T-Rex study: Quality of life assessment using novel CTCAE-PRO and FKSI-19 in first-line treatment of metastatic renal cell carcinoma with tivozanib.
Authors: Grünwald Viktor , Lange Carsten , Ziske Carsten , Hegemann Miriam , Seseke Sandra , Fieseler Claus , Standhaft Diana , Janitzky Andreas , Stauch Martina , Marschner Norbert , Distelrath Andrea , Boegemann Martin , Rau Olrik , Baumann Steffen , Hellmis Eva , Dieckhoff Jan , Brenneis Horst , Rußwurm Karen , Ivanyi Philipp , Doehn Christian 
Published: June 2023

e16549 Background: The efficacy and safety of tivozanib (TIVO) have been evaluated in pivotal randomized, clinical trials in first-line treatment of mRCC. We previously reported on effectiveness of our real-world T-Rex study. Here, we assessed the quality of life of tivozanib in a real-world patient population in Germany using NCCN-FACT FKSI-19 (FKSI-19) and tailored PRO-CTCAE questionnaires. Methods: Patients with mRCC were treated with tivozanib according to routine care in this non-interventional study (registered at BfARM #7296). Adverse events (AEs) were assessed by CTCAE 4.03. Patient reported outcomes (PRO) were assessed by Functional Kidney Cancer Symptom Index (FKSI)-19 and tailored PRO-CTCAE questionnaires, which were scored according to corresponding guidelines. Patients who reported detriments on PRO scales at any time during treatment were counted as impaired. The study was performed in accordance with the declaration of Helsinki. Results: A total of N = 32 pts. (59.4% male) received at least one cycle of treatment and were analysed. The mean age was 75.4 years (61-89) and 17 pts. (53.1%) were ≥75 years old. 93.8% had ECOG 0-1. IMDC risk was favourable/intermediate/poor in 21.8/68.8/9.4%. The most common G1/2 AEs reported by physicians were: diarrhea 34.4%, nausea 21.9%, hypotension/hypertension 15.6%. The most common G3/4 AEs were diarrhea 6.3%, cardiac dysfunction 6.3% and other 15.6%. CTCAE PRO revealed dry mouth 91.3%, fatigue 82.6%, anorexia 82.6% and dry skin 82.6% as the most frequent symptoms. CTCAE PRO that patients reported to be severe were fatigue 43.5%, dry mouth 39.1%, anorexia 34.8%, dry skin 34.8% and dysphonia 34.8%. The most frequent CTCAE PRO that interfered with usual activities were fatigue 78.3%, dyspnea 69.6% and problems with concentration 69.6%. The return rate for FKSI-19 questionnaires was 71%. Longitudinal assessment of FKSI-19 items will be reported at the meeting. Functional/wellbeing subscale indicated that all patients (100%) reported impairment for their ability to work, to enjoy life or their quality of life. FKSI-19 disease related symptom scores reported lack of appetite, lack of energy and sleep disturbances among respondents in 92.3%, 92.3% and 88.5%, respectively. Shortness of breath and diarrhea were recognized in 61.5% and 69.2% of respondents, respectively. Haematuria was reported in 7.7% and no patient complaint of fever (0%). Detailed analysis will be shown at the meeting. Conclusions: Our findings support the use of PROs to assess patient’s condition and well-being. CTCAE-PRO and FKSI-19 were able to describe impairments, which were highly relevant to patients, but may not routinely reported by physicians. Each tool adds valuable information to the complex pattern of patient’s experience during treatment. However, some items had a very low response rate and should be reconsidered for their relevance. Clinical trial information: 7296 .