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The ITP-RITUX Cohort: Rituximab in Immune ThrombocytoPenia.

Conditions
Purpura, Thrombocytopenic, Idiopathic
Autoimmune Thrombocytopenia
Registration Number
NCT01101295
Lead Sponsor
Henri Mondor University Hospital
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.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
250
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)

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Occurrence of a serious adverse events (clinical or biological events)5 years

reaction during perfusions, hypogammaglobulinemia, neutropenia,etc...

Secondary Outcome Measures
NameTimeMethod
Evaluation of the Platelet count evolution5 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 cohort5 years
Impact of rituximab on the natural history of ITP5 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 rituximab5 years

number of perfusions, dosage, retreatment.

Characteristics of the patients receiving Rituximab5 years

age, sex, duration of ITP, previous used treatment

Trial Locations

Locations (2)

Henri Mondor University Hospital

🇫🇷

Creteil, Val de Marne, France

National Reference Center for the Study of Auto Immune Cytopenia

🇫🇷

Creteil, Val de Marne, France

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