Clinical analysis of recombinant humanized thrombopoietin for treating 25 children with severe immune thrombocytopenia.
10.7534/j.issn.1009-2137.2014.02.023
- Author:
Jie ZHENG
1
,
2
,
3
,
4
;
Jing-Yao MA
1
;
Yan SU
1
;
Jing YANG
1
;
Rui-Dong ZHANG
1
;
Xuan ZHOU
1
;
Run-Hui WU
1
;
Author Information
1. Hematology Oncology Center, Beijing Key Laboratory of Pediatric Hematology and Oncology
2. Key Laboratory of Major Diseases in Children, Ministry of Education
3. National Key Discipline of Pediatrics
4. Beijing Children's Hospital, Capital Medical University, Beijing 100045, China.
- Publication Type:Journal Article
- MeSH:
Adolescent;
Child;
Child, Preschool;
Female;
Humans;
Infant;
Male;
Platelet Count;
Purpura, Thrombocytopenic, Idiopathic;
drug therapy;
immunology;
Recombinant Proteins;
therapeutic use;
Retrospective Studies;
Thrombopoietin;
therapeutic use;
Treatment Outcome
- From:
Journal of Experimental Hematology
2014;22(2):393-398
- CountryChina
- Language:Chinese
-
Abstract:
This study was aimed to evaluate the efficacy and safety of recombinant humanized thrombopoietin (rhTPO) for treating children with severe immune thrombocytopenia (ITP). A total of 25 patients with severe ITP who accepted rhTPO treatment for 14 days between December, 2009 and November, 2012 in Beijing Children's Hospital was retrospectively analyzed. The results showed that the median platelet counts of all 25 patients increased from the lowest level 4.0×10(9)/L (0×10(9)/L-10×10(9)/L) to the highest level 71×10(9)/L (14×10(9)/L-439×10(9)/L) on median 11 days (range from 3 days to 15 days). After rhTPO discontinuation, the platelet counts of patients gradually decreased. Complete response rate was 44% (11/25), response rate was 32% (8/25), non-response rate was 24% (6/25) and total response rate was 76% (19/25). The platelet count in the patients who showed complete response to rhTPO therapy reached the highest 112×10(9)/L (43×10(9)/L-439×10(9)/L) on median 12 days(range from 7 days to 15 days). The patients showed response to rhTPO treatment on median 4 days (range from 1 days to 11 days). The platelet count decreased gradually after the discontinuation of rhTPO administration but still significantly higher on 28 days than the level before the treatment (P < 0.05). 12 patients who did not respond to γ-globulin before rhTPO treatment showed response to γ-globulin after the discontinuation of rhTPO therapy. 2 patients showed mild clinical adverse reaction. It is concluded that rhTPO is an effective and safe treatment method for children with severe ITP. It will help the patient smoothly through the dangerous period of severe bleeding, but the platelet count decreases gradually after rhTPO discontinuation. Maintenance treatment is needed to consolidate the curative efficacy.
