title: Pomalidomide alone or in combination with low-dose dexamethasone in relapsed and refractory multiple myeloma: a randomized phase 2 study.
abstract: This multicenter, open-label, randomized phase 2 study assessed the efficacy and safety of pomalidomide (POM) with/without low-dose dexamethasone (LoDEX) in patients with relapsed/refractory multiple myeloma (RRMM). Patients who had received ≥2 prior therapies (including lenalidomide [LEN] and bortezomib [BORT]) and had progressed within 60 days of their last therapy were randomized to POM (4 mg/day on days 1-21 of each 28-day cycle) with/without LoDEX (40 mg/week). The primary end point was progression-free survival (PFS). In total, 221 patients (median 5 prior therapies, range 1-13) received POM+LoDEX (n = 113) or POM (n = 108). With a median follow-up of 14.2 months, median PFS was 4.2 and 2.7 months (hazard ratio = 0.68, P = .003), overall response rates (ORRs) were 33% and 18% (P = .013), median response duration was 8.3 and 10.7 months, and median overall survival (OS) was 16.5 and 13.6 months, respectively. Refractoriness to LEN, or resistance to both LEN and BORT, did not affect outcomes with POM+LoDEX (median PFS 3.8 months for both; ORRs 30% and 31%; and median OS 16 and 13.4 months). Grade 3-4 neutropenia occurred in 41% (POM+LoDEX) and 48% (POM); no grade 3-4 peripheral neuropathy was reported. POM+LoDEX was effective and generally well tolerated and provides an important new treatment option for RRMM patients who have received multiple prior therapies. This trial was registered at www.clinicaltrials.gov as #NCT00833833.
authors.affiliation: Dana-Farber Cancer Institute, Boston, MA;
authors.forename: Paul G
authors.initials: PG
authors.lastname: Richardson
authors.forename: David S
authors.initials: DS
authors.lastname: Siegel
authors.forename: Ravi
authors.initials: R
authors.lastname: Vij
authors.forename: Craig C
authors.initials: CC
authors.lastname: Hofmeister
authors.forename: Rachid
authors.initials: R
authors.lastname: Baz
authors.forename: Sundar
authors.initials: S
authors.lastname: Jagannath
authors.forename: Christine
authors.initials: C
authors.lastname: Chen
authors.forename: Sagar
authors.initials: S
authors.lastname: Lonial
authors.forename: Andrzej
authors.initials: A
authors.lastname: Jakubowiak
authors.forename: Nizar
authors.initials: N
authors.lastname: Bahlis
authors.forename: Kevin
authors.initials: K
authors.lastname: Song
authors.forename: Andrew
authors.initials: A
authors.lastname: Belch
authors.forename: Noopur
authors.initials: N
authors.lastname: Raje
authors.forename: Chaim
authors.initials: C
authors.lastname: Shustik
authors.forename: Suzanne
authors.initials: S
authors.lastname: Lentzsch
authors.forename: Martha
authors.initials: M
authors.lastname: Lacy
authors.forename: Joseph
authors.initials: J
authors.lastname: Mikhael
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Matous
authors.forename: David
authors.initials: D
authors.lastname: Vesole
authors.forename: Min
authors.initials: M
authors.lastname: Chen
authors.forename: Mohamed H
authors.initials: MH
authors.lastname: Zaki
authors.forename: Christian
authors.initials: C
authors.lastname: Jacques
authors.forename: Zhinuan
authors.initials: Z
authors.lastname: Yu
authors.forename: Kenneth C
authors.initials: KC
authors.lastname: Anderson
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000971:Antineoplastic Combined Chemotherapy Protocols; D001897:Boronic Acids / Q000008:administration & dosage; D000069286:Bortezomib; D003907:Dexamethasone / Q000008:administration & dosage*; D018572:Disease-Free Survival; D019008:Drug Resistance, Neoplasm; D005260:Female; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage; D000077269:Lenalidomide; D008297:Male; D008875:Middle Aged; D009101:Multiple Myeloma / Q000188:drug therapy*; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy; D009503:Neutropenia / Q000139:chemically induced; D011719:Pyrazines / Q000008:administration & dosage; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives*; D016896:Treatment Outcome
chemical_list: D001897:Boronic Acids; D007155:Immunologic Factors; D011719:Pyrazines; D013792:Thalidomide; D000069286:Bortezomib; D003907:Dexamethasone; C467566:pomalidomide; D000077269:Lenalidomide