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

Phase 2

Completed

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

• Registration Number: NCT01345708
• Lead Sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Brief Summary

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).

Detailed Description

Not available

Study Timeline

• Study Start: 2009-01
• Primary Completion: 2010-01
• Study Completion: 2011-01
• Status Verified: 2011-04
• Study First Submitted: 2011-04-28
• Study First Submitted QC: 2011-04-29
• Last Update Submitted: 2011-04-29
• Study First Posted: 2011-05-02
• Last Update Posted: 2011-05-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 23

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who have provided written informed consent prior to study participation, with the understanding that the consent may be withdrawn by the patient at any time without prejudice.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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
overall response	month +2	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.

Secondary Outcome Measures

Name	Time	Method
Sustained response (SR)	month +6, month +12	Sustained response (SR) was defined as Hb \> 10 g/dL at month +6 and +12, in the absence of any treatment.

Trial Locations

Locations (2)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; 🇮🇹 MIlan, Italy

Wilma Barcellini MD, UO Ematologia 2, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico; 🇮🇹 Milan, Italy