Hematology Times
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CythemiasTwo distinct subgroups of JMML identified
CMML cells
Researchers recently identified 2 distinct clinical subgroups of juvenile myelomonocytic leukemia (JMML) using gene expression profiling. The 2 subgroups have clearly different outcomes regardless of treatment, even including the recommended therapy, hematopoietic stem cell transplant. Silvia Bresolin, of the University of Padua in Italy, and researchers from Italy, Germany, the Czech Republic, Belgium, and Denmark, suggest that GEP-based profiling could help physicians make therapeutic decisions. They reported the findings March 15 online ahead of print in the Journal of Clinical Oncology. ... [Read Article] New understanding of Chuvash polycythemia
Lab mouse
A recent study suggests that inhibiting HIF-2α, and possibly several other genes, may become a beneficial therapy in the treatment of human Chuvash disease. Using a murine model expressing the R200W point mutation from the endogenous von Hippel-Lindau locus (VhlR/R), the team learned that the R200W mutation and increase in HIF activity mimic the systemic effects of chronic hypoxic exposure. Hypoxia is often associated with the development of pulmonary hypertension in Chuvash patients. The study was published February 8 in the Journal of Clinical Investigation. ... [Read Article] Deferasirox encouraging in hereditary hemochromatosis
Tissue sample
showing hemochromatosis The first trial to assess the safety and efficacy of deferasirox in patients with HFE-related hereditary hemochromatosis and non-transfusional iron overload shows encouraging results that warrant larger studies. The 2 phase, open-label, multicenter, dose-escalation study, conducted by Pradyumna Phatak, MD, of Rochester General Hospital, and colleagues, evaluated the drug at 4 levels of dosing: 5, 10, 15, and 20 mg/kg/day. ... [Read Article] INCB018424 may be first drug for myelofibrosis
Bone marrow biopsy
showing myelofibrosis INCB018424, an oral investigational dual inhibitor of JAK1 and JAK2 enzymes, is poised to be the first treatment targeted to mechanisms that underlie myelofibrosis. There is currently no approved therapy for this lethal, devastating disease. The drug—referred to as “424” by lead investigator Srdan Verstovsek, MD, PhD, from The University of Texas MD Anderson Cancer Center in Houston, improved performance status, reduced the size of the spleen, and was safe and well tolerated. ... [Read Article] Peg-IFN could become new standard therapy in MPD
Steven Jean-Jacques Kiladjian, MD, PhD
Updated results of the PVN-1 trial have researchers wondering if pegylated interferon alpha 2a (peg-IFN α2a) could replace standard therapy for myeloproliferative disorders (MPDs). Peg-IFN α2a elicited a hematologic response rate of 100% and a complete response rate of 91% at 1 year of follow up. The drug showed significant activity against the MPD clone, and only 8% of patients discontinued the drug due to side effects, which is superior to results typically seen with IFNs. ... [Read Article] |
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