This first-line protocol applies to patients with lower-risk myelodysplastic syndrome — IPSS-R up to 3.5, covering very low, low, and part of intermediate-risk cases — who present with symptomatic anaemia and haemoglobin generally below 10 g/dl, in the absence of the del(5q) cytogenetic abnormality.
The protocol is shaped by the following characteristics of this patient group:
The approach for this scenario is centred on erythropoiesis-stimulating agents as the first-choice intervention. Supportive therapy may be added to improve erythroid efficacy. The complete structured regimen — including agent selection, sequencing, and monitoring guidance — is available in the full protocol.
The primary aim is an erythroid response according to IWG 2006 criteria, expected to occur within 8–12 weeks of initiating treatment.
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)
No del(5q)
ESAs, i.e. recombinant EPO or darbepoetin (DAR), remain the first choice treatment of anaemia in most lower-risk MDS without del(5q).
Efficacy of ESAs can be improved by the addition of granulocyte colony-stimulating factor (G-CSF), but there are no data showing that one ESA is superior to another.
ESAs (especially EPO alpha) are recommended for the first-line treatment of anaemia in lower-risk MDS in patients without del(5q) [I, A].
Responses to ESA occur within 8-12 weeks of treatment. Median duration of response is 20-24 months.
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