Abstract Details

Title Using Qualitative Interviews to Improve Quantitative Assessments of Quality of Life in Patients with Neurofibromatosis 2

Topic Neuro-oncology

Presentation(s) P7 - Poster Session 7 (8:00 AM-9:00 AM)

Poster/Presentation
Number 11-006

Objective To use qualitative interviews to elucidate seemingly discordant quantitative ratings in patient reported outcomes for quality of life (QOL).

Background

Neurofibromatosis 2 (NF2) is a progressive, neurogenetic disease. Assessing QOL is important in demonstrating that new drugs improve how NF2 patients feel and function. The NFTI-QOL is the currently recommended measure to assess NF2-specific QOL in clinical trials.

Design/Methods

We interviewed 16 participants enrolled in stage one of the brigatinib arm of INTUITT-NF2 (NCT04374305) –a multicenter, adaptive platform-basket trial targeting progressive NF2-related tumors – after one year of treatment or at time of trial discontinuation.

Interviews included cognitive debriefings of the NFTI-QoL and global impression of change (GIC) scale. We also analyzed NFTI-QoL scores collected over the same time period, using a MCID of ±2 points to classify improvement or worsening in QOL.

Results 11/16 (69%) participants reported a GIC rating discordant with their change in NFTI-QoL scores. 8/13 (62%) of participants who improved on their GIC had stable (N=6) or worsened (N=2) NFTI-QoL scores. This group cited tumor stability as a major contributor to improved GIC ratings and also had lower baseline NFTI-QoL scores (median=8) than those with concordant ratings (median=12). Participants with lower NFTI-QoL scores disproportionally reported that a meaningful improvement in their symptoms would not lead to an accompanying score improvement due to inadequate NFTI-QOL response options.

Conclusions Qualitative interviews revealed two reasons for conflicting patient reported outcome results. In the setting of a progressive disease, participants can view tumor volume stability as an improvement, even with stable or worsening symptoms. Also, the NFTI-QoL may not be capturing improvement in people with a lower symptom burden. In order for the NFTI-QoL and GIC to better reflect changes in NF2 patients QOL, we recommend adding another response option to the NFTI-QoL and altering the instructions of the GIC.

Authors/Disclosures
Liesel Von Imhof, Other (Massachusetts General Hospital) PRESENTER	Ms. Von Imhof has nothing to disclose.
Scott R. Plotkin, MD, PhD, FAAN (Mass General Cancer Center)	Dr. Plotkin has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for NFlection Therapeutics. Dr. Plotkin has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Akouos. Dr. Plotkin has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for SonALAsense. Dr. Plotkin has stock in NFlection Therapeutics. Dr. Plotkin has stock in NF2 Therapeutics. Dr. Plotkin has stock in SonALAsense. Dr. Plotkin has received research support from Children's Tumor Foundation. Dr. Plotkin has received research support from Department of Defense. Dr. Plotkin has received research support from National Institutes of Health. Dr. Plotkin has received intellectual property interests from a discovery or technology relating to health care.
Kaleb H. Yohay, MD (NYU Langone Medical Center)	Dr. Yohay has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Astra Zeneca. Dr. Yohay has received publishing royalties from a publication relating to health care. An immediate family member of Dr. Yohay has received publishing royalties from a publication relating to health care.
Phioanh L. Nghiemphu, MD, FAAN (UCLA Neuro-Oncology)	Dr. Nghiemphu has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Simpson Healthcare. Dr. Nghiemphu has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Nghiemphu has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Springswork. Dr. Nghiemphu has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CIRM. Dr. Nghiemphu has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Servier. Dr. Nghiemphu has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Springworks. The institution of Dr. Nghiemphu has received research support from Erasca. The institution of Dr. Nghiemphu has received research support from Springswork. The institution of Dr. Nghiemphu has received research support from Children's Tumor Foundation. The institution of Dr. Nghiemphu has received research support from NIH-NCI . The institution of Dr. Nghiemphu has received research support from DOD. The institution of Dr. Nghiemphu has received research support from GCAR.
	No disclosure on file
Vanessa Merker (Massachusetts General Hospital)	The institution of Vanessa Merker has received research support from Children's Tumor Foundation . The institution of Vanessa Merker has received research support from NF Northeast. The institution of Vanessa Merker has received research support from Patient Centered Outcomes Research Institute. The institution of Vanessa Merker has received research support from U.S. Department of Defense . The institution of Vanessa Merker has received research support from Neurofibromatosis Therapeutic Accleration Project. Vanessa Merker has received personal compensation in the range of $10,000-$49,999 for serving as a Science Writer with Neurofibromatosis Network.

��ɫ��

��ɫ��