Identifying Patient-Reported Outcome Measures to Capture Patients’ Experiences of Symptoms and Impacts of Myelodysplastic Syndromes (MDS)
Author(s)
Nicolas Plommet1, Isabelle Petit, PharmD2, Chloe Patel, PhD3, Beverly Romero, MA4, Jennifer Quinn, PhD5, Lina Eliasson, PhD6.
1Senior Manager, Global Value & Access, Debiopharm, Lausanne, Switzerland, 2Debiopharm International, Lausanne, Switzerland, 3Sprout Health Solutions, Coventry, United Kingdom, 4Sprout Health Solutions Ltd, Germantown, MD, USA, 5Debiopharm International SA, Lausanne, Switzerland, 6Sprout Health Solutions Ltd, Pinner, United Kingdom.
1Senior Manager, Global Value & Access, Debiopharm, Lausanne, Switzerland, 2Debiopharm International, Lausanne, Switzerland, 3Sprout Health Solutions, Coventry, United Kingdom, 4Sprout Health Solutions Ltd, Germantown, MD, USA, 5Debiopharm International SA, Lausanne, Switzerland, 6Sprout Health Solutions Ltd, Pinner, United Kingdom.
OBJECTIVES: Myelodysplastic Syndromes (MDS) are a group of clonal bone marrow disorders characterised by ineffective haematopoiesis, cytopenia, and progression to acute leukaemia in some patients. MDS can cause debilitating symptoms such as fatigue, anaemia, infections, and bleeding, significantly impacting patients' quality of life. Use of patient-reported outcome (PRO) instruments is essential to understand the impact of MDS on patients’ daily lives. This study identified patient-reported symptoms and impacts of MDS and evaluated the validity of existing PRO instruments.
METHODS: A targeted literature review was conducted to identify signs, symptoms, and impacts of MDS, and existing instruments. Searches of ClinicalTrials.gov and drug labels were also conducted to identify PROs relevant to patients with MDS. A gap analysis was then 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 and impacts such as fatigue and difficulty performing daily activities. N = 21 PROs were identified from searches; n = 6 were included in the gap analysis, of which n = 1 was MDS specific (QUALMS). Only the QUALMS met most criteria for content validity and psychometric properties as outlined by regulatory guidance. When compared to the conceptual model, none of the shortlisted PRO instruments had comprehensive coverage of symptoms and impacts.
CONCLUSIONS: This study identified potential PRO instruments for use in MDS studies. However, most lacked coverage of important symptoms and impacts when compared to the conceptual model. 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’ experience of not only symptoms and impacts of MDS 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 MDS, and existing instruments. Searches of ClinicalTrials.gov and drug labels were also conducted to identify PROs relevant to patients with MDS. A gap analysis was then 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 and impacts such as fatigue and difficulty performing daily activities. N = 21 PROs were identified from searches; n = 6 were included in the gap analysis, of which n = 1 was MDS specific (QUALMS). Only the QUALMS met most criteria for content validity and psychometric properties as outlined by regulatory guidance. When compared to the conceptual model, none of the shortlisted PRO instruments had comprehensive coverage of symptoms and impacts.
CONCLUSIONS: This study identified potential PRO instruments for use in MDS studies. However, most lacked coverage of important symptoms and impacts when compared to the conceptual model. 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’ experience of not only symptoms and impacts of MDS 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
PCR127
Topic
Patient-Centered Research
Topic Subcategory
Instrument Development, Validation, & Translation, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Oncology