Rituximab in Patients With Relapsed or Refractory TTP-HUS
A Phase II Study Evaluating the Efficacy of Rituximab in the Management of Patients With Relapsed/Refractory Thrombotic Thrombocytopenic Purpura (TTP) - Hemolytic Uremic Syndrome (HUS)
  • Phase

    Phase 2
  • Study Type

  • Status

    Unknown status
  • Intervention/Treatment

    rituximab ...
  • Study Participants

The general objective of this study is to assess the efficacy and safety of Rituximab in the management of patients with refractory or relapsed thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS). There have been several case reports and case series describing the use of Rituximab in patients with TTP-HUS; however its use has not been studied in a large trial. It is hypothesized that Rituximab may ameliorate the severity of certain cases of TTP-HUS by decreasing the number of activity of B-cells which may result in decreased production of the ADAMTS13 protease inhibitor. Patients with TTP-HUS not responding to standard therapy or patients with relapsed disease may have particular benefit. Treatments that decrease the frequency of relapse or shorten the time to remission of TTP-HUS will be of benefit by decreasing the need for blood product support.
Study Started
Dec 31
Primary Completion
Jan 31
Study Completion
Jan 31
Last Update
May 19

Drug Rituximab

Rituximab will be administered on weeks 1, 2, 3, and 4 at a dose of 375 mg/m2 per infusion. Premedications (prednisone 50 mg, diphenhydramine 50 mg, acetaminophen) will be administered prior to study infusion. Patients will also be treated with plasma exchange as per institution/apheresis centre.

  • Other names: Rituxan, rituximab

Study group Experimental

All patients in the study will be in the study group and will receive rituximab. There is no "control" arm.


Inclusion Criteria:

any patient 18 years or older diagnosed with relapsed or refractory TTP-HUS requiring therapy

Exclusion Criteria:

alternate cause of hemolytic microangiopathy (evidence of DIC, malignant hypertension, vasculitis, anti-phospholipid antibody syndrome, post-partum acute renal failure)
congenital or familial TTP
TTP occuring post-stem cell, bone marrow, or solid organ transplant
drug-induced TTP
pregnancy or breast-feeding
history of hepatitis B or C infection
prior rituximab treatment
active or metastatic cancer
other causes of thrombocytopenia such as ITP, myelodysplastic syndrome, confirmed or suspected drug-induced thrombocytopenia
refusal to receive blood products
hypersensitivity to blood products, plasma products, murine proteins, or any component of the Rituximab formulation
geographic inaccessibility
co-morbid illness limiting life expectancy to less than 2 months independent of TTP
failure to provide written informed consent
No Results Posted