Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose - response relationships

Carla E. M. Hollak; Maaike de Fost; Laura van Dussen; Stephan vom Dahl; Johannes M. F. G. Aerts

doi:https://doi.org/10.1517/14656560903270520

Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose - response relationships

Carla E. M. Hollak, Maaike de Fost, Laura van Dussen, Stephan vom Dahl, Johannes M. F. G. Aerts

Research output: Contribution to journal › Review article › Academic › peer-review

23 Citations (Scopus)

Abstract

BACKGROUND: Enzyme therapy for Gaucher disease has improved the lives of many patients, by reducing the burden of their disease. Several studies have sought to determine to what extent optimal clinical outcomes are a function of the prescribed enzyme dose and its resultant costs. OBJECTIVE: Issues concerning dose - response relationships during initial and maintenance treatment phases and currently applied treatment goals have been addressed. METHODS: All studies that aimed to describe the efficacy of different doses of treatment and approaches for maintenance, such as lowering the dose or changing to less frequent infusions and the effects of drug interruptions, were reviewed. RESULTS/CONCLUSION: Dose - response relationships do exist, but doses between 30 and 60 U/kg per month may be sufficient in a large majority of patients. Goals of treatment should include important clinical end points, such as enhanced quality of life and decreased risk for malignancies and other morbidities. The relationship between these important end points and treatment schedules deserve further study

Original language	English
Pages (from-to)	2641-2652
Journal	Expert opinion on pharmacotherapy
Volume	10
Issue number	16
DOIs	https://doi.org/10.1517/14656560903270520
Publication status	Published - 2009

Access to Document

https://doi.org/10.1517/14656560903270520

Cite this

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title = "Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose - response relationships",

abstract = "BACKGROUND: Enzyme therapy for Gaucher disease has improved the lives of many patients, by reducing the burden of their disease. Several studies have sought to determine to what extent optimal clinical outcomes are a function of the prescribed enzyme dose and its resultant costs. OBJECTIVE: Issues concerning dose - response relationships during initial and maintenance treatment phases and currently applied treatment goals have been addressed. METHODS: All studies that aimed to describe the efficacy of different doses of treatment and approaches for maintenance, such as lowering the dose or changing to less frequent infusions and the effects of drug interruptions, were reviewed. RESULTS/CONCLUSION: Dose - response relationships do exist, but doses between 30 and 60 U/kg per month may be sufficient in a large majority of patients. Goals of treatment should include important clinical end points, such as enhanced quality of life and decreased risk for malignancies and other morbidities. The relationship between these important end points and treatment schedules deserve further study",

author = "Hollak, {Carla E. M.} and {de Fost}, Maaike and {van Dussen}, Laura and {vom Dahl}, Stephan and Aerts, {Johannes M. F. G.}",

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language = "English",

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pages = "2641--2652",

journal = "Expert opinion on pharmacotherapy",

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TY - JOUR

T1 - Enzyme therapy for the treatment of type 1 Gaucher disease: clinical outcomes and dose - response relationships

AU - Hollak, Carla E. M.

AU - de Fost, Maaike

AU - van Dussen, Laura

AU - vom Dahl, Stephan

AU - Aerts, Johannes M. F. G.

PY - 2009

Y1 - 2009

N2 - BACKGROUND: Enzyme therapy for Gaucher disease has improved the lives of many patients, by reducing the burden of their disease. Several studies have sought to determine to what extent optimal clinical outcomes are a function of the prescribed enzyme dose and its resultant costs. OBJECTIVE: Issues concerning dose - response relationships during initial and maintenance treatment phases and currently applied treatment goals have been addressed. METHODS: All studies that aimed to describe the efficacy of different doses of treatment and approaches for maintenance, such as lowering the dose or changing to less frequent infusions and the effects of drug interruptions, were reviewed. RESULTS/CONCLUSION: Dose - response relationships do exist, but doses between 30 and 60 U/kg per month may be sufficient in a large majority of patients. Goals of treatment should include important clinical end points, such as enhanced quality of life and decreased risk for malignancies and other morbidities. The relationship between these important end points and treatment schedules deserve further study

AB - BACKGROUND: Enzyme therapy for Gaucher disease has improved the lives of many patients, by reducing the burden of their disease. Several studies have sought to determine to what extent optimal clinical outcomes are a function of the prescribed enzyme dose and its resultant costs. OBJECTIVE: Issues concerning dose - response relationships during initial and maintenance treatment phases and currently applied treatment goals have been addressed. METHODS: All studies that aimed to describe the efficacy of different doses of treatment and approaches for maintenance, such as lowering the dose or changing to less frequent infusions and the effects of drug interruptions, were reviewed. RESULTS/CONCLUSION: Dose - response relationships do exist, but doses between 30 and 60 U/kg per month may be sufficient in a large majority of patients. Goals of treatment should include important clinical end points, such as enhanced quality of life and decreased risk for malignancies and other morbidities. The relationship between these important end points and treatment schedules deserve further study

U2 - https://doi.org/10.1517/14656560903270520

DO - https://doi.org/10.1517/14656560903270520

M3 - Review article

C2 - 19743939

SN - 1465-6566

VL - 10

SP - 2641

EP - 2652

JO - Expert opinion on pharmacotherapy

JF - Expert opinion on pharmacotherapy

IS - 16

ER -