Treatment of Lower-Risk Myelodysplastic Syndrome with del(5q) and Symptomatic Transfusion-Dependent Anaemia

This protocol covers a distinct sub-population within lower-risk MDS: patients with an IPSS-R score up to 3.5 — encompassing very low-, low-, and part of intermediate-risk IPSS-R cases — who present with symptomatic anaemia requiring regular red blood cell transfusions and carry the del(5q) chromosomal abnormality.

Clinical scenario

Symptomatic anaemia (generally Hb <10 g/dl) with transfusion dependence in the setting of del(5q) defines a therapeutically distinct group among lower-risk MDS patients, with a specific recommended treatment approach.

Treatment approach

For transfusion-dependent anaemia in lower-risk MDS with del(5q), lenalidomide is the most effective recommended agent — the complete dosing schedule and management guidance are available in the full protocol.

Clinical goals

Treatment targets red blood cell transfusion independence and cytogenetic response.

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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 anaemia (generally if Hb <10 g/dl)
Del(5q)
For transfusion-dependent anaemia of lower-risk MDS with del(5q), LEN is the most effective drug [I, A].
However, it responds to LEN in 60%–65% of patients, with a median duration of RBC transfusion independence (RBC-TI) of 2–2.5 years [I, A].
Cytogenetic response (CyR) is achieved in 50%–75% of patients (including 30%–45% complete CyR).

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