Carfilzomib, lenalidomide, dexamethasone, and cyclophosphamide (KRdc) as induction therapy for transplant-eligible, newly diagnosed multiple myeloma patients (Myeloma XI+): Interim analysis of an open-label randomised controlled trial.

<h4>Background</h4>Carfilzomib is a second-generation irreversible proteasome inhibitor that is efficacious in the treatment of myeloma and carries less risk of peripheral neuropathy than first-generation proteasome inhibitors, making it more amenable to combination therapy.<h4>Met...

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Main Authors: Graham H Jackson, Charlotte Pawlyn, David A Cairns, Ruth M de Tute, Anna Hockaday, Corinne Collett, John R Jones, Bhuvan Kishore, Mamta Garg, Cathy D Williams, Kamaraj Karunanithi, Jindriska Lindsay, Alberto Rocci, John A Snowden, Matthew W Jenner, Gordon Cook, Nigel H Russell, Mark T Drayson, Walter M Gregory, Martin F Kaiser, Roger G Owen, Faith E Davies, Gareth J Morgan, UK NCRI Haemato-oncology Clinical Studies Group
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2021-01-01
Series:PLoS Medicine
Online Access:https://journals.plos.org/plosmedicine/article/file?id=10.1371/journal.pmed.1003454&type=printable
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author Graham H Jackson
Charlotte Pawlyn
David A Cairns
Ruth M de Tute
Anna Hockaday
Corinne Collett
John R Jones
Bhuvan Kishore
Mamta Garg
Cathy D Williams
Kamaraj Karunanithi
Jindriska Lindsay
Alberto Rocci
John A Snowden
Matthew W Jenner
Gordon Cook
Nigel H Russell
Mark T Drayson
Walter M Gregory
Martin F Kaiser
Roger G Owen
Faith E Davies
Gareth J Morgan
UK NCRI Haemato-oncology Clinical Studies Group
author_facet Graham H Jackson
Charlotte Pawlyn
David A Cairns
Ruth M de Tute
Anna Hockaday
Corinne Collett
John R Jones
Bhuvan Kishore
Mamta Garg
Cathy D Williams
Kamaraj Karunanithi
Jindriska Lindsay
Alberto Rocci
John A Snowden
Matthew W Jenner
Gordon Cook
Nigel H Russell
Mark T Drayson
Walter M Gregory
Martin F Kaiser
Roger G Owen
Faith E Davies
Gareth J Morgan
UK NCRI Haemato-oncology Clinical Studies Group
author_sort Graham H Jackson
collection DOAJ
description <h4>Background</h4>Carfilzomib is a second-generation irreversible proteasome inhibitor that is efficacious in the treatment of myeloma and carries less risk of peripheral neuropathy than first-generation proteasome inhibitors, making it more amenable to combination therapy.<h4>Methods and findings</h4>The Myeloma XI+ trial recruited patients from 88 sites across the UK between 5 December 2013 and 20 April 2016. Patients with newly diagnosed multiple myeloma eligible for transplantation were randomly assigned to receive the combination carfilzomib, lenalidomide, dexamethasone, and cyclophosphamide (KRdc) or a triplet of lenalidomide, dexamethasone, and cyclophosphamide (Rdc) or thalidomide, dexamethasone, and cyclophosphamide (Tdc). All patients were planned to receive an autologous stem cell transplantation (ASCT) prior to a randomisation between lenalidomide maintenance and observation. Eligible patients were aged over 18 years and had symptomatic myeloma. The co-primary endpoints for the study were progression-free survival (PFS) and overall survival (OS) for KRdc versus the Tdc/Rdc control group by intention to treat. PFS, response, and safety outcomes are reported following a planned interim analysis. The trial is registered (ISRCTN49407852) and has completed recruitment. In total, 1,056 patients (median age 61 years, range 33 to 75, 39.1% female) underwent induction randomisation to KRdc (n = 526) or control (Tdc/Rdc, n = 530). After a median follow-up of 34.5 months, KRdc was associated with a significantly longer PFS than the triplet control group (hazard ratio 0.63, 95% CI 0.51-0.76). The median PFS for patients receiving KRdc is not yet estimable, versus 36.2 months for the triplet control group (p < 0.001). Improved PFS was consistent across subgroups of patients including those with genetically high-risk disease. At the end of induction, the percentage of patients achieving at least a very good partial response was 82.3% in the KRdc group versus 58.9% in the control group (odds ratio 4.35, 95% CI 3.19-5.94, p < 0.001). Minimal residual disease negativity (cutoff 4 × 10-5 bone marrow leucocytes) was achieved in 55% of patients tested in the KRdc group at the end of induction, increasing to 75% of those tested after ASCT. The most common adverse events were haematological, with a low incidence of cardiac events. The trial continues to follow up patients to the co-primary endpoint of OS and for planned long-term follow-up analysis. Limitations of the study include a lack of blinding to treatment regimen and that the triplet control regimen did not include a proteasome inhibitor for all patients, which would be considered a current standard of care in many parts of the world.<h4>Conclusions</h4>The KRdc combination was well tolerated and was associated with both an increased percentage of patients achieving at least a very good partial response and a significant PFS benefit compared to immunomodulatory-agent-based triplet therapy.<h4>Trial registration</h4>ClinicalTrials.gov ISRCTN49407852.
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spelling doaj-art-7fe445c9d7494b4397c8fa9d9a9d21822025-08-20T03:20:30ZengPublic Library of Science (PLoS)PLoS Medicine1549-12771549-16762021-01-01181e100345410.1371/journal.pmed.1003454Carfilzomib, lenalidomide, dexamethasone, and cyclophosphamide (KRdc) as induction therapy for transplant-eligible, newly diagnosed multiple myeloma patients (Myeloma XI+): Interim analysis of an open-label randomised controlled trial.Graham H JacksonCharlotte PawlynDavid A CairnsRuth M de TuteAnna HockadayCorinne CollettJohn R JonesBhuvan KishoreMamta GargCathy D WilliamsKamaraj KarunanithiJindriska LindsayAlberto RocciJohn A SnowdenMatthew W JennerGordon CookNigel H RussellMark T DraysonWalter M GregoryMartin F KaiserRoger G OwenFaith E DaviesGareth J MorganUK NCRI Haemato-oncology Clinical Studies Group<h4>Background</h4>Carfilzomib is a second-generation irreversible proteasome inhibitor that is efficacious in the treatment of myeloma and carries less risk of peripheral neuropathy than first-generation proteasome inhibitors, making it more amenable to combination therapy.<h4>Methods and findings</h4>The Myeloma XI+ trial recruited patients from 88 sites across the UK between 5 December 2013 and 20 April 2016. Patients with newly diagnosed multiple myeloma eligible for transplantation were randomly assigned to receive the combination carfilzomib, lenalidomide, dexamethasone, and cyclophosphamide (KRdc) or a triplet of lenalidomide, dexamethasone, and cyclophosphamide (Rdc) or thalidomide, dexamethasone, and cyclophosphamide (Tdc). All patients were planned to receive an autologous stem cell transplantation (ASCT) prior to a randomisation between lenalidomide maintenance and observation. Eligible patients were aged over 18 years and had symptomatic myeloma. The co-primary endpoints for the study were progression-free survival (PFS) and overall survival (OS) for KRdc versus the Tdc/Rdc control group by intention to treat. PFS, response, and safety outcomes are reported following a planned interim analysis. The trial is registered (ISRCTN49407852) and has completed recruitment. In total, 1,056 patients (median age 61 years, range 33 to 75, 39.1% female) underwent induction randomisation to KRdc (n = 526) or control (Tdc/Rdc, n = 530). After a median follow-up of 34.5 months, KRdc was associated with a significantly longer PFS than the triplet control group (hazard ratio 0.63, 95% CI 0.51-0.76). The median PFS for patients receiving KRdc is not yet estimable, versus 36.2 months for the triplet control group (p < 0.001). Improved PFS was consistent across subgroups of patients including those with genetically high-risk disease. At the end of induction, the percentage of patients achieving at least a very good partial response was 82.3% in the KRdc group versus 58.9% in the control group (odds ratio 4.35, 95% CI 3.19-5.94, p < 0.001). Minimal residual disease negativity (cutoff 4 × 10-5 bone marrow leucocytes) was achieved in 55% of patients tested in the KRdc group at the end of induction, increasing to 75% of those tested after ASCT. The most common adverse events were haematological, with a low incidence of cardiac events. The trial continues to follow up patients to the co-primary endpoint of OS and for planned long-term follow-up analysis. Limitations of the study include a lack of blinding to treatment regimen and that the triplet control regimen did not include a proteasome inhibitor for all patients, which would be considered a current standard of care in many parts of the world.<h4>Conclusions</h4>The KRdc combination was well tolerated and was associated with both an increased percentage of patients achieving at least a very good partial response and a significant PFS benefit compared to immunomodulatory-agent-based triplet therapy.<h4>Trial registration</h4>ClinicalTrials.gov ISRCTN49407852.https://journals.plos.org/plosmedicine/article/file?id=10.1371/journal.pmed.1003454&type=printable
spellingShingle Graham H Jackson
Charlotte Pawlyn
David A Cairns
Ruth M de Tute
Anna Hockaday
Corinne Collett
John R Jones
Bhuvan Kishore
Mamta Garg
Cathy D Williams
Kamaraj Karunanithi
Jindriska Lindsay
Alberto Rocci
John A Snowden
Matthew W Jenner
Gordon Cook
Nigel H Russell
Mark T Drayson
Walter M Gregory
Martin F Kaiser
Roger G Owen
Faith E Davies
Gareth J Morgan
UK NCRI Haemato-oncology Clinical Studies Group
Carfilzomib, lenalidomide, dexamethasone, and cyclophosphamide (KRdc) as induction therapy for transplant-eligible, newly diagnosed multiple myeloma patients (Myeloma XI+): Interim analysis of an open-label randomised controlled trial.
PLoS Medicine
title Carfilzomib, lenalidomide, dexamethasone, and cyclophosphamide (KRdc) as induction therapy for transplant-eligible, newly diagnosed multiple myeloma patients (Myeloma XI+): Interim analysis of an open-label randomised controlled trial.
title_full Carfilzomib, lenalidomide, dexamethasone, and cyclophosphamide (KRdc) as induction therapy for transplant-eligible, newly diagnosed multiple myeloma patients (Myeloma XI+): Interim analysis of an open-label randomised controlled trial.
title_fullStr Carfilzomib, lenalidomide, dexamethasone, and cyclophosphamide (KRdc) as induction therapy for transplant-eligible, newly diagnosed multiple myeloma patients (Myeloma XI+): Interim analysis of an open-label randomised controlled trial.
title_full_unstemmed Carfilzomib, lenalidomide, dexamethasone, and cyclophosphamide (KRdc) as induction therapy for transplant-eligible, newly diagnosed multiple myeloma patients (Myeloma XI+): Interim analysis of an open-label randomised controlled trial.
title_short Carfilzomib, lenalidomide, dexamethasone, and cyclophosphamide (KRdc) as induction therapy for transplant-eligible, newly diagnosed multiple myeloma patients (Myeloma XI+): Interim analysis of an open-label randomised controlled trial.
title_sort carfilzomib lenalidomide dexamethasone and cyclophosphamide krdc as induction therapy for transplant eligible newly diagnosed multiple myeloma patients myeloma xi interim analysis of an open label randomised controlled trial
url https://journals.plos.org/plosmedicine/article/file?id=10.1371/journal.pmed.1003454&type=printable
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