Significant Improvement In Health-related Quality Research Articles

Over Three Decades of Growth Hormone Treatment in Children With Chronic Kidney Disease-Associated Growth Failure Before and After Kidney Transplantation.

Growth retardation and short final height is a common complication of chronic kidney disease (CKD) beginning in childhood, with profound deleterious effects on quality of life, mental health, and social achievement. Despite optimal treatments of causative factors for growth retardation in children with CKD, more than 50% of patients reach end-stage renal failure with a height >2 SD below the mean, and most do not demonstrate "catch-up" growth after receiving a kidney transplant. Four decades ago, recombinant human growth hormone (rhGH) treatment was introduced after studies showed increased growth velocity and improved height SDS in uremic subjects. Since then, an abundance of published data showed significant improvements in health-related quality of life, and most studies revealed no significant adverse effects. Clinical practice guidelines recommended rhGH treatment in CKD Stages 3-5D and after transplantation. Despite these guidelines, this therapy remained underutilized. Most commonly cited barriers to the implementation of rhGH treatment were the need for daily injections, financial challenges, physicians' unfamiliarity with guidelines, and fear of adverse events. rhGH has been shown to improve growth and final height in short children with CKD, with minimal adverse effects. Despite data of its successful use generated over 3 decades, this treatment is underutilized. More judicious utilization of the treatment should emphasize educating patients, their care givers, and members of the multidisciplinary treating team. Additional studies are needed to assess the longer-term rhGH treatment in larger cohorts of patients, leading to additional supportive data and clearer recommendations.

Dynamic Cognitive-Motor Training versus Cognitive Computer-Based Training in People with Multiple Sclerosis: A Preliminary Randomized Controlled Trial with 2-Month Follow-Up.

Background: Recent studies underscore the intricate relationship between cognitive and motor impairments in Multiple Sclerosis (MS), often exacerbated by CNS damage compromising neural connections. These cognitive-motor deficits contribute to reduced efficiency in daily activities and heightened risks of falls and accidents. The combination of challenging cognitive-motor training in a more ecological setting could improve cognitive functions in people with MS (PwMS). Objective: This study aims to compare the impact of dynamic cognitive-motor training versus computer-based cognitive training on overall cognitive efficiency in PwMS. Methods: Thirty-eight PwMS were recruited through the neurorehabilitation services of an Institute of research and health. Twenty-four participants were randomly assigned to the Cognitive-Motor group (CMg) and Cognitive Therapy group (CTg). Participants underwent three training sessions per week for four weeks, each lasting 50 min. The primary outcome was a comprehensive cognitive assessment using the Cognitive Impairment Index (CII), and the secondary outcomes were the Multiple Sclerosis Quality of Life Questionnaire MSQOL-54 and the Stroop Color Word Interference Test (SCWT). Results: Significant differences in the CII scores across T0, T1, and T2, as indicated by Friedman's test (χ2(2) = 14.558, p = .001), were found in the CMg. A significant difference in the change in health subscale of the MSQOL-54 was observed when comparing the groups across T0, T1, and T2 (χ2(2) = 6.059, p = .048). There were also statistically significant differences for the emotional well-being (χ2(2) = 7.581, p = .023) and health distress (χ2(2) = 11.902, p = .003) subscales. Post hoc analysis showed a statistically significant improvement in health-related quality of life (HRQOL) for the former at T1 vs. T0 (Z = -2.502, p = .012 and for the latter at T2 vs. T0 (Z = -2.670, p = .008), respectively. Conclusions: Our results support the combination of cognitive-motor training to enhance cognitive functional outcomes and quality of life compared to computer-based cognitive training in PwMS.

127 Durable Treatment of Painful Diabetic Neuropathy With 10 kHz Spinal Cord Stimulation

INTRODUCTION: Approximately 25% of people with diabetes experience painful diabetic neuropathy (PDN), but conventional medical management (CMM) is ineffective for many patients. We previously presented partial study results showing that high-frequency (10 kHz) spinal cord stimulation (SCS) provides substantial symptom relief. Here we report full study results through 24-month (24M) follow-up. METHODS: We conducted a multicenter RCT to evaluate 10 kHz SCS in PDN patients. Key study inclusion criteria were: PDN symptoms = 12M refractory to medications, lower limb pain intensity = 5cm (0-10 cm visual analog scale [VAS]), and hemoglobin A1c = 10%. Patients (n = 216) were randomized 1:1 to 10 kHz SCS plus CMM or CMM alone, with optional treatment crossover at 6M. RESULTS: Patients randomized to 10 kHz SCS experienced significantly greater pain relief at 6M than those treated with CMM alone (p < 0.001). For all patients who received 10 kHz SCS, at 24M post-implantation the average pain relief was 80% from pre-implantation (p < 0.001), and the treatment responder rate was 90%. Clinician-assessed sensory function showed significant improvements with 10 kHz SCS. At 6M, 59% of patients treated with 10 kHz SCS had sensory improvement versus 9% of those treated with CMM alone (p < 0.001). At 24M, 10 kHz SCS improved sensory function for 65% of patients. Patients receiving 10 kHz SCS also experienced significant improvements in health-related quality of life (HRQoL, p < 0.001). At 24M, the mean HRQoL improvement was 2.9-4.8 times the minimum clinically important difference for patients with type 2 diabetes. CONCLUSIONS: The results demonstrate that 10 kHz SCS provides significant, durable reductions in pain and improvements in sensory function and HRQoL. Sensory function improvements support the unique disease-modifying potential of 10 kHz SCS.

A preoperative package of care for osteoarthritis, consisting of weight loss, orthotics, rehabilitation, and topical and oral analgesia (OPPORTUNITY): a two-centre, open-label, randomised controlled feasibility trial

Osteoarthritis of the knee is a major cause of disability worldwide. Non-operative treatments can reduce the morbidity but adherence is poor. We hypothesised that adherence could be optimised if behavioural change was established in the preoperative period. Therefore, we aimed to assess feasibility, acceptability, and recruitment and retention rates of a preoperative package of non-operative care in patients awaiting knee replacement surgery. We did an open-label, randomised controlled, feasibility trial in two secondary care centres in the UK. Eligible participants were aged 15-85 years, on the waiting list for a knee arthroplasty for osteoarthritis, and met at least one of the thresholds for one of the four components of the preoperative package of non-operative care intervention (ie, weight loss, exercise therapy, use of insoles, and analgesia adjustment). Participants were randomly assigned (2:1) to either the intervention group or the standard of care (ie, control) group. All four aspects of the intervention were delivered weekly over 12 weeks. Participants in the intervention group were reviewed regularly to assess adherence. The primary outcome was acceptability and feasibility of delivering the intervention, as measured by recruitment rate, retention rate at follow-up review after planned surgery, health-related quality of life, joint-specific scores, and adherence (weight change and qualitative interviews). This study is registered with ISRCTN, ISRCTN96684272. Between Sept 3 2018, and Aug 30, 2019, we screened 233 patients, of whom 163 (73%) were excluded and 60 (27%) were randomly assigned to either the intervention group (n=40) or the control group (n=20). 34 (57%) of 60 participants were women, 26 (43%) were men, and the mean age was 66·8 years (SD 8·6). Uptake of the specific intervention components varied: 31 (78%) of 40 had exercise therapy, 28 (70%) weight loss, 22 (55%) analgesia adjustment, and insoles (18 [45%]). Overall median adherence was 94% (IQR 79·5-100). At the final review, the intervention group lost a mean of 11·2 kg (SD 5·6) compared with 1·3 kg (3·8) in the control group (estimated difference -9·8 kg [95% CI -13·4 to -6·3]). A clinically significant improvement in health-related quality o life (mean change 0·078 [SD 0·195]) were reported, and joint-specific scores showed greater improvement in the intervention group than in the control group. No adverse events attributable to the intervention occurred. Participants adhered well to the non-operative interventions and their health-related quality of life improved. Participant and health professional feedback were extremely positive. These findings support progression to a full-scale effectiveness trial. Versus Arthritis.

Limited Intervention in Adult Scoliosis-A Systematic Review.

Adult scoliosis is traditionally treated with long-segment fusion, which provides strong radiographic correction and significant improvements in health-related quality of life but comes at a high morbidity cost. This systematic review seeks to examine the literature behind limited interventions in adult scoliosis patients and examine the best approaches to treatment. This is a MEDLINE- and PubMed-based literature search that ultimately included 49 articles with a total of 21,836 subjects. Our search found that long-segment interventions had strong radiographic corrections but also resulted in high perioperative morbidity. Limited interventions were best suited to patients with compensated deformity, with decompression best for neurologic symptoms and fusion needed to treat neurological symptoms secondary to up-down stenosis and to provide stability across unstable segments. Decompression can consist of discectomy, laminotomy, and/or foraminotomy, all of which are shown to provide symptomatic relief of neurologic pain. Short-segment fusion has been shown to provide improvements in patient outcomes, albeit with higher rates of adjacent segment disease and concerns for correctional loss. Interbody devices can provide decompression without posterior element manipulation. Future directions include short-segment fusion in uncompensated deformity and dynamic stabilization constructs. Limited interventions can provide symptomatic relief to adult spine deformity patients, with indications mostly in patients with balanced deformities and neurological pain.

Diagnostic and therapeutic strategies in early onset scoliosis: A current concept review.

Substantial advances in the treatment of early onset scoliosis (EOS) over the past two to three decades have resulted in significant improvements in health-related quality of life of affected children. In addition to classifications that address the marked heterogeneity of this patient population, increasing understanding of the natural history of the disease, and new implants and treatment techniques have resulted in innovations unlike any other area of pediatric orthopedics. The growing understanding of the interaction between spinal and thoracic growth, as well as dependent lung maturation, has had a lasting impact on the treatment strategy of this potentially life-threatening disease. The previous treatment approach with early corrective fusion gave way to a growth-friendly concept. Despite the steady development of new growth-friendly surgical treatment options, whose efficacy still needs to be validated, as well as a revival of conservative growth control with serial casts and/or braces, the psychosocial burden of the long lasting and complication-prone treatments remains high. As a consequence, EOS still represents one of the greatest pediatric orthopedic challenges.

Surgical treatment of nutcracker syndrome results in improved pain and quality of life

BackgroundLeft renal vein (LRV) compression, or nutcracker phenomenon, describes the compression of the LRV, most commonly between the aorta and the superior mesenteric artery. The outflow obstruction that occurs from the compression causes venous hypertension leading to the development of pelvic collaterals, lumbar vein engorgement and gonadal vein reflux. The symptoms associated with LRV compression include abdominal pain, left flank pain, back pain, headache, pelvic pain/pressure, and hematuria. Symptomatic LRV compression can cause chronic pain and disability that impedes activities of daily living. Left renal auto transplantation (LR-AT) is one mode of treatment, leading to decreased pain with no significant vascular or urological complications. Herein we present a five patient case series with symptomatic LRV compression who underwent LR-AT with improved pain and quality of life after surgery. MethodsFive patients underwent LR-AT between June 2020-December 2020 to resolve their symptomatic LRV compression. These patients were given three validated surveys pre- and post- intervention, then again at their three month follow up visit to assess their pain and health-related quality of life. ResultsThe five patients were all female with the average age of 36.8 years old (36–41) and underwent LR-AT to treat their symptomatic LRV compression. The average Numeric Rating Scale (NRS) pain score pre intervention was 8.3 (range 6.7 to 10) which improved to pain rating 5.22 (range 2.7 to 6) post intervention, p-value = 0.013. The average pain NRS score at 3 month follow up was 3.86 (range 1.3–6), p-value = 0.006 when compared to pre-intervention pain scores. The average pain intensity pre intervention was 4.5 (4 to 5) and 2.7 (1 to 4.3) post intervention, p-value = 0.024. The average pain intensity score at 3 month follow up was 2.24 (range 1.3–3.3), p-value = 0.002 when compared to pre-intervention. The VascuQoL-6 survey score pre intervention averaged score of 9.6 (range 7–12) which improved to an average score of 20.6 (range 18–24), p-value = 0.001. The average VascuQoL score at 3 month follow up was 22.6 (range 22–24), p-value = < 0.001 when compared to pre intervention QoL scores all showing a statistically significant improvement of health-related quality of life. ConclusionThe diagnosis of LRV compression can be challenging due to the non-descript symptoms and overall lack of awareness. Understanding venous tributary pathways and drainage can help clarify why patients present with unusual symptoms. Surgical treatment of LRV compression through LR-AT can improve patients’ pain and improve vascular quality of life.

Long-term health-related quality of life and symptom severity following hysterectomy, myomectomy, or uterine artery embolization for the treatment of symptomatic uterine fibroids

Few studies have directly compared different surgical procedures for uterine fibroids with respect to long-term health-related quality of life outcomes and symptom improvement. We examined differences in change from baseline to 1-, 2-, and 3-year follow-up in health-related quality of life and symptom severity among patients who underwent abdominal myomectomy, laparoscopic or robotic myomectomy, abdominal hysterectomy, laparoscopic or robotic hysterectomy, or uterine artery embolization. The COMPARE-UF registry is a multiinstitutional prospective observational cohort study of women undergoing treatment for uterine fibroids. A subset of 1384 women aged 31 to 45 years who underwent either abdominal myomectomy (n=237), laparoscopic myomectomy (n=272), abdominal hysterectomy (n=177), laparoscopic hysterectomy (n=522), or uterine artery embolization (n=176) were included in this analysis. We obtained demographics, fibroid history, and symptoms by questionnaires at enrollment and at 1, 2, and 3 years posttreatment. We used the UFS-QoL (Uterine Fibroid Symptom and Quality of Life) questionnaire to ascertain symptom severity and health-related quality of life scores among participants. To account for potential baseline differences across treatment groups, a propensity score model was used to derive overlap weights and compare total health-related quality of life and symptom severity scores after enrollment with a repeated measures model. For this health-related quality of life tool, a specific minimal clinically important difference has not been determined, but on the basis of previous research, a difference of 10 points was considered as a reasonable estimate. Use of this difference was agreed upon by the Steering Committee at the time when the analysis was planned. At baseline, women undergoing hysterectomy and uterine artery embolization reported the lowest health-related quality of life scores and highest symptom severity scores compared with those undergoing abdominal myomectomy or laparoscopic myomectomy (P<.001). Those undergoing hysterectomy and uterine artery embolization reported the longest duration of fibroid symptoms with a mean of 6.3 years (standard deviation, 6.7; P<.001). The most common fibroid symptoms were menorrhagia (75.3%), bulk symptoms (74.2%), and bloating (73.2%). More than half (54.9%) of participants reported anemia, and 9.4% women reported a history of blood transfusion. Across all modalities, total health-related quality of life and symptom severity score markedly improved from baseline to 1-year with the largest improvement in the laparoscopic hysterectomy group (Uterine Fibroids Symptom and Quality of Life: delta= [+] 49.2; symptom severity: delta= [-] 51.3). Those undergoing abdominal myomectomy, laparoscopic myomectomy, and uterine artery embolization also demonstrated significant improvement in health-related quality of life (delta= [+]43.9, [+]32.9, [+]40.7, respectively) and symptom severity (delta= [-]41.4, [-] 31.5, [-] 38.5, respectively) at 1 year, and the improvement persisted from baseline for uterine-sparing procedures during second (Uterine Fibroids Symptom and Quality of Life: delta= [+]40.7, [+]37.4, [+]39.3 SS: delta= [-] 38.5, [-] 32.0, [-] 37.7 and third year (Uterine Fibroids Symptom and Quality of Life: delta= [+] 40.9, [+]39.9, [+]41.1 and SS: delta= [-] 33.9, [-]36.5, [-] 33.0, respectively), posttreatment intervals, however with a trend toward decline in degree of improvement from years 1 and 2. Differences from baseline were greatest for hysterectomy; however, this may reflect the relative importance of bleeding in the Uterine Fibroids Symptom and Quality of Life, rather than clinically meaningful symptom recurrence among women undergoing uterus-sparing treatments. All treatment modalities were associated with significant improvements in health-related quality of life and symptom severity reduction 1-year posttreatment. However, abdominal myomectomy, laparoscopic myomectomy and uterine artery embolization indicated a gradual decline in symptom improvement and health-related quality of life by third year after the procedure.

Alpha-Stim AID cranial electrotherapy stimulation (CES) anxiety treatment: anxiety, depression and health-related quality-of-life outcomes in primary health-care social prescribing services

PurposeThis study aims to evaluate the effect of Alpha-Stim Anxiety, Insomnia and Depression (AID) cranial electrotherapy stimulation (CES) on anxiety, depression and health-related quality of life for primary care social prescribing service patients with anxiety symptoms.Design/methodology/approachOpen-label patient cohort design with no control group. A total of 33 adult patients (average age 42 years) completed six weeks of Alpha-Stim AID use. Pre- and post-intervention assessment with participant self-report measures: Patient Health Questionnaire (PHQ-9), Generalised Anxiety Disorder (GAD-7) and European Quality of Life Five Dimension (EQ-5D-5L).FindingsReliable improvement and remission rates, respectively, were 53.39% and 33.3% for GAD-7; 46.7% and 29.5% for PHQ-9. There was a significant improvement in GAD-7 and PHQ-9 with large effect sizes. EQ-5D-5L results showed significant improvements in health-related quality of life. Perceived quality of life increased by 0.17 on the health index score, with the intervention adding 1.68 quality-adjusted life years (QALYs).Practical implicationsAlpha-Stim AID can be delivered through a primary health-care social prescribing service and most patients will use as prescribed and complete treatment course. Alpha-Stim AID CES may be an effective anxiety and depression treatment for people with anxiety symptoms. The widespread roll-out of Alpha-Stim AID in health-care systems should be considered.Originality/valueTo the best of the authors’ knowledge, this is the first study to respond to the UK’s National Institute for Health and Care (NICE) request for the collection of real-world data to understand better Alpha-Stim AID in relation to people’s treatment uptake, response rates and treatment completion rates (NICE, 2021).

Effect of a Novel Handheld Photobiomodulation Therapy Device in the Management of Chemoradiation Therapy-Induced Oral Mucositis in Head and Neck Cancer Patients: A Case Series Study

Oral mucositis (OM) is a debilitating adverse event in patients undergoing treatment for cancer. This study aimed to evaluate the therapeutic effect of a novel handheld photobiomodulation therapy (PBMT) device on chemoradiation therapy (CRT)-induced OM in patients with head and neck cancer. Head and neck cancer patients undergoing CRT who developed moderate-to-severe OM during treatment were enrolled. After PBMT and at 2 and 4 weeks after PBMT, the mean value of OM grade decreased significantly from 2.63 to 2.13, 1.31, and 0.75, respectively (p &lt; 0.05, p &lt; 0.001, and p &lt; 0.001). Moreover, we observed significant improvement in health-related quality of life (HRQoL) after PBMT compared to baseline through a validated questionnaire; EORTC QLQ-C30. In the present study, the use of this PBMT device in the management of CRT-induced OM in patients with head and neck cancer was generally well tolerated and resulted in the improvement of OM. However, evidence supporting its use remains lacking owing to limitations such as the small number of participants and lack of a control group. Therefore, further mechanistic studies and large-scale randomized controlled trials are needed to confirm the effectiveness of PBMT in the treatment of CRT-induced OM, as shown in our results.

Bilateral Pallidal Deep Brain Stimulation in Meige Syndrome: Effects on Motor Function, Neuropsychological Status, and Mood.

Bilateral pallidal deep brain stimulation (DBS) has been broadly accepted as a feasible surgical procedure for treating various forms of dystonia, but its effects on motor function, neuropsychological status, and mood in patients with Meige syndrome have rarely been examined. To evaluate the effects of bilateral globus pallidus internus DBS (GPi-DBS) on the motor performance, quality of life, neuropsychological status, and mood of patients with primary Meige syndrome. Between January 2015 and April 2019, the database of 35 patients with Meige syndrome who underwent bilateral GPi-DBS in our institution was retrospectively reviewed. The severity of dystonia, health-related quality of life, cognitive function, and mood were assessed using standardized and validated rating scales at baseline. Repeat assessment of the same domains was performed at 1 year and 3 years after neurostimulation in a similar manner. One year and 3 years after bilateral GPi-DBS, Burke-Fahn-Marsden Dystonia Rating Scale movement scores were improved by 65% and 72% and Burke-Fahn-Marsden Dystonia Rating Scale disability scores were improved by 49% and 57%, respectively. The significant improvement in health-related quality of life observed at 1 year was sustained at 3 years. Relative to baseline and to the 1-year assessment, cognitive functions and mood remained stable after 3 years of neurostimulation. No deaths or life-threatening events were reported over the study period. Bilateral GPi-DBS is a safe and effective approach for medically refractory Meige syndrome that can improve motor function and quality of life without cognitive and mood side effects.

Patient-reported outcomes with direct-acting antiviral treatment for hepatitis C in West and Central Africa (TAC ANRS 12311 trial)

Patient-reported outcomes (PROs) are poorly documented for patients with chronic hepatitis C on direct-acting antiviral (DAA) treatment in low-to-middle-income countries. We documented PROs during and after DAA treatment in participants of the TAC ANRS 12311 trial (West and Central Africa). Trial participants received a 12-week regimen containing either sofosbuvir plus ribavirin (HCV genotype 2, n= 40), or sofosbuvir plus ledipasvir (HCV genotypes 1 and 4, n= 80). Health-related quality of life (SF-12), fatigue (Piper Fatigue scale), and self-reported symptoms (35-symptom list) were assessed at enrolment (Week (W) 0), during treatment (W2, W4, W8 and W12) and after treatment (W24 and W36). These PROs were compared between W0 and W36 (Wilcoxon signed-rank or McNemar tests). Mixed-effects linear regression models helped identify correlates of physical and mental quality of life component summaries (PCS and MCS) in a longitudinal analysis. Most PROs were significantly improved 24 weeks after treatment end (W36), without significant differences between treatment groups. For the post-treatment period, multivariable analysis showed significant increases in PCS for patients with cirrhosis and in MCS for patients in the sofosbuvir plus ribavirin group. A higher number of self-reported symptoms at W0 was associated with lower PCS and MCS, older age and cirrhosis with lower PCS, and male sex and HCV cure with higher PCS. Sofosbuvir-based DAA therapy was associated with a significant improvement in PROs 6 months after treatment end in patients with chronic HCV infection from Central and West Africa. These findings may guide HCV treatment providers in low-to-middle-income countries to deliver pre-treatment information concerning the benefits of DAAs beyond viral eradication. NCT02405013. Perceptions and experiences (i.e. "patient-reported outcomes") of patients with chronic hepatitis C receiving direct-acting antivirals (DAAs) are poorly documented in the African setting. This study shows significant improvements in health-related quality of life, fatigue, and self-reported symptoms 24 weeks after the end of a 12-week sofosbuvir-based DAA regimen in 120 patients from Central and West Africa. These findings substantially add to the body of knowledge about DAA therapy in the African setting. Treatment providers should be encouraged to inform patients of the benefits of DAAs beyond viral eradication, to increase treatment adherence and retention in care.

Factors associated with a clinically significant improvement in health-related quality of life after total knee arthroplasty.

The aim was to identify independent preoperative factors associated with changes in health-related quality of life (HRQoL) following total knee arthroplasty (TKA), and whether these could be used as indicators for surgery. A retrospective study of 3127 TKA patients was undertaken that included 1194 (38.2%) male and 1933 (61.8%) female patients, with a mean age of 70.5years (standard deviation 9.0). Patient demographics, body mass index and ASA grade, Oxford Knee Score (OKS) and EuroQol 5-dimension (EQ-5D) 3-level, which was used to assess HRQoL, were collected preoperatively and 2 years postoperatively. When adjusting for confounding factors, obesity grade II (p = 0.002), ASA grade 3 (p = 0.002), and a better preoperative OKS (p < 0.001) or EQ-5D score (p < 0.001) were associated with a decreased improvement in the HRQoL at 2-years. A preoperative EQ-5D of < 0.8 (95.7% specific, AUC ≥ 67.0%) or an OKS of < 36 (97.1% specific, AUC ≥ 58.1%) was associated with a clinically significant improvement HRQoL. Patella resurfacing (n = 1454, 46.5%) was not independently associated with a clinical or statistically significant improvement in HRQoL. According to preoperative factors, no subgroup of patients benefited more from patella resurfacing according to improvement in their HRQoL. ASA grade 3, grade II obesity, a better preoperative EQ-5D or OKS were independently associated with a lesser improvement in HRQoL. The thresholds identified in the EQ-5D or OKS for a clinically significant improvement in HRQoL may be used as potential indicators for referral for TKA. Patella resurfacing was not independently associated with a clinically important improvement in HRQoL. Retrospective diagnostic study, Level III.

522. Significant Improvement in Health-Related Quality of Life (HRQL) with RBX2660: Results from a Phase 3 Randomized, Placebo-Controlled Trial in Recurrent Clostridioides Difficile Infection (PUNCH CD3)

Abstract Background Recurrence of Clostridioides difficile infection (rCDI) is common - up to 35% of patients may recur. RBX2660 is a microbiota restoration therapy to reduce rCDI. Here we report 8 weeks HRQL results using the Clostridioides difficile Health-related Quality-of-Life Questionnaire (Cdiff32), a disease-specific instrument, from PUNCH CD3 (a randomized, double-blinded, placebo-controlled RBX2660 Phase 3 trial, NCT03244644). Methods Cdiff32 includes three domains (physical, mental, and social) and a total score (all range from 0 to 100 [100 best possible]). Changes in Cdiff32 from baseline to week 8 were compared between RBX2660 and placebo (PBO) using unadjusted and adjusted analyses controlling for baseline score, demographic and disease characteristics. Per trial protocol, missing data were imputed via last observation carried forward (LOCF); as-observed data were also analyzed. Patients experiencing recurrence after blinded treatment received open-label RBX2660 per physician discretion; these participants were excluded unless, per LOCF, data were available from the blinded period for week 8 use. Results A total of 206 patients (140 RBX2660, 66 PBO) were included, with similar age (mean±SD) 61.1±16.9 yrs (RBX2660) and 57.3±16.4 yrs (PBO) and baseline Cdiff32 scores. More than half of the patients had multiple comorbidities. Cdiff32 scores improved significantly from baseline to weeks 1, 4, and 8 for both arms, with greater improvements for RBX2660 through week 8 (Figs. 1 &amp; 2). At week 8, statistical differences were found for mental domain (unadjusted: 8.01±3.64; adjusted: 7.07, 95% confidence interval: [0.28, 13.86], both P&amp;lt; 0.05) and total score (adjusted: 6.11, [0.14, 12.08], P&amp;lt; 0.05), all favoring RBX2660. Results were similar for the as-observed analyses, with the adjusted physical domain also statistically favoring RBX2660. Conclusion Most patients in this study reported improved HRQL. Improvements were observed in both arms, but RBX2660-treated patients had more robust and sustained improvements with statistically significant differences in Cdiff32 scores. This study suggests that microbiome restoration therapy might positively affect HRQL; future research may link these improvements directly with microbiota changes. Disclosures Paul Feuerstadt, MD, FACG, AGAF, Ferring/Rebiotix Pharmaceuticals: Advisor/Consultant|Ferring/Rebiotix Pharmaceuticals: Grant/Research Support|Merck and Co: Advisor/Consultant|SERES Therapeutics: Advisor/Consultant|SERES Therapeutics: Grant/Research Support|Takeda Pharmaceuticals: Advisor/Consultant Erik R. Dubberke, MD, MSPH, Abbott: Advisor/Consultant|Ferring: Advisor/Consultant|Ferring: Grant/Research Support|Merck: Advisor/Consultant|Pfizer: Advisor/Consultant|Pfizer: Grant/Research Support|Seres: Advisor/Consultant|Summit: Advisor/Consultant|Synthetic Biologics: Grant/Research Support Amy Guo, PhD, Ferring Pharmaceuticals: Employee Adam Harvey, PhD, Ferring Pharmaceuticals: Employment Min Yang, MD, PhD, Analysis Group, Inc.: I am an employee of Analysis Group, Inc., which has received consulting fees from Ferring for the conduct of this study. Viviana García-Horton, PhD, Analysis Group, Inc.: Employee of Analysis Group, Inc., which received consulting fees from Ferring for the conduct of this study. Mirko Fillbrunn, PhD, Analysis Group, Inc.: I am an employee of Analysis Group, Inc., which has received consulting fees from Ferring for the conduct of this study. Glenn S. Tillotson, PhD, Ferring Pharmaceuticals: Advisor/Consultant|Paratek Pharmaceuticals: Grant/Research Support|Spero Pharmaceuticals: Advisor/Consultant|Taro Pharmaceuticals: Advisor/Consultant Lindy Bancke, PharmD, Rebiotix, a Ferring Company: Employee Kevin W. Garey, PharmD, MS, Acurx: Grant/Research Support|cidara: Advisor/Consultant|cidara: Grant/Research Support|Paratek: Grant/Research Support|Seres Health: Grant/Research Support|Summit: Grant/Research Support.

Drivers of inpatient hospitalization costs, joint-specific patient-reported outcomes, and health-related quality of life in shoulder arthroplasty for cuff tear arthropathy

Cuff tear arthropathy (CTA) can be successfully treated with various types of shoulder arthroplasty. While reverse total shoulder arthroplasty (RSA) is commonly used to treat CTA, CTA hemiarthroplasty (CTA-H, hemiarthroplasty with an extended humeral articular surface) can also be effective in patients with preserved glenohumeral elevation and an intact coracoacromial (CA) arch. As the value of arthroplasty is being increasingly scrutinized, cost containment has become a priority. The objective of this study was to assess hospitalization costs and improvements in joint-specific measures and health-related quality of life for these two types of shoulder arthroplasty in the management of CTA. Seventy-two patients (39 CTA-H and 33 RSA) were treated during the study time period using different selection criteria for each of the two procedures: CTA-H was selected in patients with retained active elevation, an intact CA arch, and an intact subscapularis, while RSA was selected in patients with pseudoparalysis or glenohumeral instability. The Simple Shoulder Test (SST) was used as a joint-specific patient-reported outcome measure. Improvement in quality-adjusted life years was measured using the Short Form 36. Costs associated with inpatient care were collected from hospital financial records. Univariate and multivariate analyses focused on determining predictors of hospitalization costs and improvements in patient-reported outcomes. Significant improvements in SST and Short Form 36 physical component scores were seen in both groups. Inpatient hospitalization costs were significantly higher in the RSA group than that in the CTA-H group ($15,074±$1614 vs. $10,389±$1948, P<.001), driven primarily by supplies including the cost of the prosthesis ($9005±$2521 vs. $4715±$2091, P<.001). The diagnosis of diabetes was an independent predictor of higher inpatient hospitalization costs for both groups. There were no independent predictors for quality-adjusted life year improvements. SST improvement in the CTA-H group was significantly higher in patients with lower preoperative SST scores. Using a standard algorithm of CTA-H for shoulders with retained active elevation and an intact CA arch and RSA for poor active elevation or glenohumeral instability, both procedures led to significant improvements in health-related quality of life and joint-specific measures. Costs were significantly lower for patients meeting the selection criteria for CTA-H. Further value analytics are needed to compare the relative cost effectiveness of RSA and CTA-H for patients with CTA having retained active elevation, intact CA arch, and intact subscapularis.

Outcomes of operative treatment for adult spinal deformity: a prospective multicenter assessment with mean 4-year follow-up.

The current literature has primarily focused on the 2-year outcomes of operative adult spinal deformity (ASD) treatment. Longer term durability is important given the invasiveness, complications, and costs of these procedures. The aim of this study was to assess minimum 3-year outcomes and complications of ASD surgery. Operatively treated ASD patients were assessed at baseline, follow-up, and through mailings. Patient-reported outcome measures (PROMs) included scores on the Oswestry Disability Index (ODI), Scoliosis Research Society-22r (SRS-22r) questionnaire, mental component summary (MCS) and physical component summary (PCS) of the SF-36, and numeric rating scale (NRS) for back and leg pain. Complications were classified as perioperative (≤ 90 days), delayed (90 days to 2 years), and long term (≥ 2 years). Analyses focused on patients with minimum 3-year follow-up. Of 569 patients, 427 (75%) with minimum 3-year follow-up (mean ± SD [range] 4.1 ± 1.1 [3.0-9.6] years) had a mean age of 60.8 years and 75% were women. Operative treatment included a posterior approach for 426 patients (99%), with a mean ± SD 12 ± 4 fusion levels. Anterior lumbar interbody fusion was performed in 35 (8%) patients, and 89 (21%) underwent 3-column osteotomy. All PROMs improved significantly from baseline to last follow-up, including scores on ODI (45.4 to 30.5), PCS (31.0 to 38.5), MCS (45.3 to 50.6), SRS-22r total (2.7 to 3.6), SRS-22r activity (2.8 to 3.5), SRS-22r pain (2.3 to 3.4), SRS-22r appearance (2.4 to 3.5), SRS-22r mental (3.4 to 3.7), SRS-22r satisfaction (2.7 to 4.1), NRS for back pain (7.1 to 3.8), and NRS for leg pain (4.8 to 3.0) (all p < 0.001). Degradations in some outcome measures were observed between the 2-year and last follow-up evaluations, but the magnitudes of these degradations were modest and arguably not clinically significant. Overall, 277 (65%) patients had at least 1 complication, including 185 (43%) perioperative, 118 (27%) delayed, and 56 (13%) long term. Notably, the 142 patients who did not achieve 3-year follow-up were similar to the study patients in terms of demographic characteristics, deformities, and baseline PROMs and had similar rates and types of complications. This prospective multicenter analysis demonstrated that operative ASD treatment provided significant improvement of health-related quality of life at minimum 3-year follow-up (mean 4.1 years), suggesting that the benefits of surgery for ASD remain durable at longer follow-up. These findings should prove useful for counseling, cost-effectiveness assessments, and efforts to improve the safety of care.

Quality care guidelines for uterine artery embolisation in women with symptomatic uterine fibroids in Australia and New Zealand: According to the AGREE-II checklist and endorsed by the Interventional Radiology Society of Australasia.

Quality care guidelines for uterine artery embolisation in women with symptomatic uterine fibroids in Australia and New Zealand: According to the AGREE-II checklist and endorsed by the Interventional Radiology Society of Australasia.

Acceptance and Commitment Therapy for inpatients with psychosis –an acceptability and feasibility single case AB designed study

BackgroundThere is a lack of research on psychological treatments for psychosis in the inpatient setting. Acceptance and Commitment Therapy for psychosis (ACTp) is a diagnose specific adaptation of the transdiagnostic ACT treatment model which has shown promising results in patients with psychosis. AimThe aim was to explore treatment effects of ACTp on inpatients with psychosis on symptoms, level of functioning, level of activity, psychological flexibility, –health-related quality of life, valued living, and to explore the acceptability and feasibility of ACTp. MethodTwelve inpatients with psychosis were treated with 3–10 daily sessions of ACTp in a non-concurrent single case AB design with additional pre-post and mid-measures. The mean age at clinic A was 48.88 (SD = 11.59) and 48.75 (SD = 16.07) at clinic B. We calculated non - overlap of all pairs (NAP) in daily measures, and reliable change index (RCI), and clinical significance in pre-post measures. ResultsHalf of the participants improved significantly on health-related quality of life and depression in daily measures and on depression and anxiety in pre-post measures. ACTp was quite acceptable and recruitment and the deliverance of therapy in the inpatient setting was feasible. ConclusionIn the present study, participants found the ACTp treatment to be comprehensive and helpful. Recruitment and treatment of inpatients were feasible, however, since most participants were discharged before ACTp protocol was completed, fewer sessions or having sessions more frequently needs further investigation. Daily measurement showed a significant improvement in health-related quality of life and depression for a majority of the participants.

Correlation of circulating tumor cells as a positive interventional biomarker in cancer patients.

e14527 Background: Circulating Tumor Cells (CTC) is a predictive biomarker for accounting disease progression and for minimal residual disease (MRD). Effect of conventional anticancer therapy on CTC count is well documented, however there is paucity of data related to effect of CAM based modality on CTC count in cancer patients. This study provides a preliminary observation about the effect of Ayurveda therapy on CTC count. Methods: The retrospective study involved stratification of 72 cancer patients undergoing cancer and maintenance treatment in a non-conventional, Ayurveda cancer treatment in India. For monitoring of prognosis in cancer patients, CTC count was assesed in patients attending Rasayu Cancer clinic. 17 cancer types, namely, breast cancer, cervix and ovarian cancer, bladder, lung, Head and neck squamous carcinoma, follicular thyroid, diffuse B-cell lymphoma, Hodgkin’s and non-Hodgkin’s, colorectal, Hepatocellular, stomach with abdominal metastasis, metastatic prostate cancer, SCC with lung and skeletal metastasis etc. Total 33 (46%) males and 39 (54.1%) female patients of various types and stages were analyzed for the presence of CTCs retrospectively. CTCs were isolated and renumerated from 1.5 ml of patient’s blood sample using OncoDiscover Liquid Biopsy Technology platform enriched with anti-EpCAM antibody immunomagnetic kit, approved by Drug Controller General of India (DCGI). CTCs were confirmed for cytokeratin+ 18 (CK18), DAPI+ and CD45-. Subsequently CTCs were imaged using Zeiss Axio Observer 7 fluorescence microscope. In 28 patients (50%), CTC was accounted for at both pre and post treatment in duration of 3-6 months. 28 patients were assessed for quality of life measured by FACT-G questionnaire. The outcome was quantified for clinico-pathological parameters; age / gender, cancer types, and CTC distribution. Results: The mean and median CTC distribution was observed to be 15.34 and 12.5, respectively. 8% patients showed the absence of any CTCs (6 subjects (1 male and 5 females). While 32 males (96%) and 34 females (87%) showed presence of CTCs. The correlation coefficient of CTC presence in male and female was significant 0.4799 (p &lt; 0.05). The Ayurveda Rasayana therapy showed significant reduction in post interventional CTC count (-3.94±1.2) (P=0.02). In addition, this group of patients also showed significant improvement in health-related quality of life as measured by the FACT-G questionnaire (P&lt;0.05). Conclusions: CTC is a validated predictive biomarker for accounting minimal residual disease, both in pre and post cancer treatments. The enumeration of CTCs represents an effective prognostic biomarker in assessing the disease progression. Reduction in CTC count was seen to be associated with improvement in health-related quality of life (QoL) which needs to be investigated further to establish correlation.

Translated article] Effect of Drug Compounding on Quality of Life in Patients With Genodermatoses: A Cross-Sectional Study

BackgroundCutaneous manifestations are complicated to treat in rare diseases. The main aim of this study was to analyze the impact of compounded drugs prepared by hospital pharmacists on the quality of life of patients with genodermatoses. Material and methodsWe undertook a cross-sectional study of patients with genodermatoses treated with topical medications compounded and dispensed by the pharmacy at Complejo Hospitalario Universitario in Pontevedra, Spain. We collected demographic data and answers to questionnaires examining generic and disease-specific quality of life, treatment satisfaction, and treatment adherence. ResultsNine patients were included. We observed a significant improvement in health-related quality of life following treatment with compounded drugs. Satisfaction with the topical medications was 2.8 on a scale of 0 (greatest satisfaction) to 25. Treatment adherence was greater than 89%. ConclusionsDrug compounding facilitates access to orphan drugs that are not available for many rare diseases. Few studies, however, have analyzed impact on quality of life in this setting. In this series of patients with genodermatoses, topical medications compounded and dispensed by a hospital pharmacy improved health-related quality of life. This preliminary study has given rise to a multicenter study of compounding for ichthyosis. We expect that analysis of a larger sample will confirm our findings.