Goldenberg, Neil A.

Introduction Clinical trials are the avenue whereby the effects of interventions on health-related outcomes are evaluated. It is imperative that these trials are diverse and inclusive to reduce disparities in overall health outcomes as individuals can experience the same disease differently. When populations are not underrepresented in clinical trials, the results are not generalizable. Despite efforts to facilitate diverse representation across clinical trials, including guidance from the United States Food and Drug Administration, gaps persist in regard to understanding approaches to achieve this goal. With this in mind, we designed an international survey-based research study with the primary objectives to understand current tools and tactics investigators are employing in their clinical research and to identify gaps to ensure diversity, equity and inclusion (DEI). Methods This study was approved through the Institutional Review Board at the University of Alabama at Birmingham. The survey was developed using QuestionPro software (Austin, TX, USA). The target population for this study was the members of the American Society of Hematology (ASH) along with individuals who attended or were presenting authors at the 2023 ASH Annual Meeting, and individuals on the “This Week in Blood email list.” Surveys were distributed via email to potential participants between April and July 2024. Reminder emails were sent on 2 occasions. The survey was also advertised via various social media platforms (e.g., Twitter, Instagram, Facebook and LinkedIn). No incentives were provided for completion of the survey. In the introductory email, participants were assured of the anonymous nature of the survey and were instructed to proceed only if they consented to participate voluntarily in this research study. Results The survey was viewed by 5171 individuals. There were 774 total responses, for a response rate of 15%. Complete responses were provided by 502 respondents. Fifty-seven percent of survey respondents were based in the United States and 56% were ASH members. Seventy-one percent of respondents were clinicians, of which the majority (74%) were primarily adult providers and 86% reported hematology as their clinical specialty, a large proportion of whom were malignant hematologists. Regarding clinical research involvement over the last 5 years, 29% identified as sub investigators, 23% were site principal investigators (PI) in multicenter studies, and 19% reported roles as overall PI for studies. Respondents reported employing tactics to enhance DEI in all phases of prospective clinical research studies. Nineteen percent stated that they engage patients in the research planning phase, in protocol design, review and revision. During the implementation phase, 21% reported outreach to community providers to increase trial awareness and encourage patient referrals. Patient groups were engaged to disseminate study findings in language that patients can understand by 22% of respondents. Among the tools that were used, the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use Guidance on Clinical Practice was the most frequently reported tool in the planning phase (24%). Respondents reported no consistent use of any tool or tactic during the implementation and dissemination phases of clinical research and a large proportion reported using no tools to enhance DEI in their studies. Seventy-four percent of respondents reported that they had received no formal training or guidance on how to incorporate DEI principles and enhance DEI in clinical research. There were mixed opinions on the importance of incorporating DEI principles into clinical research, ranging from acknowledging the significance to dismissal of the concept. The majority of survey respondents (79%) indicated willingness to participate in a learning community developed by ASH. Conclusions The findings from this survey not only shed light on what resources and strategies investigators are employing most and at what stages of their clinical research, but also highlight a critical need for more training on and awareness of resources for incorporating DEI principles into clinical research. Results of this study will empower ASH to develop comprehensive resources that resonate with the dynamic needs of trialists in the pursuit of a more equitable and effective approach to clinical research.

Abstract Abstract 4000 Poster Board III-936 Background There is insufficient information regarding the epidemiology and outcomes of DVT in children. The CDC began a consented registry in August 2003 at eight pilot Centers for Thrombosis and Thrombophilia including six pediatric sites in order to assess the scope and severity of DVT morbidity in children. Methods Data for this analytic project were extracted from the CDC thrombosis registry. We performed a nested case-control study within the registry cohort. Children and adults are eligible to enroll onto this consented registry if they have a history of thrombosis, thrombophilia or a positive family history or either. DVT site, extent, recurrence and BMI were determined clinically. In addition, thrombophilia testing batteries, imaging protocols and determinations of PTS outcomes of DVT were made locally and criteria were not standardized among participating centers. Results As of March 2009 the CDC registry included 316 cases of DVT in infants and children (ages 0-21), and 2414 cases in adults (> 21). There were 16 cases reported to have lower extremity (LE) PTS in children and 184 in adults. Table 1 displays effects of thrombus site, recurrence and BMI on PTS prevalence. The proportion of LE DVT involving the inferior vena cava (IVC) was 5.5-fold higher in children compared to adults (11% vs 2%). Furthermore, among cases of IVC involvement, the prevalence of PTS following IVC thrombosis was remarkably higher in children compared with adults (38.9 vs 6.3%, P < 0.001). The prevalence of PTS following LE DVT overall in children did not differ from adults. Results for upper extremity DVT and resultant PTS were similar. Fifteen percent of children had recurrence. Increasing number of DVT events was associated with an increased risk of PTS. BMI was higher in children who developed PTS (p = 0.06; BMI > 28, OR 3.3 [1.08, 9.59]). In addition, thrombophilia was a risk factor for developing PTS in children (p = 0.02; OR = 2.9 [CI 1.03, 8.05]), with the highest risk determined with antiphospholipid syndrome (p = 0.02; OR = 3.4 [1.09, 9.93]) and protein C deficiency (p = 0.02, OR = 10.9 [0.84, 99.3]). Discussion Children with LE DVT demonstrate a high rate of IVC thrombosis, and a higher relative risk of PTS in cases of IVC involvement. Elevated BMI and thrombophilia augment the risk of PTS in children. The limitations of this study include the lack of standardization in DVT diagnostic and PTS evaluations. The study findings support application of standardized approaches to the diagnosis and follow-up evaluation of children with DVT in order to more meaningfully determine outcomes for future interventive trials. Disclosures: Moll: Ovation: Consultancy; GTC Biotherapeutics: Consultancy; Talecris: Consultancy.