Journal of Blood Medicine最新文献

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Identification of Laboratory Biomarkers for Early Detection and Clinical Management of Post-Acute Syndrome Among Survivors of the 2013-2016 West Africa Ebola Outbreak in Sierra Leone. 2013-2016年塞拉利昂西非埃博拉疫情幸存者急性后综合征早期检测和临床管理的实验室生物标志物鉴定
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S371239
Raoul Emeric Guetiya Wadoum, Stephen Sevalie, Maurice Baimba Kargbo, Andrew Clarke, Sherry Bangura, Mariatu Kargbo, Hawa Mariama Sesay, Abdul H Kamara, Jamil Bangura, Alie F Kamara, Sophie Allieu, Hassan Rogers, Maurizio Mattei, Vittorio Colizzi, Carla Montesano, Edwin J J Momoh
{"title":"Identification of Laboratory Biomarkers for Early Detection and Clinical Management of Post-Acute Syndrome Among Survivors of the 2013-2016 West Africa Ebola Outbreak in Sierra Leone.","authors":"Raoul Emeric Guetiya Wadoum,&nbsp;Stephen Sevalie,&nbsp;Maurice Baimba Kargbo,&nbsp;Andrew Clarke,&nbsp;Sherry Bangura,&nbsp;Mariatu Kargbo,&nbsp;Hawa Mariama Sesay,&nbsp;Abdul H Kamara,&nbsp;Jamil Bangura,&nbsp;Alie F Kamara,&nbsp;Sophie Allieu,&nbsp;Hassan Rogers,&nbsp;Maurizio Mattei,&nbsp;Vittorio Colizzi,&nbsp;Carla Montesano,&nbsp;Edwin J J Momoh","doi":"10.2147/JBM.S371239","DOIUrl":"https://doi.org/10.2147/JBM.S371239","url":null,"abstract":"<p><strong>Background: </strong>The clinical management of persistent medical conditions affecting Ebola survivors, generally described as a post-Ebola syndrome, remains a public health concern. We aimed to analyze Ebola survivors' laboratory biomarkers as compared to their non-infected household relatives to identify biomarkers that could guide the identification of survivors at increased risk of developing severe at odds with the non-severe post-Ebola syndrome.</p><p><strong>Materials and methods: </strong>Data were extracted from medical records of the Ebola survivors clinic, and we included only Ebola survivor's parameters recorded during the first baseline follow-up visit 2 weeks interval after their second negative PCR result. Moreover, household non-infected family contacts of survivors visiting the clinic during the same period were recruited as community control.</p><p><strong>Results: </strong>The mean age of survivors was 32.65 (IQR: 15.5, 38.25) years, and Ebola IgG immunoglobulin was detected in all, thus confirming their status. The statistical significance (all p < 0.05) observed in monocyte percentage (MONO%), cluster of differentiation 4 percentage (CD4%), alanine aminotransferase (ALT), creatinine (CREA), and creatinine kinase (C-kinase) proved to be clinically significant as compared to the household relatives' group. Interestingly, the linear regression analysis indicated that the duration at ETU was negatively associated with lymphocyte percentage with a 5% lymphocyte decrease per day spent at ETU. Finally, there was a significant (p < 0.05) association between hematological (Hb, PCV, MCV, MCH), biochemical (ALT, CREA, C-kinase, T-cholesterol, triglycerides) parameters and the risk of developing severe complications.</p><p><strong>Conclusion: </strong>We recommend clinicians closely monitor Hb, PCV, MCV, MCH, ALT, CREA, C-kinase, T-cholesterol, triglycerides and lymphocytes as clinically relevant laboratory biomarkers to identify survivors at higher risk of developing severe post-acute syndrome upon discharge from Ebola treatment unit including headache, abdominal pain, chest pain, ocular complication, arthralgia, hearing difficulty and erectile dysfunction which can impact health-related quality of life among Ebola survivors.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"119-132"},"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/08/65/jbm-14-119.PMC9930681.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9315414","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
Prevalence of ABO and Rh Blood Group Among Volunteer Blood Donors at the Blood and Tissue Bank Service in Addis Ababa, Ethiopia. 埃塞俄比亚亚的斯亚贝巴血液和组织库服务志愿献血者中ABO和Rh血型的流行情况
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S392211
Getu Jenbere Debele, Fekadu Urgessa Fita, Melatwork Tibebu
{"title":"Prevalence of ABO and Rh Blood Group Among Volunteer Blood Donors at the Blood and Tissue Bank Service in Addis Ababa, Ethiopia.","authors":"Getu Jenbere Debele,&nbsp;Fekadu Urgessa Fita,&nbsp;Melatwork Tibebu","doi":"10.2147/JBM.S392211","DOIUrl":"https://doi.org/10.2147/JBM.S392211","url":null,"abstract":"<p><strong>Background: </strong>The discovery of the ABO blood group system and testing of blood donors highly reduced the fatalities associated with blood transfusion reactions and improved the safety of blood transfusion. Blood group antigens are found on the surface of red blood cells that are inherited biological characteristics that do not change throughout life in healthy individuals.</p><p><strong>Objective: </strong>To determine the prevalence ABO and Rh blood groups Among Volunteer Blood Donors at Ethiopian blood and tissue bank service (EBTBS), Addis Ababa.</p><p><strong>Methods: </strong>A cross-sectional study was carried out from January 2022 to May 2022, on 1700 volunteer blood donors to assess prevalence of ABO and Rh blood groups among volunteer blood donors at the Ethiopian blood and tissue bank service. All tests were performed using fully automated immunohematology analyzer (Galileo Neo Immucor). Data processing and analysis were undertaken by using Statistical Package for the Social Sciences (SPSS) version 26. An ethical clearance letter was obtained from Addis Ababa University and informed consent was also obtained from the participants of the study.</p><p><strong>Results: </strong>A total of 1700 donors were included, of which 57% of donors were males. The majority of the donors belonged to the age group between 18 and 25 years old (53%). The antigen frequencies of ABO and Rh(D) blood group system showed that O was the most prevalent blood group 44.65% followed by A (28.41%), B (21.24%), and AB (5.71%). The Rh-positive donors were more prevalent (94.82%) as oppose to the Rh-negative (5.18%).</p><p><strong>Conclusion: </strong>The knowledge of the distribution of blood groups is very important for blood banks and transfusion services which play an important role in the patient's health care. The findings of the ABO blood group in this study were comparable to other studies conducted in Ethiopia.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"19-24"},"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/51/09/jbm-14-19.PMC9868278.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10614312","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: 2
Immune Thrombocytopenia Relapse in Patients Who Received mRNA COVID-19 Vaccines [Letter]. mRNA - COVID-19疫苗接种后免疫性血小板减少症复发的研究[字母]。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S419849
Anumta Ali, Syeda Adeena Zafar, Syeda Sakina Zehra
{"title":"Immune Thrombocytopenia Relapse in Patients Who Received mRNA COVID-19 Vaccines [Letter].","authors":"Anumta Ali,&nbsp;Syeda Adeena Zafar,&nbsp;Syeda Sakina Zehra","doi":"10.2147/JBM.S419849","DOIUrl":"https://doi.org/10.2147/JBM.S419849","url":null,"abstract":"","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"377-378"},"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/69/4c/jbm-14-377.PMC10243605.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9653471","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
Multiply Relapsed Secondary CNS Non-Germinal Center Diffuse Large B-Cell Lymphoma Successfully Treated with CNS-Centric Therapy. 以中枢为中心的治疗成功治疗多发复发继发性中枢非生发中心弥漫性大b细胞淋巴瘤。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S405521
Lyndsey L Fournier, ErinMarie O Kimbrough, Muhamad Alhaj Moustafa, Ke Li, Madiha Iqbal, Vivek Gupta, Han W Tun
{"title":"Multiply Relapsed Secondary CNS Non-Germinal Center Diffuse Large B-Cell Lymphoma Successfully Treated with CNS-Centric Therapy.","authors":"Lyndsey L Fournier,&nbsp;ErinMarie O Kimbrough,&nbsp;Muhamad Alhaj Moustafa,&nbsp;Ke Li,&nbsp;Madiha Iqbal,&nbsp;Vivek Gupta,&nbsp;Han W Tun","doi":"10.2147/JBM.S405521","DOIUrl":"https://doi.org/10.2147/JBM.S405521","url":null,"abstract":"<p><p>Secondary central nervous system involvement by systemic diffuse large B-cell lymphoma (DLBCL) carries a very poor prognosis. We present a female patient who had two episodes of intracerebral central nervous system (CNS)-only relapse of systemic non-germinal center diffuse large B-cell lymphoma (NGC-DLBCL). Her treatment at initial diagnosis consisted of induction with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) and intrathecal (IT) - methotrexate (MTX) followed by consolidation with autologous stem cell transplant (ASCT) after high-dose carmustine, etoposide, cytarabine, and melphalan (BEAM) chemotherapy. She had the first CNS-only relapse 1.5 years post-ASCT and received whole brain radiation therapy (WBRT). She developed the second intracerebral CNS-only relapse 2 years post-WBRT. A CNS-centric therapeutic approach with salvage chemoimmunotherapy incorporating rituximab, high-dose methotrexate (HD-MTX), high-dose cytarabine (HiDAC), and ibrutinib was utilized for her second CNS-only relapse. She underwent consolidation with a second ASCT following high-dose carmustine (BCNU) and thiotepa chemotherapy. Given her high risk of CNS recurrence, she was started on maintenance ibrutinib. To date, she has remained in complete remission for 3 years. In our experience, multiply relapsed secondary CNS lymphoma (SCNSL) with this response is very rare. We suggest one CNS-centric therapeutic approach that can potentially salvage patients with SCNSL who have not had prior exposure to adequate CNS-directed therapies but acknowledge that additional research is necessary to validate our findings.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"455-461"},"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/14/3b/jbm-14-455.PMC10440079.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10053234","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
Efficacy of rFIXFc versus N9-GP Prophylaxis in Patients with Hemophilia B: Matching-Adjusted Indirect Comparison of B-LONG and PARADIGM 2 Trials. rFIXFc与N9-GP预防在B型血友病患者中的疗效:B- long和范式2试验的匹配调整间接比较
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S389094
Maria Elisa Mancuso, Daniel Eriksson, Aletta Falk, Zalmai Hakimi, Piotr Wojciechowski, Marlena Wdowiak, Robert Klamroth
{"title":"Efficacy of rFIXFc versus N9-GP Prophylaxis in Patients with Hemophilia B: Matching-Adjusted Indirect Comparison of B-LONG and PARADIGM 2 Trials.","authors":"Maria Elisa Mancuso,&nbsp;Daniel Eriksson,&nbsp;Aletta Falk,&nbsp;Zalmai Hakimi,&nbsp;Piotr Wojciechowski,&nbsp;Marlena Wdowiak,&nbsp;Robert Klamroth","doi":"10.2147/JBM.S389094","DOIUrl":"https://doi.org/10.2147/JBM.S389094","url":null,"abstract":"<p><strong>Purpose: </strong>For patients with hemophilia B, extended half-life factor IX (FIX) products are available for prophylaxis and for treating bleeds. Different methods are used to extend the half-lives of recombinant FIX Fc fusion protein (rFIXFc) and nonacog beta pegol (N9-GP). This affects their biodistribution and plasma FIX levels, although differences do not always correlate with clinical outcomes. A matching-adjusted indirect comparison (MAIC) of prophylaxis with rFIXFc and N9-GP was performed, based on licensed dosing in the European Union.</p><p><strong>Patients and methods: </strong>Combined rFIXFc data from the weekly and individualized interval prophylaxis arms of the B-LONG clinical trial, and N9-GP data from the 40 IU/kg once-weekly prophylaxis arm of PARADIGM 2 were used in a MAIC. Individual patient data for rFIXFc (n=87) were matched to aggregated data for N9-GP (n=29). Estimated annualized bleeding rates (ABRs) for rFIXFc were recalculated using a Poisson regression model with adjustment for over-dispersion, and compared with ABRs reported for N9-GP, using incidence rate ratios (IRRs) with 95% confidence interval (CI).</p><p><strong>Results: </strong>There was no evidence of significant differences in estimated ABRs between prophylaxis with rFIXFc and N9-GP. Analysis of pooled rFIXFc weekly and interval-adjusted dosing compared with N9-GP 40 IU/kg once weekly produced estimated ABRs of 2.59 versus 2.51 (IRR 1.03; 95% CI 0.56-1.89), as well as 1.34 versus 1.22 (IRR 1.10; 95% CI 0.42-2.91) and 1.13 versus 1.29 (IRR 0.88; 95% CI 0.47-1.63) for overall, spontaneous, and traumatic bleeding events, respectively.</p><p><strong>Conclusion: </strong>The study did not reveal any significant differences in the efficacy of rFIXFc and N9-GP prophylaxis. Given differences in trough levels (rFIXFc dosing was targeted to achieve a trough 1-3 IU/dL above baseline versus a reported estimated N9-GP mean trough of 27.3 IU/dL), interpreting plasma FIX levels as potential surrogate efficacy markers requires consideration of compound-specific pharmacokinetic profiles.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"427-434"},"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/a8/d4/jbm-14-427.PMC10390690.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9930383","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
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":"14 ","pages":"413-425"},"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
Howell-Jolly Body-Like Inclusions in Coronavirus Disease 2019 (COVID-19): Possible Novel Findings. 2019冠状病毒病(COVID-19)中的豪威尔-乔利体样内含物:可能的新发现
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S399596
Amaylia Oehadian, Ian Huang, Andini Kartikasari, Bachti Alisjahbana, Delita Prihatni
{"title":"Howell-Jolly Body-Like Inclusions in Coronavirus Disease 2019 (COVID-19): Possible Novel Findings.","authors":"Amaylia Oehadian,&nbsp;Ian Huang,&nbsp;Andini Kartikasari,&nbsp;Bachti Alisjahbana,&nbsp;Delita Prihatni","doi":"10.2147/JBM.S399596","DOIUrl":"https://doi.org/10.2147/JBM.S399596","url":null,"abstract":"<p><strong>Background: </strong>During COVID-19 pandemic, it is difficult to distinguish febrile patient infected by SARS-CoV-2 or bacterial causes. Howell-Jolly bodies are a well-known entity found in red blood cells. They are nuclear fragments, composed of deoxyribonucleic acid, commonly observed in the peripheral blood smears of hyposplenic or asplenic patients. Recently, similar inclusions often referred to as Howell-Jolly body-like inclusions (HJBLIs) have been reported in the neutrophils of patients with acquired immune deficiency syndrome (AIDS) and COVID-19 patient.</p><p><strong>Aim: </strong>To explore whether HJBLIs in peripheral blood smear could differentiate between patients with confirmed SARS-CoV-2 and bacterial pneumonia.</p><p><strong>Methods: </strong>We performed cross-sectional study using secondary data from COVID-19 database and re-evaluated peripheral blood smears to identify HJBLIs. We included confirmed COVID-19 adults age >18 years who were hospitalized in Dr. Hasan Sadikin General Hospital, Bandung, Indonesia from March 1st 2020-May 31st 2020. We also examined peripheral blood smears in patients with confirmed bacterial pneumonia as a control group. Clinical characteristics including disease severity, CURB-65 score, comorbidity, and the present of HJBLIs in peripheral blood smears were evaluated.</p><p><strong>Results: </strong>Overall, 33 patients were included: 22 were confirmed COVID-19 and 11 were confirmed bacterial pneumonia. The median (interquartile range) age in COVID-19 and patients with bacterial pneumonia were 53 years (40-64) vs 57 years (53-71), respectively. Compared with patients with bacterial pneumonia, HJBLIs were significantly higher in COVID-19 patients [21/22 (80.8%) vs 5/11 (45.5%), p 0.001].</p><p><strong>Conclusion: </strong>Howell-Jolly body-like inclusions could be a potential feature to help differentiate between COVID-19 and bacterial pneumonia.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"14 ","pages":"233-238"},"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/57/d3/jbm-14-233.PMC10066893.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9253206","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":"14 ","pages":"399-411"},"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
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":"14 ","pages":"209-211"},"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
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
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