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The Missing Link: Patient-reported Outcomes in Hematology Research

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Diego Adrianzen Herrera, MD, MSc, (@diegoah66)
Division of Hematology & Oncology, Larner College of Medicine, University of Vermont, Burlington, VT

As hematologists, we usually estimate the value of the therapies we offer through objective measures. We scan #ASH23 abstracts looking for remission rates, hemoglobin values, numbers of transfusions, and other empirical evidence. We perceive these outcomes as the measures by which we can define the effectiveness of a medical intervention. However, patients’ perspectives of their health and well-being are missing from these indicators, and we need to acknowledge that clinical staff and patients may have different perspectives on what outcomes are of most importance. As such, it has been argued that “the ultimate measure by which to judge the quality of a medical effort is whether it helps patients as they see it.”1  

The Food and Drug Administration (FDA) defines a patient-reported outcome (PRO) as “any report of the status of a patient’s health condition that comes directly from the patient without interpretation of the patient’s response by a clinician or anyone else.”2 PROs provide us with an assessment of effectiveness directly from those receiving medical interventions, filling in the blanks when it comes to how our clinical decisions can eventually impact our patients’ perception of their health. Accounting for PROs results in more cost-effective delivery of care and increases patient engagement. Several #ASH23 Oral and Poster Sessions offer important examples of how to measure and report PROs in clinical studies.  

In the session Multiple Myeloma: Clinical and Epidemiological: T Cell Redirecting Therapy Outcomes and Associated Complications, presented on December 9, Abstract 96 highlights immunotherapy-associated health-related quality of life (HRQoL) in patients with plasma cell neoplasms. Michel Delforge, MD, PhD, and colleagues utilized four validated QoL questionnaires in the phase III KarMMa-3 clinical trial to demonstrate that, when compared with standard regimens, idecabtagene vicleucel yielded faster improvement and delayed deterioration in measures of emotional, cognitive, and social functioning in patients with relapsed or refractory multiple myeloma.  

Similarly, in the December 10 session Outcomes Research – Lymphoid Malignancies: Patient Reported Outcomes in Hematological Malignancies, Abstract 667 focuses on QoL measures for lisocabtagene maraleucel among patients with relapsed and refractory mantle cell lymphoma in the TRANSCEND NHL 001 clinical trial. Using several PRO questionnaires, Michael L. Wang, MD, and colleagues identified meaningful changes in all primary QoL domains except pain, with over 50% of treated patients reporting improvements in overall health status, fatigue, and social functioning. In the same session, Abstract 668 by Guillaume Cartron, MD, PhD, and colleagues relays similar results from the TRANSCEND FL trial, which focused on lisocabtagene maraleucel in patients with relapsed and refractory follicular lymphoma. While this study did not compare outcomes between cellular therapy and alternatives such as treatment with human bispecific monoclonal antibodies, the PROs discussed therein provide further evidence supporting the benefits of cellular therapy in this complex population, for whom QoL is of crucial importance. 

The value of PROs goes beyond that for heavily pretreated patients with refractory diseases. These data can be useful for assessing novel upfront therapies for conditions with a limited number of objective outcome measures. In the December 9 session MGUS, Amyloidosis and Other Non-Myeloma Plasma Cell Dyscrasias: Clinical and Epidemiological: Screening to Vaccines–The Spectrum of Plasma Cell Disorders, Abstract 213 presents the results of an innovative study on Bruton tyrosine kinase (BTK) inhibitors for monoclonal gammopathy-related anti-myelin associated glycoprotein (MAG) IgM peripheral neuropathy. This is a population for which there has been a disconnect between traditionally reported hematologic responses and subjective improvements. Shayna R. Sarosiek, MD, and colleagues show that combination therapy with rituximab and acalabrutinib results in an 82% hematologic response but also a 57% improvement in patient-related disability scores, making a compelling argument for the addition of BTK inhibitors to the therapeutic arsenal.  

PROs can also validate objective responses and promote value-based care in patients with indolent conditions that predominantly affect QoL. In the session Outcomes Research – Lymphoid Malignancies: Practice Changing Patient-Reported Outcome Measurements and Real World Evidence Studies in Patients With Lymphoma, Abstract 381 demonstrates that patient-reported measures of HRQoL correlate with complete and partial responses to novel therapies for cutaneous lymphomas. Niloufer Khan, MD, and colleagues utilized validated scores in the phase 3 MAVORIC study, which compared mogamulizumab versus vorinostat in previously treated patients with mycosis fungoides. Their data revealed that measures of emotional, functional, social, and physical well-being correlated with response rates as measured using traditional methods. This finding is significant, as clinicians and patients are more likely to value an intervention with proven cutaneous responses if these are associated with QoL and PROs. 

As we navigate through increasingly complex and diverse treatment options, PRO assessments can be instrumental in guiding how we implement novel therapies into clinical practice and how we assemble multidisciplinary teams to care for our patients. Clinical research that incorporates PROs may be the missing link that helps us translate the objective health outcomes we value so much into more comprehensive and well-rounded care. 


1 Berwick DM. Medical associations: Guilds or leaders? BMJ. 1997;314(7094):1564-1565.  

2 FDA. Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Rockville, MD; 2009. 

Dr. Adrianzen Herrera indicated no relevant conflicts of interest. 

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