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Potential Risks to Stable Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Children With Cerebral X-linked Adrenoleukodystrophy
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“HLA-matched sibling donors are available for less than 20% of patients but historically have led to longer survival than that with HSCT from unrelated donors.”
From Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy (2024) · cites this paper as [13]
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Cites
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., et al. (2009) · Science · doi:10.1126/science.1171242 · Cited by 1,508 · cited 2×
“Among the symptomatic patients, 11 of 18 (61%) survived, but only 1 was EFS (6%) (median [IQR] EFS time, 9 [3-22] months).”
Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy
Eichler, F., Duncan, C., Musolino, P. L., Orchard, P. J., De Oliveira, S., Thrasher, A. J., Armant, M., Dansereau, C., Lund, T. C., Miller, W. P., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1700554 · Cited by 604 · cited 2×
“Among the symptomatic patients, 11 of 18 (61%) survived, but only 1 was EFS (6%) (median [IQR] EFS time, 9 [3-22] months).”
Aubourg, P., Blanche, S., Jambaqué, I., Rocchiccioli, F., Kalifa, G., Naud-Saudreau, C., Rolland, M.-O., Debré, M., Chaussain, J.-L., Griscelli, C., et al. (1990) · New England Journal of Medicine · doi:10.1056/nejm199006283222607 · Cited by 366
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Analysis of MRI patterns aids prediction of progression in X-linked adrenoleukodystrophy
Loes, D. J., Fatemi, A., Melhem, E. R., Gupte, N., Bezman, L., Moser, H. W., & Raymond, G. V. (2003) · Neurology · doi:10.1212/01.wnl.0000079050.91337.83 · Cited by 268
Berger, J., Wiesinger, C., & Eichler, F. (2015) · The Application of Clinical Genetics · doi:10.2147/tacg.s49590 · Cited by 140
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Haematopoietic stem cell transplantation in 12 patients with cerebral X-linked adrenoleukodystrophy
Baumann, M., Korenke, C., Weddige-Diedrichs, A., Wilichowski, E., Hunneman, D., Wilken, B., Brockmann, K., Klingebiel, T., Niethammer, D., Kühl, J., et al. (2003) · European Journal of Pediatrics · doi:10.1007/s00431-002-1097-3 · Cited by 47
Self-citation
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Cited by
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
Eichler, F., Duncan, C. N., Musolino, P. L., Lund, T. C., Gupta, A. O., De Oliveira, S., Thrasher, A. J., Aubourg, P., Kühl, J.-S., Loes, D. J., et al. (2024) · New England Journal of Medicine · doi:10.1056/nejmoa2400442 · Cited by 107
Self-citationTreatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy
Gupta, A. O., Raymond, G., Pierpont, E. I., Kemp, S., McIvor, R. S., Rayannavar, A., Miller, B., Lund, T. C., & Orchard, P. J. (2022) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2022.2124857 · Cited by 67
“The pattern of white matter involvement is varied, but often the splenium is the area where changes are first observed, and in about 70% of the cases the cerebral disease is primarily located in the occipital area (Figure 1) [33,34].”
BackgroundNeurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophy
Pierpont, E. I., Nascene, D. R., Shanley, R., Kenney-Jung, D. L., Ziegler, R. S., Miller, W. P., Gupta, A. O., Lund, T. C., Orchard, P. J., & Eisengart, J. B. (2020) · Neurology · doi:10.1212/wnl.0000000000009929 · Cited by 21
Metadata
- Authors
- Kühl, J.-S., Kupper, J., Baqué, H., Ebell, W., Gärtner, J., Korenke, C., Spors, B., Steffen, I. G., Strauss, G., Voigt, S., Weschke, B., Weddige, A., Köhler, W., & Steinfeld, R.
- Affiliations
- Department of Pediatric Hematology/Oncology/Hemostaseology, University Hospital Leipzig, Leipzig, Germany
- Department of Pediatric Oncology/Hematology/SCT, Charité Campus Virchow-Klinikum, Berlin, Germany
- Department of Pediatric Neurology, Charité Campus Virchow-Klinikum, Berlin, Germany
- Department of Pediatric Neurology, University Medical Center Göttingen, Göttingen, Germany
- Department of Pediatric Neurology, Klinikum Oldenburg, Oldenburg, Germany
- Department of Pediatric Radiology, Charité Campus Virchow-Klinikum, Berlin, Germany
- Department of Pediatrics, Helios-Klinikum Berlin-Buch, Berlin, Germany
- Department of Neurology, University Hospital Leipzig, Leipzig, Germany
- Venue
- JAMA Network Open
- Publisher
- American Medical Association (AMA)
- Published
- 2018-07-20
- Volume
- 1
- Issue
- 3
- Pages
- e180769
- ISSN
- 2574-3805
- PMC
- PMC6324299
- Cited by
- 43
- References
- 8
- Language
- en
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- Kühl, J.-S., Kupper, J., Baqué, H., Ebell, W., Gärtner, J., Korenke, C., Spors, B., Steffen, I. G., Strauss, G., Voigt, S., Weschke, B., Weddige, A., Köhler, W., & Steinfeld, R. (2018). Potential Risks to Stable Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Children With Cerebral X-linked Adrenoleukodystrophy. JAMA Network Open. https://doi.org/10.1001/jamanetworkopen.2018.0769
