Department of Pediatrics, Bayero University/Aminu Kano Teaching Hospital, Kano, Nigeria.
Department of Pediatrics, Murtala Mohammed Specialist Hospital, Kano, Nigeria.
Blood Adv. 2023 Oct 24;7(20):6024-6034. doi: 10.1182/bloodadvances.2023010789.
Children with sickle cell anemia (SCA) living in Nigeria are at an increased risk of malnutrition, which contributes to increased morbidity and mortality. However, evidence-based guidelines for managing malnutrition in children with SCA are lacking. To address this gap, we conducted a multicenter, randomized controlled feasibility trial to assess the feasibility and safety of treating children with SCA aged from 5 to 12 years and having uncomplicated severe acute malnutrition (body mass index z score of <-3.0). Children with SCA and uncomplicated severe acute malnutrition were randomly allocated to receive supplemental ready-to-use therapeutic food (RUTF) with or without moderate-dose hydroxyurea therapy (20 mg/kg per day). Over a 6-month enrollment period, 3190 children aged from 5 to 12 years with SCA were evaluated for eligibility, and 110 of 111 children who were eligible were enrolled. During the 12-week trial, no participants withdrew or missed visits. One participant died of unrelated causes. Adherence was high for hydroxyurea (94%, based on pill counts) and RUTF (100%, based on the number of empty sachets returned). No refeeding syndrome event or hydroxyurea-related myelosuppression occurred. At the end of the trial, the mean change in body mass index z score was 0.49 (standard deviation = 0.53), and 39% of participants improved their body mass index z score to ≥-3.0. Our findings demonstrate the feasibility, safety, and potential of outpatient treatment for uncomplicated severe acute malnutrition in children with SCA aged from 5 to 12 years in a low-resource setting. However, RUTF sharing with household and community members potentially confounded the response to malnutrition treatment. This trial was registered at clinicaltrials.gov as #NCT03634488.
尼日利亚患有镰状细胞贫血症（SCA）的儿童患营养不良的风险增加，这导致发病率和死亡率增加。然而，缺乏针对 SCA 儿童营养不良管理的循证指南。为了解决这一差距，我们进行了一项多中心、随机对照可行性试验，以评估治疗 5 至 12 岁患有不伴并发症的严重急性营养不良（体重指数 z 分数 <-3.0）的 SCA 儿童的可行性和安全性。将患有 SCA 和不伴并发症的严重急性营养不良的儿童随机分配接受补充即用型治疗食品（RUTF），或 RUTF 联合中等剂量羟基脲治疗（每天 20mg/kg）。在 6 个月的入组期间，对 3190 名 5 至 12 岁患有 SCA 的儿童进行了资格评估，符合条件的 111 名儿童中有 110 名入组。在 12 周的试验期间，没有参与者退出或错过就诊。有 1 名参与者因与研究无关的原因死亡。羟基脲（基于药片计数，94%）和 RUTF（基于返回的空袋数量，100%）的依从性高。未发生再喂养综合征事件或羟基脲相关的骨髓抑制。试验结束时，体重指数 z 分数的平均变化为 0.49（标准差=0.53），39%的参与者体重指数 z 分数提高到≥-3.0。我们的研究结果表明，在资源匮乏的环境中，对 5 至 12 岁患有 SCA 的儿童，采用门诊治疗不伴并发症的严重急性营养不良是可行的、安全的，且具有潜力。然而，RUTF 与家庭和社区成员共享可能会影响对营养不良治疗的反应。本试验在 clinicaltrials.gov 注册，编号为 NCT03634488。
