Understanding What Matters Most to Patients: Establishing the Validity of a Best-Worst Scaling Survey

NCT05061095 | Completed Results DMC

• 2 other identifiers: LCCC 2136U2CNR014637
• Study Type: observational
• Target: 51
• Locations: 1 country
• Sites: 1

Brief Summary

This study will evaluate the validity of using a survey to quantify patient preferences at the point-of-care and the potential effectiveness of the survey to improve goal-concordant care. The primary hypothesis is that by identifying the strength of patient preferences for outcomes with this survey clinicians will be able to improve goal-concordant care by aligning clinical recommendations with patients' preferences. This study will have 50 patients with newly diagnosed hematologic malignancy complete the survey throughout their disease course (up to 2 years) and conduct qualitative interviews with a subset (n = 20) of participants. The information obtained from these participants will be used to refine the survey. Interviews with oncologists and palliative care specialists (up to 10) will inform implementation.

Conditions

Hematologic Neoplasms

Keywords

leukemia; lymphoma; myeloma; myelodysplastic syndrome; best-worst scaling survey

Outcome Measures

Primary Outcomes (1)

• Content Validity of Best-Worst Scaling (BWS) Survey

  Cognitive interviewing will be used to evaluate the content validity of using a BWS survey to quantify the preferences of older patients with hematologic malignancies at the point-of-care. BWS survey asks patients a series of questions where they choose one attribute as best and one as worst - the 7 included in this survey are maintaining usual activities, living longer, avoiding dependence on others, avoiding short-term side effects, avoiding long-term side effects, avoiding hospitalizations, and avoiding high out-of-pocket costs.

  3 months after treatment decision

Secondary Outcomes (3)

• Acceptability of Best-Worst Scaling (BWS) Survey to Patients

  Up to 7 days after treatment decision

• Preliminary Efficiency of Best-Worst Scaling (BWS) Survey

  Up to 7 days after treatment decision

• Completion Rate Over Time

  Up to 1 year

Interventions

Assessment Surveys OTHER

Patients will complete surveys in three periods. An initial baseline survey, longitudinal assessments completed every 2 weeks over the course of the first three months on study (6 times maximum), and extended assessments that will happen every 3 months after the initial longitudinal period up to 2 years on the study (7 times maximum). Baseline questionnaire will collect demographic and background medical record information. Longitudinal assessments will consist of a BWS questionnaire (patients rate importance of 7 treatment goals), decisional conflict scales, EQ-5D-5L, distress thermometer, and items from PRO-CTCAE. Extended assessments will use same questionnaires as longitudinal assessments.

Qualitative Interviews OTHER

Patients will participate in 2 interviews (1 initial interview, 1 follow up). These will be cognitive interviews focusing on establishing content validity for BWS questionnaire. Will establish patient understanding of questions asked in BWS, understanding of how preferences affect treatment decisions, expected outcomes, and feedback from patients on other potentially important attributes not included in BWS.

Eligibility Criteria

• Age: 60 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants are older (≥60) patients with newly-diagnosed hematological malignancies.

You may qualify if:

• Confirmed new diagnosis of one of the following hematologic malignancies: aggressive lymphoma, multiple myeloma, CLL, CML, AML, ALL, MDS EB1 or EB2
• Age≥60
• Ability to read, understand, and communicate fluently in English
• Ability to understand and comply with study procedures
• Willingness and ability to provide written informed consent

You may not qualify if:

• Dementia, altered mental status, or psychiatric condition that would prohibit the understanding or rendering of informed consent or participation in the discrete choice experiment.
• Significant medical conditions, as assessed by the investigators, that would substantially increase the burden on the patient to complete study assessments (such as multiorgan failure, respiratory failure, or other critical illness).

Sponsors & Collaborators

• UNC Lineberger Comprehensive Cancer Center: lead
• National Institute of Nursing Research (NINR): collaborator

Study Sites (1)

Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, 27514, United States

Location

Related Publications (8)

• Richardson DR, Crossnohere NL, Seo J, Estey E, O'Donoghue B, Smith BD, Bridges JFP. Age at Diagnosis and Patient Preferences for Treatment Outcomes in AML: A Discrete Choice Experiment to Explore Meaningful Benefits. Cancer Epidemiol Biomarkers Prev. 2020 May;29(5):942-948. doi: 10.1158/1055-9965.EPI-19-1277. Epub 2020 Mar 4.

  PMID: 32132149 BACKGROUND

• Richardson DR, Oakes AH, Crossnohere NL, Rathsmill G, Reinhart C, O'Donoghue B, Bridges JFP. Prioritizing the worries of AML patients: Quantifying patient experience using best-worst scaling. Psychooncology. 2021 Jul;30(7):1104-1111. doi: 10.1002/pon.5652. Epub 2021 Feb 27.

  PMID: 33544421 BACKGROUND

• Seo J, Smith BD, Estey E, Voyard E, O' Donoghue B, Bridges JFP. Developing an instrument to assess patient preferences for benefits and risks of treating acute myeloid leukemia to promote patient-focused drug development. Curr Med Res Opin. 2018 Dec;34(12):2031-2039. doi: 10.1080/03007995.2018.1456414. Epub 2018 Apr 27.

  PMID: 29565196 BACKGROUND

• Bridges JF, Oakes AH, Reinhart CA, Voyard E, O'Donoghue B. Developing and piloting an instrument to prioritize the worries of patients with acute myeloid leukemia. Patient Prefer Adherence. 2018 Apr 27;12:647-655. doi: 10.2147/PPA.S151752. eCollection 2018.

  PMID: 29731612 BACKGROUND

• LeBlanc TW, Fish LJ, Bloom CT, El-Jawahri A, Davis DM, Locke SC, Steinhauser KE, Pollak KI. Patient experiences of acute myeloid leukemia: A qualitative study about diagnosis, illness understanding, and treatment decision-making. Psychooncology. 2017 Dec;26(12):2063-2068. doi: 10.1002/pon.4309. Epub 2016 Dec 19.

  PMID: 27862591 BACKGROUND

• Loh KP, Abdallah M, Kadambi S, Wells M, Kumar AJ, Mendler JH, Liesveld JL, Wittink M, O'Dwyer K, Becker MW, McHugh C, Stock W, Majhail NS, Wildes TM, Duberstein P, Mohile SG, Klepin HD. Treatment decision-making in acute myeloid leukemia: a qualitative study of older adults and community oncologists. Leuk Lymphoma. 2021 Feb;62(2):387-398. doi: 10.1080/10428194.2020.1832662. Epub 2020 Oct 11.

  PMID: 33040623 BACKGROUND

• Rood JAJ, Nauta IH, Witte BI, Stam F, van Zuuren FJ, Manenschijn A, Huijgens PC, Verdonck-de Leeuw IM, Zweegman S. Shared decision-making and providing information among newly diagnosed patients with hematological malignancies and their informal caregivers: Not "one-size-fits-all". Psychooncology. 2017 Dec;26(12):2040-2047. doi: 10.1002/pon.4414. Epub 2017 Apr 17.

  PMID: 28264148 BACKGROUND

• El-Jawahri A, Nelson-Lowe M, VanDusen H, Traeger L, Abel GA, Greer JA, Fathi A, Steensma DP, LeBlanc TW, Li Z, DeAngelo D, Wadleigh M, Hobbs G, Foster J, Brunner A, Amrein P, Stone RM, Temel JS. Patient-Clinician Discordance in Perceptions of Treatment Risks and Benefits in Older Patients with Acute Myeloid Leukemia. Oncologist. 2019 Feb;24(2):247-254. doi: 10.1634/theoncologist.2018-0317. Epub 2018 Aug 23.

  PMID: 30139841 BACKGROUND

MeSH Terms

Conditions

Hematologic Neoplasms; Leukemia; Lymphoma; Neoplasms, Plasma Cell; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Neoplasms by Histologic Type; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Bone Marrow Diseases

Results Point of Contact

• Title: Dr. Daniel Richardson
• Organization: Lineberger Comprehensive Cancer Center

daniel_richardson@med.unc.edu

(919) 966-4432

Study Officials

• Daniel Richardson, MD

  UNC Lineberger Comprehensive Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 27, 2021
• First Posted: September 29, 2021
• Study Start: March 21, 2022
• Primary Completion: September 26, 2023
• Study Completion: September 26, 2023
• Last Updated: December 27, 2024
• Results First Posted: December 27, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)