Pub Date : 2024-09-01Epub Date: 2024-08-02DOI: 10.1007/s12325-024-02943-5
Fernando Gómez-Peralta, Isabel Leiva-Gea, Natalia Duque, Esther Artime, Miriam Rubio de Santos
Introduction: Continuous glucose monitoring (CGM) devices allow for 24-h real-time measurement of interstitial glucose levels and have changed the interaction between people with diabetes and their health care providers. The large amount of data generated by CGM can be analyzed and evaluated using a set of standardized parameters, collectively named glucometrics. This review aims to provide a summary of the existing evidence on the use of glucometrics data and its impact on clinical practice based on published studies involving adults and children with type 1 diabetes (T1D) in Spain.
Methods: The PubMed and MEDES (Spanish Medical literature) databases were searched covering the years 2018-2022 and including clinical and observational studies, consensus guidelines, and meta-analyses on CGM and glucometrics conducted in Spain.
Results: A total of 16 observational studies were found on the use of CGM in Spain, which have shown that cases of severe hypoglycemia in children with T1D were greatly reduced after the introduction of CGM, resulting in a significant reduction in costs. Real-world data from Spain shows that CGM is associated with improved glycemic markers (increased time in range, reduced time below and above range, and glycemic variability), and that there is a relationship between glycemic variability and hypoglycemia. Also, CGM and analysis of glucometrics proved highly useful during the COVID-19 pandemic. New glucometrics, such as the glycemic risk index, or new mathematical approaches to the analysis of CGM-derived glucose data, such as "glucodensities," could help patients to achieve better glycemic control in the future.
Conclusion: By using glucometrics in clinical practice, clinicians can better assess glycemic control and a patient's individual response to treatment.
{"title":"Impact of Continuous Glucose Monitoring and its Glucometrics in Clinical Practice in Spain and Future Perspectives: A Narrative Review.","authors":"Fernando Gómez-Peralta, Isabel Leiva-Gea, Natalia Duque, Esther Artime, Miriam Rubio de Santos","doi":"10.1007/s12325-024-02943-5","DOIUrl":"10.1007/s12325-024-02943-5","url":null,"abstract":"<p><strong>Introduction: </strong>Continuous glucose monitoring (CGM) devices allow for 24-h real-time measurement of interstitial glucose levels and have changed the interaction between people with diabetes and their health care providers. The large amount of data generated by CGM can be analyzed and evaluated using a set of standardized parameters, collectively named glucometrics. This review aims to provide a summary of the existing evidence on the use of glucometrics data and its impact on clinical practice based on published studies involving adults and children with type 1 diabetes (T1D) in Spain.</p><p><strong>Methods: </strong>The PubMed and MEDES (Spanish Medical literature) databases were searched covering the years 2018-2022 and including clinical and observational studies, consensus guidelines, and meta-analyses on CGM and glucometrics conducted in Spain.</p><p><strong>Results: </strong>A total of 16 observational studies were found on the use of CGM in Spain, which have shown that cases of severe hypoglycemia in children with T1D were greatly reduced after the introduction of CGM, resulting in a significant reduction in costs. Real-world data from Spain shows that CGM is associated with improved glycemic markers (increased time in range, reduced time below and above range, and glycemic variability), and that there is a relationship between glycemic variability and hypoglycemia. Also, CGM and analysis of glucometrics proved highly useful during the COVID-19 pandemic. New glucometrics, such as the glycemic risk index, or new mathematical approaches to the analysis of CGM-derived glucose data, such as \"glucodensities,\" could help patients to achieve better glycemic control in the future.</p><p><strong>Conclusion: </strong>By using glucometrics in clinical practice, clinicians can better assess glycemic control and a patient's individual response to treatment.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11349838/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141873901","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-01Epub Date: 2024-07-31DOI: 10.1007/s12325-024-02946-2
Tobias J Lange, Pilar Escribano-Subias, Audrey Muller, Catarina C Fernandes, Martina Fontana, Tatiana Remenova, Stefan Söderberg, Sean Gaine
Introduction: Risk assessment can aid management of pulmonary arterial hypertension (PAH) and clinical decision-making. This analysis describes characteristics, treatment patterns and outcomes of patients with PAH, categorised by risk status at time of treatment escalation with selexipag in clinical settings.
Methods: Patients initiating selexipag in the ongoing multicentre, prospective EXPOSURE (EUPAS19085) study were grouped as low, intermediate-low, intermediate-high or high risk of 1-year mortality according to the ESC/ERS 4-strata method.
Results: As of November 2022, 77% (535/698) of patients initiating selexipag had data allowing for risk calculation; 14% (N = 76) were low, 31% (N = 168) intermediate-low, 34% (N = 182) intermediate-high and 20% (N = 109) high risk of 1-year mortality. Overall, patients were predominantly female (71%), with idiopathic/heritable PAH (56%) or PAH associated with connective tissue disease (CTD-PAH; 27%), median age of 60 years and prevalent (2 years) disease. From low to high risk, proportion of CTD-PAH and age increased (from 12%-40% and 46-68 years, respectively); time from diagnosis decreased and presence of cardiovascular risk factors increased. Most patients across risk groups (74-81%) initiated selexipag as part of triple oral combination therapy. Overall median (Q1, Q3) selexipag exposure duration was 10.1 (3.5, 24.1) months. Proportions of hospitalised patients increased with increasing risk group (16-42% from low to high, respectively); more hospitalisations were PAH-related for the high risk (71%) versus other risk groups (47-54%). Kaplan-Meier survival estimates were 98%, 98%, 93% and 80% at 1-year and 98%, 92%, 81% and 67% at 2-years, from low to high risk, respectively.
Conclusions: In clinical settings, selexipag is initiated across all risk groups, predominantly as triple therapy. Only 45% of patients being at low/intermediate-low risk at selexipag initiation suggests an opportunity for more frequent patient monitoring and earlier treatment escalation, given that 4-strata risk assessment was prognostic for hospitalisations and survival in this contemporary PAH cohort. A graphical abstract is available with this article.
{"title":"Four-Strata Risk Assessment in Patients with Pulmonary Arterial Hypertension Treated with Selexipag in Real-World Settings (EXPOSURE Study).","authors":"Tobias J Lange, Pilar Escribano-Subias, Audrey Muller, Catarina C Fernandes, Martina Fontana, Tatiana Remenova, Stefan Söderberg, Sean Gaine","doi":"10.1007/s12325-024-02946-2","DOIUrl":"10.1007/s12325-024-02946-2","url":null,"abstract":"<p><strong>Introduction: </strong>Risk assessment can aid management of pulmonary arterial hypertension (PAH) and clinical decision-making. This analysis describes characteristics, treatment patterns and outcomes of patients with PAH, categorised by risk status at time of treatment escalation with selexipag in clinical settings.</p><p><strong>Methods: </strong>Patients initiating selexipag in the ongoing multicentre, prospective EXPOSURE (EUPAS19085) study were grouped as low, intermediate-low, intermediate-high or high risk of 1-year mortality according to the ESC/ERS 4-strata method.</p><p><strong>Results: </strong>As of November 2022, 77% (535/698) of patients initiating selexipag had data allowing for risk calculation; 14% (N = 76) were low, 31% (N = 168) intermediate-low, 34% (N = 182) intermediate-high and 20% (N = 109) high risk of 1-year mortality. Overall, patients were predominantly female (71%), with idiopathic/heritable PAH (56%) or PAH associated with connective tissue disease (CTD-PAH; 27%), median age of 60 years and prevalent (2 years) disease. From low to high risk, proportion of CTD-PAH and age increased (from 12%-40% and 46-68 years, respectively); time from diagnosis decreased and presence of cardiovascular risk factors increased. Most patients across risk groups (74-81%) initiated selexipag as part of triple oral combination therapy. Overall median (Q1, Q3) selexipag exposure duration was 10.1 (3.5, 24.1) months. Proportions of hospitalised patients increased with increasing risk group (16-42% from low to high, respectively); more hospitalisations were PAH-related for the high risk (71%) versus other risk groups (47-54%). Kaplan-Meier survival estimates were 98%, 98%, 93% and 80% at 1-year and 98%, 92%, 81% and 67% at 2-years, from low to high risk, respectively.</p><p><strong>Conclusions: </strong>In clinical settings, selexipag is initiated across all risk groups, predominantly as triple therapy. Only 45% of patients being at low/intermediate-low risk at selexipag initiation suggests an opportunity for more frequent patient monitoring and earlier treatment escalation, given that 4-strata risk assessment was prognostic for hospitalisations and survival in this contemporary PAH cohort. A graphical abstract is available with this article.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11349848/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141854526","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-01Epub Date: 2024-07-11DOI: 10.1007/s12325-024-02928-4
Claire N Harrison, Alessandro M Vannucchi, Christian Recher, Francesco Passamonti, Aaron T Gerds, Juan Carlos Hernandez-Boluda, Abdulraheem Yacoub, Shireen Sirhan, Catherine Ellis, Bharat Patel, Bryan Strouse, Uwe Platzbecker
Introduction: Some Janus kinase (JAK) inhibitors such as ruxolitinib and fedratinib do not address and may worsen anemia in patients with myelofibrosis. In these cases, the JAK inhibitor may be continued at a reduced dose in an effort to maintain splenic and symptom control, with supportive therapy and/or red blood cell (RBC) transfusions added to manage anemia. This post hoc descriptive analysis of the phase 3 SIMPLIFY-2 trial evaluated the relative benefits of this approach versus switching to the JAK1/JAK2/activin A receptor type 1 inhibitor momelotinib in patients for whom anemia management is a key consideration.
Methods: SIMPLIFY-2 was a randomized (2:1), open-label, phase 3 trial of momelotinib versus best available therapy (BAT; 88.5% continued ruxolitinib) in JAK inhibitor-experienced patients with myelofibrosis (n = 156). Patient subgroups (n = 105 each) were defined by either baseline (1) hemoglobin (Hb) of < 100 g/L or (2) non-transfusion independence (not meeting the criteria of no transfusions and no Hb of < 80 g/L for the previous 12 weeks); outcomes have been summarized descriptively.
Results: In both subgroups of interest, week 24 transfusion independence rates were higher with momelotinib versus BAT/ruxolitinib: baseline Hb of < 100 g/L, 22 (33.3%) versus 5 (12.8%); baseline non-transfusion independent, 25 (34.7%) versus 1 (3.0%). Mean Hb levels over time were also generally higher in both subgroups with momelotinib, despite median transfusion rates through week 24 with momelotinib being comparable to or lower than with BAT/ruxolitinib. Spleen and symptom response rates with momelotinib in these subgroups were comparable to the intent-to-treat population, while rates with BAT/ruxolitinib were lower.
Conclusion: In patients with moderate-to-severe anemia and/or in need of RBC transfusions, outcomes were improved by switching to momelotinib rather than continuing ruxolitinib and using anemia supportive therapies.
{"title":"Momelotinib versus Continued Ruxolitinib or Best Available Therapy in JAK Inhibitor-Experienced Patients with Myelofibrosis and Anemia: Subgroup Analysis of SIMPLIFY-2.","authors":"Claire N Harrison, Alessandro M Vannucchi, Christian Recher, Francesco Passamonti, Aaron T Gerds, Juan Carlos Hernandez-Boluda, Abdulraheem Yacoub, Shireen Sirhan, Catherine Ellis, Bharat Patel, Bryan Strouse, Uwe Platzbecker","doi":"10.1007/s12325-024-02928-4","DOIUrl":"10.1007/s12325-024-02928-4","url":null,"abstract":"<p><strong>Introduction: </strong>Some Janus kinase (JAK) inhibitors such as ruxolitinib and fedratinib do not address and may worsen anemia in patients with myelofibrosis. In these cases, the JAK inhibitor may be continued at a reduced dose in an effort to maintain splenic and symptom control, with supportive therapy and/or red blood cell (RBC) transfusions added to manage anemia. This post hoc descriptive analysis of the phase 3 SIMPLIFY-2 trial evaluated the relative benefits of this approach versus switching to the JAK1/JAK2/activin A receptor type 1 inhibitor momelotinib in patients for whom anemia management is a key consideration.</p><p><strong>Methods: </strong>SIMPLIFY-2 was a randomized (2:1), open-label, phase 3 trial of momelotinib versus best available therapy (BAT; 88.5% continued ruxolitinib) in JAK inhibitor-experienced patients with myelofibrosis (n = 156). Patient subgroups (n = 105 each) were defined by either baseline (1) hemoglobin (Hb) of < 100 g/L or (2) non-transfusion independence (not meeting the criteria of no transfusions and no Hb of < 80 g/L for the previous 12 weeks); outcomes have been summarized descriptively.</p><p><strong>Results: </strong>In both subgroups of interest, week 24 transfusion independence rates were higher with momelotinib versus BAT/ruxolitinib: baseline Hb of < 100 g/L, 22 (33.3%) versus 5 (12.8%); baseline non-transfusion independent, 25 (34.7%) versus 1 (3.0%). Mean Hb levels over time were also generally higher in both subgroups with momelotinib, despite median transfusion rates through week 24 with momelotinib being comparable to or lower than with BAT/ruxolitinib. Spleen and symptom response rates with momelotinib in these subgroups were comparable to the intent-to-treat population, while rates with BAT/ruxolitinib were lower.</p><p><strong>Conclusion: </strong>In patients with moderate-to-severe anemia and/or in need of RBC transfusions, outcomes were improved by switching to momelotinib rather than continuing ruxolitinib and using anemia supportive therapies.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov: NCT02101268.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11349857/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141578709","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-01Epub Date: 2024-08-07DOI: 10.1007/s12325-024-02945-3
Antti A Mäkitie, Rasheed Omobolaji Alabi, Laura Pulkki-Råback, Alhadi Almangush, Jonathan J Beitler, Nabil F Saba, Primož Strojan, Robert Takes, Orlando Guntinas-Lichius, Alfio Ferlito
Background: Patients with head and neck cancer (HNC) often demonstrate stress, distress, anxiety, depression, and are at risk for suicide. These affect their quality of life (QoL) but less attention has been given to psychological variables that may impact response to treatment.
Objectives: This study aims to systematically review publications during 2013-2023 to collate evidence on the effects of psychological variables on HNC treatment outcomes.
Methods: We searched Ovid Medline, PubMed, Scopus, and Web of Science for articles that examined psychological factors related to treatment outcomes in patients with HNC.
Results: There were 29 studies (5 before treatment, 2 during, 17 after, and 5 covering the whole management trajectory) including 362,766 patients. The psychological factors were either behavioral (adjustment and coping strategy, unrealistic ideas, self-blame), cognitive (elevated risk of psychiatric co-comorbidity), or emotional (distress, depression, anxiety, nervousness, and fear of disfigurement and complications). It was found that there was a relationship between depression and decreased survival in patients with HNC. Pretreatment pain was an independent predictor of decreased survival in a large sample of patients. The distress level was approximately 54%, emotional problems ranged between 10 and 44%, while financial difficulties were identified in 54% of the patients. Sixty-nine percent of patients were reported to have used at least one cost-coping strategy within 6 months after treatment initiation. During post-treatment period, depression increased from 15% at the baseline to 29%, while the fear of recurrence was found among at least 35% of patients.
Discussion and conclusion: Several psychological factors predict QoL and survival among HNC survivors. Distress encompasses depression and anxiety, and physical burden from HNC diagnosis and treatment. Routine screening and early interventions that target distress could improve HNC survivors' QoL. A systematic and standardized measurement approach for QoL is warranted to homogenize these findings and to understand the underlying relationships.
{"title":"Psychological Factors Related to Treatment Outcomes in Head and Neck Cancer.","authors":"Antti A Mäkitie, Rasheed Omobolaji Alabi, Laura Pulkki-Råback, Alhadi Almangush, Jonathan J Beitler, Nabil F Saba, Primož Strojan, Robert Takes, Orlando Guntinas-Lichius, Alfio Ferlito","doi":"10.1007/s12325-024-02945-3","DOIUrl":"10.1007/s12325-024-02945-3","url":null,"abstract":"<p><strong>Background: </strong>Patients with head and neck cancer (HNC) often demonstrate stress, distress, anxiety, depression, and are at risk for suicide. These affect their quality of life (QoL) but less attention has been given to psychological variables that may impact response to treatment.</p><p><strong>Objectives: </strong>This study aims to systematically review publications during 2013-2023 to collate evidence on the effects of psychological variables on HNC treatment outcomes.</p><p><strong>Methods: </strong>We searched Ovid Medline, PubMed, Scopus, and Web of Science for articles that examined psychological factors related to treatment outcomes in patients with HNC.</p><p><strong>Results: </strong>There were 29 studies (5 before treatment, 2 during, 17 after, and 5 covering the whole management trajectory) including 362,766 patients. The psychological factors were either behavioral (adjustment and coping strategy, unrealistic ideas, self-blame), cognitive (elevated risk of psychiatric co-comorbidity), or emotional (distress, depression, anxiety, nervousness, and fear of disfigurement and complications). It was found that there was a relationship between depression and decreased survival in patients with HNC. Pretreatment pain was an independent predictor of decreased survival in a large sample of patients. The distress level was approximately 54%, emotional problems ranged between 10 and 44%, while financial difficulties were identified in 54% of the patients. Sixty-nine percent of patients were reported to have used at least one cost-coping strategy within 6 months after treatment initiation. During post-treatment period, depression increased from 15% at the baseline to 29%, while the fear of recurrence was found among at least 35% of patients.</p><p><strong>Discussion and conclusion: </strong>Several psychological factors predict QoL and survival among HNC survivors. Distress encompasses depression and anxiety, and physical burden from HNC diagnosis and treatment. Routine screening and early interventions that target distress could improve HNC survivors' QoL. A systematic and standardized measurement approach for QoL is warranted to homogenize these findings and to understand the underlying relationships.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11349815/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141896476","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-01Epub Date: 2024-07-25DOI: 10.1007/s12325-024-02939-1
Marcus-Alexander Wörns, Danielle Burns, Michael Paskow, Harriet Makin, Jordan Miller, Lucy M Turner, Janvi Sah
Introduction: Understanding the patient journey of hepatocellular carcinoma (HCC) may inform future clinical decision-making and enhance the patient experience. The objectives of this study were to explore the patient experience of HCC in relation to treatment options, treatment decision-making and treatment goals throughout the disease journey. This study also aimed to determine the symptoms and impacts of HCC across early, intermediate and advanced HCC.
Methods: Semi-structured 60-min interviews were conducted with n = 50 patients with HCC and n = 12 healthcare professionals (HCPs) with experience of treating patients with HCC. Interview data were analyzed using directed content analysis techniques with a hybrid inductive and deductive approach. An assessment of conceptual saturation was conducted for patients' symptom experience.
Results: Patients described treatment decisions as mostly HCP-led. In this study, surgery/resection was the most frequently offered treatment option across the HCC journey, and most patients were satisfied with the treatment options presented to them. Overall, patients described extending their overall survival (OS) and preserving quality of life (QoL) as their most important treatment goals, with patients diagnosed with advanced/unresectable HCC prioritizing QoL. HCPs also prioritized OS and progression-free survival (PFS) though reported that QoL became more important as HCC progressed. Patients experienced various symptoms across the HCC journey including fatigue, nausea, appetite loss, diarrhea and pain.
Conclusion: Overall, HCPs and patients collaborate throughout the treatment journey regarding treatment decisions and shared treatment goals. OS is critically important to patients and HCPs, though treatment goals may change depending on various clinical factors.
导言:了解肝细胞癌(HCC)患者的病程可为未来的临床决策提供依据,并改善患者的就医体验。本研究的目的是探讨患者在整个疾病过程中与治疗方案、治疗决策和治疗目标相关的 HCC 体验。本研究还旨在确定早期、中期和晚期 HCC 的症状和影响:对 n = 50 名 HCC 患者和 n = 12 名具有治疗 HCC 患者经验的医疗保健专业人员 (HCP) 进行了 60 分钟的半结构式访谈。访谈数据采用归纳和演绎混合法的定向内容分析技术进行分析。对患者的症状体验进行了概念饱和度评估:结果:患者描述的治疗决定大多由医生主导。在本研究中,手术/切除是HCC治疗过程中最常提供的治疗方案,大多数患者对提供给他们的治疗方案感到满意。总体而言,患者认为延长总生存期(OS)和保持生活质量(QoL)是他们最重要的治疗目标,而确诊为晚期/不可切除 HCC 的患者则将 QoL 放在首位。HCPs也将OS和无进展生存期(PFS)放在首位,但报告称随着HCC的进展,QoL变得更加重要。患者在整个 HCC 病程中会出现各种症状,包括疲劳、恶心、食欲不振、腹泻和疼痛:总的来说,在整个治疗过程中,医护人员和患者会就治疗决策和共同的治疗目标进行合作。尽管治疗目标可能会因各种临床因素而改变,但对患者和 HCP 而言,OS 至关重要。
{"title":"Patient Experience of Hepatocellular Carcinoma and Their Treatment Goals: An International Qualitative Study and Patient Journey Map.","authors":"Marcus-Alexander Wörns, Danielle Burns, Michael Paskow, Harriet Makin, Jordan Miller, Lucy M Turner, Janvi Sah","doi":"10.1007/s12325-024-02939-1","DOIUrl":"10.1007/s12325-024-02939-1","url":null,"abstract":"<p><strong>Introduction: </strong>Understanding the patient journey of hepatocellular carcinoma (HCC) may inform future clinical decision-making and enhance the patient experience. The objectives of this study were to explore the patient experience of HCC in relation to treatment options, treatment decision-making and treatment goals throughout the disease journey. This study also aimed to determine the symptoms and impacts of HCC across early, intermediate and advanced HCC.</p><p><strong>Methods: </strong>Semi-structured 60-min interviews were conducted with n = 50 patients with HCC and n = 12 healthcare professionals (HCPs) with experience of treating patients with HCC. Interview data were analyzed using directed content analysis techniques with a hybrid inductive and deductive approach. An assessment of conceptual saturation was conducted for patients' symptom experience.</p><p><strong>Results: </strong>Patients described treatment decisions as mostly HCP-led. In this study, surgery/resection was the most frequently offered treatment option across the HCC journey, and most patients were satisfied with the treatment options presented to them. Overall, patients described extending their overall survival (OS) and preserving quality of life (QoL) as their most important treatment goals, with patients diagnosed with advanced/unresectable HCC prioritizing QoL. HCPs also prioritized OS and progression-free survival (PFS) though reported that QoL became more important as HCC progressed. Patients experienced various symptoms across the HCC journey including fatigue, nausea, appetite loss, diarrhea and pain.</p><p><strong>Conclusion: </strong>Overall, HCPs and patients collaborate throughout the treatment journey regarding treatment decisions and shared treatment goals. OS is critically important to patients and HCPs, though treatment goals may change depending on various clinical factors.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11349831/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141756584","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-01Epub Date: 2024-08-06DOI: 10.1007/s12325-024-02923-9
Edouard Louis, Wan-Ju Lee, Leighann Litcher-Kelly, Sarah Ollis, Emma Pranschke, Kristina Fitzgerald, Ana Paula Lacerda, Ezequiel Neimark, Yuri Sanchez Gonzalez, Julian Panés
Introduction: Individuals living with Crohn's disease (CD) experience burdensome symptoms. As such, it is important to measure CD symptom severity in clinical research. The goal of this study was to evaluate the content validity, psychometric performance, and score interpretability of a new patient-reported instrument, the Crohn's Symptom Severity (CSS) questionnaire, among adolescents and adults with moderately to severely active CD.
Methods: Cognitive debriefing interviews (N = 30; n = 20 adults, n = 10 adolescents) were conducted to evaluate the content validity of the CSS. Additionally, the CSS scores were evaluated for reliability and validity using data from a phase 3 randomized clinical trial of risankizumab (NCT03105128; N = 850). Meaningful within-patient change (MWPC) thresholds were estimated using anchor-based methods.
Results: All interview participants (n = 30/30, 100.00%) reported the CSS was easy to complete and most participants (n = 28/29, 96.55%) reported that the CSS was relevant to their experience of CD. Among the clinical trial subjects (N = 850) the following was found for the CSS: mostly acceptable item-total correlations (0.26-0.79); weak to moderate inter-item correlations (r = 0.07-0.57), good internal consistency (Cronbach's α = 0.76-0.87); intraclass correlation coefficients ranged from 0.48 to 0.70, not consistently exceeding the acceptable range for test-retest reliability (0.70); acceptable convergent validity and known-groups results; and demonstrated sensitivity to change. Analyses supported an MWPC estimate of 6-11 points.
Conclusions: This study supports use of the CSS for measuring CD symptoms and sleep impact among adolescents and adults aged 16 and older with moderately to severely active CD in clinical research.
Trial registration: NCT03105128 (registration date 4 April 2017).
{"title":"Content Validity and Psychometric Evaluation of the Crohn's Symptom Severity (CSS) Questionnaire in Patients with Moderately to Severely Active Crohn's Disease.","authors":"Edouard Louis, Wan-Ju Lee, Leighann Litcher-Kelly, Sarah Ollis, Emma Pranschke, Kristina Fitzgerald, Ana Paula Lacerda, Ezequiel Neimark, Yuri Sanchez Gonzalez, Julian Panés","doi":"10.1007/s12325-024-02923-9","DOIUrl":"10.1007/s12325-024-02923-9","url":null,"abstract":"<p><strong>Introduction: </strong>Individuals living with Crohn's disease (CD) experience burdensome symptoms. As such, it is important to measure CD symptom severity in clinical research. The goal of this study was to evaluate the content validity, psychometric performance, and score interpretability of a new patient-reported instrument, the Crohn's Symptom Severity (CSS) questionnaire, among adolescents and adults with moderately to severely active CD.</p><p><strong>Methods: </strong>Cognitive debriefing interviews (N = 30; n = 20 adults, n = 10 adolescents) were conducted to evaluate the content validity of the CSS. Additionally, the CSS scores were evaluated for reliability and validity using data from a phase 3 randomized clinical trial of risankizumab (NCT03105128; N = 850). Meaningful within-patient change (MWPC) thresholds were estimated using anchor-based methods.</p><p><strong>Results: </strong>All interview participants (n = 30/30, 100.00%) reported the CSS was easy to complete and most participants (n = 28/29, 96.55%) reported that the CSS was relevant to their experience of CD. Among the clinical trial subjects (N = 850) the following was found for the CSS: mostly acceptable item-total correlations (0.26-0.79); weak to moderate inter-item correlations (r = 0.07-0.57), good internal consistency (Cronbach's α = 0.76-0.87); intraclass correlation coefficients ranged from 0.48 to 0.70, not consistently exceeding the acceptable range for test-retest reliability (0.70); acceptable convergent validity and known-groups results; and demonstrated sensitivity to change. Analyses supported an MWPC estimate of 6-11 points.</p><p><strong>Conclusions: </strong>This study supports use of the CSS for measuring CD symptoms and sleep impact among adolescents and adults aged 16 and older with moderately to severely active CD in clinical research.</p><p><strong>Trial registration: </strong>NCT03105128 (registration date 4 April 2017).</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11349822/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141892650","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Introduction: Automated bone age assessment (BAA) is of growing interest because of its accuracy and time efficiency in daily practice. In this study, we validated the clinical applicability of a commercially available artificial intelligence (AI)-powered X-ray bone age analyzer equipped with a deep learning-based automated BAA system and compared its performance with that of the Tanner-Whitehouse 3 (TW-3) method.
Methods: Radiographs prospectively collected from 30 centers across various regions in China, including 900 Chinese children and adolescents, were assessed independently by six doctors (three experts and three residents) and an AI analyzer for TW3 radius, ulna, and short bones (RUS) and TW3 carpal bone age. The experts' mean estimates were accepted as the gold standard. The performance of the AI analyzer was compared with that of each resident.
Results: For the estimation of TW3-RUS, the AI analyzer had a mean absolute error (MAE) of 0.48 ± 0.42. The percentage of patients with an absolute error of < 1.0 years was 86.78%. The MAE was significantly lower than that of rater 1 (0.54 ± 0.49, P = 0.0068); however, it was not significant for rater 2 (0.48 ± 0.48) or rater 3 (0.49 ± 0.46). For TW3 carpal, the AI analyzer had an MAE of 0.48 ± 0.65. The percentage of patients with an absolute error of < 1.0 years was 88.78%. The MAE was significantly lower than that of rater 2 (0.58 ± 0.67, P = 0.0018) and numerically lower for rater 1 (0.54 ± 0.64) and rater 3 (0.50 ± 0.53). These results were consistent for the subgroups according to sex, and differences between the age groups were observed.
Conclusion: In this comprehensive validation study conducted in China, an AI-powered X-ray bone age analyzer showed accuracies that matched or exceeded those of doctor raters. This method may improve the efficiency of clinical routines by reducing reading time without compromising accuracy.
{"title":"Validation of an AI-Powered Automated X-ray Bone Age Analyzer in Chinese Children and Adolescents: A Comparison with the Tanner-Whitehouse 3 Method.","authors":"Yan Liang, Xiaobo Chen, Rongxiu Zheng, Xinran Cheng, Zhe Su, Xiumin Wang, Hongwei Du, Min Zhu, Guimei Li, Yan Zhong, Shengquan Cheng, Baosheng Yu, Yu Yang, Ruimin Chen, Lanwei Cui, Hui Yao, Qiang Gu, Chunxiu Gong, Zhang Jun, Xiaoyan Huang, Deyun Liu, Xueqin Yan, Haiyan Wei, Yuwen Li, Huifeng Zhang, Yanjie Liu, Fengyun Wang, Gaixiu Zhang, Xin Fan, Hongmei Dai, Xiaoping Luo","doi":"10.1007/s12325-024-02944-4","DOIUrl":"10.1007/s12325-024-02944-4","url":null,"abstract":"<p><strong>Introduction: </strong>Automated bone age assessment (BAA) is of growing interest because of its accuracy and time efficiency in daily practice. In this study, we validated the clinical applicability of a commercially available artificial intelligence (AI)-powered X-ray bone age analyzer equipped with a deep learning-based automated BAA system and compared its performance with that of the Tanner-Whitehouse 3 (TW-3) method.</p><p><strong>Methods: </strong>Radiographs prospectively collected from 30 centers across various regions in China, including 900 Chinese children and adolescents, were assessed independently by six doctors (three experts and three residents) and an AI analyzer for TW3 radius, ulna, and short bones (RUS) and TW3 carpal bone age. The experts' mean estimates were accepted as the gold standard. The performance of the AI analyzer was compared with that of each resident.</p><p><strong>Results: </strong>For the estimation of TW3-RUS, the AI analyzer had a mean absolute error (MAE) of 0.48 ± 0.42. The percentage of patients with an absolute error of < 1.0 years was 86.78%. The MAE was significantly lower than that of rater 1 (0.54 ± 0.49, P = 0.0068); however, it was not significant for rater 2 (0.48 ± 0.48) or rater 3 (0.49 ± 0.46). For TW3 carpal, the AI analyzer had an MAE of 0.48 ± 0.65. The percentage of patients with an absolute error of < 1.0 years was 88.78%. The MAE was significantly lower than that of rater 2 (0.58 ± 0.67, P = 0.0018) and numerically lower for rater 1 (0.54 ± 0.64) and rater 3 (0.50 ± 0.53). These results were consistent for the subgroups according to sex, and differences between the age groups were observed.</p><p><strong>Conclusion: </strong>In this comprehensive validation study conducted in China, an AI-powered X-ray bone age analyzer showed accuracies that matched or exceeded those of doctor raters. This method may improve the efficiency of clinical routines by reducing reading time without compromising accuracy.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141858757","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Introduction: Atezolizumab, carboplatin, and etoposide (ACE) therapy is a standard of care for extensive-disease small cell lung cancer (SCLC); however, its safety data are scarce, limiting generalization to the Japanese population.
Methods: This study aimed to compare the safety of ACE versus carboplatin and etoposide (CE) therapies in Japanese patients using the Diagnosis Procedure Combination (DPC) database by comparing the incidence of adverse events (AEs). Retrospective data on clinical background and AEs were extracted from the DPC database. Incidence rates and restricted mean survival times (RMSTs) up to 6 months were analyzed for 19 clinically important AEs. Covariates were adjusted using the inverse probability weighting method.
Results: A total of 330,774 patients were identified using the International Statistical Classification of Diseases and Related Health Problems 10th Revision codes, of whom 277 were included in the ACE cohort and 478 in the CE cohort. Among the 19 AEs, the incidence of skin disorder and thyroid dysfunction was significantly higher in the ACE cohort compared with the CE cohort. The adjusted incidence rate ratios were 2.38 (95% confidence interval [CI] 1.04-5.43) for skin disorder and 6.92 (95% CI 2.00-23.89) for thyroid dysfunction. The adjusted RMST differences were - 8.2 days (95% CI - 16.0 to - 0.4 days) for skin disorder and - 8.8 days (95% CI - 15.7 to - 1.9 days) for thyroid dysfunction.
Conclusions: This study provides evidence regarding the safety of ACE combination therapy in Japanese clinical practice using the DPC database, with results comparable to those reported in pivotal clinical trials.
Trial registration: UMIN Clinical Trials Registry ID UMIN000041508.
简介:阿特珠单抗、卡铂和依托泊苷(ACE)疗法是治疗广泛病变小细胞肺癌(SCLC)的标准疗法;然而,其安全性数据很少,限制了在日本人群中的推广:本研究旨在利用诊断程序组合(DPC)数据库,通过比较不良事件(AEs)的发生率,比较ACE疗法与卡铂和依托泊苷(CE)疗法在日本患者中的安全性。从 DPC 数据库中提取了有关临床背景和 AEs 的回顾性数据。分析了19种临床重要不良反应的发生率和长达6个月的受限平均生存时间(RMST)。使用反概率加权法对协变量进行了调整:使用《疾病和有关健康问题的国际统计分类》第 10 次修订版代码共确定了 330,774 名患者,其中 277 人被纳入 ACE 队列,478 人被纳入 CE 队列。在19种AE中,ACE队列中皮肤病和甲状腺功能障碍的发病率明显高于CE队列。调整后的发病率比分别为:皮肤病 2.38(95% 置信区间 [CI] 1.04-5.43),甲状腺功能障碍 6.92(95% CI 2.00-23.89)。调整后的RMST差异为:皮肤病-8.2天(95% CI - 16.0至-0.4天),甲状腺功能障碍-8.8天(95% CI - 15.7至-1.9天):这项研究利用DPC数据库为日本临床实践中ACE联合疗法的安全性提供了证据,其结果与关键临床试验报告的结果相当:试验注册:UMIN 临床试验注册编号 UMIN000041508。
{"title":"A DPC Database Study on the Safety of Atezolizumab/Carboplatin/Etoposide in Extensive-Disease Small Cell Lung Cancer in Japanese Patients.","authors":"Motohiro Tamiya, Shunichiro Iwasawa, Yusuke Sasaki, Kosei Tajima, Yasutaka Chiba","doi":"10.1007/s12325-024-02905-x","DOIUrl":"10.1007/s12325-024-02905-x","url":null,"abstract":"<p><strong>Introduction: </strong>Atezolizumab, carboplatin, and etoposide (ACE) therapy is a standard of care for extensive-disease small cell lung cancer (SCLC); however, its safety data are scarce, limiting generalization to the Japanese population.</p><p><strong>Methods: </strong>This study aimed to compare the safety of ACE versus carboplatin and etoposide (CE) therapies in Japanese patients using the Diagnosis Procedure Combination (DPC) database by comparing the incidence of adverse events (AEs). Retrospective data on clinical background and AEs were extracted from the DPC database. Incidence rates and restricted mean survival times (RMSTs) up to 6 months were analyzed for 19 clinically important AEs. Covariates were adjusted using the inverse probability weighting method.</p><p><strong>Results: </strong>A total of 330,774 patients were identified using the International Statistical Classification of Diseases and Related Health Problems 10th Revision codes, of whom 277 were included in the ACE cohort and 478 in the CE cohort. Among the 19 AEs, the incidence of skin disorder and thyroid dysfunction was significantly higher in the ACE cohort compared with the CE cohort. The adjusted incidence rate ratios were 2.38 (95% confidence interval [CI] 1.04-5.43) for skin disorder and 6.92 (95% CI 2.00-23.89) for thyroid dysfunction. The adjusted RMST differences were - 8.2 days (95% CI - 16.0 to - 0.4 days) for skin disorder and - 8.8 days (95% CI - 15.7 to - 1.9 days) for thyroid dysfunction.</p><p><strong>Conclusions: </strong>This study provides evidence regarding the safety of ACE combination therapy in Japanese clinical practice using the DPC database, with results comparable to those reported in pivotal clinical trials.</p><p><strong>Trial registration: </strong>UMIN Clinical Trials Registry ID UMIN000041508.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11349850/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141589441","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-01DOI: 10.1007/s12325-024-02930-w
Sidney H Kennedy
{"title":"Correction: Beyond Response: Aiming for Quality Remission in Depression.","authors":"Sidney H Kennedy","doi":"10.1007/s12325-024-02930-w","DOIUrl":"10.1007/s12325-024-02930-w","url":null,"abstract":"","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11349771/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141589442","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-01Epub Date: 2024-07-30DOI: 10.1007/s12325-024-02897-8
Katherine L Gooch, Ivana Audhya, Kristen Ricchetti-Masterson, Shelagh M Szabo
Introduction: Insurance claims data and electronic health records (EHRs) have been used to characterize Duchenne muscular dystrophy (DMD) in real-world populations. The ability to assess patient-level DMD disease progression within insurance claims or EHR data infrastructures is unknown. Insurance claims and EHR data were comprehensively examined for availability and reliability of DMD outcomes that describe functional status and disease progression at the individual patient level over time.
Methods: MarketScan Commercial and Medicaid claims, and EHR-linked Clarivate open claims datasets were examined for data measuring 54 previously identified DMD-relevant outcomes in patients with DMD. Each outcome was assigned to one of five categories: functional and clinical events, clinical measures, biomarkers, functional measures, or patient-reported outcomes (PROs). Patients were identified using published coding algorithms. Annual 5-year attrition and data availability for each outcome was determined. The ability to distinguish disease severity and identify test results was also considered where applicable.
Results: A total of 1964 (MarketScan Commercial), 2007 (MarketScan Medicaid), and 10,639 (Clarivate) patients were identified. At 5 years, 31.7%, 35.1%, and 59.1% of patients remained in MarketScan Commercial, MarketScan Medicaid, and Clarivate, respectively. Claims were available for five of six functional and clinical events, with 45.5% (MarketScan Commercial), 48.0% (Clarivate), and 48.5% (MarketScan Medicaid) of patients with ≥ 1 claim for the most frequently identified clinical event (cardiomyopathy diagnosis). No data were available to describe frequency of wheelchair use or loss of ambulation. Very limited EHR data (≤ 2% of patients) were available to indicate tests were ordered for clinical measures, biomarkers, or functional assessments. No PRO notes or scores were observed. Data existed for inferring disease severity (e.g., hospitalization for cardiomyopathy); however, it was not apparent whether these events were incident.
Conclusion: Insurance claims and EHR-linked open claims data are of limited utility for holistically evaluating the progression and burden of DMD in individual patients.
{"title":"Current Challenges of Using Patient-Level Claims and Electronic Health Record Data for the Longitudinal Evaluation of Duchenne Muscular Dystrophy Outcomes.","authors":"Katherine L Gooch, Ivana Audhya, Kristen Ricchetti-Masterson, Shelagh M Szabo","doi":"10.1007/s12325-024-02897-8","DOIUrl":"10.1007/s12325-024-02897-8","url":null,"abstract":"<p><strong>Introduction: </strong>Insurance claims data and electronic health records (EHRs) have been used to characterize Duchenne muscular dystrophy (DMD) in real-world populations. The ability to assess patient-level DMD disease progression within insurance claims or EHR data infrastructures is unknown. Insurance claims and EHR data were comprehensively examined for availability and reliability of DMD outcomes that describe functional status and disease progression at the individual patient level over time.</p><p><strong>Methods: </strong>MarketScan Commercial and Medicaid claims, and EHR-linked Clarivate open claims datasets were examined for data measuring 54 previously identified DMD-relevant outcomes in patients with DMD. Each outcome was assigned to one of five categories: functional and clinical events, clinical measures, biomarkers, functional measures, or patient-reported outcomes (PROs). Patients were identified using published coding algorithms. Annual 5-year attrition and data availability for each outcome was determined. The ability to distinguish disease severity and identify test results was also considered where applicable.</p><p><strong>Results: </strong>A total of 1964 (MarketScan Commercial), 2007 (MarketScan Medicaid), and 10,639 (Clarivate) patients were identified. At 5 years, 31.7%, 35.1%, and 59.1% of patients remained in MarketScan Commercial, MarketScan Medicaid, and Clarivate, respectively. Claims were available for five of six functional and clinical events, with 45.5% (MarketScan Commercial), 48.0% (Clarivate), and 48.5% (MarketScan Medicaid) of patients with ≥ 1 claim for the most frequently identified clinical event (cardiomyopathy diagnosis). No data were available to describe frequency of wheelchair use or loss of ambulation. Very limited EHR data (≤ 2% of patients) were available to indicate tests were ordered for clinical measures, biomarkers, or functional assessments. No PRO notes or scores were observed. Data existed for inferring disease severity (e.g., hospitalization for cardiomyopathy); however, it was not apparent whether these events were incident.</p><p><strong>Conclusion: </strong>Insurance claims and EHR-linked open claims data are of limited utility for holistically evaluating the progression and burden of DMD in individual patients.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11349826/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141854525","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}