Dataset Information

Patient-reported outcome instruments used in immune-checkpoint inhibitor clinical trials in oncology: a systematic review.

ABSTRACT:

Context

Immune-checkpoint inhibitors (ICI) have shown significant benefits for overall survival across various cancer types. Patient-reported outcomes (PROs) are assessed in clinical trials as a measure of efficacy. However, it remains unclear to what extent current PRO instruments capture symptoms specific to ICI toxicities. We conducted a systematic review to identify the use and content validity of PRO instruments in ICI clinical trials in oncology.

Methods

Literature was retrieved from PubMed, Embase, PsycINFO, Medline and CINAHL databases. Articles presenting ICI clinical trials' PRO results, clinical trial study protocols, and conference abstracts stating the use of PRO measures were assessed. We evaluated the validity of identified instruments by comparing their symptom-related content with the adverse events reported in each ICI clinical trial.

Results

From database inception until January 2020, we identified 191 ICI clinical trials stating the use of PRO measures of which 26 published PRO results. The cancer-specific EORTC QLQ-C30 and the generic EQ-5D questionnaires were the most widely used instruments, often in combination with disease-specific PROs. Instruments used to report PRO symptom-related toxicities covered 45% of the most frequently reported AEs, whereas 23% of AEs were partially covered and 29% were not covered at all. Of non-covered AEs, 59% referred to the dermatologic system. Partially covered AEs related to endocrine and specific types of pain.

Conclusion

Despite the high frequency of symptom-related toxicities related to ICI, these events are only partially covered (or not addressed) by current PRO instruments, even when combined. Further research is needed to develop new strategies to tailor PRO instruments to specific ICI toxicities.

SUBMITTER: Colomer-Lahiguera S

PROVIDER: S-EPMC7364679 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Patient-reported outcome instruments used in immune-checkpoint inhibitor clinical trials in oncology: a systematic review.

Colomer-Lahiguera Sara S Bryant-Lukosius Denise D Rietkoetter Sarah S Martelli Lorraine L Ribi Karin K Fitzpatrick-Lewis Donna D Sherifali Diana D Orcurto Angela A Juergens Rosalyn R Eicher Manuela M

Journal of patient-reported outcomes 20200716 1

<h4>Context</h4>Immune-checkpoint inhibitors (ICI) have shown significant benefits for overall survival across various cancer types. Patient-reported outcomes (PROs) are assessed in clinical trials as a measure of efficacy. However, it remains unclear to what extent current PRO instruments capture symptoms specific to ICI toxicities. We conducted a systematic review to identify the use and content validity of PRO instruments in ICI clinical trials in oncology.<h4>Methods</h4>Literature was retri ...[more]

PMID: 32676785

Similar Datasets

Project description:BACKGROUND:Patient-reported outcome measures (PROMs) have become widely adopted in the care of patients with prostate cancer, but there is no validated crosswalk between two commonly used instruments, the Expanded Prostate Cancer Index Composite Short Form (EPIC-26) and the Memorial Sloan Kettering (MSK) instrument, which consists of the International Index of Erectile Function-6 (IIEF-6) questionnaire and the MSK radical prostatectomy urinary outcome scale. OBJECTIVE:To develop and validate bidirectional crosswalks between the sexual and urinary domains (single domain in MSK, separate incontinence and irritative/obstructive domains in EPIC-26) of the MSK and EPIC-26 instruments. DESIGN, SETTING, AND PARTICIPANTS:Radical prostatectomy (RP) patients completing instruments at MSK and Michigan Urological Surgery Improvement Collaborative (MUSIC) between January and May of 2017 were invited to enroll. Stratified random sampling (by institution, MSK urinary function score, and MSK erectile function score) was used to divide patient data into training and test sets. Models were developed to predict the domain score for each instrument using the other's item responses and domain scores. Performance was evaluated using capped root-mean-squared error and accuracy at established thresholds. RESULTS AND LIMITATIONS:We received 517 instruments at MSK and 1033 within MUSIC, which were assigned to training (825), post-RP test (412), and pre-RP test (313) sets. We found the crosswalks to have low error and high accuracy. Although the crosswalks are more accurate if responses to each item are known, it is possible to convert between instruments on the basis of a total domain score. CONCLUSIONS:The crosswalks are a valid way to convert between sexual and urinary domains of the MSK and EPIC-26 instruments. PATIENT SUMMARY:We developed and validated a set of formulas that allow conversion of sexual and urinary function scores between the Memorial Sloan Kettering and Expanded Prostate Cancer Index Composite Short Form patient-reported outcome questionnaires. These crosswalks allow seamless transition between the two questionnaires.

Dataset Information

Patient-reported outcome instruments used in immune-checkpoint inhibitor clinical trials in oncology: a systematic review.

Context

Methods

Results

Conclusion

Publications

Patient-reported outcome instruments used in immune-checkpoint inhibitor clinical trials in oncology: a systematic review.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets