Pub Date : 2024-10-16eCollection Date: 2024-01-01DOI: 10.1177/20406223241286677
Li Jin, Wensu Wang, Rong Zhang, Jianguo Shen, Yangyang Li, Yi Zhang
Background: Diabetic kidney disease (DKD) is a severe complication of diabetes mellitus and is associated with an increased risk of end-stage renal disease (ESRD) and cardiovascular events. Early diagnosis and monitoring of DKD are crucial for implementing appropriate interventions. This study aimed to investigate the relationship between serum renalase (RNLS) levels, DKD, and diabetic macroangiopathy in patients with type 2 diabetes mellitus (T2DM).
Objectives: This study aims to evaluate the diagnostic value of serum renalase levels in DKD and diabetic macroangiopathy.
Design: This is a retrospective case-control study.
Methods: A total of 233 participants were recruited for the study, including 115 T2DM patients without DKD or diabetic retinopathy, and 118 T2DM patients with DKD. Serum RNLS levels were measured using an enzyme-linked immunosorbent assay. Kidney function parameters and diabetic macroangiopathy risk factors were evaluated in relation to serum RNLS levels.
Results: Serum RNLS levels were significantly higher in DKD patients compared to T2DM controls (34.82 (31.68, 39.37) vs 30.52 (28.58, 33.16), p < 0.01). Multiple linear regression analysis indicated that kidney function parameters and carotid intima-media thickness were independently related to RNLS levels. The study population was divided into four groups: no DKD and no diabetic macroangiopathy, DKD without diabetic macroangiopathy, diabetic macroangiopathy without DKD, and both DKD and diabetic macroangiopathy. Analysis results showed that patients with both DKD and diabetic macroangiopathy had the highest RNLS levels. Receiver operating characteristic curve analysis demonstrated the diagnostic value of RNLS for DKD (0.76 (95% confidence interval (CI) = 0.70-0.82, p < 0.01)) and diabetic macroangiopathy (0.75 (95% CI = 0.66-0.84, p < 0.01)).
Conclusion: Circulating RNLS levels were significantly increased in patients with DKD and diabetic macroangiopathy, suggesting that RNLS may serve as an early diagnostic marker.
背景：糖尿病肾病（DKD）是糖尿病的一种严重并发症，与终末期肾病（ESRD）和心血管事件风险增加有关。早期诊断和监测 DKD 对实施适当的干预措施至关重要。本研究旨在探讨 2 型糖尿病（T2DM）患者血清肾酶（RNLS）水平、DKD 和糖尿病大血管病变之间的关系：本研究旨在评估血清肾酶水平在DKD和糖尿病大血管病变中的诊断价值：设计：这是一项回顾性病例对照研究：研究共招募了233名参与者，包括115名无DKD或糖尿病视网膜病变的T2DM患者和118名有DKD的T2DM患者。采用酶联免疫吸附测定法测量血清 RNLS 水平。评估了肾功能参数和糖尿病大血管病变风险因素与血清RNLS水平的关系：结果：与 T2DM 对照组相比，DKD 患者的血清 RNLS 水平明显更高（34.82 (31.68, 39.37) vs 30.52 (28.58, 33.16)，p p p 结论：DKD和糖尿病大血管病变患者的循环RNLS水平明显升高，表明RNLS可作为早期诊断标志物。
{"title":"The early diagnostic value of serum renalase level in diabetic kidney disease and diabetic macroangiopathy: a retrospective case-control study.","authors":"Li Jin, Wensu Wang, Rong Zhang, Jianguo Shen, Yangyang Li, Yi Zhang","doi":"10.1177/20406223241286677","DOIUrl":"10.1177/20406223241286677","url":null,"abstract":"<p><strong>Background: </strong>Diabetic kidney disease (DKD) is a severe complication of diabetes mellitus and is associated with an increased risk of end-stage renal disease (ESRD) and cardiovascular events. Early diagnosis and monitoring of DKD are crucial for implementing appropriate interventions. This study aimed to investigate the relationship between serum renalase (RNLS) levels, DKD, and diabetic macroangiopathy in patients with type 2 diabetes mellitus (T2DM).</p><p><strong>Objectives: </strong>This study aims to evaluate the diagnostic value of serum renalase levels in DKD and diabetic macroangiopathy.</p><p><strong>Design: </strong>This is a retrospective case-control study.</p><p><strong>Methods: </strong>A total of 233 participants were recruited for the study, including 115 T2DM patients without DKD or diabetic retinopathy, and 118 T2DM patients with DKD. Serum RNLS levels were measured using an enzyme-linked immunosorbent assay. Kidney function parameters and diabetic macroangiopathy risk factors were evaluated in relation to serum RNLS levels.</p><p><strong>Results: </strong>Serum RNLS levels were significantly higher in DKD patients compared to T2DM controls (34.82 (31.68, 39.37) vs 30.52 (28.58, 33.16), <i>p</i> < 0.01). Multiple linear regression analysis indicated that kidney function parameters and carotid intima-media thickness were independently related to RNLS levels. The study population was divided into four groups: no DKD and no diabetic macroangiopathy, DKD without diabetic macroangiopathy, diabetic macroangiopathy without DKD, and both DKD and diabetic macroangiopathy. Analysis results showed that patients with both DKD and diabetic macroangiopathy had the highest RNLS levels. Receiver operating characteristic curve analysis demonstrated the diagnostic value of RNLS for DKD (0.76 (95% confidence interval (CI) = 0.70-0.82, <i>p</i> < 0.01)) and diabetic macroangiopathy (0.75 (95% CI = 0.66-0.84, <i>p</i> < 0.01)).</p><p><strong>Conclusion: </strong>Circulating RNLS levels were significantly increased in patients with DKD and diabetic macroangiopathy, suggesting that RNLS may serve as an early diagnostic marker.</p>","PeriodicalId":22960,"journal":{"name":"Therapeutic Advances in Chronic Disease","volume":"15 ","pages":"20406223241286677"},"PeriodicalIF":3.3,"publicationDate":"2024-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11487514/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142475374","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}
Background: The criteria for remission in both clinical and pathological contexts for lupus nephritis (LN) remain controversial.
Objectives: To identify optimal short-term goals (remission criteria) for LN predicting long-term success at 5 years, using repeat kidney biopsy (Biopsy 2) and clinical data.
Design: Single-center observational study.
Methods: Twenty-three consecutive LN patients undergoing Biopsy 2 2 years post-induction therapy were evaluated. Two ideal long-term goals at 5 years were defined as: "A," Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) = 0 and prednisolone (PSL) ⩽5 mg/day, and "B," proteinuria ⩽0.2 g/day with a normal serum creatinine level and PSL ⩽5 mg/day. Histologically, the electron-dense deposit (EDD) score grades immune deposits based on their intensity, amount, and location. A score of ⩽1 was defined as "electron microscopy remission (ER)."
Results: Conventional renal indices failed to predict long-term goals. The short-term goals with an accuracy (area under the curve: 95% confidence interval) of ⩾0.8 predicted long-term goals: "A at 5 years" (A-5y), A-2y (0.91: 0.79-1.00), DORIS-R-2y (0.87: 0.72-1.00), EDD score (0.85: 0.70-1.00), B-2y (0.83: 0.66-0.99), and SLEDAI-R-2y (0.82: 0.66-0.98) as well as "B at 5 years" (B-5y), A-2y (0.87: 0.73-1.00), B-2y (0.87: 0.73-1.00), EDD score (0.85: 0.69-1.00), and DORIS-R-2y (0.83: 0.67-0.99). EDD scores predicted A-5y, B-5y, and PSL dose at 5 years in proportion to the score. The clinical and histological goals aligned.
Conclusion: The best predictive short-term goal was A-2y. Concordance between clinical remission (A-2y, B-2y, and DORIS-R-2y) and histological remission (ER) at 2 years suggests optimal short-term goals for LN.
{"title":"Optimal treatment targets for lupus nephritis using per-protocol repeat kidney biopsy findings at 2 years and clinical data up to 5 years: a single-center observational study.","authors":"Hidetoshi Kagawa, Ryutaro Yamanaka, Ai Matsubara, Tatsuyuki Inoue, Reika Hayashi, Natsuki Kubota, Tsutomu Hiromasa","doi":"10.1177/20406223241289074","DOIUrl":"https://doi.org/10.1177/20406223241289074","url":null,"abstract":"<p><strong>Background: </strong>The criteria for remission in both clinical and pathological contexts for lupus nephritis (LN) remain controversial.</p><p><strong>Objectives: </strong>To identify optimal short-term goals (remission criteria) for LN predicting long-term success at 5 years, using repeat kidney biopsy (Biopsy 2) and clinical data.</p><p><strong>Design: </strong>Single-center observational study.</p><p><strong>Methods: </strong>Twenty-three consecutive LN patients undergoing Biopsy 2 2 years post-induction therapy were evaluated. Two ideal long-term goals at 5 years were defined as: \"A,\" Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) = 0 and prednisolone (PSL) ⩽5 mg/day, and \"B,\" proteinuria ⩽0.2 g/day with a normal serum creatinine level and PSL ⩽5 mg/day. Histologically, the electron-dense deposit (EDD) score grades immune deposits based on their intensity, amount, and location. A score of ⩽1 was defined as \"electron microscopy remission (ER).\"</p><p><strong>Results: </strong>Conventional renal indices failed to predict long-term goals. The short-term goals with an accuracy (area under the curve: 95% confidence interval) of ⩾0.8 predicted long-term goals: \"A at 5 years\" (A-5y), A-2y (0.91: 0.79-1.00), DORIS-R-2y (0.87: 0.72-1.00), EDD score (0.85: 0.70-1.00), B-2y (0.83: 0.66-0.99), and SLEDAI-R-2y (0.82: 0.66-0.98) as well as \"B at 5 years\" (B-5y), A-2y (0.87: 0.73-1.00), B-2y (0.87: 0.73-1.00), EDD score (0.85: 0.69-1.00), and DORIS-R-2y (0.83: 0.67-0.99). EDD scores predicted A-5y, B-5y, and PSL dose at 5 years in proportion to the score. The clinical and histological goals aligned.</p><p><strong>Conclusion: </strong>The best predictive short-term goal was A-2y. Concordance between clinical remission (A-2y, B-2y, and DORIS-R-2y) and histological remission (ER) at 2 years suggests optimal short-term goals for LN.</p>","PeriodicalId":22960,"journal":{"name":"Therapeutic Advances in Chronic Disease","volume":"15 ","pages":"20406223241289074"},"PeriodicalIF":3.3,"publicationDate":"2024-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11483839/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142475373","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 : 2024-09-19DOI: 10.1177/20406223241274302
Yohannes W. Woldeamanuel, Bharati M. Sanjanwala, Robert P. Cowan
Background:Currently, there are no biomarkers for migraine.Objectives:We aimed to identify proteomic biomarker signatures for diagnosing, subclassifying, and predicting treatment response in migraine.Design:This is a cross-sectional and longitudinal study of untargeted serum and cerebrospinal fluid (CSF) proteomics in episodic migraine (EM; n = 26), chronic migraine (CM; n = 26), and healthy controls (HC; n = 26).Methods:We developed classification models for biomarker identification and natural clusters through unsupervised classification using agglomerative hierarchical clustering (AHC). Pathway analysis of differentially expressed proteins was performed.Results:Of 405 CSF proteins, the top five proteins that discriminated between migraine patients and HC were angiotensinogen, cell adhesion molecule 3, immunoglobulin heavy variable (IGHV) V-III region JON, insulin-like growth factor binding protein 6 (IGFBP-6), and IGFBP-7. The top-performing classifier demonstrated 100% sensitivity and 75% specificity in differentiating the two groups. Of 229 serum proteins, the top five proteins in classifying patients with migraine were immunoglobulin heavy variable 3-74 (IGHV 3-74), proteoglycan 4, immunoglobulin kappa variable 3D-15, zinc finger protein (ZFP)-814, and mediator of RNA polymerase II transcription subunit 12. The best-performing classifier exhibited 94% sensitivity and 92% specificity. AHC separated EM, CM, and HC into distinct clusters with 90% success. Migraine patients exhibited increased ZFP-814 and calcium voltage-gated channel subunit alpha 1F (CACNA1F) levels, while IGHV 3-74 levels decreased in both cross-sectional and longitudinal serum analyses. ZFP-814 remained upregulated during the CM-to-EM reversion but was suppressed when CM persisted. CACNA1F was pronounced in CM persistence. Pathway analysis revealed immune, coagulation, glucose metabolism, erythrocyte oxygen and carbon dioxide exchange, and insulin-like growth factor regulation pathways.Conclusion:Our data-driven study provides evidence for identifying novel proteomic biomarker signatures to diagnose, subclassify, and predict treatment responses for migraine. The dysregulated biomolecules affect multiple pathways, leading to cortical spreading depression, trigeminal nociceptor sensitization, oxidative stress, blood–brain barrier disruption, immune response, and coagulation cascades.Trial registration:NCT03231241, ClincialTrials.gov.
{"title":"Deep and unbiased proteomics, pathway enrichment analysis, and protein–protein interaction of biomarker signatures in migraine","authors":"Yohannes W. Woldeamanuel, Bharati M. Sanjanwala, Robert P. Cowan","doi":"10.1177/20406223241274302","DOIUrl":"https://doi.org/10.1177/20406223241274302","url":null,"abstract":"Background:Currently, there are no biomarkers for migraine.Objectives:We aimed to identify proteomic biomarker signatures for diagnosing, subclassifying, and predicting treatment response in migraine.Design:This is a cross-sectional and longitudinal study of untargeted serum and cerebrospinal fluid (CSF) proteomics in episodic migraine (EM; n = 26), chronic migraine (CM; n = 26), and healthy controls (HC; n = 26).Methods:We developed classification models for biomarker identification and natural clusters through unsupervised classification using agglomerative hierarchical clustering (AHC). Pathway analysis of differentially expressed proteins was performed.Results:Of 405 CSF proteins, the top five proteins that discriminated between migraine patients and HC were angiotensinogen, cell adhesion molecule 3, immunoglobulin heavy variable (IGHV) V-III region JON, insulin-like growth factor binding protein 6 (IGFBP-6), and IGFBP-7. The top-performing classifier demonstrated 100% sensitivity and 75% specificity in differentiating the two groups. Of 229 serum proteins, the top five proteins in classifying patients with migraine were immunoglobulin heavy variable 3-74 (IGHV 3-74), proteoglycan 4, immunoglobulin kappa variable 3D-15, zinc finger protein (ZFP)-814, and mediator of RNA polymerase II transcription subunit 12. The best-performing classifier exhibited 94% sensitivity and 92% specificity. AHC separated EM, CM, and HC into distinct clusters with 90% success. Migraine patients exhibited increased ZFP-814 and calcium voltage-gated channel subunit alpha 1F (CACNA1F) levels, while IGHV 3-74 levels decreased in both cross-sectional and longitudinal serum analyses. ZFP-814 remained upregulated during the CM-to-EM reversion but was suppressed when CM persisted. CACNA1F was pronounced in CM persistence. Pathway analysis revealed immune, coagulation, glucose metabolism, erythrocyte oxygen and carbon dioxide exchange, and insulin-like growth factor regulation pathways.Conclusion:Our data-driven study provides evidence for identifying novel proteomic biomarker signatures to diagnose, subclassify, and predict treatment responses for migraine. The dysregulated biomolecules affect multiple pathways, leading to cortical spreading depression, trigeminal nociceptor sensitization, oxidative stress, blood–brain barrier disruption, immune response, and coagulation cascades.Trial registration:NCT03231241, ClincialTrials.gov.","PeriodicalId":22960,"journal":{"name":"Therapeutic Advances in Chronic Disease","volume":"118 1","pages":""},"PeriodicalIF":3.5,"publicationDate":"2024-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142260587","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}
Background: Ultrasound therapy is a non-invasive technique used to address a variety of health issues.
Objectives: This systematic review and meta-analysis aim to assess the effectiveness of ultrasound therapy in alleviating pain associated with musculoskeletal diseases.
Design: This study was conducted following PRISMA guidelines, with relevant articles identified through comprehensive searches in electronic databases.
Data sources and methods: We conducted searches across multiple databases, including Scopus, PubMed, MEDLINE, ProQuest, Science Direct, CINAHL, AIM, and ELDIS. Two independent reviewers screened the titles and abstracts of the retrieved articles. We included randomized controlled trials (RCTs) and observational cohort studies published between 2010 and 2023 that evaluated ultrasound therapy for knee and shoulder skeletal disorders. The selected data were analyzed qualitatively and synthesized, with the risk of bias assessed using the RoB2 tool.
Results: Initially, 117 articles were reviewed using the search strategy, and 10 trials that met the inclusion criteria were identified. In seven of these studies, the primary musculoskeletal disorder was osteoarthritis, while three studies focused on shoulder pain and impingement. Most studies indicated that ultrasound therapy significantly reduced pain. The meta-analysis showed that ultrasound therapy was significantly more effective than other interventions for knee disorders (I2 = 51%, Z = 2.65, p = 0.008). However, for shoulder disorders, both ultrasound and other intervention methods were found to be ineffective (I2 = 93%, Z = 0.73, p = 0.46).
Conclusion: The current evidence supports the effectiveness of ultrasound therapy in reducing pain and aiding rehabilitation for knee conditions. However, there are mixed results regarding its efficacy for shoulder conditions, highlighting the need for further research in this area.
{"title":"Ultrasound therapy for pain reduction in musculoskeletal disorders: a systematic review and meta-analysis.","authors":"Haiying Guan, Yilun Wu, Xiangyang Wang, Bo Liu, Ting Yan, Razzagh Abedi-Firouzjah","doi":"10.1177/20406223241267217","DOIUrl":"10.1177/20406223241267217","url":null,"abstract":"<p><strong>Background: </strong>Ultrasound therapy is a non-invasive technique used to address a variety of health issues.</p><p><strong>Objectives: </strong>This systematic review and meta-analysis aim to assess the effectiveness of ultrasound therapy in alleviating pain associated with musculoskeletal diseases.</p><p><strong>Design: </strong>This study was conducted following PRISMA guidelines, with relevant articles identified through comprehensive searches in electronic databases.</p><p><strong>Data sources and methods: </strong>We conducted searches across multiple databases, including Scopus, PubMed, MEDLINE, ProQuest, Science Direct, CINAHL, AIM, and ELDIS. Two independent reviewers screened the titles and abstracts of the retrieved articles. We included randomized controlled trials (RCTs) and observational cohort studies published between 2010 and 2023 that evaluated ultrasound therapy for knee and shoulder skeletal disorders. The selected data were analyzed qualitatively and synthesized, with the risk of bias assessed using the RoB2 tool.</p><p><strong>Results: </strong>Initially, 117 articles were reviewed using the search strategy, and 10 trials that met the inclusion criteria were identified. In seven of these studies, the primary musculoskeletal disorder was osteoarthritis, while three studies focused on shoulder pain and impingement. Most studies indicated that ultrasound therapy significantly reduced pain. The meta-analysis showed that ultrasound therapy was significantly more effective than other interventions for knee disorders (<i>I</i> <sup>2</sup> = 51%, <i>Z</i> = 2.65, <i>p</i> = 0.008). However, for shoulder disorders, both ultrasound and other intervention methods were found to be ineffective (<i>I</i> <sup>2</sup> = 93%, <i>Z</i> = 0.73, <i>p</i> = 0.46).</p><p><strong>Conclusion: </strong>The current evidence supports the effectiveness of ultrasound therapy in reducing pain and aiding rehabilitation for knee conditions. However, there are mixed results regarding its efficacy for shoulder conditions, highlighting the need for further research in this area.</p>","PeriodicalId":22960,"journal":{"name":"Therapeutic Advances in Chronic Disease","volume":"15 ","pages":"20406223241267217"},"PeriodicalIF":3.3,"publicationDate":"2024-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11337181/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142018697","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 : 2024-07-31DOI: 10.1177/20406223241264477
Melissa Basile, Jennifer Polo, Katherine Henthorne, Joan DeCelie-Germana, Susan Galvin, Janice Wang
Background:Elexacaftor/tezacaftor/ivacaftor (ETI) has reduced many symptoms of cystic fibrosis (CF).Objectives:We sought to identify the impact of ETI on both symptoms and treatment decisions among adults with CF.Design:Participants were enrolled in a cross-sectional study. Surveys were sent via a RedCap link. Semistructured interviews were administered remotely via Microsoft Teams. Interviews were audio recorded and professionally transcribed.Methods:We assessed Cystic Fibrosis Questionnaire-Revised (CFQ-R) subscales for physical, respiratory, emotion, and treatment, and analyzed semistructured interviews covering CF treatment regimens and daily living. Quantitative and qualitative results were analyzed separately and via a mixed-methods convergence coding matrix.Results:Twenty-four adults with CF taking ETI were included. CFQ-R subscale scores (mean scores/standard deviation) were physical (82.1/22.8), respiratory (83.7/11.2), emotion (65.3/14.2), and treatment (57.5/20.1). Three themes about decision-making for non-ETI-treatments emerged: (1) How I’m feeling, (2) Not noticing a difference, and (3) Uncertainty about long-term impact of modifying treatment regimens, and we found participants weighed each of these factors in their treatment decisions. Key findings from mixed-methods analysis show that among individuals experiencing higher CFQ-R scores for physical and respiratory compared to emotion and treatment, there were statements indicating that while those participants were experiencing better physical health, many continued their burdensome treatment regimens.Conclusion:With little long-term data on the impact of reducing non-ETI treatments, participants weighed how they were feeling, treatment efficacy beliefs, and risk tolerance when making treatment decisions.
背景：Elexacaftor/tezacaftor/ivacaftor（ETI）减轻了囊性纤维化（CF）的许多症状。目标：我们试图确定 ETI 对 CF 成人患者的症状和治疗决策的影响。调查问卷通过 RedCap 链接发送。半结构式访谈通过 Microsoft Teams 进行远程管理。方法：我们评估了囊性纤维化问卷-修订版（CFQ-R）中有关身体、呼吸、情绪和治疗的分量表，并分析了涉及 CF 治疗方案和日常生活的半结构式访谈。对定量和定性结果分别进行了分析，并通过混合方法聚合编码矩阵进行了分析。CFQ-R分量表的得分（平均分/标准差）分别为身体（82.1/22.8）、呼吸（83.7/11.2）、情绪（65.3/14.2）和治疗（57.5/20.1）。关于非 ETI 治疗的决策出现了三个主题：(1) 我的感觉；(2) 没有注意到区别；(3) 不确定修改治疗方案的长期影响。混合方法分析的主要结果显示，与情绪和治疗相比，身体和呼吸方面的 CFQ-R 得分较高的人中，有一些陈述表明，虽然这些参与者的身体状况有所改善，但许多人仍在继续他们繁重的治疗方案。结论：由于有关减少非ETI 治疗影响的长期数据很少，参与者在做出治疗决定时会权衡他们的感觉、治疗效果信念和风险承受能力。
{"title":"The impact of elexacaftor/tezacaftor/ivacaftor on cystic fibrosis health-related quality of life and decision-making about daily treatment regimens: a mixed methods exploratory study","authors":"Melissa Basile, Jennifer Polo, Katherine Henthorne, Joan DeCelie-Germana, Susan Galvin, Janice Wang","doi":"10.1177/20406223241264477","DOIUrl":"https://doi.org/10.1177/20406223241264477","url":null,"abstract":"Background:Elexacaftor/tezacaftor/ivacaftor (ETI) has reduced many symptoms of cystic fibrosis (CF).Objectives:We sought to identify the impact of ETI on both symptoms and treatment decisions among adults with CF.Design:Participants were enrolled in a cross-sectional study. Surveys were sent via a RedCap link. Semistructured interviews were administered remotely via Microsoft Teams. Interviews were audio recorded and professionally transcribed.Methods:We assessed Cystic Fibrosis Questionnaire-Revised (CFQ-R) subscales for physical, respiratory, emotion, and treatment, and analyzed semistructured interviews covering CF treatment regimens and daily living. Quantitative and qualitative results were analyzed separately and via a mixed-methods convergence coding matrix.Results:Twenty-four adults with CF taking ETI were included. CFQ-R subscale scores (mean scores/standard deviation) were physical (82.1/22.8), respiratory (83.7/11.2), emotion (65.3/14.2), and treatment (57.5/20.1). Three themes about decision-making for non-ETI-treatments emerged: (1) How I’m feeling, (2) Not noticing a difference, and (3) Uncertainty about long-term impact of modifying treatment regimens, and we found participants weighed each of these factors in their treatment decisions. Key findings from mixed-methods analysis show that among individuals experiencing higher CFQ-R scores for physical and respiratory compared to emotion and treatment, there were statements indicating that while those participants were experiencing better physical health, many continued their burdensome treatment regimens.Conclusion:With little long-term data on the impact of reducing non-ETI treatments, participants weighed how they were feeling, treatment efficacy beliefs, and risk tolerance when making treatment decisions.","PeriodicalId":22960,"journal":{"name":"Therapeutic Advances in Chronic Disease","volume":"3 1","pages":""},"PeriodicalIF":3.5,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141862979","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}
Pub Date : 2024-07-31DOI: 10.1177/20406223241264539
Saima Ajaz, Mark Chamley, James Lok, Riham Soliman, Reece Khan, Karan Ahir, Monique Curtis, María Fernanda Guerra-Veloz, Kosh Agarwal
Background:Metabolic associated fatty liver disease (MAFLD) stands as the leading cause of chronic liver disease globally. Notably, individuals with metabolic risk factors, such as diabetes and obesity, exhibit a staggering prevalence of MAFLD, with estimates reaching up to 70%. However, despite its widespread occurrence, there’s a noticeable gap in understanding and awareness about MAFLD among these high-risk groups.Objectives:The main objective of this study was to assess the awareness and prevalence of MAFLD among diabetic patients who regularly receive secondary care focusing particularly on how multiethnic backgrounds and associated lifestyle preferences influence these health outcomes.Design:Cross-sectional study.Methods:Patients with type 2 diabetes (T2D) who regularly attend Lambeth Diabetes Intermediate Care Team clinics were invited to undergo MAFLD screening using FibroScan. Those who agreed to participate were provided with structured questionnaires on diet, physical activity, and MAFLD knowledge by a hepatologist. For each participant, anthropometric data, medical history, liver stiffness measurement, and controlled attenuation parameter (CAP) were documented. Steatosis was identified with a CAP value of ⩾275 dB/m, and advanced fibrosis was flagged at values of ⩾8 kPa.Results:The FibroScan data was valid in 96.4% (215), 53.5% (115/215) had steatosis and 26.2% (58/215) had liver fibrosis in this multiethnic high-risk group. Awareness of MAFLD was notably low at 30.9%. Alarmingly, 69% of patients diagnosed with liver fibrosis were unfamiliar with the condition. Additionally, understanding of MAFLD showed variation among different ethnic groups with highest levels were demonstrated in the Caucasian/White population (46%). Majority (96%) of these subjects were receiving specific lifestyle advice from healthcare professionals due to metabolic conditions and comorbidities. However, most patients preferred diets that were rich in carbohydrates (65.8%) and only 43% subjects performed moderate exercise daily highlighting lack of understanding regarding MAFLD and lifestyle management.Conclusion:There’s a pressing need for increased awareness of MAFLD, especially in multiethnic high-risk groups. Additionally, the development of cost-effective strategies to stratify risk is essential to address this growing health concern.
{"title":"Lack of awareness and ethnic polarity is a major cause of metabolic associated fatty liver disease in high-risk diabetes population in South London","authors":"Saima Ajaz, Mark Chamley, James Lok, Riham Soliman, Reece Khan, Karan Ahir, Monique Curtis, María Fernanda Guerra-Veloz, Kosh Agarwal","doi":"10.1177/20406223241264539","DOIUrl":"https://doi.org/10.1177/20406223241264539","url":null,"abstract":"Background:Metabolic associated fatty liver disease (MAFLD) stands as the leading cause of chronic liver disease globally. Notably, individuals with metabolic risk factors, such as diabetes and obesity, exhibit a staggering prevalence of MAFLD, with estimates reaching up to 70%. However, despite its widespread occurrence, there’s a noticeable gap in understanding and awareness about MAFLD among these high-risk groups.Objectives:The main objective of this study was to assess the awareness and prevalence of MAFLD among diabetic patients who regularly receive secondary care focusing particularly on how multiethnic backgrounds and associated lifestyle preferences influence these health outcomes.Design:Cross-sectional study.Methods:Patients with type 2 diabetes (T2D) who regularly attend Lambeth Diabetes Intermediate Care Team clinics were invited to undergo MAFLD screening using FibroScan. Those who agreed to participate were provided with structured questionnaires on diet, physical activity, and MAFLD knowledge by a hepatologist. For each participant, anthropometric data, medical history, liver stiffness measurement, and controlled attenuation parameter (CAP) were documented. Steatosis was identified with a CAP value of ⩾275 dB/m, and advanced fibrosis was flagged at values of ⩾8 kPa.Results:The FibroScan data was valid in 96.4% (215), 53.5% (115/215) had steatosis and 26.2% (58/215) had liver fibrosis in this multiethnic high-risk group. Awareness of MAFLD was notably low at 30.9%. Alarmingly, 69% of patients diagnosed with liver fibrosis were unfamiliar with the condition. Additionally, understanding of MAFLD showed variation among different ethnic groups with highest levels were demonstrated in the Caucasian/White population (46%). Majority (96%) of these subjects were receiving specific lifestyle advice from healthcare professionals due to metabolic conditions and comorbidities. However, most patients preferred diets that were rich in carbohydrates (65.8%) and only 43% subjects performed moderate exercise daily highlighting lack of understanding regarding MAFLD and lifestyle management.Conclusion:There’s a pressing need for increased awareness of MAFLD, especially in multiethnic high-risk groups. Additionally, the development of cost-effective strategies to stratify risk is essential to address this growing health concern.","PeriodicalId":22960,"journal":{"name":"Therapeutic Advances in Chronic Disease","volume":"44 1","pages":""},"PeriodicalIF":3.5,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141862982","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}
Pub Date : 2024-07-25DOI: 10.1177/20406223241259654
Yaming Lu, Shanshan Xiong, Mengchen Zhang, Xiaoman Zu, Jinbin Li, Ren Mao, Zhirong Zeng, Xuehua Li, Minhu Chen, Yao He
Background:Transmural healing (TH) has emerged as a potential treatment goal for Crohn’s disease (CD). However, further research is needed to confirm its benefits and risk factors associated with TH remain unclear.Objectives:We aimed to assess the value of TH based on magnetic resonance enterography (MRE) in Chinese CD patients regarding the long-term outcomes and its associated factors.Design:Retrospective, observational cohort study.Methods:Patients with CD diagnosed by colonoscopy and MRE examination between 2015 and 2022 were included. All patients were evaluated with endoscopy together with MRE within 6–12 months after baseline and followed up for at least 6 months after evaluation. The primary endpoint was the occurrence of major outcomes during the follow-up, including drug escalation, hospitalization, and surgery. The cumulative probabilities of major outcomes were calculated using Kaplan–Meier survival curves. Logistic regression analyses were used to predict TH within 6–12 months after baseline.Results:A total of 175 patients were included in the study. Of these, 69 (39.4%) patients achieved mucosal healing (MH), but only 34 (19.4%) of them achieved TH. The median follow-up duration was 17.4 months (interquartile range, 11.6–25.5), and major outcomes occurred in 58.3% of patients. A lower occurrence rate of major outcomes was noted in patients who achieved TH than in those who achieved MH only ( p = 0.012). The baseline lymphocyte/C-reactive protein ratio (LCR) [odds ratio (OR), 1.60; 95% confidence interval (CI), 1.02–2.50; p = 0.039] and bowel wall thickness (BWT) (OR, 0.72; 95% CI, 0.59–0.90; p = 0.003) were independent predictors associated with TH. According to multivariate Cox regression analysis, low LCR [hazard ratio (HR), 2.34; 95% CI, 1.51–3.64; p < 0.001], and no healing (HR, 5.45; 95% CI, 2.28–13.00; p < 0.001) were associated with an increased risk of major outcomes.Conclusion:Patients with CD who achieved TH showed improved prognosis compared to those who achieved MH only. Baseline LCR and BWT might predict TH.
{"title":"Long-term outcomes and associated factors of Crohn’s disease patients achieving transmural healing based on magnetic resonance enterography: a Chinese retrospective cohort study","authors":"Yaming Lu, Shanshan Xiong, Mengchen Zhang, Xiaoman Zu, Jinbin Li, Ren Mao, Zhirong Zeng, Xuehua Li, Minhu Chen, Yao He","doi":"10.1177/20406223241259654","DOIUrl":"https://doi.org/10.1177/20406223241259654","url":null,"abstract":"Background:Transmural healing (TH) has emerged as a potential treatment goal for Crohn’s disease (CD). However, further research is needed to confirm its benefits and risk factors associated with TH remain unclear.Objectives:We aimed to assess the value of TH based on magnetic resonance enterography (MRE) in Chinese CD patients regarding the long-term outcomes and its associated factors.Design:Retrospective, observational cohort study.Methods:Patients with CD diagnosed by colonoscopy and MRE examination between 2015 and 2022 were included. All patients were evaluated with endoscopy together with MRE within 6–12 months after baseline and followed up for at least 6 months after evaluation. The primary endpoint was the occurrence of major outcomes during the follow-up, including drug escalation, hospitalization, and surgery. The cumulative probabilities of major outcomes were calculated using Kaplan–Meier survival curves. Logistic regression analyses were used to predict TH within 6–12 months after baseline.Results:A total of 175 patients were included in the study. Of these, 69 (39.4%) patients achieved mucosal healing (MH), but only 34 (19.4%) of them achieved TH. The median follow-up duration was 17.4 months (interquartile range, 11.6–25.5), and major outcomes occurred in 58.3% of patients. A lower occurrence rate of major outcomes was noted in patients who achieved TH than in those who achieved MH only ( p = 0.012). The baseline lymphocyte/C-reactive protein ratio (LCR) [odds ratio (OR), 1.60; 95% confidence interval (CI), 1.02–2.50; p = 0.039] and bowel wall thickness (BWT) (OR, 0.72; 95% CI, 0.59–0.90; p = 0.003) were independent predictors associated with TH. According to multivariate Cox regression analysis, low LCR [hazard ratio (HR), 2.34; 95% CI, 1.51–3.64; p < 0.001], and no healing (HR, 5.45; 95% CI, 2.28–13.00; p < 0.001) were associated with an increased risk of major outcomes.Conclusion:Patients with CD who achieved TH showed improved prognosis compared to those who achieved MH only. Baseline LCR and BWT might predict TH.","PeriodicalId":22960,"journal":{"name":"Therapeutic Advances in Chronic Disease","volume":"43 1","pages":""},"PeriodicalIF":3.5,"publicationDate":"2024-07-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141786104","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}
Hidradenitis suppurativa (HS) is an inflammatory skin condition with an underlying inflammatory process. Due to the limited efficacy of available treatments, HS remains a therapeutic challenge. The safety and efficacy of tumor necrosis factor-α (TNF-α) inhibitors, adalimumab, infliximab, and etanercept, are well studied in this patient population, and in some cases, HS was unresponsive to them. In recent years, evidence has been growing regarding the application of other anti-TNFs, including certolizumab pegol (CPZ) and golimumab. We sought to evaluate the overall safety and efficacy of golimumab and CPZ in the management of HS. A comprehensive search was performed on the PubMed, Scopus, Web of Science, and Ovid Embase databases, as well as the Google Scholar search engine from initiation to 31 August 2023. A total of nine and four studies used CPZ and golimumab to treat HS, respectively. Individuals with concomitant inflammatory immune-mediated diseases, pregnant females, and patients who were refractory to previous treatments achieved a Hidradenitis Suppurativa Clinical Response following CPZ administration. Also, golimumab showed promise in treating recalcitrant HS after the failure of other treatments, such as adalimumab and anti-interleukin-1. CPZ and golimumab can be efficacious treatment options for moderate-to-severe HS, especially in patients who are unresponsive to other TNF inhibitors, such as adalimumab.
化脓性扁平湿疹（HS）是一种具有潜在炎症过程的炎症性皮肤病。由于现有治疗方法的疗效有限，HS 仍是一个治疗难题。对肿瘤坏死因子-α（TNF-α）抑制剂阿达木单抗、英夫利昔单抗和依那西普的安全性和疗效进行了深入研究，但在某些病例中，HS对这些抑制剂毫无反应。近年来，有关应用其他抗肿瘤坏死因子药物的证据越来越多，包括certolizumab pegol（CPZ）和golimumab。我们试图评估戈利木单抗和CPZ治疗HS的总体安全性和有效性。我们在PubMed、Scopus、Web of Science和Ovid Embase数据库以及Google Scholar搜索引擎上进行了全面检索，检索时间从开始到2023年8月31日。共有9项和4项研究分别使用CPZ和戈利木单抗治疗HS。伴有炎症性免疫介导疾病的患者、妊娠女性以及对既往治疗无效的患者在服用CPZ后均获得了化脓性扁桃体炎临床应答。此外，在阿达木单抗和抗白细胞介素-1等其他疗法失败后，戈利木单抗也显示出治疗顽固性HS的前景。CPZ和戈利木单抗可作为中度至重度HS的有效治疗方案，尤其适用于对阿达木单抗等其他TNF抑制剂无反应的患者。
{"title":"Golimumab and certolizumab pegol for the treatment of hidradenitis suppurativa: a literature review and future perspective","authors":"Amirhossein Heidari, Yekta Ghane, Nazila Heidari, Seyedayin Hosseini, Azadeh Goodarzi","doi":"10.1177/20406223241257342","DOIUrl":"https://doi.org/10.1177/20406223241257342","url":null,"abstract":"Hidradenitis suppurativa (HS) is an inflammatory skin condition with an underlying inflammatory process. Due to the limited efficacy of available treatments, HS remains a therapeutic challenge. The safety and efficacy of tumor necrosis factor-α (TNF-α) inhibitors, adalimumab, infliximab, and etanercept, are well studied in this patient population, and in some cases, HS was unresponsive to them. In recent years, evidence has been growing regarding the application of other anti-TNFs, including certolizumab pegol (CPZ) and golimumab. We sought to evaluate the overall safety and efficacy of golimumab and CPZ in the management of HS. A comprehensive search was performed on the PubMed, Scopus, Web of Science, and Ovid Embase databases, as well as the Google Scholar search engine from initiation to 31 August 2023. A total of nine and four studies used CPZ and golimumab to treat HS, respectively. Individuals with concomitant inflammatory immune-mediated diseases, pregnant females, and patients who were refractory to previous treatments achieved a Hidradenitis Suppurativa Clinical Response following CPZ administration. Also, golimumab showed promise in treating recalcitrant HS after the failure of other treatments, such as adalimumab and anti-interleukin-1. CPZ and golimumab can be efficacious treatment options for moderate-to-severe HS, especially in patients who are unresponsive to other TNF inhibitors, such as adalimumab.","PeriodicalId":22960,"journal":{"name":"Therapeutic Advances in Chronic Disease","volume":"102 1","pages":""},"PeriodicalIF":3.5,"publicationDate":"2024-05-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141190536","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}
Pub Date : 2024-05-18DOI: 10.1177/20406223241247643
Annemieke M. Boot, Gema Ariceta, Signe Sparre Beck-Nielsen, Maria Luisa Brandi, Karine Briot, Carmen de Lucas Collantes, Sandro Giannini, Dieter Haffner, Richard Keen, Elena Levtchenko, M. Zulf Mughal, Outi Makitie, Ola Nilsson, Dirk Schnabel, Liana Tripto-Shkolnik, M. Carola Zillikens, Jonathan Liu, Alina Tudor, Francesco Emma
Background:X-linked hypophosphatemia (XLH) is a rare, progressive disorder characterized by excess fibroblast growth factor 23 (FGF23), causing renal phosphate-wasting and impaired active vitamin D synthesis. Burosumab is a recombinant human monoclonal antibody that inhibits FGF23, restoring patient serum phosphate levels. Safety data on long-term burosumab treatment are currently limited.Objectives:This post-authorization safety study (PASS) aims to monitor long-term safety outcomes in children and adolescents (1–17 years) treated with burosumab for XLH. This first interim analysis reports the initial PASS safety outcomes.Design:A 10-year retrospective and prospective cohort study.Methods:This PASS utilizes International XLH Registry (NCT03193476) data, which includes standard diagnostic and monitoring practice data at participating European centers.Results:At data cut-off (13 May 2021), 647 participants were included in the International XLH Registry; 367 were receiving burosumab, of which 67 provided consent to be included in the PASS. Mean (SD) follow-up time was 2.2 (1.0) years. Mean (SD) age was 7.3 (4.3) years (range 1.0–17.5 years). Mean duration of burosumab exposure was 29.7 (25.0) months. Overall, 25/67 participants (37.3%) experienced ⩾1 adverse event (AE) during follow-up; 83 AEs were reported. There were no deaths, no AEs leading to treatment withdrawal, nor serious AEs related to treatment. The most frequently reported AEs were classified as ‘musculoskeletal and connective tissue disorders’, with ‘pain in extremity’ most frequently reported, followed by ‘infections and infestations’, with ‘tooth abscess’ the most frequently reported.Conclusion:In this first interim analysis of the PASS, covering the initial 2 years of data collection, the safety profile of burosumab is consistent with previously reported safety data. The PASS will provide long-term safety data over its 10-year duration for healthcare providers and participants with XLH that contribute to improvements in the knowledge of burosumab safety.Trial registration:European Union electronic Register of Post-Authorisation Studies: EUPAS32190.
{"title":"Real-world non-interventional post-authorization safety study of long-term use of burosumab in children and adolescents with X-linked hypophosphatemia: first interim analysis","authors":"Annemieke M. Boot, Gema Ariceta, Signe Sparre Beck-Nielsen, Maria Luisa Brandi, Karine Briot, Carmen de Lucas Collantes, Sandro Giannini, Dieter Haffner, Richard Keen, Elena Levtchenko, M. Zulf Mughal, Outi Makitie, Ola Nilsson, Dirk Schnabel, Liana Tripto-Shkolnik, M. Carola Zillikens, Jonathan Liu, Alina Tudor, Francesco Emma","doi":"10.1177/20406223241247643","DOIUrl":"https://doi.org/10.1177/20406223241247643","url":null,"abstract":"Background:X-linked hypophosphatemia (XLH) is a rare, progressive disorder characterized by excess fibroblast growth factor 23 (FGF23), causing renal phosphate-wasting and impaired active vitamin D synthesis. Burosumab is a recombinant human monoclonal antibody that inhibits FGF23, restoring patient serum phosphate levels. Safety data on long-term burosumab treatment are currently limited.Objectives:This post-authorization safety study (PASS) aims to monitor long-term safety outcomes in children and adolescents (1–17 years) treated with burosumab for XLH. This first interim analysis reports the initial PASS safety outcomes.Design:A 10-year retrospective and prospective cohort study.Methods:This PASS utilizes International XLH Registry (NCT03193476) data, which includes standard diagnostic and monitoring practice data at participating European centers.Results:At data cut-off (13 May 2021), 647 participants were included in the International XLH Registry; 367 were receiving burosumab, of which 67 provided consent to be included in the PASS. Mean (SD) follow-up time was 2.2 (1.0) years. Mean (SD) age was 7.3 (4.3) years (range 1.0–17.5 years). Mean duration of burosumab exposure was 29.7 (25.0) months. Overall, 25/67 participants (37.3%) experienced ⩾1 adverse event (AE) during follow-up; 83 AEs were reported. There were no deaths, no AEs leading to treatment withdrawal, nor serious AEs related to treatment. The most frequently reported AEs were classified as ‘musculoskeletal and connective tissue disorders’, with ‘pain in extremity’ most frequently reported, followed by ‘infections and infestations’, with ‘tooth abscess’ the most frequently reported.Conclusion:In this first interim analysis of the PASS, covering the initial 2 years of data collection, the safety profile of burosumab is consistent with previously reported safety data. The PASS will provide long-term safety data over its 10-year duration for healthcare providers and participants with XLH that contribute to improvements in the knowledge of burosumab safety.Trial registration:European Union electronic Register of Post-Authorisation Studies: EUPAS32190.","PeriodicalId":22960,"journal":{"name":"Therapeutic Advances in Chronic Disease","volume":"37 1","pages":""},"PeriodicalIF":3.5,"publicationDate":"2024-05-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141061543","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}
Pub Date : 2024-05-08eCollection Date: 2024-01-01DOI: 10.1177/20406223241247648
Kaituo Huang, Lingya Yao, Jing Liu, Qian Cao
In 2020, the European Medicines Agency approved subcutaneous (SC) vedolizumab (VDZ) for the maintenance treatment of adult patients with moderate to severe inflammatory bowel disease (IBD). This article reviews the efficacy, safety, persistence, pharmacology, patient satisfaction, and economic implications of transitioning to SC VDZ treatment and explores whether SC formulations can be recommended by the same guidelines as intravenous (IV) formulations. Clinical trials and real-world evidence indicate that transitioning from IV to SC VDZ in patients with IBD maintains clinical, biochemical, and patient-reported clinical remission and is well-tolerated, with no new safety issues identified, except for injection site reactions. Moreover, SC VDZ has an exposure-response relationship and low immunogenicity, is economical, and provides a high level of patient satisfaction. Owing to these advantages, transitioning may be advisable. In the future, more studies are needed to clarify the exact role of SC VDZ in IBD treatment, including optimization and transitioning strategies and individualized treatments based on baseline characteristics.
{"title":"Take vedolizumab home: transition from intravenous to subcutaneous treatment.","authors":"Kaituo Huang, Lingya Yao, Jing Liu, Qian Cao","doi":"10.1177/20406223241247648","DOIUrl":"10.1177/20406223241247648","url":null,"abstract":"<p><p>In 2020, the European Medicines Agency approved subcutaneous (SC) vedolizumab (VDZ) for the maintenance treatment of adult patients with moderate to severe inflammatory bowel disease (IBD). This article reviews the efficacy, safety, persistence, pharmacology, patient satisfaction, and economic implications of transitioning to SC VDZ treatment and explores whether SC formulations can be recommended by the same guidelines as intravenous (IV) formulations. Clinical trials and real-world evidence indicate that transitioning from IV to SC VDZ in patients with IBD maintains clinical, biochemical, and patient-reported clinical remission and is well-tolerated, with no new safety issues identified, except for injection site reactions. Moreover, SC VDZ has an exposure-response relationship and low immunogenicity, is economical, and provides a high level of patient satisfaction. Owing to these advantages, transitioning may be advisable. In the future, more studies are needed to clarify the exact role of SC VDZ in IBD treatment, including optimization and transitioning strategies and individualized treatments based on baseline characteristics.</p>","PeriodicalId":22960,"journal":{"name":"Therapeutic Advances in Chronic Disease","volume":"15 ","pages":"20406223241247648"},"PeriodicalIF":3.5,"publicationDate":"2024-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11080802/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140899542","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}
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