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

Primary Purpose

Autoimmune Hemolytic Disease (Cold Type) (Warm Type)

Status

Completed

Phase

Phase 2

Locations

Italy

Study Type

Interventional

Intervention

prednisone, low dose rituximab

About this trial

This is an interventional treatment trial for Autoimmune Hemolytic Disease (Cold Type) (Warm Type) focused on measuring Low dose rituximab, WAIHA, CHD

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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who have provided written informed consent prior to study participation, with the understanding that the consent may be withdrawn by the patient at any time without prejudice.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

Sites / Locations

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Wilma Barcellini MD, UO Ematologia 2, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Sustained response (SR)

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

Full Information

NCT ID

NCT01345708

First Posted

April 28, 2011

Last Updated

April 29, 2011

Sponsor

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Collaborators

University Hospital, Udine, Italy

1. Study Identification

Unique Protocol Identification Number

NCT01345708

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2011

Overall Recruitment Status

Completed

Study Start Date

January 2009 (undefined)

Primary Completion Date

January 2010 (Actual)

Study Completion Date

January 2011 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Collaborators

University Hospital, Udine, Italy

4. Oversight

Data Monitoring Committee

Yes

5. 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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Autoimmune Hemolytic Disease (Cold Type) (Warm Type)

Keywords

Low dose rituximab, WAIHA, CHD

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

23 (Actual)

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

prednisone, low dose rituximab

Intervention Description

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)

Primary Outcome Measure Information:

Title

overall response

Description

Time Frame

month +2

Secondary Outcome Measure Information:

Title

Sustained response (SR)

Description

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

Time Frame

month +6, month +12

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

PierMannuccio Mannucci, MD

Organizational Affiliation

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F. Sforza 28 20122, Milano.

Official's Role

Study Director

Facility Information:

Facility Name

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

City

MIlan

ZIP/Postal Code

20122

Country

Italy

Facility Name

Wilma Barcellini MD, UO Ematologia 2, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

City

Milan

ZIP/Postal Code

20122

Country

Italy

12. IPD Sharing Statement

Citations:

PubMed Identifier

18055995

Citation

Provan D, Butler T, Evangelista ML, Amadori S, Newland AC, Stasi R. Activity and safety profile of low-dose rituximab for the treatment of autoimmune cytopenias in adults. Haematologica. 2007 Dec;92(12):1695-8. doi: 10.3324/haematol.11709.

Results Reference

background

PubMed Identifier

20546023

Citation

Zaja F, Vianelli N, Volpetti S, Battista ML, Defina M, Palmieri S, Bocchia M, Medeot M, De Luca S, Ferrara F, Isola M, Baccarani M, Fanin R. Low-dose rituximab in adult patients with primary immune thrombocytopenia. Eur J Haematol. 2010 Oct;85(4):329-34. doi: 10.1111/j.1600-0609.2010.01486.x. Epub 2010 Jul 28.

Results Reference

background

PubMed Identifier

20841509

Citation

Gomez-Almaguer D, Solano-Genesta M, Tarin-Arzaga L, Herrera-Garza JL, Cantu-Rodriguez OG, Gutierrez-Aguirre CH, Jaime-Perez JC. Low-dose rituximab and alemtuzumab combination therapy for patients with steroid-refractory autoimmune cytopenias. Blood. 2010 Dec 2;116(23):4783-5. doi: 10.1182/blood-2010-06-291831. Epub 2010 Sep 14.

Results Reference

background

PubMed Identifier

22267606

Citation

Barcellini W, Zaja F, Zaninoni A, Imperiali FG, Battista ML, Di Bona E, Fattizzo B, Consonni D, Cortelezzi A, Fanin R, Zanella A. Low-dose rituximab in adult patients with idiopathic autoimmune hemolytic anemia: clinical efficacy and biologic studies. Blood. 2012 Apr 19;119(16):3691-7. doi: 10.1182/blood-2011-06-363556. Epub 2012 Jan 20.

Results Reference

derived

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

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