Myelodysplastic syndrome (MDS) is a heterogeneous group of clonal hematologic stem cell disorders characterized by ineffective hematopoiesis resulting in low blood counts, and a risk of progression to acute myeloid leukemia. Currently, there are a few FDA-approved drugs for treatment of MDS none of which are curative. Allogeneic stem cell transplantation (ASCT) is the only curative therapy. But many MDS patients have been ineligible for transplants, since the median age at diagnosis for MDS is 75 years. Sequencing of MDS genomes has identified mutations in genes implicated in RNA splicing, DNA modification, chromatin regulation, and cell signaling.
Category
Hematologic disease
Brite
Human diseases [BR:br08402]
Cardiovascular diseases
Hematologic diseases
H01481 Myelodysplastic syndrome
Human diseases in ICD-11 classification [BR:br08403]
02 Neoplasms
Neoplasms of haematopoietic or lymphoid tissues
Myelodysplastic syndromes
2A36 Myelodysplastic syndrome with isolated del(5q)
H01481 Myelodysplastic syndrome
2A37 Myelodysplastic syndrome, unclassifiable
H01481 Myelodysplastic syndrome
2A3Z Myelodysplastic syndromes, unspecified
H01481 Myelodysplastic syndrome
Jang JH, Harada H, Shibayama H, Shimazaki R, Kim HJ, Sawada K, Mitani K
Title
A randomized controlled trial comparing darbepoetin alfa doses in red blood cell transfusion-dependent patients with low- or intermediate-1 risk myelodysplastic syndromes.
