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© 2021. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.

Abstract

Purpose

To describe a series of children with extensive PNF or treatment refractory PLGG treated on a compassionate basis with trametinib.

Methods

We report on six patients with NF‐1 treated with trametinib on a compassionate basis at British Columbia Children's Hospital since 2017. Data were collected retrospectively from the patient record. RAPNO and volumetric criteria were used to evaluate the response of intracranial and extracranial lesions, respectively.

Results

Subjects were 21 months to 14 years old at the time of initiation of trametinib therapy and 3/6 subjects are male. Duration of therapy was 4–28 months at the time of this report. All patients had partial response or were stable on analysis. Two patients with life‐threatening PNF had a partial radiographic response in tandem with significant clinical improvement and developmental catch up. One subject discontinued therapy after 6 months due to paronychia and inadequate response. The most common adverse effect (AE) was grade 1–2 paronychia or dermatitis in 5/6 patients. There were no grade 3 or 4 AEs. At the time of this report, five patients remain on therapy.

Conclusion

Trametinib is an effective therapy for advanced PNF and refractory PLGG in patients with NF‐1 and is well tolerated in children. Further data and clinical trials are required to assess tolerance, efficacy and durability of response, and length of treatment required in such patients.

Details

Title
Trametinib therapy for children with neurofibromatosis type 1 and life‐threatening plexiform neurofibroma or treatment‐refractory low‐grade glioma
Author
Ronsley, Rebecca 1   VIAFID ORCID Logo  ; Hounjet, Celine D 2 ; Cheng, Sylvia 1 ; Rassekh, Shahrad Rod 1   VIAFID ORCID Logo  ; Duncan, Walter J 3 ; Dunham, Christopher 4 ; Gardiner, Jane 5 ; Ghag, Arvindera 6 ; Ludemann, Jeffrey P 7 ; Wensley, David 8 ; Wingfield Rehmus 9 ; Sargent, Michael A 10 ; Hukin, Juliette 11 

 Division of Hematology, Oncology & BMT, Department of Pediatrics, University of British Columbia, Vancouver, Canada 
 Faculty of Medicine, University of British Columbia, Vancouver, Canada 
 Division of Pediatric Cardiology, Department of Pediatrics, University of British Columbia, Vancouver, Canada 
 Division of Anatomic Pathology, Department of Pathology, University of British Columbia, Vancouver, Canada 
 Division of Pediatric Ophthalmology, Department of Surgery, University of British Columbia, Vancouver, Canada 
 Division of Pediatric Orthopedic Surgery, Department of Surgery, University of British Columbia, Vancouver, Canada 
 Division of Pediatric Otolaryngology, Department of Surgery, University of British Columbia, Vancouver, Canada 
 Division of Pediatric Respiratory Medicine, Department of Pediatrics, University of British Columbia, Vancouver, Canada 
 Division of Dermatology, Department of Pediatrics, University of British Columbia, Vancouver, Canada 
10  Division of Pediatric Neuro‐Radiology, Department of Radiology, University of British Columbia, Vancouver, Canada 
11  Division of Hematology, Oncology & BMT, Department of Pediatrics, University of British Columbia, Vancouver, Canada; Division of Neurology, Department of Pediatrics, University of British Columbia, Vancouver, Canada 
Pages
3556-3564
Section
CLINICAL CANCER RESEARCH
Publication year
2021
Publication date
Jun 2021
Publisher
John Wiley & Sons, Inc.
e-ISSN
20457634
Source type
Scholarly Journal
Language of publication
English
ProQuest document ID
2537026110
Copyright
© 2021. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.