Single-cell sequencing on PBMCs from patients with HA and HB with inhibitors reveals different immune responses to FVIII and FIX.

ABSTRACT:

Abstract

Inhibitors are the most severe complication of replacement therapy in patients with hemophilia. Previous studies, along with our clinical observations, have identified distinct incidence rates and clinical manifestations of factor VIII (FVIII) and FIX inhibitors in patients with severe hemophilia A (HA) and HB. To explore different immune responses to FVIII and FIX in patients with HA and HB and elucidate the mechanisms underlying the varying clinical manifestations of these patients, we performed single-cell sequencing on peripheral blood mononuclear cells (PBMCs) collected from 5 patients with HA and 5 with HB with inhibitors. After quality control, a total of 75 051 cells were clustered into 19 subsets. Transcriptome analysis revealed differences in the composition of l

SUBMITTER: Li E 

PROVIDER: S-EPMC12337186 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature