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Burden of Morbidity...
Burden of Morbidity after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism : A BMT Survivor Study Report
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- Wadhwa, Aman (author)
- University of Alabama
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- Chen, Yanjun (author)
- University of Alabama
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- Hageman, Lindsey (author)
- University of Alabama
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- Schlichting, Elizabeth (author)
- University of Alabama
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- Funk, Erin (author)
- University of Alabama
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- Hicks, Jessica (author)
- University of Alabama
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- Balas, Nora (author)
- University of Alabama
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- Siler, Arianna (author)
- University of Alabama
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- Wu, Jessica (author)
- University of Alabama
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- Francisco, Liton (author)
- University of Alabama
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- Holmqvist, Anna (author)
- Lund University,Lunds universitet,Pediatrik, Lund,Sektion V,Institutionen för kliniska vetenskaper, Lund,Medicinska fakulteten,Sena effekter efter barncancerbehandling,Forskargrupper vid Lunds universitet,LUCC: Lunds universitets cancercentrum,Övriga starka forskningsmiljöer,Paediatrics (Lund),Section V,Department of Clinical Sciences, Lund,Faculty of Medicine,Late effects after childhood cancer treatment,Lund University Research Groups,LUCC: Lund University Cancer Centre,Other Strong Research Environments,Skåne University Hospital
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- Gupta, Ashish (author)
- University of Minnesota
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- Lund, Troy (author)
- University of Minnesota
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- Orchard, Paul J. (author)
- University of Minnesota
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- Armenian, Saro (author)
- Beckman Research Institute of City of Hope
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- Arora, Mukta (author)
- University of Minnesota
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- Bhatia, Smita (author)
- University of Alabama
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(creator_code:org_t)
- Elsevier BV, 2022
- 2022
- English.
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In: Transplantation and cellular therapy. - : Elsevier BV. - 2666-6367. ; 28:3, s. 1-157
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Abstract
Subject headings
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- Survival after blood or marrow transplantation (BMT) for inborn errors of metabolism (IEM) is excellent; however, the burden of morbidity in long-term survivors of BMT for IEM remains understudied. This study examined the risk of chronic health conditions (CHC) in ≥2-year survivors of allogeneic BMT for IEM performed between 1974 and 2014 using the BMT Survivor Study. In this retrospective cohort study, participants (or their parents; n = 154) reported demographic data and CHCs (graded using Common Terminology Criteria for Adverse Events version 5), and transplantation characteristics were obtained from institutional databases. Unaffected siblings (n = 494) served as a comparison group. Logistic regression was used to estimated the odds of severe/life-threatening CHCs compared with siblings. Cox proportional hazards regression was used to estimate factors associated with severe/life-threatening/fatal CHCs in survivors of BMT for IEM. Survivors of allogeneic BMT for IEM (leukodystrophies, 43.5%; mucopolysaccharidoses, 41.0%) were at 12.5-fold higher odds of severe/life-threatening CHCs (95% confidence interval [CI], 5.4 to 28.9) compared with their siblings. The mean 10-year post-BMT cumulative incidence of grade 3-5 CHCs was 47.5 ± 4.0%. Reduced-intensity conditioning (RIC) was associated with a 2.7-fold higher risk (95% CI, 1.2 to 6.2; P = .02) of any grade 3-5 CHC, a 6.7-fold higher risk of grade 3-5 cardiopulmonary conditions (95% CI, 1.3 to 35.4), and a 3.0-fold higher risk of severe hearing/vision deficits (95% CI, 1.4 to 6.6). Older (age >26 years) BMT survivors were significantly less likely to graduate from college (odds ratio [OR], 0.3; 95% CI, 0.1 to 0.7) or marry (OR, 0.01; 95% CI, 0.004 to 0.07) compared with their siblings. Survivors of BMT for IEM carry a significant burden of morbidities, which affects their ability to attain adult milestones. Efforts to reduce chronic health conditions in this population are needed.
Subject headings
- 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)
Keyword
- Blood or marrow transplantation
- Chronic health conditions
- Inborn errors of metabolism
- Morbidity
Publication and Content Type
- art (subject category)
- ref (subject category)
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To the university's database
- By the author/editor
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Wadhwa, Aman
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Chen, Yanjun
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Hageman, Lindsey
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Schlichting, Eli ...
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Funk, Erin
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Hicks, Jessica
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show more...
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Balas, Nora
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Siler, Arianna
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Wu, Jessica
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Francisco, Liton
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Holmqvist, Anna
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Gupta, Ashish
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Lund, Troy
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Orchard, Paul J.
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Armenian, Saro
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Arora, Mukta
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Bhatia, Smita
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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Cancer and Oncol ...
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Hematology
- Articles in the publication
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Transplantation ...
- By the university
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Lund University