Department of Hematology & Oncology, Children's Hospital Karachi, Karachi, Pakistan.
Department of Research, Children's Hospital Karachi, Karachi, Pakistan.
Blood Adv. 2022 Dec 27;6(24):6162-6168. doi: 10.1182/bloodadvances.2022007031.
Transfusion-related complications and lack of resources in low-to-middle-income countries have led to a search for novel therapies to reduce the need for blood transfusions in patients with β-thalassemia. Hydroxyurea (HU) has demonstrated promising outcomes; additionally, thalidomide has also shown improvement in hemoglobin (Hb) levels for patients with β-thalassemia in some studies. This study presents the findings of a single-arm nonrandomized trial to evaluate the efficacy of combination therapy of HU and thalidomide in children with β-thalassemia. A total of 135 patients (median age, 6 [interquartile range, 3-10] years), 77 (57%) males and 58 (43%) females, were followed first using HU alone, for 6 months, and then using the combination of HU and thalidomide for another 6 months. The primary outcome was a response to therapy, as measured by the number of transfusions required and Hb levels, for patients while receiving HU alone and then while using the combination therapy. Study findings showed a significant decline in blood transfusion volume (P < .001) and a significant increase in median Hb levels within 3 and 6 months of the combination therapy (P < .001). Eighty-nine (65.93%) participants were good responders, 16 (11.85%) were responders, and 30 (22.22%) were nonresponders, whereas the responders had variable genetic mutations. A total of 38 adverse events were reported that resolved on supportive treatment or temporary hold of the intervention. The combination therapy demonstrated promising results and could be considered for a diverse patient population with β-thalassemia. This trial was registered at www.clinicaltrials.gov as #NCT05132270.
输血相关并发症和中低收入国家资源匮乏，促使人们寻找新的治疗方法来减少β-地中海贫血患者的输血需求。羟基脲 (HU) 已显示出良好的效果；此外，在一些研究中，沙利度胺也显示出对β-地中海贫血患者血红蛋白 (Hb) 水平的改善。本研究报告了一项单臂非随机试验的结果，该试验旨在评估 HU 和沙利度胺联合治疗β-地中海贫血儿童的疗效。共有 135 名患者（中位数年龄 6 [四分位距 3-10] 岁），77 名（57%）男性和 58 名（43%）女性，首先单独使用 HU 治疗 6 个月，然后再使用 HU 和沙利度胺联合治疗 6 个月。主要结局是单独使用 HU 治疗和联合治疗时患者所需的输血次数和 Hb 水平的治疗反应。研究结果显示，在联合治疗的 3 个月和 6 个月内，输血量显著减少（P <.001），Hb 中位数水平显著增加（P <.001）。89 名（65.93%）患者为良好反应者，16 名（11.85%）为反应者，30 名（22.22%）为无反应者，而反应者的基因突变不同。共报告了 38 例不良事件，这些事件在支持性治疗或暂时停止干预后得到解决。联合治疗显示出良好的效果，可考虑用于不同基因型的β-地中海贫血患者。该试验在 www.clinicaltrials.gov 注册，编号为 #NCT05132270。
