Treatment and outcome patterns of patients with Waldenström’s macroglobulinemia: a large, multicenter retrospective review in China

Abstract

In this study, we aimed to investigate treatment options and the prognosis of patients with WM in China. This retrospective study included 1141 patients diagnosed with symptomatic WM between January 2003 and December 2019 at 35 tertiary hospitals in 22 provinces of China. Fifty-four patients (7.3%) received monotherapy, 264 (36.0%) received chemoimmunotherapy, 395 (53.8%) received other combination regimens without rituximab, and 21 (2.9%) received ibrutinib. Using a multivariable Cox regression model, age > 65 years old, platelets <100 × 10⁹/L, serum albumin <3.5 g/dl, β2 microglobulin concentration ≥4 mg/L and LDH ≥250 IU/L predicted poor OS. In summary, our study showed that frontline treatment choices for WM are widely heterogeneous. We validated most of the established prognostic factors in the rIPSS (age >65 years, LDH ≥250 IU/L, ALB <3.5 g/dl and β2 microglobulin ≥4 mg/L) together with PLT ≤ 100 × 10⁹/L indicate a poor prognosis for patients with WM.

Keywords:

• Waldenström’s macroglobulinemia
• first-line treatment
• prognosis

Acknowledgments

The authors thank the patients and their families.

Disclosure statement

No potential conflict of interest was reported by the author(s).

Authors contributors

Cao XX, Yi SH, Jiang ZY, Li J and Qiu LG contributed to study conception and design; Cao XX, Yi SH, Jiang ZY, He JS, Yang W, Du J, Sun CY, Wu Y, Chen WM, Liu XJ, Li BZ, Li CR, Sang W, Liu QH, Chu XX, Li F, Bai O, Mao M, Fu R, Wang W, Liu LH, Wang LQ, Dong YJ, Luo J, Li ZL, Wei YQ, Zhang QK, Liu J, Ding KY, Zou L, Chen BY, Hua LM, Jing HM, He J, Wang L retrospectively reviewed patient records and contributed to data collection and patient follow-up; Cao XX, Yi SH and Jiang ZY contributed to data analysis; Cao XX wrote the paper; and all authors revised the paper and approved the submitted version.

Data availability statement

Individual participant data will not be available. Study protocol will be available beginning 9 months and ending 36 months following article publication at [email protected].

Additional information

Funding

This work was supported by institutional research funding provided by the Beijing Natural Science Foundation [Grant No. 7202160 to CXX], the Non-profit Central Research Institute Fund of the Chinese Academy of Medical Sciences [Grant No. 2019-RC-HL-001 to CXX], the CAMS Innovation Fund for Medical Sciences [Grant No. 2016-12M-1-002 to LJ] and the National Key Research and Development Program of China [Grant No. 2016YFC0901503 to LJ].