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Pomalidomide

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Small Molecules in Hematology

Part of the book series: Recent Results in Cancer Research ((RECENTCANCER,volume 212))

Abstract

Pomalidomide (originally CC-4047 or 3-amino-thalidomide) is a derivative of thalidomide that is antiangiogenic and also acts as immunomodulatory. Pomalidomide, the recent immunomodulatory agent (IMiD), has shown substantial in vitro antiproliferative and proapoptotic effects. In vivo studies have suggested limited cross-resistance between lenalidomide and pomalidomide. Moreover, pomalidomide achieved very convincing responses in relapsed and refractory multiple myeloma (RRMM) patients, including those, who are refractory to both lenalidomide and bortezomib. Since pomalidomide plus low-dose dexamethasone has shown better responses, progression-free survival (PFS) and overall survival (OS) than high-dose dexamethasone or pomalidomide alone, subsequent trials have pursued or are still investigating pomalidomide triplet combinations, using cyclophosphamide or other novel agents, such as proteasome inhibitors (PI: bortezomib, carfilzomib) or antibodies, like elotuzumab or daratumumab. Pomalidomide has also been assessed in AL amyloidosis, MPNs (myelofibrosis [MF]), Waldenstrom’s macroglobulinemia, solid tumors (sarcoma, lung cancer), or HIV, and—for AL amyloidosis and MF—has already been proven to be remarkably active. Due to its potency, pomalidomide was approved for RRMM by the US Food and Drug Administration (FDA) and by the European Medicines Agency (EMA) in 2013 and for drug combination with low-dose dexamethasone in 2015. In June 2017, the FDA further expanded approval for pomalidomide in combination with daratumumab and low-dose dexamethasone for patients with RRMM.

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Authors and Affiliations

  1. Hematology and Oncology, University of Freiburg, Hugstetter Str. 55, 79106, Freiburg, Germany

    Monika Engelhardt, Stefanie Ajayi, Heike Reinhardt, Stefan Jürgen Müller, Sandra Maria Dold & Ralph Wäsch

  2. Comprehensive Cancer Center Freiburg (CCCF), Hugstetter Str. 55, 79106, Freiburg, Germany

    Monika Engelhardt, Stefanie Ajayi & Ralph Wäsch

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  1. Monika Engelhardt
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  2. Stefanie Ajayi
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  3. Heike Reinhardt
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  4. Stefan Jürgen Müller
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  5. Sandra Maria Dold
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  6. Ralph Wäsch
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Correspondence to Monika Engelhardt .

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  1. MOLIT Institute, Cancer Center Heilbronn-Franken, SLK-Clinics, Heilbronn, Baden-Württemberg, Germany

    Uwe M. Martens

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Engelhardt, M., Ajayi, S., Reinhardt, H., Müller, S.J., Dold, S.M., Wäsch, R. (2018). Pomalidomide. In: Martens, U. (eds) Small Molecules in Hematology. Recent Results in Cancer Research, vol 212. Springer, Cham. https://doi.org/10.1007/978-3-319-91439-8_8

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  • DOI: https://doi.org/10.1007/978-3-319-91439-8_8

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