ESAs in LR-MDS: Prognostic Factors for Response Identified

A systematic literature review (SLR) published in the Journal of Clinical Medicine found that in patients with anemia and lower-risk myelodysplastic syndromes (LR-MDS), prognostic factors such as transfusion independence, high hemoglobin levels, and low serum erythropoietin (EPO) levels have a statistically significant relationship with the use of erythropoiesis-stimulating agents (ESAs).

There was a determination that the erythropoiesis-stimulating agent (ESA) response was statistically significantly correlated with hemoglobin levels, transfusion independence, and low serum erythropoietin levels.

One of the most common cytopenias in LR-MDS patients is anemia, which is usually treated with ESAs or blood transfusions, with the latter being the first-line treatment for anemia in LR-MDS patients.

To help identify patients who are less likely to benefit from the medication and provide these patients with alternative medicines, the investigators set out to better understand the impact these characteristics have on ESA response.

The investigators reported that 38 papers total—34 full-text studies and 4 conference abstracts—were included in the systematic literature review (SLR). Across all studies, the population’s median age varied between 63.1 and 82.7 years. Bone marrow blasts, age, hemoglobin level, serum ferritin level, International Prognostic Scoring System (IPSS) risk status, karyotype status, serum erythropoietin (EPO) level, and transfusion dependence/independence were the eight potential prognostic factors at baseline that were frequently reported across the included literature.

Patients who were transfusion-independent or had higher baseline hemoglobin levels were the most likely to respond to ESA medication among all the included studies; the researchers observed that this response was regarded as the most statistically significant regardless of the kind of analysis. 

Notably, prognostic variables such as age, IPSS risk status, bone marrow blasts, and others did not show “consistent, statistically significant, quantitative relationships with ESA response,” which, as the researchers found, held for many kinds of analysis.

The main conclusions of this study are consistent with prior reviews of MDS and prognostic variables that have been done. Park et al.’s literature evaluation also revealed that EPO levels were a significant predictor of the hematologic response to ESAs.2. Furthermore, these writers wrote that “treatment response is significantly more likely among those with EPO levels below versus those above various cutoffs.”

Park et al. cautioned that many of the characteristics linked to an improved prognosis have also been linked to an improved response to ESAs, as the authors of the current study have explained. They talked about the prospect that these variables might forecast disease burden more accurately than just how well an ESA works.

The researchers recognized that restricting the patient group to LR-MDS may have had an effect, since it may have prevented significant findings for variables including age, IPSS status, and karyotype status from being found.

Tatarelli et al.’s study, which looked at EPO in elderly patients, reached a similar conclusion, saying that more comprehensive research would be needed to fully comprehend the problem and that “larger studies are warranted to evaluate if EPO treatment could be worthwhile in terms of quality of life and cost-efficacy in very old patients.”

The investigators concluded that by emphasizing these important baseline clinical traits, patients with LR-MDS who display these prognostic markers may be identified before starting treatment and may be offered potentially more advantageous first-line medications.

“Future research should assess the impact of baseline ferritin levels and bone marrow blast percentages on ESA response/failure, by using multivariate analyses that include data from large patient populations in LR-MDS and consider relevant prognostic factors such as serum EPO levels, baseline hemoglobin, and transfusion-dependence status,” the investigators concluded.

For more information: Key Factors Significantly Associated With ESA Response in Lower-Risk MDS, Journal of Clinical Medicine, https://doi.org/10.3390/jcm13092702