Autoimmune Thrombocytopenia, Purpura, Thrombocytopenic, Idiopathic
Conditions
Keywords
Immune Thrombocytopenia, Rituximab, side effects, tolerance, safety, splenectomy, serum sickness
Brief summary
The primary purpose of the study is to describe by a prospective observational study the serious adverse events occurring in patients treated off-label by rituximab for Immune Thrombocytopenia.
Detailed description
Rituximab (RTX) is used in the treatment of malignant non-Hodgkin's indication for which it has been authorized since 1997 and why it is considered effective and well tolerated. Rituximab is also effective and well tolerated in combination with methotrexate in severe rheumatoid arthritis (RA) refractory to anti-TNF. To date, the RA is the only autoimmune disease in which rituximab has proven efficacy in randomized trials and has obtained authorization in this indication. There are also data from the literature, for the benefit of rituximab in other autoimmune diseases like Lupus, cryoglobulinemia associated to Hepatitis C or Sjogren's disease. Immune Thrombocytopenia (ITP) is an autoimmune disease in which there is thrombocytopenia due in part to destruction of platelets by autoantibodies. In adults, the disease is most often chronic and in the most severe forms, the treatment of choice is splenectomy. Rituximab was suggested during chronic ITP when used with 4 weekly infusions of 375 mg/m2, 60% of patients achieve an immediate response and 30 to 40% a prolonged response. These encouraging results and the apparent good tolerance advocate for using rituximab as first-line treatment for patients refractory to splenectomy. In France, Afssaps - French Health Products Safety Agency has recently issued a temporary protocol processing to authorize rituximab in this indication. Nevertheless, many questions remain unresolved, including the tolerance of long-term treatment and the risk of late relapse in responders. This justifies the creation of this register, whose establishment is recommended by Afssaps - French Health Products Safety Agency.
Interventions
None listed
Sponsors
Roche Pharma AG
Henri Mondor University Hospital
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* ITP diagnosis according to the American Society of Hematology society * Secondary ITP if the underlying disease is an autoimmune disease(Lupus,Sjogren..)
Exclusion criteria
* Previous treatment by rituximab * Secondary ITP associated to a hematologic disease (Chronic Lymphocytic Leukemia, Hodgkin Disease...) * Secondary ITP associated to a chronic infectious disease (Hepatitis B, C, HIV)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Occurrence of a serious adverse events (clinical or biological events) | 5 years | reaction during perfusions, hypogammaglobulinemia, neutropenia,etc... |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Impact of rituximab on the natural history of ITP | 5 years | Number of patients in complete response (platelet count\>100G/L), in partial response (platelet count\>50G/L and at least doubling the baseline platelet count)or in failure. Rate of diminution for the concomittant therapies for ITP. Use of emergencies treatment for ITP. |
| Modality of the administration of rituximab | 5 years | number of perfusions, dosage, retreatment. |
| Characteristics of the patients receiving Rituximab | 5 years | age, sex, duration of ITP, previous used treatment |
| Evaluation of the Platelet count evolution | 5 years | Platelet count estimated at Month 1, 3 and then every 6 months during the 5 years of follow-up. |
| Rate of splenectomy in the cohort | 5 years | — |
Countries
France
Contacts
Primary ContactBertrand GODEAU, MD
Backup ContactMehdi KHELLAF, MD
Outcome results
None listed