A Single-Arm Pilot Study With Low-Dose Rituximab Plus Standard Oral Prednisone In Idiopathic Autoimmune Hemolytic Anemia

Study Overview
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Brief Summary
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The aim of this prospective study was to evaluate the activity, safety and the duration of the response of low dose rituximab associated with standard oral prednisone as first line therapy in newly diagnosed warm autoimmune hemolytic anemia and cold hemagglutinin disease, and as second line therapy in warm autoimmune hemolytic anemia relapsed after standard oral prednisone. Further aim was to correlate the clinical response to biological parameters (cytokine and anti-erythrocyte antibody production in cultures).

Official Title
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A Single-Arm Pilot Study With Low-Dose Rituximab Plus Standard Oral Prednisone In Idiopathic Autoimmune Hemolytic Anemia

Conditions
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Autoimmune Hemolytic Disease (Cold Type) (Warm Type)

Intervention / Treatment
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* Drug: prednisone, low dose rituximab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Newly diagnosed warm or cold AIHA, defined by symptomatic anemia and positive DAT, in the absence of underlying lymphoproliferative, infectious or neoplastic disease (according to the single Center diagnostic criteria). Idiopathic warm or cold AIHA relapsed after first line treatment with oral prednisone. Aged >18 years ECOG performance status grade 0, 1 or 2 No psychiatric illness that precludes understanding concepts of the trial or signing informed consent Patients who have provided written informed consent prior to study participation, with the understanding that the consent may be withdrawn by the patient at any time without prejudice. Exclusion Criteria: Cell or humoral immunologic deficit (congenital or acquired) Any other co-existing medical or psychological condition that would preclude participation in the study or compromise ability to give informed consent Active bacterial, viral, or fungal infection requiring systemic therapy HIV or HbsAg positive (with HBV-DNA+) or HCV-Ab positive (with HCV-RNA+) patients History of malignancies within 3 years prior to study entry Concomitant immunosuppressive or cytotoxic treatment Positive pregnancy test. Lactation. The presence of associated organ-specific autoimmune diseases do not constitute exclusion criteria. Previous splenectomy does not constitute exclusion criteria

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: prednisone, low dose rituximab|Patients with warm AIHA will receive: Rituximab 100 mg i.v.weekly (fixed dose irrespective of body surface area) for 4 consecutive weeks (days +7, +14, +21, +28), and prednisone 1 mg/kg/die p.o. days from day +1 to day +30, followed by tapering (10 mg/week until 0.5/mg/kg/die, then 5 mg/week until stop) Patients with CHD will receive: Rituximab 100 mg i.v.weekly (fixed dose irrespective of body surface area) for 4 consecutive weeks (days +7, +14, +21, +28)|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| overall response | Response will be evaluated at each clinic visit. Complete response (CR) will be defined as Hb increase >2 g/dL and Hb > 12 g/dL, normalization of all haemolytic markers and no transfusion requirement;Partial response (PR) will be defined as Hb increase >2 g/dL and Hb 10-12 g/dL, and 50% reduction of transfusion. In the remaining cases there is no response. | month +2 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Sustained response (SR) | Sustained response (SR) was defined as Hb > 10 g/dL at month +6 and +12, in the absence of any treatment. | month +6, month +12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Low dose rituximab, WAIHA, CHD