Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome

Current Opinion in Hematology

Volumn 6, Issue 6, 1999, Pages 377-382

  Krivit, William ^a   Aubourg, Patrick ^b   Shapiro, Elsa ^a   Peters, Charles ^a  

^a UNIVERSITY OF MINNESOTA   (United States)

^b HÔPITAL SAINT VINCENT DE PAUL   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ADRENOLEUKODYSTROPHY; ARTICLE; BONE MARROW TRANSPLANTATION; DETERIORATION; GLOBOID CELL LEUKODYSTROPHY; HUMAN; HURLER SYNDROME; METACHROMATIC LEUKODYSTROPHY; NEUROLOGIC DISEASE; ONSET AGE; PRIORITY JOURNAL; TREATMENT OUTCOME;

ADRENOLEUKODYSTROPHY; BONE MARROW TRANSPLANTATION; HUMANS; LEUKODYSTROPHY, GLOBOID CELL; LEUKODYSTROPHY, METACHROMATIC; MUCOPOLYSACCHARIDOSIS I;

EID: 0032693385     PISSN: 10656251     EISSN: None     Source Type: Journal    
DOI: 10.1097/00062752-199911000-00004     Document Type: Article

References (49)

1. 1 Krivit W, Peters C, Shapiro EG: Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy and Sly syndromes, and Gaucher disease Type III. Curr Opin Neural 1999, 12:167-176. This is an excellent review of how microglia and perhaps astrocytes are derived from bone marrow monocyte lineage. The authors also review several conditions in which BMT can improve or stabilize neurological disease.
2. 2 Krivit W, Shapiro EG, Peters C, Kolodny EH, Wenger D, Lockman L: Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. N Engl J Med 1998, 338:1119-1126. This is a detailed report of how hematopoietic stem cell transplantation can lead to major improvement or remission from GLD.
3. 3 Kolodny EH, Raghavan S, Krivit W: Late-onset Krabbe's disease (globoid cell leukodystrophy): clinical and biochemical features of 15 cases. Dev Neurosci 1991, 13:232-239.
4. 4 Bambach, BJ, Moser HW, Blakemore K, et al.: Engraftment following in-utero bone marrow transplantation for globloid cell leukodystrophy. Bone Marrow Transplant 1997, 19:399-402.
5. 5 Blakemore, K. In-utero bone marrow transplantation for globoid cell leukodystrophy [abstract]. IBMTR Keystone March 1999. These authors report on three fetuses in whom BMT was performed in utero. Engraftation occurred in none of the fetuses. Eventually, each of these fetuses died of hyperimmune response secondary to intra-uterine transplant.
6. 6 Carrier E: Tolerance or hyper-immunity secondary to in-utero transplantation [abstract]. IBMTR Keystone March 1999.
7. 7 Kurtzberg J, Cocci G, Dubenstein P, Krivit W: Unrelated cord blood: an alternative to stem cell transplantation. In Correction of Genetic Diseases by Transplantation, edn 5 (COGENT V). London: Westminster Printing Press; 1999.
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10. 10 Loes DJ, Peters C, Krivit W: Globoid cell leukodystrophy: distinguishing early onset from late-onset disease using a brain MRI scoring method. AJNR: Am J Neuro Radiol 1999, 20:316-323. An important contribution to diagnosing infantile GLD or Krabbe's disease.
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12. 12 Dubois-Dalcq M, Feigenbaum V, Aubourg P: The neurobiology of x-linked adrenoleukodystrophy, a demyelinating peroxisomal disorder. Trends Neurosci 1999, 22:4-12. This is a most excellent review of ALD and its genetic and physiological meaning. It is a must read for anyone dealing with ALD.
13. 13 Moser HW: Adrenoleukodystrophy: phenotype, genetics, pathogenesis and therapy. Brain 1997, 120:1485-1508.
14. 14 Rajanayagam V, Shapiro EG, Krivit W, Stillman AE: Proton MR spectroscopy of childhood adrenoleukodystrophy. AJNR: Am J Neurol Radiol 1996, 17:1013-1024.
15. 15 Rajanayagm V, Balthazor, M. Shapiro EG, Krivit W, Lockman L, Stillman AE: Proton MR spectroscopy and neuropsychological testing in adrenoleukodystrophy. AJNR: Am J Neurol Radiol 1997, 18:1909-1914.
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18. 18 Shapiro EG, Peters C, Lockman L, et al.: Long-term benefit of bone marrow transplantation for childhood cerebral adrenoleukodystrophy. Lancet 1999, in press. This is an important contribution to our knowledge of the excellent results that can be obtained from BMT in patients with ALD that is moderately involved.
19. 19 Shapiro EG, Lockman LA, Balthazor M, Peters C, Krivit W: Neuropsychological and neurological function and quality of life before and after bone marrow transplantation for adrenoleukodystrophy. In Correction of Genetic Diseases by Transplantation (COGENT IV). Edited by Ringden O, Hobbs JR, Steward CG. London, Middlesex: Westminster Printing Press; 1997:52-61.
20. 20 Ziegler R, Peters C, Lockman L, et al.: Use of brain sparing regimen for treatment of severe disease in 9 patients with childhood onset of cerebral adrenoleukodystrophy. Int J Radiat Oncol Biol Phys, accepted. An excellent exposition of what can be effected for patients with severe conditions.
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26. 26 Peters C, Krivit W: Hematopoietic stem cell transplantation for inborn errors of metabolism and prospects for gene therapy. J Gene Medicine 1999, in press.
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28. 28 Koc O, Raghavan S, Peters C: Measurement of mesenchymal stem cells in patients with complete and prolonged hematopoietic engraftment. Exp Hematol, in press. This is an important report on the fact that mesenchymal stem cells are not transplanted into the recipient even if completely hematopoietically engrafted.
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31. 31 Stillman A.E, Krivit W, Shapiro EG, Lockman LA, Latchaw RE: Serial MR after bone marrow transplantation in two patients with metachromatic leukodystrophy. AJNR Am J Neuroradiol 1994, 15:1929-1932.
32. 32 Dhuna A, Toro C, Torres F, Kennedy WR, Krivit W: Longitudinal neurophysiological studies in a patient with MLD following BMT. Arch Neurol 1992, 49:1088-1092.
33. 33 Shapiro EG, Lipton ME, Krivit W: White matter dysfunction and its neuropsychological correlates: a longitudinal study of a case of metachromatic leukodystrophy treated with bone marrow transplant. J Clin Exp Neuropsychiatry 1992, 14:610-624.
34. 34 Shapiro EG, Lockman LA, Balthazor M, Krivit W: Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation. J Inherit Metab Dis 1995, 18:413-429.
35. 35 Raghavan S, Kolodny EH, Peters C, Trapp B, Krivit W: Normalization of brain Arylsulfatase A enzyme activity following bone marrow transplantation in three patients with MLD. J Exp Neurol Neuropathol, in press. This is an important observation indicative of enzyme entrance into the brain.
36. 36 Peters C, Waye JS, Vellodi A, Ringden O, Krivit W: Hematopoietic stem cell transplantation for metachromatic leukodystrophy prior to onset of clinical signs and symptoms. In Correction of Genetic Diseases by Transplantation, edn IV (COGENT IV). Edited by Ringden O, Hobbs JR, Steward CG. London, Middlesex: Westminster Printing Press; 1997:34-48.
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39. 39 Kapaun, P, Dittman R, Granitzny B, et al.: Slow progression of juvenile metachromatic leukodystrophy 6 years after bone marrow transplantation. J Child Neurol 1999, 14:222-228.
40. 40 Kidd D, Nelson J, Jones F: Long-term stabilization after bone marrow transplantation in juvenile metachromatic leukodystrophy. Arch Neurol 1998, 55:98-99.
41. 41 Polten A, Fluharty AL, Fluharty CB, von Figura K: Molecular basis of different forms of metachromatic leukodystrophy. N Engl J Med 1991, 324:18-22.
42. 42 Berger J, Loschl B, Berhnehimer H, et al.: Occurrence, distribution and phenotype of arylsulfatase A mutations in patients with metachromatic loukodystrophy. Am J Med Genet 1997, 69:335-340.
43. 43 Landrieu P, Blanche S, Vanier MT: Bone marrow transplantation in metachromatic leukodystrophy caused by saposin-B deficiency: a case report with a 3 year follow-up period. J Pediatr 1998, 133:129-132.
44. 44 Burkhardt JK, Huttler S, Klein A: Accumulation of sphingolipids in SAP-precursor (prosapason)-deficient fibroblasts occurs as intralysosomal membrane structures and can' be completely reversed by treatment with human SAP-precursor. Eur J Cell Biol 1997, 73:10-18.
45. 45 Peters C, Shapiro EG, Krivit W: Hurler syndrome: past, present and future. J Pediatr 1998, 133:7-9. An excellent review of all articles pertinent to Hurler syndrome and BMT.
46. 46 Peters C, Shapiro EG, Krivit W: Neuropsychological development in children with Hurler syndrome following hematopoietic stem cell transplantation. Pediatr Transplant 1998, 2:250-253. This is an excellent article on cognitive factors in Hurler syndrome after BMT.
47. 47 Odunusi E, Peters C, Krivit W, Ogilvie J: Genu valgum deformity in Hurler syndrome after hematopoietic stem cell transplantation: correction by surgical intervention. J Pediatr Orthop 1999, 19:270-274.
48. 48 VanHeest A, House J, Krivit W, Walker K: Surgical treatment 1 of carpal tunnel syndrome and trigger digits in children with mucopolysaccharide storage diseases. J Hand Surg Am 1998, 23:236-243.
49. 49 Hite S, Peters C, Krivit W: Recovery of odontoid process. J Pediatr Orthop 1999, in press. An outstanding contribution of BMT to a better life for patients with Hurler syndrome. Previously, the C1-C2 subluxation caused parapelgia. Presently, with the odonotid improving, we can look forward to freedom from this complication.