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 (CIBMTR)

Abstract

With improvements in hematopoietic cell transplantation (HCT) for severe aplastic anemia (SAA), there is a growing population of SAA survivors of HCT. However, there is a paucity of information regarding late effects in SAA HCT survivors. We analyzed the burden of malignant and non-malignant late effects in HCT survivors with acquired SAA. A descriptive analysis of 1,718 patients post-HCT for acquired SAA between 1995-2006 reported to the CIBMTR was conducted. Cumulative incidences (CI) of selected late effects are reported for among “condition-free” (i.e. survivors without a previous diagnosis of the specified late effect) HCT survivors with SAA. One-year overall survival for the recipients of related donor HCT was 83% (80-85) and 62% (58-66) for the recipients of unrelated donor HCT. Of the HCT recipients, 1176 (68.5%) patients with a median age at HCT of 20 years (<1-65) utilized a related donor and 542 (31.5%) patients with a median age at HCT of 20 years (<1-67) utilized an unrelated donor. The median interval from diagnosis to transplant was 3 months (<1-348) for related donor HCT and 14 months (1-318) for unrelated donor HCT. Radiation (of any type) was utilized in 6% and 78% of related and unrelated donor HCT cases; respectively. Cy-TBI (68%) was a commonly utilized conditioning regimen for unrelated donor HCT. The median follow-up is 70 months (1-160) and 67 months (3-182) for related and unrelated donor HCT; respectively. The 2-year CI of chronic GVHD was 20% (18-22) and 37% (32-41) in related and unrelated donor HCT; respectively. Table 1 demonstrates that among 1-year condition-free survivors, the CI of late effects is greater among unrelated donor HCT survivors and continues to increase. These findings suggest that HCT survviors with SAA are a robust and healthy group in general. However, subgroups of patients undergoing HCT for SAA are at-risk for late effects and must be educated about and should be monitored for late effects. Subgroups of survivors may be at-risk group for a greater burden of specific late effects. Ongoing analyses in our cohort will explore selected risk-factors for late effects after HCT for SAA.Table 1Interval specific cumulative incidence of select late effects among 1-year condition free survivors following related and unrelated donor HCT for acquired SAA between 1995 and 2006 reported to the CIBMTRHealth conditions post transplantN (at risk)Related DonorN (at risk)Unrelated DonorCumulative Incidence % (95% CI)Cumulative Incidence % (95% CI)Strokes/SeizuresOver next 2 years7071.7 (0.9-2.7)2831.3 (0.3-2.8)Over next 5 years4161.8 (1.0-2.9)1182.8 (1.2-5.1)Gonadal DysfunctionOver next 2 years7092.6 (1.6-3.8)2736.2 (3.8-9.1)Over next 5 years4193.0 (2.0-4.3)10810.5 (7.3-14.3)Renal FailureOver next 2 years7151.1 (0.5-1.9)2871.9 (0.7-3.6)Over next 5 years4251.4 (0.7-2.3)1272.4 (0.9-4.5)Avascular NecrosisOver next 2 years7241.4 (0.7-2.3)2763.2 (1.5-5.4)Over next 5 years4261.8 (1.0-2.8)1176.3 (3.6-9.7)CataractsOver next 2 years7340.6 (0.2-1.2)2842.2 (0.9-4.1)Over next 5 years4331.1 (0.5-1.9)1185.1 (2.9-8.0)Growth DisturbanceOver next 2 years7350.2 (0.0-0.7)2861.9 (0.7-3.6)Over next 5 years4330.5 (0.1-1.2)1137.2 (4.4-10.7)HypothyroidismOver next 2 years7310.7 (0.3-1.4)2860.6 (0.1-1.8)Over next 5 years4321.2 (0.5-2.1)1195.5 (2.8-9.0)Solid TumorsOver next 2 years8200.6 (0.2-1.1)2760.7 (0.1-1.9)Over next 5 years5280.7 (0.2-1.3)1211.4 (0.4-3.0) Open table in a new tab