What is the treatment of lower-risk MDS (IPSS-R up to 3.5) with symptomatic thrombocytopenia?

This protocol covers a specific sub-population of MDS patients classified as lower-risk by IPSS-R, in whom symptomatic thrombocytopenia is the principal treatment target.

Clinical Scenario

For therapeutic purposes, lower-risk MDS applies to cases with IPSS-R up to 3.5 — encompassing very low-, low-, and part of intermediate-risk patients. When symptomatic thrombocytopenia is the predominant problem, management is directed at platelet-focused intervention, with eligibility for specific approaches influenced by individual patient and marrow characteristics.

Treatment Approach (partial overview)

Several categories of therapy are considered in this setting — including androgenic therapy, agents targeting the thrombopoietin receptor pathway, and, in selected patients, immunosuppressive or hypomethylating approaches — with eligibility depending on specific marrow findings and patient features. Full selection criteria, sequencing, and evidence levels are in the complete protocol.

Treatment Goals

The primary objectives are a meaningful platelet response and a reduction in bleeding events.

Instant Access to Structured Evidence-Based Regimens

References

DOI: 10.1016/j.annonc.2020.11.002

For therapeutic purposes, the term 'lower-risk' MDS generally applies to cases with IPSS-R up to 3.5 including very low- and low-risk and part of intermediate-risk IPSS-R patients.
Symptomatic thrombocytopaenia
High-dose androgens can improve thrombocytopaenia in one-third of thrombocytopaenic lower-risk MDS, but response is generally transient [III, C].
TPO-RAs (romiplostim, eltrombopag) have some efficacy in cases of severe thrombocytopaenia but they are not approved in MDS and should only be used in patients with marrow blasts <5% [II, C].
ATG ± cyclosporine (in selected cases, as described above) and HMAs achieve platelet response in 35%–40% of cases of lower-risk MDS in addition to erythroid responses [III, C].

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