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Prolonged response after stopping TPO-RA in ITP, TP53 mutations and myelofibrosis outcomes, clinical picture of ERCC6L2 disease, where variants predispose to marrow failure and malignancy

Blood Podcast

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The Role of T-PoRA's in Sustained Responses to Initial Treatment

Of those patients who were retreated with T-PoRA, 11 out of 12 achieved a complete response. The authors did not identify any significant clinical predictors of sustained response off treatment at week 24. However, single cell RNA sequencing experiments revealed and enhanced TNF alpha signaling via NF Kappa B signature in CD8 positive T cells that relapsed after T- PoRA discontinuation.

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