What side effects are associated with this type of intervention?

Common treatments for Autoimmune Hemolytic Anemia (AIHA) include corticosteroids, immunosuppressive drugs, and enzyme inhibitors like Acalabrutinib. Corticosteroids can cause side effects such as weight gain, hypertension, and increased risk of infections. Immunosuppressive drugs may lead to bone marrow suppression, liver toxicity, and increased susceptibility to infections. Acalabrutinib, an enzyme inhibitor, may cause headaches, diarrhea, and an increased risk of infections. It is important to discuss these potential side effects with a healthcare provider to determine the most appropriate treatment plan.

What prior FDA approvals exist?

There is limited specific information on prior FDA approvals for enzyme inhibitors specifically for Autoimmune Hemolytic Anemia (AIHA). However, Acalabrutinib, which is being studied for relapsed or refractory AIHA in patients with chronic lymphocytic leukemia, works by inhibiting Bruton's tyrosine kinase (BTK), an enzyme crucial for the growth of cancer cells. Similar enzyme inhibition therapies, such as Ibrutinib, have been approved for other hematologic conditions like chronic lymphocytic leukemia, indicating a potential therapeutic approach for AIHA through enzyme inhibition.

What other types of research exist?

Based on the provided context, there are no specific novel alternatives to Acalabrutinib for Autoimmune Hemolytic Anemia mentioned. However, treatments like rituximab, which is used in other autoimmune conditions, could be considered as potential alternatives. Consulting with a healthcare provider for the latest treatment options and clinical trials is recommended.

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Bruton's Tyrosine Kinase (BTK) Inhibitor

Acalabrutinib for Autoimmune Hemolytic Anemia in Chronic Lymphocytic Leukemia

Q: What is the mechanism of action of this treatment?

Acalabrutinib, a Bruton tyrosine kinase (BTK) inhibitor, works by blocking enzymes necessary for the growth and survival of certain cancer cells, including those involved in autoimmune hemolytic anemia (AIHA). This inhibition can reduce the abnormal immune response that leads to the destruction of red blood cells in AIHA patients. Other common treatments for AIHA include corticosteroids, which suppress the immune system to decrease the production of antibodies against red blood cells, and immunosuppressive drugs like cyclophosphamide, which reduce the overall activity of the immune system. These mechanisms are crucial for AIHA patients as they help to control the immune-mediated destruction of red blood cells, thereby improving symptoms and preventing complications.

Phase 2

Waitlist Available

Led By Alexey Danilov

Research Sponsored by City of Hope Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Anemia (hemoglobin [Hgb] =< 10 g/dL; or Hgb > 10 g/dL dependent on transfusions or maintenance therapy (rituximab, cyclosporin, etc) to maintain this level of hemoglobin, and

Histologically or flow cytometry confirmed diagnosis of CLL/small lymphocytic lymphoma (SLL)

Must not have

Has difficulty with or is unable to swallow oral medication, or has significant gastrointestinal disease that would limit absorption of oral medication

Significant cardiovascular disease such as uncontrolled or symptomatic arrhythmias, congestive heart failure, or myocardial infarction within 6 months of screening, or any class 3 or 4 cardiac disease as defined by the New York Heart Association Functional Classification, or left ventricular ejection fraction (LVEF) =< 40%

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 30 days after last dose of study drug

Awards & highlights

No Placebo-Only Group

Summary

This trial studies acalabrutinib, a medication that may help stop cancer cell growth in patients with chronic lymphocytic leukemia and certain types of anemia. The medication works by blocking enzymes essential for cancer cell growth.

Who is the study for?

This trial is for patients with chronic lymphocytic leukemia who have autoimmune hemolytic anemia that hasn't improved or has returned after treatment. Participants must be able to swallow pills, not have severe infections like HIV or hepatitis, and women of childbearing age must use effective birth control. People can't join if they've had certain recent treatments, transplants, uncontrolled infections, significant heart issues, are pregnant/breastfeeding, or can't follow the study plan.

What is being tested?

The trial is testing acalabrutinib's effectiveness in treating relapsed or refractory autoimmune hemolytic anemia in chronic lymphocytic leukemia patients. It's a phase II study looking at how well acalabrutinib blocks enzymes needed for cancer cell growth.

What are the potential side effects?

Acalabrutinib may cause side effects such as bleeding problems, heart rhythm issues, high blood pressure, headache and diarrhea. Some people might experience muscle pain, fatigue or bruising easily.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have anemia or need treatment to keep my hemoglobin above 10 g/dL.

Select...

My diagnosis of CLL or SLL is confirmed by specific lab tests.

Select...

My CLL has returned or didn't respond to initial treatment with prednisone, with or without rituximab.

Select...

My kidney function, measured by creatinine clearance, is adequate.

Select...

I can perform all my self-care but may not be able to do heavy physical work.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I cannot swallow pills or have a stomach condition that affects medication absorption.

Select...

I do not have serious heart problems like recent heart attacks or severe heart failure.

Select...

I have had cancer before, but it's not currently active.

Select...

I currently have an infection that is not under control.

Select...

I had skin cancer (not melanoma) or melanoma in situ, but it's now treated with no signs of the disease.

Select...

I have not taken Ibrutinib, Acalabrutinib, or similar drugs in the last year.

Select...

I had a stem cell transplant within the last year or am on immunosuppressants for cGVHD.

Select...

I do not have an active hepatitis B or C infection.

Select...

I had early-stage cancer (like breast or cervical) that was treated, and now I show no signs of the disease.

Select...

I haven't taken more than 60 mg/day of prednisone or its equivalent for over 2 weeks in the last month.

Select...

I have been diagnosed with HIV.

Select...

I am taking vitamin K antagonists.

Select...

I have or might have a brain infection known as PML.

Select...

I have not had major surgery in the last 28 days.

Select...

My prostate cancer is being monitored without active treatment.

Select...

I have tested negative for active hepatitis B infection.

Select...

I haven't had chemotherapy, radiation, or immunotherapy in the last 21 days.

Select...

I have a history of bleeding disorders or currently have active bleeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 30 days after last dose of study drug

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 30 days after last dose of study drug

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 30 days after last dose of study drug for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Autoimmune Hemolytic Anemia (AIHA) - Overall Response Rate (ORR)

Secondary study objectives

Autoimmune hemolytic anemia - overall response rate

Autoimmune hemolytic anemia - specific relapse-free survival (RFS)

Autoimmune hemolytic anemia sustained response

+5 more

Other study objectives

Percentage of T-cell subsets among study participants

Side effects data

From 2020 Phase 2 trial • 177 Patients • NCT04346199

Headache

Septic shock

Ischaemic stroke

Chronic obstructive pulmonary disease

100%

80%

60%

40%

20%

Study treatment Arm

BSC Alone

Acalabrutinib + BSC

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (acalabrutinib)Experimental Treatment1 Intervention

Patients receive acalabrutinib PO BID on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Treatment with acalabrutinib may be continued beyond 12 cycles for a maximum of 36 cycles if, in the opinion of the treating physician, the patient might benefit from ongoing therapy.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Acalabrutinib

2020

Completed Phase 2

~2080

0 / 23Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Acalabrutinib, a Bruton tyrosine kinase (BTK) inhibitor, works by blocking enzymes necessary for the growth and survival of certain cancer cells, including those involved in autoimmune hemolytic anemia (AIHA). This inhibition can reduce the abnormal immune response that leads to the destruction of red blood cells in AIHA patients. Other common treatments for AIHA include corticosteroids, which suppress the immune system to decrease the production of antibodies against red blood cells, and immunosuppressive drugs like cyclophosphamide, which reduce the overall activity of the immune system. These mechanisms are crucial for AIHA patients as they help to control the immune-mediated destruction of red blood cells, thereby improving symptoms and preventing complications.