Trial reportNeuro-oncology2023
Phase II study of intrathecal administration of trastuzumab in patients with HER2-positive breast cancer with leptomeningeal metastasis.
Trial report in Neuro-oncology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 39 papers, 5 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
39 citing papers in PubMed, 5 syntheses or guidelines pooled it, 57 citations in OpenAlex.
- Quality-of-life measures in pharmacogenomic studies: a systematic review.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2025Pooled it
- 6th and 7th International consensus guidelines for the management of advanced breast cancer (ABC guidelines 6 and 7).Breast (Edinburgh, Scotland) · 2024Guideline
- DEGRO guideline for personalized radiotherapy of brain metastases and leptomeningeal carcinomatosis in patients with breast cancer.Strahlentherapie und Onkologie : Organ der Deutschen Rontgengesellschaft ... [et al] · 2024Guideline
- Leptomeningeal metastasis from solid tumours: EANO-ESMO Clinical Practice Guideline for diagnosis, treatment and follow-up.ESMO open · 2023Guideline
- Intrathecal trastuzumab versus alternate routes of delivery for HER2-targeted therapies in patients with HER2+ breast cancer leptomeningeal metastases.Breast (Edinburgh, Scotland) · 2023Pooled it
- Tucatinib-trastuzumab-capecitabine for treatment of leptomeningeal metastasis in women with HER2Nature cancer · 2026Trial
- Article
- Bridging the blood-brain barrier: strategies to improve delivery of biologics to tumors in the brain.Fluids and barriers of the CNS · 2026Review
- The Use of Radiotherapy in Leptomeningeal Carcinomatosis: A Systematic Review and Random-Effects Proportions Meta-Analysis.Cancers · 2026Review
- The evolving landscape of leptomeningeal metastases from NSCLC: an international, contemporary, multicenter cohort study.Annals of oncology : official journal of the European Society for Medical Oncology · 2026Article
- Case report: Systemic T-DXd and bevacizumab plus local therapy prolongs survival in breast cancer leptomeningeal metastasis: a case series and literature review.Frontiers in oncology · 2026Article
- Intrathecal pemetrexed in patients with epidermal growth factor receptor-tyrosine kinase inhibitor (EGFR-TKI) resistant refractory leptomeningeal metastasis lung adenocarcinoma: a retrospective study.Journal of thoracic disease · 2025Article
- Point/Counterpoint: Intrathecal therapy for patients with leptomeningeal metastases from solid tumors.Neuro-oncology · 2025Article
- Survival outcomes among patients with breast cancer and leptomeningeal disease.Scientific reports · 2025Article
- Drug treatment of breast cancer brain metastases: progress and challenges.Discover oncology · 2025Review
- HER2-Positive Breast Cancer-Current Treatment Management and New Therapeutic Methods for Brain Metastasis.Biomedicines · 2025Review
- The research progress on meningeal metastasis in solid tumors.Discover oncology · 2025Review
- NANO-LM: An updated scorecard for the clinical assessment of patients with leptomeningeal metastases.Neuro-oncology · 2025Article
- Leptomeningeal metastatic disease: new frontiers and future directions.Nature reviews. Clinical oncology · 2025Review
- Review
Corrections and comments
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Authors and funding
11 authors at 5 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPatients with HER2-positive breast cancer (HER2 + BC) develop central nervous system metastases twice as often as patients with luminal HER2-negative breast cancer. Leptomeningeal progression results in a drastically altered prognosis with limited therapeutic options. This phase II study was conducted to assess the efficacy of intrathecal (IT) trastuzumab in HER2 + BC patients with leptomeningeal metastasis (LM), based on a phase I dose-escalation study that had determined the recommended weekly dose of 150 mg for phase II.
methodsEligible patients received weekly administrations of 150 mg of IT trastuzumab. The primary endpoint was clinical neurologic progression-free survival (LM-related-PFS) after 8 weeks. Overall survival (OS), toxicities, and quality of life (QoL) were secondary endpoints.
resultsAmong the 19 enrolled patients, 16 (84%) had concomitant brain metastases, 15 of them had received prior radiotherapy to the brain. All patients had received at least one line of systemic anti-HER2 therapy. After 8 weeks, 14 patients (74%) were free of neurological progression. The median LM-related-PFS was 5.9 months and the median OS was 7.9 months. According to the QLQ-C30 and BN20 scales, the global health-related QoL status seemed preserved and no toxicity above grade 3 was observed.
conclusionsConducting studies on patients with LM poses significant challenges and ethical dilemmas inherent to this population. Despite some limits, this phase II study's findings in terms of clinical neurologic response and QoL's control confirms weekly administration of 150 mg of IT trastuzumab as a valuable option for HER + BC patients with LM and support the interest for further investigations.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.