ALL is a rare disease in adults. As a result, large series of homogeneously treated patients with accurate molecular and minimal residual disease (MRD) data are scarce. IKZF1 alterations are common (20%-30%) in adult ALL, but their prognostic significance remains controversial. HARMONY’s ALL team is preparing a research proposal to bring together well-annotated datasets with genetic, MRD, and follow-up data from around 1,400 adult BCP ALL patients treated in clinical trials from eight study groups. These data will be used to answer the following research questions:
Event-free survival (EFS), complete response (CR), and minimal residual disease (MRD) may be used as surrogates for overall survival (OS) in clinical trials. This may improve clinical development and trial efficiency, and accelerate the drug approval process. However, proper surrogate endpoint evaluation requires multiple trials and a reasonably-sized database. HARMONY’s AML team is preparing a research proposal to address the following research questions:
Some CLL patients develop other types of neoplasia after CLL treatment, which may or may not be the result of the therapy. HARMONY’s CLL team is preparing a research proposal to study this in more detail, addressing the following aspects:
In the longer term, this should facilitate the development of screening methods for specific cancer types in CLL patients and methods to identify patients at risk.
Most of our current knowledge about the treatment of MM is based on clinical trials. However, less than 5% of cancer patients enroll in clinical trials and data on treatments and outcomes in the remaining 95% patients are limited. HARMONY’s MM team is preparing a research proposal to identify predictors of initial response duration in a trial setting and then validate these predictors in real-world data. They will focus on patient characteristics such as stage, cytogenetics, comorbidities, and demographics. In addition, they plan to assess treatment sequencing and long-term outcomes in MM using real-world data.
NHLs represent a highly heterogeneous group of lymphoid malignancies. HARMONY’s NHL team has selected several subtypes of NHL for in-depth studies, including Diffuse Large B-cell Lymphomas (DLBCL). DLBCL is a particularly aggressive subtype that can only be cured in approximately 50% of patients. It also is the most common subtype of NHL. The major challenge in DLBCL is to improve survival of patients with refractory disease or patients who relapse early in the course of the lymphoma. At present, HARMONY’s NHL team is preparing a research proposal to address the following topics: