Trial Outcomes & Findings for Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS (NCT NCT00520468)

NCT ID: NCT00520468

Last Updated: 2012-08-07

Results Overview

Periodic bone marrow samples (every 3-6 months) to check cells related to disease before/during/after study. Response classifications categorized by the International Working Group Response Criteria for Myelodysplastic Syndrome (MDS) as: Complete Remission, Partial Response, Hematologic Improvement or No Response.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

Response evaluation within first 3 months from start of therapy, then every 3 to 6 months

Results posted on

2012-08-07

Participant Flow

Recruitment Period 6/15/04 - 6/1/09; all patients were registered at The University of Texas M.D. Anderson Cancer Center.

Of fifteen patients enrolled, fourteen patients registered were evaluable and one patient taken off study never having received study drug.

Participant milestones

Participant milestones
Measure	Cytokine-Immunotherapy Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice per week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily
Overall Study STARTED	14
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cytokine-Immunotherapy n=14 Participants Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice per week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants
Age, Categorical >=65 years	8 Participants n=5 Participants
Age Continuous	65 Years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Region of Enrollment United States	14 participants n=5 Participants

PRIMARY outcome

Timeframe: Response evaluation within first 3 months from start of therapy, then every 3 to 6 months

Population: All treated patients on study included in analysis.

Outcome measures

Outcome measures
Measure	Cytokine-Immunotherapy n=14 Participants Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice per week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily
Number of Participants With Response Complete Remission	2 participants
Number of Participants With Response Partial Response	2 participants
Number of Participants With Response Hematologic Improvement	4 participants
Number of Participants With Response No Response	6 participants

Adverse Events

Cytokine-Immunotherapy

Serious events: 11 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cytokine-Immunotherapy n=14 participants at risk Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice per week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily
Gastrointestinal disorders Diarrhea	14.3% 2/14 • Number of events 2 • 2 years 2 months
General disorders Pain	7.1% 1/14 • Number of events 1 • 2 years 2 months
Vascular disorders hemorrhage	21.4% 3/14 • Number of events 3 • 2 years 2 months
Blood and lymphatic system disorders Elevated creatinine	14.3% 2/14 • Number of events 2 • 2 years 2 months
General disorders Chest Pain	28.6% 4/14 • Number of events 4 • 2 years 2 months
General disorders Fever	7.1% 1/14 • Number of events 1 • 2 years 2 months
Nervous system disorders Confusion	7.1% 1/14 • Number of events 1 • 2 years 2 months
General disorders Dizziness	7.1% 1/14 • Number of events 1 • 2 years 2 months
Respiratory, thoracic and mediastinal disorders Dyspnea	7.1% 1/14 • Number of events 1 • 2 years 2 months

Other adverse events

Other adverse events
Measure	Cytokine-Immunotherapy n=14 participants at risk Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice per week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily
Cardiac disorders Hypertension	21.4% 3/14 • Number of events 3 • 2 years 2 months
Blood and lymphatic system disorders elevated creatinine	21.4% 3/14 • Number of events 3 • 2 years 2 months
General disorders Pain	35.7% 5/14 • Number of events 6 • 2 years 2 months
Blood and lymphatic system disorders Hypomagnesemia	28.6% 4/14 • Number of events 4 • 2 years 2 months
Gastrointestinal disorders Diarrhea	14.3% 2/14 • Number of events 2 • 2 years 2 months
Nervous system disorders Insomnia	7.1% 1/14 • Number of events 1 • 2 years 2 months
Skin and subcutaneous tissue disorders Puritis	7.1% 1/14 • Number of events 1 • 2 years 2 months
Eye disorders Blurred vision	7.1% 1/14 • Number of events 1 • 2 years 2 months
Blood and lymphatic system disorders Hyperbilirubinemia	7.1% 1/14 • Number of events 1 • 2 years 2 months

Additional Information

Gautam Borthakur, MBBS / Assistant Professor

The University of Texas M. D. Anderson Cancer Center

Phone: 713-792-7305

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place