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Tumor Engraftment in a Xenograft Mouse Model of Human Mantle Cell Lymphoma
Published on: March 30, 2018
Acalabrutinib in relapsed or refractory mantle cell lymphoma (ACE-LY-004): a single-arm, multicentre, phase 2 trial
Michael Wang1, Simon Rule2, Pier Luigi Zinzani3
1Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, Houston, TX, USA; Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Background:
Bruton tyrosine kinase is a clinically validated target in mantle cell lymphoma. Acalabrutinib (ACP-196) is a highly selective, potent Bruton tyrosine kinase inhibitor developed to minimise off-target activity.
Methods:
In this open-label, phase 2 study, oral acalabrutinib (100 mg twice per day) was given to patients with relapsed or refractory mantle cell lymphoma, until disease progression or unacceptable toxicity. The primary endpoint was overall response assessed according to the Lugano classification, and safety analyses were done in all participants. This trial is registered with ClinicalTrials.gov, number NCT02213926.
Findings:
From March 12, 2015, to Jan 5, 2016, 124 patients with relapsed or refractory mantle cell lymphoma were enrolled and all patients received treatment; median age 68 years. Patients received a median of two (IQR 1-2) previous therapies. At a median follow-up of 15·2 months, 100 (81%) patients achieved an overall response and 49 (40%) patients achieved a complete response. The Kaplan-Meier estimated medians for duration of response, progression-free survival, and overall survival were not reached; the 12-month rates were 72% (95% CI 62-80), 67% (58-75), and 87% (79-92%), respectively. The most common adverse events were primarily grade 1 or 2 and were headache (47 [38%]), diarrhoea (38 [31%]), fatigue (34 [27%]), and myalgia (26 [21%]). The most common grade 3 or worse adverse events were neutropenia (13 [10%]), anaemia (11 [9%]), and pneumonia (six [5%]). There were no cases of atrial fibrillation and one case of grade 3 or worse haemorrhage. The median duration of treatment was 13·8 months. Treatment was discontinued in 54 (44%) patients, primarily due to progressive disease (39 [31%]) and adverse events (seven [6%]).
Interpretation:
Acalabrutinib treatment provided a high rate of durable responses and a favourable safety profile in patients with relapsed or refractory mantle cell lymphoma. These findings suggest an important role for acalabrutinib in the treatment of this disease population.
Funding:
Acerta Pharma, a member of the AstraZeneca Group.
Insights
Acalabrutinib shows high response rates and durable survival in relapsed or refractory mantle cell lymphoma patients. This Bruton tyrosine kinase inhibitor offers a favorable safety profile, suggesting its potential in treating this patient population.
Area of Science:
- Oncology
- Hematology
- Pharmacology
Background:
- Bruton tyrosine kinase (BTK) is a validated therapeutic target in mantle cell lymphoma (MCL).
- Acalabrutinib (ACP-196) is a selective BTK inhibitor designed for reduced off-target effects.
Purpose of the Study:
- To evaluate the efficacy and safety of acalabrutinib in patients with relapsed or refractory MCL.
- To assess overall response rate (ORR), progression-free survival (PFS), and overall survival (OS) in this patient group.
Main Methods:
- An open-label, phase 2 study administered oral acalabrutinib (100 mg twice daily) to 124 patients with relapsed/refractory MCL.
- Treatment continued until disease progression or unacceptable toxicity, with primary endpoint being ORR per Lugano classification.
Main Results:
- High ORR of 81% (complete response: 40%) observed at a median follow-up of 15.2 months.
- Median duration of response, PFS, and OS were not reached; 12-month rates were 72% (DOR), 67% (PFS), and 87% (OS).
- Common adverse events included headache (38%), diarrhea (31%), fatigue (27%), and myalgia (21%); grade ≥3 neutropenia (10%) and anemia (9%) were most frequent.
Conclusions:
- Acalabrutinib demonstrated significant efficacy with durable responses and a manageable safety profile in relapsed/refractory MCL.
- These findings support acalabrutinib as a promising treatment option for patients with relapsed or refractory MCL.
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