Related Experiment Video

Updated: Feb 11, 2026

Fluorescence-activated Cell Sorting for Purification of Plasmacytoid Dendritic Cells from the Mouse Bone Marrow
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Fluorescence-activated Cell Sorting for Purification of Plasmacytoid Dendritic Cells from the Mouse Bone Marrow

Published on: November 4, 2016

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First pediatric experience of SL-401, a CD123-targeted therapy, in patients with blastic plasmacytoid dendritic cell

Weili Sun1, Huaying Liu2,3, Young Kim4

  • 1Department of Pediatrics, City of Hope National Medical Center, 1500 E. Duarte Road, Duarte, CA, 91010, USA. weilisun@coh.org.

Journal of Hematology & Oncology
|May 4, 2018
PubMed

Insights

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare cancer. SL-401 shows promise as a well-tolerated targeted therapy for children with BPDCN, demonstrating transient responses in some cases.

Area of Science:

  • Hematology
  • Oncology
  • Pediatric Oncology

Background:

  • Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive hematologic malignancy with poor prognosis.
  • Current treatment options for pediatric BPDCN are limited and lack standardization.
  • SL-401 targets CD123, a protein highly expressed on BPDCN cells, showing efficacy in adult trials.

Observation:

  • This study presents the first pediatric experience with SL-401 in three children diagnosed with BPDCN.
  • All patients tolerated SL-401 treatment without significant adverse events.
  • One patient with refractory disease did not respond to SL-401.

Findings:

  • Two out of three pediatric patients experienced rapid clinical improvement after SL-401 treatment.
  • Observed responses were transient, with tumor regrowth noted between treatment cycles in patients with high tumor burden.
  • SL-401 was generally well-tolerated in the pediatric cohort.

Implications:

  • SL-401 demonstrates potential as a targeted therapy for pediatric BPDCN.
  • Further investigation of SL-401 in pediatric BPDCN is warranted.
  • Understanding response duration and managing tumor burden are key for future pediatric trials.
Abstract

Keywords:
BPDCNPediatricSL-401Targeted therapy

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