Low-dose versus high-dose therapy for Gaucher disease: Goals and markers [2]

Blood

Volumn 109, Issue 1, 2007, Pages 387-

  Hollak, Carla E M ^a   De Fost, Maaike ^b   Aerts, Johannes M F G ^b   Vom Dahl, Stephan ^b  

^a ACADEMIC MEDICAL CENTER   (Netherlands)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

BIOCHEMICAL MARKER; CHITOTRIOSIDASE; GLUCOSYLCERAMIDE; IMIGLUCERASE;

CLINICAL FEATURE; DRUG DOSE REGIMEN; GAUCHER DISEASE; HUMAN; IMMUNOHISTOCHEMISTRY; LETTER; PRIORITY JOURNAL; TREATMENT OUTCOME;

EID: 33846023302     PISSN: 00064971     EISSN: 00064971     Source Type: Journal    
DOI: 10.1182/blood-2006-07-033233     Document Type: Letter

References (9)

1. Zimran A, Elstein D, Beutler E. Low-dose therapy trumps high dose again in the treatment of Gaucher disease. Blood. Prepublished on April 25, 2006, as DOI 10.1182/blood-2006-03-010801. (Now available as Blood. 2006;108:802-803.)
2. de Fost M, Hollak CE, Groener JE, et al. Superior effects of high dose enzyme replacement therapy in type 1 Gaucher disease on bone marrow involvement and chitotriosidase levels: a two center retrospective analysis. Blood. Prepublished on March 9, 2006, as DOI 10.1182/blood-2005-12-5072. (Now available as Blood. 2006;108:830-835.)
3. Hollak CE, Aerts JM, Goudsmit R, et al. Individualised low-dose alglucerase therapy for type 1 Gaucher's disease. Lancet. 1995;345:1474-1478.
4. Colburn WA, Lee JW. Biomarkers, validation and pharmacokinetic- pharmacodynamic modelling. Clin Pharmacokinet. 2003;42:997-1022.
5. Weinreb NJ, Aggio MC, Andersson HC, et al. Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients. Semin Hematol. 2004;41(suppl 5):15-22.
6. Beutler E. Enzyme replacement in Gaucher disease. PLoS Med. 2004;1:118-121.
7. Boven LA, van Meurs M, Boot RG, et al. Gaucher cells demonstrate a distinct macrophage phenotype and resemble alternatively activated macrophages. Am J Clin Pathol. 2004;122:359-369.
8. vom Dahl S, Poll LW, Häussinger D. Clinical monitoring after cessation of enzyme replacement therapy in M. Gaucher. Brit J Haematol. 2001;113:1084-1086.
9. Maas M, Hollak CE, Akkerman EM. Quantification of skeletal involvement in adults with type I Gaucher's disease: fat fraction measured by Dixon quantitative chemical shift imaging as a valid parameter. Am J Roentgenol. 2002;179:961-965.