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.
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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
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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.
After a long battle with illness and seeking many specialists, I emphasize the need to self-advocate for ongoing care following my cancer diagnosis.
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Abstract
Thanks to the efforts of our AAMDSIF bone marrow failure community and coalition partners, a bipartisan group of 105 U.S.