Complete Resolution Of Disease Research Articles

Factors associated with response to talimogene laherparepvec in the treatment of advanced melanoma

Talimogene laherparepvec (T-VEC) is currently the only United States Food and Drug Administration-approved intralesional therapy for advanced melanoma. Recent studies have assessed the integration of T-VEC with systemic immunotherapy, though the response remains variable. Therefore, we sought to identify factors associated with the response to T-VEC by conducting a retrospective, single-center analysis involving melanoma patients treated with T-VEC. In the present study, we recorded demographic and clinicopathological data, details of T-VEC treatments, prior and concurrent treatments, and clinical outcomes. The primary endpoint was the in-field overall response rate (ORR: complete + partial). Secondary endpoints included complete in-field response, defined as complete resolution of disease or a negative biopsy; disease failure-free survival (DFFS), defined from the initiation of treatment as the time to progression in patients who did not experience a disease-free interval and time to recurrence in those who did; and overall survival (OS). We used two-sample t-tests for continuous variables and Fisher’s exact test for categorical variables. DFFS and OS were further analyzed using the Kaplan–Meier method and log-rank tests for selected variables. Among the 18 patients who met the inclusion criteria, an in-field response was observed in 14 (78%) patients. Low disease burden (<5 lesions or a total diameter <5 cm) was associated with a higher in-field ORR compared to high-burden disease (100% vs. 43%, P = 0.01). There was a trend toward decreased in-field ORR for patients with >2 prior lines of systemic therapy (P = 0.18). With a median follow-up of 386 days, DFFS was associated with BRAF wild-type melanoma (P = 0.04), an in-field ORR (P = 0.007), and a measurable bystander response (P = 0.01). Two or more prior lines of immunotherapy were associated with poorer survival (P = 0.006) and worse DFFS (P = 0.02). In conclusion, despite a low sample size, we identified covariates associated with in-field ORR, DFFS, and OS, warranting further study.

Leveraging User-Friendly Mobile Medical Devices to Facilitate Early Hospital Discharges in a Pediatric Setting: A Randomized Trial Study Protocol.

Mobile technology is increasingly prevalent in healthcare, serving various purposes, including remote health monitoring and patient self-management, which could prove beneficial to early hospital discharges. This study investigates the transitional care program experience facilitating early discharges in a pediatric setting through the use of an easy-to-use mobile medical device (TytoCare™, TytoCare Ltd., Natanya, Israel). This study aims to assess the effectiveness of telehomecare in achieving complete resolution of diseases without readmission, compare the length of stay between intervention and standard care groups, and gather user and professional experiences. A randomized open-label, controlled pilot study enrolled 102 children, randomly assigned to the telehomecare (TELE) group (n = 51, adopting early hospital discharge with continued home monitoring) or the standard-of-care (STAND) group (n = 51). Primary outcomes include complete disease resolution without readmission. Secondary objectives include recording a shorter length of stay in the intervention group. Surveys on user and professional experiences were conducted. A group of 51 children declining telemedicine services (NO-TELE) was also included. In the TELE group, 100% of children achieved complete disease resolution without readmission, with a median duration of stay of 4 days, significantly shorter than the 7 days in the STAND group (p = 0.01). The telemedicine system demonstrated efficient performance and high satisfaction levels. The NO-TELE group showed no significant differences in demographics or digital technology competence. Perceived benefits of telemedicine included time and cost savings, reduced hospital stays, and technology utility and usability. This study demonstrates that user-friendly mobile medical devices effectively facilitate early hospital discharges in a pediatric setting. These devices serve as a bridge between home and hospital, optimizing care pathways.

Upfront Low-Dose Cytarabine with Prednisolone for Langerhans Cell Histiocytosis with Liver Dysfunction: A Ray of Hope

AbstractThe management of Langerhans cell histiocytosis (LCH) with accompanying liver dysfunction poses significant challenges, and this prompted the development of a modified low-dose cytarabine and prednisolone regimen. In this prospective observational study on children with LCH and liver dysfunction, four patients underwent induction and maintenance chemotherapy. The induction phase included 5 days of 100 mg/m2 cytarabine and 4 weeks of 40 mg/m2 daily prednisolone, with subsequent tapering. Maintenance included a regimen of 5 days of 100 mg/m2 cytarabine, along with oral prednisolone, repeated every 3 weeks. Complete disease resolution occurred after varying chemotherapy cycles. Three patients had liver transplants, and the chemotherapy resumed for 52 weeks after the transplant. In one child, chemotherapy was continued after reaching remission. In conclusion, a modified, less toxic low-dose cytarabine-based chemotherapy effectively managed LCH with liver dysfunction, with liver transplantation as a postremission treatment option.

Deep learning-based osteochondritis dissecans detection in ultrasound images with humeral capitellum localization.

Osteochondritis dissecans (OCD) of the humeral capitellum is a common cause of elbow disorders, particularly among young throwing athletes. Conservative treatment is the preferred treatment for managing OCD, and early intervention significantly influences the possibility of complete disease resolution. The purpose of this study is to develop a deep learning-based classification model in ultrasound images for computer-aided diagnosis. This paper proposes a deep learning-based OCD classification method in ultrasound images. The proposed method first detects the humeral capitellum detection using YOLO and then estimates the OCD probability of the detected region probability using VGG16. We hypothesis that the performance will be improved by eliminating unnecessary regions. To validate the performance of the proposed method, it was applied to 158 subjects (OCD: 67, Normal: 91) using five-fold-cross-validation. The study demonstrated that the humeral capitellum detection achieved a mean average precision (mAP) of over 0.95, while OCD probability estimation achieved an average accuracy of 0.890, precision of 0.888, recall of 0.927, F1 score of 0.894, and an area under the curve (AUC) of 0.962. On the other hand, when the classification model was constructed for the entire image, accuracy, precision, recall, F1 score, and AUC were 0.806, 0.806, 0.932, 0.843, and 0.928, respectively. The findings suggest the high-performance potential of the proposed model for OCD classification in ultrasonic images. This paper introduces a deep learning-based OCD classification method. The experimental results emphasize the effectiveness of focusing on the humeral capitellum for OCD classification in ultrasound images. Future work should involve evaluating the effectiveness of employing the proposed method by physicians during medical check-ups for OCD.

Unilateral Parotid Gland Enlargement in Newborn: Is it Suppurative Parotitis?

Acute neonatal suppurative parotitis (ANSP) is a rare disease and a novel illness affecting newborns and premature neonates are more likely to contract it. A unilateral parotid swelling and pus discharge from the Stensen’s duct is pathognomonic and sonography is the first line tool to supplement the diagnosis of parotitis. An appropriate parenteral antibiotic therapy and adequate hydration are the mainstays of treatment. Herein, we report a case of ANSP in a 12-day-old baby girl with a 48-hour history of left pre-auricular swelling where a complete resolution of disease was achieved post-treatment.

Resolution of peri-implant mucositis following standard treatment: A prospective split-mouth study.

Peri-implant mucositis (PIM) is a pathological precursor of peri-implantitis, but its pattern of conversion to peri-implantitis is unclear and complicated to diagnose clinically, while none of the available protocols yield complete disease resolution. The aim of this study was the evaluation of PIM responsiveness to standard anti-infective mechanical treatment (AIMT) at clinical and biomarker levels, and estimation of the diagnostic capacity of bone markers as surrogate endpoints and predictors. Systemically healthy outpatients presenting one implant exhibiting clinical signs of inflammation confined within the soft tissue (PIM) and one healthy control (HC) implant at a non-adjacent position were included. Clinical parameters and peri-implant crevicular fluid samples were collected baseline and 6 months following mechanical therapy, to assess the levels of RANKL, OPG, and IGFBP2. PIM clustering was performed using machine learning algorithms. Overall, 38 patients met the inclusion criteria. Therapy resulted in the reduction of all clinical and biological indicators, but respective values remained significantly higher compared to HC. Clinical examination noted 30% disease resolution at the 6-month follow-up, while 43% showed no active bone resorption. OPG showed positive prognostic value for treatment outcome, while the clustering based on active bone resorption did not differ in terms of therapeutic effectiveness. AIMT is effective in reducing the clinical and biological indicators of PIM, but complete clinical resolution was achieved in only 30% of the cases. Around one third of PIM patients exhibited active bone resorption bellow clinical detectability that was not associated with disease progression and poor treatment responsiveness.

The hidden star: mastoiditis as a late sequela of retained foreign body in the nose

BackgroundAcute mastoiditis is an intratemporal complication due to acute otitis media (AOM). Common risk factors include young age (often < 24 months), high values of C-reactive protein and previous antibiotic therapy, previous middle ear infection, or history of ear surgery. The main factor of acute mastoiditis is obstruction of the aditus antrum by granulation tissue or mucosa edema, inhibiting purulent drainage from the mastoid. Retained foreign body in the nose is a rare indirect cause of acute mastoiditis. It may be due to mucosal inflammation and accumulation of secretion causing nasal tract or nasopharyngeal infection leading to otitis media and later manifesting as an intratemporal complication.Case presentationWe report a case of acute mastoiditis as a late sequela of retained foreign body in the nose, and we also emphasize the incidental radiological finding of a star-shaped foreign body in the nose, which was a miss during the initial presentation. Further questioning revealed a history of recurrent unilateral blood-stained, foul-smelling nasal discharge, which was left untreated for years, leading to a delayed presentation of acute mastoiditis. After removing the foreign body and broad-spectrum antibiotic administration, complete disease resolution was achieved.ConclusionAlthough rare, retained foreign bodies of the nose must be ruled out in certain cases of mastoiditis. A thorough history with a high index of clinical suspicion is recommended for early diagnosis to prevent complications.

Evaluation of the adjunctive use of Er:YAG laser or erythritol powder air-polishing in the treatment of peri-implant mucositis: A randomized clinical trial.

To assess the efficacy of Er:YAG laser (ERL) and erythritol powder air-polishing (AP) in addition to the submarginal instrumentation in the non-surgical treatment of peri-implant mucositis (PM). Patients with at least one implant diagnosed with PM were included in the present 6-month randomized clinical trial (RCT). Implants were randomly assigned to one of the three treatment groups after submarginal instrumentation: AP (test 1 group), ERL (test 2 group) or no adjunctive methods (control group). The primary and secondary outcomes were, respectively, bleeding on probing (BoP) reduction and, complete disease resolution (total absence of BoP) and probing pocket depth (PPD) changes. The patient and the implant were considered the statistical unit. A multivariate logistic regression analysis was performed. A total of 75 patients were enrolled in the study. At each time point, significant BoP and PPD reductions were observed within each group. Intergroup analysis did not show statistically significant differences. Complete disease resolution ranged between 29% and 31%. The logistic regression showed that supramucosal restoration margin, PPD < 4 mm and vestibular keratinized mucosa (KM) significantly influenced the probability to obtain treatment success. The adjunctive use of AP and ERL in PM non-surgical therapy does not seem to provide any significant or clinically relevant benefit in terms of BoP and PPD reductions and complete disease resolution, over the use of submarginal instrumentation alone. Baseline PPD < 4 mm, presence of buccal KM and supramucosal restoration margin may play a role in the complete resolution of PM.

Definitive ablative stereotactic partial breast irradiation in early stage inoperable breast cancer.

This case series and literature review aims to investigate the efficacy and safety of definitive ablative radiation therapy as a treatment modality for non-operable patients with early stage breast cancer. We present two cases demonstrating the potential of this approach to achieve durable responses. We assessed the long-term response of two non-operable patients diagnosed with Stage II (cT2N0M) and Stage IA(T1bN0M0)invasive ductal carcinoma (IDC), who were deemed unfit for surgery due to significant co-morbid conditions. Definitive ablative radiation therapy was administered using stereotactic partial breast irradiation with ablative doses delivered in either a single fraction or two fractions. Serial imaging was conducted to assess treatment response and monitor adverse events. Both patients exhibited notable treatment responses following definitive ablative radiation therapy. The first patient, an 84-year-old woman, experienced a 69% reduction in tumor size over a follow-up period exceeding 2years. The second patient, an 87-year-old woman, achieved complete resolution of disease on imaging, with no signs of progression even 26month post-treatment. Both patients tolerated the treatment well, without significant treatment-related adverse events. Our case series suggests that definitive ablative radiation therapy may serve as a safe and effective treatment option for non-operable patients with early stage breast cancer. The observed durable treatment responses and minimal toxicity support the potential of this approach. Furthermore, a longer interval between ablative radiation therapy and surgery may enhance treatment response, potentially leading to increased complete pathologic response rates.

Preclinical testing of FOLR1-CAR T cells against osteosarcoma (OS).

2555 Background: Survival rates for OS are poor. Prior literature suggests folate receptor-a (FOLR1) overexpression in OS, making it a target for CAR T development. Methods: OS transcriptome data from TCGA, PCGP and NCI PIVOT programs were interrogated for FOLR1 gene expression. A 2nd generation FOLR1-CAR T cell was created by fusing the ScFv from farletuzumab (anti-FOLR1 monoclonal antibody) with a 4-1BB costimulatory and CD3z cytotoxicity domain. Non-transduced (NT) T cells served as controls. In vitro functional analysis was performed against U2OS cell line using flow cytometry (FC) and Incucyte. U2OS xenograft in NSG mice was generated via intra-femoral inoculation for local tumor and via tail vein (TV) tumor injection for pulmonary disease. Tumor engraftment was confirmed by in vivo imaging system (IVIS). Disease progression was monitored by IVIS and CT imaging. 5 million T cells were administered via TV. Circulating human T cells (HTC) collected from retro-orbital peripheral blood (PB) were detected via FC. Results: FOLR1 transcript was expressed in &gt;75% of OS in TCGA, PCGP and PIVOT. In vitro evaluation of FOLR1-CAR T demonstrated robust antitumor activity (E:T ratio 1:1) with cytotoxicity of 76.5% and 95.5% at 6 and 24 hrs. Disease progression was observed in NT T cell treated mice, with an average fold increase in IVIS radiance of 35.1 in local (n=2) and 142.8 in pulmonary disease (n=3) over 11 weeks (wks) versus complete resolution of local (n=2) and pulmonary disease (n=3) in FOLR1-CAR T treated mice (see table). NT T treated mice with local disease demonstrated significant bone destruction; no local tumors were found in FOLR1-CAR T treated mice. HTC (CD45+/CD3+) expansion was detected in PB one wk post-treatment (PT); in mice with metastasis 0.190% and 0.136% of live circulating cells were human T cells in FOLR1-CAR T and NT T treated mice, respectively. Improved overall survival was observed in FOLR1-CAR T treated mice (&gt;100 vs 87.5 days). Conclusions: FOLR1-CAR T cells appear to have in vivo activity against U2OS cell line and support further testing of safety and feasibility in an early phase clinical trial for relapsed/refractory OS. [Table: see text]

Hypereosinophilic syndrome with cutaneous manifestations successfully treated with Dupilumab as a sole agent: A case report.

Hypereosinophilic syndrome describes a process in which eosinophils in the peripheral blood are persistently increased, with variable clinical manifestations. Finding efficacious treatments for this disease can be challenging. This case describes a 72-year-old man with idiopathic hypereosinophilic syndrome with cutaneous manifestations who was successfully treated with dupilumab as a single agent therapy. There was complete clinical and biochemical resolution of disease (eosinophils levels decreased from 4.13 to 0.92) without complications.

The prevalence and changes over time of equine glandular gastric disease in a teaching herd population

SummaryBackgroundThe natural progression of equine glandular gastric disease (EGGD) in the absence of treatment has not yet been described in the literature, nor has the prevalence in a teaching herd population been reported.ObjectivesThe aims of this study were to determine the prevalence of disease in a teaching population over the study period (2019–2021) and to observe the changes over time in disease severity of naturally occurring diseases (not experimentally induced) without medical intervention.MethodsTwenty‐one horses underwent an initial gastroscopy and a repeat gastroscopy between 14 and 731 days later. Gastroscopy data were graded quantitatively and described qualitatively. Prevalence and 95% confidence intervals (CI) were calculated. The changes over time were determined by comparing initial and repeat gastroscopies.ResultsThe prevalence from initial, repeat and total number of gastroscopies was 62% (95% CI: 40.8–79.3), 71% (95% CI: 50.0–86.2) and 67% (95% CI: 51.6–79), respectively. The changes over time included worsening of disease in 29% of horses (95% CI: 13.8–50.0), improvement of disease to a lower grade in 24% (95% CI: 10.6–45.1), no change in grade in 38% (95% CI: 20.8–59.1), and complete resolution of disease to grade 0 in 10% (95% CI: 2.7–28.9).Main limitationsLimitations included a maximum of two gastroscopies per horse given COVID‐19 restrictions on data collection, and highly varied interval times between initial and repeat gastroscopies.ConclusionIn conclusion, there is a high prevalence of disease in this teaching herd. The changes over time in naturally occurring diseases without medical intervention might include worsening, improvement, no change or resolution of disease.

Efficacy of adjunctive measures in peri-implant mucositis. A systematic review and meta-analysis.

To answer the following PICO question: In systemically healthy humans with peri-implant mucositis, what is the efficacy of patient-performed or administered (by prescription) measures used adjunctively to submarginal instrumentation, as compared to submarginal instrumentation alone or combined with a negative control, in terms of reducing bleeding on probing (BOP), in randomized controlled clinical trials (RCTs) with at least 3 months of follow-up? Three databases were searched until April 2022. Weighted mean differences (WMDs) with 95% confidence intervals (CIs) and predictive intervals were calculated. Sixteen parallel RCTs corresponding to 14 studies with low/moderate risk of bias were included. Test groups showed greater reductions in BOP (%) than control groups (nstudies =16; npatients =650; WMD=14.25%; 95% CI [9.06-19.45]; p < .001; I2 = 98.7%). The greatest WMD in BOP reductions (%) were obtained by antiseptics (ns =5; np =229; WMD=22.72%; 95% CI [19.40-26.04]; p < 0.001; I2 = 94.8%), followed by probiotics (ns =6; np =260; WMD=12.11%; 95% CI [3.20-21.03]; p=.008; I2 = 93.3%) and systemic antibiotics (ns =3; np =101; WMD=5.97%; 95% CI [1.34-10.59]; p=.012; I2 = 58.1%). Disease resolution was scarcely reported (n=6). Significant clinical improvements can be obtained when professional submarginal instrumentation is combined with patient-performed or administered (by prescription) adjunctive measures, although a complete disease resolution may not be achieved.

Role of GeneXpert in diagnosis of rifampicin resistant tuberculosis in patients with cervical lymphadenopathy

Tuberculosis is one of the most common causes for cervical lymphadenopathy apart from distant metastasis and lymphoma. Early diagnosis of this disease reduces the patient’s morbidity by initiating early course of Antitubercular treatment with complete resolution of disease. Hence, for diagnosis of this condition we are searching for a modality which can give results in shorter period of time, which is more sensitive, specific and most important easier to perform. Gene Xpert is one such modality which fits into these criteria. In our case series, we are aiming to study the role of Gene Xpert in diagnosis of rifampicin resistant tuberculosis in patients with cervical lymphadenopathy. In our study, excisional biopsy from cervical region was done in 30 cases and were sent for acid fast bacilli culture, Ziehl Neelsen staining and GeneXpert studies, histopathological examination and results were analyzed.

Acute management of pneumonia in adult patients.

Acute management of pneumonia in adult patients.

HPB P43 Stereotactic ablative radiotherapy is a safe and feasible option for non-surgical candidates with colorectal liver metastases

Abstract Background Stereotactic ablative radiotherapy (SABR) has been associated with an improvement in over-all survival in patients with oligometastatic colorectal cancer with a controlled primary tumour and one to five oligometastatic lesions. We report our centre's experience of SABR as an alternative local therapy for non-surgical candidates with colorectal liver metastases. Methods All patients were identified who received SABR irradiation for colorectal liver metastases in a tertiary HPB centre between June 2016 and February 2022. SABR criteria included patients with 1–3 liver metastasis with maximum tumour diameter &amp;lt; 6 cm, liver confined disease and a suitable performance status with pre-treatment Child-Pugh status A. A retrospective review of the patient's scans, letters and MDT outcomes was performed to analyse the pathology, indications and outcomes. Results 18 patients with oligometastatic colorectal cancer received SABR to metastases in the liver over the study period. Five were female and 13 were male. Median age was 71. Performance status was 1 in the majority of cases (10/18). Only 2 patients had synchronous disease. 4 patients had concomitant metastatic disease to the lung and all 4 had had a lung resection. Half of the patients had a previous liver resection with 2 of these having two previous liver resections and 1 having 3 liver resections. 4/18 patients underwent SABR because they were deemed unfit for surgery, 6/18 were for anatomical indications with risk of an R1 resection and one was down to patient choice. Metastases were of median size 30 mm (9–87 mm). 89% (16/18) patients had a single metastasis, 1 patient had 2 metastases and 1 patient had 3 metastases. Overall survival was 19 months (4–60 months). 3 patients achieved complete resolution of disease and 4 patients had partial resolution. 6 patients were deemed to have stable disease and 4 patients had disease progression. Outcome data was not available in 1 patient. Only 2/18 pa Conclusions We observed that SABR is feasible, well tolerated and effective in this cohort of patients. Longer-term follow up data is required to assess a survival benefit.

Cutaneous Scrotal Metastasis Secondary to Primary Prostate Adenocarcinoma Responding to Immunotherapy

This case demonstrates the aggressive progression of prostate adenocarcinoma, failing 3 treatment regimens. Although the patient initially responded to treatments, he developed cutaneous metastasis. Genomic tumor analysis of scrotal tissue confirmed targetable molecular mutations. Treatment with systemic immunotherapy was initiated with complete resolution of disease. This case reveals the rare metastasis of prostate adenocarcinoma to the scrotum with advanced refractory cancer and the successful response to immunotherapy. It illustrates the importance of tumor genomic testing in advanced refractory prostate cancer.

Resolution of Disseminated Angiosarcoma in a Kidney Transplant Recipient After Treatment With Sirolimus: A Case Report

Angiosarcoma is a rare, almost universally fatal malignant neoplasm in kidney transplant recipients. No evidence-based guidelines are available for disseminated disease. Here, we report a case of a 66-year-old woman who developed disseminated angiosarcoma 4 months after living nonrelated kidney transplant. She underwent only 2 rounds of chemotherapy because of intolerable adverse effects. Her mycophenolic acid and tacrolimus were withdrawn and sirolimus use was started. In addition to its immunosuppressant effects, sirolimus has been shown to have antineoplastic properties. Remarkably, at almost 2 years post-transplant, the patient has had complete resolution of all gross metastatic disease with only immunosuppressant medication changes. This case highlights the interesting possibility that sirolimus is an effective adjunct treatment for disseminated angiosarcoma in kidney transplant recipients.

Success of Trametinib in the Treatment of Langerhans Cell Histiocytosis With Novel MAPK Pathway Mutations.

Approximately a third of patients with Langerhans cell histiocytosis (LCH) experience recurrence of disease. Genomic analysis has revealed that this condition is often driven by oncogenic mutations in the MAP kinase (MAPK) pathway, and agents that target components of this pathway have been explored as a second-line treatment for LCH. Here, we examine 2 pediatric patients with LCH and confirmed but rarely reported MAPK pathway mutations treated with trametinib, a MAP2K inhibitor approved to treat several cancers with a BRAFV600E mutation. Each patient achieved or maintained complete resolution of disease and remain on the drug with no adverse effects.

Predicting Outcomes after Mandibular Distraction Osteogenesis in Patients with Isolated Pierre-Robin Sequence

INTRODUCTION: Pierre-Robin Sequence (PRS) is characterized by micrognathia, glossoptosis, and obstructive sleep apnea (OSA). Mandibular distraction osteogenesis (MDO) is a common treatment modality to improve OSA. We aimed to evaluate the sleep study outcomes based on mandibular distraction distance on patients born with OSA secondary to micrognathia. METHODS: A retrospective chart review was conducted for patients with isolated PRS who underwent MDO at Children’s Hospital Los Angeles between January 2006 and September 2021. Relationships between demographics, preoperative sleep variables, and postoperative OSA outcomes were analyzed. Results were stratified according to OSA severity and distraction distance (30 mm vs less than 30 mm). RESULTS: A total of 71 patients met inclusion criteria. Fifty-six patients were distracted to 30 mm, whereas the remaining 15 patients experienced shorter distraction; reasons for shorter distraction included infection, parent preference, and loss to follow-up. Compared with patients distracted to less than 30 mm, those distracted to 30 mm had a significantly greater improvement in AHI when controlling for preoperative sleep study variables (p = 0.047). After MDO, significant improvement was observed in lowest oxygen saturation, AHI, highest carbon dioxide level, and highest oxygen requirement. CONCLUSION: Patients with PRS who have more severe OSA experienced greater improvements in sleep study scores after MDO with more than 89% of patients with severe OSA achieving mild or complete resolution of disease. Our results provide tangible evidence for how distracting patients to 30 mm vs less than 30 mm can improve disease parameters, which can assist clinicians and families in anticipating outcomes after MDO for patients with isolated PRS.