Pub Date : 2023-07-01DOI: 10.1097/BS9.0000000000000157
Lixian Chang, Mingchen Yan, Jingliao Zhang, Binghang Liu, Li Zhang, Ye Guo, Jing Sun, Yang Wan, Meihui Yi, Yang Lan, Yuli Cai, Yuanyuan Ren, Haihui Zheng, Aoli Zhang, Zhenyu Li, Jian Wang, Yingrui Li, Xiaofan Zhu
Children with severe aplastic anemia (SAA) face heterogeneous prognoses after immunosuppressive therapy (IST). There are few models that can predict the long-term outcomes of IST for these patients. The objective of this paper is to develop a more effective prediction model for SAA prognosis based on clinical electronic medical records from 203 children with newly diagnosed SAA. In the early stage, a novel model for long-term outcomes of SAA patients with IST was developed using machine-learning techniques. Among the indicators related to long-term efficacy, white blood cell count, lymphocyte count, absolute reticulocyte count, lymphocyte ratio in bone-marrow smears, C-reactive protein, and the level of IL-6, IL-8 and vitamin B12 in the early stage are strongly correlated with long-term efficacy (P < .05). Taken together, we analyzed the long-term outcomes of rabbit anti-thymocyte globulin and cyclosporine therapy for children with SAA through machine-learning techniques, which may shorten the observation period of therapeutic effects and reduce treatment costs and time.
{"title":"An investigation of long-term outcome of rabbit anti-thymocyte globulin and cyclosporine therapy for pediatric severe aplastic anemia.","authors":"Lixian Chang, Mingchen Yan, Jingliao Zhang, Binghang Liu, Li Zhang, Ye Guo, Jing Sun, Yang Wan, Meihui Yi, Yang Lan, Yuli Cai, Yuanyuan Ren, Haihui Zheng, Aoli Zhang, Zhenyu Li, Jian Wang, Yingrui Li, Xiaofan Zhu","doi":"10.1097/BS9.0000000000000157","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000157","url":null,"abstract":"<p><p>Children with severe aplastic anemia (SAA) face heterogeneous prognoses after immunosuppressive therapy (IST). There are few models that can predict the long-term outcomes of IST for these patients. The objective of this paper is to develop a more effective prediction model for SAA prognosis based on clinical electronic medical records from 203 children with newly diagnosed SAA. In the early stage, a novel model for long-term outcomes of SAA patients with IST was developed using machine-learning techniques. Among the indicators related to long-term efficacy, white blood cell count, lymphocyte count, absolute reticulocyte count, lymphocyte ratio in bone-marrow smears, C-reactive protein, and the level of IL-6, IL-8 and vitamin B12 in the early stage are strongly correlated with long-term efficacy (<i>P</i> < .05). Taken together, we analyzed the long-term outcomes of rabbit anti-thymocyte globulin and cyclosporine therapy for children with SAA through machine-learning techniques, which may shorten the observation period of therapeutic effects and reduce treatment costs and time.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":"5 3","pages":"180-186"},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a4/e8/bs9-5-180.PMC10400069.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9953316","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}
Pub Date : 2023-07-01DOI: 10.1097/BS9.0000000000000159
Yanan Li, Haihang Zhang, Bin Hu, Pan Wang, Wei Wang, Jing Liu
Erythropoiesis is a complex, precise, and lifelong process that is essential for maintaining normal body functions. Its strict regulation is necessary to prevent a variety of blood diseases. Normal erythropoiesis is precisely regulated by an intricate network that involves transcription levels, signal transduction, and various epigenetic modifications. In recent years, research on post-transcriptional levels in erythropoiesis has expanded significantly. The dynamic regulation of splicing transitions is responsible for changes in protein isoform expression that add new functions beneficial for erythropoiesis. RNA-binding proteins adapt the translation of transcripts to the protein requirements of the cell, yielding mRNA with dynamic translation efficiency. Noncoding RNAs, such as microRNAs and lncRNAs, are indispensable for changing the translational efficiency and/or stability of targeted mRNAs to maintain the normal expression of genes related to erythropoiesis. N6-methyladenosine-dependent regulation of mRNA translation plays an important role in maintaining the expression programs of erythroid-related genes and promoting erythroid lineage determination. This review aims to describe our current understanding of the role of post-transcriptional regulation in erythropoiesis and erythroid-associated diseases, and to shed light on the physiological and pathological implications of the post-transcriptional regulation machinery in erythropoiesis. These may help to further enrich our understanding of the regulatory network of erythropoiesis and provide new strategies for the diagnosis and treatment of erythroid-related diseases.
{"title":"Post-transcriptional regulation of erythropoiesis.","authors":"Yanan Li, Haihang Zhang, Bin Hu, Pan Wang, Wei Wang, Jing Liu","doi":"10.1097/BS9.0000000000000159","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000159","url":null,"abstract":"<p><p>Erythropoiesis is a complex, precise, and lifelong process that is essential for maintaining normal body functions. Its strict regulation is necessary to prevent a variety of blood diseases. Normal erythropoiesis is precisely regulated by an intricate network that involves transcription levels, signal transduction, and various epigenetic modifications. In recent years, research on post-transcriptional levels in erythropoiesis has expanded significantly. The dynamic regulation of splicing transitions is responsible for changes in protein isoform expression that add new functions beneficial for erythropoiesis. RNA-binding proteins adapt the translation of transcripts to the protein requirements of the cell, yielding mRNA with dynamic translation efficiency. Noncoding RNAs, such as microRNAs and lncRNAs, are indispensable for changing the translational efficiency and/or stability of targeted mRNAs to maintain the normal expression of genes related to erythropoiesis. N6-methyladenosine-dependent regulation of mRNA translation plays an important role in maintaining the expression programs of erythroid-related genes and promoting erythroid lineage determination. This review aims to describe our current understanding of the role of post-transcriptional regulation in erythropoiesis and erythroid-associated diseases, and to shed light on the physiological and pathological implications of the post-transcriptional regulation machinery in erythropoiesis. These may help to further enrich our understanding of the regulatory network of erythropoiesis and provide new strategies for the diagnosis and treatment of erythroid-related diseases.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":"5 3","pages":"150-159"},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400058/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10006851","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}
Pub Date : 2023-07-01DOI: 10.1097/BS9.0000000000000161
Sanjay K Thakur, Anil K Sinha, Dinesh K Negi, Sompal Singh
The unforeseen and uncertain life-threatening situation of the COVID-19 pandemic dramatically affected all areas of the human daily work schedule. This study was designed to assess the impact of the COVID-19 pandemic on blood transfusion services and discuss the adopted confrontation measures for uninterrupted blood supply during the pandemic situation. The data on blood donation, blood component preparation, and issue from January 2019 to December 2022 were collected from the inventory registers of the RBTC, Delhi, India. Compared to the non-pandemic year 2019, during the year 2020, all variables decreased gradually. The observed maximum decrease in variables such as blood collection (-79.16%) in the month of October, blood issue (-71.61%) in the month of August, random donor platelets (RDP) preparation (-98.09%) in the month of October, RDP issue (-86.08%) in the month of September, fresh frozen plasma (FFP) preparation (-100%) in the month of October, and FFP issue (-96.08%) in the month of July with an annual decrease of -45.52%, -42.87%, -33.00%, -59.79%, -40.98%, and -54.48%, respectively, as compared to year 2019. Compared to year 2020, in year 2021, the annual increase in blood collection, blood issue, FFP preparation, FFP issue, RDP preparation, and RDP issue was +50.20%, +21.68%, +65.31%, +78.52%, +116.23%, and +213.30%, respectively. Our study results show that the COVID-19 pandemic has significantly affected blood transfusion services at our blood bank. The adopted coping strategies to maintain the safe and uninterrupted blood transfusion chain at our blood bank gave us lessons for future preparedness if faced with a similar situation.
{"title":"Effect of COVID-19 pandemic on blood transfusion service: an experience from a regional blood transfusion center.","authors":"Sanjay K Thakur, Anil K Sinha, Dinesh K Negi, Sompal Singh","doi":"10.1097/BS9.0000000000000161","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000161","url":null,"abstract":"<p><p>The unforeseen and uncertain life-threatening situation of the COVID-19 pandemic dramatically affected all areas of the human daily work schedule. This study was designed to assess the impact of the COVID-19 pandemic on blood transfusion services and discuss the adopted confrontation measures for uninterrupted blood supply during the pandemic situation. The data on blood donation, blood component preparation, and issue from January 2019 to December 2022 were collected from the inventory registers of the RBTC, Delhi, India. Compared to the non-pandemic year 2019, during the year 2020, all variables decreased gradually. The observed maximum decrease in variables such as blood collection (-79.16%) in the month of October, blood issue (-71.61%) in the month of August, random donor platelets (RDP) preparation (-98.09%) in the month of October, RDP issue (-86.08%) in the month of September, fresh frozen plasma (FFP) preparation (-100%) in the month of October, and FFP issue (-96.08%) in the month of July with an annual decrease of -45.52%, -42.87%, -33.00%, -59.79%, -40.98%, and -54.48%, respectively, as compared to year 2019. Compared to year 2020, in year 2021, the annual increase in blood collection, blood issue, FFP preparation, FFP issue, RDP preparation, and RDP issue was +50.20%, +21.68%, +65.31%, +78.52%, +116.23%, and +213.30%, respectively. Our study results show that the COVID-19 pandemic has significantly affected blood transfusion services at our blood bank. The adopted coping strategies to maintain the safe and uninterrupted blood transfusion chain at our blood bank gave us lessons for future preparedness if faced with a similar situation.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":"5 3","pages":"209-217"},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400046/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10006853","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}
Pub Date : 2023-07-01DOI: 10.1097/BS9.0000000000000164
[This corrects the article DOI: 10.1097/BS9.0000000000000144.].
[这更正了文章DOI: 10.1097/BS9.0000000000000144.]。
{"title":"Erratum: Benefit of rituximab maintenance is associated with Follicular Lymphoma International Prognostic Index in patients with follicular lymphoma.","authors":"","doi":"10.1097/BS9.0000000000000164","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000164","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1097/BS9.0000000000000144.].</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":"5 3","pages":"219"},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400060/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9953311","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}
Pub Date : 2023-07-01DOI: 10.1097/BS9.0000000000000163
[This corrects the article DOI: 10.1097/BS9.0000000000000145.].
[这更正了文章DOI: 10.1097/BS9.0000000000000145.]。
{"title":"Erratum: Development of Auer bodies from giant inclusions associated with rough endoplasmic reticulum in acute promyelocytic leukemia.","authors":"","doi":"10.1097/BS9.0000000000000163","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000163","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1097/BS9.0000000000000145.].</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":"5 3","pages":"218"},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400052/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9953312","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}
Pub Date : 2023-07-01DOI: 10.1097/BS9.0000000000000165
Yue Liu, Lingna An, Chengfei Yang, Xiaoqi Wang, Ruihao Huang, Xi Zhang
Adoptive cell therapy (ACT) has emerged with remarkable efficacies for tumor immunotherapy. Chimeric antigen receptor (CAR) T cell therapy, as one of most promising ACTs, has achieved prominent effects in treating malignant hematological tumors. However, the insufficient killing activity and limited persistence of T cells in the immunosuppressive tumor microenvironment limit the further application of ACTs for cancer patients. Many studies have focused on improving cytotoxicity and persistence of T cells to achieve improved therapeutic effects. In this study, we explored the potential function in ACT of ginsenoside Rg1, the main pharmacologically active component of ginseng. We introduced Rg1 during the in vitro activation and expansion phase of T cells, and found that Rg1 treatment upregulated two T cell activation markers, CD69 and CD25, while promoting T cell differentiation towards a mature state. Transcriptome sequencing revealed that Rg1 influenced T cell metabolic reprogramming by strengthening mitochondrial biosynthesis. When co-cultured with tumor cells, Rg1-treated T cells showed stronger cytotoxicity than untreated cells. Moreover, adding Rg1 to the culture endowed CAR-T cells with enhanced anti-tumor efficacy. This study suggests that ginsenoside Rg1 provides a potential approach for improving the anti-tumor efficacy of ACT by enhancing T cell effector functions.
{"title":"Ginsenoside Rg1 improves anti-tumor efficacy of adoptive cell therapy by enhancing T cell effector functions.","authors":"Yue Liu, Lingna An, Chengfei Yang, Xiaoqi Wang, Ruihao Huang, Xi Zhang","doi":"10.1097/BS9.0000000000000165","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000165","url":null,"abstract":"<p><p>Adoptive cell therapy (ACT) has emerged with remarkable efficacies for tumor immunotherapy. Chimeric antigen receptor (CAR) T cell therapy, as one of most promising ACTs, has achieved prominent effects in treating malignant hematological tumors. However, the insufficient killing activity and limited persistence of T cells in the immunosuppressive tumor microenvironment limit the further application of ACTs for cancer patients. Many studies have focused on improving cytotoxicity and persistence of T cells to achieve improved therapeutic effects. In this study, we explored the potential function in ACT of ginsenoside Rg1, the main pharmacologically active component of ginseng. We introduced Rg1 during the in vitro activation and expansion phase of T cells, and found that Rg1 treatment upregulated two T cell activation markers, CD69 and CD25, while promoting T cell differentiation towards a mature state. Transcriptome sequencing revealed that Rg1 influenced T cell metabolic reprogramming by strengthening mitochondrial biosynthesis. When co-cultured with tumor cells, Rg1-treated T cells showed stronger cytotoxicity than untreated cells. Moreover, adding Rg1 to the culture endowed CAR-T cells with enhanced anti-tumor efficacy. This study suggests that ginsenoside Rg1 provides a potential approach for improving the anti-tumor efficacy of ACT by enhancing T cell effector functions.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":"5 3","pages":"170-179"},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400057/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10006854","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}
Pub Date : 2023-04-01DOI: 10.1097/BS9.0000000000000148
Jean Luc Harousseau
Immune therapy is a new avenue in the treatment of multiple myeloma (MM). The naked anti-38 antibodies daratumumab and isatuximab are already used in frontline therapy after excellent results have been achieved in relapsed/refractory MM (RRMM).The second step was the development of immune therapies targeting B cell maturation antigen (BCMA). Genetically mod- ified autologous chimeric antigen receptor (CAR)-T cell therapy BCMA-directed were initially tested in heavily pretreated patients with RRMM exposed to the 3 main therapeutic classes, immunomodulatory drugs (iMiDs), proteasome inhibitors, and anti-CD38 antibodies (triple-class exposed). Efficacy results were unprecedented in this context and Idecabtagene vicleucel (ide-cel or Abecma) was the first BCMA-directed CAR-T cell therapy approved in MM by both Federal Drug Administration (FDA) and European Medicines Agency (EMA). This approval was based on the results of the KarMMa Phase 2 study in 128 patients with RRMM who had previously received a median number of 6 lines of therapy (LOT). 1 The response rate (RR) was 73%, including 33% of complete response (CR) or better and 26% negative min- imal residual disease (MRD). However, median progression-free survival (PFS) was only 8.8 months. The Cartitude 1 Phase1b/2 evaluated ciltacabtagene autoleucel (cilta-cel) another CAR-T cell therapy with 2 BCMA-targeting single domain antibodies in 97 heavily pretreated patients with RRMM. 2 Results were recently updated and with a median follow-up of 27.7 months, 3 the results were even more impressive with a 98% RR, including 82.5% CR or better, and 92% negative MRD in evaluable patients. The 27-month PFS was 55%. With these results, Cilta-cel (Carvykti) was
{"title":"CAR-T cell therapy in myeloma: hopes and hurdles.","authors":"Jean Luc Harousseau","doi":"10.1097/BS9.0000000000000148","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000148","url":null,"abstract":"Immune therapy is a new avenue in the treatment of multiple myeloma (MM). The naked anti-38 antibodies daratumumab and isatuximab are already used in frontline therapy after excellent results have been achieved in relapsed/refractory MM (RRMM).The second step was the development of immune therapies targeting B cell maturation antigen (BCMA). Genetically mod- ified autologous chimeric antigen receptor (CAR)-T cell therapy BCMA-directed were initially tested in heavily pretreated patients with RRMM exposed to the 3 main therapeutic classes, immunomodulatory drugs (iMiDs), proteasome inhibitors, and anti-CD38 antibodies (triple-class exposed). Efficacy results were unprecedented in this context and Idecabtagene vicleucel (ide-cel or Abecma) was the first BCMA-directed CAR-T cell therapy approved in MM by both Federal Drug Administration (FDA) and European Medicines Agency (EMA). This approval was based on the results of the KarMMa Phase 2 study in 128 patients with RRMM who had previously received a median number of 6 lines of therapy (LOT). 1 The response rate (RR) was 73%, including 33% of complete response (CR) or better and 26% negative min- imal residual disease (MRD). However, median progression-free survival (PFS) was only 8.8 months. The Cartitude 1 Phase1b/2 evaluated ciltacabtagene autoleucel (cilta-cel) another CAR-T cell therapy with 2 BCMA-targeting single domain antibodies in 97 heavily pretreated patients with RRMM. 2 Results were recently updated and with a median follow-up of 27.7 months, 3 the results were even more impressive with a 98% RR, including 82.5% CR or better, and 92% negative MRD in evaluable patients. The 27-month PFS was 55%. With these results, Cilta-cel (Carvykti) was","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":"5 2","pages":"136-137"},"PeriodicalIF":0.0,"publicationDate":"2023-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10205213/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9530769","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}
Pub Date : 2023-04-01DOI: 10.1097/BS9.0000000000000145
Yong-Xin Ru, Shu-Xu Dong, Jing Liu, Brian Eyden
Giant inclusions and Auer bodies in promyeloblasts were investigated in a study which included transmission electron microscopy (TEM) for morphology and ultrastructural cytochemistry for myeloperoxidase in 10 patients with acute promyelocytic leukemia (APL). Ultrastructural cytochemistry demonstrated positive myeloperoxidase reactivity in giant inclusions, expanded rER cisternae, Auer bodies and primary granules. TEM revealed that giant inclusions were adorned by degenerated rER membrane, some of them sharing features with Auer bodies. We hypothesize a novel origin for Auer body development in promyeloblasts of APL, namely that they originate from peroxidase-positive and expanded rER cisternae, and that primary granules were directly released from these expanded rER elements, bypassing the Golgi apparatus.
{"title":"Development of Auer bodies from giant inclusions associated with rough endoplasmic reticulum in acute promyelocytic leukemia.","authors":"Yong-Xin Ru, Shu-Xu Dong, Jing Liu, Brian Eyden","doi":"10.1097/BS9.0000000000000145","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000145","url":null,"abstract":"<p><p>Giant inclusions and Auer bodies in promyeloblasts were investigated in a study which included transmission electron microscopy (TEM) for morphology and ultrastructural cytochemistry for myeloperoxidase in 10 patients with acute promyelocytic leukemia (APL). Ultrastructural cytochemistry demonstrated positive myeloperoxidase reactivity in giant inclusions, expanded rER cisternae, Auer bodies and primary granules. TEM revealed that giant inclusions were adorned by degenerated rER membrane, some of them sharing features with Auer bodies. We hypothesize a novel origin for Auer body development in promyeloblasts of APL, namely that they originate from peroxidase-positive and expanded rER cisternae, and that primary granules were directly released from these expanded rER elements, bypassing the Golgi apparatus.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":"5 2","pages":"111-117"},"PeriodicalIF":0.0,"publicationDate":"2023-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10205345/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10299795","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}
Invasive fungal diseases (IFDs) are major and lethal infectious complications for patients with neutropenia after chemotherapy. Prophylaxis with intravenous and oral suspended itraconazole (200 mg Q12h intravenously × 2 days followed by 5 mg/kg·d orally in twice) or oral suspension of posaconazole (200 mg Q8h) was administered for preventing IFDs. The only 2 episodes of proven IFDs were not included after propensity-score matching (PSM), while the incidence of possible IFDs was 8.2% (9/110) in itraconazole group and 1.8% (2/110) in posaconazole group, respectively (P = .030). In clinical failure analysis, the failure rate of posaconazole group was lower as compared to the itraconazole group (2.7% vs 10.9%, P = .016). Both intravenous-oral itraconazole and posaconazole suspension are effective in preventing IFDs, while posaconazole suspension seems more tolerable.
侵袭性真菌病(IFDs)是中性粒细胞减少患者化疗后主要的致死性感染性并发症。预防方法:静脉滴注和口服伊曲康唑悬浮液(200 mg Q12h静脉滴注× 2天,随后5 mg/kg·d口服2次)或泊沙康唑口服悬浮液(200 mg Q8h)。经倾向评分匹配(PSM)后,仅有2例确诊IFDs未被纳入,而伊曲康唑组和泊沙康唑组的可能IFDs发生率分别为8.2%(9/110)和1.8% (2/110)(P = 0.030)。在临床失败分析中,泊沙康唑组的失败率低于伊曲康唑组(2.7% vs 10.9%, P = 0.016)。静脉-口服伊曲康唑和泊沙康唑混悬液均可有效预防ifd,泊沙康唑混悬液的耐受性更强。
{"title":"Intravenous-oral itraconazole versus oral posaconazole in preventing invasive fungal diseases for acute leukemia patients.","authors":"Li Liu, Xiaolei Pei, Runzhi Ma, Yi He, Rongli Zhang, Jialin Wei, Qiaoling Ma, Weihua Zhai, Aiming Pang, Erlie Jiang, Mingzhe Han, Donglin Yang, Sizhou Feng","doi":"10.1097/BS9.0000000000000155","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000155","url":null,"abstract":"<p><p>Invasive fungal diseases (IFDs) are major and lethal infectious complications for patients with neutropenia after chemotherapy. Prophylaxis with intravenous and oral suspended itraconazole (200 mg Q12h intravenously × 2 days followed by 5 mg/kg·d orally in twice) or oral suspension of posaconazole (200 mg Q8h) was administered for preventing IFDs. The only 2 episodes of proven IFDs were not included after propensity-score matching (PSM), while the incidence of possible IFDs was 8.2% (9/110) in itraconazole group and 1.8% (2/110) in posaconazole group, respectively (<i>P</i> = .030). In clinical failure analysis, the failure rate of posaconazole group was lower as compared to the itraconazole group (2.7% vs 10.9%, <i>P</i> = .016). Both intravenous-oral itraconazole and posaconazole suspension are effective in preventing IFDs, while posaconazole suspension seems more tolerable.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":"5 2","pages":"106-110"},"PeriodicalIF":0.0,"publicationDate":"2023-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10205377/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9525569","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}
Pub Date : 2023-04-01DOI: 10.1097/BS9.0000000000000150
Jianmin Wang, Dan Yang
In a recent issue of Lancet Haematology , Xue et al 1 reported an adeno-associated virus (AAV)-based gene therapy in 10 patients with hemophilia B (HB) from China. BBM-H901, a novel vector comprised of an engineered liver-tropic AAV cap- sid (AAV843), synthesized liver-specific promoter and CpG reduced factor IX (FIX) Padua coding sequence, was infused in 10 patients (baseline FIX coagulation activity [FIX:C] were less than 2 IU/dL) after 1 week of prophylactic prednisone pretreat- ment (1 mg/kg per day). After a median follow-up of 58 weeks, mean FIX:C reached 36.9 ± 20·5 IU/dL. No FIX inhibitors or serious adverse events were observed. All patients developed high titer neutralizing antibodies against vector capsid. The concentrations of alanine aminotransferase and aspartate aminotransferase in plasma were below the upper limit of normal range in 8 patients. No FIX concentrate infusion was needed after gene therapy for these patients. This is a huge step forward in the treatment of HB in China.
{"title":"Big stride in gene therapy for hemophilia B in China.","authors":"Jianmin Wang, Dan Yang","doi":"10.1097/BS9.0000000000000150","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000150","url":null,"abstract":"In a recent issue of Lancet Haematology , Xue et al 1 reported an adeno-associated virus (AAV)-based gene therapy in 10 patients with hemophilia B (HB) from China. BBM-H901, a novel vector comprised of an engineered liver-tropic AAV cap- sid (AAV843), synthesized liver-specific promoter and CpG reduced factor IX (FIX) Padua coding sequence, was infused in 10 patients (baseline FIX coagulation activity [FIX:C] were less than 2 IU/dL) after 1 week of prophylactic prednisone pretreat- ment (1 mg/kg per day). After a median follow-up of 58 weeks, mean FIX:C reached 36.9 ± 20·5 IU/dL. No FIX inhibitors or serious adverse events were observed. All patients developed high titer neutralizing antibodies against vector capsid. The concentrations of alanine aminotransferase and aspartate aminotransferase in plasma were below the upper limit of normal range in 8 patients. No FIX concentrate infusion was needed after gene therapy for these patients. This is a huge step forward in the treatment of HB in China.","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":"5 2","pages":"138-139"},"PeriodicalIF":0.0,"publicationDate":"2023-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10205292/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9530768","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}