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Title: 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
Authors: Buchbinder, David
Nugent, Diane J.
Brazauskas, Ruta
Wang, Zhiwei
Aljurf, Mahmoud D.
Cairo, Mitchell S.
Chow, Robert
Duncan, Christine
Eldjerou, Lamis K.
Gupta, Vikas
Hale, Gregory A.
Halter, Joerg
Hayes-Lattin, Brandon M.
Hsu, Jack W.
Jacobsohn, David A.
Kamble, Rammurti T.
Kasow, Kimberly A.
Lazarus, Hillard M.
Mehta, Paulette
Myers, Kasiani C.
Parsons, Susan K.
Passweg, Jakob R.
Pidala, Joseph
Reddy, Vijay
Sales-Bonfim, Carmen M.
Savani, Bipin N.
Seber, Adriana [UNIFESP]
Sorror, Mohamed L.
Steinberg, Amir
Wood, William A.
Wall, Donna A.
Winiarski, Jacek H.
Yu, Lolie C.
Majhail, Navneet S.
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
Keywords: Hematopoietic cell transplant
Severe aplastic anemia
Late effects
Issue Date: 1-Dec-2012
Publisher: Elsevier B.V.
Citation: Biology of Blood and Marrow Transplantation. New York: Elsevier B.V., v. 18, n. 12, p. 1776-1784, 2012.
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.
ISSN: 1083-8791
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