Lenalidomide Versus Placebo in Myelodysplastic Syndromes With a Deletion 5q[31] Abnormality

The count of study participants who had no RBC transfusions for 26 consecutive weeks or more during the double-blind period.

Time frame: Up to 52 weeks

Population: The modified intent-to-treat (mITT) population included all participants with centrally-confirmed Low- or Int-1-risk MDS with del 5q\[31\] and documented RBC transfusion dependence who had received ≥ 1 dose of study drug.

The Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire (Yellen, 1997) was used to assess health-related quality of life (HRQoL). In addition to general HRQoL, the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning. The overall score range for the FACT-An is 0-188. Higher scores indicate better HRQoL.

Time frame: Baseline, Week 12

Population: Participants who had both Baseline and Week 12 FACT-An data.

The Trial Outcome Index-Anemia (TOI-An) composed of the physical and functional subscales of the FACT-G along with the Anemia subscale was used to assess health-related quality of life (HRQoL). The overall score range for the TOI-An is 0-136. Higher scores indicate better HRQoL.

Time frame: Baseline, Week 12

Population: Participants who had both Baseline and Week 12 TOI-An data.

The Trial Outcome Index-Fatigue(TOI-F) composed of the physical and functional subscales of the FACT-G along with the fatigue items from the Anemia subscale was used to assess health-related quality of life (HRQoL). The overall score range for the TOI-F is 0-108. Higher scores indicate better HRQoL.

Time frame: Baseline, Week 12

Population: Participants who had both Baseline and Week 12 TOI-F data.

Mean number of weeks that participants who achieved RBC transfusion independence for at least 182 days were able to maintain RBC transfusion independence. Both double-blind and open-label periods are included.

Time frame: up to 3 years

Population: The modified intent-to-treat (mITT) population included all participants who achieved RBC transfusion independence for at least 182 days.

Kaplan Meier estimate for median length of survival for study participants as they were randomized at the start of the study.

Time frame: up to 3 years

Population: Safety population: All randomized participants who received any Lenalidomide or placebo.

For participants who became RBC transfusion independent for at least 182 days during the double-blind study period, the mean maximum change from baseline in hemoglobin is summarized.

Time frame: Baseline, up to 52 weeks

Population: The modified intent-to-treat (mITT) population included all participants with centrally-confirmed Low- or Int-1-risk MDS with del 5q\[31\] and documented RBC transfusion dependence who had received ≥ 1 dose of study drug. MITT participants who were transfusion independent for \>= 182 study days are included.

Count of participant deaths throughout the entire study and reported by the original treatment assignment.

Time frame: up to 3 years

Population: Safety Population

The IWG criteria for bone marrow improvement: a complete remission is bone marrow sampling showing less than 5% myeloblasts with normal maturation of all cell lines, with no evidence for dysplasia. A partial remission is ≥ 50% decrease in blasts over pre-treatment. Bone marrow progression is a ≥ 50% increase in blasts that exceed the top range of the pretreatment percentile range: a) \<5% blasts b) 5-10% blasts c) 10-20% blasts d) 20-30% blasts. For example, a participant with \<5% blasts pretreatment with an on study blast increase of 50% which is now \>5% showed bone marrow progression.

Time frame: up to 52 weeks

Population: Intent to treat population. Participants represented in the treatment groups to which they were randomized. Placebo response is limited to the double-blind phase. There were 10 responders in the placebo group who achieved their response under lenalidomide treatment after crossover to open-label.

A major neutrophil response is defined by the International MDS Working Group (IWG) criteria as at least a 100% increase, or an absolute increase of ≥500/mm\^3 for participants with absolute neutrophil counts (ANC) of less than 1,500/mm\^3 before therapy, whichever is greater. A minor response for such participants is defined as an ANC increase of at least 100%, but absolute increase \<500/mm\^3.

Time frame: up to week 52

Population: The modified intent-to-treat (mITT) population included all participants with centrally-confirmed Low- or Int-1-risk MDS with del 5q\[31\] and documented RBC transfusion dependence who had received ≥ 1 dose of study drug. Additionally, the participant must have had a baseline absolute neutrophil counts (ANC) \< 1,000/mm\^3.

The International MDS Working Group (IWG) defines a major platelet response for participants with a pre-treatment platelet count of \<100,000/mm\^3 as an absolute increase of ≥30,000/mm\^3 whereas a minor response is defined as a ≥50% increase in platelet count with a net increase greater than 10,000/mm\^3 but less than 30,000/mm\^3.

Time frame: up to 52 weeks

Population: The modified intent-to-treat (mITT) population included all participants with centrally-confirmed Low- or Int-1-risk MDS with del 5q\[31\] and documented RBC transfusion dependence who had received ≥ 1 dose of study drug. Additionally, the participant must have had a baseline platelet count of \<100,000/mm\^3 to be included in the analysis.

The IWG criteria for evaluating cytogenetic response require a minimum of 20 baseline and post-baseline analyzable metaphases using conventional cytogenetic techniques. A major cytogenetic response is defined as no detectable cytogenetic abnormality if preexisting abnormality was present whereas a minor response requires ≥50% reduction in abnormal metaphases. Progression could be concluded based on as few as 3 metaphases if there were additional abnormalities. The best response is represented.

Time frame: up to 52 weeks

Population: Modified intent to treat population, which is defined as participants with centrally-confirmed Low- or Int-1-risk MDS with del 5q\[31\] and documented RBC transfusion dependence who had received ≥ 1 dose of study drug. Participants had to have had more than 1 post-baseline assessment in order to be evaluable for cytogenetic response.

Count of study participants who had no RBC transfusions during any 56 or more consecutive study days during the double-blind period.

Time frame: Up to 52 weeks

Population: The modified intent-to-treat (mITT) population included all participants with centrally-confirmed Low- or Int-1-risk MDS with del 5q\[31\] and documented RBC transfusion dependence who had received ≥ 1 dose of study drug.

Number of participants who progressed to acute myeloid leukemia during the study, summarized at three different timepoints: first 16 weeks of the double-blind study, week 52 of the double-blind study, and up to 36 months which includes the double-blind and open-label periods of the study. The counts are cumulative by timeframe.

Time frame: up to 3 years

Population: Intent to treat population. Participants represented in the treatment groups to which they were randomized.

Counts of study participants who had adverse events (AEs) during the double-blind period by MedDRA System Organ Class (SOC) and preferred term. A participant with multiple occurrences of an adverse event within a category is counted only once in that category. Adverse events were evaluated by the investigator. The National Cancer Institute (NCI)'s Common Terminology Criteria for AEs (CTCAE) was used to grade AE severity. Severity grade 3= severe and undesirable AE. Severity grade 4= life-threatening or disabling AE.

Time frame: up to week 52

Population: Safety population.