Melflufen and dexamethasone in heavily pretreated relapsed and refractory multiple myeloma
Richardson, Paul G.. (Dana-Farber Cancer Institute (Boston, Estats Units d'Amèrica). Harvard Medical School)
Oriol, Albert (Institut Germans Trias i Pujol. Institut de Recerca contra la Leucèmia Josep Carreras)
Larocca, Alessandra (Università di Torino. Azienda Ospedaliero-Universitaria Città della Salute e della Scienza)
Bladé Creixenti, Juan (Hospital Clínic i Provincial de Barcelona)
Cavo, Michele (Seràgnoli Institute of Hematology. Bologna University School of Medicine)
Rodriguez-Otero, Paula (Clínica Universidad de Navarra)
Leleu, Xavier (CHU de Poitiers)
Nadeem, Omar (Dana-Farber Cancer Institute (Boston, Estats Units d'Amèrica). Harvard Medical School)
Hiemenz, John W. (Division of Hematology-Oncology. Department of Medicine. University of Florida)
Hassoun, Hani (Memorial Sloan Kettering Cancer Center)
Touzeau, Cyrille (Université de Nantes)
Alegre, Adrian (Hospital Universitario de la Princesa (Madrid))
Paner, Agne (Rush University Medical Center)
Maisel, Christopher (Baylor Scott & White Charles A. Sammons Cancer Center)
Mazumder, Amitabha (Oncology Institute of Hope and Innovation)
Raptis, Anastasios (Universityof Pittsburgh School of Medicine)
Moreb, Jan S. (Novant Health Hematology. Novant Health Forsyth Medical Center)
Anderson, Kenneth C. (Dana-Farber Cancer Institute (Boston, Estats Units d'Amèrica). Harvard Medical School)
Laubach, Jacob P. (Dana-Farber Cancer Institute (Boston, Estats Units d'Amèrica). Harvard Medical School)
Thuresson, Sara (Oncopeptides AB)
Thuresson, Marcus (Oncopeptides AB)
Byrne, Catriona (Oncopeptides AB)
Harmenberg, Johan (Oncopeptides AB)
Bakker, Nicolaas A. (Oncopeptides AB)
Mateos, M. V (Hospital Clínico Universitario (Salamanca))

Data:	2021
Resum:	PURPOSE Melphalan flufenamide (melflufen) is a first-in-class peptide-drug conjugate that targets aminopeptidases and rapidly and selectively releases alkylating agents into tumor cells. The phase II HORIZON trial evaluated the efficacy of melflufen plus dexamethasone in relapsed and refractory multiple myeloma (RRMM), a population with an important unmet medical need. PATIENTS AND METHODS Patients with RRMM refractory to pomalidomide and/or an anti-CD38 monoclonal antibody received melflufen 40 mg intravenously on day 1 of each 28-day cycle plus once weekly oral dexamethasone at a dose of 40 mg (20 mg in patients older than 75 years). The primary end point was overall response rate (partial response or better) assessed by the investigator and confirmed by independent review. Secondary end points included duration of response, progression-free survival, overall survival, and safety. The primary analysis is complete with long-term follow-up ongoing. RESULTS Of 157 patients (median age 65 years; median five prior lines of therapy) enrolled and treated, 119 patients (76%) had triple-class-refractory disease, 55 (35%) had extramedullary disease, and 92 (59%) were refractory to previous alkylator therapy. The overall response rate was 29% in the all-treated population, with 26% in the triple-class-refractory population. In the all-treated population, median duration of response was 5. 5 months, median progression-free survival was 4. 2 months, and median overall survival was 11. 6 months at a median follow-up of 14 months. Grade.
Nota:	Clinical trial information: NCT02963493
Nota:	Altres ajuts: Oncopeptides AB
Drets:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.
Llengua:	Anglès
Document:	Article ; recerca ; Versió publicada
Publicat a:	Journal of clinical oncology, Vol. 39 Núm. 7 (january 2021) , p. 757-767, ISSN 1527-7755

DOI: 10.1200/JCO.20.02259
PMID: 33296242

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Documents de recerca > Documents dels grups de recerca de la UAB > Centres i grups de recerca (producció científica) > Ciències de la salut i biociències > Institut d'Investigació en Ciencies de la Salut Germans Trias i Pujol (IGTP) > Institut de Recerca contra la Leucèmia Josep Carreras
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