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Burden of Morbidity after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism : A BMT Survivor Study Report

Wadhwa, Aman ; Chen, Yanjun ; Hageman, Lindsey ; Schlichting, Elizabeth ; Funk, Erin ; Hicks, Jessica ; Balas, Nora ; Siler, Arianna ; Wu, Jessica and Francisco, Liton , et al. (2022) In Transplantation and cellular therapy 28(3). p.1-157
Abstract

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... (More)

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.

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type
Contribution to journal
publication status
published
subject
keywords
Blood or marrow transplantation, Chronic health conditions, Inborn errors of metabolism, Morbidity
in
Transplantation and cellular therapy
volume
28
issue
3
pages
1 - 157
publisher
Elsevier
external identifiers
  • scopus:85122302710
  • pmid:34879288
ISSN
2666-6367
DOI
10.1016/j.jtct.2021.11.023
language
English
LU publication?
yes
id
15950fc5-e84e-438c-bb79-7d8c9a30e55c
date added to LUP
2022-03-03 13:44:23
date last changed
2024-06-13 11:17:36
@article{15950fc5-e84e-438c-bb79-7d8c9a30e55c,
  abstract     = {{<p>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 &gt;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.</p>}},
  author       = {{Wadhwa, Aman and Chen, Yanjun and Hageman, Lindsey and Schlichting, Elizabeth and Funk, Erin and Hicks, Jessica and Balas, Nora and Siler, Arianna and Wu, Jessica and Francisco, Liton and Holmqvist, Anna and Gupta, Ashish and Lund, Troy and Orchard, Paul J. and Armenian, Saro and Arora, Mukta and Bhatia, Smita}},
  issn         = {{2666-6367}},
  keywords     = {{Blood or marrow transplantation; Chronic health conditions; Inborn errors of metabolism; Morbidity}},
  language     = {{eng}},
  number       = {{3}},
  pages        = {{1--157}},
  publisher    = {{Elsevier}},
  series       = {{Transplantation and cellular therapy}},
  title        = {{Burden of Morbidity after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism : A BMT Survivor Study Report}},
  url          = {{http://dx.doi.org/10.1016/j.jtct.2021.11.023}},
  doi          = {{10.1016/j.jtct.2021.11.023}},
  volume       = {{28}},
  year         = {{2022}},
}