Cross-cultural adaptation and validation of a self-reporting tool to assess health-related quality of life for Egyptians with extremity bone sarcomas in childhood or adolescence.

BACKGROUND: Validated self-reporting tools are required to evaluate the functional outcome and health-related quality of life (HRQOL) for those who had extremity bone sarcomas in their childhood or adolescence. Our study pursued cross-cultural adaptation and validation of the pediatric Toronto Extremity Salvage Score (pTESS) and Toronto Extremity Salvage Score (TESS) to assess the functional outcome for Egyptian children and adult survivors following surgeries of extremity bone sarcomas. In the modified versions of pTESS and TESS, mental domains were added to allow the evaluation of HRQOL using a specific instrument for childhood bone cancer. METHODS: The internal consistency and test-retest reliability of the studied forms were assessed with Cronbach's alpha and Intra-class coefficients (ICC), respectively. For convergent validity, correlations between scores of the generic Pediatric Quality of Life Inventory (PedsQL 4.0) and pTESS /TESS scores were reported. Factor Analysis was feasible for pTESS-leg; due to the insufficient samples, only the average inter-item correlation coefficients were reported for the remaining versions. RESULTS: Out of 233 participants, 134 responded to pTESS-leg, 53 to TESS-leg, 36 to pTESS-arm, and only 10 to TESS-arm. All versions showed excellent internal consistency (Cronbach's alpha >0.9), good test-retest reliability (ICC >0.8), moderate to strong correlations with PedsQL, and acceptable average inter-item correlation coefficients (≥0.3). Three factors were extracted for the pTESS-leg, in which all mental items were loaded on one separate factor with factor loadings exceeding 0.4. Active chemotherapy, less than one year from primary surgery, or tibial tumors were associated with significantly inferior pTESS/TESS scores in the lower extremity group. CONCLUSION: The Egyptian pTESS and TESS are valid and reliable self-reporting tools for assessing the functional outcome following surgeries for extremity bone sarcomas. The modified pTESS and TESS versions, which include additional mental domains, enabled the assessment of the overall health status of our population. Future studies should include a larger sample size and evaluate the ability of pTESS/TESS to track progress over time.

Efficacy of different salvage regimens in progressive unresectable pediatric low-grade glioma.

Multiple salvage chemotherapy regimens are used in progressive low-grade glioma (LGG), with no single regimen being more effective than any other. In the present study, different salvage therapies were compared with regard to the response rate, overall survival (OS) rate, event-free survival (EFS) rate and visual outcome in 70 patients with pediatric LGG. Age was found to significantly affect the EFS and OS rates (P<0.001). The visual outcome was the same between the three regimens. The 2-year EFS and OS rates of the vincristine/carboplatin, monthly carboplatin and weekly vinblastine regimens were 64.7 and 70.6%, 71.0 and 85.0%, and 56.0 and 62.7%, respectively (P=0.6 for EFS; P=0.56 for OS). Overall, the present study demonstrated that age had a significant impact on survival. The three salvage regimens used were equally effective with regard to the radiological response and visual outcome. However, further randomized controlled trials are required to detect the optimal salvage therapy.

MicroRNA childhood cancer catalog (M3Cs): a resource for translational bioinformatics toward health informatics in pediatric cancer.

MicroRNA childhood Cancer Catalog (M3Cs) is a high-quality curated collection of published miRNA research studies on 16 pediatric cancer diseases. M3Cs scope was based on two approaches: data-driven clinical significance and data-driven human pediatric cell line models. Based on the translational bioinformatics spectrum, the main objective of this study is to bring miRNA research into clinical significance in both pediatric cancer patient care and drug discovery toward health informatics in childhood cancer. M3Cs development passed through three phases: 1. Literature Mining: It includes external database search and screening. 2. Data processing that includes three steps: (a) Data Extraction, (b) Data Curation and annotation, (c) Web Development. 3. Publishing: Shinyapps.io was used as a web interface for the deployment of M3Cs. M3Cs is now available online and can be accessed through https://m3cs.shinyapps.io/M3Cs/. For data-driven clinical significance approach, 538 miRNAs from 268 publications were reported in the clinical domain while 7 miRNAs from 5 publications were reported in the clinical & drug domain. For data-driven human pediatric cell line models approach, 538 miRNAs from 1268 publications were reported in the cell line domain while 211 miRNAs from 177 publications in the cell line & drug domain. M3Cs acted to fill the gap by applying translational bioinformatics general pathway to transfer data-driven research toward data-driven clinical care and/or hypothesis generation. Aggregated and well-curated data of M3Cs will enable stakeholders in health care to incorporate miRNA in the clinical policy. Database URL:https://m3cs.shinyapps.io/M3Cs/.