Department of Hematology and Oncology, Beijing Children's Hospital, Capital Medical University, Nanlishi Road No. 56, Xicheng District, Beijing, 100045, People's Republic of China.
Beijing Key Laboratory of Pediatric Hematology Oncology, National Center for Children's Health, Beijing, 100045, China.
Int J Hematol. 2020 Dec;112(6):773-779. doi: 10.1007/s12185-020-02977-9. Epub 2020 Sep 3.
Immune thrombocytopenia (ITP) is one of the most common acquired immune-mediated bleeding disorders found in children. Prednisone is usually considered a first-line therapeutic agent for ITP in children. Yet, prolonged exposure to prednisone has been associated with certain side effects. This prospective randomized study comparatively assessed the efficacy and safety of short-course high-dose dexamethasone (HDD) and standard prednisone (PDN) as a first-line treatment for children with previously untreated primary ITP. Two hundred eleven children were randomized into the HDD (n = 110) and PDN (n = 101) groups. There was no difference in baseline characteristics between the two groups (p > 0.05). Early response rates were 92.7% and 93% (p = 0.923); initial response rates were 93.6% and 95% (p = 0.658) and durable response rates were 90% and 91% (p = 0.787) in the HDD and PDN groups, respectively. More remission patients in the HDD group compared with the PDN group (86.3% vs. 80.1%) at 12th month after treatment, yet no statistical difference was observed (p = 0.703). Bleeding events were 10.9% and 14.8% (p = 0.105), and bleeding score improvement rates were 78.2% and 76.2% (p = 0.284) in the HDD and PDN groups, respectively. Cushing's disease, weight gain and infection rates were higher in the PDN group compared to the HDD group (80% vs. 10%, p = 0.001; 74.2% vs. 13.6%, p = 0.001; and 26% vs. 11.8%, p = 0.012) 1 month after treatment. HDD showed non-inferior efficacy and fewer glucocorticoid-related adverse effects compared with PDN. These findings indicated that HDD could be considered as a first-line treatment in children with previously untreated primary ITP, thus replacing standard PDN.
免疫性血小板减少症 (ITP) 是儿童中最常见的获得性免疫介导性出血性疾病之一。泼尼松通常被认为是儿童 ITP 的一线治疗药物。然而，长期暴露于泼尼松会引起某些副作用。这项前瞻性随机研究比较了短程大剂量地塞米松 (HDD) 和标准泼尼松 (PDN) 作为一线治疗儿童初治原发性 ITP 的疗效和安全性。211 名儿童被随机分为 HDD(n=110)和 PDN(n=101)组。两组患者的基线特征无差异(p>0.05)。早期反应率分别为 92.7%和 93%（p=0.923）；初始反应率分别为 93.6%和 95%（p=0.658），持久反应率分别为 90%和 91%（p=0.787）。治疗 12 个月后，HDD 组的缓解患者比例高于 PDN 组(86.3% vs. 80.1%)，但无统计学差异(p=0.703)。HDD 组和 PDN 组的出血事件分别为 10.9%和 14.8%（p=0.105），出血评分改善率分别为 78.2%和 76.2%（p=0.284）。治疗 1 个月后，PDN 组的库欣病、体重增加和感染发生率高于 HDD 组(80% vs. 10%，p=0.001；74.2% vs. 13.6%，p=0.001；26% vs. 11.8%，p=0.012)。HDD 与 PDN 相比，疗效不劣效，且糖皮质激素相关不良反应发生率较低。这些发现表明，HDD 可作为儿童初治原发性 ITP 的一线治疗药物，从而替代标准 PDN。
