ESMO-Magnitude of Clinical Benefit Scale for haematological malignancies (ESMO-MCBS:H) version 1.0

B Kiesewetter; U Dafni; E G E de Vries; J Barriuso; G Curigliano; V González-Calle; M Galotti; B Gyawali; B J P Huntly; U Jäger; N J Latino; L Malcovati; S F Oosting; G Ossenkoppele; M Piccart; M Raderer; L Scarfò; D Trapani; C C Zielinski; R Wester; P Zygoura; E Macintyre; N I Cherny; ESMO-MCBS Working Group and Extended Working Group

doi:10.1016/j.annonc.2023.06.002

ESMO-Magnitude of Clinical Benefit Scale for haematological malignancies (ESMO-MCBS:H) version 1.0

Ann Oncol. 2023 Sep;34(9):734-771. doi: 10.1016/j.annonc.2023.06.002. Epub 2023 Jun 19.

Authors

B Kiesewetter¹, U Dafni², E G E de Vries³, J Barriuso⁴, G Curigliano⁵, V González-Calle⁶, M Galotti⁷, B Gyawali⁸, B J P Huntly⁹, U Jäger¹⁰, N J Latino⁷, L Malcovati¹¹, S F Oosting³, G Ossenkoppele¹², M Piccart¹³, M Raderer¹, L Scarfò¹⁴, D Trapani⁵, C C Zielinski¹⁵, R Wester¹⁶, P Zygoura¹⁷, E Macintyre¹⁸, N I Cherny¹⁹; ESMO-MCBS Working Group and Extended Working Group

Affiliations

¹ Division of Oncology, Department of Medicine I, Medical University of Vienna, Vienna, Austria.
² Laboratory of Biostatistics, School of Health Sciences, National and Kapodistrian University of Athens, Athens; Frontier Science Foundation-Hellas, Athens, Greece.
³ Department of Medical Oncology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
⁴ The Christie NHS Foundation Trust and Division of Cancer Sciences, School of Medical Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK.
⁵ European Institute of Oncology, IRCCS, Division of Early Drug Development, Milan; Department of Oncology and Hemato-Oncology, University of Milano, Milan, Italy.
⁶ Servicio de Hematología, Hospital Universitario de Salamanca-IBSAL, CIBERONC and Centro de Investigación del Cáncer-IBMCC (USAL-CSIC), Salamanca, Spain.
⁷ ESMO Head Office, Lugano, Switzerland.
⁸ Departments of Oncology, Oncology; Public Health Sciences, Queen's University, Kingston; Division of Cancer Care and Epidemiology, Queen's University, Kingston, Canada.
⁹ Cambridge Stem Cell Institute, Department of Haematology, University of Cambridge & Cambridge University Hospitals, Cambridge, UK.
¹⁰ Department of Medicine I, Clinical Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
¹¹ Department of Molecular Medicine, University of Pavia, Pavia; Department of Hematology Oncology, IRCCS S. Matteo Hospital Foundation, Pavia, Italy.
¹² Department of Haematology, VU University Medical Center, Amsterdam, The Netherlands.
¹³ Institut Jules Bordet, Hôpital Universitaire de Bruxelles (HUB), Université Libre de Bruxelles (ULB), Brussels, Belgium.
¹⁴ Strategic Research Program on CLL, Università Vita-Salute San Raffaele and IRCCS Ospedale San Raffaele, Milan, Italy.
¹⁵ Wiener Privatklinik, Central European Academy Cancer Center, Vienna, Austria.
¹⁶ Department of Hematology, Erasmus MC Cancer Institute, Rotterdam, The Netherlands.
¹⁷ Frontier Science Foundation-Hellas, Athens, Greece.
¹⁸ Onco-hématologie Biologique, AP-HP, Necker-Enfants Malades Hospital, Paris; Université Paris Cité, INSERM, CNRS, INEM F-75015, Paris, France.
¹⁹ Cancer Pain and Palliative Medicine Service, Department of Medical Oncology, Shaare Zedek Medical Center, Jerusalem, Israel. Electronic address: [email protected].

PMID: 37343663
DOI: 10.1016/j.annonc.2023.06.002

Abstract

Background: The European Society for Medical Oncology (ESMO)-Magnitude of Clinical Benefit Scale (MCBS) has been accepted as a robust tool to evaluate the magnitude of clinical benefit reported in trials for oncological therapies. However, the ESMO-MCBS hitherto has only been validated for solid tumours. With the rapid development of novel therapies for haematological malignancies, we aimed to develop an ESMO-MCBS version that is specifically designed and validated for haematological malignancies.

Methods: ESMO and the European Hematology Association (EHA) initiated a collaboration to develop a version for haematological malignancies (ESMO-MCBS:H). The process incorporated five landmarks: field testing of the ESMO-MCBS version 1.1 (v1.1) to identify shortcomings specific to haematological diseases, drafting of the ESMO-MCBS:H forms, peer review and revision of the draft based on re-scoring (resulting in a second draft), assessment of reasonableness of the scores generated, final review and approval by ESMO and EHA including executive boards.

Results: Based on the field testing results of 80 haematological trials and extensive review for feasibility and reasonableness, five amendments to ESMO-MCBS were incorporated in the ESMO-MCBS:H addressing the identified shortcomings. These concerned mainly clinical trial endpoints that differ in haematology versus solid oncology and the very indolent nature of nevertheless incurable diseases such as follicular lymphoma, which hampers presentation of mature data. In addition, general changes incorporated in the draft version of the ESMO-MCBS v2 were included, and specific forms for haematological malignancies generated. Here we present the final approved forms of the ESMO-MCBS:H, including instructions.

Conclusion: The haematology-specific version ESMO-MCBS:H allows now full applicability of the scale for evaluating the magnitude of clinical benefit derived from clinical studies in haematological malignancies.

Keywords: ESMO-MCBS; clinical benefit; clinical trials; haematological malignancies; quality of life; value frameworks.

MeSH terms

Antineoplastic Agents* / therapeutic use
Hematologic Neoplasms* / therapy
Humans
Lymphoma, Follicular* / drug therapy
Medical Oncology
Neoplasms* / drug therapy
Societies, Medical

Substances

Antineoplastic Agents