IgIV Plus Prednisone vs High-dose Dexamethasone for ITP
- Conditions
- Immune Thrombocytopenia (ITP)
- Interventions
- Drug: Intravenous immunoglobulins
- Registration Number
- NCT04968899
- Lead Sponsor
- Assistance Publique - Hôpitaux de Paris
- Brief Summary
ITP patients with low platelet count and active bleeding symptoms are at risk of life-threatening bleeding and therefore require a treatment with a rapid effect, reliable, and sustained. The combination of intravenous immunoglobulin (IVIg) and prednisone (1 mg/kg per day), is more rapidly and more frequently effective than high dose methylprednisolone to increase the platelet count. This combination is therefore usually given in patients with platelets count \< 20 x 109/L and moderate to severe bleeding manifestations. Based on common practice in France and on French ITP guidelines, on average 50 % of patients with ITP and profound thrombocytopenia do actually receive IVIg (mostly during the initial phase of the disease) corresponding to approximately 1,500 ITP patients per year in France.
Whereas IVIg is usually well tolerated, renal insufficiency and congestive heart failure may occur, moreover IVIg are costly and non-easily available with supply difficulties in many countries including France.
High dose dexamethasone (DXM) (ie: 40 mg/d for 4 days) has recently emerged as a promising treatment for ITP. One recent meta-analysis as well as a controlled prospective trial suggest that the initial overall response was higher (\> 80 %) and the time to response was shorter with dexamethasone (DXM) 40 mg/d given for 4 days compared to standard prednisone.
The investigators hypothesize that DXM could be a reasonable non-inferior alternative to IVIg, more convenient for patients with less adverse events and economically cost-effective for patients with moderate and severe bleeding manifestations.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 272
- Age ≥ 18 years ≤ 80 years
- Diagnosis of ITP whatever the duration of the disease (newly diagnosed or relapsed) according to the standard definition
- Platelet count ≤ 20 x 109/L
- Any cutaneous and/or any mucosal bleeding manifestations
- Affiliated to a social security regime
- Written consent from patient
- Symptomatic COVID-19 disease
- Life-threatening bleeding defined as Intracranial hemorrhage and/or active organ bleeding (GI tract, urinary tract or menorrhagia with at least a 2 g/dl decrease of hemoglobin value from baseline).
- Ongoing anticoagulation treatment (Therapeutic Low molecular weight heparins (LMWHs), direct oral anticoagulants (DOACs) and vitamin K antagonists (VKAs))
- Previous non-response to IVIg or DEX
- Treatment with prednisone (1 mg/kg per day) for more than 3 days
- Any, contraindications to the prescribed Ig IV or prednisone patent medicine and to Neofordex®
- Ongoing severe infection
- Severe Renal insufficiency (DFG < 45 ml.min.1.73m2)
- Severe Cardiac insufficiency (FEVG < 30 %)
- Ongoing viral infection (uncontrolled HIV, Viral hepatitis, herpes, varicella, zona).
- Uncontrolled diabetes (Acido-cetosis)
- Psychotic state not yet controlled by treatment
- Inability or refusal to understand or refusal to sign the informed consent from study participation
- Persons deprived of their liberty by judicial or administrative decision,
- Persons under legal protection (guardianship, curatorship)
- Pregnant or breastfeeding woman or ineffective contraception
- Participation in another interventional study involving human participants or being in the exclusion period at the end of a previous study involving human participants.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Control Intravenous immunoglobulins IVIg (1g/kg D1-D2) plus prednisone (1 mg/kg/day x 21 days (3 weeks)) Experimental group Neofordex® Oral dexamethasone (Neofordex®) 40 mg (Day1 to Day 4), ± an additional 4-days cycle of dexamethasone between days 10 and 21
- Primary Outcome Measures
Name Time Method Time to achieve an initial response (R) within 5 days. 5 days
- Secondary Outcome Measures
Name Time Method Proportion of early treatment switches across arms before day 5 Rates of response (R) and complete response (CR) in the two arms. at Day 28 and at 6 months Number of responders in patients with positive and negative anti-platelets antibodies in the two arms. At 6 months Number of outcome in patients with positive and negative anti-platelets antibodies in the two arms. At 6 months Time to achieve an initial complete response (CR) in the two arms between Day 1 and Day 5 complete response (CR): defined by a platelet count \> 100 x 109/L in the absence use of any other ITP directed therapies between Day 1 and Day 5
Duration of overall response from Day 1 to the end of the study in the two arms. Day 1 to 6 months Number of new bleeding manifestations between Day 1 and Day 5 in the two arms. between Day 1 and Day 5 Number of new bleeding manifestations in the two arms. Between Day 5 and Day 28 Number of adverse events in the two arms. up to 6 months
Trial Locations
- Locations (1)
Henri Mondor Hospital
🇫🇷Créteil, France