Adult patients with immune thrombocytopenia (ITP) with mild to severe bleeding manifestations. MedDRA version: 23.0 Level: PT Classification code 10083842 Term: Immune thrombocytopenia System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Interventions
Trade Name: Neofordex 40 mg comprimés
Pharmaceutical Form: Tablet
INN or Proposed INN: Neofordex 40 mg comprimés
CAS Number: 1177-87-3
Other descriptive name: DEXAMETHASONE ACETATE
Concentration unit:
Sponsors
ASSISTANCE PUBLIQUE-HOPITAUX DE PARIS
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Age = 18 years = 80 years - Diagnosis of ITP according to the standard definition - Platelet count = 20 x 109/L - Any cutaneous and/or any mucosal bleeding manifestations - Normal marrow aspirate for patients aged of 60 and over - Affiliated to a social security regime - Written consent from patient Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 190 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 82
Exclusion criteria
Exclusion criteria: - 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 (HIV, Viral hepatitis, herpes, varicella, zona) - Uncontrolled diabetes - 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate the non-inferiority in term of short-term response of oral dexamethasone given at 40 mg on days 1 to 4 compared to IVIg given at 1g/kg on days 1 and 2 in combination with prednisone (1 mg/kg per day for 3 weeks) in adult patients with immune thrombocytopenia (ITP) with mild to severe bleeding manifestations.;Secondary Objective: To compare initial rate of CR in the two arms and the delay to obtain a CR. To compare the duration of the response in the two arms. To assess the acceptability/feasibility of the protocol, by comparing the proportion of early treatment switches (before day 5) across arms. To estimate the interaction between treatment effect and the severity/risk of bleeding. To compare the initial response on bleeding manifestations in both arms. To compare the duration of hospital, stay in the two arms. To compare the efficacy (overall response rate) at Day 28 and 6 months in the two arms. To compare safety profile in the two arms. To estimate the incremental (decremental) cost effectiveness of DXM vs IVIg at 6 months given the non-inferiority design our hypothesis is that DMX is cheaper and non-inferior decrementally cost effective compared to IVIg . To assess whether the presence of anti-platelet antibodies is associated with the outcome (Chronic or Cured) at 6 months in the two arms.;Primary end point(s): Time (in days) to achieve an initial response (R) within 5 days. Initial response (R) will be defined according to international guidelines (5) as a platelet count = 30x109/L with at least a doubling of the baseline value in the absence of new bleeding and absence of the use of any other ITP directed therapies (other than the one of the treatment arm) Non-Response will be defined as platelet count < 30 x 109/L or new bleeding manifestations or use of any other ITP directed therapies including use of IVIg in the arm of DXM or DXM in the arm of IVIg. ;Timepoint(s) of evaluation of this end point: We will assess the pr | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Time (in days) to achieve an initial complete response (CR) in both arms (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) in the two arms. - Duration in time (in days) of overall response from Day 1 to the end of the study (6 months) in the two arms. - Proportion of early (before day 5) treatment switches across arms - Number of new bleeding manifestations between Day 1 and Day 5 in two arms. Bleeding manifestations will be assessed the score reported by Khellaf et al. without taking into account the age of the patient. - Rates of response (R) and complete response (CR) at 28 days, and 6 months in the two arms. - Number of bleeding manifestations between Day 5 and Day 28 in the two arms. - Number of days of hospitalization between Day 1 and Day 28 in the two arms. - Number of adverse events in the two arms. - Incremental (decremental) cost effectiveness ratio expressed in cost per responder at 6 months - Comparison of the number of responders, and outcome at 6 months in patients with positive and negative anti-platelets antibodies in the two arms. ;Timepoint(s) of evaluation of this end point: We will assess secondary endpoints at 6 months. | — |
Countries
France
Contacts
Public ContactMyriem Carrier
ASSISTANCE PUBLIQUE-HOPITAUX DE PARIS (AP-HP) (AP-HP)
Outcome results
None listed