This is a Phase II, single-arm, open-label, multinational, multicenter study of rATG in patients with low or intermediate-1 risk MDS who have either failed 1 prior treatment with growth factor(s), hypomethylating agents (5-azacitidine or decitabine), or the antiangiogenic agents lenalidomide or thalidomide, or who have never been treated for MDS (i.e., treatment-naïve patients).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
16
All patients were to be treated with rATG 3.75 mg/kg/day administered by intravenous (IV) infusion over ≥6 hours for 5 consecutive days (cumulative dose: 18.75 mg/kg)
Hopital Avicenne/University
Paris, France
St. Johannes-Hospital Duisburg
Duisburg, Germany
Medizinische Hochschule Hannover
Hanover, Germany
UMC St Radboud Centraal
Nijmegen, Netherlands
Number of Participants Who Achieved Hematologic Improvement (HI)
This is a measure of HI in the erythroid, platelet, and neutrophil lineages. Note that HI was observed in the erythroid lineage only, which is defined as a participant who had a \>=1.5 g/dL increase in hemoglobin from baseline (pretreatment value must have been \<11 g/dL) and who had a relevant reduction of units of red blood cell (RBC) transfusions by an absolute number of \>=4 RBC transfusions over 8 weeks as compared with the pretreatment transfusion number in the previous 8 weeks. These criteria were taken from the 2006 International Working Group criteria.
Time frame: 12 months
Number of Participants With Duration of HI
This is a measure of the duration of HI for those participants who achieved HI. Duration of HI is defined as the time from confirmation of HI response to the date of first documentation of HI relapse or death due to any cause, whichever occurs first.
Time frame: 36 months
Number of Participants Who Achieved Disease Remission
Disease remission is defined as a participant whose best response to therapy was a complete remission or partial remission. A complete remission is defined as: bone marrow \<=5% myeloblasts with normal maturation of all cell lines; persistent dysplasia noted; and peripheral blood hemoglobin \>=11 g/dL, platelets \>=100 x 10\^9/L, neutrophils \>=1.0 x 10\^9/L, and blasts 0%. Partial remission is defined as: all complete remission criteria if abnormal before treatment, except bone marrow blasts decreased by \>=50% over pretreatment, but still \>5%; and cellularity and morphology not relevant.
Time frame: 36 months
Duration of Disease Remission
This is a measure of the duration of overall disease remission only for those participants who achieved an overall remission. Duration of overall remission is defined as the time from first documentation of overall remission to the date of first documentation of disease relapse or death due to any cause, whichever occurs first.
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Royal Bournemouth Hospital
Bournemouth, England, United Kingdom
St. James Hospital
Leeds, England, United Kingdom
King's College Hospital
London, England, United Kingdom
Time frame: 36 months
Number of Participants Who Achieved Transfusion Independence
This is a measure of transfusion independence, which is defined as a participant with no transfusions for a period of 8 consecutive calendar weeks after first dose. Transfusion independence was to be calculated only for those participants who had documented transfusions during the 8 weeks prior to enrollment.
Time frame: 36 months
Number of Participants With Duration of Transfusion Independence
This is a measure of the duration of transfusion independence only for those participants who achieved transfusion independence. Duration of transfusion independence is defined as the longest period of time during which a participant requires no transfusions.
Time frame: 36 months
Number of Participants With a Relapse Following HI
This is a measure of relapse following HI, which is defined as a participant who experiences at least one of the following: \>=50% decrease from maximum response levels in granulocytes or platelets; \>=1.5 g/dL reduction in hemoglobin; or transfusion dependence.
Time frame: 36 months
Number of Participants With a Relapse Following Overall Remission
This is a measure of relapse following an overall remission only for participants who experienced either a complete or partial remission. Relapse following an overall remission is defined as a participant who meets any of the following criteria: a return to pretreatment bone marrow blast percentage; decrease of \>=50% from maximum remission levels in neutrophils or platelets; reduction in hemoglobin concentration by \>=1.5 g/dL from maximum remission levels; or transfusion dependence.
Time frame: 36 months
Number of Participants With Progression-free Survival
This is a measure of a progression-free survival which is defined as the time from the participant's first dose to the date of disease progression, lost to follow-up or death due to any cause, whichever occurs first.
Time frame: 36 months
Number of Participants With Transformation to Acute Myeloid Leukemia
This is a measure of transformation to acute myeloid leukemia only for participants who have bone marrow assessments. Transformation to acute myeloid leukemia is defined as the earliest date a participant experiences bone marrow blasts of \>=20% after the start of treatment.
Time frame: 36 months
Number of Participants With a Cytogenetic Response
This is a measure of cytogenic response for participants whose best response to therapy is a either a complete or partial cytogenetic response. A complete cytogenetic response is defined as the disappearance of the chromosomal abnormality without appearance of new ones. A partial cytogenetic response is defined as at least 50% reduction of the chromosomal abnormality.
Time frame: 36 months
Number of Participants With a Marrow Remission
This is a measure of bone marrow complete remission for participants who experience a remission. Bone marrow complete remission is defined as a bone marrow assessment of \<=5% myeloblasts and decrease by \>=50% over pretreatment.
Time frame: 36 months