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Updated: Aug 25, 2025
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Published on: November 26, 2013
Intravenous thrombolysis in CADASIL: report of two cases and a systematic review
Francesca Pescini1,2, Sara Torricelli3, Martina Squitieri3
1Emergency Department, Stroke Unit, AOU Careggi, Florence, Italy. francesca.pescini@unifi.it.
Insights
Intravenous thrombolysis with alteplase for acute ischemic stroke in Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL) patients appears safe and effective, despite limited data. This treatment may be considered for CADASIL patients experiencing acute stroke.
Area of Science:
- Neurology
- Genetics
- Vascular Medicine
Background:
- Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL) is a genetic small vessel disease causing recurrent strokes and dementia.
- Acute stroke management in CADASIL is challenging due to bleeding risks and unproven thrombolytic efficacy.
- Clinicians may encounter CADASIL patients with acute ischemic stroke within the thrombolysis window.
Approach:
- This study reports two CADASIL patients treated with intravenous alteplase for acute ischemic stroke.
- A systematic literature review was conducted up to August 2, 2022, including case reports and series on CADASIL patients with acute stroke.
- The review focused on epidemiological data, efficacy, and safety of intravenous thrombolysis in this population.
Key Points:
- Both reported CADASIL patients received intravenous thrombolysis without complications and showed good clinical outcomes.
- The literature review identified three case reports of CADASIL patients treated with alteplase, with no reported bleeding complications.
- Limited but available data suggest potential benefits of intravenous thrombolysis in acute stroke for CADASIL patients.
Conclusions:
- Intravenous thrombolysis for acute ischemic stroke in CADASIL patients is a viable consideration.
- While data are scarce, current evidence indicates a favorable safety and efficacy profile.
- Further research is warranted to establish definitive treatment guidelines for stroke in CADASIL.
Background:
Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL) is a genetic small vessel disease responsible for recurrent ischemic strokes, often with a progressive course leading to dementia and disability. On MRI, lacunes, microbleeds, and severe white matter alterations are typical features of the disease. In case of acute stroke, because of the bleeding risk associated with the disease and the doubtful efficacy of fibrinolytic treatment in a disease with poor evidence of thrombosis, the efficacy of intravenous thrombolysis remains unproven. Nevertheless, stroke is a frequent occurrence in CADASIL patients, and clinicians not unlikely may face in the emergency room the situation of a CADASIL patient with an acute stroke within the time window for thrombolysis.
Objective:
We report on two CADASIL patients treated with intravenous alteplase for acute ischemic stroke, and we present a review of literature aimed to report epidemiological data, efficacy and safety of intravenous thrombolysis in CADASIL patients.
Methods:
We performed a systematic review of medical literature published until August 2, 2022. Case reports and series in English language reporting on CADASIL patients and acute stroke were included.
Results:
Both patients were treated with intravenous thrombolysis without complications and had a good clinical outcome. The systematic review identified three case reports of CADASIL patients who were treated with intravenous alteplase for acute ischemic stroke; no bleedings complications were described.
Conclusions:
Available data on intravenous thrombolysis in CADASIL patients are scarce but suggest that this treatment can be taken into consideration for these patients.
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