Using ibrutinib to treat autoimmune hemolytic anemia in patients with chronic lymphocytic leukemia

Ibrutinib for the Treatment of Autoimmune Hemolytic Anemia in Patients With Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma or CLL-like Monoclonal B-cell Lymphocytosis

Phase 2 Interventional Gruppo Italiano Malattie EMatologiche dell'Adulto · NCT05694312

This study is testing if the drug ibrutinib can help adults with chronic lymphocytic leukemia who also have autoimmune hemolytic anemia feel better.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment45 (estimated)
Ages18 Years and up
SexAll
SponsorGruppo Italiano Malattie EMatologiche dell'Adulto Academic / other
Drugs / interventionsrituximab, ibrutinib
Locations3 sites (Florence and 2 other locations)
Trial IDNCT05694312 on ClinicalTrials.gov

What this trial studies

This multicenter, phase II study evaluates the efficacy of ibrutinib in treating autoimmune hemolytic anemia (AIHA) in adult patients diagnosed with chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), or CLL-like monoclonal B-cell lymphocytosis (MBL). Participants will receive ibrutinib at a dosage of 420 mg per day for up to 12 cycles of 28 days, provided there is no progression of CLL or unacceptable toxicity. The study includes a follow-up period of one year after treatment completion to assess long-term outcomes.

Who should consider this trial

Good fit: Ideal candidates are adults over 18 with a diagnosis of CLL/SLL or CLL-like MBL and active AIHA that is either relapsed, steroid-resistant, or steroid-dependent.

Not a fit: Patients with DAT-negative active AIHA or those who do not meet the inclusion criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from AIHA associated with CLL/SLL.

How similar studies have performed: While the use of ibrutinib has been explored in other contexts, this specific application for AIHA in CLL/SLL is novel and has not been extensively tested in prior studies.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

1. Diagnosis of CLL/small lymphocytic lymphoma (SLL) or CLL-like monoclonal B-cell lymphocytosis (MBL) according to IWCLL guidelines.
2. Patients \>18 years old
3. Active AIHA (wAIHA or CAD) that i) is relapsed after previous treatment with corticosteroids (with or without rituximab), or ii) is steroid-resistant (failure to obtain hematologic response within 3 weeks on at least 1 mg/kg predniso(lo)ne), or iii) is steroid-dependent (need to continue on predniso(lo)ne at a dose of \>10 mg/day to maintain a response). AIHA is defined as: anemia (hemoglobin ≤10 g/dL; or hemoglobin \>10 g/dL dependent on transfusions to maintain this level of hemoglobin) and laboratory evidence of hemolysis (presence of 3 of 4 markers: increased reticulocyte count, increased indirect bilirubin, increased lactate dehydrogenase, decreased haptoglobin) and positive DAT (either IgG DAT, C3 DAT or both).
4. Eligibility of patients with DAT-negative active AIHA should be confirmed by the Principal Investigator and co-Principal Investigator for the trial.
5. Signed written informed consent according to ICH/EU/GCP and national local laws.

Exclusion Criteria:

1. Contraindication to ibrutinib therapy as per treating physician's discretion.
2. Contraindication to ibrutinib therapy as per ibrutinib data sheet (severe hepatic impairment, known allergy to the drug or to one of the excipients, concomitant treatment with warfarin or other vitamin K antagonists).
3. Previous exposure to ibrutinib as CLL-directed therapy.
4. Other CLL/SLL- or AIHA-directed treatment at the time of enrollment in the study, other than corticosteroids.
5. Female patients who are currently in pregnancy or are willing to be pregnant or are lactating.

Where this trial is running

Florence and 2 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Autoimmune Hemolytic AnemiaChronic Lymphocytic LeukemiaSmall Lymphocytic LymphomaMonoclonal B-Cell Lymphocytosis CLL-Type
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.