Clinicians use the presence or absence of minimal residual disease as a metric by which to judge the efficacy of therapy in patients with hematological malignancies. A new review article interrogates whether that approach aligns with patient-centered outcomes.
Assessing whether a patient still has minimal residual disease (MRD) following therapy for hematological malignancies has become a common metric for assessing the success of treatment, but a new study asks whether such an evaluation produces conclusions that align with meaningful improvements in a patient’s quality of life.
MRD is a straightforward way to characterize patients following treatment for diseases like lymphoma, leukemia, and multiple myeloma (MM). Patients who are found to still have a small number of cancer cells after treatment are labeled “MRD positive”; those without any presence of the disease are termed “MRD negative.”
Sumeet Panjabi, PhD, of Amgen Inc, and colleagues wanted to find out whether MRD status correlates with real-world quality-of-life improvements for patients following treatment. In search of an answer, Panjabi and colleagues constructed a targeted literature review to find studies that looked at MRD and patient-centered outcomes (PCOs).
The results of the review were presented at the Professional Society for Health Economics and Outcomes Research’s ISPOR 2020, which was held as a virtual conference due to the ongoing coronavirus disease 2019 pandemic.
For the purposes of the study, PCOs were defined as health-related quality of life (HRQOL), utilities, health care resource use, and cost-effectiveness. The authors pulled full-text studies from within the past decade, as well as conference abstracts and health technology assessment submissions for the previous 5 years. The study was broadened to include all hematological malignancies.
The search yielded a total of 8 publications, and Panjabi and colleagues report that in general those publications showed that MRD negativity had positive impacts on PCO; however, they note that the research is still limited, and the results are not without contradiction.
For instance, the studies, which mostly used an MRD sensitivity threshold of 10-4, varied somewhat by diagnosis. One study of patients with MM found that patients who started at a similar baseline status and achieved MRD-negative status had a better HRQOL than those who remained MRD-positive. Similarly, 2 studies of chronic lymphoblastic leukemia (CLL) and acute lymphoblastic leukemia (ALL) also showed that QOL was improved in patients with no evidence of disease. Yet, a different study of patients with chronic myeloid leukemia (CML) found patients who were MRD negative had higher symptom burdens than their MRD-positive counterparts after 24 months, Panjabi and colleagues wrote.
Four studies that looked at health care utilization and cost found MRD-negative patients had lower rates of both usage and cost. Meanwhile, 2 studies that looked specifically at the cost-effectiveness of the targeted therapy blinatumomab (Blincyto) in patients with ALL found the drug was cost-effective largely because its use tended to correlate with high MRD response rates, which in turn translated to lower health care costs and longer relapse-free durations.
Panjabi and colleagues conclude that the existing literature is promising, but insufficient.
“The beneficial impact of deeper responses in the form of MRD negativity on PCOs was demonstrated in these few studies,” they conclude. “However, these data are limited and the relationship between MRD status and PCOs needs to be explored further in future studies.”
Panjabi S, Gebregergish S, Abbas Z, Buchanan J, Yuan Z. Relationship between minimal residual disease (MRD) and patient outcomes in haematology indications: a targeted literature review. Abstract presented at: ISPOR 2020; May 2020; Orlando, FL. Accessed May 29, 2020. https://www.ispor.org/heor-resources/presentations-database/presentation/intl2020-3182/100052