treatment

SRSF2-mutated MDS: bortezomib STAT?

Original Publication Date
Article Source
External Web Content
In this issue of Blood, Takashima et al show that reduced STAT1 abundance in SRSF2-mutant myelodysplastic syndrome (MDS) cells confers protection against interferon (IFN)-driven cell suppression relative to normal wild-type (WT) cells.1 Importantly, the authors demonstrate that…

Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis After Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation

Original Publication Date
Article Source
External Web Content
Abstract Purpose Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for advanced hematologic malignancies. HSCT using human leukocyte antigen (HLA)-mismatched donors is historically associated with inferior survival. Patients from underrepresented…

Understanding the Paroxysmal Nocturnal Hemoglobinuria Journey

Status
Recruiting
Study Date (Range)
-
Bone Marrow Disease(s)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Age Group
21 years and older
The purpose of this study is to better understand the treatment journey and experiences of people living with Paroxysmal Nocturnal Hemoglobinuria (PNH), specifically those currently in a “watch and wait” period after being diagnosed.

PLEDGE – Patients’ Long-term Experience on VOYDEYA (Danicopan): Gathering Evidence in the Real-World

Status
Recruiting
Study Date (Range)
-
Bone Marrow Disease(s)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Age Group
18 years and older
The IRB-approved observational Phase IV PLEDGE study is set-up to collect real-world data on adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) with symptomatic extravascular hemolysis who recently started VOYDEYATM (danicopan) as add-on treatment to ULTOMIRIS®…
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