Exceptional long-term responses from OCEAN and HORIZON trials: melflufen-dexamethasone as an expansion of treatment options for relapsed/refractory multiple myeloma in the era of new immunotherapies?

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00179906%3A_____%2F25%3A10504822" target="_blank" >RIV/00179906:_____/25:10504822 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00216208:11150/25:10504822

Výsledek na webu

<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=ulhNs7U6TK" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=ulhNs7U6TK</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1007/s00432-025-06326-3" target="_blank" >10.1007/s00432-025-06326-3</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Exceptional long-term responses from OCEAN and HORIZON trials: melflufen-dexamethasone as an expansion of treatment options for relapsed/refractory multiple myeloma in the era of new immunotherapies?

Popis výsledku v původním jazyce

Alkylating agents have represented the first effective drug class in multiple myeloma (MM) but, since the introduction of novel effective drugs, their use has progressively decreased and is currently relegated to autologous stem cell transplant (ASCT) and few other settings. Nevertheless, the combination of melflufen (a peptide-drug conjugate pro-drug of melphalan) and dexamethasone was approved by the U.S. Food &amp; Drug Administration (FDA) for triple-class refractory (TCR) patients after &gt;= 4 prior lines of therapy (LOT) following results of HORIZON clinical trial (NCT02963493). This combination was subsequently withdrawn as it was not associated with improved overall survival (OS) as compared to pomalidomide-dexamethasone (OCEAN clinical trial, NCT03151811). However, since a post-hoc analysis showed a benefit in OS for patients without prior ASCT or with a time to progression (TTP) &gt; 36 months after ASCT, the European Medicines Agency (EMA) has approved melflufen-dexamethasone for TCR patients after &gt;= 3 LOT, including specification that TTP must be &gt;= 3 years in patients with prior ASCT. In this paper, we report three cases of patients receiving the combination melflufen-dexamethasone in the aforementioned clinical trials in three hematologic centers across Europe and achieving exceptionally long responses as compared to the overall enrolled populations, with good tolerability. Further, we discuss the potential use of this chemotherapy-based regimen in the era of novel immunotherapies.

Název v anglickém jazyce

Exceptional long-term responses from OCEAN and HORIZON trials: melflufen-dexamethasone as an expansion of treatment options for relapsed/refractory multiple myeloma in the era of new immunotherapies?

Popis výsledku anglicky

Alkylating agents have represented the first effective drug class in multiple myeloma (MM) but, since the introduction of novel effective drugs, their use has progressively decreased and is currently relegated to autologous stem cell transplant (ASCT) and few other settings. Nevertheless, the combination of melflufen (a peptide-drug conjugate pro-drug of melphalan) and dexamethasone was approved by the U.S. Food &amp; Drug Administration (FDA) for triple-class refractory (TCR) patients after &gt;= 4 prior lines of therapy (LOT) following results of HORIZON clinical trial (NCT02963493). This combination was subsequently withdrawn as it was not associated with improved overall survival (OS) as compared to pomalidomide-dexamethasone (OCEAN clinical trial, NCT03151811). However, since a post-hoc analysis showed a benefit in OS for patients without prior ASCT or with a time to progression (TTP) &gt; 36 months after ASCT, the European Medicines Agency (EMA) has approved melflufen-dexamethasone for TCR patients after &gt;= 3 LOT, including specification that TTP must be &gt;= 3 years in patients with prior ASCT. In this paper, we report three cases of patients receiving the combination melflufen-dexamethasone in the aforementioned clinical trials in three hematologic centers across Europe and achieving exceptionally long responses as compared to the overall enrolled populations, with good tolerability. Further, we discuss the potential use of this chemotherapy-based regimen in the era of novel immunotherapies.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30204 - Oncology

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2025

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Journal of Cancer Research and Clinical Oncology

ISSN

0171-5216

e-ISSN

1432-1335

Svazek periodika

151

Číslo periodika v rámci svazku

11

Stát vydavatele periodika

DE - Spolková republika Německo

Počet stran výsledku

10

Strana od-do

288

Kód UT WoS článku

001591244900001

EID výsledku v databázi Scopus

2-s2.0-105018295641