Treatment preferences among individuals with primary hyperoxaluria type 1 (PH1): a real-world study

Area of Science:

• Rare genetic disorders
• Nephrology
• Metabolic diseases

Background:

• Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder characterized by excessive oxalate production, leading to kidney and organ damage.
• Nedosiran is a treatment for PH1 in the U.S. for patients aged 9 years and older with an eGFR of at least 30 mL/min/1.73 m².
• Nedosiran can be self- or caregiver-administered at home, with fixed dosing available for patients 12 years and older.

Purpose of the Study:

• To understand treatment preferences among adults with PH1 in a real-world setting.
• To identify challenges associated with current treatment administration methods for PH1.
• To inform the development of more patient-centric PH1 therapies.

Main Methods:

• A cross-sectional, web-based survey was conducted among U.S.-based adults (≥18 years) diagnosed with PH1.
• The survey included a 20-25 minute questionnaire.
• Data collection occurred from October to December 2023.

Main Results:

• Participants (N=39) showed a strong preference for self-administration at home, with 72% favoring self-injection over healthcare provider (HCP)-administered injections.
• Convenience factors such as frequency of administration, time required, and place of administration were highly important.
• A majority preferred non-weight-based dosing (64%) and found quarterly injections (56%) easy or very easy, but 67% preferred monthly injections at home over quarterly at a medical facility.

Conclusions:

• Patients with PH1 prioritize treatment convenience and lifestyle compatibility.
• Self-administration, home-based treatments, and predictable dosing regimens are key preferences for individuals with PH1.
• These findings highlight the need for patient-centered approaches in developing and delivering PH1 therapies.