Single-arm Pilot Study Research Articles

The Sublingual Microcirculation in Critically Ill Children with Septic Shock Undergoing Hemoadsorption: A Pilot Study.

Background: The importance of perfusion-guided resuscitation in septic shock has recently emerged. We explored whether the use of hemoadsorption led to a potential beneficial role in microvascular alterations in this clinical setting. Methods: A pre-planned secondary analysis of a Phase-II interventional single-arm pilot study (NCT05658588) was carried out, where 17 consecutive septic shock children admitted into PICU were treated with continuous renal replacement therapy (CRRT) and CytoSorb. Thirteen patients were eligible to be investigated with sublingual microcirculation at baseline, 24, 48, 72 and 96 h from the onset of blood purification. Patients achieving a microvascular flow index (MFI) ≥ 2.5 and/or proportion of perfused vessels (PPV) exceeding 90% by 96 h were defined as responders. Results: In 10/13 (77%), there was a significant improvement in MFIs (p = 0.01) and PPVs% (p = 0.04) between baseline and 24 h from the end of treatment. Eight patients displayed a high heterogenicity index (HI > 0.5) during blood purification and among these, five showed an improvement by the end of treatment (HI < 0.5). Conclusions: In this pilot study, we have found a potential association between CytoSorb hemoadsorption and a microcirculation improvement in pediatric patients with septic shock, particularly when this observation has been associated with hemodynamic improvement.

Feasibility and acceptability of a telehealth intervention for improving peer relationships for adolescents with neurofibromatosis type 1: a single-arm pilot study.

Elevated rates of social difficulties are evident for children and adolescents with neurofibromatosis type 1 (NF1) but the effects of social skills interventions have not been investigated for this population. The Program for the Education and Enrichment of Relational Skills (PEERS®), a widely established social skills intervention in autism spectrum disorders with expansion to other conditions, was recently modified to be offered virtually. This study examined the feasibility and acceptability of this telehealth intervention. 27 adolescents with NF1 with social skills difficulties and at least 1 caregiver enrolled in the study. 19 of those participants (Mage = 14.21 years, SD = 1.63; 7 females; 79% White) completed PEERS® via telehealth in a single-arm pilot study. Dropout rates, attendance records, helpfulness of the curriculum topics and caregiver-reported acceptability, including ratings on the Treatment Acceptability Questionnaire, were examined. Low study drop out (30% of enrolled participants; 14% of participants who began the intervention) and high attendance rates were observed. Caregivers found sessions related to common, everyday interactions most helpful. Adolescents indicated sessions related to having get-togethers and social nuances (e.g., humor) as most helpful. Caregiver ratings indicated acceptability of the intervention. This investigation supported the feasibility and acceptability of telehealth PEERS®, a social skills intervention program, among adolescents with NF1 and their caregivers based on attendance patterns as well as appraisal of the curriculum and telehealth modality.

An incentive-based text-messaging intervention to reduce maternal alcohol use during pregnancy and lactation in South Africa (MaRISA study): Findings from a single-arm pilot study.

South Africa has the highest rate of fetal alcohol spectrum disorders (FASD) globally. As with alcohol use during pregnancy, alcohol consumption while breastfeeding adversely impacts infant development. We pilot tested an incentive-based text-messaging intervention to reduce alcohol use during pregnancy and lactation in South Africa. A single-arm pilot trial was conducted over 3 months in healthcare facilities in Cape Town, South Africa. Pregnant and breastfeeding participants tested positive for recent alcohol use by urinalysis. The three-month intervention had two components, contingency management of alcohol abstinence confirmed by urinalysis twice weekly and weekly health-related text messaging from an evidence-based brief intervention. We collected twice weekly urine samples for measurement of ethyl glucuronide (EtG), an alcohol biomarker, and measures of self-reported alcohol and drug use, violence exposure, and mental health at six weeks and three months post-enrollment. Sixty participants were enrolled, of whom 31 were pregnant and 29 lactating. The number of days with four or more drinks in the past month decreased from 9 days at baseline, on average, to 1-3 days (p-value range: 0.144-0.010) at follow-up timepoints. There were statistically significant increases in the proportions of participants with alcohol-negative urine tests (p < 0.001). The percentages of participants breastfeeding while using alcohol decreased from baseline to the end of 3 months in the overall sample and among those enrolled postpartum, though these were not significant (p-value range: 0.255-0.147). Maternal depression scores also decreased among participants enrolled postpartum (p = 0.054). Emotional abuse by the main partner, but neither physical nor sexual abuse, significantly decreased at both follow-ups in the overall sample (p = 0.032) and among participants enrolled while pregnant (p = 0.015). This study is among the first to pilot test an incentive-based text-messaging intervention for maternal alcohol use and other outcomes such as depression and violence exposure. Further testing is warranted in a well-powered, randomized controlled trial.

Digital Phenotyping of Geriatric Depression Using a Community-Based Digital Mental Health Monitoring Platform for Socially Vulnerable Older Adults and Their Community Caregivers: 6-Week Living Lab Single-Arm Pilot Study.

Despite the increasing need for digital services to support geriatric mental health, the development and implementation of digital mental health care systems for older adults have been hindered by a lack of studies involving socially vulnerable older adult users and their caregivers in natural living environments. This study aims to determine whether digital sensing data on heart rate variability, sleep quality, and physical activity can predict same-day or next-day depressive symptoms among socially vulnerable older adults in their everyday living environments. In addition, this study tested the feasibility of a digital mental health monitoring platform designed to inform older adult users and their community caregivers about day-to-day changes in the health status of older adults. A single-arm, nonrandomized living lab pilot study was conducted with socially vulnerable older adults (n=25), their community caregivers (n=16), and a managerial social worker over a 6-week period during and after the COVID-19 pandemic. Depressive symptoms were assessed daily using the 9-item Patient Health Questionnaire via scripted verbal conversations with a mobile chatbot. Digital biomarkers for depression, including heart rate variability, sleep, and physical activity, were measured using a wearable sensor (Fitbit Sense) that was worn continuously, except during charging times. Daily individualized feedback, using traffic signal signs, on the health status of older adult users regarding stress, sleep, physical activity, and health emergency status was displayed on a mobile app for the users and on a web application for their community caregivers. Multilevel modeling was used to examine whether the digital biomarkers predicted same-day or next-day depressive symptoms. Study staff conducted pre- and postsurveys in person at the homes of older adult users to monitor changes in depressive symptoms, sleep quality, and system usability. Among the 31 older adult participants, 25 provided data for the living lab and 24 provided data for the pre-post test analysis. The multilevel modeling results showed that increases in daily sleep fragmentation (P=.003) and sleep efficiency (P=.001) compared with one's average were associated with an increased risk of daily depressive symptoms in older adults. The pre-post test results indicated improvements in depressive symptoms (P=.048) and sleep quality (P=.02), but not in the system usability (P=.18). The findings suggest that wearable sensors assessing sleep quality may be utilized to predict daily fluctuations in depressive symptoms among socially vulnerable older adults. The results also imply that receiving individualized health feedback and sharing it with community caregivers may help improve the mental health of older adults. However, additional in-person training may be necessary to enhance usability. ClinicalTrials.gov NCT06270121; https://clinicaltrials.gov/study/NCT06270121.

Feasibility and acceptability of an acceptance and commitment therapy for caregivers of dementia: a single arm trial emphasizing adult protection

PurposeCaregivers for older adults play a crucial role in protecting those diagnosed with dementia. These caregivers often experience heightened anxiety, depression and burdens associated with their responsibilities. Sufficient support and efficient coping mechanisms alleviate distress and caregiving burdens, thereby enhancing and protecting adults. Acceptance and commitment therapy (ACT) fosters acceptance of internal experiences and promotes behaviors aligned with personal values. This single-arm pilot study aims to evaluate the feasibility, acceptability and preliminary effects of an online ACT intervention (Online Acceptance and Commitment Therapy Intervention for Caregivers [OACTIC]) on well-being aspects among dementia caregivers, emphasizing the importance of tailored interventions to protect older adults and their caregivers.Design/methodology/approachThe caregiver must be ≥21 years of age (i.e. a parent, spouse, partner, sibling or adult child), or they must be the primary caregiver of older adults with dementia for ≥12 months and have scores that indicate moderate or higher on the scale of anxiety, depression and poor quality of life. Participants received an online-based ACT over six weekly 1-h sessions. The following outcome measures were administered at baseline (T1), immediately post-intervention (T2) and three-month follow-up (T3): Generalized Anxiety Disorder-7, Patient Health Questionnaire-9, Zarit Burden Interview, The Experience of Suffering Measure and Acceptance and Action Questionnaire-II.FindingsAll 45 participants completed the study, with 93.3% rating their OACTIC experience as “completely satisfied.”Originality/valueOACTIC (Trial Registration# AEARCTR-0012366) is a novel study conducted in Pakistan, offering a cost-effective and time-efficient treatment approach that enhances the psychological well-being and protects older adults and their caregivers.

Favorable liver and skeletal muscle changes in patients with MASLD and T2DM receiving glucagon-like peptide-1 receptor agonist: A prospective cohort study.

To investigate changes in skeletal muscle mass and fat fraction in patients with metabolic dysfunction-associated steatotic liver disease (MASLD) and type 2 diabetes mellitus (T2DM) undergoing treatment with Semaglutide for 6months. This single-arm pilot study included 21 patients with MASLD who received semaglutide for T2DM. Body weight, metabolic parameters, liver enzymes, fibrosis markers, skeletal muscle index (cm2/m2), and fat fraction (%) at the L3 level using the two-point Dixon method on magnetic resonance imaging (MRI), as well as liver steatosis and liver stiffness assessed using MRI-based proton density fat fraction (MRI-PDFF) and MR elastography, respectively, were prospectively examined before and 6 months after semaglutide administration. The mean age of the patients was 53 years and 47.6% were females. The median liver steatosis-fraction (%) and skeletal muscle steatosis-fraction values (%) significantly decreased (22.0 vs 12.0; P = .0014) and (12.8 vs 9.9; P = .0416) at baseline and 6 months, respectively, while maintaining muscle mass during treatment. Semaglutide also dramatically reduced hemoglobin A1c (%) (6.8 vs 5.8, P = .0003), AST (IU/L) (54 vs 26, P < .0001), ALT (IU/L) (80 vs 34, P = .0004), and γ-GTP (IU/L) levels (64 vs 34, P = .0007). Although not statistically significant, Body weight (kg) (79.9 vs 77.4), body mass index (BMI) (kg/m2) (28.9 vs 27.6), and liver stiffness (kPa) (28.9 vs 27.6) showed a decreasing trend. Fibrosis markers such as M2BPGi, type IV collagen, and skeletal muscle area did not differ. Semaglutide demonstrated favorable effects on liver and skeletal muscle steatosis, promoting improved liver function and diabetic status.

174P A single-arm open-label pilot study of combination therapy of TACE in combination with ablation and durvalumab in unresectable hepatocellular carcinoma patients (TAD study)

174P A single-arm open-label pilot study of combination therapy of TACE in combination with ablation and durvalumab in unresectable hepatocellular carcinoma patients (TAD study)

CheMo4METPANC: A randomized phase 2 study with combination chemotherapy (gemcitabine and nab-paclitaxel), chemokine (C-X-C) motif receptor 4 inhibitor (motixafortide), and immune checkpoint blockade (cemiplimab) compared to chemotherapy alone in metastatic treatment-naïve pancreatic adenocarcinoma.

TPS4208 Background: Metastatic pancreatic ductal adenocarcinoma (mPDAC) carries a 5-year relative survival of 3%. Immune checkpoint inhibitors have thus far failed to improve outcomes due to an immunosuppressive tumor microenvironment (TME). Signaling through the C-X-C motif chemokine receptor 4 (CXCR4)/C-X-C motif chemokine ligand 12 (CXCL12) axis results in exclusion of anti-tumor immune cells in mouse PDAC models. Longitudinal survival studies testing combinations of CXCR4i, aPD-1, and gemcitabine in the KPC mouse model of PDAC demonstrated a survival benefit with the combination of all three agents. Results from a multicenter, investigator-initiated, single arm pilot study evaluating the preliminary safety and efficacy ofMotixafortide (CXCR4i), Cemiplimab (aPD1), Gemcitabine, and Nab-paclitaxel (MCGN) in 11 patients with treatment-naïve mPDAC were recently presented. Seven patients (64%) achieved a partial response, including 6 confirmed partial responses, and 10 patients (91%) had disease control, compared to historic partial response and disease control rates of 23% and 48%, respectively, with gemcitabine and nab-paclitaxel (GN). Preliminary median progression-free survival (PFS) was 9.6 months compared to historic median PFS of 5.5 months with GN. Analysis of paired pre- and on-treatment biopsy samples from demonstrated an increase in CD8+ T-cell density in tumors from all 11 patients treated with MCGN (p=0.007). Based on these promising results, the protocol was amended from a single arm expansion totaling 40 patients to a randomized phase 2 study. Methods: This is an open-label, multicenter, investigator-initiated phase 2 clinical trial. Patients are randomized 2:1 to receive MCGN or GNalone (N=108). The primary endpoint is progression free survival. The study has 80% power to detect an improvement in PFS from 6 to 9.2 months (HR 0.65) with a one-sided alpha of 0.20. One interim analysis for futility is planned when 50% of the PFS events are observed. Secondary objectives include response rate, disease control rate, duration of clinical benefit and overall survival. Patients will undergo optional paired biopsies which will be analyzed in conjunction with samples collected from the phase I pilot study. Exploratory studies include interrogation of the TME using quantitative multiplex immunofluorescence and single-cell RNA sequencing. The randomized phase 2 trial is open to enrollment and one patient has been randomized as of 1/29/2024. Clinical trial information: NCT04543071 .

Music therapy group for women outpatients with major depressive disorder under treatment as usual in a psychiatric institution: non-randomized pilot trial

Major Depressive Disorder (MDD) is a prevalent mental illness in the world. Pharmacological treatment is typically the first choice, but Humanistic Music Therapy (HMT) may offer additional benefits when added to Treatment As Usual (TAU). This single-arm pilot study aimed to assess the effectiveness of Group HMT as an intervention for adult women diagnosed with MDD, in a psychiatric institution. The study comprised 12 outpatient women with MDD who received more than one year of pharmacological treatment. A pre-test/post-test design using the Hamilton Depression (HAM-D-21), Personal and Social Performance (PSP), and Global Assessment of Functioning (GAF) scales were employed to evaluate symptom severity and patient changes. Results indicated a significant decrease in HAM-D scores from 15.7 to 6.6 (p ≤ .005), a notable improvement in personal and social functioning (PSP scale) (p ≤ .05), and an increase in global activity level (GAF scale) (p ≤ .05). In conclusion, adult women with MDD receiving TAU in a psychiatric institution experienced a significant reduction in depressive symptoms and enhancement in global activity, personal, and social functioning when HMT was added to their treatment regimen. This study is limited by being non-randomized, unblinded, and, with a small population. A randomized controlled trials are required to confirm the benefits of HMT.

Evaluating the feasibility of a Mobile Audio Companion (Elly) to reduce anxiety and to improve patient-reported outcomes among patients with cancer.

11120 Background: Anxiety is one of the most commonly reported symptoms among cancer patients, and is a known risk factor for reduced health-related quality of life and other negative patient-reported outcomes (PROs). Mobile health applications (apps) are tools that can be used flexibly in patients’ preferred environments with the potential to reduce anxiety and improve quality of life. The current study sought to examine the feasibility and preliminary efficacy of a mobile audio companion app, Elly, to improve anxiety and other critical PROs among cancer patients. Methods: This was a single-site, single-arm pilot study conducted over 6-months. Adults 18 years or older with a diagnosis of cancer of any type, received treatment within 6 months of consent, and had access to an iPhone were eligible. The primary outcome was change in mean anxiety t-scores (NIH PROMIS anxiety short form (8a)) administered electronically via REDCap at baseline, 1 (primary timepoint), 3, and 6 months. Secondary outcomes included changes in NIH PROMIS depression, loneliness, social support, and perceived stress scores from baseline to 6 months. Changes in NIH PROMIS T-scores at each time point were calculated and analyzed using paired t-tests. Results: Of 53 patients who consented for the study, 38 patients (72%) were included in the analysis ( Mage: 52.8 years, SD: 11.7, 84.2% female). Most participants reported a history of breast cancer (50%), followed by ovarian (16%), hematologic (11%), and other cancer types (24%) Phone incompatibility and technological challenges were the most common reasons for screen-fails (n=15). All participants downloaded and used the app at least once, with an average of 154 app usage events across participants recorded over the study period (median: 64, range 11-1186 times). The mean baseline anxiety T-score was 63.5 (SD: 2.1). Anxiety levels decreased among participants over time, with a mean difference in anxiety T-scores of -1.9 at 30 days, -3.3 at 90 days, and -3.0 at 6 months from baseline, which was statistically (p&lt;0.05) and clinically significant. Participants also reported improvements in perceived stress, depression symptomatology, social support, and loneliness, although statistical significance was not reached. Conclusions: Mobile apps, such as Elly, may feasibly be used to support patient-reported anxiety as well as other PROs, especially among those who present with high levels of anxiety at baseline. Efforts to increase access to mobile health apps and provide technical support for patients should be made increase equity across the cancer survivor population. Larger, multi-center randomized controlled trials are needed to build on these findings.

Continuous glucose monitoring and rates of hyperglycemia during chemotherapy for early-stage breast cancer.

e24134 Background: Diabetes (DM) and hyperglycemia (HG) during chemotherapy increase the risk of treatment-related toxicities. The prevalence of HG and daily patterns of HG over the course of chemotherapy in patients with early-stage breast cancer (ESBC) are currently unknown. Methods: We conducted a prospective single-arm pilot study of continuous glucose monitoring in patients with ESBC receiving chemotherapy from 12/2020 – 2/2022. Eligibility criteria included: diagnosis of Stage I-III BC, age ≥18, and planned chemotherapy with concurrent corticosteroid supportive care use. Patients were excluded if they were receiving other systemic corticosteroids or insulin. Patients wore FreeStyle Libre Pro continuous glucose monitoring sensors from the time of chemotherapy initiation (± 7 days) through completion. The sensors detected interstitial glucose levels every 15 minutes and were reapplied by the study team every 2-3 weeks. Primary endpoints were: (1) prevalence of HG, defined as number of patients with ≥1 glucose reading ≥140 mg/dL and (2) among those who developed HG, the proportion of time spent hyperglycemic, defined as number of readings ≥140 mg/dL divided by total number of readings. Key secondary endpoints included prevalence of prediabetes (pre-DM) and diabetes (DM) at baseline and changes in A1c from baseline to weeks 12 and 24. The analysis was stratified by baseline A1c (euglycemia [A1c &lt; 5.7%], pre-DM [A1c 5.7%-6.4%], or DM [A1c ≥6.5% and clinical diagnosis of DM prior to study enrollment]). Results: Among 20 evaluable patients, the median age at baseline was 59 (range 34-78) and median body mass index (BMI) was 29.5 kg/m2 (range 19.5-41.6). The most common chemotherapy regimens included docetaxel/cyclophosphamide (25%), weekly paclitaxel ± trastuzumab (20%), paclitaxel followed by doxorubicin/cyclophosphamide (15%), docetaxel/carboplatin/trastuzumab/pertuzumab (15%), and cyclophosphamide/methotrexate/fluorouracil (15%). All 20 patients developed HG. Of 124,165 total readings, 17% were ≥140 mg/dL and mean glucose was 112.7 mg/dL. At baseline, n = 10 were euglycemic, n = 7 had pre-DM, and n = 3 had DM. By cohort, the proportion of time spent hyperglycemic was 3.9% (euglycemia group), 10% (pre-DM group), and 73.3% (DM group) (p &lt; .0001). Mean glucose values were 95.5 mg/dL (euglycemia group), 104.5 mg/dL (pre-DM group), and 183.0 mg/dL (DM group) (p &lt; .0001). Change in mean A1c from baseline to weeks 12 and 24 varied significantly across groups. Conclusions: Among patients receiving chemotherapy for ESBC, we found evidence of HG in all patients, and the proportion of time spent hyperglycemic during chemotherapy varied significantly by baseline A1c. Further interventions should evaluate the benefits of improved glucose control among patients with baseline A1c &gt; 5.7%. Clinical trial information: NCT04473378 .

Effect of a 12-week remote exercising program (ExIOnc) on quality of life and symptoms of older patients with cancer in Brazil.

1623 Background: Leading organizations, including ASCO, recommend physical activity for patients with cancer. However, numerous barriers hinder patients from maintaining regular physical activity, particularly among older adults, who have lower participation in exercise programs. This study aims to evaluate the effects of a 12-week supervised remote exercise program on the health-related quality of life (HRQOL) of older patients with cancer (aged ≥65) undergoing treatment in Brazil. Methods: This was a single-arm pilot study evaluating a remote physical activity program which combined supervised and remote exercise. Eligible patients were contacted through WhatsApp for the initial assessment and received guidance on weekly exercises. The Vedius platform was employed to demonstrate proper home exercise techniques. The prescribed regimen consisted of 3-5 hours per week (4-6 days a week), encompassing both resistance and aerobic exercise. Outcome measures included overall HRQOL (FACT-G, scale 0-108) and symptom assessment (ESAS). Assessments were conducted at baseline and after 12 weeks. Descriptive statistics were calculated, and linear mixed models were employed to analyze changes from before to after the intervention. Results: Out of 46 approached patients, 41 patients were enrolled, with a median age of 70 years (range 65-88). Of the participants, 56.1% were female, 70.7% were white, and 58.2% were married, with 53.5% holding a college degree. The most common cancer types were breast (26.8%), genitourinary (22.0%), and lung (17.1%), all diagnosed at advanced stages. A significant improvement was observed in overall HRQOL (MT1=83.2 to MT2=94.4, P=0.001). Improvements were also seen in the severity of various symptoms, including pain (MT1=1.6 to MT2=0.7, P=0.001), fatigue (MT1=3.8 to MT2=1.3, P=0.001), nausea (MT1=0.8 to MT2=0.2, P=0.01), depression (MT1=2.6 to MT2=0.8, P=0.001), anxiety (MT1=3.2 to MT2=1.2, P=0.001), drowsiness (MT1=1.8 to MT2=0.4, P=0.001), and appetite (MT1=2.2 to MT2=0.6, P=0.001). Conclusions: This study demonstrates a positive impact of a 12-week remote physical activity program on HRQOL and symptom management for older patients with cancer, and set the stage for future randomized trials. Despite the challenges posed by cancer treatment, incorporating such programs into patient care could be an effective strategy to enhance overall well-being and mitigate treatment-related symptoms.

Evaluating a New Digital App-Based Program for Heart Health: Feasibility and Acceptability Pilot Study.

Cardiovascular disease (CVD) is the leading cause of death in the United States, affecting a significant proportion of adults. Digital health lifestyle change programs have emerged as a promising method of CVD prevention, offering benefits such as on-demand support, lower cost, and increased scalability. Prior research has shown the effectiveness of digital health interventions in reducing negative CVD outcomes. This pilot study focuses on the Lark Heart Health program, a fully digital artificial intelligence (AI)-powered smartphone app, providing synchronous CVD risk counseling, educational content, and personalized coaching. This pilot study evaluated the feasibility and acceptability of a fully digital AI-powered lifestyle change program called Lark Heart Health. Primary analyses assessed (1) participant satisfaction, (2) engagement with the program, and (3) the submission of health screeners. Secondary analyses were conducted to evaluate weight loss outcomes, given that a major focus of the Heart Health program is weight management. This study enrolled 509 participants in the 90-day real-world single-arm pilot study of the Heart Health app. Participants engaged with the app by participating in coaching conversations, logging meals, tracking weight, and completing educational lessons. The study outcomes included participant satisfaction, app engagement, the completion of screeners, and weight loss. On average, Heart Health study participants were aged 60.9 (SD 10.3; range 40-75) years, with average BMI indicating class I obesity. Of the 509 participants, 489 (96.1%) stayed enrolled until the end of the study (dropout rate: 3.9%). Study retention, based on providing a weight measurement during month 3, was 80% (407/509; 95% CI 76.2%-83.4%). Participant satisfaction scores indicated high satisfaction with the overall app experience, with an average score of ≥4 out of 5 for all satisfaction indicators. Participants also showed high engagement with the app, with 83.4% (408/489; 95% CI 80.1%-86.7%) of the sample engaging in ≥5 coaching conversations in month 3. The results indicated that participants were successfully able to submit health screeners within the app, with 90% (440/489; 95% CI 87%-92.5%) submitting all 3 screeners measured in the study. Finally, secondary analyses showed that participants lost weight during the program, with analyses showing an average weight nadir of 3.8% (SD 2.9%; 95% CI 3.5%-4.1%). The study results indicate that participants in this study were satisfied with their experience using the Heart Health app, highly engaged with the app features, and willing and able to complete health screening surveys in the app. These acceptability and feasibility results provide a key first step in the process of evidence generation for a new AI-powered digital program for heart health. Future work can expand these results to test outcomes with a commercial version of the Heart Health app in a diverse real-world sample.

A Tailored Intervention for Improving Diabetes Self-care Among Adults With Visual Impairment: A Pilot Study

ObjectivesOur aim in this study was to develop and evaluate a tailored intervention for improving diabetes self-care among people with visual impairment (TID-VI) in South Korea. MethodsThe TID-VI program was designed around the barriers, resources, and perceptual factors to promote diabetes self-care in those with visual impairment (VI). A single-arm pilot study was conducted to evaluate the feasibility and preliminary effects of the intervention. Diabetes self-efficacy, self-care behaviours, depression, health-related quality of life, and clinical outcomes (fasting blood glucose, glycated hemoglobin [A1C], lipids, and blood pressure) were measured before and up to 2 months after the 12-week intervention. ResultsAll 14 participants completed TID-VI. Diabetes self-efficacy, diabetes self-care behaviours, depression, and health-related quality of life showed improvement from baseline that was sustained at 2 months. Although high- and low-density lipoprotein also improved, there were no differences in blood glucose, A1C, total cholesterol, or blood pressure at 2 months. ConclusionsA theory-driven, tailored intervention specific to the needs of adults with VI can produce substantial improvements in patient-reported quality of life and health status outcomes, although the benefits are yet to be confirmed in a controlled study.

#2264 Efficacy and safety of oral iron supplementation with liposomal iron in non-dialysis chronic kidney disease patients with iron deficiency

Abstract Background and Aims Iron deficiency (ID) is very common in patients with non-dialysis dependent chronic kidney disease (NDD-CKD). In these patients, oral iron supplementation is recommended as first-line treatment for ID. However, the efficacy of oral iron supplementation is frequently impaired by a higher incidence of adverse gastrointestinal side effects. Liposomal iron is a compound with ferric pyrophosphate enclosed in a phospholipid nucleus, providing high gastrointestinal absorption and bioavailability and a lower incidence of side effects. However, few data are available on its efficacy in patients with NDD-CKD. Method This single-arm pilot study was carried out in in patients with NDD-CKD stages 1-5 and ID, defined as serum ferritin (SF)&amp;lt;100 ng/mL and/or transferrin saturation (TSAT)&amp;lt; 20%, consecutively seen in two Nephrology units (Naples and Bari) from 01/12/2022 to 01/04/2023. We assessed the efficacy of six months of liposomal iron therapy in correcting ID across three visits after baseline (month 1, 3 and 6). All patients received liposomal iron (30 mg/day twice daily for the first month followed by 30 mg daily for the remaining five months). The primary endpoints were the achievement of SF ≥100 ng/mL and TSAT ≥20%. Secondary outcomes were hemoglobin (Hb) changes and safety of liposomal iron; adherence to the iron supplementation was assessed by monthly pill counting. Changes of continuous variables during the study were analyzed by linear mixed models assuming an unstructured covariance matrix; this was done to take into account the correlation between repeated measures and missing points for patients not completing the study. Change of prevalence of corrected SF and TSAT values from baseline to month 1, 3 and 6 was analyzed by McNemar test. Results 38 patients (65.3 ± 15.2 years, 52.6% males, 35.1% diabetes, eGFR 38.5 ± 28.8 mL/min/1.73 m2) received at least one dose of liposomal iron (safety population). Efficacy population included 34 patients with at least one control visit after baseline. Clinical characteristics of patients enrolled in the two clinics did not differ. In efficacy population, baseline SF was 44 (IQR 19-82) ng/mL and TSAT 14.3 ± 5.9%, corresponding to a percentage of SF&amp;lt;100 ng/mL of 78.9%, and TSAT&amp;lt;20% in 89.5%; 64.7% had both parameters below the cut-offs. Anemia (defined as Hb&amp;lt;12 g/dL in women and &amp;lt;13 g/dL in men) was detected in 22/34 patients. Liposomal iron induced a progressive increase of TSAT from 14.3 ± 5.9%, at baseline to 20.0 ± 7.6% at month-6 (P = 0.009) whereas no difference was detected for SF (Fig. 1A and 1C). Accordingly, at the end of study, the prevalence of TSAT&amp;gt;20% significantly increased from 11.8% to 50.0% (P = 0.002) while correction of SF, mildly improved (from 23.5% to 30.0%, P = 0.214, Fig. 1B and 1D). When patients were stratified by anemia, a significant improvement of TSAT was observed in anemic patients (from 13.3 ± 5.8% to 20.2 ± 8.1%, P = 0.012), whereas no significant improvement was found in patients without anemia (P = 0.093). Adherence to iron supplementation was 100% in patients completing the study. Hb values did not significantly increase at month-6 in either anemic (+0.60 ± 1.39 g/dL, P = 0.258) or non-anemic (+0.08 ± 0.78 g/dL, P = 1.0) patients. In safety population (n = 38), 7 patients dropped out (18.4%). In particular, treatment was discontinued in 4 patients in the first month (2 for diarrhea, 1 for dyspepsia and constipation, and 1 for death), 1 patient after 3 months (switch to intravenous iron for symptomatic anemia), and 2 after five months (1 for dyspepsia and 1 for death). Conclusion The use of liposomal iron in patients with NDD-CKD is associated with a partial correction of functional ID (TSAT&amp;lt;20%), with no significant effect on iron storage. The small sample size prevents any causative conclusion, though it may provide some insights into using these compounds in patients with CKD.

Multimodal digital health treatments for Chronic Migraine associated with Medication Overuse Headache: a literature appraisal and results of a single-arm open trial (the BE-HOME program).

Mindfulness-based treatments gained popularity for migraine treatment. In this manuscript we report the results of a single-arm open pilot study that evaluated the impact of a multimodal web-based intervention combining home-based medication withdrawal, patients' education, and online mindfulness-based interventions. We aimed to address whether our program had the ability to show a change in the observed parameters and the study should therefore be intended as an early phase trial. Consecutive patients with chronic migraine associated with medication overuse headache were enrolled, followed-up for 12months, in a program that included home-based medication withdrawal, education on the correct use of drugs and lifestyle issues, prescription of tailored pharmacological prophylaxis, and attendance to six online mindfulness-based sessions. We tested the effect of the program on improving headache frequency, medication intake, quality of life (QoL), headache impact, depression, self-efficacy, and pain catastrophizing. A total of 37 patients completed the study (10 dropped out). We observed a large improvement in headache frequency, medication intake, headache impact, and QoL, a moderate improvement in pain catastrophizing and a mild improvement in depression symptoms; 70% to 76% of patients achieved 50% or more reduction in headache frequency from baseline to each follow-up (p < .01). The results of our multimodal program showed significant improvements in headache frequency, medication intake, and patient-reported outcomes. Future studies are needed to better identify patients who might benefit most from Digital Health Interventions and to demonstrate at least an equivalence in outcome with in-person programs carried out in hospital settings.

Interactive Malaysian Childhood Healthy Lifestyle (i-MaCHeL) programme: a single-arm pilot study

BackgroundThe growing obesity epidemic in Malaysia presents a public health challenge that requires innovative intervention strategies. In an effort to address this problem, an Interactive Malaysian Childhood Healthy Lifestyle (i-MaCHeL) programme, which is a web-based initiative designed for preschool child–parent dyads offers a novel approach. Nevertheless, the success of such a web-based intervention depends on several interrelated factors. This research aims to examine the feasibility of i-MaCHeL in the Malaysian context, its usability for preschool child–parent dyads, and the acceptability of the programme among these user groups.MethodsThis was a single-arm pilot study involving 46 child–parent dyads recruited from six government preschools in Terengganu, Malaysia. The preschools were selected using a cluster random sampling technique at the preschool level. The intervention feasibility was determined based on the retention rate of participants in the pilot study. The System Usability Scale (SUS) and intervention process evaluation were used to assess the usability and acceptability of the web-based i-MaCHeL programme.ResultsThe retention data demonstrated that 42 out of 46 participants completed the 13-week intervention programme, which showed that the overall retention rate was 91.3%. A mean (SD) SUS score of 84.70 (13.82) was obtained from parents, indicating that the web-based i-MaCHeL had an acceptable usability level. The mean scores of the process evaluation items ranged from 4.52 (0.63) to 4.83 (0.38), demonstrating that the web-based i-MaCHeL was highly accepted by the parents. The acceptability data also indicated that at least 92.9% (39/42) of the parents agreed/strongly agreed that the web content, programme duration, intervention dose, WhatsApp group, and delivery mode were appropriate.ConclusionsAccording to these findings, the i-MaCHeL intervention using a web-based approach was feasible, usable, and acceptable as part of a weight-related behaviour change intervention for preschool child–parent dyads. This pilot study demonstrated that the web-based i-MaCHeL was feasible and promising for delivering weight-related behavioural intervention to child–parent dyads.Trial registrationClinicalTrials.gov, NCT04711525. Registered on January 15, 2021.

Abstract PO5-12-09: A Single-Arm Pilot Study of the Feasibility and Efficacy of Electro-Acupuncture in Subjects with Chemotherapy-Induced Peripheral Neuropathy

Abstract Background: Chemotherapy-induced peripheral neuropathy (CIPN) is a very common dose-limiting side effect of many cancer treatments. However, the optimal treatment for CIPN remains unclear. Electroacupuncture (EA) is a non-pharmacologic treatment that combines traditional acupuncture with electrical stimulation. EA is being examined for CIPN and has shown modest benefits. Design: This is a pilot, single-center, prospective, single-arm, non-blinded study. All subjects had residual grade ≥2 CIPN after having received curative intent chemotherapy for stage I-III breast cancer, completed at least 3 months prior to study enrollment. Patients received 10 sessions of electro-acupuncture, administered by a licensed professional, over the course of 7 weeks. A sub-group of patients also had baseline and post-treatment skin punch biopsies to assess intra-epithelial nerve density (IEND). The primary objective was to determine the feasibility of completing a 10-treatment EA program in this patient population. Feasibility was defined as ≥15 subjects completing ≥8 EA treatments. Secondary endpoints included neuropathic pain, as assessed by the Brief Pain Inventory-Short Form (BPI-SF), and change in the quality of life, as assessed by the Functional Assessment of Cancer Therapy/Gynecologic Oncology Group-Neurotoxicity (FACT/GOG-NTX) subscale. Results: Twenty eligible female subjects were included in the study, with a median age of 66.5 years (range 45-81 years old). All patients received taxane chemotherapy, with 15 (75%) patients receiving weekly paclitaxel, 4 (8%) patients receiving Taxotere every 3 weeks, and 1 (2%) patient received weekly abraxane. The duration between chemotherapy completion and EA treatment initiation ranged between 4-92 months, with a median of 29 months. Eight (40%) patients were taking gabapentin, 2 (10%) on duloxetine, 1 (5%) pregabalin, and 3 (15%) on NSAIDS before and during the study. The study met its primary endpoint of feasibility, with 18 of 20 (90%) patients completing 8 or more EA sessions. Pain level, as assessed by the worst pain score on BPI-SF, improved significantly from a mean of 6.3 to 3.7, 2 weeks after completion of EA treatments (p = .0058). FACT/GOG-NTX quality of life measurement, “I am bothered by side effects of my treatment” also significantly improved from a mean score of 2.1 to 0.8 (p = .0177). There were no major adverse events (AEs) related to EA. Nine mild AEs were noted among 6 (30%) patients; localized skin biopsy site infections: G1=3, G2=3, nonlocalized cellulitis G3=1, Fatigue G1-2 =2. The localized skin biopsy site infections led to the discontinuation of further skin biopsy procedures. [We will also present data on the IEND for the nine patients who have paired before and after skin punch biopsies – this will be available at the time of the conference.] Conclusion: Electroacupuncture is a feasible treatment for CIPN. Furthermore, this pilot study did show a benefit for perceived pain and quality of life. Further studies will need to be conducted in regards to longevity of response. As larger studies in the future confirm the benefit of acupuncture, insurance payors are more likely to cover this important service. Citation Format: Nikitha Vobugari, John Paul Liang, Loni Savage, Dharamvir Jain, Kai Sun, Hanh Mai, Jenny Chang, Monica Desai, Tejal Patel, Polly Niravath. A Single-Arm Pilot Study of the Feasibility and Efficacy of Electro-Acupuncture in Subjects with Chemotherapy-Induced Peripheral Neuropathy [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO5-12-09.

Abstract PO1-20-01: A Prospective Single-Arm Pilot Interventional Trial to Assess Individual Breast Cancer Risk Prior to Gender-Affirming Chest Masculinization Surgery

Abstract Background: The number of individuals who identify as transgender and/or gender-diverse (TGD) is steadily increasing and the number of individuals seeking chest masculinization surgery, commonly called “top-surgery,” is also increasing. In persons assigned female at birth, top surgery removes most breast tissue to allow the chest to appear masculine. However, top surgery is not equivalent to oncologic risk-reducing mastectomies (RRM), which aim to remove all breast tissue to reduce future breast cancer (BC) risk. While top surgery has significant psychological and health benefits, its impact on future BC risk is unclear, especially for those individuals who are at high-risk for future BC development (defined as ≥17% by international consensus guidelines) and/or have a pathogenic germline mutation for BC. The aims of this study are to 1) determine the percentage of TGD persons assigned female at birth who are considering undergoing top surgery and are at elevated BC risk using validated risk models, 2) determine the percentage of TGD persons at elevated BC risk who choose to undergo RRM as part of top surgery, and 3) assess and compare self-perceived BC risk compared to calculated risk. Methods: A single-institution prospective single-arm pilot study is currently underway from March 2023 through January 2024. Individuals assigned female at birth, who are age ≥18 years, and considering undergoing top surgery, but have not yet undergone top surgery, are eligible to enroll (goal accrual N=35). Participants are being actively recruited form our institution’s Comprehensive LGBTQIA+ Inclusion Health Clinic and Plastic Surgery Clinic. Consented participants undergo a personalized BC risk assessment the validated IBIS and Gail (for those ≥35 years old) BC risk models. Those with an average lifetime BC risk (&amp;lt; 17%) are recommended to continue top surgery as planned/no intervention, participants with a moderate lifetime BC risk (17-30%) are counseled they may consider RRM as part of their top surgery operation given that sparse data exists in this space, while those with a high lifetime BC risk ( &amp;gt;30% or pathogenic germline variant) are counseled on the potential benefits of RRM. Participants with a family history suggestive of a hereditary cancer syndrome meet with a certified Genetics counselor for consideration of genetic testing, per NCCN guidelines. Results: We anticipate reporting 1) general demographic description of the cohort, 2) the percentage who have an elevated lifetime BC risk and/or a pathogenic genetic mutation, 3) the percentage of those with an elevated risk who choose to undergo RRM as part of top surgery, and 4) report the differences between self-perceived vs calculated lifetime BC risk. Conclusion: Our findings will inform TGD persons and surgeons on the utility of a personalized BC risk assessment prior to top surgery to assist in surgical decision-making, report the accuracy of self-perceived BC risk in TGD persons, and potentially change the standard pre-operative evaluation for TGD persons considering top surgery to include a formal BC risk assessment to ensure high-risk individuals have the option to undergo RRM to appropriately reduce their future BC risk. Citation Format: Chandler Cortina, Anna Purdy, Samantha Stachowiak, Kristen Klement, Sarah Sasor, Kate Krucoff, Andrew Petroll, Kevin Robertson, Annie Lakatos, Erin Doren. A Prospective Single-Arm Pilot Interventional Trial to Assess Individual Breast Cancer Risk Prior to Gender-Affirming Chest Masculinization Surgery [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO1-20-01.

Abstract PO4-22-12: Robot-assisted nipple-sparing mastectomy using da Vinci SP single-port system: primary results from the pilot trial RASPIM-01

Abstract Background Robot-assisted breast surgery was developed in 2014 for nipple-sparing mastectomy through a concealable lateral chest wall incision smaller. Although successful experiences have been reported using a multi-arm surgical robotic system, collisions between robotic arms and instruments often result in the discontinuation of console workflow and limit the performance of the surgical robot. Compared to multi-armed robot systems, the SP system is single-armed, equipped with flexible instruments containing multiple joints that may avoid instrument collision, a controllable camera reducing visual dead spots, and a designated non-third-party port that allows single-port entry and full deployment of the instrument. The RASPIM-01 trial (NCT05448963) is a single-armed pilot study assessing the feasibility of robot-assisted nipple-sparing mastectomy (NSM) using a new SP system. Method This study is a pilot trial conducted in a single-arm, non-randomized design with a recruitment of 30 participants. The SP surgical robot is used in NSM through the lateral chest wall incisions. The major inclusion criteria are 1) Breast cancer with preoperative clinical tumor sizes less than 5 cm, with an adequate tumor-skin distance of at least 3mm, and without nipple-areolar involvement in at least 1cm around the nipple by image, 2) Breast cancer up to clinical stage IIIA (T3, N1-2) showing adequate response to neoadjuvant therapy and meeting criteria 1), 3) Germline pathogenic/likely pathogenic BRCA1 or 2 variant carriers with or without a breast cancer diagnosis, requiring unilateral or bilateral therapeutic mastectomy or prophylactic mastectomy. Exclusion criteria include extensive breast skin or nipple involvement such as inflammatory breast cancer, Paget’s disease, nipple discharge associated with malignancy, image suggestive of cancer involvement of the nipple and subareolar tissue, and stage III breast cancer without response to neoadjuvant therapy, and previous radiotherapy of the surgical side breast. The endpoint measurements include Primary endpoint: The ability to complete nipple-sparing mastectomy with an SP system in the per-protocol population without additional assistant port or conversion to open surgery. Statistical analysis includes point estimation with a 95% confidence interval to analyze the mean or proportion of key performance parameters. No interim analysis will be performed due to the limited number intended to recruit. Results The study recruited 30 breast cancer patients and conducted 30 NSMs, while no prophylactic mastectomy case was recruited. The median age was 44 years old, BMI was 22.4 (18.9-30.0) kg/m2. Median tumor size was 3.0 (1.0-14.6) cm, and axillary lymph node metastases were present in 7 cases (23.3%). T2 (13 cases, 43.3%) tumors were the majority, followed by Tis (10 cases, 33.3%), T1 (5 cases, 16.7%), and T3 (2 cases, 6.7%). The anatomical stages of invasive cancer were Ia (16.7%), IIa (26.7%), IIb (16.7%), and IIIa (6.7%), among them 9 (30.3%) received neoadjuvant chemotherapy before surgery. All NSMs met the primary endpoint of successful completion using the SP robot system without conversion to open mastectomy or the addition of an assistant port. Median console time was 108.5 (73-285) min and docking time was 2.5 (1-9) min. Specimen weight was 311.6 (107-838) gm, while 2 (6.7%) cases had their nipple excised due to cancer-involved margin reported from the frozen section. Twenty-six (86.6%) cases received autologous free flap reconstruction, among which 25 (83.3%) cases received DIEP, and 1 (3.3%) received PAP reconstruction. Four cases received implant (2 cases, 6.7%) or tissue expander (3 cases, 6.7%). Conclusion This is the first NSM clinical trial operated with SP surgical robot system and met its primary endpoint showing the feasibility to conduct NSM. Secondary and exploratory endpoints data will be reported after the completion of clinical follow-ups. Citation Format: Wen-Ling Kuo, Chia-Huei Chu, Jung-ju Huang. Robot-assisted nipple-sparing mastectomy using da Vinci SP single-port system: primary results from the pilot trial RASPIM-01 [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO4-22-12.