The current state of understanding of oncology expanded access programs in Malaysia.

INTRODUCTION: An expanded access program (EAP) is a regulatory mechanism that provides access to an investigational drug, which is not approved for use, in treating life-threatening conditions when all the standard-ofcare treatments are exhausted. MATERIALS AND METHODS: An online, anonymous, voluntary survey was conducted to assess the level of knowledge and understanding about EAPs among Malaysian oncologists using SurveyMonkey® between April 2020 and June 2020. Oncologists who had enquired about EAP in the past, were invited at random to participate in the survey. Participants who did not provide consent or failed to complete the survey were excluded. RESULTS: A total of 15 oncologists participated in the survey, from both public (46.6%) and private (46.6%) practices. Most respondents (80%) had filed between 1 to 10 EAP applications in the past 12 months. For 73.3% respondents, resources or training were not provided for EAPs from institutions. Around 53% of the respondents reported that their knowledge of EAPs and application processes including country regulations is 'good'. The majority of respondents (73.3%) reported that the educational modules on an overview of EAPs, country regulations and the EAP application process will be beneficial. Most participants received information about the existing EAPs either by reaching out to a pharmaceutical sponsor or through another health care provider and some received information about the existing EAPs through their institutions or patients/caregivers. Most of the respondents recommended that pharmaceutical companies should have readily available information related to the availability and application of EAPs for all pipeline products on their websites. DISCUSSION: EAPs are crucial treatment access pathways to provide investigational drugs to patients who have exhausted their treatment options and are not eligible for participation in clinical trials. Malaysian oncologists have a fair understanding about the EAPs and the application processes. CONCLUSION: Additional training and awareness are needed for Malaysian oncologists to upscale the utilisation of EAPs.

MicroRNA expression profile of a Malaysian Bajau family with familial mitochondrial neurogastrointestinal encephalomyopathy.

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a rare, autosomal recessive disorder associated with mutations in the thymidine phosphorylase (TYMP) gene. The main objective of this study was to characterize the genetic profiles of the deceased proband's family members (N = 4) using DNA sequencing and to determine miRNA deregulation in MNGIE using miRNA microarray profiling and bioinformatic analysis. We found that the genetic profile of the younger sister showed similar TYMP gene mutations as that of the proband with the exception of a heterozygous mutation in exon 10. The miRNA microarray revealed 55 significantly up-regulated and 65 significantly down-regulated miRNAs. These miRNAs have been implicated in various mitochondrial dynamics such as energy metabolism, Krebs cycle, mitochondria-associated apoptosis, and mitophagy. In conclusion, we demonstrate that blood miRNAs are deregulated in the pathogenesis of MNGIE and these changes may have therapeutic implications. Further experimental studies will be required to elucidate the functional miRNA-mRNA interactions in MNGIE.

A comparative study of the learning style preferences of students with learning disabilities and students who are gifted.

The purpose of this study was to investigate whether significant group, gender, and grade differences existed in the learning style preferences of students with learning disabilities and students who are gifted, using the Learning Style Inventory (LSI) (Dunn, Dunn, & Price, 1987). Subjects consisted of 53 students with learning disabilities and 64 gifted students randomly selected from two large public high schools. They were 10th-, 11th-, and 12th-grade students, aged 15 to 17. A three-way analysis of variance on the LSI raw scores revealed significant group differences in preferences for light, design, and kinesthetic modality, and in motivation, persistence, responsibility, parent motivation, and teacher motivation. Significant gender and grade differences were found in preferences for mobility and learning in the afternoon, respectively. A stepwise discriminant analysis revealed six variables that significantly discriminated between the two groups. Findings imply that incorporating the learning styles of students with learning disabilities and students who are gifted is important for individualizing educational programming.

Comparison of self-concepts of students identified as gifted and regular students.

The purpose of the present study was to compare the self-concepts of 40 eighth grade students identified as gifted and 40 regular eighth grade students using the Piers-Harris Children's Self-concept Scale. A t test indicated a significant group difference on the behavior component only and no significant differences by gender. More similarities than differences were noted in self-concepts of these students identified as gifted and regular students.