PMID- 36114629
OWN - NLM
STAT- MEDLINE
DCOM- 20230606
LR  - 20230610
IS  - 2038-2529 (Electronic)
IS  - 0300-8916 (Print)
IS  - 0300-8916 (Linking)
VI  - 109
IP  - 3
DP  - 2023 Jun
TI  - Establishment of 6 pediatric rhabdomyosarcoma patient's derived xenograft models 
      closely recapitulating patients' tumor characteristics.
PG  - 314-323
LID - 10.1177/03008916221110266 [doi]
AB  - INTRODUCTION: The prognosis for patients with metastatic and recurrent pediatric 
      rhabdomyosarcoma (RMS) remains poor. The availability of preclinical models is 
      essential to identify promising treatments We established a series of pediatric 
      RMS patient derived xenografts (PDXs), all faithfully mirroring primary tumor 
      characteristics and representing a unique tool for clarifying the biological 
      processes underlying RMS progression and relapse. METHODS: Fresh tumor samples 
      from 12 RMS patients were implanted subcutaneously in both flanks of 
      immunocompromised mice. PDXs were considered as grafted after accomplishing three 
      passages in mice. Characterization of tumor tissues and models was performed by 
      comparing both morphology and immunoistochemical and fluorescence in situ 
      hybridization (FISH) characteristics. RESULTS: Six PDXs were established, with a 
      successful take rate of 50%. All models closely mirrored parental tumor 
      characteristics. An increased grafting rate for tumors derived from patients with 
      worse outcome (p = 0.006) was detected. For 50% PDXs grafting occurred when the 
      corresponding patient was still alive. CONCLUSION: Our findings increase the 
      number of available RMS PDX models and strengthen the role of PDXs as useful 
      preclinical tools for patients with unmet medical needs and to develop 
      personalized therapies.
FAU - Gasparini, Patrizia
AU  - Gasparini P
AUID- ORCID: 0000-0002-9548-3724
AD  - Tumor Genomics Unit, Department of Research, Fondazione IRCCS Istituto Nazionale 
      dei Tumori, Milan, Italy.
FAU - Casanova, Michela
AU  - Casanova M
AD  - Paediatric Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy.
FAU - Centonze, Giovanni
AU  - Centonze G
AD  - First Pathology Division, Department of Pathology and Laboratory Medicine, 
      Fondazione IRCCS Istituto Nazionale dei Tumori di Milano, Milan, Italy.
FAU - Borzi, Cristina
AU  - Borzi C
AUID- ORCID: 0000-0003-2850-6559
AD  - Tumor Genomics Unit, Department of Research, Fondazione IRCCS Istituto Nazionale 
      dei Tumori, Milan, Italy.
FAU - Bergamaschi, Luca
AU  - Bergamaschi L
AD  - Paediatric Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy.
FAU - Collini, Paola
AU  - Collini P
AD  - Soft Tissue and Bone Pathology, Histopathology and Pediatric Pathology Unit, 
      Department of Diagnostic Pathology and Laboratory Medicine, Fondazione IRCCS 
      Istituto Nazionale dei Tumori, Milan, Italy.
FAU - Testi, Adele
AU  - Testi A
AD  - Laboratory of Molecular Pathology, Department of Pathology, Fondazione IRCCS 
      Istituto Nazionale dei Tumori, Milan, Italy.
FAU - Chiaravalli, Stefano
AU  - Chiaravalli S
AD  - Paediatric Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy.
FAU - Massimino, Maura
AU  - Massimino M
AD  - Paediatric Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy.
FAU - Sozzi, Gabriella
AU  - Sozzi G
AD  - Tumor Genomics Unit, Department of Research, Fondazione IRCCS Istituto Nazionale 
      dei Tumori, Milan, Italy.
FAU - Ferrari, Andrea
AU  - Ferrari A
AUID- ORCID: 0000-0002-4724-0517
AD  - Paediatric Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy.
FAU - Moro, Massimo
AU  - Moro M
AUID- ORCID: 0000-0003-2562-4007
AD  - Tumor Genomics Unit, Department of Research, Fondazione IRCCS Istituto Nazionale 
      dei Tumori, Milan, Italy.
LA  - eng
PT  - Journal Article
DEP - 20220916
PL  - United States
TA  - Tumori
JT  - Tumori
JID - 0111356
SB  - IM
MH  - Humans
MH  - Animals
MH  - Mice
MH  - Xenograft Model Antitumor Assays
MH  - Heterografts
MH  - In Situ Hybridization, Fluorescence
MH  - Prognosis
MH  - *Rhabdomyosarcoma/genetics
MH  - Disease Models, Animal
PMC - PMC10248287
OTO - NOTNLM
OT  - Rhabdomyosarcoma
OT  - patient-derived xenografts
OT  - personalized therapy
COIS- The author(s) declared no potential conflicts of interest with respect to the 
      research, authorship, and/or publication of this article.
EDAT- 2022/09/18 06:00
MHDA- 2023/06/06 06:42
PMCR- 2023/06/08
CRDT- 2022/09/17 00:52
PHST- 2023/06/06 06:42 [medline]
PHST- 2022/09/18 06:00 [pubmed]
PHST- 2022/09/17 00:52 [entrez]
PHST- 2023/06/08 00:00 [pmc-release]
AID - 10.1177_03008916221110266 [pii]
AID - 10.1177/03008916221110266 [doi]
PST - ppublish
SO  - Tumori. 2023 Jun;109(3):314-323. doi: 10.1177/03008916221110266. Epub 2022 Sep 
      16.