Farber's disease without central nervous system involvement: Bone-marrow transplantation provides a promising new approach [1]

Annals of the Rheumatic Diseases

Volumn 65, Issue 12, 2006, Pages 1665-1666

  Ehlert, K ^a   Roth, J ^a   Frosch, M ^a   Fehse, N ^b   Zander, N ^b   Vormoor, J ^a,c  

^a UNIVERSITY HOSPITAL MÜNSTER   (Germany)

^b UNIVERSITY MEDICAL CENTER HAMBURG EPPENDORF   (Germany)

^c NEWCASTLE UNIVERSITY   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ACYLSPHINGOSINE DEACYLASE; ANTIINFLAMMATORY AGENT; BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; IMMUNOSUPPRESSIVE AGENT; METHOTREXATE; STEROID; THYMOCYTE ANTIBODY;

ANALGESIA; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; BONE MARROW TRANSPLANTATION; CENTRAL NERVOUS SYSTEM; CLINICAL FEATURE; FARBER DISEASE; GRAFT VERSUS HOST REACTION; HUMAN; JUVENILE RHEUMATOID ARTHRITIS; LETTER; NEUROTOXICITY; PHYSIOTHERAPY; PRIORITY JOURNAL; SURGICAL TECHNIQUE; VEIN OCCLUSION;

ADULT; CERAMIDE TRIHEXOSIDASE; CHILD, PRESCHOOL; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LYSOSOMAL STORAGE DISEASES; MALE; TREATMENT OUTCOME;

EID: 33751262990     PISSN: 00034967     EISSN: None     Source Type: Journal    
DOI: 10.1136/ard.2005.048322     Document Type: Letter

References (7)

1. Moser HW, Linke T, Fensom AH, Levade T, Sandhoff K. Acid ceramidase deficiency: Farber lipogranulomatosis. In: Scriver CR, ed. Metabolic and molecular bases for inherited disease. New York: McGraw-Hill, 2001:3573-85.
2. Burek C, Roth J, Koch HG, Harzer K, Los M, Schulze-Osthoff K. The role of ceramide in receptor- and stress-induced apoptosis studied in acidic ceramidase-deficient Farber disease cells. Oncogene 2001;20:0493-502.
3. Souillet G, Guiband P, Fensom AH, Maire I, Zabot MT. Outcome of displacement bone marrow transplantation in Farbers disease: a report of a case. In: Hobbs JR, ed. Correction of certain genetic diseases by transplantation. London: COGENT, 1989:137-41.
4. Yeager AM, Armfield-Uhas K, Coles CD, Davis PC, Krause WL, Moser HW. Bone marrow transplantation for infantile ceramidase deficiency (Farber disease). Bone Marrow Transplant 2000;26:357-63.
5. Vormoor J, Ehlert K, Groll AH, Koch HG, Frosch M, Roth J. Successful hematopoietic stem cell transplantation in Farber disease. J Pediatr 2004;144:132-4.
6. Burck U, Moser HW, Goebel HH, Gruttner R, Held KR. A case of lipogranulomatosis Farber: some clinical and ultrastructural aspects. Eur J Pediatr 1985;143:203-8.
7. Peters C, Balthazor M, Shapiro EG, King RJ, Kollman C, Hegland JD, et al. Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood 1996;87:4894-902.