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BackgroundA growing number of cancer and hematopoietic stem cell transplant (HSCT) survivors require long-term follow-up with optimal communication schemes, and patients' compliance is crucial. Adolescents have various unmet needs. Regarding self-report of symptoms and health status, users of mobile apps showed enhanced compliance. Currently, HSCT aftercare at the HSCT outpatient clinic of the St. Anna Children’s Hospital in Vienna, Austria, is based on handwritten diaries, carrying various disadvantages. Recently, we developed the prototype of a web-based, self-monitoring gamified mobile app tailored for adolescents: the INTERACCT (Integrating Entertainment and Reaction Assessment into Child Cancer Therapy) app. ObjectiveThis observational, prospective study evaluated the usability of the INTERACCT app for tracking real-time self-reported symptoms and health status data in adolescent HSCT patients and a healthy matched control group. The primary outcome of the study was the quality of the self-reported medical information. We hypothesized that the mobile app would provide superior medical information for the clinicians than would the handwritten diaries. MethodsHealth data were reported via paper diary and mobile app for 5 consecutive days each. The quality of medical information was rated on a 5-point scale independently and blinded by two HSCT clinicians, and the duration of use was evaluated. A total of 52 participant questionnaires were assessed for gaming patterns and device preferences, self-efficacy, users’ satisfaction, acceptability, and suggestions for improvement of the mobile app. Interrater reliability was calculated with the intraclass correlation coefficient, based on a two-way mixed model; one-way repeated-measures analysis of variance and t tests were conducted post hoc. Descriptive methods were used for correlation with participants’ demographics. For users’ satisfaction and acceptability of the mobile app, the median and the IQR were calculated. ResultsData from 42 participants—15 patients and 27 healthy students—with comparable demographics were evaluated. The results of our study indicated a superiority of the quality of self-reported medical data in the INTERACCT app over traditional paper-and-pencil assessment (mobile app: 4.14 points, vs paper-based diary: 3.77 points, P=.02). The mobile app outperformed paper-and-pencil assessments mainly among the patients, in particular among patients with treatment-associated complications (mobile app: 4.43 points, vs paper-based diary: 3.73 points, P=.01). The mobile app was used significantly longer by adolescents (≥14 years: 4.57 days, vs ≤13 years: 3.14 days, P=.03) and females (4.76 days for females vs 2.95 days for males, P=.004). This corresponds with a longer duration of use among impaired patients with comorbidities. User satisfaction and acceptability ratings for the mobile app were high across all groups, but adherence to entering a large amount of data decreased over time. Based on our results, we developed a case vignette of the target group. ConclusionsOur study was the first to show that the quality of patient-reported medical information submitted via the INTERACCT app embedded in a serious game is superior to that submitted via a handwritten diary. In light of these results, a refinement of the mobile app supported by a machine learning approach is planned within an international research project.

Within the last few decades, we have witnessed tremendous advancements in the study of pediatric low-grade gliomas (pLGG), leading to a much-improved understanding of their molecular underpinnings. Consequently, we have achieved successful milestones in developing and implementing targeted therapeutic agents for treating these tumors. However, the community continues to face many unknowns when it comes to the most effective clinical implementation of these novel targeted inhibitors or combinations thereof. Questions encompassing optimal dosing strategies, treatment duration, methods for assessing clinical efficacy, and the identification of predictive biomarkers remain unresolved. Here, we offer the consensus of the international pLGG coalition (iPLGGc) clinical trial working group on these important topics and comment on clinical trial design and endpoint rationale. Throughout, we seek to standardize the global approach to early clinical trials (phase I and II) for pLGG, leading to more consistently interpretable results as well as enhancing the pace of novel therapy development and encouraging an increased focus on functional endpoints as well and quality of life for children faced with this disease.

Background: We report on the characterization and introduction of a novel prognostic score for patients undergoing stereotactic body radiotherapy (SBRT) for the treatment of single and multiple pulmonary metastases (PMs) derived from head and neck cancer (HNC). Methods: In this retrospective study, we examined selected factors associated with progression‐free survival (PFS) and overall survival (OS) among 59 patients with HNC treated with SBRT for a total of 118 PMs, between 2009 and 2023. Factors related to survival were included in the prognostic scoring system. Results: Prognostic factors including histology, age, number of metastases, and performance status at first SBRT were weighted differently depending on the strength of correlation to PFS and OS. Total prognostic scores (HAMP) ranged from 13 to 24 points, with a cut‐off total score of ≤18 scoring points for patients in a high‐risk (HR) subcohort, and of ≥19 scoring points for patients in a low‐risk group (LR). Median PFS (23.8 vs. 5.5 months, p < 0.001) and OS (61.3 vs. 16.4 months, p < 0.001) were significantly longer in the low‐risk group compared to the high‐risk group. Conclusion: The HAMP score might be a convenient tool to facilitate individualized treatment decisions and appropriate follow‐up. The accuracy and reliability of the score requires further evaluation in prospective studies. [ABSTRACT FROM AUTHOR]

Aim: Associations between serum biomarkers S100 calcium‐binding protein B (S100B) and neuron‐specific enolase (NSE) in neonates with hypoxic‐ischaemic encephalopathy (HIE) offer contradicting data in regard to neurocognitive outcome. The aim of our study was to provide another dataset to answer this question if S100B or NSE correlates to outcome in neonatal HIE. In addition, we investigate whether amplitude‐integrated EEG (aEEG) or magnetic resonance imaging (MRI) also has predictive value. Methods: In neonates with HIE born in Vorarlberg, Austria, (n = 34) from 2012to 2020, NSE and S100B serum levels were measured on day one. aEEG was installed at admission and MRI performed within 7 days. Surviving children (n = 27) were categorised as good or poor outcome by using an age‐appropriate neurocognitive test and a standardised neurological follow‐up. Positive and negative predictive values and receiver operating characteristic curves were calculated to evaluate the prognostic value. Results: S100B showed best positive and negative predictive value, 72.7% and 90.5%, respectively, and a significant area under the curve of 0.820. NSE showed a positive and a negative predictive value of 43.8% and 81.3% and an area under the curve of 0.757. Severely abnormal aEEG and abnormal MRI significantly correlated with outcome (p = 0.024 and 0.001 respectively). Conclusion: S100B and NSE on day one, severely abnormal aEEG and abnormal MRI show a significant correlation and good predictive value for neurocognitive outcome. [ABSTRACT FROM AUTHOR]

Few high-quality instruments are available to comprehensively screen for multiple interpersonal traumatic childhood experiences differentiated by developmental stage. One instrument assessing various forms of childhood trauma is the Maltreatment and Abuse Chronology of Exposure (MACE-X, Teicher & Parigger, 2015), which allows for the assessment of age at exposure for every item. The aim of this study is the development of a shorter version, which is suitable for screening. In- and out-patients (n = 719) of a somatic clinic were assessed with the German version of the 75-item MACE. Descriptive statistics, a nonparametric item response theory model (Mokken Scale Analysis), and factor analysis were combined to select items for a screening version. A screener (MACE-SCREEN) with 39 items allocated to five scales was developed. Scales differentiate between abuse types and neglect, direct experience vs. witnessing violence, and the age of the perpetrator (adults vs. peers). The scales showed good internal consistency (Alpha.78 to.91) and the total score a strong correlation with the total score of the long version (r =.96). This screening version is still comprehensive as it assesses exposure to various forms of interpersonal traumatic events, including peer violence and witnessing domestic abuse. As it also allows for the concurrent assessment of age at exposure, it lends itself to assessing exposure across developmental stages. [ABSTRACT FROM AUTHOR]

Background: Adverse childhood experiences (ACEs) are associated with posttraumatic and complex posttraumatic stress disorder symptoms in adulthood (PTSD/cPTSD), as well as reduced epistemic trust (trust in the authenticity and personal relevance of interpersonally transmitted information) and impaired personality functioning. The present work aims to investigate the predictive value of epistemic trust--the capacity for social learning--on the mediating effect of personality functioning in the association of ACEs and PTSD/cPTSD. Methods: We conducted structural equation modeling (SEM) based on representative data of the German population (N = 2,004). Personality functioning (OPD-SQS) was applied as a mediator between ACEs and PTSD/cPTSD (ITQ), while epistemic trust (ETMCQ) was added as predictor for OPD-SQS. TLI, CFI, and RMSEA (95%-CI) determined the models' fit. Results: N = 477 (23.8%) participants reported at least one ACE and n = 218 (10.9%) reported ≥4 ACEs. Fit indices were good for both PTSD (TLI = 0.96; CFI = 0.99; RMSEA = 0.06; 95%CI: 0.041-0.078) and cPTSD (TLI = 0.96; CFI = 0.99; RMSEA = 0.06; 95%CI: 0.043-0.081). ACEs were significantly associated with cPTSD (b = 0.44, p < 0.001) and PTSD (b = 0.29, p < 0.001), explaining 20 and 8% of its variance. Adding personality functioning as a mediator increased the explained variance of cPTSD and PTSD to 47 and 19% while the direct association between ACEs and cPTSD/PTSD decreased (b = 0.21/b = 0.17), thus, indicating a partial mediation. Including epistemic trust substantially increased the explained variance for personality functioning (41%) compared to ACEs as a single predictor (16%). Conclusion: We add to previous research emphasizing the association between ACEs and PTSD/cPTSD symptoms. Offering insights on underlying mechanisms, we show that epistemic trust and personality functioning are relevant mediators. Since both are modifiable by psychotherapy, knowledge about the role of these constructs can inform research on psychotherapeutic interventions and prevention. [ABSTRACT FROM AUTHOR]