Luspatercept reduced transfusion burden in MDS patients

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Alan F. List, MD
© ASH/Scott Morgan 2018
Results of the randomized, phase 3 Medalist trial show that the erythroid maturation agent luspatercept can reduce transfusion burden in patients with anemia due to myelodysplastic syndromes (MDS) and ring sideroblasts. Almost 38% of luspatercept-treated patients achieved red blood cell (RBC) transfusion independence for 8 weeks... [Read Article]
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FDA approves generic decitabine for MDS

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Micrograph showing MDS
The U.S. Food and Drug Administration has approved Lupin’s decitabine product, a generic version of Otsuka Pharmaceutical Co. Ltd.’s Dacogen, to treat patients with myelodysplastic syndromes (MDS). Lupin’s decitabine for injection (50 mg, single-dose vial) is approved to treat patients with intermediate-1, intermediate-2, and high-risk MDS. This includes previously treated, untreated,... [Read Article]
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Combo worsens platelet recovery in MDS

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Eltrombopag (Promacta)
Photo courtesy of GSK
Results from the phase 3 SUPPORT trial suggest there is no role for the combination of eltrombopag and azacitidine in patients with intermediate- or high-risk myelodysplastic syndromes (MDS), according to investigators. Adding eltrombopag to azacitidine worsened platelet recovery, reduced the overall response rate, and did not improve overall... [Read Article]
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Researchers develop genetics-based prognostic tool for MDS

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Micrograph showing MDS
Researchers have developed a new risk model for primary myelodysplastic syndromes (MDS) that integrates genetic and clinical information. The research team considered the current standard for prognostication—the revised International Prognostic Scoring System (IPSS-R)—to be too complex, limited to newly diagnosed cases, and missing information on mutations and age. So they devised a... [Read Article]
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Sequencing informs prognosis after HSCT in MDS

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HSCT preparation
Photo by Chad McNeeley
Gene sequencing early after transplant may provide important prognostic information in patients with myelodysplastic syndromes (MDS), according to a new study. Patients who had disease-associated mutations in the bone marrow 30 days after hematopoietic stem cell transplant (HSCT) were significantly more likely to experience disease progression and have lower... [Read Article]
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HIF1A could be therapeutic target for MDS

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Lab mouse
The transcription factor HIF1A could be a therapeutic target for “a broad spectrum” of patients with myelodysplastic syndromes (MDS), according to researchers. Preclinical experiments indicated that HIF1A fuels the biological processes that cause different types of MDS. Researchers also found that inhibiting HIF1A reversed MDS symptoms and prolonged survival in mouse models of... [Read Article]
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AMP publishes report on DNA variants in CMNs

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Polycythemia vera
Image courtesy of AFIP
A new report addresses the clinical relevance of DNA variants in chronic myeloid neoplasms (CMNs). The report is intended to aid clinical laboratory professionals with the management of most CMNs and the development of high-throughput pan-myeloid sequencing testing panels. The authors list 34 genes they consider “critical” for sequencing... [Read Article]
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Familial risk of myeloid malignancies

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Three generations of
women in a family
A large study has revealed “the strongest evidence yet” supporting genetic susceptibility to myeloid malignancies, according to a researcher. The study showed that first-degree relatives of patients with myeloid malignancies had double the risk of developing a myeloid malignancy themselves, when compared to the general population. The researchers... [Read Article]
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Study could change treatment of MLSM7

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Study authors Tamara
Lamprecht, Jason Schwartz,
Jing Ma, and Jeffrey Klco
Photo from St. Jude
Children’s Research
Hospital/Justin Veneman
New findings could help improve treatment of an inherited bone marrow disorder known as myelodysplasia and leukemia syndrome with monosomy 7 (MLSM7), according to researchers. While studying families affected by MLSM7, researchers identified germline mutations... [Read Article]
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Treatments, disease affect spermatogonia in boys

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Male germinal epithelium
showing spermatogonia,
spermatocytes, spermatids,
and spermatozoa
Image from Dreamstime
Alkylating agents, hydroxyurea (HU), and certain non-malignant diseases can significantly deplete spermatogonial cell counts in young boys, according to research published in Human Reproduction. Boys who received alkylating agents to treat cancer had significantly lower spermatogonial cell counts than control subjects or boys... [Read Article]
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