TY - JOUR
T1 - Factors affecting symptom presentation in an early-phase clinical trials clinic patient population
AU - George, Goldy C.
AU - Mendoza, Tito R.
AU - Iwuanyanwu, Eucharia C.
AU - Manandhar, Meryna
AU - Afshar, Solmaz F.
AU - Piha-Paul, Sarina A.
AU - Tsimberidou, Apostolia
AU - Naing, Aung
AU - Cleeland, Charles S.
AU - Hong, David S.
N1 - Publisher Copyright:
© 2019, Springer Science+Business Media, LLC, part of Springer Nature.
PY - 2020/8/1
Y1 - 2020/8/1
N2 - Increasing numbers of oncology therapies are being approved based on early-phase single-arm studies. Yet, little is known regarding the use of patient-reported outcomes in single-arm oncology trials testing novel therapies. We examined patient-reported symptom severity and symptom interference with activity- (WAW: work, general activity, walking) and mood-(REM: relations with others, enjoyment of life, mood) related functioning, and their association with factors known to influence symptom severity reporting, in early-phase clinical trials clinic patients. Patients completed the validated MD Anderson Symptom Inventory, containing 13 severity items and six interference items, each rated on a 0–10 scale (higher scores = worse symptom severity/interference). Performance status (ECOG-PS) and age were ascertained. Multiple linear regression was performed. In 248 phase I patients (51% female, 90% ECOG 0–1, and 74% ≤65 years), 67% of patients had ≥seven concurrent symptoms of any severity level, and 51% of patients described ≥three concurrent symptoms as moderate-to-severe (severity rating ≥ 5). Composite symptom severity, WAW and REM were worse in patients with ECOG-PS ≥ 2 vs. 0–1, and worse in patients with ECOG-PS = 1 than in patients with ECOG-PS = 0. Compared with patients over 65y, adolescent and young adult (AYA) patients (18y-39y) and patients aged 40y to 65y had worse composite symptom severity. As expected, being employed full-time/retired was associated with better symptom profiles in phaseI patients. The variation of symptom burden by performance status and age suggest that these factors need to be considered in the design of early-phase trials, particularly if patient-reported symptoms are used as primary/secondary/exploratory endpoints.
AB - Increasing numbers of oncology therapies are being approved based on early-phase single-arm studies. Yet, little is known regarding the use of patient-reported outcomes in single-arm oncology trials testing novel therapies. We examined patient-reported symptom severity and symptom interference with activity- (WAW: work, general activity, walking) and mood-(REM: relations with others, enjoyment of life, mood) related functioning, and their association with factors known to influence symptom severity reporting, in early-phase clinical trials clinic patients. Patients completed the validated MD Anderson Symptom Inventory, containing 13 severity items and six interference items, each rated on a 0–10 scale (higher scores = worse symptom severity/interference). Performance status (ECOG-PS) and age were ascertained. Multiple linear regression was performed. In 248 phase I patients (51% female, 90% ECOG 0–1, and 74% ≤65 years), 67% of patients had ≥seven concurrent symptoms of any severity level, and 51% of patients described ≥three concurrent symptoms as moderate-to-severe (severity rating ≥ 5). Composite symptom severity, WAW and REM were worse in patients with ECOG-PS ≥ 2 vs. 0–1, and worse in patients with ECOG-PS = 1 than in patients with ECOG-PS = 0. Compared with patients over 65y, adolescent and young adult (AYA) patients (18y-39y) and patients aged 40y to 65y had worse composite symptom severity. As expected, being employed full-time/retired was associated with better symptom profiles in phaseI patients. The variation of symptom burden by performance status and age suggest that these factors need to be considered in the design of early-phase trials, particularly if patient-reported symptoms are used as primary/secondary/exploratory endpoints.
KW - Adolescent and young adult (AYA)
KW - Clinical trial
KW - ECOG performance status
KW - Patient reported outcome
KW - Symptom
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U2 - 10.1007/s10637-019-00879-0
DO - 10.1007/s10637-019-00879-0
M3 - Article
C2 - 31761957
AN - SCOPUS:85075390789
SN - 0167-6997
VL - 38
SP - 1166
EP - 1174
JO - Investigational New Drugs
JF - Investigational New Drugs
IS - 4
ER -