Kabuyi Paul Lumbala, Mbayabo Gloire, Ngole Mamy, Zola Aimé Lumaka, Race Valerie, Matthijs Gert, Van Geet Chris, Tshilobo Prosper Lukusa, Devriendt Koenraad, Mikobi Tite Minga
Department of Pediatrics University of Kinshasa Kinshasa Democratic Republic of the Congo.
Center of Human Genetics Faculty of Medicine University of Kinshasa Kinshasa Democratic Republic of the Congo.
EJHaem. 2023 Jul 19;4(3):595-601. doi: 10.1002/jha2.735. eCollection 2023 Aug.
: Despite a high incidence of sickle cell anemia, hydroxyurea (HU) treatment is rarely used in the DR Congo. This study aims to assess the efficacy of HU, the incidence of side effects that may limit its use in adults and to determine the dose needed for clinical improvement in patients. : In a prospective study, patients received an initial dose of 15 mg/kg/day which was increased by 5 mg/kg every 6 months, up to a maximum of 30 mg/kg/day. The response and side effects to HU were evaluated biologically and clinically every 3 months during a 2-year period. : Seventy adult patients with a moderate or severe clinical phenotype initiated treatment. Only minor side effects were reported. At the end of the 2-year treatment phase, 45 (64.3%) had dropped out, of whom 33 were without a clear reason. Clinical and biological improvement was more marked during the first year. There was a reduction in severe vaso-occlusive crises ( < 0.001), need for transfusion ( < 0.001), and hospitalization days ( = 0.038). Fetal hemoglobin (HbF) levels increased on average 2.9 times after 12 months ( < 0.001). The increase in mean corpuscular volume was greater in the first year ( < 0.001) than in the second year ( = 0.041). The decrease in leukocytes ( < 0.001) was significant during the first year. In 70% of patients, the 20 mg/kg/day dose was needed to reach the 20% HbF threshold. : HU is effective and well tolerated. The magnitude of the response varies from one patient to another. Improvement of clinical manifestations is achieved in most patients with a relatively low dose. Effective implementation of HU treatment will require improved adherence to treatment.
尽管镰状细胞贫血发病率很高，但羟基脲（HU）治疗在刚果民主共和国很少使用。本研究旨在评估HU的疗效、可能限制其在成人中使用的副作用发生率，并确定患者临床改善所需的剂量。：在一项前瞻性研究中，患者接受初始剂量为15mg/kg/天，每6个月增加5mg/kg，最高可达30mg/kg/天。在2年期间，每3个月从生物学和临床方面评估对HU的反应和副作用。：70名具有中度或重度临床表型的成年患者开始治疗。仅报告了轻微副作用。在2年治疗阶段结束时，45名（64.3%）患者退出，其中33名没有明确原因。临床和生物学改善在第一年更为明显。严重血管闭塞性危机减少（<0.001）、输血需求减少（<0.001）和住院天数减少（=0.038）。12个月后胎儿血红蛋白（HbF）水平平均增加2.9倍（<0.001）。第一年平均红细胞体积的增加大于第二年（<0.001）（=0.041）。第一年白细胞减少（<0.001）显著。70%的患者需要20mg/kg/天的剂量才能达到20%HbF阈值。：HU有效且耐受性良好。反应程度因患者而异。大多数患者使用相对较低剂量即可实现临床表现的改善。有效实施HU治疗需要提高治疗依从性。
