Publications by authors named "S Lanfranconi"
Background: The Phase 1/2 Treat_CCM randomized controlled trial for people with familial cerebral cavernous malformations (FCCMs) confirmed the safety of propranolol and suggested beneficial effects on intracerebral hemorrhage or new focal neurological deficits, but the effects on patient-reported outcome measures have not been reported.
Methods: Participants completed self-reported questionnaires at baseline, 1 and 2 years. Depression was assessed with the Beck Depression Inventory-II (BDI-2); Anxiety with the State-Trait Anxiety Inventory X1 and X2 (STAI X-1 and STAI X-2); and Quality of Life with the Short Form 36 (SF-36), split into the physical and mental component scales (PCS and MCS).
Background: Cerebral Cavernous Malformation (CCM) is a rare cerebrovascular disease, characterized by the presence of multiple vascular malformations that may result in intracerebral hemorrhages (ICHs), seizure(s), or focal neurological deficits (FND). Familial CCM (fCCM) is due to loss of function mutations in one of the three independent genes KRIT1 (CCM1), Malcavernin (CCM2), or Programmed Cell death 10 (PDCD10/CCM3). The aim of this study was to identify plasma protein biomarkers of fCCM to assess the severity of the disease and predict its progression.
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- The study investigates whether combining intravenous thrombolysis (IVT) with endovascular thrombectomy (EVT) is more effective and safe for patients with mild acute ischemic stroke and large vessel occlusion compared to IVT alone.
- A total of 1,037 patients were analyzed, with 312 undergoing matched comparisons. The results showed that IVT plus EVT was linked to worse functional outcomes despite no significant safety advantage when compared to IVT alone.
- Patients receiving the combination treatment experienced higher rates of certain hemorrhagic events, indicating the need for careful evaluation when considering treatment methods for mild strokes.
Article Synopsis
- The study aims to determine if quantitative susceptibility mapping (QSM) can predict hemorrhagic signs in patients with cerebral cavernous malformations (CCMs) after one year.
- Researchers enrolled familial CCM patients and utilized 3-T MRI scans to measure QSMmax, finding higher susceptibility in hemorrhagic CCMs compared to non-hemorrhagic ones.
- The results indicated that QSMmax has good predictive accuracy for whether CCMs would still show hemorrhagic signs or remain stable after one year, highlighting its potential for follow-up assessments in larger studies.
Background: Observations in people with cerebral cavernous malformations, and in preclinical models of this disorder, suggest that the β-blocker propranolol might reduce the risk of intracerebral haemorrhage. We aimed to evaluate the safety and efficacy of prolonged treatment with propranolol to reduce the incidence of symptomatic intracerebral haemorrhage or focal neurological deficit in people with familial cerebral cavernous malformations.
Methods: We conducted a randomised, open-label, blinded-endpoint, phase 2 pilot trial (Treat_CCM) at six national reference centres for rare diseases in Italy.