Journal of Blood Medicine最新文献

{"title":"Clinical and Genomic Profile of Primary Cranial Neurolymphomatosis.","authors":"Emily B Wolf, Robin Imperial, Liuyan Jiang, Amit K Agarwal, Han W Tun","doi":"10.2147/JBM.S459123","DOIUrl":"10.2147/JBM.S459123","url":null,"abstract":"<p><p>Primary cranial neurolymphomatosis (PCNL) is a rare subtype of primary CNS lymphoma (PCNSL) in which infiltrative lymphomatous involvement is confined to cranial nerves. Here, we report a case of PCNL with successful genomic profiling. A 57-year-old male had a lengthy prediagnostic phase spanning approximately 30 months, characterized by multiple episodes of cranial neuropathies managed by steroids. At the time of diagnosis, the patient had right-sided cranial neuropathies involving cranial nerves (CN) V, VI, and VII. Pathological findings of the right cavernous lesion biopsy were consistent with large B-cell lymphoma-infiltrating nerve fibers. The clinical course was aggressive and refractory, characterized by relentless progression with the development of cervical spinal neurolymphomatosis, cerebrospinal fluid involvement, and ependymal and intraparenchymal cerebral involvement, despite multiple lines of therapy, including chemoimmunotherapy, Bruton's tyrosine kinase inhibitor, radiation, autologous stem cell transplant, chimeric antigen receptor T-cell therapy (CAR-T), and whole-brain radiation. The patient survived for 22 months from the time of the initial diagnosis and 52 months after the first episode of cranial neuropathy. Next-generation sequencing identified mutations (MYD88, CD79b, and PIM1) that are frequently observed in PCNSL. The unusual findings included a total of 22 mutations involving PIM1, indicating a highly active aberrant somatic hypermutation and two missense CXCR4 mutations. CXCR4 mutations have never been described in PCNSL and may have implications for disease biology and therapeutic interventions. We provide a literature review to further elucidate PCNL.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"291-303"},"PeriodicalIF":2.1,"publicationDate":"2024-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11212808/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141468125","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}

{"title":"Successful Treatment of Steroid-Refractory Immune Thrombocytopenia in a Patient Developing Multiple Myeloma While on Immune Checkpoint Inhibitor Therapy for Lung Cancer: A Case Report.","authors":"Yudai Hayashi, Masao Tsukada, Daisuke Shinoda, Marina Matsui, Kanichi Iwama, Koichi Kajiwara, Kozai Yasuji","doi":"10.2147/JBM.S468921","DOIUrl":"10.2147/JBM.S468921","url":null,"abstract":"<p><p>Immune checkpoint inhibitor-related thrombocytopenia (irTCP) is a relatively rare immune-related adverse event (irAE); however, overall survival may worsen when it occurs. Prolonged use of high-dose steroids can diminish the effectiveness of immune checkpoint inhibitor (ICI) therapy on the primary disease because of T lymphocyte suppression, thus early tapering is necessary. We experienced a rare case of a 79-year-old male who concurrently developed irTCP and multiple myeloma (MM) during treatment with ICIs for lung adenocarcinoma. The patient exhibited severe thrombocytopenia and elevated serum IgA levels. Based on various tests, we diagnosed MM and irTCP. Despite administering the standard bortezomib plus dexamethasone (Bd therapy) treatment for MM, there was no response and the irTCP was steroid-resistant. Consequently, we administered a regimen including daratumumab (DPd therapy) for steroid-resistant irTCP and refractory MM, which resulted in a response. As a result, we were able to avoid prolonged use of high-dose steroids and the patient is stable without exacerbation of lung adenocarcinoma for 1 year and 5 months after the onset of MM. To our knowledge, there are no cases of MM developing during ICI treatment and this is the first case report in which daratumumab was effective for the treatment of irTCP.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"285-290"},"PeriodicalIF":2.1,"publicationDate":"2024-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11198016/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141450585","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}

{"title":"Haematological Indices in Acute Coronary Syndrome Patients in Ethiopia: A Comparative Cross-Sectional Study.","authors":"Samuel Tadesse, Esayas Kebede Gudina, Daniel Yilma, Elsah Tegene Asefa, Tilahun Yemane, Andualem Mossie","doi":"10.2147/JBM.S457371","DOIUrl":"10.2147/JBM.S457371","url":null,"abstract":"<p><strong>Background: </strong>Numerous biomarkers are used as diagnostic, prognostic, and predictive indicators of myocardial ischemia. The most commonly used biomarkers are cardiac troponin I (Tn-I) and creatinine kinase (CK-MB). However, in developing nations, their availability in primary care settings is extremely limited. In such situations, easily available assays such as complete blood count (CBC) should be investigated as prognostic indicators in individuals with acute coronary syndrome (ACS).</p><p><strong>Objective: </strong>This study aimed to compare the pattern of haematological indices and blood cell ratios of ACS patients compared with apparently healthy controls.</p><p><strong>Methods: </strong>Patients diagnosed with ACS were recruited consecutively between 01 May 2022 and 31 October 2023 at Jimma Medical Center (JMC). Biochemical analyses and complete blood counts were performed. Analysis of variance was performed to compare the continuous variables. Spearman correlation coefficient tests were performed to correlate hematologic parameters with high sensitive troponin-I (hs-Tn-I) levels.</p><p><strong>Results: </strong>This study enrolled 220 participants (110 patients with ACS and age, sex, and place of residence matched 110 non-ACS controls). From ACS group 99 (90%) were diagnosed with ST-elevated myocardial infarction. The ACS group had a significantly greater mean platelet volume (MPV), white blood cell count, red cell distribution width (RDW), neutrophil-to-lymphocyte ratio, and platelet-to-lymphocyte ratio. The RDW (r = 0.248, p = 0.009) and MPV (r = 0.245, p = 0.009) were significantly positively correlated with hs-Tn-I levels in the ACS group. MPV, RDW, and monocyte count were significantly higher in non-survivor ACS patients (p <0.05).</p><p><strong>Conclusion: </strong>The significant differences observed in haematological parameters between individuals with ACS and healthy controls suggest the potential utility of these easily accessible and cost-effective diagnostics in predicting future morbidity and ACS risk. Incorporating these routine evaluations into clinical practice could enhance risk assessment and improve patient outcomes.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"275-284"},"PeriodicalIF":2.1,"publicationDate":"2024-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11193461/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141442735","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}

{"title":"The Characteristics of Compound Heterozygosity for Hemoglobin G-Makassar with Hb E in Malaysia.","authors":"Roszymah Hamzah, Ahmad Sabry Mohamad, Norafiza Mohd Yasin, Ezalia Esa, Guo Chen, Veena Selvaratnam","doi":"10.2147/JBM.S432849","DOIUrl":"10.2147/JBM.S432849","url":null,"abstract":"<p><strong>Background: </strong>Human hemoglobin of G-Makassar and hemoglobin E (Hb E) are hemoglobin variants that affect Beta (β) globin. Hb G-Makassar is a very rare variant while Hb E is estimated to affect at least one million people worldwide. Both Hb G-Makassar and Hb E can be inherited in the heterozygous, homozygous or compound heterozygous state. This case series describes the characteristics of four individuals with compound heterozygosity for Hb G-Makassar/Hb E cases in Malaysia. To the best of our knowledge, these are the only four individuals with this genotype reported in the literature.</p><p><strong>Case series: </strong>We present four cases of compound heterozygosity for Hb G-Makassar/Hb E identified from October 2014 to January 2021. All the cases were incidental findings whereby the screening Hb analysis showed the presence of peaks in both Hb S and Hb E zones on capillary electrophoresis (CE) and cation-exchange high-performance liquid chromatography (HPLC). Molecular analysis confirmed the findings of compound heterozygous Hb G-Makassar/Hb E. Two cases had a history of anemia secondary to unrelated conditions that resolved with treatment of the underlying cause. The other two cases were asymptomatic individuals who were detected through Malaysia's National Thalassemia Screening program. On the last follow-up, all the individuals were well, non-transfusion dependent, and had no reported history of chronic anemia, bleeding, hemolysis or thromboembolism complications.</p><p><strong>Conclusion: </strong>The cases reported here highlight the possibilities for rare compound heterozygous states in multi-ethnicity populations such as Malaysia. Compound heterozygous Hb G-Makassar/Hb E individuals are clinically silent with laboratory values suggesting microcytic and hypochromic red blood cells. Further local epidemiology or population studies with genotyping tests are required for a better understanding of the diversity of its clinical phenotype.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"255-264"},"PeriodicalIF":2.0,"publicationDate":"2024-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11144427/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141199763","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}

{"title":"Can COVID-19 Increase Platelet in Adult Immune Thrombocytopenia During the TPO-RA Administration? A Real-World Observational Study.","authors":"Xiaoyu Wang, Yingqiao Zhu, Dan Liu, Lijun Zhu, Juan Tong, Changcheng Zheng","doi":"10.2147/JBM.S457545","DOIUrl":"10.2147/JBM.S457545","url":null,"abstract":"<p><strong>Introduction: </strong>COVID-19 infection has brought new challenges to the treatment of adult patients with immune thrombocytopenia (ITP). In adult ITP patients, there have been no relevant reports exploring the incidence, clinical characteristics, and risk factors of platelet elevation after COVID-19 infection.</p><p><strong>Materials and methods: </strong>A total of 66 patients with previously diagnosed ITP from December 2022 to February 2023 in a single-center were collected and analyzed for this real-world clinical retrospective observational study.</p><p><strong>Results: </strong>In the platelet count increased group (n = 19), 13 patients (68.4%) were using thrombopoietin receptor agonists (TPO-RA) treatment at the time of COVID-19 infection; the median platelet count was 52 (2-207) ×10⁹/L at the last visit before infection and 108 (19-453) ×10⁹/L at the first visit after infection. In the platelet count stable group (n = 19) and platelet count decreased group (n = 28), 9 (47.4%) and 8 (28.6%) patients were using TPO-RA at the time of infection, respectively. ITP patients treated with TPO-RA had a significantly higher risk of increased platelet count than those not treated with TPO-RA at the time of infection (platelet count increased group vs platelet count decreased group: OR: 5.745, p = 0.009; platelet count increased group vs the non-increased group: OR: 3.616, p = 0.031). In the platelet count increased group, the median platelet count at 6 months post-infection was 67 (14-235) × 10⁹/L, which was significantly higher than the platelet level at the last visit before infection (p = 0.040).</p><p><strong>Conclusion: </strong>This study showed that some adult ITP patients had an increase in platelet count after COVID-19 infection, and this phenomenon was strongly associated with the use of TPO-RA at the time of infection. Although no thrombotic events were observed in this study, it reminds clinicians that they should be alert to the possibility of thrombotic events in the long-term management of adult ITP patients during the COVID-19 pandemic.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"217-225"},"PeriodicalIF":2.0,"publicationDate":"2024-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11088401/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140911208","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}

{"title":"Cardiovascular Consequences of Sickle Cell Disease.","authors":"Salem Bahashwan, Rahaf Mohammad Almuhanna, Maryam Taher Al Hazza, Reem Wajdi Baarma, Abdulrahman Yousif AlNajjar, Faris Sameer Siddiqui, Shouq Ziyad Fatani, Ahmed Barefah, Hatem Alahwal, Abdullah Almohammadi, Osman Radhwi, Alaa S Algazzar, Eman M Mansory","doi":"10.2147/JBM.S455564","DOIUrl":"10.2147/JBM.S455564","url":null,"abstract":"<p><strong>Background: </strong>Sickle cell disease is an inherited blood disorder which can lead to severe complications, particularly in the cardiovascular and respiratory systems, potentially resulting in arrhythmias, pulmonary hypertension (PH), and cardiomegaly. This study aims to investigate the risk of PH and arrhythmias in adult SCD patients.</p><p><strong>Methods: </strong>Retrospective analysis of medical records from King Abdulaziz University Hospital (KAUH) for patients with SCD aged 15 and above between 2009 and 2021. The study included 517 patients, with echocardiograms and electrocardiograms assessed according to the European Society of Cardiology/the European Respiratory Society (ESC/ERS) guidelines for categorizing PH risk (low, moderate, high) and detecting arrhythmias. Data analysis employed the Statistical Package for the Social Sciences (SPSS), utilizing quantitative and qualitative data representation. Multivariate logistic regression identified independent risk factors with odds ratios at a 95% confidence interval (CI).</p><p><strong>Results: </strong>Among participants, 50.3% were male, with a total sample average age of 34.45 ± 9.28 years. Results indicated that 1.4% of patients experienced arrhythmias, 3.7% had a moderate PH risk, and 3.3% were classified as high PH risk. Logistic regression revealed significant independent risk factors for PH and arrhythmia in patients with SCD, with chronic kidney disease (CKD) carrying the highest odds (26.4 times higher odds of PH and 15.36 times higher odds of arrhythmias).</p><p><strong>Conclusion: </strong>Patients with SCD are at risk for developing PH and various arrhythmias but are often underdiagnosed. Key risk factors for PH included CKD, liver cirrhosis, and pre-existing cardiac conditions. Arrhythmias were significantly associated with CKD and pre-existing cardiac conditions. To mitigate these risks, we recommend involving a multidisciplinary healthcare team in the care of adult patients with SCD. Future prospective studies are advised for early detection of PH and arrhythmias in hemoglobinopathy patients, potentially reducing mortality.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"207-216"},"PeriodicalIF":2.0,"publicationDate":"2024-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11086457/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140911260","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}

{"title":"The Montana Interfacility Blood Network: A Novel Lifesaving \"Hand-off\" for the Optimal Care of Rural Patients.","authors":"Gordon M Riha, Alyssa Johnson, Sadie Arnold, Michael S Englehart, Simon J Thompson","doi":"10.2147/JBM.S442134","DOIUrl":"10.2147/JBM.S442134","url":null,"abstract":"<p><strong>Purpose: </strong>The state of Montana encompasses and defines rural health care as it is known in the United States (US) today. This vast area is punctuated by pockets of health care availability with varying access to blood products for transfusion. Furthermore, timely transport is frequently challenged by weather that may limit air transportation options, resulting in multiple hours in ground transport to definitive care.</p><p><strong>Patients and methods: </strong>The Montana State Trauma Care Committee (MT-STCC) developed the Montana Interfacility Blood Network (MT-IBN) to ensure blood availability in geographically distanced cases where patients may otherwise not survive. The index case that led to the formal development of the MT-IBN is described, followed by a second case illustrating the IBN process.</p><p><strong>Results: </strong>This process and development manuscript details the innovative efforts of MT-STCC to develop this fledgling idea unique to rural US health care. We review guidelines that have been developed to define broad aspects of the MT-IBN including the reason to share resources, proper packaging, paperwork necessary for transfer, and how to provide resources directly to the patient. Finally, we describe implementation within the state.</p><p><strong>Conclusion: </strong>The MT-IBN was developed by MT-STCC to facilitate the hand-off of lifesaving blood to patients being transported by ground to definitive care in Montana without having to stop at an intermediary facility. This has already led to lives saved in areas that are limited in blood availability due to rurality.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"141-146"},"PeriodicalIF":2.0,"publicationDate":"2024-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10961070/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140206896","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}

{"title":"Prevalence and Associated Factors of Anemia among Newborns at Jimma Medical Center, South-west Ethiopia.","authors":"Gebeyaw Arega Berihun, Girum Tesfaye, Wondimagegn Adissu, Edosa Tadasa, Kidist Adamu, Abinet Tantu Kombe, Lealem Gedefaw","doi":"10.2147/JBM.S443312","DOIUrl":"10.2147/JBM.S443312","url":null,"abstract":"<p><strong>Background: </strong>Newborn anemia is among the most common hematological problems and it can cause asymptomatic or severe to acute life-threatening events. It leads to impairment in brain maturation and development, tissue hypoxia, and stunted growth and then arrested growth if left untreated. The prevalence of anemia among newborns ranges from 23.4-66% in sub-Saharan Africa. But, there is limited information in Ethiopia regarding the prevalence of newborn anemia and its risk factors. Therefore, this study aimed to determine the prevalence of newborn anemia and its associated factors at Jimma Medical Center (JMC), South-west Ethiopia.</p><p><strong>Methods: </strong>A hospital-based cross-sectional study design was implemented from January 14 to February 28, 2021, involving 288 full-term newborns by employing consecutive convenient sampling technique for study participant selection. Socio-demographic data and other associated factors were collected through interviews and a review of medical records by a structured questionnaire. Three mL umbilical cord blood samples from each newborn were collected and analyzed for a complete blood count by an automated hematological analyzer. Data were entered into Epi Data version 3.1 and exported to Statistical Package for Social Science version 20 for analysis. Binary logistic regression were used to identify the predictors of newborn anemia.</p><p><strong>Results: </strong>The overall prevalence of anemia among newborns was 26.4%; of them, 65.8%, 25%, and 9.2% were mild, moderate, and severe anemia types, respectively. Maternal vegetable consumption habit (AOR = 0.26, 95% CI: 0.11, 0.62) and maternal anemia (AOR = 0.34, 95% CI: 0.17, 0.69) were significantly associated with anemia in newborns.</p><p><strong>Conclusion: </strong>In general, newborn anemia in this study was a moderate public health problem. Based on this study, early screening of anemia among newborns may reduce further complications. Prevention of maternal anemia during pregnancy by improving their nutritional status especially vegetable consumption had a positive impact on reducing anemia among newborns.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"129-140"},"PeriodicalIF":2.0,"publicationDate":"2024-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10950678/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140174852","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}

{"title":"Case Study: Rosai-Dorfman Disease and Its Multifaceted Aspects.","authors":"Daniela Oliveira Werneck Rodrigues, Roberta Wolp Diniz, Leonardo Cunha Dentz, Monica de Albuquerque Costa, Roberto Heleno Lopes, Lucas Fernandes Suassuna, Jane Rocha Duarte Cintra, Christian Domenge","doi":"10.2147/JBM.S436720","DOIUrl":"10.2147/JBM.S436720","url":null,"abstract":"<p><p>Rosai-Dorfman Disease (RDD) is a rare non-Langerhans histiocytosis, usually self-limited and presenting with massive, painless, bilateral cervical lymphadenopathy, with or without constitutional symptoms. Extranodal disease is frequently present, and may happen in the absence of lymph node involvement, symptomatology and differential diagnosis will depend on the site affected and fatal cases may occur. The authors present two cases of Rosai-Dorfman disease (RDD), diagnosed through immunohistochemistry, with different progressions, one with complete remission and one culminating in death, highlighting the variety of presentations and the diagnostic difficulty. RDD is a rare condition with clinical presentations similar to several diseases, and should be considered in the differential diagnosis of lymphadenopathy with extranodal lesions.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"123-128"},"PeriodicalIF":2.0,"publicationDate":"2024-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10941986/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140143461","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}

{"title":"A Post-Authorization Safety Surveillance Study to Report Clinical Experience with Purified Factor IX Concentrate in Pediatric Patients with Hemophilia B.","authors":"Zoran Igrutinović, Hélène Louise Hooimeijer, Karim Kentouche, Jaco Botha, Peter L Turecek, Marta Kokot-Kierepa, Hanna T Gazda","doi":"10.2147/JBM.S425617","DOIUrl":"10.2147/JBM.S425617","url":null,"abstract":"<p><strong>Introduction: </strong>Purified factor IX (FIX) concentrate (IMMUNINE^®, Takeda Manufacturing Austria AG, Vienna, Austria) is indicated for the treatment and prophylaxis of bleeding episodes in patients with congenital hemophilia B. Data on the use of purified FIX concentrate in patients ≤6 years old with congenital hemophilia B are limited.</p><p><strong>Aim: </strong>Document real-world clinical experience with purified FIX concentrate in routine practice for pediatric patients with hemophilia B.</p><p><strong>Methods: </strong>This prospective post-authorization safety surveillance study enrolled patients ≤6 years old with moderate or severe hemophilia B (baseline FIX ≤5%) who were prescribed purified FIX concentrate, as determined by the treating physician. The planned observation period for each patient was either 12 months or ≥50 exposure days, whichever occurred first. The primary endpoints were the occurrence of treatment-related adverse events (AEs) and serious AEs (SAEs), and inhibitor development.</p><p><strong>Results: </strong>Thirteen male patients (mean ± standard deviation age, 3.80 ± 1.76 years) enrolled and received ≥1 treatment with purified FIX concentrate. Thirty-two AEs were reported in 6 patients; 4 were SAEs. No AEs were considered related to purified FIX concentrate. No patients developed inhibitory antibodies. Inhibitor testing was not conducted in 2 patients. Eighteen bleeding episodes were treated with purified FIX concentrate in 6 patients. Hemostatic efficacy was rated as either \"excellent\" or \"good\" in all patients with an available rating.</p><p><strong>Conclusion: </strong>No treatment-related AEs were reported, and purified FIX concentrate was shown to be effective in treating and preventing bleeding episodes in pediatric patients ≤6 years old with hemophilia B.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"15 ","pages":"113-122"},"PeriodicalIF":2.0,"publicationDate":"2024-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10933513/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140119551","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}