The HARMONY Alliance is a Public-Private Partnership for Big Data in Hematology including over 100 organizations such as European medical associations, hospitals, research institutes, patient organizations, pharmaceutical and IT companies. HARMONY uses Big Data analytics to accelerate the development of more effective treatments for blood cancer patients. The HARMONY Alliance is funded by the Innovative Medicines Initiative (IMI) (now called Innovative Health Initiative, IHI) of the European Commission and is currently running two projects: HARMONY (January 2017-June 2023) and HARMONY PLUS (October 2020-September 2023). In addition to the research projects that exploit the HARMONY Big Data Platform, the HARMONY Alliance is running Delphi surveys to develop core outcome sets (COS) for HMs, as well as Health Technology Assessment projects and multi-stakeholder activities. Read more at: https://www.harmony-alliance.eu/
Various groups participate in HARMONY, including EMN. HARMONY Research Project Leads and Key Opinion Leaders in Multiple Myeloma are Prof. Pieter Sonneveld, Prof. Mario Boccadoro, and Prof. Jesus San-Miguel.
An important Multiple Myeloma project is being coordinated by the EMN within HARMONY, and it involves many European clinical centers. The title of the is: “SECOND REVISION OF THE INTERNATIONAL STAGING SYSTEM (R2-ISS) FOR MULTIPLE MYELOMA”, which is being conducted through the HARMONY BigData Platform.
EMN PROJECT COORDINATOR: Dr. Mattia D’Agostino, Torino, Italy
CONTRIBUTING COUNTRIES & GROUPS: Study centers in locations including Italy, Spain, The Netherlands, Nordic countries, Germany and the UK have provided data for incorporation into the MM database. The conduct of a study with such a large number of patients with MM is unprecedented in Europe.
BACKGROUND: The clinical outcome of patients with newly diagnosed Multiple Myeloma is heterogeneous with wide-ranging survival times. The Revised-International Staging System was developed in 2015 (Palumbo et al. J Clin Oncol. 2015;33(26):2863-9) to stratify subgroups of patients with MM with differing prognosis and survival. However, this tool has only partly guided therapeutic choices and, according to it, 62% of patients were classified into the intermediate-risk category (R-ISS II), possibly including patients with different risk levels of progression/death. Moreover, in the R-ISS, high-risk cytogenetic abnormalities were considered as present if at least one among del(17p), t(4;14), or t(14;16) was detected, while emerging data showed that having more than one high-risk cytogenetic abnormality predicts poorer outcomes.
AIMS: Validate and/or further improve the Revised-International Staging System for use in patients with Multiple Myeloma; Compare different therapeutic approaches in different groups of patients according to the prognostic group.
PROJECT DESCRIPTION: The EMN, under the European Union-funded HARMONY project, collected individual patient data from a large cohort of 10843 young and elderly patients with newly diagnosed Multiple Myeloma to improve risk stratification and propose a revision of the current R-ISS (R2-ISS). In this project, the EMN analyzed the prognostic value of each single baseline risk feature in an additive fashion, including 1q gain/amplification in the risk calculation. An additive scoring system based on the top features predicting progression-free survival and overall survival was developed and validated.
RESULTS: Official data and the new score were published on JCO on 23 May, 2022. Briefly, in the training set (N = 7,072), at a median follow-up of 75 months, ISS, del(17p), lactate dehydrogenase, t(4;14), and 1q+ had the highest impact on PFS and OS. These variables were all simultaneously present in 2,226 patients. A value was assigned to each risk feature according to their OS impact (ISS-III 1.5, ISS-II 1, del(17p) 1, high lactate dehydrogenase 1, and 1q+ 0.5 points). Patients were stratified into four risk groups according to the total additive score: low (Second Revision of the International Staging System [R2-ISS]-I, 19.2%, 0 points), low-intermediate (II, 30.8%, 0.5-1 points), intermediate-high (III, 41.2%, 1.5-2.5 points), high (IV, 8.8%, 3-5 points). Median OS was not reached versus 109.2 versus 68.5 versus 37.9 months, and median PFS was 68 versus 45.5 versus 30.2 versus 19.9 months, respectively. The score was validated in an independent validation set (N = 3,771, of whom 1,214 were with complete data to calculate R2-ISS) maintaining its prognostic value.
In conclusion, the R2-ISS is a simple prognostic staging system allowing a better stratification of patients with intermediate-risk NDMM. The additive nature of this score fosters its future implementation with new prognostic variables.