Single-cell landscape of hematopoiesis and immunological responses in patients with severe aplastic anemia treated with immunosuppression and eltrombopag

We performed single cell RNA sequencing, and VDJ sequencing of TCR and BCR of bone marrow samples (BMMMNCs and sorted CD34+ HSPCs) from 20 patients with severe aplastic anemia (SAA), pre- and post-treatment, to understand disease pathogenesis and response to treatment. RNA profiles of single bone marrow hematopoietic cells of 20 SAA patients and 4 healthy donors were generated by deep sequencing using Illumina NovaSeq.