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Questionnaires
Patients in the RobExReha-Patients group received training with the RobExReha device. A session consisted of the donning and setup of the device, subsequent gaming therapy, termination of the session and doffing of the device. Amount and mode of robotic support as well as the length of the gaming sessions were adjusted...
PMC10422755
Statistical methods
±
Both patient groups filled out the Quebec User Evaluation of Satisfaction with assistive Technology (QUEST) [As the RobExReha-Patients group used the HoloLens for the gaming environment, they additionally filled out the “presence in augmented reality” (pAR) questionnaire (adapted from [The therapists completed a questi...
PMC10422755
Results
PMC10422755
Safety
temporary shoulder pain, shoulder pain
ADVERSE EVENTS, ADVERSE EVENT
No serious adverse events occurred and none of the patients mentioned feeling unsafe at any time. However, one study-related non-serious adverse event was registered: temporary shoulder pain, which vanished within an hour after the therapeutic intervention. This patient had an unstable sitting posture in the wheelchair...
PMC10422755
Usability
PMC10422755
Feasibility and technical aspects
spasticity
Concerning the robotic set up, the positioning of the LBR arm on the cart (Fig. For three patients, the arm weighing process did not function as intended. As a result, the default weight of 0.01 kg was shown by the robot and the weight needed to be estimated and corrected manually. These patients suffered pronounced sp...
PMC10422755
Video protocol – donning and doffing times
pain
Starting and preparing the RobExReha system included the activation of the system and HoloLens, the connection of the HoloLens to the robot, donning the elbow brace onto the patient’s arm and connecting it to the robot flange, weighing of the arm as well as preparation and donning of the HoloLens. This collectively too...
PMC10422755
Questionnaires
PMC10422755
Discussion
orthosis
Results of the Quebec User Evaluation of Satisfaction with assistive technology (QUEST) patients’ questionnaire: Scores can range from 1 (not satisfied at all) to 5 (highly satisfied). Asterisks indicate significant differences. Circles indicate outliersThe results of the QUEST (see Fig. The RobExReha-Patients found t...
PMC10422755
Safety and clinical feasibility
shoulder pain
CONSTRICTION, MINOR
The results suggest that the use of the RobExReha system was safe and feasible in neurological patients. None of the patients mentioned safety concerns. The occurrence of shoulder pain in one patient, however, highlights that special caution should be taken when treating patients with limited stability in the trunk and...
PMC10422755
Donning and doffing
Most patients found that the device was relatively easy to put on. From a therapist’s perspective, only one therapist mentioned difficulties, specifically in the connection process of the elbow brace to the flange: sometimes the robot seemed to be in a slightly incorrect position. The large range of donning times of th...
PMC10422755
Patients’ perspective
PMC10422755
Usability questionnaires
Due to the prototype stage of the RobExReha device, we anticipated lower usability in the QUEST when compared to the Armeo Reference Group. However, this was only confirmed for the overall score and the items “reliability and safety” and “easy to use”. Yet, usability was generally rated positive by the patients in all ...
PMC10422755
Workload
The overall workload in the RTLX tended to be higher in the RobExReha group with a mean score of 41.7 compared to the Reference Group (mean: 34.1) without reaching a significant difference neither in the overall score nor domain-wise. Those are slightly lower compared to global work load scores, where video gaming had ...
PMC10422755
3D perception
stroke, impairments in stereoscopic vision, reduced mobility of the cervical spine
STROKE, STILL
The gaming environment in AR was an important focus of this study. Specifically, concerns regarding the spatial perception of stroke patients when using the HoloLens were of interest. The feasibility of using the HoloLens in neurological patients as shown by Rohrbach et al. [Regarding the AR perception by the patients,...
PMC10422755
Therapists’ perspective
STILL
The system was rated by the therapists in the UEQ-S as “leading edge”, “inventive”, “interesting” and “exciting”, despite some improvable features (as mentioned above) and the limitations in system stability. The overall usability as measured with the SUS was 60.5 ± 21.8 (25-82.5), which corresponds to an average of an...
PMC10422755
Potential optimization
cognitive and motor function, spasticity
MINOR
Although the RobExReha system was generally perceived as inventive and usable, possibilities to improve the system could be identified: The positioning of the LBR should be adjusted to better accommodate the targeted range of motion and avoid conflicts with the axis boundaries of the LBR arm.Another solution for the we...
PMC10422755
Limitations
left-sided impairments, stroke
STROKE
Due to the prototype stage, the RobExReha system was only available for training of the right arm, which led to limitations in patient inclusion. The Reference Group, contrarily, additionally included patients with left-sided impairments. This could lead to bias; for example, in stroke patients, as the affected hemisph...
PMC10422755
Conclusion
We investigated a novel robotic arm therapy system in combination with an augmented reality serious game. The system demonstrated to be feasible in patients with impairments of the upper extremity from neurological causes and good cognitive function. Notably, the good acceptance and perception of the game using the Hol...
PMC10422755
Acknowledgements
We would like to thank Christian Frede and his team (AmbiGate GmbH, Tübingen) for their contribution to the software of the system and Dr. Winfried Ilg (Centre for Integrative Neuroscience, Tübingen) for useful discussions and consultation. We want to thank our colleagues at BEC GmbH, Fraunhofer IPA and Schön Klinik th...
PMC10422755
Author’s contributions
MH, FM
AC: methodology, formal analysis, investigation, data curation, writing – original draft, visualization, project administration SR: investigation, resources, writing – original draft, MH: writing – review & editing, conceptualisation, methodology, project administration, JL: resources, writing – original draft, TA: met...
PMC10422755
Funding
The development of the RobExReha device and the clinical study were done within the scope of the “RobExReha”-project funded by the German Federal Ministry of Education and Research (BMBF) under the funding code 16SV7849.
PMC10422755
Data Availability
The dataset supporting the conclusion of this article is available on reasonable personal request.
PMC10422755
Declarations
PMC10422755
Competing interests
This study was performed while SR was employed at BEC GmbH and JL at Fraunhofer IPA, who manufactured together with Ambigate GmbH the RobExReha system.
PMC10422755
Ethics approval and consent to participate
DER
This study conforms with the Declaration of Helsinki, all participants provided informed consent prior to enrolment. An ethical approval was obtained (Ethikkommission der Bayerischen Landesärztekammer) and the trial was registered at DRKS (DRKS00022136).
PMC10422755
Consent for publication
Not applicable.
PMC10422755
References
PMC10422755
Background
kidney damage, autosomal dominant polycystic kidney disease, ADPKD
ADPKD, PROGRESSION, AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE
Tolvaptan slows expansion of kidney volume and kidney function decline in adults with autosomal dominant polycystic kidney disease (ADPKD). Progression during childhood could be treated before irreversible kidney damage occurs, but trial data are lacking. We evaluated the safety and efficacy of tolvaptan in children/ad...
PMC10101612
Methods
ADPKD
ADPKD
This was the 1-year, randomized, double-blind, portion of a phase 3b, two-part trial being conducted at 20 academic pediatric nephrology centers. Key eligibility criteria were ADPKD and eGFR ≥60 ml/min per 1.73 m
PMC10101612
Results
Among the 91 randomized (group 1,
PMC10101612
Conclusions
ADPKD
ADPKD
Tolvaptan exhibited pharmacodynamic activity in pediatric ADPKD. Aquaretic effects were manageable, with few discontinuations.
PMC10101612
Clinical Trial registry name and registration number:
ADPKD
ADPKD, AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE
Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease) NCT02964273.
PMC10101612
Introduction
hereditary kidney disorders, ADPKD, impairment of kidney function, Autosomal dominant polycystic kidney disease
ADPKD, KIDNEY FAILURE, DISEASE PROGRESSION, AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE
Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common hereditary kidney disorders, characterized by the progressive formation and growth of cysts, leading to impairment of kidney function and eventually kidney failure.Given the phenotypic variability, uncertainty exists regarding the management...
PMC10101612
Methods
PMC10101612
Participants
Study methods have been reported.Exclusion criteria included alanine aminotransferase/aspartate aminotransferase ≥1.5 × upper limit of normal or any medical condition that could interfere with the evaluation of the trial objectives or endanger participant safety.
PMC10101612
Trial Design
This was a phase 3b, two-part trial (EudraCT number: 2016-000187-42;
PMC10101612
Treatments
Participants received tolvaptan or placebo daily in a split-dose regimen, with the first dose taken upon waking and the second 8–9 hours later (
PMC10101612
End Points
early-stage disease
Given that treatment effects on TKV or kidney function are difficult to measure in a pediatric population with early-stage disease, pharmacodynamic parameters were considered the most appropriate coprimary end points: change from baseline in spot urine osmolality and spot urine specific gravity at week 1 in all partici...
PMC10101612
Assessments
Postscreening assessment visits occurred at baseline (day 1), week 1, and monthly for 12 months. Participants aged 12–17 years underwent TKV assessment by MRI performed according to published standards at screening and month 12.
PMC10101612
Statistical Analyses
SECONDARY
A sample size of ≥60 participants aged 12–17 years was planned. It was expected that approximately 40 participants aged 4–11 years would also enroll for an anticipated total enrollment of 100 participants. As data were to be summarized descriptively, no formal power calculations to determine sample size were undertaken...
PMC10101612
Results
PMC10101612
Baseline Characteristics
autosomal dominant polycystic kidney disease, ADPKD, anemia
ANEMIA, POLYCYSTIC KIDNEY DISEASE, NEPHROLITHIASIS, ADPKD, AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE, COLONIC DIVERTICULITIS
Demographic characteristics in the total population were similar across treatment arms (Table Baseline demographic and clinical characteristics of children and adolescents enrolled in a randomized, placebo-controlled clinical trial of tolvaptanPKD, polycystic kidney disease; ADPKD, autosomal dominant polycystic kidney ...
PMC10101612
Exposure to Study Medication
In all randomized participants, exposure of ≥361 days was achieved in 33 of 48 participants (69%) in the tolvaptan arm and 29 of 43 participants (67%) in the placebo arm. The average daily dose was 41 (SD 17) mg in the tolvaptan arm and 51 (SD 14) mg in the placebo arm. The most frequent modal total daily doses at mont...
PMC10101612
Pharmacodynamics
nonadherence, tuberous sclerosis
SECONDARY, TUBEROUS SCLEROSIS
For the first coprimary efficacy end point (all participants), least squares (LS) mean (±SEM) reduction from baseline in spot urine osmolality (premorning dose) after 1 week of daily dosing was greater in the tolvaptan arm (−390 [28] mOsm/kg) than in the placebo arm (−90 [29] mOsm/kg; Coprimary, key secondary, and rela...
PMC10101612
Safety and Tolerability
death, bleeding, overdose, pelvic pain, fracture, cirrhosis, fibrosis, hematuria, dizziness, kidney pain, intentional self-injury, pollakiuria, deaths
CHOLESTASIS AND JAUNDICE, BLEEDING, ADVERSE EVENTS, EVENT, CIRRHOSIS, EVENTS, HEPATIC FAILURE, FIBROSIS, HEMATURIA, NONINFECTIOUS HEPATITIS, ADVERSE EVENT, VIRAL PERICARDITIS, PETIT MAL EPILEPSY, HYPERTENSIVE CRISIS
Treatment-emergent AEs were reported for 44 of 48 participants (92%) in the tolvaptan group and 42 of 43 participants (98%) in the placebo group during phase A of the trial. The most frequent AEs are shown in Table Summary of adverse events and listing of individual treatment-emergent adverse events (by system organ cl...
PMC10101612
Discussion
ADPKD, aquaretic adverse, orthostatic hypotension
ADPKD, SECONDARY, DRUG-INDUCED LIVER INJURY, EVENTS, ORTHOSTATIC HYPOTENSION
We report the first phase 3, interventional clinical trial of tolvaptan in children and adolescents with ADPKD. The current report focuses on the randomized, placebo-controlled, 1-year portion (phase A) of the study. In the absence of standardized criteria for identifying the potential for rapid progression in the pedi...
PMC10101612
Supplementary Material
PMC10101612
Acknowledgments
Autosomal Dominant Polycystic Kidney Disease
AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE
The development of this manuscript was supported by Otsuka Pharmaceutical Development & Commercialization, Inc. (Rockville, MD). Editorial and writing services, including writing of a first draft, were provided by Andrew J. Horgan, PhD, of BioScience Communications, Inc. (New York, NY), activities that were also funded...
PMC10101612
Disclosures
KIDNEY DISEASES
M.A. Cadnapaphornchai reports consultancy agreements with Otsuka Pharmaceutical, honoraria from Otsuka, and an advisory or leadership role for Otsuka Tolvaptan pediatric steering committee. A. Dandurand reports employment with Cerevel Therapeutics and was an employee of Otsuka at the time of the study. A. Dandurand's s...
PMC10101612
Funding
This research was funded by Otsuka Pharmaceutical Development & Commercialization, Inc. (Rockville, MD).
PMC10101612
Author Contributions
A. Dandurand, D.M. Mekahli, F. Schaefer, L. Shi, S.E. Shoaf, and K. Sikes conceptualized the study; M.A. Cadnapaphornchai, A. Dandurand, L.A. Greenbaum, L. Guay-Woodford, M. Litwin, D.M. Mekahli, F. Schaefer, T. Seeman, S.E. Shoaf, and K. Sikes were responsible for investigation; M.A. Cadnapaphornchai, A. Dandurand, L....
PMC10101612
Data Sharing Statement
To submit inquiries related to Otsuka clinical research, or to request access to individual participant data (IPD) associated with any Otsuka clinical trial, please visit
PMC10101612
References
PMC10101612
Supplementary Information
In a randomised trial, we found that integrated maternal HIV and infant health services through the end of breastfeeding were significantly associated with the primary outcome of engagement in HIV care and viral suppression at 12 months postpartum, compared to the standard of care. Here, we quantitatively explore poten...
PMC10598190
Keywords
Open access funding provided by University of Cape Town.
PMC10598190
Introduction
HIV infections
HIV INFECTIONS
Increases in the availability and uptake of antiretroviral therapy (ART), including under guidelines of universal ART for pregnant and breastfeeding women, have led to massive reductions in new paediatric HIV infections [An approach which has been posited to reduce disengagement from care is the integration of HIV care...
PMC10598190
Methods
This analysis draws on data from the MCH-ART trial, which evaluated integrated services during the postpartum period. The design and primary results of the trial have been previously reported [
PMC10598190
MCH-ART Intervention
SECONDARY
Women who attended the immediate postpartum study visit and were currently breastfeeding [471 (75%) of 628 women enrolled into antenatal follow-up] were enrolled into postpartum follow-up and randomly allocated. Random allocation was to the MCH-ART intervention (integrated concurrent and co-located maternal ART and pae...
PMC10598190
Measures
Depression, HIV and HIV treatment knowledge [Depression, Viral suppression
DISORDERS
Table  Factors explored as (A) effect modifiers and (B) mediators of the intervention effect, across timepoints of assessmentAbbreviations: EPDS: Edinburgh Postnatal Depression Scale; K-10: Kessler-10 scale; AUDIT-C: Alcohol Use Disorders Identification Test – Consumption.At various antenatal and postpartum timepoints,...
PMC10598190
Data Analysis
Data were analysed using STATA V14.2 (Stata Corporation, College Station, Texas, USA) and R (R Foundation for Statistical Computing, Vienna, Austria). To assess modifiers of the intervention effect, we used stratified χ
PMC10598190
Results
Depression
DISORDERS
Between March 2013 and April 2014, 628 pregnant women living with HIV were enrolled into antenatal follow-up. Of these women, 471 (75%) attended the immediate postpartum study visit and reported breastfeeding, and were enrolled into the MCH-ART trial. Compared to those enrolled, women who were excluded had entered ante...
PMC10598190
Modifiers of the Intervention Effect
Depression
DISORDERS
Psychosocial characteristics relevant to the analyses of effect modification are summarised in Table  Forest plot of primary outcome across subgroups of psychosocial characteristics measured prior to randomisation Intervention effect, stratified by potential effect modifiersAbbreviations: EPDS: Edinburgh Postnatal Depr...
PMC10598190
Mediators of the Intervention Effect
No appreciable differences were observed in HIV or treatment knowledge, ART medication beliefs or adherence self-efficacy between the intervention and control group at study measurement visits after randomisation (Table  Potential postpartum mediators of the intervention effect, by trial arm (intervention versus contro...
PMC10598190
Discussion
SECONDARY
Using data from the MCH-ART study, which showed that integrated maternal and child health care during the postpartum period significantly increased retention in HIV care and viral suppression [Taken together, the intervention appeared to be most effective among women experiencing added vulnerability due to poverty, uni...
PMC10598190
Conclusions
Despite these limitations, this analysis provides novel and important findings related to modifiers and mediators of the effectiveness of integrated care for postpartum women living with HIV. Our findings suggest that integrated services are associated with significantly better patient-provider relationships over time....
PMC10598190
Acknowledgements
AIDS
PEDIATRIC AIDS, AIDS, EMERGENCY
The authors would like to thank the women who participated in this study, as well as the study staff for their support of this research. This research was supported by the President’s Emergency Plan for AIDS Relief (PEPFAR) through the National Institute of Child Health and Human Development (NICHD), grant numbers 1R01...
PMC10598190
Authors’ Contribution
LM and EJA conceived of and designed the parent study, with input from RHR and CAM. KBrittain, TP, and AZ implemented the research, data collection and data management. KBrittain and KBrown performed the data analysis. KBrittain wrote the first draft of the manuscript. All authors critically reviewed the manuscript and...
PMC10598190
Funding
AIDS
PEDIATRIC AIDS, AIDS, EMERGENCY
Open access funding provided by the University of Cape Town. This research was supported by the President’s Emergency Plan for AIDS Relief (PEPFAR) through the National Institute of Child Health and Human Development (NICHD), grant numbers 1R01HD074558. Additional funding comes from the Elizabeth Glaser Pediatric AIDS ...
PMC10598190
Data Availability
All relevant data are within the paper.
PMC10598190
Code Availability
N/A.
PMC10598190
Declarations
PMC10598190
Conflict of interest
The authors have no conflicts of interest to declare.
PMC10598190
Ethics Approval
This study was performed in line with the principles of the 1964 Declaration of Helsinki and its later amendments. The study was approved by the Faculty of Health Sciences Human Research Ethics Committee at the University of Cape Town, and the Institutional Review Board of Columbia University Medical Centre.
PMC10598190
Consent to Participate
All participants provided written informed consent prior to enrolment.
PMC10598190
Consent for publication
Not applicable.
PMC10598190
References
PMC10598190
Background
depression
Only about half the people with depression seek professional health care services. To constitute the different predictors and associating variables of health care utilisation, we model the process and aim to test our hypothesised
PMC9831378
Method
depressive symptoms
All variables are examined in an online study (baseline, three- and six-month follow-up). The sample consisted of adults with depressive symptoms (PHQ-9 sum score ≥ 8), currently not receiving mental health care treatment. To examine the prediction of variables explaining help-seeking behaviour, a path model analysis w...
PMC9831378
Results
Altogether, 1368 participants (
PMC9831378
Conclusion
The
PMC9831378
Trial registration
German Clinical Trials Register: DRKS00023557. Registered 11 December 2020. World Health Organization, Universal Trial Number: U1111–1264-9954. Registered 16 February 2021.
PMC9831378
Supplementary Information
The online version contains supplementary material available at 10.1186/s12889-022-14937-5.
PMC9831378
Keywords
Open Access funding enabled and organized by Projekt DEAL.
PMC9831378
Methods
This study is part of a project funded by the German Research Foundation, with a published study protocol [
PMC9831378
Sample & power analysis
depressive
Participants without current professional treatment and with at least mild depressive symptoms were included (PHQ-9 sum score ≥ 8; 48). In total, Because the path analysis is done in addition to the analyses reported in the study protocol [
PMC9831378
Statistical analysis
REGRESSIONS
No missing scale values are detected within the relevant data. Total drop-out rate was 28.14% between baseline assessment and both follow-ups together (i.e., participation in either three- or six-month follow up). To examine potential reasons for attrition we conducted logistic regressions in which dropout is defined a...
PMC9831378
Results
PMC9831378
Sample characteristics
The final sample size consisted of
PMC9831378
Discussion
The aim of this study was to test a comprehensive The For the prediction of For Regarding superordinate variables, tWe hypothesised associations between
PMC9831378
Limitations
depression, psychiatric
ATTRITION
Our analyses are based exclusively on self-reported data. New instruments yet lacking full psychometric validation were used for continuum beliefs, causal beliefs, self-efficacy measures and treatment experience. However, the new questionnaires are based on existing validated items and therefore should still be interpr...
PMC9831378
Conclusion
In summary, we were able to replicate findings of various influences on the help-seeking process expanding existing research of help-seeking variables and stigma through an intermediary level of subjective illness representations according to the
PMC9831378
Acknowledgements
Not applicable.
PMC9831378
Authors’ contributions
GS, LJP
The manuscript was equally developed and drafted by TM and LJP. TM and LP contributed to the design of the study and the acquisition of the data. TM and LJP analysed and interpreted the data. ST and HoM contributed to the conception of the work. ST, HoM, SiS, and GS revised the draft. GS and SiS manage the project. All...
PMC9831378
Funding
Open Access funding enabled and organized by Projekt DEAL. The study was funded by the DFG (German Research Foundation,
PMC9831378
Availability of data and materials
The datasets generated during the current study are available from the corresponding author on reasonable request.
PMC9831378
Declarations
PMC9831378
Ethics approval and consent to participate
The study procedure was developed in accordance with the Declaration of Helsinki and has been approved by the Ethics Commission of the University Medicine Greifswald (BB 061/18) and Leipzig (514/19-lk). Participants included in the study gave electronic informed consent following comprehensive written information about...
PMC9831378
Consent for publication
Not applicable.
PMC9831378
Competing interests
The authors declare that they have no competing interests.
PMC9831378