The transition from children to young people living with home mechanical ventilation.

PURPOSE: This study aimed to examine how young people living with Home Mechanical Ventilation experience the transition from childhood to young adulthood in relation to everyday life, perceived health and transition into adult professional healthcare. METHODS: Nine young adults (three females and six males aged 18-31) were interviewed, and data was primary analysed using phenomenological hermeneutics. In the actual study, data was reworked using secondary analysis as described by Beck. Two interviewees were ventilated invasively and six non-invasively, and one was treated with continuous positive airway pressure (CPAP). RESULTS: The results are presented in two main categories. First; moving towards adulthood; and second, To handle changes in health and healthcare contacts. The study highlights the importance of ongoing social relations and being part of a socializing and physically active community. The transfer from paediatric to adult healthcare was solid and worked out well but was a process in which the participants struggled to find their own voice. CONCLUSIONS: The transition into adulthood is a sensitive and challenging time for young people with HMV, but stable, close relationships and a well-organized transfer can enable this group to feel safe and able to find and use their own voice.

Therapeutic targeting of RNA for neurological and neuromuscular disease.

Neurological and neuromuscular diseases resulting from familial, sporadic, or de novo mutations have devasting personal, familial, and societal impacts. As the initial product of DNA transcription, RNA transcripts and their associated ribonucleoprotein complexes provide attractive targets for modulation by increasing wild-type or blocking mutant allele expression, thus relieving downstream pathological consequences. Therefore, it is unsurprising that many existing and under-development therapeutics have focused on targeting disease-associated RNA transcripts as a frontline drug strategy for these genetic disorders. This review focuses on the current range of RNA targeting modalities using examples of both dominant and recessive neurological and neuromuscular diseases.

Quantifying Muscle Regeneration: Activated Muscle Satellite Cells and New Regenerated Myofibers in Chronic and Acute Degeneration Models.

Regeneration is a remarkable characteristic of the skeletal muscle. Triggered by common lesions, regeneration is stimulated resulting in muscle fiber repair and restoration of muscle homeostasis in normal muscle. In genetic dystrophic muscle, the cycle of degeneration/regeneration is an endless loop that leads to impaired regeneration and substitution of muscle fibers by connective and adipose tissue, causing muscle weakness. Identification and characterization of muscle regeneration steps can help discover potential therapy targets for muscle diseases and aging. Muscle regeneration markers such as the number of satellite cells in the muscle, the proportion of activated satellite cells, and the quantity of regenerating muscle fiber can be quantified using immunolabeling.Here we are presenting a quantitative method to measure muscle regeneration that can be applied to different proposals. To demonstrate the protocol applicability, we used models for acute and chronic muscle injuries. As model of acute degeneration, a wild-type C57BL6 mice with muscle injury induced by electroporation was used, and the muscle was analyzed after 5 and 10 days post-injury. DMDmdx mouse muscle was used as a model of chronic degeneration. The methodologies presented here are among the gold standard methodologies for muscle regeneration analysis and can be easily applied to any type of muscle regeneration study.

Ultrasound evaluation and grading of neuromuscular disease in lower extremities among diabetic patients.

OBJECTIVE: To explore the clinical utility of ultrasound in evaluating and grading neuromuscular diseases in the lower extremities of patients with diabetes mellitus. METHODS: A total of 126 inpatients from the Department of Diabetes at Zhangzhou Affiliated Hospital of Fujian Medical University, China, were recruited from June 2020 to December 2022. The cohort included 69 patients with type 2 diabetes mellitus (T2DM) and diabetic peripheral neuropathy (DPN group) and 57 patients with T2DM but without DPN (non-DPN group). Additionally, 80 healthy controls were included. High-frequency ultrasound was used to scan the common peroneal, sural, and tibial nerves, measuring their transverse (D1) and anteroposterior (D2) diameters, and calculating the cross-sectional area (CSA). Changes in the internal echo of the extensor digitorum brevis (EDB) muscle, including maximum thickness and CSA, were also recorded. The DPN group was further subdivided based on disease duration to assess ultrasonic changes over time and the statistical significance of these variations. RESULTS: Ultrasonic changes such as uneven internal echo reduction, ill-defined epineurial boundaries, and obscured cribriform structures were most prevalent in the DPN group. Significant differences in ultrasound parameters (D1, D2, CSA) were observed among the groups (all P<0.05), with the most pronounced changes in the DPN group. In patients with a disease duration of over 15 years, a significant increase in CSA of lower extremity nerves and a decrease in CSA of the EDB were noted compared to those in the 5-10 years subgroup (19.89±0.98 vs 19.00±0.94; 5.25±0.74 vs 5.93±0.94; all P<0.05). CONCLUSIONS: High-frequency ultrasound provides a valuable imaging basis for diagnosing and monitoring DPN, demonstrating significant changes in nerve and muscle parameters among diabetic patients.

Factors associated with noninvasive ventilation usage in patients with hypoventilation disorders.

Study Objectives: The objective of this study was to investigate the association between demographic, clinical, and interface factors and noninvasive ventilation (NIV) usage. Methods: A retrospective cohort analysis of 478 patients prescribed NIV from 2013 to 2021 was performed. Demographic factors, clinical indications for NIV, and interface factors were collected, and linear regression was conducted to evaluate the association between these variables and NIV usage (hour/night). Results: The average usage of the cohort was 6.5 hour/night ±â€…4.6, with an average age of 57 years ±â€…16 and body mass index (BMI) of 40.5kg/m2 ±â€…14.7. The cohort was mostly male (n = 290, 60.6%). The most common indications for NIV prescription were high-pressure requirement for obstructive sleep apnea (HPR, n = 190, 39.7%), neuromuscular disease (NMD, n = 140, 29.3%), and obesity hypoventilation syndrome (OHS, n = 111, 23.2%). A diagnosis of NMD was a significant predictor of higher NIV usage (8.0 ±â€…6.1 hour/night) in multivariate analysis (p = .036). The HPR subcohort had the lowest usage of all indications. Age and BMI did not predict usage. A nasal interface (p < .01) and lower expiratory positive airway pressure (EPAP) setting (p < .001) were associated with increased NIV usage. Conclusions: This study highlights the multifaceted nature of NIV usage. Where demographic factors were not consistent predictors of usage, interface, and clinical indication were associated with usage. These findings highlight that the HPR users are a group at risk of low usage.

Assessment of Chronic Hypercapnic Respiratory Failure.

Undiagnosed chronic hypercapnic respiratory failure may be encountered during the evaluation of sleep-related breathing disorders at the sleep clinic. This article reviews the mechanism of chronic hypercapnic respiratory failure and the systematic approach to the assessment of specific sleep disorders associated with nocturnal hypoventilation encountered in clinical practice.

Initiation of Chronic Non-invasive Ventilation.

Initiation of home non-invasive ventilation (NIV) requires careful consideration of the patient's condition, motivation, expectations, wishes, and social circumstances. The decision to start NIV depends on a combination of factors including patient symptoms and objective evidence of nocturnal hypoventilation. A solid understanding of the underlying pathophysiology is key to a systematic and well-balanced clinical approach to titrating NIV. The location where NIV is initiated is not the most relevant issue, provided that it is a comfortable, safe environment in which adequate monitoring can be assured. The majority of patients prefer their own home for treatment initiation.

Telemonitoring in Non-invasive Ventilation.

Telemonitoring in non-invasive ventilation is constantly evolving to enable follow-up of adults and children. Depending on the device and manufacturer, different ventilator variables are displayed on web-based platforms. However, high-granularity measurement is not always available remotely, which precludes breath-by-breath waveforms and precise monitoring of nocturnal gas exchange. Therefore, telemonitoring is mainly useful for monitoring utilization of the device, leaks, and respiratory events. Coordinated relationships between patients, homecare providers, and hospital teams are necessary to transform available data into diagnosis and actions. Telemonitoring is time and cost-consuming. The balance between cost, workload, and clinical benefit should be further evaluated.

Palliative Care and Noninvasive Ventilation.

Palliative care is important for many patients who require noninvasive ventilation. The particular needs of patients with neuromuscular disease and chronic obstructive pulmonary disease are explored. Advance care planning is explored with tips for undertaking this important communication task. Brief comments regarding symptom burden, weaning, voluntary assisted dying, and self-care are included.

Impact of Disease-modifying Therapies on Respiratory Function in People with Neuromuscular Disorders.

Spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) are neuromuscular disorders that affect muscular function. The most common causes of morbidity and mortality are respiratory complications, including restrictive lung disease, ineffective cough, and sleep-disordered breathing. The paradigm of care is changing as new disease-modifying therapies are altering disease trajectory, outcomes, expectations, as well as patient and caregiver experiences. This article provides an overview on therapeutic advances for SMA and DMD in the last 10 years, with a focus on the effects of disease-modifying therapies on respiratory function.

Ultrasonographic Assessment of Diaphragm Function to Predict Need for Mechanical Ventilation and its Liberation in Patients with Neuromuscular Disorders: An Observational Cohort Pilot Study.

BACKGROUND: Management of assisted ventilation and determining the optimal timing for discontinuation presents a significant clinical obstacle in patients affected by neuromuscular (NM) diseases. This study aimed to evaluate the efficacy of ultrasound in appraising diaphragmatic function for predicting the necessity of intubation and determining the opportune moment to discontinue mechanical ventilation (MV) in patients with NM disorders. METHODS: The study was conducted in adult patients with NM diseases requiring inpatient care in the high-dependency neurology ward and the intensive care unit. Ultrasonographic assessment of diaphragmatic excursion (DE) and diaphragmatic thickness fraction (DTF) was conducted at the patient's bedside every 48 h for ventilated patients and every 72 h for nonventilated patients until they were weaned from the ventilator or discharged home. Qualitative data are expressed as percentages or numbers, and quantitative data are represented as mean ± standard deviation. Unpaired t-tests were employed to compare continuous variables, and χ2 tests were used for categorical variables. Contingency table analysis was used to compute relative risks in comparing the baseline DE and DTF with the sequential changes in these values. RESULTS: In cases in which the baseline left DE measured less than 1 cm, the relative risk for the requirement of ventilation was 2.5 times higher, with a confidence interval of 0.62-0.99 (P = 0.19). Notably, a bilateral reduction in DE within the initial 48 h of admission was identified as predictive of need for intubation. When comparing ventilated and nonventilated patients, it was observed that the mean DE values for the left and right sides in ventilated patients (0.74 and 0.79) were significantly lower than those in nonventilated patients (1.3 and 1.66), with corresponding P values of 0.05 and 0.01, respectively. Furthermore, a decline in right DE by more than 50% within 72 h of admission presented a relative risk of 3.3 for the necessity of ventilation, with a confidence interval of 1.29-8.59 (P = 0.01). Duration of ventilation ranged from 2 to 45 days, with an average of 13.14 days, whereas the mean ventilator-free days recorded was 13.57. Notably, a sequential increase in bilateral DE correlated with an extended duration of ventilator-free days. CONCLUSIONS: The presence of a baseline left DE of less than 1 cm, a consecutive decrease in DE measurements within 48 h, and a comparative reduction in right DE of more than 50% within the initial 3 days are indicators associated with the requirement for MV in patients with NM disease. Furthermore, the upward trajectory of DE in mechanically ventilated patients is linked to an increased number of days free from ventilator support, suggesting its potential to forecast earlier weaning.

Clinically relevant mouse models of severe spinal muscular atrophy with respiratory distress type 1.

Spinal Muscular Atrophy with Respiratory Distress (SMARD1) is a lethal infantile disease, characterized by the loss of motor neurons leading to muscular atrophy, diaphragmatic paralysis, and weakness in the trunk and limbs. Mutations in IGHMBP2, a ubiquitously expressed DNA/RNA helicase, have been shown to cause a wide spectrum of motor neuron disease. Though mutations in IGHMBP2 are mostly associated with SMARD1, milder alleles cause the axonal neuropathy, Charcot-Marie-Tooth disease type 2S (CMT2S), and some null alleles are potentially a risk factor for sudden infant death syndrome (SIDS). Variant heterogeneity studied using an allelic series can be informative in order to create a broad spectrum of models that better exhibit the human variation. We previously identified the nmd2J mouse model of SMARD1, as well as two milder CMT2S mouse models. Here, we used CRISPR-Cas9 genome editing to create three new, more severe Ighmbp2 mouse models of SMARD1, including a null allele, a deletion of C495 (C495del) and a deletion of L362 (L362del). Phenotypic characterization of the IGHMBP2L362del homozygous mutants and IGHMBP2C495del homozygous mutants respectively show a more severe disease presentation than the previous nmd2J model. The IGHMBP2L362del mutants lack a clear denervation in the diaphragm while the IGHMBP2C495del mutants display a neurogenic diaphragmatic phenotype as observed in SMARD1 patients. Characterization of the Ighmbp2-null model indicated neo-natal lethality (median lifespan = 0.5 days). These novel strains expand the spectrum of SMARD1 models to better reflect the clinical continuum observed in the human patients with various IGHMBP2 recessive mutations.

Living with adult-onset myotonic dystrophy type 1: a scoping review.

PURPOSE: To identify the existing literature on experiences of living with adult-onset myotonic dystrophy type 1 (DM1) from people with adult-onset DM1, their caregivers and health care professionals. MATERIALS AND METHODS: Following the framework of Arksey and O'Malley, a literature search was performed in five databases in October-November 2022. An updated search was conducted in December 2023. Studies were eligible if they reported on experiences related to living with DM1 from people with adult-onset DM1, caregivers or healthcare professionals. Qualitative, quantitative, and mixed method studies were included. Key findings were categorized using the International Classification of Functioning, Disability and Health (ICF) components. RESULTS: 11 out of 1842 studies were included, of which five had a quantitative design, five had a qualitative design and one study had a mixed methods design. The studies reported on multiple experiences of living with adult-onset DM1 from the perspectives of people with the disease and their caregivers. All components of the ICF were reported in the studies; activity and participation and personal factors were the most reported. CONCLUSION: Adult-onset DM1 is a complex disease with great biopsychosocial impact making it challenging to live with for those diagnosed with DM1 as well as their caregivers.

Issues with hands or arms, myotonia, fatigue, impaired sleep or daytime sleepiness, and symptoms of depression in everyday life should be addressed in the follow-up of people with adult-onset myotonic dystrophy type 1 (DM1) to facilitate increased participation in daily life.Challenges related to activity and participation should be addressed in the follow-up of people with adult-onset DM1 to help facilitate increased activity and participation in everyday life.Interventions targeting caregiver needs are necessary to help them cope with living with a person with adult-onset DM1 and to minimize the negative impact DM1 has on their lives.

Oximetry and carbon dioxide screening for ventilatory requirements in children with spinal muscular atrophy type 1-3.

INTRODUCTION: Despite disease modifying treatments (DMT), assisted ventilation is commonly required in children with Spinal Muscular Atrophy (SMA). Guidelines suggest screening with oximetry and transcutaneous carbon dioxide (TcCO2) for sleep disordered breathing (SDB). AIM: To determine the utility of pulse oximetry and TcCO2 as a screen for SDB and the need for Non-Invasive Ventilation (NIV) in children with SMA type 1-3. METHODS: A prospective cohort study was conducted in Queensland, Australia. Full diagnostic PSG was completed in DMT naïve children with SMA. Pulse oximetry and TcCO2 were extracted from PSG. Apnoea-hypopnoea indices (AHI) criteria were applied to PSG results to define the need for NIV. Abnormal was defined as: ≤3 months of age [mo] AHI≥10 events/hour; >3mo AHI ≥5 events/hour. Receiver operating characteristic curves were calculated for abnormal PSG and pulse oximetry/TcCO2 variables, and diagnostic statistics were calculated. RESULTS: Forty-seven untreated children with SMA were recruited (type 1 n = 13; 2 n = 21; 3 n = 13) ranging from 0.2 to 18.8 years old (median 4.9 years). Oxygen desaturation index ≥4 % (ODI4) ≥20events/hour had sensitivity 82.6 % (95 % CI 61.2-95.0) and specificity of 58.3 % (95 % CI 36.6-77.9). TcCO2 alone and combinations of oximetry/TcCO2 had low diagnostic ability. The same methodology was applied to 36 children who were treated (type 1 n = 7; type 2 n = 17; type n = 12) and oximetry±TcCO2 had low diagnostic ability. CONCLUSION: ODI4 ≥20events/hour can predict the need for NIV in untreated children with SMA. TcCO2 monitoring does not improve the PPV. If normal however, children may still require a diagnostic PSG. Neither oximetry nor TcCO2 monitoring were useful screening tests in the children treated with DMT.

What is normal age-related thigh muscle composition among 45- to 84-year-old adults from the UK Biobank study.

A loss of skeletal muscle mass and an increase in intramuscular fat are known to occur as we enter middle and older age, but the expected changes or normative values have remained unknown. The primary reason for this is that imaging studies are difficult and expensive to conduct, and consequently, the sample sizes have remained small. The development of the UK Biobank which provides access to a large magnetic resonance imaging (MRI) data set of more than 50,000 participants provides an opportunity to finally address this question of normative values for each age group. The study's primary aim was to determine the age-related changes in thigh muscle composition (e.g., thigh fat-free muscle volume and intramuscular fat) between the ages of 45 and 84 years. The second aim was to analyse associations between thigh fat-free muscle volume and intramuscular fat with lifestyle behaviours (smoking, alcohol consumption, and physical activity), leg pain, and bone mineral density. Fifty thousand three hundred thirty-two participants were included in the study. Total fat-free thigh muscle declined between the ages of 45 and 84 years, while intramuscular fat of the thigh continued to increase. The changes were stable between these age groups. The mean volume of fat-free muscle ranged from 11.16 (SD: 1.40) to 13.26 L (SD: 1.85) in adult males and 7.60 (SD: 0.97) to 8.80 L (SD 1.29) in females between the ages of 45 and 84 years. For intramuscular fat, the change among women was from 6.94% (SD: 1.59) in the 45 to 54 years age bracket to 8.83% (SD: 1.92) in the 75 to 84 age bracket, while for men, it was 5.83% (SD: 1.30) in the 45 to 54 age bracket to 7.85% (SD 1.89) in the 75 to 84 age bracket. The total fat-free muscle volume and intramuscular fat percentage provided can be used for the purpose of reference standards or normative values for adults in the age groups provided. Fat-free muscle and intramuscular fat were found to be associated with a range of health, activity, and leg pain outcomes, and these should be investigated in a follow-up longitudinal imaging study.

Perspectives of Young People with Neuromuscular Diseases Regarding Their Choice of Educational Programs and Possibilities to Complete Program Requirements.

Young people with impaired functioning and/or disability do not receive as much education or as high an education as fully functioning young people, thus limiting their job opportunities. Therefore, the aim of this study was to investigate perspectives of young people with neuromuscular diseases regarding their choice of educational programs and possibilities to complete program requirements to gain knowledge for use in future counselling and the development of a national questionnaire survey. The design for this study was qualitative using the interpretive description methodology and Anthony Giddens' theory on modernity and self-identity. Data were generated through two focus group interviews with seven people between 18 and 30 years of age. Beginning and completing an education was influenced by the creation of identity: the importance of experiencing demands and expectations, the meaning of social relations when learning, and the consequences of accessibility for educational opportunities. The participants' sense of self-identity was built by testing boundaries and developing images through social relations with peers, parents, and teachers. How they acted and behaved in the social arena of school and education influenced their choices and chances of completing educational programs.

Neuromuscular disease auxiliary diagnosis using a portable magnetomyographic system.

The measurement of electromyography (EMG) signals with needle electrodes is widely used in clinical settings for diagnosing neuromuscular diseases. Patients experience pain during needle EMG testing. It is significant to develop alternative diagnostic modalities. This paper proposes a portable magnetomyography (MMG) measurement system for neuromuscular disease auxiliary diagnosis. Firstly, the design and operating principle of the system are introduced. The feasibility of using the system for auxiliary diagnosis of neuromuscular diseases is then studied. The magnetic signals and needle EMG signals of thirty subjects were collected and compared. It is found that the amplitude of muscle magnetic field signal increases during mild muscle contraction, and the signal magnitudes of the patients are smaller than those of normal subjects. The diseased muscles tested in the experiment can be distinguished from the normal muscles based on the signal amplitude, using a threshold value of 6 pT. The MMG diagnosis results align well with the needle EMG diagnosis. In addition, the MMG measurement indicates that there is a persistence of spontaneous activity in the diseased muscle. The experimental results demonstrate that it is feasible to auxiliary diagnose neuromuscular diseases using the portable MMG system, which offers the advantages of non-contact and painless measurements. After more in-depth, systematic, and quantitative research, the portable MMG could potentially be used for auxiliary diagnosis of neuromuscular diseases.

Pelvic Fixation for Non-Ambulatory Patients with Neuromuscular Scoliosis Treated with Magnetically Controlled Growing Rods-A 4-Year Comparison of Two Different Fixation Methods.

Background/Objectives: This study aims to analyze the efficacy and safety of the two pelvic fixation systems, S-hooks (SH) and lumbar-sacral-alar-iliac (SAI) screws, when used in association with magnetically controlled growing rods (MCGRs) in non-ambulatory children with severe neuromuscular scoliosis (NMS). Methods: Forty-one patients were retrospectively examined and subdivided corresponding to ilium hook fixation or SAI screws. The major curve correction (%) and pelvic obliquity (PO) correction (%) were assessed utilizing scoliosis plain film radiographs over time. Complications and unplanned return to the operating room (UPROR) were recorded. Patient-specific pre- and postoperative values were included in a backward stepwise regression model to assess UPROR. Results: Mean age at index intervention was 9.4 years. Preoperative main curve was 81° and PO was 22°. 21 and 20 patients were categorized into the SH and SAI subgroups, respectively. Initial curve correction was significantly better in the SAI subgroup (63 vs. 42% in the SH, p = 0.045), while PO correction was equally good. Curve and PO correction were maintained throughout the follow-up period of 55 months. UPROR rate was 38% in the SH subgroup, and 5% in the SAI subgroup (p = 0.010). Regression analysis identified postoperative curve correction as predictive value for UPROR (p = 0.006). Conclusions: SAI screw fixation has a low UPROR rate and achieves superior curve correction. S-hooks are a viable option to correct PO and NMS in children with high operative risk to reduce operative time, but revision surgery is not uncommon.

Possible sex and racial disparities in myasthenia gravis care.

INTRODUCTION/AIMS: Given the importance of early diagnosis and treatment of myasthenia gravis (MG), it is critical to understand disparities in MG care. We aimed to determine if there are any differences in testing, treatment, and/or access to neurologists for patients of varying sex and race/ethnicity with MG. METHODS: We used a nationally representative healthcare claims database of privately insured individuals (2001-2018) to identify incident cases of MG using a validated definition. Diagnostic testing, steroid-sparing agents, intravenous immunoglobulin (IVIG), plasma exchange (PLEX), and thymectomy were defined using drug names or CPT codes. Steroid use was defined using AHFS class codes. We also determined whether an individual had a visit to a neurologist and the time between primary care and neurologist visits. Logistic regression determined associations between sex and race/ethnicity and testing, treatments, and access to neurologists. RESULTS: Female patients were less likely to get a computed tomography (CT) chest (odds ratio (OR) 0.73, 95% confidence interval (CI): 0.64-0.83), receive steroids (OR: 0.85, 95% CI: 0.75-0.97), steroid-sparing agents (OR: 0.84, 95% CI: 0.72-0.97), and IVIG or PLEX (OR: 0.80, 95% CI: 0.67-0.95). Black patients were less likely to receive steroids (OR: 0.78, 95% CI: 0.63-0.96). No significant disparities were seen in access to neurologists. DISCUSSION: We found healthcare disparities in MG treatment with female and Black patients receiving less treatment than men and those of other races/ethnicities. Further research and detailed assessments accounting for individual patient factors are needed to confirm these apparent disparities.

Permanent cutaneous vesicostomy: a pragmatic approach to safely manage lower urinary tract dysfunction in pediatric patients with chronic and life-limiting conditions and neuropathic bladders.

Introduction: Lower urinary tract dysfunction (LUTD) in cerebral palsy (CP) and other neuromuscular diseases can present with chronic retention that leads to hydronephrosis, recurrent urinary tract infections (UTI), and stone formation. Whenever the conservative treatment of LUTD fails for any reason, it is considered to be complicated LUTD, in which a surgical approach is warranted. Cutaneous vesicostomy (CV) is a simple, well-tolerated, and potentially reversible procedure that protects the upper tracts. We describe our experience using CV for this complex population. Materials and methods: Children with CP and other neuromuscular diseases admitted to pediatric long-term care units for palliative care between 2015 and 2019 were included in the study. They present multi-system involvement, polypharmacy, and Gross Motor Function Classification System levels of 4 or 5. We retrospectively studied this population's indications and results of CV. Results: Of the 52 admitted patients, 18 presented LUTD with UTI (n:18; 100%), stones (n:5; 28%), progressive hydroureteronephrosis (n:3; 17%), or stones (n:2; 11%). Conservative initial management (catheterizations, prophylaxis antibiotics) was effective in half the cases. The remaining nine were defined as complicated LUTD and underwent CV. After a mean follow-up of 11.3 months, the follow-up showed improved hydronephrosis in all nine (100%) patients. Recurrent UTIs were no longer seen in eight of nine patients, although three patients required bladder irrigations; bladder stones did not recur after CV; the kidney stones needed further intervention. Revision of the CV was required in two (11%) cases at 12 and 24 months postoperatively due to stoma stenosis. Conclusion: CV is a relatively simple and effective procedure representing a pragmatic solution for managing complicated LUTD in complex long-term institutionalized pediatric palliative care patients with neuropathic bladders.