Pilot Study Research Articles

Evaluation of Safety and Beneficial Health Effects of the Human-Milk Strain Bifidobacterium breve DSM32583: An Infant Pilot Trial.

Human milk promotes the growth of bifidobacteria in the infant gut. Adding bifidobacterial species to infant formula may contribute to increasing their presence in the gut of formula-fed infants. Therefore, the safety and anti-infectious effects of Bifidobacterium breve DSM32583, a breast milk isolate, were assessed in a pilot trial involving 3-month-old infants. The infants were randomly assigned to either the probiotic (PG) or the control (CG) groups. All the infants consumed the same formula, although it was supplemented with the strain (1 × 107 cfu/g of formula) in the PG. Overall, 160 infants (80 per group) finished the intervention. Infants in CG gained more weight compared to PG (p < 0.05), but the weights for age Z-scores at 6 months were within the normal distribution for this age group. The rates of infections affecting the gastrointestinal and respiratory tracts and antibiotic therapy were significantly lower in the PG. The bifidobacterial population and the level of short-chain fatty acids were higher (p < 0.05) in the fecal samples of PG infants. No adverse events related to formula consumption were observed. In conclusion, the administration of an infant formula with B. breve DSM32583 was safe and exerted potential beneficial effects on gut health.

Determining the Degree of Perceptible Static Eyelid Asymmetry and Effect of Face Inversion: A Cross-sectional Pilot Study.

To determine the degree of static eyelid asymmetry required to be perceptible and whether this is affected by image inversion. Images of 3 volunteers were digitally manipulated to have eyelid asymmetry of 0.5 mm, 1 mm, or 1.5 mm of 3 different types, upper lid ptosis, upper lid retraction, and lower lid retraction. Forty-nine laypersons stated whether the images were symmetrical or asymmetrical. A separate inversion survey, completed by 29 clinicians, consisted of symmetrical images and 1 mm asymmetrical images, with half being inverted. Upper lid ptosis, upper lid retraction, and lower lid retraction were not detected by most laypeople at 0.5 mm of severity (18.9%, 6.7%, 18.9% detection, respectively) but all 3 were detected by the majority of participants once asymmetry reached 1 mm severity (65.7%, 61.8%, 51.0% detection, respectively) and rose to over 70% identification at 1.5 mm (92.2%, 73.5%, 73.5% detection, respectively). Inversion of the images led to 19.7% lower rates of correct identification of asymmetrical faces compared with images presented in the correct orientation (80.7% asymmetry identified in normal images, 61.0% inverted, p < 0.001). All asymmetries were detectable by a majority of laypersons at a severity of 1 mm. Image inversion decreases a clinician's ability to detect a 1 mm asymmetry, indicating an impaired asymmetry perception in the intraoperative view. This study provides research to counsel patients with varying degrees of eyelid asymmetry.

Head Injury Treatment With Healthy and Advanced Dietary Supplements: A Pilot Randomized Controlled Trial of the Tolerability, Safety, and Efficacy of Branched Chain Amino Acids in the Treatment of Concussion in Adolescents and Young Adults.

Concussion is a common injury in the adolescent and young adult populations. Although branched chain amino acid (BCAA) supplementation has shown improvements in neurocognitive and sleep function in pre-clinical animal models of mild-to-moderate traumatic brain injury (TBI), to date, no studies have been performed evaluating the efficacy of BCAAs in concussed adolescents and young adults. The goal of this pilot trial was to determine the efficacy, tolerability, and safety of varied doses of oral BCAA supplementation in a group of concussed adolescents and young adults. The study was conducted as a pilot, double-blind, randomized controlled trial of participants ages 11-34 presenting with concussion to outpatient clinics (sports medicine and primary care), urgent care, and emergency departments of a tertiary care pediatric children's hospital and an urban tertiary care adult hospital, between June 24, 2014 and December 5, 2020. Participants were randomized to one of five study arms (placebo and 15 g, 30 g, 45 g, and 54 g BCAA treatment daily) and followed for 21 days after enrollment. Outcome measures included daily computerized neurocognitive tests (processing speed, the a priori primary outcome; and attention, visual learning, and working memory), symptom score, physical and cognitive activity, sleep/wake alterations, treatment compliance, and adverse events. In total, 42 participants were randomized, 38 of whom provided analyzable data. We found no difference in our primary outcome of processing speed between the arms; however, there was a significant reduction in total symptom score (decrease of 4.4 points on a 0-54 scale for every 500 g of study drug consumed, p value for trend = 0.0036, [uncorrected]) and return to physical activity (increase of 0.503 points on a 0-5 scale for every 500 g of study drug consumed, p value for trend = 0.005 [uncorrected]). There were no serious adverse events. Eight of 38 participants reported a mild (not interfering with daily activity) or moderate (limitation of daily activity) adverse event; there were no differences in adverse events by arm, with only two reported mild adverse events (both gastrointestinal) in the highest (45 g and 54 g) BCAA arms. Although limited by slow enrollment, small sample size, and missing data, this study provides the first demonstration of efficacy, as well as safety and tolerability, of BCAAs in concussed adolescents and young adults; specifically, a dose-response effect in reducing concussion symptoms and a return to baseline physical activity in those treated with higher total doses of BCAAs. These findings provide important preliminary data to inform a larger trial of BCAA therapy to expedite concussion recovery.

Mindful Night-to-Day: A Pilot Feasibility Trial of a Mindfulness-Based Insomnia and Symptom Management Intervention for Patients with Hematologic Cancer

ABSTRACT Objectives Patients with hematologic cancer experience severe symptoms (i.e. insomnia, fatigue, pain, distress). Few interventions addressing insomnia and other symptoms exist for this population. Mindfulness-Based Therapy for Insomnia (MBTI) may be appropriate but has only been tested in healthy outpatients. This study aimed to develop and test an adapted MBTI protocol for hematologic cancer patients. Methods Patient (n = 3) and clinician (n = 1) focus groups, and user-testing (N = 5) informed adaptation of Mindful Night-to-Day (MBTI+). A single-arm pilot trial (N = 32) evaluated feasibility (accrual, attrition, adherence), acceptability (intervention satisfaction), and changes to insomnia symptom severity (Insomnia Severity Index; primary outcome) and secondary outcomes (fatigue, pain, distress, pre-sleep arousal, mindfulness, symptom management self-efficacy) at baseline, post-intervention, and 1-month post-intervention. Descriptive statistics and paired sample t-tests were conducted. Results Qualitative feedback informed MBTI+ content, format, and delivery. Mindfulness was used to increase symptom awareness (sleepiness vs. fatigue). Meditations and behavioral skills were applied to inpatient treatment. MBTI+ met feasibility (N = 32/12 months; 8.1% attrition; 83.8% adherence) and acceptability (M = 3.52/4.00) benchmarks. Insomnia symptom severity decreased (d = 1.20) from baseline to post-intervention, as did most secondary outcomes. Conclusions MBTI+ was feasible, acceptable, and showed promise for benefits throughout inpatient and outpatient treatment. Findings warrant further evaluation in a randomized trial.

An alternating-intervention pilot trial on the impact of an informational handout on patient-reported outcomes and follow-up after lung cancer screening.

Lung cancer screening (LCS) can reduce lung cancer mortality; however, poor understanding of results may impact patient experience and follow-up. We sought to determine whether an informational handout accompanying LCS results can improve patient-reported outcomes and adherence to follow-up. This was a prospective alternating intervention pilot trial of a handout to accompany LCS results delivery. Patients undergoing LCS in a multisite program over a 6-month period received a mailing containing either: 1) a standardized form letter of LCS results (control) or 2) the LCS results letter and the handout (intervention). A two-sided informational handout on commonly asked questions after LCS created through iterative mixed-methods evaluation with both LCS patients and providers. The primary outcomes of 1)patient understanding of LCS results, 2)correct identification of next steps in screening, and 3)patient distress were measured through survey. Adherence to recommended follow-up after LCS was determined through chart review. Outcomes were compared between the intervention and control group using generalized estimating equations. 389 patients were eligible and enrolled with survey responses from 230 participants (59% response rate). We found no differences in understanding of results, identification of next steps in follow-up or distress but did find higher levels of knowledge and understanding on questions assessing individual components of LCS in the intervention group. Follow-up adherence was overall similar between the two arms, though was higher in the intervention group among those with positive findings (p = 0.007). There were no differences in self-reported outcomes between the groups or overall follow-up adherence. Those receiving the intervention did report greater understanding and knowledge of key LCS components, and those with positive results had a higher rate of follow-up. This may represent a feasible component of a multi-level intervention to address knowledge and follow-up for LCS. ClinicalTrials.gov NCT05265897.

Usability and preliminary effectiveness of an app-based physical activity and education program for people with hip or knee osteoarthritis – a pilot randomized controlled trial

BackgroundHip and knee osteoarthritis (OA) are highly prevalent worldwide. The guidelines recommend physical activity and education as the core treatments for osteoarthritis. Digital health has the potential to engage people in physical activity and disease management. Therefore, we conducted a pilot trial to assess the usability and preliminary effectiveness of an app-based physical activity and education program (Join2Move) compared to usual care for people with hip and/or knee OA in Germany.MethodsA randomized controlled pilot study was conducted. Individuals with diagnosed or self-reported knee and hip OA were included. Allocation to the intervention or control group was randomized. The intervention group received the Join2Move program. The Join2Move program was previously developed as a website and evaluated in the Netherlands. For the current study, the program was translated and adapted to the German context and adjusted from a website to an app. The control group received usual care. The primary outcomes were usability and preliminary effectiveness (pain and physical functioning). Measurements were taken at baseline and at twelve weeks. The data analysis was performed using SPSS (IBM SPSS Statistics 29.0).ResultsSixty participants, with a mean age of 61.9 (SD ± 7.2) years, were allocated to the intervention (n = 32) or the control group (n = 28) and included in the analysis. The majority of participants had knee OA (68%), and 12% had hip and knee OA. The dropout rate was n = 11 (18%). No adverse events were reported. Usability was rated as acceptable (mean System Usability Scale = 71.3/100) with a wide range (32.5 to 100). Statistically significant between-group differences were found only for pain (mean difference 8.52 (95% CI 1.01 to 16.04), p = 0.027).ConclusionsJoin2Move demonstrated acceptable usability. The preliminary results of the pilot trial indicate the potential of a stand-alone app for the treatment of patients with hip or knee OA. However, the acceptable usability of Join2Move limits its recommendation for everyone. There appears to be room for improvement in app usability and in identifying patients for whom the app is suitable and the right time to use a stand-alone app.Trial registrationGerman Clinical Trials Register DRKS00027164.

An open label pilot trial of low-dose lithium for young people at ultra-high risk for psychosis.

Lithium, even at low doses, appears to offer neuroprotection against a wide variety of insults. In this controlled pilot, we examined the safety (i.e., side-effect profile) of lithium in a sample of young people identified at ultra-high risk (UHR) for psychosis. The secondary aim was to explore whether lithium provided a signal of clinical efficacy in reducing transition to psychosis compared with treatment as usual (TAU). Young people attending the PACE clinic at Orygen, Melbourne, were prescribed a fixed dose (450 mg) of lithium (n = 25) or received TAU (n = 78). The primary outcome examined side-effects, with transition to psychosis, functioning and measures of psychopathology assessed as secondary outcomes. Participants in both groups were functionally compromised (lithium group GAF = 56.6; monitoring group GAF = 56.9). Side-effect assessment indicated that lithium was well-tolerated. 64% (n = 16) of participants in the lithium group were lithium-adherent to week 12. Few cases transitioned to psychosis across the study period; lithium group 4% (n = 1); monitoring group 7.7% (n = 6). There was no difference in time to transition to psychosis between the groups. No group differences were observed in other functioning and symptom domains, although all outcomes improved over time. With a side-effect profile either comparable to, or better than UHR antipsychotic trials, lithium might be explored for further research with UHR young people. A definitive larger trial is needed to determine the efficacy of lithium in this cohort.

Clinical Remote Monitoring of Individuals With Spinal Cord Injury at Risk for Pressure Injury Recurrence Using mHealth: Protocol for a Pilot, Pragmatic, Hybrid Implementation Trial.

Pressure injuries are one of the most challenging secondary conditions for individuals with spinal cord injuries and related disorders (SCI/D) owing to inherent, lifelong risk factors that include a lack of sensory and motor function below the level of injury and reliance on a wheelchair for daily mobility, resulting in prolonged periods of sitting. Although many factors contribute to the development of pressure injuries, the pressure between the skin and a surface is always a factor and the development of injury is dependent on the magnitude and duration of the pressure. Clinically, broad recommendations for relieving pressure are used because we know very little about the unique day-to-day life patterns of the individual wheelchair user. Typically, it is after the occurrence of a pressure injury that the therapist will check equipment fit and the effectiveness of pressure offloading and ask about other surfaces they sit on in their home and community. This time-lapsed, largely self-reported data are fraught with recall bias and inaccuracies that the therapist incorporates into a plan of care. This study's objective is to pilot-test the implementation and clinical effectiveness of a telehealth model of care combined with our mobile health (mHealth) Assisted Weight-Shift device for remote monitoring of factors related to maintaining skin health and wheelchair setup. Our overall hypothesis is that this study will result in an effective implementation plan, and the enhanced connected model of care using remote monitoring of pressure management will result in pilot-level, improved clinical outcomes for adults with spinal cord injury at high risk for pressure injury recurrence. For all aims, we will use a mixed methods design using an exploratory, sequential approach to include the strengths of both qualitative and quantitative data. For aims 1 and 2, we will iteratively collect qualitative data from therapists, patients with SCI/D, and other stakeholders. For aim 3, we will perform a hybrid effectiveness-implementation randomized controlled trial to pilot-test the intervention. The projected results include an iteratively developed and tested implementation plan that meets moderate to high levels of acceptability, feasibility, and appropriateness. Additionally, the pilot trial results are expected to show positive trends in relevant clinical outcomes related to reduced pressure injury incidence, recurrence, and improved healing when compared with the standard of care. Currently, 6 participants have been recruited for our aim-1 qualitative study. This study will expand upon our previous study to move the Assisted Weight-Shift system into routine clinical care, which was a strong desire of adults with SCI/D for improved individualized care plans to prevent pressure injuries. The results of this study will guide the next steps in a full, hybrid effectiveness-implementation trial with the goal of improving care to prevent pressure injuries. DERR1-10.2196/51849.

Development and Implementation of an eHealth Oncohematonootric Program: Descriptive, Observational, Prospective Cohort Pilot Study.

In oncohematology, both the development of the disease and the side effects of antineoplastic treatment often take a toll on patients' physical and nutritional well-being. In this era of digital transformation, we launched a pioneering project for oncohematologic patients to promote adherence to a healthy lifestyle and improve their physical and nutritional well-being. We aim to achieve this goal by involving doctors and nutritionists through the Nootric app. This study aims to assess the impact of the use of eHealth tools to facilitate nutrition and well-being in oncohematologic patients. We also aim to determine the usefulness of physical-nutritional management in improving tolerance to chemotherapy treatments within routine clinical practice. We designed a descriptive, observational, longitudinal, prospective cohort pilot study that included a total of 22 patients from March to May 2022 in the Vinalopó University Hospital. The inclusion criteria were adults over 18 years of age diagnosed with oncohematological pathology in active chemotherapy treatment. An action plan was created to generate alerts between the doctor and the nutritionist. In the beginning, the patients were trained to use the app and received education highlighting the importance of nutrition and physical exercise. Sociodemographic, clinical-biological-analytical (eg, malnutrition index), health care impact, usability, and patient adherence data were collected. Tolerance to chemotherapy treatment and its health care impact were evaluated. We included 22 patients, 11 (50%) female and 11 (50%) male, ranging between 42 and 84 years of age. Among them, 13 (59%) were adherents to the program. The most frequent diseases were lymphoproliferative syndromes (13/22, 59%) and multiple myeloma (4/22, 18%). Moreover, 15 (68%) out of 22 patients received immunochemotherapy, while 7 (32%) out of 22 patients received biological treatment. No worsening of clinical-biological parameters was observed. Excluding dropouts and abandonments (n=9/22, 41%), the adherence rate was 81%, established by calculating the arithmetic mean of the adherence rates of 13 patients. No admission was observed due to gastrointestinal toxicity or discontinuation of treatment related to alterations in physical and nutritional well-being. In addition, only 5.5% of unscheduled consultations were increased due to incidents in well-being, mostly telematic (n=6/103 consultation are unscheduled). Additionally, 92% of patients reported an improvement in their nutritional habits (n=12/13), and up to 45% required adjustment of medical supportive treatment (n=5/11). There were no cases of grade 3 or greater gastrointestinal toxicity. All of this reflects improved tolerance to treatments. Patients reported a satisfaction score of 4.3 out of 5, while professionals rated their satisfaction at 4.8 out of 5. We demonstrated the usefulness of integrating new technologies through a multidisciplinary approach. The Nootric app facilitated collaboration among the medical team, nutritionists, and patients. It enabled us to detect health issues related to physical-nutritional well-being, anticipate major complications, and mitigate potentially avoidable risks. Consequently, there was a decrease in unscheduled visits and admissions related to this condition.

Anlotinib in Chinese patients aged ≥70 years with advanced non-squamous non-small cell lung cancer without prior chemotherapy: a multicenter, single-arm pilot trial.

Based on pharmacoeconomics, drug availability and actual treatment, optimal treatment regimens for Chinese non-small-cell lung carcinoma (NSCLC) patients over 70 years old are needed. This multicenter, single-arm pilot trial enrolled patients with advanced non-squamous NSCLC who refused systemic chemotherapy. Eligible patients received anlotinib (12 mg/day, d1-14, Q3W) until disease progression, intolerant toxicities, or withdrawal from the study. The primary endpoint was progression-free survival (PFS). Forty-nine patients were screened between January 2019 and September 2021, of whom 40 patients were eligible. The median age was 76 years. With a median follow-up period of 16.20 (95% CI: 8.77, 25.10) months, the median PFS was 5.45 months (95% CI: 3.52-9.23) and the median overall survival was 10.32 months (95% CI: 6.44-12.78). Three patients achieved a partial response and 34 had stable disease, with an objective response rate of 7.5% and a disease control rate of 92.5%. Thirty-three (82.5%; 33/40) patients reported treatment-related adverse events (TRAEs) of any grade, and the incidence rate of grade ≥3 TRAEs was 35% (14/40). The most common grade ≥3 TRAEs were hypertension (4/40; 10.0%), hand-foot syndrome (3/40; 7.5%), and proteinuria (2/40; 5.0%). Anlotinib treatment was feasible and safe in Chinese elderly patients with advanced non-squamous NSCLC who did not receive any systemic chemotherapy.

Preventing eating pathology among elite adolescent ballet dancers: a pilot trial of the Athlete Body Project

ABSTRACT Adolescent ballet dancers are commonly recognized as aesthetic athletes who are at higher risk for body dissatisfaction, disordered eating, and eating disorders. Although athletic participation can generally protect against negative mental and physical health outcomes, idealization of a body ideal specific to ballet can confer unique vulnerability for eating pathology. Despite this known vulnerability, rigorous efforts to prevent eating pathology among young dancers are lacking. Previously, we reported on a trial of the Female Athlete Body Project adapted for professional female ballet dancers. The current study tested a subsequent adaptation, the Athlete Body Project (ABP), tailored for younger dancers, including a version for boys. This non-randomized, uncontrolled pilot trial tested ABP among boys and girls in two elite ballet schools (N = 23), formatted in-person and via video-conferencing. Program delivery met benchmarks for feasibility and acceptability across gender and delivery formats, and dancers provided positive qualitative feedback. Overall results suggest the potential of ABP in intervening on pre-post eating pathology and body appreciation, and significantly decreasing drive for muscularity among young ballet dancers. Taken together, this pilot work addresses a gap in the field and advances the needed development of eating disorder prevention programming for young, high-risk aesthetic athletes.

Concordance of Blood Glucose and CGM During a Pilot Trial of Automated Insulin Delivery in Type 1 Diabetes Pregnancies.

Customized and standard automated insulin delivery (AID) systems for use in pregnancies of women with preexisting type 1 diabetes (T1D) are being developed and tested to achieve pregnancy appropriate continuous glucose monitoring (CGM) targets. Guidance on the use of CGM for treatment decisions during pregnancy in the United States is limited. Ten pregnant women with preexisting T1D participated in a trial evaluating at-home use of a pregnancy-specific AID system. Seven-point self-monitoring of blood glucose (SMBG) was compared to the closest sensor glucose (Dexcom G6 CGM) value biweekly to assess safety and reliability based on the 20%/20 mg/dL criteria. All participants completed the study with 7 participants satisfying the safety and reliability criteria with a mean absolute relative difference of 10.3%. Three participants did not fulfill the criteria, mainly because the frequency of SMBG did not meet the requirements. Dexcom G6 CGM is safe and accurate in the real-world setting for use in pregnant women with preexisting T1D with reduced SMBG testing as part of a pregnancy-specific AID system.

Abstract CT031: A pilot clinical trial of neoadjuvant modified FOLFIRINOX plus nivolumab in borderline resectable pancreas cancer

Abstract Background: Combinations of chemotherapy and checkpoint inhibitors have failed to show improvements in pancreas cancer (PC) but all studies reported to date have been in the metastatic setting. Perioperative strategies of PD1 inhibitor combinations have shown promise in many malignancies. The A021501 trial established modified FOLFIRINOX (mFFX) as a reference neoadjuvant regimen for patients (pts) with borderline resectable pancreas cancer (BRPC). Here, we present clinical and pathologic results from a phase 1/2 investigator-initiated study investigating the safety and efficacy of mFFX and Nivolumab in pts with BRPC. Methods: Pts with ECOG PS 0-1 and BRPC confirmed by qualified surgeons were treated with a maximum of 6 cycles of mFFX (oxaliplatin 85 mg/m2, irinotecan 150 mg/m2, leucovorin 400 mg/m2 and infusional 5-fluorouracil 2400 mg/m2 over 46 hours) on days 1 and 15 of a 28-day cycle and Nivolumab (480 mg Iv q 4 weeks). The primary endpoint was safety (including post-operative fistula formation) and pathologic response to neoadjuvant therapy. Secondary endpoints included progression-free survival (PFS), overall response rate (ORR) and overall survival (OS). RNA sequencing was performed on 16 post-treatment resected tumors, 5 patient-matched diagnostic pre-treatment biopsies, and 9 resected tumors from non-trial patients treated with mFFX alone. Results: Overall, 28 pts (median age 68, M: 16, F, 12) were enrolled. 26 (93%) pts completed at least 3 cycles of mFFX and Nivo and 24 (86%) pts underwent surgery (23 R0/1R1). There were no grade 2 post-operative fistulas observed and the rates of grade 3 or higher adverse events was 55% with all being related to mFFX and none attributed to nivolumab. At a median follow-up of 24 months, the median PFS was 34.8 months, and the median OS was 35.1 months. Among pts who underwent pancreatectomy, the 18-month OS rate was 90%. There were 2 pathologic complete responses and 2 near complete responses. Compared to pre-treatment biopsies, RNA sequencing from resected specimens revealed higher CD8 (P=0.018) and Granzyme A (P=0.024) expression. In patients with pathologically node negative disease (n=11), elevated Granzyme A expression was associated with significantly improved PFS (P=0.046). Adenosine-related gene expression increased in 50% (n=8) of post-treatment samples (P&amp;lt;0.022) and correlated with expression of adenosine-generating CD73 (P=0.008). Conclusions: Neoadjuvant mFOLFIRINOX and Nivolumab was associated with favorable rates of R0 resection, PFS and OS relative to historical data in patients with BRPC in this pilot trial. The addition of Nivolumab did not increase rates of G3 AE’s and there were no unexpected safety signals. The addition of Nivolumab to neoadjuvant FOLFIRINOX may benefit some patients by enhancing cytolytic T cell function and decreasing immunosuppressive adenosine. This is the first trial reported with a PD1 inhibitor in neoadjuvant pancreas cancer and a Phase II trial is ongoing. Citation Format: Zev A. Wainberg, Jason Link, David Dawson, Lee Rosen, Stephen Kim, Mark Girgis, Jon King, Joe Hines, Saeed Sadeghi, Olga Olevsky, Deborah J. Wong, Harsimran Multani, Jenna Davis, Lisa Yonemoto, Ann Marie Siney, Christine Kivork, Chi-Hong Tseng, Timothy Donahue. A pilot clinical trial of neoadjuvant modified FOLFIRINOX plus nivolumab in borderline resectable pancreas cancer [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 2 (Late-Breaking, Clinical Trial, and Invited Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(7_Suppl):Abstract nr CT031.

A Novel Intervention to Reduce Fear of Progression and Trauma Symptoms in Advanced Cancer Using Written Exposure to Worst-Case Scenarios.

Background: Adults with advanced cancer experience profound future uncertainty, reflected in elevated fear of cancer progression (FoP) and cancer-related trauma symptoms. These symptoms are associated with physical symptom burden and poorer quality of life, and few interventions exist to manage them. Objective: To develop and pilot a written exposure-based coping intervention (EASE) focused on worst-case scenarios among adults with advanced cancer reporting elevated cancer-related trauma symptoms or FoP. Design: A single-arm intervention development and pilot trial. Participants: The trial enrolled 29 U.S. adults with stage III or stage IV solid tumor cancer (n = 24) or incurable or higher-risk blood cancer (n = 5) reporting elevated cancer-related trauma symptoms or FoP. Among those screened, 74% were eligible, with an eligible-to-enrolled rate of 85%. Design/Measurements: EASE was delivered over five 1:1 videoconferencing sessions. Feasibility and acceptability were evaluated via attendance, surveys, and exit interviews. Outcomes were assessed at five time points through 3-month (FU1, main assessment of interest) and 4.5-month (FU2) follow-up. Results: Participant and interventionist feedback was used to iteratively refine EASE. Among participants, 86% (25/29) completed all five sessions and FU1; surveys and exit interviews indicated high acceptability. Primary outcomes of cancer-related trauma symptoms and FoP improved significantly from pre to both follow-ups by predominantly large effect sizes. Secondary outcomes of anxiety, depression, hopelessness, fear of death/dying, and fatigue, and most process measures improved significantly by FU1 or FU2. Conclusions: EASE, a novel adaptation of written exposure therapy, is a promising approach to reducing FoP and cancer-related trauma symptoms among adults with advanced cancer that warrants further study.

Abstract CT293: eCAN Project : The EU Joint Action on strengthening eHealth including telemedicine and telemonitoring for cancer prevention and care

Abstract The integration of telemedicine tools such as teleconsultation and telemonitoring in cancer patients care may allow to improve person-centered care and patients’ empowerment. The eCAN JA (https://ecanja.eu/) explores the role of telemedicine tools in clinical trials focusing on tele-rehabilitation and tele-psychological support in different populations of cancer patients in 10 European countries.The main aim is to assess the effect of teleconsultation and telemonitoring program focused on rehabilitation and psychological support for cancer patients on patient reported outcomes measures (PROMs) in three different pilot randomized controlled trials. MethodsThe pilot trials have been launched in September 2023 in 10 European countries with a multicentric, prospective, randomized, open label design in patients affected by Breast Cancer (BC) (Pilot 1a), Head and Neck (H&amp;N) (Pilot 1b) and advanced cancer (Pilot 2). According to the sample size calculation, the aim is to enroll a minimum of 354 patients, in 18 European cancer centers. Patients are randomly assigned either to the intervention or control group using a 1:1 ratioThe main objectives of the pilots are: 1-To assess the effect of teleconsultation program and telemonitoring focused on rehabilitation for patients with BC (1a) and H&amp;N (1b) cancer on the PROMs (quality of life (QoL) and pain) compared to usual care.2-To assess the effect of teleconsultation program and telemonitoring focused on psychological support for patients with advanced cancer (pilot 2) on the PROMs (QoL and distress thermometer) compared to usual care. Patients in the intervention group receive weekly teleconsultation during 8 weeks through the secure Edumeet platform. In pilot 1, tele-rehabilitation training is performed by a remote physiotherapist and consist of a series of revalidation exercises. In pilot 2, tele-psychological support is performed by a remote psychologist and consist of techniques for managing emotions and stress.PROMs data will be collected longitudinally during the intervention with dedicated digital application (eCAN app) and distant Telemonitoring with eCAN dashboard. Patients in the control group receive usual (in person) care. Results64/354 patients have been enrolled at present. An interim analysis is planned for the end of January 2024 in order to assess feasibility, patients compliance and dropout rate.. ConclusionThe integration of telemedicine tools such as teleconsultation and telemonitoring may allow to improve person-centered care and patients’ empowerment. The results of the eCAN project will improve our knowledge on benefits and risks for teleconsultation and telemonitoring in cancer patients care and will facilitate health care professionals’ skills in communication with patients and their families in the context of remote care and telemedicine. Co-funded by the European Union (Grant number: 101075326). Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or HaDEA. Neither the European Union nor the granting authority can be held responsible for them. Citation Format: Andrea Pace, Vittorio Castaldo, Marco G. Paggi, Efthyvoulos Kyriacou, Christina Plomariti, Magdalena Rosinska, Tugce Schmitt, Victoria Leclercq, eCAN WP5 research team. eCAN Project : The EU Joint Action on strengthening eHealth including telemedicine and telemonitoring for cancer prevention and care [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 2 (Late-Breaking, Clinical Trial, and Invited Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(7_Suppl):Abstract nr CT293.

Abstract CT232: Intense exercise for survival among men with metastatic prostate cancer: 12-month feasibility results from the INTERVAL-GAP4 trial pilot site at Edith Cowan University, Australia

Abstract Introduction: Men with prostate cancer undertaking moderate-to-vigorous exercise have a marked reduction of 30-60% in all-cause and cancer-related mortality based on observational studies. In 2016, the Intense Exercise for Survival among Men with Metastatic Prostate Cancer (INTERVAL-GAP4) was launched to determine if supervised exercise improves overall survival in men with metastatic prostate cancer. Here, we describe the demographic characteristics, completion rates, exercise adherence, and safety of the first 12 months of the intervention at the pilot site, Edith Cowan University in Perth, Australia. Methods: INTERVAL-GAP4 is a multi-center global randomized controlled phase III trial. Patients are randomly allocated (1:1) to: (intervention) a high intensity combined resistance and aerobic exercise supervised program for 1 year tapering to self-management in year 2; or (control) self-directed unsupervised exercise with print materials. Patients were stratified by site and by disease/treatment status (metastatic hormone-sensitive prostate cancer [mHSPC] or metastatic castration-resistant prostate cancer [mCRPC] and if the latter, treatment modality). The study did not meet recruitment goals and closed to further enrollment in February 2023. Results: 240 participants were evaluated between April 2016-Feb 2023, 60 patients were consented, 52 participants were randomized, and two patients did not receive the allocation, resulting in 50 participants (27 intervention and 23 control). Main reasons for exclusion were not meeting clinical criteria (N=81), time commitment (N=24), unable to contact (N=22), not interested (N=19), and poor physical function (N=13). Median age at randomization was 72 years (IQR: 68, 77), median body mass index (BMI) was 31.7 kg/m2 (IQR: 27.5, 35.7), 94% identified as white, and time since diagnosis was 5 years (IQR: 1, 9). 13 participants (26%) had mHSPC and 37 (74%) had mCRPC. Completion rates were the following at 6 months: 84% completed surveys, 62% exercise testing, and 66% biological samples. Of 42 alive at 12 months: 85% completed surveys, 73% testing, and 73% biological samples. Median exercise adherence was 90% (IQR: 78, 97) for the first 12 months of the study. Adherence was not significantly different for those with mCRPC vs. mHSPC status. There were 10 SAEs in first 12 months; all (100%) were unrelated to the intervention. Conclusions: Exercise training was feasible and safe in men with metastatic prostate cancer with no difference observed in exercise session adherence by disease status. Survey completion was high, while in-person completion rates were affected by the COVID-19 pandemic. Additional analysis of the entire study population (N=145) is ongoing and will be compared with the pilot site. Citation Format: Stacey A. Kenfield, Nicolas H. Hart, Jennette Sison, June M. Chan, Kerry S. Courneya, Fred Saad, Robert U. Newton. Intense exercise for survival among men with metastatic prostate cancer: 12-month feasibility results from the INTERVAL-GAP4 trial pilot site at Edith Cowan University, Australia [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 2 (Late-Breaking, Clinical Trial, and Invited Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(7_Suppl):Abstract nr CT232.

Novel Performance Rating Instruments for Gynecological Procedures in Primary Care: A Pilot Study.

Improving training and confirming the acquisition of gynecological procedure skills for family physicians (FPs) is crucial for safe healthcare delivery. The objectives of this study were to (a) develop performance rating instruments for four gynecological procedures, and (b) pilot them to provide preliminary validity evidence using modern validity theory. Sixteen academic FPs and gynecologists participated in a modified Delphi technique to develop procedure-specific checklists (PSCs) for four procedures: intrauterine device insertion, endometrial biopsy, punch biopsy of the vulva, and routine pessary care. We modified a previously validated global rating scale (GRS) for ambulatory settings. Using prerecorded videos, 19 academic FPs piloted instruments to rate one first-year and one second-year family medicine resident's performance. They were blinded to the level of training. We compared the mean scores for PSCs and GRS for each procedure using paired samples t tests and Cohen'sdto estimate effect sizes. Consensus on items for the final PSCs was reached after two Delphi rounds. PSC and GRS scores were numerically higher for the second-year resident than the first-year resident for every procedure, with statistically significant differences for six of eight comparisons (P&lt;.05). All comparisons demonstrated large effect sizes (Cohen'sd&gt;0.8). Both instruments received high scores for ease of use by raters. We developed novel performance rating instruments for four gynecological procedures and provided preliminary validity evidence for their use for formative feedback in a simulation setting. This pilot study suggests that these instruments may facilitate the training and documentation of family medicine trainees' skills in gynecological procedures.

A Randomized Controlled Pilot Trial of a Brief Dialectical Behavior Therapy Skills Training-Informed Intervention for Test Anxiety

ABSTRACT Data testing the effectiveness of third-wave interventions to decrease college students’ test anxiety is sparse. We conducted a randomized controlled trial with college students to examine whether an intervention using several DBT skills (DBT; Linehan, 2015a, 2015b) would decrease test anxiety. Participants (N = 48) were randomly assigned to one of two conditions, the brief DBT condition (n = 26) or the control condition (n = 22), a brain-training activity group. Participants in both conditions met for a single, two-hour session. After the intervention, participants completed an exam in their psychology course. Results indicated that individuals in the DBT intervention group had greater reductions in levels of test anxiety and state anxiety, and greater increases in mindfulness, than individuals in the control group. This study provides data supporting DBT skills training as a promising treatment for students with test anxiety.

Electronic health intervention to manage symptoms of immunotherapy in patients with cancer (SOFIA): Results from a randomized controlled pilot trial.

For patients receiving immune checkpoint inhibitors, early detection of immune-related adverse events (irAEs) is critical for one's safety. To this end, a smartphone app (SOFIA) was developed that featured the assessment of electronic patient-reported outcomes (ePROs) focusing on irAEs as well as a set of comprehensive supportive information. Its feasibility and preliminary efficacywere evaluated in a randomized controlled trial (RCT). Patients who received immune checkpoint inhibition therapy were randomly assigned to an intervention group (IG) or a control group (CG; care as usual). During the 12-week intervention period, IG patients used SOFIA to report twice weekly ePROs and receive cancer- and immunotherapy-relevant contents. Before a patient's next clinical visit, the physician in charge was given the ePRO reports. The primary objective was to test the feasibility of SOFIA. Furthermore, the preliminary efficacy of SOFIA for health-related quality of life (HRQOL), psychosocial outcomes, and medical data was examined. Clinical outcomes were assessed at baseline (T0), post-intervention (T1), and a 3-month follow-up (T2). Seventy-one patients were randomized to the IG (n=34) or the CG (n=37). SOFIA showed high feasibility and acceptance. At T1, patients in the IG reported significantly better HRQOL and role functioning and less depression, distress, and appetite loss. No significant differences were revealed regarding medical data, the utilization of supportive care services, or survival. SOFIA showed high feasibility and acceptance and improved HRQOL and psychosocial outcomes. These results suggest further evaluation of efficacy in a large-scale confirmatory multicenter RCT.

REporting quality of PilOt randomised controlled trials in surgery (REPORTS): a methodological survey protocol

IntroductionThe aim of this methodological review is to evaluate the completeness of reporting of surgical pilot and feasibility randomised trials as per the Consolidated Standards of Reporting Trials (CONSORT) extension...