January 23rd 2024
Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
December 26th 2023
FDA Approves Pemigatinib for Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement
August 26th 2022The FDA Friday approved pemigatinib (Pemazyre), a selective fibroblast growth factor (FGFR) inhibitor, to treat adults who have relapsed or refractory (R/R) myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement, a very rare and aggressive cancer.
Read More
Better HRQOL, Health Literacy Seen in Systemic Mastocytosis vs Mast Cell Activation Syndrome
August 22nd 2022Systemic mastocytosis and mast cell activation syndrome both negatively impact health-related quality of life (HRQOL), with mast cell activation syndrome patients reporting more significant impacts on everyday life.
Read More
Large-scale Studies Needed to Guide Newborn Screening for Rare Diseases
August 2nd 2022Despite the known potential benefits of newborn screening, the exact long-term clinical benefits and cost-effectiveness in large cohorts remain uncertain due to a lack of large-scale longitudinal research.
Read More
Bacteria Behind Rare Disease Melioidosis Found in US for First Time
July 27th 2022For the first time, the CDC has identified in Mississippi dirt and water samples a bacteria that causes a rare and sometimes fatal disease called melioidosis, which is common in low-income tropical countries and one that scientists have been warning about for several years.
Read More
Inflammatory Cytokine Profiles Are Similar in MDS and Unexplained Cytopenias, Study Suggests
July 21st 2022Clonal cytopenias of undetermined significance often manifest prior to myelodysplastic syndromes (MDS), and a recent study found similarities in the cytokine profiles of the distinct conditions.
Read More
Hypomethylating Agents Can Produce Long Remission of MPNs but May Contribute to Morbidity, Mortality
July 4th 2022Hypomethylating agents are a widely used treatment option in acute myeloid leukemia secondary to myeloproliferative neoplasms (MPNs), but careful monitoring of thromboembolic risk is needed for optimal outcomes.
Read More
Assessing Longitudinal Trends in Essential Thrombocythemia, Polycythemia Vera Blood Counts
July 2nd 2022A recent study identified trends in blood counts, but did not find statistically significant associations between achieving hematological remission and clinical outcomes in polycythemia vera (PV) or essential thrombocythemia (ET).
Read More
Population-Based Study Finds Racial Disparities in MDS Outcomes
June 29th 2022Research presented at the 2022 American Society of Clinical Oncology Annual Meeting shows White patients with myelodysplastic syndrome (MDS) have worse overall survival outcomes than African American patients, contrary to previous findings in other cancer types.
Read More
Poor Health-Related Quality of Life, Many Burdens in Rare Diseases, Study Finds
June 18th 2022People with rare diseases experience significant diagnostic delays, high out-of-pocket costs, travel burden for specialty care, and insufficient dental and psychological support, according to new findings.
Read More
Transfusion-Dependent Patients With MDS Vary in Treatment Experiences, Preferences
June 17th 2022Transfusion dependence is common for patients with myelodysplastic syndromes (MDS), but real-world data show inconsistent patient experiences and varied transfusion practices between countries.
Read More
Study: Suggested Cyclosporine Levels Posttransplant May Be Too Low to Prevent GVHD
June 4th 2022Preventing graft-versus-host disease (GVHD) after allogeneic stem cell transplant is a key aspect of patient management, but current cyclosporine concentration guidelines may not be sufficient, according to a recent study.
Read More
CLAD, Pulmonary GVHD Have Similar Clinical Traits, Research Needs
May 19th 2022Fine-tuning clinical phenotypes and identifying effective treatments remain challenging in chronic lung allograft dysfunction (CLAD) after lung transplantation and chronic graft-versus-host disease (GVHD) after allogeneic hematopoietic stem-cell transplantation.
Read More
RAS Inhibitors May Hold Potential as Preventive for Acute Chest Syndrome in Sickle Cell Disease
May 15th 2022A retrospective study assessed hospital readmission rates in patients with sickle cell disease and acute chest syndrome who took angiotensin-converting enzyme inhibitors or angiotensin II receptor blockers vs those who did not.
Read More
Review Highlights Treatment Landscape, Insights on Pregnancy Management for ET or PV
May 8th 2022Polycythemia vera and essential thrombocythemia are both slow-progressing conditions and treated mainly with symptom management, but novel agents and gene targets have changed the landscape in recent years.
Read More
Poorer Outcomes Associated With Recurrent aGVHD Warrant Independent Recognition, Says Study
May 2nd 2022After a median follow-up of 535 days, analysis showed that recurrent acute graft-versus-host disease (RaGVHD) carried several implications for survival, leading the researchers to call for further risk stratification of aGVHD.
Read More
Real-world Study Shows Ruxolitinib May Reduce Arterial Thrombosis in Polycythemia Vera
April 27th 2022Previous studies have shown ruxolitinib effective in symptom relief and hematocrit control for hydroxyurea-resistant polycythemia vera, but its role in disease progression is not yet clear.
Read More