Multiple Clinicians Research Articles

Gender identity in Klinefelter Syndrome: a patient-centered approach to treatment

Introduction There is increasing evidence that gender dysphoria (GD) is more prevalent in the Klinefelter Syndrome (KS) population than in males in the general population; however, the exact incidence is uncertain. The aim of this study was to further explore the prevalence of gender-related issues, the role that physical characteristics play in gender identity, and the issues surrounding Hormone Replacement Therapy (HRT) in KS. Methods As part of a registered Quality Improvement Project (QIP), one online 23-point questionnaire on KS patient attitudes toward gender identity was shared with members of the Klinefelter Syndrome Association (KSA). In total, 139 anonymous responses were collected between December 2021 and January 2023. The questionnaire was developed with the guidance of multiple clinicians (including gender psychiatrists, urologists, psychosexual medicine specialists, and endocrinologists) and patient Delphi rounds. Data was reviewed and analyzed by 4 independent researchers within the QIP team. Results Only 53% of KS patients responding to this survey fully identified as male and 19% stated that they did not enjoy living as the sex on their birth certificate, with 43% considering changing aspects of their physical appearance to better match their gender. Regarding HRT, 67% of respondents were receiving Testosterone Replacement Therapy (TRT). 63% wanted TRT and 17% wanted estrogen, including 6% of TRT users who would prefer estrogen instead. 36% that were currently receiving TRT did not identify as male, and 3 participants stated that they have GD. Conclusion These results indicate that a significant proportion of KS patients do not fully identify with the male gender and are unhappy living as the sex on their birth certificate. Although TRT worked for most, its use should be discussed carefully with those with gender identity concerns.

7336 Decades In The Dark: The Long Road To Diagnosing And Treating Hypophosphatasia

Abstract Disclosure: V.A. Mendpara: None. G. Madrigal Loria: None. L.Z. Khan: None. Background: Hypophosphatasia (HPP) is a rare bone disorder that results from deficient activity of the tissue non-specific isoenzyme alkaline phosphatase (ALP).[1] This case highlights a patient whose diagnosis of this treatable genetic condition was significantly delayed for almost two decades, despite receiving care at a prominent academic institution. The patient's persistently low ALP levels in routine lab tests were overlooked by multiple clinicians, leading to a delay in her care and eventual treatment. Case presentation: A 61-year-old Caucasian woman presented at the endocrinology clinic with complaints of severe fatigue, generalized pain, multiple childhood fractures, and ongoing dental issues. The rheumatology clinic to which she was referred for polyarthralgia diagnosed her with Hypophosphatasia (HPP) after noting chronically low alkaline phosphatase (ALP) levels from her previous charts ranging from 15 to 24 U/L (normal range: 34 to 123 U/L) over two decades. Her serum calcium, parathyroid hormone (PTH), and 25-hydroxy vitamin D levels were within normal ranges. A DEXA scan revealed normal bone density, effectively ruling out osteoporosis. Further investigation showed elevated Vitamin B6 levels at 219.6 nmol/L (normal range: 20.0 to 125.0 nmol/L), raising suspicion for HPP. The patient's family history provided additional insight, with her mother having a history of short stature and fractures and her son experiencing premature tooth loss at the age of 2. A genetic evaluation identified a heterozygous pathogenic variant in the ALPL gene (c.881A>C, p.Asp294Ala), confirming the diagnosis of autosomal dominant HPP. Initiating treatment with Asfotase alfa, a medication targeting HPP, resulted in a marked improvement in the patient's symptoms. Discussion: This case shows the diagnostic complexities of HPP, stressing the significance of actively screening for this condition via ALP levels in routine laboratory testing or during osteoporosis evaluations. The diagnostic process is critical to distinguish HPP from primary osteoporosis, as treatment and potential risks differ significantly. Inadvertent use of bisphosphonates can worsen hypomineralization and lead to atypical femoral fractures in HPP patients. Asfotase alfa enzyme replacement therapy, available since 2015, offers a targeted approach by replacing the enzyme defect, thereby mitigating symptoms. Heightened awareness among healthcare providers of HPP in adults holds the potential to enhance diagnosis rates and facilitate improved treatment outcomes for this highly treatable condition.[1] Primer on the Metabolic Bone Diseases and Disorders of Mineral Metabolism, Eighth Edition. ASBMR. Presentation: 6/2/2024

Who Uses Pathways Around Care Management?

Clinical pathways are developed by clinicians but used for various purposes in the US health care system. Pathways were designed to result in the best possible clinical and financial outcomes. Real-world care manage­ment involves multiple clinicians, administrators, and other organizations re­sponsible for benefits administration outside of direct clinical care. As a result, there are many different uses for clinical pathways. This pilot study provides a brief literature review on users, uses, and a recent electronic survey to de­termine how users use pathways along with their feedback around their value. Key findings from this limited sample indicate a variety of uses for pathways re­ported primarily by nurses, nursing staff, or practice administration. This prelim­inary effort aids in documenting the value and real-world use of care pathways in the diverse care provider marketplace that plans to be studied in a larger sur­vey with updated questions. This area of clinical pathways remains an important effort to better understand and provide a guide for those working in this grow­ing field of clinical-economic work in the health care system.

Perinatal continuity of care for mothers with depressive symptoms: perspectives of mothers and clinicians.

Mothers with mild to moderate depression in pregnancy are at risk of developing postpartum depression. Midwife-led continuity of care may support maternal mental health throughout the perinatal period. Research is needed to better understand how continuity of care may support mothers experiencing depression in pregnancy. This study aimed to investigate the perspectives of mothers with mild to moderate depression and clinicians regarding continuity of care in the perinatal period. Fourteen mothers and clinicians participated in individual interviews or a focus group. Analysis was conducted using inductive reflexive thematic analysis with a constructivist orientation. From the perspectives of mothers and clinicians, continuity of care during the antenatal period benefitted mothers' mental health by providing connection and rapport, information about pregnancy and referral options, and reassurance about whether pregnancy symptoms were normal. The experience of seeing multiple clinicians was noted by mothers to increase distress while participants discussed the value of extending continuity of care into the postpartum period, including having someone familiar checking in on them. The importance of having a second opinion and not always relying on a single provider during pregnancy was highlighted by some mothers and clinicians. Mothers also described how multiple modes of communication with a midwife can be helpful, including the ease and accessibility of text or email. Mothers and clinicians perceived benefits of continuity of care for maternal mental health. Offering midwife-led continuity of care to mothers with mild to moderate depression during the perinatal period is recommended.

Patient-clinician interactions in shared diabetes/nephrology consultations - A qualitative observation study.

The incidence of chronic disease is increasing worldwide which, in turn, increases the demand for healthcare services. To meet these demands, healthcare systems are adapting their services in order to reduce treatment costs and ensure coherence for patients with multiple diseases. One form of adaptation is shared outpatient consultations between internal medical specialties. However, little is known about how patients interact with multiple clinicians in shared consultations. This project aimed to explore how patients with diabetes and chronic kidney disease interact with multiple clinicians in a shared outpatient setting. We performed a qualitative ethnographic study, combining focused participant observations with informal field interviews. We included 17 participants, nine males and eight females with a mean age of 67.3 in the project. The data analysis was guided by Braun and Clarke's reflexive thematic analysis and Arthur Kleinman's theory of illness and disease. We found one over-arching theme: 'A consultation which encompassed both illness and disease' and four subthemes: (1) 'The medical focal point' pertained to the focus on physiological measurements in dialogue between patients and clinicians. (2) 'The possibility of negotiations' illustrated how decisions about dialysis and pharmacological treatment were based on negotiations. (3) 'Speaking different languages' displayed how patients used alternative illness-based explanations whereas clinicians tended to use biomedical language. (4) 'Perceptions of everyday life' concerned what patients considered was best for them when managing their illness and everyday lives. Patients present information on how they balance life with physiological and psychosocial challenges. When clinicians employ a biomedical perspective, opportunities to gain information on patients' illness behaviours or cues to negotiate are missed. Patients prioritise functioning on a daily level over following treatment regimes. These findings are tenuous and require verification in similar studies in similar settings. Shared Outpatient Clinic, Patient-clinician interactions.

Leveraging an Electronic Health Record Patient Portal to Help Patients Formulate Their Health Care Goals: Mixed Methods Evaluation of Pilot Interventions.

Persons with multiple chronic conditions face complex medical regimens and clinicians may not focus on what matters most to these patients who vary widely in their health priorities. Patient Priorities Care is a facilitator-led process designed to identify patients' priorities and align decision-making and care, but the need for a facilitator has limited its widespread adoption. The aims of this study are to design and test mechanisms for patients to complete a self-directed process for identifying priorities and providing their priorities to clinicians. The study involved patients of at least 65 years of age at 2 family medicine practices with 5 physicians each. We first tested 2 versions of an interactive website and asked patients to bring their results to their visit. We then tested an Epic previsit questionnaire derived from the website's questions and included standard previsit materials. We completed postintervention phone interviews and an online survey with participating patients and collected informal feedback and conducted a focus group with participating physicians. In the test of the first website version, 17.3% (35/202) of invited patients went to the website, 11.4% (23/202) completed all of the questions, 2.5% (5/202) brought results to their visits, and the median session time was 43.0 (IQR 28.0) minutes. Patients expressed confusion about bringing results to the visit. After clarifying that issue in the second version, 15.1% (32/212) of patients went to the website, 14.6% (31/212) completed the questions, 1.9% (4/212) brought results to the visit, and the median session time was 35.0 (IQR 35.0) minutes. In the test of the Epic questionnaire, 26.4% (198/750) of patients completed the questionnaire before at least 1 visit, and the median completion time was 14.0 (IQR 23.0) minutes. The 8 main questions were answered 62.9% (129/205) to 95.6% (196/205) of the time. Patients who completed questionnaires were younger than those who did not (72.3 vs 76.1 years) and were more likely to complete at least 1 of their other assigned questionnaires (99.5%, 197/198) than those who did not (10.3%, 57/552). A total of 140 of 198 (70.7%) patients responded to a survey, and 86 remembered completing the questionnaire; 78 (90.7%) did not remember having difficulty answering the questions and 57 (68.7%) agreed or somewhat agreed that it helped them and their clinicians to understand their priorities. Doctors noted that the sickest patients did not complete the questionnaire and that the discussion provided a good segue into end-of-life care. Embedding questionnaires assaying patient priorities into patient portals holds promise for expanding access to priorities-concordant care.

Hospital staff perspectives on the cost and efficiency of peripheral intravenous catheter use: a case study from three Australian hospitals.

Peripheral intravenous catheters (PIVCs) are required by most hospitalised patients. Difficult intravenous access (DIVA) makes insertion challenging, with poor patient outcomes, treatment delays and resource waste from multiple insertion attempts, often by multiple clinicians. This exploratory qualitative case study aimed to investigate how clinical and executive hospital staff view PIVC insertions for patients with DIVA from a cost and efficiency perspective. Fifteen semi-structured interviews were conducted with staff from three large, urban Australian hospitals. Data was thematically analysed, with four themes generated: (1) PIVCs are not considered from a cost or resource use perspective; (2) resources required for successful PIVC insertion are variable and unpredictable; (3) limited funding and support exist for advanced skill and ultrasound-guided insertion; and (4) processes for PIVC training and competency are inefficient. Investment in advanced PIVC inserters (with ultrasound-guided cannulation skills, and ability to train and assess novice inserters), with clear escalation pathways to these clinicians may reduce inefficiencies and waste associated with difficult PIVC insertions.

Continuity and coordination of care for childhood cancer survivors with multiple chronic conditions: Results from the Childhood Cancer Survivor Study.

Continuity and coordination-of-care for childhood cancer survivors with multiple chronic conditions are understudied but critical for appropriate follow-up care. From April through June 2022, 800 Childhood Cancer Survivor Study participants with two or more chronic conditions (one or more severe/life-threatening/disabling) were emailed the "Patient Perceived Continuity-of-Care from Multiple Clinicians" survey. The survey asked about survivors' main (takes care of most health care) and coordinating (ensures follow-up) provider, produced three care-coordination summary scores (main provider, across multiple providers, patient-provider partnership), and included six discontinuity indicators (e.g., having to organize own care). Discontinuity (yes/no) was defined as poor care on one or more discontinuity item. Chi-square tests assessed associations between discontinuity and sociodemographics. Modified Poisson regression models estimated prevalence ratios (PRs) for discontinuity risk associated with the specialty and number of years seeing the main and coordinating provider, and PRs associated with better scores on the three care-coordination summary measures. Inverse probability weights adjusted for survey non-participation. A total of 377 (47%) survivors responded (mean age 48 years, 68% female, 89% non-Hispanic White, 78% privately insured, 74% ≥college graduate); 147/373 (39%) reported discontinuity. Younger survivors were more likely to report discontinuity (chi-square p=.02). Seeing the main provider ≤3 years was associated with more prevalent discontinuity (PR; 95%CI) (1.17; 1.02-1.34 vs≥10 years). Cancer specialist main providers were associated with less prevalent discontinuity (0.81; 0.66-0.99 vs. primary care). Better scores on all three care-coordination summary measures were associated with less prevalent discontinuity: main provider (0.73; 0.64-0.83), across multiple providers (0.81; 0.78-0.83), patient-provider partnership (0.85; 0.80-0.89). Care discontinuity among childhood cancer survivors is prevalent and requires intervention.

One Year Follow-Up of a 4-Year-Old Caucasian Girl Diagnosed with Stage IV Grade C Localized Periodontitis.

Stage IV grade C localized periodontitis (pre-puberal localized aggressive periodontitis/LPP), an extremely rare form of periodontal disease, occurs in otherwise healthy individuals (no signs of dental plaque/calculus) due a hyper-aggressive auto-immune response to high periodontopathic bacteria levels. Methods: A 4-year-old Caucasian girl with unusually high mobility of the deciduous lower left canine and localized gingival inflammation was misrecognized by multiple clinicians (initially diagnosed with hypophosphatasia, genetic and metabolic disorders, all turning negative), over a period of 4-6 months, despite initial radiographs showing clear pathognomonic signs. The LPP diagnostic was made by the last clinician, but by then the tooth was lost. Similar inflammation signs appeared around the lower deciduous right canine. X-ray examination showed similar bone and periodontal loss as previously seen, while periodontopathic bacteria tested highly positive. The patient received both mechanical cleaning and ten days of systemic antibiotic treatment (Augmentin and Metronidazole). Results: Two months later, inflammation signs disappeared, with periodontal regeneration radiologically present, and only small periodontopathic bacteria precursor concentrations. Conclusions: Despite initial periodontal loss, an adequate treatment can keep under control an LPP disease. Moreover, bone and periodontal regeneration appears if periodontopathic bacteria scores are kept lower, showing the importance of fast adequate diagnostic and treatment.

Clues for Early Diagnosis of MEN2B Syndrome Before Medullary Thyroid Carcinoma.

Early onset medullary thyroid carcinoma, later pheochromocytomas, and nonspecific extra-endocrine features (hypermobility and persistent constipation) are part of the clinical phenotype of Multiple Endocrine Neoplasia type 2B (MEN2B). A de novo pathogenic M918T variant in the rearranged during transfection proto-oncogene is usually identified. Affected children are often seen by multiple clinicians over a long period before consideration of a diagnosis of MEN2B, with metastatic medullary thyroid carcinoma often the precipitator. We describe the clinical presentation and course of 5 children ultimately diagnosed with MEN2B in New South Wales and the Australian Capital Territory, Australia between 1989 and 2021. All cases had intestinal ganglioneuromatosis that could have prompted an earlier diagnosis. Population wide newborn genomic screening for rare diseases is on the horizon. We propose that MEN2B genomic screening should be included in newborn screening programs and that careful exclusion of intestinal ganglioneuromatosis would allow earlier identification leading to improved clinical outcomes.

Systems Forces Leading to Feeding Tube Placement in Patients with Advanced Dementia: A Qualitative Exploration of Clinical Momentum.

Background: Older adults with serious illness near the end-of-life often receive invasive treatments. We developed a conceptual model called clinical momentum that describes system-level forces producing a trajectory of care that is difficult to modify and contributes to overtreatment. We sought to evaluate the empirical fit of our model by examining an event with clear guidelines against intervention: permanent feeding tube placement in patients with advanced dementia. Methods: We screened three hospitals and identified patients 65 years and older with advanced dementia who received a permanent feeding tube. We interviewed 34 family members and clinicians. We coded transcripts and characterized factors that arose during the course of care and their relationships to feeding tube placement. We used abductive analysis to compare the data with theory and identify areas of discordance and alignment. Results: We found that the course of care started with a temporary tube to correct an acute problem. As problems were identified, multiple clinicians were consulted to address a specific problem without collective discussion of the patient's health trajectory. Eventually, clinicians had to address the temporary tube, which was framed to families as a decision to place a permanent feeding tube or withdraw treatment. Conclusion: Elements of the model-including recognition-primed decision-making, "fix-it," and sunk costs-contributed to placement of a feeding tube, which set in motion a path toward intervention long before a goals-of-care conversation occurs. Clinical momentum expands our understanding of overtreatment at the end-of-life and may reveal opportunities to reduce other nonbeneficial interventions.

The Importance of Conceptual Origins: The Case of the Just-Right Challenge.

Core concepts in occupational therapy have often developed over time with contributions from multiple clinicians and scholars. This makes it difficult to determine exactly from where, or from whom, ideas originated. Without original sources, we cannot chart the historical development of ideas and honor those whose scholarship made it possible. This article documents the process undertaken to properly cite one such common concept in occupational therapy, the just-right challenge; illustrates the value of doing so; and advocates for the importance of thorough historical scholarship.

Auditory brainstem implants: an analysis of adverse events in the MAUDE database

ObjectiveAuditory Brainstem Implants (ABI) are used to restore hearing in patients lacking appropriate cochlear anatomy and/or cochlear nerve. The objective of this study was to examine the Manufacture and User Facility Device Experience (MAUDE) database to analyze adverse events. Study designThis is a study of a multi-institutional database maintained by the US FDA. SettingA database analysis was performed via collaboration of multiple clinicians at tertiary referral centers. MethodsThe MAUDE database was queried for Medical Device Reports (MDRs) relating to ABIs. MDRs were identified using the advanced search term “Implant, Auditory Brainstem” and reviewing all reports with the basic search term “Brainstem Implant”. All collected reports were individually reviewed. ResultsA total of 265 individual patient reports were reviewed, of which 55 reports met inclusion criteria. Reports regarding audiologic outcome included failure to provide hearing benefit (n = 27), implant failure/device malfunction (n = 10), and device non-use (n = 6). Postoperative complications included local skin infection (n = 3), CSF leak (n = 3), elevated ICP (n = 1), surgical site dehiscence (n = 1), swelling (n = 1), seroma formation requiring drainage (n = 1), and meningitis (n = 2). Two patients had dislodged magnets during 1.5 Tesla MRI acquisition. There were 35 instances of full explantation of the device and 1 partial removal; 13 patients had a new device implanted following explantation. ConclusionsPoor hearing results, device failure, and non-use were commonly reported causes for explanation in this analysis. This information can aid physicians in counseling patients and family members and managing device expectations.

Prescription and Dispensation of QT-Prolonging Medications in Individuals Receiving Hemodialysis

Individuals with dialysis-dependent kidney failure have numerous risk factors for medication-related adverse events, including receipt of care by multiple clinicians and initiation of some QT-prolonging medications with known risk of torsades de pointes (TdP), which is associated with higher risk of sudden cardiac death. Little is known about the prescription and dispensation patterns of QT-prolonging medications among people receiving dialysis, hindering efforts to reduce drug-related harm from these and other medications in this high-risk population. To examine prescription and dispensation patterns of QT-prolonging medications with known TdP risk and selected interacting medications prescribed to individuals receiving hemodialysis. This cross-sectional study included patients 60 years or older who were enrolled in Medicare Parts A, B, and D receiving in-center hemodialysis from January 1 to December 31, 2019. Analyses were conducted from October 20, 2022, to June 16, 2023. New-user prescriptions for the 7 most frequently filled QT-prolonging medications characterized by the timing of the new prescription relative to acute care encounters, the type of prescribing clinician and pharmacy that dispensed the medication, and concomitant use of selected medications known to interact with the 7 most frequently filled QT-prolonging medications with known TdP risk. The main outcomes were the frequencies of the most commonly filled and new-use episodes of QT-prolonging medications; the timing of medication fills relative to acute care events; prescribers and dispensing pharmacy characteristics for new use of medications; and the frequency and types of new-use episodes with concurrent use of potentially interacting medications. Of 20 761 individuals receiving hemodialysis in 2019 (mean [SD] age, 74 [7] years; 51.1% male), 10 992 (52.9%) filled a study drug prescription. Approximately 80% (from 78.6% for odansetron to 93.9% for escitalopram) of study drug new-use prescriptions occurred outside of an acute care event. Between 36.8% and 61.0% of individual prescriptions originated from general medicine clinicians. Between 16.4% and 26.2% of these prescriptions occurred with the use of another QT-prolonging medication. Most potentially interacting drugs were prescribed by different clinicians (46.3%-65.5%). In this cross-sectional study, QT-prolonging medications for individuals with dialysis-dependent kidney failure were commonly prescribed by nonnephrology clinicians and from nonacute settings. Prescriptions for potentially interacting medications often originated from different prescribers. Strategies aimed at minimizing high-risk medication-prescribing practices in the population undergoing dialysis are needed.

Lateral bone augmentation with a composite graft covered with a stretched and pinned collagen membrane: A retrospective case series using cone-beam computed tomography.

(1) To assess the effectiveness of the Sausage Technique™ when applied for lateral bone augmentation by multiple experienced clinicians; (2) To identify risk indicators for a poor outcome and to assess the need for adjunctive surgery. All patients who had been treated with the Sausage Technique™ for lateral bone augmentation by three experienced surgeons between January 2019 and December 2021 were included in a retrospective case series. The Sausage Technique™ technique includes the use of autogenous bone chips and deproteinized bovine bone mineral (1:1 ratio), covered with a stretched and pinned collagen membrane. The increase in alveolar width between the pre-operative situation and 9 months was assessed at different levels on superimposed cone-beam CT scans. Twenty-five augmentations performed in 25 patients (17 males, 8 females, mean age 51 years) were available for evaluation. Mean alveolar width increased from 4.35 to 7.43 mm at 3 mm below the crest. The mean increase of 3.08 mm (95% CI 2.10-4.06; p < 0.001) was significant. The outcome of non-containing single implant sites was significantly worse than the outcome of other sites (MD 2.67 mm; p = 0.008). The need for regrafting was 4% and the need for soft tissue augmentation was 48%. Twenty percent of the patients needed soft tissue augmentation due to a lack of keratinized mucosa width, and 32% due to a lack of buccal convexity. The former was mainly needed at multiple implant sites, whereas the latter was mainly required at single implant sites. All implant survived and remained healthy until the final follow-up. The Sausage Technique™ is an effective bone augmentation technique. Non-containing single implant sites were associated with a poor outcome and adjunctive soft tissue augmentation was needed in about half of the patients.

Experiences of people with multiple sclerosis and clinicians in using cognitive behavioural therapies for hidden symptoms: a systematic review and meta-aggregation.

Cognitive behavioural therapies (CBTs) are a standard of care for treatment of many 'hidden symptoms' in people with MS (PwMS), such as stress, depression, and fatigue. However, these interventions can vary widely in formatting and may not be tailored for PwMS. To optimize CBTs for MS, understanding the experiences of PwMS and clinicians is essential. This systematic review and meta-aggregation synthesizes existing qualitative data on stakeholder perspectives of CBTs for PwMS. Systematic searches across five major electronic databases were conducted. Studies reporting qualitative data were identified. Two reviewers performed screening, quality assessment, data extraction, and certainty of evidence assessments. Meta-aggregation was performed as per the Joanna Briggs Institute approach, entailing qualitative data extraction, developing categories, and synthesizing overall findings. Twenty-eight studies were included in this review, comprising data from 653 PwMS and 47 clinicians. In the meta-aggregation, 122 qualitative results were extracted and grouped into nine categories. Categories were then combined into six synthesized findings: (1) setting the context-life with MS, (2) reasons for participating in CBTs, (3) acceptability of and experiences with participating in CBTs, (4) perceived benefits of CBTs, (5) perceived challenges with CBTs, and (6) suggestions to improve CBTs for PwMS. A range of benefits including psychological, social, and lifestyle improvements were reported, but varied based on the design of the CBT intervention. Future CBT interventions should be tailored to participant needs, delivered in group settings, offer online options, and be delivered by a trained facilitator familiar with MS. Further exploration of the ideal CBT design for PwMS, as well as engagement with caregivers and clinicians treating MS, is warranted.

Rehabilitation Treatment Specification System for Voice Therapy: Application to Everyday Clinical Care.

Rehabilitation intervention descriptions often do not explicitly identify active ingredients or how those ingredients lead to changes in patient functioning. The Rehabilitation Treatment Specification System (RTSS) provides guidance to identify the critical aspects of any rehabilitation therapy and supported the development of standardly named ingredients and targets in voice therapy (Rehabilitation Treatment Specification System for Voice Therapy [RTSS-Voice]). This study sought to test the content validity of the RTSS-Voice and determine if the RTSS-Voice can be used to identify commonalities and differences in treatment (criterion validity) across clinicians in everyday clinical practice. Five speech-language pathologists from different institutions videotaped one therapy session for 59 patients diagnosed with a voice or upper airway disorder. Specifications were created for each video, and iterative rounds of revisions were completed with the treating clinician and two RTSS experts until consensus was reached on each specification. All 59 sessions were specified without the addition of any targets or ingredients. There were two frequent targets: (a) increased volition and (b) decreased strained voice quality. There were three frequent ingredients: (a) information regarding the patient's capability and motivation to perform a therapeutic behavior, (b) knowledge of results feedback, and (c) opportunities to practice voicing with improved resonance and mean airflow. Across sessions treating vocal hyperfunction, there was large variability across clinicians regarding the types and number of treatment components introduced, types of feedback provided, and vocal practice within spontaneous speech and negative practice. The RTSS and the RTSS-Voice demonstrated strong content validity, as they comprehensively characterized 59 therapy sessions. They also demonstrated strong criterion validity, as commonalities and differences were identified in everyday voice therapy for vocal hyperfunction across multiple clinicians. Future work to translate RTSS principles and RTSS-Voice terms into clinical documentation can help to understand how clinician and patient variability impacts outcomes and bridge the research-practice gap. https://doi.org/10.23641/asha.24796875.

Translating AD Clinical Trial Results to Meaningful Benefits: Expectations, Definitions, Challenges and Opportunities.

AbstractBackgroundClinical trials in Alzheimer’s disease (AD) are designed and powered to detect treatment impact on validated research outcome measures necessary for regulatory approval. However, what constitutes a clinically meaningful benefit, how to define, measure and translate it from the results of clinical trial results to multiple stakeholders, including clinicians and patients requires better clarification.MethodsReview and synthesis of recent publications (Assuncao, Sperling, et al. Alz Res Therapy 2022; Petersen, Aisen Andrews, Atri, et al. Alzheimers Dementia. 2023 in press) and AD therapeutics developments to provide overview and frameworks for translating AD clinical trial results to meaningful benefits with respect to expectations, definitions, challenges and opportunities that may resonate with multiple stakeholders, including patients, patient care partners and clinicians.ResultsRational for realistic expectations of putative AD disease‐modifying treatments (DMTs) are reviewed including expectations of not improving symptoms but of slowing disease progression and modestly moderating between‐group (treatment versus placebo) clinical decline. Definitions postulated to represent clinically meaningful benefits are reviewed. Confusion, misunderstanding or misinterpretations in regards to representation of thresholds for meaningful within‐patient progression are discussed, including recent misinterpretation of necessary thresholds for determining meaningful group‐level differences having created misguided expectations for clinical meaningfulness in DMT clinical trial results. Discuss confounding minimal detectable change (MCD) and utilizing between‐group differences (to assess outcome efficacy) with and to define minimal clinically important differences (MCID) (an individual, within‐patient change in outcome assessment). Within‐patient change thresholds are not intended to assess meaningfulness of differences between group‐level changes over time, but may represent meaningful within‐patient trajectories via complementary (e.g. responder/progressor) analyses. Multiple frameworks show the challenges and opportunities to multidimensionally better represent and translate results to face‐valid and patient‐centered representations to resonate with AD stakeholders. These include expanded outcomes (e.g. neuropsychiatric symptoms, socioeconomic burden); patient‐ and caregiver‐reported outcomes; complementary analyses (standardized effect‐sizes, RR/OR, NNT, Time‐to‐Event, Time‐Equivalent‐Gained); and concepts such as predictive benefit (via a disease biomarker/core domain) and cumulative benefits (accrual and potentially increasing over time).ConclusionExpanded data, analyses and frameworks for defining and translating clinically meaningful patient‐centered benefit to multiple stakeholders, particularly patients, care‐partners and clinicians, are proposed.

Supporting complex oncology workflows with telemedicine.

521 Background: Telemedicine is now foundational in health care, including oncology. Cancer care is complex and requires an integrated care model that involves multiple participants. The use of telemedicine in oncology requires careful consideration of workflows and patients’ needs. Our team reviewed existing telemedicine technology options, but none fit the needs of ongoing, complex cancer care delivery. We describe the development and implementation of a home-grown telemedicine system to support complex oncology workflows in an efficient and scalable manner at a high-volume specialty cancer center. Methods: Using an internally developed application, MSK Telemedicine, we created a technology-enabled workflow that mimicked the positive aspects of an in-person interaction for the patient, the clinician, and administrative staff. The patient (i) can invite friends and family to the visit, (ii) are notified when the clinician is running late, (iii) can join the visit without downloading special software, (iv) can receive visit reminders based on their preferences, (v) can engage with the telemedicine platform in multiple languages (vi) have translators join the call, (vi) provide research consents and participate in clinical trials virtually, and (vii) have multiple clinicians join the virtual visit. We assessed telemedicine volume, duration of the remote encounter, patient satisfaction and help desk measures from August 1, 2021 to May 31, 2023. Patients could provide feedback immediately after the visit using star ratings on a scale of 1-5, 5 being the highest score. After the first telemedicine visit using MSK Telemedicine, patients also had the opportunity to answer a comprehensive patient satisfaction questionnaire. Results: Telemedicine represented 28% of the outpatient visit volume. During the study period, the platform supported a total of 483,499 visits with 67% including more than 2 people. Clinicians requested translator services for 1,289 of visits (Nov 2022 – May 2023) and 25,411 reminders were sent in languages other than English. Patients provided 5,635 research consents. The overall patient satisfaction was 4.84 (n=53,902). Patients reported high ease of use (4.83), audio quality (4.83) and video quality (4.84). 90% of patients would recommend telemedicine to other patients and 91% would have another telemedicine visit with their provider (n=29,804). Over 96% of telemedicine calls lasted over 3 minutes, a proxy to identify successful visits. Since the implementation of the platform, the monthly number of patient calls to the help desk decreased significantly (from 1,691 in March 2022 to 585 in March 2023). Conclusions: The MSK Telemedicine system is an example of a high performing system that is successfully meeting the unique and complex needs of cancer care delivery while providing a seamless patient experience. Future research is underway to evaluate the impact of this system on additional patient and provider outcomes.

Eustachian Tube Balloon Dilation: A Comprehensive Analysis of Adverse Events.

Eustachian tube balloon dilation (ETBD) has been Food and Drug Administration (FDA) approved for refractory Eustachian tube dysfunction since 2016. While ETBD is generally seen as safe, the complication profile has not been well defined. The objective of this study was to utilize the FDA manufacturer and user facility device experience (MAUDE) database to better assess adverse events (AE) related to ETBD. This is a study of a multiinstitutional database maintained by the U.S. FDA. A database analysis was performed via the collaboration of multiple clinicians at tertiary referral centers. The FDA MAUDE database was queried for all medical device reports (MDR) related to ETBD devices from January 2012 to November 2022. Eighty-eight unique MDR were identified, 16 of which met inclusion criteria. Three MDRs were classified as device-related (18.8%); none resulted in an AE. Thirteen MDRs (81.3%) were patient-related; all were classified as AEs. The most common AE was postoperative subcutaneous emphysema (n = 6, 46.2%). Of the patients with subcutaneous emphysema, there was a wide range of severity. The most severe AE (n = 1, 6.3%) was postoperative stroke secondary to carotid artery dissection. Though ETBD is generally seen as a safe procedure, there have been several concerning AEs reported to date. Increased awareness of ETBD complications can serve as a primer for improved patient education and counseling during the informed consent process and aid surgeons in clinical decision-making. Future studies with standardized reporting protocols are warranted to create a central registry for ETBD.