Drug therapy in patients with severe forms of sickle cell anemia: A nonrandomized clinical trial of combining l-carnitine with hydroxycarbamide therapy

Q4 Medicine

Journal of Applied Hematology Pub Date : 2022-10-01 DOI:10.4103/joah.joah_23_22

Safaa A. Khaled, Israa M. Ashry

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引用次数: 3

Abstract

BACKGROUND AND AIM: Combining Hydroxycarbamide (hydroxyurea) therapy and L-Carnitine (HU+LC) was proved effective and tolerable in patients with thalassemia intermedia; however, its role in sickle cell anemia (SCA) was not investigated. This study aimed to assess various aspects of treatment with HU + LC in adult patients with severe forms of SCA. PATIENTS AND METHODS: Ninety-one adult SCA patients were recruited, then distributed into four treatment groups: Group 1 – 37 patients treated with HU + LC; Group 2 – 16 patients treated with HU; Group 3 – 18 patients treated with LC; and Group 4 – 20 patients received supportive treatment only. Their baseline data were recorded then reassessed 8–12 weeks later. Their compliance was assessed subjectively. RESULTS: Patients were nearly matched as regards gender, age, baseline hematologic, and biochemical data. Compared to baseline, HU + LC treatment significantly increased hemoglobin and reduced reticulocytes, white blood cells, mean corpuscular hemoglobin concentration, and lactate dehydrogenase (LDH). Compared with other lines, HU + LC significantly reduced number of painful episodes, blood transfusions, and in hospital days per year. Adding LC to HU improved patient compliance, with mild side effects. Residence, side effects, baseline hemoglobin (Hb), and Hb F% affected patients' compliance. Both gender and age did not show any influence on response to treatment while side effects, compliance, baseline Hb, and LDH displayed a significant effect. CONCLUSION: Combining LC with HU for the treatment of adult SCA patients showed superior hematologic and clinical effectiveness. The response was more obvious in severe cases. However, health education about the combination is needed to improve patients' compliance and therapeutic responses. Trial registration: Clinical Trials.gov, NCT05081349. https://clinicaltrials.gov/ct2/show/NCT05081349.

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严重镰状细胞性贫血患者的药物治疗：左旋肉碱与羟胺联合治疗的非随机临床试验

背景与目的：羟基脲联合左旋肉碱治疗中间型地中海贫血疗效确切，耐受性好；然而，它在镰状细胞性贫血（SCA）中的作用尚未得到研究。本研究旨在评估成年严重SCA患者HU+LC治疗的各个方面。患者和方法：招募91名成年SCA患者，然后分为四个治疗组：第1组-37名接受HU+LC治疗的患者；第2-16组患者接受HU治疗；第3-18组患者接受LC治疗；第4-20组患者仅接受支持性治疗。他们的基线数据被记录下来，然后在8-12周后重新评估。他们的依从性是主观评估的。结果：患者在性别、年龄、基线血液学和生化数据方面几乎匹配。与基线相比，HU+LC治疗显著增加了血红蛋白，减少了网织红细胞、白细胞、平均红细胞血红蛋白浓度和乳酸脱氢酶（LDH）。与其他线相比，HU+LC显著减少了疼痛发作次数、输血次数和每年住院天数。在HU中添加LC可改善患者依从性，副作用轻微。住院时间、副作用、基线血红蛋白（Hb）和Hb F%影响患者的依从性。性别和年龄对治疗反应没有任何影响，而副作用、依从性、基线Hb和LDH表现出显著影响。结论：LC联合HU治疗成人SCA患者具有良好的血液学和临床疗效。严重病例的反应更为明显。然而，需要对联合用药进行健康教育，以提高患者的依从性和治疗反应。试验注册：Clinical Trials.gov，NCT05081349。https://clinicaltrials.gov/ct2/show/NCT05081349.

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