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Ronja Thallner,1 Christoph Gumbinger,1 Anja Hohmann,1 Antje Wick,1 Wolfgang Wick,1 Loraine Busetto1,2 1Department of Neurology, Heidelberg University Hospital, Heidelberg, Germany; 2Institute for Medical Virology, Goethe University Frankfurt, University Hospital, Frankfurt am Main, GermanyCorrespondence: Loraine Busetto, Institute for Medical Virology, Goethe University Frankfurt, University Hospital, Paul-Ehrlich-Straße 40, Frankfurt am Main, 60590, Germany, Tel/Fax +49 69 6301-86103, Email busetto@med.uni-frankfurt.dePurpose: There is a lack of evidence regarding how patients with malignant brain tumor and their relatives experience participation in neurooncological clinical trials. Similarly, insights from the perspective of trial staff caring for this group of patients are missing. This study aims to investigate patient, relative and trial staff experiences regarding participation in clinical neurooncological trials.Methods: Within a qualitative exploratory study, 29 semi-structured interviews with brain tumor patients, relatives and trial staff were conducted and analyzed using reflexive thematic analysis (RTA) by Braun and Clarke. A patient researcher and patient council were involved in data analysis and interpretation.Results: Four themes were developed reflecting significant aspects of the trial experience: 1. “It all revolves around hope”; 2. “Trial participation: experiencing unique medical care”; 3. “Everyone’s roles are changing”; 4. “Communication as a possible area of conflict”. Experiencing trial participation and general medical treatment were found to be interconnected to such a degree that they were often not meaningfully distinguished by patients and relatives.Conclusion: In addition to assessing traditional endpoints for patient outcomes, we recommend increased emphasis on investigating the impact of the “soft” components constituting trial participation. Due to the interconnectedness of medical treatment and trial participation, we recommend further investigation in comparison to experiences in regular care. A deeper understanding of trial participation is needed to inform improvements for patient experiences and staff satisfaction alongside medical and scientific progress.Plain Language Summary: The treatment options available to patients with (malignant) brain tumors are currently very limited. Therefore, patients are sometimes offered to participate in a clinical trial. This means that they receive an experimental treatment (eg new medicine) for which it is not yet clear whether it works better than regular medical care. Currently, little is known about how this group of patients, their relatives and the hospital staff who care for them experience the participation in these clinical trials – which is what we aimed to explore in our study reported here.Based on interviews with patients, relatives and staff, we found that:trial participation mainly revolves around hope;trial participation entails experiencing unique medical care;trial participation significantly changes the previous roles of patients, relatives and staff;trial participation intensifies communication as a possible area of conflict.By providing information on how patients, relatives and staff make sense of their trial experiences, this study constitutes an important addition to the traditional focus of clinical trials on medical and scientific endpoints (eg progression-free survival). This may help clinicians and researchers involved in cancer research and treatment to understand why “unsuccessful” trials can still be perceived as positive by patients or how hopeful communication may support their patients even when perceived as “unrealistic” from the clinicians’ perspective. An in depth understanding of trial participation from the perspective of those affected is needed for improved care experiences alongside medical and scientific progress for cancer treatment.Keywords: cancer research, brain tumor, qualitative research, care experiences

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Background and purpose: We quantified peripheral nerve lesions in adults with 5q‐linked spinal muscular atrophy (SMA) type 3 by analysing the magnetization transfer ratio (MTR) of the sciatic nerve, and tested its potential as a novel biomarker for macromolecular changes. Methods: Eighteen adults with SMA 3 (50% SMA 3a, 50% SMA 3b) and 18 age‐/sex‐matched healthy controls prospectively underwent magnetization transfer contrast imaging in a 3‐Tesla magnetic resonance scanner. Two axial three‐dimensional gradient echo sequences, with and without an off‐resonance saturation rapid frequency pulse, were performed at the right distal thigh. Sciatic nerve regions of interest were manually traced on 10 consecutive axial slices in the images generated without off‐resonance saturation, and then transferred to corresponding slices generated by the sequence with the off‐resonance saturation pulse. Subsequently, MTR and cross‐sectional areas (CSAs) of the sciatic nerve were analysed. In addition, detailed neurologic, physiotherapeutic and electrophysiologic examinations were conducted in all patients. Results: Sciatic nerve MTR and CSA reliably differentiated between healthy controls and SMA 3, 3a or 3b. MTR was lower in the SMA 3 (P < 0.0001), SMA 3a (P < 0.0001) and SMA 3b groups (P = 0.0020) than in respective controls. In patients with SMA 3, MTR correlated with all clinical scores, and arm nerve compound motor action potentials (CMAPs). CSA was lower in the SMA 3 (P < 0.0001), SMA 3a (P < 0.0001) and SMA 3b groups (P = 0.0006) than in controls, but did not correlate with clinical scores or electrophysiologic results. Conclusions: Magnetization transfer ratio is a novel imaging marker that quantifies macromolecular nerve changes in SMA 3, and positively correlates with clinical scores and CMAPs. [ABSTRACT FROM AUTHOR]

Background and purpose: Although patient‐centredness is considered a key component of high‐quality neurological care, it is unclear to what extent it can or should be implemented during the acute phase. Using acute stroke as an example, the aim was to identify critical junctures for patient‐centredness along the acute care pathway from the perspectives of patients, relatives and staff. Methods: A qualitative multi‐method study was conducted including 27 non‐participant observations and 37 semi‐structured interviews with patients, relatives and staff. Junctures were defined as critical when mentioned (as problematic) in two or three information sources (i.e. observations, staff interviews, or patient and relative interviews), as potentially critical when mentioned in one, and as uncritical when not mentioned. Results: Post‐procedure communication after thrombectomy, patients' stay at the stroke unit and decision‐making around transfer, discharge and rehabilitation were identified as critical junctures for patient‐centredness. Arrival at the emergency department and the (thrombectomy) treatment itself were identified as uncritical junctures, whilst history‐taking and treatment preparation, the treatment decision and patients' stay at the intensive care unit were identified as potentially critical junctures. Conclusions: In acute stroke care, patients, relatives and staff prioritize fast over patient‐centred decision‐making in the most time‐critical phases, especially before and during treatment. This is reversed after the procedure, when difficulties arise implementing a patient‐centred approach in clinical practice. To improve patient‐centredness where it is most needed, clear guidelines and accessible resources are recommended. Future research should investigate whether insights from acute phases of stroke care are applicable to other neurological conditions as well. [ABSTRACT FROM AUTHOR]

Aims: Mutations of isocitrate dehydrogenase (IDH)1/2 affect almost all astrocytomas of WHO grade II and III. A subset of IDH‐mutant astrocytic tumours progresses to IDH‐mutant glioblastoma or presents with the histology of a glioblastoma at first presentation. We set out here to assess the molecular spectrum of IDH‐mutant glioblastomas. Methods: We performed an integrated molecular analysis of a mono‐centric cohort (n = 97); assessed through genome‐wide DNA methylation analysis, copy‐number profiling and targeted next generation sequencing using a neurooncology‐tailored gene panel. Results: Of these 97 IDH‐mutant glioblastomas, 68 had a glioblastoma at first presentation ('de novo' IDH‐mutant glioblastoma) and 29 emerged from a prior low‐grade lesion ('evolved' IDH‐mutant glioblastoma). Unsupervised hierarchical clustering of DNA methylation data disclosed that IDH‐mutant glioblastoma ('de novo' and 'evolved') formed a distinct group separate from other diffuse glioma subtypes. Homozygous deletions of CDKN2A/B were found to be associated with shorter survival. Conclusions: This study demonstrates DNA methylation patterns in IDH‐mutant glioblastoma to be distinct from lower‐grade astrocytic counterparts but homogeneous within de novo and evolved IDH‐mutant glioblastomas, and identifies CDKN2A as a marker for possible genetic sub‐stratification. [ABSTRACT FROM AUTHOR]

Copyright of Best Practice Onkologie is the property of Springer Nature and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)