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A Critical Review of Sickle Cell Disease Burden and Challenges in Sub-Saharan Africa. 撒哈拉以南非洲镰状细胞病负担和挑战的重要审查。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S406196
Obi Peter Adigwe, Solomon Oloche Onoja, Godspower Onavbavba
{"title":"A Critical Review of Sickle Cell Disease Burden and Challenges in Sub-Saharan Africa.","authors":"Obi Peter Adigwe,&nbsp;Solomon Oloche Onoja,&nbsp;Godspower Onavbavba","doi":"10.2147/JBM.S406196","DOIUrl":"https://doi.org/10.2147/JBM.S406196","url":null,"abstract":"<p><p>Sickle cell disease is caused by an abnormality of the β-globin gene and is characterised by sickling of the red blood cells. Globally, sub-Saharan African countries share the highest burden of the disease. This study aimed at critically reviewing studies focusing on challenges of sickle cell anaemia in sub-Saharan Africa. A literature search was carried out in five major databases. Articles that met the inclusion criteria were included in the bibliometric review and critical analysis. A majority of the studies were undertaken in the West African region (85.5%), followed by Central Africa (9.1%). Very few studies had been undertaken in East Africa (3.6%), whilst the Southern African region had the fewest studies (1.8%). Distribution in relation to country revealed that three quarters of the studies were carried out in Nigeria (74.5%), followed by the Democratic Republic of the Congo (9.1%). According to healthcare settings, a strong majority of the studies were undertaken in tertiary health care facilities (92.7%). Major themes that emerged from the review include interventions, cost of treatment, and knowledge about sickle cell disease. Public health awareness and promotion as well as improving the quality of sickle cell centers for prompt management of patients with sickle cell disorder was identified as a critical strategy towards reducing the burden of the disease in sub-Saharan Africa. To achieve this, governments in countries located in this region need to adopt a proactive strategy in addressing gaps that have been identified in this study, as well as instituting other relevant measures, such as continuous media engagement and public health interventions relating to genetic counselling. Reforms in other areas that can help reduce the disease burden, include training of practitioners and equipping sickle cell disease treatment centers according to World Health Organization specifications.</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/69/d6/jbm-14-367.PMC10239624.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9592710","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
Anemia Among Women Using Family Planning at Public Health Facilities in Ambo Town, Central Ethiopia: Multi-Center Cross-Sectional Study. 埃塞俄比亚中部安博镇公共卫生机构计划生育妇女贫血:多中心横断面研究
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S400191
Shalama Lekasa Nagari, Gudina Egata, Ame Mehadi, Tahir Ahmed Hassen, Temam Beshir Raru, Mohammed Abdurke, Mohammed Yuya, Shemsedin Abdulkadir, Hiwot Berhanu, Kedir Teji Roba
{"title":"Anemia Among Women Using Family Planning at Public Health Facilities in Ambo Town, Central Ethiopia: Multi-Center Cross-Sectional Study.","authors":"Shalama Lekasa Nagari,&nbsp;Gudina Egata,&nbsp;Ame Mehadi,&nbsp;Tahir Ahmed Hassen,&nbsp;Temam Beshir Raru,&nbsp;Mohammed Abdurke,&nbsp;Mohammed Yuya,&nbsp;Shemsedin Abdulkadir,&nbsp;Hiwot Berhanu,&nbsp;Kedir Teji Roba","doi":"10.2147/JBM.S400191","DOIUrl":"https://doi.org/10.2147/JBM.S400191","url":null,"abstract":"<p><strong>Background: </strong>Anemia affects more than a quarter of non-pregnant women over the globe, with Sub-Saharan Africa bearing a disproportionate share. Although the use of family planning is beneficial in reducing anemia, lack of scientific study on anemia among family planning users of reproductive-age women is notable, particularly in the study setting. The purpose of this study was to determine the extent of anemia and associated factors in women who used family planning.</p><p><strong>Methods: </strong>A cross-sectional multi-centered study was conducted from March 3 to 29, 2019, among 443 non-pregnant reproductive age (15 to 49 years) women receiving family planning services in Ambo town. Sample size was calculated using Epi-info version 7 software. Participants were selected by systematic random sampling technique. Trained data collectors collected data using a structured pretested questionnaire, as well as venous blood and stool samples. Epi-Data and SPSS were used to enter and analyze data. The effect of independent variables on the outcome variable was determined by binary logistic regression analysis with adjusted odds ratio at 95% confidence interval and 5% margin of error. P-value <0.05 was used to declare statistical significance.</p><p><strong>Results: </strong>This study revealed 28% (95% CI:23.9%, 32.3%) magnitude of anemia. Age of 25-35 years [AOR:2.84, 95% CI:1.74, 4.64], implantable family planning method [AOR: 0.34, 95% CI: 0.12, 0.96], no previous use of family planning [AOR:2.62, 95% CI: 1.62, 4.24], household food insecurity [AOR: 2.04, 95% CI: 1.06, 3.93], parasite infestations [AOR:2.01, 95% CI: 1.12, 3.63], and regular intake of coffee/tea within 30 minutes post meal [AOR:3.85, 95% CI:1.24, 11.92] were independently associated with anemia.</p><p><strong>Conclusion: </strong>Anemia is a moderate public health concern among reproductive-age women receiving family planning services in the study area. There are missed opportunities to address the anemia burden during family planning services. This study emphasizes the importance of nutritional screening for early detection and targeted interventions for healthcare workers in reducing missed opportunities to prevent and control anemia in vulnerable populations.</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/1f/94/jbm-14-83.PMC9922510.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10794022","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
RNAi for the Treatment of People with Hemophilia: Current Evidence and Patient Selection. RNAi治疗血友病:当前证据和患者选择。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S390521
Sara Boyce, Savita Rangarajan
{"title":"RNAi for the Treatment of People with Hemophilia: Current Evidence and Patient Selection.","authors":"Sara Boyce,&nbsp;Savita Rangarajan","doi":"10.2147/JBM.S390521","DOIUrl":"https://doi.org/10.2147/JBM.S390521","url":null,"abstract":"<p><p>Severe hemophilia is associated with spontaneous, prolonged and recurrent bleeding. Inadequate prevention and treatment of bleeding can lead to serious morbidity and mortality. Due to the limitations of intravenous clotting factor replacement, including the risk of inhibitory antibodies, innovative novel therapies have been developed that have dramatically changed the landscape of hemophilia therapy. Ribonucleic acid interference (RNAi) has brought the opportunity for multiple strategies to manipulate the hemostatic system and ameliorate the bleeding phenotype in severe bleeding disorders. Fitusiran is a RNAi therapeutic that inhibits the expression of the natural anticoagulant serpin antithrombin. Reduction in antithrombin is known to cause thrombosis if coagulation parameters are otherwise normal and can rebalance hemostasis in severe hemophilia. Reports from late stage clinical trials of fitusiran in hemophilia A and B participants, with and without inhibitory antibodies to exogenous clotting factor, have demonstrated efficacy in preventing bleeding events showing promise for a future \"universal\" prophylactic treatment of individuals with moderate-severe hemophilia.</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/25/a0/jbm-14-317.PMC10132380.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9746638","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
Managing the Cerebrovascular Complications of Sickle Cell Disease: Current Perspectives. 处理镰状细胞病的脑血管并发症:目前的观点。
IF 2
Journal of Blood Medicine Pub Date : 2023-01-01 DOI: 10.2147/JBM.S383472
Jennifer Light, Maria Boucher, Jacquelyn Baskin-Miller, Mike Winstead
{"title":"Managing the Cerebrovascular Complications of Sickle Cell Disease: Current Perspectives.","authors":"Jennifer Light,&nbsp;Maria Boucher,&nbsp;Jacquelyn Baskin-Miller,&nbsp;Mike Winstead","doi":"10.2147/JBM.S383472","DOIUrl":"https://doi.org/10.2147/JBM.S383472","url":null,"abstract":"<p><p>The importance of protecting brain function for people with sickle cell disease (SCD) cannot be overstated. SCD is associated with multiple cerebrovascular complications that threaten neurocognitive function and life. Without screening and preventive management, 11% of children at 24% of adults with SCD have ischemic or hemorrhagic strokes. Stroke screening in children with SCD is well-established using transcranial Doppler ultrasound (TCD). TCD velocities above 200 cm/s significantly increase the risk of stroke, which can be prevented using chronic red blood cell (RBC) transfusion. RBC transfusion is also the cornerstone of acute stroke management and secondary stroke prevention. Chronic transfusion requires long-term management of complications like iron overload. Hydroxyurea can replace chronic transfusions for primary stroke prevention in a select group of patients or in populations where chronic transfusions are not feasible. Silent cerebral infarction (SCI) is even more common than stroke, affecting 39% of children and more than 50% of adults with SCD; management of SCI is individualized and includes careful neurocognitive evaluation. Hematopoietic stem cell transplant prevents cerebrovascular complications, despite the short- and long-term risks. Newer disease-modifying agents like voxelotor and crizanlizumab, as well as gene therapy, may treat cerebrovascular complications, but these approaches are investigational.</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/f5/6c/jbm-14-279.PMC10112470.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9753979","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
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":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/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":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/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
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":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/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":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/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
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":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/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
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
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