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Outcomes for children with recurrent/refractory atypical teratoid rhabdoid tumor: A single institution study with molecular correlation
  • +17
  • Santhosh A. Upadhyaya,
  • Steven S. Carey,
  • Jie Huang,
  • Jason R. Myers,
  • Roya Mostafavi,
  • Brent Orr,
  • Sandeep Kumar Dhanda,
  • Layna H. Michalik,
  • Ruth Tatevossian,
  • Paul Klimo,
  • Frederick Boop,
  • Congyu Lu,
  • Edgar Sioson,
  • Xin Zhou,
  • Kim Nichols,
  • Thomas Merchant,
  • David Ellison,
  • Giles Robinson,
  • Arzu Onar,
  • Amar Gajjar
Santhosh A. Upadhyaya
University of Michigan Department of Pediatrics

Corresponding Author:[email protected]

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Steven S. Carey
St Jude Children's Research Hospital
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Jie Huang
St Jude Children's Research Hospital
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Jason R. Myers
St Jude Children's Research Hospital
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Roya Mostafavi
Le Bonheur Children's Hospital
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Brent Orr
St Jude Children's Research Hospital
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Sandeep Kumar Dhanda
St Jude Children's Research Hospital Department of Oncology
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Layna H. Michalik
St Jude Children's Research Hospital Department of Oncology
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Ruth Tatevossian
St Jude Children's Research Hospital Department of Oncology
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Paul Klimo
St Jude Children's Research Hospital
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Frederick Boop
St Jude Children's Research Hospital
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Congyu Lu
St Jude Children's Research Hospital
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Edgar Sioson
St Jude Children's Research Hospital
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Xin Zhou
St Jude Children's Research Hospital
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Kim Nichols
St Jude Children's Research Hospital Department of Oncology
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Thomas Merchant
St Jude Children's Research Hospital Department of Radiation Oncology
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David Ellison
St Jude Children's Research Hospital
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Giles Robinson
St Jude Children's Research Hospital Department of Oncology
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Arzu Onar
St Jude Children's Research Hospital
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Amar Gajjar
St Jude Children's Research Hospital Department of Oncology
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Abstract

Background: Survival data for recurrent or treatment refractory pediatric atypical teratoid rhabdoid tumor (AT/RT), and its association to molecular groups is extremely limited. Methods: Single-institution retrospective study of sixty-four children <21 years old with AT/RT that was recurrent or refractory to frontline therapies (PD) treated at St. Jude Children’s Research Hospital from January 2000 to December 2020. Demographic, clinicopathologic, treatment, molecular grouping (SHH, TYR and MYC) and germline SMARCB1/SMARCA4 mutational data were collected. Progression-free survival (PFS2: time from initial PD to subsequent first progression) and overall survival (OSpostPD: time from PD to death/last follow up) were estimated by Kaplan–Meier analysis. Results: Median age at and time from initial diagnosis to PD were 2.1 years (range: 0.5-17.9 years) and 5.4 months (range: 0.5-125.6 months), respectively. Only 5/64 children (7.8%) are alive at median follow-up of 10.9 (range: 4.2-18.1) years from PD. The 2/5-year PFS2 and OSpostPD were 3.1% (±1.8%)/1.6% (±1.1%) and 20.3% (±4.8%)/7.3% (±3.5%), respectively. Children with TYR group (n=10) had a better OSpostPD compared to those with MYC (n=11) (2-year survival estimates: 60.0% ±14.3% vs. 18.2% ±9.5%; p=0.019), or those with SHH (n=21; 4.8% ±3.3%; p=0.014). In univariate analyses, OSpostPD was better with older age at diagnosis (p=0.037), female gender (p=0.008), and metastatic site of PD compared to local or combined sites of PD (p<0.001). Conclusions: Children with recurrent/refractory AT/RT have dismal outcomes. Older age at diagnosis, female gender, TYR group, and metastatic site of PD were associated with relatively longer survival in our study.
06 Mar 2024Submitted to Pediatric Blood & Cancer
06 Mar 2024Submission Checks Completed
06 Mar 2024Assigned to Editor
06 Mar 2024Review(s) Completed, Editorial Evaluation Pending
18 Mar 2024Reviewer(s) Assigned
09 Jun 2024Submission Checks Completed
09 Jun 2024Assigned to Editor
10 Jun 2024Review(s) Completed, Editorial Evaluation Pending
10 Jun 2024Reviewer(s) Assigned
04 Jul 2024Editorial Decision: Accept