Clinical trial NCT01101295: The ITP-RITUX Cohort: Rituximab in Immune ThrombocytoPenia.
Official title: The ITP-RITUX Cohort: An Observational Study on Rituximab Off-label Use for Immune ThrombocytoPenia.
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Purpura, Thrombocytopenic, Idiopathic
Autoimmune Thrombocytopenia
Keywords: Immune Thrombocytopenia
Rituximab
side effects
tolerance
safety
splenectomy
serum sickness
Lead sponsor: Henri Mondor University Hospital
Sponsor class: OTHER
Collaborators: name: Roche Pharma AG
First posted: 2010-04-09
Enrollment: 250
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Immune ThrombocytoPenia (ITP) patients according to the American Society of Hematology (ASH).

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.

Eligibility criteria: 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)

Primary outcomes:
measure: Occurrence of a serious adverse events (clinical or biological events)
timeFrame: 5 years
Secondary outcomes:
measure: Impact of rituximab on the natural history of ITP
timeFrame: 5 years
measure: Modality of the administration of rituximab
timeFrame: 5 years
measure: Characteristics of the patients receiving Rituximab
timeFrame: 5 years
measure: Evaluation of the Platelet count evolution
timeFrame: 5 years
measure: Rate of splenectomy in the cohort
timeFrame: 5 years
Other outcomes:
Locations:
city: Creteil
country: France
facility: Henri Mondor University Hospital
state: Val de Marne
city: Creteil
country: France
facility: National Reference Center for the Study of Auto Immune Cytopenia
state: Val de Marne
Study officials:
affiliation: National Reference Center for Study of Cytopenia
name: Bertrand GODEAU, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Khellaf M, Charles-Nelson A, Fain O, Terriou L, Viallard JF, Cheze S, Graveleau J, Slama B, Audia S, Ebbo M, Le Guenno G, Cliquennois M, Salles G, Bonmati C, Teillet F, Galicier L, Hot A, Lambotte O, Lefrere F, Sacko S, Kengue DK, Bierling P, Roudot-Thoraval F, Michel M, Godeau B. Safety and efficacy of rituximab in adult immune thrombocytopenia: results from a prospective registry including 248 patients. Blood. 2014 Nov 20;124(22):3228-36. doi: 10.1182/blood-2014-06-582346. Epub 2014 Oct 7.