| Repeat abdominal ultrasound evaluation of 100 patients with type I Gaucher disease treated with enzyme replacement therapy for up to 7 years. | |
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MedLine Citation:
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PMID: 11960391 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
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With the advent of enzyme replacement therapy for Gaucher disease, there was a need to assess response to treatment repeatedly over relatively short periods of time; hence abdominal ultrasound was used in our referral clinic because it was convenient, safe and inexpensive. In 100 treated patients monitored for 2-7 years, nearly all had reduction in hepatosplenomegaly. The per cent reduction was more dramatic in patients who started treatment when older than 16 years. This effect may be explained by a correlation between per cent reduction and degree of baseline organomegaly. Because children often initiate therapy sufficiently early to preclude developing massive organomegaly, the observed per cent reduction is commensurately less. For all patients treated for more than five years, plateau-ing in response was seen after the first three years of treatment. This study permits us to reconsider less frequent evaluations of organ volumes. Our findings also underscore the efficacy of enzyme replacement, but raise the question of the need for dose reduction as nearly normal organ volumes are achieved. |
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Authors:
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Michael Patlas; Irith Hadas-Halpern; Ayala Abrahamov; Ari Zimran; Deborah Elstein |
Publication Detail:
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Type: Journal Article |
Journal Detail:
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Title: The hematology journal : the official journal of the European Haematology Association / EHA Volume: 3 ISSN: 1466-4860 ISO Abbreviation: Hematol. J. Publication Date: 2002 |
Date Detail:
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Created Date: 2002-04-17 Completed Date: 2003-07-25 Revised Date: 2004-11-17 |
Medline Journal Info:
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Nlm Unique ID: 100965523 Medline TA: Hematol J Country: England |
Other Details:
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Languages: eng Pagination: 17-20 Citation Subset: IM |
Affiliation:
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Gaucher Clinic, Shaare-Zedek Medical Center, Jerusalem, Israel. |
Export Citation:
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APA/MLA Format Download EndNote Download BibTex |
| MeSH Terms | |
Descriptor/Qualifier:
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Abdomen
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ultrasonography Adolescent Adult Aged Child Child, Preschool Enzymes / administration & dosage, pharmacology, therapeutic use* Female Follow-Up Studies Gaucher Disease / drug therapy, ultrasonography* Hepatomegaly / drug therapy Humans Male Middle Aged Retrospective Studies Splenectomy Splenomegaly / drug therapy Treatment Outcome |
| Chemical | |
Reg. No./Substance:
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0/Enzymes |
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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