Outcomes Measured in Multiple Myeloma Trials in the Past 10 Years: Focus on Minimal Residual Disease and Therapy Response
Author(s)
Mimi Choon-Quinones, MBA, PhD, LL.M1, George D. Obeng, MSc2, Tamas Agh, MSc, PhD, MD3, Attila Imre, MSc4, Benjamin Asiedu-Ayeh, BSc2, Sampson Kawuo, BSc2, Asmau Mohammed Tukur, MPhil2, Inusah Mohammed, MSc2, Akosua Pokuaah Obeng, MPhil2, Brian Durie, Prof5, Jean-Luc HAROUSSEAU, Prof6, Anja Seckinger, MD7, Dirk Hose, Prof7, Marcell Csanádi, MSc, PhD3;
1Partners For Patients NGO, Lugano, Switzerland, 2Syreon Research Africa, Accra, Ghana, 3Syreon Research Institute, Budapest, Hungary, 4Semmelweis University, Center for Health Technology Assessment, Budapest, Hungary, 5Cedars Sinai Outpatient Cancer Center, Los Angeles, CA, USA, 6Institut de Cancérologie de l'Ouest, Saint Herblain, France, 7Vrije Universiteit Brussel, Jette, Belgium
1Partners For Patients NGO, Lugano, Switzerland, 2Syreon Research Africa, Accra, Ghana, 3Syreon Research Institute, Budapest, Hungary, 4Semmelweis University, Center for Health Technology Assessment, Budapest, Hungary, 5Cedars Sinai Outpatient Cancer Center, Los Angeles, CA, USA, 6Institut de Cancérologie de l'Ouest, Saint Herblain, France, 7Vrije Universiteit Brussel, Jette, Belgium
OBJECTIVES: Multiply myeloma (MM) treatment has advanced over the last decades, leading to unprecedented response-rates and depth. Deep responses quantifiable as “minimal residual disease” (MRD) correlate, especially if prolonged over time, with longer progression-free and overall-survival. According to FDA and EMA, there is a need for standardizing MRD measurement in the trials’ eco-space and establishing consistent thresholds for clinical decision-making. Here, we aimed to provide an overview of how MRD and therapy response were assessed in MM trials initiated over the past years.
METHODS: The database of clinicaltrials.gov was searched on the December 18th, 2024 for the condition of MM from January 1st 2014. No limitation was applied for interventions, locations or study status. The downloaded database was screened for trials specifically focusing on therapy for only MM. Data were collected on MRD and therapy response related to outcome definition and type of measurement.
RESULTS: After screening the 1952 downloaded trials, 1258 investigated specifically MM treatment, and among these 1139 were interventional study. Therapy response was assessed in 978 interventional trials (85.9%), while 330 (29.0%) assessed MRD. For completed interventional trials MRD was measured in 15.1%, for active trials it was 42.4%, while for withdrawn/terminated ones it was 11.3%. MRD was more commonly assessed in the past 5 years (39.2% of interventional trials), compared to the previous period (17.4%). In trials, where MRD was assessed, the technology used for the assessment was reported only in 46.4% (153/330). Among these 153 trials, next-generation sequencing was used in 60.1%, flow cytometry in 53.6% and both methods in 13.7%.
CONCLUSIONS: Our study found that there is a significant increment in the number of trials investigating MRD and almost half of the ongoing ones include such outcomes. The growing adoption of MRD assessment reflects its increasing importance in evaluating therapeutic efficacy and predicting patient outcomes.
METHODS: The database of clinicaltrials.gov was searched on the December 18th, 2024 for the condition of MM from January 1st 2014. No limitation was applied for interventions, locations or study status. The downloaded database was screened for trials specifically focusing on therapy for only MM. Data were collected on MRD and therapy response related to outcome definition and type of measurement.
RESULTS: After screening the 1952 downloaded trials, 1258 investigated specifically MM treatment, and among these 1139 were interventional study. Therapy response was assessed in 978 interventional trials (85.9%), while 330 (29.0%) assessed MRD. For completed interventional trials MRD was measured in 15.1%, for active trials it was 42.4%, while for withdrawn/terminated ones it was 11.3%. MRD was more commonly assessed in the past 5 years (39.2% of interventional trials), compared to the previous period (17.4%). In trials, where MRD was assessed, the technology used for the assessment was reported only in 46.4% (153/330). Among these 153 trials, next-generation sequencing was used in 60.1%, flow cytometry in 53.6% and both methods in 13.7%.
CONCLUSIONS: Our study found that there is a significant increment in the number of trials investigating MRD and almost half of the ongoing ones include such outcomes. The growing adoption of MRD assessment reflects its increasing importance in evaluating therapeutic efficacy and predicting patient outcomes.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
CO198
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment, Clinician Reported Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Oncology