This multicenter randomized, open-label study aimed to compare the efficacy and safety of eltrombopag combining rituximab with eltrombopag in China adult ITP patients .This study was be conducted in adult ITP patients who had not responded to or had relapsed after previous treatment of ITP, including first line therapy and /or splenectomy.
The primary objective of this study was to evaluate the efficacy and safety of eltrombopag combining rituximab treating previously treated ITP patients compared to eltrombopag. The secondary objective was to evaluate the efficacy of eltrombopag combining rituximab in ITP patients with positive autoantibody compared to eltrombopag .In addition, health-related quality of life (HRQoL) measure was assessed in all participants. 224 eligible subjects were randomized to either eltrombopag combining rituximab or eltrombopag treatment in 1:1 ratio. 112 enrolled patients are randomly picked up to take eltrombopag combining with rituximab at the indicated dose. 112 enrolled patients are randomly picked up to take eltrombopag at the indicated dose. The initial dose of eltrombopag administration was an oral 75 mg once daily in all participants .The dose of eltrombopag was adjusted according to the subject platelet count during the period from week 1 to week 24. Subjects in eltrombopag combining rituximab treatment group received single dose infusion of rituximab 375 mg/m(2) within 14 days after enrollment.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
224
After enrollment,all subjects receive eltrombopag treatment,the initial dose of eltrombopag administration was an oral 75 mg once daily.Complete blood count including platelet count was done once a week.The dose of eltrombopag was adjusted according to the subject platelet count during the period from week 1 to week 24. If the platelet count \>250×10\^9/L, the eltrombopag will stop until the platelet count \<30×10\^9/L. All subjects receive single dose infusion of rituximab 375 mg/m(2) within 14 days after enrollment. Efficacy and safety will be evaluated at Week 4, Week 8, and Week 12.
After enrollment,all subjects receive eltrombopag treatment,the initial dose of eltrombopag administration was an oral 75 mg once daily.Complete blood count including platelet count was done once a week.The dose of eltrombopag was adjusted according to the subject platelet count during the period from week 1 to week 24. If the platelet count \>250×10\^9/L, the eltrombopag will stop until the platelet count \<30×10\^9/L. Efficacy and safety will be evaluated at Week 4, Week 8, and Week 12.
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Tianjin, China
RECRUITINGTreatment response
Number of participants achieving a platelet count \>=30×10\^9/L and at least doubling of the baseline count at Week 4, Week 8, and Week 12 .
Time frame: From the start of study treatment (Day 1) up to the end of week 12.
Drug efficacy
Number of participants achieving a platelet count \>=30×10\^9/L at week 4, week 8, and week 12 in ITP patients with positive autoantibody
Time frame: From the start of study treatment (Day 1) up to the end of week 4, week 8 and week 12.
Long-term treatment response
Number of participants achieving a platelet count \>=30×10\^9/L at week 16, week 20, and week 24.
Time frame: From the start of study treatment (Day 1) up to the end of week 16, week 20 and week 24
Time to Response
Time to response is defined as time from the start of treatment to the first time of achieving a platelet count \>=30×10\^9/L and at least doubling of the baseline count during the whole 24 weeks.
Time frame: From the start of study treatment (Day 1) up to the end of week 24
Duration of response
Total duration of time a participant had a platelet count \>=30×10\^9/L
Time frame: From the start of study treatment (Day 1) up to the end of week 24.
Evaluation of effectiveness
Number of participants that reduced or discontinued baseline concomitant ITP medications during the whole 24 weeks.
Time frame: From the start of study treatment (Day 1) up to the end of week 24.
Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
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The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.
Time frame: From the start of study treatment (Day 1) up to the end of week 24.
Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)
In all participants ,use ITP-PAQ to assess the HRQoL before and after treatment.
Time frame: From the start of study treatment (Day 1) up to the end of week 24.
Functional Assessment of Chronic Illness Therapy fatigue subscale (FACIT-F)
In all participants ,use FACIT-F to assess the HRQoL before and after treatment.
Time frame: From the start of study treatment (Day 1) up to the end of week 24.
Number of Participants with side effects of the drugs
Side effects of the drugs included fever, headache, serum disease,hypotension, rashes, infection liver injury, hypokalaemia, etc.
Time frame: From the start of study treatment (Day 1) up to the end of week 24.