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Scientific Reports May 2024Capmatinib is a potent selective mesenchymal-epithelial transition inhibitor approved in 2020 for the treatment of metastatic non-small cell lung cancer. As real-world...
Capmatinib is a potent selective mesenchymal-epithelial transition inhibitor approved in 2020 for the treatment of metastatic non-small cell lung cancer. As real-world evidence is very limited, this study evaluated capmatinib-induced adverse events through data mining of the FDA Adverse Event Reporting System database. Four disproportionality analysis methods were employed to quantify the signals of capmatinib-related adverse events. The difference in capmatinib-associated adverse event signals was further investigated with respect to sex, age, weight, dose, onset time, continent, and concomitant drug. A total of 1518 reports and 4278 adverse events induced by capmatinib were identified. New significant adverse event signals emerged, such as dysphagia, dehydration, deafness, vocal cord paralysis, muscle disorder, and oesophageal stenosis. Notably, higher risk of alanine aminotransferase and aspartate aminotransferase increases were observed in females, especially when capmatinib was combined with immune checkpoint inhibitors. Compared with Europeans and Asians, Americans were more likely to experience peripheral swelling, especially in people > 65 years of age. Renal impairment and increased blood creatinine were more likely to occur with single doses above 400 mg and in Asians. This study improves the understanding of safety profile of capmatinib.
Topics: Humans; Male; Female; United States; Middle Aged; Aged; United States Food and Drug Administration; Pharmacovigilance; Adverse Drug Reaction Reporting Systems; Benzamides; Adult; Triazines; Carcinoma, Non-Small-Cell Lung; Aged, 80 and over; Young Adult; Lung Neoplasms; Adolescent; Imidazoles
PubMed: 38762672
DOI: 10.1038/s41598-024-62356-w -
Frontiers in Pediatrics 2024Brown-Vialetto-Van Laere (BVVL) syndrome is an extremely rare autosomal recessive progressive motoneuron disease that is caused by a defect in the riboflavin transporter...
BACKGROUND
Brown-Vialetto-Van Laere (BVVL) syndrome is an extremely rare autosomal recessive progressive motoneuron disease that is caused by a defect in the riboflavin transporter genes SLC52A2 and SLC52A3. BVVL syndrome has a variable age of presentation, and it is characterized by progressive auditory neuropathy, bulbar palsy, stridor, muscle weakness, and respiratory compromise secondary to diaphragmatic and vocal cord paralysis. BVVL syndrome has a poor prognosis in the absence of treatment, including morbidity with quadriparesis and sensorineural hearing loss, with mortality in the younger age group. Early administration of riboflavin is associated with prolonged survival, low morbidity, and reversal of some clinical manifestations.
CASE PRESENTATION
We describe an 18-month-old male infant with progressive pontobulbar palsy, loss of developmental milestones, and a clinical picture suggestive of chronic inflammatory demyelinating neuropathy. A nerve conduction study revealed axonal neuropathy, while molecular analysis revealed a homozygous mutation in one of the riboflavin transporter genes, SLC52A3, confirming BVVL syndrome. The patient needed long-term respiratory support and a gastrostomy tube to support feeding. With high-dose riboflavin supplementation, he experienced moderate recovery of motor function.
CONCLUSION
This report highlights the importance of considering BVVL syndrome in any patient who presents with the clinical phenotype of pontobulbar palsy and peripheral axonal neuropathy, as early riboflavin treatment may improve or halt disease progression, thus reducing the associated mortality and morbidity.
PubMed: 38745833
DOI: 10.3389/fped.2024.1377515 -
Acta Otorhinolaryngologica Italica :... May 2024
Review
Topics: Humans; Laryngeal Neoplasms; Postoperative Complications; Laryngectomy; Laryngoscopy
PubMed: 38745512
DOI: 10.14639/0392-100X-suppl.1-44-2024-N2969 -
Radiology. Imaging Cancer May 2024The "puffed cheek" technique is routinely performed during CT neck studies in patients with suspected oral cavity cancers. The insufflation of air within the oral... (Review)
Review
The "puffed cheek" technique is routinely performed during CT neck studies in patients with suspected oral cavity cancers. The insufflation of air within the oral vestibule helps in the detection of small buccal mucosal lesions, with better delineation of lesion origin, depth, and extent of spread. The pitfalls associated with this technique are often underrecognized and poorly understood. They can mimic actual lesions, forfeiting the technique's primary purpose. This review provides an overview of the puffed cheek technique and its associated pitfalls. These pitfalls include pneumoparotid, soft palate elevation that resembles a nasopharyngeal mass, various tongue displacements or distortions that obscure tongue lesions or mimic them, sublingual gland herniation, an apparent exacerbation of the airway edema, vocal cord adduction that hinders glottic evaluation, and false indications of osteochondronecrosis in laryngeal cartilage. Most stem from a common underlying mechanism of unintentional Valsalva maneuver engaged in by the patient while trying to perform a puffed cheek, creating a closed air column under positive pressure with resultant surrounding soft-tissue displacement. These pitfalls can thus be avoided by instructing the patient to maintain continuous nasal breathing while puffing out their cheek during image acquisition, preventing the formation of the closed air column. CT, Head/Neck © RSNA, 2024.
Topics: Humans; Cheek; Tomography, X-Ray Computed; Mouth Neoplasms; Insufflation
PubMed: 38727566
DOI: 10.1148/rycan.230211 -
Cureus Apr 2024We discuss a novel approach to resecting a large 1.5 cm intracordal schwannoma via direct laryngoscopy with combined endoscopic and microlaryngoscopic techniques....
We discuss a novel approach to resecting a large 1.5 cm intracordal schwannoma via direct laryngoscopy with combined endoscopic and microlaryngoscopic techniques. Removing relatively bulky masses within the vocal cord soft tissue can be challenging secondary to difficult visualization of the operative field during direct laryngoscopy. We describe a case where a bulky atypical spindle cell schwannoma was removed via direct laryngoscopy via combined endoscopic and microlaryngoscopic techniques. The tumor obstructed 40% of the visual field of the laryngoscope. In this case, a 44-year-old female presented to the head and neck surgery clinic with 1.5 years of progressive hoarseness. On fiberoptic laryngoscopy, a mass was noted medializing the right true vocal cord. The patient was taken to surgery and after intubation and suspension with a Dedo laryngoscope, the mass was removed trans-orally through the laryngoscope with visualization using a combination of rigid and flexible endoscopy as well as with a microscope. Although visualization can sometimes be reduced using direct laryngoscopy, surgical excision of relatively large laryngeal masses can be performed in selected cases without the need to approach the masses trans-cervically.
PubMed: 38721193
DOI: 10.7759/cureus.57823 -
Translational Pediatrics Apr 2024Innominate artery compression syndrome (IAS) is caused by an abnormally originating innominate artery compressing the trachea anteriorly. One option to relieve such...
BACKGROUND
Innominate artery compression syndrome (IAS) is caused by an abnormally originating innominate artery compressing the trachea anteriorly. One option to relieve such compression is an anterior aortopexy (AA). We describe our technique of an AA via a partial upper median sternotomy.
CASE DESCRIPTION
Nine consecutive patients underwent AA for IAS via a partial upper median sternotomy from July 2017 to November 2020 at two US teaching hospitals. The median age was 9 months [interquartile range (IQR), 3-16.5 months]. The male to female ratio was 1.25. All patients had >70% compression by flexible bronchoscopy. Two patients had previous surgeries. The median follow-up was 6 months (IQR, 4-8.5 months). The indications for the operation were: acute life-threatening events (ALTEs) (4/9 patients), recurrent intubation (4/9), and severe stridor with >70% luminal reduction (1/9). Technical success (defined as ≤20% residual stenosis) was achieved in 78% (7/9) of the patients. The two patients with unsuccessful AAs required either a tracheal resection or an innominate artery reimplantation. Both achieved full symptom resolution. Overall, 78% (7/9) of patients experienced full symptom resolution. Of the two patients without full symptom resolution, one had mild stridor at 6 months post-operation. The other patient without full resolution is awaiting further vocal cord surgery for an associated glottic pathology.
CONCLUSIONS
A partial upper sternotomy provides a very versatile approach to an AA for IAS. In addition to facilitating an adequate AA, a partial upper sternotomy provides options for direct tracheal surgery or an innominate artery reimplantation in case an optimal result is not obtained by an AA.
PubMed: 38715670
DOI: 10.21037/tp-23-597 -
Scientific Reports May 2024A wide variety of treatments have been developed to improve respiratory function and quality of life in patients with bilateral vocal fold paresis (BVFP). One...
A wide variety of treatments have been developed to improve respiratory function and quality of life in patients with bilateral vocal fold paresis (BVFP). One experimental method is the electrical activation of the posterior cricoarytenoid (PCA) muscle with a laryngeal pacemaker (LP) to open the vocal folds. We used an ovine (sheep) model of unilateral VFP to study the long-term effects of functional electrical stimulation on the PCA muscles. The left recurrent laryngeal nerve was cryo-damaged in all animals and an LP was implanted except for the controls. After a reinnervation phase of six months, animals were pooled into groups that received either no treatment, implantation of an LP only, or implantation of an LP and six months of stimulation with different duty cycles. Automated image analysis of fluorescently stained PCA cross-sections was performed to assess relevant muscle characteristics. We observed a fast-to-slow fibre type shift in response to nerve damage and stimulation, but no complete conversion to a slow-twitch-muscle. Fibre size, proportion of hybrid fibres, and intramuscular collagen content were not substantially altered by the stimulation. These results demonstrate that 30 Hz burst stimulation with duty cycles of 40% and 70% did not induce PCA atrophy or fibrosis. Thus, long-term stimulation with an LP is a promising approach for treating BVFP in humans without compromising muscle conditions.
Topics: Animals; Sheep; Disease Models, Animal; Vocal Cord Paralysis; Electric Stimulation Therapy; Laryngeal Muscles; Humans; Pacemaker, Artificial; Vocal Cords; Female
PubMed: 38714750
DOI: 10.1038/s41598-024-60875-0 -
Langenbeck's Archives of Surgery May 2024In recent years, there has been an increasing prevalence of patients with papillary thyroid microcarcinoma (PTMC) without lymph node involvement in medical centers...
BACKGROUND
In recent years, there has been an increasing prevalence of patients with papillary thyroid microcarcinoma (PTMC) without lymph node involvement in medical centers worldwide. For patients who are unable to undergo active surveillance (AS) and are afraid of postoperative complications, conformal thyroidectomy may be a suitable option to ensure both preservation of function and complete removal of the tumor.
METHODS
The patients in the cohort during 2010 to 2015 were retrospectively enrolled strictly following the inclusion and exclusion criteria. The observation and control groups were defined based on the surgical approach, with patients in the observation group undergoing conformal thyroidectomy and patients in the control group undergoing lobectomy. Event-free survival (EFS), the interval from initial surgery to the detection of recurrent or metastatic disease, was defined as the primary observation endpoint.
RESULTS
A total of 319 patients were included in the study, with 124 patients undergoing conformal thyroidectomy and 195 patients undergoing lobectomy. When compared to lobectomy, conformal thyroidectomy demonstrated reduced hospital stays, shorter operative times, and lower rates of vocal cord paralysis and hypoparathyroidism. Furthermore, the mean bleeding volume during the operation and the rate of permanent hypothyroidism were also lower in the conformal thyroidectomy group than in the lobectomy group. However, there was no statistically significant difference observed in the 5- and 10-year EFS between the two groups.
CONCLUSIONS
Conformal thyroidectomy had advantages in perioperative management and short-term complication rates, with an EFS that was not inferior to that of lobectomy. Thus, conformal thyroidectomy is a feasible option for low-risk PTMC patients.
Topics: Humans; Thyroidectomy; Female; Male; Thyroid Neoplasms; Retrospective Studies; Middle Aged; Carcinoma, Papillary; Adult; Follow-Up Studies; Feasibility Studies; Cohort Studies; Postoperative Complications; Treatment Outcome; Operative Time
PubMed: 38714551
DOI: 10.1007/s00423-024-03333-9 -
Cureus Apr 2024Dysphagia is a common symptom encountered in clinical practice, typically associated with a wide range of etiologies, including structural abnormalities, inflammatory...
Dysphagia is a common symptom encountered in clinical practice, typically associated with a wide range of etiologies, including structural abnormalities, inflammatory conditions, neoplasms, and neurological disorders. However, the combination of subcutaneous emphysema, vocal cord palsy, enlarged arytenoids, and pooling of saliva in a dysphagic patient represents a rare and intriguing presentation. A 33-year-old female presented at a tertiary care hospital in Western India with hoarseness of voice, difficulty in swallowing, productive cough, and neck pain for two months with an abrupt increase in the severity of all symptoms in two days. A history of chewable tobacco use for six years was disclosed. Clinical evaluation revealed a thin build with platynychia and conjunctival pallor, dental staining, drooling of saliva, the presence of extensive subcutaneous emphysema on palpation of the neck, and absent laryngeal crepitus. Endoscopic evaluation was suggestive of right vocal cord palsy and enlarged, congested arytenoid cartilages, post-cricoid growth with pooling of saliva in bilateral pyriform fossae. A CT scan of the neck showed a 2x3 cm neoplastic growth in the hypopharynx, with subcutaneous emphysema and free air foci in the head and neck region, prompting an immediate tracheostomy and biopsy of the hypopharyngeal growth with Ryle's tube insertion. Squamous cell carcinoma was confirmed on the biopsy report. Due to its rarity, the possible underlying cause of idiopathic subcutaneous emphysema should be sought whenever encountered in clinical practice since these patients are potentially misdiagnosed. A high index of suspicion among clinicians, along with a consideration of the constellation of other symptoms and clinical features of a possible underlying hypopharyngeal cancer whenever encountering such patients is of key importance for prompting further investigations and treatment.
PubMed: 38711727
DOI: 10.7759/cureus.57727 -
American Journal of Otolaryngology 2024To develop a multi-instance learning (MIL) based artificial intelligence (AI)-assisted diagnosis models by using laryngoscopic images to differentiate benign and...
OBJECTIVE
To develop a multi-instance learning (MIL) based artificial intelligence (AI)-assisted diagnosis models by using laryngoscopic images to differentiate benign and malignant vocal fold leukoplakia (VFL).
METHODS
The AI system was developed, trained and validated on 5362 images of 551 patients from three hospitals. Automated regions of interest (ROI) segmentation algorithm was utilized to construct image-level features. MIL was used to fusion image level results to patient level features, then the extracted features were modeled by seven machine learning algorithms. Finally, we evaluated the image level and patient level results. Additionally, 50 videos of VFL were prospectively gathered to assess the system's real-time diagnostic capabilities. A human-machine comparison database was also constructed to compare the diagnostic performance of otolaryngologists with and without AI assistance.
RESULTS
In internal and external validation sets, the maximum area under the curve (AUC) for image level segmentation models was 0.775 (95 % CI 0.740-0.811) and 0.720 (95 % CI 0.684-0.756), respectively. Utilizing a MIL-based fusion strategy, the AUC at the patient level increased to 0.869 (95 % CI 0.798-0.940) and 0.851 (95 % CI 0.756-0.945). For real-time video diagnosis, the maximum AUC at the patient level reached 0.850 (95 % CI, 0.743-0.957). With AI assistance, the AUC improved from 0.720 (95 % CI 0.682-0.755) to 0.808 (95 % CI 0.775-0.839) for senior otolaryngologists and from 0.647 (95 % CI 0.608-0.686) to 0.807 (95 % CI 0.773-0.837) for junior otolaryngologists.
CONCLUSIONS
The MIL based AI-assisted diagnosis system can significantly improve the diagnostic performance of otolaryngologists for VFL and help to make proper clinical decisions.
Topics: Humans; Artificial Intelligence; Vocal Cords; Laryngoscopy; Male; Leukoplakia; Female; Middle Aged; Aged; Diagnosis, Computer-Assisted; Machine Learning; Diagnosis, Differential; Adult; Algorithms; Laryngeal Neoplasms
PubMed: 38703609
DOI: 10.1016/j.amjoto.2024.104342