在社区卫生中心使用新型镰状细胞治疗剂的结果和障碍

Anne H. Metzger, M. Anim, C. Johnson
{"title":"在社区卫生中心使用新型镰状细胞治疗剂的结果和障碍","authors":"Anne H. Metzger, M. Anim, C. Johnson","doi":"10.12974/2312-5411.2021.08.1","DOIUrl":null,"url":null,"abstract":"Sickle cell disease is genetic red blood cell disorder transmitted via an autosomal recessive mutation due to valine replacing glutamicacid on the beta globulin chain of the hemoglobin molecule. The disease impacts millions of people worldwide majority living in sub-Saharan Africa and India and impacts approximately 100,000 Americans mostly those of African descent. [2-3] In 2019, two novel treatment agents for sickle cell anemia, crizanlizumab (Adakveo) and voxelotor (Oxbryta) were approved by the United States Food and Drug Administration (US FDA) [7, 8]. Both medications offer sickle cell patients improved control of their disease by reducing sickling of the red blood cells (voxelotor) and the painful effects of vaso-occlusive crises, (crizanlizumab). We studied the effects of crizanlizumab and voxelotor on a population of patients in a sickle cell clinic. Fifty-two charts were reviewed for inclusion in the study; 12 patients were using crizanlizumab and 12 patients were using voxelotor. Eight patients met criteria for evaluation of crizanlizumab and 7 patients for voxelotor. Of all data collected, the only significant difference between baseline measures and post-therapy measures was for voxelotor and hemoglobin levels at baseline and at 3 or more months post therapy. This was a small study which reflects the experience of one clinic; sickle cell providers must continue to address the social determinants of health, psychosocial and psychological needs of their patients in addition to prescribing these novel medications. ","PeriodicalId":91541,"journal":{"name":"Journal of hematology research","volume":" ","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2021-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"1","resultStr":"{\"title\":\"Outcomes and Barriers to Use of Novel Sickle Cell Therapeutic Agents in a Community Health Center\",\"authors\":\"Anne H. Metzger, M. Anim, C. Johnson\",\"doi\":\"10.12974/2312-5411.2021.08.1\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Sickle cell disease is genetic red blood cell disorder transmitted via an autosomal recessive mutation due to valine replacing glutamicacid on the beta globulin chain of the hemoglobin molecule. The disease impacts millions of people worldwide majority living in sub-Saharan Africa and India and impacts approximately 100,000 Americans mostly those of African descent. [2-3] In 2019, two novel treatment agents for sickle cell anemia, crizanlizumab (Adakveo) and voxelotor (Oxbryta) were approved by the United States Food and Drug Administration (US FDA) [7, 8]. Both medications offer sickle cell patients improved control of their disease by reducing sickling of the red blood cells (voxelotor) and the painful effects of vaso-occlusive crises, (crizanlizumab). We studied the effects of crizanlizumab and voxelotor on a population of patients in a sickle cell clinic. Fifty-two charts were reviewed for inclusion in the study; 12 patients were using crizanlizumab and 12 patients were using voxelotor. Eight patients met criteria for evaluation of crizanlizumab and 7 patients for voxelotor. Of all data collected, the only significant difference between baseline measures and post-therapy measures was for voxelotor and hemoglobin levels at baseline and at 3 or more months post therapy. This was a small study which reflects the experience of one clinic; sickle cell providers must continue to address the social determinants of health, psychosocial and psychological needs of their patients in addition to prescribing these novel medications. \",\"PeriodicalId\":91541,\"journal\":{\"name\":\"Journal of hematology research\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2021-06-02\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"1\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of hematology research\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.12974/2312-5411.2021.08.1\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of hematology research","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.12974/2312-5411.2021.08.1","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 1

摘要

镰状细胞病是一种遗传性红细胞疾病,通过常染色体隐性突变传播,原因是缬氨酸取代了血红蛋白分子β -球蛋白链上的谷氨酸。这种疾病影响了全世界数百万人,大多数生活在撒哈拉以南非洲和印度,影响了大约10万美国人,其中大部分是非洲人后裔。[2-3] 2019年,两种新型镰状细胞性贫血治疗药物crizanlizumab (Adakveo)和voxelotor (Oxbryta)获得了美国食品和药物管理局(US FDA)的批准[7,8]。这两种药物通过减少红细胞的镰状细胞(voxelotor)和血管闭塞危像的痛苦影响(crizanlizumab),为镰状细胞患者改善疾病控制。我们研究了crizanlizumab和voxelotor对镰状细胞临床患者群体的影响。本研究审查了52个图表;12例患者使用crizanlizumab, 12例患者使用voxelotor。8例患者符合crizanlizumab的评估标准,7例患者符合voxelotor的评估标准。在收集的所有数据中,基线测量和治疗后测量之间唯一的显著差异是基线和治疗后3个月或更长时间的体素和血红蛋白水平。这是一个小型研究,反映了一个诊所的经验;镰状细胞提供者必须继续处理健康的社会决定因素,以及患者的社会心理和心理需求,除了开这些新型药物。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
Outcomes and Barriers to Use of Novel Sickle Cell Therapeutic Agents in a Community Health Center
Sickle cell disease is genetic red blood cell disorder transmitted via an autosomal recessive mutation due to valine replacing glutamicacid on the beta globulin chain of the hemoglobin molecule. The disease impacts millions of people worldwide majority living in sub-Saharan Africa and India and impacts approximately 100,000 Americans mostly those of African descent. [2-3] In 2019, two novel treatment agents for sickle cell anemia, crizanlizumab (Adakveo) and voxelotor (Oxbryta) were approved by the United States Food and Drug Administration (US FDA) [7, 8]. Both medications offer sickle cell patients improved control of their disease by reducing sickling of the red blood cells (voxelotor) and the painful effects of vaso-occlusive crises, (crizanlizumab). We studied the effects of crizanlizumab and voxelotor on a population of patients in a sickle cell clinic. Fifty-two charts were reviewed for inclusion in the study; 12 patients were using crizanlizumab and 12 patients were using voxelotor. Eight patients met criteria for evaluation of crizanlizumab and 7 patients for voxelotor. Of all data collected, the only significant difference between baseline measures and post-therapy measures was for voxelotor and hemoglobin levels at baseline and at 3 or more months post therapy. This was a small study which reflects the experience of one clinic; sickle cell providers must continue to address the social determinants of health, psychosocial and psychological needs of their patients in addition to prescribing these novel medications. 
求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
自引率
0.00%
发文量
0
期刊最新文献
New Insights into the Nature of the 5q- Deletion Syndrome Based on Quantitative Measurement of BAALC- Expressing Stem Cell Burdens Novel Simple Approach for Differentiating Concentrated or Diluted Blood Samples, Hematological Disorders and Organ Dysfunctions in Acute Care Settings-A Global Perspective Trisomy 8 is Associated with Favorable Outcome in the Patients with Myelodysplastic Syndromes Treated with Allogeneic Hematopoietic Stem Cell Transplantation Multiple Myeloma Presenting as Acute Kidney Failure Secondary to Lambda Light Chain Deposition Multicenter Study of Pyruvate Kinase Deficiency in Argentina
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1