Effective treatment of α-mannosidosis by allogeneic hematopoietic stem cell transplantation

Journal of Pediatrics

Volumn 144, Issue 5, 2004, Pages 569-573

  Grewal, Satkiran S ^a   Shapiro, Elsa G ^b   Krivit, William ^b   Charnas, Lawrence ^b   Lockman, Lawrence A ^b   Delaney, Kathleen A ^b   Davies, Stella M ^b   Wenger, David A ^b   Rimell, Frank L ^b   Abel, Susan ^a   Grovas, Alfred C ^b   Orchard, Paul J ^b   Wagner, John E ^b   Peters, Charles ^b  

^a UNIVERSITY OF MINNESOTA   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA MANNOSIDASE;

ADAPTIVE BEHAVIOR; ADULT; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; CASE REPORT; CLINICAL EXAMINATION; CLINICAL FEATURE; COGNITION; DONOR; ENZYMATIC ASSAY; ENZYME ACTIVITY; FEMALE; FOLLOW UP; HEARING; HUMAN; MALE; MANNOSIDOSIS; NEUROPHYSIOLOGY; PATIENT MONITORING; PERCEPTION DEAFNESS; PHYSICAL EXAMINATION; PRESCHOOL CHILD; PRIORITY JOURNAL; SKELETON MALFORMATION; SPEECH ANALYSIS; TREATMENT OUTCOME; UNITED STATES; UNIVERSITY HOSPITAL; VERBAL MEMORY;

EID: 2342551124     PISSN: 00223476     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jpeds.2004.01.025     Document Type: Article

References (18)

1. Thomas GH. Disorders of glycoprotein degradation: alpha-mannosidosis, beta-mannosidosis, fucosidosis, and sialidosis. In: Schriver CR, Beaudet AL, Sly WS, Valle D, editors. The metabolic and molecular bases of inherited disease. 8th edition New York: McGraw-Hill; 2001. p. 3507-33.
2. Autio S, Louhimo T, Helenius M. The clinical course of mannosidosis. Ann Clin Res 1982;14:93-7.
3. Chester MA, Lundblad A, Ockerman P-A, Autio S. Mannosidosis. In: Durand P, O'Brien JS, editors. Genetic errors of glycoprotein metabolism. Berlin: Springer-Verlag; 1982. p. 89-122.
4. Wenger DA, Williams C. Screening for lysosomal disorders. In: Hommes FA, editor. Techniques in diagnostic human biochemical genetics, a laboratory manual. New York: Wiley-Liss; 1991. p. 587-617.
5. Yunis JJ, Lewandowski RC Jr, Sanfilippo SJ, Tsai MY, Foni I, et al. Clinical manifestations of mannosidosis: a longitudinal study. Am J Med 1976;61:841-8.
6. Shapiro E, Krivit W, Lockman L, Jambaque I, Peters C, Cowan M, et al. Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy. Lancet 2000;356:713-8.
7. Krivit W, Aubourg P, Shapiro E, Peters C. Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome. Curr Opin Hematol 1999;6:377-82.
8. Peters C, Shapiro EG, Krivit W. Neuropsychological development in children with Hurler syndrome following hematopoietic stem cell transplantation. Pediatr Transplant 1998;2:250-3.
9. Krivit W, Shapiro EG, Peters C, Wagner JE, Cornu G, Kurtzberg J, et al. Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. N Engl J Med 1998;338:1119-26.
10. 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;18:413-29.
11. Burgess J, Traycoff C, Hardwick A, Smith A, Hangoc G, Ishizawa L, et al. Separation of clinical quantities of CD34+ cells from human marrow using immunomagnetic procedures. Prog Clin Biol Res 1994;389:293-302.
12. Scharf SJ, Smith AG, Hansen JA, McFarland C, Erlich HA. Quantitative determination of bone marrow transplant engraftment using fluorescent polymerase chain reaction primers for human identity markers. Blood 1995;85:1954-63.
13. Glucksberg H, Storb R, Fefer A, Buckner CD, Neiman PE, Clift RA, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HLA-matched sibling donors. Transplantation 1974; 18:295-304.
14. Olsen I, Dean MF, Harris G, Muir H. Direct transfer of a lysosomal enzyme from lymphoid cells to deficient fibroblasts. Nature 1981;291: 244-7.
15. Walkley SU, Thrall MA, Dobrenis K, Huang M, March PA, Siegel DA, et al. Bone marrow transplantation corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease. Proc Natl Acad Sci U S A 1994;91:2970-4.
16. Northern JL, Downs MP. Hearing and hearing loss in children. In: Northern JL, Downs MP, editors. Hearing in children. 4th ed. Baltimore: Williams and Wilkins; 1991. p. 1-29.
17. Will A, Cooper A, Hatton C, Sardharwalla IB, Evans DI, Stevens RF. Bone marrow transplantation in the treatment of alpha-mannosidosis. Arch Dis Child 1987;62:1044-9.
18. Wall DA, Grange DK, Goulding P, Daines M, Luisiri A, Kotagal S. Bone marrow transplantation for the treatment of alpha-mannosidosis. J Pediatr 1998;133:282-5.