The ITP-RITUX Cohort: Rituximab in Immune ThrombocytoPenia. (ITP-RITUX)

The ITP-RITUX Cohort: An Observational Study on Rituximab Off-label Use for Immune ThrombocytoPenia.

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.

Study Overview

• Status: Unknown
• Conditions: Purpura, Thrombocytopenic, Idiopathic; Autoimmune 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.

• Study Type: Observational
• Enrollment (Anticipated): 250

Contacts and Locations

• Study Contact: Name: Bertrand GODEAU, MD; Phone Number: 0033149812900; Email: bertrand.godeau@hmn.aphp.fr
• Study Contact Backup: Name: Mehdi KHELLAF, MD; Phone Number: 0033149812076; Email: mehdi.khellaf@hmn.aphp.fr

Study Locations

• France
  • Val de Marne
    • Creteil, Val de Marne, France, 94010
      • Recruiting
      • Henri Mondor University Hospital
      • Contact: Mehdi KHELLAF, MD; Phone Number: 0033149812076; Email: mehdi.khellaf@hmn.aphp.fr
      • Sub-Investigator: Mehdi KHELLAF, MD
    • Creteil, Val de Marne, France, 94010
      • Recruiting
      • National Reference Center for the Study of Auto Immune Cytopenia
      • Contact: Mehdi KHELLAF, MD; Phone Number: 0033149812076; Email: mehdi.khellaf@hmn.aphp.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Immune ThrombocytoPenia (ITP) patients according to the American Society of Hematology (ASH).

Description

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 Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of a serious adverse events (clinical or biological events)	reaction during perfusions, hypogammaglobulinemia, neutropenia,etc...	5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Impact of rituximab on the natural history of ITP	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.	5 years
Modality of the administration of rituximab	number of perfusions, dosage, retreatment.	5 years
Characteristics of the patients receiving Rituximab	age, sex, duration of ITP, previous used treatment	5 years
Evaluation of the Platelet count evolution	Platelet count estimated at Month 1, 3 and then every 6 months during the 5 years of follow-up.	5 years
Rate of splenectomy in the cohort		5 years

Collaborators and Investigators

• Sponsor: Henri Mondor University Hospital
• Collaborators: Roche Pharma AG
• Investigators: Principal Investigator: Bertrand GODEAU, MD, National Reference Center for Study of Cytopenia

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start: April 1, 2010
• Primary Completion (Anticipated): April 1, 2012
• Study Completion (Anticipated): April 1, 2017

Study Registration Dates

• First Submitted: April 5, 2010
• First Submitted That Met QC Criteria: April 8, 2010
• First Posted (Estimate): April 9, 2010

Study Record Updates

• Last Update Posted (Estimate): March 21, 2012
• Last Update Submitted That Met QC Criteria: March 20, 2012
• Last Verified: March 1, 2012

More Information

Terms related to this study

• Keywords: safety; Rituximab; tolerance; Immune Thrombocytopenia; side effects; splenectomy; serum sickness
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
• Other Study ID Numbers: GECAI