PMID- 29565196
OWN - NLM
STAT- MEDLINE
DCOM- 20191007
LR  - 20220201
IS  - 1473-4877 (Electronic)
IS  - 0300-7995 (Print)
IS  - 0300-7995 (Linking)
VI  - 34
IP  - 12
DP  - 2018 Dec
TI  - Developing an instrument to assess patient preferences for benefits and risks of 
      treating acute myeloid leukemia to promote patient-focused drug development.
PG  - 2031-2039
LID - 10.1080/03007995.2018.1456414 [doi]
AB  - Objective Acute myeloid leukemia (AML) is a progressive blood cancer with few 
      effective treatment options. As part of a patient-focused drug development (PFDD) 
      initiative led by the Leukemia and Lymphoma Society (LLS), this study sought to 
      use a community-centered approach to develop and pilot an instrument to measure 
      patient preferences for the benefits and risks of treating AML. Methods 
      Instrument development was informed by a literature review, engagement with 
      expert stakeholders (n = 12), engagement with community stakeholders, and 
      pre-testing. A discrete-choice experiment (DCE), in which participants made 
      choices between 16 pairs of hypothetical treatments, was developed with five 
      attributes: event-free survival, complete remission, time in hospital, short-term 
      side-effects, and long-term side-effects. A pilot test was conducted and analyzed 
      using conditional logistic regression. Results are presented using relative 
      attribute importance (RAI) scores. Results Patients with AML and caregivers were 
      engaged in developing (n = 15), pre-testing (n = 13), and pilot testing (n = 26) 
      the instrument. The pilot included patients with AML (n = 18) and caregivers of 
      living or deceased patients with AML (n = 8). Participants had a mean age of 50 
      years (range =24-81), and were mostly college educated (n = 22), privately 
      insured (n = 21), and employed (n = 13). Based on the DCE, complete remission was 
      identified as the most important attribute (RAI =10), followed by event-free 
      survival (3.7), time in hospital (2.8), long-term side-effects (2.3), and 
      short-term side-effects (2.1). Conclusion The mixed-methods approach to PFDD was 
      welcomed by all stakeholders and there was strong endorsement to implement this 
      DCE as part of a national survey. Key points for decision makers The Leukemia and 
      Lymphoma Society (LLS) initiated an independent effort to promote patient-focused 
      drug development (PFDD). This study presents the development and piloting of a 
      preference study as a first step in this initiative. Results of this pilot study 
      were used to guide a PFDD meeting to discuss the lived experience of patients and 
      caregivers affected by AML. Productive engagement by all patients, caregivers, 
      and stakeholders throughout the process resulted in strong endorsement of the 
      project's approach and recognition of the need to conduct a national study.
FAU - Seo, Jaein
AU  - Seo J
AD  - a Department of Health Policy and Management , Johns Hopkins Bloomberg School of 
      Public Health , Baltimore , MD , USA.
FAU - Smith, B Douglas
AU  - Smith BD
AD  - b Johns Hopkins Sidney Kimmel Comprehensive Cancer Center , Baltimore , MD , USA.
FAU - Estey, Elihu
AU  - Estey E
AD  - c Clinical Research Division , Fred Hutchinson Cancer Research Center , Seattle , 
      WA , USA.
AD  - d Department of Medical Oncology , University of Washington , Seattle , WA , USA.
FAU - Voyard, Ernest
AU  - Voyard E
AD  - e The Leukemia & Lymphoma Society , Ryebrook , NY , USA.
FAU - O' Donoghue, Bernadette
AU  - O' Donoghue B
AD  - e The Leukemia & Lymphoma Society , Ryebrook , NY , USA.
FAU - Bridges, John F P
AU  - Bridges JFP
AD  - a Department of Health Policy and Management , Johns Hopkins Bloomberg School of 
      Public Health , Baltimore , MD , USA.
LA  - eng
GR  - U01 FD004977/FD/FDA HHS/United States
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Research Support, U.S. Gov't, P.H.S.
DEP - 20180427
PL  - England
TA  - Curr Med Res Opin
JT  - Current medical research and opinion
JID - 0351014
SB  - IM
MH  - Adult
MH  - Aged
MH  - Aged, 80 and over
MH  - Caregivers/psychology
MH  - Data Collection
MH  - Decision Making
MH  - Drug Development/*methods
MH  - Female
MH  - Humans
MH  - Leukemia, Myeloid, Acute/*drug therapy
MH  - Logistic Models
MH  - Male
MH  - Middle Aged
MH  - *Patient Preference
MH  - Pilot Projects
MH  - Risk Assessment
MH  - Young Adult
PMC - PMC8799376
MID - NIHMS1576924
OTO - NOTNLM
OT  - Acute myeloid leukemia (AML)
OT  - Community engagement
OT  - Instrument development
OT  - Stated preference
EDAT- 2018/03/23 06:00
MHDA- 2019/10/08 06:00
PMCR- 2022/01/28
CRDT- 2018/03/23 06:00
PHST- 2018/03/23 06:00 [pubmed]
PHST- 2019/10/08 06:00 [medline]
PHST- 2018/03/23 06:00 [entrez]
PHST- 2022/01/28 00:00 [pmc-release]
AID - 10.1080/03007995.2018.1456414 [doi]
PST - ppublish
SO  - Curr Med Res Opin. 2018 Dec;34(12):2031-2039. doi: 10.1080/03007995.2018.1456414. 
      Epub 2018 Apr 27.