Post-transplant adaptive function in childhood cerebral adrenoleukodystrophy

Elizabeth I. Pierpont, Erin McCoy, Kelly E. King, Rich S. Ziegler, Ryan Shanley, David Nascene, Gerald V. Raymond, Rachel Phelan, Troy C. Lund, Paul J. Orchard, Weston P. Miller

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

Objective: Hematopoietic stem cell transplantation (HSCT) is the only treatment known to slow or halt inflammatory demyelination among boys with the cerebral form of X-linked adrenoleukodystrophy (cALD), a devastating childhood condition affecting the central nervous system. HSCT can lead to a range of adverse outcomes including fatality. Previous studies have examined the potential predictors of post-HSCT survival and neurologic functioning. However, little is known about patients' daily-life adaptive functional outcomes (i.e., ability to communicate, maintain social relationships, and independently execute tasks of daily living). The purpose of this retrospective cohort study was to identify which patient characteristics and treatment-related variables predict long-term adaptive function among the survivors of HSCT for cALD. Methods: We obtained caregiver ratings of adaptive functioning of 65 transplant survivors at an average of 4.6 years (range: 1.0–24.1 years) post-HSCT. Using linear regression with penalized maximum likelihood estimation, we modeled the relative contribution of pre-transplant neurocognitive test performance, MRI severity, transplant regimen, and length of time since transplant on patient adaptive functioning outcomes. Results: Higher radiographic disease severity and poorer performance on baseline neurocognitive tests requiring fine motor skills and visual perception were associated with inferior adaptive functioning after HSCT. Use of radiation during the transplant preparative regimen also predicted poorer adaptive outcomes. Interpretation: In addition to radiological disease severity, baseline neurocognitive test performance is associated with post-transplant adaptive functional outcomes. Neurocognitive measures may play an important role in prognostic counseling and post-transplant treatment planning for patients considering HSCT for cALD.

Original languageEnglish (US)
Pages (from-to)252-261
Number of pages10
JournalAnnals of Clinical and Translational Neurology
Volume5
Issue number3
DOIs
StatePublished - Mar 2018

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Adrenoleukodystrophy
Hematopoietic Stem Cell Transplantation
Transplants
Survivors
Visual Perception
Motor Skills
Aptitude
Demyelinating Diseases
Nervous System
Caregivers
Counseling
Linear Models
Cell Survival
Cohort Studies
Therapeutics
Central Nervous System
Retrospective Studies
Radiation

All Science Journal Classification (ASJC) codes

  • Neuroscience(all)
  • Clinical Neurology

Cite this

Pierpont, E. I., McCoy, E., King, K. E., Ziegler, R. S., Shanley, R., Nascene, D., ... Miller, W. P. (2018). Post-transplant adaptive function in childhood cerebral adrenoleukodystrophy. Annals of Clinical and Translational Neurology, 5(3), 252-261. https://doi.org/10.1002/acn3.526
Pierpont, Elizabeth I. ; McCoy, Erin ; King, Kelly E. ; Ziegler, Rich S. ; Shanley, Ryan ; Nascene, David ; Raymond, Gerald V. ; Phelan, Rachel ; Lund, Troy C. ; Orchard, Paul J. ; Miller, Weston P. / Post-transplant adaptive function in childhood cerebral adrenoleukodystrophy. In: Annals of Clinical and Translational Neurology. 2018 ; Vol. 5, No. 3. pp. 252-261.
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Pierpont, EI, McCoy, E, King, KE, Ziegler, RS, Shanley, R, Nascene, D, Raymond, GV, Phelan, R, Lund, TC, Orchard, PJ & Miller, WP 2018, 'Post-transplant adaptive function in childhood cerebral adrenoleukodystrophy', Annals of Clinical and Translational Neurology, vol. 5, no. 3, pp. 252-261. https://doi.org/10.1002/acn3.526

Post-transplant adaptive function in childhood cerebral adrenoleukodystrophy. / Pierpont, Elizabeth I.; McCoy, Erin; King, Kelly E.; Ziegler, Rich S.; Shanley, Ryan; Nascene, David; Raymond, Gerald V.; Phelan, Rachel; Lund, Troy C.; Orchard, Paul J.; Miller, Weston P.

In: Annals of Clinical and Translational Neurology, Vol. 5, No. 3, 03.2018, p. 252-261.

Research output: Contribution to journalArticle

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T1 - Post-transplant adaptive function in childhood cerebral adrenoleukodystrophy

AU - Pierpont, Elizabeth I.

AU - McCoy, Erin

AU - King, Kelly E.

AU - Ziegler, Rich S.

AU - Shanley, Ryan

AU - Nascene, David

AU - Raymond, Gerald V.

AU - Phelan, Rachel

AU - Lund, Troy C.

AU - Orchard, Paul J.

AU - Miller, Weston P.

PY - 2018/3

Y1 - 2018/3

N2 - Objective: Hematopoietic stem cell transplantation (HSCT) is the only treatment known to slow or halt inflammatory demyelination among boys with the cerebral form of X-linked adrenoleukodystrophy (cALD), a devastating childhood condition affecting the central nervous system. HSCT can lead to a range of adverse outcomes including fatality. Previous studies have examined the potential predictors of post-HSCT survival and neurologic functioning. However, little is known about patients' daily-life adaptive functional outcomes (i.e., ability to communicate, maintain social relationships, and independently execute tasks of daily living). The purpose of this retrospective cohort study was to identify which patient characteristics and treatment-related variables predict long-term adaptive function among the survivors of HSCT for cALD. Methods: We obtained caregiver ratings of adaptive functioning of 65 transplant survivors at an average of 4.6 years (range: 1.0–24.1 years) post-HSCT. Using linear regression with penalized maximum likelihood estimation, we modeled the relative contribution of pre-transplant neurocognitive test performance, MRI severity, transplant regimen, and length of time since transplant on patient adaptive functioning outcomes. Results: Higher radiographic disease severity and poorer performance on baseline neurocognitive tests requiring fine motor skills and visual perception were associated with inferior adaptive functioning after HSCT. Use of radiation during the transplant preparative regimen also predicted poorer adaptive outcomes. Interpretation: In addition to radiological disease severity, baseline neurocognitive test performance is associated with post-transplant adaptive functional outcomes. Neurocognitive measures may play an important role in prognostic counseling and post-transplant treatment planning for patients considering HSCT for cALD.

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