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Late Mortality afte...
Late Mortality after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism : A Report from the Blood or Marrow Transplant Survivor Study-2 (BMTSS-2)
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- Wadhwa, Aman (författare)
- University of Alabama
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- Chen, Yanjun (författare)
- University of Alabama
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- Holmqvist, Anna (författare)
- Lund University,Lunds universitet,Pediatrik, Lund,Sektion V,Institutionen för kliniska vetenskaper, Lund,Medicinska fakulteten,Paediatrics (Lund),Section V,Department of Clinical Sciences, Lund,Faculty of Medicine,Skåne University Hospital
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- Wu, Jessica (författare)
- University of Alabama
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- Ness, Emily (författare)
- University of Alabama
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- Parman, Mariel (författare)
- University of Alabama
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- Kung, Michelle (författare)
- University of Alabama
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- Hageman, Lindsey (författare)
- University of Alabama
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- Francisco, Liton (författare)
- University of Alabama
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- Braunlin, Elizabeth (författare)
- University of Minnesota
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- Miller, Weston (författare)
- Sangamo Therapeutics,University of Minnesota
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- Lund, Troy (författare)
- University of Minnesota
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- Armenian, Saro (författare)
- City of Hope National Medical Center
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- Arora, Mukta (författare)
- University of Minnesota
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- Orchard, Paul (författare)
- University of Minnesota
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- Bhatia, Smita (författare)
- University of Alabama
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(creator_code:org_t)
- Elsevier BV, 2019
- 2019
- Engelska.
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Ingår i: Biology of Blood and Marrow Transplantation. - : Elsevier BV. - 1083-8791. ; 25:2, s. 328-334
- Relaterad länk:
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http://dx.doi.org/10...
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http://www.bbmt.org/...
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https://lup.lub.lu.s...
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https://doi.org/10.1...
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Abstract
Ämnesord
Stäng
- Allogeneic blood or marrow transplantation (BMT) is currently considered the standard of care for patients with specific inborn errors of metabolism (IEM). However, there is a paucity of studies describing long-term survival and cause-specific late mortality after BMT in these patients with individual types of IEM. We studied 273 patients who had survived ≥2 years after allogeneic BMT for IEM performed between 1974 and 2014. The most prevalent IEM in our cohort were X-linked adrenoleukodystrophy (ALD; 37.3%), Hurler syndrome (35.1%), and metachromatic leukodystrophy (MLD; 10.2%). Conditional on surviving ≥2 years after BMT, the overall survival for the entire cohort was 85.5 ± 2.4% at 10 years and 73.5 ± 3.7% at 20 years. The cohort had a 29-fold increased risk of late death compared with an age- and sex-matched cohort from the general US population (95% CI, 22- to 38-fold). The increased relative mortality was highest in the 2- to 5-year period after BMT (standardized mortality ratio [SMR], 207; 95% confidence interval [CI], 130 to 308) and declined with increasing time from BMT, but remained elevated for ≥21 years after BMT (SMR, 9; 95% CI, 4 to 18). Sequelae from the progression of primary disease were the most common causes of late mortality in this cohort (76%). The use of T cell-depleted grafts in patients with ALD and Hurler syndrome was a risk factor for late mortality. Younger age at BMT and use of busulfan and cyclosporine were protective in patients with Hurler syndrome. Our findings demonstrate relatively favorable overall survival in ≥2-year survivors of allogeneic BMT for IEM, although primary disease progression continues to be responsible for the majority of late deaths.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Cancer och onkologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Cancer and Oncology (hsv//eng)
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
Nyckelord
- Blood or marrow transplantation
- Inborn errors of metabolism
- Late mortality
Publikations- och innehållstyp
- art (ämneskategori)
- ref (ämneskategori)
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Till lärosätets databas
- Av författaren/redakt...
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Wadhwa, Aman
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Chen, Yanjun
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Holmqvist, Anna
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Wu, Jessica
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Ness, Emily
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Parman, Mariel
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visa fler...
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Kung, Michelle
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Hageman, Lindsey
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Francisco, Liton
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Braunlin, Elizab ...
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Miller, Weston
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Lund, Troy
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Armenian, Saro
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Arora, Mukta
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Orchard, Paul
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Bhatia, Smita
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