In-Depth Diagnostic Testing in Older Patients with Myelodysplastic Syndromes

A new multicenter study coauthored by Cleveland Clinic investigators shows that the survival benefit of complete diagnostic evaluation (CDE) may not outweigh the associated potential costs, pain and anxiety in older patients with myelodysplastic syndromes (MDS). The findings, presented at the American Society of Clinical Oncology’s 2021 annual meeting, reflect experience in fee-for-service Medicare patients and point to a need for more careful patient selection to eliminate unnecessary CDEs while maximizing therapeutic outcomes.

MDS is the most common myeloid malignancy in the United States. CDE to definitively establish the diagnosis of MDS requires at minimum a bone marrow biopsy and other supporting studies including fluorescence in situ hybridization and chromosomal analysis.

“Because these tests are painful and expensive, clinicians have debated whether they’re necessary for better care in certain patients,” says Sudipto Mukherjee, MD, PhD, MPH, staff in the Department of Hematology and Oncology at Cleveland Clinic Cancer Center and lead author of the study. “We conducted this study to answer that question.”

Study at a glance

Approximately 17,000 MDS patients aged 66 or older identified in a Medicare database from 2011 to 2014 were included in the study. All patients included in the study had no more than one cytopenia and no dependence on blood transfusions in the 16 weeks before or after diagnosis of MDS.

To assess the value of CDE in this population, the investigators focused on variables that provided clinical context about the decision regarding the testing. They included age, race, sex, number of Elixhauser comorbid conditions, nursing home status, prior history of cancer, gastrointestinal bleeding and chronic kidney disease (CKD). Impact on outcomes, and whether CDE led to therapy initiation, particularly use of erythropoiesis stimulating agents (ESAs), hypomethylating agents (HMAs) and lenalidomide, were also explored.

Combinations of factors that resulted in little clinical justification for CDE were determined with a classification and regression-tree analysis, a machine learning approach. Survival outcomes in patients who had and had not undergone CDE were compared with a Cox proportional hazards regression analysis.

Choosing the appropriate patients

Overall, just over half (51%) of the study population received CDE. Nearly half of them (46.6%) were aged 80 or older, 33.6% had at least five chronic conditions and 4.8% were nursing home residents.

In MDS patients that had an isolated cytopenia and no transfusion needs, the investigators found that 46% of those aged 80 years or older and 57.7% of those aged 66 to 79 years underwent CDE in the absence of a history of CKD or colonoscopy or received HMA or ESA post CDE. In the group that had no cytopenias, 56.2% of those who received CDE did not have a prior history of cancer or progress to acute myeloid leukemia in the first year following MDS diagnosis or received HMA or ESA.

In adjusted regression analyses, no survival benefit was associated with receipt of CDE (P = 0.24).

“Our findings show that careful patient selection may reduce unnecessary CDE for older MDS patients and should be a priority in clinical decision-making as a way to promote patient-centered care and minimize use of low-value procedures that may be of limited benefit,” concludes Dr. Mukherjee.