PDF(999 KB)
rhTPO联合大剂量地塞米松治疗儿童难治性免疫性血小板减少症的临床分析
卢园园, 管娜, 孟庆红, 李明磊, 刘云云, 王莹
中国当代儿科杂志 ›› 2018, Vol. 20 ›› Issue (7) : 534-537.
PDF(999 KB)
PDF(999 KB)
rhTPO联合大剂量地塞米松治疗儿童难治性免疫性血小板减少症的临床分析
Efficacy of recombinant human thrombopoietin combined with high-dose dexamethasone in the treatment of refractory immune thrombocytopenia in children
目的 探讨重组人血小板生成素（rhTPO）联合大剂量地塞米松治疗儿童难治性免疫性血小板减少性紫癜（ITP）的疗效及安全性。方法 58例一线治疗无效的ITP患儿随机分为rhTPO治疗组（31例）和地塞米松治疗组（27例）。地塞米松组每28天静滴大剂量地塞米松4 d （每日0.6 mg/kg），连续两个循环。rhTPO组在地塞米松治疗的基础上皮下注射rhTPO （每日300 U/kg）14 d。评价治疗第3、7、14天和第1、2、3个月末的总有效率（显效+有效）和治疗中的不良反应。结果 与DXM组相比，治疗第7天、14天、1个月，rhTPO联合治疗患儿的显效率、总有效率均较高（P < 0.05）；治疗第2个月，rhTPO联合治疗患儿仅总有效率较高（P < 0.05）。DXM组在治疗第一周发生肝损1例。两组患儿均未出现高血压、发热、皮疹、过敏反应、乏力等不良事件。结论 rhTPO联合大剂量地塞米松治疗难治性ITP的有效率高，而且安全。
Objective To explore the effcacy and safety of recombinant human thrombopoietin (rhTPO) combined with high-dose dexamethasone (DXM) in the treatment of children with refractory immune thrombocytopenic purpura (ITP).Methods Fifty-eight ITP children who had failed frst-line therapy were randomly divided into two groups:DXM treatment (n=27) and rhTPO + DXM treatment (n=31). The DXM treatment group received two continuous cycles of DXM treatment; in each cycle, patients received high-dose DXM (0.6 mg/kg daily) by intravenous drip for 4 days every 28 days. The rhTPO group received subcutaneous injection of rhTPO (300 U/kg daily) for 14 days additional to DXM treatment. The overall response rate (marked response rate+slight response rate) and adverse reactions were evaluated after 3, 7, and 14 days and 1, 2, and 3 months of treatment.Results After 7 and 14 days and 1 month of treatment, the rhTPO+DXM treatment group had a signifcantly higher marked response rate and a signifcantly higher overall response rate than the DXM treatment group (P < 0.05). After 2 months of treatment, the rhTPO + DXM treatment group had a signifcantly higher overall response rate than the DXM group (P < 0.05). One patient in the DXM treatment group had liver damage during the frst week of treatment. There was no hypertension, fever, rash, allergy, or weakness in the two groups.Conclusions rhTPO combined with high-dose DXM is an effective and safe approach for treating refractory ITP.
[1] Kühne T. Update on the Intercontinental Cooperative ITP Study Group (ICIS) and on the Pediatric and Adult Registry on chronic ITP (PARC ITP)[J]. Pediatr Blood Cancer, 2013, 60(Suppl 1):S15-S18.
[2] Wei Y, Ji XB, Wang YW, et al. High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia:a prospective multicenter randomized trial[J].Blood, 2016, 127(3):296-302.
[3] Wendling F, Maraskovsky E, Debili N, et al. cMpl ligand is a humoral regulator of megakaryocytopoiesis[J]. Nature, 1994, 369(6481):571-574.
[4] 李燕, 黄琴, 王超, 等. 大剂量地塞米松联合重组人血小板生成素治疗48例成人初治重症原发免疫性血小板减少症的有效性及安全性[J]. 中华血液学杂志, 2016, 37(2):134-137.
[5] 黄月婷, 刘晓帆, 薛峰, 等. 重组人血小板生成素治疗41例儿童原发免疫性血小板减少症的疗效及安全性[J]. 中华血液学杂志, 2015, 36(6):511-514.
[6] 马洁, 傅玲玲, 陈振萍, 等. 大剂量地塞米松治疗38例原发免疫性血小板减少症患儿的临床研究[J]. 中国血液学杂志, 2016, 37(10):912-915.
[7] 中华医学会儿科分会血液学组. 儿童原发性免疫性血小板减少症诊疗建议[J]. 中华儿科杂志, 2013, 51(5):382-384.
[8] Mazzucconi MG, Fazi P, Bernasconi S, et al. Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura:a GIMEMA experience[J]. Blood, 2007, 109(4):1401-1407.
[9] Bénichou C. Criteria of drug-induced liver disorders. Report of an international consensus meeting[J]. J Hepatol, 1990, 11(2):272-276.
[10] Wörmann B. Clinical indications for thrombopoietin and thrombopoietin-receptor agonists[J]. Transfus Med Hemother, 2013, 40(5):319-325.
[11] Bussel JB, Lakkaraja M. Thrombopoietic agents:there is still much to learn[J]. Presse Med, 2014, 43(4 Pt 2):e69-e78.
[12] 廖小梅, 王迎, 邓承祺, 等. 血小板生成素与慢性特发性血小板减少性紫癜的相关研究[J]. 中华血液学杂志, 2001, 22:473-475.
[13] Nakazaki K, Hosoi M, Hangaishi A, et al. Comparison between pulsed high-dose dexamethasone and daily corticosteroid therapy for adult primary immune thrombocytopenia:a retrospective study[J]. Intern Med, 2012, 51(8):859-863.
[14] Hedlund-Treutiger I, Henter JI, Elinder G, et al. Randomized study of IVIg and high-dose dexamethasone therapy for children with chronic idiopathic thrombocytopenic purpura[J]. J Pediatr Hematol Oncol, 2003, 25(2):139-144.
[15] 魏炜. 大剂量地塞米松联合rhTPO治疗初治重症原发免疫性血小板减少症疗效观察[J]. 中国继续医学教育, 8(33):202-204.
[16] 贾小军. 重组人血小板生成素联合糖皮质激素治疗原发性免疫性血小板减少症临床疗效分析[J]. 医药卫生, 2015, 1(3):8-10.
[17] 周洪静,郝云良. 大剂量地塞米松联合重组人血小板生成素治疗重症免疫性血小板减少症的临床疗效研究[J]. 国际输血及血液学杂志, 2016, 39(4):325-328.
