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

Sponsor

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico (Other)

Overall Status

Completed

CT.gov ID

NCT01345708

Collaborator

University Hospital, Udine, Italy (Other)

Enrollment

Locations

Duration (Months)

11.5

Patients Per Site

0.5

Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Autoimmune Hemolytic Disease (Cold Type) (Warm Type)	Drug: prednisone, low dose rituximab	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

23 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Jan 1, 2009

Actual Primary Completion Date :

Jan 1, 2010

Actual Study Completion Date :

Jan 1, 2011

Outcome Measures

Primary Outcome Measures

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

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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico	MIlan		Italy	20122
2	Wilma Barcellini MD, UO Ematologia 2, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico	Milan		Italy	20122