Refractory Anemia With Excess Blasts

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Retrieved

2021-01-23

Source

Orphanet

Trials

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Genes

TET2, FANCB, CD34, RUNX1, CCR7, MECOM, KMT2A, EPO, GATA2, HPSE2, ACSL5, SF3B1, ACSL6, AKAP12, DLK1, CIP2A, IGHV1-12, ACHE, NSD1, VEGFA, DERL1, ATAD1, ASXL1, ILDR1, MIR29B1, MIR29B2, VIM2P, MOGS, SRSF2, U2AF1, THY1, BCL2, BCR, CBL, CDKN2B, CHEK1, CSF3, ETV6, FLT3, HLA-DRB1, ITGA2B, MCL1, NCAM1, NRAS, RARS1, BCHE, SPN, THPO, U2AF1L5

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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Refractory anemia with excess blasts (RAEB) is a frequent severe subtype of myelodysplastic syndrome (MDS; see this term) characterized by cytopenias with unilineage or multilineage dysplasia and 5% to 19% blasts in bone marrow or blood.

Epidemiology

Exact prevalence is unknown but RAEB accounts for about 40% of patients with MDS which has a prevalence estimated to be 1/25,000 to 1/33,000. The disease tends to occur in older adults with a male predominance.

Clinical description

Two types of RAEB are recognized: type 1 and type 2 (RAEB-1 and RAEB-2; see these terms) which are differentiated based on the percentage of blasts in bone marrow or blood. Patients usually present with symptoms related to one or more cytopenias. Survival is lower in patients with RAEB-2 and there is a higher rate of transformation into acute myeloid leukemia in these patients. Most RAEB patients die from neutropenia-, thrombocytopenia- and anemia-related complications.

Etiology

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