Venetoclax monotherapy as front-line therapy for blastic plasmacytoid dendritic cell neoplasm

Ankit Mitesh Shah; Denise Pereira; Julio Poveda; Trent Peng Wang

doi:10.1016/j.lrr.2023.100380

Venetoclax monotherapy as front-line therapy for blastic plasmacytoid dendritic cell neoplasm

Leuk Res Rep. 2023 Jul 3:20:100380. doi: 10.1016/j.lrr.2023.100380. eCollection 2023.

Authors

Ankit Mitesh Shah¹, Denise Pereira², Julio Poveda³, Trent Peng Wang²

Affiliations

¹ University of Miami Miller School of Medicine, Miami, FL, USA.
² Division of Transplantation & Cellular Therapy, University of Miami Miller School of Medicine, Sylvester Comprehensive Cancer Center, 1411 NW 12th Ave, Suite 231, Miami, FL 33136, USA.
³ Department of Pathology, University of Miami Miller School of Medicine, Miami, FL, USA.

Abstract

Venetoclax is an approved treatment for relapsed/refractory Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). We report a unique case of venetoclax monotherapy used for front-line induction and as a bridge to allogeneic hematopoietic stem cell transplantation (HCT). Venetoclax therapy resulted in rapid complete resolution of skin lesions, however, treatment interruption due to neutropenia led to brisk cancer recurrence. Fortunately, the patient responded to re-challenge and was able to undergo HCT. Venetoclax is active in the first-line treatment setting for BPDCN, however its effect on blood counts and durability of response should be further studied.

Keywords: Allogeneic hematopoietic stem cell transplantation; Blastic plasmacytoid dendritic cell neoplasm; Targeted therapy; Venetoclax.

Publication types

Case Reports

Grants and funding

K12 CA226330/CA/NCI NIH HHS/United States