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

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

Author Buchbinder, David Google Scholar
Nugent, Diane J. Google Scholar
Brazauskas, Ruta Google Scholar
Wang, Zhiwei Google Scholar
Aljurf, Mahmoud D. Google Scholar
Cairo, Mitchell S. Google Scholar
Chow, Robert Google Scholar
Duncan, Christine Google Scholar
Eldjerou, Lamis K. Google Scholar
Gupta, Vikas Google Scholar
Hale, Gregory A. Google Scholar
Halter, Joerg Google Scholar
Hayes-Lattin, Brandon M. Google Scholar
Hsu, Jack W. Google Scholar
Jacobsohn, David A. Google Scholar
Kamble, Rammurti T. Google Scholar
Kasow, Kimberly A. Google Scholar
Lazarus, Hillard M. Google Scholar
Mehta, Paulette Google Scholar
Myers, Kasiani C. Google Scholar
Parsons, Susan K. Google Scholar
Passweg, Jakob R. Google Scholar
Pidala, Joseph Google Scholar
Reddy, Vijay Google Scholar
Sales-Bonfim, Carmen M. Google Scholar
Savani, Bipin N. Google Scholar
Seber, Adriana Autor UNIFESP Google Scholar
Sorror, Mohamed L. Google Scholar
Steinberg, Amir Google Scholar
Wood, William A. Google Scholar
Wall, Donna A. Google Scholar
Winiarski, Jacek H. Google Scholar
Yu, Lolie C. Google Scholar
Majhail, Navneet S. Google Scholar
Institution Childrens Hosp Orange Cty
CIBMTR Med Coll Wisconsin
Med Coll Wisconsin
King Faisal Specialist Hosp & Res Ctr
New York Med Coll
Dana Farber Canc Inst
Univ Florida
Princess Margaret Hosp
Univ S Florida
Univ Basel Hosp
Oregon Hlth & Sci Univ
Childrens Natl Med Ctr
Baylor Coll Med
Univ N Carolina Hosp
Univ Hosp Case
Univ Arkansas Med Sci
Cincinnati Childrens Hosp Med Ctr
Tufts Med Ctr
Florida Ctr Cellular Therapy
Univ Fed Parana
Vanderbilt Univ
Universidade Federal de São Paulo (UNIFESP)
Fred Hutchinson Canc Res Ctr
Mt Sinai Med Ctr
Univ Manitoba
Karolinska Univ Hosp
Louisiana State Univ
Dept Natl Marrow Donor Program
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 I 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.
Keywords Hematopoietic cell transplant
Severe aplastic anemia
Late effects
Language English
Sponsor Public Health Service Grant from the National Cancer Institute
National Heart, Lung, and Blood Institute
National Institute of Allergy and Infectious Diseases
National Cancer Institute
Health Resources and Services Administration/Department of Health and Human Services
Office of Naval Research
Grant number Public Health Service Grant from the National Cancer Institute: U24-CA76518
National Heart, Lung, and Blood Institute: 5U01HL069294
Office of Naval Research: N00014-06-1-0704
Office of Naval Research: N00014-08-1-0058
Date 2012-12-01
Published in Biology of Blood and Marrow Transplantation. New York: Elsevier B.V., v. 18, n. 12, p. 1776-1784, 2012.
ISSN 1083-8791 (Sherpa/Romeo, impact factor)
Publisher Elsevier B.V.
Extent 1776-1784
Access rights Open access Open Access
Type Article
Web of Science ID WOS:000311593900003

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