Clinician-reported Outcomes Research Articles

Women benefit from endovenous ablation with fewer complications: Analysis of the Vascular Quality Initiative Varicose Vein Registry

ObjectiveTo evaluate the association between gender and long-term clinician-reported and patient-reported outcomes after endovenous ablation procedures. MethodsThis retrospective cohort study of prospectively collected data from the Vascular Quality Initiative’s Varicose Vein Registry included patients undergoing endovenous ablation procedures on truncal veins with or without treatment of perforating veins between 2015 and 2019. A univariate analysis included comparisons of preprocedural, postprocedural, and periprocedural change in Venous Clinical Severity Score (VCSS) and total symptom score by gender. Rates of complications including deep vein thrombosis, endovenous heat-induced thrombosis, leg pigmentation, blistering, paresthesia, incisional infection, and any postprocedural complications were reported by gender. Multivariable analysis leveraged linear regression to examine how gender affected the relationships between patient characteristics, complication rates, and periprocedural change in VCSS score and total symptom score. ResultsOf 9743 patients who met the inclusion criteria, 3090 (31.7%) were men and 6653 (68.2%) were women. The perioperative change in VCSS score was greater for men than women (average –4.46 for men vs –4.13 for women; P < .0001). Perioperative change in total symptom score was greater for women than for men (average –10.64 for women vs –9.64 for men; P < .0001). Women had lower incidence of any leg complication (6.1% vs 8.6%; P = .001) endovenous heat-induced thrombosis (1.1% vs 2.2%; P = .002), and infection (0.4% vs 0.7%; P = .001). In multivariable analysis, among patients with a body mass index of more than 40, presence of deep reflux, and preoperative Clinical, Etiologic, Anatomic, and Physiologic classification of 2, women had a greater periprocedural change in VCSS score than men. ConclusionsWomen benefited from endovenous ablation similarly as men, with a lower incidence of postprocedural complications. Gender may be useful for patient selection and counseling for endovenous ablation, with particular usefulness among patients with a high body mass index, presence of deep reflux, and preoperative Clinical, Etiologic, Anatomic, and Physiologic classification of 2.

Outcomes reporting in systematic reviews on vital pulp treatment: A scoping review for the development of a core outcome set.

BackgroundA large number of research reports on vital pulp treatment (VPT) has been published over the last two decades. However, heterogeneity in reporting outcomes of VPT is a significant challenge for evidence synthesis and clinical decision‐making.ObjectivesTo identify outcomes assessed in VPT studies and to evaluate how and when outcomes are measured. A subsidiary aim was to assess evidence for selective reporting bias in the included studies. The results of this review will be used to inform the development of a core outcome set (COS) for endodontic treatments.MethodsMultiple healthcare bibliographic databases, including PubMed/MEDLINE, Ovid EMBASE, Scopus, Cochrane Database of Systematic Reviews and Web of Science were searched for systematic reviews published between 1990 and 2020, reporting on VPT. Screening, data extraction and risk of bias assessment were completed independently by two reviewers. Outcomes' information was extracted and aligned with a healthcare taxonomy into five core areas: survival, clinical/physiological changes, life impact, resource use and adverse events.ResultsThirty‐six systematic reviews were included, 10 reporting on indirect pulp capping or selective caries removal, nine on direct pulp capping, eight on pulpotomy and nine on combined VPTs. There was considerable variation in the outcomes reported in these reviews and their included studies. Clinician‐reported outcomes were used considerably more often than patient‐reported outcomes. A range of instruments and time points were used for measuring outcomes. Several of the reviews were assessed as having low risk of selective reporting bias, but many did not specifically report this domain, whilst others did not provide risk of bias assessment at all.DiscussionConsiderable variation in selection of outcomes and how and when they are measured and reported was evident, and this heterogeneity has implications for evidence synthesis and clinical decision‐making.ConclusionsWhilst there is a lack of consistency, several potentially important outcomes for VPT, including pulp survival, incidence of post‐operative pain and need for further intervention, have been identified which could inform the development of a COS for endodontic treatment.RegistrationCore Outcome Measures in Effectiveness Trials (COMET) (No. 1879).

Evaluating the impact of education on clinician integration of guidelines, real-world data, and patient perspectives on gastroenteropancreatic neuroendocrine tumors (GEP-NETs).

e23005 Background: Targeted therapies for somatostatin receptor-positive NETs have seen significant developments in recent years. Approved and investigational somatostatin analogs for GEP-NETs are changing the treatment landscape for these cancers allowing for better management of difficult-to-treat heterogeneous tumors. To improve competence in applying these advances and to consider patient perspectives in treatment selection, an educational initiative was designed in collaboration with the Healing NET Foundation. Methods: A 1-hour CME activity was broadcast live-online in October 2021 and remains on-demand through October 2022 at OMedLive.com. The first module of the CME activity focused on real-world and quality-of-life data, and consensus guidelines related to somatostatin analogs (SSA) and peptide receptor radiotherapies (PRRT). The second module of the CME activity highlighted patient perspectives based on interviews of real patient with SSA and PRRT experience. This second section was distributed via social media through the Healing NET Foundation. Knowledge and competence questions were administered pre-, immediate post-, and 2 mos. post-activity. McNemar tests compared paired responses (pre/post &amp; pre/2 mos) with Cohen’s d for effect size. Results: As of 01/28/22, 238 clinicians have participated in the activity, 80% of whom are physicians, advanced practitioners (NP/PA), and nurses. Through social distribution, the patient perspectives were viewed by 300 patients/caregivers. Across the four CME test questions low baseline knowledge/competence was observed when selecting treatment with SSA based on reported progression-free survival and time to deterioration of quality of life from clinical trial data. Clinical considerations based on patient perspectives and prophylactic regimens with PRRT also revealed low baseline knowledge/competence. Statistically significant improvements were seen for pre/post paired responses across all four CME questions with gains ranging from 25% to 53%. The greatest challenge managing a patient with GEP-NET, was identified as adherence to treatment schedules. At 2-mos. follow-up, 90% and 95% reported improved behavioral impact on both clinical practice and patient experience/outcomes, respectively. Qualitative data including clinician write-in examples of behavioral impact and patient perspectives from the interviews will be shared. Conclusions: Assessments reveal a positive impact of live-online education on clinical practice when sharing patient perspectives in context of clinical updates. Open-ended responses to behavioral impact questions illustrated clear improvements in clinician-reported patient experience and outcomes, clinical practice management, and knowledge of SSA and PRRT for patients with GEP-NETs.

Abstract No. 591 Systematic review of use of patient-reported outcome measures in interventional radiology

A patient-reported outcome (PRO) is any outcome metric reported directly by the patient, in contradistinction to clinician-reported outcomes, which have traditionally dominated clinical research. This systematic review evaluates the ways in which PROs have been utilized in the interventional radiology literature.

Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis

BackgroundGeneralized myasthenia gravis (gMG) is a rare, chronic, and debilitating autoimmune disease. Activation of the complement system by autoantibodies against the postsynaptic acetylcholine receptor (AChR) leads to destruction of the postsynaptic membrane and disruption of neuromuscular transmission. This trial evaluated ravulizumab, a long-acting inhibitor of terminal complement protein C5, as a treatment for gMG.MethodsIn this randomized, double-blind, placebo-controlled, multinational trial, we randomly assigned (1:1) patients with anti-AChR antibody-positive gMG to intravenous ravulizumab or placebo for 26 weeks. Patients received a loading dose on day 1, followed by maintenance doses on day 15 and every 8 weeks thereafter. The primary end point and first secondary end point (change from baseline to week 26 in patient-reported Myasthenia Gravis–Activities of Daily Living [MG-ADL] scale and clinician-reported Quantitative Myasthenia Gravis [QMG] total scores, respectively) were compared between the ravulizumab- and placebo-treated groups.ResultsIn total, 175 patients were enrolled. Ravulizumab significantly increased the magnitude of mean changes from baseline to week 26 versus placebo in MG-ADL (−3.1 vs. −1.4; P<0.001) and QMG (−2.8 vs. −0.8; P<0.001) total scores. Improvements in both measures occurred within 1 week of ravulizumab initiation and were sustained through week 26. QMG total scores improved by 5 points or more in a significantly greater proportion of ravulizumab-treated patients than of those receiving placebo (30.0% vs. 11.3%; P=0.005). No notable differences in adverse events were observed.ConclusionsRavulizumab demonstrated rapid and sustained improvements in both patient- and clinician-reported outcomes and had a side effect and adverse-event profile that did not limit treatment in adults with anti-AChR antibody-positive gMG. (Funded by Alexion, AstraZeneca Rare Disease; ClinicalTrials.gov number, NCT03920293; EudraCT number, 2018-003243-39.)

Higher Pelvic Incidence Was Associated with a Higher Risk of Sagittal Malposition of Femoral Component and Poor Outcomes of Primary Total Knee Arthroplasty: A Retrospective Cohort Analysis.

Spine-pelvis-lower extremity sagittal alignment is regarded as a global sagittal balance. Currently, there are few studies evaluating the pelvic and femoral sagittal alignment during total knee arthroplasty (TKA). This retrospective study aims to elucidate how pelvic and femoral sagittal alignment affect clinical outcomes of primary TKA for osteoarthritis (OA) and determine the proper range of femoral sagittal alignment. Patient-reported outcome measures (PROMs), including the Knee Society Score (KSS), Western Ontario and McMaster Universities (WOMAC), and patient satisfaction scores, and clinician-reported outcomes (CROs), including range of motion (ROM) and pelvic and femoral sagittal parameters, of 67 cases were evaluated (89 knees) before and 1 year after TKA. The angle between the distal femur anterior cortex line and flange of the femoral component (FC) was defined as the α angle. Correlations between the α angle and PROM and CRO were investigated using multivariate and secondary regression analyses. Patients were further divided into four cohorts (A, B, C, and D) according to the α angle, and comparisons of their postoperative PROM and ROM scores were performed. Postoperative PROM and ROM scores improved significantly compared with the preoperative scores (p < 0.01). Only the α angle was significantly associated with postoperative knee extension among all PROM and CRO indexes (p = 0.001). Secondary regression demonstrated a convex upward function, and the scores were the highest at α angles of 0.57, 0.96, and -1.42 degrees for postoperative KSS, satisfaction, and range of knee extension, respectively (p < 0.01). However, the concave upward degree was the lowest at an α angle of 0.33 degrees for pelvic incidence (p < 0.001). Bonferroni's paired comparisons indicated that postoperative KSS and satisfaction of the cohort B (0 degrees ≤ α angle ≤ 3 degrees) were better than those of other cohorts (p < 0.0125). The results indicate that surgeons should pay more attention to the sagittal alignment of FC in patients with increased pelvic incidence, the distal femoral anterior cortex is recommended as an anatomic landmark, and 0 to 3 degrees might be "safe zones" of the sagittal flexion of FC in TKA. This study reflects the level of evidence III.

The British Orthopaedic Surgery Surveillance study: slipped capital femoral epiphysis: the epidemiology and two-year outcomes from a prospective cohort in Great Britain.

AimsThe aim of this study was to inform the epidemiology and treatment of slipped capital femoral epiphysis (SCFE).MethodsThis was an anonymized comprehensive cohort study, with a nested consented cohort, following the the Idea, Development, Exploration, Assessment, Long-term study (IDEAL) framework. A total of 143 of 144 hospitals treating SCFE in Great Britain participated over an 18-month period. Patients were cross-checked against national administrative data and potential missing patients were identified. Clinician-reported outcomes were collected until two years. Patient-reported outcome measures (PROMs) were collected for a subset of participants.ResultsA total of 486 children (513 hips) were newly affected, with a median of two patients (interquartile range 0 to 4) per hospital. The annual incidence was 3.34 (95% confidence interval (CI) 3.01 to 3.67) per 100,000 six- to 18-year-olds. Time to diagnosis in stable disease was increased in severe deformity. There was considerable variation in surgical strategy among those unable to walk at diagnosis (66 urgent surgery vs 43 surgery after interval delay), those with severe radiological deformity (34 fixation with deformity correction vs 36 without correction) and those with unaffected opposite hips (120 prophylactic fixation vs 286 no fixation). Independent risk factors for avascular necrosis (AVN) were the inability of the child to walk at presentation to hospital (adjusted odds ratio (aOR) 4.4 (95% CI 1.7 to 11.4)) and surgical technique of open reduction and internal fixation (aOR 7.5 (95% CI 2.4 to 23.2)). Overall, 33 unaffected untreated opposite hips (11.5%) were treated for SCFE by two-year follow-up. Age was the only independent risk factor for contralateral SCFE, with age under 12.5 years the optimal cut-off to define ‘at risk’. Of hips treated with prophylactic fixation, none had SCFE, though complications included femoral fracture, AVN, and revision surgery. PROMs demonstrated the marked impact on quality of life on the child because of SCFE.ConclusionThe experience of individual hospitals is limited and mechanisms to consolidate learning may enhance care. Diagnostic delays were common and radiological severity worsened with increasing time to diagnosis. There was unexplained variation in treatment, some of which exposes children to significant risks that should be evaluated through randomized controlled trials.Cite this article: Bone Joint J 2022;104-B(4):519–528.

The British Orthopaedic Surgery Surveillance study: Perthes' disease: the epidemiology and two-year outcomes from a prospective cohort in Great Britain.

AimsThe aim of this study was to evaluate the epidemiology and treatment of Perthes’ disease of the hip.MethodsThis was an anonymized comprehensive cohort study of Perthes’ disease, with a nested consented cohort. A total of 143 of 144 hospitals treating children’s hip disease in the UK participated over an 18-month period. Cases were cross-checked using a secondary independent reporting network of trainee surgeons to minimize those missing. Clinician-reported outcomes were collected until two years. Patient-reported outcome measures (PROMs) were collected for a subset of participants.ResultsOverall, 371 children (396 hips) were newly affected by Perthes’ disease arising from 63 hospitals, with a median of two patients (interquartile range 1.0 to 5.5) per hospital. The annual incidence was 2.48 patients (95% confidence interval (CI) 2.20 to 2.76) per 100,000 zero- to 14-year-olds. Of these, 117 hips (36.4%) were treated surgically. There was considerable variation in the treatment strategy, and an optimized decision tree identified joint stiffness and age above eight years as the key determinants for containment surgery. A total of 348 hips (88.5%) had outcomes to two years, of which 227 were in the late reossification stage for which a hip shape outcome (Stulberg grade) was assigned. The independent predictors of a poorer radiological outcome were female sex (odds ratio (OR) 2.27 (95% CI 1.19 to 4.35)), age above six years (OR 2.62 (95% CI (1.30 to 5.28)), and over 50% radiological collapse at inclusion (OR 2.19 (95% CI 0.99 to 4.83)). Surgery had no effect on radiological outcomes (OR 1.03 (95% CI 0.55 to 1.96)). PROMs indicated the marked effect of the disease on the child, which persisted at two years.ConclusionDespite the frequency of containment surgery, we found no evidence of improved outcomes. There appears to be a sufficient case volume and community equipoise among surgeons to embark on a randomized clinical trial to definitively investigate the effectiveness of containment surgery.Cite this article: Bone Joint J 2022;104-B(4):510–518.

Developing a core outcome set for assessing clinical safety outcomes of cardiovascular diseases in clinical trials of integrated traditional Chinese medicine and Western medicine: study protocol

BackgroundIntegrative medicine is commonly used in China. Researchers prefer to report efficacy outcomes rather than safety outcomes in clinical trials; thus, evidence regarding safety in integrative medicine is unclear. Developing a core outcome set (COS) for safety outcomes is necessary. In this study, a representative example of the methodology for developing COS to assess safety outcomes of cardiovascular diseases in clinical trials investigating integrated medicine will be developed.Methods and analysisSafety information will be extracted from package inserts and through systematic reviews of treatments for cardiovascular diseases (including angina pectoris, myocardial infarction, heart failure, arrhythmia, and hypertension) to develop an extensive list of safety outcomes, which will then be categorized according to whether subjective or objective outcomes. Questionnaires for clinician-reported safety outcomes and patient-reported safety outcomes will be developed. Two rounds of the Delphi survey will then be conducted for different stakeholders (traditional Chinese medicine clinicians and researchers in cardiovascular diseases, Western medicine clinicians and researchers in cardiovascular diseases, integrated medicine clinicians and researchers of cardiovascular diseases, pharmacologists, methodologists of evidence-based medicine, and patients). After round 2 of the Delphi analysis, a face-to-face consensus meeting will be held to determine the final COS for assessing safety outcomes in cardiovascular diseases.DiscussionA COS for safety outcomes in cardiovascular diseases may improve the consistency of reporting results and will help identify potential bias of selective reporting in the future.Trial registrationThis study was registered in the Core Outcome Measures in Effectiveness Trials database as study 1564.

Minimally invasive retroperitoneal lymph node dissection for men with testis cancer: a retrospective cohort study of safety and feasibility

To describe the perioperative safety, functional and immediate post-operative oncological outcomes of minimally invasive RPLND (miRPLND) for testis cancer. We performed a retrospective multi-centre cohort study on testis cancer patients treated with miRPLND from 16 institutions in eight countries. We measured clinician-reported outcomes stratified by indication. We performed logistic regression to identify predictors for maintained postoperative ejaculatory function. Data for 457 men undergoing miRPLND were studied. miRPLND comprised laparoscopic (n = 56) or robotic (n = 401) miRPLND. Indications included pre-chemotherapy in 305 and post-chemotherapy in 152 men. The median retroperitoneal mass size was 32mm and operative time 270min. Intraoperative complications occurred in 20 (4%) and postoperative complications in 26 (6%). In multivariable regression, nerve sparing, and template resection improved ejaculatory function significantly (template vs bilateral resection [odds ratio (OR) 19.4, 95% confidence interval (CI) 6.5-75.6], nerve sparing vs non-nerve sparing [OR 5.9, 95% CI 2.3-16.1]). In 91 men treated with primary RPLND, nerve sparing and template resection, normal postoperative ejaculation was reported in 96%. During a median follow-up of 33months, relapse was detected in 39 (9%) of which one with port site (< 1%), one with peritoneal recurrence and 10 (2%) with retroperitoneum recurrences. The low proportion of complications or peritoneal recurrences and high proportion of men with normal postoperative ejaculatory function supports further miRPLND studies.

Patient-reported outcome measures (PROMs) and palliative-care clinician reported outcomes (ClinROs) mutually improve pain and other symptoms assessment of hospitalized cancer-patients.

Patients with malignant diseases are known to have a high symptom burden including pain, and insufficient treatment of pain in this population has been frequently documented. To promote the integration of specialized palliative care and hematology and oncology, this study investigated disease, treatment, and comorbidity related symptoms as well as functional capacity and health-related quality of life (HQoL) by patient-reported outcome measures (PROMs) and clinician-reported outcome measures (ClinROs) among inpatients in a comprehensive cancer center. This cross-sectional study was carried out in a large comprehensive cancer centre of both oncological and hematological inpatients. It combined the use of PROMs and ClinROs. A high symptom burden was reported with fatigue and appetite loss as the most frequent symptoms, and role function being the most impaired function. Further, a low HQoL score was associated with a high number of symptoms/impairments. More than half of all patients reported pain in the last 24h. Out of 95 patients with average pain >0 in the last 24h, 71% were treated with opioids and 24% were treated with adjuvant analgesic (AA) defined as antiepileptics, antidepressants and prednisolone. Out of 57 patients with average pain >0 in the last24h and possible neuropathic pain, 33% were treated with AAs. A high odds ratio for moderate/severe pain in patients with possible neuropathic pain mechanisms was observed. This study did not only emphasize the need for systematic use of PROMs to identify symptoms and needs for inpatients, but also displayed why PROMs supported by ClinROs are a prerequisite to deliver truly individualized and high-quality patient-centered care. This study calls for continuous training of health care professionals to deliver high-quality treatment of pain. Further, it contributes to the growing recognition, that palliative care and standard care must be integrated to strengthen patient-centered care.

Effect of plasma rich in growth factors on quality of life following mandibular third molar removal: a double-blind randomized controlled trial

The objective of this study was to investigate the effect of plasma rich in growth factors (PRGF) on patient- and clinician-reported outcomes following mandibular third molar removal. Seventy-four patients requiring surgical removal of a unilateral impacted mandibular third molar under local anaesthesia were recruited into the study. PRGF was prepared for all patients irrespective of study arm allocation. Reviews were conducted 3 days (T1) and 7 days (T2) postoperatively. Primary outcome measures were pain (numerical rating scale, NRS), OHIP-14 (Oral Health Impact Profile-14), and postoperative symptom severity scale (PoSSe) data. Secondary outcome measures including mouth opening, dry socket, socket healing, and analgesic consumption were also explored. The statistical analysis was performed using analysis of covariance and the χ2 test. NRS pain scores were higher in the PRGF group at T1, demonstrating borderline significance (mean difference 1.0; P = 0.06), with no difference at T2. PoSSe scores did not differ between the groups, with the exception of the ‘interference with daily activities’ subscale at T1, where PRGF group patients scored 1.2 units higher (P = 0.02). OHIP-14 scores demonstrated a 25% increased likelihood of PRGF patients reporting discomfort on eating at T1 (P = 0.02), with no statistical significance at T2. Secondary outcomes did not differ between the groups. No difference in clinical or quality of life outcomes was observed for patients receiving adjunctive PRGF in third molar sockets.

Updating and Integrating Core Outcome Sets for Localised, Locally Advanced, Metastatic, and Nonmetastatic Castration-resistant Prostate Cancer: An Update from the PIONEER Consortium

ContextHarmonisation of outcome reporting and definitions for clinical trials and routine patient records can enable health care systems to provide more efficient outcome-driven and patient-centred interventions. We report on the work of the PIONEER Consortium in this context for prostate cancer (PCa). ObjectiveTo update and integrate existing core outcome sets (COS) for PCa for the different stages of the disease, assess their applicability, and develop standardised definitions of prioritised outcomes. Evidence acquisitionWe followed a four-stage process involving: (1) systematic reviews; (2) qualitative interviews; (3) expert group meetings to agree standardised terminologies; and (4) recommendations for the most appropriate definitions of clinician-reported outcomes. Evidence synthesisFollowing four systematic reviews, a multinational interview study, and expert group consensus meetings, we defined the most clinically suitable definitions for (1) COS for localised and locally advanced PCa and (2) COS for metastatic and nonmetastatic castration-resistant PCa. No new outcomes were identified in our COS for localised and locally advanced PCa. For our COS for metastatic and nonmetastatic castration-resistant PCa, nine new core outcomes were identified. ConclusionsThese are the first COS for PCa for which the definitions of prioritised outcomes have been surveyed in a systematic, transparent, and replicable way. This is also the first time that outcome definitions across all prostate cancer COS have been agreed on by a multidisciplinary expert group and recommended for use in research and clinical practice. To limit heterogeneity across research, these COS should be recommended for future effectiveness trials, systematic reviews, guidelines and clinical practice of localised and metastatic PCa. Patient summaryPatient outcomes after treatment for prostate cancer (PCa) are difficult to compare because of variability. To allow better use of data from patients with PCa, the PIONEER Consortium has standardised and recommended outcomes (and their definitions) that should be collected as a minimum in all future studies.

Comparison of Patient and Clinician Reported Outcomes Following (Chemo)Radiotherapy for Bladder Cancer

Purpose: To evaluate whether there is sufficient correlation between patient reported outcomes (PRO) and clinician reported outcomes (CRO) in bladder cancer follow-up post-radiotherapy to use solely one data method to reduce trial follow-up burden on patients, clinicians and trial programmes.

'Physio-EndEA' Study: A Randomized, Parallel-Group Controlled Trial to Evaluate the Effect of a Supervised and Adapted Therapeutic Exercise Program to Improve Quality of Life in Symptomatic Women Diagnosed with Endometriosis.

Aim: The ‘Physio-EndEA’ study aims to explore the potential benefits of a therapeutic exercise program (focused on lumbopelvic stabilization and tolerance to exertion) on the health-related quality of life (HRQoL) of symptomatic endometriosis women. Design: The present study will use a parallel-group randomized controlled trial design. Methods: A total of 22 symptomatic endometriosis women will be randomized 1:1 to the Physio-EndEA or usual care groups. The ‘Physio-EndEA’ program will consist of a one-week lumbopelvic stabilization learning phase followed by an eight-week phase of stretching, aerobic and resistance exercises focused on the lumbopelvic area that will be sequentially instructed and supervised by a trained physiotherapist (with volume and intensity progression) and adapted daily to the potential of each participant. The primary outcome measure is HRQoL. The secondary outcome measures included clinician-reported outcomes (pressure pain thresholds, muscle thickness and strength, flexibility, body balance and cardiorespiratory fitness) and patient-reported outcomes (pain intensity, physical fitness, chronic fatigue, sexual function, gastrointestinal function and sleep quality). Discussion: Findings of this study will help to identify cost-effective non-pharmacological options (such as this exercise-based intervention) that may contribute to the improvement of HRQoL in symptomatic endometriosis women.

Relevant impact of surgery on quality of life for adolescent idiopathic scoliosis

To determine the value of scoliosis surgery, it is necessary to evaluate outcomes in domains that matter to patients. Since randomized trials on adolescent idiopathic scoliosis (AIS) are scarce, prospective cohort studies with comparable outcome measures are important. To enhance comparison, a core set of patient-related outcome measures is available. The aim of this study was to evaluate the outcomes of AIS fusion surgery at two-year follow-up using the core outcomes set. AIS patients were systematically enrolled in an institutional registry. In all, 144 AIS patients aged ≤ 25 years undergoing primary surgery (median age 15 years (interquartile range 14 to 17) were included. Patient-reported (condition-specific and health-related quality of life (QoL); functional status; back and leg pain intensity) and clinician-reported outcomes (complications, revision surgery) were recorded. Changes in patient-reported outcome measures (PROMs) were analyzed using Friedman's analysis of variance. Clinical relevancy was determined using minimally important changes (Scoliosis Research Society (SRS)-22r), cut-off values for relevant effect on functioning (pain scores) and a patient-acceptable symptom state (PASS; Oswestry Disability Index). At baseline, 65 out of 144 patients (45%) reported numerical rating scale (NRS) back pain scores > 5. All PROMs significantly improved at two-year follow-up. Mean improvements in SRS-22r function (+ 1.2 (SD 0.6)), pain (+ 0.6 (SD 0.8)), and self-image (+ 1.1 (SD 0.7)) domain scores, and the SRS-22r total score (+ 0.5 (SD 0.5)), were clinically relevant. At two-year follow-up, 14 out of 144 patients (10%) reported NRS back pain > 5. Surgical site infections did not occur. Only one patient (0.7%) underwent revision surgery. Relevant improvement in functioning, condition-specific and health-related QoL, self-image, and a relevant decrease in pain is shown at two-year follow-up after fusion surgery for AIS, with few adverse events. Contrary to the general perception that AIS is a largely asymptomatic condition, nearly half of patients report significant preoperative back pain, which reduced to 10% at two-year follow-up. Cite this article: Bone Joint J2022;104-B(2):265-273.

Mobility endpoints in marketing authorisation of drugs: what gets the European medicines agency moving?

BackgroundMobility is defined as the ability to independently move around the environment and is a key contributor to quality of life, especially in older age. The aim of this study was to evaluate the use of mobility as a decisive outcome for the marketing authorisation of drugs by the European Medicines Agency (EMA).MethodsFifteen therapeutic areas which commonly lead to relevant mobility impairments and alter the quantity and/or the quality of walking were selected: two systemic neurological diseases, four conditions primarily affecting exercise capacity, seven musculoskeletal diseases and two conditions representing sensory impairments. European Public Assessment Reports (EPARs) published by the EMA until September 2020 were examined for mobility endpoints included in their ‘main studies’. Clinical study registries and primary scientific publications for these studies were also reviewed.ResultsFour hundred and eighty-four EPARs yielded 186 relevant documents with 402 ‘main studies’. The EPARs reported 153 primary and 584 secondary endpoints which considered mobility; 70 different assessment tools (38 patient-reported outcomes, 13 clinician-reported outcomes, 8 performance outcomes and 13 composite endpoints) were used. Only 15.7% of those tools distinctly informed on patients’ mobility status. Out of 402, 105 (26.1%) of the ‘main studies’ did not have any mobility assessment. Furthermore, none of these studies included a digital mobility outcome.ConclusionsFor conditions with a high impact on mobility, mobility assessment was given little consideration in the marketing authorisation of drugs by the EMA. Where mobility impairment was considered to be a relevant outcome, questionnaires or composite scores susceptible to reporting biases were predominantly used.

Guanidinoacetate–creatine in secondary progressive multiple sclerosis: a case report

Acute secondary progressive multiple sclerosis (SPMS) is characterized by escalating neurological disability, with limited disease-modifying therapeutic options. A 48-year-old woman with acute SPMS being treated with interferon beta-1a and oral corticosteroids presented as a clinical outpatient with no disease-modifying effects after treatment. A decision was made to treat her with a combination of guanidinoacetate and creatine for 21 days. She had made clinical progress at follow-up, with the intensity of fatigue dropping from severe to mild. Magnetic resonance spectroscopy revealed increased brain choline, creatine, N-acetylaspartate, and glutathione. Patients with SPMS may benefit from guanidinoacetate–creatine treatment in terms of patient- and clinician-reported outcomes; this requires additional study.

Clinical Outcome Assessment in Cancer Rehabilitation and the Central Role of Patient-Reported Outcomes.

Simple SummaryAfter completion of acute cancer treatment, it is important to support patients in recovering physically and psychologically and to help them regain their social life. This is the goal of cancer rehabilitation. If we want to know which rehabilitation interventions are helpful, we must measure their effects. This can be done by asking clinicians, testing patients’ performance, observing their behaviors, or by asking patients directly about their experience. This paper focuses on reports from the patients. We give an overview of available questionnaires and offer advice regarding their use. Furthermore, we discuss how to integrate them into clinical practice and research. The most promising way to collect such data are electronic systems, which offer many advantages. The goal of assessing the patient perspective is to help patients, clinicians, and health insurance providers to decide which rehabilitation interventions suit patients’ needs, and therefore, which ones should be chosen and reimbursed.The aim of cancer rehabilitation is to help patients regain functioning and social participation. In order to evaluate and optimize rehabilitation, it is important to measure its outcomes in a structured way. In this article, we review the different types of clinical outcome assessments (COAs), including Clinician-Reported Outcomes (ClinROs), Observer-Reported Outcomes (ObsROs), Performance Outcomes (PerfOs), and Patient-Reported Outcomes (PROs). A special focus is placed on PROs, which are commonly defined as any direct report from the patient about their health condition without any interpretation by a third party. We provide a narrative review of available PRO measures (PROMs) for relevant outcomes, discuss the current state of PRO implementation in cancer rehabilitation, and highlight trends that use PROs to benchmark value-based care. Furthermore, we provide examples of PRO usage, highlight the benefits of electronic PRO (ePRO) collection, and offer advice on how to select, implement, and integrate PROs into the cancer rehabilitation setting to maximize efficiency.

NCOG-18. RELATIONSHIP BETWEEN RANO-PRO WORKING GROUP STANDARDIZED PRIORITY CONSTRUCTS AND DISEASE PROGRESSION AMONG MALIGNANT GLIOMA PATIENTS AS MEASURED THROUGH CLINICAL OUTCOMES ASSESSMENTS

Abstract Recognizing the importance of clinical outcomes assessments (COA), the RANO-PRO Working Group recommends inclusion of core symptoms/functions in clinical care/research for malignant glioma patients. This study evaluated the association between the recommended symptoms (pain, perceived cognition, seizures, aphasia, treatment-specific symptoms) and functions (physical: weakness, walking; and role/social: work, usual activities) and disease progression in these patients. MDASI-Brain Tumor and EQ-5D-3L scores, Karnofsky Performance Status (KPS), and Neurologic Function Score (NFS) were evaluated in relation to disease progression by chi-square tests, independent- and paired-samples t-tests, adjusted for multiple comparisons. Our sample included 336 patients with malignant glioma; 82% white, 64% male, median age=52 (21-79). Imaging study revealed disease progression for 46% of patients. All symptoms except seizures and difficulty concentrating were worse in the group whose imaging showed disease progression versus stable disease, as well as the functions of walking, work, activity, and self-care (0.8 &amp;lt; difference &amp;lt; 1.8). Patients with disease progression were 4 times more likely to have a poor KPS (≤ 80) and worse NFS. Among patients with disease progression (n=112), all symptoms, except seizures, worsened from first assessment to time of progression. Up to 22% of patients reported worsening mobility, self-care, and usual activity; 46% and 35% had worsened KPS and NFS, respectively. Seven symptoms and functions were each individually reported by at least 10% of patients as having worsened the most. Worsening of symptoms and functions was not observed among patients with stable disease, except in difficulty understanding. Identified core symptoms/functions worsen at the time of progression demonstrating the relationship between priority constructs and a traditional tumor response measure while highlighting the importance of longitudinal collection of COA. The pattern of worsening was observed via both patient- and clinician-reported outcomes, emphasizing the utility of COA in clinical care and clinical trials.