Trial Outcomes & Findings for Lenalidomide and Prednisone in Treating Patients With Myelofibrosis (NCT NCT00227591)

Last Updated: 2014-05-21

Results Overview

Response was evaluated for Anemia and Spleen: Major anemia response: hemoglobin increase to within normal limits in the absence of transfusion. Minor anemia response: hemoglobin improvement of at least 2 grams per deciliter independent of transfusion support, or achievement of transfusion independence in transfusion-dependent patients. Major spleen response: normalization of spleen size to the range of 12-14 centimeters by ultrasound. Minor spleen response: a 50% or more decrease in excess spleen size by ultrasound. Complete remission (CR): complete resolution of disease-related symptoms, splenomegaly, normalization of peripheral blood count, white cell differential and smear, and normalization of bone marrow histology. Partial remission (PR): a major or minor response in anemia or splenomegaly. Overall Response (OR)=CR + PR, assessed among eligible, treated patients.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

48 participants

Primary outcome timeframe

Assessed at the end of cycle 3

Results posted on

2014-05-21

Participant Flow

This study accrued 48 cases between December 2, 2005 and March 9, 2007. Per two-stage design, the study was suspended on May 31, 2006 for a toxicity and response analysis. Since more than 4 patients achieved a response, the study was reactivated on December 5, 2006. Accrual continued to a total of 48 patients and terminated on March 9, 2007.

Participant milestones

Participant milestones
Measure	Lenalidomide Lenalidomide 10 mg/day plus prednisone X 28 days X 3 cycles
Overall Study STARTED	48
Overall Study Began Treatment	47
Overall Study COMPLETED	47
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Lenalidomide Lenalidomide 10 mg/day plus prednisone X 28 days X 3 cycles
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Lenalidomide and Prednisone in Treating Patients With Myelofibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Lenalidomide n=47 Participants Lenalidomide 10 mg/day plus prednisone X 28 days X 3 cycles
Age, Categorical <=18 years	0 Participants n=39 Participants
Age, Categorical Between 18 and 65 years	25 Participants n=39 Participants
Age, Categorical >=65 years	22 Participants n=39 Participants
Age, Continuous	63 years STANDARD_DEVIATION 11.2 • n=39 Participants
Sex: Female, Male Female	21 Participants n=39 Participants
Sex: Female, Male Male	26 Participants n=39 Participants
Region of Enrollment United States	47 participants n=39 Participants

PRIMARY outcome

Timeframe: Assessed at the end of cycle 3

Population: 6 ineligible patients were excluded from the analysis.

Outcome measures

Outcome measures
Measure	Lenalidomide n=42 Participants Lenalidomide 10 mg/day plus prednisone X 28 days X 3 cycles
Overall Response Rate	0.26 Proportion of participants Interval 0.14 to 0.42

Adverse Events

Lenalidomide

Serious events: 47 serious events

Other events: 47 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Lenalidomide n=47 participants at risk Lenalidomide 10 mg/day plus prednisone X 28 days X 3 cycles
Blood and lymphatic system disorders Lymphopenia	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Blood and lymphatic system disorders Neutropenia	44.7% 21/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
General disorders Pain NOS	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Cardiac disorders Pericardial effusion	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Cardiac disorders Pulmonary hypertension	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Musculoskeletal and connective tissue disorders Soft tissue necrosis	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Nervous system disorders Syncope	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Blood and lymphatic system disorders Thrombocytopenia	19.1% 9/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Vascular disorders Thrombosis	4.3% 2/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Metabolism and nutrition disorders ALT Increased	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Gastrointestinal disorders Abdomen pain	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Metabolism and nutrition disorders Alkaline Phosphatase Increased	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Blood and lymphatic system disorders Anemia	44.7% 21/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Cardiac disorders Atrial fibrillation	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Musculoskeletal and connective tissue disorders Back pain	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Metabolism and nutrition disorders Bilirubin Increased	4.3% 2/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Gastrointestinal disorders Diarrhea without prior colostomy	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
General disorders Dizziness	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Respiratory, thoracic and mediastinal disorders Dyspnea	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Vascular disorders Edema limb	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
General disorders Fatigue	6.4% 3/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Cardiac disorders Heart block sick sinus syndrome	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Metabolism and nutrition disorders Hyperglycemia	4.3% 2/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Metabolism and nutrition disorders Hyperkalemia	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Metabolism and nutrition disorders Hyperuricemia	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Infections and infestations Infection with gr3-4 neutrophils	2.1% 1/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Blood and lymphatic system disorders Leukopenia	29.8% 14/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.

Other adverse events

Other adverse events
Measure	Lenalidomide n=47 participants at risk Lenalidomide 10 mg/day plus prednisone X 28 days X 3 cycles
Metabolism and nutrition disorders AST increased	17.0% 8/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Blood and lymphatic system disorders Anemia	91.5% 43/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Metabolism and nutrition disorders Anorexia	12.8% 6/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Metabolism and nutrition disorders Bilirubin Increased	17.0% 8/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Gastrointestinal disorders Constipation	21.3% 10/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Gastrointestinal disorders Diarrhea witout prior colostomy	44.7% 21/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
General disorders Dizziness	19.1% 9/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Gastrointestinal disorders Dyspepsia muco	17.0% 8/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Vascular disorders Edema limb	31.9% 15/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Musculoskeletal and connective tissue disorders Extremity limb pain	14.9% 7/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
General disorders Fatigue	40.4% 19/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
General disorders Headache	12.8% 6/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Metabolism and nutrition disorders Hyperglycemia	27.7% 13/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Metabolism and nutrition disorders Hypokalemia	17.0% 8/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
General disorders Insomnia	17.0% 8/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Blood and lymphatic system disorders Leukopenia	61.7% 29/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Blood and lymphatic system disorders Neutropenia	46.8% 22/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
General disorders Pain NOS	12.8% 6/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Skin and subcutaneous tissue disorders Pruritus	10.6% 5/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Skin and subcutaneous tissue disorders Rash	19.1% 9/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
General disorders Sweating	12.8% 6/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
General disorders Taste disturbance	12.8% 6/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
Blood and lymphatic system disorders Thrombocytopenia	57.4% 27/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.
General disorders Weight loss	14.9% 7/47 • During treatment (up to 6 months) and for 30 days after the end of treatment.

Additional Information

Study Statistician

ECOG Statistical Office

Phone: 617-632-3012

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60