Trial Outcomes & Findings for Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence (NCT NCT01178281)

NCT ID: NCT01178281

Last Updated: 2019-07-17

Results Overview

RBC-transfusion independence was defined as the absence of RBC transfusions for any consecutive 84-day interval.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

267 participants

Primary outcome timeframe

168 days

Results posted on

2019-07-17

Participant Flow

Participants in the global study were enrolled at 72 clinical centers in 15 countries. In addition, the China-specific extension study enrolled participants with myeloproliferative neoplasm (MPN)-associated myelofibrosis and severe anemia not receiving red blood cell (RBC)-transfusions at 5 sites in China.

Participants in the global study were randomized 2:1 to receive blinded pomalidomide or placebo. All participants in the China extension received open-label pomalidomide. Randomization was stratified by age (≤ vs \>65 years), white blood cells (\< or ≥25 × 10⁹/L) and baseline transfusion requirement (≤ vs \>4 units RBC/28 days over the prior 84 days).

Participant milestones

Participant milestones
Measure	Pomalidomide 0.5 mg Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit (defined as a reduction from Baseline of ≥ 50% in RBC-transfusion frequency during the prior 84-day interval) could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	Placebo Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	China Extension: Pomalidomide 0.5 mg Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion. Participants who experienced anemia response could continue treatment until the response was lost or other criteria for treatment discontinuation applied.
Overall Study STARTED	168	84	15
Overall Study Received Study Drug	167	83	15
Overall Study COMPLETED	168	84	15
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Results of the global study and China extension study were analyzed separately.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pomalidomide 0.5 mg n=168 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who experienced clinical benefit (defined as a reduction from Baseline of ≥ 50% in RBC-transfusion frequency during the prior 84-day interval) could continue to receive pomalidomide until the definition of clinical benefit was no longer met or other criteria for treatment discontinuation applied.	Placebo n=84 Participants Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	China Extension: Pomalidomide 0.5 mg n=15 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion. Participants who experienced anemia response could continue treatment until the response was lost or other criteria for treatment discontinuation applied.	Total n=267 Participants Total of all reporting groups
Disease Sub-type Post-essential thrombocythemia myelofibrosis	23 Participants n=168 Participants	11 Participants n=84 Participants	3 Participants n=15 Participants	37 Participants n=267 Participants
Disease Sub-type Missing	1 Participants n=168 Participants	0 Participants n=84 Participants	0 Participants n=15 Participants	1 Participants n=267 Participants
Age, Continuous Global study	69.0 years n=168 Participants • Results of the global study and China extension study were analyzed separately.	69.0 years n=84 Participants • Results of the global study and China extension study were analyzed separately.	—	69.0 years n=252 Participants • Results of the global study and China extension study were analyzed separately.
Age, Continuous China extension study	—	—	63.0 years n=15 Participants • Results of the global study and China extension study were analyzed separately.	63.0 years n=15 Participants • Results of the global study and China extension study were analyzed separately.
Sex: Female, Male Female	41 Participants n=168 Participants	28 Participants n=84 Participants	6 Participants n=15 Participants	75 Participants n=267 Participants
Sex: Female, Male Male	127 Participants n=168 Participants	56 Participants n=84 Participants	9 Participants n=15 Participants	192 Participants n=267 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=168 Participants	1 Participants n=84 Participants	0 Participants n=15 Participants	4 Participants n=267 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	144 Participants n=168 Participants	79 Participants n=84 Participants	15 Participants n=15 Participants	238 Participants n=267 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	21 Participants n=168 Participants	4 Participants n=84 Participants	0 Participants n=15 Participants	25 Participants n=267 Participants
Race/Ethnicity, Customized American Indian or Alaskan Native	1 Participants n=168 Participants	0 Participants n=84 Participants	0 Participants n=15 Participants	1 Participants n=267 Participants
Race/Ethnicity, Customized Asian	20 Participants n=168 Participants	11 Participants n=84 Participants	15 Participants n=15 Participants	46 Participants n=267 Participants
Race/Ethnicity, Customized Black or African American	2 Participants n=168 Participants	3 Participants n=84 Participants	0 Participants n=15 Participants	5 Participants n=267 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islanders	1 Participants n=168 Participants	0 Participants n=84 Participants	0 Participants n=15 Participants	1 Participants n=267 Participants
Race/Ethnicity, Customized White	122 Participants n=168 Participants	66 Participants n=84 Participants	0 Participants n=15 Participants	188 Participants n=267 Participants
Race/Ethnicity, Customized Other	3 Participants n=168 Participants	0 Participants n=84 Participants	0 Participants n=15 Participants	3 Participants n=267 Participants
Race/Ethnicity, Customized Missing	19 Participants n=168 Participants	4 Participants n=84 Participants	0 Participants n=15 Participants	23 Participants n=267 Participants
Baseline RBC Transfusion Burden Global study	2.7 units per 28 days n=168 Participants • Data for the global study and China extension study were analyzed separately.	2.8 units per 28 days n=84 Participants • Data for the global study and China extension study were analyzed separately.	—	2.7 units per 28 days n=252 Participants • Data for the global study and China extension study were analyzed separately.
Baseline RBC Transfusion Burden China extension study	—	—	0.0 units per 28 days n=15 Participants • Data for the global study and China extension study were analyzed separately.	0.0 units per 28 days n=15 Participants • Data for the global study and China extension study were analyzed separately.
Eastern Cooperative Oncology Group (ECOG) Performance Status 0 (Fully active)	53 Participants n=168 Participants	22 Participants n=84 Participants	5 Participants n=15 Participants	80 Participants n=267 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status 1 (Restricted but ambulatory)	85 Participants n=168 Participants	47 Participants n=84 Participants	9 Participants n=15 Participants	141 Participants n=267 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status 2 (Ambulatory but unable to work)	30 Participants n=168 Participants	15 Participants n=84 Participants	1 Participants n=15 Participants	46 Participants n=267 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status 3 (Limited self-care)	0 Participants n=168 Participants	0 Participants n=84 Participants	0 Participants n=15 Participants	0 Participants n=267 Participants
Eastern Cooperative Oncology Group (ECOG) Performance Status 4 (Completely disabled)	0 Participants n=168 Participants	0 Participants n=84 Participants	0 Participants n=15 Participants	0 Participants n=267 Participants
Disease Sub-type Primary myelofibrosis	127 Participants n=168 Participants	65 Participants n=84 Participants	10 Participants n=15 Participants	202 Participants n=267 Participants
Disease Sub-type Post-polycythemia vera myelofibrosis	17 Participants n=168 Participants	8 Participants n=84 Participants	2 Participants n=15 Participants	27 Participants n=267 Participants

PRIMARY outcome

Timeframe: 168 days

Population: Global study intent to treat population (ITT) which includes all participants randomized to either of the two study drugs, regardless of whether or not any study drug was actually taken.

RBC-transfusion independence was defined as the absence of RBC transfusions for any consecutive 84-day interval.

Outcome measures

Outcome measures
Measure	Pomalidomide 0.5 mg n=168 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	Placebo n=84 Participants Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	China Extension: Pomalidomide 0.5 mg Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion.
Percentage of Participants Who Achieved RBC-Transfusion Independence	17.3 percentage of participants Interval 11.88 to 23.84	16.7 percentage of participants Interval 9.42 to 26.38	—

PRIMARY outcome

Timeframe: From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks.

Population: China extension intent-to-treat population, which includes all participants enrolled in the China extension study.

A response in the China extension study was defined as an increase in hemoglobin ≥ 15 g/L above baseline value (in the absence of RBC transfusion) for ≥ 84 consecutive days.

Outcome measures

Outcome measures
Measure	Pomalidomide 0.5 mg n=15 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	Placebo Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	China Extension: Pomalidomide 0.5 mg Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion.
China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days	1 Participants	—	—

SECONDARY outcome

Timeframe: From first dose of study drug up to end of study; median follow-up time was 19.1 months in the pomalidomide 0.5 mg arm and 17.6 months in the placebo arm.

Population: Global study intent-to-treat population

The time from randomization to the death or to the latest date when participants are known to be alive. Overall survival was analyzed using Kaplan-Meier method; participants who were alive or lost to follow-up were censored at the latest date they were known to be alive.

Outcome measures

Outcome measures
Measure	Pomalidomide 0.5 mg n=168 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	Placebo n=84 Participants Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	China Extension: Pomalidomide 0.5 mg Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion.
Overall Survival	24.2 months Interval 19.5 to 33.5	26.2 months Interval 18.0 to 32.6	—

SECONDARY outcome

Timeframe: From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm.

Population: Global study intent-to-treat population with an 84-day RBC-transfusion independence response

The duration of RBC-transfusion independence is the time from the date at which the first RBC-transfusion independence started to the date of another RBC-transfusion given at least 84 days after the time the transfusion independence started. The duration of the RBC-transfusion independence was analyzed using the Kaplan-Meier method. Data were censored at the end of the treatment phase for participants who had not received another RBC-transfusion after the start of transfusion independence by the end of treatment phase.

Outcome measures

Outcome measures
Measure	Pomalidomide 0.5 mg n=29 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	Placebo n=14 Participants Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	China Extension: Pomalidomide 0.5 mg Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion.
Duration of RBC-Transfusion Independence	NA months Interval 4.8 to Could not be calculated due to the low number of participants with an event	5.8 months Interval 3.0 to Could not be calculated due to the low number of participants with an event	—

SECONDARY outcome

Timeframe: 168 days

Population: Global study intent-to-treat population with an 84-day RBC-transfusion independence response

Time to response was measured from first dose of study drug to the start of the first response. The start date of the response was defined as one day after the last date of an RBC-transfusion for participants who received a RBC-transfusion after the first dose, and as the date of the first dose of study drug for participants who received no RBC-transfusions during the 84 days after the first dose of study drug.

Outcome measures

Outcome measures
Measure	Pomalidomide 0.5 mg n=29 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	Placebo n=14 Participants Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	China Extension: Pomalidomide 0.5 mg Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion.
Time to RBC-Transfusion Independence	6.9 weeks Interval 0.1 to 20.1	2.4 weeks Interval 0.1 to 15.4	—

SECONDARY outcome

Timeframe: From the first dose of study drug until 28 days after last dose; median treatment duration was 23.7 weeks in the pomalidomide arm, 23.9 weeks in the placebo arm, and 24.0 weeks in the China extension pomalidomide arm.

Population: Participants who received at least 1 dose of study drug

A TEAE is an adverse event (AE) that starts on or after the first dose of study drug. The severity of each AE was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE),Version 4.0 and according to the following scale: Grade 1 = Mild (transient or mild discomfort; no limitation in activity; no medical intervention/therapy required); Grade 2 = Moderate (mild to moderate limitation in activity, some assistance may be needed; minimal medical intervention/therapy required); Grade 3 = Severe (marked limitation in activity, assistance usually required; medical intervention/therapy required, hospitalization possible); Grade 4 = Life-threatening (extreme limitation in activity, significant assistance or medical intervention/therapy required, hospitalization or hospice care probable); Grade 5 = Death Drug-related (related) AEs are those suspected by the Investigator as being related to administration of study drug

Outcome measures

Outcome measures
Measure	Pomalidomide 0.5 mg n=167 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	Placebo n=83 Participants Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	China Extension: Pomalidomide 0.5 mg n=15 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion.
Number of Participants With Treatment-emergent Adverse Events (TEAE) Adverse event suspected as related to study drug	90 Participants	32 Participants	3 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) Adverse event leading to dose interruption	48 Participants	17 Participants	2 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) Drug-related AE leading to dose interruption	26 Participants	6 Participants	1 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) Related AE leading to study drug discontinuation	21 Participants	8 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) Grade 3/4 adverse event	100 Participants	44 Participants	4 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) Any adverse event (AE)	164 Participants	81 Participants	12 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) AE leading to discontinuation of study drug	53 Participants	14 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) Grade 3/4 AE related to study drug	45 Participants	13 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) Grade 3/4 AE leading to study drug discontinuation	33 Participants	9 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) Grade 3/4 AE leading to dose interruption	36 Participants	14 Participants	1 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) Grade 5 adverse event	17 Participants	10 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) Grade 5 AE related to study drug	1 Participants	3 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) Serious adverse event (SAE)	76 Participants	29 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) SAE related to study drug	24 Participants	7 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) SAE leading to discontinuation of study drug	31 Participants	8 Participants	0 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAE) SAE leading to dose interruption	22 Participants	7 Participants	0 Participants

SECONDARY outcome

Population: Analysis of healthcare resource utilization data were not collected during the study and no analysis were performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline and Days 85 and 169

Population: Global study intent-to-treat population with available data at baseline and each time point

EQ-5D is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D includes 2 components: the EQ-5D health state profile (descriptive system) and the EQ-5D visual analog scale (VAS). For the health state profile participants rate their perceived health state today on 5 dimensions: mobility, selfcare, usual activities, pain/discomfort, and anxiety/depression on a Likert-type scale from 1 to 3, where 1 = "no problems," 2 = "some problems," and 3 = "extreme problems." The EQ-5D Health Utility Index (HUI) was generated from the five health state domain scores, and ranges from -0.594 (worst) and 1 (best) imaginable health state, with -0.594 representing an "unconscious" health state.

Outcome measures

Outcome measures
Measure	Pomalidomide 0.5 mg n=162 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	Placebo n=82 Participants Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	China Extension: Pomalidomide 0.5 mg Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion.
Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score Day 85	-0.0385 score on a scale Standard Deviation 0.2480	-0.0298 score on a scale Standard Deviation 0.2129	—
Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score Day 169	-0.0202 score on a scale Standard Deviation 0.1666	0.0766 score on a scale Standard Deviation 0.1546	—

SECONDARY outcome

Timeframe: Baseline and Days 85 and 169

Population: Global study intent-to-treat population with available data at baseline and each time point.

EQ-5D is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D includes 2 components: the EQ-5D health state profile (descriptive system) and the EQ-5D visual analog scale (VAS). On the VAS the participant rates his/her health state on a line from 0 (worst imaginable health) to 100 (best imaginable health).

Outcome measures

Outcome measures
Measure	Pomalidomide 0.5 mg n=161 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	Placebo n=81 Participants Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	China Extension: Pomalidomide 0.5 mg Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion.
Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale Day 85	2.0 units on a scale Standard Deviation 20.78	-1.4 units on a scale Standard Deviation 14.07	—
Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale Day 169	2.9 units on a scale Standard Deviation 22.85	0.3 units on a scale Standard Deviation 24.25	—

SECONDARY outcome

Timeframe: Baseline and Days 85 and 169

Population: Global study intent-to-treat population with available data at baseline and each time point.

The FACT-An is a 47-item, cancer-specific questionnaire consisting of a core 27-item general questionnaire measuring the four general domains of QoL (physical, social/family, emotional and functional well-being), and an additional 20-item anemia questionnaire (FACT-An Anemia subscale) that measures 13 fatigue-associated items (FACT-F Fatigue subscale) and seven non-fatigue-related items. Each item is scored using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a bit; and 4 = Very much). FACT-An total score is calculated by adding all the FACT-An subscales together. The total score ranges from 0-188 with higher scores representing better QOL.

Outcome measures

Outcome measures
Measure	Pomalidomide 0.5 mg n=159 Participants Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	Placebo n=81 Participants Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.	China Extension: Pomalidomide 0.5 mg Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion.
Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score Day 169	6.2 units on a scale Standard Deviation 20.49	11.9 units on a scale Standard Deviation 18.60	—
Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score Day 85	-2.1 units on a scale Standard Deviation 20.32	4.3 units on a scale Standard Deviation 18.73	—

Adverse Events

Global Study: Pomalidomide 0.5 mg

Serious events: 76 serious events

Other events: 155 other events

Deaths: 115 deaths

Global Study: Placebo

Serious events: 29 serious events

Other events: 76 other events

Deaths: 54 deaths

China Extension: Pomalidomide 0.5 mg

Serious events: 0 serious events

Other events: 12 other events

Deaths: 8 deaths

Serious adverse events

Other adverse events

Additional Information

Anne McClain

Celgene Corporation

Phone: 1-888-260-1599

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Results from a center cannot be submitted for publication before results of multicenter study are published unless it is more than 1 year since study completion. Then Investigator can publish if the manuscript is submitted to Celgene 60 days prior to submission. If Celgene decides a publication would hinder drug development, Investigator must delay submission for up to 90 days. Investigator must delete confidential information before submission or defer publication to permit patent applications.
Publication restrictions are in place

Restriction type: OTHER