Wolman disease successfully treated by bone marrow transplantation

Bone Marrow Transplantation

Volumn 26, Issue 5, 2000, Pages 567-570

  Krivit, W ^a   Peters, C ^a   Dusenbery, K ^b   Ben Yoseph, Y ^c   Ramsay, N K C ^a   Wagner, J E ^a   Anderson, R ^d  

^a UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF MINNESOTA   (United States)

^c WAYNE STATE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d WAKE FOREST SCHOOL OF MEDICINE   (United States)

Author keywords

Bone marrow transplantation; Lysosomal acid lipase; Wolman disease

Indexed keywords

ACID LIPASE; ALANINE AMINOTRANSFERASE; ALKALINE PHOSPHATASE; BILIRUBIN; CHOLESTEROL; CYCLOPHOSPHAMIDE; CYCLOSPORIN; DEXAMETHASONE; HYDROCORTISONE; IMMUNOGLOBULIN A; IMMUNOGLOBULIN E; IMMUNOGLOBULIN G; IMMUNOGLOBULIN M; METHYLPREDNISOLONE; THYMOCYTE ANTIBODY; TRIACYLGLYCEROL;

ADRENAL DISEASE; ALLOGENIC BONE MARROW TRANSPLANTATION; ARTICLE; AUTOSOMAL RECESSIVE INHERITANCE; CALCIFICATION; CASE REPORT; CHOLESTEROL BLOOD LEVEL; CLINICAL FEATURE; DIARRHEA; ENZYME ACTIVITY; FAILURE TO THRIVE; FOLLOW UP; GRAFT VERSUS HOST REACTION; HUMAN; HYPERBILIRUBINEMIA; HYPERTENSION; IMMUNOSUPPRESSIVE TREATMENT; MALE; MALNUTRITION; NEPHROTOXICITY; PRESCHOOL CHILD; PRIORITY JOURNAL; TREATMENT OUTCOME; TRIACYLGLYCEROL BLOOD LEVEL; WHOLE BODY RADIATION; WOLMAN DISEASE;

EID: 0033828329     PISSN: 02683369     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.bmt.1702557     Document Type: Article

References (21)

1. Generalized xanthomatosis with calcified adrenals
2. Primary familial xanthomatosis with involvement and calcification of the adrenals: Report of two more cases in siblings of a previously described infant
3. Lipid accumulation and acid lipase deficiency in fibroblasts from a family with Wolman's disease and their apparent correction in vitro
4. Acid lipase deficiency: Wolman disease and cholesteryl ester storage disease
5. Alteration of murine serum lipase activity after allogeneic bone marrow transplantation
6. Wolman's disease: Review of treatment with bone marrow transplantation and considerations for the future
7. Wolman's disease treated by displacement bone marrow transplantation
8. Total body irradiation in conditioning regimens for bone marrow transplantation
9. Bone marrow graft engineering by counterflow centrifugal elutriation. Results of a phase I-III clinical trial
10. Restriction fragment length polymorphism (RFLP) as markers of engraftment in allogeneic bone marrow transplantation
11. Cloning and expression of cDNA encoding human lysosomal acid lipase/cholesteryl ester hydrolase. Similarities to gastric and lingual lipases
12. Bone marrow transplantation in thalassemia: Modifications of hepatic iron overload and associated lesions after long-term engrafting
13. Morbidity and mortality due to liver disease in children undergoing allogeneic bone marrow transplantation: A 10-year prospective study
14. Marrow transplantation for patients with thalassemia: Results in class 3 patients
15. Niemann-Pick disease type C (a cellular cholesterol lipidosis) treated by bone marrow transplantation
16. New lysosomal acid lipase gene mutants explain the phenotype of Wolman disease and cholesteryl ester storage disease
17. Cholesteryl ester storage disease: Relationship between molecular defects and in situ activity of lysosomal acid lipase
18. Targeted disruption of the mouse lysosomal acid lipase gene: Long-term survival with massive cholesteryl ester and triglyceride storage
19. Molecular and enzymatic analysis of lysosomal acid lipase in cholesteryl ester storage disease
20. Different missense mutations in histidine-108 of lysosomal acid lipase cause cholesteryl ester storage disease in unrelated compound heterozygous and hemizygous individuals
21. Lysosomal acid lipase mutations that determine phenotype in Wolman and cholesterol ester storage disease