Multicenter Pilot Study Research Articles

Exploring the Potential of Electronic Patient-Reported Outcome Measures to Inform and Assess Care in Sarcoma Centers: A Longitudinal Multicenter Pilot Study.

Electronic patient-reported outcome measures (ePROMs) are useful tools to assess care needs of patients diagnosed with cancer and to monitor their symptoms along the illness trajectory. Studies regarding the application of ePROMs by advanced practice nurses (APNs) specialized in sarcoma care and the use of such electronic measures for care planning and assessing quality of care are lacking. To explore the potential of ePROMs in clinical practice for assessing the patient's quality of life, physical functionality, needs, and fear of progression, as well as distress and the quality of care in sarcoma centers. A multicenter longitudinal pilot study design was chosen. Three sarcoma centers with and without APN service located in Switzerland were included. The instruments EQ-5D-5L, Pearman Mayo Survey of Needs, the National Comprehensive Cancer Network Distress Thermometer, PA-F12, and Toronto Extremity Salvage Score were used as ePROMs. Data were analyzed descriptively. Overall, 55 patients participated in the pilot study; 33 (60%) received an intervention by an APN, and 22 (40%) did not. Patients in sarcoma centers with APN service reported overall higher scores in quality of life and functional outcome. The number of needs and distress level were lower in sarcoma centers with APN service. No differences were found with respect to patients' fear of progression. Most of the ePROMs proved to be reasonable in clinical practice. PA-F12 has shown low clinical relevance. Using ePROMs appears to be reasonable to obtain clinically relevant patient information and to evaluate the quality of care in sarcoma centers.

18 - A multicentre pilot study on intravesical hyaluronic acid and chondroitin sulphate administration in the refractory Bacillus Calmette Guerin-induced chemical cystitis

18 - A multicentre pilot study on intravesical hyaluronic acid and chondroitin sulphate administration in the refractory Bacillus Calmette Guerin-induced chemical cystitis

Assessing the Subjective Effectiveness of Sensorimotor Insoles (SMIs) in Reducing Pain: A Descriptive Multicenter Pilot Study.

This pilot study aimed to investigate the use of sensorimotor insoles in pain reduction, different orthopedic indications, and the wearing duration effects on the development of pain. Three hundred and forty patients were asked about their pain perception using a visual analog scale (VAS) in a pre-post analysis. Three main intervention durations were defined: VAS_post: up to 3 months, 3 to 6 months, and more than 6 months. The results show significant differences for the within-subject factor "time of measurement", as well as for the between-subject factor indication (p < 0.001) and worn duration (p < 0.001). No interaction was found between indication and time of measurements (model A) or between worn duration and time of measurements (model B). The results of this pilot study must be cautiously and critically interpreted, but may support the hypothesis that sensorimotor insoles could be a helpful tool for subjective pain reduction. The missing control group and the lack of confounding variables such as methodological weaknesses, natural healing processes, and complementary therapies must be taken into account. Based on these experiences and findings, a RCT and systematic review will follow.

Value of prolonged scrotal drainage after penile prosthesis implantation: a multicenter prospective nonrandomized pilot study.

We aimed to understand the risks and benefits of post-inflatable penile prosthesis (IPP) implantation drainage and optimal duration. Our patients were divided into 3 groups: Group 1 (n = 114) had no drain placed, Group 2 had a drain placed for 24 h (n = 114) and Group 3 had a drain placed for 72 h (n = 117). Postoperative scrotal hematoma and prosthesis infection rates were compared between the groups. The patients from Group 3 demonstrated a statistically significant lower incidence of hematoma on the 10th postoperative day: (n = 1, 0.9%) compared to Group 2: (n = 11, 9.6%) and Group 1: (n = 8, 7%), (p = 0.013). However, on the 3rd postoperative day, there was a statistically significant lower incidence of hematoma in both Groups 3 and 2: (0.9% and 6.1%, respectively) vs. Group 1: (11.4%), (p = 0.004). Hematoma rates followed the same group order after the first day of surgery: 1.7% (n = 2), 5.3% (n = 6), and 8.8% (n = 10), respectively, (p = 0.05). Five patients (4.4%) in Group 1 and four patients (3.5%) in Group 2 developed an IPP associated infection, opposed to only a single patient (0.85%) in Group 3, (p = 0.210). We concluded that prolonged scrotal drainage for 72 h after virgin IPP implantation significantly reduces hematoma and infection rates.

IPSCs-based generation of vascular smooth muscle cells to study genotype-phenotype correlation in patients with ACTA2-related hereditary thoracic aortic aneurysm and dissection (HTAAD): A multicentric pilot study

Hereditary thoracic aortic aneurysm and dissection (HTAAD) is an autosomal dominant disorder characterized by dilatation or dissection of the thoracic aorta in families without Marfan- or related syndromes. ACTA2 gene, encoding alpha-smooth muscle actin, is the most frequent cause of non-syndromic HTAAD (12 to 21%). The penetrance is incomplete, with a dominant negative effect, and probably affected by the mutation location. Thus, the cumulative risk to aortic event ranges from 76% at the age of 85 in the classical form, to 100% by the age of 25 years in the highly penetrant ACTA2 variant at p.R179 which is associated with multisystem smooth muscle cell dysfunction syndrome (SMDS). SMDS is characterized by patent ductus arteriosus or aorto-pulmonary fenestration, early aortic dissection without dilatation, and cerebrovascular or coronary disease (moya-moya, stenosis). In clinical practice, this clinical heterogeneity doesn’t allow a relevant and personalized health care prevention. We proposed to develop and validate a new cellular model to explore in vitro the functional effects of ACTA2 variants (Class V) and to identify key mediator of vascular smooth muscle cells (VSMCs) dysfunction. Three patients with different ACTA2 pathogenic variants (p.G.148R, p.Thr212Lys, p.P355G) were included. Their phenotype was aortic dissection before 30-years old, and a more severe phenotype with ischemic stroke at 13-years old mimicking the SMDS for the patient with the pG.148R variant. Peripheric blood mononuclear cells (PBMCs) were collected from patient blood samples and induced pluripotent stem cells (iPSCs) were reprogrammed. Human iPSCs were differentiated toward the neural crest lineage, and subsequently to VSMCs. VSMCs were analyzed for their proliferation rate and migration capacity. Their actin polymerization was examined. To identify phenotypic difference between these three ACTA2 variants we performed RNAseq analysis. Patients VSMCs from healthy controls were used as controls. We observed difference of proliferation using FACS analysis, and of migration between these three iPSC-derived VSMCs compared to controls. Transcriptomic analysis is currently analysed and results will be presented at the congress. Our study identifies a robust protocol to obtain iPSC-derived VSMCs and provides novel insight on the genotype-phenotype correlation in patients with ACTA2 mutations.

Design of a multi-institutional neurocognitive discovery study in adult congenital heart disease (MINDS-ACHD)

Neurocognitive dysfunction (NCD) is a common comorbidity among children with congenital heart disease (CHD). However, it is unclear how underlying CHD and its sequelae combine with genetics and acquired cardiovascular and neurological disease to impact NCD and outcomes across the lifespan in adults with CHD. The Multi-Institutional Neurocognitive Discovery Study in Adults with Congenital Heart Disease (MINDS-ACHD) is a partnership between the Pediatric Heart Network (PHN) and the Adult Alliance for Research in Congenital Cardiology (AARCC) that examines objective and subjective neurocognitive function and genetics in young ACHD. This multicenter cross-sectional pilot study is enrolling 500 young adults between 18 and 30 years with moderate or severe complexity CHD at 14 centers in North America. Enrollment includes 4 groups (125 participants each): 1) d-looped Transposition of the Great Arteries (d-TGA); 2) Tetralogy of Fallot (TOF); 3) single ventricle (SV) physiology; and 4) "other moderately or severely complex CHD." Participants complete the standardized tests from the NIH Toolbox Cognitive Battery, the NeuroQoL, the Hospital Anxiety and Depression Scale, and the PROMIS Global QoL measure. Clinical and demographic variables are collected by interview and medical record review, and an optional biospecimen is collected for genetic analysis. Due to the COVID-19 pandemic, participation may be done remotely. Tests are reviewed by a Neurocognitive Core Laboratory. MINDS-ACHD is the largest study to date characterizing NCD in young adults with moderate or severely complex CHD in North America. Its results will provide valuable data to inform screening and management strategies for NCD in ACHD and improve lifelong care.

Effects of activation of an alcohol metabolic gene, cigarette smoking, and alcohol intake on the incidence of metachronous gastric cancer in patients who underwent endoscopic resection for gastric cancer: A multicenter retrospective pilot study.

Metachronous gastric cancer (GC) frequently occurs in patients who have undergone endoscopic resection (ER) for GC. We evaluated the risk for development of metachronous GC following ER for GC based on genetic polymorphisms for alcohol dehydrogenase-1B (ADH1B) and aldehyde dehydrogenase-2 (ALDH2), as well as alcohol consumption and smoking habits. We studied 77 patients who underwent ER for GC (median follow-up of 84 months). Genotyping of ADH1B/ALDH2 was performed using saliva sampling. Histories of alcohol consumption and smoking before and after ER and Helicobacter pylori eradication were documented. Multivariate analyses revealed that homozygous slow-metabolizing ADH1B (hazard ratio [HR] = 2.38, P < 0.13), heavy smoking (HR = 2.36, P < 0.09), and cigarette smoking after ER (HR = 2.47, P < 0.10) were not independently associated with the risk of secondary GC development. We analyzed the cessation status of the 38 patients who were classified as heavy smokers before ER based on their smoking habits after the ER and divided them into a cessation group (n = 27, non-smokers after ER) and a non-cessation group (n = 11). Cumulative incidence curves of secondary GC in the cessation and non-cessation groups revealed 5-year incidence rates of 19.0% and 45.0%, respectively (P = 0.02). Continued cigarette smoking, at a high level, may be an important risk factor for the development of metachronous GC. Advice for smoking cessation should be given.

Clinical effectiveness of training for awareness, resilience, and action for adolescents and young adults with depression: The pilot phase of a multicenter randomized controlled trial.

Depression is a top-ranking global health concern increasing in magnitude. Available treatments for adolescents and young adults are not convincingly effective and relapse rates remain high. Training for Awareness, Resilience and Action (TARA) is a group treatment program targeting specific pathophysiological mechanisms of depression in young people. TARA is feasible, acceptable, preliminarily efficacious in depressed American adolescents, and it affects postulated brain-circuitry. As an initial step of a multicenter randomized controlled trial (RCT) we performed a single-arm multicenter pilot-study on TARA. Thirty-five depressed individuals (15-21 years old, 28 females) received TARA for 12 weeks face-to-face or online. Data was collected before (T0), during, and after the intervention (T1). The trial was pre-registered at clinicaltrials.gov, NCT Registration: identifier [NCT04747340]. Feasibility outcomes included recruitment, attendance rates, and session ratings. Adverse events were recorded weekly and extracted from medical records at the end of the trial. Primary effectiveness outcome was self-rated depression severity on Reynolds Adolescent Depression scale 2nd ed. at T1. Secondary outcomes were Children's Depression Rating Scale-revised (CDRS-R) and Multidimensional Anxiety Scale for Children (MASC) at T1. TARA was feasible and safe in the present trial. No significant RADS-2-change was seen (adjusted mean difference -3.26, 95 % CI -8.35 to 1.83; p= 0.20), however a significant decrease in CDRS-R scores is reported (adjusted mean difference -9.99, 95% CI -14.76 to -5.22; p < 0.001). MASC-scores did not change significantly (adjusted mean difference 1.98, 95% CI -0.96 to 4.91; p=0.18). Additional feasibility aspects are presented and discussed. Limitations include substantial loss-to-follow-up, no randomization to control, and that some participants received concomitant treatment(s). The Coronavirus pandemic complicated both implementation and interpretation of the trial. In conclusion TARA was feasible and safe in depressed adolescents and young adults. Preliminary signs of effectiveness were seen. The initiated RCT will be important and worthwhile to conduct, and several improvements to the design are suggested based on the present results. clinicaltrials.gov, identifier NCT04747340.

Cerebrolysin as an Early Add-on to Reperfusion Therapy: Risk of Hemorrhagic Transformation after Ischemic Stroke (CEREHETIS), a prospective, randomized, multicenter pilot study

BackgroundCerebrolysin could mitigate reperfusion injury and hemorrhagic transformation (HT) in animal models of acute ischemic stroke.MethodsThis was a prospective, randomized, open-label, parallel-group with active control, multicenter pilot study. Cerebrolysin (30 mL/day over 14 days) was administered concurrently with alteplase (0.9 mg/kg) in 126 patients, whereas 215 control patients received alteplase alone. The primary outcomes were the rate of any and symptomatic HT assessed from day 0 to 14. The secondary endpoints were drug safety and functional outcome measured with the National Institutes of Health Stroke Scale (NIHSS) on day 1 and 14, and the modified Rankin scale (mRS) on day 90. Advanced brain imaging analysis was applied on day 1 and 14 as a marker for in vivo pharmacology of Cerebrolysin.ResultsCerebrolysin treatment resulted in a substantial decrease of the symptomatic HT rate with an odds ratio (OR) of 0.248 (95% CI: 0.072–0.851; p = 0.019). No serious adverse events attributed to Cerebrolysin occurred. On day 14, the Cerebrolysin arm showed a significant decrease in the NIHSS score (p = 0.045). However, no difference in the mRS score was observed on day 90. A substantial improvement in the advanced brain imaging parameters of the infarcted area was evident in the Cerebrolysin group on day 14.ConclusionsEarly add-on of Cerebrolysin to reperfusion therapy was safe and significantly decreased the rate of symptomatic HT as well as early neurological deficit. No effect on day 90 functional outcome was detected. Improvements in the imaging metrics support the neuroprotective and blood–brain barrier stabilizing activity of Cerebrolysin.Trial registrationName of Registry: ISRCTN.Trial Registration Number: ISRCTN87656744.Trial Registration Date: 16/02/2021.

Feasibility and Safety of Tailored Lymphadenectomy Using Sentinel Node-Navigated Surgery in Patients with High-Risk T1 Esophageal Adenocarcinoma

BackgroundSelective lymphadenectomy using sentinel node-navigated surgery (SNNS) might offer a less invasive alternative to esophagectomy in patients with high-risk T1 esophageal adenocarcinoma (EAC). The aim of this study was to evaluate the feasibility and safety of a new treatment strategy, consisting of radical endoscopic resection of the tumor followed by SNNS.MethodsIn this multicenter pilot study, ten patients with a radically resected high-risk pT1cN0 EAC underwent SNNS. A hybrid tracer of technetium-99m nanocolloid and indocyanine green was injected endoscopically around the resection scar the day before surgery, followed by preoperative imaging. During surgery, sentinel nodes (SNs) were identified using a thoracolaparoscopic gammaprobe and fluorescence-based detection, and subsequently resected. Endpoints were surgical morbidity and number of detected and resected (tumor-positive) SNs.ResultsLocalization and dissection of SNs was feasible in all ten patients (median 3 SNs per patient, range 1–6). The concordance between preoperative imaging and intraoperative detection was high. In one patient (10%), dissection was considered incomplete after two SNs were not identified intraoperatively. Additional peritumoral SNs were resected in four patients (40%) after fluorescence-based detection. In two patients (20%), a (micro)metastasis was found in one of the resected SNs. One patient experienced neuropathic thoracic pain related to surgery, while none of the patients developed functional gastroesophageal disorders.ConclusionsSNNS appears to be a feasible and safe instrument to tailor lymphadenectomy in patients with high-risk T1 EAC. Future research with long-term follow-up is warranted to determine whether this esophageal preserving strategy is justified for high-risk T1 EAC.

Prenatal diagnosis by trio exome sequencing in fetuses with ultrasound anomalies: A powerful diagnostic tool.

Introduction: Prenatal ultrasound (US) anomalies are detected in around 5%-10% of pregnancies. In prenatal diagnosis, exome sequencing (ES) diagnostic yield ranges from 6% to 80% depending on the inclusion criteria. We describe the first French national multicenter pilot study aiming to implement ES in prenatal diagnosis following the detection of anomalies on US. Patients and methods: We prospectively performed prenatal trio-ES in 150 fetuses with at least two US anomalies or one US anomaly known to be frequently linked to a genetic disorder. Trio-ES was only performed if the results could influence pregnancy management. Chromosomal microarray (CMA) was performed before or in parallel. Results: A causal diagnosis was identified in 52/150 fetuses (34%) with a median time to diagnosis of 28 days, which rose to 56/150 fetuses (37%) after additional investigation. Sporadic occurrences were identified in 34/56 (60%) fetuses and unfavorable vital and/or neurodevelopmental prognosis was made in 13/56 (24%) fetuses. The overall diagnostic yield was 41% (37/89) with first-line trio-ES versus 31% (19/61) after normal CMA. Trio-ES and CMA were systematically concordant for identification of pathogenic CNV. Conclusion: Trio-ES provided a substantial prenatal diagnostic yield, similar to postnatal diagnosis with a median turnaround of approximately 1 month, supporting its routine implementation during the detection of prenatal US anomalies.

Performance of Brazilian Pediatric Risk of Severity Model for Illness (Brprism) Compared to Pediatric Index of Mortality and Pediatric Risk of Mortality 2

Introduction: The best prognosis score with which to evaluate high-risk patients upon admission into pediatric intensive care is not well established in resource-limited settings. The objective of study was to formulate a risk-of-illness severity model for pediatric mortality to be applied upon PICU admission in resource-limited settings. Methods: Our study was designed to develop an illness severity index and a prognostic model for critically ill children. A prospective, observational multicenter pilot study, performed between February 1995 and October 1999, evaluated the variables, methodology and statistical techniques for the development of a model. A single-center prospective cohort study, performed between November 1999 and October 2004, collected information from consecutive admissions into the Pediatric Intensive Care Unit (PICU) at a high-complexity university, teaching, and reference hospital in São Paulo, Brazil. Results: In the pilot study, 1,459 patients (a PICU mortality rate of 16%) were included, and in the second study, 1,033 patients (a PICU mortality rate of 13.9% and a hospital mortality rate of 6.9% after PICU discharge) were included. We used multivariable regression to determine two probabilistic models; the first addressed survival and the overall probability of death (hospital plus PICU deaths), and the second was conditional (i.e., PICU death). An illness severity index stratified these probabilities into three risk strata: low-, medium- and high-risk patients. In the final step, the new death probabilities were estimated using a Bayesian adjustment. Conclusions: The model estimates three probabilities (survival, death in the PICU and death in the hospital after PICU discharge) stratified into three risk categories. To the best of our knowledge, this is the first study using a Bayesian adjustment to determine a prognosis and illness severity, and it should enable us to make therapeutic adjustments and provide appropriate counseling for high-risk patients in resource-limited settings.

Level I axillary dissection in patients with breast cancer and tumor-involved sentinel lymph node after NAC is not sufficient for adequate nodal staging.

The purpose of the study was to investigate the oncological sufficiency of level I axillary dissection for adequate histological nodal staging (ypN) in patients with breast cancer and tumor-involved sentinel lymph node (SLN) after neoadjuvant chemotherapy (NAC). A prospective multicentre pilot study took place from 01.01.2018 to 30.11.2020 in three mammary centres in the Czech Republic in patients with breast cancer after NAC (NCT03556397). Patients in the cohort with positive histological frozen section of SLN were indicated to separate axillary dissection of levels I and II. Sixty-one patients with breast cancer after NAC were included in the study according to inclusion and exclusion criteria. Twelve patients with breast cancer and tumour involved SLN after NAC were further included in the analysis. Two (16.7%) patients had positive non-sentinel lymph nodes in level I only, one (8.3%) patient had positive lymph nodes in level II only, and seven (58.3%) patients had positive lymph nodes in both levels. Level I axillary dissection in a patient with tumour involved SLN after NAC would have resulted in understaging in five (41.7%) patients, mostly ypN1 instead of ypN2. According to our pilot result, level I axillary dissection is not sufficient in terms of adequate histological nodal staging in breast cancer patients after NAC, and level II axillary dissection should not be omitted.

Abstract No. 574 Safety and Feasibility of a PEEK Polymer Transpedicular Vertebral System to Treat Vertebral Compression Fracture: A Multicenter Pilot Study

Abstract No. 574 Safety and Feasibility of a PEEK Polymer Transpedicular Vertebral System to Treat Vertebral Compression Fracture: A Multicenter Pilot Study

Drained versus non-drained inflatable penile prosthesis implantation: A multicenter prospective pilot study

Drained versus non-drained inflatable penile prosthesis implantation: A multicenter prospective pilot study

Finger doses due to 68Ga-labelled pharmaceuticals in PET departments—results of a multi-centre pilot study

Introduction: Although the use of 68Ga has increased substantially in nuclear medicine over the last decade, there is limited information available on occupational exposure due to 68Ga. The purpose of this study is to determine the occupational extremity exposure during the preparation, dispensing and administration of 68Ga-labelled radiopharmaceuticals. Method: Workers in eight centres wore a ring dosimeter for all tasks involving 68Ga-labelled radiopharmaceuticals for a minimum of one month. Additionally, the fingertip dose was monitored in two centres and the hand with the highest ring dose during 68Ga procedures was also identified in one centre. Results: The median normalised ring dose for 68Ga procedures was found to be 0.25 mSv GBq−1 (range 0.01–3.34). The normalised 68Ga ring doses recorded in this study are similar to that found in the literature for 18F. This study is consistent with previous findings that the highest extremity dose is found on the non-dominant hand. A limited sub study in two of the centres showed a median fingertip to base of the finger dose ratio of 4.3. Based on this median ratio, the extrapolated annual 68Ga fingertip dose for 94% of the workers monitored in this study would be below Category B dose limit (150 mSv) and no worker would exceed Category A dose limit (500 mSv). Conclusion: When appropriate shielding and radiation protection practices are employed, the extremity dose due to 68Ga is comparable to that of 18F and is expected to be well below the regulatory limits for the majority of workers.

P320 Confocal Laser Endomicroscopy to predict the efficacy of vedolizumab as first-line biotherapy in ulcerative colitis

Abstract Background There is no biomarker to guide first-line of biotherapy in ulcerative colitis (UC). The main objective of this multicenter pilot study was to demonstrate the feasibility of identifying a4b7 and/or TNF-a expressing cells by dual-band confocal endomicroscopy (CLE) into the mucosa to predict the response to vedolizumab as first-line biotherapy. NCT02878083. Methods Patients with moderate to severe UC, naïve of biotherapy were prospectively included and received vedolizumab. Patients were evaluated clinically, biologically, endoscopically and histologically at week 22. Nonresponding patients were subsequently treated by adalimumab and were evaluated at week 30. Prior to the initiation of vedolizumab, during endoscopy, 8 biopsies were taken for extemporaneous and ex vivo analysis by CLE, histology and confocal microscopy. Fresh biopsies were treated with vedolizumab coupled with FITC and adalimumab coupled with Alexa-Fluor to analyze their respective fluorescence (figure) in one step with the Dual-band CLE system (Mauna Kea Technologies, France). The number and the surface of positive stained areas visualized by CLE were counted and compared between responders and non-responders. Blood samples were collected before each infusion and were used to analyze the immune cells (LT, LB, NK, monocytes) by FACS and vedolizumab trough levels. Frozen biopsies were analyzed by confocal microscopy (Opal Akoya multiplexing; Tyramide signal amplification) after labeling by an anti-a4b7 antibody. Results 19 patients out of the 20 initially planned were included. Clinical remission, endoscopic improvement (Mayo ≤ 1), endoscopic remission (Mayo 0) at W22 were respectively 58%, 58% and 45%. 18 out of 19 patients (95%) could be analyzed by CLE. The number and surface of vedolizumab-FITC stained areas were numerically superior in responder patients at week22; by contrast, no difference was found for the areas marked by adalimumab-Alexa-fluor. A threshold value of 6 areas marked in CLE by the vedolizumab-FITC at week 0 discriminated responders and non-responders at week 22 with a Se of 78% and a Sp of 85%. No difference of circulating cell population has been identified between responders and non-responders and the vedolizumab trough levels were not predictive of the response. In confocal microscopy, 15 patients have been analyzed; the percentage of a4b7-binding cells was not different between responders (n=9) and non-responders (n=6) Conclusion This study confirms the feasibility of extemporaneous identification by dual-band CLE of the mucosal cells expressing a4b7 and/or TNF-a. Results suggest that only the proportion of cells binding vedolizumab-FITC in the mucosa was indicative of the response to vedolizumab

Wearable sensor-based performance status assessment in cancer: A pilot multicenter study from the Alliance for Clinical Trials in Oncology (A19_Pilot2).

Clinical performance status is designed to be a measure of overall health, reflecting a patient's physiological reserve and ability to tolerate various forms of therapy. Currently, it is measured by a combination of subjective clinician assessment and patient-reported exercise tolerance in the context of daily living activities. In this study, we assess the feasibility of combining objective data sources and patient-generated health data (PGHD) to improve the accuracy of performance status assessment during routine cancer care. Patients undergoing routine chemotherapy for solid tumors, routine chemotherapy for hematologic malignancies, or hematopoietic stem cell transplant (HCT) at one of four sites in a cancer clinical trials cooperative group were consented to a six-week prospective observational clinical trial (NCT02786628). Baseline data acquisition included cardiopulmonary exercise testing (CPET) and a six-minute walk test (6MWT). Weekly PGHD included patient-reported physical function and symptom burden. Continuous data capture included use of a Fitbit Charge HR (sensor). Baseline CPET and 6MWT could only be obtained in 68% of study patients, suggesting low feasibility during routine cancer treatment. In contrast, 84% of patients had usable fitness tracker data, 93% completed baseline patient-reported surveys, and overall, 73% of patients had overlapping sensor and survey data that could be used for modeling. A linear model with repeated measures was constructed to predict the patient-reported physical function. Sensor-derived daily activity, sensor-derived median heart rate, and patient-reported symptom burden emerged as strong predictors of physical function (marginal R2 0.429-0.433, conditional R2 0.816-0.822). Trial Registration: Clinicaltrials.gov Id NCT02786628.

Gestalt clinical severity score (GCSS) as a predictor of patient severity of illness or injury

ObjectiveTo determine if clinical judgement is accurate to predict the severity of injury or illness, and can be used at patient arrival when other formal scoring systems are not yet available. DesignA multicenter pilot study using a prospective observational convenience sample of patients arriving by EMS to the emergency department (ED) or Trauma Center. SettingTwo urban, Level 1 trauma centers at academic tertiary care hospitals. PatientsMedical and trauma patients age 18 and older transported by EMS (N = 216). Exclusion criteria (prior to arrival): intubation, assisted ventilation (BVM or NPPV), CPR in progress, prisoners, or previously present motor or speech deficits. MeasurementsCompletion of a novel 15-point scale of Verbal, Motor, and Facial Expression within 1–2 min of arrival by a clinician outside of the treatment team. Primary endpoint was the immediate disposition from the ED or Trauma Center: Home, Brief Observation (<24 h), Admission to Floor, ICU (OR and IR as surrogates since these patients ultimately go to the ICU), or Morgue. ResultsUnivariate analysis revealed a strong, positive monotonic correlation between GCSS and disposition (Rho = 0.693, p < .0001). Multivariable logistic regression revealed the “best” model included GCSS and age (group 18–44 years old versus all the other age groups) (p < .0001). There was a 156% increase in the odds of being discharged home (versus being admitted) for a one-unit increase in GCSS (OR = 2.56, 95% CI 1.94, 3.37). ConclusionsPhysicians can make accurate predictions of severity of injury and illness using a gestalt method and the scoring system we have developed as patient disposition correlates well with GCSS score. GCSS is most accurate with the 18–44 age group.

Wearable sensor-based performance status assessment in cancer: A pilot multicenter study from the Alliance for Clinical Trials in Oncology (A19_Pilot2)

Wearable sensor-based performance status assessment in cancer: A pilot multicenter study from the Alliance for Clinical Trials in Oncology (A19_Pilot2)