Ibrutinib Effective As First-line Therapy in Waldenström's Macroglobulinemia

Share this content:
The efficacy of ibrutinib as a first-line therapy for WM has not been extensively examined.
The efficacy of ibrutinib as a first-line therapy for WM has not been extensively examined.

Ibrutinib is a safe and highly active treatment among previously untreated patients with Waldenström's macroglobulinemia (WM), according to a poster to be presented at the 2017 American Society of Hematology (ASH) Annual Meeting.

Ibrutinib — a Bruton tyrosine kinase (BTK) inhibitor — is currently used to treat patients with WM who have previously received treatment, but its efficacy as a first-line therapy in treatment-naïve patients is unknown.

Continue Reading Below

For this prospective phase 2 study (ClinicalTrials.gov Identifier: NCT02604511), researchers enrolled 30 patients with untreated WM and administered ibrutinib 420 mg daily. Patients' baseline characteristics included median serum IgM of 4369, and median bone marrow disease involvement was 65%. All patients had MYD88 mutation-positive disease, and 47% of patients had the CXCR4 mutation.

Median time on therapy was 8.1 months; median time on therapy for patients with CXCR4 wild-type and CXCR4 mutation was 9.4 months compared with 8.0 months, respectively (P = .98). The overall response rate was 96.7%, major response rate (greater than partial response) was 80%, and very good partial response (VGPR) was achieved by 17% of patients. No patients achieved complete response.

Median serum IgM levels decreased from 4380 to 1786 (P =.0001) at best response; at baseline, 60% of patients had a serum IgM greater than 3000 mg/dL compared with just 7% of patients at best response (P <.0001). At best response, median bone marrow involvement was reduced to 20% from 65% (P <.0001), and 70% and 80% of patients with adenopathy and splenomegaly, respectively, had a reduction or resolution of these conditions. Patients also had an increase in median hemoglobin levels, from 10.3 to 13.6 g/dL (P <.0001).

Mutated CXCR4 was associated with delayed patient response to ibrutinib.

The authors concluded that “[o]ur findings provide the first report of activity and safety of ibrutinib in previously untreated and symptomatic patients with Waldenström's macroglobulinemia, and show that ibrutinib is highly active and well-tolerated as a single agent, with no unexpected toxicities.”


Treon SP, Gustine J, Meid K, et al. Ibrutinib is highly active as first line therapy in symptomatic Waldenstrom's macroglobulinemia. Oral presentation at: American Society of Hematology 59th Annual Meeting & Exposition; December 9-12, 2017; Atlanta, GA.

You must be a registered member of ONA to post a comment.

Sign Up for Free e-newsletters

Regimen and Drug Listings


Bone Cancer Regimens Drugs
Brain Cancer Regimens Drugs
Breast Cancer Regimens Drugs
Endocrine Cancer Regimens Drugs
Gastrointestinal Cancer Regimens Drugs
Genitourinary Cancer Regimens Drugs
Gynecologic Cancer Regimens Drugs
Head and Neck Cancer Regimens Drugs
Hematologic Cancer Regimens Drugs
Lung Cancer Regimens Drugs
Other Cancers Regimens
Rare Cancers Regimens
Skin Cancer Regimens Drugs