[A multi-center clinical trial of recombinant human thrombopoietin in chronic refractory idiopathic thrombocytopenic purpura].
OBJECTIVE
To evaluate the efficacy and safety of human recombinant thrombopoietin (rhTPO, a product of Sunshine Pharmaceutical Co Ltd, China) in chronic refractory idiopathic thrombocytopenic purpura.


METHODS
Eighty-two patients with chronic refractory idiopathic purpura received daily subcutaneous administration of rhTPO at a dose of 1.0 micro g/kg for a maximum of 14 doses.


RESULTS
After the beginning of treatment, the median platelet counts increased from 15.5 (6.0 - 24.0) x 10(9)/L to 27.5 (16.0-47.0) x 10(9)/L, 35.0 (20.5-78.0) x 10(9)/L and 77.0 (41.8-119.5) x 10(9)/L on the fifth, seventh and fifteenth day, respectively (P < 0.01). After the discontinuation of rhTPO administration, the platelet count decreased gradually. On the twenty-eighth day, the median platelet count was 76.5 (35-120.3) x 10(9)/L, which was still significantly higher than the level before the treatment (P < 0.01). The overall response rate was 85.3%. The rate in the group with remarkable response (platelet count > or = 100 x 10(9)/L without bleeding) was 58.5% and that in the good response group (platelet count > or = 50 x 10(9)/L or 30 x 10(9)/L higher than the count before the treatment, without bleeding) was 26.8%. Only 3 patients had mild clinical untoward reactions. Low titer (1:5) of anti-TPO antibody in serum was detected with ELISA in one of the sixteen patients who received the test on the twenty-first and twenty-eighth day. Neutralizing test using TPO-dependent cell line showed that the positive serum had no neutralizing activity on rhTPO.


CONCLUSIONS
Consecutive subcutaneous injection of rhTPO for a maximum of 14 days was associated with a temporary elevation in platelet counts in patients with chronic refractory ITP with tolerable adverse effects.
Zhao
Yong-qiang
YQ
Department of Hematology, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Science, Beijing 100730, China. zhaoyq@pumch.ac.cn
Wang
Qing-Yu
QY
Zhai
Ming
M
Xu
Jian
J
Chen
Xie-qun
XQ
Liu
Wen-li
WL
Zhang
Mei
M
Song
Shan-jun
SJ
Wang
Jian-min
JM
Meng
Fan-yi
FY
Shan
Yuan-dong
YD
D000328:Adult; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000627:therapeutic use; D013926:Thrombopoietin / Q000008:administration & dosage / Q000627:therapeutic use*
D011994:Recombinant Proteins; D013926:Thrombopoietin