{"title":"b级团队:平等但不同?","authors":"Luke Pembroke","doi":"10.2478/jhp-2021-0014","DOIUrl":null,"url":null,"abstract":"Abstract As a person with haemophilia B, I have known there are differences between haemophilia A and haemophilia B and their respective treatment throughout my life – though I was shocked when I learnt about the impact inhibitors can have when it comes to bleeding. Despite being very rare, as well as difficult to manage, in a recent survey reported by Chaplin et al., many nurses had experience in managing haemophilia B inhibitors. Nurses in the survey also thought extended half-life (EHL) factor products would remain the optimal treatment for haemophilia B in 2025. Ongoing clinical trials for novel molecules like concuzimab and fitusiran signal the start of more treatment options for haemophilia B, and the development of gene therapy has focused on haemophilia B in the first instance. But the fact remains that the pharmaceutical industry has focused on developing treatments for the larger haemophilia A market. Could this have distorted perceptions around treatment? In a further ‘perception bias’ that impacts management, some nurses feel there are differences in bleeding phenotype between haemophilia A and B. Garner et al.'s paper discussing rIX-FX, suggests that treatment adherence is better in haemophilia B due to lower dosing frequency, making it an easier treatment option than for haemophilia A. The patient perception may be somewhat different. While dosing schedules in haemophilia B have been more consistent for longer, there has been less pharmacokinetic modelling in haemophilia B and, arguably, less opportunity for truly tailored treatment. Gene therapy has been shown to be more ‘successful’ for haemophilia B than haemophilia A, but emicizumab has raised questions about the need for gene therapy in haemophilia A. Having an ‘emi-equivalent’ for haemophilia B will raise the same questions and may give people haemophilia B and inhibitors an effective treatment that is as transformative as emicizumab has been in the haemophilia A population.","PeriodicalId":372940,"journal":{"name":"The Journal of Haemophilia Practice","volume":"7 1","pages":"0"},"PeriodicalIF":0.0000,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"The B-team: Equal but different?\",\"authors\":\"Luke Pembroke\",\"doi\":\"10.2478/jhp-2021-0014\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Abstract As a person with haemophilia B, I have known there are differences between haemophilia A and haemophilia B and their respective treatment throughout my life – though I was shocked when I learnt about the impact inhibitors can have when it comes to bleeding. Despite being very rare, as well as difficult to manage, in a recent survey reported by Chaplin et al., many nurses had experience in managing haemophilia B inhibitors. Nurses in the survey also thought extended half-life (EHL) factor products would remain the optimal treatment for haemophilia B in 2025. Ongoing clinical trials for novel molecules like concuzimab and fitusiran signal the start of more treatment options for haemophilia B, and the development of gene therapy has focused on haemophilia B in the first instance. But the fact remains that the pharmaceutical industry has focused on developing treatments for the larger haemophilia A market. Could this have distorted perceptions around treatment? In a further ‘perception bias’ that impacts management, some nurses feel there are differences in bleeding phenotype between haemophilia A and B. Garner et al.'s paper discussing rIX-FX, suggests that treatment adherence is better in haemophilia B due to lower dosing frequency, making it an easier treatment option than for haemophilia A. The patient perception may be somewhat different. While dosing schedules in haemophilia B have been more consistent for longer, there has been less pharmacokinetic modelling in haemophilia B and, arguably, less opportunity for truly tailored treatment. Gene therapy has been shown to be more ‘successful’ for haemophilia B than haemophilia A, but emicizumab has raised questions about the need for gene therapy in haemophilia A. Having an ‘emi-equivalent’ for haemophilia B will raise the same questions and may give people haemophilia B and inhibitors an effective treatment that is as transformative as emicizumab has been in the haemophilia A population.\",\"PeriodicalId\":372940,\"journal\":{\"name\":\"The Journal of Haemophilia Practice\",\"volume\":\"7 1\",\"pages\":\"0\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2021-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"The Journal of Haemophilia Practice\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.2478/jhp-2021-0014\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"The Journal of Haemophilia Practice","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.2478/jhp-2021-0014","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Abstract As a person with haemophilia B, I have known there are differences between haemophilia A and haemophilia B and their respective treatment throughout my life – though I was shocked when I learnt about the impact inhibitors can have when it comes to bleeding. Despite being very rare, as well as difficult to manage, in a recent survey reported by Chaplin et al., many nurses had experience in managing haemophilia B inhibitors. Nurses in the survey also thought extended half-life (EHL) factor products would remain the optimal treatment for haemophilia B in 2025. Ongoing clinical trials for novel molecules like concuzimab and fitusiran signal the start of more treatment options for haemophilia B, and the development of gene therapy has focused on haemophilia B in the first instance. But the fact remains that the pharmaceutical industry has focused on developing treatments for the larger haemophilia A market. Could this have distorted perceptions around treatment? In a further ‘perception bias’ that impacts management, some nurses feel there are differences in bleeding phenotype between haemophilia A and B. Garner et al.'s paper discussing rIX-FX, suggests that treatment adherence is better in haemophilia B due to lower dosing frequency, making it an easier treatment option than for haemophilia A. The patient perception may be somewhat different. While dosing schedules in haemophilia B have been more consistent for longer, there has been less pharmacokinetic modelling in haemophilia B and, arguably, less opportunity for truly tailored treatment. Gene therapy has been shown to be more ‘successful’ for haemophilia B than haemophilia A, but emicizumab has raised questions about the need for gene therapy in haemophilia A. Having an ‘emi-equivalent’ for haemophilia B will raise the same questions and may give people haemophilia B and inhibitors an effective treatment that is as transformative as emicizumab has been in the haemophilia A population.