New-diagnosed PCNSL Treated With Methotrexate (MTX) and Orelabrutinib-based Regimen

A Retrospective Case-control Study of Newly-diagnosed Primary Central Nervous System Lymphoma (PCNSL) Treated With Methotrexate (MTX)-Based Chemotherapy in Combination With Orelabrutinib

This study was a single-centre, retrospective study that retrospectively collected first-line PCNSL patients receiving methotrexate-based chemotherapy ± orelabrutinib at Huashan Hospital of Fudan University. The study was divided into two retrospective cohorts, Cohort A was a cohort of patients receiving methotrexate-based chemotherapy alone, and Cohort B was a cohort of patients receiving methotrexate-based chemotherapy + orelabrutinib.

Study Overview

• Status: Completed
• Conditions: Primary Central Nervous System Lymphoma
• Intervention / Treatment: Drug: Orelabrutinib

Detailed Description

The study was divided into two retrospective cohorts of approximately 35-40 patients each. Cohort A was the cohort of patients who received methotrexate-based chemotherapy alone, and cohort B was the cohort of patients who received methotrexate-based chemotherapy + orelabrutinib. Statistical analyses such as propensity score analysis (PSM) and inverse probability weighted (IPTW) analysis were performed, focusing on comparing and analysing the short-term efficacy data (ORR, CR) and long-term efficacy data (PFS, OS) of the two cohorts.

• Study Type: Observational
• Enrollment (Actual): 136

Contacts and Locations

Study Locations

• China
  • Shanghai, China
    • Department of Hematology, Huashan Hospital, Fudan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

PCNSL is a rare and highly aggressive subtype of primary extranodal non-Hodgkin's lymphoma with a distinctive clinical presentation, and more than 95% of the pathological types are diffuse large B-cell lymphomas.The current National Comprehensive Cancer Network (NCCN) guidelines recommend first-line induction therapy with high-dose methotrexate (HD-MTX) in combination with rituximab, but long-term efficacy is still unsatisfactory, with 50% of patients relapsing at around 2 years

Description

Inclusion Criteria:

• Pathologically confirmed primary central nervous system lymphoma
• Methotrexate-based chemotherapy ± orelabrutinib regimen as first-line treatment for patients

Exclusion Criteria:

• Patients receiving ≤2 cycles of treatment without efficacy evaluation
• Those with uncontrolled or significant cardiovascular disease, including: uncontrolled diabetes mellitus, severe cardiac, pulmonary, hepatic, renal insufficiency) and haematological, endocrine system lesions, history of other uncontrollable malignancies

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cohort A; Cohort A was the cohort of patients who received methotrexate-based chemotherapy alone
Cohort B; cohort B was the cohort of patients who received methotrexate-based chemotherapy + orelabrutinib.	Drug: Orelabrutinib; Combination of orelabrutinib on a methotrexate-based chemotherapy regimen with a starting dose of 150 mg/d

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective response rate (ORR)	ORR is defined as the proportion of patients with a best response of CR, CRu or PR during induction therapy	At the end of induction therapy, Cycle 6-8 (each cycle is 28 days)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
PFS	Progression-free survival is calculated from the date of start of therapy until the date of first documented progress or death due to any cause	Up to 2 years

Collaborators and Investigators

• Sponsor: Huashan Hospital
• Investigators: Principal Investigator: Tong Chen, Huashan Hospital; Study Director: Tong Chen, Huashan Hospital

Study record dates

Study Major Dates

• Study Start (Actual): August 10, 2023
• Primary Completion (Actual): January 30, 2024
• Study Completion (Actual): July 30, 2024

Study Registration Dates

• First Submitted: October 30, 2023
• First Submitted That Met QC Criteria: October 30, 2023
• First Posted (Actual): November 3, 2023

Study Record Updates

• Last Update Posted (Estimated): September 16, 2025
• Last Update Submitted That Met QC Criteria: September 13, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Lymphoma; orelabrutinib
• Other Study ID Numbers: KY2023-677

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No