Prapti A. Patel, Yazan F. Madanat, Andrew J. Belli, Eric Hansen, Heidi Foss, Molly Schulte, Ching-Kun Wang
Research matters
When you’re focusing on the future of cancer care, it helps to start with a clear and accurate picture of the present.
Ben A Derman, Andrew J. Belli, Ching-Kun Wang, Eric Hansen, Spencer S Langerman, Andrzej Jakubowiak, Brian C-H Chiu
Multiple myeloma (MM) risk stratification schemata such as the International Staging System (ISS) and Revised-ISS (R-ISS) were derived from clinical trial subjects made up predominately of younger White individuals with adequate renal function. It is unknown whether these prognostic indices are applicable to all patients with newly diagnosed (ND) MM, especially among Black individuals, older adults, and those with renal dysfunction. The R-ISS expanded on the ISS by including and serum lactate dehydrogenase (LDH) and high-risk cytogenetic abnormalities (HRCA) identified by fluorescence in-situ hybridization (FISH), but HRCA may not translate into poor prognosis for older adults and for Black individuals. We sought to create an inclusive risk prognostic index for NDMM using real-world data derived from electronic health records.
Ankit Kansagra, Eric Hansen, Andrew J. Belli, Stefanie Goran, Ching-Kun Wang
Multiple myeloma (MM) is a heterogeneous disease with wide variability in outcomes. The presence of cytogenetic abnormalities in MM is of critical importance for prognosis and risk stratification. However, patients who may or may not have sufficient cytogenetic abnormalities to classify as high-risk can still experience rapid disease progression despite therapy, or functional high risk (FHR) disease. These two high risk cohorts comprise vulnerable subpopulations who have a significant burden of disease, and it is critical that we understand the underlying patient characteristics and optimal treatment sequence. We sought to investigate these two high risk patient populations treated in the contemporary real-world practice setting.
Sikander Ailawadhi, Ching-Kun Wang, Andrew J. Belli, Catarina Jansson Blixt, Diana Cripps, Stojan Zavisic, Karthik Ramasamy
Melphalan flufenamide (melflufen) is a first-in-class peptide-drug conjugate that targets aminopeptidases and rapidly releases alkylating agents into tumor cells. The single-arm, open-label, Phase 2 HORIZON study (NCT02963493), conducted in Europe and the USA, demonstrated in patients with heavily pre-treated relapsed/refractory multiple myeloma (RRMM), including those with triple-class-refractory and extramedullary disease, that melflufen in combination with dexamethasone showed clinically meaningful efficacy and a manageable safety profile (Richardson PG, et al. J Clin Oncol. 2021;39:757-767). Comparative trials between all regimens/agents are not feasible, and hence, real-world datasets may be valuable to allow the comparison of patient outcomes in single-arm clinical trials with similar patient populations in the real world. The objective of the REAL-world Myeloma (REALM) study was to compare clinical outcomes between patients in the COTA database and those of patients in the HORIZON study in order to compare routine care with melflufen.
Feng Wang, Boris Gorsh, Maral DerSarkissian, Prani Paka, Rachel Bhak, Natalie Boytsov, Miriam L. Zichlin, Leah Sansbury, Christopher W. Yee, Shannon Ferrante, Anamika Khanal, Ahmed Noman, Mei Sheng Duh
Patients with relapsed/refractory multiple myeloma (RRMM) resistant to multiple drug classes remain a high unmet need population, despite advances in MM patient care. The objective of this study was to assess real-world treatment patterns and outcomes in patients with RRMM who had failed multiple prior lines of therapy (LOT) and were refractory to multiple drug classes to identify gaps in their treatment pathways.
Donna R. Rivera, Henry J. Henk, Elizabeth Garrett-Mayer, Jennifer B. Christian, Andrew J. Belli, Suanna S. Bruinooge, Janet L. Espirito, Connor Sweetnam, Monika A. Izano, Yanina Natanzon, Nicholas J. Robert, Mark S. Walker, Aaron B. Cohen, Marley Boyd, Lindsey Enewold, Eric Hansen, Rebecca Honnold, Lawrence Kushi, Pallavi S. Mishra Kalyani, Ruth Pe Benito, Lori C. Sakoda, Elad Sharon, Olga Tymejczyk, Emily Valice, Joseph Wagner, Laura Lasiter, Jeff D. Allen
The purpose of this study was to evaluate the potential collective opportunities and challenges of transforming real-world data (RWD) to real-world evidence for clinical effectiveness by focusing on aligning analytic definitions of oncology end points. Patients treated with a qualifying therapy for advanced non-small cell lung cancer in the frontline setting meeting broad eligibility criteria were included to reflect the real-world population. Although a trend toward improved outcomes in patients receiving PD-(L)1 therapy over standard chemotherapy was observed in RWD analyses, the magnitude and consistency of treatment effect was more heterogeneous than previously observed in controlled clinical trials. The study design and analysis process highlighted the identification of pertinent methodological issues and potential innovative approaches that could inform the development of high-quality RWD studies.
Laura Lasiter, Olga Tymejczyk, Elizabeth Garrett-Mayer, Shrujal Baxi, Andrew J. Belli, Marley Boyd, Jennifer B. Christian, Aaron B. Cohen, Janet L. Espirito, Eric Hansen, Connor Sweetnam, Nicholas J. Robert, Mackenzie Small, Mark D. Stewart, Monika A. Izano, Joseph Wagner, Yanina Natanzon, Donna R. Rivera, Jeff Allen
In prior work, Friends of Cancer Research convened multiple data partners to establish standardized definitions for oncology real-world end points derived from electronic health records (EHRs) and claims data. Here, we assessed the performance of real-world overall survival (rwOS) from data sets sourced from EHRs by evaluating the ability of the end point to reflect expected differences from a previous randomized controlled trial across five data sources, after applying inclusion/exclusion criteria. The KEYNOTE-189 clinical trial protocol of platinum doublet chemotherapy (chemotherapy) vs. programmed cell death protein 1 (PD-1) in combination with platinum doublet chemotherapy (PD-1 combination) in first-line nonsquamous metastatic non-small cell lung cancer guided retrospective cohort selection. The Kaplan-Meier product limit estimator was used to calculate 12-month rwOS with 95% confidence intervals (CIs) in each data source. Cox proportional hazards models estimated hazard ratios (HRs) and associated 95% CIs, controlled for prognostic factors. Once the inclusion/exclusion criteria were applied, the five resulting data sets included 155 to 1,501 patients in the chemotherapy cohort and 36 to 405 patients in the PD-1 combination cohort. Twelve-month rwOS ranged from 45% to 58% in the chemotherapy cohort and 44% to 68% in the PD-1 combination cohort. The adjusted HR for death ranged from 0.80 (95% CI: 0.69, 0.93) to 1.15 (95% CI: 0.71, 1.85), controlling for age, gender, performance status, and smoking status. This study yielded insights regarding data capture, including ability of real-world data to precisely identify patient populations and the impact of criteria on end points. Sensitivity analyses could elucidate data set–specific factors that drive results.
Ankit Kansagra, Benjamin Derman, Andrew Belli, Eric Hansen, Stefanie Goran, Ching-Kun Wang
African Americans (AA) are disproportionately affected by multiple myeloma (MM) with over double the incidence and mortality rates as compared to white patients. Although there have been significant therapeutic advances for the treatment of MM over the last 20 years, research has suggested that these benefits may not be uniform across racial groups. We sought to explore racial disparities in multiple myeloma using contemporary real-world data (RWD).
Peter Feng Wang, Leah Sansbury, Maral DerSarkissian, Shannon Ferrante, Rachel Bhak, Boris Gorsh, Miriam L. Zichlin, Christopher W. Yee, Natalie Boytsov, Anamika Khanal, Prani Paka, Ahmed Noman, Mei Sheng Duh
Real-world data on treatment patterns and outcomes in patients with RRMM resistant to multiple drug classes are limited.
X. Le, J. Martinalbo, A. Holynskyj, W.C. Rhodes, W-H. Wu, J. Kim, V. Pretre, F. Ye, J. Morrissette
Tx options for METex14 NSCLC have been limited to chemotherapy (Ctx), immunotherapy (IO) and multikinase inhibitors (MKi) until the FDA approvals of selective MET inhibitors (METi) from 2020. Timing of NGS testing in relation to systemic first-line Tx (1L) initiation and Tx selection patterns for METex14 NSCLC pts in the real-world prior to those approvals warrants evaluation to guide future practice.
