Complete Resolution Research Articles

Efficacy and Safety of Crisaborole 2% Ointment in the Treatment of Mild-to-moderate Atopic Dermatitis – A Prospective, Open-label Study at a Tertiary Care Centre in Eastern India

Objectives: Atopic dermatitis (AD) is a common occurrence worldwide, where topical corticosteroids and calcineurin inhibitors, along with antihistamines, are being used for the treatment of mild-to-moderate AD. Crisaborole 2% ointment, a phosphodiesterase-4 inhibitor, has been recently approved for AD treatment by the Food and Drug Administration. Crisaborole is proposed to have lesser side effects in comparison to its counterparts. However, the impact of crisaborole on the Indian population has only been studied in the paediatric population and has not been widely explored. We conducted this study to find the safety and efficacy of crisaborole 2% ointment in mild-to-moderate AD in the Indian population. Materials and Methods: Thirty consecutive mild-to-moderate AD patients were included in the study. The patients were advised to apply crisaborole 2% ointment at the site of involvement twice daily for 28 days. The evaluation was done at baseline and day 28 using the SCORing AD (SCORAD), investigator static global assessment (ISGA) and eczema-associated severity index (EASI) scores. The patients were followed up to report any side effects. Results: There was a statistically significant decrease in the mean SCORAD, ISGA and EASI scores after the end of treatment. According to SCORAD, there were 58.3% of patients with moderate AD which reduced to 41.6% at the end of treatment. ISGA defined treatment success as only being achieved by 36.6% of patients, out of which complete resolution was achieved only by 20% of patients at the end of treatment. The mean EASI scores showed a considerable decreasing trend from 8.47 at baseline to 4.73 at the end of the study. Six patients withdrew from the study due to exacerbation of symptoms, possibly burning at the site of application. Conclusion: Although crisaborole reduced symptoms, it did not provide any instant relief to symptoms of AD, leading to discontinuation in a few patients, making it a viable drug for maintenance rather than a first-line therapy.

Bronchiectasis with Transmediastinal Herniation of the Left Upper Lobe in a 3-Year-Old Child: A Case Report

Background: Bronchiectasis is a disorder marked by the destruction of smooth muscle and elastic tissue caused by inflammation, resulting in the permanent expansion of bronchi and bronchioles. It can occur following a single severe episode or repeated episodes of pneumonia, as well as exposure to tuberculosis. The Case: A child reported with cough and cold for 7 days, with mild fever. He was admitted to the hospital due to breathing difficulties and facial swelling. The clinical exam showed crepitation, wheezing, and pectus carinatum. Patient has history of multiple hospital admissions due to pneumonia and respiratory distress and exposure to tuberculosis. His mother was diagnosed and treated for tuberculosis when he was 3 months old. Condition of the patient was evaluated using ultrasonographic examination, chest radiograph and High-Resolution Computed Tomography of thorax. Conclusion: High-resolution Computed Tomography (HRCT) scanning is the preferred diagnostic test as it helps to identify the pathologic changes and the exact extent through which it has taken place. Early intervention plays a critical role in reducing severe complications like hemoptysis and cor pulmonale. The current treatment options consist of antibiotics, bronchodilators, anti-inflammatory medications, and physical therapy. The patient was treated using steroids, anti-microbials and inhalational bronchodilators. Complete symptom resolution was noted in two weeks from date of admission. He also seemed to be doing well in the follow-up visit, one week post discharge. Severe cases may require injectable antibiotics. As a widespread condition in India, early diagnosis and treatment with suitable antimicrobials is critical for a positive outcome.

Clinical application of mineralized collagen scaffolds in surgical treatment of skull defects

To explore the clinical application value of mineralized collagen (MC) bone scaffolds in repairing various types of skull defects, and to assess the suitability and repair effectiveness of porous MC (pMC) scaffolds, compact MC (cMC) scaffolds, and biphasic MC composite (bMC) scaffolds. A retrospective analysis was conducted on the clinical data of 105 patients who underwent skull defect repair with pMC, cMC, or bMC between October 2014 and April 2022. The cohort included 63 males and 42 females, ranging in age from 3 months to 55 years, with a median age of 22.7 years. Causes of defects included craniectomy after traumatic surgery in 37 cases, craniotomy in 58 cases, tumor recurrence or intracranial hemorrhage surgery in 10 cases. Appropriate MC scaffolds were selected based on the patient's skull defect size and age: 58 patients with defects <3 cm² underwent skull repair with pMC (pMC group), 45 patients with defects ≥3 cm² and aged ≥5 years underwent skull repair with cMC (cMC group), and 2 patients with defects ≥3 cm² and aged <5 years underwent skull repair with bMC (bMC group). Postoperative clinical follow-up and imaging examinations were conducted to evaluate bone regeneration, the biocompatibility of the repair materials, and the occurrence of complications. All 105 patients were followed up 3-24 months, with an average of 13 months. No material-related complication occurred in any patient, including skin and subcutaneous tissue infection, excessive ossification, and rejection. CT scans at 6 months postoperatively showed bone growth in all patients, and CT scans at 12 months postoperatively showed complete or near-complete resolution of bone defects in all patients, with 58 cases repaired in the pMC group. The CT values of the defect site and the contralateral normal skull bone in the pMC group at 12 months postoperatively were (1 123.74±93.64) HU and (1 128.14±92.57) HU, respectively, with no significant difference ( t=0.261, P=0.795). MC exhibits good biocompatibility and osteogenic induction ability in skull defect repair. pMC is suitable for repairing small defects, cMC is suitable for repairing large defects, and bMC is suitable for repairing pediatric skull defects.

Rhinolith Within a Giant Concha Bullosa: A Case Report and Review of Literature.

Concha bullosa is a common anatomical variation involving pneumatization of the middle turbinate. Although usually asymptomatic, a large concha bullosa can obstruct nasal airflow and cause related symptoms. Rhinoliths, calcified formations typically found in the inferior meatus, rarely appear within concha bullosa. This report presents a rare case of a rhinolith within a giant concha bullosa in a 15-year-old male experiencing progressive, unilateral nasal obstruction. Computed tomography (CT) revealed a densely-calcified mass inside of an extensive concha bullosa, accompanied by nasal septal deviation and maxillary sinus opacity. The patient underwent successful endoscopic sinus surgery with excision of the concha bullosa's lateral and medial laminae, facilitating complete rhinolith removal and correction of septal deviation. Postoperative follow-up over 6 months showed complete symptom resolution with no recurrence. This case underscores the importance of CT imaging in evaluating unusual nasal obstructions and highlights endoscopic sinus surgery as an effective approach for extensive concha bullosa. The findings emphasize careful consideration of anatomical variations in clinical practice and contribute to the limited literature on rhinolith formation within concha bullosa. Further studies are warranted to understand the pathogenesis of rhinoliths in nasal anatomical variations.

Treatment-emergent central sleep apnea resolves with lower inspiratory pressure.

Treatment emergent central sleep apnea (TECSA) is an important problem during therapy with continuous positive airway pressure (CPAP) in patients with obstructive sleep apnea (OSA). Here we tested a device designed to improve CPAP comfort through reducing IPAP (-Com) to determine if such a reduction in IPAP could eliminate central apneas in patients with TECSA. Since increasing tidal volume (potentially via IPAP increments) has been suggested as a possible mechanism contributing to TECSA onset, our hypothesis was that reducing IPAP would yield a drop in the central apnea index (CAI). The addition of a known resistance (-Com device) that reduces IPAP was implemented into the CPAP circuit during the second half of CPAP titrations in a cohort of community-dwelling patients who developed TECSA during a split-night CPAP titration. CAI was quantified from the sleep periods without and with -Com in place. 1613 patients underwent CPAP titration with 19 of them developing TECSA during the titration. The addition of -Com resulted in complete resolution of TECSA in all patients with adequate sleep data under all conditions (N=13), yielding a significant reduction in the CAI (17.3 ± 11.0 vs. 1.5 ± 1.7 events/h without and with -Com, respectively, P<0.001). -Com virtually resolved all instances of TECSA, suggesting that reducing IPAP could be an effective strategy for managing the occurrence of central respiratory events in OSA patients using CPAP.

Factors Influencing Favourable Clinical Outcomes in Idiopathic Intracranial Hypertension with Venous Sinus Stenosis Stenting.

This study aimed to investigate the factors influencing favourable clinical outcomes in idiopathic intracranial hypertension (IIH) patients with venous sinus stenosis (VSS) who received stenting treatment. In this prospective cohort study, we analyzed atotal of 172 IIH patients with VSS treated with venous sinus stenting. Clinical outcomes were categorized as either "complete resolved" or "improved". We employed multivariable logistic regression modeling to identify independent factors associated with the clinical outcomes. The predictive accuracy of the model was assessed using receiver operating characteristic (ROC) analysis. Out of the total cohort, 126 patients exhibited asymptomatic clinical outcomes, while 46patients showed improved clinical outcomes. Patients with asymptomatic clinical outcomes demonstrated notably lower preoperative BMI (26.8 kg/m2 versus 28.7 kg/m2, P = 0.013). In multivariate analysis, preoperative BMI (odds ratio [OR] = 1.108, 95% confidence interval [CI] = 1.009-1.216) was an independent factor of complete resolution of the symptoms. In the receiver operating characteristic (ROC) analysis, the identified cut-off BMI for the highest sensitivity(0.5) and specificity (0.754) was determined to be 29.3 kg/m2. Lower BMI may be associated with the complete resolution of symptoms in IIH patients with VSS who received venous sinus stenting treatment.

Treatment of Hereditary Leukonychia with Intramatricial Triamcinolone: Case Report

Introduction: Hereditary leukonychia is a rare genetic nail disorder characterized by whitening of the nail plate, which is sometimes due to mutations in the phospholipase C delta-1 (PLCδ1) gene. While leukonychia is typically asymptomatic, it carries significant psychosocial burden, and patients often report that others comment that they look like they are wearing nail polish. There are no known treatment options. Case Presentation: A 39-year-old Kuwaiti male with autosomal recessive variant of PLCδ1-related non-syndromic leukonychia affecting nine fingernails presented for treatment because he was socially stigmatized. Treatment with 30% glycolic acid chemical peel was ineffective. Because there is evidence suggesting a link between PLCδ1-related hereditary leukonychia and abnormal keratinization, we hypothesized that intralesional steroids, which inhibit keratinocyte activity and reduce inflammation within the nail matrix, may be an effective treatment. Following treatment with intramatricial triamcinolone, the patient experienced complete resolution of leukonychia in five fingernails and partial improvement in the remaining four. Conclusion: We present a case of successful treatment of hereditary leukonychia due to a phospholipase C δ-1 gene mutation with intramatricial triamcinolone. This case highlights the potential of this treatment approach for leukonychia and warrants further investigation in larger cohort studies.

Case Report: Surgical treatment of type B aortic dissection in an adult with double aortic arch

BackgroundDouble aortic arch (DAA) with type B aortic dissection in adults is a rare aortic vascular disease. The abnormal anatomical structure of the aortic arch in such patients presents significant challenges in the selection of surgical approaches, and there is a notable lack of exploration into endovascular repair approaches that simultaneously preserve asymptomatic vascular rings.Case descriptionA 43-year-old female patient was admitted due to recurrent chest and back pain lasting for over a month. Computed tomography angiography (CTA) indicated a double aortic arch anomaly with localized dissection of the descending aorta. Esophagography with barium swallow revealed vascular indentation on the upper and middle thoracic esophagus, with mild to moderate local narrowing. Based on a comprehensive preoperative evaluation of the imaging and the patient's clinical history, a thoracic endovascular aortic repair (TEVAR) procedure was performed. Considering that the deformity did not cause any clinical symptoms and that the vessel diameter and distance from the proximal anchoring area were sufficient, the posterior section of the dominant arch was chosen as the proximal anchorage zone, and a stent with proximal bare zone was deployed to maintain blood flow to the distal non-dominant arch and preserve the integrity of the vascular ring. Follow-up CTA scans at one- and six-month post-operation showed that the aortic stent was well-positioned, with no visible primary lesion. The patient reported complete resolution of chest pain and no difficulties with swallowing or breathing.ConclusionIn adult patients with DAA complicated by aortic dissection, the abnormal anatomy of the aortic arch poses significant challenges in making treatment decisions. After a comprehensive, multidimensional evaluation of the patient's medical history, CTA, and esophagography, we successfully performed TEVAR procedure. This case provides new insights into the surgical strategy for treating such rare conditions.

Intercostal bypass graft pseudoaneurysm repaired with percutaneous glue embolization utilizing CT needle guidance: A case report.

Pseudoaneurysm formation in connective tissue patients postoperatively can be a challenging and life-threatening problem to treat. Repair of an aorta to intercostal bypass graft pseudoaneurysm post open thoracoabdominal aortic aneurysm repair by percutaneous glue embolization utilizing Dyna CT Needle Guidance technology is a safe, minimally invasive method with low morbidity and short post-procedural recovery. A 30-year-old male with suspected connective tissue disorder who developed a Type B aortic dissection complicated by limb threatening right lower extremity ischemia treated with left-to-right fem-fem bypass and RLE fasciotomies. His thoracic aortic dissection subsequently was repaired urgently for persistent chest pain and rapid aortic expansion by open thoracoabdominal aortic aneurysm repair with aorta to intercostal bypass graft. Seven weeks postop patient presented with hemoptysis and was noted to have an enlarging T9 intercostal pseudoaneurysm requiring repair. Post discharge follow-up thoracic CTA performed showed complete resolution of the T9 intercostal pseudoaneurysm. Therefore, percutaneous NBCA glue embolization with iGuide technology is a viable alternative to open thoracic pseudoaneurysm repair and offers distinct advantages over other imaging modalities for localization.

Successful treatment of chronic pulmonary aspergillosis in a patient with early pulmonary tuberculosis and COVID-19: a case report.

Chronic pulmonary aspergillosis (CPA) often develops in residual lesions of pulmonary tuberculosis (PTB). Every year, 112,000 to 160,000 people worldwide will develop post-PTB CPA. The simultaneous occurrence of CPA with the first episode of PTB is rare. During the COVID-19 pandemic, COVID-19- associated invasive aspergillosis (CAPA) occurred in patients receiving high doses of corticosteroids and mechanical ventilation. However, CPA and COVID-19 are rarely reported simultaneously. This case study presents a patient with CPA in the first episode of PTB during hospitalization for COVID-19. The favorable evolution is highlighted, including the resolution of the cavitation and fungal ball with appropriate and early treatment. A 48-year-old female patient from the Central West of Brazil was admitted with a history of cough, yellow sputum, fever, and significant weight loss for two months. The respiratory symptoms worsened one week before admission. She tested positive for COVID-19 by RT-PCR. She had a history of hypertension and diabetes. Clinical examination revealed tachypnea, slurred speech, and hypoxia. She presented with hyperglycemia, obesity, hypertension, and an episode of hemoptysis. Chest CT revealed cavitation in the right upper lobe with a 45 mm aspergilloma, multifocal morning opacities, and nodular opacities. Laboratory tests confirmed the PTB with positive sputum for acid-fast bacilli and positive culture for Mycobacterium tuberculosis. The sputum culture also showed Aspergillus spp. She received early treatment for bacterial pneumonia with ceftriaxone, dexamethasone, enoxaparin, an anti-TB regimen, and itraconazole. There was a progressive clinical improvement and the patient was discharged after 15 days. She completed six months of anti-TB therapy and 13 months of itraconazole treatment for CPA, with complete resolution of the cavitation and aspergilloma. This case study presents a unique case of CPA that manifested as simple aspergilloma and was diagnosed concurrently with the initial episode of PTB in a COVID-19 patient with obesity, hypertension, and diabetes. Remarkably, the fungal ball and cavitation regressed spontaneously. The favorable clinical and radiological results highlight the importance of comprehensive treatment approaches for concurrent respiratory infections and emphasize the need to investigate CPA and PTB during COVID-19 hospitalization.

Efficacy and safety of 5-aminolevulinic acid photodynamic therapy in refractory genital lichen sclerosus

BackgroundGenital lichen sclerosus (GLS) is a chronic inflammatory skin disease affecting the genital and anal areas. Treatment of refractory remains a challenge in clinical practice. ObjectiveThis study aimed to evaluate the efficacy and safety of 5-aminolevulinic acid photodynamic therapy (5-ALA PDT) for refractory GLS. MethodsThirty-five patients with histopathologically confirmed GLS (including 32 females and 3 males) were treated with 5-ALA PDT. Lesion pretreatment was performed to enhance photosensitizer penetration, and fluorescence diagnosis was used for precise point-to-point treatment. Efficacy and safety were systematically evaluated by assessing treatment outcomes, symptom improvement, side effects and maintenance of efficacy over a 6-month follow-up period. ResultsAfter treatment, most patients experienced significant relief of pruritus, improved skin elasticity and color, and reduced lesion area. After 6 treatment sessions, 11 patients (31.43%) achieved complete resolution of pruritus, with 3 patients (8.57%) achieving complete remission after 3 sessions. Cattaneo scores decreased significantly after treatment and during follow-up (P<0.05). Adverse reactions were mainly mild pain, which could be relieved by ice compression or oral analgesics. Conclusions5-ALA PDT shows significant efficacy and good safety for refractory GLS, improving the clinical symptoms and signs of patients, and warrants wider clinical application.

Arachnoidal cyst of the brain with post-traumatic hemorrhage: case report and literature review

Introduction. Arachnoid cysts (AC) occur in 1-3% of children. There is sacs with spinal fluid between brain and arachnoid membrane. Only about 6% of intracranial AC manifest clinically. AC can be complicated by hemorrhage with traumatic brain injury and spontaneously. Materials and methods. We have described a clinical case of AC, complicated by hemorrhage with the subdural hematoma after brain injury and literature review. There is unusually that after drainage of the hematoma, complete resolution of the AK was noted (the only such case described in the literature). Conclusion. The asymptomatic AC can be complicated by hemorrhage due brain injury or spontaneously. Due to the high elastic properties of the brain in children, complete regression of even long-existing AC is possible.

Use of ionized plasma jet therapy in the treatment of xanthelasma palpebrarum

Objectives: To evaluate the safety and efficacy of ionized plasma therapy for the treatment of xanthelasma palpebrarum. Material and Methods: A total of 35 patients were enrolled for the study. Patients were screened as per the predefined inclusion and exclusion criteria. The patients were treated with ionized plasma jet therapy (IPJT) plasma machines. Pre- and post-sizes of the xanthelasma lesions were noted along with the side effects, and the patients were followed up for a period of 90 days. Results: The complete resolution of the xanthelasma lesion was observed in 28 out of 35 patients, with two patients requiring two sessions, five patients developing hypopigmentation, and 1 patient having hyperpigmentation as a side effect, which was resolved after 4 weeks. Conclusions: Based on the results, ionized plasma therapy for xanthelasma palpebrarum can be recommended as a safe and effective option.

Serendipitous Discovery of Lipemia Retinalis in a Premature Infant: A Case Report

Introduction Lipemia retinalis (LR) is a rare ocular condition characterized by a creamy white appearance of the retinal vessels, which is indicative of significantly elevated serum triglyceride (TG) levels. This condition is particularly noteworthy in pediatric populations, especially in preterm infants who are at risk for various metabolic disorders. Case Presentation We report a case of LR in a 44-day-old preterm infant identified during routine retinopathy of prematurity (ROP) screening. Unusual retinal findings led to metabolic investigations, revealing elevated TG levels confirming LR. The infant was managed with a dietary regimen including skimmed milk, resulting in a substantial decrease in TG levels and complete resolution of the retinal changes. Conclusion Fundus examination serves as a portal to the systemic health of an individual, enabling rare identification of LR, indicative of underlying dyslipidemia. This case underscores the necessity for comprehensive metabolic screening in preterm infants, particularly during ROP assessments. Furthermore, it emphasizes the effectiveness of dietary interventions in managing LR. The favorable outcomes stress the significance of early detection of LR in pediatric patients and implementing nutritional strategies to enhance metabolic well-being, thereby averting severe hypertriglyceridemia complications.

Choroidal Response to Intravitreal Bevacizumab Injections in Treatment-Naïve Macular Neovascularization Secondary to Chronic Central Serous Chorioretinopathy

Background/Objectives: To evaluate the impact of intravitreal bevacizumab (IVB) therapy on anatomical and visual outcomes in patients with macular neovascularization (MNV) secondary to chronic central serous chorioretinopathy (cCSC). Methods: This retrospective observational study reviewed the medical records of treatment-naïve patients diagnosed with cCSC complicated by MNV and treated with IVB injections over a 5-year period. The presence of MNV was confirmed using optical coherence tomography angiography (OCTA). Best-corrected visual acuity (BCVA), subfoveal choroidal thickness (SFCT), and subretinal fluid (SRF) were recorded pre- and post-IVB treatment. Results: Twenty-two eyes of 22 patients (mean age, 68 ± 11 years) were included. After a mean follow-up of 21.0 ± 14.6 months, SRF significantly decreased from baseline (176.86 ± 115.62 µm) to the final follow-up (80.95 ± 87.32 µm, p = 0.003). A greater SRF reduction was associated with more injections (&gt;7) (p = 0.047). However, no significant changes were observed in BCVA (p &gt; 0.05) or SFCT (p &gt; 0.05), irrespective of follow-up duration or injection frequency. Complete resolution of SRF was achieved in nine patients (40.9%), and a significantly greater reduction in SFCT was observed in complete responders compared to non-responders (p = 0.03). Conclusions: IVB therapy significantly reduced SRF in cCSC patients with secondary MNV, though it did not lead to visual improvement or significant changes in SFCT. However, greater choroidal thinning in patients with complete fluid resorption may suggest distinct underlying mechanisms or alternative sources of subretinal fluid beyond the MNV itself.

Compassionate access to virus-specific T cells for adoptive immunotherapy over 15 years

Adoptive T-cell immunotherapy holds great promise for the treatment of viral complications in immunocompromised patients resistant to standard anti-viral strategies. We present a retrospective analysis of 78 patients from 19 hospitals across Australia and New Zealand, treated over the last 15 years with “off-the-shelf” allogeneic T cells directed to a combination of Epstein–Barr virus (EBV), cytomegalovirus (CMV), BK polyomavirus (BKV), John Cunningham virus (JCV) and/or adenovirus (AdV) under the Australian Therapeutic Goods Administration’s Special Access Scheme. Most patients had severe post-transplant viral complications, including drug-resistant end-organ CMV disease, BKV-associated haemorrhagic cystitis and EBV-driven post-transplant lymphoproliferative disorder. Adoptive immunotherapy is well tolerated with few adverse effects. Importantly, 46/71 (65%) patients show definitive clinical improvement including reduction in viral load, clinical symptoms and complete resolution of end-organ disease. In addition, seven high-risk patients remain disease free. Based on this long-term encouraging clinical experience, we propose that a dedicated nationally funded centre for anti-viral cellular therapies should be considered to provide T cell therapies for critically ill patients for compassionate use.

Spontaneous resolution of a giant bulla in a patient presenting with COVID-19 with presumed superadded bacterial infection.

Bullous emphysema is a chronic disease characterized by bullae, or air spaces in the lungs. Giant bullae exceed one-third of the hemithorax volume and are usually treated via bullectomy. We present the case of a 35-year-old man who presented to hospital with a history of COVID-19 infection and seven days of chest pain and dyspnoea. A giant left upper lobe fluid-filled bulla was identified on computed tomography. He was discharged with a course of antibiotics. A radiograph performed one month after presentation revealed an unchanged giant bulla. However, a chest radiograph and computed tomography nine months after initial presentation showed complete spontaneous resolution of the bulla. Bullectomy was deemed unnecessary. Cases of spontaneous bullae resolution, or autobullectomy, are rare. Our case implicates the role of infectious processes in autobullectomy. Serial imaging monitoring and delayed cardiothoracic assessment may be prudent to assess bullectomy necessity.

Successful novel use of dupilumab for gastrointestinal involvement of idiopathic hypereosinophilic syndrome: case report and review of the literature.

Hypereosinophilic syndrome (HES) is characterized by blood and tissue hypereosinophilia leading to organ damage. Gastrointestinal involvement is the third most common manifestation. We present a patient with idiopathic HES with secondary eosinophilic esophagitis (EoE), gastritis, and enteritis, corticosteroids-dependent, azathioprine- and mepolizumab-refractory. The patient achieved clinical and histopathologic remission following dupilumab treatment. A 28year-old female presented with chronic episodic nausea and emesis since childhood and initial diagnosis of primary eosinophilic gastrointestinal disease (EGID), improved with corticosteroids, refractory to azathioprine. She was found to have peripheral eosinophilia and multifactorial anemia, with iron, B12, and folate deficiencies. Esophageal, gastric, duodenal, and terminal ileum biopsies showed significant eosinophilic infiltrate. Bone marrow biopsy at age 31 confirmed HES diagnosis. By age 32, she became total parental nutrition (TPN)-dependent. She failed trials of benralizumab and mepolizumab [anti-interleukin (IL)-5 inhibitors], and cromolyn (mast-cell stabilizer). After developing new esophageal stricture, we initiated dupilumab (IL-4/13 inhibitor), recently FDA-approved for EoE. After 9weeks, esophageal stricture, gut tissue eosinophilia, and prior intestinal ulcerations resolved. She ceased TPN and is tolerating a non-restricted diet, with complete symptom resolution. Our patient's complete remission with dupilumab shows promise for broadening its use in treating GI involvement in HES, along with primary EGIDs.

Zenker’s Per-Oral Endoscopic Myotomy (Z-POEM): The Ideal First Line Therapy for Treatment of Zenker’s Diverticulum

Zenker's diverticulum is a mucosal herniation at the pharyngoesophageal junction. While open surgical myotomy is the conventional treatment, robust evidence on third-space flexible endoscopic myotomy is lacking. We assessed safety, effectiveness and patient-reported outcomes of per-oral endoscopic myotomy (Z-POEM) in the largest reported single-centre experience with this technique. Retrospective cohort study was performed of consecutive patients undergoing Z-POEM in a tertiary hospital thoracic surgery practice from 2018-2024. Data collected included demographics, comorbidities, clinical and radiographic (pre/post-operative esophagram) outcomes, and validated patient-reported quality-of-life outcomes (Dakkak and Eckardt scores). Descriptive analyses were used to describe the population, outcomes, and complications. Aggregate and individual-patient symptom trajectories were charted over time. Fifty-seven patients (57.9% female, n=33) with a mean age of 74 years underwent Z-POEM. Median diverticulum size was 2 cm (IQR 2-3). All patients reported subjective symptom improvement and showed complete resolution of obstructed barium flow on postoperative esophagrams. At median follow-up of 9 months, all Z-POEM patients reported clinically-important improvements in objective symptom scores, with symptom trajectories showing rapid and durable improvement up to 5 years postoperatively. Four patients (7%) experienced complications. Majority of the cohort (66.7%) was unsuitable for rigid stapled myotomy, mainly due to short diverticulum or limited neck/mouth movement. No patients required repeat myotomy. We demonstrate significant and sustained relief of patient-reported outcomes/symptoms following Z-POEM, including for small diverticuli, with low complication rates. Its safety and versatility has made it the new standard of care locally; Z-POEM can be adopted by third-space endoscopy-trained thoracic surgeons.

Subretinal Gene Therapy Drug AGTC-501 for XLRP Phase 1/2 Multicenter Study (HORIZON): 24-Month Safety and Efficacy Results: Subretinal Gene Therapy AGTC-501 for XLRP Ph 1/2 24M Results

PurposeTo evaluate the safety and efficacy of subretinal gene therapy using AGTC-501 (rAAV2tYF-GRK1-RPGR) in male participants with X-linked retinitis pigmentosa (XLRP). DesignPhase 1/2, open-label, dose-escalation study. MethodsSetting: Four centers in the United States. Patient or Study Population: Twenty-nine males with XLRP and confirmed pathogenic RPGR variants. Mean age was 31.6 years (range 15-55). Intervention: Subretinal injection of AGTC-501 at doses ranging from 2.48 × 1010 to 1.99 × 1012 vg/eye administered in one eye per participant. Subretinal injection sites initially targeted the peripheral retina and then transitioned to the macula with successive cohorts. Main Outcome Measures: Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), laboratory parameters, and immunological responses. Efficacy was evaluated by mesopic microperimetry mean sensitivity. ResultsAll 29 participants experienced ≥1 TEAE. Eleven (38%) experienced ≥1 grade 3 TEAE. Six (21%) experienced ≥1 ocular SAE related to AGTC-501, including retinal detachment (n=4), subcapsular cataract (n=1), and glaucoma (n=1). Two (6.9%) experienced non-ocular treatment-emergent SAEs. Immunological findings did not indicate safety concerns. Three of 4 participants at the highest dose exhibited concerning retinal pigment epithelial changes. Half the participants at the highest tolerated dose (6.8 × 1011vg/eye) maintained ≥7 dB improvement in ≥5 loci at 24 months. ConclusionsSubretinal AGTC-501 was generally well-tolerated. Despite all participants experiencing at least one TEAE, most of these events were mild in nature, exhibited complete resolution, and were associated with the subretinal injection procedure itself rather than the study agent. The highest dose exhibited an unfavorable risk-benefit profile due to the development of RPE changes. Although this group had the highest improvement in retinal sensitivity, our team has decided not to continue this dose in future clinical trials. Preliminary efficacy was observed at the maximum tolerated dose. Further studies are warranted to assess long-term safety and efficacy of AGTC-501 for XLRP treatment.