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Phase II study of pomalidomide in high-risk relapsed and refractory multiple myeloma

Leukemia volume 28, pages 2413–2415 (2014)Cite this article

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Pomalidomide (Pom) is an IMiD immunomodulatory agent that is now FDA approved for treatment of patients who have received ⩾2 prior therapies, including lenalidomide (Len) and bortezomib (Bor), and have demonstrated disease progression on or within 60 days of completion of the last line of therapy.1 There are limited data evaluating efficacy of Pom in high-risk RRMM with prior exposure/refractoriness to Len and how best to combine it with other available multiple myeloma (MM) therapies for this patient population. Herein, we are reporting a novel, response-adaptive, phase II clinical trial design that used Pom in high-risk RRMM with sequential addition of specific MM therapies.

The study was approved by the UAMS IRB. All subjects were informed of the investigational nature of this study and gave written voluntary consent in accordance with institutional and federal guidelines. Patients with MM and high-risk features that has relapsed or resistant to ⩾1 prior MM therapy were eligible for enrollment. High-risk features were defined by having high risk on gene expression profiling (GEP) (GEP 702 or GEP803 signatures), elevated LDH or having abnormal metaphase cytogenetics at the time of disease relapse or progression. Patients with RRMM over 18 years of age and ECOG performance status 0–2 were eligible. All subjects were required to take aspirin or alternate prophylactic anticoagulation while on study.

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Acknowledgements

Clinical trial and translational GEP studies were supported by Celgene Corporation.

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Authors and Affiliations

  1. Myeloma Institute for Research and Therapy, University of Arkansas for Medical Sciences, Little Rock, AR, USA

    S Z Usmani, Q Zhang, S Yaccoby, D Steward, S Panozzo, N Petty, S Waheed, F Van Rhee & B Barlogie

  2. Levine Cancer Institute/Carolinas Healthcare System, Charlotte, NC, USA

    S Z Usmani

  3. Cancer Research and Biostatistics, Seattle, WA, USA

    K Stratton, P Qu, E Hansen, A Hoering & J Crowley

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  1. S Z Usmani
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  6. E Hansen
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  8. S Panozzo
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  14. B Barlogie
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Corresponding author

Correspondence to S Z Usmani.

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Competing interests

SZU is a consultant to Celgene, Millennium, Onyx and Sanofi. He has received research funding from ArrayBioPharma, Celgene, Onyx, Janssen, Pharmacyclics, and speaking honoraria from Celgene, Millennium and Onyx. BB has received research funding from Celgene and Novartis. He is a consultant to Celgene and Genzyme and has received speaking honoraria from Celgene and Millennium. BB is a co-inventor on patents and patent applications related to use of GEP in cancer medicine.

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Usmani, S., Zhang, Q., Stratton, K. et al. Phase II study of pomalidomide in high-risk relapsed and refractory multiple myeloma. Leukemia 28, 2413–2415 (2014). https://doi.org/10.1038/leu.2014.248

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