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INTRODUCTION

Diffuse cutaneous systemic sclerosis (dcSSc) is a debilitating autoimmune disease with a high mortality rate. Autologous hematopoietic stem cell transplantation (HSCT) improves long-term outcome in dcSSc, but optimal timing of HSCT has not been established yet. It may seem preferable to initiate immunosuppressive therapy before proceeding to HSCT. However when ineffective, disease progression can lead to contra-indication for HSCT. Rational pharmacotherapy in dcSSc is key considering the impact of disease and risks and costs involved. Determination of the optimal treatment strategy in dcSSc was adopted to the research agenda of the Dutch Society of Rheumatology.

 

RESEARCH QUESTION

Is HSCT as first-line treatment superior to HSCT as “rescue therapy” in early dcSSc patients?

 

HYPOTHESIS

(A) First-line HSCT, versus (B) immunosuppressive therapy followed by rescue HSCT in case of progression, improves outcomes and the health-economical perspective.

 

STUDY DESIGN

Randomized, multicenter, open label trial

 

STUDY POPULATION Adults with early severe dcSSc

 

INTERVENTION

A. First-line autologous HSCT (using a non-myeloablative regimen)

versus

B. Monthly iv cyclophosphamide pulses for 1 year, followed by 1 year of oral mycophenolate. Rescue HSCT in case of non-response.

 

OUTCOME MEASURES

Primary outcome: Event free survival (EFS)

Secondary outcomes include treatment safety, the CRISS (composite score), number of HSCT in arm B or post HSCT immunosuppressant use in arm A, quality of life, daily functioning, cost-efficiency.

 

SAMPLE SIZE/DATA-ANALYSIS The sample size is 120 patients. To compare EFS between groups, a Kaplan Meyer analysis will be performed and cox regression will be used for prognostic factors (determined a priori). The effect of covariates will be evaluated using Cox-regression. For binary outcomes proportions will be calculated and differences tested using Chi-square/Fisher exact tests. The effects of covariates will be evaluated using logistic regression.

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