Clinical trial NCT03119974: Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in ITP
Official title: Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in Immune Thrombocytopenia: a Prospective Multicenter Open Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Persistent or Chronic ITP
Keywords: ITP
TPO-RAS
Thrombopoietin-receptor agonists
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2017-04-19
Enrollment: 48
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Thrombopoietin-receptor agonists (Tpo-RAs) have profoundly changed the management of ITP. However, today, there are no international recommendations concerning the long-term use of these costly, potentially pro-thrombotic agents, and that could induce bone marrow fibrosis in case of prolonged treatment. Tpo-RAs have been thought to play only a supporting role in ITP management. But our center along with many other research centers, have reported unexpected cases of durable remission after Tpo-RAs discontinuation in adult chronic ITP. In these retrospective studies, more than 20 % of patients were able to achieve prolonged remission.

The purpose of this study is to demonstrate that a substantial proportion of ITP patients may achieve a prolonged response after Tpo-RA discontinuation.

The investigators developed, in this study, a standardized procedure to discontinue Eltrombopag and Romiplostim, wherein the dose will be slowly tapered to limit the risk of bleeding. In case of relapse after Tpo-RA discontinuation, the decision to start a new therapy will be based on the clinician's judgment.


Eligibility criteria: Inclusion Criteria:

1. Age \>= 18 years
2. Diagnosis of ITP according to the standard definition
3. Disease duration of more than 3 months at Tpo-RA initiation
4. Platelet count \> 100 x 109/L for more than 2 months on Tpo-RA Therapy, with at least 3 platelet counts \> 100 x 109/L
5. Blood count lasting for less than 7 days
6. Normal marrow aspirate for patients aged of 60 and over
7. Informed signed consent
8. Treatment with Tpo-RA for at least 3 months

Exclusion Criteria:

1) Anticoagulation or anti-platelet treatment 2) Recent treatment with corticosteroids ± intravenous immunoglobulins (less than 2 months) 3) Rituximab or splenectomy within the 2 months preceding the Tpo-RA initiation 4) Rituximab or splenectomy after Tpo-RA initiation/RA initiation 5) Previous failure of Tpo-RA discontinuation 6) Pregnant or breastfeeding women 7) No affiliation to a social security scheme or other social protection scheme 8) Inability or refusal to understand or refusal to sign the informed consent from study participation 9) Patient deprived of freedom or under legal protection (guardianship, curatorship) 10) Hypersensitivity to Romiplostin or to any of the excipients or to E. coli derived proteins

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Primary outcomes:
measure: The proportion of patients achieving an overall response (complete response and response) at week 24 (6 months). The criteria of response will be defined according to international terminology
timeFrame: week 24 (6 months)
Secondary outcomes:
measure: The rate of overall response after Tpo-RAs discontinuation
timeFrame: 24 and 52 weeks
measure: The duration of overall response after Tpo-RAs discontinuation.
timeFrame: 24 and 52 weeks
measure: The number of bleeding events during the reduction period and along the study period at weeks 4, 8, 12,24,36, 52
timeFrame: at weeks 4, 8, 12,24,36, 52
measure: The rate of the response to a new course of Tpo-RAs in case of relapse after Tpo-RAs discontinuation at one year
timeFrame: 52 weeks
measure: The delay of the response to a new course of Tpo-RAs in case of relapse after Tpo-RAs discontinuation
timeFrame: 52 weeks
measure: To identify predictive factors, for overall prolonged response
timeFrame: Weeks 24
Other outcomes:
Locations:
city: Creteil
country: France
facility: Henri-Mondor Hospital
Study officials:
affiliation: Assistance Publique - Hôpitaux de Paris
name: Matthieu Mahevas, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: