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

This study has been completed.
Sponsor:
Collaborator:
University Hospital, Udine, Italy
Information provided by:
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
ClinicalTrials.gov Identifier:
NCT01345708
First received: April 28, 2011
Last updated: April 29, 2011
Last verified: April 2011
  Purpose

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

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico:

Primary Outcome Measures:
  • overall response [ Time Frame: month +2 ] [ Designated as safety issue: Yes ]
    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) [ Time Frame: month +6, month +12 ] [ Designated as safety issue: Yes ]
    Sustained response (SR) was defined as Hb > 10 g/dL at month +6 and +12, in the absence of any treatment.


Enrollment: 23
Study Start Date: January 2009
Study Completion Date: January 2011
Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)
Intervention Details:
    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)

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01345708

Locations
Italy
Wilma Barcellini MD, UO Ematologia 2, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Milan, Italy, 20122
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
MIlan, Italy, 20122
Sponsors and Collaborators
Fondazione Ospedale
University Hospital, Udine, Italy
Investigators
Study Director: PierMannuccio Mannucci, MD Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F. Sforza 28 20122, Milano.
  More Information

Publications:
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Wilma Barcellini, MD, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano
ClinicalTrials.gov Identifier: NCT01345708     History of Changes
Other Study ID Numbers: Eudra CT 2008-006713-25
Study First Received: April 28, 2011
Last Updated: April 29, 2011
Health Authority: Italy: The Italian Medicines Agency

Keywords provided by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico:
Low dose rituximab
WAIHA
CHD

Additional relevant MeSH terms:
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Hemolysis
Anemia
Hematologic Diseases
Autoimmune Diseases
Immune System Diseases
Pathologic Processes
Prednisone
Rituximab
Anti-Inflammatory Agents
Therapeutic Uses
Pharmacologic Actions
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Immunologic Factors
Antirheumatic Agents

ClinicalTrials.gov processed this record on August 28, 2014