The purpose of this study is to examine the effect of G-CSF on disease free survival and overall survival in aplastic anaemia patients who also receive ATG and Cyclosporin A.
Open label, randomized, controlled study of G-CSF, ATG and Cyclosporin A versus ATG and Cyclosporin A. Subjects will be evaluated for hematologic response through day 240. Subjects who do not demonstrate a partial or complete remission by day 120 will be randomized to receive either a second course of ATG or continue their current regimen. Subjects who do demonstrate a partial or complete remission will continue their current regimen through day 240 or maintenance of a complete remission for 30 days. The last day of study treatment will be day 240.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
205
Yes/no addition of G-CSF
Yes/no early retreatment with ATG
Failure free survival
To evaluate the effect of G-CSF on failure free survival and mortality in study subjects also receiving ATG and Cyclosporin A \& time to hematologic response (failure defined as death, non-response or requirement of further treatment).
Time frame: day 240
Haematological response
The proportion of subjects who achieve a hematologic response
Time frame: day 240
Severe Infections
Incidence of severe infections
Time frame: day 240
Benefit of addition of G-CSF
The benefit due to the addition of G-CSF on death rate (i), days of hospitalization (ii), and duration of antibiotic treatment (iii)
Time frame: day 240
Complete remission
Time to achieving a complete remission within 120 days
Time frame: day 120
Relapse rate
The relapse rate among responders
Time frame: 2year
Blood count
Median blood counts among subjects who achieve transfusion independence
Time frame: day 240
Severity of the disease
The proportion of subjects who have a change in severity of disease (e.g. improvement from very severe to severe aplastic anemia)
Time frame: day 365
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University Hospital
Pilsen, Czechia
CHU Angers
Angers, France
Avicenne Hospital
Bobigny, France
University Hospital
Brest, France
CHU Clemenceau
Caen, France
CHU de Caen
Caen, France
Henri Mondor
Créteil, France
CHU Limoges
Limoges, France
Paoli-Calmettes Institute
Marseille, France
CHU Montpellier
Montpellier, France
...and 61 more locations
Retreatment with ATG
Proportion of subjects who respond to re-treatment with ATG,
Time frame: day 240
Safety
The safety of G-CSF in subjects treated with G-CSF, ATG and Cyclosporin A, compared to subjects who receive ATG and Cyclosporin A
Time frame: 6year