单中心儿童原发性慢性难治性免疫性血小板减少症临床特点分析

朱筱旌; 马静瑶; 谷昊; 胡玉; 刘会青; 杨碧熙; 张佳璐; 赵莎莎; 吴润晖

doi:10.13201/j.issn.1004-2806.2021.11.007

单中心儿童原发性慢性难治性免疫性血小板减少症临床特点分析

- 国家儿童医学中心, 首都医科大学附属北京儿童医院血液肿瘤中心, 儿童血液病与肿瘤分子分型北京市重点实验室, 儿科学国家重点学科, 儿科重大疾病研究教育部重点实验室(北京, 100045)
基金项目:
国家自然科学基金课题（No：81970111）；北京市自然科学基金课题（No：7192064）；北京市医院管理中心儿科学科协同发展中心“儿科专项”（No：XTZD20180205）；国家科技重大专项（No：2017ZX09304029004）

详细信息

通讯作者: 吴润晖,E-mail:runhuiwu@hotmail.com

中图分类号: R558.2

收稿日期: 2021-06-26

Clinical characteristics of primary chronic refractory immune thrombocytopenia in single center in children

- National Children's Medical Center, Beijing Children's Hospital Affiliated to Capital Medical University Blood Tumor Center, Beijing Key Laboratory of Children's Blood Disease and Tumor Molecular Typing, National Key Discipline of Pediatrics, Key Laboratory of the Ministry of Education for Research on Major Pediatric Diseases, Beijing, 100045, China

More Information

Corresponding author: WU Runhui, E-mail: runhuiwu@hotmail.com

Received Date: 26 June 2021

摘要

摘要: 目的：探讨儿童原发性慢性难治性免疫性血小板减少症患者的临床基本特征，为制定诊疗策略提供依据。方法：回顾性收集2016年4月至2019年5月我院血液肿瘤中心收治的符合慢性/难治性（血小板数量持续<20×10⁹/L）血小板减少症诊断患儿、在首次来该院诊疗时既往临床资料，并分析总结。结果：共收集到符合入组要求患儿67例，其中男35例，女32例，男∶女=1.09∶1.00；中位发病年龄4.39岁（范围6个月~12.16岁），中位病程1.75年（范围1个月~10年），中位血小板基线值5×10⁹/L（范围0~18×10⁹/L），极重度血小板减少（<10×10⁹/L）52例（77.61%）；Buchanan出血评分≥3分仅7例（10.45%），在极重度血小板减少中仅6例（8.96%）。全部患儿既往反复应用提升血小板一线治疗：丙种球蛋白中位应用次数2（范围0~9）次/年，糖皮质激素类药物中位应用天数84（范围0~365） d/年；部分患儿既往应用二线治疗：3例（4.48%）使用细胞毒药物，2例（2.99%）使用雄激素，27例（40.30%）使用利妥昔单抗，4例（5.97%）使用环孢素，1例（1.49%）使用霉酚酸酯；33例（49.25%）使用2种以上药物。结论：儿童原发性慢性难治性免疫性血小板减少症起病多在学龄前期，男女发病无明显差别；以极重度血小板减少为主，但严重出血少见；因病情迁延，多反复使用激素、丙种球蛋白治疗，并多联合其他对儿童毒副作用大的三线药物治疗；目前急需探索安全、有效的靶向分层精准治疗。
- 儿童 /
- 原发性慢性难治性血小板减少症 /
- 临床特点 /
- 治疗
Abstract: Objective: To explore the clinical characteristics of children with primary chronic refractory immune thrombocytopenia, and to provide the basis for making diagnosis and treatment strategies.Methods: From April 2016 to May 2019, the patients with chronic/refractory thrombocytopenia(the number of platelets continuously<20×10⁹/L) admitted to the blood tumor center of our hospital were retrospectively collected, and the previous clinical data were analyzed and summarized when they first came to the hospital for diagnosis and treatment.Results: A total of 67 cases were collected, including 35 males and 32 females, the ratio of male to female was 1.09:1.00. The median age was 4.39 years(range 6 months to 12.16 years), median duration was 1.75 years(range 1 month to 10 years), the baseline value of median platelet was 5×10⁹/L(range 0-18×10⁹/L), and 52 cases(77.61%) were extremely severe thrombocytopenia(<10×10⁹/L). Buchanan bleeding scores ≥ 3 was in only 7 cases(10.45%), and only 6 cases(8.96%) were very severe thrombocytopenia. In all cases, the first-line treatment of promoting platelets had been repeatedly used:median times of application of gamma globulin were 2(range 0-9) times/year, and the number of days of median application of glucocorticoids was 84(range 0-365) days/year. Partial cases used second-line treatment:3 cases(4.48%) used cytotoxic drugs, 2 cases(2.99%) used androgen, 27 cases(40.30%) used rituximab, 4 cases(5.97%) used cyclosporine, 1 case(1.49%) used mycophenolate mofetil. Thirty-three cases(49.25%) used more than 2 kinds of drugs.Conclusion: The onset period of children's primary chronic refractory immune thrombocytopenia is mostly in the pre-school period, and there is no significant difference between male and female. Most of patients are severe thrombocytopenia, but severe bleeding is rare. Because of the prolonged course of the disease, patients are generally treated with glucocorticoids and gamma globulin repeatedly, even combining with other third-line drugs with great toxicity and adverse events on children. At present, it is urgent to explore safe and effective treatment with precisely targeted.
- children /
- primary chronic refractory thrombocytopenia /
- clinical characteristics /
- treatment

HTML全文

参考文献(14)

[1]	Grainger JD, Thind S.A practical guide to the use of eltrombopag in children with chronic immune thrombocytopenia[J].Pediatr Hematol Oncol, 2017, 34(2):73-89.
[2]	Shaw J, Klipatrick K, Eisen M, et al.The incidence and clinical burden of immune thrombocytopenia in pediatric patients in the United States[J].Platelets, 2020, 31(3):307-314.
[3]	杨仁池.难治性原发免疫性血小板减少症定义及临床处理[J].中国实用内科杂志, 2017, 37(2):96-98.
[4]	Buchanan GR, Adix L.Grading of hemorrhage in children with idiopathic thrombocytopenic purpura[J].J Pediatr, 2002, 141(5):683-688.
[5]	Despotovic JM, Grimes AB.Pediatric ITP:is it different from adult ITP?[J].Hematology Am Soc Hematol Educ Program, 2018, 2018(1):405-411.
[6]	付盼.儿童原发性血小板减少症临床分析及预后因素探讨[D].上海:上海交通大学, 2017.
[7]	Giordano P, Lassandro G, di Meo NA, et al.A Narrative Approach to Describe QoL in Children With Chronic ITP[J].Front Pediatr, 2019, 7:163.
[8]	Jaime-Pérez JC, Aguilar-Calderón P, Jiménez-Castillo RA, et al.Treatment outcomes and chronicity predictors for primary immune thrombocytopenia:10-year data from an academic center[J].Ann Hematol, 2020, 99(11):2513-2520.
[9]	侯明.免疫性血小板减少症的治疗进展[J].临床血液学杂志, 2020, 33(1):6-9.
[10]	胡玉, 马洁, 马静瑶, 等.联合应用重组人血小板生成素安全有效治疗婴幼儿重症原发免疫性血小板减少症——单中心数据分析[J].临床血液学杂志, 2020, 33(3):166-169, 175.
[11]	Neunert C, Terrell DR, Arnold DM, et al.American Society of Hematology 2019 guidelines for immune thrombocytopenia[J].Blood Adv, 2019, 3(23):3829-3866.
[12]	Matzdorff A, Meyer O, Ostermann H, et al.Immune Thrombocytopenia-Current Diagnostics and Therapy:Recommendations of a Joint Working Group of DGHO, OGHO, SGH, GPOH, and DGTI[J].Oncol Res Treat, 2018, 41 Suppl 5:1-30.
[13]	Tarantino MD, Despotovic J, Roy J, et al.Romiplostim treatment for children with immune thrombocytopenia:Results of an integrated database of five clinical trials[J].Pediatr Blood Cancer, 2020, 67(11):e28630.
[14]	Li Y, He Z, Chen Q, et al.The efficacy and safety of low-dose rituximab in immune thrombocytopenia:a systematic review and meta-analysis[J].Platelets, 2019, 30(6):690-697.