Protocolized Diuretic Strategy as a Treatment Algorithm for Cardiorenal Syndrome

Thalassemia is a disorder of hemoglobin (Hb) synthesis characterized by chronic hemolysis. In β-thalassemias major (β-TM), patients require regular transfusion at an early age due to severe anemia. Subsequently, intensive chelation therapy is initiated to mitigate the effects of the resultant iron overload. Clinical disease burden and the demanding treatment can affect health-related quality of life (HRQoL) outcomes in this population. The aim of this study was to assess HRQoL outcomes in Egyptian pediatric thalassemia patients. Patients were enrolled simultaneously from the hematology clinic at the National Research Institute in Cairo, Egypt. The Arabic version of SF36 tool was used to assess HRQoL outcomes. Socioeconomic data were collected by patient and parent interviews. Clinical data were collected by review of medical records. One hundred and thirty patients and 60 controls were enrolled, with a mean age of 5.4 ± 3.2 years and 6.3 ± 3.0, respectively. The HRQoL outcome scores were lower in all domains in the thalassemia group compared to the control group (p = 0.0001). Transfusion-dependent (TD) patients had lower HRQoL scores compared to nontransfusion-dependent (NTD) patients (p = 0.0001). Patient education and maternal education were independently associated with better HRQoL scores (p = 0.007, p = 0.028, respectively). Residents of rural areas reported lower scores compared to urban residents (p = 0.026). Thalassemia was associated with lower HRQoL scores, in all domains, compared to HRQoL in unaffected controls. Chronic transfusion independence, patient education, and maternal education were all associated with higher HRQoL scores. Psychological, social, and economic support for families with thalassemia are all essential tools to improve HRQoL outcomes.

138. Radiographic malalignment has a far greater impact on clinical outcomes than perioperative and postoperative complications in ASD surgery

Data regarding health-related quality of life (HRQoL) in primary sclerosing cholangitis (PSC) are sparse and have only been studied cross-sectionally in a disease which runs a fluctuating and unpredictable course. We aim to describe HRQoL longitudinally by using repeated measurements in a population-based cohort. Every 3 months from May 2017 up to August 2020, patients received digital questionnaires at home. These included the EQ-5D, 5-D Itch, patient-based SCCAI and patient-based HBI. The SF-36, measuring HRQoL over eight dimensions as well as a physical component summary (PCS) and mental component summary (MCS) score, was sent annually. Data were compared with Dutch reference data and a matched IBD disease control from the population-based POBASIC cohort. Mixed-effects modelling was performed to identify factors associated with HRQoL. Three hundred twenty-eight patients completed 2576 questionnaires. A significant reduction of small clinical relevance in several mean HRQoL scores was found compared with the Dutch reference population: 46.4 versus 48.0, p=.018 for PCS and 47.5 versus 50.5, p=.004 for MCS scores. HRQoL outcomes were significantly negatively associated with coexisting active IBD (PCS -12.2, p < .001 and MCS -12.0, p < .001), which was not the case in case of quiescent IBD. Decreasing HRQoL scores were also negatively associated with increasing age (PCS -0.1 per 10 years, p=.002), female sex (PCS -2.8, p < .001), diagnosis of AIH overlap (PCS -3.7, p=.059), end-stage liver disease (PCS -3.7, p=.015) and presence of itch (PCS -9.2, p < .001 and MCS -3.1, p=.078). The odds of reporting a clinically relevant reduction in EQ-5D scores showed seasonal variation, being lowest in summer (OR=0.48 relative to spring, p=.037). In patients with liver transplant, HRQoL outcomes were comparable to the Dutch general population. PSC patients report impaired HRQoL of small clinical relevance compared with the general population. After liver transplantation, HRQoL scores are at comparable levels to the general population. HRQoL scores are associated with potentially modifiable factors such as itch and IBD activity.

Improving Medical and Psychiatric Outcomes Among Individuals With Bipolar Disorder: A Randomized Controlled Trial

Predictors of health-related quality of life after non-catastrophic injury sustained in a road traffic crash.

THE EFFECT OF NURSE PRACTITIONER-LED CARE IN TERTIARY CARE ON HEALTH-RELATED QUALITY OF LIFE IN ADULT PATIENTS WITH ATRIAL FIBRILLATION- RESULTS OF A RANDOMIZED CONTROLLED TRIAL

ABSTRACTBackground: The analgesic effect of long-acting opioids, such as transdermal fentanyl, has been demonstrated in patients with cancer, neuropathic pain and chronic low back pain (CLBP). However, the broader effect of long-acting opioids on the patient's health-related quality of life (HRQoL) is less well known.Objective: To evaluate HRQoL outcomes in CLBP patients treated with transdermal fentanyl.Research design and methods: An observational study was conducted at 17 clinical centers in the US. Eligible patients had CLBP diagnosis for at least 3 months and were taking short-acting opioids chronically, and then initiated transdermal fentanyl treatment. Patients completed the Treatment Outcomes in Pain Survey (TOPS), which includes the SF‐36 Health Survey, at baseline and ≥ 9 weeks of treatment. The HRQoL burden of CLBP was determined by comparing CLBP patients’ SF‐36 scores to the general US population and low back pain patient norms. HRQoL outcomes were determined by comparing baseline and follow-up TOPS and SF‐36 scores. Additionally, HRQoL outcomes were evaluated across patient groups stratified by changes in pain intensity ratings as measured by an 11-point numerical rating scale.Results: At baseline CLBP patients (N = 131) scored one-to-two standard deviations (SD) below age and gender adjusted SF‐36 general population norms (MANOVA F = 127.1, p < 0.0001) and significantly lower than low back pain norms (MANOVA F = 125.3, p < 0.0001). At follow-up, significant improvement ( p < 0.05) was observed on six of the SF‐36 scales and both SF‐36 summary measures and five of the six TOPS pain-related scales. The magnitude of change in scores in effect size units among these scales ranged from 0.17 to 0.80, which are considered small to large effect size changes. HRQoL score improvement was greatest among patients experiencing the greatest pain relief.Conclusion: CLBP patients who chronically used short-acting opioids showed tremendous HRQoL burden. Favorable HRQoL outcomes were observed among patients who reported pain relief.

Effect of a pre-operative web-based dietary self-management intervention on patient satisfaction, body weight and quality of life of esophageal cancer patients: A prospective, observational study

For the past 40 years, quantitative methods and psychometric analyses have been an integral component for developing and evaluating the measurement properties of patient-reported and health-related quality of life (HRQL) outcome measures. Advances in the development of new quantitative methods and in the application of these new methods have increased our understanding of the relationship among physiologic, clinical, and HRQL outcomes and improved the development and evaluation of new health outcome instruments. In this section, we are pleased to provide a good cross section of the quantitative methods applied to HRQL and other patient-reported outcome measures in this issue of Quality of Life Research. The twelve papers included in this section cover a range in topics from various approaches to evaluating longitudinal HRQL data, evaluating theoretical models, and applying advanced psychometric methods to understanding conceptual equivalence across countries or in analyzing PRO item-level data. First, there are several articles summarizing advanced quantitative methods of handling longitudinal data. For example, Anota et al. [1] summarize the issues and definitions associated with time-to-deterioration type analyses, with illustrations from early breast cancer and metastatic pancreatic cancer samples. A number of different definitions for deterioration in HRQL outcomes can be specified, and these decisions have implications for the results of the time-to-deterioration analyses. The authors provide some guidance on the use of time-to-deterioration versus time-todefinitive-deterioration. In the article by de Bock et al. [2], Rasch analysis is used to handle informative intermittent missing data for longitudinal comparisons of PRO data. They developed several simulations with varying amounts of informative and non-informative missing data and applied longitudinal Rasch mixed models and linear mixed models. The two analysis methods were comparable when there was little missing data (\15 %), but the longitudinal Rasch mixed models performed better when there was greater missing data ([15 %). Terrin et al. [3] evaluated prediction models for transplant-related mortality based on HRQL data in a study pediatric hematopoietic stem cell transplant patients. Joint models were used to analyze the longitudinal HRQL data and the time-to-mortality data within the same statistical analysis using a single likelihood function. They found that trajectories of HRQL outcomes predicted transplant-related mortality in pediatric hematopoietic stem cell transplant patients, even after adjusting survival for baseline demographic and clinical characteristics. The next set of several articles evaluate theoretical models for understanding the relationship between clinical and HRQL measures. Mayo et al. [4] evaluated the Wilson– Cleary model [5] in patients recovering from a recent stroke. They apply structural equation models (SEM) to examine the relationship among biological variables, symptoms, functional outcomes, and health perceptions in 533 patients D. A. Revicki (&) Outcomes Research, Evidera, 7101 Wisconsin Ave., Suite 1400, Bethesda, MD 201814, USA e-mail: dennis.revicki@evidera.com

OBJECTIVE The emphasis on health-related quality of life (HRQOL) outcomes is increasing, along with an emphasis on evidence-based medicine. However, there is a notable paucity of validated HRQOL instruments for the pediatric population. Furthermore, no standardization or consensus currently exists concerning which HRQOL outcome measures ought to be used in pediatric neurosurgery. The authors wished to identify HRQOL outcomes used in pediatric neurosurgery research over the past 10 years, their frequency, and usage trends. METHODS Three top pediatric neurosurgical journals were reviewed for the decade from 2005 to 2014 for clinical studies of pediatric neurosurgical procedures that report HRQOL outcomes. Similar studies in the peer-reviewed journal Pediatrics were also used as a benchmark. Publication year, level of evidence, and HRQOL outcomes were collected for each article. RESULTS A total of 31 HRQOL studies were published in the pediatric neurosurgical literature over the study period. By comparison, there were 55 such articles in Pediatrics. The number of publications using HRQOL instruments showed a significant positive trend over time for Pediatrics (B = 0.62, p = 0.02) but did not increase significantly over time for the 3 neurosurgical journals (B = 0.12, p = 0.5). The authors identified a total of 46 different HRQOL instruments used across all journals. Within the neurosurgical journals, the Hydrocephalus Outcome Questionnaire (HOQ) (24%) was the most frequently used, followed by the Health Utilities Index (HUI) (16%), the Pediatric Quality of Life Inventory (PedsQL) (12%), and the 36-Item Short Form Health Survey (SF-36) (12%). Of the 55 articles identified in Pediatrics, 22 (40%) used a version of the PedsQL. No neurosurgical study reached above Level 4 on the Oxford Centre for Evidence-Based Medicine (OCEBM) system. However, multiple studies from Pediatrics achieved OCEBM Level 3, several were categorized as Level 2, and one reached Level 1. CONCLUSIONS The frequency of studies using HRQOL outcomes in pediatric neurosurgical research has not increased over the past 10 years. Within pediatric neurosurgery, high-quality studies and standardization are lacking, as compared with contemporary studies in Pediatrics. In general, although the HOQ, HUI, PedsQL, and SF-36 instruments are emerging as standards in pediatric neurosurgery, even greater standardization across the specialty is needed, along with the design and implementation of more rigorous studies.

Adult spinal deformity (ASD) is usually rigid and requires a combined anterior–posterior approach for deformity correction. Anterior lumbar interbody fusion (ALIF) allows direct access to the disc space and placement of a large interbody graft. A larger interbody graft facilitates correction of ASD. However, an anterior approach carries significant risks. Lateral lumbar interbody fusion (LLIF) through a minimally invasive approach has recently been used for ASD. The present study was performed to evaluate the effectiveness of oblique lumbar interbody fusion (OLIF) in the treatment of ASD. We performed a retrospective study utilizing the data of 74 patients with ASD. The inclusion criteria were lumbar coronal Cobb angle > 20°, pelvic incidence (PI)–lumbar lordosis (LL) mismatch > 10°, and minimum follow–up of 2 years. Patients were divided into two groups: ALIF combined with posterior spinal fixation (ALIF+PSF) (n = 38) and OLIF combined with posterior spinal fixation (OLIF+PSF) (n = 36). The perioperative spinal deformity radiographic parameters, complications, and health-related quality of life (HRQoL) outcomes were assessed and compared between the two groups. The preoperative sagittal vertical axis (SVA), LL, PI–LL mismatch, and lumbar Cobb angles were similar between the two groups. Patients in the OLIF+PSF group had a slightly higher mean number of interbody fusion levels than those in the ALIF+PSF group. At the final follow–up, all radiographic parameters and HRQoL scores were similar between the two groups. However, the rates of perioperative complications were higher in the ALIF+PSF than OLIF+PSF group. The ALIF+PSF and OLIF+PSF groups showed similar radiographic and HRQoL outcomes. These observations suggest that OLIF is a safe and reliable surgical treatment option for ASD.

This study of UK patients examines clinical, health-related quality of life (HRQOL) and economic outcomes associated with iron chelation therapy (ICT). Desferrioxamine (DFO) (Desferal; Novartis, Switzerland) and Deferiprone (Ferriprox; Apotex, Canada) are ICTs used to treat iron overload. DFO requires 8-to 12-hour infusions a minimum of five times per week. Deferiprone is administered in an oral daily regimen. Although pharmacologically efficacious, clinical effectiveness of ICT within the real-world setting is yet to be fully elucidated. A naturalistic cohort study of 60 patients (beta-thalassaemia, n=40; sickle cell disease, n=14; myelodysplastic syndromes, n=6; 63% female) receiving ICT in four UK treatment centres was conducted. Serum ferritin level data were abstracted from medical charts. Compliance, HRQOL, satisfaction and resource utilisation data were collected from interviews. Maximum ICT costs were estimated using the resource utilisation data associated with DFO. Mean serum ferritin levels, generally, remained elevated despite ICT. Compliance was suboptimal and HRQOL scores were lower than population norms. The total estimated mean weighted annual per-patient cost of DFO treatment was approximately pound19,000. DFO-related equipment, DFO drug, and home healthcare were estimated to account for 43%, 19% and 24% of costs, respectively. Other more minor components of total annual costs were for in-patient infusions, ICT home delivery services and monitoring costs. Generally, patients are not achieving target serum ferritin thresholds despite chronic treatment for iron overload. ICT appears to negatively impact HRQOL; compliance with ICT is poor; and, in the case of DFO, treatment costs well exceed the cost of DFO alone. These results suggest that current ICT in the real-world setting is suboptimal with respect to various clinical, HRQOL and economic outcomes.

To explore the association of brain-derived neurotrophic-factor (BDNF) Val66Met polymorphism with both baseline health related quality of life (HRQOL) scores and improvement in HRQOL scores in Chinese major depressive patients treated with fluoxetine. Patients with major depressive disorder (MDD) took fluoxetine (20 mg/day) for 6 weeks. The HRQOL was measured with the Medical Outcomes Study Short Form-36 (SF-36) at baseline and at 6th week. Patients were genotyped for Val66Met polymorphism of BDNF gene. There was a significant association between social function (SF) and BDNF Val66Met polymorphism, and patients with Met/Met genotype had better SF (compared with Val/Val P = 0.004; compared with Val/Met P = 0.005). A significant association was found between improvement in SF and BDNF Val66Met polymorphism, and patients with Met/Met genotype had poorer improvement in SF (compared with Val/Val P = 0.010; compared with Val/Met P = 0.001). Similar association was found between improvement in mental component summary (MCS) and BDNF Val66Met polymorphism, and patients with Met/Met genotype had poorer improvement in MCS (compared with Val/Val P = 0.066; compared with Val/Met P = 0.006). These results indicate that there may be association between BDNF Val66Met polymorphism and both baseline HRQOL (SF) scores and improvement in HRQOL (SF, MCS) scores in Chinese major depressive patients treated with fluoxetine.

Commentary--goodbye M(C)ID! Hello MID, where do you come from?

PRO59 ASSOCIATION BETWEEN CLINICAL OUTCOMES AND QUALITY OF LIFE IN PATIENTS WITH HEREDITARY TRANSTHYRETIN AMYLOIDOSIS