Patient Reported Outcomes

The NTAP Patient Reported Outcome (PRO) Initiative aims to develop reliable measures that assess the impact of plexiform neurofibromas (pNFs) on patients with neurofibromatosis type 1 (NF1).

This program is leading the creation and validation of PRO measures to be used as treatment outcomes that are the most relevant to patients, and which can be used as primary or secondary endpoints in clinical trials and product labeling applications. There are currently no PRO measures that have been validated specifically for use in patients with pNFs.

Current NTAP-funded PRO projects:

Development of patient reported outcomes system for patients with neurofibromatosis type 1-associated plexiform neurofibromas using a mixed method approach

Lai_JinsheiJin-Shei Lai, Ph.D. OTR/L, Feinberg School of Medicine, Northwestern University
This project aims to create a computer-based patient reported outcome (PRO) measure for use with patients 5 years and older who have neurofibromatosis type 1 and plexiform neurofibromas. The tool produced by this work will have multiple domains, including pain, stigma, organ dysfunction and others identified through the concept elicitation phase of the study. Methodology used to create the Patient Reported Outcomes Measurement Information System (PROMIS) and the Quality of Life for Patients with Neurological Disorders (Neuro-QOL) will be used as the basis for the methodology that will guide this work. Items from the PROMIS Pediatric Pain Interference bank, the Neuro-QOL Pediatric Pain bank, and the Neuro-QOL Pediatric Stigma bank will be modified and supplemented with new items as needed. Item Response Theory will be used to analyze the psychometric properties of the items generated.

Narrative: Read about the project’s background, goals, specific aims, and preliminary results (auspices of Dr. Jin-Shei Lai, js-lai@northwestern.edu)

Publication 1: Lai, J.S., Jensen, S.E., Patel, Z., Listernick, R., Charrow, J. (2017). Using a qualitative approach to conceptualize concerns of patients with neurofibromatosis Type 1 associated plexiform neurofibromas (pNF) across the lifespan. American Journal of Medical Genetics Part A, 173(1), 79-87.

Publication 2: Patel, Z.S., Jensen, S.E., Lai, J.S. (2016). Considerations for conducting qualitative research with pediatric patients for the purpose of PRO development. Quality of Life Research, 25, 2193-2199.

Development of a child neurofibromatosis type 1 health related quality of life measure

Swigonski_Nancy

Nancy Swigonski, M.D., M.P.H., University of Indiana
This project aims to create a written patient reported outcome (PRO) measure for use with individuals ages 5-24 with neurofibromatosis type 1 (NF1). The tool produced by this work will have multiple domains, including pain, neurologic dysfunction, organ dysfunction, parasthesias, stigma, perceived physical appearance, worry, treatment anxiety and others identified through the concept elicitation phase of the study. Methodology used to create the PedsQL NF1 Adult Module will be used as the basis for the methodology that will guide this work. Items from the PedsQL NF1 Adult Module will be modified and supplemented with new items as needed to create the proposed measure. Validation will include the use of the PedsQL 4.0 Generic Score Scale. Parallel parent-observer reports will be developed for children ages 5-18 years.

NarrativeRead about the project’s background, goals, specific aims, methods, preliminary results, and impact (auspices of Dr. Nancy Swigonski, nswigons@iupui.edu)

PublicationNutakki K, Varni JW, Steinbrenner S, Draucker CB, Swigonski NL. Development of the pediatric quality of life inventory neurofibromatosis type 1 module items for children, adolescents and young adults: qualitative methods. J Neurooncol. 2016 Dec 23.

The development and validation of patient reported outcome measures to assess pain in Individuals

Pam Wolters, Ph.D., National Cancer InstituteWolters_Pam
This project aims to create two PROs for use with people ages 6 and above who have neurofibromatosis type 1 and plexiform neurofibromas. The tools produced by this work will measure pain intensity and pain interference. For pain intensity, this team proposes to modify the Numerical Rating Scale-11 (NRS-11); construct validity will be assessed using the Visual Analog Scale and the Faces Pain Scale-Revised. The proposal includes work to validate the NRS-11 for children ages 6-7, as it currently is validated for individuals ages 8 and above. For pain interference, the Pain Interference Index will be modified; construct validity will be assessed using the Functional Disability Inventory.

NarrativeRead about the project’s background, goals, specific aims, and preliminary results (auspices of Dr. Pam Wolters, woltersp@mail.nih.gov)

Development of a needs-based quality of life patient reported outcome measure specific patients with neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (pNFs)

Stephen McKenna Ph.D., AFBPsS, C.psychol, Galen ResearchMcKenna_Stephen
This project aims to create a written patient reported outcome (PRO) measure for use with people ages 18 and above who have neurofibromatosis type 1 (NF1) and plexiform neurofibromas (pNFs). The tool produced by this work will be a needs-based quality of life instrument with items focusing on needs fulfillment rather than specific symptoms or functioning. The measure aims to determine the impact of stigma, pain, organ compression, neurological dysfunction and any other concerns identified through the concept elicitation phase of the study. Utilizing Rasch analysis, the proposed tool would be unidimensional and provide a valid single score, which would be complementary to symptom and functioning scales. The tool will be developed in both UK English and US English.

Narrative: Read about the project’s background, goals, methods, and preliminary results (auspices of Dr. Stephen McKenna, smckenna@galen-research.com)