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Treatment options for lower-risk myelodysplastic syndromes. Where are we now?

ABSTRACT: Myelodysplastic syndromes (MDS) are a spectrum of clonal stem-cell disorders characterized clinically by bone-marrow failure. Resultant cytopenias are responsible for significant mortality and decreased quality of life in patients with MDS. In patients with low-risk MDS (LR-MDS), anemia is the most common cytopenia and erythropoiesis-stimulating agents (ESA) are usually used as first-line therapy. Those patients who become refractory to ESA have a poor survival. Available treatment options such as lenalidomide, hypomethylating agents, and immunosuppressive therapy can provide some hematologic response among selected subsets of patients, however durable responses are limited, and these agents can carry significant adverse effects. Chronic transfusions help to alleviate symptoms of anemia but still carry risks associated with transfusion and iron overload. Luspatercept, recently approved for those LR-MDS with ring sideroblasts refractory to ESA, was found to have an improvement in transfusion independence with a well-tolerated safety profile. While anemia is the most common cytopenia, thrombocytopenia and neutropenia management is challenging and the co-occurrence of these cytopenias with anemia may dictate the choice of therapy. In this article, we review LR-MDS and discuss the optimal use of current treatment options and explore new therapeutic options on the horizon.

SUBMITTER: Volpe VO

PROVIDER: S-EPMC7812395 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature

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Treatment options for lower-risk myelodysplastic syndromes. Where are we now?

Volpe Virginia O VO Komrokji Rami S RS

Therapeutic advances in hematology 20210114

Myelodysplastic syndromes (MDS) are a spectrum of clonal stem-cell disorders characterized clinically by bone-marrow failure. Resultant cytopenias are responsible for significant mortality and decreased quality of life in patients with MDS. In patients with low-risk MDS (LR-MDS), anemia is the most common cytopenia and erythropoiesis-stimulating agents (ESA) are usually used as first-line therapy. Those patients who become refractory to ESA have a poor survival. Available treatment options such ...[more]

PMID: 33505645

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Project description:The majority of myelodysplastic syndrome (MDS) patients belong to the International Prognostic Scoring System (IPSS) and IPSS-revised (IPSS-R) lower-risk categories. Their precise diagnostics and prognostic stratification is often a challenge, but may ensure the optimization of therapy. The availability of diverse treatment options has significantly improved the quality of life and survival of this group of patients. Anemia is the most relevant cytopenia in terms of frequency and symptoms in lower-risk MDS, and may be treated successfully with erythropoietic stimulating agents, provided a careful selection is performed on the basis of IPSS-R, endogenous erythropoietin levels, and transfusion independence. Doses and duration of therapy of erythropoietic-stimulating agents (ESAs) are critical to determine efficacy. In case a patient fails ESA treatment, the available options may include lenalidomide (approved for del5q positive cases), hypomethylating agents, and a rather large number of experimental agents, whose clinical trials should be offered to a larger number of MDS patients. The choice for second-line treatment must take into account biologic, cytogenetic, and molecular-identified characteristics of individual patients, as well as frailty and comorbidities. Other cytopenias are less frequently presenting as isolated. Specific therapy for thrombocytopenia has been proposed in experimental clinical trials with thrombomimetic agents that have shown good efficacy, but raised some safety concern. Although neutropenia is targeted symptomatically with growth factor supportive care, the immunosuppressive treatments are indicated mainly for pancytopenic, hypoplastic lower-risk MDS; they are not widely used because of their toxicity, despite the fact that they may induce responses. Finally, hematopoietic stem cell transplant is the curative option also for lower-risk MDS and timing should be carefully evaluated, balancing toxicity and the possibility of survival advantage. Finally, even when considered suitable for lower-risk MDS, transplant application is limited to the rarer fit and younger MDS patient.

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Treatment options for lower-risk myelodysplastic syndromes. Where are we now?

Publications

Treatment options for lower-risk myelodysplastic syndromes. Where are we now?

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