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Optical genome mapping identifies hidden structural variation in acute myeloid leukemia: Two case reports. 光学基因组图谱发现急性髓性白血病中隐藏的结构变异:两个病例报告。
Pub Date : 2024-11-08 DOI: 10.1016/j.htct.2024.06.011
Harmanpreet Singh, Nikhil Shri Sahajpal, Vivek Gupta, Jaspreet Farmaha, Ashutosh Vashisht, Ashis K Mondal, Ravindra Kolhe
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引用次数: 0
Development and validation of the self-regulation of blood donation scale for blood donors. 开发和验证献血者献血自律量表。
Pub Date : 2024-11-07 DOI: 10.1016/j.htct.2024.09.2482
Fahimeh Ranjbar Kermani, Sedigheh Amini Kafi-Abad, Mahtab Maghsudlu, Kamran Mousavi Hosseini, Fatemeh Mohammadali, Atefeh MohammadJafari

Background: Because knowledge of blood donor motivation is crucial in guiding recruitment and retention efforts, the present study aimed at developing and validating a new scale as a multidimensional measure of blood donation motivation from the perspective of the self- determination theory.

Methods: This study was conducted in three phases from September 2022 to May 2023. The first phase involved developing a draft scale based on a literature review. In the second phase, face and content validity were performed. The third phase used a cross-sectional design to assess the construct validity and internal consistency of the initial scale following administration to blood donors with a history of at least one previous whole blood donation who visited the largest blood transfusion center in Iran. Of the 420 subjects who were recruited using a mixed sampling method, 343 who fully completed the initial version of the scale were subjected to an construct validity assessment using exploratory factor analysis with Equamax rotation and internal consistency using McDonald's omega coefficient.

Main results: The initial version of the scale consisted of 30 survey items with both a content validity ratio and a content validity index of 0.99. Exploratory factor analysis identified 24 items; grouped in six regulation factors (non-regulation, external regulation, introjected regulation, identified regulation, integrated regulation, and intrinsic regulation). The factors demonstrated adequate internal consistency with ω values ranging from 0.60 to 0.79.

Conclusion: The present study provides psychometric support for the newly developed questionnaire to evaluate donation motivation among blood donors in Iran or in other countries with similar language, and religious and cultural values.

背景:由于了解献血者的献血动机对于指导招募和留住献血者的工作至关重要,本研究旨在从自我决定理论的角度开发和验证一个新的量表,作为衡量献血动机的多维量表:本研究从 2022 年 9 月至 2023 年 5 月分三个阶段进行。第一阶段是在文献综述的基础上编制量表草案。在第二阶段,进行了面效和内容效度验证。第三阶段采用横断面设计,在对访问伊朗最大输血中心的至少有一次全血献血史的献血者施测后,评估初始量表的结构效度和内部一致性。在采用混合抽样法招募的 420 名受试者中,有 343 人完整填写了量表的初始版本,我们采用探索性因子分析和 Equamax 旋转法对其进行了结构效度评估,并采用麦当劳欧米茄系数对其进行了内部一致性评估:量表的初始版本由 30 个调查项目组成,其内容效度比和内容效度指数均为 0.99。探索性因子分析确定了 24 个条目;分为六个调节因子(非调节、外在调节、内隐调节、识别调节、综合调节和内在调节)。这些因子具有足够的内部一致性,ω 值在 0.60 至 0.79 之间:本研究为新开发的问卷提供了心理测量学支持,该问卷可用于评估伊朗或其他具有类似语言、宗教和文化价值观的国家献血者的献血动机。
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引用次数: 0
Assessing treatment response in thrombotic thrombocytopenic purpura: Beyond the platelet count. 评估血栓性血小板减少性紫癜的治疗反应:超越血小板计数。
Pub Date : 2024-11-07 DOI: 10.1016/j.htct.2024.06.012
Cilomar Martins de Oliveira Filho, Ibidunni Bode-Sojobi, Barbara D Lam, Stephanie Conrad, Jonathan Berry, Brian J Carney
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引用次数: 0
Fluvastatin suppresses hemin-induced cell death, reactive oxygen species generation, and elevated labile iron pool. 氟伐他汀可抑制血红素诱导的细胞死亡、活性氧生成和可溶性铁池的升高。
Pub Date : 2024-11-07 DOI: 10.1016/j.htct.2024.09.2480
Shion Imoto, Katsuyasu Saigo, Mari Kono, Ayako Ohbuchi, Tohru Sawamura, Yuji Mizokoshi, Takashi Suzuki

Background: In transfusion-related iron overload, macrophage/reticuloendothelial cells are the first site of haem-derived iron accumulation. The prevention of haem-induced cytotoxicity in macrophages may represent a target for iron overload treatment. Deferasirox, an oral iron chelator, has been used to treat transfusion-related iron overload however, low adherence to the therapy is an issue. Statins, which are widely used for the prevention of atherosclerotic cardiovascular diseases, also have anti-oxidative and anti-inflammatory effects independent of their lipid lowering ones. Whether statins can suppress hemin-induced cytotoxicity and enhance the cytoprotective effects of deferasirox are important considerations to improve transfusion-related iron overload treatment. This study also evaluated the effects of eltrombopag, a thrombopoietin receptor agonist.

Materials and methods: Human monocytic THP-1 cells were pretreated with statins, deferasirox, and/or eltrombopag, followed by treatment with hemin. Cell viability, reactive oxygen species generation, and the intracellular labile iron pool were measured using flow cytometry.

Results: Fluvastatin and another four statins suppressed hemin-induced cell death, reactive oxygen species generation, and increases in the labile iron pool. Moreover, fluvastatin enhanced the suppressive effect of deferasirox on hemin-induced cell death. The effects of eltrombopag were similar to those of the statins.

Conclusion: The safety of statins is well established. When used in combination with fluvastatin or other statins, the suppressive effects of deferasirox on hemin-induced cytotoxicity in THP-1 cells were amplified. Further research is necessary to see whether statins will act in the same way in vivo or in human primary monocytes/macrophages.

背景:在输血相关的铁超载中,巨噬细胞/网状内皮细胞是血源性铁积聚的第一个部位。预防巨噬细胞中由血液诱导的细胞毒性可能是治疗铁超载的一个目标。地拉罗司是一种口服铁螯合剂,已被用于治疗输血相关的铁超载,但治疗依从性低是一个问题。他汀类药物被广泛用于预防动脉粥样硬化性心血管疾病,除降脂作用外,还具有抗氧化和抗炎作用。他汀类药物能否抑制血清素诱导的细胞毒性并增强地拉羅司的细胞保护作用,是改善输血相关铁过载治疗的重要考虑因素。材料和方法:用他汀类药物、地拉罗司和/或艾曲波帕格预处理人单核细胞 THP-1 细胞,然后用海明处理。使用流式细胞仪测量细胞活力、活性氧生成和细胞内可变铁池:结果:氟伐他汀和另外四种他汀类药物抑制了海明诱导的细胞死亡、活性氧生成和可溶性铁池的增加。此外,氟伐他汀还增强了去铁胺对海明诱导的细胞死亡的抑制作用。艾曲波帕的效果与他汀类药物相似:结论:他汀类药物的安全性已得到公认。当与氟伐他汀或其他他汀类药物联合使用时,地拉羅司对 THP-1 细胞中海明诱导的细胞毒性的抑制作用被放大。他汀类药物是否会在体内或人类原代单核细胞/巨噬细胞中以同样的方式发挥作用,还需要进一步研究。
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引用次数: 0
The effect of modulating platelet reactive oxygen species by the addition of antioxidants to prevent clearance of cold-stored platelets. 通过添加抗氧化剂调节血小板活性氧以防止冷藏血小板清除的效果。
Pub Date : 2024-10-18 DOI: 10.1016/j.htct.2024.09.2479
Rufeng Xie, Yiming Yang, Xueyu Jiang, Li Gao, Juan Sun, Jie Yang

Background: It is known that the rapid clearance of cold-stored platelets is attributed to various storage lesions, including an abnormal increase in reactive oxygen species when platelets are exposed to cold temperatures. As an antioxidant, N-acetylcysteine exhibits some significant effects on scavenging various reactive oxygen species and inhibiting cell damage and apoptosis.

Aims: This study aimed to investigate the effects of N-acetylcysteine on reducing reactive oxygen species production and protecting cold-stored platelets from phagocytosis and clearance, and to determine the optimal concentration of N-acetylcysteine.

Methods: Platelet concentrates were divided into three groups: room-temperature-stored platelets, cold-stored platelets, and cold-stored platelets with the addition of different concentrations of N-acetylcysteine. After five days of storage, reactive oxygen species production, lipid peroxidation levels, activation marker expressions, GPIb/ɑ desialylation with exposure of glycan residues and other quality parameters of platelets were measured and compared between the groups. Phagocytosis of platelets was detected by phorbol 12-myristate 13-acetate-activated THP-1 or Hep G2 cells. Moreover, the recovery of infused platelets was measured in severe combined immunodeficient mice at different timepoints.

Results: After 5 days of storage, cytoplasmic reactive oxygen species significantly increased in chilled compared to non-chilled platelets; they were notably reduced with the addition of N-acetylcysteine, particularly at a concentration of 5 mM. Compared with chilled platelets, the P-selectin and phosphatidylserine expressions, as well as exposure of GPIb/ɑ glycan residues, were significantly reduced with 5 mM of N-acetylcysteine. Phagocytosis of platelets by THP-1 or Hep G2 cells was significantly lower in 5 mM of N-acetylcysteine compared to cold-stored platelets without N-acetylcysteine.

Conclusions: This study demonstrated correlations between reactive oxygen species production and their pro-oxidant effects on platelet clearance after cold storage. The addition of N-acetylcysteine at an appropriate concentration do not only protects chilled platelets from storage lesions caused by reactive oxygen species overproduction but also prevents platelet phagocytosis in vitro and clearance in vivo, thereby extending circulating time.

背景:众所周知,冷藏血小板的快速清除是由于各种贮存病变造成的,包括血小板暴露于低温时活性氧的异常增加。目的:本研究旨在探讨 N-乙酰半胱氨酸对减少活性氧生成、保护冷藏血小板不被吞噬和清除的作用,并确定 N-乙酰半胱氨酸的最佳浓度:将浓缩血小板分为三组:室温储存血小板、低温储存血小板和添加不同浓度 N-乙酰半胱氨酸的低温储存血小板。储存五天后,测量并比较了各组血小板的活性氧生成、脂质过氧化水平、活化标志物表达、GPIb/ɑ去酰化与糖残基暴露以及其他质量参数。通过磷酸-12-肉豆蔻酸-13-乙酸酯激活的 THP-1 或 Hep G2 细胞检测血小板的吞噬作用。此外,还在不同时间点测定了严重合并免疫缺陷小鼠输注血小板的恢复情况:结果:储存 5 天后,冷藏血小板与非冷藏血小板相比,细胞质中的活性氧明显增加;加入 N-乙酰半胱氨酸后,活性氧明显减少,尤其是在浓度为 5 mM 时。与冷藏血小板相比,P-选择素和磷脂酰丝氨酸的表达以及 GPIb/ɑ 聚糖残基的暴露在 5 mM N-乙酰半胱氨酸的作用下明显减少。与不含 N-乙酰半胱氨酸的冷藏血小板相比,5 mM N-乙酰半胱氨酸可明显降低 THP-1 或 Hep G2 细胞对血小板的吞噬作用:本研究证明了活性氧的产生与它们对冷藏后血小板清除的促氧化作用之间的相关性。添加适当浓度的 N-乙酰半胱氨酸不仅能保护冷藏血小板免受活性氧生成过多导致的储存损伤,还能防止血小板在体外被吞噬和在体内被清除,从而延长循环时间。
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引用次数: 0
Assessment of transfusion-induced iron overload with T2*MRI in survivors of childhood acute lymphoblastic leukemia: A case control study. 利用 T2*MRI 评估儿童急性淋巴细胞白血病幸存者输血引起的铁超载:病例对照研究
Pub Date : 2024-10-18 DOI: 10.1016/j.htct.2024.09.2478
Laila M Sherief, Mohamed Beshir, Sahar N Saleem, Wesam Elmozy, Mona Elkalioubie, Basma K Soliman, Amr M Fawzy, Mona Alsharkawy, Diana Hanna

Introduction: Childhood acute lymphoblastic leukemia survivors receiving multiple packed red blood transfusions may be at risk of vital organ iron deposition causing long-term complications. This study was undertaken to assess the prevalence and severity of iron overload in the liver and heart by magnetic resonance imaging.

Methods: A case-control study was conducted on 60 acute lymphoblastic leukemia survivors aged from 6 to 18 years and 60 healthy, age- and sex-matched children as a control group. The hematological profile, and serum ferritin was assessed and the iron content of the liver and heart was measured by T2* magnetic resonance imaging.

Results: Twenty-six (43.3 %) and two (3.3 %) patients had elevated liver and myocardial iron concentrations, respectively. The statistics show a significantly positive correlation between liver T2* magnetic resonance and serum ferritin. The total volume of blood transfused and duration of follow up were associated with elevated liver iron concentrations (p-values = 0.036 and 0.028 respectively). Myocardial T2* magnetic resonance lacked correlation with serum ferritin and transfusion therapy CONCLUSION: Liver iron overload was detected in children and adolescents after acute lymphoblastic leukemia therapy. The risk of iron overload was related mainly to the transfusion burden during therapy. These patients need monitoring after therapy to assess their need for future chelation therapy.

导言:儿童急性淋巴细胞白血病幸存者在接受多次充盈红细胞输血后,可能面临重要器官铁沉积导致长期并发症的风险。本研究旨在通过磁共振成像评估肝脏和心脏铁超载的发生率和严重程度:方法:研究人员对 60 名年龄在 6 至 18 岁之间的急性淋巴细胞白血病幸存者进行了病例对照研究,并以 60 名年龄和性别匹配的健康儿童作为对照组。研究评估了血液学特征和血清铁蛋白,并通过 T2* 磁共振成像测量了肝脏和心脏的铁含量:结果:分别有 26 名(43.3%)和 2 名(3.3%)患者的肝脏和心肌铁含量升高。统计数据显示,肝脏 T2* 磁共振成像与血清铁蛋白呈明显正相关。输血总量和随访时间与肝脏铁浓度升高有关(p 值分别为 0.036 和 0.028)。心肌 T2* 磁共振与血清铁蛋白和输血治疗缺乏相关性 结论:在急性淋巴细胞白血病治疗后的儿童和青少年中发现肝脏铁超载。铁超载的风险主要与治疗期间的输血负担有关。这些患者需要在治疗后接受监测,以评估他们今后是否需要接受螯合疗法。
{"title":"Assessment of transfusion-induced iron overload with T2*MRI in survivors of childhood acute lymphoblastic leukemia: A case control study.","authors":"Laila M Sherief, Mohamed Beshir, Sahar N Saleem, Wesam Elmozy, Mona Elkalioubie, Basma K Soliman, Amr M Fawzy, Mona Alsharkawy, Diana Hanna","doi":"10.1016/j.htct.2024.09.2478","DOIUrl":"https://doi.org/10.1016/j.htct.2024.09.2478","url":null,"abstract":"<p><strong>Introduction: </strong>Childhood acute lymphoblastic leukemia survivors receiving multiple packed red blood transfusions may be at risk of vital organ iron deposition causing long-term complications. This study was undertaken to assess the prevalence and severity of iron overload in the liver and heart by magnetic resonance imaging.</p><p><strong>Methods: </strong>A case-control study was conducted on 60 acute lymphoblastic leukemia survivors aged from 6 to 18 years and 60 healthy, age- and sex-matched children as a control group. The hematological profile, and serum ferritin was assessed and the iron content of the liver and heart was measured by T2* magnetic resonance imaging.</p><p><strong>Results: </strong>Twenty-six (43.3 %) and two (3.3 %) patients had elevated liver and myocardial iron concentrations, respectively. The statistics show a significantly positive correlation between liver T2* magnetic resonance and serum ferritin. The total volume of blood transfused and duration of follow up were associated with elevated liver iron concentrations (p-values = 0.036 and 0.028 respectively). Myocardial T2* magnetic resonance lacked correlation with serum ferritin and transfusion therapy CONCLUSION: Liver iron overload was detected in children and adolescents after acute lymphoblastic leukemia therapy. The risk of iron overload was related mainly to the transfusion burden during therapy. These patients need monitoring after therapy to assess their need for future chelation therapy.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142570803","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A comprehensive consolidation of data on the connection between CDKN2A polymorphisms and the susceptibility to childhood acute lymphoblastic leukemia. 关于 CDKN2A 多态性与儿童急性淋巴细胞白血病易感性之间关系的综合数据。
Pub Date : 2024-10-08 DOI: 10.1016/j.htct.2024.05.017
Maryam Aghasipour, Fatemeh Asadian, Seyed Alireza Dastgheib, Abolhasan Alijanpour, Ali Masoudi, Maedeh Barahman, Mohammad Golshan-Tafti, Reza Bahrami, Amirmasoud Shiri, Hossein Aarafi, Kazem Aghili, Hossein Neamatzadeh

Background: Acute lymphoblastic leukemia is the predominant neoplastic ailment in childhood. Prior research has already established noteworthy connections between CDKN2A polymorphisms and susceptibility to this childhood leukemia, however, substantial associations are still awaiting validation. This investigation was undertaken to examine the correlation between CDKN2A polymorphisms and the risk of acute lymphoblastic leukemia in children.

Methods: Acquisition of information encompassed the exploration of diverse databases including PubMed, Scopus, EMBASE, and China National Knowledge Infrastructure (CNKI) until January 10, 2024. An estimation of associations was achieved utilizing odds ratios with 95% confidence intervals.

Results: A total of 22 case-control studies encompassing 10,203 cases of acute lymphoblastic leukemia and 36,424 healthy controls were included. Within this pool of studies, 14 focused on rs3731217, comprising 5396 cases and 15,787 controls, whereas eight studies investigated rs3731249, comprising 4807 cases and 20,637 controls. The aggregated data showed that the rs3731217 variant offers protection against acute lymphoblastic leukemia. Nevertheless, when subgroups are analyzed according to ethnicity, it becomes clear that the rs3731217 polymorphism significantly influences susceptibility, particularly among individuals of Caucasian and African descent with no such association being observed in children of Asian origin. Nevertheless, the rs3731249 polymorphism displayed a noteworthy correlation with vulnerability to pediatric acute lymphoblastic leukemia.

Conclusion: The aggregated data revealed that the rs3731217 variation offers protection against the development of pediatric acute lymphoblastic leukemia and the rs3731249 polymorphism is significantly correlated with susceptibility.

背景:急性淋巴细胞白血病是儿童时期最主要的肿瘤性疾病。先前的研究已经确定了 CDKN2A 多态性与这种儿童白血病易感性之间值得注意的联系,然而,实质性的联系仍有待验证。这项调查旨在研究 CDKN2A 多态性与儿童患急性淋巴细胞白血病风险之间的相关性:方法:在 2024 年 1 月 10 日前,对包括 PubMed、Scopus、EMBASE 和中国国家知识基础设施(CNKI)在内的各种数据库进行了信息采集。利用带有 95% 置信区间的几率比对相关性进行了估计:结果:共纳入了 22 项病例对照研究,包括 10,203 例急性淋巴细胞白血病病例和 36,424 例健康对照。在这些研究中,14 项研究重点研究了 rs3731217,包括 5396 例病例和 15787 例对照;8 项研究调查了 rs3731249,包括 4807 例病例和 20637 例对照。综合数据显示,rs3731217 变体可预防急性淋巴细胞白血病。不过,如果根据种族对亚组进行分析,就会发现 rs3731217 多态性对易感性有显著影响,尤其是在高加索人和非洲人后裔中,而在亚裔儿童中则没有观察到这种关联。然而,rs3731249 多态性与小儿急性淋巴细胞白血病的易感性有显著的相关性:综合数据显示,rs3731217 变异可防止小儿急性淋巴细胞白血病的发生,而 rs3731249 多态性与易感性显著相关。
{"title":"A comprehensive consolidation of data on the connection between CDKN2A polymorphisms and the susceptibility to childhood acute lymphoblastic leukemia.","authors":"Maryam Aghasipour, Fatemeh Asadian, Seyed Alireza Dastgheib, Abolhasan Alijanpour, Ali Masoudi, Maedeh Barahman, Mohammad Golshan-Tafti, Reza Bahrami, Amirmasoud Shiri, Hossein Aarafi, Kazem Aghili, Hossein Neamatzadeh","doi":"10.1016/j.htct.2024.05.017","DOIUrl":"https://doi.org/10.1016/j.htct.2024.05.017","url":null,"abstract":"<p><strong>Background: </strong>Acute lymphoblastic leukemia is the predominant neoplastic ailment in childhood. Prior research has already established noteworthy connections between CDKN2A polymorphisms and susceptibility to this childhood leukemia, however, substantial associations are still awaiting validation. This investigation was undertaken to examine the correlation between CDKN2A polymorphisms and the risk of acute lymphoblastic leukemia in children.</p><p><strong>Methods: </strong>Acquisition of information encompassed the exploration of diverse databases including PubMed, Scopus, EMBASE, and China National Knowledge Infrastructure (CNKI) until January 10, 2024. An estimation of associations was achieved utilizing odds ratios with 95% confidence intervals.</p><p><strong>Results: </strong>A total of 22 case-control studies encompassing 10,203 cases of acute lymphoblastic leukemia and 36,424 healthy controls were included. Within this pool of studies, 14 focused on rs3731217, comprising 5396 cases and 15,787 controls, whereas eight studies investigated rs3731249, comprising 4807 cases and 20,637 controls. The aggregated data showed that the rs3731217 variant offers protection against acute lymphoblastic leukemia. Nevertheless, when subgroups are analyzed according to ethnicity, it becomes clear that the rs3731217 polymorphism significantly influences susceptibility, particularly among individuals of Caucasian and African descent with no such association being observed in children of Asian origin. Nevertheless, the rs3731249 polymorphism displayed a noteworthy correlation with vulnerability to pediatric acute lymphoblastic leukemia.</p><p><strong>Conclusion: </strong>The aggregated data revealed that the rs3731217 variation offers protection against the development of pediatric acute lymphoblastic leukemia and the rs3731249 polymorphism is significantly correlated with susceptibility.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142515298","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Epidemiologic profile of hemoglobinopathies in Benin. 贝宁血红蛋白病的流行病学概况。
Pub Date : 2024-10-08 DOI: 10.1016/j.htct.2024.07.008
Selma Gomez, Adjile Edjide Roukiyath Amoussa, Edwige Dedjinou, Manasse Kakpo, Pélagie Gbédji, Nouhoum Amossou Soulé, Bernice Quenum

Background: Sickle cell disease is the most common inherited blood disorder in the world with the birth of approximately 300,000 newborns screened each year. In 2009, the World Health Organization ranked the fight against sickle cell disease among the priorities for the Africa regions. The best way to prevent this incurable disease remains, on one hand systematic screening at birth, and on the other the proscription of risky union between heterozygous subjects.

Aim: The aim of this study was to analyze the epidemiological profile of sickle cell disease and other hemoglobinopathies in Benin and determine more up-to-date prevalence rates of the disease within the population.

Methods: The hemoglobin profiles of 2910 study participants were determined by quantitative electrophoresis. Samples with abnormal hemoglobin results were subjected to a complete blood count.

Results: Our study population was balanced between males (1528) and females (1382) with a sex ratio of 1.1. The mean age ranged from eight years in the pediatric group to 26 years in adults. The hemoglobin electrophoresis profiles found were as follows: 59.7 % Hb AA (normal), 21.7 % Hb AS, 10.2 % Hb AC, 3.1 % Hb SS, 3.7 % Hb SC, and 1.6 % of the rare phenotypes (Hb AD, Hb AE, Hb AF, Hb A/β-thal, Hb SD, Hb SF, Hb CC and Hb C/β-thal). Participants with abnormal hemoglobin presented a normochromic normocytic anemia. A total of 356 (12 %) people knew their profile compared to 2554 (88 %) who did not.

Conclusion: The high prevalence of hemoglobinopathies found in this study highlights in importance of screening in the Benin population.

背景:镰状细胞病是世界上最常见的遗传性血液疾病,每年约有 30 万新生儿接受筛查。2009 年,世界卫生组织将防治镰状细胞病列为非洲地区的优先事项之一。预防这种不治之症的最佳方法,一方面是在新生儿出生时进行系统筛查,另一方面是禁止杂合子之间的危险结合。目的:本研究旨在分析贝宁镰状细胞病和其他血红蛋白病的流行病学概况,并确定该疾病在人口中的最新患病率:采用定量电泳法测定了 2910 名研究参与者的血红蛋白状况。对血红蛋白结果异常的样本进行了全血细胞计数:我们的研究对象中男性(1528 人)和女性(1382 人)比例均衡,性别比为 1.1。平均年龄从儿童组的 8 岁到成人组的 26 岁不等。血红蛋白电泳图谱如下:59.7% 为血红蛋白 AA(正常),21.7% 为血红蛋白 AS,10.2% 为血红蛋白 AC,3.1% 为血红蛋白 SS,3.7% 为血红蛋白 SC,1.6% 为罕见表型(血红蛋白 AD、血红蛋白 AE、血红蛋白 AF、血红蛋白 A/β-thal、血红蛋白 SD、血红蛋白 SF、血红蛋白 CC 和血红蛋白 C/β-thal)。血红蛋白异常的参与者表现为正常色素性正常细胞性贫血。共有 356 人(12%)知道自己的情况,而不知道的有 2554 人(88%):本研究发现血红蛋白病的发病率很高,这凸显了在贝宁人口中进行筛查的重要性。
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引用次数: 0
Benefits of BV-AVD (Brentuximab vedotin, doxorubicin, vinblastine, and dacarbazine) versus ABVD (doxorubicin hydrochloride, bleomycin sulfate, vinblastine sulfate, dacarbazine) in patients with advanced-stage Hodgkin's lymphoma: an analysis of the ECHELON 1 trial by the GATLA group using the Delphi Method. BV-AVD(布伦妥昔单抗韦多汀、多柔比星、长春新碱和达卡巴嗪)与 ABVD(盐酸多柔比星、硫酸博来霉素、硫酸长春新碱和达卡巴嗪)对晚期霍奇金淋巴瘤患者的益处:GATLA 小组使用德尔菲法对 ECHELON 1 试验进行的分析。
Pub Date : 2024-09-25 DOI: 10.1016/j.htct.2024.07.007
Astrid Pavlovsky, Juan Ignacio Garcia Altuve, Amalia Cerutti, Lorena Fiad, Nicolás Kurgansky, Fernando Warley, Florencia Negri Aranguren

Introduction: The BV-AVD (Brentuximab vedotin, doxorubicin, vinblastine, and dacarbazine) combination for first-line treatment of advanced stage Hodgkin's lymphoma has been approved by regulatory authorities and included in international guidelines. However, several factors influence its incorporation as standard of care.

Materials and methods: A group of experts from different institutions was identified and, using the Delphi method, an analysis of the results of the ECHELON 1 trial for the indication of BV-AVD over ABVD (doxorubicin hydrochloride, bleomycin sulfate, vinblastine sulfate, dacarbazine) in patients with Hodgkin's lymphoma Stages III and IV in Argentina was done. The clinical and academic experience of the authors and the context of the Argentine healthcare system were considered.

Results and discussion: Seven statements on general aspects of the management of Hodgkin's lymphoma and nine on specific aspects related to the use of BV-AVD over ABVD reached a consensus of agreement. There was a strong expert consensus in favor of indicating BV-AVD in the presence of extranodal disease or pulmonary disease. Moderate to severe neuropathy, pregnancy and drug allergy were considered absolute contraindications to prescribe BV.

Conclusions: The authors agreed that BV-AVD could be considered a new treatment option in high-risk patients. However health system-dependent factors (such as high cost, lack of availability, reimbursement difficulties, irregular delivery, and issues with granulocyte-colony stimulating factor availability) could pose limitations for this prescription. While awaiting new data from clinical trials and real-world studies, these recommendations can represent a useful tool for hematologists in different parts of the world.

简介BV-AVD (Brentuximab vedotin、多柔比星、长春新碱和达卡巴嗪)联合疗法用于晚期霍奇金淋巴瘤的一线治疗,已获得监管机构批准并被纳入国际指南。然而,有几个因素影响着它被纳入标准治疗:确定了一个由来自不同机构的专家组成的小组,并采用德尔菲法分析了 ECHELON 1 试验的结果,该试验的目的是在阿根廷的霍奇金淋巴瘤 III 期和 IV 期患者中将 BV-AVD 用于 ABVD(盐酸多柔比星、硫酸博来霉素、硫酸长春新碱、达卡巴嗪)的适应症。研究考虑了作者的临床和学术经验以及阿根廷医疗系统的背景:就霍奇金淋巴瘤管理的一般方面有七项声明,就使用 BV-AVD 而非 ABVD 的具体方面有九项声明达成了一致意见。专家们一致赞成在出现结节外疾病或肺部疾病时使用 BV-AVD。中度至重度神经病变、妊娠和药物过敏被视为开具 BV 的绝对禁忌症:作者们一致认为,BV-AVD 可被视为高危患者的一种新的治疗选择。然而,医疗系统的相关因素(如费用高昂、缺乏可用性、报销困难、不规则分娩以及粒细胞集落刺激因子的可用性问题)可能会限制这种处方的使用。在等待来自临床试验和实际研究的新数据的同时,这些建议可为世界各地的血液学专家提供有用的工具。
{"title":"Benefits of BV-AVD (Brentuximab vedotin, doxorubicin, vinblastine, and dacarbazine) versus ABVD (doxorubicin hydrochloride, bleomycin sulfate, vinblastine sulfate, dacarbazine) in patients with advanced-stage Hodgkin's lymphoma: an analysis of the ECHELON 1 trial by the GATLA group using the Delphi Method.","authors":"Astrid Pavlovsky, Juan Ignacio Garcia Altuve, Amalia Cerutti, Lorena Fiad, Nicolás Kurgansky, Fernando Warley, Florencia Negri Aranguren","doi":"10.1016/j.htct.2024.07.007","DOIUrl":"10.1016/j.htct.2024.07.007","url":null,"abstract":"<p><strong>Introduction: </strong>The BV-AVD (Brentuximab vedotin, doxorubicin, vinblastine, and dacarbazine) combination for first-line treatment of advanced stage Hodgkin's lymphoma has been approved by regulatory authorities and included in international guidelines. However, several factors influence its incorporation as standard of care.</p><p><strong>Materials and methods: </strong>A group of experts from different institutions was identified and, using the Delphi method, an analysis of the results of the ECHELON 1 trial for the indication of BV-AVD over ABVD (doxorubicin hydrochloride, bleomycin sulfate, vinblastine sulfate, dacarbazine) in patients with Hodgkin's lymphoma Stages III and IV in Argentina was done. The clinical and academic experience of the authors and the context of the Argentine healthcare system were considered.</p><p><strong>Results and discussion: </strong>Seven statements on general aspects of the management of Hodgkin's lymphoma and nine on specific aspects related to the use of BV-AVD over ABVD reached a consensus of agreement. There was a strong expert consensus in favor of indicating BV-AVD in the presence of extranodal disease or pulmonary disease. Moderate to severe neuropathy, pregnancy and drug allergy were considered absolute contraindications to prescribe BV.</p><p><strong>Conclusions: </strong>The authors agreed that BV-AVD could be considered a new treatment option in high-risk patients. However health system-dependent factors (such as high cost, lack of availability, reimbursement difficulties, irregular delivery, and issues with granulocyte-colony stimulating factor availability) could pose limitations for this prescription. While awaiting new data from clinical trials and real-world studies, these recommendations can represent a useful tool for hematologists in different parts of the world.</p>","PeriodicalId":94026,"journal":{"name":"Hematology, transfusion and cell therapy","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142376479","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Venetoclax with low-dose cytarabine, a forgotten combination in patients with acute myeloid leukemia ineligible for intensive chemotherapy: a systematic review. Venetoclax与小剂量阿糖胞苷--不符合强化化疗条件的急性髓性白血病患者的忘我组合:系统综述。
Pub Date : 2024-09-23 DOI: 10.1016/j.htct.2024.07.006
Lauro Fabián Amador-Medina, Erick Crespo-Solís, Francisco Javier Turrubiates-Hernández, Karla Edith Santibañez-Bedolla

Background: Based on the VIALE-A and VIALE-C studies, the Food and Drug Administration approved venetoclax in 2020 in combination with azacitidine or low-dose cytarabine for the treatment of patients with acute myeloid leukemia ineligible for intensive chemotherapy. After the publication of these studies, venetoclax/azacitidine was assumed to be superior to venetoclax/low-dose cytarabine; however, these studies were not designed to demonstrate superiority between these combinations. Therefore, we conducted a systematic review to describe overall survival, complete remission rate, and composite complete remission rate to assess response of these two regimens in patients with newly diagnosed acute myeloid leukemia who are ineligible for intensive chemotherapy.

Materials and methods: The PubMed and Web of Science databases were searched for retrospective studies and complete remission, composite complete remission, and overall survival rates were recorded.

Results: Only 11 of the 815 publications identified were eligible to be included n this review, ten studies evaluated the venetoclax/azacitidine combination and one study evaluated the venetoclax/low-dose cytarabine combination. The median overall survival for venetoclax/azacitidine was 10.75 months, whereas for venetoclax/low-dose cytarabine the median overall survival had not been reached at the time of publication. Composite complete remission was 63.3 % for venetoclax/azacitidine and 90 % for venetoclax/low-dose cytarabine. Adverse events were similar for both combinations.

Conclusions: A limited number of studies investigating the venetoclax/low-dose cytarabine combination exist. Based on the available data, the superiority of venetoclax/azacitidine over venetoclax/low-dose cytarabine cannot be assumed for all acute myeloid leukemia patients who are ineligible for intensive chemotherapy. Venetoclax/low-dose cytarabine can still be considered as an option for the drug combinations currently under investigation.

背景:基于VIALE-A和VIALE-C研究,美国食品和药物管理局于2020年批准了venetoclax与阿扎胞苷或小剂量阿糖胞苷联合用于治疗不符合强化化疗条件的急性髓性白血病患者。这些研究发表后,人们认为 Venetoclax/阿扎胞苷优于 Venetoclax/小剂量阿糖胞苷;然而,这些研究并非旨在证明这些组合之间的优越性。因此,我们进行了一项系统性综述,以描述新诊断的急性髓性白血病患者的总生存期、完全缓解率和复合完全缓解率,评估这两种方案对不符合强化化疗条件的患者的反应:在PubMed和Web of Science数据库中检索回顾性研究,记录完全缓解率、复合完全缓解率和总生存率:在815篇文献中,只有11篇符合纳入本综述的条件,其中10篇研究评估了venetoclax/阿扎胞苷联合疗法,1篇研究评估了venetoclax/小剂量阿糖胞苷联合疗法。Venetoclax/阿扎胞苷的中位总生存期为10.75个月,而Venetoclax/小剂量阿糖胞苷的中位总生存期在发表时尚未达到。Venetoclax/阿扎胞苷的复合完全缓解率为63.3%,Venetoclax/小剂量阿糖胞苷的复合完全缓解率为90%。两种组合的不良反应相似:研究文尼他克/小剂量阿糖胞苷组合的研究数量有限。根据现有数据,对于所有不符合强化化疗条件的急性髓性白血病患者,不能认为 Venetoclax/azacitidine 优于 Venetoclax/ 小剂量阿糖胞苷。对于目前正在研究的药物组合,仍可考虑将 Venetoclax/ 小剂量阿糖胞苷作为一种选择。
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Hematology, transfusion and cell therapy
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