Multidimensional Assessment of Patient-Reported-Outcomes in a Multicenter Cohort of Inborn Errors of Immunity

Area of Science:

• Immunology
• Patient-Reported Outcomes Research

Background:

• Patient-reported outcomes (PROs) are vital for lifelong disease management in inborn errors of immunity (IEIs).
• Existing research often focuses narrowly on specific IEI subgroups or immunoglobulin replacement therapy (IgRT) routes, limiting comprehensive understanding.

Purpose of the Study:

• To evaluate health-related quality of life (HRQoL) and IgRT treatment satisfaction (TS) in a diverse IEI patient cohort.
• To identify factors influencing HRQoL and TS to improve patient well-being.

Main Methods:

• Cross-sectional survey of 500 IEI patients on IgRT, assessing TS (TSQM-9) and HRQoL (KINDL/SF-36).
• Integration of patient/caregiver-reported data with clinical information.
• Analysis of factors impacting HRQoL and TS across different IEI diagnoses and treatment parameters.

Main Results:

• Non-Primary Antibody Deficiency (PAD) diagnoses, comorbidities, absence from school/work, and systemic adverse reactions negatively impacted HRQoL.
• Severe infections and hospitalizations adversely affected both HRQoL and TS.
• Subcutaneous IgRT, especially at home, correlated with higher TS due to convenience and reduced absenteeism, but did not influence adult HRQoL.

Conclusions:

• IEI patient well-being and satisfaction are multifactorial, not solely determined by the IgRT route.
• Minimizing disease/treatment burdens and promoting societal participation (e.g., school/work attendance) can enhance patient self-esteem and coping mechanisms.