MPNs Clinical Trials Anthony J Messina MSc CCRP 12 Quentin E OBrien MPH 2 Vasily Andrianov MD 1 Keren R Moss MD 1 Laura Vidal MD ID: 931560
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Slide1
Underreporting
of Patient Reported Outcomes (PRO) in Myeloproliferative Neoplasms (
MPNs)
Clinical
Trials
Anthony J. Messina
,
MSc
, CCRP 1,2 Quentin E. O’Brien, MPH 2* Vasily Andrianov, MD 1 Keren R. Moss, MD 1* Laura Vidal, MD1* Liat Vidal, MD, MSc 1*
Conclusion
A
substantial
number
of MPN randomized trials did not include a PRO measure. Disease-specific PRO measures were used in 52% of the studies that included a PRO.In 37% of trials, PRO data was not reported. Even when reported, the report was often late after the initial report and reported partially. ImplicationsThe collection of PRO data should result in routine, timely and appropriate reporting as part of the trial outcome publication to allow for a thorough assessment of investigational drug treatment effects. Investigations should focus on causal factors that influence the underreporting of PRO data inclusion in trial publications such as: (1) Logistics with trial design (2) Missing data or difficulty with data collection in patients with advanced or progressive disease (3) Utilization of disease-specific PRO measures.
BackgroundPatient-reported outcomes provide unique and meaningful insight about the effect of a treatment from a patient’s view. Patients with MPNs today can live with their disease for long periods of time. Therefore a comprehensive evaluation of treatment effectiveness should consider the patient burden of the disease and treatment effect. ObjectiveTo evaluate the frequency at which PRO measures are utilized as study endpoints and are made publicly available when trial results are published.MethodsWe searched Citeline® Trialtrove database for randomized clinical trials including patients with Myeloproliferative Neoplasms, initiated between 2006-2016, utilizing at least 1 PRO.We searched PubMed Medline (Ovid), Google Scholar, ClinicalKey, and CINAHL databases for publications associated with the trial, and recorded type of publication year, number of randomized patients, and reported outcomes.
1- Syneos Health Oncology & Hematology, 2- George Washington University School of Medicine and Health Science *Non-ASH Members There are no relationships to disclose.
Abbreviations: AFFIRM v2, Armed Forces Institute of Rehabilitation Medicine; CML, Chronic Myelogenous Leukemia; Comb, Combined; EORTC-QLQ-C30,European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30; EQ-5D, EuroQol 5 Dimensions; FACT-An, Functional Assessment of Cancer Therapy Anemia; FACT-BRM, Functional Assessment of Cancer Therapy biologic response modifier; FACT-G, Functional Assessment of Cancer Therapy General; FACT-Leu, Functional Assessment of Cancer Therapy Leukemia; HRQOL, Health Related Quality of Life; MFS-AF, Myelofibrosis Symptom Assessment Form; MPNs, Myeloproliferative Neoplasms; MPN-SAF, Myeloproliferative Neoplasms Symptom Form; OS, overall survival; PGIC, Patient Global Impression of Change; PRO, Patient Reported Outcome; PROM, Patient Reported Outcome Measure; PV, Polycythemia Vera; PSIS, Pruritus Symptom Impact Scale; Qol, Quality of Life; SF-36, Short Form Health Survey 36 Item; TSS, Total Symptom Score; W/O, Without; WPAI, Work Productivity and Activity Impairment Questionnaire
Trials identified through database searching
n=343
Inclusion Criteria applied n=35
n=30
Trials excluded
n= 5
Exclusion Criteria
Supportive care (n=2)Lack of publication or trial terminated w/0 results ( n=3)
Inclusion CriteriaMyeloproliferative NeoplasmsTrials conducted between 2006-2016Randomized clinical trials
Trials
with
≥1 PRO endpointn=19
CMLn=8
Mastocytosis n=2
PV
n=4
Myelofibrosis
n=2
Comb. MPN
n=3
Trials with no PRO endpointn=11
Results
19 trials (19/30; 63%) included at least one PRO assessment as an endpoint (Table 2). Among these 19 trials, a variety of PROs was utilized to measure symptoms, functional health, psychological and somatic HRQOL (Figure 2).
Figure 1
Figure 2
Table 1
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Seven
studies (36.8%) that included a PRO
failed
to publish PRO data.
13/19 trials that had a PRO measure (68.4%) were sponsored by industry, three (15.8%) by industry-academic, and three (15.8%) solely by academic institutions.
Trial Characteristics n/N%Total number of MPN studies identified35100Potentially included studies 30 Disease states Chronic Myelogenous Leukemia (CML)20/3557.1Polycythemia Vera (PV)2/355.7Primary Myelofibrosis 5/3514.3Mastocytosis2/355.7Essential Thrombocythemia1/352.9Combination of MPNs3/358.6*Values were rounded to a single decimal point
Publication Resultsn/N%Included Studies (Studies with ≥1 PRO)19/3063.3PRO as primary endpoint3/1915.8PRO as secondary or exploratory endpoint16/1984.2Studies with published PRO data12/1963.2Comprehensive PRO data published 9/1947.4Partial PRO data published3/1915.88PRO data in initial publication6/1250.0PRO data published >6 months after initial publication4/1233.3*Values were rounded to a single decimal point
Table 2