Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report
Abstract
Cerebral adrenoleukodystrophy (cALD) remains a devastating neurodegenerative disease; only allogeneic hematopoietic cell transplantation (HCT) has been shown to provide long-term disease stabilization and survival. Sixty boys undergoing HCT for cALD from 2000 to 2009 were analyzed. The median age at HCT was 8.7 years; conditioning regimens and allograft sources varied. At HCT, 50% demonstrated a Loes radiographic severity score ≥ 10, and 62% showed clinical evidence of neurologic dysfunction. A total of 78% (n = 47) are alive at a median 3.7 years after HCT. The estimate of 5-year survival for boys with Loes score < 10 at HCT was 89%, whereas that for boys with Loes score ≥ 10 was 60% (P = .03). The 5-year survival estimate for boys absent of clinical cerebral disease at HCT was 91%, whereas that for boys with neurologic dysfunction was 66% (P = .08). The cumulative incidence of transplantation-related mortality at day 100 was 8%. Post-transplantation progression of neurologic dysfunction depended significantly on the pre-HCT Loes score and clinical neurologic status. We describe the largest single-institution analysis of survival and neurologic function outcomes after HCT in cALD. These trials were registered at www.clinicaltrials.gov as #NCT00176904, #NCT00668564, and #NCT00383448.
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Patients displaying mild neurological impairment along with discernible MRI involvement appear to achieve the most favorable outcomes after HCT.
“Patients displaying mild neurological impairment along with discernible MRI involvement appear to achieve the most favorable outcomes (46, 47).”
From X-linked adrenoleukodystrophy and primary adrenal insufficiency (2023) · cites this paper as [47]
Allogeneic hematopoietic cell transplantation (HCT) has the potential to arrest the progression of the disease in individuals with inflammatory cerebral involvement.
“Allogenic hematopoietic cell transplantation (HCT) has the potential to arrest the progression of the disease in individuals with inflammatory cerebral involvement.”
From X-linked adrenoleukodystrophy and primary adrenal insufficiency (2023) · cites this paper as [47]
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- Authors
- Miller, W. P., Rothman, S. M., Nascene, D., Kivisto, T., DeFor, T. E., Ziegler, R. S., Eisengart, J., Leiser, K., Raymond, G., Lund, T. C., Tolar, J., & Orchard, P. J.
- Affiliations
- University of Minnesota (US)
- Kennedy Krieger Institute (US)
- ORCID
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2011-08-18
- Volume
- 118
- Issue
- 7
- Pages
- 1971-1978
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- OpenAlex
- W2147261346
- Cited by
- 290
- References
- 6
- Citations by year
- 2026: 15 · 2025: 16 · 2024: 19 · 2023: 11 · 2022: 22 · 2021: 30 · 2020: 19 · 2019: 32 · 2018: 25 · 2017: 26 · 2016: 18 · 2015: 14 · 2014: 17 · 2013: 10 · 2012: 13
- FWCI
- 9.13
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Blood (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Health Sciences › Medicine › Hematology › Hematopoietic Stem Cell Transplantation
- Keywords
- cerebral adrenoleukodystrophy, allogeneic hematopoietic stem cell transplantation, childhood, survival, neurological function, neurologic dysfunction, transplantation-related mortality, disease progression, conditioning regimen
- MeSH terms
- Adolescent, Adrenoleukodystrophy, Child, Child, Preschool, Graft vs Host Disease, Humans, Infant, Male, Transplantation, Homologous, Cohort Studies, Survival Analysis, Treatment Outcome, Hematopoietic Stem Cell Transplantation, Disease Progression, Transplantation Conditioning, Young Adult
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