Eltrombopag for the treatment of primary immune thrombocytopenia in 23 pediatric patients.
10.3760/cma.j.issn.0253-2727.2019.12.012
- Author:
Yue Ting HUANG
1
;
Xiao Fan LIU
2
;
Yun Fei CHEN
2
;
Rong Feng FU
2
;
Wei LIU
2
;
Feng XUE
2
;
Lei ZHANG
2
;
Ren Chi YANG
2
Author Information
1. Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Tianjin 300020, China (Huang Yueting is working on the Department of Hematology, the First Affiliated Hospital of Xiamen University, Xianmen 361003, China).
2. Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Tianjin 300020, China.
- Publication Type:Journal Article
- Keywords:
Child;
Drug toxicity;
Eltrombopag;
Thrombocytopenia;
Treatment outcome
- MeSH:
Adolescent;
Benzoates;
Child;
Female;
Humans;
Hydrazines;
Male;
Purpura, Thrombocytopenic, Idiopathic;
Pyrazoles;
Retrospective Studies;
Treatment Outcome
- From:
Chinese Journal of Hematology
2019;40(12):1031-1034
- CountryChina
- Language:Chinese
-
Abstract:
Objective: To evaluate the efficacy and safety of eltrombopag in the treatment of pediatric primary immune thrombocytopenia (ITP) . Methods: The clinical characteristics of 23 pediatric ITP patients who received eltrombopag from May 2015 to March 2019 were retrospectively analyzed. Eltrombopag started with an initial dose of 12.5-50.0 mg/d and the maximum dose was 75.0 mg/d. Results: Among 23 children, there were 11 boys and 12 girls with median age 11.0 (2.0-17.0) years. Four cases were newly diagnosed ITP, the other 8 of persistent ITP and 11 of chronic ITP. The duration of eltrombopag application ranged from 4.5 to 95 weeks (8/23 still ongoing) . The median platelet (PLT) counts at 2 weeks, 4 weeks, 3 months and the 6 months after treatment were 40 (4-170) ×10(9)/L, 20 (4-130) ×10(9)/L, 60 (4-110) ×10(9)/L, and 70 (18-160) ×10(9)/L, which were all significantly higher than that before treatment 14 (2-82) ×10(9)/L (z=-3.440, P=0.001; z=-1.964, P=0.049; z=-4.339, P<0.001;z=-5.794, P<0.001 respectively) . The overall response rate was 60.87% (14/23 cases) . The median time to PLT count ≥30×10(9)/L was 10.5 (3-42) days. Seven patients (30.43%) responded within the first week, and 10 cases (43.48%) achieved PLT counts ≥30×10(9)/L within 2 weeks. All patients were divided into three groups according to the age (<6 years old, 6-12 years old, 13-17 years old) . The response rates were similar in three groups, as 33.33%, 60.00%, 85.71%, respectively. WHO bleeding scores as 0, 1, 2 were corresponded to 4, 12 and 7 patients before treatment. Patient numbers changed to 13, 7, 3 with bleeding scores 0, 1, 2 respectively after treatment (χ(2)=7.558, P=0.006) . Eltrombopag was well tolerated, the common adverse events included elevated transaminase (4 cases) and serum bilirubin (4 cases) ; mild nausea (1 case) , vomiting (1 case) and dizziness (1 case) . No drug withdrawal occurred due to adverse events. Conclusion: Eltrombopag is safe and effective in pediatric patients with primary ITP.
