Rare Disease

Bulky Splenomegaly Associated With Worse Outcomes in Patients With MF Undergoing Allo-HCT

October 29th 2020, 7:30pm


Findings from a retrospective analysis support the use of splenectomy for patients with progressive disease and large splenomegaly.

Researchers Outline Novel Treatment Approaches Being Studied in Myelofibrosis

October 24th 2020, 12:00pm


Their study outlines several treatments being investigated for the treatment of patients with myelofibrosis and other myeloproliferative neoplasms, generally after they have become resistant or intolerant to Janus kinase inhibition.

What Drives Leukemic Transformation of Polycythemia Vera, Essential Thrombocythemia?

October 23rd 2020, 4:30pm


New findings from a small study are suggesting that time to leukemic transformation may be driven by distinct time-dependent molecular mechanisms.

Depression Worsens Symptom Burden in Patients With MPNs, Study Says

October 16th 2020, 4:30pm


Results from a large international survey indicated that depression worsened the systemic symptom burden for patients.

OOP Orphan Drug Spending Rising Higher for Children Than Adults

October 10th 2020, 10:45am


Orphan drug spending to treat rare conditions in children is increasingly burdening payers and families, a recent study said.

Pinpointing Peripheral Blood Blasts and the Impact on OS in Myelofibrosis

October 9th 2020, 7:30pm


Researchers identified 3 groups with distinctive patterns of overall survival (OS), among which they determined additional prognostic value in patients with <5% bone marrow blasts.

National Study Finds 2-Fold Risk of IBD Among Patients With MPNs

October 3rd 2020, 11:00am


The study of over 8000 patients with myeloproliferative neoplasms (MPNs) also found that these patients were 40% more likely to have a prior diagnosis of inflammatory bowel disease (IBD).

Survey Results Point to Differences Between Adult, Pediatric MPNs

October 1st 2020, 9:30pm


Historically, there has been a lack of understanding around clinical and laboratory features and prognostic factors for children with myeloproliferative neoplasms (MPNs), as MPNs are especially rare in this patient population.

Real-World Study Shows Promising Efficacy, Safety of Ropeg in MPN

September 25th 2020, 7:00pm


Based on assessments of ropeginterferon alpha-2b in a real-world setting, researchers highlighted the safety, tolerance, and efficacy of the treatment in Philadelphia-negative myeloproliferative neoplasm (MPNs).

Exploring New Perspectives in Diagnosis, Prognosis, and Treatment of PV

September 22nd 2020, 9:45pm


In a review of the polycythemia vera (PV) landscape, researchers outline new perspectives in the diagnosis, prognosis, and treatment of the disease.