Experience In Treating Patients Research Articles

Abstract PO1-22-03: Radiation Toxicity and Treatment Outcomes in Breast Cancer Patients with ATM Pathogenic and VUS Mutations: Implications for Personalized Therapy

Abstract Introduction: Previous studies have shown that ATM (ataxia-telangiectasia mutated) mutations may be associated with increased radiation sensitivity and altered treatment response. However, there is a limited understanding of the clinical implications and optimal management strategies for breast cancer patients with ATM mutations receiving radiotherapy (RT). The aim of our study is to assess the impact of ATM mutations on radiation response and treatment outcomes in breast cancer patients undergoing RT. Material and Methods: A total of 79 female patients diagnosed with breast cancer were included in the study, with 31 (39.3%) having pathogenic (P) and likely pathogenic (LP) variants (all of the variants were heterozygous) and 48 (60.8%) having VUS variants detected through whole ATM gene sequencing screening for patients in accordance with NCCN clinical practice guideline. Statistical analysis, including descriptive, graphical, and inferential methods, such as the Kolmogorov-Smirnov test, Mann-Whitney U Test, and Chi-square tests, was conducted using SPSS version 25.0 to evaluate the normal distribution of continuous variable scores and perform quantitative and qualitative comparisons between groups at a 95% confidence level (p < 0.05). Results: The median age of the patients was 44 years (range: 24-74), with 75% of them being 50 years old or younger .The rate of breast cancer in patients with VUS variants was significantly higher in those aged 50 and below compared to those with P/LP variants (83% vs. 61%, p=0.028). There was no statistically significant difference in clinical stage, tumour histological type, and molecular subtype classification based on ATM gene variant type (P/LP vs. VUS) among the patients (p>0.05), but the rate of high tumour grade was significantly higher in the VUS variant group (65% vs. 40%, p=0.034). The rate of patients who received RT was 87%, with 26 patients receiving radiation to the breast or chest wall and 43 patients receiving additional regional lymph node irradiation. There were no statistically significant differences in surgical and systemic treatment types and RT dose based on ATM gene variant type (P/LP vs. VUS) (p>0.05). Among the patients who received RT, 33 (48%) experienced early-stage skin reactions, and 15 (22%) experienced late-stage skin reactions according to Common Terminology Criteria for Adverse Events (CTCAE) v5. No Grade 3, 4 and 5 early and late toxicity were observed . The rate of early (83% vs. 23%; p< 0.001) and late (35% vs. 13%; p=0.029) skin reactions was significantly higher in the P/LP variant group compared to the VUS variant group. Grade I lymphedema was observed in 2 (2.5%) patients, 1 (3.2%) in the P/LP group and 1 (2.1%) in the VUS group (p>0.05). Among the 23 patients who underwent breast reconstruction, 1 (4%) had implant revision and 1 (4%) experienced implant loss, both of which were observed in the pathogenic variant group (p>0.05). Conclusion: Limited clinical data exists on RT toxicity in breast cancer patients with ATM gene mutations, and clear guidelines for decision-making are lacking. Our study revealed higher rates of acute and chronic skin reactions in the P/LP variant group, highlighting the need for further investigation into the impact of these variants on treatment outcomes and patient management. However, it is crucial to manage these reactions effectively and not allow them to hinder treatment. Additional research and interventions are warranted to better understand and address these reactions, aiming to enhance the overall treatment experience and outcomes for patients with ATM gene mutations. Citation Format: Evrim Tezcanli, Seda Eroz, Ugur Ozbek, Ahmet Yesilyurt, CIHAN URAS, Nuran Bese. Radiation Toxicity and Treatment Outcomes in Breast Cancer Patients with ATM Pathogenic and VUS Mutations: Implications for Personalized Therapy [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO1-22-03.

Treatment seeking experiences of ACS patients in Yogyakarta, Indonesia during COVID-19 pandemic: A qualitative study.

Delay in treatment seeking is recognized as a major contributor for Acute Coronary Syndrome (ACS) mortality in Indonesia. Despite the significance of timely treatment, decline in admission and delay in presentation of patients with ACS were consistently reported during the COVID-19 pandemic. These suggested that treatment seeking performance of patients during the pandemic might be different from the previous period. This qualitative study aimed to explore treatment seeking behaviour, barriers in seeking medical treatment, and experiences of patients with ACS in Yogyakarta, Indonesia during COVID-19 pandemic. In depth-interviews were carried out with 30 patients, who were hospitalized with ACS at one of the three selected hospitals in Yogyakarta during the pandemic period. Thematic analysis was performed to create vital explanations for treatment seeking practices of patients with ACS during pandemic. Three significant themes were identified: treatment seeking decisions, barriers in seeking medical treatment during COVID-19, and experiencing both good and bad impression from entering and staying in the hospital. The intensity of ACS symptoms and fear of COVID-19 infection dominated the delay in seeking medical treatment. Strict safety measures, religious belief, and fear of ACS helped patients overcome barriers and seek medical treatment during pandemic. ACS patients also had convenient medical care during the pandemic and believed medical staff would provide excellent care to them. However, visit restriction policy could cause psychological discomfort. Increase awareness of ACS symptoms and the risk of delays ACS treatment are essential to support patients' decisions to seek medical helps in a timely manner at any situations including pandemic. Interventions aim at alleviating psychological distress should also be designed and implemented to improve treatment experiences of ACS patients, who sought medical treatment during the pandemic crisis.

Patient listening on social media for patient-focused drug development: a synthesis of considerations from patients, industry and regulators.

Patients, life science industry and regulatory authorities are united in their goal to reduce the disease burden of patients by closing remaining unmet needs. Patients have, however, not always been systematically and consistently involved in the drug development process. Recognizing this gap, regulatory bodies worldwide have initiated patient-focused drug development (PFDD) initiatives to foster a more systematic involvement of patients in the drug development process and to ensure that outcomes measured in clinical trials are truly relevant to patients and represent significant improvements to their quality of life. As a source of real-world evidence (RWE), social media has been consistently shown to capture the first-hand, spontaneous and unfiltered disease and treatment experience of patients and is acknowledged as a valid method for generating patient experience data by the Food and Drug Administration (FDA). While social media listening (SML) methods are increasingly applied to many diseases and use cases, a significant piece of uncertainty remains on how evidence derived from social media can be used in the drug development process and how it can impact regulatory decision making, including legal and ethical aspects. In this policy paper, we review the perspectives of three key stakeholder groups on the role of SML in drug development, namely patients, life science companies and regulators. We also carry out a systematic review of current practices and use cases for SML and, in particular, highlight benefits and drawbacks for the use of SML as a way to identify unmet needs of patients. While we find that the stakeholders are strongly aligned regarding the potential of social media for PFDD, we identify key areas in which regulatory guidance is needed to reduce uncertainty regarding the impact of SML as a source of patient experience data that has impact on regulatory decision making.

Own experience in catheter angiography and interventional treatment of patients with probable vertebrobasilar insufficiency

The vertebrabasilar (VB) system plays an essential role in the circulation of main structures of nervous system. About 25% of ischemic brain strokes are located in the vertebrobasilar circulation. 20-25% of them are due to atherosclerosis, leading to formation of hemodynamically significant stenoses in both vertebral arteries. Catheter angiography proves and visualise with great accuracy vascular pathology along the course of the extracranial segments of cerebral arteries. Interventional treatment in patients with significant vertebral artery stenoses is an established and safe method of treatment, complementing optimal drug therapy, and preceding surgical treatment.

Retracted: Analysis of the Effect of the Communication Ability of Nurses in Outpatient Infusion Room on the Treatment Experience of Patients and Their Families

Retracted: Analysis of the Effect of the Communication Ability of Nurses in Outpatient Infusion Room on the Treatment Experience of Patients and Their Families

Real-World Outcomes and Digitally Monitored Quality of Life in Voxelotor-Treated Patients with Sickle Cell Disease

Background Voxelotor is a haemoglobin oxygen-affinity modulator that leads to inhibited red blood cell sickling, has demonstrated reduced markers of haemolysis and anaemia improvement, and is one of a small number of novel disease-modifiers approved in the US for sickle cell disease (SCD) treatment. It has marketing authorisation in the UK and is currently undergoing reimbursement appraisal. The modelled impact of voxelotor on health-related quality of life (HRQoL)remains a key issue in current trial evidence. Therefore, the need for real-world data and understanding of patient-reported outcomes and experiences following treatment remains critical. Aims This work sought to characterise the side effects, HRQoL, and wearable-captured sleep and activity metrics of SCD patients in the UK, before and after treatment initiation. Methods Feedback from 19 SCD patients who had received voxelotor was captured through an online survey (Aug 2022-Mar 2023). 15 of these patients were also consented and enrolled within an artificial intelligence (AI) platform of over 600 SCD patients that combined genomic, medical record, real-time wearable biometric, and patient-reported outcomes data into a single platform. This included a mobile phone app for day-to-day QoL questionnaire (EQ-5D-5L) entry and an FDA-cleared smartwatch to capture sleep quality and physical activity. Convenience sampling selected data for a 17-patient (89%) subcohort with available pre- and post-voxelotor initiation EQ-5D-5L scores, either through the platform or follow-up survey snapshots. For each patient, this was analysed up to the date on which they completed the survey. EQ-5D-5L was also analysed in comparison to platform patients who had hydroxyurea (HU) in the past (n=19) or were receiving HU at the time of analysis (n=60), in addition to those with no recorded HU use (n=116). Additional analysis compared pre- and post-initiation activity levels (n=6), 4 of whom also had sleep quality data. Results The 19 surveyed patients had received voxelotor for a mean ± SD (range) of 194 ± 415 (17-1,826) days. 11 (58%) had also received HU and 1 (5%) received crizanlizumab. The mean age was 33 ± 13 (14-52) years, 12 (63%) were male, and 16 (84%) had HbSS. Side effects were reported in 8 (42%) patients, mostly diarrhea (6, 32%), stomach pains (2, 11%), and nausea/vomiting (2, 11%). Comparison of the 17-patient pre- to post-voxelotor cohort showed a statistically significant (p<0.001) increase in pooled EQ-5D-5L scores, from 0.558 ± 0.314 to 0.761 ± 0.262 (Figure 1). Post-initiation EQ-5D-5L for these 17 patients was significantly higher than for those receiving HU at 0.731 ± 0.232 (p=0.023) or who had previously received HU at 0.704 ± 0.256 (p=0.040) (Figure 2). Analysis of pre- and post-initiation wearables data saw increased deep sleep in 50% (2/4) and decreased nightly wakeups in 50% (2/4) of patients. Pooled pre- and post-initiation data showed a significant difference in deep sleep (43% vs. 47%, p=0.011). The mean number of steps and distance increased for 50% (3/6) of patients. While soft activity (Metabolic Equivalent of Task (METs): <3, e.g., sleeping, sitting quietly, slow walk) decreased for 67% (4/6) of patients, moderate activity (MET: 3-6, e.g., fast walk, cleaning, gardening) increased in 67% (4/6) and intense activity (MET: >6, e.g., running, hiking) increased in 60% (3/5). Conclusions Our data present a different lens to the voxelotor treatment experience of patients in the UK, with a side effect profile similar to that of other published studies. We demonstrated that EQ-5D-5L increased by 0.203 pre- to post-initiation, and was 0.030 and 0.057 higher than those receiving HU and those who had previously received HU, respectively, in the voxelotor-treated group. These are in line with the ranges of minimally important differences (0.030-0.098) reported for the EQ-5D-5L in other chronic diseases. As such and despite the limitations of small sample size and variability in pre- to post-initiation changes, a statistically significant improvement in HRQoL was observed after initiating voxelotor, as well as in comparison to HU treatment pathways. Similarly, pre- and post-initiation wearables data demonstrated early positive trends in voxelotor's impacts on sleep and activity using an FDA-cleared device, highlighting a need for further research into these metrics and the impact of SCD-modifying treatment.

Treatment and Coaching Experience of Individuals with Relapsed/Refractory Multiple Myeloma: Qualitative Insights during Treatment with Isatuximab

Background: Understanding the treatment experience of patients (pts) with relapsed/refractory multiple myeloma (RRMM) is important to providing timely, appropriate supportive care. A multi-center trial (NCT05053607) is examining patient experience with isatuximab for treatment of RRMM, triangulating quantitative data from patient-reported outcomes (PRO) and wearable devices, along with qualitative data collected via individual interviews. Here we present outcomes from the qualitative data analysis, the aim of which was to describe experience with diagnosis and treatment, as well as the digital life coaching (DLC) intervention. Study Design and Methods: In a prospective, quasi-experimental study, 50 individuals aged ≥18 years are consented and enrolled in a 3-month DLC intervention. PROs are collected at baseline and up to once per month for 3 months in addition to physical activity data collected via wrist-worn activity tracker. Up to 10 participants were purposefully selected to participate in an individual interview of up to 1 hour at one time point between weeks 8-12 of study participation. Qualitative descriptive analysis was used to analyze study data and summarize key themes. Results: A total of 8 individuals participated in qualitative interviews (2 men and 6 women). One identified as African American, 1 as Hispanic, and 6 as White. The mean age of participants was 67.6 (R= 47-86); average time since diagnosis was 8.25 (R=1-15); and average lines of therapy was 4.25 (R= 3-8). Responses were organized into three themes, focused on experience with: diagnosis and prior lines of therapy; current isatuximab inclusive therapy; and DLC, with highlighted quotes represented in the Table. Participants self-reported the most common symptoms of prior lines of therapy were hair loss, gastrointestinal symptoms (e.g., nausea, reflux, upset stomach), and “steroid-related” symptoms of food cravings and insomnia. Previous lines of therapy were discontinued due to either treatment toxicity or disease progression; the most commonly reported treatment toxicities were blood clots, neuropathy, dizziness, and immunosuppression with associated infections or pneumonia. Related specifically to their isatuximab-treatment regimens, most participants reported feeling a “burst of energy” but few to no side effects at the end of treatment. Several commented on the time commitment of having to go to the cancer center for infusions. Most stated the frequency and duration is acceptable given the outcomes of treatment. Insights into the measures of success of treatment ranged from how individuals felt while receiving therapy, to what degree therapy interfered with their ability to participate in activities of daily living, and the effect of treatment on their RRMM status. Several participants specifically spoke by name to the response of their Kappa free light chain test results being significantly lower. When asked about what they found most supportive during treatment, knowledge was the most common response. They also reported family, having a plan, and spirituality/faith as helpful. Regarding their coaching experience on study all described it as “positive,” however, two reported that the content felt fundamental to them since they had been managing their diagnosis for several years. Benefits included having someone, particularly other than a family member, to speak to about their health, increased attention on healthy behaviors such as nutrition and exercise, and enhancing access to information and education about their diagnosis, treatment, and management. Conclusions: Qualitative interviews among individuals with RRMM provided insight into the challenges but also hope associated with chronic management of their condition. Treatment with isatuximab regimens was perceived as logistically manageable with few side effects. Though some individuals felt their long-term experience with MM made the coaching less impactful, coaching was generally perceived as a positive experience, with many individuals self-reporting benefits related to health behaviors and outcomes.

Cytokine Release Syndrome: The Patient, Caregiver and Healthcare Professional Experience

Background: Cytokine release syndrome (CRS) is a supraphysiologic response to T-cell engaging immunotherapy that can negatively impact the treatment experience of patients with non-Hodgkin lymphoma (NHL) and other blood cancers. We explored patient, caregiver and healthcare professional (HCP) perspectives on key burdens and unmet needs associated with CRS. Methods: Existing insights from disease-level patient journeys (follicular lymphoma, diffuse large B-cell lymphoma, multiple myeloma [MM]) and reports from focus groups with a CRS patient working group from the USA, UK, Canada, and Belgium were assessed to identify key questions. Further interviews were conducted (November 2022-January 2023) in the USA, UK, Germany, and Austria to address these questions with patients, caregivers and HCPs. HCP insights were collected via 60-minute virtual interviews; patient and caregiver insights were collected via video homework tasks and 60-minute virtual interviews. Outputs were validated by the working group, consisting of patients with CRS experience and representatives of patient organizations. Results: A total of 34 HCPs (hemato-oncologists, n=24; nurses, n=10; Tables 1 & 2) with varying degrees of experience of managing CRS were interviewed, along with 9 patients and 3 caregivers (associated diagnoses: NHL, n=6; MM, n=5; acute lymphocytic leukemia, n=1). All patients had experienced CRS (Grade 1-3) as a result of CAR T-cell (n=6) or bispecific antibody (n=3) therapy. Insights were based on five focus areas: 1) patient education and information; 2) CRS diagnosis and management; 3) CRS in different healthcare archetypes; 4) digital literacy and the role of technology in mitigating/monitoring/managing CRS; and 5) the caregiver perspective. CRS education was a primary unmet need, but overall awareness and knowledge of CRS was strong among the patient sample. Patients noted the life-threatening potential of CRS, but preferred to be on active treatment. HCP communication to patients focused on CRS being an expected occurrence rather than a marker of treatment failure, the need for in-patient admission in some cases and/or close monitoring, and the critical role of caregivers. Generally, HCPs were familiar with CRS and the procedures for its monitoring and management, but the variability in time to onset, severity of symptoms, and potential impact on quality of life (QOL) warranted caution. Logistics emerged as a common barrier, with CRS treatment often administered at major hospitals, as local, community-based clinics do not currently have the required capacity/capability to manage CRS; this could be achieved via the provision of training and support. Uncertainty remains regarding the broader roll out of bispecific antibodies, given the variability in CRS treatment between countries. Patients, HCPs and caregivers were aligned on their preferences for remote monitoring for CRS. All three groups expressed the need for simple, easy-to-use solutions that integrate seamlessly with existing systems and do not cause undue concern with false alarms, complex technical set-up and patient user experience. Patients emphasized the need for prompt and assured access to their medical team in case of an emergency. HCPs expressed a range of opinions on the feasibility of adopting such solutions. Some were open to using new tools and saw the potential for alleviating resource challenges. Others had reservations about interoperability and questions around over-informing patients with detailed data. Patients were open to adopting new technologies, if they added tangible benefit to their QOL, avoided additional logistical burden and improved their experience. Conclusions: There is strong awareness among patients, caregivers, and HCPs of the risks associated with CRS and the potential impact on QOL. HCPs with experience in administering cellular immunotherapies are confident in monitoring and managing CRS; however, its unpredictability and logistical burden need to be addressed. There is value in adopting digital solutions for CRS monitoring and management. Concerns remain around interoperability, data stewardship, and integration and validation within current hospital systems. Patients' concerns regarding the useability of such digital solutions and assured data privacy also need to be addressed as part of solution development in the CRS space.

Azacitidine Treatment in MDS: A Systematic Literature Review and Meta-Analysis Comparing the Efficacy of Real World Data with Randomized Controlled Trials

Background: Myelodysplastic Syndromes (MDS) are a group of myeloid malignancies affecting the hematopoietic stem and progenitor cells and occurring mainly in elderly individuals. Two hypomethylating agents (HMA), Azacitidine (AZA) and Decitabine (DAC), have shown efficacy in treatment of MDS patients and received approval based on randomized controlled trials (RCTs) more than a decade ago. Since then, many publications have reported real world experience on HMA treatment in MDS patients, which showed a reduced efficacy of HMA than the RCT. The reasons for this difference have been extensively discussed but a systematic review never performed. Aim: We performed a systematic literature review and meta-analysis to compare the efficacy of real world data with randomized controlled trials and to identify potential cofactors for different outcomes. Material and Methods: We performed a systematic literature search from observational studies (OBSTs) describing real world experience of AZA treatment in MDS patients using Embase, Ovid Medline, and Web of Science databases. Patient characteristics, IPSS/IPSS-R, and outcome (response rates, overall survival) were collected from OBSTs that were performed between 2003 and 2023. Kaplan-Meier curves with log-rank tests were used to assess differences in survival outcomes. An inverse variance weighted meta-analysis was performed by using restricted mean survival times or proportions (response rate outcome). The restricted mean survival time and the standard error at 12, 24 and 36 months was calculated by using the R package survival. The pooled effect and prediction intervals from random effect models were reported. Results: In our systematic literature search, data from OBSTs describing real world experience of AZA treatment in MDS patients could be extracted and compared to the only RCT published with AZA (Fenaux P et al., Lancet Oncology 2009). Patient and disease characteristics were similar for AZA-RCT and OBST (respect of gender ratio, median age, IPPS/IPSS-R). Restricted mean survival time and standard error of the OBSTs and AZA-RCT showed no significant difference for the time points at 12 months (10.0 [95%-CI: 9.6; 10.3] months (OBSTs) vs 10.0 [95%-CI: 9.5; 10.5] months (AZA-RCT); figure part A) and 24 months (15.5 [95%-CI: 14.6; 16.5] months (OBSTs) vs. 16.9 [95%-CI: 15.7; 18.2] months (AZA-RCT); figure part B). At 36 months, the restricted mean survival time of OBSTs was 18.7 [95%-CI: 17.3; 20.1] months compared to 22.3 [95%-CI: 20.2; 24.5] months of the AZA-RCT (figure part C), which was different in favor of the RCT. Using random effect models, the AZA treatment response rate was 0.51 [95%-CI: 0.47; 0.56] for the OBSTs and 0.49 [95%-CI: 0.42; 0.57] for the AZA-RCT (figure part D). Conclusion: Compared to AZA-RCT data, restricted mean survival time (for time points 12 months and 24 months) and response rate of OBSTs describing real world data of AZA treatment in MDS patients are comparable. Regarding the observation time point at 36 months, the lower restricted mean survival time of the OBSTs compared to AZA-RCT may be most likely explained by study patient selection bias.

Kawasaki Disease Associated Acute Abdomen: Most Require No Surgery.

To summarize the clinical features and treatment experiences of patients with Kawasaki disease (KD)-associated acute abdomen (KD-AA). We conducted a retrospective case-control study of patients with KD-AA treated at our hospital between January 2006 and November 2022. Of the 917 children with KD, 43 (4.7%) presented with AA. Of these, 33 with complete information were included in the KD-AA group. Patients with KD-AA were significantly older, with higher neutrophil rate, C-reactive protein, procalcitonin, and alanine transaminase levels and lower hemoglobin, albumin, and serum sodium levels. Additionally, more patients with KD-AA presented with aseptic meningitis and KD shock syndrome than those with KD alone (all p<0.05). The two groups did not differ in the incidence of intravenous immunoglobulin (IVIG) resistance, incomplete KD, or coronary artery abnormalities. All patients received aspirin and IVIG therapy, with nine receiving a second dose of IVIG and 11 receiving corticosteroids. Only two patients with KD-AA underwent surgery, and the prognosis of all patients with KD-AA was good. KD-AA should be suspected in febrile children with abdominal symptoms. Prompt diagnosis of KD-AA is important for early effective treatment to avoid unnecessary surgical harm. KD, complicated by acute abdomen, has a good prognosis.

Cytogenetic risk explored within an electronic patient reported outcomes (ePROs) remote symptom monitoring platform among patients with acute myelogenous leukemia (AML).

349 Background: Patients with AML and poor-risk cytogenetic abnormalities often have suboptimal treatment response and poor long-term survival, Electronic Patient Reported Outcomes (ePROs) integration into routine oncology practice have been shown to improve patient time on therapy, survival, and resource utilization. This study explores the use of an ePRO platform to explore differences in treatment experience and ePRO compliance in patients with AML and poor and other-risk cytogenetics. Methods: Adult patients with AML undergoing treatment were enrolled in the Carevive remote symptom monitoring (RSM) platform between March 1, 2021 and April 20, 2023. Patients received baseline and weekly surveys to assess patient-reported symptom burden, physical function, overall health, and survey compliance throughout treatment. Baseline characteristics included age, sex, race, frailty status (modified Geriatric Assessment, CARE survey item, and/or self-reported activity level), comorbidities, and social determinants of health. Symptoms (derived from the PRO-CTCAE), physical function (PROMIS 4a), and quality of life (QoL) (EORTC QLQ-C30, items #29-30) were assessed weekly. Results were stratified by poor vs. other cytogenetic risk. As this was an exploratory study, statistical significance was not reported. Results: Of a total of 124 patients, 47 (38%) had poor-risk and 77 (62%) had other-risk disease by cytogenetics. Weekly survey compliance was comparable (68% vs 69%), and median age was 66 (range 35-88) and 69 (range 21-88), respectively. About 50% of all patients were male, 76% were White, and 69% had ≥1 comorbidity. Patients with other-risk were younger with 40% fewer comorbidities (mean 0.95, median 0, range 0-5) than patients with poor cytogenetic risk (mean 1.36, median 1, range 0-5). Overall symptoms were reported 12% more frequently among patients with other risk (1.49 symptoms per patient-week) compared to patients with poor risk (1.33 symptoms per patient-week). Fatigue was reported by 20% of patients across all patient-weeks for both risk groups; shortness of breath was reported 6% and 10% across all patient-weeks for poor and other risk groups, respectively. Although baseline treatment bother was 17% higher for poor risk patients, over time, QoL and treatment bother were comparable between both groups, and physical function was directionally higher. Conclusions: This study illustrates the feasibility of using remote symptom monitoring for evaluating patients with poor vs. other cytogenetic risk in routine care, with relatively high survey compliance in a real-world setting. Despite poor prognostic status, the experience of poor cytogenetic risk patients was comparable to other cytogenetic risk patients and reported directionally lower levels of symptom burden and higher levels of function over time.

Enhancing the Security and Privacy of eHealth Records through Blockchain-based Management: A Comprehensive Framework

Progress in information technology is transforming the healthcare sector with the goal of enhancing medical services, diagnostics, and continuous monitoring through wearable devices, among other benefits, while also lowering expenses. This digital transformation enhances the convenience of computing, storing, and retrieving medical records, ultimately leading to improved treatment experiences for patients. Electronic health record systems have come under fire for centralized control, faults, and attack points with transferring data custodians. These systems are frequently utilized for the interchange of health information among healthcare stakeholders. The main objective is to overcome information asymmetry and data breaches commonly encountered in the Electronic Health Record (EHR) system. This study introduces a decentralized and trustless architecture aimed at securely storing patients' medical records and granting access to authorized individuals, including healthcare providers and patients themselves. The research primarily focuses on bolstering the security and privacy of healthcare data management systems using blockchain technology. To address the issue of blockchain scalability, an off-chain scaling approach is proposed, utilizing an underlying medium to store large volumes of data. This is achieved through the integration of Elliptic Curve Cryptography (ECC) and the Interplanetary File System (IPFS). The proposed system provides a secure and efficient method for storing and sharing sensitive healthcare data while ensuring confidentiality and data integrity.

Changes in Patient Discourse: A Qualitative Study Based on the Treatment Experience of Chinese Patients with Somatization Symptoms.

This qualitative study examines the characteristics exhibited by Chinese patients with somatization symptoms during their treatment process, focusing on changes in illness interpretation and language use. A semi-structured in-depth interview was conducted with 10 patients receiving treatment in a clinical psychology department of a general hospital who reported somatic symptoms as their main complaint. The interview data were recorded and transcribed, and analyzed using interpretive phenomenological analysis. Two core themes emerged from the analysis: avoidance at the utterance level; and at the semantic level, power and contestation. Patients with somatization symptoms exhibit avoidance behaviors, and their experience of illness and the therapeutic process impact their discourse. Professionals should pay attention to patients' own interpretations, cultural background and acceptance of the illness.

Experience in Treatment of Patients with First Type Secondary Endoleaks

INTRODUCTION: First type secondary endoleaks (EL) require quickest elimination, primarily using the endovascular methods. Despite the existence of a sufficiently large number of methods of their elimination, the results of treatment remain ambiguous.&#x0D; AIM: Analysis of our own experience of elimination of the first type ELs in patients after endoprosthetics of infrarenal abdominal aortic aneurysm.&#x0D; MATERIALS AND METHODS: The study is retrospective with prospective observation. All patients underwent computed tomography (CT) for identification of the type of EL and choosing a method of its elimination. The term first type secondary EL was understood as EL which developed in the long-term period after the primarily negative CT-angiography after endoprosthetic repair of the aorta.&#x0D; RESULTS: ELs of Ia type were diagnosed in 14 cases, in one case EL of Ib type was identified. On average, first type secondary ELs were identified in 34 months after the primary operation for endograft implantation. In 2 of 15 patients, EL were symptomatic. The elimination of the first type EL required an individualized approach in all cases. The main methods of elimination of the first type EL were: elongation using Jotec E-iliac (n = 1); coiling with implantation of Gore cuff and Chimney-stenting of the left renal artery (LRA; n = 2); implantation of Gore cuff (n = 4); reinforcement with Aptus EndoAnchors (n = 2); Cuff Gore implantation and LRA stenting by Chimney-technique (n = 1); implantation of Cuff Jotec and reinforcement with Aptus EndoAnchors (n = 1); aneurysmal sac coiling (n = 2); Gore cuff implantation with fixation with EndoAnchors and LRA stenting using Chimney-technique (n = 1). The technical success of EL elimination was 100% (in all cases, the success was confirmed by intraoperative angiography, as well as by CT angiography or ultrasound duplex scanning with contrast enhancement after surgery), there were no deaths. In the long-term period (from 6 to 60 months), re-interventions were performed in two cases, fatal outcome happened in one case, after a late open conversion for elimination of EL.&#x0D; CONCLUSION: In 13% of cases, first type ELs were symptomatic, and their elimination required individual approach with taking into account anatomical factors and the implanted graft. The medium-term results of the elimination of the first type EL are good, no fatal cases have been reported, and the number of reinterventions has reached 20%, which requires monitoring in the postoperative period.

1394P Real-world experience in treatment of patients with non-small cell lung cancer with BRAF and cMET Exon 14 skipping mutations

1394P Real-world experience in treatment of patients with non-small cell lung cancer with BRAF and cMET Exon 14 skipping mutations

Predictors of Poor Treatment Experience in Cervical Cancer Patients Receiving Definitive Chemoradiation and Brachytherapy Boost: A Prospective Study

Cervical cancer patients experience high overall symptom burden. Here, we analyzed patient reported outcomes (PROs) to identify disease, treatment, or patient-related characteristics that can predict poor treatment experience. Cervical cancer patients treated with definitive chemoradiation (CRT) at a single institution enrolled on a prospective trial evaluating PROs between 2021-2023 were included. Patients received PRO questionnaires at baseline (BL) and 2-7 days after final brachytherapy implant (post-BT). Data was collected using the EORTC-QLQ-C30, which is a validated metric scored on a 4-point Likert scale (1 = not at all, 2 = a little, 3 = quite a bit, 4 = very much). Poor treatment experience was defined as low physical function [score >2], significant overall symptom burden [> population mean], or substantial nausea/vomiting (N/V), diarrhea, pain, or fatigue [score >2] after BT. Potential predictors included age, menopause status, stage, radiation field size, BT modality (PDR vs HDR), marital status, high baseline financial toxicity [score >2], depression [score >2], worse social function [score >2] and poor emotional function [score >2]. Logistic regression modeling was performed and p<0.05 were considered significant. A total of 36 patients completed BL and post-BT PRO metrics. Median age was 42 (range, 18-85), 22% (n = 8) of patients had localized disease, 75% (n = 27) had regional disease, and 3% (n = 1) had distant disease. Low BL social function was associated with high symptom burden after BT (HR 12.5, 95% CI 2.3-68.2, p = 0.004), significant N/V (HR 19.0, 95% CI 1.9-191.0, p = 0.012), high rates of fatigue (HR 9.29, 95% CI 1.6-54.8, p = 0.014), and overall poor physical function after treatment (HR 5.67, 95% CI 1.1-30.1, p = 0.042). High BL financial toxicity was predictive of elevated symptom burden after BT (HR 12.0, 95% CI 2.2-66.0, p = 0.004) and substantial fatigue (HR 7.33, 95% CI 1.5-36.7, p = 0.015). Significant depression at BL was also associated with high rates of N/V (HR 9.78, 95% CI 1.4-66.9, p = 0.02). Patient age, menopausal status, disease stage, radiation field size, BT modality, and marital status were not significantly predictive for symptom burden or physical function after treatment. Patients with poor baseline social function, high financial toxicity, and depression are at risk for increased symptom burden. Screening for these factors may provide an opportunity to intervene early and improve patient treatment experience.

Analysis of Institutional DIBH Coaching Program for Surface Guided-DIBH Patients

Our institute has implemented a surface guided-DIBH (SG-DIBH) coaching program which involves consultation, pre-treatment (CT-sim) and treatment. We would like to analyze the effectiveness of the program. A total of 72 left breast cancer patients between 1st Apr 2022 to 9th Dec 2022 were registered for radiation treatment. During consultation, oncologist selected suitable patients based on the following criteria: a) age of 18-70; b) left breast cancer, right breast cancer with internal mammary nodes irradiation or dextrocardia; c) no lung/cardiac disease history & d) volunteer for SG-DIBH technique. The eligible patients were then trained by the coaching therapist using a teaching video and practiced at home. During CT simulation, patients were assessed according to the DIBH evaluation form. The evaluation components included patient's compliancy and understanding, the differences of lateral skin marking (free breathing, FB vs DIBH), duration of breath hold and reproducibility. Patients who passed the evaluation were scanned under both FB and DIBH for SG-DIBH treatment. IMRT-FFF 6 to 7 fields were planned. During SG-DIBH treatment, first 3 fractions and weekly CBCT were taken. Patients were encouraged to continue DIBH practice at home throughout whole course of the treatment and they were given 3 identical survey forms (5 questions) at the beginning, middle and end of treatment. The measure for the success of this coaching program would be number of breath-holds, duration of treatment time, treatment accuracy (CBCT matching) and survey results. There were 48 patients who were eligible for DIBH coaching program, however, only 24 patients had passed the coaching evaluation and 20 patients were treated with SG-DIBH technique successfully. The mean of number of breath-hold and treatment time was 7 times and 7.9 minutes. Total of 123 CBCT images were studied. The setup errors were (0.242±0.180) cm, (0.152±0.137) cm, (0.202±0.165) cm, (0.684±0.640) degrees, (0.816±0.767) degrees, (0.912±0.707) degrees in lateral, longitudinal, vertical, pitch, roll and yaw directions. According to the survey analysis, the number of times to practice at home decreased as the treatment went by. An improvement was seen in patients' self-evaluation in mastering DIBH technique with proper coaching program (from 60% to 90%). Patients' anxiety in performing DIBH were alleviated greatly towards the end of the treatment (from 47% to 15%). 100% of the patients were willing to go for DIBH treatment if given a second chance and additional suggestions claimed that professional clinical teams and coaching program were important for their DIBH treatment journey. A comprehensive DIBH coaching program can effectively identify SG-DIBH patient's suitability. Patient compliancy, treatment accuracy and treatment experience can be enhanced with good coaching program. The involvement of clinical team from consultation to pre-treatment and treatment stage is essential for a successful SG-DIBH treatment.

Reconstruction of Moderately and Severely Atrophic Scalp-A Multicentric Experience in Surgical Treatment of Patients Irradiated for Tinea Capitis in Childhood and Surgical Algorithm.

Background and Objectives: Before the introduction of griseofluvin, the use of X-ray radiation was the treatment of choice for tinea capitis. More than half a century later various types of tumors have been found to be associated with childhood irradiation due to tinea capitis, most commonly cancers of the head and neck, as well as brain tumors. The often unusually aggressive and recurrent nature of these tumors necessitates the need for repeated surgeries, while the atrophic skin with an impaired vascular supply due to radiation often poses an additional challenge for defect reconstruction. We present our experience in the surgical treatment of such patients. Materials and Methods: This is a retrospective cohort study. In this study, 37 patients treated for acquired defects of the scalp with a history of irradiation therapy due to tinea capitis in childhood were included in this study, 24 male and 13 female patients. The mean age at the first appointment was 60.6 ± 7.8, with the youngest included patient being 46 and the oldest being 75 years old. Patients' characteristics, surgical treatment, and complications were analyzed and a reconstructive algorithm was developed. Results: Local flaps were used for reconstruction in 34 patients, direct sutures were used in 10 patients and 20 patients received split-thickness skin grafts for coverage of both primary and secondary defects for reconstruction of flap donor sites. One regional flap and one dermal substitute covered by an autologous skin graft were also used for reconstruction. Complications occurred in 43.2% of patients and were significantly associated with the presence of comorbidities (p = 0.001), aseptic bone necrosis (p = 0.001), as well as skin atrophy in frontal, occipital, and parietal region (p = 0.001, p = 0.042 and p = 0.001, respectively). A significant correlation between major complications and moderate skin atrophy was found only in the parietal region (p = 0.026). Conclusions: Unfortunately, many protocols developed for scalp reconstruction are not applicable in the setting of severe or diffuse scalp skin atrophy associated with high tumor recurrence rate and radiation-induced vascular impairment, such as in tinea capitis patients in Serbia. An algorithm has been developed based on the authors' experience in managing these patients.

Real-world experience of OnabotulinumtoxinA treatment in female patients with chronic migraine: a qualitative study using in-depth interviews

Background Chronic migraine (CM) causes great disability and affects an individual’s quality of life. OnabotulinumtoxinA (OBT-A, Botox®) was the first prophylactic treatment specifically indicated for CM. The aim of this study was to describe the experiences of women with CM treated with OBT-A. Materials and Methods The study design is a qualitative descriptive study. A purposeful sampling of 30 women (mean age, 42.7; standard deviation, 10.6) who had received at least two administrations of OBT-A for CM (PREEMPT protocol) was performed. Data collection included in-depth interviews and researchers’ field notes. A thematic analysis was carried out according to qualitative research guidelines. Results Five themes were identified: (a) A long way to go before Botox®, (b) First time hearing about the treatment and its expectations, (c) The administration of Botox®, (d) Treatment effects, and (e) Follow-up. Patients described a long history of treatment failures prior to the start of OBT-A treatment. Information about this migraine treatment came from the neurologist; following the information, patients had high expectations, including unrealistic expectations regarding the onset and duration of effect. They acknowledged fear of the injections and some discomfort due to the procedure. With treatment, participants reported better migraine control and an improvement in their quality of life. Follow-up had some barriers, such as delayed appointments for subsequent doses, but also strengths, such as effectiveness and few side effects. Conclusions Qualitative research offers insight into how patients with CM experience treatment with OBT-A. Our results highlight some relevant aspects that should be considered when providing OBT-A treatment.

Evaluating Response Trends of Chlormethine/Mechlorethamine Gel in Patients With Stage I-IIA Mycosis Fungoides: Analysis of Individual Patient Data From a Randomized Controlled Phase II Study to Facilitate Optimal Treatment Experiences

IntroductionChlormethine (CL) gel was approved for treatment of mycosis fungoides based on the pivotal 201 trial (NCT00168064). Data visualization from individual patients is a powerful tool for discovery of hidden treatment trends. Here, we present a post hoc analysis of individual patient data from the pivotal trial to provide a more granular depiction of treatment and response changes over time, with an emphasis on end of treatment status. Materials and MethodsIndividual patient response data were plotted over a 12-month treatment period to visualize patient experiences while using CL gel. Responder status was assigned according to end-of-treatment Composite Assessment of Index Lesion Severity (CAILS) score, and patients were classified as early (≤4 months) or late responders based on timing of response. Baseline and active treatment characteristics were compared between early and late responders, and baseline body surface area (BSA) was compared between responders and patients with stable or progressive disease. ResultsData from 123 patients with baseline and postbaseline results were included. At the end of treatment, 64.2%/55.3% were responders, 30.9%/34.1% had stable disease, and 4.9%/10.6% had progressive disease by CAILS and mSWAT, respectively. Among patients who responded to treatment, 64.6% and 35.4% were early and late responders, respectively. Response pattern analysis also identified patients with an intermittent response or initial progressive disease. Baseline BSA was not associated with responder status. Late responders had longer treatment duration and higher postbaseline plaque elevation, while early responders had a higher frequency of dermatitis. ConclusionsResults presented here can facilitate optimal treatment experiences for patients starting CL gel.