Pomalidomide-dexamethasone in refractory multiple myeloma: Long-term follow-up of a multi-cohort phase II clinical trial

Sikander Ailawadhi, Joseph R Mikhael, B. R. Laplant, K. M. Laumann, Shaji K Kumar, Vivek Roy, David M Dingli, Peter Leif Bergsagel, F. K. Buadi, S Vincent Rajkumar, Rafael Fonseca, Morie Gertz, Prashant Kapoor, Taimur Sher, S. R. Hayman, Alexander Keith Stewart, Angela Dispenzieri, R. A. Kyle, Wilson Gonsalves, C. B. ReederYi Lin, R. S. Go, N. Leung, Taxiarchis Kourelis, J. A. Lust, Stephen J Russell, Asher A Chanan Khan, Martha Lacy

Research output: Contribution to journalArticle

7 Citations (Scopus)

Abstract

Despite therapeutic advances, multiple myeloma remains incurable, with limited options for patients with refractory disease. We conducted a large, multi-cohort clinical trial testing various doses and treatment schedules of pomalidomide and dexamethasone (Pom/dex) in patients with refractory multiple myeloma. Overall, 345 patients were enrolled to six cohorts based on number and type of prior lines of therapy, pomalidomide dose and schedule. Median prior lines of therapy were three with near universal prior exposure to proteasome inhibitors and/or immunomodulatory drugs. A confirmed response rate of 35% was noted for all cohorts (range 23-65%) with higher responses in cohorts with fewer prior lines of therapy. Median time to confirmed response was ∼ 1/22 months and the longest progression-free survival and overall survival seen in any cohort were 13.1 and 47.9 months, respectively. Observed adverse reactions were as expected, with myelosuppression and fatigue being the most common hematologic and non-hematologic adverse events (AEs), respectively. Longer durations of treatment and response, higher response rates and fewer AEs were noted with the 2 mg pomalidomide dose. This is the longest follow-up data for Pom/dex in refractory multiple myeloma and will help shape the real-world utilization of this regimen.

Original languageEnglish (US)
Pages (from-to)719-728
Number of pages10
JournalLeukemia
Volume32
Issue number3
DOIs
StatePublished - Mar 1 2018

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Phase II Clinical Trials
Multiple Myeloma
Dexamethasone
Appointments and Schedules
Therapeutics
Proteasome Inhibitors
Disease-Free Survival
Fatigue
pomalidomide
Clinical Trials
Survival
Pharmaceutical Preparations

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

Cite this

@article{71f5cefe30f6441e801911dcc44d52f5,
title = "Pomalidomide-dexamethasone in refractory multiple myeloma: Long-term follow-up of a multi-cohort phase II clinical trial",
abstract = "Despite therapeutic advances, multiple myeloma remains incurable, with limited options for patients with refractory disease. We conducted a large, multi-cohort clinical trial testing various doses and treatment schedules of pomalidomide and dexamethasone (Pom/dex) in patients with refractory multiple myeloma. Overall, 345 patients were enrolled to six cohorts based on number and type of prior lines of therapy, pomalidomide dose and schedule. Median prior lines of therapy were three with near universal prior exposure to proteasome inhibitors and/or immunomodulatory drugs. A confirmed response rate of 35{\%} was noted for all cohorts (range 23-65{\%}) with higher responses in cohorts with fewer prior lines of therapy. Median time to confirmed response was ∼ 1/22 months and the longest progression-free survival and overall survival seen in any cohort were 13.1 and 47.9 months, respectively. Observed adverse reactions were as expected, with myelosuppression and fatigue being the most common hematologic and non-hematologic adverse events (AEs), respectively. Longer durations of treatment and response, higher response rates and fewer AEs were noted with the 2 mg pomalidomide dose. This is the longest follow-up data for Pom/dex in refractory multiple myeloma and will help shape the real-world utilization of this regimen.",
author = "Sikander Ailawadhi and Mikhael, {Joseph R} and Laplant, {B. R.} and Laumann, {K. M.} and Kumar, {Shaji K} and Vivek Roy and Dingli, {David M} and Bergsagel, {Peter Leif} and Buadi, {F. K.} and Rajkumar, {S Vincent} and Rafael Fonseca and Morie Gertz and Prashant Kapoor and Taimur Sher and Hayman, {S. R.} and Stewart, {Alexander Keith} and Angela Dispenzieri and Kyle, {R. A.} and Wilson Gonsalves and Reeder, {C. B.} and Yi Lin and Go, {R. S.} and N. Leung and Taxiarchis Kourelis and Lust, {J. A.} and Russell, {Stephen J} and {Chanan Khan}, {Asher A} and Martha Lacy",
year = "2018",
month = "3",
day = "1",
doi = "10.1038/leu.2017.258",
language = "English (US)",
volume = "32",
pages = "719--728",
journal = "Leukemia",
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publisher = "Nature Publishing Group",
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TY - JOUR

T1 - Pomalidomide-dexamethasone in refractory multiple myeloma

T2 - Long-term follow-up of a multi-cohort phase II clinical trial

AU - Ailawadhi, Sikander

AU - Mikhael, Joseph R

AU - Laplant, B. R.

AU - Laumann, K. M.

AU - Kumar, Shaji K

AU - Roy, Vivek

AU - Dingli, David M

AU - Bergsagel, Peter Leif

AU - Buadi, F. K.

AU - Rajkumar, S Vincent

AU - Fonseca, Rafael

AU - Gertz, Morie

AU - Kapoor, Prashant

AU - Sher, Taimur

AU - Hayman, S. R.

AU - Stewart, Alexander Keith

AU - Dispenzieri, Angela

AU - Kyle, R. A.

AU - Gonsalves, Wilson

AU - Reeder, C. B.

AU - Lin, Yi

AU - Go, R. S.

AU - Leung, N.

AU - Kourelis, Taxiarchis

AU - Lust, J. A.

AU - Russell, Stephen J

AU - Chanan Khan, Asher A

AU - Lacy, Martha

PY - 2018/3/1

Y1 - 2018/3/1

N2 - Despite therapeutic advances, multiple myeloma remains incurable, with limited options for patients with refractory disease. We conducted a large, multi-cohort clinical trial testing various doses and treatment schedules of pomalidomide and dexamethasone (Pom/dex) in patients with refractory multiple myeloma. Overall, 345 patients were enrolled to six cohorts based on number and type of prior lines of therapy, pomalidomide dose and schedule. Median prior lines of therapy were three with near universal prior exposure to proteasome inhibitors and/or immunomodulatory drugs. A confirmed response rate of 35% was noted for all cohorts (range 23-65%) with higher responses in cohorts with fewer prior lines of therapy. Median time to confirmed response was ∼ 1/22 months and the longest progression-free survival and overall survival seen in any cohort were 13.1 and 47.9 months, respectively. Observed adverse reactions were as expected, with myelosuppression and fatigue being the most common hematologic and non-hematologic adverse events (AEs), respectively. Longer durations of treatment and response, higher response rates and fewer AEs were noted with the 2 mg pomalidomide dose. This is the longest follow-up data for Pom/dex in refractory multiple myeloma and will help shape the real-world utilization of this regimen.

AB - Despite therapeutic advances, multiple myeloma remains incurable, with limited options for patients with refractory disease. We conducted a large, multi-cohort clinical trial testing various doses and treatment schedules of pomalidomide and dexamethasone (Pom/dex) in patients with refractory multiple myeloma. Overall, 345 patients were enrolled to six cohorts based on number and type of prior lines of therapy, pomalidomide dose and schedule. Median prior lines of therapy were three with near universal prior exposure to proteasome inhibitors and/or immunomodulatory drugs. A confirmed response rate of 35% was noted for all cohorts (range 23-65%) with higher responses in cohorts with fewer prior lines of therapy. Median time to confirmed response was ∼ 1/22 months and the longest progression-free survival and overall survival seen in any cohort were 13.1 and 47.9 months, respectively. Observed adverse reactions were as expected, with myelosuppression and fatigue being the most common hematologic and non-hematologic adverse events (AEs), respectively. Longer durations of treatment and response, higher response rates and fewer AEs were noted with the 2 mg pomalidomide dose. This is the longest follow-up data for Pom/dex in refractory multiple myeloma and will help shape the real-world utilization of this regimen.

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