Withdrawal of enzyme replacement therapy in Gaucher's disease

Although enzyme replacement therapy is safe and effective in ameliorating the signs and symptoms of Gaucher's disease, some patients have withdrawn from treatment. The purpose of this study was to evaluate the response to withdrawal and to discuss the implications for patients currently on unal...

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Bibliographic Details
Published in:British journal of haematology 2000-08, Vol.110 (2), p.488-492
Main Authors: Elstein, Deborah, Abrahamov, Ayala, Hadas‐Halpern, Irith, Zimran, Ari
Format: Article
Language:English
Subjects:
Adolescent
Adult
Biological and medical sciences
Child, Preschool
Enzyme Therapy
Errors of metabolism
ethics
Female
Gaucher Disease - blood
Gaucher Disease - complications
Gaucher Disease - therapy
Gaucher's disease
Hemoglobins - analysis
Hepatomegaly - etiology
Humans
Lipids (lysosomal enzyme disorders, storage diseases)
Male
Medical sciences
Metabolic diseases
Middle Aged
Platelet Count
Severity of Illness Index
Splenomegaly - etiology
Treatment Outcome
Treatment Refusal
treatment withdrawal
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Summary:Although enzyme replacement therapy is safe and effective in ameliorating the signs and symptoms of Gaucher's disease, some patients have withdrawn from treatment. The purpose of this study was to evaluate the response to withdrawal and to discuss the implications for patients currently on unaltered therapy regimens since the advent of treatment. Fifteen patients, who had been treated with enzyme replacement for 5–56 months and then withdrew for 8–47 months, were assessed for changes in haematological parameters and in liver and spleen index volume. Despite non‐uniformity of duration of on and off periods, degree of organomegaly, anaemia and thrombocytopenia, most patients did not revert to respective baseline values in most parameters after withdrawal. None of the patients suffered exacerbation of bone involvement or had new or aggravated pulmonary hypertension. Adult patients with stable Gaucher's disease may be withdrawn from therapy for circumscribed periods without forfeiting most gains accrued during enzyme therapy. Therefore, stopping and restarting may be considered in some patients. Alternatively, maintenance at reduced dosage and/or frequency may be appropriate in some adult patients who are stable or non‐responsive after the first years of enzyme therapy. This caveat does not apply to children.
ISSN:0007-1048
1365-2141
DOI:10.1046/j.1365-2141.2000.02177.x