Research

Empowering Healthcare with Real-World Evidence

Dive into our repository of groundbreaking research papers, where we merge clinical expertise with data driven insights to revolutionize patient care and treatment strategies.

Group 79
Sharlene Dong MD, Ankit J. Kansagra MD, Gurbakhash Kaur MDMA, Anna Barcellos MPH, Andrew J. Belli MPH, Laura L. Fernandes PhD, Eric Hansen MS, Jacob Ambrose MS, Claire Bai MS, Christina M. Zettler, Ching-Kun Wang MD
Real-world data (RWD) is being increasingly used to support clinical and regulatory decisions where utilization of clinical trial data is difficult or impractical. With this expanded utilization comes a need to ensure RWD is of sufficient quality and completeness to adequately answer relevant research questions. Given the importance of overall survival (OS) for oncology research, treatment and care, complete and accurate real-world mortality data capture is of critical importance. Currently the industry standard practice aggregates mortality data across available data sources to maximize data capture. This study sought to validate and benchmark a composite real-world mortality variable against the gold standard data source from the National Death Index (NDI).
Luciano Costa MDPhD, Thomas W LeBlanc MD, Hans Tesch MD, Pieter Sonneveld MD PhD, Ryan Kyle, Liliya Sinyavskaya, Patrick Hlavacek MPH, Aster Meche, Jinma Ren, Alex Schepart PharmD, Didem Aydin MD PhD, Guido Nador MD, Marco DiBonaventura PhD
Elranatamab(ELRA) is a BCMAxCD3 bispecific antibody being investigated for the treatment of relapsed/refractory multiple myeloma (MM). The phase 2 MagnetisMM-3 (MM-3; NCT04649359) trial was single-armed; the aim of this study was to contextualize the efficacy data from MM-3 with two real-world (RW) external control arms.
Muhammad Saad Hamid MD, Claire Bai MS, Chelsea Perelgut BSN RN, Helena Yelovich MD, Christina M. Zettler, Laura L. Fernandes PhD, Andrew J. Belli MPH, Eric Hansen MS, Ching-Kun Wang MD
Follicular lymphoma (FL) accounts for about 35% of all non-Hodgkin lymphomas and 70% of indolent lymphomas in the United States. Despite relatively high 1L remission rates, a clinically high-risk subgroup experiences early disease progression within 24 months of 1L initiation (POD24). As the understanding of this population evolves, a treatment paradigm is yet to be established. This study aimed to describe the characteristics of patients who experienced POD24 to 1L therapy and their outcomes following receipt of select 2L treatment sequences using a contemporary real-world (rw) dataset.
Joshua F. Zeidner MD, Priyanka Mehta MDFRCPath MRCP, Benyam Muluneh, Sarah Bertoli, Pierre-Yves Dumas MDPhD, Arnaud Pigneux MD PhD, Cédric Fernandez, Hélène Derrien, Christian Recher MD PhD
Intensive chemotherapy (IC) remains the main backbone therapy in fit, newly diagnosed, acute myeloid leukemia (AML) patients. Clinical trials assessing the efficacy of IC under-represent select subgroups of patients, such as those with certain comorbidities, unique characteristics, or rare genomic abnormalities. Real-world data provides additional insights into treatment patterns and clinical outcomes in patients with AML receiving first-line IC. REAL-IDH is an international network that generates evidence to improve real-world understanding of AML, including patients with a mutation in the gene encoding isocitrate dehydrogenase 1 (m IDH1). This abstract presents data from the REAL-IDH study, specifically on treatment patterns and outcomes in newly diagnosed AML patients who were treated with IC, including those with or without m IDH1 disease.
Rebecca Bystrom MD, Laura L. Fernandes PhD, Joseph Wynne MDPhD, Dianne Pulte MD, Eric Hansen MS, Andrew J. Belli MPH, Anna Barcellos MPH, Christina M. Zettler, Jonathon Vallejo PhD, Kelly Norsworthy MD, Angelo De Claro MD, Catherine Lerro PhD, Marc R Theoret MD, Ching-Kun Wang MD, Donna Rivera MS PharmD
A unique subgroup of AML is defined by the presence of the TP53 mutation, with worse overall survival (OS) and limited therapeutic options (Döhner et al, Blood 2022; Daver et al, J Hematol Oncol 2023). Clinical trials are essential for establishing the efficacy and safety of new treatment options in AML; yet, they may not be generalizable to patients with TP53 mutation, who are often older adults with comorbidities and prior treatment exposure. Thus, the optimal treatment for TP53 mutated AML is unclear. Real-world data (RWD) can provide insights into demographic and clinical characteristics, as well as the utilization, effectiveness, and safety of treatment regimens used in routine clinical practice. This RWD study aimed to describe the characteristics, first line treatment patterns, and outcomes in patients with ND-AML with TP53 mutation in a real-world cohort of adult patients in the US.
Tycel Philips MD, Laurie H. Sehn MD MPH, Anthony Wang MPH, PhD, Junhua Yu PhD, Elizabeth H. Marchlewicz PhD, Rajesh Kamalakar MS, Kavita Sail PhD, Donald Arnette PhD, Shibing Yang PhD, Alex Mutebi PhD, Fernando Rivas Navarro MD, Gilles Salles MD PhD
Follicular lymphoma (FL) is the most common indolent subtype of non-Hodgkin lymphoma. Despite the generally indolent nature of the condition, there are subgroups of FL patients who may not have an indolent experience, with their disease not responding to multiple lines of therapy. Thus, optimization of novel therapy could improve patient outcomes. This analysis examined treatment patterns, overall response rates (ORR), and complete response (CR) rates by line of therapy (LOT) in relapsed/refractory (R/R) FL in third-line or later (3L+) therapy.
Ralph Boccia, Hong Xiao, Dimana Miteva, Min Che, Tao Gu PhD, Ali McBride PharmD, Mrudula B. Glassberg, Aylin Yucel
Myelodysplastic syndromes (MDS) are a group of rare, clonal bone marrow malignancies characterized by ineffective erythropoiesis. Erythropoiesis-stimulating agents (ESAs) are a treatment option for anemia in patients with lower-risk MDS (LR-MDS); however as only one in three patients respond to ESAs, there is a need for additional treatment options. In April 2020, luspatercept was approved for the treatment of anemia in adult patients with LR-MDS who failed to respond to ESAs. The aim of this study was to investigate real-world treatment patterns and early clinical outcomes in adult patients in the United States with LR-MDS receiving luspatercept.
Matthew S. Davids MD MMSc, Jacob Ambrose MS, Enrico De Nigris MSc, Jennifer Prescott, Siyang Leng MD MS, Mohammed Z.H. Farooqui DO, Shravanthi R. Gandra PhD MBA, Christina M. Zettler, Laura L. Fernandes PhD, Ching-Kun Wang MD, Mazyar Shadman MD MPH
The chronic lymphocytic leukemia (CLL) treatment landscape is rapidly evolving, and real-world (rw) outcomes of patients receiving 2L+ therapy in the current treatment paradigm are incompletely understood. This study examined the rw outcomes of patients with CLL/ small lymphocytic lymphoma (SLL) receiving 2 or more lines of therapy (LOTs) using a large, contemporary rw dataset.
Adam Schoenfeld, Chen Hu, Ravi Rajaram, Josephine Feliciano, Urmila Chandran, Charlene Wong, U Hariharan, Iftekhar Kalsekar, Tianyi Wang, Qing Huang, Aisha Hasan
Immunotherapy with PD-(L)1-based strategies has rapidly transformed the treatment landscape for non-driver mutation metastatic non-small cell lung cancer (NSCLC). However, it is unclear how recent immunotherapy approvals have translated into real world practice patterns and patient outcomes. This study examined treatment patterns and attrition rates by line of therapy (LOT) among real-world patients with non-driver mutation metastatic NSCLC in the era of immunotherapy.
Hans C. Lee MD, Sundar Jagannath MD, Devender Dhanda PhD, Teofilia Acheampong MPH, PhD, Pallavi Patwardhan PhD BSPharm, Thomas S. Marshall PharmD, Suvina Amin MPH, Tao Gu PhD, Sikander Ailawadhi
With rapid treatment advances in multiple myeloma (MM), more patients receive the 3 main classes of MM treatments (immunomodulatory imide drug [IMiD] agents, proteasome inhibitors [PIs], and anti-CD38 monoclonal antibodies [mAbs]) in earlier lines of therapy and become TCE. This is a prerequisite for subsequent treatment with some novel therapies eg, anti-B-cell maturation antigen (BCMA) agents. Published evidence of RW clinical outcomes in patients with TCE MM is scarce in the evolving RRMM treatment landscape. The objective of this study was to assess RW clinical outcomes in patients with TCE RRMM who received treatment post-TCE.