Trial Outcomes & Findings for High-dose Cytarabine/Mitoxantrone Followed by Autotransplantation for t-MDS/t-AML (NCT NCT00774046)

Results Overview

Complete remission (CR): \<5% bone marrow blasts with recovery of peripheral blood counts; complete cytogenetic remission, the disappearance of any pre-existing cytogenetic abnormality Partial remission (PR): \>5% bone marrow blasts, but less than the pre-treatment blast percentage within the bone marrow Resistant disease (RD): no significant cytoreduction in bone marrow leukemic cells from pre-treatment levels Not evaluable (NE): patients who died during induction chemotherapy or who withdrew from follow-up before assessment could be made

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

32 participants

Primary outcome timeframe

Day 28-40

Results posted on

2014-02-11

Participant Flow

Participant milestones

Participant milestones
Measure	Induction Chemotherapy Followed by Stem Cell Transplant Ara-C Mitoxantrone Etoposide Stem cell mobilization Autologous transplant
Screening STARTED	116
Screening COMPLETED	60
Screening NOT COMPLETED	56
Enrollment STARTED	60
Enrollment COMPLETED	32
Enrollment NOT COMPLETED	28
Induction Therapy STARTED	32
Induction Therapy COMPLETED	32
Induction Therapy NOT COMPLETED	0
Stem Cell Mobilization STARTED	10
Stem Cell Mobilization COMPLETED	7
Stem Cell Mobilization NOT COMPLETED	3
Autologous Transplant STARTED	4
Autologous Transplant COMPLETED	4
Autologous Transplant NOT COMPLETED	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Induction Chemotherapy Followed by Stem Cell Transplant Ara-C Mitoxantrone Etoposide Stem cell mobilization Autologous transplant
Screening Not eligible	56
Enrollment Desired other therapy/refused	28
Stem Cell Mobilization Count too low (2), sudden death (1)	3

Baseline Characteristics

High-dose Cytarabine/Mitoxantrone Followed by Autotransplantation for t-MDS/t-AML

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Patients n=32 Participants Ara-C Mitoxantrone Etoposide
Age, Continuous	56 years n=99 Participants
Sex: Female, Male Female	18 Participants n=99 Participants
Sex: Female, Male Male	14 Participants n=99 Participants
Region of Enrollment United States	32 participants n=99 Participants

PRIMARY outcome

Timeframe: Day 28-40

Complete remission (CR): \<5% bone marrow blasts with recovery of peripheral blood counts; complete cytogenetic remission, the disappearance of any pre-existing cytogenetic abnormality Partial remission (PR): \>5% bone marrow blasts, but less than the pre-treatment blast percentage within the bone marrow Resistant disease (RD): no significant cytoreduction in bone marrow leukemic cells from pre-treatment levels Not evaluable (NE): patients who died during induction chemotherapy or who withdrew from follow-up before assessment could be made

Outcome measures

Outcome measures
Measure	All Patients n=32 Participants Ara-C Mitoxantrone Etoposide
Response to Induction Chemotherapy (CR or PR)	81.2 percentage of participants Interval 63.6 to 92.8

PRIMARY outcome

Timeframe: Up to 2000 days

Outcome measures

Outcome measures
Measure	All Patients n=32 Participants Ara-C Mitoxantrone Etoposide
Overall Survival	399 Days Interval 125.0 to 812.0

PRIMARY outcome

Timeframe: Up to 2000 days

Relapse is defined as bone marrow blasts \>5% if the patient had achieved a complete remission, or the recurrence of any clonal cytogenetic abnormality.

Outcome measures

Outcome measures
Measure	All Patients n=32 Participants Ara-C Mitoxantrone Etoposide
Relapse-free Survival	415 Days Interval 141.0 to Due to censoring, the 75th percentile was not reached.

SECONDARY outcome

Timeframe: 1-5 days from initiation of stem cell collection

Population: Subset of patients in CR who underwent mobilization and attempted stem cell collection.

Feasibility is the ability to cryopreserve \>=2.0 x 10\^6 CD34+ cells/kg

Outcome measures

Outcome measures
Measure	All Patients n=10 Participants Ara-C Mitoxantrone Etoposide
Feasibility of Stem Cell Collection	70 percentage of participants Interval 35.0 to 93.0

SECONDARY outcome

Timeframe: 1-5 days from initiation of stem cell collection

Population: Subset of patients in CR who underwent mobilization and attempted stem cell collection.

Outcome measures

Outcome measures
Measure	All Patients n=10 Participants Ara-C Mitoxantrone Etoposide
Numbers of Stem Cells Collected	4.50 10^6 CD34+ cells/kg Interval 0.0 to 30.31

SECONDARY outcome

Timeframe: Up to 817 days

Population: Subset of patients undergoing autologous stem cell transplant

Outcome measures

Outcome measures
Measure	All Patients n=4 Participants Ara-C Mitoxantrone Etoposide
Overall Survival in Patients Undergoing Autologous Stem Cell Transplant	294 Days Interval 133.0 to Largest observation censored at 817 days

SECONDARY outcome

Timeframe: Up to 883 days

Population: Subset of patients undergoing autologous stem cell transplant

Outcome measures

Outcome measures
Measure	All Patients n=4 Participants Ara-C Mitoxantrone Etoposide
Disease-free Survival in Patients Undergoing Autologous Stem Cell Transplant	367 Days Interval 95.0 to Largest observation censored at 883 days

Adverse Events

All Patients

Serious events: 7 serious events

Other events: 31 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	All Patients n=32 participants at risk Ara-C Mitoxantrone Etoposide
Infections and infestations Neutropenic fever	3.1% 1/32 • Number of events 1 • 2 months
Infections and infestations Infection with grade 4 neutrophils	9.4% 3/32 • Number of events 3 • 2 months
General disorders Hemorrhage	3.1% 1/32 • Number of events 1 • 2 months
General disorders Sudden death	3.1% 1/32 • Number of events 1 • 2 months
Gastrointestinal disorders Hemorrhage	3.1% 1/32 • Number of events 1 • 2 months

Other adverse events

Other adverse events
Measure	All Patients n=32 participants at risk Ara-C Mitoxantrone Etoposide
Infections and infestations Neutropenic fever	59.4% 19/32 • 2 months
Infections and infestations Infection/sepsis	50.0% 16/32 • 2 months
Blood and lymphatic system disorders Thrombocytopenia	25.0% 8/32 • 2 months
Cardiac disorders Left ventrcular dysfunction	12.5% 4/32 • 2 months
Gastrointestinal disorders Non-infectious diarrhea	25.0% 8/32 • 2 months
General disorders Anemia	9.4% 3/32 • 2 months
Respiratory, thoracic and mediastinal disorders Pneumonia	9.4% 3/32 • 2 months
Skin and subcutaneous tissue disorders Rash	31.2% 10/32 • 2 months
Respiratory, thoracic and mediastinal disorders Respiratory failure	6.2% 2/32 • 2 months
Cardiac disorders Hypotension	12.5% 4/32 • 2 months
Cardiac disorders Tachycardia	6.2% 2/32 • 2 months
Cardiac disorders Atrial fibrillation	6.2% 2/32 • 2 months
General disorders Mental status changes	6.2% 2/32 • 2 months

Additional Information

Dr. Lucy A. Godley

University of Chicago

Phone: 773-702-4140

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place