The DOI matches its Crossref record; 3 of the 3 references it cites were found in a registry or an index.
Outcomes of Unrelated Umbilical Cord Blood Transplantation for X-Linked Adrenoleukodystrophy
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“Among patients with cerebral adrenoleukodystrophy and other inherited metabolic disorders, allogeneic transplantation has been associated with a 100-day mortality rate of 8%, a 1-year mortality rate of 18%, a rate of graft failure of 6 to 14%, a rate of serious infection of 29%, a rate of acute grade 2, 3, or 4 GVHD of 8 to 45%, and a rate of chronic GVHD of up to 21%.5,10,22–24”
From Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy (2017) · cites this paper as [23]
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Cites
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
Moser, H. W., Loes, D. J., Melhem, E. R., Raymond, G. V., Bezman, L., Cox, C. S., & Lu, S.-E. (2000) · Neuropediatrics · doi:10.1055/s-2000-9236 · Cited by 210
Takemoto, Y., Suzuki, Y., Horibe, R., Shimozawa, N., Wanders, R. J. A., & Kondo, N. (2003) · Brain and Development · doi:10.1016/s0387-7604(03)00033-0 · Cited by 101
Research works printed but not linked: 19
- Moser, 1980, Adrenoleukodystrophy: studies of the phenotype, genetics and biochemistry, Johns Hopkins Med J, 147, 217
- Schaumburg, 1975, Adrenoleukodystrophy, Arch Neurol, 32, 577, 10.1001/archneur.1975.00490510033001 · DOI · Paper page
- Moser, 1994, The gene responsible for adrenoleukodystrophy encodes a peroxisomal membrane protein, Hum Mol Genet, 3, 265, 10.1093/hmg/3.2.265 · DOI · Paper page
- Krivit, 1999, Curr Opin Neurol, 12, 167, 10.1097/00019052-199904000-00007 · DOI
- Suzuki, 2000, Bone marrow transplantation for the treatment of X-linked adrenoleukodystrophy, J Inherit Metab Dis, 23, 453, 10.1023/A:1005656029200 · DOI
- Sauer, 2004, Hematopoietic stem cell transplantation for mucopolysaccharidoses and leukodystrophies, Klin Padiatr, 216, 163, 10.1055/s-2004-822629 · DOI
- Peters, 2004, Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999, Blood, 104, 881, 10.1182/blood-2003-10-3402 · DOI · Paper page
- Gluckman, 1989, Hematopoietic reconstitution in a patient with Fanconi’s anemia by means of umbilical-cord blood from an HLA-identical sibling, N Engl J Med, 321, 1174, 10.1056/NEJM198910263211707 · DOI
- Kurtzberg, 1996, Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients, N Engl J Med, 335, 157, 10.1056/NEJM199607183350303 · DOI
- Martin, 2006, Results of the Cord Blood Transplantation Study (COBLT): outcomes of unrelated umbilcal cord transplantation in pediatric patients with lysosomal and peroxisomal storage disorders, Biol Blood MarrowTransplant, 12, 184, 10.1016/j.bbmt.2005.09.016 · DOI
- Staba, 2004, Cord-blood transplants from unrelated donors with Hurlers syndrome, N Engl J Med, 350, 1960, 10.1056/NEJMoa032613 · DOI
- Escolar, 2005, Transplantation of umbilical-cord blood in babies with infantile Krabbe’s disease, N Engl J Med, 352, 2069, 10.1056/NEJMoa042604 · DOI
- Jacobson, 2001, Oral busulfan pharmacokinetics and engraftment in children with Hurler Syndrome and other inherited metabolic storage diseases undergoing hematopoetic stem cell transplant, Bone Marrow Transplant, 27, 855, 10.1038/sj.bmt.1703010 · DOI
- Rubinstein, 1995, Processing and cryopreservation of placental/umbilical cord blood for unrelated bone marrow reconstitution, Proc Natl Acad Sci U S A, 92, 10119, 10.1073/pnas.92.22.10119 · DOI
- Przepiorka, 1995, 1994 Consensus conference on acute GVHD grading, Bone Marrow Transplant, 15, 825
- Loes, 1994, Adrenoleukodystrophy: a scoring method for brain MR observations, Am J Neuroradiol, 15, 1761
- Society, 1994, Guideline nine: guidelines on evoked potentials, J Clin Neurophysiol, 11, 40, 10.1097/00004691-199401000-00010 · DOI
- Moser, 2005, Follow up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo’s oil, Arch Neurol, 62, 1073, 10.1001/archneur.62.7.1073 · DOI · Paper page
- Smalley, 1966, Two cases of busulfan toxicity, Ann Intern Med, 64, 154, 10.7326/0003-4819-64-1-154 · DOI
Cited by
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
Cartier, N., & Aubourg, P. (2010) · Brain Pathology · doi:10.1111/j.1750-3639.2010.00394.x · Cited by 151
Kemper, A. R., Brosco, J., Comeau, A. M., Green, N. S., Grosse, S. D., Jones, E., Kwon, J. M., Lam, W. K. K., Ojodu, J., Prosser, L. A., et al. (2017) · Genetics in Medicine · doi:10.1038/gim.2016.68 · Cited by 109
Kühl, J.-S., Suarez, F., Gillett, G. T., Hemmati, P. G., Snowden, J. A., Stadler, M., Vuong, G. L., Aubourg, P., Köhler, W., & Arnold, R. (2017) · Brain · doi:10.1093/brain/awx016 · Cited by 78
Metadata
- Authors
- Beam, D., Poe, M. D., Provenzale, J. M., Szabolcs, P., Martin, P. L., Prasad, V., Parikh, S., Driscoll, T., Mukundan, S., Kurtzberg, J., & Escolar, M. L.
- Venue
- Biology of Blood and Marrow Transplantation
- Publisher
- Elsevier BV
- Published
- 2007-06
- Volume
- 13
- Issue
- 6
- Pages
- 665-674
- ISSN
- 1083-8791
- Cited by
- 83
- References
- 3
- Language
- en
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