RESUME: Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence

• Sponsor: Celgene (Industry)
• Overall Status: Completed
• CT.gov ID: NCT01178281
• Collaborator: (none)
• Enrollment: 267
• Locations: 87
• Arms: 3
• Actual Duration (Months): 92.2
• Patients Per Site: 3.1
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

The objective of this study is to determine whether pomalidomide is safe and effective in reversing red blood cell (RBC)-transfusion-dependence in persons with myeloproliferative neoplasm (MPN)-associated myelofibrosis (global study) and in reversing anemia in Chinese with MPN-associated myelofibrosis and severe anemia not receiving RBC-transfusions (China extension study only)

Condition or Disease	Intervention/Treatment	Phase
Primary Myelofibrosis; MPN-associated Myelofibrosis	Drug: Pomalidomide 0.5 mg; Drug: Placebo; Drug: Pomalidomide	Phase 3

Detailed Description

The multicenter global study was conducted in 15 countries including Australia, Austria, Belgium, Canada, China, France, Germany, Italy, Japan, the Netherlands, Russia, Spain, Sweden, the United Kingdom, and the United States. The global study enrolled participants with myeloproliferative neoplasm (MPN)-associated myelofibrosis and RBC-transfusion-dependence. Participants were randomly assigned to receive pomalidomide or placebo in a blinded fashion.

In most countries participating in the global study, RBC-transfusions are typically given for a hemoglobin level <80-90 g/L. In China, RBC-transfusions are rarely given unless the hemoglobin level is <60 g/L. Consequently, few Chinese with MPN-associated myelofibrosis meet RBC-transfusion-dependence criteria of the global study. A China-specific extension was developed to test the ability of pomalidomide to improve severe anemia (defined as a hemoglobin < 80 g/L for ≥ 84 days in persons not receiving RBC-transfusions).

The China-specific extension study consisted of a single-arm, open-label study in adults with MPN-associated myelofibrosis and severe anemia not receiving RBC transfusions with the objective of describing the frequency of anemia response.

The Global (intent-to-treat [ITT] and safety) population in the main study and the China extension (ITT and safety) population are mutually exclusive.

Study Design

• Study Type: Interventional
• Actual Enrollment: 267 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Compare Efficacy and Safety of Pomalidomide in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Red Blood Cell-Transfusion-Dependence
• Actual Study Start Date: Sep 8, 2010
• Actual Primary Completion Date: Jan 1, 2013
• Actual Study Completion Date: May 15, 2018

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 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.	Drug: Pomalidomide 0.5 mg; Pomalidomide 0.5 mg capsule taken by mouth once daily. Immunomodulatory agent with demonstrated efficacy in the treatment of subjects with RBC-transfusion-dependence associated with MNP-associated myelofibrosis.; Other Names: CC-4047; Pomalyst; Imnovid
Placebo Comparator: 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.	Drug: Placebo; Placebo Comparator to active drug; Placebo capsule taken by mouth once daily
Experimental: 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.	Drug: Pomalidomide; Pomalidomide 0.5 mg capsule taken by mouth once daily.; Other Names: CC-4047 Pomalyst Imnovid

Outcome Measures

Primary Outcome Measures

1. Percentage of Participants Who Achieved RBC-Transfusion Independence [168 days]

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

2. China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days [From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks.]

   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.

Secondary Outcome Measures

1. Overall Survival [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.]

   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.

2. Duration of RBC-Transfusion Independence [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.]

   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.

3. Time to RBC-Transfusion Independence [168 days]

   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.

4. Number of Participants With Treatment-emergent Adverse Events (TEAE) [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.]

   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

5. Healthcare Resource Utilization [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.]

6. Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score [Baseline and Days 85 and 169]

   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.

7. Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale [Baseline and Days 85 and 169]

   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).

8. Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score [Baseline and Days 85 and 169]

   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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Age ≥ 18 years

• Myeloproliferative-neoplasm (MPN)-associated myelofibrosis

• RBC-transfusion-dependence (global study):

• Average RBC-transfusion frequency ≥ 2 units/28 days over at least the 84 days immediately prior to randomization. There must be no interval > 42 days without ≥ 1 RBC-transfusion.

• Only RBC-transfusions given when the hemoglobin ≤ 90 g/L³ are scored in

determining eligibility.

• RBC-transfusions due to bleeding are not scored in determining eligibility.

• RBC-transfusions due to chemotherapy-induced anemia are not scored in determining eligibility.

• Severe anemia (China-specific extension):

• ≥ 2 hemoglobin concentrations ≤ 80 g/L for ≥ 84 days immediately before the day of enrollment.

• No RBC-transfusion within 6 months prior to enrollment.

• Hemoglobin ≤ 130 g/L at randomization (global study); ≤ 80 g/L at enrollment in the China-specific extension.

• Bone marrow biopsy within 6 months (global study only).

• Inappropriate to receive blood cell or bone marrow allotransplant, erythropoietin and androgenic steroids

• Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.

• Agree to follow pregnancy precautions as required by the protocol.

• Agree to receive counseling related to teratogenic and other risks of pomalidomide.

• Agree not to donate blood or semen.

Exclusion Criteria:

• Prior blood cell or bone marrow allotransplant.

• Use of drugs to treat MPN-associated myelofibrosis ≤ 30 days before starting study drug.

• Treatment with erythropoietin or androgenic steroids ≤ 84 days before starting study drug.

• Anemia due to reasons other than MPN-associated myelofibrosis.

• Pregnant or lactating females.

• More than 10% blasts by bone marrow examination or more than 10% blasts in blood in consecutive measurements spanning at least 8 weeks

• Prior history of malignancies,other than the disease being studied, unless the subject has been free of the malignancy for ≥ 5 years with the following exceptions:

• Carcinoma in situ of the cervix

• Carcinoma in situ of the breast

• Incidental histologic finding of prostate cancer (T 1a or T 1b using TNM [tumor, nodes, metastasis] clinical staging system)

• Human immunodeficiency virus (HIV) infection, active hepatitis B virus (HBV) or hepatitis C virus (HCV) infections.

• Prior treatment with pomalidomide.

• Allergic reaction or rash after treatment with thalidomide or lenalidomide

• Any of the following laboratory abnormalities:

• Neutrophils < 0.5x10^9 /L

• Platelets < 25 x 10^9 /L

• Estimated glomerular filtration rate (kidney function) < 30 mL/min/1.73 m²

• Aspartate aminotransferase (AST) and alanine transaminase (ALT) > 3.0 x upper limit of normal (ULN)

• Total bilirubin ≥ 4 x ULN;

• Uncontrolled hyperthyroidism or hypothyroidism.

• Deep venous thrombosis (DVT) or pulmonary embolus (PE) < 6 months before starting study drug

• Clinically-important heart disease within the past 6 months

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Mayo Clinic	Scottsdale	Arizona	United States	85259
2	UCLA School of Medicine	Los Angeles	California	United States	90095
3	University of Florida Shands Cancer Center	Gainesville	Florida	United States	32610
4	Mayo Clinic	Jacksonville	Florida	United States	32224
5	University of Illinois at Chicago	Chicago	Illinois	United States	60612
6	University of Michigan Comprehensive Cancer Center	Ann Arbor	Michigan	United States	48109
7	Mayo Clinic	Rochester	Minnesota	United States	55905
8	Mount Sinai School of Medicine Brookdale University Hospital	Brooklyn	New York	United States	11212
9	Weill Medical College of Cornell University	New York	New York	United States	10021
10	Ruttenberg Treatment Center	New York	New York	United States	10029
11	Memorial Sloan Kettering Cancer Center	New York	New York	United States	10065
12	Medicine Taussig Cancer Institute	Cleveland	Ohio	United States	44195
13	Thomas Jefferson University	Philadelphia	Pennsylvania	United States	19107
14	Avera Hematology and Transplant	Sioux Falls	South Dakota	United States	57105
15	MD Anderson Cancer Center	Houston	Texas	United States	77030
16	University of Utah	Salt Lake City	Utah	United States	84132
17	Fred Hutchinson Cancer Center	Seattle	Washington	United States	98109
18	Gosford Hospital	Gosford	New South Wales	Australia	2250
19	Royal North Shore Hospital	St. Leonards	New South Wales	Australia	2065
20	Frankston Hospital	Frankston	Victoria	Australia	3199
21	Royal Melbourne Hospital	Parkville	Victoria	Australia	3050
22	Medizinische Universitatklinik Graz	Graz		Austria	A-8036
23	Medizinische Universitat Innsbruck	Innsbruck		Austria	A-6020
24	Medizinische Universitat Wien	Vienna		Austria	A-1190
25	Algemeen Ziekenhuis Sint-Jan	Brugge		Belgium	8000
26	Grand Hopital de Charleroi	Charleroi		Belgium	6000
27	Universitaire Ziekenhuis Leuven Gathuisberg	Leuven		Belgium	3000
28	Cross Cancer Institute	Edmonton	Alberta	Canada	T6G lz2
29	Vancouver General Hospital	Vancouver	British Columbia	Canada	V5Z 1M9
30	Princess Margaret Hospital	Toronto	Ontario	Canada	M5G 2M9
31	Centre Hospitalier de L'Universite de Montreal	Montreal,		Canada
32	Peking University People's Hospital	Beijing		China	100044
33	Peking Union Medical College Hospital	Beijing		China	100730
34	Jiangsu Province Hospital	Jiangsu		China	210029
35	Shanghai Ruijin Hospital	Shanghai		China	200025
36	West China Hospital, Sichuan University	Sichuan		China	610041
37	Blood Disease Hospital Chinese Academy of Medical Sciences	Tianjin		China	300041
38	Hopital Albert Michallon	La Tronche		France	38043
39	Hopital Saint Vincent de Paul	Lille		France	59020
40	CHU Dupuytren	Limoges		France	87042
41	Hopital Saint-Louis	Paris		France	75475
42	CHRU - Hopital du Haut Leveque	Pessac		France	33604
43	Hopitaux Universitaires de Strasbourg, CHU Haute-Pierre	Strasbourg		France	67098
44	Hopital Purpan	Toulouse		France	31059
45	Institut Gustave Roussy	Villejuif		France	94805
46	Universitatsklinikum Aachen	Aachen		Germany	52074
47	Medizinische Hochschule Hannover	Hannover		Germany	30625
48	Universitatsklinikum Leipzig	Leipzig		Germany	04103
49	Johannes Wesling Klinikum Minden	Minden		Germany	32429
50	Universitatsklinikum Ulm	Ulm		Germany	89081
51	Azienda Ospedaliera Universitaria Consorziale Policlinico di Bari	Bari		Italy	70124
52	Ospedali Riuniti di Bergamo	Bergamo		Italy	24128
53	Azienda Ospedaliera Universitaria Careggi	Firenze		Italy	50134
54	Azienda Ospedaliera Universitaria Federico II di Napoli	Napoli		Italy	80131
55	Azienda Ospedaliera San Luigi Gonzaga	Orbassano		Italy	10043
56	IRCCS Fondazione Policlinico San Matteo, Universita di Pavia, Centro per lo Studio della Mielofibrosi	Pavia		Italy	27100
57	IRCCS Fondazione Policlinico San Matteo, Universita di Pavia, Ematologia	Pavia		Italy	27100
58	Ospedale di Circolo e Fondazione Macchi Varese	Varese		Italy	21100
59	Juntendo University Hospital	Bunkyou-ku		Japan	113-8431
60	Kyushu University Hospital	Fukuoka City		Japan	812-8582
61	Tokai University Hospital	Isehara City		Japan	259-1193
62	Kyoto University Hospital	Kyoto City		Japan	606-8507
63	Nagasaki University Hospital	Nagasaki City		Japan	852-8501
64	Tokyo Medical University Hospital	Shinjuku		Japan	160-0023
65	VU University Medical Center	Amsterdam		Netherlands	1081 HV
66	Erasmus Medish Centrum	Rotterdam		Netherlands	3015 CE
67	University Medical Center Utrecht	Utrecht		Netherlands	3584 CX
68	Wojewodzki Szpital Specjalistyczny im. F.Chopina	Rzeszow		Poland	35-055
69	Samodzielny Publiczny Szpital Kliniczny Nr 1 PAM	Szczecin		Poland	71-242
70	Centralny Szpital Kliniczny MSWiA	Warsaw		Poland	02-507
71	Russian Scientific Haematology Centre	Moscow		Russian Federation	125167
72	Federal State Institution Russian Scientific-research Institute of Hematology and Transfusiology of Federal Medical-Biological Agency of Russia	Saint-Petersburg		Russian Federation	191024
73	State Pavlov Medical University	Saint-Petersburg		Russian Federation	196022
74	Federal State Institution "Federal Centre of Heart, Blood and Endocrinology of Rosmedtechnologies named after V.A. Almazov"	Saint-Petersburg		Russian Federation	197341
75	Hospital Clinic I Provincial de Barcelona	Barcelona		Spain	08036
76	Hospital Universitario Puerta De Hierro Majadahonda	Majadahonda		Spain	28222
77	Hospital Clinico de Salamanca	Salamanca		Spain	37007
78	Hospital Clinico de Valencia	Valencia		Spain	46010
79	Skane University Hospital	Lund		Sweden	22185
80	Karolinska University Hospital Huddinge	Stockholm		Sweden	14185
81	Belfast City Hospital	Belfast		United Kingdom	BT9 7AB
82	Beatson Oncology Centre	Glasgow		United Kingdom	G12 0YN
83	John Radcliffe Hospital NHS Trust	Headington		United Kingdom	OX3 9DU
84	St. Thomas Hospital	London		United Kingdom	SE1 9RT
85	Hammersmith Hospital	London		United Kingdom	W12 ONN
86	Freeman Hospital	Newcastle upon Tyne		United Kingdom	NE7 7DN
87	Royal Hallamshire Hospital	Sheffield		United Kingdom	S10 2JF

Sponsors and Collaborators

• Celgene

Investigators

• Study Director: Robert Peter P Gale, MD, Ph.D., Celgene Corporation

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Celgene
• ClinicalTrials.gov Identifier: NCT01178281
• Other Study ID Numbers: CC-4047-MF-002; 2010-018965-42
• First Posted: Aug 10, 2010
• Last Update Posted: Jul 17, 2019
• Last Verified: Jun 1, 2019

• Keywords provided by Celgene: Myelofibrosis; Post-polycythemia vera myelofibrosis; Post-essential thrombocythemia myelofibrosis; RBC-transfusion-dependence; Primary Myelofibrosis
• Additional relevant MeSH terms: Neoplasms; Primary Myelofibrosis; Myeloproliferative Disorders; Thalidomide; Pomalidomide

Study Results

Participant Flow

Recruitment Details	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.
Pre-assignment Detail	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).

Arm/Group Title	Pomalidomide 0.5 mg	Placebo	China Extension: Pomalidomide 0.5 mg
Arm/Group Description	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.	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.	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.
Period Title: Overall Study
STARTED	168	84	15
Received Study Drug	167	83	15
COMPLETED	168	84	15
NOT COMPLETED	0	0	0

Baseline Characteristics

Arm/Group Title	Pomalidomide 0.5 mg	Placebo	China Extension: Pomalidomide 0.5 mg	Total
Arm/Group Description	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.	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.	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 of all reporting groups
Overall Participants	168	84	15	267
Age (years) [Median (Full Range) ]
Global study	69.0	69.0	69.0
China extension study	63.0	63.0
Sex: Female, Male (Count of Participants)
Female	41 24.4%	28 33.3%	6 40%	75 28.1%
Male	127 75.6%	56 66.7%	9 60%	192 71.9%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	3 1.8%	1 1.2%	0 0%	4 1.5%
Not Hispanic or Latino	144 85.7%	79 94%	15 100%	238 89.1%
Unknown or Not Reported	21 12.5%	4 4.8%	0 0%	25 9.4%
Race/Ethnicity, Customized (Count of Participants)
American Indian or Alaskan Native	1 0.6%	0 0%	0 0%	1 0.4%
Asian	20 11.9%	11 13.1%	15 100%	46 17.2%
Black or African American	2 1.2%	3 3.6%	0 0%	5 1.9%
Native Hawaiian or Other Pacific Islanders	1 0.6%	0 0%	0 0%	1 0.4%
White	122 72.6%	66 78.6%	0 0%	188 70.4%
Other	3 1.8%	0 0%	0 0%	3 1.1%
Missing	19 11.3%	4 4.8%	0 0%	23 8.6%
Baseline RBC Transfusion Burden (units per 28 days) [Median (Inter-Quartile Range) ]
Global study	2.7	2.8	2.7
China extension study	0.0	0.0
Eastern Cooperative Oncology Group (ECOG) Performance Status (Count of Participants)
0 (Fully active)	53 31.5%	22 26.2%	5 33.3%	80 30%
1 (Restricted but ambulatory)	85 50.6%	47 56%	9 60%	141 52.8%
2 (Ambulatory but unable to work)	30 17.9%	15 17.9%	1 6.7%	46 17.2%
3 (Limited self-care)	0 0%	0 0%	0 0%	0 0%
4 (Completely disabled)	0 0%	0 0%	0 0%	0 0%
Disease Sub-type (Count of Participants)
Primary myelofibrosis	127 75.6%	65 77.4%	10 66.7%	202 75.7%
Post-polycythemia vera myelofibrosis	17 10.1%	8 9.5%	2 13.3%	27 10.1%
Post-essential thrombocythemia myelofibrosis	23 13.7%	11 13.1%	3 20%	37 13.9%
Missing	1 0.6%	0 0%	0 0%	1 0.4%

Outcome Measures

1. Primary Outcome

Title	Percentage of Participants Who Achieved RBC-Transfusion Independence
Description	RBC-transfusion independence was defined as the absence of RBC transfusions for any consecutive 84-day interval.
Time Frame	168 days

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Pomalidomide 0.5 mg	Placebo
Arm/Group Description	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.	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.
Measure Participants	168	84
Number (95% Confidence Interval) [percentage of participants]	17.3 10.3%	16.7 19.9%

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Pomalidomide 0.5 mg, Placebo
	Comments
	Type of Statistical Test	Other
	Comments
Statistical Test of Hypothesis	p-Value	1.000
	Comments
	Method	Fisher Exact
	Comments

2. Primary Outcome

Title	China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days
Description	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.
Time Frame	From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks.

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	China Extension: Pomalidomide 0.5 mg
Arm/Group Description	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.
Measure Participants	15
Count of Participants [Participants]	1 0.6%

3. Secondary Outcome

Title	Overall Survival
Description	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.
Time Frame	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.

Outcome Measure Data

Analysis Population Description

Global study intent-to-treat population

Arm/Group Title	Pomalidomide 0.5 mg	Placebo
Arm/Group Description	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.	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.
Measure Participants	168	84
Median (95% Confidence Interval) [months]	24.2	26.2

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Pomalidomide 0.5 mg, Placebo
	Comments
	Type of Statistical Test	Other
	Comments
Statistical Test of Hypothesis	p-Value	0.929
	Comments
	Method	Log Rank
	Comments

4. Secondary Outcome

Title	Duration of RBC-Transfusion Independence
Description	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.
Time Frame	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.

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Pomalidomide 0.5 mg	Placebo
Arm/Group Description	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.	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.
Measure Participants	29	14
Median (95% Confidence Interval) [months]	NA	5.8

5. Secondary Outcome

Title	Time to RBC-Transfusion Independence
Description	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.
Time Frame	168 days

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Pomalidomide 0.5 mg	Placebo
Arm/Group Description	Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression.	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.
Measure Participants	29	14
Median (Full Range) [weeks]	6.9	2.4

6. Secondary Outcome

Title	Number of Participants With Treatment-emergent Adverse Events (TEAE)
Description	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
Time Frame	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.

Outcome Measure Data

Analysis Population Description

Participants who received at least 1 dose of study drug

Arm/Group Title	Pomalidomide 0.5 mg	Placebo	China Extension: Pomalidomide 0.5 mg
Arm/Group Description	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.	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.	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.
Measure Participants	167	83	15
Any adverse event (AE)	164 97.6%	81 96.4%	12 80%
Adverse event suspected as related to study drug	90 53.6%	32 38.1%	3 20%
Adverse event leading to dose interruption	48 28.6%	17 20.2%	2 13.3%
Drug-related AE leading to dose interruption	26 15.5%	6 7.1%	1 6.7%
AE leading to discontinuation of study drug	53 31.5%	14 16.7%	0 0%
Related AE leading to study drug discontinuation	21 12.5%	8 9.5%	0 0%
Grade 3/4 adverse event	100 59.5%	44 52.4%	4 26.7%
Grade 3/4 AE related to study drug	45 26.8%	13 15.5%	0 0%
Grade 3/4 AE leading to study drug discontinuation	33 19.6%	9 10.7%	0 0%
Grade 3/4 AE leading to dose interruption	36 21.4%	14 16.7%	1 6.7%
Grade 5 adverse event	17 10.1%	10 11.9%	0 0%
Grade 5 AE related to study drug	1 0.6%	3 3.6%	0 0%
Serious adverse event (SAE)	76 45.2%	29 34.5%	0 0%
SAE related to study drug	24 14.3%	7 8.3%	0 0%
SAE leading to discontinuation of study drug	31 18.5%	8 9.5%	0 0%
SAE leading to dose interruption	22 13.1%	7 8.3%	0 0%

7. Secondary Outcome

Title	Healthcare Resource Utilization
Time Frame	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.

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Pomalidomide 0.5 mg	Placebo
Arm/Group Description	Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression.	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.
Measure Participants	0	0

8. Secondary Outcome

Title	Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score
Description	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.
Time Frame	Baseline and Days 85 and 169

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Pomalidomide 0.5 mg	Placebo
Arm/Group Description	Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression.	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.
Measure Participants	162	82
Day 85	-0.0385 (0.2480)	-0.0298 (0.2129)
Day 169	-0.0202 (0.1666)	0.0766 (0.1546)

9. Secondary Outcome

Title	Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale
Description	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).
Time Frame	Baseline and Days 85 and 169

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Pomalidomide 0.5 mg	Placebo
Arm/Group Description	Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression.	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.
Measure Participants	161	81
Day 85	2.0 (20.78)	-1.4 (14.07)
Day 169	2.9 (22.85)	0.3 (24.25)

10. Secondary Outcome

Title	Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score
Description	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.
Time Frame	Baseline and Days 85 and 169

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Pomalidomide 0.5 mg	Placebo
Arm/Group Description	Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression.	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.
Measure Participants	159	81
Day 85	-2.1 (20.32)	4.3 (18.73)
Day 169	6.2 (20.49)	11.9 (18.60)

Adverse Events

Time Frame	All-cause mortality data are reported from first dose of study drug up to end of study; maximum follow-up time was 85 months. Adverse events are reported 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.
Adverse Event Reporting Description	The safety population includes all participants who received at least one dose of study drug; one participant randomized to pomalidomide and one participant randomized to placebo in the global study did not receive treatment and are excluded from the safety population.
Arm/Group Title	Global Study: Pomalidomide 0.5 mg		Global Study: Placebo		China Extension: Pomalidomide 0.5 mg
Arm/Group Description	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.		Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who experienced clinical benefit could continue to receive placebo until the definition of clinical benefit was no longer met or other criteria for treatment discontinuation applied.		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.
All Cause Mortality
	Global Study: Pomalidomide 0.5 mg		Global Study: Placebo		China Extension: Pomalidomide 0.5 mg
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	115/167 (68.9%)		54/83 (65.1%)		8/15 (53.3%)
Serious Adverse Events
	Global Study: Pomalidomide 0.5 mg		Global Study: Placebo		China Extension: Pomalidomide 0.5 mg
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	76/167 (45.5%)		29/83 (34.9%)		0/15 (0%)
Blood and lymphatic system disorders
ANAEMIA	10/167 (6%)		3/83 (3.6%)		0/15 (0%)
DISSEMINATED INTRAVASCULAR COAGULATION	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
FEBRILE NEUTROPENIA	3/167 (1.8%)		1/83 (1.2%)		0/15 (0%)
HAEMOLYSIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
HAEMOLYTIC ANAEMIA	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
NEUTROPENIA	1/167 (0.6%)		1/83 (1.2%)		0/15 (0%)
PANCYTOPENIA	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
SPLENIC EMBOLISM	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
SPLENIC INFARCTION	1/167 (0.6%)		1/83 (1.2%)		0/15 (0%)
SPLENOMEGALY	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
THROMBOCYTOPENIA	3/167 (1.8%)		2/83 (2.4%)		0/15 (0%)
Cardiac disorders
ACUTE CORONARY SYNDROME	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
ACUTE MYOCARDIAL INFARCTION	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
ATRIAL FIBRILLATION	2/167 (1.2%)		0/83 (0%)		0/15 (0%)
CARDIAC ARREST	2/167 (1.2%)		0/83 (0%)		0/15 (0%)
CARDIAC FAILURE	5/167 (3%)		1/83 (1.2%)		0/15 (0%)
CARDIAC FAILURE ACUTE	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
CARDIAC FAILURE CONGESTIVE	3/167 (1.8%)		0/83 (0%)		0/15 (0%)
CONDUCTION DISORDER	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
CORONARY ARTERY DISEASE	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
Gastrointestinal disorders
ABDOMINAL DISTENSION	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
ASCITES	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
DIARRHOEA	0/167 (0%)		2/83 (2.4%)		0/15 (0%)
ENTEROCOLITIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
GASTRIC HAEMORRHAGE	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
GASTROINTESTINAL HAEMORRHAGE	2/167 (1.2%)		1/83 (1.2%)		0/15 (0%)
HAEMORRHOIDAL HAEMORRHAGE	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
HAEMORRHOIDS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
MELAENA	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
VOMITING	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
General disorders
ASTHENIA	2/167 (1.2%)		0/83 (0%)		0/15 (0%)
FATIGUE	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
GENERALISED OEDEMA	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
INFLUENZA LIKE ILLNESS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
MULTI-ORGAN FAILURE	2/167 (1.2%)		2/83 (2.4%)		0/15 (0%)
NON-CARDIAC CHEST PAIN	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
OEDEMA PERIPHERAL	4/167 (2.4%)		0/83 (0%)		0/15 (0%)
PYREXIA	5/167 (3%)		3/83 (3.6%)		0/15 (0%)
SUDDEN CARDIAC DEATH	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
SUDDEN DEATH	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
Hepatobiliary disorders
CHOLECYSTITIS	1/167 (0.6%)		1/83 (1.2%)		0/15 (0%)
Infections and infestations
ATYPICAL PNEUMONIA	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
BACTEROIDES BACTERAEMIA	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
BRONCHITIS	2/167 (1.2%)		0/83 (0%)		0/15 (0%)
BRONCHOPULMONARY ASPERGILLOSIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
CELLULITIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
DEVICE RELATED INFECTION	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
ENTEROCOLITIS INFECTIOUS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
ERYSIPELAS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
GASTROINTESTINAL CANDIDIASIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
INFECTIOUS PLEURAL EFFUSION	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
LUNG INFECTION	3/167 (1.8%)		1/83 (1.2%)		0/15 (0%)
LYMPHADENITIS BACTERIAL	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
NECROTISING FASCIITIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
PARONYCHIA	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
PNEUMOCYSTIS JIROVECI PNEUMONIA	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
PNEUMONIA	6/167 (3.6%)		4/83 (4.8%)		0/15 (0%)
SEPSIS	1/167 (0.6%)		1/83 (1.2%)		0/15 (0%)
SEPTIC SHOCK	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
SIALOADENITIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
STAPHYLOCOCCAL SEPSIS	2/167 (1.2%)		0/83 (0%)		0/15 (0%)
URINARY TRACT INFECTION	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
UROSEPSIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
WOUND INFECTION	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
Injury, poisoning and procedural complications
FALL	1/167 (0.6%)		1/83 (1.2%)		0/15 (0%)
INJURY	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
SUBDURAL HAEMATOMA	1/167 (0.6%)		1/83 (1.2%)		0/15 (0%)
SUBDURAL HAEMORRHAGE	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
TRANSFUSION-RELATED CIRCULATORY OVERLOAD	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
Investigations
ASPARTATE AMINOTRANSFERASE INCREASED	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
PLATELET COUNT INCREASED	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
WHITE BLOOD CELL COUNT INCREASED	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
Metabolism and nutrition disorders
DECREASED APPETITE	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
DEHYDRATION	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
DIABETIC KETOACIDOSIS	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
HYPOGLYCAEMIA	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
Musculoskeletal and connective tissue disorders
BACK PAIN	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
B-CELL LYMPHOMA	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
BASAL CELL CARCINOMA	1/167 (0.6%)		1/83 (1.2%)		0/15 (0%)
HISTIOCYTOSIS HAEMATOPHAGIC	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
MALIGNANT MELANOMA	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
MYELOFIBROSIS	9/167 (5.4%)		3/83 (3.6%)		0/15 (0%)
OESOPHAGEAL ADENOCARCINOMA	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
Nervous system disorders
CEREBROVASCULAR ACCIDENT	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
CONVULSION	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
LOSS OF CONSCIOUSNESS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
SYNCOPE	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
VIITH NERVE PARALYSIS	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
Psychiatric disorders
ABNORMAL BEHAVIOUR	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
Renal and urinary disorders
CALCULUS BLADDER	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
CALCULUS URETERIC	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
NEPHROLITHIASIS	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
NEPHROTIC SYNDROME	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
RENAL COLIC	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
RENAL FAILURE	3/167 (1.8%)		0/83 (0%)		0/15 (0%)
RENAL FAILURE ACUTE	1/167 (0.6%)		1/83 (1.2%)		0/15 (0%)
RENAL FAILURE CHRONIC	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
URINARY RETENTION	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
URINARY TRACT OBSTRUCTION	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
Respiratory, thoracic and mediastinal disorders
DYSPNOEA	1/167 (0.6%)		1/83 (1.2%)		0/15 (0%)
EPISTAXIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
HYPOXIA	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
INTERSTITIAL LUNG DISEASE	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
PNEUMONITIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
PULMONARY EMBOLISM	2/167 (1.2%)		0/83 (0%)		0/15 (0%)
PULMONARY HYPERTENSION	2/167 (1.2%)		0/83 (0%)		0/15 (0%)
RESPIRATORY FAILURE	2/167 (1.2%)		0/83 (0%)		0/15 (0%)
Skin and subcutaneous tissue disorders
ACUTE FEBRILE NEUTROPHILIC DERMATOSIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
DRUG ERUPTION	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
LEUKOCYTOCLASTIC VASCULITIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
PETECHIAE	0/167 (0%)		1/83 (1.2%)		0/15 (0%)
RASH MACULO-PAPULAR	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
SKIN ULCER	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
Vascular disorders
AXILLARY VEIN THROMBOSIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
DEEP VEIN THROMBOSIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
HYPOTENSION	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
INTERMITTENT CLAUDICATION	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
JUGULAR VEIN THROMBOSIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
PHLEBITIS	1/167 (0.6%)		0/83 (0%)		0/15 (0%)
Other (Not Including Serious) Adverse Events
	Global Study: Pomalidomide 0.5 mg		Global Study: Placebo		China Extension: Pomalidomide 0.5 mg
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	155/167 (92.8%)		76/83 (91.6%)		12/15 (80%)
Blood and lymphatic system disorders
ANAEMIA	12/167 (7.2%)		5/83 (6%)		2/15 (13.3%)
NEUTROPENIA	25/167 (15%)		4/83 (4.8%)		0/15 (0%)
THROMBOCYTOPENIA	21/167 (12.6%)		12/83 (14.5%)		1/15 (6.7%)
Cardiac disorders
ARRHYTHMIA	2/167 (1.2%)		1/83 (1.2%)		1/15 (6.7%)
Gastrointestinal disorders
ABDOMINAL DISTENSION	11/167 (6.6%)		1/83 (1.2%)		0/15 (0%)
ABDOMINAL PAIN	22/167 (13.2%)		11/83 (13.3%)		0/15 (0%)
ABDOMINAL PAIN UPPER	10/167 (6%)		3/83 (3.6%)		0/15 (0%)
CONSTIPATION	24/167 (14.4%)		9/83 (10.8%)		0/15 (0%)
DIARRHOEA	35/167 (21%)		17/83 (20.5%)		2/15 (13.3%)
NAUSEA	21/167 (12.6%)		16/83 (19.3%)		0/15 (0%)
VOMITING	12/167 (7.2%)		7/83 (8.4%)		0/15 (0%)
General disorders
ASTHENIA	23/167 (13.8%)		8/83 (9.6%)		0/15 (0%)
CHEST PAIN	2/167 (1.2%)		0/83 (0%)		1/15 (6.7%)
FATIGUE	35/167 (21%)		17/83 (20.5%)		3/15 (20%)
OEDEMA	1/167 (0.6%)		0/83 (0%)		1/15 (6.7%)
OEDEMA PERIPHERAL	53/167 (31.7%)		14/83 (16.9%)		1/15 (6.7%)
PYREXIA	30/167 (18%)		9/83 (10.8%)		2/15 (13.3%)
Hepatobiliary disorders
HEPATIC CYST	1/167 (0.6%)		0/83 (0%)		1/15 (6.7%)
HEPATIC FUNCTION ABNORMAL	1/167 (0.6%)		0/83 (0%)		1/15 (6.7%)
Infections and infestations
BRONCHITIS	9/167 (5.4%)		4/83 (4.8%)		2/15 (13.3%)
NASOPHARYNGITIS	13/167 (7.8%)		0/83 (0%)		0/15 (0%)
PNEUMONIA	10/167 (6%)		2/83 (2.4%)		1/15 (6.7%)
UPPER RESPIRATORY TRACT INFECTION	10/167 (6%)		5/83 (6%)		1/15 (6.7%)
URINARY TRACT INFECTION	12/167 (7.2%)		2/83 (2.4%)		0/15 (0%)
Injury, poisoning and procedural complications
FALL	7/167 (4.2%)		5/83 (6%)		0/15 (0%)
LIGAMENT SPRAIN	1/167 (0.6%)		0/83 (0%)		1/15 (6.7%)
Investigations
WEIGHT DECREASED	11/167 (6.6%)		3/83 (3.6%)		1/15 (6.7%)
WHITE BLOOD CELL COUNT DECREASED	2/167 (1.2%)		0/83 (0%)		1/15 (6.7%)
WHITE BLOOD CELL COUNT INCREASED	0/167 (0%)		1/83 (1.2%)		1/15 (6.7%)
Metabolism and nutrition disorders
DECREASED APPETITE	20/167 (12%)		7/83 (8.4%)		0/15 (0%)
HYPERGLYCAEMIA	3/167 (1.8%)		2/83 (2.4%)		1/15 (6.7%)
HYPERURICAEMIA	7/167 (4.2%)		5/83 (6%)		0/15 (0%)
Musculoskeletal and connective tissue disorders
ARTHRALGIA	6/167 (3.6%)		8/83 (9.6%)		0/15 (0%)
MUSCLE SPASMS	9/167 (5.4%)		5/83 (6%)		0/15 (0%)
PAIN IN EXTREMITY	11/167 (6.6%)		6/83 (7.2%)		0/15 (0%)
Nervous system disorders
DIZZINESS	17/167 (10.2%)		12/83 (14.5%)		0/15 (0%)
SOMNOLENCE	5/167 (3%)		5/83 (6%)		0/15 (0%)
Renal and urinary disorders
CALCULUS URETERIC	1/167 (0.6%)		0/83 (0%)		1/15 (6.7%)
NEPHROLITHIASIS	3/167 (1.8%)		0/83 (0%)		1/15 (6.7%)
Respiratory, thoracic and mediastinal disorders
COUGH	24/167 (14.4%)		9/83 (10.8%)		1/15 (6.7%)
DYSPNOEA	28/167 (16.8%)		8/83 (9.6%)		1/15 (6.7%)
DYSPNOEA EXERTIONAL	10/167 (6%)		3/83 (3.6%)		0/15 (0%)
EPISTAXIS	8/167 (4.8%)		5/83 (6%)		0/15 (0%)
PLEURAL EFFUSION	5/167 (3%)		2/83 (2.4%)		1/15 (6.7%)
Skin and subcutaneous tissue disorders
NIGHT SWEATS	10/167 (6%)		3/83 (3.6%)		0/15 (0%)
PRURITUS	12/167 (7.2%)		5/83 (6%)		0/15 (0%)
RASH	11/167 (6.6%)		2/83 (2.4%)		0/15 (0%)

Limitations/Caveats

[Not Specified]

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Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

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.