Unclassified Myelodysplastic Syndrome

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Retrieved

2021-01-23

Source

Orphanet

Trials

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Genes

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Drugs

2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one, Allogeneic ex vivo expanded umbilical cord blood cells, Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus, Arsenic trioxide ( Arsenic Trioxide Accord, Arsenic trioxide Mylan, Arsenic trioxide medac, TRISENOX ), Asunercept, Azacitidine ( AZACITIDINE ACCORD, AZACITIDINE BETAPHARM, AZACITIDINE CELGENE, AZACITIDINE MYLAN, VIDAZA ), Decitabine ( DACOGEN ), Fully human IgG1 antibody specific for CD33, Humanised IgG4 monoclonal antibody to the human toll-like receptor type 2, Imatinib mesilate ( GLEEVEC, GLIVEC, IMATINIB TEVA, IMATINIB TEVA B.V. ), Lenalidomide ( LENALIDOMIDE ACCORD, REVLIMID ), Lintuzumab, Luspatercept ( REBLOZYL ), Magrolimab, Mocetinostat, NEDD8-activating enzyme (NAE) inhibitor, Rigosertib, Sapacitabine, Sotatercept, Stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells, Thalidomide ( THALIDOMIDE CELGENE, THALOMID ), Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3), haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate

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Unclassified myelodysplastic syndrome (MDS-U) is a subtype of myelodysplastic syndrome (MDS; see this term) with atypical features of uncertain clinical significance.

Epidemiology

Prevalence is unknown but MDS-U appears to account for a very low proportion of MSD cases.

Clinical description

Unclassified MDS covers cases that cannot be allocated to another subtype of MSD after a full diagnostic work-up has been performed including clinical, morphologic, immunophenotypic, or cytogenetic testing. One or more cytopenias are a standard feature of MDS-U but other clinical features are variable. The following cases are considered to fall into this subtype: features of refractory cytopenia with uni- or multi-lineage dysplasia (RCUD, RCMD, see these terms) but with 1% blasts in peripheral blood, unilineage dysplasia with pancytopenia, and persistent cytopenias without morphological features of MDS associated with characteristic cytogenetic abnormalities.

Etiology

The etiology is not known but is thought to involve inherited susceptibility or hematopoietic stem cell damage.