title: Activity of pomalidomide in patients with immunoglobulin light-chain amyloidosis.
abstract: Immunoglobulin light-chain (AL) amyloidosis is a rare, incurable plasma cell disorder. Its therapy has benefited immensely from the expanding drug armamentarium available for multiple myeloma. Pomalidomide in combination with weekly dexamethasone (Pom/dex) is active among patients with relapsed myeloma. In the present study, we explored the Pom/dex combination in patients with previously treated AL. Patients were eligible for this prospective phase 2 trial if they had had at least one prior regimen and if they had reasonably preserved organ function. Patients were treated with oral Pom/dex. Thirty-three patients were enrolled. The median age was 66 years. Median time from diagnosis to on-study was 37 months. Eighty-two percent had cardiac involvement. The confirmed hematologic response rate was 48%, with a median time to response of 1.9 months. Organ improvement was documented in 5 patients. The median overall and progression-free survival rates were 28 and 14 months, respectively; the 1-year overall and progression-free survival rates were 76% and 59%, respectively. There was a discordance between the hematologic response and the N-terminal pro-brain natriuretic peptide response. The most common grade 3-5 adverse events, regardless of attribution, were neutropenia and fatigue. We conclude that pomalidomide appears to be a valuable drug covering an unmet clinical need in patients with previously treated AL. The trial is registered at www.clinicaltrials.gov as NCT00558896.
authors.affiliation: Division of Hematology, Department of Medicine, Mayo Clinic Rochester, Rochester, NY, USA. dispenzieri.angela@mayo.edu
authors.forename: Angela
authors.initials: A
authors.lastname: Dispenzieri
authors.forename: Francis
authors.initials: F
authors.lastname: Buadi
authors.forename: Kristina
authors.initials: K
authors.lastname: Laumann
authors.forename: Betsy
authors.initials: B
authors.lastname: LaPlant
authors.forename: Suzanne R
authors.initials: SR
authors.lastname: Hayman
authors.forename: Shaji K
authors.initials: SK
authors.lastname: Kumar
authors.forename: David
authors.initials: D
authors.lastname: Dingli
authors.forename: Steven R
authors.initials: SR
authors.lastname: Zeldenrust
authors.forename: Joseph R
authors.initials: JR
authors.lastname: Mikhael
authors.forename: Robert
authors.initials: R
authors.lastname: Hall
authors.forename: S Vincent
authors.initials: SV
authors.lastname: Rajkumar
authors.forename: Craig
authors.initials: C
authors.lastname: Reeder
authors.forename: Rafael
authors.initials: R
authors.lastname: Fonseca
authors.forename: P Lief
authors.initials: PL
authors.lastname: Bergsagel
authors.forename: A Keith
authors.initials: AK
authors.lastname: Stewart
authors.forename: Vivek
authors.initials: V
authors.lastname: Roy
authors.forename: Thomas E
authors.initials: TE
authors.lastname: Witzig
authors.forename: John A
authors.initials: JA
authors.lastname: Lust
authors.forename: Stephen J
authors.initials: SJ
authors.lastname: Russell
authors.forename: Morie A
authors.initials: MA
authors.lastname: Gertz
authors.forename: Martha Q
authors.initials: MQ
authors.lastname: Lacy
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D000686:Amyloidosis / Q000188:drug therapy* / Q000378:metabolism / Q000473:pathology; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D003907:Dexamethasone / Q000009:adverse effects / Q000627:therapeutic use*; D018572:Disease-Free Survival; D004359:Drug Therapy, Combination / Q000009:adverse effects; D005260:Female; D006403:Hematologic Tests; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000378:metabolism*; D008297:Male; D008875:Middle Aged; D010950:Plasma Cells / Q000187:drug effects* / Q000378:metabolism / Q000473:pathology; D013792:Thalidomide / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use
chemical_list: D000970:Antineoplastic Agents; D007147:Immunoglobulin Light Chains; D013792:Thalidomide; D003907:Dexamethasone; C467566:pomalidomide