title: Pomalidomide (CC4047) plus low-dose dexamethasone as therapy for relapsed multiple myeloma.
abstract: 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.
authors.affiliation: Division of Hematology, Mayo Clinic College of Medicine, Rochester, MN 55905, USA. lacy.martha@mayo.edu
authors.forename: Martha Q
authors.initials: MQ
authors.lastname: Lacy
authors.forename: Suzanne R
authors.initials: SR
authors.lastname: Hayman
authors.forename: Morie A
authors.initials: MA
authors.lastname: Gertz
authors.forename: Angela
authors.initials: A
authors.lastname: Dispenzieri
authors.forename: Francis
authors.initials: F
authors.lastname: Buadi
authors.forename: Shaji
authors.initials: S
authors.lastname: Kumar
authors.forename: Philip R
authors.initials: PR
authors.lastname: Greipp
authors.forename: John A
authors.initials: JA
authors.lastname: Lust
authors.forename: Stephen J
authors.initials: SJ
authors.lastname: Russell
authors.forename: David
authors.initials: D
authors.lastname: Dingli
authors.forename: Robert A
authors.initials: RA
authors.lastname: Kyle
authors.forename: Rafael
authors.initials: R
authors.lastname: Fonseca
authors.forename: P Leif
authors.initials: PL
authors.lastname: Bergsagel
authors.forename: Vivek
authors.initials: V
authors.lastname: Roy
authors.forename: Joseph R
authors.initials: JR
authors.lastname: Mikhael
authors.forename: A Keith
authors.initials: AK
authors.lastname: Stewart
authors.forename: Kristina
authors.initials: K
authors.lastname: Laumann
authors.forename: Jacob B
authors.initials: JB
authors.lastname: Allred
authors.forename: Sumithra J
authors.initials: SJ
authors.lastname: Mandrekar
authors.forename: S Vincent
authors.initials: SV
authors.lastname: Rajkumar
mesh_terms: 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*
chemical_list: D013792:Thalidomide; D003907:Dexamethasone; C467566:pomalidomide