Corticosteroid-resistant or Relapsed ITP
Conditions
Keywords
Immune Thrombocytopenia, Recombinant human thrombopoietin, avatrombopag, switching
Brief summary
Thrombopoietin receptor agonists (TPO-RAs) represent a highly effective and well-tolerated second-line ITP treatment that provides excellent responses.If there is cross-resistance between 2 drugs for the treatment of adult ITP is still unkonwn.The purpose of this study is to investigate the efficacy and safety of switching avatrombopag and rh-TPO in adults with ITP.
Detailed description
Thrombopoietin Receptor Agonists (TPO-RAs) are novel treatments for patients with chronic Primary Immune Thrombocytopenia (ITP). According to the findings of mechanism-based studies, rhTPO competes with endogenous TPO for binding to TPO-R while avatrombopag has an additive effect with endogenous TPO, indicating that the treatment mechanism and side-effect profiles could be somewhat different between these drugs. 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 avatrombopag and rh-TPO in adults with ITP.This is a non-interventional study. Patients who fail previous steroids and receive rh-TPO and then switch to avatrombopag or vice versa will be enrolled. The reason for switch will be recorded. The efficacy, safety, and patient/physician preference will be assessed and compared between the two agents.
Interventions
None listed
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.Failed initial glucocorticosteroid treatment, 4.Applying rhTPO or Eltrombopag as subsequent treatment 5.Switch from rh-TPO to eltrombopag or vice versa 6.Normal neutrophils 7.Available follow-up at least 6 weeks after switching
Exclusion criteria
1. HIV positive status, or active infection of HBV or HCV 2. Suffering from a serious or progressive disease, which, in the investigator's judgment, put the subject at undue risk for participation in this study (i.e. cancer or pre-cancer, immunocompromised, uncontrolled diabetes, epilepsy, severe cardio-cerebrovascular disease(s) (i.e. stroke, idiopathic aortic stenosis, aneurysm, hypertrophic obstructive cardiomyopathy, ischaemic heart disease, tachyarrhythmias, severe heart failure \[classified as NYHA III-IV\], severe lung dysfunctions, etc)) 3. History of thrombosis plus two or more risk factors as defined in Caprini thrombosis risk assessment model 4. Lactating or pregnant women, or WOCBP who are unwilling to use highly effective contraceptive measures during the study period 5. Abnormal liver and renal functions: AST or ALT or total bilirubin ≥1.5 × ULN, and/or creatinine ≥176.8 μmol/L 6. Women of childbearing potential (WOCBP) that are pregnant or wish to become pregnant during the prospective phase of the study. 7. Other conditions which the investigator considers inappropriate for enrollment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Initial response after switching | 4 weeks | Rate of response at 4 weeks after switching from rhTPO to avatrombopag or vise versa |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Initial response after switching according to the reasons of switching | 4 weeks | Rate of response at 1 month after switching according to the reasons of switching,such as lack of efficacy, Platelet count fluctuations, development of adverse events,patient's or doctor's preference |
| Rate of response at 12 weeks after switching according to the reasons of switching | 12 weeks | Rate of response at 12 weeks after switching according to the reasons of switching,such as lack of efficacy, Platelet count fluctuations, development of adverse events,patient's or doctor's preference |
| Time to response | 4 weeks | Time to CR or R from switching |
| Durable response | 24 weeks | The maintenance of platelet count ≥ 30 x 10\^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 24 weeks follow-up. |
| Response rate at 12 weeks after switching | 12 weeks | Rate of response at 12 weeks after switching from rhTPO to avatrombopag or vise versa |
| Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) | 24 weeks | In all participants ,use ITP-PAQ to assess the Health Related Quality of Life(HRQoL) before and after treatment. |
| Functional Assessment of Chronic Illness Therapy fatigue subscale (FACIT-F) | 24 weeks | In all participants ,use FACIT-F to assess the Health Related Quality of Life(HRQoL) before and after treatment. |
| Safety assessment | 24 weeks | Number of Participants with side effects of the drugs |
| Incidence of bleeding events | 24 weeks | Incidence of clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale |
Contacts
Primary ContactHaixia Fu, MD
Backup ContactYun He, MD
Outcome results
None listed