Several previous studies (clinical and non-clinical) hypothesize that treatment with deferasirox causes a hematological improvement in transfused patients with low and intermediate-1 risk myelodysplastic syndrome. The purpose of this study was to assess the presence of genetic biomarkers predictive for hematologic response by the use of gene expression profiling of bone marrow aspirates obtained from MDS patients with or without hematological response.
This trial was terminated due to low enrollment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Enrollment
1
Patients experiencing a hematological response and patients not experiencing a hematological response while on deferasirox treatment received a new bone marrow aspirate in order to investigate the presence of differential gene expression between those two groups
Patients are already on commercial deferasirox before entering the study.
Novartis Investigative Site
Liège, Belgium
Fold Increase/Decrease in Gene Transcription From Baseline Bone Marrow Aspirate of Responders Versus Non-responders'
Using next-generation sequencing, gene expression profiling in responder and non-responder patients were to be performed on existing bone marrow aspirate samples. Gene transcription were then to be compared between the two groups and the fold increase/decrease in differentially expressed genes were to be calculated.
Time frame: 18 months
Time to Response
The time to response is defined as the time (in months) between the date of deferasirox initiation and the date of the first documented hematological response only in the responder group.
Time frame: 18 months
Changes in Serum Ferritin Levels
From baseline to time of response (responder group) or time to last follow up (non-responders)
Time frame: Baseline, 18 months
Deferasirox Dose Used
Deferasirox dose is defined as the average daily dose (mg/kg/d) given to the patient from treatment initiation to the emergence of hematological response in the responder group or the time of enrollment in the study in the non-responder group.
Time frame: 18 months
Changes in Serum Transferrin Levels
From baseline to time of response (responder group) or time to last follow up (non-responders)
Time frame: Baseline, 18 months
Changes in Transferrin Saturation Levels
From baseline to time of response (responder group) or time to last follow up (non-responders)
Time frame: Baseline, 18 months
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