ABSTRACT
Background: Biomarker-based therapies are increasingly used in cancer patients outside clinical trials. Systematic assessment of patient-reported outcomes (PRO) is warranted to take patients' perspectives during biomarker-based therapies into consideration. We assessed the feasibility of an electronic PRO assessment via a smartphone application. Methods: An interdisciplinary expert panel developed a smartphone application based on symptom burden and health-related quality of life (HRQoL) metrics reported in a retrospective analysis of 292 neuro-oncological patients. The app included validated assessments of health-related quality of life (HRQoL), the burden of symptoms, and psychological stress. Feasibility and usability were tested in a pilot study. Semi-structured interviews with patients and health care professionals (HCP) were conducted, transcribed, and analyzed according to Mayring´s qualitative content analysis. Furthermore, we assessed compliance and descriptive data of ePROs. Results: A total of 14 patients have been enrolled, (9 female, 5 male). A total of 4 HCPs, 9 patients, and 1 caregiver were interviewed regarding usability/feasibility. The main advantages were the possibility to complete questionnaires at home and comfortable implementation in daily life. Compliance was high, for example, 82% of the weekly distributed NCCN distress thermometer questionnaires were answered on time, however, with interindividual variability. We observed a median distress score of 5 (range 0-10, 197 results, n = 12, weekly assessed) and a median Global health score of 58.3 according to the EORTC QLQ-C30 instrument (range 16.7-100, 77 results, n = 12, monthly assessed). Conclusions: This pilot study proved the feasibility and acceptance of the app. We will therefore expand its application during biomarker-guided therapies to enable systematic PRO assessments.
ABSTRACT
Background: The clinical utility of molecular profiling and targeted therapies for neuro-oncology patients outside of clinical trials is not established. We aimed at investigating feasibility and clinical utility of molecular profiling and targeted therapy in adult patients with advanced tumors in the nervous system within a prospective observational study. Methods: molecular tumor board (MTB)@ZPM (NCT03503149) is a prospective observational precision medicine study for patients with advanced tumors. After inclusion of patients, we performed comprehensive molecular profiling, formulated ranked biomarker-guided therapy recommendations based on consensus by the MTB, and collected prospective clinical outcome data. Results: Here, we present initial data of 661 adult patients with tumors of the nervous system enrolled by December 31, 2021. Of these, 408 patients were presented at the MTB. Molecular-instructed therapy recommendations could be made in 380/408 (93.1%) cases and were prioritized by evidence levels. Therapies were initiated in 86/380 (22.6%) cases until data cutoff. We observed a progression-free survival ratio >1.3 in 31.3% of patients. Conclusions: Our study supports the clinical utility of biomarker-guided therapies for neuro-oncology patients and indicates clinical benefit in a subset of patients. Our data might inform future clinical trials, translational studies, and even clinical care.
ABSTRACT
BACKGROUND: It is the aim of this study to conceptualize a scheme for patellar-height analysis suitable for knees without and with implanted endoprosthesis. Furthermore, patellar-height and change of patellar-height by total-knee-arthroplasty (TKA) should be studied and correlated with clinical outcomes. We hypothesize that not only True-Patella-Infera (TPI) but also Pseudo-Patella-Infera (PPI) might be correlated with elevated anterior-knee-pain and reduced range-of-motion. METHODS: Several patellar-height indices were measured by three independent raters in two passes. Indices were analyzed and intra- and interobserver agreements were determined. For radiographic and clinical evaluation of 92 knee arthroplasties, the Insall-Salvati Index (ISI) and the Caton-Deschamps Index (CDI) were used to determine patellar-height and especially the amounts of TPI and PPI. Patellar-height was correlated with clinical outcome parameters: pain, ROM, Oxford Knee Score, Knee Society Score, Kujala Score, SF-36, Tegner and Lysholm. RESULTS: All used patellar-height indices demonstrated good intra- and interobserver agreement. Low preoperative ROM was correlated with low postoperative ROM after primary TKA. Postoperatively, 2.2% of the patients had a TPI and 9.8% had a PPI. Patella infera was not correlated with reduced subjective or objective clinical outcome parameters. CONCLUSIONS: Preoperative ROM is a good predictor of postoperative ROM after primary TKA. Patellar-height analysis can be sufficiently performed using the ISI and the CDI, respectively the derived CDI (dCDI) in cases with implanted endoprosthesis. As an alternative to the CDI/dCDI, the BPI/mBPI can be used. There seems to be a certain threshold of acceptable patellar-height reduction, in which clinical outcome is not deteriorated.
ABSTRACT
PURPOSE: The original Caton-Deschamps index (oCDI) detects functional patella height. It cannot be used in knees with an implanted endoprosthesis. The "modified Caton-Deschamps index" (mCDI) for knee arthroplasty can miss pseudo-patella-infera (PPI), which is common after TKA. A derivate of the oCDI could be a simple analogue to the index published in 1982 using a modified tibial reference point at the anterior proximal point of the inlay, which can indirectly be located on the lateral knee radiograph. It was the aim of this study to determine the intra- and inter-rater agreement of a derived Caton-Deschamps index (dCDI) for knee arthroplasty. We hypothesized that the derived Caton-Deschamps index (dCDI) is a reliable radiological measure for patella height in knee arthroplasty. METHODS: Several patella height indices were measured by three independent raters in two passes. The second pass was performed after 6 weeks in random order. Intra- and inter-observer agreements were determined and analyzed using the intraclass correlation coefficient (ICC). For radiographic evaluation, digital lateral radiographs of 150 knees before and after primary TKA were used. RESULTS: We found high interrater reliability for all analyzed indices. We found the highest agreements for the ISI preop (ICC = 0.914) and postop (ICC = 0.920), respectively. We also found very good intra-rater reliability for the CDI (ICCpreop = 0.954), dCDI (ICCpostop = 0.945), ISI (ICCpreop = 0.960; ICCpostop=0.940) and BPI (ICCpreop = 0.969; ICCpostop = 0.955). Fourteen cases (9.3%) with insignificant PPI were found. CONCLUSION: The derived Caton-Deschamps index (dCDI) can easily be used in knee arthroplasty and demonstrated high intra- and interrater agreement, which was similar to other commonly used and established patella height indices.
