3357 - A global patient-driven Facebook study in a very rare sarcoma: Health-related quality of life in Epithelioid Hemangioendothelioma (EHE) patients

Background

EHE is a vascular sarcoma with an incidence of ≤ 1 per 1 million/year, and an unpredictable clinical course with significant symptom burden. Recruiting EHE patients for studies is difficult and health-related quality of life (HRQoL) in these patients is unknown. We aimed to study the impact of EHE symptom burden on HRQoL.

Methods

The study was initiated after EHE patients’ foundations approached our research group to study HRQoL. After ethical approval, patients were recruited from the global EHE Facebook group from May-October 2018. Data were collected using the unique, well-established online PROFILES (Patient Reported Outcomes Following Initial treatment and Long-term Evaluation of Survivorship) registry. Latent class cluster analysis was performed to identify groups based on 10 frequently reported symptoms. Differences in HRQoL (EORTC-QLQ-C30) between clusters were examined.

Results

Of 138 patients who registered in PROFILES, 115 (83%) completed the survey. Three clusters of EHE patients were identified with low (A) intermediate (B) and high (C) symptom burden. Highly symptomatic patients had clinically relevant lower scores on HRQoL compared to the other two groups (p < 0.001) (Table). These patients suffered mostly from pain, insomnia and fatigue and more often had bone/pleural lesions.Table:

1680P HRQoL per cluster

a: cluster A vs B, b: cluster A vs C, c: cluster B vs C

Conclusions

For the first time, we showed that a Facebook patient group enabled HRQoL research in EHE. One third of patients was highly symptomatic and experienced reduced functioning in daily life. Collaboration with patients and the established online PROFILES system facilitated successful recruitment in this ultra-rare sarcoma and offers perspectives for future research.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

W.T. van der Graaf: Research grant / Funding (institution): Bayer; Research grant / Funding (institution): Novartis. I.M.E. Desar: Research grant / Funding (institution): Novartis. All other authors have declared no conflicts of interest.