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What Is the Role of HSCT in Pediatric, Young Adult Patients With BPDCN?

By Melissa Badamo - Last Updated: April 5, 2024

The role of hematopoietic stem cell transplant (HSCT) in pediatric, adolescent, and young adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) remains unclear, according to a recent study presented at the 2024 American Society of Pediatric Hematology/Oncology (ASPHO) Conference.

Jeremy Connors, MD, of the University of Texas MD Anderson Cancer Center, and colleagues studied HSCT as consolidative treatment in this patient population because “the prognostic indicators have not been established, hindering standardized treatment approaches, and rendering the role and timing of HSCT ambiguous.”

The investigators reviewed 21 case reports of patients 20 years old and younger who underwent therapy for BPDCN, including HSCT. The median age was nine years.

As initial therapy, 10 patients received acute lymphoblastic leukemia-based regimens, four received acute myeloid leukemia-based regimens, four received non-Hodgkin lymphoma-based regimens, two received CHOP-based regimens, and one received tagraxofusp monotherapy.

Eighteen patients (85.7%) were in complete remission at the time of HSCT, and three patients had persistent disease. Following first and second relapse, two patients underwent a second HSCT.

The median overall survival was 16 months. Three patients died, one from pulmonary toxicity after conditioning and two from therapy-related myeloid disease.

“While there is more success in the initial therapy of these patients, especially when an acute lymphoid leukemia-based regimen is utilized, definitive indicators of increased risk for relapse are illusive,” Dr. Connors and colleagues wrote. “Further study is needed as to predictors of relapse, and thus those who would benefit most from early HSCT.”



Connors J, Ramakrishnan R, Elgehiny A, et al. “Hematopoietic stem cell transplant in the pediatric/AYA blastic plasmacytoid dendritic cell neoplasm.” Poster #408. Presented at the 2024 American Society of Pediatric Hematology/Oncology (ASPHO) Conference; April 3–6, 2024; Seattle, Washington.

Original Source: What Is the Role of HSCT in Pediatric, Young Adult Patients With BPDCN? | Blood Cancers Today

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