Patient-based Questionnaire Research Articles

Symptom content analysis of OSA questionnaires: time to identify and improve relevance of diversity of OSA symptoms?

Obstructive sleep apnea (OSA) is a heterogeneous condition covering many clinical phenotypes in terms of the diversity of symptoms. Patient-based OSA screening questionnaires used in routine practice contain significantly varying contents that can impact the reliability and validity of the screening. We investigated to what extent common patient-based OSA screening questionnaires differ or overlap in their item content by conducting a rigorous, methodical, and quantified content overlap analysis. We conducted an item content analysis of 11 OSA screening questionnaires validated in adult populations and characterized their overlap using a 4-step approach: (1) selection of OSA screening questionnaires, (2) item extraction and selection, (3) extraction of symptoms from items, and (4) assessment of content overlap with the Jaccard index (from 0, no overlap, to 1, full overlap). We extracted 72 items that provided 25 distinct symptoms from 11 selected OSA questionnaires. The overlap between them was weak (mean Jaccard index: 0.224; ranging from 0.138 to 0.329). All questionnaires contained symptoms of the "OSA symptom" dimension (eg, snoring or witnessed apneas). The STOP-BANG (0.329) and the Berlin (0.280) questionnaires exhibited the highest overlap content. Ten symptoms (40%) were investigated in only 1 questionnaire. The heterogeneity of content and the low overlap across these questionnaires reflect the challenges of screening OSA. The different OSA questionnaires potentially capture varying aspects of the disorder, with the risk of biased results in studies. Suggestions are made for better OSA screening and refinement of clinical OSA phenotypes. Gauld C, Baillieul S, Martin VP, etal. Symptom content analysis of OSA questionnaires: time to identify and improve relevance of diversity of OSA symptoms? J Clin Sleep Med. 2024;20(7):1105-1117.

Mid-term outcomes of three commonly used surgical reconstructions for scapholunate instability.

This retrospective analysis reports the mid-term (>2 years) outcomes of capsulodesis, (modified) Brunelli tenodesis and bone-ligament-bone reconstruction for scapholunate ligament instability. In total, 60 patients (64 wrists) returned the Patient-Rated Wrist Evaluation and Disabilities of the Arm, Shoulder, and Hand questionnaires. Of these participants, 42 (46 wrists) returned for a wrist examination and radiographs. In the questionnaire group, the mean follow-up for capsulodesis was 11.8 years, tenodesis 5.9 years and bone-ligament-bone 8.9 years. In the objective outcomes group, the mean follow-up was 10.2 years for capsulodesis, 5.7 for tenodesis and 8.9 for bone-ligament-bone. In all groups, some patients showed radiographic deterioration, which did not always correlate with poor functional outcome. With a mean follow-up greater than 5 years, there was no substantial difference in clinical outcomes and patient-based outcome questionnaires between capsulodesis, tenodesis or bone-ligament-bone reconstruction for the treatment of scapholunate instability. This study emphasizes the need for further research. Surgeons should use the technique that produces the fewest complications.Level of evidence III.

Conventional versus lateral fasciotomy for prevention of lateral femoral cutaneous nerve injury in the non-fan-type nerve in total hip arthroplasty with direct anterior approach.

Lateral femoral cutaneous nerve (LFCN) injury is a potential complication after the direct anterior approach for total hip arthroplasty (DAA-THA). The aim of this study was to determine how the location of the fasciotomy in DAA-THA affects LFCN injury. In this trial, 134 patients were randomized into a lateral fasciotomy (n = 67) or a conventional fasciotomy (n = 67) group. This study was a dual-centre, double-blind, prospective randomized controlled two-arm trial with parallel group design and a 1:1 allocation ratio. The primary endpoint was the presence of LFCN injury, which was determined by the presence of numbness, decreased sensation, tingling, jolt-like sensation, or pain over the lateral aspect of the thigh, excluding the surgical scar, using a patient-based questionnaire. The secondary endpoints were patient-reported outcome measures (PROMs) using the Western Ontario and McMaster Universities osteoarthritis index (WOMAC), Japanese Orthopaedic Association Hip Disease Evaluation Questionnaire (JHEQ), and the Forgotten Joint Score-12 (FJS-12). Assessments were obtained three months after surgery. The incidence of LFCN injury tended to be lower in the lateral fasciotomy group (p = 0.089). In the lateral fasciotomy group, there were no significant differences in the mean PROM scores between patients with and without LFCN injury (FJS-12: 54.42 (SD 15.77) vs 65.06 (SD 26.14); p = 0.074; JHEQ: 55.21 (SD 12.10) vs 59.72 (SD 16.50); p = 0.288; WOMAC: 82.45 (SD 6.84) vs 84.40 (SD 17.91); p = 0.728). In the conventional fasciotomy group, there were significant differences in FJS-12 and JHEQ between patients with and without LFCN injury (FJS-12: 43.21 (SD 23.08) vs 67.28 (SD 20.47); p < 0.001; JHEQ: 49.52 (SD 13.97) vs 59.59 (SD 15.18); p = 0.012); however, there was no significant difference in WOMAC (76.63 (SD 16.81) vs 84.16 (SD 15.94); p = 0.107). The incidence of LFCN injury at three months after THA was comparable between the lateral and conventional fasciotomy groups. Further studies are needed to assess the long-term effects of these approaches.

A randomized trial to evaluate the preventive effect of lafutidine on chemotherapy-induced peripheral neuropathy in patients treated with carboplatin and paclitaxel for lung cancer.

Chemotherapy-induced peripheral neuropathy (CIPN) has a significant impact on the therapeutic efficacy of chemotherapy and patients' quality of life. The aim of this study was to assess the preventive effect of lafutidine on CIPN. Patients were randomly assigned (1:1) to carboplatin and paclitaxel chemotherapy with lafutidine 10 mg twice daily (lafutidine group) or without lafutidine (control group). Peripheral neuropathy in both groups was assessed with the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 and two patient-based questionnaires, the Patient Neurotoxicity Questionnaire (PNQ) and the Functional Assessment of Cancer Therapy/Gynecologic Oncology Group-Neurotoxicity (FACT/GOG-Ntx). The primary outcome was the incidence of grade 2 or higher peripheral neuropathy in CTCAE version 5.0. The target number of cases was set at approximately 40. In total, 18 patients were screened, and 16 patients were assigned to the lafutidine group (n=9) or control group (n=7) between January 2021 and January 2023. Due to poor recruitment, the target number of cases was not reached. Grade 2 or higher neuralgia was 22.2% in the lafutidine group and 14.3% in the control group. Grade 2 or higher peripheral sensory neuropathy was 100% in the lafutidine group and 71.4% in the control group (P=0.175). Grade 3 or higher peripheral neuropathy was not detected in either group. There was no significant difference in PNQ scores between the two groups. Median FACT/GOGNtx scores after the fourth cycle tended to be lower in the lafutidine group than in the control group. There was no statistically significant difference in progression free survival (PFS) between the two groups. There were no adverse events due to lafutidine administration. Although the preventive effect of lafutidine on CIPN could not be demonstrated statistically, lafutidine FACT/GOG-Ntx scores showed a trend toward decreased neurotoxicity as chemotherapy proceeded. More reliable studies using lafutidine on the prevention of CIPN should be conducted. Japan Registry of Clinical Trials identifier: jRCTs021200031.

Prognostic Factors for the Postoperative Improvement of Spinal Cord-Related Neuropathic Pain in Patients with Degenerative Cervical Myelopathy.

The number of patients with degenerative cervical myelopathy (DCM) requiring surgical treatment has markedly increased in today's aging society. Such patients often exhibit impaired activities of daily living because of motor dysfunction as well as neuropathic pain (NeP). Although many studies have demonstrated the safety and efficacy of surgical treatment for DCM, residual postoperative NeP has not been well described. Therefore, this study aimed to identify the predictors of postoperative NeP improvement in patients with DCM. This retrospective study included 92 outpatients with postoperative chronic NeP (≥3 months) related to DCM. Data were obtained from clinical information, magnetic resonance imaging (MRI) findings, and patient-based questionnaires using the Neuropathic Pain Symptom Inventory (NPSI) and the Brief Scale for Psychiatric Problems in Orthopaedic Patients. Univariate and multivariate analyses were performed for patients with NPSI improvement rates <30% and ≥30% to identify prognostic factors. Among 92 patients, 61 (66.3%) had residual NeP, with a low improvement rate even after surgery. The independent negative prognostic factors for NeP improvement after surgery were older age at operation (odds ratio (OR): 0.932), longer symptom duration before surgery (OR: 0.589), and higher preoperative NPSI score (OR: 0.932). The cut-off value of symptom duration before surgery for postoperative NeP improvement was 1 year. By contrast, the preoperative Japanese Orthopaedic Association score and MRI findings, including signal intensity change and the degree of spinal cord compression, were not associated with postoperative NeP improvement. Moreover, even in patients with an NPSI improvement rate ≥30%, the NPSI subscores for deep pain and paresthesia/dysesthesia remained high. Discrepancies between physician- and image-based assessments and patient-based assessments were identified as factors associated with improvement in postoperative NeP. Our findings are important for both spine surgeons and patients to manage patient expectations with respect to recovery during the postoperative course.

Clinical impact of short limited lumbar fusion for adult spinal deformity with postural and radiological abnormalities

Extensive surgical spinopelvic fusion for patients with adult spinal deformity (ASD) to achieve optimal radiological parameters should be avoided. The aim of this study was to review clinical and imaging findings in patients with ASD with postural and radiological abnormalities who underwent a novel three-level limited lumbar fusion as two-stage surgery in an attempt to propose a better tolerated alternative to spinopelvic long fusion to the pelvis. The subjects were 26 patients with a minimum follow-up period of 2 years. Cobb angle, C7 sagittal vertical axis, and pelvic incidence (PI) minus lumbar lordosis (LL) were significantly improved after surgery and maintained at follow-up. Most radiological parameters were corrected with lateral interbody fusion (LIF) as the initial surgery, and few with posterior fusion. PI-LL remained high after limited lumbar fusion, but scores on patient-based questionnaires and sagittal and coronal tilt in gait analysis improved. In cases with postoperative progression of proximal junctional kyphosis (11.5%), residual L1–L2 local kyphosis after LIF was the most significant radiological feature. In some cases of ASD with postural abnormalities, short limited lumbar fusion gives sufficient postoperative clinical improvement with preservation of spinal mobility and activities of daily living. The range of fixation should be determined based on radiological parameters after LIF to avoid postoperative complications.

Lateral versus conventional fasciotomy for prevention of lateral femoral cutaneous nerve injury in total hip arthroplasty with direct anterior approach: a study protocol for a dual-center, double-blind, randomized controlled trial

BackgroundAn incision for total hip arthroplasty (THA) via the direct anterior approach (DAA) is generally made outside of the space between the sartorius and tensor fasciae latae muscles to prevent lateral femoral cutaneous nerve (LFCN) injury. Anatomical studies have revealed that the LFCN courses between the sartorius and tensor fasciae latae muscles. When the LFCN branches radially while distributing in the transverse direction from the sartorius muscle to the tensor fasciae latae muscle, it is called the fan type. Studies suggest that damage to the fan type LFCN is unavoidable during conventional fasciotomy.We previously demonstrated that injury to non-fan variation LFCN occurred in 28.6% of patients who underwent THA by fasciotomy performed 2 cm away from the intermuscular space. This suggests that the conventional approach also poses a risk of LFCN injury to non-fan variation LFCN. LFCN injury is rarely reported in the anterolateral approach, which involves incision of fascia further away than the DAA. The purpose of this study is to investigate how the position of fasciotomy in DAA affects the risk of LFCN injury.MethodsWe will conduct a prospective, randomized, controlled study. All patients will be divided into a fan variation and a non-fan variation group using ultrasonography before surgery. Patients with non-fan variation LFCN will receive conventional fasciotomy and lateral fasciotomy in the order specified in the allocation table created in advance by our clinical trial center. The primary endpoint will be the presence of LFCN injury during an outpatient visit using a patient-based questionnaire. The secondary endpoints will be assessed based on patient-reported outcomes at 3 months after surgery in an outpatient setting using the Western Ontario and McMaster Universities Osteoarthritis Index, the Japanese Orthopaedic Association Hip-disease Evaluation Questionnaire, and the Forgotten-Joint Score-12.DiscussionWe hypothesize that the incidence of LFCN injury due to DAA-THA is reduced by making the incision further away from where it is typically made in conventional fasciotomy.If our hypothesis is confirmed, it will reduce the disadvantages of DAA and improve patient satisfaction.Trial registrationUMIN Clinical Trials Registry, UMIN000035945. Registered on 20 February, 2019.

Symptom-based pharmacotherapy for neuropathic pain related to spinal disorders: results from a patient-based assessment

Existing guidelines advocate an updated therapeutic algorithm for chronic neuropathic pain (NeP), but pharmacotherapeutic management should be individualized to pain phenotypes to achieve higher efficacy. This study was aimed to evaluate the efficacy of medications, based on NeP phenotypes, and to propose symptom-based pharmacotherapy. This retrospective study was enrolled 265 outpatients with chronic NeP related to spinal disorders. The patients were classified into three groups: spinal cord-related pain, radicular pain, and cauda equina syndrome. Data were obtained from patient-based questionnaires using Neuropathic Pain Symptom Inventory (NPSI) and the Brief Scale for Psychiatric Problems in Orthopaedic Patients, and from clinical information. The proportions of patients with ≥ 30% and ≥ 50% reduction in NPSI score for each pain subtype (spontaneous pain, paroxysmal pain, evoked pain, and paresthesia/dysesthesia) and drugs were evaluated. The pain reduction rate was significantly lower in patients with spinal cord-related pain, especially for paresthesia/dysesthesia. For spinal cord-related pain, duloxetine and neurotropin had insufficient analgesic effects, whereas mirogabalin was the most effective. Pregabalin or mirogabalin for radicular pain and duloxetine for cauda equina syndrome are recommended in cases of insufficient analgesic effects with neurotropin. The findings could contribute to better strategies for symptom-based pharmacotherapeutic management.

Japanese translation and linguistic validation of King's Parkinson's Disease Pain Scale (KPPS) and King's Parkinson's Disease Pain Questionnaire (KPPQ)

Pain is one of the most frequent non-motor symptoms associated with Parkinson's disease (PD) and it has a great impact on patient's quality of life. Thus, its quantitative evaluation is critical in establishing therapeutic evidence. The King's Parkinson's Disease Pain Scale (KPPS) was introduced as a scale of pain specific to PD in 2015. As a follow-up to the evaluator-based KPPS, the patient-based questionnaire, the King's Parkinson's Disease Pain Questionnaire (KPPQ), was introduced in 2018. We developed a linguistically validated Japanese version of the KPPS and KPPQ, and the process of its construction is reported in this study.

POSA44 The Societal Impact of Multiple Sclerosis in France: Results from the Petals Study

Multiple sclerosis (MS) is a debilitating neurodegenerative disease characterized by chronic inflammation, demyelination, and neuronal loss. MS places a considerable financial burden on society. However, data quantifying the total societal burden including extra costs, forgone income and production losses endured by MS patients, their caregivers and employers in France is lacking. The present study, PETALS, was undertaken to address the existing gap and estimate the financial burden (direct and indirect) imposed by MS in the French population. Between October and November 2020, 208 French adults with a confirmed diagnosis of MS were recruited via MSCopilot®, a MS self-assessment digital tool, and several MS patients’ networks. A patient-based questionnaire consisting of 142 questions was used to retrieve patients' insights. Indirect costs were estimated using a combination of top-down and bottom-up approaches. Data on direct costs were retrieved from Assurance Maladie. Out-of-pocket expenses (OOPE) incurred by MS patients were also reported. All costs were expressed in €2020. Data from the survey were extrapolated to the overall French MS population. The societal cost of MS was estimated to be €2.7 Billion (Bn) a year in France. Direct costs amount to €1.4 Bn a year. Indirect costs amount to €1.3 Bn a year. Loss of income/costs due to reduction in work time contributed over half of the indirect cost (53.3%), followed by health-related aids/benefits (42.5%). Furthermore, OOPEs incurred by MS patients contributed to a notable proportion of the total annual costs (over €90 million). The results from the current study showed that MS not only affected the productivity and income of individuals but also the working time of caregivers. Understanding the magnitude of the socioeconomic burden posed by MS may provide valuable insights for optimizing resource allocation and devising effective disease management strategies that can reduce its impact.

The 4-Snip Ampullotomy: A New Surgical Technique for Managing Acquired Punctal Stenosis

Retrospective case note review was completed for all eyes undergoing the new 4-snip ampullotomy procedure from September 2014 - February 2017. Anatomical success was defined as patency of the lacrimal punctum on slit-lamp biomicroscopy with ability to pass a 27-gauge cannula at follow-up and functional success on symptomatic improvement as reported by the patient. A patient-based subjective questionnaire on symptom improvement and procedural experience was posted to each patient anonymously.

Symptom-based characteristics and treatment efficacy of neuropathic pain related to spinal disorders

BackgroundSymptom-based therapeutic management is required for neuropathic pain (NeP) to achieve higher treatment efficacy. In spinal disorders, which have a high prevalence of NeP, neurological symptoms are classified into myelopathy, radiculopathy, and cauda equina syndrome. The characteristics of pain and the treatment efficacy for each of these symptoms require clarification. MethodsA retrospective patient-based outcome study was conducted in 265 outpatients with chronic NeP (≥3 months) related to spinal disorders. The patients were classified into three groups according to their neurological symptoms: spinal cord-related pain, radicular pain, and cauda equina syndrome. Data were obtained from patient-based questionnaires using the Neuropathic Pain Symptom Inventory (NPSI) and the Brief Scale for Psychiatric Problems in Orthopaedic Patients (BS-POP), and from clinical information. ResultsMost of the patients with NeP had a NPSI score >10 (moderate to severe pain) and 40% had psychiatric problems. The common subtype of NeP was spontaneous pain and paresthesia/dysesthesia in patients with radicular pain and cauda equina syndrome, whereas more severe paresthesia/dysesthesia was particularly prominent in patients with spinal cord-related pain. The pain reduction rate was significantly lower in these latter patients, especially in association with residual paresthesia/dysesthesia. ConclusionsThe characteristics and treatment efficacy of NeP in patients with spinal disorders varied according to neurological symptoms. Effective treatment was difficult, especially for paresthesia/dysesthesia in patients with spinal cord-related pain. These findings enhance the understanding of the underlying mechanisms of pain and could help in design of symptom-based therapeutic management.

Symptoms of Medication Withdrawal in Parkinson's Disease: Considerations for Informed Consent in Patient-Oriented Research.

Dopamine medication withdrawal in Parkinson's disease (PD)is commonly employed in clinical practice and can be required for participation in research studies. When asked to withdraw from medications, participants often enquire as to what symptoms they should expect. This study sought to improve the informed consent process by identifying patient-reported symptoms when dopamine treatment is withheld. We also sought to provide clinical guidance regarding the extent of these symptoms and consider participant willingness to undergo these assessments. Participants were recruited from community-based PD programs and support groups in Nashville, Tennessee, USA. A patient-based questionnaire determined the frequency and severity of motor and nonmotor symptoms. The questionnaire also assessed whether patients would be willing to abstain from medication at a future date and under what circumstances. A total of 31/90 participants reported willingness to withdraw from dopaminergic medications for clinical or research purposes. Tremor, walking, and balance were the most common motor symptoms that worsened during this time. Sleep dysfunction, constipation, and tremor were noted as the most severe symptoms. Of note, 10% of participants indicated that they would not be willing to go off medications again, suggesting that a minority of patients find this to be most discomforting. When prompted for a reason why participants would be willing to come off of their medications again, "for clinical purposes" was selected the most. Study teams should list these symptoms in the applications to their institutional review board and in the informed consent to provide guidance for participants.

Assessment of patients' satisfaction following coverage of gingival recessions: Questionnaire-based case series

Background/Aim: One of the most important goals of periodontal plastic surgery constitutes the predictable root coverage. Despite the thorough documentation that several surgical approaches can be implemented for the successful treatment of gingival recession (GR), only limited papers reported data on the evaluation according to patient's opinion. Thus, the forthcoming study is aiming in the patient-based assessment of the outcome of root coverage procedures. Material and Methods: Sixteen subjects (eight male, eight female; mean age 43.75 years, range: 23-73 years) ,with twenty one GRs, constituted the sample of this study and the root coverage procedures were executed in the period from 2011 to 2019. A patient-based questionnaire was used to assess the level of subjects' concern on various aspects related to the GR, preand post-operatively. Patient's overall post-operative satisfaction was evaluated with a VAS. Results: After a period of at least 6 months of the surgical treatment, the majority of the subjects indicated that they were not concerned for all the examined criteria. The criteria with the highest improvement (reported as improvement by at least two levels of concern preand post-operatively) were the following: 1) fear to lose the involved teeth and 2) esthetics of the area of the recession defect. With regard to esthetics' criteria, the majority of the patients indicated post-operatively the examined criteria as not important. The esthetics' criteria yielding the highest improvement were: 1) position of the teeth and 2) the fact that the teeth showed longer. Mean patient's overall post-operative satisfaction was 82,3 (range: 0-100). Conclusions: The majority of the subjects were satisfied from the implemented root coverage procedures. Few studies on the international literature have evaluated patient satisfaction following therapy. Comprehensive, multi-center studies, comprised of a large sample and a using a standardized approach are needed in future research.

Adaptation to Spanish and Validation of the Rhinitis Control Assessment Test questionnaire.

The Rhinitis Control Assessment Test (RCAT) is a patient-based questionnaire that is widely used to evaluate control of rhinitis. Objective: To develop and validate a Spanish version of the RCAT (RCATe). After translation and cultural adaptation of the original RCAT, this multicenter, observational, prospective study evaluated the properties/attributes of the RCATe by assessing its validity, reliability, responsiveness, effect size, minimal important difference and cut point. The recruited sample comprised 252 allergic rhinitis (AR) patients from 27 allergy and otolaryngology departments in hospitals throughout Spain. Significant and strong correlations were found between the RCATe and the total nasal symptom score and the visual analog scale (-0.79 and -0.77, respectively; P<.0001). The RCATe revealed significant differences between patients grouped in the different categories of severity or duration of AR (P<.001). The internal consistency (Cronbach α) was good (0.84), and the test-retest reliability was moderate (0.54 evaluated by the physician and 0.49 by the patient). The responsiveness to change was high and significant for RCATe (P<.0001) and correlated linearly with the improvement in AR. The overall effect size was 1.62. The cut-off point to identify patients with adequate control of AR was >20 (area under the receiver operating characteristic curve, 0.746; sensitivity, 58.3%; specificity, 90.9%). The psychometric evaluation and validation of the RCATe indicated good reliability, validity, and responsiveness, thus suggesting that it is effective for measuring control of AR symptoms by Spanish-speaking patients.

Objective evaluation of postoperative changes in real-life activity levels in the postoperative course of lumbar spinal surgery using wearable trackers

BackgroundLumbar spinal disease causes disabilities in performing daily activities. Operative treatments are aimed at pain relief and rapid return to routine activity. Patient-based outcome measures are used to evaluate pathologies and therapeutic effects associated with lumbar spinal disease. Nevertheless, it remains unknown as to how much such treatment improves activity levels.The purpose of the current study was to measure changes in activity levels before and after lumbar spinal surgery using a wearable activity tracker and to analyze the differences between results and patient-based outcomes.MethodsSixty patients who underwent lumbar surgery were studied. The physical activity of participants was objectively evaluated using a wearable Micro-Motion logger system (Actigraph). We measured the amount of activity before and at 1, 3, 6, and 12 months after the surgery to evaluate postoperative changes. The Japanese Orthopaedic Association Back Pain Evaluation Questionnaire, Oswestry Disability Index, Roland-Morris Disability Questionnaire and visual analog scale were used to assess patient-based outcomes of pain and activities of daily living-related scores; we analyzed the relationships between scores and actual activity levels.ResultsThe amount of actual activity decreased significantly 1 month after the surgery compared to that during the preoperative period, which then improved after 3 months postoperatively (p < 0.01). Furthermore, there was a significant improvement 6 months after the surgery compared to that during the preoperative period (p < 0.05). The changes in activity for each period were strongly correlated, regardless of the period. In contrast, a significant improvement was observed at 1 month after the surgery in almost all items of the patient-based questionnaires (p < 0.05).ConclusionsThe objective activity tracker demonstrated that lumbar surgery results in the amount of activity decreasing 1 month just after surgery followed by gradual postoperative recovery within 3 months. By contrast, patient-based outcomes showed improvement in 1 month that was significantly different from the change in actual activity, indicating a gap between patient-oriented clinical scores and their actual activities.

Broadening horizons with 225Ac-DOTATATE targeted alpha therapy for gastroenteropancreatic neuroendocrine tumour patients stable or refractory to 177Lu-DOTATATE PRRT: first clinical experience on the efficacy and safety.

The objective of this study was to investigate and present the early results on the efficacy, safety, and quality of life of 225Ac-DOTATATE targeted alpha therapy (TAT) in patients with advanced, progressive, 177Lu-DOTATATE refractory, and somatostatin receptor (SSTR) expressing metastatic GEP-NETs. In this prospective study, we recruited patients with metastatic GEP-NETs who were stable or progressive disease on 177Lu-DOTATATE therapy. Systemic TAT using 225Ac-DOTATATE was performed in all the patients with 225Ac-DOTATATE (100kBq/kg body weight) at an interval of 8weeks. The primary end point was to assess the objective response (measured by RECIST 1.1 and functional M.D. Anderson criteria). The secondary end points included biochemical response assessment as per the Italian Trials in Medical Oncology (ITMO), adverse event profile as per CTCAE v5.0, and clinical response assessment by the quality of life (assessed with EORTC QLQ-GI.NET21 patient-based questionnaire). Between April 2018 and March 2019, 32 patients (17 females, 15 males, mean age 52 ± 9.2years, 35-72years) with either stable disease after completing 177Lu-DOTATATE therapy (14, 44%) or progressive disease on 177Lu-DOTATATE therapy (18, 56%) were included in the study. The morphological response was assessed in 24/32 patients that revealed partial remission in 15 and stable disease in 9. There was no documented disease progression or deaths in the median follow-up of 8months (range 2-13months). There was a significant decrease in the plasma chromogranin level post-225Ac-DOTATATE therapy (P < 0.0001). Our short-term clinical results indicate 225Ac-DOTATATE TAT as a promising treatment option which adds a new dimension in patients who are refractory to 177Lu-DOTATATE therapy or have reached the maximum prescribed cycles of 177Lu-DOTATATE therapy.

Changes in the incidence of cervical lesions owing to the development of rheumatoid arthritis treatment and the impact of cervical lesions on patients’ quality of life

Objectives: To clarify changes in the incidence of cervical lesions in rheumatoid arthritis (RA) patients with advanced treatment and the impact of cervical lesions on the patients’ quality of life (QOL).Methods: Incidence of radiographic cervical lesions in 1333 RA patients in 2015 was compared with that in our 1999 survey. The association between cervical lesions and QOL evaluated using three different patient-based questionnaires was also analyzed.Results: The incidence of atlantoaxial subluxation (AAS), vertical subluxation (VS), and subaxial subluxation (SAS) in 2015 decreased by 50%, 75%, and 5%, respectively, compared to the 1999 survey. Although QOL, evaluated using the Japanese Orthopedic Association Cervical Myelopathy Evaluation Questionnaire (JOACMEQ; specific to myelopathy), deteriorated as the cervical lesion progressed, there was no association between cervical lesion progression and QOL evaluated using the Short Form-8™ (SF-8™; comprehensive health-related QOL). Cervical lesion progression was also associated with QOL deterioration evaluated using the Health Assessment Questionnaire Disability Index (HAQ-DI; specific to RA), but age and disease duration had stronger influences.Conclusion: The incidence of cervical lesions decreased in 2015 compared to 1999. Cervical lesion progression may be associated with QOL deterioration due to myelopathy. Age and disease duration have more impact on disease-specific QOL.

Relationship between patient-based scoring systems and the activity level of patients measured by wearable activity trackers in lumbar spine disease.

To evaluate whether a relationship exists between patient-based scoring systems and the activity level of patients with low back pain (LBP) by using wearable activity trackers, and to determine whether activity level was affected by patient factors. The subjects were 66 patients with LBP. The physical activity of participants was objectively evaluated using the Micro-Motion logger (Actigraph). The activity level was analyzed with the mean active count of the proportional-integrating mode (PMAC) and zero-crossing mode. Clinical symptoms were evaluated using the Japanese Orthopaedic Association Back Pain Evaluation Questionnaire (JOABPEQ), Roland-Morris Disability Questionnaire, the Oswestry Disability Index, and visual analog scale (VAS). The relationships between each item of the patient-based questionnaire and activity level, and the influence of individual factors (age, sex, body mass index [BMI], low back pain, and muscle mass) on the activity level were evaluated. In each domain of the JOABPEQ, lumbar spine dysfunction and social life dysfunction were correlated with PMAC (r = 0.327 and 0.321, respectively). The low back pain VAS scores were correlated with PMAC (r = - 0.246). Multiple regression analysis shows that individual factors affecting the activity level of patients with LBP were sex, BMI, low back pain, and muscle mass in PMAC (p < 0.01). Some domains of the questionnaires were correlated with activity level, but others were not. Additionally, the activity level of patients with LBP was affected by sex, BMI, LBP, and skeletal muscle mass index. These slides can be retrieved under Electronic Supplementary Material.

Development of MRC Centre MRI calf muscle fat fraction protocol as a sensitive outcome measure in Hereditary Sensory Neuropathy Type 1

ObjectivesHereditary sensory neuropathy type 1 (HSN1) is a rare, slowly progressive neuropathy causing profound sensory deficits and often severe motor loss. L-serine supplementation is a possible candidate therapy but the...