Treatment preferences towards thrombopoietin-receptor agonists for immune thrombocytopenia and experience of disease (TRAPeze): Italy cohort

ABSTRACT

Objective

Identify patient preference towards thrombopoietin-receptor agonists (TPO-RAs) and determine the clinical and social impact of immune thrombocytopenia (ITP) in Italy.

Methods

The Thrombopoietin-Receptor Agonist Patient experience (TRAPeze) survey collected responses from Italian residents from 17th January to 28th February 2022. TRAPeze utilized a discrete choice experiment (DCE) to elicit patient preferences towards TPO-RA attributes and a patient burden survey (PBS) to determine ITP disease characteristics and social impact.

Results

Seventy-six respondents completed the DCE, of which 69 completed both the DCE and PBS (mean [range] age 45 [18.0–73.0] years, 80% female). TPO-RA attributes with the greatest influence over respondent choice were method of administration (odds ratio [OR] 2.96; 95% confidence interval [CI] 2.16–4.06), drug-food interactions (OR 1.48; 95% CI 1.17–1.86) and frequency of dosing (OR 1.32; 95% CI 1.15–1.52). Respondents were more likely to prefer therapies administered orally over subcutaneous injection (OR 3.76; 95% CI 2.51–5.63), once weekly over once daily (OR 1.83; 95% CI 1.26–2.65), and therapies without food restrictions over with restrictions (OR 1.58; 95% CI 1.17–2.14).

The most frequently reported symptoms were bruising (82%), petechiae (65%) and fatigue (64%). Most respondents (84%) felt ITP impacted familial relationships and 71% of employed respondents reported fatigue influencing their ability to work, with 31% reducing working hours.

Conclusion

Although responses indicated a moderate perception of general health, ITP clearly impacted respondent work and social life. Our findings demonstrate respondents preferred TPO-RAs delivered orally, with less frequent dosing and without food restrictions.

KEYWORDS:

• Immune thrombocytopenia
• thrombopoietin-receptor agonists
• treatment preferences
• discrete choice experiment
• real-world

Acknowledgements

The authors wish to thank all respondents for contributing to the survey. We are grateful for the contributions of Barbara Lovrencic from the Italian ITP patient association (Associazione Italiana Porpora Immune Thrombocitopenica) and thank the members of the association for their participation in this study. Sobi reviewed and provided feedback on the studies.

Disclosure statement

AL reports honoraria from Amgen, Novartis, Grifols and Sobi, and consultancy fees from Grifols. BL reports consultancy fees from Novartis, Ucb and Sobi. VM reports consultancy fees from Amgen, Bayer, Novartis and Sobi, and research funding from Grifols. AN reports honoraria from Amgen, Angle, Argenx, Dova, Novartis, Ono and Shionogi, and consultancy fees from Amgen, Angle, Argenx and Dova. MM reports consultancy fees from Novartis, Sobi and UCB. KW and DE are employees of Sobi. EG, ODP and SP are employees of Wickenstones and report funding from Sobi for this research.

Data accessibility

Data supporting the findings presented here are available from the corresponding author upon reasonable request.

Additional information

Funding

This research was funded by Swedish Orphan Biovitrum AB (Sobi™).