Therapeutic Advances in Hematology最新文献

{"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}

{"title":"Using T-lymphocyte subsets at engraftment to predict the risk of acute graft-versus-host disease in patients with thalassemia major: development of a new predictive nomogram.","authors":"Hongwen Xiao, Gaohui Yang, Qiulin Huang, Zhenbin Wei, Zhaoping Gan, Meiqing Wu, Zeyan Shi, Huicheng Huang, Zhaofang Pan, Lianjin Liu, Lingling Shi, Zhongming Zhang, Rongrong Liu, Yongrong Lai","doi":"10.1177/20406207241294054","DOIUrl":"10.1177/20406207241294054","url":null,"abstract":"<p><strong>Background: </strong>Acute graft-versus-host disease (aGvHD) is the primary cause of mortality following allogeneic hematopoietic cell transplantation (HCT).</p><p><strong>Objectives: </strong>This study aimed to predict the risk of aGvHD after HCT in patients with thalassemia major using a novel predictive nomogram.</p><p><strong>Design: </strong>A retrospective study was used to develop the prediction model.</p><p><strong>Methods: </strong>We performed retrospective analyses on 402 consecutive thalassemia patients who underwent HCT. Risk factors for aGvHD were analyzed using Cox proportional regression models. T-lymphocyte subsets were collected from 240 patients at the time of neutrophil engraftment. Least Absolute Shrinkage and Selection Operator regression was utilized to screen the indices, with cut-off values established through restricted cubic spline (RCS) regression. The predictive model was developed by integrating these T-lymphocyte subsets with clinical features, aiming to enhance the accuracy of aGvHD risk prediction.</p><p><strong>Results: </strong>Among 402 thalassemia patients analyzed post-transplantation, significant independent risk factors for aGvHD included matched unrelated donors, haploid-related donors, peripheral blood stem cell infusions, and donor age older than 40 years. Our RCS analysis indicated a marked increase in aGvHD risk when CD4+ T-cell counts exceeded 36 cells/μL and CD8+ T-cell counts exceeded 43 cells/μL during neutrophil engraftment. The integration of T-lymphocyte subsets with clinical risk factors into a Cox regression model demonstrated good predictive performance for assessing aGvHD risk.</p><p><strong>Conclusion: </strong>This study presents a novel model designed to predict aGvHD in thalassemia patients post-transplantation by utilizing T-lymphocyte data at the time of engraftment. The model facilitates the creation of personalized treatment plans, aiming to minimize the incidence of aGvHD and improve patient outcomes.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241294054"},"PeriodicalIF":3.4,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11632902/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142814168","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":"Efficacy and safety of avatrombopag in the treatment of chemotherapy-induced thrombocytopenia in children with acute lymphoblastic leukemia: a single-center retrospective study.","authors":"Huiyan Yang, Jingyu Gao, Yongsheng Ruan, Zhaokun Chen, Ruihan Fang, Lei Zhang, Zhibiao Wang, Tiantian Yi, Qian Zhang, Yang Luo, Libai Chen, Xuedong Wu","doi":"10.1177/20406207241304300","DOIUrl":"10.1177/20406207241304300","url":null,"abstract":"<p><strong>Background: </strong>Chemotherapy-induced thrombocytopenia (CIT) commonly exacerbates the difficulty of cancer treatment, increasing bleeding risks and potentially reducing chemotherapy dosage, ultimately impacting its efficacy. However, there are limited studies about avatrombopag application in acute lymphoblastic leukemia (ALL) CIT.</p><p><strong>Objectives: </strong>We aimed to evaluate the efficacy and safety of avatrombopag in treating CIT patients diagnosed with ALL.</p><p><strong>Design: </strong>This retrospective study, using propensity score matching, included 42 pairs of cases treated with and without avatrombopag (CAT: 54 cases, CAT+: 30 cases).</p><p><strong>Methods: </strong>Data of CIT-ALL children were retrospectively collected. The primary endpoint was platelet count (PC) response rate on day 10 ± 2 (defined as an increase of PC to ⩾75 × 10⁹/L with the exclusion of platelet transfusion). Secondary efficacy endpoints, safety endpoints, and factors that predict PC response were also analyzed.</p><p><strong>Results: </strong>In the avatrombopag group, the PC response rate was prominently higher on day 10 ± 2 (89.1%) versus the control group (56.4%, <i>p</i> = 0.005). On day 10 ± 2, the difference in median PC change from baseline was predominantly distinct in the avatrombopag group compared to the control group (<i>p</i> = 0.001). In the avatrombopag group, platelet recovery to ⩾25 and ⩾50 × 10⁹/L was faster (<i>p</i> = 0.001, <i>p</i> = 0.002), and quicker platelet reaching ⩾75 × 10⁹/L and ⩾100 × 10⁹/L was achieved (<i>p</i> = 0.023, <i>p</i> = 0.011). The avatrombopag group not only increased the nadir PC (<i>p</i> = 0.009) but also reduced the total platelet transfusion compared to the control group (<i>p</i> = 0.047). Only one case (2.4%) experienced bleeding events after medication. Nine cases of secondary thrombocythemia were noted without other adverse events. There was no difference in event-free survival between the two groups (<i>p</i> = 0.648). Drug administration was prediction factor for PC response.</p><p><strong>Conclusion: </strong>Avatrombopag is a potentially safe and effective treatment option for CIT in pediatric ALL.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241304300"},"PeriodicalIF":3.4,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11622298/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142802235","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":"Design of an international, phase IV, open-label study of simoctocog alfa in women/girls with hemophilia A undergoing surgery (NuDIMENSION).","authors":"Natascha Marquardt, Florian Langer, Katharina Holstein, María Teresa Álvarez Román, Ramiro Núñez Vázquez, Predrag Miljić, Nicolas Drillaud, Laurent Ardillon, Anna-Elina Lehtinen, Rita Carlotta Santoro, Mariasanta Napolitano, Sergio Siragusa, Gillian Gidley, Martina Jansen, Sigurd Knaub, Johannes Oldenburg","doi":"10.1177/20406207241300040","DOIUrl":"https://doi.org/10.1177/20406207241300040","url":null,"abstract":"<p><strong>Background: </strong>Although hemophilia A mainly affects males, carriers (defined as females with hemophilia A, as well as symptomatic or asymptomatic hemophilia A carriers) are at risk of excessive bleeding, particularly during trauma or during surgical procedures. Clinical trials have focused on male patients with severe disease, and data for females are limited. Improved, evidence-based treatment guidelines for management of hemophilia A carriers are required.</p><p><strong>Objectives and design: </strong>The NuDIMENSION study is a phase IV, prospective, open-label, single-arm study that will evaluate the perioperative efficacy and safety of simoctocog alfa (Nuwiq^®), a recombinant factor VIII (FVIII), in women/girls with hemophilia A undergoing major surgery. The study will be conducted at approximately 15 centers worldwide. Women/girls aged ⩾12 years, with mild or moderate hemophilia A (residual FVIII activity (FVIII:C) ⩾1% to <40%) and with no current/past FVIII inhibitors are eligible. All patients must be scheduled to undergo a major surgical procedure during which simoctocog alfa will be administered.</p><p><strong>Methods and analysis: </strong>The primary endpoint is overall perioperative hemostatic efficacy (\"success\" or \"failure\") of simoctocog alfa. Hemostatic efficacy will be assessed at the end of surgery and at the end of the postoperative period (i.e., completion of wound healing), with overall adjudication by an Independent Data Monitoring Committee. Safety endpoints will include the incidences of thrombotic events and FVIII inhibitor development. The aim is to recruit 28 patients to achieve 26 evaluable surgeries.</p><p><strong>Ethics: </strong>Ethical approval will be received from institutional review boards/independent ethics committees, and the study will be conducted in compliance with the Declaration of Helsinki.</p><p><strong>Discussion: </strong>Data from NuDIMENSION will generate much-needed evidence on surgical management of women/girls with hemophilia A, which will help to enable the development of treatment guidelines specific for such patients.</p><p><strong>Trial registration: </strong>CT EU 2022-502061-17-00; NCT05936580.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241300040"},"PeriodicalIF":3.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11610030/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142772625","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":"Efficacy of emicizumab in patients with severe haemophilia A without factor VIII inhibitors in Germany: evaluation of real-life data documented by the smart medication eDiary.","authors":"Carmen Escuriola Ettingshausen, Wolfgang Eberl, Hermann Eichler, Ronald Fischer, Christina Hart, Katharina Holstein, Ralf Knöfler, Jürgen Kreutz, Caspar David Kühnöl, Wolfgang A Miesbach, Christian Pfrepper, Andreas Rösch, Ulrich J Sachs, Karolin Trautmann-Grill, Wolfgang Mondorf","doi":"10.1177/20406207241295653","DOIUrl":"https://doi.org/10.1177/20406207241295653","url":null,"abstract":"<p><strong>Background: </strong>Systematically documented data on real-world use of emicizumab, a bispecific antibody factor (F)VIII mimetic, are still lacking in people with severe haemophilia A (PwSHA). Smart medication, a real-time, online platform, monitors treatment administration and outcomes for people with haemophilia A in Germany.</p><p><strong>Objective: </strong>To evaluate annualised bleeding rates (ABRs) and annualised joint bleeding rates (AJBRs), using data documented in the smart medication eDiary, for PwSHA receiving emicizumab.</p><p><strong>Design: </strong>Data for 97 PwSHA without FVIII inhibitors who started emicizumab treatment between 1 January 2018 and 31 March 2023, with >24 weeks of documentation after switching from FVIII replacement, were collected in the smart medication eDiary. Those with ⩾24 weeks of pre-emicizumab data were included for analysis 24 weeks before and after switching.</p><p><strong>Methods: </strong>The primary objective was to evaluate ABR and AJBR for treated bleeds. The proportion of bleed-free participants was calculated and administration frequency for FVIII and emicizumab were collected. The mean dosing frequencies for FVIII replacement and emicizumab were also evaluated.</p><p><strong>Results: </strong>The mean calculated ABR and AJBR were 0.64 and 0.39, respectively, after initiating emicizumab. For those with documentation before starting emicizumab (<i>n</i> = 58), ABR decreased by 79.6% and AJBR decreased by 90.8%. The proportion of bleed-free participants increased by 21.3%, and joint bleed-free participants increased by 18.2%. The median FVIII dosing frequency was every 3.5 days (<i>n</i> = 54; range: 1.0-20.8); median emicizumab dosing frequency was every 11.2 days (<i>N</i> = 97; range: 6.6-29.4).</p><p><strong>Conclusion: </strong>Real-world data collected using the smart medication eDiary provide insights into efficacy outcomes after switching from FVIII replacement to emicizumab prophylaxis. Bleeds, including joint bleeds, decreased after switching.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"15 ","pages":"20406207241295653"},"PeriodicalIF":3.4,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11607772/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142772638","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}