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Towards graft- versus-host disease-free alternative donor transplant platforms for patients with acquired aplastic anemia

Author(s): 
Oved JH, Bacigalupo A, DeZern AE
Primary Author: 
Oved JH
Journal Title: 
Haematologica
Original Publication Date: 
May 2025

Hematopoietic stem cell transplantation is a well-established treatment option for acquired

What are the most important quality of life domains for patients with aplastic anemia and paroxysmal nocturnal hemoglobinuria?

Author(s): 
Taylor KJ, Singer S, Langemeijer S, Kelly RJ, Arnold L, Panse J, Patriquin CJ, Nishimura JI, Piggin M, Burmester PO
Primary Author: 
Taylor KJ
Journal Title: 
Annals of Hematology
Original Publication Date: 
May 2025

Disease Burden at the Time of Transplantation Is a Primary Predictor of Outcomes in Pediatric MDS: A Single-Center Experience

Author(s): 
Dahlberg A, Stevenson P, Bhatt NS, Burroughs L, Carpenter PA, Summers C, Tarlock K, Thakar MS, Milano F, Deeg HJ, Bleakley M
Primary Author: 
Dahlberg A
Journal Title: 
Cancers
Original Publication Date: 
May 2025

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.

Bone Marrow Disease(s): 

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