Response to venetoclax in patients with blastic plasmacytoid dendritic cell neoplasm

Kirsty M. Sharplin; Catherine Vassiliou; James Nguyen; Bryone J. Kuss; Stephen B. Ting; Hilda Mangos; Hamish Scott; Anna Brown; Piers Blombery; Andrew Wei; David M. Ross

doi:10.1016/j.leukres.2025.107678

Response to venetoclax in patients with blastic plasmacytoid dendritic cell neoplasm

Kirsty M. Sharplin, Catherine Vassiliou, James Nguyen, Bryone J. Kuss, Stephen B. Ting, Hilda Mangos, Hamish Scott, Anna Brown, Piers Blombery, Andrew Wei, David M. Ross

College of Medicine and Public Health

Research output: Contribution to journal › Letter › peer-review

Abstract

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) is a rare, aggressive hematopoietic neoplasm with a median survival of 12 months. Cutaneous involvement occurs in > 90 % of cases and few cases present solely as leukemia. Central nervous system (CNS) involvement is common. Treatment approaches lack consensus. Tagraxofsp-erzs, a CD123-directed cytotoxin, is the only approved targeted therapy. Chemotherapy regimens for acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), or lymphoma are often repurposed. Durable remissions are seen only with allogeneic haematopoietic stem-cell transplantation (allo-HSCT).

BCL2 overexpression in BPDCN supports the rationale for venetoclax use, first validated in murine models. Herein we report five additional cases, adding to nineteen previously reported cases of venetoclax use in BPDCN and examine the genetic underpinnings and alternative anti-apoptotic protein upregulation following venetoclax therapy.

Original language	English
Article number	107678
Number of pages	7
Journal	Leukemia Research
Volume	152
DOIs	https://doi.org/10.1016/j.leukres.2025.107678
Publication status	Published - May 2025

Keywords

Blastic Plasmacytoid Dendritic Cell Neoplasm
hematopoietic neoplasms
patient outcomes
case studies

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title = "Response to venetoclax in patients with blastic plasmacytoid dendritic cell neoplasm",

abstract = "Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) is a rare, aggressive hematopoietic neoplasm with a median survival of 12 months. Cutaneous involvement occurs in > 90 % of cases and few cases present solely as leukemia. Central nervous system (CNS) involvement is common. Treatment approaches lack consensus. Tagraxofsp-erzs, a CD123-directed cytotoxin, is the only approved targeted therapy. Chemotherapy regimens for acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), or lymphoma are often repurposed. Durable remissions are seen only with allogeneic haematopoietic stem-cell transplantation (allo-HSCT).BCL2 overexpression in BPDCN supports the rationale for venetoclax use, first validated in murine models. Herein we report five additional cases, adding to nineteen previously reported cases of venetoclax use in BPDCN and examine the genetic underpinnings and alternative anti-apoptotic protein upregulation following venetoclax therapy.",

keywords = "Blastic Plasmacytoid Dendritic Cell Neoplasm, hematopoietic neoplasms, patient outcomes, case studies",

author = "Sharplin, {Kirsty M.} and Catherine Vassiliou and James Nguyen and Kuss, {Bryone J.} and Ting, {Stephen B.} and Hilda Mangos and Hamish Scott and Anna Brown and Piers Blombery and Andrew Wei and Ross, {David M.}",

year = "2025",

month = may,

doi = "10.1016/j.leukres.2025.107678",

language = "English",

volume = "152",

journal = "Leukemia Research",

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T1 - Response to venetoclax in patients with blastic plasmacytoid dendritic cell neoplasm

AU - Sharplin, Kirsty M.

AU - Vassiliou, Catherine

AU - Nguyen, James

AU - Kuss, Bryone J.

AU - Ting, Stephen B.

AU - Mangos, Hilda

AU - Scott, Hamish

AU - Brown, Anna

AU - Blombery, Piers

AU - Wei, Andrew

AU - Ross, David M.

PY - 2025/5

Y1 - 2025/5

N2 - Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) is a rare, aggressive hematopoietic neoplasm with a median survival of 12 months. Cutaneous involvement occurs in > 90 % of cases and few cases present solely as leukemia. Central nervous system (CNS) involvement is common. Treatment approaches lack consensus. Tagraxofsp-erzs, a CD123-directed cytotoxin, is the only approved targeted therapy. Chemotherapy regimens for acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), or lymphoma are often repurposed. Durable remissions are seen only with allogeneic haematopoietic stem-cell transplantation (allo-HSCT).BCL2 overexpression in BPDCN supports the rationale for venetoclax use, first validated in murine models. Herein we report five additional cases, adding to nineteen previously reported cases of venetoclax use in BPDCN and examine the genetic underpinnings and alternative anti-apoptotic protein upregulation following venetoclax therapy.

AB - Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) is a rare, aggressive hematopoietic neoplasm with a median survival of 12 months. Cutaneous involvement occurs in > 90 % of cases and few cases present solely as leukemia. Central nervous system (CNS) involvement is common. Treatment approaches lack consensus. Tagraxofsp-erzs, a CD123-directed cytotoxin, is the only approved targeted therapy. Chemotherapy regimens for acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), or lymphoma are often repurposed. Durable remissions are seen only with allogeneic haematopoietic stem-cell transplantation (allo-HSCT).BCL2 overexpression in BPDCN supports the rationale for venetoclax use, first validated in murine models. Herein we report five additional cases, adding to nineteen previously reported cases of venetoclax use in BPDCN and examine the genetic underpinnings and alternative anti-apoptotic protein upregulation following venetoclax therapy.

KW - Blastic Plasmacytoid Dendritic Cell Neoplasm

KW - hematopoietic neoplasms

KW - patient outcomes

KW - case studies

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U2 - 10.1016/j.leukres.2025.107678

DO - 10.1016/j.leukres.2025.107678

M3 - Letter

AN - SCOPUS:105001713211

SN - 0145-2126

VL - 152

JO - Leukemia Research

JF - Leukemia Research

M1 - 107678

ER -

Response to venetoclax in patients with blastic plasmacytoid dendritic cell neoplasm

Abstract

Keywords

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