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Pharmacologically Treated Anxiety and Depression in People Diagnosed with von Willebrand Disease: Matched Cohort Study. 血管性血友病患者的药物治疗焦虑和抑郁:匹配队列研究
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S407993
Katrina Wilcox Hagberg, Susan Jick, Gülden Özen, Ping Du
{"title":"Pharmacologically Treated Anxiety and Depression in People Diagnosed with von Willebrand Disease: Matched Cohort Study.","authors":"Katrina Wilcox Hagberg,&nbsp;Susan Jick,&nbsp;Gülden Özen,&nbsp;Ping Du","doi":"10.2147/JBM.S407993","DOIUrl":"https://doi.org/10.2147/JBM.S407993","url":null,"abstract":"<p><strong>Background: </strong>People diagnosed with von Willebrand disease (VWD) have reduced quality of life versus the general population, and there is limited evidence of increased rates of anxiety and/or depression among people diagnosed with VWD.</p><p><strong>Aim: </strong>To understand the association between VWD and mental health outcomes.</p><p><strong>Design and setting: </strong>A retrospective cohort study was conducted using the UK Clinical Practice Research Datalink (CPRD) GOLD database (1988-2016).</p><p><strong>Methods: </strong>People diagnosed with VWD were matched 1:10 to randomly selected people in the database without VWD based on sex, birth year ±2 years, CPRD record start year ±2 years, and general practice attended. Individuals were followed from VWD diagnosis or match date to censoring (first event date, CPRD end date, or death). Treated anxiety and treated depression were identified by a diagnostic Read Code and a prescription for anxiety/depression medication recorded within 90 days of each other, after VWD diagnosis/match date.</p><p><strong>Results: </strong>Treated anxiety was recorded in 89 of 1119 (8.0%) people diagnosed with VWD and 624 of 10,423 (6.0%) without VWD (age- and sex-adjusted incidence rate ratio [IRR], 1.37; 95% confidence interval [CI], 1.10-1.71). Treated depression was recorded in 119 of 1083 (11.0%) people diagnosed with VWD and 846 of 9845 (8.6%) without VWD (adjusted IRR, 1.35; 95% CI, 1.11-1.63). Females aged 20-39 and 0-19 years were at greatest risk for treated anxiety and treated depression, respectively.</p><p><strong>Conclusion: </strong>Higher rates of treated anxiety and depression were observed among people diagnosed with versus without VWD, predominantly in young females.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/4b/c5/jbm-14-413.PMC10349568.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10201661","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}
引用次数: 0
Prolonged Exposure to Caplacizumab as Rescue Therapy in Refractory Immune Thrombotic Thrombocytopenic Purpura. 长期暴露于卡普拉珠单抗作为难治性免疫性血栓性血小板减少性紫癜的抢救治疗。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S395248
George M Rodgers, Misa Berndt, Megan Fonteno, Jeffrey A Gilreath
{"title":"Prolonged Exposure to Caplacizumab as Rescue Therapy in Refractory Immune Thrombotic Thrombocytopenic Purpura.","authors":"George M Rodgers,&nbsp;Misa Berndt,&nbsp;Megan Fonteno,&nbsp;Jeffrey A Gilreath","doi":"10.2147/JBM.S395248","DOIUrl":"https://doi.org/10.2147/JBM.S395248","url":null,"abstract":"<p><p>We describe a case of refractory thrombotic thrombocytopenic purpura (7 lines of therapy) in which caplacizumab was used over a 6-month period as rescue therapy. Caplacizumab maintained the patient in clinical remission until successful immunosuppression was achieved resulting in normal ADAMTS13 levels. This case illustrates the utility of caplacizumab therapy in treating refractory TTP.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/d7/f5/jbm-14-209.PMC9987240.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9084127","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}
引用次数: 0
Pharmacokinetic-Pharmacodynamic Comparison of Recombinant and Plasma-Derived von Willebrand Factor in Patients with von Willebrand Disease Type 3. 重组血管性血友病因子与血浆源性血管性血友病3型患者药动学-药效学比较
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S395845
Alexander Bauer, Sofia Friberg-Hietala, Giovanni Smania, Martin Wolfsegger
{"title":"Pharmacokinetic-Pharmacodynamic Comparison of Recombinant and Plasma-Derived von Willebrand Factor in Patients with von Willebrand Disease Type 3.","authors":"Alexander Bauer,&nbsp;Sofia Friberg-Hietala,&nbsp;Giovanni Smania,&nbsp;Martin Wolfsegger","doi":"10.2147/JBM.S395845","DOIUrl":"https://doi.org/10.2147/JBM.S395845","url":null,"abstract":"<p><strong>Background: </strong>Recombinant von Willebrand factor (rVWF, vonicog alfa, Vonvendi/Veyvondi, Takeda Pharmaceuticals USA, Lexington, MA) and several plasma-derived VWF/factor VIII (pdVWF/FVIII) concentrates are available for treating bleeding episodes in patients with von Willebrand disease (VWD).</p><p><strong>Purpose: </strong>To develop population pharmacokinetic (PK)/pharmacodynamic (PD) models that describe VWF:ristocetin cofactor (VWF:RCo) activity and its relationship with FVIII activity (FVIII:C) over time following intravenous administration of either rVWF or a pdVWF/FVIII concentrate (VWF:RCo/FVIII:C 2.4:1) in patients with VWD; to use the final PK/PD models for an in silico comparison of rVWF and pdVWF/FVIII.</p><p><strong>Methods: </strong>The population PK model for rVWF was based on data from four clinical studies in which rVWF was administered to adult patients with VWD type 1, 2 or 3 (phase 1: NCT00816660; phase 3: NCT01410227 and NCT02283268) or severe hemophilia A (phase 1: EudraCT 2011-004314-42). The PK and PK/PD models for pdVWF/FVIII were based on data from the phase 1 study (NCT00816660) in patients with type 3 VWD who received either rVWF plus recombinant FVIII (rFVIII, octocog alfa, ADVATE<sup>®</sup>, Takeda Pharmaceuticals USA, Lexington, MA, USA) or pdVWF/FVIII.</p><p><strong>Results: </strong>There was a marked difference in clearance following rVWF administration compared with pdVWF/FVIII in type 3 VWD, leading to a ~1.75 longer mean residence time (ie, persistence of VWF:RCo activity in the body) and half-life for rVWF versus pdVWF/FVIII. Simulations showed that following repeated administration of rVWF (50 IU/kg), a FVIII:C activity of >40 IU/dL can be maintained for the full 72 h dosing interval.</p><p><strong>Conclusion: </strong>The slower elimination of VWF:RCo following rVWF administration results in a prolonged effect on FVIII turnover compared with pdVWF/FVIII administration.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b4/a8/jbm-14-399.PMC10276593.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10035922","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}
引用次数: 0
Validation of the International Consensus Group Criteria for Slide Review Following Automated Complete Blood Count at Jimma Medical Center, Ethiopia. 埃塞俄比亚Jimma医疗中心自动全血细胞计数后载玻片检查国际共识组标准的验证。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S402940
Girum Tesfaye Kiya, Aklilu Getachew Mamo, Sintayehu Asaye Biya, Dejene Gebre Gobena, Natal Demeke, Tilahun Yemane Shenkutie
{"title":"Validation of the International Consensus Group Criteria for Slide Review Following Automated Complete Blood Count at Jimma Medical Center, Ethiopia.","authors":"Girum Tesfaye Kiya,&nbsp;Aklilu Getachew Mamo,&nbsp;Sintayehu Asaye Biya,&nbsp;Dejene Gebre Gobena,&nbsp;Natal Demeke,&nbsp;Tilahun Yemane Shenkutie","doi":"10.2147/JBM.S402940","DOIUrl":"https://doi.org/10.2147/JBM.S402940","url":null,"abstract":"<p><strong>Background: </strong>The international consensus group suggested criteria for action following automated complete blood count and white blood cell differential analysis. These criteria were set based on data from laboratories of developed countries. It is highly important to validate the criteria in developing countries where infectious diseases are still rampant that can affect blood cell count and morphology. Thus, this study aimed to validate the consensus group criteria for slide review at Jimma Medical Center, Ethiopia from November 1, 2020 to February 30, 2021.</p><p><strong>Methods: </strong>The study comprised a total of 1685 patient samples from the daily laboratory workload of CBC analysis. The samples were collected in K2-EDTA tubes (Becton Dickinson) and analyzed using Coulter DxH 800 and Sysmex XT-1880 hematology analyzers. A slide review was done on two Wright-stained slides for each sample. All statistical analyses were performed using SPSS version 20 software.</p><p><strong>Results: </strong>There were 39.8% positive findings, the majority of which were related to red blood cells. The false negative and false positive rates for Sysmex and Coulter analyzer were 2.4% vs 4.8%; and 4.6% vs 4.7%, respectively. The false negative rate was unacceptably higher when we used physicians' triggered slide review, which was 17.3% and 17.9% for Sysmex and Coulter analyzers, respectively.</p><p><strong>Conclusion: </strong>Generally, the consensus group rules are suitable to use in our setting. However, we might still need to modify the rules, particularly to reduce the review rates. It is also necessary to confirm the rules with case mixes proportionally derived from the source population.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/01/14/jbm-14-213.PMC10046196.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9278553","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}
引用次数: 0
Congenital Methemoglobinemia: First Confirmed Case in the Arab Population with a Novel Variant in the CYB5R Gene in the State of Qatar: A Case Report. 先天性高铁血红蛋白血症:卡塔尔阿拉伯人群中CYB5R基因新变体的首例确诊病例:病例报告。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S395865
Abdulrahman Al-Abdulmalek, Reem Al-Sulaiman, Mohammad Abu-Tineh, Mohamed A Yassin
{"title":"Congenital Methemoglobinemia: First Confirmed Case in the Arab Population with a Novel Variant in the <i>CYB5R</i> Gene in the State of Qatar: A Case Report.","authors":"Abdulrahman Al-Abdulmalek,&nbsp;Reem Al-Sulaiman,&nbsp;Mohammad Abu-Tineh,&nbsp;Mohamed A Yassin","doi":"10.2147/JBM.S395865","DOIUrl":"https://doi.org/10.2147/JBM.S395865","url":null,"abstract":"<p><p>Methemoglobinemia (MetHb) is a rare hematological condition characterized by high methemoglobin levels in the blood. It happens when hemoglobin is oxidized, resulting in hypoxia and cyanosis, which may occur in inherited or acquired forms. Inherited or congenital methemoglobinemia is a rare autosomal recessive condition and has never been reported in the Arab population. Here we report a case of a 22-year-old Arab man with a positive family history who presented with bluish discoloration of the fingers and lips and was found to have methemoglobinemia. Genetic study on the patient and his family revealed compound heterozygous variants in the CYB5R3 Exon 5 c.431G>A p.Gly144Asp likely pathogenic variant and CYB5R3 Exon 9 c.871G>A p.Val291Met variant of unknown significance. We suggest that the novel c.871G>A p.Val291Met variant could be causative for methemoglobinemia.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10072335/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9325092","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}
引用次数: 0
Successful CNS-Centric Therapeutic Management and Genomic Profiling of Primary Cranial Vault Diffuse Large B-Cell Lymphoma. 原发性颅穹窿弥漫性大b细胞淋巴瘤以中枢神经系统为中心的成功治疗管理和基因组分析。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S391094
Jordan Davis, ErinMarie O Kimbrough, Muhamad Alhaj Moustafa, Liuyan Jiang, Vivek Gupta, Ephraim Parent, Han W Tun
{"title":"Successful CNS-Centric Therapeutic Management and Genomic Profiling of Primary Cranial Vault Diffuse Large B-Cell Lymphoma.","authors":"Jordan Davis,&nbsp;ErinMarie O Kimbrough,&nbsp;Muhamad Alhaj Moustafa,&nbsp;Liuyan Jiang,&nbsp;Vivek Gupta,&nbsp;Ephraim Parent,&nbsp;Han W Tun","doi":"10.2147/JBM.S391094","DOIUrl":"https://doi.org/10.2147/JBM.S391094","url":null,"abstract":"<p><p>Primary cranial vault lymphoma (PCVL) is a rare lymphoma involving the skull with or without extra- and intracranial extension. Most cases of PCVL are diffuse large B-cell lymphoma (DLBCL). We report a case of primary cranial vault diffuse large B-cell lymphoma (PCV-DLBCL) that was successfully treated with anthracycline-based chemoimmunotherapy (CIT) alternating with central nervous system (CNS)-directed CIT with high-dose methotrexate and high-dose cytarabine. CNS-centric therapy was given for suspected cerebral cortical involvement and presumed elevated risk of CNS recurrence. The patient has remained in complete remission for 4.25 years following treatment. We suggest that PCV-DLBCL is potentially curable with CNS-directed therapy. Additionally, we provide genomic profiling results indicating an indeterminate cell of origin and multiple genetic mutations which are not frequently seen in DLBCL.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/1b/de/jbm-14-49.PMC9879025.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10589969","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}
引用次数: 0
Association Between Helicobacter pylori Infection and Anemia Among Adult Dyspeptic Patients Attending Kiryandongo General Hospital, Uganda. 乌干达Kiryandongo总医院成人消化不良患者幽门螺杆菌感染与贫血的关系
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S392146
Daisy Asiimwe, Isaac Bangi, Jospeh Esanyu, Daniel Ojok, Benedict Okot, Clinton Olong, Robert Wagubi, Godfrey Kisembo, Fred Sempijja, Enoch Muwanguzi, Benson Okongo
{"title":"Association Between <i>Helicobacter pylori</i> Infection and Anemia Among Adult Dyspeptic Patients Attending Kiryandongo General Hospital, Uganda.","authors":"Daisy Asiimwe,&nbsp;Isaac Bangi,&nbsp;Jospeh Esanyu,&nbsp;Daniel Ojok,&nbsp;Benedict Okot,&nbsp;Clinton Olong,&nbsp;Robert Wagubi,&nbsp;Godfrey Kisembo,&nbsp;Fred Sempijja,&nbsp;Enoch Muwanguzi,&nbsp;Benson Okongo","doi":"10.2147/JBM.S392146","DOIUrl":"https://doi.org/10.2147/JBM.S392146","url":null,"abstract":"<p><strong>Purpose: </strong>To determine the prevalence of anemia and its association with <i>Helicobacter pylori</i> infection among adult dyspeptic patients.</p><p><strong>Patients and methods: </strong>A cross-sectional study was conducted among 283 dyspeptic patients at Kiryandongo General Hospital, in Uganda. A structured questionnaire was administered to capture demographic and clinical characteristics of study participants. Four milliliters of blood were then collected into an EDTA vacutainer for Complete Blood Count (CBC) and analyzed using HUMA COUNT 30<sup>TS</sup>, and peripheral blood smears were made and stained using Giemsa stain. Anemia was defined as hemoglobin levels <12g/dl in females and <13g/dl in men according to the World Health Organization (WHO). <i>Helicobacter pylori (H. pylori)</i> stool antigen test was performed using Whole power <i>H. pylori</i> Ag rapid test device, and saline stool preparation was examined for intestinal parasites. Chi-squared test and Logistic regression were performed to determine association, and a p-value of ≤0.05 was considered statistically significant.</p><p><strong>Results: </strong>The overall prevalence of <i>Helicobacter pylori</i> infection was 42.4% (120/283). The prevalence of anemia among <i>H. pylori</i>-infected patients was 25.8% (31/120) and 15.3% (25/163) among <i>H. pylori</i>-negative counterparts. <i>H. pylori</i> infection was significantly associated with anemia (p-value 0.042), age (p-value 0.02, 0.009), water sources (p-value 0.0049,) and intestinal parasitic infestation (p-value 0.02), respectively.</p><p><strong>Conclusion: </strong>This study has shown that the prevalence of <i>H. pylori</i> infection and anemia is high among dyspeptic patients at Kiryandongo General Hospital. <i>H. pylori</i> infection was found associated with anemia, age, water sources, and intestinal parasitic infestation. Routine screening of anemia in <i>H. pylori</i>-infected individuals and further studies to explore the relationship between anemia and <i>H. pylori</i> disease is highly recommended.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e6/c8/jbm-14-57.PMC9883989.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10592548","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}
引用次数: 1
Safe Blood Donation from Donors Using Antihypertensive Medication. A Multi-Center Retrospective Quality Study from South-East Norway. 使用抗高血压药物的献血者的安全献血。挪威东南部一项多中心回顾性质量研究。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S390609
Kathrine M Neuman Johnsen, Karin Magnussen, Christian Erstad, Sadaf Nabi Bhatti, Lise Sofie H Nissen-Meyer
{"title":"Safe Blood Donation from Donors Using Antihypertensive Medication. A Multi-Center Retrospective Quality Study from South-East Norway.","authors":"Kathrine M Neuman Johnsen,&nbsp;Karin Magnussen,&nbsp;Christian Erstad,&nbsp;Sadaf Nabi Bhatti,&nbsp;Lise Sofie H Nissen-Meyer","doi":"10.2147/JBM.S390609","DOIUrl":"https://doi.org/10.2147/JBM.S390609","url":null,"abstract":"<p><strong>Purpose: </strong>In Norway, blood donors using antihypertensive medication were deferred until 2015. Following revision of the national directive, these donors could be allowed, providing stable dose for at least 3 months, adequate blood pressure control and no adverse effects caused by the therapy. The new practice was evaluated by a quality study where the major aim was to establish whether donations from blood donors on antihypertensive medication pose a risk to the donor. The risk was assessed by counting the number and categorizing the adverse events related to blood donation. In addition, the quantitative effect of including these donors was calculated.</p><p><strong>Subjects and methods: </strong>In this retrospective quality study, blood donors on antihypertensive therapy were recruited from four different blood centers to fill out a questionnaire. A total of 265 donors answered questions regarding their health status, type of medication used, and adverse events connected to blood donation both before and after starting the therapy.</p><p><strong>Results: </strong>No severe adverse events were observed in donors on antihypertensive medications. The amount of mild adverse events, as exhibited by only 7 persons (0.46%) in this donor population, was the same as for donors without hypertensive treatment.</p><p><strong>Conclusion: </strong>Blood donation from persons on antihypertensive therapy poses no extra risk of severe adverse events, given the use of screening criteria to identify and bleed only low-risk donors.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/43/74/jbm-14-337.PMC10164375.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9498262","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}
引用次数: 1
Switching from Sucrose-Formulated rFVIII to Octocog Alfa (BAY 81-8973) Prophylaxis Improves Bleed Outcomes in the LEOPOLD Clinical Trials. 在LEOPOLD临床试验中,从蔗糖配方的rFVIII转向Octocog Alfa (BAY 81-8973)预防可改善出血结局。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S405624
Gili Kenet, Thomas Moulton, Brian M Wicklund, Sanjay P Ahuja, Miguel Escobar, Johnny Mahlangu
{"title":"Switching from Sucrose-Formulated rFVIII to Octocog Alfa (BAY 81-8973) Prophylaxis Improves Bleed Outcomes in the LEOPOLD Clinical Trials.","authors":"Gili Kenet,&nbsp;Thomas Moulton,&nbsp;Brian M Wicklund,&nbsp;Sanjay P Ahuja,&nbsp;Miguel Escobar,&nbsp;Johnny Mahlangu","doi":"10.2147/JBM.S405624","DOIUrl":"https://doi.org/10.2147/JBM.S405624","url":null,"abstract":"<p><strong>Introduction: </strong>Previous clinical trials established the efficacy and safety of sucrose-formulated recombinant factor (F) VIII (rFVIII-FS/Kogenate FS®/Helixate FS®) and octocog alfa (BAY 81-8973/Kovaltry®; LEOPOLD trials).</p><p><strong>Aim: </strong>To report the results of a post hoc subgroup analysis assessing efficacy and safety outcomes in patients with hemophilia A who were receiving rFVIII-FS prior to enrolling into the LEOPOLD I Part B and LEOPOLD Kids Part A clinical trials and switching to octocog alfa.</p><p><strong>Methods: </strong>LEOPOLD I Part B (NCT01029340) and LEOPOLD Kids Part A (NCT01311648) were octocog alfa Phase 3, multinational, open-label studies in patients with severe hemophilia A aged 12-65 years and ≤12 years, respectively. Annualized bleeding rate (ABR) was the efficacy endpoint for both studies. Safety endpoints included adverse events (AEs) and development of FVIII inhibitors.</p><p><strong>Results: </strong>Of the 113 patients in both LEOPOLD trials, 40 (35.4%) patients received rFVIII-FS prophylaxis pre-study and had data available for pre-study total ABR. In LEOPOLD I Part B (n = 22, 35.5%), median (Q1; Q3) total ABR decreased from 2.5 (0.0; 9.0) pre-study to 1.0 (0.0; 6.8), and from 1.0 (0.0; 6.0) pre-study to 0.0 (0.0; 6.02) in LEOPOLD Kids Part A (n = 18, 35.3%). Octocog alfa was well tolerated, and no patients had drug-related serious AEs or inhibitors.</p><p><strong>Conclusion: </strong>Treatment with octocog alfa prophylaxis appeared to have a favorable risk-benefit profile compared with rFVIII-FS and thus could be an effective and improved alternative strategy for individualized treatment for children, adolescent and adult patients with severe hemophilia A currently on rFVIII-FS treatment.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/6e/16/jbm-14-379.PMC10257928.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9624145","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}
引用次数: 0
"Severe Anemia: A Case Report of an Uncommon Precipitant of Schizophrenia Relapse". 《重度贫血:精神分裂症复发罕见诱因1例》。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S407722
Krittisak Anuroj, Siwat Chongbanyatcharoen, Romteera Chiencharoenthanakij
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