Abstract | BACKGROUND: OBJECTIVES: SUBJECTS AND METHODS: Data were recorded at a single centre for 9 paediatric patients. Assessment of response included serial measurements of haemoglobin (Hb) concentrations, platelet count, angiotensin-converting enzyme (ACE) and total acid phosphatase (TAP) levels. Growth was assessed by serial determinations of body weight and height, plotted against standard growth charts. Organ size (liver and spleen) was measured clinically and also radiologically, where possible. RESULTS: In this low-dose imiglucerase treatment group: (i) there was a significant increase in Hb over time--normal Hb levels were achieved in 7 of the 9 patients after a mean of 3.7 years; (ii) platelet counts increased over time, reaching normal levels in 7 patients; (iii) there was a significant decrease in both ACE and TAP over time; (iv) heights and weights of the subjects increased significantly over time with treatment, normalising to the expected growth percentiles; and (v) organ size (liver and spleen) reduced with therapy in all patients measured. CONCLUSION: ERT with low-dose imiglucerase (+/- 10 U/kg/fortnight) ameliorates Gaucher disease-associated anaemia and thrombocytopenia. Low-dose ERT is effective and may be considered in resource-poor clinical situations when other alternatives are not available.
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Authors | R Heitner, S Arndt, J B Levin |
Journal | South African medical journal = Suid-Afrikaanse tydskrif vir geneeskunde
(S Afr Med J)
Vol. 94
Issue 8
Pg. 647-51
(Aug 2004)
ISSN: 0256-9574 [Print] South Africa |
PMID | 15352589
(Publication Type: Journal Article)
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Chemical References |
- Glucosylceramidase
- imiglucerase
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Topics |
- Adolescent
- Child
- Child, Preschool
- Cohort Studies
- Dose-Response Relationship, Drug
- Drug Administration Schedule
- Female
- Follow-Up Studies
- Gaucher Disease
(diagnosis, drug therapy)
- Glucosylceramidase
(administration & dosage)
- Humans
- Infusions, Intravenous
- Linear Models
- Long-Term Care
- Male
- Prospective Studies
- Risk Assessment
- Time Factors
- Treatment Outcome
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