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. 2025 Aug 16.
doi: 10.1007/s40271-025-00762-6. Online ahead of print.

A Qualitative Study on Patient Experience with Signs, Symptoms, and Daily Impacts of Immune Thrombocytopenia

Waleed Ghanima  1 , Nichola Cooper  2 , Sylvie Bozzi  3 , Ahmed Daak  4 , Imene Gouia  3 , Matias Cordoba  4 , Javier Barrio  5 , Michael Kostikas  6 , Owen Cooper  7 , Howard Liebman  8
Affiliations

A Qualitative Study on Patient Experience with Signs, Symptoms, and Daily Impacts of Immune Thrombocytopenia

Waleed Ghanima et al. Patient. .

Abstract

Background: Primary immune thrombocytopenia (ITP) adversely impacts a patient's health-related quality of life (HRQoL).

Objective: This study explored patients' experiences with signs, symptoms, and impacts of ITP and updated the conceptual disease model for HRQoL in patients with ITP.

Methods: Adult patients with ITP were included in the study. Patients with any comorbidity where fatigue was a key symptom (e.g., anemia) were excluded. A concept elicitation interview assessed signs, symptoms, and impacts from the patients' perspective, and cognitive debriefing assessed the validity of the selected patient-reported outcome instruments (ITP-Patient Assessment Questionnaire, Patient Global Impression of Severity of Fatigue, Patient Global Impression on Severity, and Patient Global Impression on Change scales). Symptoms/impacts mentioned by ≥ 50% of patients and a "bothersomeness" rating of ≥ 5 were considered salient. The preliminary conceptual model was updated based on the concepts reported by more than one patient.

Results: A total of 18 patients (mean age 42.7 ± standard deviation 14.7 years; female 78%) were interviewed. Six salient symptoms included fatigue (94%; n = 17), bruising (83%; n = 15), petechiae (72%; n = 13), difficulty staying awake during the day, difficulty falling asleep, and difficulty staying asleep (all symptoms related to sleep: 50%, n = 9). Six key impacts that emerged included those on work (72%; n = 13), worry and fear (67%; n = 12), limited physical or sporting activities, the perceived need to be cautious to avoid getting hurt, and family, friends, and social life (all 56%; n = 10).

Conclusion: This study highlighted six key symptoms of ITP and its impacts on patients with ITP. The concept elicitation findings were used to update the conceptual model, and-during cognitive debriefing-patients found the patient-reported outcome instruments easy to understand and relevant to their experience.

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Conflict of interest statement

Declarations. Conflict of Interest: Waleed Ghanima has received fees for participation on advisory boards from Amgen, Novartis, Pfizer, Principia Biopharma Inc—a Sanofi Company, Sanofi, SOBI, Grifols, UCB, Argenx, Cellphire, Alpine, Kedrion, HiBio, Hutchmed, and Takeda; lecture honoraria from Amgen, Novartis, Pfizer, Bristol Myers Squibb, SOBI, Grifols, Sanofi, and Bayer; and research grants from Bayer, BMS/Pfizer, UCB, and Sanofi. Nichola Cooper has received research support from Novartis and Rigel and honoraria for participation on advisory boards or speaking at educational events from Sanofi, Novartis, Sobi, Griffols, Rigel, and Argenx. Sylvie Bozzi, Ahmed Daak, Imene Gouia, and Matias Cordoba are employees of Sanofi and may own stock and/or stock options in Sanofi. Javier Barrio, Michael Kostikas, and Owen Cooper are employees of IQVIA. Howard Liebman has received consultation fees from Amgen, Novartis, Sanofi, and Sobi and clinical trial research support through the USC Norris Cancer Center, Abcuro, Janssen, and Sanofi. Funding: Open access funding provided by University of Oslo (incl Oslo University Hospital). This study was funded by Sanofi. Ethics Statement: The study adhered to the Declaration of Helsinki, Good Clinical Practice and applicable regulatory requirements. The study protocol, interview guide, and patient communication templates were approved by a WIRB-Copernicus Group institutional review board, and all patients provided written informed consent to participate. Patient Consent: All the included patients provided written informed consent to participate. Consent for Publication: Not applicable. Data Availability: Further data are available from the corresponding author upon reasonable request. Permission to Reproduce Material from Other Sources: Not applicable. Author Contributions: WG, NC, SB, AD, IG, MC, and HL designed/conceived of this study. JB, MK, and OC performed the research. JB, MK, and OC coordinated the data collection. WG, NC, SB, AD, IG, MC, HL, JB, MK, and OC analyzed and interpreted the data. All authors critically reviewed the draft and approved the final version for publication.

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