TY - JOUR
T1 - Frontline Hyper-CVAD Plus Venetoclax for Pediatric Blastic Plasmacytoid Dendritic Cell Neoplasm
AU - He, Jiasen
AU - Garcia, Miriam B.
AU - Connors, Jeremy S.
AU - Nuñez, Cesar A.
AU - Quesada, Andres E.
AU - Gibson, Amber
AU - Roth, Michael
AU - Cuglievan, Branko
AU - Pemmaraju, Naveen
AU - McCall, David
N1 - Publisher Copyright:
© 2023 Wolters Kluwer Health, Inc. All rights reserved.
PY - 2023/11/1
Y1 - 2023/11/1
N2 - Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy, especially in pediatrics, that can involve the bone marrow, skin, lymph nodes, and central nervous system (CNS). Given its variable clinical presentation, coupled with an immunohistochemistry pattern (CD4, CD56, TCF4, TCL-1, and CD123 positivity) that differs from other myeloid neoplasms, the diagnosis of BPDCN can be missed. Limited data are available to guide the treatment of pediatric BPDCN. Herein, we report a case of a pediatric patient who had BPDCN with central nervous system, orbital, and skin involvement. This patient achieved complete remission after receiving modified hyper-CVAD (hyperfractionated cyclophosphamide, vincristine, doxorubicin, and dexamethasone with venetoclax and intrathecal chemotherapy. He remains disease-free 200 days after receiving a stem cell transplant. This represents the first known published pediatric case using a modified hyper-CVAD plus venetoclax regimen for treating a pediatric BPDCN patient in the frontline setting.
AB - Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy, especially in pediatrics, that can involve the bone marrow, skin, lymph nodes, and central nervous system (CNS). Given its variable clinical presentation, coupled with an immunohistochemistry pattern (CD4, CD56, TCF4, TCL-1, and CD123 positivity) that differs from other myeloid neoplasms, the diagnosis of BPDCN can be missed. Limited data are available to guide the treatment of pediatric BPDCN. Herein, we report a case of a pediatric patient who had BPDCN with central nervous system, orbital, and skin involvement. This patient achieved complete remission after receiving modified hyper-CVAD (hyperfractionated cyclophosphamide, vincristine, doxorubicin, and dexamethasone with venetoclax and intrathecal chemotherapy. He remains disease-free 200 days after receiving a stem cell transplant. This represents the first known published pediatric case using a modified hyper-CVAD plus venetoclax regimen for treating a pediatric BPDCN patient in the frontline setting.
KW - CNS involvements
KW - modified hyper-CVAD
KW - pediatric BPDCN
KW - venetoclax
UR - http://www.scopus.com/inward/record.url?scp=85175356250&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85175356250&partnerID=8YFLogxK
U2 - 10.1097/MPH.0000000000002748
DO - 10.1097/MPH.0000000000002748
M3 - Article
C2 - 37661300
AN - SCOPUS:85175356250
SN - 1077-4114
VL - 45
SP - E1001-E1004
JO - Journal of Pediatric Hematology/Oncology
JF - Journal of Pediatric Hematology/Oncology
IS - 8
ER -