Consensus recommendations on management of selumetinib-associated adverse events in pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas.

Background: Selumetinib is the first approved treatment for pediatric patients with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN) in the EU and US, as well as in multiple other countries. Evidence for the management of selumetinib-associated adverse events (AEs) is mostly limited to clinical trials and expanded-access programs. We gathered a panel of European healthcare practitioners with clinical experience prescribing selumetinib and/or managing pediatric patients with NF1-PN to provide recommendations on the prevention and management of AEs. Methods: A modified Delphi approach was used to develop the recommendations among the group of experts. Initial statements were developed from a literature review of current management recommendations and regulatory reports. The panel refined the statements and rated the extent to which they agreed with them in 2 sessions and a follow-up survey. The panel comprised 2 pediatric neuro-oncologists, 1 pediatric oncologist, 1 pediatrician, 1 neuropediatrician, 1 oncologist, 1 neurologist, 2 psychologists, and 1 dermatologist. Results: The experts agreed on the relative frequency and impact of AEs potentially associated with selumetinib. Consensus-level agreement was reached for 36 statements regarding the prevention and management of AEs potentially associated with selumetinib. Experts recommended treatments for AEs based on their experience. Conclusions: The development of a variety of consensus statements indicates expert agreement on best practices for the prevention and management of AEs potentially associated with selumetinib in pediatric patients with NF1-PN. These events are generally manageable and should be considered alongside treatment benefit. Information sharing is warranted as further experience is gained.

Preparing Children for Invasive Medical Cancer Treatment with "My Logbook": Preliminary Results of a Pilot Study.

Pediatric cancer is one of the most burdensome chronic diseases, necessitating a variety of severe medical interventions. As a result, the disease and its treatment cause numerous acute and long-term medical, psychological, and socioeconomic strains for young patients and their families. Therefore, psychosocial care using evidence-based interventions (EBIs) before, during, and after medical treatments is essential to ensure that patients receive adequate information and to minimize the adverse emotional and psychosocial impacts such as insecurity, fear, and shame. The present study reports the first promising results of applying cancer-specific psychosocial methods developed in the quality improvement project "My Logbook." The four assessed tools are specifically designed to adequately prepare pediatric cancer patients for surgery, chemotherapy, radiotherapy, and stem cell transplantation. Self and proxy ratings were used to assess the patients' subjective knowledge and emotional well-being before and after each intervention session. The results showed that patient-centered interventions using various creative and developmentally adapted methodologies (e.g., psychoeducation, crafting, games) have the potential to effectively enhance patient health literacy (V = 120.5, p < .001, r = 0.33) and well-being as manifested in more positive (slope = 0.121, p = .016) and less negative (slope = - 0.350, p < .001) or neutral emotions (slope = - 0.202, p = .002). These findings highlight the importance of developing and implementing psychosocial tools in pediatric oncology to prevent psychological overload and negative emotions and to increase subjective control beliefs, autonomy, and empowerment. Moreover, the effective application and systematic evaluation of evidence-based psychosocial tools can facilitate the establishment of standardized guidelines for psychosocial care in pediatric oncology. Thereby, the final goal is to ensure the quality of care and to use education to increase the quality of life for all pediatric cancer patients.Trial registration: ClinicalTrials.gov Identifier: NCT04474678 (July 17, 2020).

The oracle of D-A-Ch - Results of a Delphi Survey for the development of the evidence- and consensus-based tool "My Logbook".

OBJECTIVE: The present report describes the results of four delphi surveys conducted within the quality improvement project "My Logbook" which aims to translate evidence-based standards for psychosocial care in pediatric oncology into a practical consensus-based tool. METHODS: In four consecutive delphi surveys a total of n=153 international, multi-disciplinary experts rated the content, method, and design of the different booklets of "My Logbook" which a local expert group had conceptualized. After each survey, the feedback was incorporated, and the changes were evaluated in a final consensus vote by the quality assurance panel of the PSAPOH. RESULTS: While some surveys led to a review on a page level, most booklets as a whole reached the consensus-level of approval. Over the course of the surveys, any revisions and comments were incorporated in the booklets, and approval rates increased steadily. DISCUSSION: The delphi surveys ensured the integration of multi-disciplinary, international expertise, uncovering issues such as language barriers and the need for a user manual that would not have been evident in first line . The incorporation of the input led to a continuous improvement of the tool, reflected in steadily increasing acceptance rates in the consecutive survey rounds. CONCLUSION: The incorporation of the expert input as well as the additional development of a user manual resulted in a final version of the "My Logbook" apt for the interdisciplinary application in pediatric oncology in the entire DACH-region.

Bridging the gap: A quality improvement project to implement psychosocial care standards into clinical practice in pediatric oncology.

BACKGROUND: Psychosocial guidelines and standards systematically describe stressors and resources in particularly challenging situations and hence serve as a basis for interventions to achieve defined psychosocial goals. Despite fundamental principles and guidelines for psychosocial methods, the quality of provided care varies considerably depending on setting, provision, and profession. The purpose of the present protocol is to illustrate the development and evaluation of the standardized psychological intervention "My Logbook", a practical guide accompanying children through all stages of treatment by directly translating current quality standards of psychosocial care into practice. METHODS: In an evidence-based set-up, using face-to-face discussions and telephone conferences, a multi-professional team of local experts decide on critical disease-related issues, structure, content (information and intervention elements) and design of the quality improvement tool. Via delphi surveys an extended expert team is asked to rate the content, method, and design of all booklets which is concluded by a final agreement by the specialist group for quality assurance of the psychosocial working group in the Society for Pediatric Oncology (PSAPOH). The developed tools are piloted in an international multicenter study to evaluate the patient-reported outcome and feasibility and to integrate practical views of patients, as well as psychosocial and interdisciplinary professionals into the further development of the "My Logbook". DISCUSSION: The iterative development of the "My Logbook" including local and international experts as well as the patient and practical perspective allow for the design of a process-oriented, consensus - and evidence-based tool directly translating the S3-Guideline into clinical practice. Feasibility and applicability are fostered through an iterative process of constant evaluation and adaptation of the tool by international experts and through the clinical experience gathered in the multi-centered pilot study. Furthermore, the systematic evaluation of the tool by patients, psychosocial, and interdisciplinary professionals enables the identification of persisting gaps between evidence-based standards and clinical practice, discrepancies between the various stakeholders' perspectives as well as regional differences in feasibility, thereby directly linking practice and research. The preliminary results emphasize that psychological support can be standardized, enabling an evaluation and optimization of psychosocial care which future studies need to assess in multicenter clinical randomized controlled trials.

Do we still need IQ-scores? Misleading interpretations of neurocognitive outcome in pediatric patients with medulloblastoma: a retrospective study.

Over the past decades, many studies used global outcome measures like the IQ when reporting cognitive outcome of pediatric brain tumor patients, assuming that intelligence is a singular and homogeneous construct. In contrast, especially in clinical neuropsychology, the assessment and interpretation of distinct neurocognitive domains emerged as standard. By definition, the full scale IQ (FIQ) is a score attempting to measure intelligence. It is established by calculating the average performance of a number of subtests. Therefore, FIQ depends on the subtests that are used and the influence neurocognitive functions have on these performances. Consequently, the present study investigated the impact of neuropsychological domains on the singular "g-factor" concept and analysed the consequences for interpretation of clinical outcome. The sample consisted of 37 pediatric patients with medulloblastoma, assessed 0-3 years after diagnosis with the Wechsler Intelligence Scales. Information processing speed and visuomotor function were measured by the Trailmaking Test, Form A. Our findings indicate that FIQ was considerably impacted by processing speed and visuomotor coordination, which leaded to an underestimation of the general cognitive performance of many patients. One year after diagnosis, when patients showed the largest norm-deviation, this effect seemed to be at its peak. As already recommended in international guidelines, a comprehensive neuropsychological test battery is necessary to fully understand cognitive outcome. If IQ-tests are used, a detailed subtest analysis with respect to the impact of processing speed seems essential. Otherwise patients may be at risk for wrong decision making, especially in educational guidance.