Ja Min Byun, Sung-Soo Park, Sung-Soo Yoon, Ari Ahn, Myungshin Kim, Jung Yeon Lee, Young-Woo Jeon, Seung-Hwan Shin, Seung-Ah Yahng, Youngil Koh, Chang-Ki Min
Background: The goal of induction therapy for multiple myeloma (MM) is to achieve adequate disease control. Current guidelines favor triplet (bortezomib-lenalidomide-dexamethasone; VRd) or quadruplet regimens (daratumumab, bortezomib-thalidomide-dexamethasone; D-VTd). In the absence of a direct comparison between two treatment regimens, we conducted this study to compare the outcomes and safety of VRd and D-VTd.
Methods: Newly diagnosed MM patients aged >18 years who underwent induction therapy followed by autologous stem cell transplantation (ASCT) between November 2020 and December 2021 were identified. Finally, patients with VRd (N=37) and those with D-VTd (N=43) were enrolled.
Results: After induction, 10.8% of the VRd group showed stringent complete remission (sCR), 21.6% showed complete response (CR), 35.1% showed very good partial response (VGPR), and 32.4% showed partial response (PR). Of the D-VTd group, 9.3% showed sCR, 34.9% CR, 48.8% VGPR, and 4.2% PR (VGPR or better: 67.6% in VRd vs. 93% in D-VTd, P=0.004). After ASCT, 68.6% of the VRd group showed CR or sCR, while 90.5% of the D-VTd group showed CR or sCR (P=0.016). VRd was associated with an increased incidence of skin rash (P=0.044). Other than rashes, there were no significant differences in terms of adverse events between the two groups.
Conclusion: Our study supports the use of a front-line quadruplet induction regimen containing a CD38 monoclonal antibody for transplant-eligible patients with newly diagnosed MM.
{"title":"Advantage of achieving deep response following frontline daratumumab-VTd compared to VRd in transplant-eligible multiple myeloma: multicenter study.","authors":"Ja Min Byun, Sung-Soo Park, Sung-Soo Yoon, Ari Ahn, Myungshin Kim, Jung Yeon Lee, Young-Woo Jeon, Seung-Hwan Shin, Seung-Ah Yahng, Youngil Koh, Chang-Ki Min","doi":"10.5045/br.2023.2023005","DOIUrl":"https://doi.org/10.5045/br.2023.2023005","url":null,"abstract":"<p><strong>Background: </strong>The goal of induction therapy for multiple myeloma (MM) is to achieve adequate disease control. Current guidelines favor triplet (bortezomib-lenalidomide-dexamethasone; VRd) or quadruplet regimens (daratumumab, bortezomib-thalidomide-dexamethasone; D-VTd). In the absence of a direct comparison between two treatment regimens, we conducted this study to compare the outcomes and safety of VRd and D-VTd.</p><p><strong>Methods: </strong>Newly diagnosed MM patients aged >18 years who underwent induction therapy followed by autologous stem cell transplantation (ASCT) between November 2020 and December 2021 were identified. Finally, patients with VRd (N=37) and those with D-VTd (N=43) were enrolled.</p><p><strong>Results: </strong>After induction, 10.8% of the VRd group showed stringent complete remission (sCR), 21.6% showed complete response (CR), 35.1% showed very good partial response (VGPR), and 32.4% showed partial response (PR). Of the D-VTd group, 9.3% showed sCR, 34.9% CR, 48.8% VGPR, and 4.2% PR (VGPR or better: 67.6% in VRd vs. 93% in D-VTd, P=0.004). After ASCT, 68.6% of the VRd group showed CR or sCR, while 90.5% of the D-VTd group showed CR or sCR (P=0.016). VRd was associated with an increased incidence of skin rash (P=0.044). Other than rashes, there were no significant differences in terms of adverse events between the two groups.</p><p><strong>Conclusion: </strong>Our study supports the use of a front-line quadruplet induction regimen containing a CD38 monoclonal antibody for transplant-eligible patients with newly diagnosed MM.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a0/ef/br-58-2-83.PMC10310489.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10092905","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}
Background: With the emergence of the coronavirus disease 2019 (COVID-19) and inability of healthcare systems to control the disease, various therapeutic theories with controversial responses have been proposed. Plasmapheresis was administered as a medication. However, the knowledge of its efficacy and indications is inadequate. This study evaluated the use of plasmapheresis in critically ill patients with cancer.
Methods: This randomized clinical trial was conducted on 86 patients with malignancies, including a control group (N=41) and an intervention group (N=45) with severe COVID-19 during 2020-21. Both groups were treated with routine medications for COVID-19 management according to national guidelines, and plasmapheresis was applied to the intervention group. C-reactive protein (CRP), D-dimer, ferritin, lactate dehydrogenase, hemoglobin, and white blood cell, polymorphonuclear, lymphocyte, and platelet levels were measured at admission and at the end of plasmapheresis. Other variables included neutrophil recovery, intensive care unit admission, intubation requirements, length of hospital stay, and hospitalization outcomes.
Results: CRP(P<0.001), D-dimer (P<0.001), ferritin (P=0.039), and hemoglobin (P=0.006) levels were significantly different between the groups after the intervention. Neutrophil recovery was remarkably higher in the case than in the control group (P<0.001). However, plasmapheresis did not affect the length of hospital stay (P=0.076), which could have significantly increased survival rates (P<0.001).
Conclusion: Based on the study findings, plasmapheresis led to a significant improvement in laboratory markers and survival rate in patients with severe COVID-19. These findings reinforce the value of plasmapheresis in cancer patients as a critical population suffering from neutropenia and insufficient immune responses.
{"title":"Efficacy of plasmapheresis in neutropenic patients suffering from cytokine storm because of severe COVID-19 infection.","authors":"Alireza Sadeghi, Somayeh Sadeghi, Mohammad Saleh Peikar, Maryam Yazdi, Mehran Sharifi, Safie Ghafel, Farzin Khorvash, Behrooz Ataei, Mohammad Reza Safavi, Elahe Nasri","doi":"10.5045/br.2023.2022201","DOIUrl":"https://doi.org/10.5045/br.2023.2022201","url":null,"abstract":"<p><strong>Background: </strong>With the emergence of the coronavirus disease 2019 (COVID-19) and inability of healthcare systems to control the disease, various therapeutic theories with controversial responses have been proposed. Plasmapheresis was administered as a medication. However, the knowledge of its efficacy and indications is inadequate. This study evaluated the use of plasmapheresis in critically ill patients with cancer.</p><p><strong>Methods: </strong>This randomized clinical trial was conducted on 86 patients with malignancies, including a control group (N=41) and an intervention group (N=45) with severe COVID-19 during 2020-21. Both groups were treated with routine medications for COVID-19 management according to national guidelines, and plasmapheresis was applied to the intervention group. C-reactive protein (CRP), D-dimer, ferritin, lactate dehydrogenase, hemoglobin, and white blood cell, polymorphonuclear, lymphocyte, and platelet levels were measured at admission and at the end of plasmapheresis. Other variables included neutrophil recovery, intensive care unit admission, intubation requirements, length of hospital stay, and hospitalization outcomes.</p><p><strong>Results: </strong>CR<i>P</i>(<i>P</i><0.001), D-dimer (<i>P</i><0.001), ferritin (<i>P</i>=0.039), and hemoglobin (<i>P</i>=0.006) levels were significantly different between the groups after the intervention. Neutrophil recovery was remarkably higher in the case than in the control group (<i>P</i><0.001). However, plasmapheresis did not affect the length of hospital stay (P=0.076), which could have significantly increased survival rates (<i>P</i><0.001).</p><p><strong>Conclusion: </strong>Based on the study findings, plasmapheresis led to a significant improvement in laboratory markers and survival rate in patients with severe COVID-19. These findings reinforce the value of plasmapheresis in cancer patients as a critical population suffering from neutropenia and insufficient immune responses.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/9c/b7/br-58-2-91.PMC10310491.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10110839","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}
Sua Noh, Sang A Kim, Ji Yun Lee, Jeong Ok Lee, Soo Mee Bang
Fig. 1. Clinical course and changes in laboratory findings. With steroid treatment plus plasmapheresis, the patient’s hemoglobin levels and platelet counts recovered rapidly. Renal biopsy was performed during the administration of high-dose steroids. Fig. 2. Light microscopy of the kidney biopsy. (A) Endothelial swelling and some intraglomerular thrombi were observed on H&E-stained specimens (×500). (B) Segmental occlusion of the glomerular capillary lumen and thickening of the shrunken capillary wall with double contours were observed using PAS staining (×400). A case report of thrombotic thrombocytopenic purpura-like syndrome after Coronavirus disease 2019 vaccination
{"title":"A case report of thrombotic thrombocytopenic purpura-like syndrome after Coronavirus disease 2019 vaccination.","authors":"Sua Noh, Sang A Kim, Ji Yun Lee, Jeong Ok Lee, Soo Mee Bang","doi":"10.5045/br.2023.2023036","DOIUrl":"https://doi.org/10.5045/br.2023.2023036","url":null,"abstract":"Fig. 1. Clinical course and changes in laboratory findings. With steroid treatment plus plasmapheresis, the patient’s hemoglobin levels and platelet counts recovered rapidly. Renal biopsy was performed during the administration of high-dose steroids. Fig. 2. Light microscopy of the kidney biopsy. (A) Endothelial swelling and some intraglomerular thrombi were observed on H&E-stained specimens (×500). (B) Segmental occlusion of the glomerular capillary lumen and thickening of the shrunken capillary wall with double contours were observed using PAS staining (×400). A case report of thrombotic thrombocytopenic purpura-like syndrome after Coronavirus disease 2019 vaccination","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e2/39/br-58-2-116.PMC10310487.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10111926","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}
TO THE EDITOR: Early T-cell precursor acute lymphoblastic leukemia (ETP-ALL) arises from early thymic progenitor cells that migrate from the bone marrow to the thymus and have the potential to differentiate into myeloid/dendritic or T cells. Gene expression profiling has revealed that ETP cells share similarities with hematopoietic stem cells and myeloid progenitor cells. ETP leukemic cells do not express CD1a, CD8, and CD5 (negative to dim). Instead, these cells express ≥1 stem cell/myeloid markers. However, in addition to other ETP-ALL diagnostic criteria, near-ETP-ALL usually shows brighter CD5 [1]. In the literature, the data on ETP-ALL varies markedly, ranging from outcomes poorer than those of other T-ALL to comparable outcomes, with complete remission (CR) rates ranging from 70% to more than 90% [2-5]. Combination chemotherapy is the mainstay treatment. ETP-ALL represents a high-risk subtype of ALL. These outcomes highlight the need for alternative therapeutic approaches that are prognosis-based, and ideally, aiming for minimal residual disease (MRD)negative remission, which may help in preventing relapse. Here, we present the results of a retrospective study on ETP/near-ETP-ALL in the Department of Hemato-Oncology of a dedicated cancer hospital in North India.
{"title":"End of induction MRD assessment based early treatment intensification with novel agents in ETP-ALL- may be the way forward.","authors":"Pritish Chandra Patra, Sujay Rainchwar, Reema Singh, Rohan Halder, Pallavi Mehta, Megha Verma, Rayaz Ahmed, Jyoti Shankar Raichaudhuri, Dinesh Bhurani, Narendra Agrawal, Suman Pramanik","doi":"10.5045/br.2023.2022241","DOIUrl":"https://doi.org/10.5045/br.2023.2022241","url":null,"abstract":"TO THE EDITOR: Early T-cell precursor acute lymphoblastic leukemia (ETP-ALL) arises from early thymic progenitor cells that migrate from the bone marrow to the thymus and have the potential to differentiate into myeloid/dendritic or T cells. Gene expression profiling has revealed that ETP cells share similarities with hematopoietic stem cells and myeloid progenitor cells. ETP leukemic cells do not express CD1a, CD8, and CD5 (negative to dim). Instead, these cells express ≥1 stem cell/myeloid markers. However, in addition to other ETP-ALL diagnostic criteria, near-ETP-ALL usually shows brighter CD5 [1]. In the literature, the data on ETP-ALL varies markedly, ranging from outcomes poorer than those of other T-ALL to comparable outcomes, with complete remission (CR) rates ranging from 70% to more than 90% [2-5]. Combination chemotherapy is the mainstay treatment. ETP-ALL represents a high-risk subtype of ALL. These outcomes highlight the need for alternative therapeutic approaches that are prognosis-based, and ideally, aiming for minimal residual disease (MRD)negative remission, which may help in preventing relapse. Here, we present the results of a retrospective study on ETP/near-ETP-ALL in the Department of Hemato-Oncology of a dedicated cancer hospital in North India.","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/7a/7a/br-58-2-112.PMC10310492.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10110838","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}
REFERENCES 1. Munker R, Labopin M, Esteve J, Schmid C, Mohty M, Nagler A. Mixed phenotype acute leukemia: outcomes with allogeneic stem cell transplantation. A retrospective study from the Acute Leukemia Working Party of the EBMT. Haematologica 2017; 102:2134-40. 2. Shimoni A. Relapse of acute leukemia after a second allogeneic stemcell transplantation; is there any hope for cure? Bone Marrow Transplant 2022;57:336-7. 3. DiNardo CD, Jonas BA, Pullarkat V, et al. Azacitidine and venetoclax in previously untreated acute myeloid leukemia. N Engl J Med 2020;383:617‐29. 4. Schuler E, Wagner-Drouet EM, Ajib S, et al. Treatment of myeloid malignancies relapsing after allogeneic hematopoietic stem cell transplantation with venetoclax and hypomethylating agents-a retrospective multicenter analysis on behalf of the German Cooperative Transplant Study Group. Ann Hematol 2021;100:959‐68. 5. Qasrawi A, Gomes V, Chacko CA, et al. Acute undifferentiated leukemia: data on incidence and outcomes from a large population-based database. Leuk Res 2020;89:106301. 6. Pan R, Hogdal LJ, Benito JM, et al. Selective BCL-2 inhibition by ABT-199 causes on-target cell death in acute myeloid leukemia. Cancer Discov 2014;4:362‐75. 7. Fleischmann M, Scholl S, Frietsch JJ, et al. Clinical experience with venetoclax in patients with newly diagnosed, relapsed, or refractory acute myeloid leukemia. J Cancer Res Clin Oncol 2022;148:3191‐202. 8. Pratz KW, Jonas BA, Pullarkat V, et al. Measurable residual disease response and prognosis in treatment-naïve acute myeloid leukemia with venetoclax and azacitidine. J Clin Oncol 2022; 40:855‐65. 9. Wang N, He J, Liu F. Venetoclax in combination with hypomethylating agents for the treatment of treatment-naive B/myeloid mixed-phenotype acute leukemia and relapsed/refractory acute myeloid leukemia: a report of 3 cases. Chemotherapy 2022;67:178-82. 10. Klocke H, Dong ZM, O'Brien C, et al. Venetoclax and decitabine for T/myeloid mixed-phenotype acute leukemia not otherwise specified (MPAL NOS). Case Rep Hematol 2020;2020:8811673. 11. Zhao P, Ni M, Ma D, et al. Venetoclax plus azacitidine and donor lymphocyte infusion in treating acute myeloid leukemia patients who relapse after allogeneic hematopoietic stem cell transplantation. Ann Hematol 2022;101:119‐30. 12. Lasica M, Anderson MA. Review of venetoclax in CLL, AML and multiple myeloma. J Pers Med 2021;11:463. 13. Mei M, Aldoss I, Marcucci G, Pullarkat V. Hypomethylating agents in combination with venetoclax for acute myeloid leukemia: update on clinical trial data and practical considerations for use. Am J Hematol 2019;94:358-62. 14. Rausch CR, DiNardo CD, Maiti A, et al. Venetoclax dosing in combination with antifungal agents: real world experience in patients with acute myeloid leukemia. Blood (ASH Annual Meeting Abstracts) 2019;134(Suppl 1):2640.
{"title":"An alternative approach to confirm mixed lineage involvement in acute leukemia with <i>KMT2A</i> rearrangement-an illustrative report.","authors":"Debadrita Ray, Sreejesh Sreedharanunni, Anand Balakrishnan, Praveen Sharma, Nabhajit Mallik, Srinivasan Peyam, Man Updesh Singh Sachdeva","doi":"10.5045/br.2023.2023084","DOIUrl":"https://doi.org/10.5045/br.2023.2023084","url":null,"abstract":"REFERENCES 1. Munker R, Labopin M, Esteve J, Schmid C, Mohty M, Nagler A. Mixed phenotype acute leukemia: outcomes with allogeneic stem cell transplantation. A retrospective study from the Acute Leukemia Working Party of the EBMT. Haematologica 2017; 102:2134-40. 2. Shimoni A. Relapse of acute leukemia after a second allogeneic stemcell transplantation; is there any hope for cure? Bone Marrow Transplant 2022;57:336-7. 3. DiNardo CD, Jonas BA, Pullarkat V, et al. Azacitidine and venetoclax in previously untreated acute myeloid leukemia. N Engl J Med 2020;383:617‐29. 4. Schuler E, Wagner-Drouet EM, Ajib S, et al. Treatment of myeloid malignancies relapsing after allogeneic hematopoietic stem cell transplantation with venetoclax and hypomethylating agents-a retrospective multicenter analysis on behalf of the German Cooperative Transplant Study Group. Ann Hematol 2021;100:959‐68. 5. Qasrawi A, Gomes V, Chacko CA, et al. Acute undifferentiated leukemia: data on incidence and outcomes from a large population-based database. Leuk Res 2020;89:106301. 6. Pan R, Hogdal LJ, Benito JM, et al. Selective BCL-2 inhibition by ABT-199 causes on-target cell death in acute myeloid leukemia. Cancer Discov 2014;4:362‐75. 7. Fleischmann M, Scholl S, Frietsch JJ, et al. Clinical experience with venetoclax in patients with newly diagnosed, relapsed, or refractory acute myeloid leukemia. J Cancer Res Clin Oncol 2022;148:3191‐202. 8. Pratz KW, Jonas BA, Pullarkat V, et al. Measurable residual disease response and prognosis in treatment-naïve acute myeloid leukemia with venetoclax and azacitidine. J Clin Oncol 2022; 40:855‐65. 9. Wang N, He J, Liu F. Venetoclax in combination with hypomethylating agents for the treatment of treatment-naive B/myeloid mixed-phenotype acute leukemia and relapsed/refractory acute myeloid leukemia: a report of 3 cases. Chemotherapy 2022;67:178-82. 10. Klocke H, Dong ZM, O'Brien C, et al. Venetoclax and decitabine for T/myeloid mixed-phenotype acute leukemia not otherwise specified (MPAL NOS). Case Rep Hematol 2020;2020:8811673. 11. Zhao P, Ni M, Ma D, et al. Venetoclax plus azacitidine and donor lymphocyte infusion in treating acute myeloid leukemia patients who relapse after allogeneic hematopoietic stem cell transplantation. Ann Hematol 2022;101:119‐30. 12. Lasica M, Anderson MA. Review of venetoclax in CLL, AML and multiple myeloma. J Pers Med 2021;11:463. 13. Mei M, Aldoss I, Marcucci G, Pullarkat V. Hypomethylating agents in combination with venetoclax for acute myeloid leukemia: update on clinical trial data and practical considerations for use. Am J Hematol 2019;94:358-62. 14. Rausch CR, DiNardo CD, Maiti A, et al. Venetoclax dosing in combination with antifungal agents: real world experience in patients with acute myeloid leukemia. Blood (ASH Annual Meeting Abstracts) 2019;134(Suppl 1):2640.","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/50/e0/br-58-2-120.PMC10310490.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9791403","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 : 2023-06-30Epub Date: 2023-04-07DOI: 10.5045/br.2023.2022238
Sena Kim, Sora Lim, Farnaz Razmkhah, Jaebok Choi
{"title":"Overexpression of S100A9 in donor T cells is associated with reconstitution of gut microbiota and outcome of allogeneic hematopoietic stem cell transplantation.","authors":"Sena Kim, Sora Lim, Farnaz Razmkhah, Jaebok Choi","doi":"10.5045/br.2023.2022238","DOIUrl":"10.5045/br.2023.2022238","url":null,"abstract":"","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2023-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/76/68/br-58-2-105.PMC10310486.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10109762","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}
Suresh Hanagavadi, Priyanka Indoria, K S Rajashekar, Rajat Hegde, Smita Hegde, Sujayendra Kulkarni, Chanda Varshini Sindhiya, Vardendra Kulkarni, Suyamindra S Kulkarni, Pramod B Gai
This article has been withdrawn as the request of the author(s) and/or Editors. The Publisher apologizes for any inconvenience this may cause.
应作者和/或编辑的要求,本文已被撤回。对于由此造成的任何不便,出版商深表歉意。
{"title":"WITHDRAWN: Mutational analysis of the <i>F8</i> gene and phenotypic characterization of Hemophilia A.","authors":"Suresh Hanagavadi, Priyanka Indoria, K S Rajashekar, Rajat Hegde, Smita Hegde, Sujayendra Kulkarni, Chanda Varshini Sindhiya, Vardendra Kulkarni, Suyamindra S Kulkarni, Pramod B Gai","doi":"10.5045/br.2023.2022237","DOIUrl":"https://doi.org/10.5045/br.2023.2022237","url":null,"abstract":"<p><p>This article has been withdrawn as the request of the author(s) and/or Editors. The Publisher apologizes for any inconvenience this may cause.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-06-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9675490","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}
The prognosis of patients with chronic phase (CP) chronic myeloid leukemia (CML) has significantly improved due to the development of potent BCR::ABL1 tyrosine kinase inhibitors (TKIs). However, approximately 15‒20% of patients ultimately experience treatment failure due to resistance or intolerance to TKI therapy. As the prognosis of patients in whom multiple TKIs fail remains poor, an optimal therapeutic approach is required to treat the condition. Asciminib, an allosteric inhibitor that targets ABL1 myristoyl pocket, has been approved by the Food and Drug Administration for use in patients with CP-CML resistant or intolerant to ≥2 prior TKIs or those with T315I mutation. In a phase 1 trial, asciminib monotherapy showed a relatively favorable safety profile and potent efficacy in patients with and without the T315I mutation. In a subsequent phase 3 trial, asciminib treatment was associated with a significantly higher major molecular response rate and lower discontinuation rate than bosutinib in patients with CP-CML for whom two previous TKIs failed. Several clinical trials are being performed in various clinical settings to evaluate the role of asciminib as a frontline treatment for newly diagnosed CP-CML, either as a single agent or in combination with other TKIs as a second-line or additive treatment to improve treatment-free or deep remission. This review summarizes the incidence, available therapies, and outcomes of patients with CP-CML who experienced treatment failure, the mechanism of action, preclinical and clinical data, and ongoing trials for asciminib.
{"title":"Asciminib: the first-in-class allosteric inhibitor of BCR::ABL1 kinase.","authors":"Eun-Ji Choi","doi":"10.5045/br.2023.2023017","DOIUrl":"https://doi.org/10.5045/br.2023.2023017","url":null,"abstract":"<p><p>The prognosis of patients with chronic phase (CP) chronic myeloid leukemia (CML) has significantly improved due to the development of potent BCR::ABL1 tyrosine kinase inhibitors (TKIs). However, approximately 15‒20% of patients ultimately experience treatment failure due to resistance or intolerance to TKI therapy. As the prognosis of patients in whom multiple TKIs fail remains poor, an optimal therapeutic approach is required to treat the condition. Asciminib, an allosteric inhibitor that targets ABL1 myristoyl pocket, has been approved by the Food and Drug Administration for use in patients with CP-CML resistant or intolerant to ≥2 prior TKIs or those with T315I mutation. In a phase 1 trial, asciminib monotherapy showed a relatively favorable safety profile and potent efficacy in patients with and without the T315I mutation. In a subsequent phase 3 trial, asciminib treatment was associated with a significantly higher major molecular response rate and lower discontinuation rate than bosutinib in patients with CP-CML for whom two previous TKIs failed. Several clinical trials are being performed in various clinical settings to evaluate the role of asciminib as a frontline treatment for newly diagnosed CP-CML, either as a single agent or in combination with other TKIs as a second-line or additive treatment to improve treatment-free or deep remission. This review summarizes the incidence, available therapies, and outcomes of patients with CP-CML who experienced treatment failure, the mechanism of action, preclinical and clinical data, and ongoing trials for asciminib.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/2f/45/br-58-s1-s29.PMC10133857.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9455553","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}
Chimeric antigen receptor (CAR) T-cell therapy presents a revolutionary advancement in personalized cancer treatment. During the production process, the patient's own T-cells are genetically engineered to express a synthetic receptor that binds to a tumor antigen. CAR T-cells are then expanded for clinical use and infused back into the patient's body to attack cancer cells. Although CAR T-cell therapy is considered a major breakthrough in cancer immunotherapy, it is not without limitations. In this review, we discuss the barriers to effective CAR T-cell therapy in Korea.
{"title":"Practical issues in CAR T-cell therapy.","authors":"Ja Min Byun","doi":"10.5045/br.2023.2023015","DOIUrl":"https://doi.org/10.5045/br.2023.2023015","url":null,"abstract":"<p><p>Chimeric antigen receptor (CAR) T-cell therapy presents a revolutionary advancement in personalized cancer treatment. During the production process, the patient's own T-cells are genetically engineered to express a synthetic receptor that binds to a tumor antigen. CAR T-cells are then expanded for clinical use and infused back into the patient's body to attack cancer cells. Although CAR T-cell therapy is considered a major breakthrough in cancer immunotherapy, it is not without limitations. In this review, we discuss the barriers to effective CAR T-cell therapy in Korea.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2023-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/3e/5f/br-58-s1-s11.PMC10133847.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9826121","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}