What are the most important quality of life domains for patients with aplastic anemia and paroxysmal nocturnal hemoglobinuria?
The Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria Treatment
Disease Burden at the Time of Transplantation Is a Primary Predictor of Outcomes in Pediatric MDS: A Single-Center Experience
Background: Hematopoietic cell transplantation (HCT) remains the only curative therapy for pediatric myelodysplastic syndrome (MDS) in all but rare cases. While HCT outcomes for pediatric MDS are similar across the largest registry and single-center trials, factors identified as contributing to inferior outcomes vary from study to study. We performed an analysis to provide more clarity on the prognostic implications of disease characteristics, including blast burden and cytogenetic abnormalities, in the current era.
New Approvals in Low- and Intermediate-Risk Myelodysplastic Syndromes
Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: Phase 2 ESCALATE trial
Key Points
Eltrombopag combined with IST demonstrated a trend towards a favorable ORR in pediatric patients with r/r SAA.
The PK profile for eltrombopag was similar to that observed in patients with ITP, and no unexpected safety signals were observed in children
Donor regulatory T-cell therapy to prevent graft-versus-host disease
Key Points
Treg-engineered donor graft prevents acute and chronic GVHD.
Patients treated with Treg-engineered graft who develop GVHD respond to primary corticosteroid therapy at a high rate.
Taking Charge of My Care and Advocacy Following a Cancer Diagnosis
After a long battle with illness and seeking many specialists, I emphasize the need to self-advocate for ongoing care following my cancer diagnosis.
Prognostic Significance of Monocytic-like Phenotype in AML patients treated with Venetoclax and Azacytidine
Key Points
The impact of ABO compatibility on allogeneic hematopoietic cell transplantation outcomes: a contemporary and comprehensive study from the transplant complications working party of the EBMT
Abstract