Treatments and Outcomes of Untreated Cerebral Cavernous Malformations in CHina.

• Conditions: Cerebral Cavernous Malformations
• Interventions: Procedure: Surgery; Procedure: Observation

• Registration Number: NCT03467295
• Lead Sponsor: First Affiliated Hospital of Fujian Medical University

Brief Summary

This nationwide multicenter prospective cohort study will collect the treatment information and outcomes of the patients with previously untreated cavernous malformations (U-CMs) in China (at least 2000 patients from 20 centers). The investigators aim to determine the effect of different treatments on long-term outcomes in patients with untreated cerebral cavernous malformations.

Detailed Description

Despite the availability of microsurgical excision and stereotactic radiosurgery for cavernoma treatment and known genetic causes of most familial forms of cavernoma, uncertainties remain about cause, diagnosis, prognosis, treatment, and care. According to the recently published CM treatment guideline, surgical and conservative treatments are both first-line therapeutic options in previously untreated CMs (U-CMs). However, given current data, neurosurgeons are still unable to counsel patients on which would be the best option. To address the absence of high level evidence to support cerebral CM treatment decisions, there is a need for a large sample size study of cerebral CM treatment with a prospective concurrent group. China has the largest population in the world and may have the most extensive and valuable clinical resources of U-CMs. But, till now, the current treatment and outcome situation of the disease in China is not well known. Thus, the investigators are conducting a nationwide multicenter prospective registry study in China to determine the effect of different treatments on long-term outcomes in patients with untreated cerebral cavernous malformations.At least 2000 patients with untreated cerebral cavernous malformation will be enrolled from 20 Grade Ⅲ A level hospitals distributed all over China. Clinical data and laboratory data are prospectively collected by electric case report form (CRF) and uploaded online by each neurosurgery center to form the prospective clinical database in First Affiliated Hospital of Fujian Medical University. This is a cohort follow-up study across a 5-year period with a 2 years interval of enrollment and 3 years follow up for each patient.

Study Timeline

• Study Start: 2018-01-01
• Status Verified: 2018-03
• Study First Submitted: 2018-03-09
• Study First Submitted QC: 2018-03-09
• Last Update Submitted: 2018-03-15
• Study First Posted: 2018-03-16
• Last Update Posted: 2018-03-19
• Primary Completion: 2023-03-31
• Study Completion: 2023-03-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 2000

Inclusion Criteria

• Definite diagnosis of cerebral CM on the basis of brain MRI or pathologic examination;
• Patients without any surgical intervention (microsurgery, radiosurgery, or multimodality treatment) before enrolment;
• Informed consent, and willing to accept long-term follow-up.

Exclusion Criteria

• Patients receiving emergency surgery due to acute intracranial hematoma and resultant brain hernia;
• Patients with other never system diseases, such as aneurysms, tumors or other vascular malformations except venous development anomaly;
• Patients with severe underlying disease, which affects the patient's functional status and life expectancy;
• Patients with severe mental or psychologic disease.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Surgically treated group	Surgery	Surgical removal of intracerebral CMs by craniotomy with or without following stereotactic radiosurgery.
Conservatively treated group	Observation	Observation with the best medicine administration and supportive treatment are performed.

Primary Outcome Measures

Name	Time	Method
Poor outcome	3 years	The primary outcome of this study is patients with poor outcome (mRS\>2 lasting at least 1year) at the last follow up.

Secondary Outcome Measures

Name	Time	Method
symptomatic hemorrhage	3 years	Requiring acute or subacute onset symptoms (any of headache, epileptic seizure, impaired consciousness, or new/worsened brain function deficit referable to the anatomic location of the CM) accompanied by radiological, pathological, surgical, or rarely only cerebrospinal fluid evidence of recent extra- or intralesional hemorrhage.
drug refractory epilepsy	3 years	Failure of adequate trials of two tolerated, appropriately chosen and used antiepileptic drug schedules (whether as monotherapies or in combination) to achieve sustained seizure freedom.
All-cause mortality	3 years	Death caused by all the causes

Trial Locations

Locations (1)

The first affiliated hospital of fujian medical university; 🇨🇳 Fuzhou, Fujian, China