Identifying Patient-Reported Outcome Measures to Capture Patients’ Experiences of Symptoms and Impacts of Acute Myeloid Leukemia (AML)
Author(s)
Nicolas Plommet1, Isabelle Petit, PharmD2, Anna de la Motte, PhD3, Vanessa Cooper, BSc, MSc, PhD4, Jennifer Quinn, PhD5, Sarah Clifford, MSc, PhD6, Lina Eliasson, PhD7.
1Senior Manager, Global Value & Access, Debiopharm, Lausanne, Switzerland, 2Debiopharm International, Lausanne, Switzerland, 3Sprout Health Solutions, Merchantville, NJ, USA, 4Sprout Health Solutions, Pinner, United Kingdom, 5Debiopharm International SA, Lausanne, Switzerland, 6Sprout Health Solutions, Los Angeles, CA, USA, 7Sprout Health Solutions Ltd, Pinner, United Kingdom.
1Senior Manager, Global Value & Access, Debiopharm, Lausanne, Switzerland, 2Debiopharm International, Lausanne, Switzerland, 3Sprout Health Solutions, Merchantville, NJ, USA, 4Sprout Health Solutions, Pinner, United Kingdom, 5Debiopharm International SA, Lausanne, Switzerland, 6Sprout Health Solutions, Los Angeles, CA, USA, 7Sprout Health Solutions Ltd, Pinner, United Kingdom.
OBJECTIVES: Acute Myeloid Leukaemia (AML) is an aggressive haematologic malignancy characterised by rapid progression and poor outcomes despite treatment availability. Patients with AML often experience debilitating symptoms such as fatigue, infections, bleeding, and impaired quality of life due to both the disease and its intensive treatments. Use of patient-reported outcomes (PROs) is critical to assess symptoms and understand the full impact of AML on patients' daily lives. This study identified patient-reported symptoms and impacts of AML and evaluated the validity of existing instruments.
METHODS: A targeted literature review was conducted to identify signs, symptoms and impacts of AML and existing instruments. In parallel, searches of ClinicalTrials.gov and drug labels were conducted to identify PROs relevant to patients with AML. A gap analysis was conducted on candidate PROs to evaluate content validity and psychometric properties against regulatory guidance. A preliminary conceptual model was developed. Concepts were mapped to the shortlisted PROs.
RESULTS: The conceptual model encompassed symptoms including fatigue, fever and pain, and physical, social, cognitive, lifestyle and emotional/psychological impacts. N = 27 PRO instruments were identified from searches; n = 5 were included in the gap analysis, of which n = 2 were AML-specific. None of the PROs met full criteria for content and psychometric validation as outlined by regulatory guidance. When compared to the conceptual model, the disease-specific AML-QoL and FACT-Leu, and the general oncology measure EORTC QLQ-C30 had the most comprehensive coverage.
CONCLUSIONS: This study identified potential PROs for use in AML studies. Further validation of these measures within the specific context of use is needed. As therapeutic innovation evolves, further research is also needed to capture patients’ experiences, not only of symptoms and impacts of AML, but also the impacts of different treatments to ensure the instruments remain fit-for-purpose.
METHODS: A targeted literature review was conducted to identify signs, symptoms and impacts of AML and existing instruments. In parallel, searches of ClinicalTrials.gov and drug labels were conducted to identify PROs relevant to patients with AML. A gap analysis was conducted on candidate PROs to evaluate content validity and psychometric properties against regulatory guidance. A preliminary conceptual model was developed. Concepts were mapped to the shortlisted PROs.
RESULTS: The conceptual model encompassed symptoms including fatigue, fever and pain, and physical, social, cognitive, lifestyle and emotional/psychological impacts. N = 27 PRO instruments were identified from searches; n = 5 were included in the gap analysis, of which n = 2 were AML-specific. None of the PROs met full criteria for content and psychometric validation as outlined by regulatory guidance. When compared to the conceptual model, the disease-specific AML-QoL and FACT-Leu, and the general oncology measure EORTC QLQ-C30 had the most comprehensive coverage.
CONCLUSIONS: This study identified potential PROs for use in AML studies. Further validation of these measures within the specific context of use is needed. As therapeutic innovation evolves, further research is also needed to capture patients’ experiences, not only of symptoms and impacts of AML, but also the impacts of different treatments to ensure the instruments remain fit-for-purpose.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR126
Topic
Patient-Centered Research
Topic Subcategory
Instrument Development, Validation, & Translation, Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology