STRATUS: Evaluation of Safety of Pomalidomide in Combination With Dexamethasone (Low Dose) in Patients With Refractory or Relapsed and Refractory Multiple Myeloma

• Sponsor: Celgene (Industry)
• Overall Status: Completed
• CT.gov ID: NCT01712789
• Collaborator: (none)
• Enrollment: 682
• Locations: 112
• Arm: 1
• Actual Duration (Months): 85.1
• Patients Per Site: 6.1
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

The primary purpose of the study is to evaluate the safety and efficacy and to generate PK and biomarker data for the combination of pomalidomide and low-dose dexamethasone in patients with refractory or relapsed and refractory multiple myeloma.

The study consists of a Screening phase within 28 days prior to cycle 1 day 1, a Treatment phase and a Follow-up phase which starts within 28 days of discontinuation from study treatment, every 3 months for up to 5 years.

In addition, the collection of steady-state PK data from a large population will enable robust population PK and assess Pomalidomide exposure response analyses.

The exploratory objectives of the study are to investigate potential markers predictive of POM response or resistance and pharmacodynamic markers.

Condition or Disease	Intervention/Treatment	Phase
Multiple Myeloma	Drug: Pomalidomide; Drug: Dexamethasone	Phase 3

Study Design

• Study Type: Interventional
• Actual Enrollment: 682 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Multicenter, Single-arm, Open-label Study With Pomalidomide in Combination With Low Dose Dexamethasone in Subjects With Refractory or Relapsed and Refractory Multiple Myeloma
• Actual Study Start Date: Nov 6, 2012
• Actual Primary Completion Date: Dec 11, 2019
• Actual Study Completion Date: Dec 11, 2019

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Pomalidomide plus Dexamethasone Pomalidomide 4mg by mouth (PO) daily days 1 through 21 of a 28 day cycle and dexamethasone 40mg/day PO for those ≤75 years of age or 20mg/day for those greater than 75 years of age on Days 1, 8, 15 and 22 of a 28 day cycle.	Drug: Pomalidomide; Oral Pomalidomide at the starting dose of 4 mg on Days 1-21 of a 28-day cycle; Drug: Dexamethasone; Oral Low dose Dexamethasone at the starting dose of 40mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle.

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Treatment Emergent Adverse Events (TEAE) [From the first dose of study treatment up to 28 days following the last dose of study treatment. The median duration of treatment with pomalidomide and LD-dex was 21.4 weeks.]

   An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen in a subject during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the subject's health, regardless of etiology. Any worsening (i.e., any significant adverse change in the frequency or intensity of a pre- existing condition) was considered an AE. The severity of AEs were graded based on the symptoms according to version 4.0 of the National Cancer Institute Common Terminology Criteria for Adverse Events. Second primary malignancies were monitored as events of interest and considered as part of the assessment of AEs. A SAE = AE occurring at any dose that: Results in death; Is life-threatening Requires inpatient hospitalization or prolongation of existing hospitalization Results in persistent or significant disability/incapacity Is a congenital anomaly/birth defect

Secondary Outcome Measures

1. Overall Response [Response was assessed at each treatment cycle and at treatment discontinuation; median duration of treatment with pomalidomide and LD-dex was 21.4 weeks]

   Overall response rate (ORR) was defined as the percentage of participants with a stringent complete response (sCR), complete response (CR), very good partial response (VGPR) or partial response (PR) according to the International Myeloma Working Group uniform response criteria (IMWG URC) assessed by the Investigator. Responses must have been confirmed at at least 2 consecutive assessments before the institution of any new therapy with no known evidence of progressive or new bone lesions

2. Time to Response [Response was assessed at each treatment cycle and at treatment discontinuation; median duration of treatment with pomalidomide and LD-dex was 21.4 weeks]

   Time to response was defined as the time from treatment enrollment to the first documentation of response (sCR, CR, VGPR or PR) based on IMWG criteria.

3. Kaplan Meier Estimate of Duration of Response [From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months]

   Duration of response, calculated for responders only, was defined as time from the initial documented response (SCR, CR, VGPR or PR) to the first confirmed disease progression, or death if no disease progression was recorded. Participants without a documented progression were censored at the time of their last tumor assessment.

4. Kaplan Meier Estimate of Progression Free Survival (PFS) According to the European Medicines Agency Guidelines [From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months]

   Progression free survival was calculated as the time from study enrollment, defined as the IVRS enrollment date, until either PD or death (any cause). Participants without an event (either a documented PD or death) at the time of study end were censored at the time of their last documented disease assessment or at the IVRS enrollment date if no disease assessment was conducted.

5. Kaplan Meier Estimate of Time to Progression [From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months]

   Time to progression was calculated as the time from study enrollment until first recorded disease progression as determined by the site investigator based on the IMWG criteria, or until death due to progression. Participants not experiencing a documented progression were censored at the time of their last tumor assessment (or at the time of trial enrollment if no assessment was conducted).

6. Kaplan Meier Estimate of Overall Survival (OS) [From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months]

   Overall survival was calculated as the time from study enrollment, defined as the IVRS enrollment date, until death due to any cause. Participants who did not have death data at the time of study end/analysis were censored at the time they were last known to be alive.

7. Pomalidomide Exposure - Apparent (Oral) Clearance (CL/F) [Cycles 1, 2, 3, 4, 5, 6]

   Pharmacokinetic (PK) parameters are derived from pomalidomide concentration versus time data.

8. Pomalidomide Exposure - Apparent Volume of Distribution (V/F) [Cycles 1, 2, 3, 4, 5, 6]

   Pharmacokinetic (PK) parameters are derived from Pomalidomide concentration versus time data.

9. Cytogenetic Analysis [Study entry]

   Cytogenetic analysis was to be performed using fluorescence in situ hybridization (FISH) methodology at a local laboratory, to evaluate the relationship between cytogenetic profiles and the combination of POM and LD-DEX in terms of response and outcome.

Eligibility Criteria

Criteria

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

Inclusion Criteria:

• Patients ≥18 years old, who must understand and voluntarily sign an Informed Consent.

• Patients must have documented diagnosis of Multiple Myeloma and have measurable disease.

• Patients must have undergone prior treatment with ≥ 2 treatments lines, of anti-myeloma therapy.

• Patients must have either refractory or relapsed and refractory disease.

• Patients must have received at least 2 consecutive cycles of prior treatment that include lenalidomide and bortezomib, either alone or in combination regimens.

• Patients must have received adequate alkylator therapy

Exclusion Criteria:

• Prior history of malignancies, other than Multiple Myeloma.

• Previous therapy with Pomalidomide, hypersensitivity to thalidomide and lenalidomide or dexamethasone.

• Patients who received an allogeneic bone marrow or allogeneic peripheral blood stem cell transplant.

• Patients who are planning for or who are eligible for stem cell transplant.

• Patients who received major surgery and any anti-myeloma drug therapy within the last 14 days of starting study treatment.

• Patients with a current disease that can interfere with protocol procedures or study treatment.

• Patients unable or unwilling to undergo antithrombotic prophylactic treatment.

• Pregnant or breastfeeding females.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Medical University of Graz	Graz		Austria	A-8036
2	Medizinische Universitat Innsbruck	Innsbruck		Austria	6020
3	Wilhelminenspital Vienna	Vienna		Austria	1160
4	Medical University of Vienna	Vienna		Austria	A-1090
5	AZ St-Jan Brugge Oostende AV	Brugge		Belgium	8000
6	Institut Jules Bordet	Brussels		Belgium	1000
7	VUB Vrije Universiteit Brussel	Brussel		Belgium	1090
8	UZ Gent	Gent		Belgium	9000
9	UZ Leuven	Leuven		Belgium	3000
10	Centre Hospitalier Universitaire de Liege	Liege		Belgium	4000
11	CHU Mont -Godinne	Yvoir		Belgium	5530
12	Aarhus University Hospital	Aarhus		Denmark	8000
13	Odense University Hospital	Odense		Denmark	DK-5000
14	Vejle Hospital	Vejle		Denmark	7100
15	Tartu University Hospital Clinic	Tartu		Estonia	51014
16	Helsingin yliopistollinen keskussairaala	Helsinki		Finland	FI-00290
17	Turku University Hospital	Turku		Finland	20521
18	Centre Hospitalier de la cote basque	Bayonne		France	64109
19	Hopital Henri Mondor	Créteil		France	94010
20	Hopital A. Michallon	La Tronche		France	38700
21	CHRU Claude Huriez	Lille		France	59037
22	Institut Paoli Calmette Hematologie	Marseille cedex		France	13273
23	CHU Hotel Dieu	Nantes		France	44093
24	Hopital Saint Antoine	Paris		France	75012
25	Service Hemato-Immunologie Hopital St Louis	Paris		France	75475
26	Centre Hospitalier Lyon Sud	Pierre Bénite		France	69495
27	CHU de Reims	Reims cedex		France	51092
28	Hematologie - CHU Purpan	Toulouse		France	31059
29	CHRU Hopital Bretonneau	Tours cedex		France	37044
30	CHU Nancy Hematology	Vandoeuvre les Nancy		France	54511
31	Charite, Campus Benjamin Franklin Universitatsmedizin Berlin	Berlin		Germany	12200
32	Klinikum Chemnitz	Chemnitz		Germany	D-09113
33	Universitatsklinikum Carl Gustav Carus	Dresden		Germany	01307
34	Universitatsklinkikum DusseldorfKlinik fur Hamatologie, Onkologie und klin. Immunoligie	Dusseldorf		Germany	D-40225
35	Universitatsklinikum Essen-	Essen		Germany	45122
36	Universitatsklinikum Freiburg Medizinische Klinik und Poliklinik	Freiburg		Germany	D-79106
37	Abt Haematologie - Onkologie / Allg. Krankenhaus Altona	Hamburg		Germany	D-22763
38	Universitatsklinikum Heidelberg	Heidelberg		Germany	69120
39	Universitatsklinikum Jena	Jena		Germany	O7740
40	University of Schleswig Holstein	Kiel		Germany	24105
41	Klinikum der Universitat zu Koln	Köln		Germany	50937
42	Universitatsklinikum Leipzig	Leipzig		Germany	04103
43	Universitatsklinik MuensterMedizinische Klinik A	Muenster		Germany	48129
44	TU München - Klinikum rechts der Isar	München		Germany	81675
45	UKT Universitaetsklinikum Tuebingen	Tuebingen		Germany	72076
46	University Hospital of Ulm	Ulm		Germany	89081
47	Universitatsklinikum Wurzburg	Würzburg		Germany	97080
48	University of Athens	Athens		Greece	11528
49	Cork University HospitalHaematology Consultant	Wilton	Cork	Ireland
50	Mater Misericordiae University Hospital	Dublin		Ireland	Dublin 7
51	University Hospital Galway	Galway		Ireland	ST46QG
52	Ospedali Riuniti di Ancona	Ancona		Italy	60126
53	A.O. Policlinico - Università di Bari	Bari		Italy	70124
54	University of Bologna	Bologna		Italy	40138
55	Ospedale Ferrarotto	Catania		Italy	95124
56	Fondazione IRCCS Istituto Nazionale dei Tumori	Milano		Italy	20133
57	ASST Grande Ospedale Metropolitano Niguarda, Milano	Milano		Italy	20162
58	Universita degli Studi di Padova	Padova		Italy	35128
59	Casa di Cura La Maddalena	Palermo		Italy	90146
60	Ospedale Civile di Piacenza	Piacenza		Italy	29100
61	Azienda Ospedaliero-Universitaria Pisana	Pisa		Italy	56126
62	Universita degli Studi di Roma La Sapienza - Azienda Policlinico Umberto I	Roma		Italy	00168
63	Ospedale Sant'Eugenio	Rome		Italy	00144
64	Azienda Ospedaliera San Giovanni Battista - Ospedale Molinette	Torino		Italy	10126
65	Azienda Ospedaliero-Universitaria Santa Maria della Misericordia die Udine	Udine		Italy	33100
66	Ospedale San Bortolo	Vicenza		Italy	36100
67	VU University Medical Center VU Medisch Centrum	Amsterdam		Netherlands	1081 HV
68	Haga Hospital	Den Haag		Netherlands	2545 CH
69	Universitair Medisch Centrum Groningen	Groningen		Netherlands	9713 GZ
70	Erasmus Medical Center	Rotterdam		Netherlands	3015 CE
71	University Medical Center Utrecht	Utrecht		Netherlands	3584 CX
72	Oslo University Hospital, Rikshospitalet HF	Oslo		Norway	0424
73	St. Olavs Hospital Trondheim	Trondheim		Norway	N-7006
74	Akademia Medyczna w Gdansku Katedra i Klinika Hematologii i Transplantologii	Gdansk		Poland	80-211
75	Szpitala Uniwersyteckiego w. Krakowie	Kraków		Poland	31 501
76	Instytut Hematologii i Transfuzjologii w Warszawie	Warszawa		Poland	02-776
77	Hospital Universitario de Coimbra- Hospitais de Universidade de Coimbra	Coimbra		Portugal	3000-075
78	Instituto Portugues de Oncologia de Lisboa	Lisboa		Portugal	1099-023
79	Hospital de Santa Maria	Lisboa		Portugal	1649-035
80	Hospital Geral de Santo António - Serviço de Hematologia Clínica	Porto		Portugal	4099-001
81	University Hospital Bratislava - Hospital Ss Cyril and Methodius	Bratislava		Slovakia	85107
82	Hospital Universitari Germans Trias i Pujol	Badalona (Barcelona)		Spain	8916
83	Hospital de la Santa Creu i Sant Pau	Barcelona		Spain	08025
84	Hospital Universitario de Canarias	La Laguna (Tenerife)		Spain	38320
85	Hospital de La Princesa	Madrid		Spain	28006
86	Hospital Ramon y Cajal	Madrid		Spain	28034
87	Hospital Clinico San Carlos	Madrid		Spain	28040
88	Hospital 12 de Octubre	Madrid		Spain	28041
89	Hospital Universitario Virgen De La Victoria	Malaga		Spain	29010
90	Hospital Morales Meseguer	Murcia		Spain	30008
91	Clinica Universidad de Navarra	Pamplona		Spain	31008
92	Hospital Universitario de Salamanca	Salamanca		Spain	37007
93	Hospital de Donosti	San Sebastián (Guipuzcoa)		Spain	20014
94	Hospital Clinico Universitario De Santiago De Compostela	Santiago De Compostela		Spain	15706
95	Hospital Virgen de la Salud	Toledo		Spain	45004
96	Hospital Universitari i Politecnic La Fe de Valencia	Valencia		Spain	46026
97	Universitetssjukhuset i Lund	Lund		Sweden	SE-22185
98	Karolinska University HospitalSolna	Stockholm		Sweden	SE 17176
99	Universitatsspital Bern	Bern		Switzerland	3010
100	Hopitaux Universitaires de Geneve-HUG	Genèva		Switzerland	1211
101	University Hospital Zurich	Zurich		Switzerland	8091
102	Ankara University Medical Faculty Cebeci Hospital	Ankara		Turkey	06590
103	Ege University Medical School	Izmir		Turkey	35100
104	Belfast City Hospital Haematology Department	Belfast Northern Ireland		United Kingdom	BT9 7AB
105	Kent and Canterbury Hospital	Canterbury/Kent		United Kingdom	CT1 3NG
106	Leeds Teaching Hospitals Trust	Leeds		United Kingdom	LS9 7TF
107	Royal Liverpool University Hospital	Liverpool		United Kingdom	L7 8XP
108	Christie Hospital NHS Foundation Trust	Manchester		United Kingdom	M20 4BX
109	Newcastle Hospital Foundation Trust	Newcastle upon Tyne		United Kingdom	NE7 7DN
110	Royal Marsden Hospital	Sutton (Surrey)		United Kingdom	SM2 5PT
111	Southmead Hospital	Westbury-on-Trym/ Bristol		United Kingdom	BS10 5NB
112	New Cross Hospital	Wolverhampton		United Kingdom	WV10 0QP

Sponsors and Collaborators

• Celgene

Investigators

• Study Director: Teresa Peluso, MBBS, DCPSA, Bristol-Myers Squibb

Study Documents (Full-Text)

• Study Protocol - May 30, 2013
• Statistical Analysis Plan - Mar 6, 2015

More Information

Publications

• Dimopoulos MA, Palumbo A, Corradini P, Cavo M, Delforge M, Di Raimondo F, Weisel KC, Oriol A, Hansson M, Vacca A, Blanchard MJ, Goldschmidt H, Doyen C, Kaiser M, Petrini M, Anttila P, Cafro AM, Raymakers R, San-Miguel J, de Arriba F, Knop S, Röllig C, Ocio EM, Morgan G, Miller N, Simcock M, Peluso T, Herring J, Sternas L, Zaki MH, Moreau P. Safety and efficacy of pomalidomide plus low-dose dexamethasone in STRATUS (MM-010): a phase 3b study in refractory multiple myeloma. Blood. 2016 Jul 28;128(4):497-503. doi: 10.1182/blood-2016-02-700872. Epub 2016 May 25.
• Moreau P, Dimopoulos MA, Richardson PG, Siegel DS, Cavo M, Corradini P, Weisel K, Delforge M, O'Gorman P, Song K, Chen C, Bahlis N, Oriol A, Hansson M, Kaiser M, Anttila P, Raymakers R, Joao C, Cook G, Sternas L, Biyukov T, Slaughter A, Hong K, Herring J, Yu X, Zaki M, San-Miguel J. Adverse event management in patients with relapsed and refractory multiple myeloma taking pomalidomide plus low-dose dexamethasone: A pooled analysis. Eur J Haematol. 2017 Sep;99(3):199-206. doi: 10.1111/ejh.12903. Epub 2017 Jun 14.
• Qian X, Dimopoulos MA, Amatangelo M, Bjorklund C, Towfic F, Flynt E, Weisel KC, Ocio EM, Yu X, Peluso T, Sternas L, Zaki M, Moreau P, Thakurta A. Cereblon gene expression and correlation with clinical outcomes in patients with relapsed/refractory multiple myeloma treated with pomalidomide: an analysis of STRATUS. Leuk Lymphoma. 2019 Feb;60(2):462-470. doi: 10.1080/10428194.2018.1485915. Epub 2018 Aug 2.
• Siegel DS, Weisel KC, Dimopoulos MA, Baz R, Richardson P, Delforge M, Song KW, San Miguel JF, Moreau P, Goldschmidt H, Cavo M, Jagannath S, Yu X, Hong K, Sternas L, Zaki M, Palumbo A. Pomalidomide plus low-dose dexamethasone in patients with relapsed/refractory multiple myeloma and moderate renal impairment: a pooled analysis of three clinical trials. Leuk Lymphoma. 2016 Dec;57(12):2833-2838. Epub 2016 Jun 7.

• Responsible Party: Celgene
• ClinicalTrials.gov Identifier: NCT01712789
• Other Study ID Numbers: CC-4047-MM-010; 2012-001888-78
• First Posted: Oct 24, 2012
• Last Update Posted: Jan 10, 2022
• Last Verified: Dec 1, 2021

• Keywords provided by Celgene: Multiple Myeloma; Relapsed Multiple Myeloma; Relapsed and refractory Multiple Myeloma; Pomalidomide; Dexamethasone
• Additional relevant MeSH terms: Multiple Myeloma; Neoplasms, Plasma Cell; Dexamethasone; Pomalidomide

Study Results

Participant Flow

Recruitment Details	The study was conducted at 112 sites: 4 in Austria, 7 in Belgium, 3 in Denmark, 1 in Estonia, 2 in Finland, 13 in France, 17 in Germany, 1 in Greece, 3 in Ireland, 15 in Italy, 5 in the Netherlands, 2 in Norway, 3 in Poland, 4 in Portugal, 1 in Slovakia, 15 in Spain, 2 in Sweden, 3 in Switzerland, 2 in Turkey, and 9 in the United Kingdom.
Pre-assignment Detail	Study participants had to have either refractory or relapsed and refractory disease, defined as documented disease progression during or within 60 days of completing their last myeloma therapy to be eligible to participate in the trial.

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Period Title: Overall Study
STARTED	682
Received Study Treatment	676
COMPLETED	0
NOT COMPLETED	682

Baseline Characteristics

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Overall Participants	682
Age (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	65.4 (9.10)
Sex: Female, Male (Count of Participants)
Female	301 44.1%
Male	381 55.9%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	52 7.6%
Not Hispanic or Latino	626 91.8%
Unknown or Not Reported	4 0.6%
Race/Ethnicity, Customized (Count of Participants)
Asian	3 0.4%
Black or African American	4 0.6%
White	669 98.1%
Other	2 0.3%
Missing	4 0.6%
Eastern Cooperative Oncology Group (ECOG) Performance Status (Count of Participants)
0 = Fully active, no restrictions	295 43.3%
1 = Restricted activity but ambulatory	319 46.8%
2 = Ambulatory and capable of all self-care	67 9.8%
3 = Limited self-care	1 0.1%
4 = Completely disabled	0 0%
Time since diagnosis (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	6.15 (3.649)
Beta 2 Microglobulin (mg/L) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [mg/L]	5.48 (4.713)
Serum Light Chain Type (Count of Participants)
Kappa	364 53.4%
Lambda	230 33.7%
No Serum Light chain Type Detected	16 2.3%
Test Not Performed	72 10.6%
Serum Heavy Chain Type (Count of Participants)
Immunoglobulin A (IgA)	145 21.3%
Immunoglobulin D (IgD)	5 0.7%
Immunoglobulin E (IgE)	0 0%
Immunoglobulin G (IgG)	388 56.9%
Immunoglobulin M (IgM)	4 0.6%
No serum heavy chain type detected	68 10%
Test not performed	72 10.6%
Renal Function (Cockcroft Gault Creatinine Clearance) (Count of Participants)
< 30 mL/min	12 1.8%
30 - < 45 mL/min	57 8.4%
45 - < 60 mL/min	168 24.6%
60 - < 80 mL/min	190 27.9%
≥ 80 mL/min	250 36.7%
Missing	5 0.7%
Corrected Serum Calcium (mmol/L) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [mmol/L]	2.43 (0.231)

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Treatment Emergent Adverse Events (TEAE)
Description	An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen in a subject during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the subject's health, regardless of etiology. Any worsening (i.e., any significant adverse change in the frequency or intensity of a pre- existing condition) was considered an AE. The severity of AEs were graded based on the symptoms according to version 4.0 of the National Cancer Institute Common Terminology Criteria for Adverse Events. Second primary malignancies were monitored as events of interest and considered as part of the assessment of AEs. A SAE = AE occurring at any dose that: Results in death; Is life-threatening Requires inpatient hospitalization or prolongation of existing hospitalization Results in persistent or significant disability/incapacity Is a congenital anomaly/birth defect
Time Frame	From the first dose of study treatment up to 28 days following the last dose of study treatment. The median duration of treatment with pomalidomide and LD-dex was 21.4 weeks.

Outcome Measure Data

Analysis Population Description

The safety population includes all enrolled participants who received at least one dose of the IP.

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Measure Participants	676
≥ TEAE	673 98.7%
≥ 1 TEAE Related to Pomalidomide (POM)	527 77.3%
≥ 1 TEAE Related to LD-Dex	448 65.7%
≥ 1 TEAE Related to Either POM or LD-Dex	575 84.3%
≥ 1 Grade (Gr) 3 or 4 TEAE	606 88.9%
≥ 1 Gr 3 or 4 TEAE Related to (R/T) POM	417 61.1%
≥ 1 Gr 3 or 4 TEAE R/T LD-Dex	226 33.1%
≥ 1 Gr 3 or 4 TEAE R/T Either POM or LD-Dex	448 65.7%
≥ 1 Grade 5 TEAE	127 18.6%
≥ 1 Grade 5 TEAE R/T POM	14 2.1%
≥ 1 Grade 5 TEAE R/T LD-Dex	16 2.3%
≥ 1 Grade 5 TEAE R/T either POM or LD-Dex	18 2.6%
≥ 1 Serious TEAE	448 65.7%
≥ 1 Serious TEAE R/T POM	187 27.4%
≥ 1 Serious TEAE R/T LD-Dex	146 21.4%
≥ 1 Serious TEAE R/T Either POM or LD-Dex	215 31.5%
≥ 1 Serious TEAE Leading to (L/T)Stopping of POM	36 5.3%
≥ 1 Serious TEAE L/T Stopping of LD-Dex	34 5%
≥1 Serious TEAE L/T Stopping either POM or LD-Dex	37 5.4%
≥ 1 TEAE L/T to Stopping of POM	54 7.9%
≥ 1 TEAE L/T to Stopping of LD-DEX	61 8.9%
≥ 1 TEAE L/T to Stopping of Either POM or LD-DEX	63 9.2%
≥1 Study Drug Related TEAE (L/T) Stopping POM	30 4.4%
≥1 Study Drug Related TEAE L/T Stopping LD-Dex	19 2.8%
≥1 Drug Related TEAE L/T Stopping LD-Dex or POM	38 5.6%
≥ 1 TEAE L/T to Reduction (R/D) of POM	164 24%
≥ 1 TEAE L/T to R/D of LD-DEX	150 22%
≥ 1 TEAE L/T to R/D of Either POM or LD-DEX	244 35.8%
≥ 1 Study Drug Related TEAE L/T to R/D of POM	142 20.8%
≥ 1 Study Drug Related TEAE L/T to R/D of LD-DEX	135 19.8%
≥1 StudyDrug Related TEAE L/T to R/D POM or LD-DEX	224 32.8%
≥ 1 TEAE L/T to Interruption (I/R) of POM	455 66.7%
≥ 1 TEAE L/T to I/R of LD-DEX	434 63.6%
≥ 1 TEAE L/T to I/R of either POM or LD-DEX	470 68.9%
≥ 1 Study Drug Related TEAE L/T to I/R of POM	294 43.1%
≥ 1 Study Drug Related TEAE L/T to I/R of LD-DEX	185 27.1%
≥1 StudyDrug Related TEAE L/T to I/R POM or LD-DEX	333 48.8%

2. Secondary Outcome

Title	Overall Response
Description	Overall response rate (ORR) was defined as the percentage of participants with a stringent complete response (sCR), complete response (CR), very good partial response (VGPR) or partial response (PR) according to the International Myeloma Working Group uniform response criteria (IMWG URC) assessed by the Investigator. Responses must have been confirmed at at least 2 consecutive assessments before the institution of any new therapy with no known evidence of progressive or new bone lesions
Time Frame	Response was assessed at each treatment cycle and at treatment discontinuation; median duration of treatment with pomalidomide and LD-dex was 21.4 weeks

Outcome Measure Data

Analysis Population Description

The intent to treat (ITT) population was defined as all enrolled participants (participants who received an interactive voice response system (IVRS) enrollment date) regardless of whether they received any investigational product (IP) or not.

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Measure Participants	682
Number (95% Confidence Interval) [Percentage of Participants]	33.4 4.9%

3. Secondary Outcome

Title	Time to Response
Description	Time to response was defined as the time from treatment enrollment to the first documentation of response (sCR, CR, VGPR or PR) based on IMWG criteria.
Time Frame	Response was assessed at each treatment cycle and at treatment discontinuation; median duration of treatment with pomalidomide and LD-dex was 21.4 weeks

Outcome Measure Data

Analysis Population Description

Includes participants with a response (sCR, VGPR or PR).

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Measure Participants	228
Median (Full Range) [Weeks]	8.1

4. Secondary Outcome

Title	Kaplan Meier Estimate of Duration of Response
Description	Duration of response, calculated for responders only, was defined as time from the initial documented response (SCR, CR, VGPR or PR) to the first confirmed disease progression, or death if no disease progression was recorded. Participants without a documented progression were censored at the time of their last tumor assessment.
Time Frame	From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months

Outcome Measure Data

Analysis Population Description

Includes participants with a response (sCR, VGPR or PR).

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Measure Participants	228
Median (95% Confidence Interval) [Months]	7.9

5. Secondary Outcome

Title	Kaplan Meier Estimate of Progression Free Survival (PFS) According to the European Medicines Agency Guidelines
Description	Progression free survival was calculated as the time from study enrollment, defined as the IVRS enrollment date, until either PD or death (any cause). Participants without an event (either a documented PD or death) at the time of study end were censored at the time of their last documented disease assessment or at the IVRS enrollment date if no disease assessment was conducted.
Time Frame	From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months

Outcome Measure Data

Analysis Population Description

The intent to treat (ITT) population was defined as all enrolled participants (participants who received an interactive voice response system (IVRS) enrollment date) regardless of whether they received any investigational product (IP) or not.

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Measure Participants	682
Median (95% Confidence Interval) [Months]	4.6

6. Secondary Outcome

Title	Kaplan Meier Estimate of Time to Progression
Description	Time to progression was calculated as the time from study enrollment until first recorded disease progression as determined by the site investigator based on the IMWG criteria, or until death due to progression. Participants not experiencing a documented progression were censored at the time of their last tumor assessment (or at the time of trial enrollment if no assessment was conducted).
Time Frame	From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months

Outcome Measure Data

Analysis Population Description

The intent to treat (ITT) population was defined as all enrolled participants (participants who received an interactive voice response system (IVRS) enrollment date) regardless of whether they received any investigational product (IP) or not.

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Measure Participants	682
Median (95% Confidence Interval) [Months]	4.8

7. Secondary Outcome

Title	Kaplan Meier Estimate of Overall Survival (OS)
Description	Overall survival was calculated as the time from study enrollment, defined as the IVRS enrollment date, until death due to any cause. Participants who did not have death data at the time of study end/analysis were censored at the time they were last known to be alive.
Time Frame	From enrollment to the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months

Outcome Measure Data

Analysis Population Description

The intent to treat (ITT) population was defined as all enrolled participants (participants who received an interactive voice response system (IVRS) enrollment date) regardless of whether they received any investigational product (IP) or not.

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Measure Participants	682
Median (95% Confidence Interval) [Months]	11.9

8. Secondary Outcome

Title	Pomalidomide Exposure - Apparent (Oral) Clearance (CL/F)
Description	Pharmacokinetic (PK) parameters are derived from pomalidomide concentration versus time data.
Time Frame	Cycles 1, 2, 3, 4, 5, 6

Outcome Measure Data

Analysis Population Description

Pharmacokinetic population Pharmacokinetic samples from 476 participants included in studies CC-4047-MM-005 and CC 4047 MM 010 were used for the population PK analysis of pomalidomide

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Measure Participants	476
Median (95% Confidence Interval) [Liters/hour]	6.02

9. Secondary Outcome

Title	Pomalidomide Exposure - Apparent Volume of Distribution (V/F)
Description	Pharmacokinetic (PK) parameters are derived from Pomalidomide concentration versus time data.
Time Frame	Cycles 1, 2, 3, 4, 5, 6

Outcome Measure Data

Analysis Population Description

Pharmacokinetic population Pharmacokinetic samples from 476 participants included in studies CC-4047-MM-005 and CC 4047 MM 010 were used for the population PK analysis of pomalidomide

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Measure Participants	476
Median (95% Confidence Interval) [Liters]	75.10

10. Secondary Outcome

Title	Cytogenetic Analysis
Description	Cytogenetic analysis was to be performed using fluorescence in situ hybridization (FISH) methodology at a local laboratory, to evaluate the relationship between cytogenetic profiles and the combination of POM and LD-DEX in terms of response and outcome.
Time Frame	Study entry

Outcome Measure Data

Analysis Population Description

Statistical analysis could not be performed due to high level of missing cytogenetic data as well as cytogenetic probe analysis being non- compliant with study requirements which lead to an inability to accurately identify specific cytogenetic abnormalities.

Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
Measure Participants	0

Adverse Events

Time Frame	All-cause mortality is reported from enrollment until the end of follow-up; median time on follow-up was 10.9 (range 0 - 81) months. Adverse events are reported from first dose of study drug up to 28 days after last dose; median duration of treatment was 21.4 weeks.
Adverse Event Reporting Description	Second primary malignancies were monitored as events of interest and considered as part of the assessment of AEs.
Arm/Group Title	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
Arm/Group Description	Participants received 4 mg pomalidomide (POM) by mouth (PO) on Days 1 to 21 of each 28-day treatment cycle and low dose dexamethasone (LD-Dex) PO at the starting dose of 40 mg/day (≤ 75 years old) or 20 mg/day (> 75 years old) on Days 1, 8, 15 and 22 of a 28-day cycle until the documentation of confirmed progressive disease (PD), intolerable toxicity, death, withdrawal of participation in the study/consent, lost to follow-up, or as long as they benefited from therapy according to the opinion of the responsible study investigator.
All Cause Mortality
	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
	Affected / at Risk (%)	# Events
Total	598/682 (87.7%)
Serious Adverse Events
	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
	Affected / at Risk (%)	# Events
Total	448/676 (66.3%)
Blood and lymphatic system disorders
Anaemia	15/676 (2.2%)
Bone marrow failure	1/676 (0.1%)
Disseminated intravascular coagulation	1/676 (0.1%)
Febrile bone marrow aplasia	1/676 (0.1%)
Febrile neutropenia	34/676 (5%)
Heparin-induced thrombocytopenia	1/676 (0.1%)
Hyperviscosity syndrome	3/676 (0.4%)
Leukopenia	1/676 (0.1%)
Neutropenia	20/676 (3%)
Pancytopenia	3/676 (0.4%)
Thrombocytopenia	13/676 (1.9%)
Cardiac disorders
Acute coronary syndrome	1/676 (0.1%)
Acute myocardial infarction	1/676 (0.1%)
Angina pectoris	1/676 (0.1%)
Atrial fibrillation	17/676 (2.5%)
Atrial flutter	3/676 (0.4%)
Atrioventricular block	1/676 (0.1%)
Cardiac amyloidosis	2/676 (0.3%)
Cardiac arrest	2/676 (0.3%)
Cardiac disorder	1/676 (0.1%)
Cardiac failure	12/676 (1.8%)
Cardiac failure congestive	2/676 (0.3%)
Cardio-respiratory arrest	2/676 (0.3%)
Coronary artery stenosis	1/676 (0.1%)
Myocardial infarction	4/676 (0.6%)
Nodal arrhythmia	1/676 (0.1%)
Palpitations	1/676 (0.1%)
Sinus bradycardia	1/676 (0.1%)
Eye disorders
Conjunctival haemorrhage	1/676 (0.1%)
Exophthalmos	1/676 (0.1%)
Gastrointestinal disorders
Abdominal pain	3/676 (0.4%)
Abdominal pain upper	2/676 (0.3%)
Anal fistula	1/676 (0.1%)
Constipation	1/676 (0.1%)
Diarrhoea	6/676 (0.9%)
Diverticulum intestinal	1/676 (0.1%)
Gastritis	2/676 (0.3%)
Gastrointestinal amyloidosis	1/676 (0.1%)
Gastrointestinal haemorrhage	1/676 (0.1%)
Haemorrhoidal haemorrhage	1/676 (0.1%)
Haemorrhoids	1/676 (0.1%)
Ileus	1/676 (0.1%)
Intestinal infarction	1/676 (0.1%)
Large intestinal haemorrhage	1/676 (0.1%)
Large intestine perforation	1/676 (0.1%)
Nausea	3/676 (0.4%)
Pancreatitis	1/676 (0.1%)
Pancreatitis acute	1/676 (0.1%)
Rectal haemorrhage	1/676 (0.1%)
Rectal ulcer	1/676 (0.1%)
Upper gastrointestinal haemorrhage	1/676 (0.1%)
Volvulus	1/676 (0.1%)
Vomiting	4/676 (0.6%)
General disorders
Asthenia	5/676 (0.7%)
Death	4/676 (0.6%)
Disease progression	1/676 (0.1%)
Euthanasia	1/676 (0.1%)
Fatigue	5/676 (0.7%)
General physical health deterioration	45/676 (6.7%)
Generalised oedema	1/676 (0.1%)
Hyperpyrexia	2/676 (0.3%)
Hyperthermia	1/676 (0.1%)
Malaise	2/676 (0.3%)
Mucosal inflammation	1/676 (0.1%)
Multiple organ dysfunction syndrome	1/676 (0.1%)
Non-cardiac chest pain	1/676 (0.1%)
Oedema	1/676 (0.1%)
Oedema peripheral	2/676 (0.3%)
Pain	2/676 (0.3%)
Pyrexia	41/676 (6.1%)
Hepatobiliary disorders
Cholecystitis	1/676 (0.1%)
Cholecystitis acute	1/676 (0.1%)
Cholelithiasis	1/676 (0.1%)
Gallbladder obstruction	1/676 (0.1%)
Hepatic failure	1/676 (0.1%)
Hyperbilirubinaemia	1/676 (0.1%)
Liver disorder	1/676 (0.1%)
Immune system disorders
Drug hypersensitivity	1/676 (0.1%)
Infections and infestations
Abdominal infection	1/676 (0.1%)
Abscess limb	1/676 (0.1%)
Anal abscess	1/676 (0.1%)
Appendicitis	1/676 (0.1%)
Arthritis bacterial	1/676 (0.1%)
Atypical pneumonia	4/676 (0.6%)
Bacteraemia	2/676 (0.3%)
Bacterial diarrhoea	1/676 (0.1%)
Biliary sepsis	1/676 (0.1%)
Bronchitis	9/676 (1.3%)
Bronchopulmonary aspergillosis	4/676 (0.6%)
Bursitis infective	1/676 (0.1%)
Campylobacter gastroenteritis	1/676 (0.1%)
Catheter site infection	1/676 (0.1%)
Cellulitis	2/676 (0.3%)
Cerebral aspergillosis	1/676 (0.1%)
Citrobacter infection	1/676 (0.1%)
Clostridial sepsis	1/676 (0.1%)
Clostridium difficile colitis	2/676 (0.3%)
Cystitis	1/676 (0.1%)
Device related infection	2/676 (0.3%)
Diverticulitis	2/676 (0.3%)
Ear infection	1/676 (0.1%)
Erysipelas	1/676 (0.1%)
Escherichia infection	1/676 (0.1%)
Escherichia sepsis	4/676 (0.6%)
Escherichia urinary tract infection	1/676 (0.1%)
Folliculitis	1/676 (0.1%)
Gastroenteritis	5/676 (0.7%)
Gastroenteritis norovirus	1/676 (0.1%)
Herpes zoster infection neurological	1/676 (0.1%)
Infection	14/676 (2.1%)
Infective exacerbation of chronic obstructive airways disease	1/676 (0.1%)
Influenza	5/676 (0.7%)
Leishmaniasis	1/676 (0.1%)
Listeria sepsis	1/676 (0.1%)
Listeriosis	1/676 (0.1%)
Localised infection	1/676 (0.1%)
Lower respiratory tract infection	22/676 (3.3%)
Lung infection	16/676 (2.4%)
Meningitis pneumococcal	1/676 (0.1%)
Meningococcal sepsis	1/676 (0.1%)
Neutropenic sepsis	5/676 (0.7%)
Oral fungal infection	1/676 (0.1%)
Oral infection	1/676 (0.1%)
Osteomyelitis bacterial	1/676 (0.1%)
Otitis externa	1/676 (0.1%)
Parainfluenzae virus infection	1/676 (0.1%)
Pharyngitis	1/676 (0.1%)
Pneumococcal sepsis	1/676 (0.1%)
Pneumocystis jirovecii pneumonia	3/676 (0.4%)
Pneumonia	117/676 (17.3%)
Pneumonia influenzal	3/676 (0.4%)
Pneumonia pneumococcal	5/676 (0.7%)
Pneumonia respiratory syncytial viral	2/676 (0.3%)
Pneumonia staphylococcal	2/676 (0.3%)
Pneumonia streptococcal	2/676 (0.3%)
Pneumonia viral	1/676 (0.1%)
Pseudomonal sepsis	3/676 (0.4%)
Pulmonary sepsis	3/676 (0.4%)
Respiratory moniliasis	1/676 (0.1%)
Respiratory tract infection	11/676 (1.6%)
Rhinovirus infection	1/676 (0.1%)
Sepsis	14/676 (2.1%)
Septic shock	15/676 (2.2%)
Sinusitis bacterial	1/676 (0.1%)
Soft tissue infection	1/676 (0.1%)
Staphylococcal infection	1/676 (0.1%)
Staphylococcal sepsis	3/676 (0.4%)
Staphylococcal skin infection	1/676 (0.1%)
Streptococcal bacteraemia	1/676 (0.1%)
Streptococcal infection	2/676 (0.3%)
Systemic candida	1/676 (0.1%)
Systemic infection	1/676 (0.1%)
Tracheobronchitis	2/676 (0.3%)
Upper respiratory tract infection	8/676 (1.2%)
Urinary tract infection	11/676 (1.6%)
Urinary tract infection bacterial	1/676 (0.1%)
Urosepsis	5/676 (0.7%)
Vascular device infection	1/676 (0.1%)
Injury, poisoning and procedural complications
Ankle fracture	1/676 (0.1%)
Clavicle fracture	1/676 (0.1%)
Contusion	1/676 (0.1%)
Fall	3/676 (0.4%)
Febrile nonhaemolytic transfusion reaction	1/676 (0.1%)
Femoral neck fracture	2/676 (0.3%)
Femur fracture	4/676 (0.6%)
Head injury	1/676 (0.1%)
Humerus fracture	4/676 (0.6%)
Ilium fracture	2/676 (0.3%)
Joint dislocation	1/676 (0.1%)
Perineal injury	1/676 (0.1%)
Post-traumatic pain	1/676 (0.1%)
Rib fracture	1/676 (0.1%)
Spinal compression fracture	1/676 (0.1%)
Subdural haematoma	1/676 (0.1%)
Tibia fracture	1/676 (0.1%)
Traumatic fracture	1/676 (0.1%)
Traumatic intracranial haemorrhage	1/676 (0.1%)
Investigations
Blood creatinine increased	6/676 (0.9%)
Blood immunoglobulin A increased	1/676 (0.1%)
C-reactive protein increased	2/676 (0.3%)
International normalised ratio increased	1/676 (0.1%)
Monoclonal immunoglobulin present	3/676 (0.4%)
Neutrophil count decreased	2/676 (0.3%)
Platelet count decreased	1/676 (0.1%)
Protein urine present	1/676 (0.1%)
White blood cell count decreased	2/676 (0.3%)
Metabolism and nutrition disorders
Decreased appetite	2/676 (0.3%)
Dehydration	5/676 (0.7%)
Diabetes mellitus inadequate control	1/676 (0.1%)
Hyperamylasaemia	1/676 (0.1%)
Hypercalcaemia	30/676 (4.4%)
Hyperglycaemia	3/676 (0.4%)
Hyperkalaemia	2/676 (0.3%)
Hypocalcaemia	1/676 (0.1%)
Hypoglycaemia	1/676 (0.1%)
Hypokalaemia	1/676 (0.1%)
Hyponatraemia	2/676 (0.3%)
Metabolic acidosis	1/676 (0.1%)
Tumour lysis syndrome	3/676 (0.4%)
Musculoskeletal and connective tissue disorders
Arthralgia	2/676 (0.3%)
Arthritis	1/676 (0.1%)
Back pain	7/676 (1%)
Bone lesion	1/676 (0.1%)
Bone pain	7/676 (1%)
Gouty arthritis	1/676 (0.1%)
Musculoskeletal chest pain	1/676 (0.1%)
Musculoskeletal pain	2/676 (0.3%)
Neck pain	2/676 (0.3%)
Osteolysis	2/676 (0.3%)
Pain in extremity	3/676 (0.4%)
Pathological fracture	7/676 (1%)
Spinal pain	4/676 (0.6%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal cell carcinoma	6/676 (0.9%)
Bowen's disease	2/676 (0.3%)
Gastrointestinal stromal tumour	1/676 (0.1%)
Invasive breast carcinoma	1/676 (0.1%)
Metastases to liver	1/676 (0.1%)
Metastases to meninges	1/676 (0.1%)
Monoclonal gammopathy	1/676 (0.1%)
Plasma cell leukaemia	1/676 (0.1%)
Plasma cell myeloma	4/676 (0.6%)
Plasmacytoma	10/676 (1.5%)
Squamous cell carcinoma	1/676 (0.1%)
Squamous cell carcinoma of skin	7/676 (1%)
Squamous cell carcinoma of the tongue	2/676 (0.3%)
Nervous system disorders
Cauda equina syndrome	1/676 (0.1%)
Cerebral haematoma	1/676 (0.1%)
Cognitive disorder	1/676 (0.1%)
Coma	1/676 (0.1%)
Depressed level of consciousness	2/676 (0.3%)
Disturbance in attention	1/676 (0.1%)
Encephalopathy	1/676 (0.1%)
Facial nerve disorder	1/676 (0.1%)
Generalised tonic-clonic seizure	1/676 (0.1%)
Haemorrhage intracranial	2/676 (0.3%)
Headache	1/676 (0.1%)
IIIrd nerve paresis	1/676 (0.1%)
Intracranial aneurysm	1/676 (0.1%)
Lateral medullary syndrome	1/676 (0.1%)
Motor dysfunction	1/676 (0.1%)
Paraesthesia	1/676 (0.1%)
Paraplegia	1/676 (0.1%)
Parkinsonism	1/676 (0.1%)
Peripheral motor neuropathy	1/676 (0.1%)
Peripheral sensorimotor neuropathy	1/676 (0.1%)
Presyncope	1/676 (0.1%)
Ruptured cerebral aneurysm	1/676 (0.1%)
Seizure	2/676 (0.3%)
Somnolence	1/676 (0.1%)
Spinal cord compression	3/676 (0.4%)
Subarachnoid haemorrhage	1/676 (0.1%)
Syncope	2/676 (0.3%)
Psychiatric disorders
Confusional state	4/676 (0.6%)
Depression	1/676 (0.1%)
Disorientation	1/676 (0.1%)
Renal and urinary disorders
Acute kidney injury	26/676 (3.8%)
Crush syndrome	1/676 (0.1%)
Haematuria	1/676 (0.1%)
Nephrolithiasis	1/676 (0.1%)
Oliguria	2/676 (0.3%)
Renal failure	23/676 (3.4%)
Renal impairment	1/676 (0.1%)
Renal vascular thrombosis	1/676 (0.1%)
Urinary retention	1/676 (0.1%)
Reproductive system and breast disorders
Scrotal cyst	1/676 (0.1%)
Respiratory, thoracic and mediastinal disorders
Acute pulmonary oedema	1/676 (0.1%)
Acute respiratory failure	1/676 (0.1%)
Bronchospasm	2/676 (0.3%)
Chronic obstructive pulmonary disease	1/676 (0.1%)
Cough	1/676 (0.1%)
Dyspnoea	15/676 (2.2%)
Dyspnoea exertional	1/676 (0.1%)
Epistaxis	5/676 (0.7%)
Haemoptysis	1/676 (0.1%)
Hypoxia	1/676 (0.1%)
Interstitial lung disease	1/676 (0.1%)
Orthopnoea	1/676 (0.1%)
Pleural effusion	6/676 (0.9%)
Pleurisy	2/676 (0.3%)
Pneumonitis	1/676 (0.1%)
Pneumothorax	1/676 (0.1%)
Productive cough	1/676 (0.1%)
Pulmonary congestion	1/676 (0.1%)
Pulmonary embolism	5/676 (0.7%)
Pulmonary haemorrhage	1/676 (0.1%)
Pulmonary oedema	5/676 (0.7%)
Respiratory acidosis	1/676 (0.1%)
Respiratory distress	1/676 (0.1%)
Respiratory failure	14/676 (2.1%)
Skin and subcutaneous tissue disorders
Rash	1/676 (0.1%)
Skin necrosis	1/676 (0.1%)
Social circumstances
Social stay hospitalisation	1/676 (0.1%)
Vascular disorders
Aortic stenosis	1/676 (0.1%)
Deep vein thrombosis	6/676 (0.9%)
Haematoma	1/676 (0.1%)
Hypertension	1/676 (0.1%)
Hypotension	4/676 (0.6%)
Shock haemorrhagic	1/676 (0.1%)
Other (Not Including Serious) Adverse Events
	Pomalidomide Plus Low Dose Dexamethasone (LD-Dex)
	Affected / at Risk (%)	# Events
Total	646/676 (95.6%)
Blood and lymphatic system disorders
Anaemia	327/676 (48.4%)
Leukopenia	92/676 (13.6%)
Neutropenia	382/676 (56.5%)
Thrombocytopenia	236/676 (34.9%)
Gastrointestinal disorders
Abdominal pain	34/676 (5%)
Constipation	163/676 (24.1%)
Diarrhoea	118/676 (17.5%)
Nausea	96/676 (14.2%)
Vomiting	49/676 (7.2%)
General disorders
Asthenia	155/676 (22.9%)
Fatigue	197/676 (29.1%)
Oedema peripheral	108/676 (16%)
Pyrexia	178/676 (26.3%)
Infections and infestations
Bronchitis	70/676 (10.4%)
Nasopharyngitis	57/676 (8.4%)
Pneumonia	38/676 (5.6%)
Respiratory tract infection	58/676 (8.6%)
Upper respiratory tract infection	50/676 (7.4%)
Urinary tract infection	54/676 (8%)
Investigations
Blood creatinine increased	49/676 (7.2%)
C-reactive protein increased	34/676 (5%)
Neutrophil count decreased	47/676 (7%)
Weight decreased	47/676 (7%)
Metabolism and nutrition disorders
Decreased appetite	69/676 (10.2%)
Hypercalcaemia	40/676 (5.9%)
Hyperglycaemia	40/676 (5.9%)
Hypokalaemia	52/676 (7.7%)
Musculoskeletal and connective tissue disorders
Arthralgia	42/676 (6.2%)
Back pain	105/676 (15.5%)
Bone pain	65/676 (9.6%)
Muscle spasms	96/676 (14.2%)
Muscular weakness	35/676 (5.2%)
Musculoskeletal chest pain	54/676 (8%)
Musculoskeletal pain	37/676 (5.5%)
Pain in extremity	54/676 (8%)
Nervous system disorders
Dizziness	55/676 (8.1%)
Headache	49/676 (7.2%)
Peripheral sensory neuropathy	79/676 (11.7%)
Tremor	45/676 (6.7%)
Psychiatric disorders
Confusional state	37/676 (5.5%)
Insomnia	76/676 (11.2%)
Respiratory, thoracic and mediastinal disorders
Cough	141/676 (20.9%)
Dyspnoea	117/676 (17.3%)
Epistaxis	47/676 (7%)
Skin and subcutaneous tissue disorders
Hyperhidrosis	37/676 (5.5%)
Pruritus	36/676 (5.3%)
Rash	54/676 (8%)

Limitations/Caveats

[Not Specified]

More Information

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 > 1 year since study completion. Then, Investigator can publish if manuscript is submitted to Celgene 60 days prior to submission. If Celgene decides publication would hinder drug development, Investigator must delay submission for up to 90 additional days. Investigator must delete confidential information before submission and defer publication to permit patent applications.