Abstract Details

Title Adult Patients' Experiences With Selumetinib Treatment Versus Placebo for Neurofibromatosis Type One-Plexiform Neurofibroma Associated Symptoms and Their Impacts: A Qualitative Sub-study of a Phase Three, Placebo-controlled Trial (KOMET)

Topic Neuro-oncology

Presentation(s) S13 - Neuro-oncology: Advances in Diagnosis and Treatments (2:12 PM-2:24 PM)

Poster/Presentation
Number 007

Objective To enhance understanding of patient experiences with selumetinib (ARRY-142886, AZD6244; MEK1/2 inhibitor) treatment versus placebo in adults with NF1-PN in KOMET (NCT04924608).

Background KOMET evaluated efficacy and safety of selumetinib in adults with NF1 and symptomatic, inoperable PN. At study initiation, no approved therapies existed.

Design/Methods A qualitative interview sub-study was conducted with KOMET participants in Germany, UK, Italy, Spain, Canada and USA. Trained moderators conducted three 1:1 semi-structured interviews via teleconference (February 2022–December 2024). Participants were enrolled in KOMET and consented to the sub-study.

Results Sub-study participants were aged 18-59 years, 52% were women and 80% White. At interview 1 (baseline: N=25, n=13 selumetinib; n=12 placebo), main locations of target PNs were head/neck (n=7, 28%) and trunk (n=6, 24%). Most common symptoms were chronic pain (80%), sensitivity/numbness (48%), episodic/spike pain (36%), swelling (32%), and itching (28%). Highest reported impacts were on restricted body movements (56%), exercise (52%), and impact on school/work (44%). At interview 2 (cycle 12, N=22 [n=12 selumetinib; n=10 placebo]), all except one selumetinib participants reported improvements in symptom and impact burden with noticeable decreases in pain, tingling, PN size, and resulting improvement in sleep and fatigue and body movements, while placebo participants observed no notable changes. By interview 3 (~20 months post-randomization: N=16, n=8 selumetinib; n=8 crossover [12 cycles placebo + 8 cycles selumetinib]), the observed changes from interview 2 were sustained or further improved for most selumetinib participants. Crossover participants (~8 months on selumetinib post-crossover) reported similar benefits as the selumetinib group participants, including improved sleep, mobility, and a greater ability to engage in physical and social activities.

Conclusions This study provides insights into the experience of adults with NF1-PN during KOMET, and further demonstrates the positive effect of selumetinib on symptoms by adding to the objective parameters and patient-reported outcomes in the primary analysis publication.

Authors/Disclosures
Ayo Adeyemi, PhD PRESENTER	Dr. Adeyemi has received personal compensation for serving as an employee of Alexion. Dr. Adeyemi has or had stock in Alexion.
Angela Swampillai, MBBS, BSc, MRCP, FRCR	Dr. Swampillai has received personal compensation in the range of $0-$499 for serving as a market research with Adivo Associates.
Ignacio Blanco, MD, PhD	Dr. Blanco has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Blanco has received personal compensation in the range of $500-$4,999 for serving as a Consultant for SpringWorks. The institution of Dr. Blanco has received research support from Asociació Catalana Neurofibromatosis (AcNefi). The institution of Dr. Blanco has received research support from Spanish Health Ministry.
Carolina Barnett Tapia, MD	Dr. Barnett Tapia has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Barnett Tapia has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NMD pharma. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for argenx. The institution of Dr. Barnett Tapia has received research support from Department of defense. The institution of Dr. Barnett Tapia has received research support from Muscular dystrophy canada. The institution of Dr. Barnett Tapia has received research support from MgNet. The institution of Dr. Barnett Tapia has received research support from NIH. The institution of Dr. Barnett Tapia has received research support from Cartesian. The institution of Dr. Barnett Tapia has received research support from Alexion. Dr. Barnett Tapia has received intellectual property interests from a discovery or technology relating to health care.
Yoomin Lee, Medical Student	Miss Lee has nothing to disclose.
Angela M. Mastronuzzi, MD, PhD	Dr. Mastronuzzi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ipsen.
Cordula Matthies, MD, PhD	Prof. Matthies has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Boston Scientific. Prof. Matthies has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion/ Astra Zeneca. Prof. Matthies has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Boston Scientific. The institution of Prof. Matthies has received research support from German Research Council.
silverio perrotta, MD	Prof. perrotta has nothing to disclose.
Marica Eoli, MD	Dr. Eoli has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion.
Hans H. Shuhaiber, MD	The institution of Dr. Shuhaiber has received research support from SPRINGWORKS . The institution of Dr. Shuhaiber has received research support from RECURSION . The institution of Dr. Shuhaiber has received research support from AstraZeneca. The institution of Dr. Shuhaiber has received research support from CTF. The institution of Dr. Shuhaiber has received research support from NFLECTION .
Gail Doughton, PhD	Dr. Doughton has received personal compensation for serving as an employee of AstraZeneca. An immediate family member of Dr. Doughton has received personal compensation for serving as an employee of AstraZeneca. Dr. Doughton has stock in AstraZeneca. An immediate family member of Dr. Doughton has stock in AstraZeneca.
Randolph de la Rosa Rodriguez, MD	Dr. de la Rosa Rodriguez has received personal compensation for serving as an employee of Alexion. Dr. de la Rosa Rodriguez has stock in Astrazeneca.
Said C. Farschtschi, MD	Dr. Farschtschi has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion Pharma. Dr. Farschtschi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for SpringWorks Therapeutics. The institution of Dr. Farschtschi has received research support from Bundesverband Neurofibromatose Germany. The institution of Dr. Farschtschi has received research support from Nothing is Forever e.V. Germany.

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