10.7), avascular necrosis 6.3% (95% CI: 3.6–9.7), hypothyroidism 5.5% (95% CI: 2.8–9.0), and cataracts 5.1% (95% CI: 2.9–8.0). Our results indicated that all patients undergoing HCT for SAA remain at-risk for late effects and must be counseled about and should be monitored for late effects for the remainder of their lives.