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December 8, 2025BloodOpen Access

Optimizing establishment of high need pediatric Acute Myeloid Leukemia (AML) patient derived xenograft (PDX) models

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Authors

MRMax RochetteSGS GollapudiASAlexandra M. Stevens

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Overview

Evaluating engraftment efficacy and disease burden in pediatric AML using patient-derived xenografts and immunodeficient mice.

Key Points

  • To optimize engraftment and establish more pediatric acute myeloid leukemia patient-derived xenograft models.
  • Utilized patient samples with rare genetic fusions for engraftment in immunodeficient mice.
  • Samples conditioned with busulfan before being injected into NRGS and MISTRG mice.
  • Engraftment tracked through flow cytometry and clinical monitoring of mice.
  • Four of twelve viable patient samples achieved engraftment (33%).
  • Engrafted cells showed significant cell expansion, particularly CBFA2T3::GLIS2 (517 fold increase).
  • Disease burden was primarily in bone marrow, indicating difficulty in identifying extramedullary disease.

Cite This Study

Rochette et al. (2025) studied this question.

synapsesocial.com/papers/69362f604fa91c937236dccdhttps://doi.org/10.1182/blood-2025-1515
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Abstract P38: Expanding the repertoire of physiologically relevant AML models to support new target drug discovery, compound screening, and predicting patient responses in clinical trials2024
  2. 2Addressing a Pre-Clinical Pipeline Gap: Development of the Pediatric Acute Myeloid Leukemia Patient-Derived Xenograft Program at Texas Children’s Hospital at Baylor College of Medicine2024 · 9 citations
  3. 3Abstract 4515: Clinically relevant AML modeling with <i>in vitro</i> bone marrow niche and <i>in vivo</i> PDX approaches.2026
  4. 4A platform of robust patient-derived leukemia models covering subgroups for which no cell lines exist2025 · 1 citations
  5. 5Advancing Precision Therapy in Pediatric Acute Myeloid Leukemia Through PDX Models and Mitochondrial Targeting2026