Changes In QOL Scores Research Articles

Effects of ganaxolone on non-seizure outcomes in CDKL5 Deficiency Disorder: Double-blind placebo-controlled randomized trial

CDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy. Ganaxolone, a neuroactive steroid, reduces the frequency of major motor seizures in children with CDD. This analysis explored the effect of ganaxolone on non-seizure outcomes. Children (2–19 years) with genetically confirmed CDD and ≥ 16 major motor seizures per month were enrolled in a double-blind randomized placebo-controlled trial. Ganaxolone or placebo was administered three times daily for 17 weeks. Behaviour was measured with the Anxiety, Depression and Mood Scale (ADAMS), daytime sleepiness with the Child Health Sleep Questionnaire, and quality of life with the Quality of Life Inventory-Disability (QI-Disability) scale. Scores were compared using ANOVA, adjusted for age, sex, number of anti-seizure mediations, baseline 28-day major motor seizure frequency, baseline developmental skills, and behaviour, sleep or quality of life scores. 101 children with CDD (39 clinical sites, 8 countries) were randomized. Median (IQR) age was 6 (3–10) years, 79.2 % were female, and 50 received ganaxolone. After 17 weeks of treatment, Manic/Hyperactive scores (mean difference 1.27, 95%CI –2.38,-0.16) and Compulsive Behaviour scores (mean difference 0.58, 95%CI -1.14,-0.01) were lower (improved) in the ganaxolone group compared with the placebo group. Daytime sleepiness scores were similar between groups. The total change in QOL score for children in the ganaxolone group was 2.6 points (95%CI -1.74,7.02) higher (improved) than in the placebo group but without statistical significance. Along with better seizure control, children who received ganaxolone had improved behavioural scores in select domains compared to placebo.

QOL-23. BASELINE TOTAL BRAIN VOLUME PREDICTS FOR POORER QOL AND REDUCED OVERALL SURVIVAL AFTER CRANIAL RADIOTHERAPY IN OLDER PATIENTS WITH GLIOBLASTOMA (GBM): RESULTS FROM THE PROSPECTIVE BRITER STUDY

Abstract GBM incidence peaks in the 7th and 8th decades of life with prognosis worsening with age. Short-course partial brain radiotherapy, +/- chemotherapy, extends survival but there is no validated evidence for prediction of individual risk of acute radiotherapy related side effects. BRITER, a prospective multicentre observational trial, recruited patients with newly diagnosed GBM aged ≥ 65 planned for cranial radiotherapy. Baseline MRI scans were analysed for 5 putative markers of brain resilience including relative total brain volume (ratio of cerebrospinal fluid (CSF) volume to total intracranial volume (TIV)) and their relationship to change in QoL. 126 patients were enrolled; mean age 72 years (range 65-83); 67% men, 33% women. 4% had a radiological diagnosis, 19% biopsy and 77% had debulking surgery. 21% received palliative radiotherapy alone (25% 30Gy/6#, 75% 40Gy/15#), 79% received radiotherapy with concurrent TMZ (47% 40Gy/15#, 53% 60Gy/30#). Median OS was 11.3 months. After accounting for age, sex, treatment and baseline MoCA score, there was a significant relationship between baseline CSF:TIV ratio and change in QoL score at 8 weeks post treatment. For each unit point of increase in CSF:TIV ratio, there was a corresponding decrease in EORTC QLC C30 QoL score by a factor of 1.27 (coefficient -1.27, 95% CI -3.24 to -0.19 p = 0.02). 35 participants were too unwell to complete questionnaires or had died by the 8 week follow up visit. In this subgroup, post hoc logistic regression showed baseline CSF:TIV ratio was related to risk of non-attendance (OR 1.35, 95% CI 1.01-1.80, p = 0.04). Cox regression models were fitted for time to death in 88 participants who had analysable baseline MRI sequences. Baseline CSF:TIV was associated with worsened OS (HR 1.41, 95% CI 1.19-1.66, p < 0.001). This unique prospective study indicates that baseline imaging parameters can predict the impact of cranial radiotherapy on QoL and OS.

NQPC-21 QOL EVALUATION IN THE TREATMENT COURSE OF CNS LYMPHOMA

Abstract Background Primary central nervous system lymphoma (PCNSL) is the second common primary malignant brain tumor. Compared to glioma, the treatment period is often longer, but there have been few research reports on QOL evaluation during the treatment yet. In this study, we retrospectively examined the QOL scores obtained during and following treatment of PCNSL to determine the association between changes in the QOL score and response to treatment, with particular interest in the issue in the evaluation over time. Methods Patients with newly-diagnosed PCNSL who were treated and evaluated for QOL by EORTC QLQ-C30/BN-20 at our facility since April 2016 were included. Results A total of 69 patients were included, with a median age of 69, and a male to female ratio of 41:32. The median KPS was 70, and the median MMSE score was 29 points, indicating that a majority of the patients who could undergo the testing retained adequate cognitive function. There were 38 patients (55.1%) who maintained CR during the QOL evaluation period, and their QOL function scores tended to be maintained above the baseline. In contrast, 31 patients (44.9%) who relapsed during the QOL evaluation period showed a tendency to decline in global health at the time of recurrence or evaluation immediately before recurrence, with the worsened symptom score reflecting the localization of recurrence. Any associations of changes in QOL score with therapeutic methods were identified. Changes in QOL scores were rather frequently observed with exacerbation of comorbidities, perhaps due to a high incidence of PCNSL in the elderly patients. Conclusions EORTC QLQ-C30/BN-20 enabled QOL evaluation that might reflect treatment effect in PCNSL. The reporting rate to the questionnaire was low in patients with substantial brain dysfunction, and QOL evaluation may also be affected by comorbidities. Further investigation of influencing factors on QOL evaluation is warranted.

Stereotactic Pelvic Adjuvant Radiation Therapy in Cancers of the Uterus (SPARTACUS): A Multicenter Prospective Trial Evaluating Acute Toxicities and Patient Reported Outcomes

Adjuvant radiation plays a significant role in reducing locoregional recurrences in uterine cancers. Standard treatment consists of daily radiation for 5 weeks which can be challenging for patients and the healthcare system, especially during the COVID pandemic. Hypofractionated radiotherapy has been evaluated and established in other pelvic malignancies. This study aims to evaluate the acute urinary and bowel toxicities, and patient reported outcomes following stereotactic hypofractionated adjuvant radiation for endometrial cancer.This is a prospective phase I/II trial in which patients with endometrial cancer planned for adjuvant radiation received 30 Gy in 5 fractions, every other day or once weekly. Treatment was delivered at two centers with volumetric arc radiation therapy with a body-vacuum immobilization, bowel enema and 3D image-guidance. Toxicity assessment, outcomes and patient reported quality of life (QOL, EORTC core QLQ-C30 and endometrial EN24) were collected at baseline, fractions (F) 3 and 5, and at regular follow-up intervals. Higher scores represent better global QOL/health status or worse symptoms (scale 0 - 100). Changes in QOL over time were investigated with linear mixed-effects models. A P-value threshold of 0.05 was used for statistical significance. A change in QOL score of ≥ 10 points was considered clinically significant.The median age of the 41 enrolled patients is 66 (range: 51 - 88). Histologies included 29 endometrioid adenocarcinoma, 8 serous/clear cell, 1 carcinosarcoma, and 3 dedifferentiated. Thirty patients had stage 1 disease while 3 had stage 2 and 8 stage 3. Seven patients received sequential chemotherapy and 3 had additional vault brachytherapy. Median follow-up is 9 months, with worst toxicity (GI or GU) of grade 1 and 2 in 63% and 24% respectively. No patients have experienced a grade 3 or higher toxicity. Patient-reported diarrhea and gastrointestinal domain scores were statistically significantly worse than baseline at F5 (mean paired difference = 27.2; 8.7, P < .005) and 6 weeks (mean paired difference = 7.9; 5.1, P < 0.05), and returned to baseline levels at 12 weeks (Table 1). The only clinically significant change (≥ 10) from baseline was in diarrhea at F5. There were no significant changes in urinary domain, overall health and quality of life scores. No locoregional recurrences have been found; 3 patients recurred distantly, of which 2 died of metastatic disease.Stereotactic hypofractionated radiation for uterine cancers is feasible and well-tolerated with short-term follow-up. Longer follow-up and future randomized studies are needed to further evaluate this treatment.

Increased Operative Difficulty and Poorer Outcomes after Multiple Endotherapies for Chronic Pancreatitis: An Analysis of 48 Consecutive Frey's Procedures

Introduction: Chronic pancreatitis (CP) is being increasingly treated by endotherapy as part of the step-up approach. Multiple sessions of endotherapy have led to shrinking indications and delayed referrals for surgery. This study analyses the impact of multiple endotherapy sessions on difficulty of Frey's procedure and outcomes of surgery. Material and Methods: This prospective study included 48 consecutive Frey's procedures done for CP between 2016 and 2019 at our tertiary hepatopancreatobiliary centre. Demographic data, duration of CP, number of endotherapies [≥3 (group A) or < 3 (group B)], operative difficulty [operative time, intra-operative blood loss], pain relief [Visual Analog Scale (VAS)], and quality of life [EORTC-QLQ-C30 questionnaire (QoL score)] at 6 months were recorded. Results: 28 (58.33%) out of 48 patients were in group A, and 20 (41.67%) in group B. The mean operative time was 153.22±24.1 minutes in group A vs 138.33±15.29 minutes in group B (p< 0.05). The mean blood loss was 156.11±43.28 mL in group A compared to 116.04±34.42 mL in group B (p< 0.01). Improvement in VAS at 6 months was 3.22±0.83 in group A and 4.79±1.58 in group B (p< 0.01). Improvement in QoL score at 6 months was 36.44±9.34 in group A and 35.62±12.42 in group B (p=0.85). Conclusion: Multiple endotherapy sessions increase the operative time and intra-operative blood loss, thus increasing the operative difficulty, in addition to giving poorer pain relief. Early referral for surgery without subjecting patient to multiple endotherapies would improve operative and post-operative outcomes.OP04-21Correlation of number of endotherapies with operative difficulty and outcomesParameter≥3endotherapies (group A)<3 endotherapies (group B)p-valueNumber of patients28 (58.33%)20 (41.67%)Mean duration of surgery (minutes)153.22 ± 24.1138.33 ± 15.29<0.05*Mean blood loss (mL)156.11 ± 43.28116.04 ± 34.42<0.01*Change in VAS at 6 months3.22 ± 0.834.79 ± 1.58<0.01*Change in QoL score at 6 months36.44 ± 9.3435.62 ± 12.420.85 Open table in a new tab

Quality of life and factors that affect osteoporotic hip fracture patients in Thailand

ObjectivesThe aim of this study was to report the health-related quality of life (HRQoL) over the first year after osteoporotic hip fracture and to identify the factors associated with HRQoL in Police General Hospital in Thailand. MethodsA prospective cohort study was conducted. 139 subjects who had osteoporotic hip fracture were recruited, which they will be assessed during the preinjury recall and at 3, 6, and 12 months after the fracture. HRQoL was measured as Thailand health state utility values (THSUVs), which derived from EuroQol-5-Dimension-3-Level. Factors associated with the change in QoL were analyzed. ResultsOne hundred thirty-six patients with a hip fracture were included for analysis. Their mean age was 82 ± 11 years old. The mean ± standard deviation (SD) of QoL score and EQ VAS at 12-month follow-up period were lower than before the fracture ([0.68 ± 0.105 vs. 0.55 ± 0.183] and [85 ± 10.5 vs. 79 ± 19.2]). The mean ± SD of QoL score and EQ VAS, compared before and after the fracture was estimated at −0.13 ± 0.164 and −6 ± 14.7 (P = 0.01). The positive influences on the change in QoL score were present in the subject, who are younger than 80 years old, had normal or high body mass index (BMI), and had operative treatment. ConclusionsOsteoporotic hip fractures incurred substantial loss in HRQoL which was markedly impaired at first 3 months after hip fracture and after that HRQoL was improved but did not return to prefracture levels. So primary prevention of fall is more important. The changes in HRQoL, was influenced by age, BMI, and operative treatment.

Treatment efficacy of sacral nerve stimulation in slow transit constipation: a two-phase, double-blind randomized controlled crossover study.

Sacral nerve stimulation (SNS) is a potential treatment for constipation refractory to standard therapies. However, there have been no randomized controlled studies examining its efficacy. In patients with slow transit constipation, we evaluated the efficacy of suprasensory and subsensory SNS compared with sham, in a prospective, 18-week randomized, double-blind, placebo-controlled, two-phase crossover study. The primary outcome measure was the proportion of patients who, on more than 2 days/week for at least 2 of 3 weeks, reported a bowel movement associated with a feeling of complete evacuation. After 3 weeks of temporary peripheral nerve evaluation (PNE), all patients had permanent implantation and were randomized to subsensory/sham (3 weeks each) and then re-randomized to suprasensory/sham (3 weeks each) with a 2-week washout period between each arm. Daily stool dairies were kept, and quality of life (QoL; SF36) was measured at the end of each arm. Between November 2006 and March 2012, 234 constipated patients were assessed, of whom 59 were willing and deemed eligible to participate (4 male; median age 42 years). Of the 59 patients, 16 (28%) responded to PNE. Fifty-five patients went on to permanent SNS implantation. The proportion of patients satisfying the primary outcome measure did not differ between suprasensory (30%) and sham (21%) stimulations, nor between subsensory (25%) and sham (25%) stimulations. There were no significant changes in QoL scores. In patients with refractory slow transit constipation, SNS did not improve the frequency of complete bowel movements over the 3-week active period.

Paper 25: Impact of Hip Arthroscopy for Femoroacetabular Impingement on Quality of Life

In a prospective, consecutive series of 611 patients, the largest reported to date, we have found that arthroscopic surgery for FAI improves the QoL in 75% of the patients. The benefit of hip arthroscopy for the treatment of femoroacetabular impingement (FAI) on quality of life (QOL) needs further exploration. We prospectively collected data on 611 patients, the largest series reported, who underwent hip arthroscopy for FAI over a period of five years under the care of a single surgeon. The minimum follow-up was one year with a mean follow-up of three years. The responses to the Harris hip score were translated using Rosser index matrix, to QoL score. The mean QoL score increased from 0.946 (−1.486 to 0.995) to 0.974 (0.7 to 1) at one year after surgery (p<0.001). It was noted that the mean QoL score in males was significantly (p<0.001) better than females, both before surgery and at one year after surgery; although the mean change in the QoL score was not statistically different (M=0.02,F=0.04;p=0.12). Linear regression analysis revealed that the significant predictors of change in QoL score were pre-operative QoL score (p<0.001) and the gender (p=0.02). The change in QoL score showed moderate (?=−0.66;p<0.001) negative correlation with the pre-operative QoL score. The QoL scores improved in 74.5%, remained unchanged in 15.6%; while it deteriorated in 9.9% of the patients at one year after surgery. In a prospective, consecutive series of 611 patients, the largest reported to date, we have found that arthroscopic surgery for FAI improves the QoL in 75% of the patients. The pre-operative QoL score and gender were significant predictors of the change in QoL.

Change in International Prostate Symptom Storage Subscore After Long-term Medical Therapy in BPH Patients: Finasteride and Alpha-blocker Combination Therapy in Men With Moderate-to-severe LUTS/BPH in Korea

To evaluate the efficacy and the change of International Prostate Symptom Score (IPSS) storage subscore after combination therapy (α-blocker and finasteride) in patients with lower urinary tract symptoms (LUTS) consistent with moderate-to-severe benign prostatic hyperplasia (BPH) and compared with α-blocker-only treatment. A total of 1315 patients seen in 5 urology centers in Korea from January 2001 to August 2007, with LUTS secondary to BPH were enrolled. Patients were divided into 4 groups according to treatment pattern (α-blocker monotherapy vs combination therapy) and IPSS storage subscores (≥6 vs <6). The changes from baseline in prostate volume, prostate-specific antigen concentration, IPSS, and Q(max.) were analyzed. We also determined the statistical differences among 4 groups in the IPSS, Q(max.), quality of life score (QoL), and prostate volume. Of the 1315 men, 217 (16.5%) completed the month 48 visit. All groups showed significant improvements in the IPSS total score, IPSS voiding subscores, and QoL at 1 year. However, the high storage subscore group at baseline showed a significantly higher improvement in IPSS total scores and IPSS voiding subscores compared with the low storage subscore group at year 4. IPSS storage subscores were improved only in patients with high storage subscores at baseline, especially in combination treatment group. The mean change in QoL score from baseline to year 4 was significantly improved in high storage subscore groups. Data from this retrospective analysis suggest that long-term combination treatment would be beneficial, especially to patients with severe storage symptoms at baseline.

A multicenter, randomized, double-blind, placebo-controlled, dose-finding trial of a long-acting formulation of octreotide in promoting weight loss in obese adults with insulin hypersecretion

To compare changes in weight in obese patients who received long-acting octreotide (octreotide LAR) at one of three dose levels (20, 40, or 60 mg) or placebo over 6 months and to identify the lowest dose of octreotide LAR that safely achieved optimal weight loss. Randomized, double-blind, placebo-controlled trial of octreotide LAR at three dose levels. A total of 172 adults (28 men and 144 women) with at least moderate obesity (body mass index (BMI) range 30-65 kg/m2) and evidence of insulin hypersecretion were enrolled. Patients were predominantly either Caucasian (50.0%) or African American (45.3%). The mean age (38 +/- 11 year), weight (110.7 +/- 23 kg), and BMI (39.8 +/- 6.5 kg/m2) were similar across the four treatment groups. Efficacy measures included weight, BMI, fasting serum glucose; triglycerides; percentage of total body fat and abdominal fat as measured by dual-energy X-ray absorptiometry; skin fold thickness; waist-to-hip circumference; leptin; percentage of carbohydrates, fat, and protein ingested; nutritional evaluation (including dietary analysis--3-day food record); quality of life (QoL; using the Impact of Weight on Quality of Life-Lite); Beck Depression Inventory; and Carbohydrate Craving Questionnaire. Safety measures included medical history, vital signs, physical examinations, hematology, blood chemistries, thyroid function tests, hemoglobin A1c, gallbladder ultrasound, electrocardiograms, and adverse events. After 6 months of treatment, patients receiving 40 or 60 mg of octreotide LAR experienced statistically significant weight loss compared to baseline, with mean differences from placebo in percent weight change of -1.98 and -1.87%, respectively. This finding was accompanied by statistically significant mean decreases in BMI compared to baseline, that is, a mean decrease of 0.73 and 0.79 kg/m2 for the 40 and 60 mg treatment arms, respectively. The observed weight loss was progressive during the 6-month treatment in the two higher dose groups. The lowest dose to reach statistical significance in weight loss after 6 months' treatment was 40 mg. Post hoc analysis revealed a 3.5-3.8% weight loss at month 6 in the two higher dose groups among Caucasian patients having insulin secretion greater than the median of the cohort, defined as CIR(gp) (corrected insulin response at the glucose peak) > or = 1.43. There were no statistically significant changes in QoL scores, body fat, leptin concentration, Beck Depression Inventory, or macronutrient intake. Mean changes of blood glucose AUC(0-180 min) during an oral glucose tolerance test in patients taking octreotide LAR were 39-40 mg/dl h higher than those on placebo. A total of 7-21% of the patients taking octreotide LAR reached a 5% or greater decrease in body weight from Baseline, compared to 11% for the placebo group. This was not statistically significant. The most common adverse events included diarrhea, headache, cholelithiasis, nausea, and abdominal pain. Octreotide LAR given at 40 or 60 mg resulted in statistically significant weight loss. A post hoc analysis stratifying patients by race and CIR(gp) indicated that Caucasian patients with the greater degree of insulin hypersecretion appeared to derive the most benefit from treatment. The observed safety profile was consistent with the known effects of octreotide from previous studies.

Improvement in quality of life of children with acute Crohn's disease does not parallel mucosal healing after treatment with exclusive enteral nutrition.

Crohn's disease is a chronic debilitating disorder affecting a child's physical and emotional well-being. Recent emphasis on 'quality of life' (QOL) has led to re-evaluation of available medical treatments. To assess prospectively change in QOL, clinical disease activity and intestinal mucosal inflammation in active paediatric Crohn's disease after treatment with exclusive enteral nutrition. In addition, we evaluated whether change in QOL could predict changes in paediatric Crohn's disease activity index (PCDAI) and mucosal inflammation (endoscopic and histologic). The IMPACT II questionnaire was used prospectively and longitudinally in 26 consecutively recruited children [16 males (67%), median 14 years, s.d. = 1.7 years] with active Crohn's disease (PCDAI > 20). They were treated with a new polymeric enteral feed (ACD004, Nestle) for a period of 8 weeks. All had PCDAI, QOL and endoscopic assessment at the time of diagnosis and after 8 weeks of treatment. Twenty-three of 26 children achieved a clinical remission at 8 weeks, with improvement in the QOL scores (P < 0.05). The change in QOL score after treatment was predictive of achieving a clinical remission, but not of histological improvement. Although children may find dietary restrictions difficult, this study confirms a clear improvement in QOL after treatment with exclusive enteral nutrition. However, improvement in QOL scores is not reflected by improvement in mucosal inflammation. Whilst improving QOL remains a core principal in patient management, the long-term consequences of ongoing mucosal inflammation must be better understood before relying only on short-term QOL measures to dictate treatment choices.

Quality of life pre and post epilepsy surgery

The aim of this work was to assess the health-related quality of life (HRQL) of patients pre and post surgical treatment for epilepsy. A total of 145 patients were interviewed during their pre-surgical assessment on the telemetry unit, Queen Square. The HRQL assessment comprised the quality of life assessment schedule (QOLAS), the EuroQol EQ-5D and the epilepsy surgery inventory (ESI-55). A total of 40 patients were followed up, of which 22 had undergone surgery and achieved 75% or greater reduction in seizures. The QOLAS scores for the patients who achieved 75% or greater seizure reduction post-op were significantly lower (i.e. improved HRQL) compared to baseline. The descriptive data suggest that the EQ-5D may not be capturing all of the QOL issues of relevance to patients with chronic, intractable epilepsy and the EQ-5D may not be valid for this group. Most patients queried the visual analogue scale (VAS) which asks for an overall rating of the respondent’s self-perceived health. The most frequent comments, from 42% of patients, was that ‘health’ did not include their epilepsy. Despite this, the group whose seizures were reduced had significantly higher VAS scores at follow-up. We can conclude that the VAS is sensitive to clinical change. The baseline EQ-5D utility and follow-up scores were compared. There were no significant changes in QOL scores for either group. The patients who achieved 75% or greater reduction in seizures post-op scored significantly higher (i.e. better QOL) on 2/3 composite scores of the ESI-55 at follow-up. The QOLAS, the EQ-5D VAS and the ESI-55 were sensitive to clinically defined outcome. The results for the EQ-5D profile and the EQ-5D utility suggest that the EQ-5D is not a valid and responsive instrument for use in patients with intractable epilepsy.

Are changes in QOL scores meaningful to patients?

Are changes in QOL scores meaningful to patients?