Vol 13, No 3-4 (2022)
Review paper
Published online: 2023-02-16

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Therapeutic options in high-risk myelodysplastic syndrome

Marcela Maksymowicz1, Sara Moqbil1, Piotr Machowiec1, Monika Podhorecka2
Hematology in Clinical Practice 2022;13(3-4):97-111.


Myelodysplastic syndromes (MDS) constitute a heterogeneous group of diseases characterised by ineffective haematopoiesis, dysplasia and cytopenias. The treatment for high-risk MDS (HR-MDS) depends on individual factors such as the stage of the disease, age, comorbidities, and infections.

Allogeneic haematopoietic stem cell transplantation (allo-HSCT) with reduced intensity conditioning has allowed more HR-MDS patients to be transplant-eligible, regardless of age. Hypomethylating agents, including azacitidine and decitabine, remain the standard of care for HR-MDS patients who are not qualified for curative allo-HSCT. Combination therapy of azacitidine with some new drugs resulted in higher response rates than azacitidine in monotherapy. Other targeted therapies are under investigation. They include HMA with different antibodies targeting immune checkpoints — programmed cell death (ligand) 1, cytotoxic T lymphocyte antigen 4, T-cell immunoglobulin mucin-3 or cluster of differentiation 47. Larger studies are necessary to confirm their efficacy in the treatment of HR-MDS.

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