Conducting biomedical research using smartphones is a novel approach to studying health and disease that is only beginning to be meaningfully explored. Gathering large-scale, real-world data to track ...disease manifestation and long-term trajectory in this manner is quite practical and largely untapped. Researchers can assess large study cohorts using surveys and sensor-based activities that can be interspersed with participants' daily routines. In addition, this approach offers a medium for researchers to collect contextual and environmental data via device-based sensors, data aggregator frameworks, and connected wearable devices. The main aim of the SleepHealth Mobile App Study (SHMAS) was to gain a better understanding of the relationship between sleep habits and daytime functioning utilizing a novel digital health approach. Secondary goals included assessing the feasibility of a fully-remote approach to obtaining clinical characteristics of participants, evaluating data validity, and examining user retention patterns and data-sharing preferences. Here, we provide a description of data collected from 7,250 participants living in the United States who chose to share their data broadly with the study team and qualified researchers worldwide.
Participant recruitment can be a significant bottleneck in carrying out research studies. Connected health and mobile health platforms allow for the development of Web-based studies that can offer ...improvement in this domain. Sleep is of vital importance to the mental and physical health of all individuals, yet is understudied on a large scale or beyond the focus of sleep disorders. For this reason and owing to the availability of digital sleep tracking tools, sleep is well suited to being studied in a Web-based environment.
The aim of this study was to investigate a method for speeding up the recruitment process and maximizing participant engagement using a novel approach, the Achievement Studies platform (Evidation Health, Inc, San Mateo, CA, USA), while carrying out a study that examined the relationship between participant sleep and daytime function.
Participants could access the Web-based study platform at any time from any computer or Web-enabled device to complete study procedures and track study progress. Achievement community members were invited to the study and assessed for eligibility. Eligible participants completed an electronic informed consent process to enroll in the study and were subsequently invited to complete an electronic baseline questionnaire. Then, they were asked to connect a wearable device account through their study dashboard, which shared their device data with the research team. The data were used to provide objective sleep and activity metrics for the study. Participants who completed the baseline questionnaires were subsequently sent a daily single-item Sleepiness Checker activity for 7 consecutive days at baseline and every 3 months thereafter for 1 year.
Overall, 1156 participants enrolled in the study within a 5-day recruitment window. In the 1st hour, the enrollment rate was 6.6 participants per minute (394 per hour). In the first 24 hours, the enrollment rate was 0.8 participants per minute (47 participants per hour). Overall, 1132 participants completed the baseline questionnaires (1132/1156, 97.9%) and 1047 participants completed the initial Sleepiness Checker activity (1047/1156, 90.6%). Furthermore, 1000 participants provided activity-specific wearable data (1000/1156, 86.5%) and 982 provided sleep-specific wearable data (982/1156, 84.9%).
The Achievement Studies platform allowed for rapid recruitment and high study engagement (survey completion and device data sharing). This approach to carrying out research appears promising. However, conducting research in this way requires that participants have internet access and own and use a wearable device. As such, our sample may not be representative of the general population.
Introduction Understanding the patient voice and experience is an integral part of clinical care. The American Sleep Apnea Association organized and hosted a Patient-Focused Medical Product ...Development (PFMPD) meeting for the FDA in June 2018. The PFMPD meeting was the first of its kind, focused on medical products in addition to medications. Prior to the PFMPD, similar Patient-Focused Drug Development (PFDD) meetings were held by the FDA beginning in 2012 as a forum for patient communities about their experiences living with their respective conditions and the treatments they are using. Methods A national survey was conducted. It was comprised of 32-items that were a combination of multiple choice and open-text fields. Sections included diagnosis, symptoms, impact of sleep apnea on daily living, treatments and impact of treatments. The survey was developed as a “fit-for-purpose” instrument for use in conjunction with the FDA’s PFDD initiative and was therefore informed by the focus of past PFDD meeting surveys and related medical literature. It was tested and refined by project staff and in a group of patients. It took an average of 17 minutes to complete. The survey was widely publicized, sent to email lists and promoted within social media. All responses were anonymous. Results The survey was available for completion between April and August 2018 and attracted a total of 5,630 responses, 85% of whom were sleep apnea patients and 14% were family or friends of a patient. 57% of respondents were female and 85% were diagnosed by a physician. 34% of respondents on CPAP continued to report moderate to severe daytime symptoms. 53% identified potential long-term consequences of OSA on health and lifespan as their top concern. 70% reported using CPAP, and of those, 82% reported using it 7 nights per week and 6.2 hours per night. Conclusion The PFMPD survey conducted by the ASAA is a rich source of the sleep apnea patient’s experience encompassing recognition, diagnosis, symptoms, and treatment. Support (If Any) The FDA AWAKE meeting was hosted by the American Sleep Apnea Association.
Although treatment of obstructive sleep apnea (OSA) with positive airway pressure (PAP) therapy is effective, adherence is often poor. Understanding the patient perspective is needed to inform ...adherence-promoting interventions. This qualitative study assessed the experiences, preferences, facilitators, and barriers surrounding PAP therapy for the management of OSA in patients from adolescence to older adulthood.
Eligible participants ages 19 and older were identified from administrative health care claims; adolescent participants ages 12-18 and their parents/caregivers were identified via electronic health records of a tertiary sleep specialty clinic at a large children's hospital. Forty English-speaking patients and 10 parents of adolescents diagnosed with OSA and prescribed PAP therapy completed semistructured 60-minute telephone interviews conducted by a trained facilitator. Common themes and illustrative quotes were identified.
Themes around OSA diagnosis, initiating OSA treatment, learning about OSA/PAP, decision to start PAP, PAP benefits and challenges, and reasons for nonadherence were identified. Participants suggested design and delivery changes to improve PAP devices. Issues unique to adolescents and their parents were discussed.
The unique perspectives of patients regarding PAP therapy should be taken into consideration when developing interventions to increase PAP adherence and improve clinical care. Based on identified themes, opportunities for intervention may exist at all stages of care, from diagnosis to treatment initiation. Involving partners, parents, and other caregivers in PAP therapy may be beneficial for optimizing adherence.
Simon SL, Stephenson JJ, Haynes K, et al. The lived experience of positive airway pressure therapy in patients with obstructive sleep apnea across the lifespan: a qualitative study.
. 2024;20(3):407-416.
Introduction This study sought to take advantage of technological and methodological advancements in the field of Mobile Health and apply them to a large-scale longitudinal research study on sleep ...and activity. Large-scale studies of sleep patterns, quality, and associated characteristics substantiated by objective data are lacking. Utilizing a novel platform, we sought to gain further insights into the relationship between sleep and activity while simultaneously speeding up the recruitment process and maximizing data completeness. Methods A subset of Achievement community members were invited to the study and assessed for eligibility. Eligible participants completed an electronic consent process and a series of baseline questionnaires. Participants were asked to connect a wearable device through the study dashboard, which allowed objective sleep and activity data to be collected. Participants could access the study platform from any computer or connected mobile device. After successful completion of the baseline questionnaires, participants were sent a daily single-item Sleepiness Checker activity (Karolinska Sleepiness Scale) for 7 consecutive days at baseline and every 3 months thereafter for 1 year. Results During a 5-day recruitment period, 1156 participants enrolled in the study. 98% of enrolled participants completed baseline questionnaires and 91% completed a baseline sleepiness checker activity. Mean days of sleepiness checker completed was 5.7±1.7 (1-7), with 51% of participants completing 7 consecutive days. 85% of participants provided sleep-specific wearable data, and 87% provided activity-specific wearable data. At baseline, wearable data indicated that participants slept an average of 302.8±98.1 minutes per night (63-576) and self-reported TST was 391.2±108 minutes. Participant retention at one year was 60%, with 40% of those participants completing 7/7 days of the sleepiness checker. Conclusion Conducting online, community-based longitudinal studies that include objective sleep and activity data is an innovative approach to expanding sleep research. As is the case with all large community-based studies, data quality and representativeness of the sample to the overall population must be carefully examined. Support (If Any) This study was supported in part by the American Sleep Apnea Association and Evidation Health.
Mobile technologies offer the potential to reduce the costs of conducting clinical trials by collecting high-quality information on health outcomes in real-world settings that are relevant to ...patients and clinicians. However, widespread use of mobile technologies in clinical trials has been impeded by their perceived challenges. To advance solutions to these challenges, the Clinical Trials Transformation Initiative (CTTI) has issued best practices and realistic approaches that clinical trial sponsors can now use. These include CTTI recommendations on technology selection; data collection, analysis, and interpretation; data management; protocol design and execution; and US Food and Drug Administration submission and inspection. The scientific principles underpinning the clinical trials enterprise continue to apply to studies using mobile technologies. These recommendations provide a framework for including mobile technologies in clinical trials that can lead to more efficient assessment of new therapies for patients.
To report the results of a phase II clinical trial of de-intensified chemoradiotherapy for patients with human papillomavirus-associated oropharyngeal squamous cell carcinoma.
Major inclusion ...criteria were (1) having American Joint Committee on Cancer (AJCC) 7th edition T0-T3, N0-N2c, M0 (AJCC 8th edition T0-T3, N0-N2, M0), (2) being p16 positive, and (3) reporting minimal or remote smoking history. Treatment was limited to 60 Gy intensity-modulated radiotherapy with concurrent intravenous cisplatin 30 mg/m
once per week. Patients with T0-T2 N0-1 (AJCC 7th edition) did not receive chemotherapy. All patients had a 10- to 12-week post-treatment positron emission tomography/computed tomography to assess for neck dissection. The primary end point was 2-year progression-free survival. Secondary end points included 2-year local-regional control, distant metastasis-free survival and overall survival, and patient-reported outcomes (European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire and the patient-reported outcomes version of the Common Terminology Criteria for Adverse Events).
One hundred fourteen patients were enrolled (median follow-up of 31.8 months), with 81% having a minimum follow-up of 2 years. Eighty percent of patients had 10 or fewer tobacco pack-years. Two-year local-regional control, distant metastasis-free survival, progression-free survival, and overall survival were as follows: 95%, 91%, 86%, and 95%, respectively. Mean pre- and 2-year post-treatment European Organisation for Research and Treatment of Cancer quality of life scores were as follows: global, 79/84 (lower worse); swallowing, 8/9 (higher worse); and dry mouth, 14/45 (higher worse). Mean pre- and 2-year post-treatment patient-reported outcomes version of the Common Terminology Criteria for Adverse Events scores (0 to 4 scale, higher worse) were as follows: swallowing, 0.5/0.7, and dry mouth, 0.4/1.3. Thirty-four percent of patients required a feeding tube (median, 10.5 weeks; none permanent). There were no grade 3 or higher late adverse events.
Clinical outcomes with a de-intensified chemoradiotherapy regimen of 60 Gy intensity-modulated radiotherapy with concurrent low-dose cisplatin are favorable in patients with human papillomavirus-associated oropharyngeal squamous cell carcinoma. Neither neoadjuvant chemotherapy nor routine surgery is needed to obtain favorable results with de-escalation.
To perform a prospective, multi-institutional, phase 2 study of a substantial decrease in concurrent chemoradiation therapy (CRT) intensity as primary treatment for favorable-risk, human ...papillomavirus-associated oropharyngeal squamous cell carcinoma.
The major inclusion criteria were: (1) T0 to T3, N0 to N2c, M0; (2) human papillomavirus or p16 positive; and (3) minimal/remote smoking history. Treatment was limited to 60 Gy intensity modulated radiation therapy with concurrent weekly intravenous cisplatinum (30 mg/m(2)). The primary study endpoint was pathologic complete response (pCR) rate based on required biopsy of the primary site and dissection of pretreatment positive lymph node regions, regardless of radiographic response. Power computations were performed for the null hypothesis that the pCR rate is 87% and n=40, resulting in a type 1 error of 14.2%. Secondary endpoint measures included physician-reported toxicity (Common Toxicity Terminology for Adverse Events, CTCAE), patient-reported symptoms (PRO-CTCAE), and modified barium swallow studies.
The study population was 43 patients. The pCR rate was 86% (37 of 43). The incidence of CTCAE grade 3/4 toxicity and PRO-CTCAE severe/very severe symptoms was as follows: mucositis 34%/45%, general pain 5%/48%, nausea 18%/52%, vomiting 5%/34%, dysphagia 39%/55%, and xerostomia 2%/75%. Grade 3/4 hematologic toxicities were 11%. Thirty-nine percent of patients required a feeding tube for a median of 15 weeks (range, 5-22 weeks). There were no significant differences in modified barium swallow studies before and after CRT.
The pCR rate with decreased intensity of therapy with 60 Gy of IMRT and weekly low-dose cisplatinum is very high in favorable-risk oropharyngeal squamous cell carcinoma, with evidence of decreased toxicity compared with standard therapies. ClinicalTrials.gov ID: NCT01530997.
Plasma circulating tumor human papillomavirus DNA (ctHPVDNA) is a sensitive and specific biomarker of human papillomavirus (HPV)-associated oropharyngeal squamous cell carcinoma (OPSCC). We ...investigated whether longitudinal monitoring of ctHPVDNA during post-treatment surveillance could accurately detect clinical disease recurrence.
A prospective biomarker clinical trial was conducted among patients with nonmetastatic HPV-associated (p16-positive) OPSCC. All patients were treated with curative-intent chemoradiotherapy (CRT). Patients underwent a 3-month post-CRT positron emission tomography/computed tomography scan and were thereafter clinically evaluated every 2-4 months (years 1-2), then every 6 months (years 3-5). Chest imaging was performed every 6 months. Blood specimens were collected every 6-9 months for analysis of plasma ctHPVDNA using a multianalyte digital polymerase chain reaction assay. The primary endpoint was to estimate the negative predictive value (NPV) and positive predictive value (PPV) of ctHPVDNA surveillance.
One hundred fifteen patients were enrolled, and 1,006 blood samples were analyzed. After a median follow-up time of 23 months (range, 6.1-54.7 months), 15 patients (13%) developed disease recurrence. Eighty-seven patients had undetectable ctHPVDNA at all post-treatment time points, and none developed recurrence (NPV, 100%; 95% CI, 96% to 100%). Twenty-eight patients developed a positive ctHPVDNA during post-treatment surveillance, 15 of whom were diagnosed with biopsy-proven recurrence. Sixteen patients had 2 consecutively positive ctHPVDNA blood tests, 15 of whom developed biopsy-proven recurrence. Two consecutively positive ctHPVDNA blood tests had a PPV of 94% (95% CI, 70% to 99%). Median lead time between ctHPVDNA positivity and biopsy-proven recurrence was 3.9 months (range, 0.37-12.9 months).
Detection of ctHPVDNA in two consecutive plasma samples during post-treatment surveillance has high PPV and NPV for identifying disease recurrence in patients with HPV-associated oropharyngeal cancer and may facilitate earlier initiation of salvage therapy.
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