Recombinant human thrombopoietin in combination with cyclosporin A as a novel therapy in corticosteroid-resistant primary immune thrombocytopenia.
BACKGROUND
The management of patients with refractory immune thrombocytopenia (ITP) is challenging, as there is no standard treatment option. The aim of this study was to investigate the efficacy of recombinant human thrombopoietin (rhTPO) in combination with cyclosporin A (CsA) for the management of patients with corticosteroid-resistant primary ITP.


METHODS
Thirty-six patients with corticosteroid-resistant ITP were randomly divided into an observation group and control group. In the observation group, 19 patients received subcutaneous injection of rhTPO at a dose of 1 µg/kg (300 U/kg) once daily up to day 14. Simultaneously they also received oral CsA at a dose of 1.5-2.0 mg/kg twice daily for three months. In the control group, rhTPO alone was administered subcutaneously at 1 µg/kg once daily in the other 17 ITP patients for 14 consecutive days and then the treatment was withdrawn.


RESULTS
There was no significant difference in the response rate at the end of the first week after treatment initiation between the observation group and the control group (63.2% vs. 58.8%, P > 0.05), neither was there at the end of the second week (89.5% vs. 94.1%, P > 0.05). However, the relapse rate in the observation group was significantly lower than that in control group at the end of the first (17.7% vs. 50.0%, P < 0.05), second (29.4% vs. 68.8%, P < 0.05) and the third month (29.4% vs. 87.5%, P < 0.01). In addition, rhTPO plus CsA were well tolerated and adverse events recorded were mild.


CONCLUSIONS
Combination therapy with rhTPO and CsA was effective in the management of patients with corticosteroidresistant ITP, with a relatively short time to response and low recurrence rate. It might be considered as a potential secondline treatment regimen for ITP.
Department of Hematology, Qilu Hospital, Shandong University, Jinan, Shandong 250012, China; Department of Hematology, Affiliated Hospital of Medical College, Qingdao University, Qingdao, Shandong 266003, China.
Zhong-Guang
ZG
Cui
Xin-Guang
XG
Liu
Ping
P
Qin
Ming
M
Hou
Shao-Ling
SL
Wu
Jun
J
Peng
Hong-Guo
HG
Zhao
Hong-Yi
HY
Wang
Chun-Ting
CT
Zhao
D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D000328:Adult; D000368:Aged; D016572:Cyclosporine / Q000008:administration & dosage / Q000627:therapeutic use*; D004351:Drug Resistance; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D011994:Recombinant Proteins / Q000627:therapeutic use*; D013921:Thrombocytopenia / Q000188:drug therapy*; D013926:Thrombopoietin / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome; D055815:Young Adult
D000305:Adrenal Cortex Hormones; D011994:Recombinant Proteins; D016572:Cyclosporine; D013926:Thrombopoietin