10 Dec How imaging core labs drive success in malignant hematology trials
Malignant Hematology is the term that refers to blood cancers, like leukemia, lymphoma, and multiple myeloma. According to research published in The Lancet, hematological malignancies account for around 6.6% of total cancer cases and 7.2% of total cancer-related deaths, projected to reach almost 5 million worldwide by the year 2030. Hematological malignancies often require close monitoring and intensive therapy, and despite a range of best-in-class and advanced targeted therapies becoming available over the last decade, high rates of recurrence remain a challenge.
Pharmaceutical companies continue to drive innovation in this therapeutic area, leveraging the ongoing accumulation of scientific understanding, but given that hematological oncology trials are fundamentally different from solid tumor studies, trial design and trial execution require expert guidance. Small patient populations, heterogeneous disease biology, rapidly evolving standards of care, and ethical considerations contribute to the need for careful consideration when designing and running these clinical trials.
Imaging core labs allow pharmaceutical companies to coordinate and generate robust imaging and clinical data, improving the reliability of response assessment in complex hematologic malignancies. This article considers the unique challenges of malignant hematology research, highlighting how imaging core labs can drive success in malignant hematology trials.
Milestone approvals in malignant hematology
The therapeutic landscape of malignant hematology treatments has experienced exponential growth over the last decade, with milestone approvals branching across chimeric antigen receptor T (CAR-T) therapies, bispecific T cell engagers, small-molecule inhibitors, antibody-drug conjugates, and recombinant immunotoxins. A systematic review covering the last decade of FDA-approved drugs for hematological malignancies evaluates 52 different drug approvals from 2011 to 2021.
Given this growth, it is no surprise that global biopharmaceutical companies are showing increasing interest in this area of research, which has seen significant market growth aligned with advancements in therapeutic approaches. A diverse patient population combined with potential high-value treatments leads to investment and results in novel drug approvals, providing hope for those living with malignant hematology disorders. With that being said, malignant hematology trials are inherently complex, requiring extensive therapeutic area expertise, infrastructure, and understanding to achieve success.
Challenges unique to malignant hematology trials
Sponsors must take advantage of the infrastructure and principles of solid tumor trials, while developing study designs, endpoints, and recruitment procedures reflective of a much smaller, more nuanced cohort of patients with blood-based cancers.
Challenges unique to malignant hematology trials include:
- Low disease incidence and a rare patient population
- Highly specialized and limited investigator pool
- Complex and evolving disease classifications
- Rapidly changing treatment landscape
- Unique and intricate endpoints
- First-in-class therapies and site inexperience
- Data complexity and interpretation challenges
- Global variability in standards of care
- Burdensome procedures impacting patient recruitment
- Operational challenges at community sites
Malignant hematology imaging modalities
Imaging plays an important role in malignant hematology trials, providing early insight into treatment response and helping to quantify disease burden. Importantly, imaging is disease-specific, and must be aligned with the underlying disease biology. FDG-PET/CT imaging, for example, provides researchers with various ways to capture treatment effect, including maximum standardized uptake value, and is commonly used to evaluate non-Hodgkin lymphomas. MRI can capture lymph node involvement, marrow infiltration, and CNS involvement, particularly valuable in multiple myeloma. While CT provides a foundational assessment across malignant disease, it can also help to quantify lymph node size, organ involvement, and structural changes to evaluate therapeutic response.
Common malignant hematology imaging modalities include:
- Positron Emission Tomography (PET)
- Computed Tomography (CT)
- Magnetic Resonance Imaging (MRI)
While these modalities offer valuable insight when applied correctly, they come with limitations. Many hematologic malignancies are diffuse rather than localized, while some present with focal lesions, this heterogeneity affects imaging selection and interpretation. Variability in acquisition, reconstruction, and reader interpretation can alter trial outcomes, and imaging data must be integrated early into the protocol design phase to ensure interpretability and regulatory compliance.
Surrogate-based endpoints in malignant hematology
Traditional endpoints, such as overall survival, can take many years to evaluate, placing an increased emphasis on surrogate endpoints in malignant hematology trials. Compared to prognostic markers, surrogate endpoints can be measured early but can still capture the expected effect of an investigational therapy. That being said, not all surrogate endpoints can answer the same question, and if not properly validated, they can fail to accurately reflect the true clinical benefit of a treatment. The question must be asked, “Does the use of a clinically meaningful endpoint condemn the feasibility of the trial?”
Surrogate endpoints are becoming essential, and many studies rely on markers such as progression-free survival (PFS), disease-free or event-free survival, response rate, objective response rate, and time to progression to assess treatment response. Alongside this, a number of complex hematologic endpoints are often considered, including white blood cell, neutrophil, and blast counts, bone marrow assessments, and measurements of organs like the liver and spleen. Minimal residual disease (MRD) and molecular endpoints are also becoming increasingly important surrogate measures across leukemias, lymphomas, and multiple myeloma.
The challenge, however, is that imaging is often underestimated, even when it’s central to trial outcomes. Proper imaging data acquisition and central review are critical for generating robust, unbiased results that regulators can rely on, and must be integrated from the outset of clinical trial design.
How imaging core labs drive success in malignant hematology trials?
An experienced imaging core lab can be instrumental in the success of a malignant hematology trial. Imaging core labs provide centralized data collection, quality control, blinded independent central review, and standardized imaging and clinical data analysis, covering both traditional and surrogate imaging endpoints. Malignant hematology trials are becoming a competitive landscape for sponsors, who have to balance a relatively small cohort of patients, in comparison to solid tumor trials, and an even smaller pool of hematology specialist researchers and clinicians.
Perceptive Imaging, having supported over 370 malignant hematology trials and 70 regulatory approvals, has proven it can meet the challenges of malignant hematology trials head on. Perceptive leverages its well-established infrastructure, standardized processes, and network of expertise to overcome any challenges before they cause delays, ensuring consistent imaging quality, reliable assessments, and smoother trial operations across all regions.
Learn more about Perceptive Imaging and contact an imaging solutions specialist.
Resources
The Lancet. Global landscape and trends in lifetime risks of haematologic malignancies in 185 countries: population-based estimates from GLOBOCAN 2022. https://www.thelancet.com/journals/eclinm/article/PIIS2589-5370(25)00125-7/fulltext
Cancers. FDA-Approved Drugs for Hematological Malignancies-The Last Decade Review. https://pubmed.ncbi.nlm.nih.gov/35008250/
Blood. What clinicians should know about surrogate end points in hematologic malignancies. https://www.sciencedirect.com/science/article/pii/S0006497124009698
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Last Updated on December 10, 2025 by Marie Benz MD FAAD