The choice of drug treatment in advanced soft tissue sarcoma (STS) continues to be a challenge considering efficacy, QoL and toxicity. Unlike other cancer types, where integrating patient reported outcomes (PRO) has proven to be beneficial for QoL, there is no such evidence in patients with STS yet.
This multi-center study explored the effect of a comprehensive intervention on QoL in patients with STS undergoing treatment with trabectedin (Yondelis®). Seven hospitals were cluster-randomized into control cluster (CC with electronic [e] assessment of PRO) or interventional cluster (IC including ePRO and expert-consensus based treatment suggestions). The center, where the multidisciplinary treatment suggestions were created, served as a reference center (RC). Outcomes included QoL (measured with FACT-G), symptoms (MDASI), anxiety and depression (HADS) and pain intensity and interference (BPI). The explorative primary endpoint was change of FACT-G total score (range 0-108) after nine weeks.
80 patients (50% male; mean age: 58.1 years, range: 22-87 years) were included. After nine weeks, decrease in QoL was smaller in IC (-2.04) than in the CC (-5.5) and RC (-4.75). Nevertheless, this trend failed to reach statistical significance (p = 0.235). Improved, but not significant (p = 0.247) median progression free survival was observed in IC (277 days) and CC (279 days) than in RC (126 days). Other PRO showed non-significant, but yet medium to large effects.
This is the first study to include ePRO in patients with STS. The clinically important benefit in QoL observed in the IC could serve as a proof of principle to strengthen patient care. Beyond proofing statistical significance of the clinically meaningful effects, this study is an important prerequisite for future research in this area.
Clinical trial identification
Legal entity responsible for the study
B. Kasper: Consultancy: Bayer, Clinigen, Eisai, Lilly; Honoraria: Bayer, Eisai, Lilly, Novartis, PharmaMar, Pfizer; Financing of research: PharmaMar. M. Schuler: Research Grant: PharmaMar. All other authors have declared no conflicts of interest.Table: 1606PD
Sociodemographic data at baseline
|Interventional cluster (IC), n = 39||Control cluster (CC), n = 29||Reference center (RC), n = 12||p-value|
|Age, mean (SD) Range (years)||58.39 (11.68), 38-87||55.80 (14.92), 22-80||63.00 (15.63), 34 - 82||0.440|
|Tumor entity Leiomyosarcoma Liposarcoma Others||18 10 15||9 10 15||5 3 7|
|ECOG 0 1 2||20 15 3||14 13 0||5 7 0||0.300|
|Patient Reported Outcomes||IC, n = 3||CC, n = 18||RC, n = 8||Cohens d||p-value|
|FACT-G total Mean change after 9 weeks||-2.04||-5.50||-4.75||0.72||0.235|
|HADS depression Mean change after 9 weeks||0.65||1.44||-0.25||0.91||0.181|
|HADS anxiety Mean change after 9 weeks||0.00||0.78||-0.88||0.72||0.235|
|BPI pain severity Mean change after 9 weeks||0.48||0.01||-0.47||0.72||0.235|
|BPI pain interference Mean change after 9 weeks||0.62||0.15||-0.23||0.70||0.241|
|MDASI, symptom severity Mean change after 9 weeks||0.60||0.29||-0.23||0.68||0.247|
|MDASI, symptom interference Mean change after 9 weeks||1.03||1.00||0.21||0.04||0.482|