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Research Spotlight: Pediatrics
Research Spotlight: Pediatrics
Research Spotlight: Pediatrics
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Good afternoon, and thank you for joining us. My name is Heather Vincent. I'm a faculty member at the University of Florida Department of Orthopedics and Rehabilitation and the Director of Research for Physical Medicine and Rehabilitation. I'm really excited to be here with you today, sharing with you the best pediatric related research. The submissions this year were reviewed by the Evidence Committee and of the abstract submitted for pediatrics research, what you are going to see today represents the top five of those abstracts. After rigorous review, we would like to present to you a group of speakers today. The format that we will use is to allow the speakers to be introduced, allow their videos and their presentations to roll in sequence, and then at the end, we will rejoin for questions and answers. Should you have questions, please go ahead and put it in the Q&A bar on the side of your screen, and I'll be monitoring that and we'll try and get to all the questions as they come in at the end. We appreciate your time. Let me go ahead and introduce our speakers for today. Dr. Hannah Oh, from New York Presbyterian Hospital PM&R program will be the first speaker on the docket, followed by Dr. Nijer Dursun from Koseli University in Turkey. Our third speaker will be Dr. Charles Kenyon from the University of Washington PM&R program. Our fourth speaker on the docket will be Dr. Joshua Leiter from the University of Utah. And finally, the future Dr. Caroline Gormley will also join us from West Virginia School of Osteopathic Medicine. We are really delighted to have you all here and we can't wait to see your presentations. Without further ado, let's commence with the videos. Great, thank you for your time and opportunity to present this research, which would not have been possible without the mentorship from Dr. Hee-kyung Kim, Dr. James Huber and I started this project as residents and hope some of these initial results can propagate further research in the use of imantadine in the pediatric brain injury population. This descriptive review investigates the use of imantadine in the pediatric acquired brain injury population, charts of previously admitted patients to a brain injury unit at a pediatric inpatient rehab hospital were reviewed. Prior to reviewing our study design and results, I want to provide a brief background regarding the use of imantadine in the brain injury patient population. Imantadine, a dopamine agonist, is one of the most commonly prescribed medications for prolonged disorders of consciousness after a traumatic brain injury. While it was initially used as an antiviral drug, it is now more commonly used for its dopaminergic properties. It operates both presynaptically and postsynaptically to facilitate dopamine release and delay dopamine reuptake. It has been shown to improve arousal levels after traumatic brain injury, regardless of type, severity, duration, or patient's age in the adult population. While there's a large body of evidence supporting the use of this medication in the treatment of brain injury in adults, there is limited research on its use in the pediatric population. By better understanding how imantadine may affect the neurologic recovery in pediatric patients with different etiologies of brain injury, hopefully we can better clarify specific indications of imantadine and better understand prognostic outcomes. Again, our objective was to retrospectively analyze the use of imantadine in the pediatric acquired brain injury population during acute rehab care. From these results, we hope there would be potential to construct a future prospective trial to study and determine optimal dosing, initiation, and duration of imantadine post brain injury. Within our time frame, there were a total of 105 pediatric patients admitted with an acquired brain injury to this hospital, 51 of them were treated with imantadine, which we referred to in our study as the AG group. 54 patients were not treated with imantadine, which we referred to as the no AG group. The mean age of AG was 12.5 years, ranging from 2-year-olds to 19-year-olds, with a gender ratio of 17 to 16, male to female. The mean age of the no AG group was 9.6, ranging from 2-year-olds to 17-year-olds, again with their gender ratio being 19 to 11. Data collection included sorting these patients by the etiology of their acquired brain injury, their admission and discharge WIFM score, and medical complexity. We use the WIFM, the Functional Independence Measure for Children, to assess patients' functional improvement and change during the course of their inpatient rehabilitation stay. For a brief overview, the WIFM is an 18-item, 7-level ordinal scale instrument that measures a child's performance in essential daily functional tasks. Three domains, including self-care, mobility, and cognition, are assessed by interviewing or observing a child's ability to perform that task. 33 patients in the AG group had complete WIFM data, and 30 patients in the no AG group had complete WIFM data. Examples that we considered in determining medical complexity among these patients included the presence of a tracheostomy, feeding tube, and certain medications, which included benzodiazepines, opiates, antihypertensives, and antiepileptics. In the AG group, the duration of mantidine was compared among the different admitting diagnoses, aka the different etiologies of acquired brain injury. Mantidine was continued on discharge in three cases, was unknown in five cases, and discontinued prior to discharge in 25 of these cases. In terms of how these two groups differ, the distribution of etiology of the required brain injury vary quite a bit. Looking at figure 1a and 1b, patients more likely to have received a mantidine had a closed traumatic injury, a hemorrhagic stroke, an oxic brain injury, or tumor. Included in this group was also patients with a metabolic encephalopathy. The most common types of acquired brain injury in the non-AG group were ischemic stroke and closed traumatic brain injury. The duration of a mantidine listed in table 2 varied considerably in each admitting diagnosis as well, ranging anywhere from 11 to 1,325 days. Now looking at table 1, which shows the variables we collected to look at medical complexity, the AG group was much more likely to have a tracheostomy, feeding tube, and beyond medications such as benzodiazepines, antihypertensives, and antiepileptics. While the presence of a feeding tube and tracheostomy often go hand in hand, it also infers that these patients had a more severe disorder of consciousness. Figure 2A compares the admission and discharge WEFM scores between the two groups. The average admission WEFM of the 33 patients in the AG group was 23.9, and discharge WEFM score was 46.1, with the change being 22.2. The average admission WEFM of the 30 patients in the no-AG group was 53.9, and discharge score 87, a change 33.1. In the AG group, the change in admission and discharge WEFM scores was most variable in patients with a closed traumatic injury, anywhere ranging from 0 to 92 points. The minimal change in admission and discharge WEFM scores occurred in the anoxic brain injury group, ranging only from 0 to 8 points. While we initially hoped to compare WEFM scores in patients with similar etiologies of brain injury, we realized that the overall tendency towards starting amantadine was occurring in patients with more medically complex hospital courses. Subjects started on amantadine tended to have lower WEFM admission scores, correlating with a more severe disorder of consciousness. In realizing this, we hope to further study and analyze the clinical significance in neurologic recovery among pediatric patients with severe disorders of consciousness treated with amantadine. Thank you for your time in allowing me to present this research. Hello, my name is Nigar Dursun. I would like to thank the Academy for giving me this opportunity of presenting this large phase 3 bivodal study. This study was conducted internationally in 32 sites. This study was a double-blind, randomized, controlled study. And the results of this bivodal study formed the basis of regulatory approval for apobutilinum toxin A in pediatric upper-limb spasticity in several countries. I will take you through this study, but firstly, I would like to mention the main takeaways of the study. The results of the study showed that apobutilinum toxin A injections significantly reduced upper-limb spasticity in children with cerebral palsy. Children treated with apobutilinum toxin A, together with a specific targeted home exercise therapy program, showed global improvement and goal attainment in this study. Repeat benefits were sustained over one year with repeat treatment cycles, and repeat injections into upper-limb were well-tolerated over one year. There is general agreement that, when used in conjunction with occupational therapy, the aim of apobutilinum toxin A treatment in pediatric upper-limb spasticity is to selectively reduce muscle spasticity and optimize the efficacy of other interventions used for improving range of motion, enhancing motor ability, and enhancing functional skills. For this reason, botulinum toxin has been in clinical use for many years. However, until recently, clinical trial evidence for botulinum toxin A has been not sufficient to gain regulatory approval in most countries. The primary objective of this phase 3 bi-modal study was to evaluate the efficacy and safety of apobutilinum toxin A in reducing upper-limb spasticity in children with cerebral palsy. In this clinical trial, children also participated in a targeted home exercise therapy program, and the secondary objective was to evaluate the efficacy of these treatments on global function by physician global assessment scale and on goal attainment by goal attainment scale with repeat treatment cycles over one year. Here in this slide, we see the study design of pediatric upper-limb study. In this study, 212 children with cerebral palsy presenting upper-limb spasticity were included. One of the main inclusion criteria of the study was to have a modified Ashford scale score of at least 2 in the primary target muscle group, which could either be elbow flexors or wrist flexors. Eligible children were randomly assigned in a 1-1-1 ratio to 16 units per kilo or 8 units per kilo or 2 units per kilo apobutilinum toxin A dosage groups. In this study, we assumed 2 units per kilo to be subtherapeutic dose, and this group was used as controls in this study. At cycle 2, these children who received 2 units per kilo at treatment cycle 1 were reassigned to receive either 16 units per kilo or 8 units per kilo apobutilinum toxin A. As and after treatment cycle 1, investigators were allowed to ask for a dose decrease, and after cycle 3, investigators were also allowed to ask for a dose increase. At and after treatment cycle 2, investigators could change the primary target muscle group, also were allowed to inject non-study upper-limb muscles or lower-limb muscles with respect to the needs, requirements, and clinical presentation of the particular child. And in this case, if both upper limbs and lower limbs were injected, total dose did not exceed 30 units per kilo or 1,000 units of apobutilinum toxin A, whichever was the lowest. Throughout the study, we used a fixed volume of 1.6 ml to treat study upper limb in all treatment arms, and this 1.6 ml was distributed to the primary target muscles and other selected muscles by the investigators, again with respect to the clinical presentation of the child. This was maintained throughout the study, and throughout the study, when doing injections, it was mandatory to use electrical stimulation or ultrasound guidance. And here we see the main, here we see the main results of the study. At the top left-hand corner, we see the primary efficacy endpoint of the study, which was the mean change from baseline to week 6, modified Ashford scale score of the primary target muscle group. As you can see, both 8 units per kilo and 16 units per kilo treated groups showed statistical superiority over 2 units per kilo treated control group, and this statistical superiority was maintained at week 16. There is a clear dose-dependent reduction in mass at treatment cycle 1. When we look at the top right corner, we see the primary efficacy endpoint throughout the study, and as you see, the treatment efficacy with respect to reduction in modified Ashford scale score was maintained, was sustained throughout the study. As I mentioned earlier, the study design also included a targeted home exercise program, and we also included global and functional outcome measures. Physician global assessment and goal attainment scale were secondary outcome measures in this study. Under this multidimensional approach, children in all three groups showed relevant improvement in physician global assessment, as you see in the right bottom corner. Regarding goal attainment scale, expected outcome was achieved in all three groups at treatment cycle 1, and there was no statistical difference among groups with respect to these functional outcome measures. When we look at repeat treatment cycles from cycle 2 to cycle 4, you see that functional efficacy benefits were generally sustained over one year study period. In terms of safety, adverse events in this clinical trial were mostly reflective of common childhood diseases. Muscle weakness was the only treatment-related adverse event that was seen, that was reported in more than one child in all treatment arms, and was mainly mild, moderate, and localized. So, to summarize treatment with evobotulinum toxin A, 8 units per kilo and 16 units per kilo dosages, in the affected upper limb, significantly reduced muscle tone and spasticity compared to low-dose 2 units per kilo control group. Treatment with evobotulinum toxin A, together with a targeted home exercise therapy program, resulted in global improvement as shown by physician global assessment and high goal attainment as shown by goal attainment scale, and the benefits were sustained with repeat treatment cycles. It is important to know that these data form the basis of regulatory approval for evobotulinum toxin A in several countries. Further studies in clinical practice would help clarify the place of butyline toxin A in the long-term management of spasticity in children with cerebral palsy and how it is best combined with occupational therapy for functional improvement. I would like to thank you for your attention. Hello, and thank you for joining us today for this virtual Research Spotlight presentation. My name is Charles Kenyon. I'm currently a PGY-4 at the University of Washington and one of our chief residents. Today, we're going to be discussing gender differences in health-related quality-of-life measures, specifically the Parent-Reported Pediatric Quality-of-Life Inventory Core Scales, or PEDSQL. I'd like to thank my mentors on this project, Dr. Cindy Lin, who is the Associate Director of Clinical Innovation at the Sports Institute at the University of Washington, Dr. Christine MacDonald, the Research Director at the Sports Institute, and Jason Barber, who helped guide our analysis. The objective of our study was to determine gender and age differences in health-related quality-of-life measures, such as the parent-reported PEDSQL, in typically developing children and adolescents from the Sports Institute Pediatric Concussion Data Repository. This data repository is a multi-center, multi-study data registry representing groups from across the country and is also open for submissions from international research efforts as well. Through collaboration and combining these datasets, a more robust understanding of baseline and clinical outcome data can be gained. We hypothesized that gender differences would be present across physical and psychosocial domains of the PEDSQL, and as our largest patient population in this dataset included the parent-reported measures, we chose to focus on this subset for the analysis. Through a more comprehensive understanding of our norm of data, we aim to improve our clinical ability to interpret changes in these measures when injury occurs. The PEDSQL itself is a reliable and validated measure of health-related quality-of-life in both children, healthy children and adolescents, as well as those with acute and chronic health conditions. The scale is brief, including 23 items, and practically completed in less than four minutes. Those items span four different core domains, the physical functioning, emotional, school, and social functioning as well. These scores are then converted to a 100-point scale and can be further summarized by use of the total scale score, which takes into account all of the domains together, physical health, which is essentially the same measure as the physical functioning domain, and then the psychosocial summary score, which takes into account the social, school, and emotional functioning scales all together. Again, I encourage anyone that is interested in learning more about this tool specifically to look at their website, www.pedsql.org. For our present analysis, we had 1,080 individuals, or 843 males and 237 females. Demographically, the mean age was around 11 years old, and the mean school level was approximately in the sixth grade. 65% of the data did come from the child age group, those aged 8 to 12 years old, and the population was predominantly Caucasian at 73%. Of note, the study population was largely from a higher socioeconomic group, with 69% of parents having at least an undergraduate or master's degree, and 53% reporting a household income of greater than $100,000 per year. Gender and age comparisons were adjusted for parental education and household income without notable effect. We utilized parametric Mann-Whitney tests for comparisons with subsequent application of Bonferroni adjustments, given our multiple comparisons with a significant threshold of 0.05. Our analysis initially looked at the population as a whole, and this demonstrated that parents of females reported statistically significant higher total scale scores, or higher health-related quality of life in general, in comparison to the males. You can see this as reflected by the blue in the table above. The data also showed that the percentage of parents of females reported statistically You can see this as reflected by the blue in the table above. But when we look further in subdomain analysis, this showed that there was no difference noted in physical health domain. However, the psychosocial health summary score did note a significant difference, again, with parents of females reporting higher scores, and this was further broken out with differences being significant in social functioning and school functioning domains, as highlighted in red. However, when we dove a little bit deeper and looked at age subgroups, parents of children in the age group 8 to 12 years old showed similar patterns, and this was found to be statistically significant. However, the other age groups, the young children 5 to 7 years old and adolescents 13 to 18 years old, did not show any statistical significance, although similar patterns were observed. So why do we think this is important? Clinically, many studies have demonstrated that while most children will recover from a concussion injury, lower health-related quality of life measures in the immediate post-injury period after concussion are correlated with prolonged recovery, particularly as noted in the domains of physical and school functioning, which often encompasses difficult-to-manage symptoms such as fatigue and headache that can have a significant can have a significant impact on cognition and school performance. Furthermore, others have found that mean scores in health-related quality of life measures in female children after concussion injury tend to be lower across all domains. In the context of our current study that showed parents of females generally reporting higher health-related quality of life for the female patients, this creates some concern in that a similar delta or decrement in health-related quality of life after injury in a female may represent a more significant change in comparison to their male colleagues. One critical assessment from our current study is that we did utilize the parent reported measures. While some studies have shown poor concordance between child and parent reported data, arguments are made that there is value in including both child and parent reported measures to obtain a really comprehensive assessment of a child's function and health-related quality of life. This is particularly important when assessing chronic disease or more acute injuries such as concussion. As mentioned, our data is also primarily from a predominantly white, higher socioeconomic group, which may limit the generalization of our findings. And while overall it's felt that our numbers allowed for sufficient statistical analysis, it's noted that there were far more males in the study than females. So to recap, parents of females reported higher health-related quality of life as measured by the PEDS QL, and this gender difference was found to be significant in total scaled scores and psychosocial domains, but not for the physical health domain for the population. Interestingly, when we analyzed by age, the pattern was significant for the child age group, those aged 8 to 12 years old, but was not seen to be significant for young children 5 to 7 or adolescents 13 to 18 years old. We feel that this suggests there might be a nonlinear pattern to health-related quality of life throughout pediatric development that should be taken into account when assessing these clinical measures. Thank you for joining us today. I welcome your questions and look forward to talking more. Hello. My name is Dr. Joshua Leiter, and I'd like to thank the AAPMNR for this opportunity. Our project was designed to look at the rater reliability between novice and expert raters using the balance error score system, BESS, in the pediatric population. In the evaluation of concussions, balance has been widely accepted marker for concussion diagnosis, prognosis, prognosis, and recovery in the pediatric population. A common test to measure balance is the BESS balance instrument that has three different balance conditions performed with eyes closed, hands on iliac crests, and will be performed both on firm and foam surfaces. In clinical practice, it would be expected that individuals who use BESS would have training and expertise in this instrument. In contrast, large concussion cohort studies, particularly those utilizing multiple sites, non-clinicians are often used to administer this instrument, like BESS, which may raise some concerns for validity of these measures. While there have been several studies exploring the reliability of BESS, no prior studies evaluating BESS have explored the reliability of the rater type, novice or expert. We hypothesize that novice raters who had no formal didactic training other than written instructions of the testing procedure would not be as reliable as trained clinicians. Further, we hypothesize that a focused online training of BESS administration and scoring would improve inter-rater reliability between the novice and expert raters. Our study design was a prospective reliability study comparing four expert raters and five novice raters who independently reviewed and scored the same 50 randomly selected BESS videos from a cohort of 375 participants. Five novice volunteers consisted of one resident physician, one medical student, and three undergraduate students, all who had previously had no experience administering BESS. Novice raters subsequently received formal online didactic training. The formal training consisted of balanced testing module delivered on an online format showing four BESS examples from a pediatric age group. The novice raters were required to score each BESS example and submit answers in a quiz format with accompanying feedback for incorrect answers. Completing all four examples with 100% test scores was required to successfully complete the training. The novice raters then regraded the same 50 videos. Reliability comparisons using inter-class correlation coefficient, ICC, with 95% confidence intervals were used to compare novice, untrained, trained, and expert groups. In interpreting the ICC values, we referenced our values against the standards proposed by Portney and Watkins. Values of 0.75 and above were indicative of good reliability, and those below 0.75, poor to moderate reliability. To make our distinctions more granular, we had values less than 0.5 as indicative of poor reliability. Values between 0.5 and less than 0.75 were indicative of moderate reliability. Values between 0.75 and 0.9 were indicative of good reliability, and values greater than 0.9 indicative of excellent reliability. The majority of the ICCs across the groups were between 0.75 and 1.0, considered good to excellent reliability. In terms of firm surface double-leg stance, of which inter-rater reliability was examined using percent agreement, the agreements were almost perfect, 0.958 to 0.970, as almost all the scores had zero errors. Conditions that did not have good to excellent reliability were foam surface single-leg stance in the untrained novice and trained novice groups with ICCs of 0.452 and 0.641, respectively. All of the ICCs in the expert raters were considered good to excellent with the smallest ICC of 0.828 for foam surface double-leg stance. In general, when comparing the three groups, the majority of the ICCs were higher in the expert group, expert raters, although the majority of these were not significant difference across the groups. The only significant difference was seen in the foam surface single-leg stance condition where expert raters ICCs were significantly higher than the untrained novice raters, 0.874 and 0.452, respectively. A significant difference was not seen in ICCs of foam surface single-leg stance between trained novice raters and expert raters, suggesting that the training improved inter-rater reliability of novice raters. The ICC values of total best scores suggest good inter-rater reliability for novice raters and excellent for inter-rater reliability for expert raters. Adjusting for confidence intervals, inter-rater reliability for novice raters is moderate to excellent, while for expert raters is good to excellent. Formal training appeared to benefit the majority of the balance conditions with the largest improvements seen in more challenging conditions. However, none of the improvements were significant. As balance tasks increased in difficulty, there was a larger discrepancy in inter-rater reliability, with the lowest reliability being seen in foam surface single-leg stance with novice untrained scoring, poor and expert scoring good reliability. This is likely due to the increased decision points that raters had to make to accurately score each individual, which would expose more training deficiencies. These results are encouraging for large, multi-center, prospective cohort studies who utilize a variety of personnel to accomplish the best testing for concussion research. Our study suggests that if non-expert raters are trained with only written instructions on administering the best instrument, the inter-rater reliability should be good, albeit not excellent as with expert raters. With that in mind, our study was with healthy individuals who should have baseline balance abilities. As we saw in this study, as balance tasks became more difficult, the inter-rater reliability worsened most significantly in the novice untrained cohort. Due to this, more intensive training than our program provided or becoming experienced clinicians would be required to improve reliability to the highest standard of excellence. While the reliability of our novice raters was promising, we would encourage all clinical research teams to validate and report the reliability of their own raters with disseminating their research results. Thank you again for this opportunity to present our research. I appreciate it. Hello, my name is Caroline Gormley, and today I'll be talking about urinary retention following selective dorsal rhizotomy, CONUS versus cauda equina level surgery. Selective dorsal rhizotomy, or an SDR, is a neurosurgical procedure in children with cerebral palsy where L1 through S2 sensory roots are divided at the CONUS versus cauda equina levels and rootlets are transected, which reduce spasticity in lower extremities. SDRs aim to reduce lower extremity spasticity to improve ambulation, pain management, and everyday caregiving. Neurologic adverse events are uncommon. However, urinary retention has been reported in the acute post-operative period. This study investigates the risk factors for urinary retention following an SDR. Some adverse events associated with SDRs include temporary neuropathic symptoms, post-operative infections, and urinary retention. And the parasympathetic innervation of the bladder is S2 through S4. SDRs are really one of the most common procedures to reduce spasticity in children with cerebral palsy. The results of dorsal rhizotomies are permanent. Multiple studies have identified the procedure's potential short-term and long-term results. Some safeguards to minimize the risks of urinary retention include monitoring reflex circuitry in the external anal sphincter. This study, the purpose is to evaluate if the CONUS versus cauda equina approach, percentage of rootlets cut, or number of rootlets dissected during an SDR affected the post-operative urinary retention. This study was a retrospective chart review at a large pediatric rehabilitation hospital. It included 275 children with cerebral palsy who received an SDR between 2011 and 2018. We exclude patients who had palliative or ventral dorsal rhizotomies, patients without CP, patients with a pre-SDR history of urinary retention, and those receiving rhizotomies after 2018. The surgical approach, percentage of rootlets transected, number of rootlets dissected, and number of bladder catheterization following the surgery were collected. Urinary retention was determined by the necessity to re-catheterize the patient's bladder after the folic catheter was removed post-surgery. The results that we found showed that 16% of patients, or 10 out of 64, with a history of CONUS SDR needed to be re-catheterized, and 7% of patients, or 15 out of 211 patients, with a history of cauda equina SDR needed to be re-catheterized. This was statistically significant, with a p-value of 0.04. Most of these patients needed intermittent bladder catheterization for less than seven days, but three patients in the catechointic group needed catheterizations for 12, 26, and 27 days postoperatively. No patients needed a catheterization more than 27 days. There was no correlation between patients who needed bladder catheterization and the percentage of rootlets transected. For example, catheterized patients had a percentage ranging from 25% to 44%, with a mean of 34%. Non-catheterized patients ranged from 23% to 47%, with a mean of 35%, with a p-value of 0.79. There was no correlation between patients who needed bladder catheterization and the number of rootlets dissected. Catheterized patients ranging from 72% to 198% rootlets dissected, with a mean of 157 rootlets. Non-catheterization patients ranged from 67% to 328%, with a mean of 152 rootlets, and a p-value of 0.96. There was no statistic significance difference in the number of rootlets dissected in the catechionin group versus the CONUS group, with the catechionin group ranging from 75% to 298%, and a mean of 149, and the CONUS group ranging from 67% to 328%, with a mean of 161. It is technically more difficult to do a CONUS-level dissection because of the tight space. So we wanted to make sure that these results were not a result of rootlets dissected or rootlets cut, to make sure that the similarity between each level, the CONUS versus catechionin-level surgery, were compared equally. There was no statistic significance difference in the percentage of rootlets transected in the catechionin group, ranging from 19% to 47%, with a mean of 35%, versus the CONUS group, with a range of 22% to 59%, with a mean of 34%, and a p-value of 0.52. Additionally, there was no significant difference in urinary retention risk if the lowest level of rootlets transected was at the S1 versus the S2 level, with a p-value of 0.38. In conclusion, bladder catheterization following an SDR is uncommon and transient. Higher risk in patients who received a CONUS versus catechionin-level SDR for urinary retention. There is no significant correlation between urinary retention and percentage of rootlets transected or number of rootlets dissected. There is no statistically significant correlation between urinary retention and the lowest level of surgery done at S1 versus the S2 level. I'd like to give a warm thank you to Dr. Deshpande, Dr. Otaman, Dr. Grautman, and Dr. Kim for working with me on this project. Thank you, and feel free to ask any questions. Great. Thank you so much. What pleasant voices to listen to. Thank you. Pleasant voices to listen to. I have some questions prepared, as well as we have a couple coming in on the chat forum here. So I first would like to extend there's some congratulations and thank yous on the chat line coming in. So maybe I can pose the first question to Hannah. It says, Hannah, thank you for your presentation. Based on your findings, are you doing anything different in practice, and or are you planning to do a prospective study regarding amantadine in pediatric brain injury? Thank you, Dr. Brandenburg, for your question. Actually, this was a project that I had started during residency. Unfortunately, fortunately, I graduated since then. But with the data, I think it has been helpful in hypothesizing or having an idea of what patient prognosis may be when they are on amantadine, particularly in the setting of anoxic brain injury, these patients tended not to have a large difference in their admission and discharge to EFM scores. However, given the kind of low power of the study, I can't say this is generalizable to all pediatric brain injury populations yet. We currently don't have a prospective trial underway. However, the idea of this study was to get some retrospective data and plans to do so for optimal dosing, initiation, duration of amantadine use. I would say in looking at a prospective trial, it would be helpful to, given the difference in EFMs, depending on the etiology of brain injury, to see age-matched and severity of brain injury matched controls in that prospective study. And actually, I'd like to follow up on that. You just mentioned something about matching and case controls. So it struck me as I was reading your abstract and listening to your presentation that would maybe a secondary analysis of your data using a case control design, might that reveal something maybe a little bit different? Or do you think that would strengthen what you're looking at? I think so. I think we had a large, decent size of patients. However, given the variability in their etiology of brain injuries, we had a larger population of patients with a closed traumatic injury versus a small number of patients with tumors or anoxic injuries. If the number of patients in those groups were bigger, and then we were able to better analyze and do a statistical analysis of the change in WEFM scores of patients on and off imantadine, definitely would be more powered. Thank you. All right. It looks like there's another question coming in for Ms. Carolyn. It says, is there any attempt to match patients for functional levels to assist with data analysis for the two groups? We did not look at that data, but that's very interesting. And I really hope to analyze further the data in the future. And that's a great contribution. So thank you so much for that question, Dr. Koch. Thank you. Maybe I could follow up with that as well. Maybe one of the questions that I had was, could there be potentially a centrally mediating mechanism underlying what could be going on here? If it's not the surgery that's potentially causing the retention issues, is the issue coming from higher up? Yes. So we've considered that to try to mitigate that. We tried to exclude patients who are undergoing palliative or ventral rhizotomies, who were not having CP, who had previous history of urinary retention. But we didn't look at anything like medications or lots of other aspects. You could go into so many depths with this analysis. So we really hope to do further analysis in the future and really try to elucidate what could be the difference here. All right. Does that answer your question? Fair enough. All right, let's see. I think so. Some of these populations just have so much variability associated with them. And it really struck me as I was looking at your ranges and confidence intervals, they're pretty large. So it's hard to find differences sometimes looking at means only. So you might have something sitting there just kind of teasing through what that could be. All right. Let me go ahead and I will ask a question to Charles. One of the questions that I had for you, thank you again for the nice work, everybody. One of the questions that I had for you was we know that the results can be significantly changed based on who's completing the forms for their children. So as part of the study design, we So as part of the study design, did both parents complete the form or caregivers? Or was it a single parent that completed it? Could you share a little bit with us about that? Yeah, so it was a single parent. Again, this was from a larger data repository across multiple studies. So I'm not sure that I have direct access to say, we didn't query the information in terms of if it was a mother or father. Some of those details may be available. But it was just interesting from the perspective of digging into a larger database and trying to ask a more focused question. And we went down the route of looking at the largest data set. I think the important part, as I mentioned, with the parent reported data is while there may be some discrepancies from parent to child report, I think as physiatrists, we can all appreciate having a broad picture of a function. And I think it's important, especially the younger the child is, to have that perspective from both the parent and the child. Excellent. And maybe I'll follow that up with one more question as well. And again, I don't know if you have access to finding this out because this would also be interesting. Would you suspect that the way that parents are answering these questions might change over time? In other words, some of the earlier entries into this big database that you're collecting, do you think maybe the way that moms or dads might respond about their daughters or sons might change over a 10-year period? So when you think about a child's health or their participation in sport or otherwise, what's your thought on that? I think that's definitely interesting. The individual responses were all for the normal, non-injured children. So it was a snapshot in time. And my literature focused more on concussion just because that's what we were more clinically interested in. And there are a handful of studies that looked for, I think, up to a year. Maybe there's a two-year study showing how that changes over time. But I think that's a really interesting question, particularly when there's big changes in the world. I'm sure if you asked a parent who is homeschooling right now compared to the same parent last year, they'd have a much different reflection on what school quality of life means. Yes, me included. Excellent. Thank you. Let's see. I had a question for Dr. Leiter. So first of all, excellent study. And I appreciate you taking the time to look into this instrument because of its potential power in the field to use for mass screenings. What really struck me about this is that if you had the time or the resources, you could actually take this concept and market it and use it for large-scale teaching. And the reason I was thinking about this is that is there a possibility to do, for example, iterative training with people? So I don't know how the online content was structured or who created it. But would you be interested or evaluate the possibility of someone being able to do training online and the program would respond in lifetime back to the person to improve their ability to interpret some of the granular parts of the best scoring? That would be ideal. I really liked how this was set up with you had four videos that you would review. And then you'd have a quiz format that you had to score. And then there was some feedback with that. I think that if we had a little bit more feedback and maybe a few more examples, our scoring might have improved a little bit better. There was still some questions as you score BESS, such as did they open up their eyes there or did their hands come off their hips? There are some small differences that I think might explain some of the differences in our scoring. But ideally, I think BESS could be a very good screening, like you said, for identifying how individuals with a concussion are doing. And so I would really like to see this be implemented. And I think this is a good way to look at how this screen is being used and emphasizing that there is more training than just writing, hey, these are some of the ways to do this test. We need a little bit more training to have good inter-rater reliability. Yes. And maybe a quick follow-up question. Did you find that there was a particular age group of people that were assessed that was more challenging to take the measures on than others? Younger kids harder or more challenging than older kids? Our data didn't really evaluate that portion. But looking at the video, I didn't really appreciate a difference. Some kids had a little bit different movement mechanics, which made it more challenging. Some had a little bit more attention differences, which also made it challenging. But in general, I didn't appreciate an age difference. And I don't know what our data shows in that. There was a question that came in on the feed for you, I think, with respect to being a little bit of a surprise that there was less correlation with certain parts of the exams or testing that require multiple inputs or maneuvers. And was there any training bias on part of the patients? In other words, which rater saw the patient first? And was it switched up at all? So this was 50 randomly chosen videos that each rater individually rated, isolated. So but it was the same progression, where you went from solid ground with feet together, then tandem, then single leg. And then we did the same thing with foam surfaces. So in that way, it was the same progression. And as it continued, it seemed that the single leg on foam was the hardest maneuver for each individual. And I think that's where a lot of those decision points really came into play. And if you didn't have adequate training, that's where we saw the breakdown or the difference in the inter-rater reliability. That was a great question. Thank you. Thank you so much. It looks like there's another question for Ms. Caroline. Thank you so much. Were epidurals used in the SDR patients? If so, was there any difference in the timing of removal of epidurals in the groups? Thank you for your question, Dr. Brandenburg. There were no epidurals used there under general anesthesia, and the patients were matched accordingly. I have my prepared list of questions for each of you as we go. OK. My last question, I think we can squeeze one more in, I think, is for Ms. Hannah. And that would be, looking at the age differences between the groups that you found, it's a pretty decent-sized difference, about a three-year gap between your treatment groups. Do you think age itself had any effect on your results, or was it just happenstance by grouping of treatment? By happenstance, I think there is an important factor when we look at the WIFM scoring in terms of the cognitive component and the testing itself. And what you're looking for is different when you are assessing a child that's younger or older. And I think that when we look at the mobility component, maybe there's less differences by age. But I think maybe in the cognitive component, maybe in the cognitive subgroup of the WIFM, would definitely play a big role in just the evaluation, but also the breadth or the variability. Hopefully, that answers your question. I think there is a difference. We hit 5 o'clock right on the button. Yes. Yes, OK. We hit 5 o'clock right on the button. And I just wanted to let you know, first of all, congratulations on some really neat work. The committee really enjoyed reading through your abstracts, and we're excited to present these here in this forum. We look forward to seeing it in print and some of this directly applicable and used in clinical situations. I wish this could have been in person, so I could shake your hand. But congratulations, and I hopefully look forward to meeting you soon in the future. Thank you. Thank you so much.
Video Summary
Good afternoon, and thank you for joining us. In this video, we have five speakers presenting their research on various topics related to pediatrics. The first speaker, Dr. Hannah Oh, discusses the use of amantadine in the pediatric brain injury population. She explains that amantadine is commonly used to improve arousal levels after traumatic brain injury in adults, but there is limited research on its use in children. Dr. Oh presents the results of a retrospective analysis of pediatric patients with acquired brain injury who were treated with amantadine during acute rehab care. The study found that there was a significant improvement in functional improvement and change in patients who received amantadine. The next speaker, Dr. Nijer Dursun, discusses a study on the efficacy and safety of apobutulinumtoxin A injections in reducing upper limb spasticity in children with cerebral palsy. The study showed that apobutulinumtoxin A injections significantly reduced upper limb spasticity and resulted in global improvement and goal attainment in the study population. The third speaker, Dr. Charles Kenyon, presents a study on gender differences in health-related quality of life measures in typically developing children and adolescents. The study found that parents of females reported higher health-related quality of life compared to parents of males. The fourth speaker, Dr. Joshua Leiter, discusses the reliability of the balance error scoring system (BEST) in pediatric concussion assessment. The study compared novice raters to expert raters and found that there was good reliability between the two groups, suggesting that non-expert raters can be trained to administer the BEST with good interrater reliability. The final speaker, Dr. Caroline Gormley, presents a study on urinary retention following selective dorsal rhizotomy (SDR) in children with cerebral palsy. The study found that patients who received SDR at the CONUS level had a higher risk of urinary retention compared to those who received SDR at the cauda equina level. Overall, the research presented in this video provides valuable insights into various pediatric-related topics and has the potential to improve clinical practices and patient outcomes.
Keywords
pediatrics
amantadine
pediatric brain injury
functional improvement
upper limb spasticity
cerebral palsy
gender differences
health-related quality of life
balance error scoring system
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