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
{"title":"emicizumab在德国无因子VIII抑制剂的严重血友病A患者中的疗效：对智能药物eDiary记录的真实数据的评估","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":null,"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":3.4000,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11607772/pdf/","citationCount":"0","resultStr":"{\"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\":null,\"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\":3.4000,\"publicationDate\":\"2024-11-29\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11607772/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Therapeutic Advances in Hematology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/20406207241295653\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Therapeutic Advances in Hematology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/20406207241295653","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/1/1 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
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.
Background: 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.
Objective: To evaluate annualised bleeding rates (ABRs) and annualised joint bleeding rates (AJBRs), using data documented in the smart medication eDiary, for PwSHA receiving emicizumab.
Design: 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.
Methods: 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.
Results: The mean calculated ABR and AJBR were 0.64 and 0.39, respectively, after initiating emicizumab. For those with documentation before starting emicizumab (n = 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 (n = 54; range: 1.0-20.8); median emicizumab dosing frequency was every 11.2 days (N = 97; range: 6.6-29.4).
Conclusion: 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.
期刊介绍：
Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
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