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镰状细胞病的羟基脲疗法:低收入和中等收入环境下医疗服务提供者的知识与处方模式

Hydroxyurea therapy in sickle cell disease: knowledge and prescription patterns among care providers in a low-and middle-income setting.

作者信息

Ofakunrin Akinyemi O D, Okpe Edache Sylvanus, Olaosebikan Rasaq, Ukpoju-Ebonyi Onyeka Mary, Afolaranmi Tolulope Olumide, Kilson Dangkat Bitrus, Oguche Stephen

机构信息

Department of Paediatrics, Faculty of Clinical Sciences, University of Jos &Jos University Teaching Hospital, Jos, Nigeria.

Department of Paediatrics, Federal University of Health Sciences Teaching Hospital, Otukpo, Nigeria.

出版信息

Niger Med J. 2025 Apr 3;66(1):198-209. doi: 10.71480/nmj.v66i1.675. eCollection 2025 Jan-Feb.

DOI:10.71480/nmj.v66i1.675
PMID:40309548
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12038620/
Abstract

BACKGROUND

Hydroxyurea is a safe, effective, and well-tolerated disease-modifying therapy for patients with sickle cell disease (SCD), leading to reduced morbidity, mortality, and an improved quality of life. However, its underutilization, driven by inadequate knowledge and variability in use among SCD care providers, may limit its benefits. This study assessed the knowledge and prescription patterns of hydroxyurea among SCD care providers in Jos, Nigeria.

METHODOLOGY

A cross-sectional survey of 132 physicians managing SCD was conducted to collect data on socio-demographics, hydroxyurea knowledge, and prescription patterns using a proforma. Knowledge was assessed using 17 questions (maximum score: 17). Scores above 12 (50th percentile) were classified as "good," while scores of 12 or below were categorized as "poor." Prescription patterns were evaluated against the National Institutes of Health (2014) and British Society for Haematology (2018) guidelines. Data were analyzed using descriptive and inferential statistics.

RESULTS

Sixty-seven (50.8%) of the 132 physicians had inadequate knowledge of hydroxyurea's clinical benefits and safety. Only 35 (26.5%) were aware of available treatment guidelines, and 32 (24.2%) had ever prescribed hydroxyurea. Among prescribers, nine (28.1%) used inappropriate criteria to initiate treatment and six (18.8%) prescribed below recommended doses. The median maximum daily prescribed dose was 750mg, whereas five (15.6%) physicians did not exceed 200mg, irrespective of patient weight. Treatment guidelines were not followed by 25 (78.1%) of prescribers.

CONCLUSION

This study revealed a high prevalence of inadequate knowledge and inconsistent hydroxyurea prescription practices among SCD care providers. Targeted training is essential to enhance hydroxyurea utilization and ensure adherence to standardized treatment guidelines, ultimately improving patient outcomes.

摘要

背景

羟基脲是一种用于镰状细胞病(SCD)患者的安全、有效且耐受性良好的病情改善疗法,可降低发病率、死亡率并改善生活质量。然而,由于SCD护理提供者知识不足以及使用存在差异,导致其未得到充分利用,这可能会限制其益处。本研究评估了尼日利亚乔斯市SCD护理提供者对羟基脲的知识和处方模式。

方法

对132名管理SCD的医生进行了横断面调查,使用一份表格收集有关社会人口统计学、羟基脲知识和处方模式的数据。通过17个问题(最高分:17分)评估知识水平。得分高于12分(第50百分位数)被归类为“良好”,而得分12分及以下被归类为“差”。根据美国国立卫生研究院(2014年)和英国血液学学会(2018年)的指南评估处方模式。使用描述性和推断性统计分析数据。

结果

132名医生中有67名(50.8%)对羟基脲的临床益处和安全性知识不足。只有35名(26.5%)知晓现有治疗指南,32名(24.2%)曾开过羟基脲处方。在开处方者中,9名(28.1%)使用不适当的标准开始治疗,6名(18.8%)的处方剂量低于推荐剂量。规定的最大每日剂量中位数为750毫克,而5名(15.6%)医生无论患者体重如何,剂量都不超过200毫克。25名(78.1%)开处方者未遵循治疗指南。

结论

本研究表明,SCD护理提供者中知识不足和羟基脲处方做法不一致的情况很普遍。有针对性的培训对于提高羟基脲的利用率和确保遵循标准化治疗指南至关重要,最终可改善患者预后。

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本文引用的文献

1
Development of multi-level standards of care recommendations for sickle cell disease: Experience from SickleInAfrica.镰状细胞病多级护理建议标准的制定:来自非洲镰状细胞病项目的经验
Front Genet. 2023 Jan 12;13:1052179. doi: 10.3389/fgene.2022.1052179. eCollection 2022.
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Sickle cell disease in children: an update of the evidence in low- and middle-income settings.儿童镰状细胞病:低收入和中等收入环境下的证据更新
Arch Dis Child. 2023 Feb;108(2):108-114. doi: 10.1136/archdischild-2021-323633. Epub 2022 Jun 15.
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Level of utilization and provider-related barriers to the use of hydroxyurea in the treatment of sickle cell disease patients in Jos, North-Central Nigeria.在尼日利亚中北部乔斯,羟脲在治疗镰状细胞病患者中的使用情况及其与提供者相关的障碍。
Afr Health Sci. 2021 Jun;21(2):765-774. doi: 10.4314/ahs.v21i2.36.
4
Moderate fixed-dose hydroxyurea for primary prevention of strokes in Nigerian children with sickle cell disease: Final results of the SPIN trial.中等剂量固定剂量羟基脲用于尼日利亚镰状细胞病患儿中风的一级预防:SPIN试验的最终结果
Am J Hematol. 2020 Sep;95(9):E247-E250. doi: 10.1002/ajh.25900. Epub 2020 Jul 10.
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Effective use of hydroxyurea for sickle cell anemia in low-resource countries.在资源匮乏国家有效使用羟基脲治疗镰状细胞贫血
Curr Opin Hematol. 2020 May;27(3):172-180. doi: 10.1097/MOH.0000000000000582.
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Effectiveness and Safety of Hydroxyurea in the Treatment of Sickle Cell Anaemia Children in Jos, North Central Nigeria.羟脲在尼日利亚中北部乔斯治疗镰状细胞贫血儿童的疗效和安全性。
J Trop Pediatr. 2020 Jun 1;66(3):290-298. doi: 10.1093/tropej/fmz070.
7
Patterns of prescribing hydroxyurea for sickle cell disease patients from a central hospital, Saudi Arabia.沙特阿拉伯一家中心医院为镰状细胞病患者开具羟基脲的模式。
Hematol Rep. 2019 Feb 19;11(1):7860. doi: 10.4081/hr.2019.7860.
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Hydroxyurea for Children with Sickle Cell Anemia in Sub-Saharan Africa.在撒哈拉以南非洲,用羟脲治疗镰状细胞贫血儿童。
N Engl J Med. 2019 Jan 10;380(2):121-131. doi: 10.1056/NEJMoa1813598. Epub 2018 Dec 1.
9
Low-dose hydroxycarbamide therapy may offer similar benefit as maximum tolerated dose for children and young adults with sickle cell disease in low-middle-income settings.在中低收入环境下,低剂量羟基脲疗法对于患有镰状细胞病的儿童和年轻人可能与最大耐受剂量疗法具有相似的疗效。
F1000Res. 2018 Sep 4;7. doi: 10.12688/f1000research.14589.1. eCollection 2018.
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Guidelines for the use of hydroxycarbamide in children and adults with sickle cell disease: A British Society for Haematology Guideline.镰状细胞病儿童和成人使用羟基脲的指南:英国血液学学会指南
Br J Haematol. 2018 May;181(4):460-475. doi: 10.1111/bjh.15235. Epub 2018 May 6.