Details

Autor(en) / Beteiligte

• Pemmaraju, Naveen
• Kantarjian, Hagop
• Sweet, Kendra
• Wang, Eunice
• Senapati, Jayastu
• Wilson, Nathaniel R.
• Konopleva, Marina
• Frankel, Arthur E.
• Gupta, Vikas
• Mesa, Ruben
• Ulrickson, Matthew
• Gorak, Edward
• Bhatia, Sumeet
• Budak-Alpdogan, Tulin
• Mason, James
• Garcia-Romero, Maria Teresa
• Lopez-Santiago, Norma
• Cesarman-Maus, Gabriela
• Vachhani, Pankit
• Lee, Sangmin
• Bhatt, Vijaya Raj
• Blum, William
• Walter, Roland B.
• Bixby, Dale
• Gojo, Ivana
• Duvic, Madeleine
• Rampal, Raajit K.
• de Lima, Marcos
• Foran, James
• Fathi, Amir T.
• Hall, Aric Cameron
• Jacoby, Meagan A.
• Lancet, Jeffrey
• Mannis, Gabriel
• Stein, Anthony S.
• Mims, Alice
• Rizzieri, David
• Olin, Rebecca
• Perl, Alexander
• Schiller, Gary
• Shami, Paul
• Stone, Richard M.
• Strickland, Stephen
• Wieduwilt, Matthew J.
• Daver, Naval
• Ravandi, Farhad
• Vasu, Sumithira
• Guzman, Monica
• Roboz, Gail J.
• Khoury, Joseph
• Qazilbash, Muzaffar
• Aung, Phyu P.
• Cuglievan, Branko
• Madanat, Yazan
• Kharfan-Dabaja, Mohamed A.
• Pawlowska, Anna
• Taylor, Justin
• Tallman, Martin
• Dhakal, Prajwal
• Lane, Andrew A.

Titel

North American Blastic Plasmacytoid Dendritic Cell Neoplasm Consortium: position on standards of care and areas of need

Ist Teil von

• Blood, 2023-02, Vol.141 (6), p.567-578

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2023

Link zum Volltext

Quelle

MEDLINE

Beschreibungen/Notizen

• Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy with historically poor outcomes and no worldwide consensus treatment approach. Unique among most hematologic malignancies for its frequent cutaneous involvement, BPDCN can also invade other extramedullary compartments, including the central nervous system. Generally affecting older adults, many patients are unfit to receive intensive chemotherapy, and although hematopoietic stem cell transplantation is preferred for younger, fit individuals, not all are eligible. One recent therapeutic breakthrough is that all BPDCNs express CD123 (IL3Rα) and that this accessible surface marker can be pharmacologically targeted. The first-in-class agent for BPDCN, tagraxofusp, which targets CD123, was approved in December 2018 in the United States for patients with BPDCN aged ≥2 years. Despite favorable response rates in the frontline setting, many patients still relapse in the setting of monotherapy, and outcomes in patients with relapsed/refractory BPDCN remain dismal. Therefore, novel approaches targeting both CD123 and other targets are actively being investigated. To begin to formally address the state of the field, we formed a new collaborative initiative, the North American BPDCN Consortium (NABC). This group of experts, which includes a multidisciplinary panel of hematologists/oncologists, hematopoietic stem cell transplant physicians, pathologists, dermatologists, and pediatric oncologists, was tasked with defining the current standard of care in the field and identifying the most important research questions and future directions in BPDCN. The position findings of the NABC’s inaugural meetings are presented herein. Pemmaraju and colleagues of the North American Blastic Plasmacytoid Dendritic Cell Neoplasm Consortium summarize the latest biological insights, the importance of an accurate diagnosis, and the data on the activity of targeted agents. The front-line tagraxofusp, a conjugate of the diphtheria toxin and interleukin-3, and venetoclax are among these agents. The investigators also emphasize the role of central nervous system prophylaxis and outline challenges in developing optimized regimens to treat this rare and difficult malignancy.