Clinical trial NCT01345708: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autoimmune Hemolytic Disease (Cold Type) (Warm Type)
Keywords: Low dose rituximab
WAIHA
CHD
Lead sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Sponsor class: OTHER
Collaborators: name: University Hospital, Udine, Italy
First posted: 2011-05-02
Enrollment: 23
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


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have provided written informed consent prior to study participation, with the understanding that the consent may be withdrawn by the patient at any time without prejudice.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

Primary outcomes:
measure: overall response
timeFrame: month +2
Secondary outcomes:
measure: Sustained response (SR)
timeFrame: month +6, month +12
Other outcomes:
Locations:
city: MIlan
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
city: Milan
country: Italy
facility: Wilma Barcellini MD, UO Ematologia 2, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Study officials:
affiliation: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via F. Sforza 28 20122, Milano.
name: PierMannuccio Mannucci, MD
role: STUDY_DIRECTOR
References:
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.
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.
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.
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.