Pub Date : 2024-10-08DOI: 10.1007/s44313-024-00034-6
Valentina Perrone, Melania Leogrande, Maria Cappuccilli, Luca Degli Esposti
Purpose: This real-world analysis described the Hemophilia A (HA) population in Italy, evaluating drug utilization and consumption of factor VIII (FVIII) products of patients under prophylaxis and on-demand therapy.
Methods: From Jan-2017 to Jun-2022, male patients with HA were identified through prescriptions of FVIII products [extended half-life FVIII, standard half-life recombinant FVIII, and plasma-derived FVIII (EHL FVIII, SHL rFVIII, and pdFVIII, respectively)], or emicizumab or FVIII plus von Willebrand factor or HA-related hospitalization using administrative flows of Italian healthcare entities. Patients on treatment with FVIII products during 2021-2022 were stratified by treatment regimen (prophylaxis/on-demand). The mean annual consumption expressed in International Units (IU) of EHL FVIII and SHL FVIII in patients treated during 2021-2022 having at least 12-month follow-up were assessed.
Results: Among included HA patients, 145 (39.5%) received EHL FVIII and 222 (60.5%) SHL FVIII. Of 165 patients on prophylaxis, 105 (64%) received an EHL FVIII and 60 (36%) an SHL FVIII. The mean annual consumption of FVIII was 336,700 IU (median 319,000 IU) for EHL FVIII and 440,267 IU (median 360,500 IU) for SHL FVIII. Specifically, for patients on EHL FVIII, the most common drugs were efmoroctocog alfa (N = 51) and damoctocog alfa pegol (N = 50), followed by turoctocog alfa pegol (N = 25) and rurioctocog alfa pegol (N = 19). Of 702 HA patients initially treated with FVIII products, 74 (10.5%) switched to emicizumab during follow-up.
Conclusion: These findings revealed an extensive use of EHL FVIII products, suggesting growing efforts from clinicians to optimize prophylactic strategies and achieve better bleeding protection.
{"title":"Real-world insights into the management of hemophilia A in Italy: treatment patterns and healthcare resource utilization.","authors":"Valentina Perrone, Melania Leogrande, Maria Cappuccilli, Luca Degli Esposti","doi":"10.1007/s44313-024-00034-6","DOIUrl":"10.1007/s44313-024-00034-6","url":null,"abstract":"<p><strong>Purpose: </strong>This real-world analysis described the Hemophilia A (HA) population in Italy, evaluating drug utilization and consumption of factor VIII (FVIII) products of patients under prophylaxis and on-demand therapy.</p><p><strong>Methods: </strong>From Jan-2017 to Jun-2022, male patients with HA were identified through prescriptions of FVIII products [extended half-life FVIII, standard half-life recombinant FVIII, and plasma-derived FVIII (EHL FVIII, SHL rFVIII, and pdFVIII, respectively)], or emicizumab or FVIII plus von Willebrand factor or HA-related hospitalization using administrative flows of Italian healthcare entities. Patients on treatment with FVIII products during 2021-2022 were stratified by treatment regimen (prophylaxis/on-demand). The mean annual consumption expressed in International Units (IU) of EHL FVIII and SHL FVIII in patients treated during 2021-2022 having at least 12-month follow-up were assessed.</p><p><strong>Results: </strong>Among included HA patients, 145 (39.5%) received EHL FVIII and 222 (60.5%) SHL FVIII. Of 165 patients on prophylaxis, 105 (64%) received an EHL FVIII and 60 (36%) an SHL FVIII. The mean annual consumption of FVIII was 336,700 IU (median 319,000 IU) for EHL FVIII and 440,267 IU (median 360,500 IU) for SHL FVIII. Specifically, for patients on EHL FVIII, the most common drugs were efmoroctocog alfa (N = 51) and damoctocog alfa pegol (N = 50), followed by turoctocog alfa pegol (N = 25) and rurioctocog alfa pegol (N = 19). Of 702 HA patients initially treated with FVIII products, 74 (10.5%) switched to emicizumab during follow-up.</p><p><strong>Conclusion: </strong>These findings revealed an extensive use of EHL FVIII products, suggesting growing efforts from clinicians to optimize prophylactic strategies and achieve better bleeding protection.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"59 1","pages":"30"},"PeriodicalIF":2.3,"publicationDate":"2024-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11461399/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142394161","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-28DOI: 10.1007/s44313-024-00023-9
Young-Shil Park, Ki-Young Yoo, Sang Kyu Park, Taiju Hwang, Aeran Jung, Eun Jin Choi
Purpose: This study aimed to investigate the pharmacokinetics (PK) of factor VIII (FVIII) in Korean patients, as limited information is available on the PK of FVIII in this population.
Methods: We collected the FVIII PK results from patients with moderate-to-severe hemophilia A using myPKFiT. PK variations were assessed according to age, blood type, inhibitor history, von Willebrand factor antigen (vWF:Ag) level, and body mass index. Additionally, the correlation between the PK profile and prophylaxis regimen was specifically analyzed for each product in severe cases.
Results: The PK data of 48 and 81 patients treated with octocog alfa and rurioctocog alfa pegol, respectively, were obtained. The median half-lives of octocog alfa and rurioctocog alfa pegol were 9.9 (range: 6.3-15.2) h and 15.3 (range: 10.4-23.9) h, respectively. The PK profiles for each product did not differ according to age group; however, blood type-O patients had shorter half-lives and time to 1% compared to non-blood type-O patients. In regression analysis, the PK of octocog alfa showed a statistically significant difference according to age, whereas the PK of rurioctocog alfa pegol correlated with vWF:Ag. Only the frequency of rurioctocog alfa pegol use showed a statistically significant difference in relation to time to 1%, although the coefficient of determination was small.
Conclusion: This study confirmed significant interpatient variation in the PK of FVIII among Korean patients with hemophilia A. To achieve optimized prophylaxis, personalizing the regimen based on the PK profile of each individual patient is essential.
{"title":"Evaluation of FVIII pharmacokinetic profiles in Korean hemophilia A patients assessed with myPKFiT: a retrospective chart review.","authors":"Young-Shil Park, Ki-Young Yoo, Sang Kyu Park, Taiju Hwang, Aeran Jung, Eun Jin Choi","doi":"10.1007/s44313-024-00023-9","DOIUrl":"10.1007/s44313-024-00023-9","url":null,"abstract":"<p><strong>Purpose: </strong>This study aimed to investigate the pharmacokinetics (PK) of factor VIII (FVIII) in Korean patients, as limited information is available on the PK of FVIII in this population.</p><p><strong>Methods: </strong>We collected the FVIII PK results from patients with moderate-to-severe hemophilia A using myPKFiT. PK variations were assessed according to age, blood type, inhibitor history, von Willebrand factor antigen (vWF:Ag) level, and body mass index. Additionally, the correlation between the PK profile and prophylaxis regimen was specifically analyzed for each product in severe cases.</p><p><strong>Results: </strong>The PK data of 48 and 81 patients treated with octocog alfa and rurioctocog alfa pegol, respectively, were obtained. The median half-lives of octocog alfa and rurioctocog alfa pegol were 9.9 (range: 6.3-15.2) h and 15.3 (range: 10.4-23.9) h, respectively. The PK profiles for each product did not differ according to age group; however, blood type-O patients had shorter half-lives and time to 1% compared to non-blood type-O patients. In regression analysis, the PK of octocog alfa showed a statistically significant difference according to age, whereas the PK of rurioctocog alfa pegol correlated with vWF:Ag. Only the frequency of rurioctocog alfa pegol use showed a statistically significant difference in relation to time to 1%, although the coefficient of determination was small.</p><p><strong>Conclusion: </strong>This study confirmed significant interpatient variation in the PK of FVIII among Korean patients with hemophilia A. To achieve optimized prophylaxis, personalizing the regimen based on the PK profile of each individual patient is essential.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"59 1","pages":"29"},"PeriodicalIF":2.3,"publicationDate":"2024-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11358568/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142082216","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-12DOI: 10.1007/s44313-024-00027-5
Kootae Park, Soon Hyo Kwon
Kidney disease is a frequent complication of multiple myeloma and other malignancies associated with monoclonal gammopathies. Additionally, dysproteinemia-related kidney disease can occur independently of overt multiple myeloma or hematologic malignancies. Monoclonal gammopathy of renal significance (MGRS) is a spectrum of disorders in which a monoclonal immunoglobulin produced by a benign or premalignant B-cell or plasma cell clone causes kidney damage. MGRS-associated renal disease manifests in various forms, including immunoglobulin-associated amyloidosis, monoclonal immunoglobulin deposition diseases (light chain, heavy chain, and combined light and heavy chain deposition diseases), proliferative glomerulonephritis with monoclonal immunoglobulin deposits, C3 glomerulopathy with monoclonal gammopathy, and light chain proximal tubulopathy. Although MGRS is a nonmalignant or premalignant hematologic condition, it has significant renal implications that often lead to progressive kidney damage and, eventually, end-stage kidney disease. This review discusses the epidemiology, pathogenesis, and management of MGRS and focuses on the perspective of nephrologists.
{"title":"Monoclonal gammopathy of renal significance from the perspective of nephrologists.","authors":"Kootae Park, Soon Hyo Kwon","doi":"10.1007/s44313-024-00027-5","DOIUrl":"10.1007/s44313-024-00027-5","url":null,"abstract":"<p><p>Kidney disease is a frequent complication of multiple myeloma and other malignancies associated with monoclonal gammopathies. Additionally, dysproteinemia-related kidney disease can occur independently of overt multiple myeloma or hematologic malignancies. Monoclonal gammopathy of renal significance (MGRS) is a spectrum of disorders in which a monoclonal immunoglobulin produced by a benign or premalignant B-cell or plasma cell clone causes kidney damage. MGRS-associated renal disease manifests in various forms, including immunoglobulin-associated amyloidosis, monoclonal immunoglobulin deposition diseases (light chain, heavy chain, and combined light and heavy chain deposition diseases), proliferative glomerulonephritis with monoclonal immunoglobulin deposits, C3 glomerulopathy with monoclonal gammopathy, and light chain proximal tubulopathy. Although MGRS is a nonmalignant or premalignant hematologic condition, it has significant renal implications that often lead to progressive kidney damage and, eventually, end-stage kidney disease. This review discusses the epidemiology, pathogenesis, and management of MGRS and focuses on the perspective of nephrologists.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"59 1","pages":"28"},"PeriodicalIF":2.3,"publicationDate":"2024-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11319560/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141917740","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-08DOI: 10.1007/s44313-024-00024-8
Young Shil Park, Ji Kyoung Park, Jeong A Park, Hee Jo Baek, Jae Hee Lee, Chur Woo You, Chuhl Joo Lyu, Eun Jin Choi
Background: To investigate the clinical treatment status, such as treatment regimen, bleeding events, and drug dose, in patients with hemophilia B in South Korea.
Methods: In this retrospective chart review, data of patients with hemophilia B from eight university hospitals were collected. Demographic and clinical data, treatment data, such as regimen and number of injections, dose of factor IX concentrate, and bleeding data were reviewed. Descriptive analyses were performed with annual data for 2019, 2020, and 2021, as well as the three years consecutively.
Results: The medical records of 150 patients with hemophilia B between January 1, 2019, and December 31, 2021, were collected. Among these, 72 (48.0%) were severe, 47 (31.3%) were moderate, and 28 (18.7%) were mild. The results showed approximately two times more patients receiving prophylaxis as those receiving on-demand therapy, with 66.1% of patients receiving prophylaxis in 2019, 64.9% in 2020, and 72.1% in 2021. Annualized bleeding rates were 2.2% (± 3.1) in 2019, 1.8% (± 3.0) in 2020, and 1.8% (± 2.9) in 2021 among patients receiving prophylaxis. For the doses of factor IX concentrate, patients receiving prophylaxis received an average of 41.6 (± 11.9) IU/Kg/Injection in 2019, 45.7 (± 12.9) IU/Kg/Injection in 2020, and 60.1 (± 24.0) IU/Kg/Injection in 2021.
Conclusions: Clinically, prophylaxis is more prevalent than reported. Based on insights gained from current clinical evidence, it is expected that the unmet medical needs of patients can be identified, and physicians can evaluate the status of patients and actively manage hemophilia B using more effective treatment strategies.
背景:调查韩国血友病 B 患者的临床治疗状况,如治疗方案、出血事件和药物剂量:调查韩国 B 型血友病患者的临床治疗状况,如治疗方案、出血事件和药物剂量:在这项回顾性病历审查中,收集了 8 家大学医院的 B 型血友病患者数据。方法:在这项回顾性病历审查中,收集了 8 家大学医院的血友病 B 患者数据,审查了人口统计学和临床数据、治疗数据(如治疗方案和注射次数、浓缩因子 IX 的剂量)以及出血数据。对 2019 年、2020 年和 2021 年以及连续三年的年度数据进行了描述性分析:收集了 150 名 B 型血友病患者在 2019 年 1 月 1 日至 2021 年 12 月 31 日期间的医疗记录。其中,72 人(48.0%)为重度,47 人(31.3%)为中度,28 人(18.7%)为轻度。结果显示,接受预防性治疗的患者约为按需治疗患者的两倍,2019 年接受预防性治疗的患者占 66.1%,2020 年占 64.9%,2021 年占 72.1%。在接受预防性治疗的患者中,2019 年的年化出血率为 2.2%(± 3.1),2020 年为 1.8%(± 3.0),2021 年为 1.8%(± 2.9)。就浓缩因子 IX 的剂量而言,接受预防性治疗的患者在 2019 年平均接受 41.6(± 11.9)IU/Kg/注射,2020 年平均接受 45.7(± 12.9)IU/Kg/注射,2021 年平均接受 60.1(± 24.0)IU/Kg/注射:在临床上,预防性治疗比报告的更为普遍。结论:临床上,预防性治疗比报告的更为普遍。根据从当前临床证据中获得的见解,预计可以确定患者未满足的医疗需求,医生可以评估患者的状况,并采用更有效的治疗策略积极管理 B 型血友病。
{"title":"Clinical data on treatment regimen and use of medication among patients with hemophilia B in Korea.","authors":"Young Shil Park, Ji Kyoung Park, Jeong A Park, Hee Jo Baek, Jae Hee Lee, Chur Woo You, Chuhl Joo Lyu, Eun Jin Choi","doi":"10.1007/s44313-024-00024-8","DOIUrl":"10.1007/s44313-024-00024-8","url":null,"abstract":"<p><strong>Background: </strong>To investigate the clinical treatment status, such as treatment regimen, bleeding events, and drug dose, in patients with hemophilia B in South Korea.</p><p><strong>Methods: </strong>In this retrospective chart review, data of patients with hemophilia B from eight university hospitals were collected. Demographic and clinical data, treatment data, such as regimen and number of injections, dose of factor IX concentrate, and bleeding data were reviewed. Descriptive analyses were performed with annual data for 2019, 2020, and 2021, as well as the three years consecutively.</p><p><strong>Results: </strong>The medical records of 150 patients with hemophilia B between January 1, 2019, and December 31, 2021, were collected. Among these, 72 (48.0%) were severe, 47 (31.3%) were moderate, and 28 (18.7%) were mild. The results showed approximately two times more patients receiving prophylaxis as those receiving on-demand therapy, with 66.1% of patients receiving prophylaxis in 2019, 64.9% in 2020, and 72.1% in 2021. Annualized bleeding rates were 2.2% (± 3.1) in 2019, 1.8% (± 3.0) in 2020, and 1.8% (± 2.9) in 2021 among patients receiving prophylaxis. For the doses of factor IX concentrate, patients receiving prophylaxis received an average of 41.6 (± 11.9) IU/Kg/Injection in 2019, 45.7 (± 12.9) IU/Kg/Injection in 2020, and 60.1 (± 24.0) IU/Kg/Injection in 2021.</p><p><strong>Conclusions: </strong>Clinically, prophylaxis is more prevalent than reported. Based on insights gained from current clinical evidence, it is expected that the unmet medical needs of patients can be identified, and physicians can evaluate the status of patients and actively manage hemophilia B using more effective treatment strategies.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"59 1","pages":"27"},"PeriodicalIF":2.3,"publicationDate":"2024-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11310372/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141903148","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-07DOI: 10.1007/s44313-024-00030-w
Jeong Suk Koh, Ik-Chan Song
Anemia is frequently observed in patients with cancer owing to anticancer chemotherapy, radiation therapy, and inflammatory responses. This often leads to functional iron deficiency, characterized by adequate iron stores but impaired use of iron for red blood cell production. This condition, termed functional iron deficiency anemia (IDA), is identified by a ferritin level of 30-500 µg/dL and a transferrin saturation < 50%. Functional iron deficiency often develops with the prolonged use of erythropoiesis-stimulating agents, leading to a diminished response to anemia treatment. Although oral iron supplementation is common, intravenous iron is more effective and recommended in such cases. Recent studies have shown that ferric carboxymaltose (FCM) is effective in treating functional IDA in patients with cancer. However, because of its potential to induce asymptomatic severe phosphate deficiency, it is important to closely monitor phosphate levels in patients receiving FCM.
{"title":"Functional iron deficiency anemia in patients with cancer.","authors":"Jeong Suk Koh, Ik-Chan Song","doi":"10.1007/s44313-024-00030-w","DOIUrl":"10.1007/s44313-024-00030-w","url":null,"abstract":"<p><p>Anemia is frequently observed in patients with cancer owing to anticancer chemotherapy, radiation therapy, and inflammatory responses. This often leads to functional iron deficiency, characterized by adequate iron stores but impaired use of iron for red blood cell production. This condition, termed functional iron deficiency anemia (IDA), is identified by a ferritin level of 30-500 µg/dL and a transferrin saturation < 50%. Functional iron deficiency often develops with the prolonged use of erythropoiesis-stimulating agents, leading to a diminished response to anemia treatment. Although oral iron supplementation is common, intravenous iron is more effective and recommended in such cases. Recent studies have shown that ferric carboxymaltose (FCM) is effective in treating functional IDA in patients with cancer. However, because of its potential to induce asymptomatic severe phosphate deficiency, it is important to closely monitor phosphate levels in patients receiving FCM.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"59 1","pages":"26"},"PeriodicalIF":2.3,"publicationDate":"2024-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11306885/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141898564","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-02DOI: 10.1007/s44313-024-00029-3
Ho-Young Yhim
Cancer-associated venous thromboembolism (VTE) significantly impacts morbidity and mortality. The introduction of direct oral anticoagulants over the past decade has revolutionized VTE treatment in patients with active cancer, offering potential advantages over traditional therapies. However, uncertainties persist regarding the optimal selection and dosage of anticoagulants, particularly in patients with specific risk factors for bleeding, such as certain cancer types (e.g., upper gastrointestinal cancer, genitourinary cancer, primary or metastatic brain tumor, and hematologic malignancies) and specific patient characteristics (e.g., renal dysfunction and thrombocytopenia). Recent data on the thrombotic risk associated with low thrombotic burden VTE, such as subsegmental pulmonary embolism and isolated distal deep vein thrombosis, underscore the need for updated management strategies in daily clinical practice. This review aims to explore these issues and highlight the evolving landscape of cancer-associated VTE management.
{"title":"Proper application of anticoagulation therapy on cancer-associated venous thrombosis.","authors":"Ho-Young Yhim","doi":"10.1007/s44313-024-00029-3","DOIUrl":"10.1007/s44313-024-00029-3","url":null,"abstract":"<p><p>Cancer-associated venous thromboembolism (VTE) significantly impacts morbidity and mortality. The introduction of direct oral anticoagulants over the past decade has revolutionized VTE treatment in patients with active cancer, offering potential advantages over traditional therapies. However, uncertainties persist regarding the optimal selection and dosage of anticoagulants, particularly in patients with specific risk factors for bleeding, such as certain cancer types (e.g., upper gastrointestinal cancer, genitourinary cancer, primary or metastatic brain tumor, and hematologic malignancies) and specific patient characteristics (e.g., renal dysfunction and thrombocytopenia). Recent data on the thrombotic risk associated with low thrombotic burden VTE, such as subsegmental pulmonary embolism and isolated distal deep vein thrombosis, underscore the need for updated management strategies in daily clinical practice. This review aims to explore these issues and highlight the evolving landscape of cancer-associated VTE management.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"59 1","pages":"25"},"PeriodicalIF":2.3,"publicationDate":"2024-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11296996/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141876264","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-09DOI: 10.1007/s44313-024-00026-6
Seug Yun Yoon, Sung-Eun Lee
{"title":"Treatment with ropeginterferon alfa-2b in patients with hydroxyurea resistant or intolerant polycythemia vera in South Korea: one-year results from a phase 2 study.","authors":"Seug Yun Yoon, Sung-Eun Lee","doi":"10.1007/s44313-024-00026-6","DOIUrl":"10.1007/s44313-024-00026-6","url":null,"abstract":"","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"59 1","pages":"23"},"PeriodicalIF":2.3,"publicationDate":"2024-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11233476/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141560040","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-04DOI: 10.1007/s44313-024-00022-w
Kyung-Nam Koh, Su Hyun Yoon, Sung Han Kang, Hyery Kim, Ho Joon Im
Histiocytic neoplasms are rare diseases involving macrophages, dendritic cells, and monocytes. They include Langerhans cell histiocytosis (LCH), Erdheim-Chester disease (ECD), Rosai-Dorfman disease (RDD), juvenile xanthogranuloma (JXG), and histiocytic sarcoma. Histiocytic neoplasms are characterized by varied clinical courses and prognoses, necessitating a nuanced understanding of their classification, epidemiology, and clinical manifestations. Genetic studies have revealed somatic mutations, predominantly in the MAPK pathway, suggesting a clonal neoplastic nature. This review covers the current understanding of histiocytic neoplasms, molecular pathophysiology, with a particular focus on mutations in genes such as BRAF, MAP2K1, and the PI3K-AKT signaling pathways, and evolving treatment strategies, especially focusing on LCH, ECD, RDD, and JXG. The treatment landscape has evolved with advancements in targeted therapies. BRAF inhibitors, such as vemurafenib and dabrafenib, have shown efficacy, especially in high-risk LCH cases; however, challenges remain, including relapse post-treatment discontinuation, and adverse effects. MEK inhibitors have also demonstrated effectiveness, and cobimetinib has recently been approved for use in adults. Further research is required to determine the optimal treatment duration and strategies for managing therapy interruptions. Advancements in molecular genetics and targeted therapies have revolutionized the management of histiocytic neoplasms. However, ongoing research is crucial for optimizing patient outcomes.
{"title":"Advancements in the understanding and management of histiocytic neoplasms.","authors":"Kyung-Nam Koh, Su Hyun Yoon, Sung Han Kang, Hyery Kim, Ho Joon Im","doi":"10.1007/s44313-024-00022-w","DOIUrl":"10.1007/s44313-024-00022-w","url":null,"abstract":"<p><p>Histiocytic neoplasms are rare diseases involving macrophages, dendritic cells, and monocytes. They include Langerhans cell histiocytosis (LCH), Erdheim-Chester disease (ECD), Rosai-Dorfman disease (RDD), juvenile xanthogranuloma (JXG), and histiocytic sarcoma. Histiocytic neoplasms are characterized by varied clinical courses and prognoses, necessitating a nuanced understanding of their classification, epidemiology, and clinical manifestations. Genetic studies have revealed somatic mutations, predominantly in the MAPK pathway, suggesting a clonal neoplastic nature. This review covers the current understanding of histiocytic neoplasms, molecular pathophysiology, with a particular focus on mutations in genes such as BRAF, MAP2K1, and the PI3K-AKT signaling pathways, and evolving treatment strategies, especially focusing on LCH, ECD, RDD, and JXG. The treatment landscape has evolved with advancements in targeted therapies. BRAF inhibitors, such as vemurafenib and dabrafenib, have shown efficacy, especially in high-risk LCH cases; however, challenges remain, including relapse post-treatment discontinuation, and adverse effects. MEK inhibitors have also demonstrated effectiveness, and cobimetinib has recently been approved for use in adults. Further research is required to determine the optimal treatment duration and strategies for managing therapy interruptions. Advancements in molecular genetics and targeted therapies have revolutionized the management of histiocytic neoplasms. However, ongoing research is crucial for optimizing patient outcomes.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"59 1","pages":"22"},"PeriodicalIF":2.3,"publicationDate":"2024-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11224208/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141499323","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-06-07DOI: 10.1007/s44313-024-00021-x
Ka-Won Kang
Surgical patients are at risk of postoperative complications and mortality, necessitating preoperative patient optimization through the identification and correction of modifiable risk factors. Although preoperative platelet transfusions aim to reduce the risk of bleeding, their efficacy remains uncertain. Similarly, red blood cell transfusion in patients with anemia does not reduce the risk of postoperative mortality and may exacerbate complications. Therefore, developing individualized strategies that focus on correcting preoperative complete blood count abnormalities and minimizing transfusion requirements are essential. This review aimed to examine complete blood count abnormalities and appropriate transfusion strategies to minimize postoperative complications.
{"title":"Preoperative consultation for determining the appropriate transfusion strategy.","authors":"Ka-Won Kang","doi":"10.1007/s44313-024-00021-x","DOIUrl":"10.1007/s44313-024-00021-x","url":null,"abstract":"<p><p>Surgical patients are at risk of postoperative complications and mortality, necessitating preoperative patient optimization through the identification and correction of modifiable risk factors. Although preoperative platelet transfusions aim to reduce the risk of bleeding, their efficacy remains uncertain. Similarly, red blood cell transfusion in patients with anemia does not reduce the risk of postoperative mortality and may exacerbate complications. Therefore, developing individualized strategies that focus on correcting preoperative complete blood count abnormalities and minimizing transfusion requirements are essential. This review aimed to examine complete blood count abnormalities and appropriate transfusion strategies to minimize postoperative complications.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"59 1","pages":"21"},"PeriodicalIF":2.2,"publicationDate":"2024-06-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11161442/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141284995","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}