Long-term Safety Study of Treatment With the Thrombopoietin Agonists Eltrombopag and Romiplostim in Patients With Immune Thrombocytopenia (ITP)

Long-term Safety Study of Treatment With the Thrombopoietin Agonists Eltrombopag and Romiplostim in Patients With Primary Immune Thrombocytopenia (ITP)

Status

Withdrawn

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01443351

Enrollment

Registered

2011-09-29

Start date

2015-03-31

Completion date

2019-08-31

Last updated

2019-09-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Idiopathic Thrombocytopenic Purpura

Keywords

ITP, romiplostim, eltrombopag, bone marrow fibrosis, long-term safety

Brief summary

Thrombopoietin Receptor Agonists (TPO-ra) are novel treatment modalities for patients with refractory Primary Immune Thrombocytopenia (ITP), but only few data are available for long-term effects of these drugs. In this observational study, effects and adverse effects including evaluation of bone marrow biopsies done at fixed intervals will be recorded from ITP patients treated with TPO-ra. For some patients, blood samples will be collected for research use.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Adult patients with ITP * platelet counts \< 25 x10\*9/L or \< 50 x10\*9/L with bleeding symptoms * meets criteria for treatment with TPO-ra * Females must use contraceptives when applicable for at least three months before inclusion

Exclusion criteria

* Pregnancy or nursing * Former thromboembolic events excluding one incidence of deep venous thrombosis as complication to surgery or pregnancy or one incidence of cerebral embolism as complication to atrial fibrillation * Liver insufficiency (for eltrombopag only) * TPO-ra contraindications (e.g. allergy) * TPO-ra treatments less than 6 months prior to inclusion

Design outcomes

Primary

Measure	Time frame	Description
Number of patients with sustained response at 6 months follow-up	6 months	Response is defined as platelet count \> 30.000 mio/L and no bleeding

Secondary

Measure	Time frame	Description
Yearly response rates	5 years	Number of patients with response to treatment at 1, 2, 3, 4, and 5-year follow-up
Frequency of relapse	5 years	—
Rate of splenectomy	5 years	—
Development of reticulin fibrosis in bone marrow biopsies	5 years	—

Countries

Denmark

Outcome results