Journal of Blood Medicine最新文献

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Erythrocyte Alloimmunization and Transfusion Strategies in Sickle Cell Disease: A Single-Center Analysis. 镰状细胞病的红细胞异体免疫和输血策略:单中心分析。
IF 2.7
Journal of Blood Medicine Pub Date : 2025-10-03 eCollection Date: 2025-01-01 DOI: 10.2147/JBM.S548152
Abdulaziz Yusuf, Abrar Ahmad, Hesham A El-Beshbishy, Heba Badie Gong, Chahed Walid Chahdah, Tahani Bakhsh
{"title":"Erythrocyte Alloimmunization and Transfusion Strategies in Sickle Cell Disease: A Single-Center Analysis.","authors":"Abdulaziz Yusuf, Abrar Ahmad, Hesham A El-Beshbishy, Heba Badie Gong, Chahed Walid Chahdah, Tahani Bakhsh","doi":"10.2147/JBM.S548152","DOIUrl":"https://doi.org/10.2147/JBM.S548152","url":null,"abstract":"<p><strong>Aim: </strong>Alloimmunization (the production of antibodies against foreign red blood cell (RBC) antigens) is a significant complication in patients with sickle cell disease (SCD) who require chronic transfusion. This retrospective study examined the distribution of ABO and Rh phenotypes in SCD patients at Dr. Soliman Fakeeh Hospital in Jeddah (DSFH-J) and their implications for alloimmunization risk. The high immunogenicity of the K antigen in the Kell system, second only to that of the D antigen in the Rh system, makes it a frequent target.</p><p><strong>Results: </strong>Among 241 patients with SCD, the most common blood group was O (58.5%), followed by A (26.97%), B (12.03%), and AB (2.9%). The majority of patients (93.36%) were Rh-positive (D antigen-present). Among Rh antigens, the e antigen was the most prevalent (97.51%), while C antigen and c antigen were detected in 68.04% and 75.52% of patients, respectively. Within the Kell system, K was found in 8.29% of the study population. The most common antibodies detected were anti-E (20%) and anti-C (15%), indicating Rh incompatibilities to be a major concern. Kell system antibodies (anti-K) accounted for 12.5% of cases, and unidentified alloantibodies represented 17.5%. Although antibodies from other blood group systems (such as Kidd, Duffy, Lutheran, and MNS) were detected at low frequencies, their presence and known clinical significance in causing transfusion reactions underscore the need for extended RBC phenotyping to include these systems.</p><p><strong>Conclusion: </strong>The observed distribution of Rh phenotypes and the presence of alloantibodies beyond the prevalent ones highlights the need for extended RBC phenotyping to include other blood group systems, such as Kidd and Duffy. Establishing a national blood donor registry with comprehensive RBC antigen data is a crucial step toward ensuring safer transfusions. Standardizing blood screening protocols across hospitals in Saudi Arabia and introducing routine extended RBC typing before transfusions would minimize alloimmunization risks and improve the overall patient safety.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"16 ","pages":"445-455"},"PeriodicalIF":2.7,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12502970/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145251166","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
Safety of Recombinant von Willebrand Factor in the Treatment of von Willebrand Disease: Real-World Data from an EU Post-Authorization Safety Study. 重组血管性血友病因子治疗血管性血友病的安全性:来自欧盟授权后安全性研究的真实世界数据
IF 2.7
Journal of Blood Medicine Pub Date : 2025-10-02 eCollection Date: 2025-01-01 DOI: 10.2147/JBM.S512634
Susan M Sinclair, Yi Ba, Kayode Badejo
{"title":"Safety of Recombinant von Willebrand Factor in the Treatment of von Willebrand Disease: Real-World Data from an EU Post-Authorization Safety Study.","authors":"Susan M Sinclair, Yi Ba, Kayode Badejo","doi":"10.2147/JBM.S512634","DOIUrl":"https://doi.org/10.2147/JBM.S512634","url":null,"abstract":"<p><strong>Introduction: </strong>In Europe, recombinant von Willebrand factor (rVWF) is approved for the prevention and treatment of hemorrhage or surgical bleeding in adults with von Willebrand disease (VWD) for whom desmopressin alone is ineffective or contraindicated. Real-world data on rVWF safety are limited.</p><p><strong>Aim: </strong>To assess the safety of rVWF in real-world European clinical practice.</p><p><strong>Methods: </strong>EU post-authorization safety study (NCT05265078, EUPAS45617) was a multicenter, retrospective, non-interventional study conducted in adults with VWD who received rVWF at 1 of 30 participating sites in Europe (January 2019-March 2023). Data were collected retrospectively for ≥7 days and ≤6 months after the first rVWF infusion, and similarly, after each subsequent rVWF course. Primary outcomes were the risk of hypersensitivity reactions, thromboembolic events, and VWF/factor VIII (FVIII) inhibitor formation when used for hemorrhage treatment or prevention/treatment of surgical bleeding; and the association of thromboembolic events with concurrent use of FVIII for hemorrhage treatment or prevention/treatment of surgical bleeding.</p><p><strong>Results: </strong>In the primary analysis, 87 patients received 203 rVWF treatment courses. In total, 2 hypersensitivity-related AEs of mild severity occurred in 1 patient who received rVWF (0.00068 events per person-day at risk), and 1 thromboembolic AE of moderate severity (venous thrombosis) was reported in 1 patient (0.00127 events per person-week at risk). There were no reports of VWF or FVIII inhibitor formation. The association between thromboembolic events and the concurrent use of rVWF and FVIII could not be assessed because no patient received rVWF in conjunction with FVIII.</p><p><strong>Conclusion: </strong>In this EU post-authorization safety study, the risks of hypersensitivity reactions and thromboembolic events with rVWF were low and there were no reports of VWF or FVIII inhibitor formation. Overall, no new safety signals were identified in this European real-world study when rVWF was used for the prevention or treatment of hemorrhage or surgical bleeding in VWD.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"16 ","pages":"457-467"},"PeriodicalIF":2.7,"publicationDate":"2025-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12499246/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145244550","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
Non-Traumatic Fat Embolism Syndrome in Two HbSS Sickle Cell Disease Patients: A Case Series and Review. 2例HbSS镰状细胞病患者的非创伤性脂肪栓塞综合征:病例系列和回顾
IF 2.7
Journal of Blood Medicine Pub Date : 2025-10-01 eCollection Date: 2025-01-01 DOI: 10.2147/JBM.S532980
Mohammed Amouri Alzayer, Salah Abohelaika, Mousa Alhaddad, Mazen Al Zayer, Ahmed J Alali, Abdullah Mohammad Al Abbas, Nabeel J Al Eid, Fatimah A Al Saeed, Hussain H Al Saeed
{"title":"Non-Traumatic Fat Embolism Syndrome in Two HbSS Sickle Cell Disease Patients: A Case Series and Review.","authors":"Mohammed Amouri Alzayer, Salah Abohelaika, Mousa Alhaddad, Mazen Al Zayer, Ahmed J Alali, Abdullah Mohammad Al Abbas, Nabeel J Al Eid, Fatimah A Al Saeed, Hussain H Al Saeed","doi":"10.2147/JBM.S532980","DOIUrl":"10.2147/JBM.S532980","url":null,"abstract":"<p><p>Non-traumatic fat embolism syndrome (FES) in sickle cell disease (SCD) is associated with high morbidity and mortality. This severe complication arises from bone marrow necrosis, which subsequently can lead to fat embolism syndrome and multi-organ failure. A key challenge in diagnosing and managing this syndrome lies in the absence of established diagnostic criteria and the variability of its clinical presentation. Instant red cell exchange transfusion could be lifesaving once the syndrome is suspected, and the use of therapeutic plasma exchange, as suggested in the literature, can improve outcomes. Here, we report two cases of SCD with FES that were managed successfully with blood transfusion and plasma exchange. Both of them rapidly deteriorated and required intensive care unit admission, where they received red cell exchange followed by plasma exchange. They both made a good recovery and were discharged from the hospital within two weeks.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"16 ","pages":"437-443"},"PeriodicalIF":2.7,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12497365/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145238623","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
Iron Overload and Its Impact on Liver Function and Lipid Profiles in Transfusion-Dependent β-Thalassemia Patients in Sana'a City. 萨那市输血依赖性β-地中海贫血患者铁超载及其对肝功能和脂质谱的影响
IF 2.7
Journal of Blood Medicine Pub Date : 2025-09-30 eCollection Date: 2025-01-01 DOI: 10.2147/JBM.S538996
Ali Al-Shami, Mokhtar Alzomor
{"title":"Iron Overload and Its Impact on Liver Function and Lipid Profiles in Transfusion-Dependent β-Thalassemia Patients in Sana'a City.","authors":"Ali Al-Shami, Mokhtar Alzomor","doi":"10.2147/JBM.S538996","DOIUrl":"10.2147/JBM.S538996","url":null,"abstract":"<p><strong>Background: </strong>β-thalassemia major (βTM) is a severe genetic blood disorder that necessitates regular blood transfusions, which often lead to iron overload and associated complications, including liver dysfunction and dyslipidemia.</p><p><strong>Objective: </strong>To investigate the relationship between iron overload, liver function abnormalities, and lipid profile disturbances in transfusion-dependent β-thalassemia patients in Sana'a, Yemen.</p><p><strong>Methods: </strong>A cross-sectional study was conducted among 53 participants recruited from the Yemeni Association for Thalassemia Patients and Genetic Blood Disorders in Sana'a City. Participants were divided into four groups: healthy controls, β-thalassemia patients receiving regular blood transfusions with or without iron chelation therapy (ICT), and nontrans fused thalassemia patients. Clinical data were collected using structured questionnaires, and Biochemical and haematological parameters, including serum ferritin, serum iron, GPT (ALT), bilirubin, triglycerides (TG), HDL, LDL, cholesterol, hemoglobin (Hb), and white blood cell (WBC) counts, were measured. The data were analyzed using SPSS Version 20. Normality was assessed with the ShapiroWilk test. Parametric tests, including independent sample ttests and ANOVA, were used to compare continuous variables. Categorical data were analyzed with the chi-square test. A Bonferroni correction was applied to adjust for multiple comparisons.</p><p><strong>Results: </strong>Serum ferritin levels above 1,000 ng/mL were considered elevated, and iron levels were significantly higher in transfusion-dependent patients, particularly those receiving blood for >5 years or >250 mL per transfusion. Group II (patients receiving blood transfusions with ICT) and Group III (patients receiving blood transfusions without ICT) showed significantly elevated ferritin and serum iron levels compared to Group IV (non-transfused patients). Patients with high ferritin levels also exhibited significantly elevated GPT and direct bilirubin, indicating liver damage, with the highest levels observed in Group II and Group III. Furthermore, these patients had higher triglycerides and lower HDL, LDL, and total cholesterol, consistent with dyslipidemia. Haematological parameters showed reduced hemoglobin and RBCs, and increased WBCs among patients with iron overload.</p><p><strong>Conclusion: </strong>Iron overload is strongly associated with liver dysfunction and dyslipidemia in transfusion-dependent β-thalassemia patients. The findings suggest that iron chelation therapy helps reduce the impact of iron overload on liver function and lipid metabolism. Routine monitoring of ferritin, liver enzymes, and lipid profiles is essential for managing these complications. Effective iron chelation therapy is critical, and improving access to ICT and establishing better follow-up strategies are needed to mitigate the long-term consequences of iron overload.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"16 ","pages":"425-436"},"PeriodicalIF":2.7,"publicationDate":"2025-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12495971/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145232648","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
Journeying Towards Excellence in the Care of Patients with Haemophilia and Other Inherited Bleeding Disorders From a Developing World: Insights From Tanzania. 来自发展中国家的血友病和其他遗传性出血性疾病患者的卓越护理之旅:来自坦桑尼亚的见解。
IF 2.7
Journal of Blood Medicine Pub Date : 2025-09-10 eCollection Date: 2025-01-01 DOI: 10.2147/JBM.S537383
Stella Samson Rwezaula, Samson D Mtoba, Rashid A Gosse, Rebecca W Mwakichako, Abdallah Raphael Makalla, Martha Loiseyeki Bruckman, Luhongedzo Gerson Matandala, Linda Alphey Shao, Hedwiga Francis Swai, Michelle Sholzberg, Jerome Teitel, Amos Rodger Mwakigonja, Oluyemi Akinloye
{"title":"Journeying Towards Excellence in the Care of Patients with Haemophilia and Other Inherited Bleeding Disorders From a Developing World: Insights From Tanzania.","authors":"Stella Samson Rwezaula, Samson D Mtoba, Rashid A Gosse, Rebecca W Mwakichako, Abdallah Raphael Makalla, Martha Loiseyeki Bruckman, Luhongedzo Gerson Matandala, Linda Alphey Shao, Hedwiga Francis Swai, Michelle Sholzberg, Jerome Teitel, Amos Rodger Mwakigonja, Oluyemi Akinloye","doi":"10.2147/JBM.S537383","DOIUrl":"10.2147/JBM.S537383","url":null,"abstract":"<p><strong>Introduction: </strong>Haemophilia, an X-linked recessive bleeding disorder caused by mutations in the <i>F8</i> and <i>F9</i> genes, remains profoundly underdiagnosed in resource-limited settings. In Tanzania, it is estimated that fewer than 10% of individuals with haemophilia are diagnosed. Centralized services, limited diagnostic capacity, and underutilization of available therapies pose major barriers to timely and effective care. This review reports on the activities undertaken to establish and expand haemophilia care in Tanzania, charting the journey toward excellence.</p><p><strong>Methods: </strong>This is a comprehensive review of national program reports, registry data from the Haemophilia Society of Tanzania, and interviews with key personnel directly involved in the development of haemophilia services between 2021 and April 2025.</p><p><strong>Results: </strong>Between 2021 and 2025, Tanzania made significant strides in strengthening haemophilia care. A multidisciplinary workforce was trained both locally and internationally in specialized diagnosis, treatment, and management of haemophilia and other inherited bleeding disorders. Fourteen haemophilia clinics were established, including a national comprehensive clinic at Muhimbili National Hospital and satellite clinics across zonal and regional hospitals, providing screening, diagnosis, and comprehensive care. As of 17th April 2025, a total of 473 patients had been registered and were receiving structured care through this network.</p><p><strong>Conclusion: </strong>The integration of specialized haematological training, stakeholder engagement, decentralization of services through 14 clinics, and the development of a national registry and clinical guidelines have markedly enhanced Tanzania's capacity to diagnose and manage haemophilia and other inherited bleeding disorders. Moving forward, it is imperative to expand regional coverage, intensify community-based awareness and screening initiatives, strengthen capacity building and research efforts, and establish a specialized centre of excellence dedicated to advancing haemophilia care, professional training, research, and innovation.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"16 ","pages":"413-423"},"PeriodicalIF":2.7,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12433640/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069669","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
Multifaceted Clinical Spectrum of Vitamin B12 Deficiency - a Case Report and Literature Review. 维生素B12缺乏症的多方面临床谱-一个病例报告和文献综述。
IF 2.7
Journal of Blood Medicine Pub Date : 2025-08-25 eCollection Date: 2025-01-01 DOI: 10.2147/JBM.S524466
Ewa Pustelnik, Katarzyna Pikora, Magdalena Zofia Hartman, Marta Kurzeja, Joanna Czuwara, Paweł Łaguna
{"title":"Multifaceted Clinical Spectrum of Vitamin B12 Deficiency - a Case Report and Literature Review.","authors":"Ewa Pustelnik, Katarzyna Pikora, Magdalena Zofia Hartman, Marta Kurzeja, Joanna Czuwara, Paweł Łaguna","doi":"10.2147/JBM.S524466","DOIUrl":"10.2147/JBM.S524466","url":null,"abstract":"<p><strong>Background: </strong>Vitamin B12 (cobalamin) deficiency is a well-known cause of hematologic and neurological disorders; however, its presentation can be highly variable, leading to diagnostic challenges. The etiology is diverse: while the most common cause is dietary insufficiency, other potential causes include malabsorption syndromes, autoimmune gastritis, gastrointestinal disorders, chronic infections, and genetic defects. Clinical presentation varies significantly, ranging from clinically silent macrocytosis to life-threatening anemia or pancytopenia. Neurological and psychiatric manifestations may include vision and gait impairment, depression, and cognitive dysfunction. Given this complexity, vitamin B12 deficiency can mimic other conditions, often leading to a delay in diagnosis.</p><p><strong>Case presentation: </strong>A 15-year-old male was admitted in critical condition with severe anemia, thrombocytopenia, jaundice, progressive weight loss, fatigue, gait disturbances, and vision impairment. Initially, Evan's syndrome was suspected, but further laboratory investigations, including a peripheral blood smear and elevated mean corpuscular volume (MCV), led to the diagnosis of profound vitamin B12 deficiency. Additional workup revealed chronic atrophic gastritis as the underlying cause. The patient was treated with vitamin B12 injections, leading to significant hematologic and neurological improvement, weight gain, and resolution of psychiatric symptoms. However, optic nerve atrophy was detected as a late complication.</p><p><strong>Conclusion: </strong>This case emphasizes the need to consider vitamin B12 deficiency in pediatric patients with unexplained hematologic, neurological, and psychiatric symptoms, particularly when associated with chronic atrophic gastritis. Early recognition and intervention are crucial to preventing irreversible complications, such as optic neuropathy. Given the multidisciplinary nature of its presentation, this case serves as an important reminder for pediatricians, hematologists, gastroenterologists, and neurologists to maintain a high index of suspicion for vitamin B12 deficiency in complex clinical scenarios.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"16 ","pages":"391-411"},"PeriodicalIF":2.7,"publicationDate":"2025-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12396523/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144955421","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
Greater Efficacy of Cidofovir Than of Foscarnet for the Treatment of Acyclovir-Resistant Herpes Simplex Virus Infection After Allogeneic Hematopoietic Stem Cell Transplantation. 西多福韦治疗同种异体造血干细胞移植后无环韦耐药单纯疱疹病毒感染的疗效优于磷膦酸钠。
IF 2.7
Journal of Blood Medicine Pub Date : 2025-08-22 eCollection Date: 2025-01-01 DOI: 10.2147/JBM.S527721
Jun Kong, Bin Chen, Yilei Ma, Lin An, Yao Lu, Nan Chen, Xiaodong Mo
{"title":"Greater Efficacy of Cidofovir Than of Foscarnet for the Treatment of Acyclovir-Resistant Herpes Simplex Virus Infection After Allogeneic Hematopoietic Stem Cell Transplantation.","authors":"Jun Kong, Bin Chen, Yilei Ma, Lin An, Yao Lu, Nan Chen, Xiaodong Mo","doi":"10.2147/JBM.S527721","DOIUrl":"10.2147/JBM.S527721","url":null,"abstract":"<p><p>Herpes simplex virus (HSV) infection is a common problem in patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Severe HSV infections can cause pneumonia, encephalitis, meningitis and other lesions, thus requiring caution. Acyclovir is the drug of choice for the prevention of HSV infection. Acyclovir resistance caused refractory HSV infection which is a severe complication after allo-HSCT. Cidofovir may be effective for acyclovir-resistant patients; however, the efficacy of systemic cidofovir administration for acyclovir-resistant patients after allo-HSCT remains unknown. We describe a 67-year-old female patient with multiple ulcers and erosions of the periocular, perioral, and oral mucosa after haploidentical allo-HSCT. Poor results were observed after the treatment of acyclovir and foscarnet. In particular, an adverse effect of low potassium and renal damage has been observed with foscarnet. Genetic testing suggested an acyclovir-resistant herpes simplex virus type 1 (HSV1) infection with T287M mutation of gene <i>UL23</i>. The patient's infection was cured after intravenous treatment with cidofovir. Systemic administration of cidofovir may be useful for patients with acyclovir-resistant HSV1 infection after haploidentical allo-HSCT.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"16 ","pages":"385-390"},"PeriodicalIF":2.7,"publicationDate":"2025-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12379988/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144955382","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
First Report of Hematopoietic Stem Cell Transplantation for Children Diagnosed with Wiskott-Aldrich Syndrome in Vietnam. 越南诊断为Wiskott-Aldrich综合征的儿童进行造血干细胞移植的首例报道。
IF 2.7
Journal of Blood Medicine Pub Date : 2025-08-12 eCollection Date: 2025-01-01 DOI: 10.2147/JBM.S528827
Le Nguyen-Ngoc-Quynh, Binh Nguyen-Thanh, Anh Thi Van Nguyen, Ha Dang-Thi, Anh Ha-Phuong, Quynh Bui-Thi-Thuy, Huong Le-Thi-Minh, Ngoc Nguyen-Bao, Minh Le-Duc, Phuong Ha-Thi, Chi Le-Quynh, Mai Nguyen-Thi-Phuong, Tien Ngo-Manh, Lan Bui-Ngoc, Toan Duong-Khanh, Tung Cao-Viet, Dien Tran-Minh
{"title":"First Report of Hematopoietic Stem Cell Transplantation for Children Diagnosed with Wiskott-Aldrich Syndrome in Vietnam.","authors":"Le Nguyen-Ngoc-Quynh, Binh Nguyen-Thanh, Anh Thi Van Nguyen, Ha Dang-Thi, Anh Ha-Phuong, Quynh Bui-Thi-Thuy, Huong Le-Thi-Minh, Ngoc Nguyen-Bao, Minh Le-Duc, Phuong Ha-Thi, Chi Le-Quynh, Mai Nguyen-Thi-Phuong, Tien Ngo-Manh, Lan Bui-Ngoc, Toan Duong-Khanh, Tung Cao-Viet, Dien Tran-Minh","doi":"10.2147/JBM.S528827","DOIUrl":"10.2147/JBM.S528827","url":null,"abstract":"<p><strong>Background: </strong>Awareness of inborn error immunity, such as Wiskott-Aldrich syndrome (WAS), is still lacking in Vietnam. The shortage of clinical immunologists and transplantation teams lead to poor prognosis for patients.</p><p><strong>Objective: </strong>Describe initial data about hematopoietic stem cell transplantation (HSCT) for WAS.</p><p><strong>Methods: </strong>Retrospective analyzing 15 procedures on 13 patients at the Vietnam National Children's Hospital from 2020 to 2024.</p><p><strong>Results: </strong>The median age at HSCT was 34 months (range: 17-86). Of the patients, 73.3% received myeloablative conditioning based on busulfan, while 26.7% underwent reduced-intensity conditioning. Donor sources included matched sibling donors (MSD, 20.0%), unrelated cord blood (UCB, 33.3%), phenotypically identical family donor (MFD, 6.7%), and mismatched related donors (MMRD, 40.0%). The median stem cell dose was 4.9 × 10^6/kg of the recipient's body weight (range: 0.33 to 10.4 × 10^6/kg). Neutrophil and platelet engraftment occurred at a median of 14 days (range: 10-19) and 48 days (range: 14-143), respectively. By day +30, 73.3% of patients achieved full donor chimerism. One patient experienced graft failure, and another faced graft rejection two months post-transplant, both of whom underwent a second transplant with different donors. The overall survival rate was 92.3% with a median follow-up of 23 months (range: 6-53), with one patient died from chronic graft-versus-host disease (cGVHD). All surviving patients achieved normalization of platelet counts.</p><p><strong>Conclusion: </strong>HSCT offers significant benefits to WAS patients, achieving an excellent overall survival rate.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"16 ","pages":"373-383"},"PeriodicalIF":2.7,"publicationDate":"2025-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12357556/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144873354","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
An Increased Plasma D-Dimer Levels During Different Stages of Normal and Complicated Pregnancies Among Women in Bisha, Saudi Arabia. 沙特阿拉伯比沙妇女正常妊娠和复杂妊娠不同阶段血浆d -二聚体水平升高
IF 2.7
Journal of Blood Medicine Pub Date : 2025-08-06 eCollection Date: 2025-01-01 DOI: 10.2147/JBM.S523353
Tareg M Belali
{"title":"An Increased Plasma D-Dimer Levels During Different Stages of Normal and Complicated Pregnancies Among Women in Bisha, Saudi Arabia.","authors":"Tareg M Belali","doi":"10.2147/JBM.S523353","DOIUrl":"10.2147/JBM.S523353","url":null,"abstract":"<p><strong>Introduction: </strong>Hypercoagulability is one of the most reported state among Pregnant women. During pregnancy, the concentrations of D-dimer increase in a trimester-dependent- manner. The presence of gestational diabetes (GD) and gestational hypertension (GH) lead to further elevation in D-dimer levels. Pregnant women are at an increased risk of developing Venous thromboembolism (VTE), which can be fatal for both the mother and fetus. Elevated D-dimer levels during pregnancy could result in misdiagnosing other thrombotic diseases, emphasizing the need for further confirmatory testing for VTE. It is crucial to observe the plasma D-dimer concentrations among pregnant women at maternity and children's hospitals as it plays a role in guiding the anticoagulant treatments and minimizing the incidence of VTE among pregnant women.</p><p><strong>Aim: </strong>The main objective of the current study was to detect the changes in the plasma D-dimer concentrations in healthy and complicated pregnancies across different gestational trimesters among women at the Maternity and Children's Hospital, Bisha, Saudi Arabia.</p><p><strong>Methods: </strong>A cross-sectional study was conducted from March 2022 to March 2023, involving the analysis of plasma samples collected from 230 pregnant and non-pregnant women. Three samples were collected from each subject in each trimester. D-dimer measurement was conducted using an ACL Elite Pro Automated analyzer.</p><p><strong>Results: </strong>The findings of this study show that D-dimer levels increased progressively throughout the pregnancy trimesters across all study groups. The increase was more noticeable among women with gestational diabetes (278.39 ± 29.808 ng/mL) and gestational hypertension (320.63 ± 12.157 ng/mL), suggesting that these complications influence D-dimer levels more significantly than healthy and multiple pregnancies. Notably, a positive correlation was found between age and D-dimer levels across all groups (p < 0.05), with the highest mean levels in the 41-48 age group.</p><p><strong>Conclusion: </strong>D-dimer levels rise progressively throughout pregnancy and are significantly elevated in women with gestational diabetes and hypertension. These findings underscore the importance of interpreting D-dimer values in the context of gestational age and pregnancy complications. Additionally, maternal age influences D-dimer concentrations, emphasizing the need to interpret results within the context of age-related physiological changes during pregnancy.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"16 ","pages":"349-358"},"PeriodicalIF":2.7,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12336379/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144821535","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
Exploring the Relationship Between Personalization of Care and Participation in Sport Activities Among People with Severe Hemophilia A Across Europe: Post Hoc Analysis of the CHESS II Study. 探索欧洲严重血友病A患者个性化护理与参与体育活动之间的关系:国际象棋II研究的事后分析
IF 2.7
Journal of Blood Medicine Pub Date : 2025-08-06 eCollection Date: 2025-01-01 DOI: 10.2147/JBM.S521079
Tom Blenkiron, Enrico Ferri Grazzi, Tom Burke, Maureen Watt, Kimberly H Davis
{"title":"Exploring the Relationship Between Personalization of Care and Participation in Sport Activities Among People with Severe Hemophilia A Across Europe: Post Hoc Analysis of the CHESS II Study.","authors":"Tom Blenkiron, Enrico Ferri Grazzi, Tom Burke, Maureen Watt, Kimberly H Davis","doi":"10.2147/JBM.S521079","DOIUrl":"10.2147/JBM.S521079","url":null,"abstract":"<p><strong>Purpose: </strong>To describe the demographic and clinical characteristics of patients with hemophilia A receiving different levels of treatment personalization (TP), and to assess the relationship between TP and sport active time (SAT).</p><p><strong>Patients and methods: </strong>This post hoc analysis of the CHESS II study used data from physician-completed patient record forms and patient self-completion forms for adult males receiving prophylaxis for severe hemophilia A in Europe between November 2018 and October 2020. SAT was assessed using propensity score matching (PSM) across levels of TP, including pharmacokinetic (PK)-guided and non-PK-guided.</p><p><strong>Results: </strong>Of 54 patients, 32 (59.3%) received TP. Of these, 22 (68.8%) and 10 (31.3%) received non-PK-guided and PK-guided treatment, respectively. Median age varied between the TP and no-TP groups (29.5 and 34.0 years, respectively). Median (IQR) annual bleeding incidence was higher with non-PK-guided vs PK-guided TP (4.0 [3.0-8.0] vs 3.5 [2.0-4.0]). Median (IQR) problem joints were similar with non-PK-guided and PK-guided TP (1.0 [0.0-1.0] and 1.0 [0.0-2.0]). Patients in the TP vs no-TP group had higher median (IQR) SAT per month (3.3 [1.8-6.2] vs 1.8 [0.7-5.0] hours). Median (IQR) SAT per month was higher with PK-guided vs non-PK-guided TP (4.0 [3.0-20.0] vs 3.0 [1.3-5.3] hours). After controlling for confounding in the PSM model, SAT remained higher with TP vs no-TP and with PK-guided vs non-PK-guided TP. In both PSM models, P values were <0.05 for the average treatment effect and <0.01 for the average treatment effect on the treated. Sensitivity analyses confirmed the robustness of the PSM.</p><p><strong>Conclusion: </strong>Patients receiving TP vs no-TP had higher median SAT. Of those with TP, those receiving PK-guided vs non-PK-guided TP experienced lower bleeding rates and higher SAT. PK-guided TP may help patients to be more active, potentially gaining the clinical and psychosocial benefits of exercise.</p>","PeriodicalId":15166,"journal":{"name":"Journal of Blood Medicine","volume":"16 ","pages":"359-372"},"PeriodicalIF":2.7,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12336377/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144821536","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
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