TY - JOUR
T1 - Finding a consensus on time-to-event endpoints definitions in marginal zone lymphoma
T2 - A Delphi method
AU - Bommier, Côme
AU - Lambert, Jérôme
AU - Nowakowski, Grzegorz
AU - Zucca, Emanuele
AU - Thieblemont, Catherine
N1 - Funding Information:
To all the experts from the MZL community that gave their time to take our survey, and more specifically to the Rounds 2-3 experts: Luca Arcaini (Pavia, Italy), Mark Bishton (Nottingham, UK), Christian Buske (Ulm, Germany), Guillaume Cartron (Montpellier, France), Luciano Cascione (Bellizona, Switzerland), Loïc Chartier (Lyon, France), Annarita Conconi (Biella, Italy), Anthony Davies (Southampton, UK), Martin Dreyling (München, Germany), Andres Ferreri (Milano, Italy), Christohper Flowers (Houston, USA), Norma Gutierrez (Salamanca, Spain), Emilio Iannitto (Palermo, Italy), Jean-Philippe Jais (Paris, France), Peter Johnson (Southampton, UK), Clémentine Joubert (Lyon, France), Noémie Lang (Genève, Swittzerland), Isidore Lossos (Miami, USA), Stefano Luminari (Modena, Italy), Peter Martin (New York, USA), Carlos Montalbàn (Madrid, Spain), Franck Morschhauser (Lille, France), Nicolas Mounier (Nice, France), Urban Novak (Bern, Switzerland), Grzegorz Nowakowski (Rochester, USA), Davide Rossi (Bellinzona, Switzerland), Gilles Salles (New York, USA), Stephen Smith (Seattle, USA), Anasthasios Stathis (Bellinzona, Switzerland), Catherine Thieblemont (Paris, France), Hervé Tilly (Caen, France), Judith Trotman (Sydney, Australia). Côme Bommier is funded by Inserm/ITMO/Aviesan (FRFT-DOC21) as part of his PhD program.
Funding Information:
To all the experts from the MZL community that gave their time to take our survey, and more specifically to the Rounds 2‐3 experts: Luca Arcaini (Pavia, Italy), Mark Bishton (Nottingham, UK), Christian Buske (Ulm, Germany), Guillaume Cartron (Montpellier, France), Luciano Cascione (Bellizona, Switzerland), Loïc Chartier (Lyon, France), Annarita Conconi (Biella, Italy), Anthony Davies (Southampton, UK), Martin Dreyling (München, Germany), Andres Ferreri (Milano, Italy), Christohper Flowers (Houston, USA), Norma Gutierrez (Salamanca, Spain), Emilio Iannitto (Palermo, Italy), Jean‐Philippe Jais (Paris, France), Peter Johnson (Southampton, UK), Clémentine Joubert (Lyon, France), Noémie Lang (Genève, Swittzerland), Isidore Lossos (Miami, USA), Stefano Luminari (Modena, Italy), Peter Martin (New York, USA), Carlos Montalbàn (Madrid, Spain), Franck Morschhauser (Lille, France), Nicolas Mounier (Nice, France), Urban Novak (Bern, Switzerland), Grzegorz Nowakowski (Rochester, USA), Davide Rossi (Bellinzona, Switzerland), Gilles Salles (New York, USA), Stephen Smith (Seattle, USA), Anasthasios Stathis (Bellinzona, Switzerland), Catherine Thieblemont (Paris, France), Hervé Tilly (Caen, France), Judith Trotman (Sydney, Australia). Côme Bommier is funded by Inserm/ITMO/Aviesan (FRFT‐DOC21) as part of his PhD program.
Publisher Copyright:
© 2022 John Wiley & Sons Ltd.
PY - 2022/12
Y1 - 2022/12
N2 - A recent systematic review reported that trials involving patients with marginal zone lymphoma (MZL) show marked heterogeneity both in the choice and definitions of primary and secondary endpoints, thus hampering comparability between trials. The main objective of this study was to reach consensus, through a Delphi process, on the definitions of four time-to-event endpoints in MZL trials, by surveying clinicians and methodologists involved in the conduct of clinical trials including patients with MZL. We polled a panel of leading international experts involved in MZL trials by means of self-administered sequential questionnaires in 2021. Of these 105 experts, 62 responded to the Round 1 questionnaire regarding the definitions of progression-free survival (PFS), event-free survival (EFS), time-to-failure (TTF), and time-to-next-treatment (TTNT). Afterward, we therefore focused the Round 2 and 3 questionnaires among principal investigators, coinvestigators, and trial methodologists. Consensus was reached when there was a >80% agreement on all potential events (11 choices) of each endpoint. Participants in our survey reached consensus on three of the four time-to-event endpoints definitions. Consensus was reached on the definitions of PFS and TTNT after Round 1, of TTF after Round 2, and was not reached for EFS after Round 3. The disagreement concerned the event “treatment discontinuation” in EFS definition. The main interest of our study was to elicit investigator's interest in the importance of consistently defining endpoints in MZL trials and to highlight that composite endpoints should not be encouraged. Fifteen years after the last consensus statement on time-to-event endpoints definitions issued in Lugano (2007), both the review of literature and survey of international investigators agree on the inconsistency of endpoints definitions used within the MZL community. Hopefully, revised standardized definitions of endpoints shall be provided at the upcoming International Conference on Malignant Lymphoma in 2023.
AB - A recent systematic review reported that trials involving patients with marginal zone lymphoma (MZL) show marked heterogeneity both in the choice and definitions of primary and secondary endpoints, thus hampering comparability between trials. The main objective of this study was to reach consensus, through a Delphi process, on the definitions of four time-to-event endpoints in MZL trials, by surveying clinicians and methodologists involved in the conduct of clinical trials including patients with MZL. We polled a panel of leading international experts involved in MZL trials by means of self-administered sequential questionnaires in 2021. Of these 105 experts, 62 responded to the Round 1 questionnaire regarding the definitions of progression-free survival (PFS), event-free survival (EFS), time-to-failure (TTF), and time-to-next-treatment (TTNT). Afterward, we therefore focused the Round 2 and 3 questionnaires among principal investigators, coinvestigators, and trial methodologists. Consensus was reached when there was a >80% agreement on all potential events (11 choices) of each endpoint. Participants in our survey reached consensus on three of the four time-to-event endpoints definitions. Consensus was reached on the definitions of PFS and TTNT after Round 1, of TTF after Round 2, and was not reached for EFS after Round 3. The disagreement concerned the event “treatment discontinuation” in EFS definition. The main interest of our study was to elicit investigator's interest in the importance of consistently defining endpoints in MZL trials and to highlight that composite endpoints should not be encouraged. Fifteen years after the last consensus statement on time-to-event endpoints definitions issued in Lugano (2007), both the review of literature and survey of international investigators agree on the inconsistency of endpoints definitions used within the MZL community. Hopefully, revised standardized definitions of endpoints shall be provided at the upcoming International Conference on Malignant Lymphoma in 2023.
KW - Delphi
KW - event-free survival
KW - progression-free survival
KW - time-to-event endpoints
KW - time-to-failure
KW - time-to-next-treatment
UR - http://www.scopus.com/inward/record.url?scp=85127420355&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85127420355&partnerID=8YFLogxK
U2 - 10.1002/hon.2988
DO - 10.1002/hon.2988
M3 - Letter
C2 - 35299287
AN - SCOPUS:85127420355
SN - 0278-0232
VL - 40
SP - 1086
EP - 1089
JO - Hematological Oncology
JF - Hematological Oncology
IS - 5
ER -