Abstract
What is this summary about?
This summary describes a publication about a study called SPRINT. The SPRINT study included 50 children with neurofibromatosis type 1 (NF1) and plexiform neurofibroma (PN) that could not be removed with surgery. PNs are tumors that grow along nerves and can cause various problems for children, such as pain, changes to appearance, and muscle weakness. In SPRINT, the study team wanted to learn whether a medication called selumetinib was able to shrink the PN caused by NF1 (also known as NF1-related PN), and if shrinking PNs helped relieve children of the problems caused by it. To assess how selumetinib might help, children had scans to measure the size of their PN, completed questionnaires, and had a variety of other tests done by their doctor. Their caregivers also completed questionnaires about their child. The children took selumetinib capsules twice a day on an empty stomach.
What were the results?
The results showed that selumetinib was able to shrink the PN for most children (68%). The results also showed that the problems caused by the children’s PNs mostly improved while on selumetinib treatment. SPRINT also showed that the side effects of selumetinib were mainly mild and could be managed by doctors.
What do the results mean?
Before SPRINT, there were not many treatment options for children with NF1 and PN as there were no medications that had been shown to shrink PN, and surgery was not always possible. SPRINT showed that this medication shrinks most PNs and could help children with NF1 and PN. In April 2020, selumetinib was approved by the US Food and Drug Administration (FDA) because of the results of SPRINT. Selumetinib was the first and, as of February 2024, is the only medicine that can be prescribed by doctors to help children with NF1-related PN.
Clinical Trial Registration: NCT01362803 (SPRINT) (ClinicalTrials.gov)
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Conflicts of interest disclosure
Andrea M. Gross has served as an unpaid scientific advisor for Alexion, AstraZeneca Rare Disease, and SpringWorks Therapeutics. Brigitte C. Widemann has served as an unpaid scientific advisor for Alexion, AstraZeneca Rare Disease, and SpringWorks Therapeutics. Pamela L. Wolters, Andrea Baldwin, Colette Achée, Sarah E. Hart, and Lindsay Brewer declare no conflicts of interest.
Financial disclosure
This study was funded by the following organizations: AstraZeneca through a Cooperative Research and Development Agreement with the NCI CTEP (provision of selumetinib and funding for the pharmacokinetic analysis and clinical trial support), a grant (U54 CA196519-04) from the Developmental and Hyperactive Ras Tumor (DHART) Specialized Program of Research Excellence (funding for the pharmacodynamic studies), and the Neurofibromatosis Therapeutic Acceleration Program (NTAP) (funding for trial conduct at the Children’s Hospital of Philadelphia and Cincinnati Children’s Hospital Medical Center).
Writing disclosure
Editorial/medical writing assistance in the preparation of this PLSP was provided by Emily Clark, PhD and Connie Feyerherm, MSci of OPEN Health Communications (London, UK), with financial support from Alexion, AstraZeneca Rare Disease.
Open access
This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 Unported License. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/
Acknowledgments
We would like to express our thanks to the SPRINT study team: E. Dombi, P. Whitcomb, M.J. Fisher, B. Weiss, A.R. Kim, M. Bornhorst, A.C. Shah, S. Martin, S.M. Paul, K. Heisey, M. Smith, J.O. Blakeley, S.M. Steinberg, and D.J. Venzon.