Experimental therapy of sickle cell disease. Use of hydroxyurea.

S Charache
{"title":"Experimental therapy of sickle cell disease. Use of hydroxyurea.","authors":"S Charache","doi":"","DOIUrl":null,"url":null,"abstract":"<p><strong>Purpose: </strong>Therapy of sickle cell disease with hydroxyurea is experimental.</p><p><strong>Patients and methods: </strong>We have begun a randomized blinded clinical trial to determine its clinical utility. The efficacy of this drug is unproved and its risks, which include mutagenesis, teratogenesis and carcinogenesis, are poorly understood. These risks are explicitly stated in our consent forms. A significant number of patients who are asked to enroll refuse to enter the study. This refusal is probably because of individual variations in perception of risk and personal inconvenience, as well as differences in perception of personal benefit. We have a few hints as to which patients are more likely to produce increased amounts of fetal hemoglobin, but our findings do not indicate which patients are most likely to show a good clinical response.</p><p><strong>Results: </strong>Our study group decided not to treat patients under 18 years of age with hydroxyurea until clinical efficacy of the drug is proved in adults. We have criteria for selecting patients for entry into our ongoing study, but the criteria are based more on study design than on an estimate of present or future severity of the manifestations of sickle cell disease.</p><p><strong>Conclusions: </strong>Features of our previous study and results of the present trial may be helpful in defining indications for bone marrow transplantation in children with sickle cell disease.</p>","PeriodicalId":22558,"journal":{"name":"The American journal of pediatric hematology/oncology","volume":"16 1","pages":"62-6"},"PeriodicalIF":0.0000,"publicationDate":"1994-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"The American journal of pediatric hematology/oncology","FirstCategoryId":"1085","ListUrlMain":"","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0

Abstract

Purpose: Therapy of sickle cell disease with hydroxyurea is experimental.

Patients and methods: We have begun a randomized blinded clinical trial to determine its clinical utility. The efficacy of this drug is unproved and its risks, which include mutagenesis, teratogenesis and carcinogenesis, are poorly understood. These risks are explicitly stated in our consent forms. A significant number of patients who are asked to enroll refuse to enter the study. This refusal is probably because of individual variations in perception of risk and personal inconvenience, as well as differences in perception of personal benefit. We have a few hints as to which patients are more likely to produce increased amounts of fetal hemoglobin, but our findings do not indicate which patients are most likely to show a good clinical response.

Results: Our study group decided not to treat patients under 18 years of age with hydroxyurea until clinical efficacy of the drug is proved in adults. We have criteria for selecting patients for entry into our ongoing study, but the criteria are based more on study design than on an estimate of present or future severity of the manifestations of sickle cell disease.

Conclusions: Features of our previous study and results of the present trial may be helpful in defining indications for bone marrow transplantation in children with sickle cell disease.

镰状细胞病的实验治疗。羟基脲的使用。
目的:羟基脲治疗镰状细胞病是实验性的。患者和方法:我们已经开始了一项随机盲法临床试验,以确定其临床效用。这种药物的疗效尚未得到证实,其风险,包括诱变、致畸和致癌,也知之甚少。这些风险在我们的同意书中都有明确说明。有相当数量的患者被要求参加这项研究。这种拒绝可能是因为个体对风险和个人不便的感知存在差异,以及对个人利益的感知存在差异。我们有一些提示,哪些患者更有可能产生胎儿血红蛋白量增加,但我们的发现并没有表明哪些患者最有可能表现出良好的临床反应。结果:我们的研究组决定在证实该药物在成人中的临床疗效之前,不使用羟基脲治疗18岁以下的患者。我们有选择患者进入我们正在进行的研究的标准,但这些标准更多地基于研究设计,而不是基于对镰状细胞病目前或未来表现严重程度的估计。结论:我们先前研究的特点和本试验的结果可能有助于确定镰状细胞病儿童骨髓移植的适应症。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
自引率
0.00%
发文量
0
×
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
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
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 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信