Patient perspectives on pregnancy affected by hemolytic disease of the infant and newborn or fetal and neonatal alloimmune thrombocytopenia

Abstract Introduction Rare alloantibody-related conditions of pregnancy, including hemolytic disease of the fetus and newborn (HDFN) and fetal and neonatal alloimmune thrombocytopenia (FNAIT), are serious, potentially life-threatening conditions. Currently, little is known about the experience, well-being, and unmet needs of the maternal patients affected by these conditions. Furthermore, the relevance and suitability of disease-agnostic patient-reported outcome (PRO) instruments in capturing these experiences remains to be evaluated. Methods Qualitative, semi-structured interviews were conducted with participants who had ≥ 1 pregnancy diagnosed with HDFN or FNAIT. Open-ended concept elicitation interviews provided direct insights into the patient experience. From these, condition-specific thematic analyses allowed for concepts to be applied to existing PRO measures, such as the Short Form-36 Health Survey v2 Acute (SF-36v2 Acute) and the EuroQoL-5 Dimension 5 Levels (EQ-5D-5L), to evaluate the suitability of these disease-agnostic measures in capturing the maternal participant experience. Further cognitive debriefing with participants on additional PRO measures provided insight into item-level suitability, relevance, and feasibility of recall periods from the participants’ perspective. The Mother-Generated Index (MGI) was also provided to participants to complete at home. Results Ten participants with pregnancies affected by HDFN and 8 participants with FNAIT were included. Median age was 35.5 to 40.5 years, with most being White/Caucasian, college-educated, and employed. During pregnancy, impacts on emotional well-being were most prevalent across HDFN and FNAIT, and these were accompanied by physical, social, and work-related impacts. Postnatal experiences frequently involved emotional impacts and experiences with the neonatal intensive care unit. Long-term effects and concerns about future pregnancies were also commonly reported. Most concepts were represented by the SF-36v2 Acute/EQ-5D-5L, except for future family planning. Cognitive debriefing of additional PRO measures showed overall high relevance and suitability, with minor item-specific issues. Furthermore, MGI findings mirrored interview themes and provided broader perspectives on meaningful impacts in other areas. Conclusion These findings add valuable insights into the patient experience of pregnancies affected by rare alloantibody-related conditions, revealing broad and long-lasting effects on emotional, physical, social, and other well‑being domains. These results support the suitability of SF-36v2 Acute and EQ-5D-5L for capturing patient-reported experiences during pregnancies affected by HDFN or FNAIT.