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

David Buchbinder, Diane J. Nugent, Ruta Brazauskas, Zhiwei Wang, Mahmoud D. Aljurf, Mitchell S. Cairo, Robert Chow, Christine Duncan, Lamis K. Eldjerou, Vikas Gupta, Gregory A. Hale, Joerg Halter, Brandon M. Hayes-Lattin, Jack W. Hsu, David A. Jacobsohn, Rammurti T. Kamble, Kimberly A. Kasow, Hillard M. Lazarus, Paulette Mehta, Kasiani C. MyersSusan K. Parsons, Jakob R. Passweg, Joseph Pidala, Vijay Reddy, Carmen M. Sales-Bonfim, Bipin N. Savani, Adriana Seber, Mohamed L. Sorror, Amir Steinberg, William A. Wood, Donna A. Wall, Jacek H. Winiarski, Lolie C. Yu, Navneet S. Majhail

Research output: Contribution to journalArticle

21 Scopus citations

Abstract

With improvements in hematopoietic cell transplant (HCT) outcomes for severe aplastic anemia (SAA), there is a growing population of SAA survivors after HCT. However, there is a paucity of information regarding late effects that occur after HCT in SAA survivors. This study describes the malignant and nonmalignant late effects in survivors with SAA after HCT. A descriptive analysis was conducted of 1718 patients post-HCT for acquired SAA between 1995 and 2006 reported to the Center for International Blood and Marrow Transplant Research (CIBMTR). The prevalence and cumulative incidence estimates of late effects are reported for 1-year HCT survivors with SAA. Of the HCT recipients, 1176 (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 transplantation 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 1 late effect and 1% had multiple late effects. For 1-year survivors of URD HCT, 13% had 1 late effect and 2% had multiple late effects. Among survivors of MSD HCT, the cumulative incidence estimates of developing late effects were all <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 5 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, must be counseled about, and should be monitored for late effects for the remainder of their lives.

Original languageEnglish (US)
Pages (from-to)1776-1784
Number of pages9
JournalBiology of Blood and Marrow Transplantation
Volume18
Issue number12
DOIs
StatePublished - Dec 2012

Keywords

  • Allogeneic
  • Hematopoietic cell transplant
  • Late effects
  • Severe aplastic anemia
  • Survivorship

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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    Buchbinder, D., Nugent, D. J., Brazauskas, R., Wang, Z., Aljurf, M. D., Cairo, M. S., Chow, R., Duncan, C., Eldjerou, L. K., Gupta, V., Hale, G. A., Halter, J., Hayes-Lattin, B. M., Hsu, J. W., Jacobsohn, D. A., Kamble, R. T., Kasow, K. A., Lazarus, H. M., Mehta, P., ... Majhail, N. S. (2012). 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. Biology of Blood and Marrow Transplantation, 18(12), 1776-1784. https://doi.org/10.1016/j.bbmt.2012.06.018