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.
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DOI：
10.1016/j.bbmt.2012.06.018
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发表时间：
2012-12
影响因子：
4.3
通讯作者：
Majhail, Navneet S.
中科院分区：
文献类型：
--
作者：
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;Sorror, Mohamed L.;Steinberg, Amir;Wood, William A.;Wall, Donna A.;Winiarski, Jacek H.;Yu, Lolie C.;Majhail, Navneet S.
随着重型再生障碍性贫血（SAA）造血细胞移植（HCT）结局的改善，HCT后SAA幸存者人数不断增加。然而，关于SAA幸存者HCT后发生的晚期效应的信息很少。本研究描述了HCT后SAA幸存者的恶性和非恶性晚期效应。对1995-2006年期间向CIBMTR报告的1,718例HCT后获得性SAA患者进行了描述性分析。报告了SAA患者1年HCT存活者的晚期效应患病率和累积发生率。在HCT受者中，分别有1，176例（68.5%）和542例（31.5%）患者接受了匹配的同胞供体（MSD）或无关供体（URD）HCT。HCT时的中位年龄为20岁。从诊断到移植的中位间隔时间为3个月，MSD HCT和14个月，URD HCT。MSD和URD HCT幸存者的中位随访时间分别为70个月和67个月。整个队列的1年、2年和5年总生存率分别为76%（95%置信区间[CI]：74-78）、73%（95% CI：71-75）和70%（95% CI：68-72）。在MSD HCT的1年存活者中，6%有一种晚期效应，1%有多种晚期效应。对于URD HCT的1年存活者，13%有一个晚期效应，2%有多个晚期效应。在MSD HCT的存活者中，发生晚期效应的累积发生率均低于3%，且未随时间增加。相反，对于URD HCT的接受者，5年内发生几种晚期效应的累积发生率超过3%：性腺功能障碍10.5%（95% CI：7.3-14.3），生长障碍7.2%（95% CI：4.4-10.7）、缺血性坏死6.3%（95% CI：3.6-9.7）、甲状腺功能减退5.5%（95% CI：2.8-9.0）和白内障5.1%（95% CI：2.9-8.0）。我们的研究结果表明，所有接受HCT治疗SAA的患者仍有晚期效应的风险，必须对其进行咨询，并应在其余生中监测晚期效应。
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.
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影响因子：
20.3
作者：
Deeg, H. Joachim;O'Donnell, Margaret;Storer, Barry
通讯作者：
Storer, Barry
影响因子：
20.3
作者：
Sanders, Jean E.;Woolfrey, Ann E.;Storb, Rainer F.
通讯作者：
Storb, Rainer F.
影响因子：
6.5
作者：
Kahl, C;Leisenring, W;Storb, R
通讯作者：
Storb, R
DOI：
10.3324/haematol.2010.026682
发表时间：
2010-12-01
期刊：
HAEMATOLOGICA-THE HEMATOLOGY JOURNAL
影响因子：
--
作者：
Gupta, Vikas;Eapen, Mary;Marsh, Judith C. W.
通讯作者：
Marsh, Judith C. W.
影响因子：
20.3
作者：
Deeg, HJ;Socie, G;Storb, R
通讯作者：
Storb, R