Corticosteroid-resistant or Relapsed ITP
Conditions
Keywords
Immune Thrombocytopenia, Recombinant human thrombopoietin, Eltrombopag, switching
Brief summary
Thrombopoietin Receptor Agonists (TPO-ra) are novel treatments for patients with refractory Primary Immune Thrombocytopenia (ITP). Rh-TPO and eltrombopag increase the number of platelets through different mechanism. If there is cross-resistance between 2 drugs for the treatment of adult ITP is still no answer. The purpose of this study is to investigate the efficacy and safety of switching eltrombopag and Rh-TPO in adults with ITP.
Detailed description
Non-interventional study. Patients who fail previous steroids and receive rh-TPO and then switch to EPAG or vice versa will be enrolled. The reason for switch will be recorded. Patients in the rh-TPO group were given rh-TPO 300 U/kg once daily for 21 days, and those in the eltrombopag group were given eltrombopag 50mg once daily for 6 weeks. Rh-TPO and eltrombopag were terminated any time the platelet counts increased above 100 × 10\^9/L in the rh-TPO group and 300 × 10\^9/L in the eltrombopag group. The efficacy, safety, and patient/physician preference will be assessed and compared between the two agents.
Interventions
DRUGEltrombopag
Patients will be given eltrombopag 50mg once daily for 6 weeks.
DRUGRecombinant human thrombopoietin (rh-TPO)
Patients will be given rh-TPO 300 U/kg once daily for 21 days.
Sponsors
Peking University People's Hospital
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
1.18 years or older 2.Primary ITP 3.Platelet count ≤ 30 × 109/l 4.Normal neutrophils, reticulocyte count, creatinine and liver enzyme values 5.Available follow-up of 2 months at least for each period 6.Failed initial glucocorticosteroid treatment 7.Unwillingness to accept splenectomy or failed splenectomy \-
Exclusion criteria
1. HIV, hepatitis B or C, Helicobacter pylori infection 2. Malignancy 3. Congenital or acquired immunologic deficit 4. History of thrombosis plus two or more risk factors 5. Nursing or pregnant women 6. Abnormal liver and renal functions: AST/ALT/total bilirubin ≥1.5 × ULN, creatinine ≥1.5 mg/dl 7. Severe heart and lung dysfunctions -
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Response Rate at 6 Weeks After Switching | 6 weeks | The percentage of patients who have reached platelet count ≥ 50×10\^9/L at 6 weeks after switching. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Treatments Associated Adverse Events | 6 weeks | Adverse event/serious adverse event associated with study drugs during 6 weeks after switching |
| Reasons of Switching | 6 weeks | Reasons of switching eltrombopag and rh-TPO will be recorded, including lack of efficacy, patient preference, side effects, platelet count fluctuation |
| Number of Participants With Bleeding Events | 6 weeks | Number of participants with bleeding events of the two groups during 6 weeks after switching |
| TOR (Time to Response) | 6 weeks | The time to achieve platelet count ≥ 50×10\^9/L after switching. |
| DOR (Duration of Response) | 6 weeks | The duration of achieving platelet count ≥ 50×10\^9/L after switching. |
Countries
China
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Recombinant Human Thrombopoietin (Rh-TPO) Group Patients who fail previous steroids and eltrombopag and then switch to Rh-TPO will be enrolled. The reason for switch will be recorded. Patients will be given rh-TPO 300 U/kg once daily for 21 days. Rh-TPO will be terminated any time the platelet counts increased above 100 × 10\^9/L. The efficacy, safety, and patient/physician preference will be assessed.
Recombinant human thrombopoietin (rh-TPO): Patients will be given rh-TPO 300 U/kg once daily for 21 days. | 10 |
| Eltrombopag Group Patients who fail previous steroids and rh-TPO and then switch to eltrombopag will be enrolled. The reason for switch will be recorded. Patients will be given eltrombopag 50mg once daily for 6 weeks. Eltrombopag will be terminated any time the platelet counts increased above 300× 10\^9/L.The efficacy, safety, and patient/physician preference will be assessed.
Eltrombopag: Patients will be given eltrombopag 50mg once daily for 6 weeks. | 10 |
| Total | 20 |
Baseline characteristics
| Characteristic | Eltrombopag Group | Total | Recombinant Human Thrombopoietin (Rh-TPO) Group |
|---|---|---|---|
| Age, Continuous | 28 years | 29 years | 30 years |
| Race and Ethnicity Not Collected | — | 0 Participants | — |
| Region of Enrollment China | 10 participants | 20 participants | 10 participants |
| Sex: Female, Male Female | 6 Participants | 11 Participants | 5 Participants |
| Sex: Female, Male Male | 4 Participants | 9 Participants | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 10 | 0 / 10 |
| other Total, other adverse events | 1 / 10 | 1 / 10 |
| serious Total, serious adverse events | 0 / 10 | 0 / 10 |
Outcome results
Primary
Response Rate at 6 Weeks After Switching
The percentage of patients who have reached platelet count ≥ 50×10\^9/L at 6 weeks after switching.
Time frame: 6 weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Eltrombopag Group | Response Rate at 6 Weeks After Switching | 10 Participants |
| Recombinant Human Thrombopoietin (Rh-TPO) Group | Response Rate at 6 Weeks After Switching | 10 Participants |
Secondary
DOR (Duration of Response)
The duration of achieving platelet count ≥ 50×10\^9/L after switching.
Time frame: 6 weeks
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Eltrombopag Group | DOR (Duration of Response) | 3 weeks |
| Recombinant Human Thrombopoietin (Rh-TPO) Group | DOR (Duration of Response) | 4 weeks |
Secondary
Number of Participants With Bleeding Events
Number of participants with bleeding events of the two groups during 6 weeks after switching
Time frame: 6 weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Eltrombopag Group | Number of Participants With Bleeding Events | 2 Participants |
| Recombinant Human Thrombopoietin (Rh-TPO) Group | Number of Participants With Bleeding Events | 4 Participants |
Secondary
Reasons of Switching
Reasons of switching eltrombopag and rh-TPO will be recorded, including lack of efficacy, patient preference, side effects, platelet count fluctuation
Time frame: 6 weeks
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Eltrombopag Group | Reasons of Switching | lack of efficacy | 2 Participants |
| Eltrombopag Group | Reasons of Switching | patient preference | 5 Participants |
| Eltrombopag Group | Reasons of Switching | side effects | 3 Participants |
| Eltrombopag Group | Reasons of Switching | platelet count fluctuation | 0 Participants |
| Recombinant Human Thrombopoietin (Rh-TPO) Group | Reasons of Switching | platelet count fluctuation | 2 Participants |
| Recombinant Human Thrombopoietin (Rh-TPO) Group | Reasons of Switching | lack of efficacy | 2 Participants |
| Recombinant Human Thrombopoietin (Rh-TPO) Group | Reasons of Switching | side effects | 5 Participants |
| Recombinant Human Thrombopoietin (Rh-TPO) Group | Reasons of Switching | patient preference | 1 Participants |
Secondary
TOR (Time to Response)
The time to achieve platelet count ≥ 50×10\^9/L after switching.
Time frame: 6 weeks
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Eltrombopag Group | TOR (Time to Response) | 5 weeks |
| Recombinant Human Thrombopoietin (Rh-TPO) Group | TOR (Time to Response) | 2 weeks |
Secondary
Treatments Associated Adverse Events
Adverse event/serious adverse event associated with study drugs during 6 weeks after switching
Time frame: 6 weeks
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eltrombopag Group | Treatments Associated Adverse Events | 3 participants |
| Recombinant Human Thrombopoietin (Rh-TPO) Group | Treatments Associated Adverse Events | 1 participants |