Access Program Research Articles

Impact of a guaranteed access program to imatinib on the survival of patients with chronic myeloid leukemia.

This work aimed to evaluate the impact of a guaranteed access program to imatinib on the survival of patients with Chronic Myeloid Leukemia. We carried out a retrospective, observational, and analytical study of the database of patients diagnosed with Chronic Myeloid Leukemia of the Instituto Nacional de Cancerología and the Hospital General de México Dr. Eduardo to assess overall survival based on guaranteed access or not to imatinib. With an average follow-up of 99months, all patients' estimated 20-year overall survival was 72% (95% CI, 76-67). A significant difference was found in the 20-year survival probability in favor of patients with guaranteed access 76% (95% CI, 81-71) vs. 61% (95% CI, 69-52) (p < 0.001), in addition to those in which they had better attachment 81.2% (95% CI, 85-76) vs. 44.9% (95% CI, 52-37) (p < 0.001). CML is the most frequent chronic leukemia in Mexico. It mainly affects the economically active population (mean age 40), and the prognosis in our country has improved, emulating developed countries; however, the results depend on access to treatment and proper monitoring.

Exploring the role of the Recovery College model as a transformative tool for recovery-oriented practice: perceived benefits and perspectives from health practitioners in Quebec, Canada.

Mental health practitioners (MHPs), including occupational therapists (OTs), need support to adopt a truly recovery-oriented practice. Like other practitioners, if OTs often embrace the principles of recovery as a philosophical foundation for their practice, these principles may not always reflect in their attitudes, behaviors or in their interventions. While further research is needed to demonstrate the positive effects of recovery-oriented training programs on MHPs' attitudes and practice, there is a need to explore novel training programs. The Recovery College (RC) model is one of the interventions that are designed to facilitate these changes in practice, through co-production and co-delivery of recovery-focused courses curriculum. Although the perceived benefits and outcomes of RC courses are widely documented, very few studies focus specifically on what MHPs gain from them or on their global experience. The aim of this article is to describe the experience of MHPs learners in RC courses and the perceived benefits on their practice. An exploratory descriptive qualitative study was conducted. Data were collected through semi-structured interviews and analyzed using Miles and Huberman's stepwise qualitative analysis method. Participants were 13 MHPs working in community organizations or healthcare institutions and who participated as learners in a RC, in the province of Quebec, Canada. Ten themes emerged from the qualitative analysis. Participants expressed their perspectives on the format of the courses, their initial expectations and their recommendations. They also identified the types of knowledge they shared during the courses. Participants reported changes in their practice, raised awareness on their clinical and personal issues, improved well-being and recovery. Group composition, interactions within the group, complementarity of the different types of knowledge, and pedagogical design and learning activities were identified as key ingredients of RC. This study highlighted RCs' role in enriching MHPs clinically and personally. RC curriculum and courses drive changes in practice and attitudes towards service users. RCs may assist MHPs reflect on practice and improve their clinical reasoning. This study advances understanding of a promising, accessible training program for adopting a recovery-oriented practice amid a paradigm shift among MHPs and OTs.

Establishing an autogenous vascular access program in a Guatemalan comprehensive pediatric nephrology center.

The Guatemalan Foundation for Children with Kidney Diseases collaborated with Bridge of Life, a not-for-profit charitable organization, to establish a vascular access program. We reviewed our experience with graded surgical responsibility and structured didactic training, creating arteriovenous fistulas (AVF) for Guatemalan children. Pediatric vascular access missions were completed from 2015 to 2023 and analyzed retrospectively. Follow-up was completed by the Guatemalan pediatric surgeons, nephrologists, and nursing staff. AVF patency and patient survival were evaluated by Kaplan-Meier life-table analysis with univariate and multivariable association between patient demographic variables by Cox proportional hazards models. Among a total of 153 vascular access operations, there were 139 new patient procedures, forming the study group for this review. The mean age was 13.6years, 42.6% were female, and the mean BMI was 17.3. Radial or ulnar artery-based direct AVFs were established in 100 patients (71.9%) and ten of the 25 transposition procedures. Brachial artery inflow was required in 29 direct AVFs (20.9%). Two patients underwent femoral vein transpositions. Access-related distal ischemia was not encountered. Seven of the AVF patients later required access banding for arm edema; all had previous dialysis catheters (mean = 9, range 4-12). Primary and cumulative patency rates were 84% and 86% at 12months and 64% and 81% at 24months, respectively. The median follow-up was 12months. Overall patient survival was 84% and 67% at 12 and 24months, respectively. There were no deaths related to AVF access. Safe and functional AVFs were established in a teaching environment within a Guatemalan comprehensive pediatric nephrology center.

Real-world demographic and clinical profiles of patients with treatment-resistant depression initiated on esketamine nasal spray

Objective ESKALE is a French, multicentre, observational study of adults with treatment-resistant depression (TRD) treated with esketamine. This interim analysis describes baseline demographic and clinical characteristic evolution in patients included and treated from early access program to post-marketing launch. Methods Data were collected from medical records and included patient characteristics, disease history at esketamine initiation, use of neurostimulation, the patient’s care pathway, and the number of antidepressant treatment lines prescribed prior to esketamine initiation. Descriptive statistics were used for each cohort: the early access program ‘Temporary Authorisation for Use’ (ATU), post-ATU, and post-launch cohorts. Results The overall ESKALE cohort (N = 160 included; n = 157 treated with esketamine; average age 49.0 years; 66.2% female) demonstrated moderate-to-severe depression according to clinical assessment and a mean Montgomery-Åsberg Depression Rating Scale score of 32.6 (8.0); however, severity, subtype, and comorbidities were heterogeneous across the cohorts. Earlier use of esketamine and prior to alternative treatments occurred during the later cohorts. Conclusion These findings demonstrated a high burden of TRD in these patients and that esketamine is used in TRD treatment regardless of their disease severity, subtype, or existing comorbidities. These results also suggest that esketamine is potentially a clinically useful alternative treatment, particularly with healthcare professionals gaining greater familiarity with and easier access to esketamine.

Screening for Anxiety, Depression and Panic in older adults in a Family Medicine Unit in Sinaloa, Mexico

Introduction: The most prevalent mental disorders in Primary Care are Anxiety, Depression and Panic. These are one of the main causes of health demand in hospital stay services. Objective: to determine the frequency of mental disorders in older adults at the Family Medicine Unit No. 35, Culiacán, Sinaloa, México in the period from March to November 2024. Methods: Descriptive cross-sectional study. Older adults aged 60 to 75 years without a diagnosis of any mental disorder or syndrome were included. Sample of 379 participants. Simple random probabilistic sampling. A socio-demographic data sheet and the PHQ-4 + PHQ-TP scale were used. Descriptive and inferential statistics (Chi square) p ≤0,05 were used, it adhered to ethical and legal criteria. Results: The average age was 65,59 years. 53,3 % were men, 52 % were married, 50,7 % were currently working, 35,1 % had primary education and 76 % lived in urban areas. 34 % had anxiety, 28,2 % depression and 30,6 % panic. A significant relationship was found between anxiety and marital status (χ²=5,814, p=0,05) and schooling (χ²=12,845, p=0,00). Depression was related to schooling (χ²=18,479, p=0,00) and current job (χ²=8,528, p=0,003). Panic was related to sex (χ²=17,688, p=0,00), marital status (χ²=10,935, p=0,004), current job (χ²=6,273, p=0,001) and education (χ²=13,092, p=0,004). Conclusions: Anxiety was the most common mental disorder among older adults, highlighting the need to promote accessible and effective mental health programs that address the particular needs of this age group.

The ‘Forelle’ Early Market Access (FEMA) programme: South Africa’s solution to astringency and mealiness in ‘Forelle’ pears

The ‘Forelle’ Early Market Access (FEMA) programme: South Africa’s solution to astringency and mealiness in ‘Forelle’ pears

Pooled Fecal Allogeneic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from the Early Access Program in Europe

Pooled Fecal Allogeneic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from the Early Access Program in Europe

1651P Characteristics, tolerance and effectiveness of patients aged more or less than 75 years treated with [177Lu]Lu-PSMA-617 as part of France’s early access program

1651P Characteristics, tolerance and effectiveness of patients aged more or less than 75 years treated with [177Lu]Lu-PSMA-617 as part of France’s early access program

1628P Factors influencing clinical and biological response in patients treated with [177Lu]Lu-PSMA-617 under France's early access program

1628P Factors influencing clinical and biological response in patients treated with [177Lu]Lu-PSMA-617 under France's early access program

805MO Real-world efficacy and safety of tisagenlecleucel (CTL019) for relapse or refractory follicular lymphoma patients included in the early access program through the French DESCAR-T registry

805MO Real-world efficacy and safety of tisagenlecleucel (CTL019) for relapse or refractory follicular lymphoma patients included in the early access program through the French DESCAR-T registry

225P Nivolumab for cancer of unknown primary (CUP): Clinical efficacy and biomarker analysis from NivoCUP2 expanded access program (WJOG14620M)

225P Nivolumab for cancer of unknown primary (CUP): Clinical efficacy and biomarker analysis from NivoCUP2 expanded access program (WJOG14620M)

Investigating the impact of Accessible Pedagogies on the experiences and engagement of students with language and/or attentional difficulties

AbstractStudents with disability tend to report lower levels of school engagement. To date, research has focused on building students’ extrinsic motivation and self-regulation with limited consideration of the impact of instructional barriers. In this mixed-methods study, we investigated the effect of teachers’ participation in the Accessible Pedagogies™ Program of Learning on the classroom experiences and engagement of 56 Year 10 students with disabilities impacting language and information processing. When asked in interviews what their teacher did to help them pay attention and to understand, students described teachers’ increased use of practices that were the focus of the program. Self-report questionnaire data revealed a positive, statistically significant increase in cognitive engagement for students whose teachers participated in Accessible Pedagogies™. No increase was observed for a Comparison Group. Findings suggest that the reduction of extraneous language and cognitive load through teachers’ use of Accessible Pedagogies™ may have helped students deploy available mental effort to engage in learning, rather than expend that effort to overcome unnecessary instructional barriers. Future research will investigate the impact of Accessible Pedagogies™ with larger samples and a wider range of students.

Effectiveness of risankizumab induction and maintenance therapy for refractory Crohn’s disease: a real-world experience from a preapproval access programme and early access to medicines scheme

ObjectiveSince approval in Crohn’s disease (CD) of risankizumab, there has been widespread use. Real-world data are, however, limited and our aim is to address that gap.Design/methodWe performed a retrospective, observational...

CT-198 Pooled Fecal Allogeneic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from the Early Access Program in Europe

CT-198 Pooled Fecal Allogeneic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from the Early Access Program in Europe

Capmatinib efficacy for METex14 non-small cell lung cancer patients: Results of the IFCT-2104 CAPMATU study

BackgroundCapmatinib is a selective MET inhibitor with demonstrated efficacy in a phase II study of non-small cell lung cancer (NSCLC) patients harboring METex14 mutations. However, the real-world outcomes of capmatinib are largely unknown. From June 2019, the French Early Access Program (EAP) provided capmatinib to METex14 NSCLC patients who were ineligible for or for whom first-line standard therapies had failed. MethodsIFCT-2104 CAPMATU was a multicenter study that included all METex14 NSCLC patients who received capmatinib as part of the EAP until August 2021. The primary endpoints were time to treatment failure (TTF), progression-free survival (PFS), overall survival (OS) and objective response rate (ORR). ResultsA total of 146 patients were included. The median age was 74.9 years, 56.6 % were never-smokers, and 32.4 % had brain metastases. The median TTF, median PFS and median OS from capmatinib initiation were 5.1 months (95 % CI 4.2–6.0), 4.8 months (95 % CI 4.0–6.0) and 10.4 months (95 % CI 8.3–13.2), respectively. Evaluation of the best response to capmatinib was available for 134 patients and resulted in an ORR of 55.3 % (95 % CI 46.8 %-63.6 %). The median PFS was 7.7 months for treatment-naïve patients and 6.0 and 4.1 months for patients who had received one or 2 + prior lines of treatment, respectively. For patients with brain metastases, the median PFS was 3.0 months. Capmatinib had a known and manageable safety profile, with grade 3 to 4 adverse events, mostly peripheral edema (8.2 %), occurring in 17.8 % of patients. ConclusionIn this large real-world study of METex14 NSCLC patients, the efficacy of capmatinib was confirmed, with a manageable safety profile, even in patients with brain metastases and in those who received several lines of treatment. This study reinforces the key role of capmatinib for these patients.

Mykonos: A pulsed power driver for science and innovation

Sandia National Laboratories has been operating the Mykonos linear transformer driver (LTD) in a five-cavity configuration since 2014. The machine operates at 1MA output current, 500kV output voltage, with a 10–90% current rise time of 85ns, which enables small scale physics and engineering pulsed power experiments. Mykonos provides hands-on pulsed power experimental training for students and staff alongside senior Sandia scientists in an environment that is more accessible than the Z Facility. Over the years, we have fielded and accumulated a wide variety of optical, x-ray and electrical diagnostics and we are preparing to open this facility to outside users. Here, we are presenting the pulsed power and diagnostic capability of Mykonos as well as some recent experiments that have been performed on the facility. The goal of this publication is to attract researchers across the pulsed power and high energy density (HED) community to collaborate with Sandia on exciting, innovative science and to train the next generation of researchers for the National Nuclear Security Agency (NNSA) and the nation. As such, we have established a Mykonos Academic Access Program (MAAP) as part of ZNetUS to enable academic utilization of the Mykonos Pulsed Power Facility.

Analysis of Consumer Preferences in the Fitness Services Market of China

This article examines consumer preferences in the fitness services market in China, driven by the growing popularity of fitness and healthy living. Understanding fitness preferences among the population is increasingly important for developing effective fitness programs and marketing strategies. Chinese society is transitioning from traditional lifestyles to more active and healthy ones, leading to a rise in interest in physical activity and entertainment, as well as the emergence of new fitness facilities and programs. Insights into fitness preferences within the context of Chinese society can aid fitness industry enterprises and public health organizers in designing more effective and accessible programs for a broad audience. The contemporary Chinese market is one of the largest and fastest-growing globally. Economic changes in recent decades have led to a significant improvement in living standards and the emergence of new consumer trends among the Chinese population. However, these changes necessitate a thorough analysis of Chinese consumers' preferences to identify key factors influencing their choices. This study is based on a survey of 257 adults from various sociocultural groups across different regions of China. The research aims to determine the problems and prospects of engaging various population segments in health fitness activities and their consumer preferences. The results indicate varied satisfaction levels with fitness outcomes between men and women, with time constraints and lack of motivation being the main obstacles to fitness participation. Financial limitations and the distance to fitness centers also impact, albeit to a lesser extent. The most valued services for a comfortable fitness experience are massage, showers, and personal training programs. Personalized training sessions and online consultations received mixed reviews. These findings highlight the importance of addressing barriers to fitness participation and developing targeted strategies to enhance the quality of life for Chinese consumers.

The African American Transplant Access Program: Mitigating Disparities in Solid Organ Transplantation

Black Americans experience end-stage kidney disease (ESKD) at a disproportionately higher rate than other racial and ethnic groups in the United States. Kidney transplantation provides the best outcomes for patients with ESKD. However, Black patients frequently have decreased access to kidney transplantation. This article summarizes the robust literature on disparities in transplantation for Black patients and presents a practical solution to this complex issue by redesigning the transplant access process for Black patients challenged by structural and institutional barriers. The authors provide a detailed overview of a novel African American Transplant Access Program (AATAP) with its pillars of cultural congruency, trust, health literacy, and psychosocial support. This overview includes a thorough description of the program’s conceptualization, the process of creating the program, the preliminary results, and guidance to establish similarly culturally congruent transplant access programs.

The whole community? Assessing FEMA’s inclusion of Tribal governments in hazard mitigation efforts

The Federal Emergency Management Agency (FEMA) has long advocated for what it calls a “Whole Community approach” to disaster resilience and recovery. This philosophy holds that the priorities of all governmental, commercial, and interest groups should be considered, and their capabilities leveraged, in preparing for and responding to disasters. According to FEMA, federally recognized Tribal governments are part of the “Whole Community.” In this paper we use systematic content analysis techniques to examine policy documents derived from the Hazard Mitigation Assistance grant program to assess whether and how FEMA has taken the concrete policy steps necessary to include Tribal governments in the “Whole Community.” We find that while FEMA has expressed interest in a more equitable and accessible program that serves the needs of Tribal governments, it has taken few practical steps toward this goal.

A Medical Student-Inspired Advocacy Effort to Improve Pediatric Mental Health Services

The demand for pediatric mental health services has exceeded the supply of mental health providers for decades and increased significantly during the COVID-19 pandemic. Given the federal administration’s interest in passing a major funding bill to address the COVID-19 pandemic, the authors recognized an opportunity to collaborate in an advocacy effort to request federal financial support for Pediatric Mental Health Care Access Programs (PMHCAPs). The challenges of the pandemic and limited travel became assets as the advocacy team, who lived hundreds of miles apart in 2 states, met virtually with each other and with Congressional staff during February of 2021 on a tight timeline. In March of 2021, President Biden signed the American Rescue Plan that provided $80 million for PMHCAPs.