Hemolytic Disease of the Fetus and Newborn (HDFN): A Mixed-Methods Preference Study
Author(s)
Divya Mohan, PhD1, Adele Barlassina, PhD2, Marco Boeri, BSc, MSc, PhD1, Molly Sherwood, BA3, Ellen M. Janssen, BA, PhD4, Alexis Krumme, ScD4, Laura M. Bozzi, MS, PhD5.
1OPEN Health HEOR & Market Access, London, United Kingdom, 2OPEN Health HEOR & Market Access, Rotterdam, Netherlands, 3Allo Hope Foundation, Tuscaloosa, AL, USA, 4Global Epidemiology Organization, Johnson & Johnson, Titusville, NJ, USA, 5Global Epidemiology Organization, Johnson & Johnson, Raritan, NJ, USA.
1OPEN Health HEOR & Market Access, London, United Kingdom, 2OPEN Health HEOR & Market Access, Rotterdam, Netherlands, 3Allo Hope Foundation, Tuscaloosa, AL, USA, 4Global Epidemiology Organization, Johnson & Johnson, Titusville, NJ, USA, 5Global Epidemiology Organization, Johnson & Johnson, Raritan, NJ, USA.
Presentation Documents
OBJECTIVES: Little is known about patient preferences for the treatment of Hemolytic Disease of the Fetus and Newborn (HDFN), mainly due to its rarity. To fill this gap, we conducted a mixed methods study to understand patient preferences for treatment of pregnancies affected by severe HDFN.
METHODS: The study consisted of three stages: a quantitative best-worst scaling (BWS) survey to prioritize qualities of HDFN treatment, qualitative interviews to gain in-depth insights into disease and treatment experience, and a quantitative threshold technique (TT) survey to measure acceptable benefit-risk tradeoffs. A patient advocacy group educated the study team on the disease impact, ensured relevance of study design, facilitated recruitment, and supported interpretation of results. The study recruited people who had a pregnancy severely affected by HDFN in the past 5 years, over 18 years of age or older during that pregnancy, and resident in the US or Canada.
RESULTS: 28 participants, the mean age was 35 and the median number of intrauterine transfusions (IUT) received in their most recent pregnancy was 3. In the BWS, preventing severe complications and long-term complications for the baby were identified as most important. The qualitative analysis highlighted different aspects of the pregnancy experience across 3 themes: diagnosis, monitoring, and delivery. In the TT, in exchange for a reduction in one IUT, 50% of participants accepted up to 10% risk of serious infection for the baby, in the first year of life; the majority (62%) would accept maximum 10 days in the NICU; and 40% would accept greater than 30% risk for maternal complications.
CONCLUSIONS: While results show preference heterogeneity, participants want to reduce the number of IUTs limiting complications for the baby. These findings demonstrate the need to understand the complex decisions mothers make when considering treatment for their unborn child during pregnancy.
METHODS: The study consisted of three stages: a quantitative best-worst scaling (BWS) survey to prioritize qualities of HDFN treatment, qualitative interviews to gain in-depth insights into disease and treatment experience, and a quantitative threshold technique (TT) survey to measure acceptable benefit-risk tradeoffs. A patient advocacy group educated the study team on the disease impact, ensured relevance of study design, facilitated recruitment, and supported interpretation of results. The study recruited people who had a pregnancy severely affected by HDFN in the past 5 years, over 18 years of age or older during that pregnancy, and resident in the US or Canada.
RESULTS: 28 participants, the mean age was 35 and the median number of intrauterine transfusions (IUT) received in their most recent pregnancy was 3. In the BWS, preventing severe complications and long-term complications for the baby were identified as most important. The qualitative analysis highlighted different aspects of the pregnancy experience across 3 themes: diagnosis, monitoring, and delivery. In the TT, in exchange for a reduction in one IUT, 50% of participants accepted up to 10% risk of serious infection for the baby, in the first year of life; the majority (62%) would accept maximum 10 days in the NICU; and 40% would accept greater than 30% risk for maternal complications.
CONCLUSIONS: While results show preference heterogeneity, participants want to reduce the number of IUTs limiting complications for the baby. These findings demonstrate the need to understand the complex decisions mothers make when considering treatment for their unborn child during pregnancy.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR12
Topic
Patient-Centered Research
Topic Subcategory
Patient Engagement
Disease
SDC: Pediatrics, SDC: Rare & Orphan Diseases