Patient-Reported AutoImmunity Secondary to Cancer immunothErapy

Abstract Background Patient-reported outcomes (PROs) promote patient centeredness in clinical trials; however, in the field of rapidly emerging and clinically impressive immunotherapy, data on PROs are limited. Methods We systematically identified all immunotherapy approvals from 2011 through 2018 and assessed the analytic tools and reporting quality of associated PRO reports. For randomized clinical trials (RCTs), we developed a novel 24-point scoring scale: the PRO Endpoints Analysis Score based on 24 criteria derived from the recommendations of the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium. Results We assessed 44 trial publications supporting 42 immunotherapy approvals. PROs were published for 21 of the 44 (47.7%) trial publications. Twenty-three trials (52.3%) were RCTs and 21 (47.7%) pertained to single-arm trials. The median time between primary clinical outcomes publications and their corresponding secondary PRO publications was 19 months (interquartile range = 9-29 months). Of the 21 PRO reports, 4 (19.0%) reported a specific hypothesis, and most (85.7%) used descriptive statistics. Three (3 of 21 [14.3%]) studies performed a control for type I error. As for RCTs, 14 of 23 (60.9%) published PRO data, including 13 (56.5%) that published a secondary dedicated manuscript. One-half of these 14 trials scored less than 13 points on the 24-point PRO Endpoints Analysis Score. The mean score was 12.71 (range = 5-17, SD = 3.71), and none met all the recommendations of the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium. Conclusions Suboptimal reporting of PROs occurs regularly in cancer immunotherapy trials. Increased efforts are needed to maximize the value of these data in cancer immunotherapy development and approval.

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Trends in PRO reporting in clinical trials leading to cancer immunotherapy drug approvals from 2011 to 2019.

Journal of Clinical Oncology ◽

10.1200/jco.2020.38.5_suppl.105 ◽

2020 ◽

Vol 38 (5_suppl) ◽

pp. 105-105

Author(s):

Houssein Safa ◽

Monica Tamil ◽

Adi Diab ◽

Adele Semaan ◽

Jad Chahoud

Keyword(s):

Clinical Trials ◽

Cancer Immunotherapy ◽

Current Trend ◽

Type I ◽

Patient Centeredness ◽

Quality Reporting ◽

Study Protocols ◽

Fda Approval ◽

Patient Reported ◽

Drug Approvals

105 Background: Patient-reported outcomes (PROs) are a primary tool to evaluate the effect of a given drug on the health-related quality of life from a patient’s perspective and to maximize the value of patient-centeredness in drug development and approval. In this study, we aimed to evaluate the current trend in reporting, analysis and interpretation of PRO data. Methods: We conducted a systematic review of the FDA archives, identifying all cancer immunotherapy drug approvals between the years 2011 and 2019. We then retrieved the clinical trials that respectively led to these drug approvals from PubMed and ClinicalTrials.gov. We systematically screened for PROs and assessed their analytic tools and interpretation methods that were collected from the published journal articles and their study protocols. If one FDA approval was supported by more than one clinical trial, we included all studies in our review. An FDA approval was considered to include PROs if they were reported in at least one of the supporting trials. Results: Thirty-seven clinical trials leading to 35 immunotherapy drug approvals were identified. More than half (54%) did not publish any PROs. While PROs were reported in the primary clinical outcomes manuscript of 4 trials (11%) and in a secondary separately published paper for 13 trials (35%). The median time between the primary and secondary papers was 22 months (range: 5 - 40). In the 17 published PROs, the hypothesis was broad in 12 (71%), not reported in 4 (24%) and specific in only 1 (6%). Ten (59%) were reported as exploratory endpoints, five (29%) as secondary endpoints, and two (12%) did not specify the PRO reporting in their endpoints. The most common PRO instruments were EQ-5D (71%) and QLQ-C30 (65%). Control for type I error was needed but not done in 15 (88%). Fourteen (82%) described an approach for dealing with missing PRO assessments. None reported on significant differences based on race and ethnicity of participants. Conclusions: Suboptimal reporting and delays in publication of PROs occur regularly in cancer immunotherapy trials. Increased efforts are needed to enhance standardization and quality reporting of PROs to maximize the value of this data in cancer immunotherapy drug approval.

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Patient reported benefit of the efficacy of speech therapy in dysphonia

Clinical Otolaryngology ◽

10.1046/j.1365-2273.1998.0138n.x ◽

1998 ◽

Vol 23 (3) ◽

pp. 284-285 ◽

Cited By ~ 2

Author(s):

Mackenzie ◽

Deary ◽

Sellars ◽

Wilson

Keyword(s):

Speech Therapy ◽

Patient Reported

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Cancer Immunotherapy

10.1016/s1877-1173(19)x0006-x ◽

2019 ◽

Keyword(s):

Cancer Immunotherapy

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Association of Physical Morbidity and Health-Related Quality of Life in a Representative Sample of Older German People

European Journal of Health Psychology ◽

10.1027/2512-8442/a000019 ◽

2018 ◽

Vol 25 (4) ◽

pp. 140-151

Author(s):

Markus A. Wirtz ◽

Matthias Morfeld ◽

Elmar Brähler ◽

Andreas Hinz ◽

Heide Glaesmer

Keyword(s):

Quality Of Life ◽

Representative Sample ◽

Short Form ◽

Physical Symptoms ◽

German Population ◽

Health Related Quality ◽

Patient Reported ◽

Related Quality ◽

Health Related

Abstract. The association between health-related quality of life (HRQoL; Short-Form Health Survey-12; SF-12) and patient-reported morbidity-related symptoms measured by the Patient Health Questionnaire-15 (PHQ-15) is analyzed in a representative sample of older people in the general German population. Data from 1,659 people aged 60 to 85 years were obtained. Latent class analysis identified six classes of patients, which optimally categorize clusters of physical symptoms the participants reported: musculoskeletal impairments (39.8%), healthy (25.7%), musculoskeletal and respiratory/cardiac impairments (12.8%), musculoskeletal and respiratory impairments, along with bowel and digestion problems (12.9%), general impairments (4.9%), and general impairments with no bowel and digestion problems (4.8%). The participants’ SF-12 Physical Health Scores (η2 = .39) and their Mental Health Scores (η2 = .28) are highly associated with these latent classes. These associations remain virtually identical after controlling for age. The results provide evidence that profiles of patient-reported physical impairments correspond strongly with reduced HRQoL independently from aging processes.

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