In the dynamic field of hematology and oncology, finding effective treatments for Waldenstrom macroglobulinemia (WM) is crucial. A recent study led by Professor Shuhua Yi from the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, has made significant strides in this area. This research analyzed first- and second-line therapies in 377 patients with WM, demonstrating that sequential treatment escalation markedly improves survival rates. By transitioning from cytotoxic drugs to rituximab, bortezomib, or Bruton tyrosine kinase inhibitors, patients saw significant benefits. These findings, presented at a major medical conference, underscore the collaborative effort and innovation driving progress in hematology and improving patient outcomes.This study aims to identify the optimal treatment strategies for patients with relapsed Waldenstrom macroglobulinemia (WM), focusing on the impact of sequential treatment escalation on improving survival outcomes.
A retrospective analysis was conducted on 377 patients with WM who were treated at the Blood Disease Hospital, Chinese Academy of Medical Sciences, between June 2000 and September 2021. The study compared the outcomes of first-line and second-line therapies, with an emphasis on patients who underwent sequential treatment escalation. Treatment regimens were categorized into cytotoxic drugs, rituximab (R)-based regimens, bortezomib (V)-based regimens, and Bruton tyrosine kinase inhibitor (BTKi)-based regimens.
In the research, Professors Yi observed that the Major Response Rates (MRR) were 65.1% for first-line therapy and 67.9% for second-line therapy, with no significant difference between the two (P = 0.678). The Progression-Free Survival (PFS) for first-line therapy (PFS1) was 56.3 months, while for second-line therapy (PFS2) it was 40.7 months, showing a statistically significant difference (P = 0.03). In the Targeted Drugs Group, PFS1 and PFS2 were comparable, with durations of 60.7 months and 44.7 months respectively, and the difference was not statistically significant (P = 0.21). The Sequential Treatment Escalation Group demonstrated a higher MRR (80.6% vs. 47.1%), a longer PFS2 (50.4 months vs. 23.5 months), and a significantly longer post-relapse overall survival, with a median that has not been reached compared to 50.7 months for the control group (P = 0.039).
(Blood Science. 6(1):e00179, January 2024.)
The study highlights that sequential treatment escalation—transitioning from cytotoxic drugs to R-/V-/BTKi-based regimens or from R-/V-based to BTKi-based regimens—significantly improves survival outcomes for patients with relapsed WM. This approach results in higher response rates and extended progression-free and overall survival times.
Patients treated with targeted drug regimens (R-, V-, or BTKi-based) demonstrated significantly better outcomes compared to those treated with conventional cytotoxic drugs. For second-line therapies, BTKi-based regimens had the highest MRR (80.6%) and overall response rate (ORR) of 96.8%. The study also showed that patients in the sequential treatment escalation group had better outcomes than those in the non-escalation group, with significantly longer PFS2 and post-relapse overall survival.
Sequential treatment escalation is a promising strategy for improving survival in patients with relapsed WM. By utilizing advancements in targeted therapies, this approach can lead to significant improvements in major response rates and survival outcomes. Future research should continue to explore and validate these findings in larger, independent cohorts to refine treatment protocols and optimize patient outcomes.
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