Therapeutic Advances in Hematology最新文献

{"title":"Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France.","authors":"Hervé Chambost, Yohann Repessé, Fabienne Genre-Volot, Dominique Desprez, Sabine Marie Castet, Stéphane Vanderbecken, Meriem Zidi, Corinne Gandossi, Eveline Nüesch, Helena Palmborg, Elena Santagostino","doi":"10.1177/20406207241311535","DOIUrl":"10.1177/20406207241311535","url":null,"abstract":"<p><strong>Background: </strong>More real-world data are needed to complement existing phase III studies on the efficacy and safety of recombinant factor IX Fc fusion protein (rFIXFc) in people with haemophilia B.</p><p><strong>Objectives: </strong>We report final data from the B-SURE study, evaluating the real-world usage and effectiveness of rFIXFc in France.</p><p><strong>Methods: </strong>Previously treated patients (all ages/severities) received on-demand or prophylactic rFIXFc during B-SURE. Annualised bleeding rate (ABR), injection frequency (IF) and factor consumption (FC) were prospectively evaluated for patients on rFIXFc prophylaxis (primary endpoints). Six months of retrospective factor IX (FIX) data were collected for comparison; patients with ⩾3 months of treatment pre- and post-switch to rFIXFc were analysed.</p><p><strong>Design: </strong>B-SURE was a 24-month, prospective, non-interventional, real-world study across haemophilia treatment centres in France.</p><p><strong>Results: </strong>Ninety-one male patients enrolled across 21 centres (34% <18 years, 89% severe haemophilia B). Eighty-four patients received prophylaxis at rFIXFc initiation; mean prospective observation period was 21.5 months. Sixty-eight of 84 patients had prior FIX prophylaxis; on rFIXFc prophylaxis, these patients achieved low median ABR (1.2), IF (47.45 injections/year) and mean FC (2844 IU/kg/year). Compared with previous FIX, mean ABR was reduced by 40% (<i>n</i> = 63); mean IF and FC were reduced by 38.20 injections/year and 1008 IU/kg/year (<i>n</i> = 57). In patients with prior FIX on-demand (<i>n</i> = 15), mean ABR reduced by 84% on rFIXFc prophylaxis (<i>n</i> = 14), mean IF reduced by 2.13 injections/year and mean FC increased by 381.8 IU/kg/year (<i>n</i> = 15). Most physicians and patients were satisfied/highly satisfied with rFIXFc prophylaxis. rFIXFc was well tolerated with no new safety concerns.</p><p><strong>Conclusion: </strong>Findings support the safety and effectiveness of rFIXFc, with reduced IF and FC while maintaining/improving bleed protection. <b><i>Trial registration</i>:</b> NCT03655340.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"16 ","pages":"20406207241311535"},"PeriodicalIF":3.4,"publicationDate":"2025-01-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11770721/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143053726","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A nomogram based on InLDH and InNLR for predicting disseminated intravascular coagulation in patients with heat stroke.","authors":"Lulu Wan, Gan Lin, Jiale Yang, Anwei Liu, Xuezhi Shi, Jinhu Li, Lian Xie, Ronglin Chen, Huasheng Tong","doi":"10.1177/20406207241311386","DOIUrl":"10.1177/20406207241311386","url":null,"abstract":"<p><strong>Background: </strong>Heat stroke (HS), a potentially fatal heat-related illness, is often accompanied by disseminated intravascular coagulation (DIC) early, resulting in a poorer prognosis. Unfortunately, diagnosis by current DIC scores is often too late to identify DIC. This study aims to investigate the predictors and predictive model of DIC in HS to identify DIC early.</p><p><strong>Methods: </strong>This retrospective study analyzed clinical data of patients with HS in a tertiary hospital from January 1, 2008 to December 31, 2020. Univariate and multivariate logistic regression analyses were employed to identify the risk factors for DIC in HS. The predictive models based on these risk factors were constructed and externally validated, and their predictive efficacy was evaluated using receiver operating characteristic curves.</p><p><strong>Results: </strong>A total of 219 HS patients, including 49 with DIC, were included. The independent risk factors for DIC were identified as follows: neutrophil percentage (Neu%), lymphocyte count, lymphocyte percentage (Lym%), creatine kinase-MB (CKMB), lactate dehydrogenase (LDH), aspartate aminotransferase (AST), neutrophil-lymphocyte ratio (NLR), monocyte-lymphocyte ratio (MLR), and rhabdomyolysis (RM). After logarithmization, the final predictive model based on the logarithm of lactate dehydrogenase (InLDH; odds ratio (OR) = 9.266, 95% confidence interval (95%CI; 4.379-19.607), <i>p</i> < 0.0001) and the logarithm of neutrophil-lymphocyte ratio (InNLR; OR = 3.393, 95%CI (1.834-6.277), <i>p</i> < 0.0001) was constructed with the largest area under the curve (0.928). A nomogram incorporating InLDH and InNLR was developed and showed excellent discrimination and calibration capabilities.</p><p><strong>Conclusion: </strong>Nine independent risk factors were identified for the occurrence of DIC in HS patients. The predictive model based on InLDH and InNLR can effectively predict the incidence of DIC. A nomogram based on InLDH and InNLR was developed to facilitate early identification and timely treatment of DIC in HS patients.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"16 ","pages":"20406207241311386"},"PeriodicalIF":3.4,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11719444/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142972036","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic significance of MRD and its correlation with arsenic concentration in pediatric acute promyelocytic leukemia: a retrospective study by SCCLG-APL group.","authors":"Zhong Fan, Liang-Chun Yang, Yi-Qiao Chen, Wu-Qing Wan, Dun-Hua Zhou, Hui-Rong Mai, Wan-Li Li, Li-Hua Yang, He-Kui Lan, Hui-Qin Chen, Bi-Yun Guo, Zi-Jun Zhen, Ri-Yang Liu, Guo-Hua Chen, Xiao-Qin Feng, Cong Liang, Li-Na Wang, Yu-Li, Jie-Si Luo, Dan-Ping Huang, Xue-Qun Luo, Bin Li, Li-Bin Huang, Xiao-Li Zhang, Yan-Lai Tang","doi":"10.1177/20406207241311774","DOIUrl":"https://doi.org/10.1177/20406207241311774","url":null,"abstract":"<p><strong>Background: </strong>Treatment outcomes for acute promyelocytic leukemia (APL) have improved with all-trans-retinoic acid and arsenic trioxide, yet relapse remains a concern, especially in pediatric patients. The prognostic value of minimal residual disease (MRD) post-induction and the impact of arsenic levels during induction on MRD are not fully understood.</p><p><strong>Objectives: </strong>To evaluate the relationship between post-induction MRD levels and relapse-free survival (RFS) in pediatric APL patients, and to investigate the correlation between blood arsenic concentration levels during induction therapy and MRD status.</p><p><strong>Design: </strong>A retrospective analysis of pediatric APL patients enrolled in a clinical trial from September 2011 to July 2020.</p><p><strong>Methods: </strong>We assessed the relationship between RFS and post-induction MRD levels using the log-rank test. The optimal MRD cut-off was determined using the \"surv_cutpoint\" function in the survminer R package. Arsenic concentration levels were monitored in 16 patients on days 7 and 14 of induction therapy, and Spearman correlation was used to analyze the relationship between arsenic concentrations and MRD levels.</p><p><strong>Results: </strong>Among 176 pediatric APL patients, with a median follow-up of 6 years, 4 relapsed. Patients with MRD >3.1% had significantly lower RFS compared to those with MRD ⩽3.1% (94.6% vs 100%, <i>p</i> = 0.023). In addition, a negative correlation was found between blood arsenic concentration levels and post-induction MRD levels. Lower arsenic concentrations were associated with higher MRD levels, with significant correlations observed for trough concentrations (<i>R</i> = -0.666, <i>p</i> = 0.005) and peak concentrations (<i>R</i> = -0.499, <i>p</i> = 0.049) on day 7.</p><p><strong>Conclusion: </strong>Our study highlights the prognostic significance of post-induction MRD assessment in pediatric APL. We also demonstrate a negative correlation between blood arsenic concentration levels and MRD, suggesting that lower arsenic concentrations during induction therapy may contribute to a higher MRD burden. These findings may inform strategies to optimize treatment and improve outcomes in pediatric APL.<b>Trial registration:</b> www.clinicaltrials.gov (NCT02200978).</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"16 ","pages":"20406207241311774"},"PeriodicalIF":3.4,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11707783/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142955479","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Single-center experience of venetoclax combined with azacitidine in young patients with newly diagnosed acute myeloid leukemia.","authors":"Xuezhu Xu, Rui Liu, Hongli Chen, Ruoyu Yang, Gongzhizi Gao, Aili He, Fangxia Wang","doi":"10.1177/20406207241311776","DOIUrl":"https://doi.org/10.1177/20406207241311776","url":null,"abstract":"<p><strong>Background: </strong>Medical resources, especially blood products, were in short supply during the COVID-19. Less intensive therapy with hypomethylating agents/venetoclax (VEN) seems an effective treatment option for patients with acute myeloid leukemia (AML).</p><p><strong>Objectives: </strong>To retrospectively analyze the efficacy and safety of VEN combined with azacitidine (AZA) in young adult patients with newly diagnosed (ND) AML.</p><p><strong>Design: </strong>This was a retrospective study.</p><p><strong>Methods: </strong>The clinical data of 25 AML patients treated with the VEN + AZA regimen from January 2021 to December 2023 at our center were collected, compared with a randomized historical study cohort that was administered intensive chemotherapy (IC) from January 2018 to December 2019.</p><p><strong>Results: </strong>No rate of complete remission/complete remission with incomplete count recovery differences observed between the two arms reached statistical significance. Compared to traditional IC, minimal residual disease (MRD)-negative remission was achieved more quickly in patients treated with VEN + AZA regimens (after cycle 1: 8% in the IC group vs 56% in the VEN group, <i>p</i> = 0.0004; after cycle 2: 16% in the IC group vs 72% in the VEN group, <i>p</i> = 0.0001), especially in those AML patients who had a poor prognosis. The dependency of transfusion of red blood cell (RBC) and platelets during induction treatment was significantly lower in the VEN + AZA group (RBC: <i>p</i> = 0.0269; platelet: <i>p</i> = 0.0054). Compared with the standard IC, the incidence rate of non-hematological adverse events in VEN + AZA group was significantly decreased (infection: 100% vs 20%, <i>p</i> = 0.0001; gastrointestinal side effects: 48% vs 12%, <i>p</i> = 0.0055). The total hospitalization cost of the VEN group was significantly less than that of the IC group (<i>p</i> = 0.0395).</p><p><strong>Conclusion: </strong>In conclusion, our study indicated that VEN + AZA with a higher MRD-negative remission rate and less toxic appeared to be a therapy option for young patients with ND AML. However, further well-designed studies with larger numbers of patients are needed to confirm the benefits of VEN + AZA in this population.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"16 ","pages":"20406207241311776"},"PeriodicalIF":3.4,"publicationDate":"2025-01-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11705358/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142955482","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hematopoietic stem cell microtransplantation: current situation and challenges.","authors":"Zhengyang Li, Yuanyuan Yang, Hongwei Peng, Fei Li","doi":"10.1177/20406207241310332","DOIUrl":"https://doi.org/10.1177/20406207241310332","url":null,"abstract":"<p><p>Allogeneic hematopoietic stem cell transplantation (allo-HSCT) stands as a cornerstone in the treatment of hematological malignancies, recognized for its remarkable efficacy. However, the persistent challenge of graft-versus-host disease (GVHD) continues to represent a significant barrier, often being the leading cause of nonrelapse mortality after allo-HSCT. To address this limitation, hematopoietic stem cell microtransplantation (MST) has emerged as a novel therapeutic strategy that synergistically combines chemotherapy, allo-HSCT, and cellular immunotherapy. This innovative approach is designed to retain the patient's immune function, promote the establishment of microchimerism, and achieve a potent graft-versus-tumor (GVT) response, all while significantly minimizing the risk of GVHD. MST has primarily been applied in the treatment of hematological malignancies, where it has demonstrated promising outcomes, including marked improvements in complete remission rates, overall survival rates, and progression-free survival rates. Moreover, MST facilitates hematopoietic recovery, decreases the likelihood of infections, and reduces the incidence of GVHD, thus contributing to an improved quality of life for patients. A deeper and more comprehensive understanding of MST's mechanisms could enhance its clinical utility and integration into standard treatment protocols. This review aims to explore the underlying mechanisms, current clinical applications, and challenges of MST, shedding light on its potential role in advancing the management of hematological malignancies.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"16 ","pages":"20406207241310332"},"PeriodicalIF":3.4,"publicationDate":"2025-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11694307/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142932663","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Optimal use of BTK inhibitors in Waldenström's macroglobulinemia: combination or single drug approach?","authors":"Eirini Solia, Efstathios Kastritis","doi":"10.1177/20406207241308771","DOIUrl":"10.1177/20406207241308771","url":null,"abstract":"<p><p>Waldenström macroglobulinemia is an indolent B-cell lymphoma which although remains incurable, there are a lot of treatment options. Today, Bruton tyrosine kinase inhibitors have a central role in the management of the disease either as monotherapy or combination with other regimens, due to their efficacy, ease of administration, and safety profile. However, there is still active clinical investigation to further increase their efficacy and improve safety profile. Combinations based on BTK inhibitors may offer advantages. Second- and third-generation BTK inhibitors are also evaluated in combinations aiming to improve the depth of response, overcome genetic factors associated with poorer outcomes and reduce toxicity and duration of therapy.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241308771"},"PeriodicalIF":3.4,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11672393/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142903580","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Current landscape of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitors and regulators.","authors":"Julia J Shi, Yusuf M Ozcan, Carlos I Ayala Santos, Hetalkumari Patel, Jamile Shammo, Taha Bat","doi":"10.1177/20406207241307500","DOIUrl":"10.1177/20406207241307500","url":null,"abstract":"<p><p>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder which is caused by mutations in phosphatidylinositol glycan class A leading to hemolysis of red blood cells via complement inhibition. The first treatment for PNH, eculizumab, was FDA approved in 2007. Since then, many new treatment options for PNH have arisen. This critical review will examine all medications available for PNH on the US market, highlight several major medications in development, and discuss the risks and treatment considerations associated with each option. It is not intended to address PNH clonal dynamics, disease presentation, or discussions on when to initiate treatment.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241307500"},"PeriodicalIF":3.4,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11672493/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142903579","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcomes and management of invasive procedures in participants with hemophilia A post gene therapy: a post hoc analysis of the GENEr8-1 phase III trial.","authors":"Doris V Quon, Jiaan-Der Wang, Michael Wang, Dominic Pepperell, Young-Shil Park, Gili Kenet, Johnny Mahlangu, Teh-Liane Khoo, Tara M Robinson, Konstantia-Maria Chavele, Steven W Pipe","doi":"10.1177/20406207241304645","DOIUrl":"10.1177/20406207241304645","url":null,"abstract":"<p><strong>Background: </strong>Hemophilia A is caused by coagulation factor VIII (FVIII) deficiency and increases bleeding risk during invasive procedures.</p><p><strong>Objectives: </strong>To investigate FVIII concentrate use and bleeding outcomes for invasive procedures after valoctocogene roxaparvovec gene transfer.</p><p><strong>Design: </strong>This manuscript presents post hoc analysis of the phase III GENEr8-1 trial.</p><p><strong>Methods: </strong>A post hoc analysis was performed for GENEr8-1, a global, single-arm, open-label, phase III trial that enrolled 134 adults with severe hemophilia A. FVIII activity and bleeding were evaluated after 2 years of follow-up. Invasive procedures were reviewed and categorized as major or minor. FVIII activity was measured with a chromogenic assay. Bleeding was self-reported by participants. Principal investigators completed questionnaires about perioperative management.</p><p><strong>Results: </strong>In total, 111 invasive procedures were performed in 65 participants during GENEr8-1 as of the data cut. Procedures performed with FVIII treatment included 33 minor and 11 major procedures. The remaining 67 invasive procedures were minor and performed without FVIII treatment. When considering these 67 minor procedures, 43/46 investigators completing the questionnaires reported that the gene-therapy-derived FVIII activity was sufficient for the type of procedure. Minor procedures performed without FVIII treatment were associated with participants' higher mean endogenous FVIII activity (50.5 IU/dL) compared with major procedures (14.2 IU/dL) or minor procedures (16.4 IU/dL) performed with concomitant FVIII. Fourteen participants experienced 18 procedure-related bleeds (13 co-occurring with FVIII use). Participants who received FVIII treatment for procedure-related bleeds had numerically lower mean endogenous FVIII activity than those who did not receive FVIII treatment.</p><p><strong>Conclusion: </strong>Invasive procedures were safely performed in participants following treatment with valoctocogene roxaparvovec. The questionnaire responses from investigators generally suggest they used endogenous FVIII activity derived from valoctocogene roxaparvovec to inform clinical decisions in a manner comparable to exogenously administered FVIII, and more commonly prescribed supplementary FVIII concentrate in the peri-procedural period for participants with lower FVIII activity levels.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241304645"},"PeriodicalIF":3.4,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11660064/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142877875","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Demographics, clinical characteristics, and real-world treatment patterns among patients with beta-thalassemia: a retrospective medical record abstraction study.","authors":"Maria Domenica Cappellini, Mrudula B Glassberg, Juliana Meyers, Maria Jimenez, Tram Nham, Luciana Bueno, Jan Sieluk, Aylin Yucel, Ferras Alashkar","doi":"10.1177/20406207241298088","DOIUrl":"10.1177/20406207241298088","url":null,"abstract":"<p><strong>Background: </strong>Beta-thalassemias (BTs) are characterized by deficient or absent synthesis of the beta-globin subunit, leading to anemia. Patient characteristics and treatment patterns in these patients may vary.</p><p><strong>Objective: </strong>This retrospective study evaluated demographics, clinical characteristics, and treatment patterns in patients with transfusion-dependent BT (TDT) and non-transfusion-dependent BT (NTDT).</p><p><strong>Methods: </strong>Medical records of adults with TDT or NTDT in the United Kingdom, France, Germany, Spain, and Canada with ⩾5 years of history within the practice were evaluated.</p><p><strong>Results: </strong>Among patients with TDT (<i>N</i> = 118), mean (standard deviation (SD)) age was 36.1 (11.9) years, and 28.8% were female; among patients with NTDT (<i>N</i> = 96), mean (SD) age was 36.6 (9.8) years, and 38.5% were female. Among patients with TDT, 21.2% received transfusions every 2 weeks or more frequently, 28.8% every 3 weeks, 26.3% every 4 weeks, and 21.2% less frequently than 4 weeks. Patients with TDT had a mean (SD) of 2.4 (0.6) units of blood transfused per transfusion, with a pretransfusion hemoglobin (Hb) level of 6.9 (1.3). In total, 84.4% of patients with NTDT had at least one transfusion, and the mean (SD) number of transfusions among patients with NTDT was 15.9 (15.9). Among patients with NTDT, the mean (SD) units of blood per transfusion were 2.2 (0.6) units, and the mean (SD) Hb level prior to transfusion was 7.4 (1.2) g/dL. Iron chelation therapy was received by 70.3% of TDT patients and 45.8% of NTDT patients.</p><p><strong>Conclusion: </strong>This study found that both patients with NTDT and TDT have low pretransfusion Hb levels. A high number of patients, especially patients with TDT, were not treated according to the current recommendations on target hemoglobin level, thereby highlighting the importance of national reference centers for improving long-term outcomes and quality of life in these patients.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241298088"},"PeriodicalIF":3.4,"publicationDate":"2024-12-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11648021/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142839782","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mechanisms of resistance to histone deacetylase inhibitors in acute leukemia.","authors":"Mohammad Amin Akbarzadeh, Yosra Vaez-Gharamaleki, Mohammad-Salar Hosseini","doi":"10.1177/20406207241306553","DOIUrl":"10.1177/20406207241306553","url":null,"abstract":"","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241306553"},"PeriodicalIF":3.4,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11635846/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142819146","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}