Citations

OBJECTIVE Imaging surveillance with serial MRI, or a "wait-and-scan" approach, is a management option for patients with small or medium-sized vestibular schwannomas (VSs). Prior publications have indicated no distinct quality of life advantage to upfront treatment compared with initial wait-and-scan management. However, imaging surveillance is dependent on patient adherence to follow-up. In this study, the authors aimed to identify rates and predictors of patient loss to follow-up during wait-and-scan management of sporadic VS. METHODS A single-center study was conducted including all patients from 2013 to 2018 who had undergone upfront imaging surveillance of sporadic VS. Patient data were retrospectively obtained from the electronic medical record. Outcomes of interest included loss to follow-up unrelated to death and inconsistent adherence to imaging surveillance recommendations. Logistic regression analyses were conducted to evaluate factors associated with loss to follow-up. RESULTS Over a 6-year study period, 270 patients underwent initial imaging surveillance of a sporadic VS. The median tumor diameter was 8.6 mm (range 1–28.9 mm). At the time of censoring, 106 patients (39.3%) had received treatment, 157 (58.1%) had been advised to continue follow-up, and 7 (2.6%) had died of non–VS-related causes. In total, 73 patients (27.0%) were completely lost to follow-up prior to the first treatment or death. Additionally, 60 patients (22.2%) missed at least 1 MRI follow-up or imaging follow-up was delayed by more than 1 year. Multivariable logistic regression identified an out-of-state residence (OR 3.05, 95% CI 1.58–5.89, p = 0.0009) and a smaller tumor size (unit OR per 1-mm increase in size, OR 0.88, 95% CI 0.83–0.95, p = 0.0006) to be associated with loss to follow-up. Patients living ≥ 350 miles from the hospital or with tumors ≤ 3 mm at the time of initial clinic evaluation were most likely to be lost to follow-up. Only a smaller tumor size was associated with an increased risk of inconsistent imaging follow-up (unit OR per 1-mm increase in size, OR 0.92, 95% CI 0.87–0.98, p = 0.007). CONCLUSIONS Patients undergoing imaging surveillance of VS are at risk for loss to follow-up and inconsistent imaging surveillance. Patients with smaller tumors or those living farther away from the treating institution are at highest risk for being lost to follow-up.

Abstract Disclosure: B.M. Biller: Consulting Fee; Self; HRA Pharmaceuticals, Novartis Pharmaceuticals, Sparrow, Strongbridge Biopharma. Grant Recipient; Self; Strongbridge Biopharma. A. Gilis-Januszewska: Speaker; Self; Ipsen, Novartis Pharmaceuticals, Novo Nordisk, Recordati, Teva Pharmaceutical Industries Ltd.. M. Doknic: None. A.M. Pico: None. M. Fleseriu: Consulting Fee; Self; Ascendis, GlaxoSmithKline, Johnson &Johnson, Karyopharm, Novo Nordisk, Pfizer, Inc., Pharmacosmos. Research Investigator; Self; Ascendis. G. Raverot: None. A.M. Isidori: None. Y. Takahashi: None. J.M. Garcia: None. J.M. Silverstein: None. I. Bancos: None. E. Huang: Employee; Self; Ascendis Pharma, Inc. J. Kang: Employee; Self; Ascendis Pharma, Inc. A.S. Komirenko: Employee; Self; Ascendis Pharma, Inc. L. Domrzalski: Employee; Self; Ascendis Pharma, Inc. A. Shu: Employee; Self; Ascendis Pharma, Inc. K. Yuen: Advisory Board Member; Self; Amryt, Ascendis, Corcept, Crinetics, Novo Nordisk, Recordati, Strongbridge, Xeris. Grant Recipient; Self; Amryt, Ascendis, Corcept, Crinetics. Speaker; Self; Corcept, Novo Nordisk, Recordati. Background: Adult GHD (aGHD) is characterized by metabolic abnormalities due to insufficient growth hormone (GH) production. Lonapegsomatropin was designed to provide sustained release of unmodified somatropin, with the identical amino acid sequence as endogenous GH, and once-weekly injection reduces the burden of daily GH replacement therapy. MethodsForesiGHt was a randomized, parallel-3-arm, placebo(PL)-controlled (double-blind) and active-controlled (open-label) trial designed to establish the efficacy and safety of lonapegsomatropin in aGHD. It evaluated 259 adults with GHD across 21 countries who were GH treatment-naïve or not treated with GH in the prior year, randomized 1:1:1 to receive lonapegsomatropin, once-weekly PL, or daily somatropin (dGH). Fixed dosing was based on age and oral estrogen intake, and designed to be comparable across lonapegsomatropin and dGH arms. Following a 12-week (w) titration period to target maintenance dose, fixed doses were administered for 26w. ResultsBaseline (BL) characteristics were similar between arms. Lonapegsomatropin demonstrated superiority on the primary efficacy endpoint of change from BL in trunk percent fat at Week 38 vs PL (lonapegsomatropin -1.7% vs PL 0.4%, LS mean difference -2.0%, p < 0.0001) and on the key secondary efficacy endpoints of change from BL in total body lean mass (lonapegsomatropin 1.6 kg vs PL -0.1 kg, LS mean difference 1.7 kg, p < 0.0001) and change from BL in trunk fat mass (lonapegsomatropin -0.5 kg vs PL 0.2 kg, LS mean difference -0.7 kg, p = 0.0053). Mean total exposure and maintenance doses were similar for lonapegsomatropin and dGH arms, with larger changes from BL in average IGF-I SDS mean at Week 38 in the lonapegsomatropin arm (1.4) vs dGH arm (0.5). To assess outcomes at comparable weekly IGF-I exposure, a post-hoc analysis was performed in a subset of participants with average IGF-I SDS ≤1.75 SDS at Week 38. Mean IGF-I SDS for these subsets were similar (lonapegsomatropin -0.1, dGH -0.5), and comparable effects in fat and lean tissue compartments were demonstrated (change from BL in trunk percent fat mean -2.4% and -2.6% respectively; change from BL in total body lean mass mean 1.7 kg and 1.4 kg respectively). In the safety population, the incidence of severe AEs was low (lonapegsomatropin, 3.4%; placebo, 1.2%; dGH, 2.3%) and the incidence of treatment-related AEs was similar (lonapegsomatropin 24.7%, dGH 22.1%). Injection site reaction incidence was low and similar for lonapegsomatropin (4.5%), dGH (5.8%) and placebo (4.8%), and A1c levels remained stable in all treatment arms. ConclusionsThe results of the foresiGHt trial indicate that lonapegsomatropin is a safe, efficacious, and tolerable replacement for endogenous GH. Once-weekly dosing may be impactful for adults with GHD who typically manage multiple other medical therapies. Presentation: 6/3/2024

OBJECTIVE Advances in surgical technology and microneurosurgery have led to increased utilization of so-called minimally invasive approaches, including the supraorbital eyebrow (SE) and minipterional (MPT) approaches for lesions involving the interpeduncular region. This study aimed to describe and compare anatomical landmarks, along with highlighting the advantages and disadvantages of the SE and MPT approaches to the interpeduncular region. METHODS Ten formalin-fixed, latex-injected cadaveric specimens were used to perform bilateral SE and MPT approaches to the interpeduncular region. The operative depth of each approach to key anatomical landmarks was measured. Forty-five axial thin-slice computed tomography studies were reviewed to calculate the operative angles, with consideration of the midline as a reference. A 3D interactive anatomical model generated through the photogrammetry scanning technique was described. RESULTS The depths of the operative corridors of the SE and MPT approaches to the interpeduncular fossa were 83.4 ± 1.8 mm and 67.7 ± 3.2 mm, respectively (p < 0.001). The mean angle of the MPT approach to the interpeduncular fossa was significantly wider than the one provided by the SE approach (39.9° ± 5.1° vs 28.4° ± 3.6°, p < 0.001). The interpeduncular region can consistently be accessed through the carotid-oculomotor triangle with the SE approach, as well as with the MPT approach. Furthermore, the SE route offered adequate access to the interpeduncular fossa through the opticocarotid triangle. The MPT route provided direct access to the upper prepontine cistern and anterior mesencephalic zone (AMZ). CONCLUSIONS The MPT approach provides a wider and shorter operative corridor and can be employed for lesions in the interpeduncular region with extension to the prepontine cistern and ventrolateral midbrain lesions requiring access through the AMZ. The SE approach is better suited for ventromedial midbrain lesions requiring access via the interpeduncular fossa safe entry zone. Additional studies analyzing these approaches in a clinical setting will help to delineate their reliability and efficacy.

Background: Cutaneous collagenous vasculopathy (CCV) is a rare acquired microangiopathy that commonly affects middle-aged, fair-skinned individuals. It presents with telangiectatic lesions involving the lower extremities. Histologic analysis is mandatory for diagnosis. Objectives: To describe and characterize the patients diagnosed with CCV at the Mayo Clinic. Methods: A multicenter retrospective observational analysis was performed on patients with a clinical and histological diagnosis of CCV at the Mayo Clinic in Florida, Arizona, and Rochester from January 2000 to October 2023. Results: The study included 34 patients, 22 (64.7%) females. The median age at diagnosis was 52 years (range, 12-80 years). CCV lesions were found to affect the lower extremities in 29 (85.3%) patients, followed by the trunk in 14 (41.2%). Nineteen (55.9%) patients presented 1 concomitant medical condition, 8 (23.5%) 2 conditions, and 5 (14.7%) 3 or more medical conditions, such as arterial hypertension 10 (29.4%), type 2 diabetes mellitus 3 (8.8%), hyperlipidemia 3 (8.8%), cardiac disease 3 (8.8%), and hematologic malignancy 3 (8.8%). Thirty-one patients (91.2%) were taking medications, with 19 (55.9%) taking 3 or more. Pathology staining revealed periodic acid-Schiff positivity in 23 of 34 cases (67.6%), and collagen IV in 26 of 34 cases (47.1%). Treatments included pulsed dye laser 3 (8.8%) and topical sirolimus 1 (2.9%). Conclusions: CCV is an uncommon microangiopathy that affects pediatric, middle-aged, and elderly patients. It involves the lower extremities, trunk, and upper extremities. Patients often present with associated metabolic and cardiovascular conditions and are usually taking at least 1 medication. Treatment options are limited.