TY - JOUR
T1 - Blastic plasmacytoid dendritic cell neoplasm
T2 - a comprehensive review in pediatrics, adolescents, and young adults (AYA) and an update of novel therapies
AU - Cuglievan, Branko
AU - Connors, Jeremy
AU - He, Jiasen
AU - Khazal, Sajad
AU - Yedururi, Sireesha
AU - Dai, Julia
AU - Garces, Sofia
AU - Quesada, Andres E.
AU - Roth, Michael
AU - Garcia, Miriam
AU - McCall, David
AU - Gibson, Amber
AU - Ragoonanan, Dristhi
AU - Petropoulos, Demetrios
AU - Tewari, Priti
AU - Nunez, Cesar
AU - Mahadeo, Kris M.
AU - Tasian, Sarah K.
AU - Lamble, Adam J.
AU - Pawlowska, Anna
AU - Hammond, Danielle
AU - Maiti, Abhishek
AU - Haddad, Fadi G.
AU - Senapati, Jayatsu
AU - Daver, Naval
AU - Gangat, Naseema
AU - Konopleva, Marina
AU - Meshinchi, Soheil
AU - Pemmaraju, Naveen
N1 - Publisher Copyright:
© 2023, The Author(s).
PY - 2023/9
Y1 - 2023/9
N2 - Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy that can involve the bone marrow, peripheral blood, skin, lymph nodes, and the central nervous system. Though more common in older adults, BPDCN has been reported across all age groups, including infants and children. The incidence of pediatric BPDCN is extremely low and little is known about the disease. Pediatric BPDCN is believed to be clinically less aggressive but often with more dissemination at presentation than adult cases. Unlike adults who almost always proceed to a hematopoietic stem cell transplantation in first complete remission if transplant-eligible, the majority of children can be cured with a high-risk acute lymphoblastic leukemia-like regimen. Hematopoietic stem cell transplantation is recommended for children with high-risk disease, the definition of which continues to evolve, or those in relapse and refractory settings where outcomes continue to be dismal. Novel agents used in other hematologic malignancies and CD123 targeted agents, including chimeric antigen receptor T-cells and monoclonal/bispecific antibodies, are being brought into research and practice. Our goal is to provide a comprehensive review of presentation, diagnosis, and treatment by review of pediatric cases reported for the last 20 years, and a review of novel targeted therapies and therapies under investigation for adult and pediatric patients.
AB - Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy that can involve the bone marrow, peripheral blood, skin, lymph nodes, and the central nervous system. Though more common in older adults, BPDCN has been reported across all age groups, including infants and children. The incidence of pediatric BPDCN is extremely low and little is known about the disease. Pediatric BPDCN is believed to be clinically less aggressive but often with more dissemination at presentation than adult cases. Unlike adults who almost always proceed to a hematopoietic stem cell transplantation in first complete remission if transplant-eligible, the majority of children can be cured with a high-risk acute lymphoblastic leukemia-like regimen. Hematopoietic stem cell transplantation is recommended for children with high-risk disease, the definition of which continues to evolve, or those in relapse and refractory settings where outcomes continue to be dismal. Novel agents used in other hematologic malignancies and CD123 targeted agents, including chimeric antigen receptor T-cells and monoclonal/bispecific antibodies, are being brought into research and practice. Our goal is to provide a comprehensive review of presentation, diagnosis, and treatment by review of pediatric cases reported for the last 20 years, and a review of novel targeted therapies and therapies under investigation for adult and pediatric patients.
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U2 - 10.1038/s41375-023-01968-z
DO - 10.1038/s41375-023-01968-z
M3 - Review article
C2 - 37452102
AN - SCOPUS:85164784026
SN - 0887-6924
VL - 37
SP - 1767
EP - 1778
JO - Leukemia
JF - Leukemia
IS - 9
ER -