Pomalidomide (CC4047) plus low-dose dexamethasone as therapy for relapsed multiple myeloma.
OBJECTIVE
Thalidomide and lenalidomide are immunomodulatory drugs (IMiDs) that produce high remission rates in the treatment of multiple myeloma. Pomalidomide is a new IMiD with high in vitro potency. We report, to our knowledge, the first phase II trial of pomalidomide administered in combination with low-dose dexamethasone for the treatment of relapsed or refractory multiple myeloma.


METHODS
Pomalidomide was administered orally at a dose of 2 mg daily on days 1 through 28 of a 28-day cycle. Dexamethasone 40 mg daily was administered orally on days 1, 8, 15, and 22 of each cycle. Responses were recorded using the criteria of the International Myeloma Working Group.


RESULTS
Sixty patients were enrolled. Thirty-eight patients (63%) achieved confirmed response including complete response in three patients (5%), very good partial response in 17 patients (28%), and partial response in 18 patients (30%). Responses were seen in 40% of lenalidomide-refractory patients, 37% of thalidomide-refractory patients, and 60% of bortezomib-refractory patients. Responses were seen in 74% of patients with high-risk cytogenetic or molecular markers. Toxicity consisted primarily of myelosuppression. Grade 3 or 4 hematologic toxicity consisted of anemia (5%), thrombocytopenia (3%), and neutropenia (32%). One patient (1.6%) had a thromboembolic event. The median progression-free survival time was 11.6 months and was not significantly different in patients with high-risk disease compared with patients with standard-risk disease.


CONCLUSIONS
The combination of pomalidomide and low-dose dexamethasone is extremely active in the treatment of relapsed multiple myeloma, including high response rates in patients refractory to other novel agents.
Lacy
Martha Q
MQ
Division of Hematology, Mayo Clinic College of Medicine, Rochester, MN 55905, USA. lacy.martha@mayo.edu
Hayman
Suzanne R
SR
Gertz
Morie A
MA
Dispenzieri
Angela
A
Buadi
Francis
F
Kumar
Shaji
S
Greipp
Philip R
PR
Lust
John A
JA
Russell
Stephen J
SJ
Dingli
David
D
Kyle
Robert A
RA
Fonseca
Rafael
R
Bergsagel
P Leif
PL
Roy
Vivek
V
Mikhael
Joseph R
JR
Stewart
A Keith
AK
Laumann
Kristina
K
Allred
Jacob B
JB
Mandrekar
Sumithra J
SJ
Rajkumar
S Vincent
SV
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage*; D003907:Dexamethasone / Q000008:administration & dosage*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009101:Multiple Myeloma / Q000188:drug therapy*; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy*; D013792:Thalidomide / Q000008:administration & dosage / Q000031:analogs & derivatives*
D013792:Thalidomide; D003907:Dexamethasone; C467566:pomalidomide