Koffi Kouassi Gustave, Dieket Ruth, N'dhatz Emeraude, Abenan Nelly Eloise, Silué Alexis Dohoma, Kamara Ismael, Kouakou Boidy, Nanho Danho Clotaire
Hematology Teaching Hospital of Yopougon, Abidjan 21 BP 632, Ivory Coast.
University Felix Houphouet-Boigny GPE-Abidjan, Abidjan, Ivory Coast.
Anemia. 2025 Mar 25;2025:3576890. doi: 10.1155/anem/3576890. eCollection 2025.
Very few trials of hydroxyurea efficacy and safety have been conducted in sub-Saharan Africa. We aimed to evaluate the efficacy and safety of hydroxyurea and its utility in low-resource settings. We conducted a prospective comparative trial in patients with SCA. 128 patients were enrolled and divided into two groups. 68 patients were treated with hydroxyurea at a dose of 10-20 mg/kg/day and 62 patients in a control group without hydroxyurea. The endpoints evaluated were feasibility, safety, and benefit (laboratory variables, sickle cell-related events, transfusions). The patients assigned to hydroxyurea treatment had a lower annual rate of crises than the control group (median 2.9 vs. 5.3 crises per year, =0.001), a lower annual rate of hospitalizations (median 2.2 vs. 4.7, =0.002), and a lower annual rate of transfusions (median 1.3 vs. 5.1, =0.001). We observed a significant increase in Hb F from 11.77% to 14.6% (=0.001) in patients treated with hydroxyurea. We also observed a significant increase in the mean Hb level from 7.3 g/dL to 9.2 g/dL in patients treated with hydroxyurea (=0.004). Patients treated with hydroxyurea also have a beneficial effect on WBC and platelet levels by reducing leukocytosis and thrombocytosis. The annual number of infectious complications was significantly lower in the group of patients treated with hydroxyurea. Hydroxyurea has an important clinical benefit by reducing the incidence of vaso-occlusive events, infections, and transfusions, which translates into fewer hospitalizations. The main problem is that it is not accessible to most of our patients who live in poor socioeconomic conditions.
在撒哈拉以南非洲地区，针对羟基脲疗效和安全性开展的试验极少。我们旨在评估羟基脲的疗效、安全性及其在资源匮乏地区的实用性。我们对镰状细胞贫血（SCA）患者进行了一项前瞻性对照试验。共纳入128例患者并分为两组。68例患者接受羟基脲治疗，剂量为10 - 20毫克/千克/天，62例患者作为未接受羟基脲治疗的对照组。评估的终点指标包括可行性、安全性和获益情况（实验室指标、镰状细胞相关事件、输血情况）。接受羟基脲治疗的患者年度危机发生率低于对照组（中位数分别为每年2.9次和5.3次危机，P = 0.001），年度住院率较低（中位数分别为2.2次和4.7次，P = 0.002），年度输血率也较低（中位数分别为1.3次和5.1次，P = 0.001）。我们观察到接受羟基脲治疗的患者血红蛋白F（Hb F）从11.77%显著升至14.6%（P = 0.001）。我们还观察到接受羟基脲治疗的患者平均血红蛋白水平从7.3克/分升显著升至9.2克/分升（P = 0.004）。接受羟基脲治疗的患者对白细胞和血小板水平也有有益影响，可减轻白细胞增多和血小板增多。接受羟基脲治疗的患者组年度感染并发症数量显著更低。羟基脲通过降低血管闭塞性事件、感染和输血的发生率具有重要临床获益，这意味着住院次数减少。主要问题是，我们大多数生活在社会经济条件较差环境中的患者无法获得羟基脲治疗。
