Perspectives of Patients and Caregivers on Relapsed/Refractory Acute Myeloid Leukemia Treatments: A Mixed-Methods Interview Study
Author(s)
Sophie Van Tomme, PhD1, Joshua Libbrecht, MBA2, Rosanne Janssens, MSc, PhD3, Harrison Clarke, MSc4, Paul Cordero, PharmD, PhD5, Stephanie Tcherny-Lessenot, MD6, Matthew Quaife, PhD7.
1SANOFI, Amsterdam, Netherlands, 2Sanofi, Cambridge, MA, USA, 3Evidera, Brussels, Belgium, 4Evidera, Bethesda, MD, USA, 5Sanofi, London, United Kingdom, 6Sanofi, Paris, France, 7Evidera, London, United Kingdom.
1SANOFI, Amsterdam, Netherlands, 2Sanofi, Cambridge, MA, USA, 3Evidera, Brussels, Belgium, 4Evidera, Bethesda, MD, USA, 5Sanofi, London, United Kingdom, 6Sanofi, Paris, France, 7Evidera, London, United Kingdom.
OBJECTIVES: Understanding the perspectives of patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) is key to improving outcomes and supporting shared treatment decision-making. This study aimed to explore patient and caregiver experiences with AML treatments and their views on treatment risks and benefits.
METHODS: Interviews were conducted with R/R AML patients and informal caregivers in the US and UK. Interviews included open-ended discussions based on a semi-structured guide and ranking exercises to identify the most important benefits and burdensome risks. Qualitative data were analyzed thematically, and quantitative data descriptively.
RESULTS: The study included 15 R/R AML patients (median age: 57, n=10 US, n=5 UK) and 15 caregivers (median age: 39, n=10 US, n=5 UK). Patients were diagnosed a median of 2.8 years previously and had undergone a median of five treatments. Extreme tiredness (n=7/15), increased risk of infections (n=6/15), and digestive problems (n=6/15) were the most frequently reported short-term side-effects experienced, and concentration problems and reduced appetite/weight loss the most frequently reported long-term side-effects (n=7/15). Overall survival was ranked as the most important treatment benefit by 10/15 patients and 9/15 caregivers, followed by achieving remission (n=3/15 patients, n=5/15 caregivers), and improving quality of life (n=1/15 for both). Participants described that quality of life becomes more important as time progresses; a UK patient noted: “[at diagnosis] your plan is to stay alive, whereas now is to live my life”. Increased infection risk was ranked the most burdensome side-effect by patients (n=5/15), while caregivers considered organ damage risk most important (n=4/15). Infusion-related reactions were not reported as a major concern for either group.
CONCLUSIONS: Findings emphasize the importance of survival and remission to R/R AML patients and caregivers and highlight concerning side-effects of infection and organ damage. This understanding can help tailor treatment approaches to better align with patient and caregiver priorities.
METHODS: Interviews were conducted with R/R AML patients and informal caregivers in the US and UK. Interviews included open-ended discussions based on a semi-structured guide and ranking exercises to identify the most important benefits and burdensome risks. Qualitative data were analyzed thematically, and quantitative data descriptively.
RESULTS: The study included 15 R/R AML patients (median age: 57, n=10 US, n=5 UK) and 15 caregivers (median age: 39, n=10 US, n=5 UK). Patients were diagnosed a median of 2.8 years previously and had undergone a median of five treatments. Extreme tiredness (n=7/15), increased risk of infections (n=6/15), and digestive problems (n=6/15) were the most frequently reported short-term side-effects experienced, and concentration problems and reduced appetite/weight loss the most frequently reported long-term side-effects (n=7/15). Overall survival was ranked as the most important treatment benefit by 10/15 patients and 9/15 caregivers, followed by achieving remission (n=3/15 patients, n=5/15 caregivers), and improving quality of life (n=1/15 for both). Participants described that quality of life becomes more important as time progresses; a UK patient noted: “[at diagnosis] your plan is to stay alive, whereas now is to live my life”. Increased infection risk was ranked the most burdensome side-effect by patients (n=5/15), while caregivers considered organ damage risk most important (n=4/15). Infusion-related reactions were not reported as a major concern for either group.
CONCLUSIONS: Findings emphasize the importance of survival and remission to R/R AML patients and caregivers and highlight concerning side-effects of infection and organ damage. This understanding can help tailor treatment approaches to better align with patient and caregiver priorities.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR185
Topic
Patient-Centered Research
Disease
Oncology