Stereotactic Aspiration of Brain Stem Hematoma: a Prospective, Multi-center, Randomized Controlled Trial

1. Research Background and Rationale Primary brainstem hemorrhage (PBSH) represents the most severe and life-threatening form of spontaneous intracerebral hemorrhage. Due to its critical anatomical location involving vital centers, it is associated with high mortality (30-day mortality rates ranging from 47% to 80%) and extremely poor neurological outcomes. For a long time, the optimal treatment strategy for PBSH, particularly the value of surgical intervention, has been a subject of significant global controversy. Influential international guidelines, such as those from the American Heart Association/American Stroke Association (AHA/ASA), still regard surgery for PBSH as a relative or absolute contraindication, primarily due to the lack of high-level evidence.

Concurrently, with advancements in neuroimaging, neuronavigation, and minimally invasive surgical techniques, several medical centers in China have progressively explored surgical interventions for PBSH, including stereotactic aspiration and neuroendoscopic evacuation, accumulating considerable clinical experience. The 2022 Chinese Expert Consensus on the Diagnosis and Treatment of Primary Brainstem Hemorrhage suggests that surgical treatment can be considered for specific patients, such as those with hematoma volume ≥5 ml and a Glasgow Coma Scale (GCS) score ≤7, following thorough assessment. However, the consensus is largely based on retrospective analyses or small case series, providing limited evidence that falls short of guiding widespread clinical practice.

The core dilemma in current clinical decision-making lies in the disconnect between theory and practice. Theoretically, early hematoma evacuation may relieve compression on brainstem structures and facilitate neurological recovery. In practice, however, the risks inherent to surgery, heterogeneity in patient selection, variations in surgical timing and technique, and other factors leave it unclear whether surgical intervention offers a net clinical benefit. Therefore, conducting a rigorously designed and properly executed multicenter randomized controlled trial (RCT) to compare the impact of stereotactic minimally invasive aspiration versus standard medical conservative treatment on patient outcomes is of urgent and paramount importance. Such a study is essential to fill the existing evidence gap and establish a scientific treatment protocol. 2. Research Objectives and Core Content 2.1 Research Objective The primary objective of this study is to systematically compare the efficacy and safety of stereotactic brainstem hematoma aspiration versus conservative treatment alone in eligible patients with PBSH through a prospective, multicenter, randomized controlled trial. The focus is on evaluating the difference in the rate of death and disability (using the Modified Rankin Scale [MRS] score as the core metric) at 3 months post-treatment. The aim is to provide high-quality, generalizable evidence-based medical data to inform clinical decision-making for brainstem hemorrhage.

2.2 Research Content

This study focuses on the most challenging patient population in clinical practice: those with moderate-to-severe primary PBSH. By randomly assigning patients who meet strict inclusion and exclusion criteria to either the surgical intervention group (stereotactic aspiration) or the medical treatment group (standard conservative therapy), the study will objectively evaluate, within a unified and standardized treatment and follow-up framework:

1. Whether surgical intervention can significantly improve patients' neurological functional outcomes (primary endpoint).
2. Differences between the two groups in the incidence of various complications (e.g., re-bleeding, infection, hydrocephalus), length of intensive care unit (ICU) stay, and total hospital length of stay (secondary endpoints).
3. The safety profile and spectrum of adverse events associated with surgical treatment.
4. Study Design and Methodological Highlights 3.1 Overall Design This is a multicenter, prospective, single-blind (with outcome assessors blinded), randomized controlled study.