Long-term outcomes of IMiD-based trials in patients with immunoglobulin light-chain amyloidosis: a pooled analysis

Rahma Warsame, Betsy LaPlant, Shaji K. Kumar, Kristina Laumann, Gabriela Perez Burbano, Francis K. Buadi, Morie A. Gertz, Robert A. Kyle, Martha Q. Lacy, David Dingli, Nelson Leung, Suzanne R. Hayman, Prashant Kapoor, Yi L. Hwa, Amie Fonder, Miriam Hobbs, Wilson I. Gonsalves, Taxiarchis Kourelis, John Lust, Stephen J. RussellSteven Zeldenrust, Yi Lin, Eli Muchtar, Ronald S. Go, S. Vincent Rajkumar, Angela Dispenzieri

Research output: Contribution to journalArticlepeer-review

7 Scopus citations

Abstract

Rarity of light-chain amyloidosis (AL) makes randomized studies challenging. We pooled three phase II studies of immunomodulatory drugs (IMiDs) to update survival, toxicity, and assess new response/progression criteria. Studies included were lenalidomide-dexamethasone (Len-Dex) (n = 37; years: 2004–2006), cyclophosphamide-Len-Dex (n = 35; years: 2007–2008), and pomalidomide-Dex (n = 29; years: 2008–2010) trial. Primary endpoint was hematologic response. Overall survival (OS) was calculated from registration to death and progression-free survival (PFS) was calculated from registration to progression or death. Hematologic, cardiac, and renal response/progression was assessed using the modern criteria. Analysis included 101 patients, with a median age of 65 years, 61% male, 37 newly diagnosed (ND), and 64 relapsed/refractory (RR). Median follow-up was 101 months (range 17–150) and 78% of patients died. OS and PFS for pooled cohort were 31 and 15 months, respectively. Forty-eight patients achieved a hematologic response; for ND, 10 patients (28%) achieved ≥VGPR (very good partial response) and 8 (14%) among the RR. Only cardiac stage was prognostic for OS. Common grade ≥3 toxicities were hematologic, fatigue, and rash, and were similar among studies. Hematologic and renal responses occurred more frequently and rapidly using modern response criteria; cardiac response was less frequent but occurred quickly. IMiDs can result in long progression-free intervals/survival with tolerable toxicities. The new response/progression criteria were rapid and allows for tailoring therapy.

Original languageEnglish (US)
Article number4
JournalBlood cancer journal
Volume10
Issue number1
DOIs
StatePublished - Jan 1 2020

ASJC Scopus subject areas

  • Hematology
  • Oncology

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