scholarly journals POS0128 PHYSICIAN AND PATIENT PERCEPTIONS OF SURGICAL PROCEDURES FOR KNEE OA ACROSS JAPAN, THE US AND 5 EU COUNTRIES: RESULTS OF A REAL-WORLD STUDY

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 275.2-276
Author(s):  
N. Fukui ◽  
P. G. Conaghan ◽  
K. Togo ◽  
N. Ebata ◽  
L. Abraham ◽  
...  
Keyword(s):  
Surgical Procedures ◽  
Primary Care Physicians ◽  
Total Joint Replacement ◽  
Functional Improvement ◽  
Published Data ◽  
Knee Oa ◽  
Obesity And Diabetes ◽  
Patient Reported ◽  
The Us ◽  
Effective Option

Background:Patients with knee osteoarthritis (OA) who do not achieve adequate pain relief and functional improvement with a combination of non-pharmacologic and pharmacologic therapies are recommended an arthroplasty as an effective option to relieve severe pain and functional limitations. However, some patients are reluctant to undergo surgical interventions, and clinicians may choose to avoid or delay surgery due to safety risks and/or the financial cost. It is of interest to understand if the use and perception of surgery differs between countries, however, few published data exist.Objectives:To demonstrate how surgery and the use of surgical procedures differs across Japan, United States of America (US) and 5 major European countries (EU5) and to evaluate patient perception towards surgery.Methods:Data were drawn from the Adelphi OA Disease Specific Programme (2017-18), a point-in-time survey of primary care physicians (PCP), rheumatologists (rheums), orthopaedic surgeons (orthos) and their OA patients. Patients with physician-diagnosed knee OA were included and segmented into two categories: had previous surgery (PS) and never had surgery (NS). A Fisher’s exact test was performed on the two groups. Physicians reported on patient demographics; whether patients had undergone surgery; type of surgery; success of surgery; how success was defined; and reasons for wanting to delay surgery. Patients reported their willingness to undergo surgery; reasons for not wanting surgery; how successful their surgery was; and how they defined this success.Results:Physician/patient reported data were available for 302,230 (Japan), 527,283 (US) and 1487,726 (EU5) patients with diagnosed knee OA. Patients were categorised by their physicians as mild (40% Japan; 34% US; 24% EU5), moderate (49% Japan; 49% US; 56% EU5) or severe (9% Japan; 17% US; 19% EU5). Patients in Japan were more likely to be female (78% vs 54% US; 58% EU5), older (73 vs 65 US; 66 EU5) and have a lower BMI than patients in the US and EU5. Obesity and diabetes were much less prevalent among patients in Japan. One in ten patients in Japan had undergone a surgery (10%), far fewer than in the US (22%) or EU5 (17%). When surgery was performed, this was more likely to be a total joint replacement (TJR) in Japan, whereas in the EU and US, arthroscopic washout was more commonly performed.For over half of Japanese patients (56%), successful surgery was more likely to be defined as having no more pain (vs. 35% US; 14% EU5). Improved mobility and a reduction in pain were also commonly reported reasons. Physicians (in each region) were more likely to suggest pain reduction, rather than no pain, and improved mobility as markers of success. Patients in Japan were much more likely to say they would not agree to surgery if recommended by their doctor, or were unsure (84% vs. 68% US; 62% EU5). The main reason for patient reluctance in Japan was fear of surgery, whereas in the US and EU5 the main reason given was that surgery was not needed. This finding was also evident among physicians in Japan, who frequently reported that patient reluctance was a key reason for delaying surgery. Physicians in Japan, do however, report that patient request was one of their main triggers for recommending surgery (45% vs 20% US; 16% EU5).Conclusion:Although surgery can be an effective option for those with OA who have exhausted other treatment options, some patients are reluctant to undergo surgery out of fear, especially in Japan, possibly due to the higher patient age. Physicians aiming to delay surgery were driven by patient reluctance in Japan, whereas cost to patient was a bigger factor in the US and EU5. The higher level of TJR vs. other surgery options among patients in Japan may suggest physicians are looking for higher levels of efficacy.Disclosure of Interests:Naoshi Fukui Speakers bureau: Pfizer, Consultant of: Pfizer, Philip G Conaghan Speakers bureau: Abbvie, Novartis, Consultant of: AstraZeneca, BMS, Eli Lilly, EMD Serono, Flexion Therapeutics, Galapagos, Gilead, Novartis, Pfizer, Kanae Togo Shareholder of: Pfizer, Employee of: Pfizer, Nozomi Ebata Shareholder of: Pfizer, Employee of: Pfizer, Lucy Abraham Shareholder of: Pfizer, Employee of: Pfizer, James Jackson: None declared, Jessica Jackson: None declared, Mia Berry: None declared, Hemant Pandit Paid instructor for: Bristol Myers Squibb, Consultant of: Johnson and Johnson, Grant/research support from: GSK

scholarly journals Validation and Generalizability of Preoperative PROMIS Scores to Predict Postoperative Success in Foot and Ankle Patients

2018 ◽  
Vol 39 (7) ◽  
pp. 763-770 ◽  
Author(s):  
Michael R. Anderson ◽  
Jeff R. Houck ◽  
Charles L. Saltzman ◽  
Man Hung ◽  
Florian Nickisch ◽  
...  
Keyword(s):  
Significant Proportion ◽  
Area Under The Curve ◽  
Functional Improvement ◽  
Published Data ◽  
Foot And Ankle ◽  
Threshold Values ◽  
Meaningful Improvement ◽  
Geographic Population ◽  
Ankle Surgery ◽  
Patient Reported

Background: A recent publication reported preoperative Patient-Reported Outcomes Measurement Instrumentation System (PROMIS) scores to be highly predictive in identifying patients who would and would not benefit from foot and ankle surgery. Their applicability to other patient populations is unknown. The aim of this study was to assess the validation and generalizability of previously published preoperative PROMIS physical function (PF) and pain interference (PI) threshold t scores as predictors of postoperative clinically meaningful improvement in foot and ankle patients from a geographically unique patient population. Methods: Prospective PROMIS PF and PI scores of consecutive patient visits to a tertiary foot and ankle clinic were obtained between January 2014 and November 2016. Patients undergoing elective foot and ankle surgery were identified and PROMIS values obtained at initial and follow-up visits (average, 7.9 months). Analysis of variance was used to assess differences in PROMIS scores before and after surgery. The distributive method was used to estimate a minimal clinically important difference (MCID). Receiver operating characteristic curve analysis was used to determine thresholds for achieving and failing to achieve MCID. To assess the validity and generalizability of these threshold values, they were compared with previously published threshold values for accuracy using likelihood ratios and pre- and posttest probabilities, and the percentages of patients identified as achieving and failing to achieve MCID were evaluated using χ2 analysis. Results: There were significant improvements in PF ( P < .001) and PI ( P < .001) after surgery. The area under the curve for PF (0.77) was significant ( P < .01), and the thresholds for achieving MCID and not achieving MCID were similar to those in the prior study. A significant proportion of patients (88.9%) identified as not likely to achieve MCID failed to achieve MCID ( P = .03). A significant proportion of patients (84.2%) identified as likely to achieve MCID did achieve MCID ( P < .01). The area under the curve for PROMIS PI was not significant. Conclusions: PROMIS PF threshold scores from published data were successful in classifying patients from a different patient and geographic population who would improve with surgery. If functional improvement is the goal, these thresholds could be used to help identify patients who will benefit from surgery and, most important, those who will not, adding value to foot and ankle health care. Level of evidence: Level II, Prospective Comparative Study

scholarly journals The Effect of Ankle Arthrodesis on the Biomechanical Function of the Foot

2017 ◽  
Vol 2 (3) ◽  
pp. 2473011417S0002
Author(s):  
Michal Kozanek ◽  
James Brodsky ◽  
Justin Kane
Keyword(s):  
Kinetic Parameters ◽  
Patient Reported Outcomes ◽  
Sagittal Plane ◽  
Ankle Arthrodesis ◽  
Functional Improvement ◽  
Multiple Objective ◽  
Published Data ◽  
Kinematic Parameters ◽  
Patient Reported ◽  
End Stage

Category: Ankle, Ankle Arthritis, Basic Sciences/Biologics, Hindfoot Introduction/Purpose: Several studies on the effect on gait of total ankle arthroplasty (TAA) have demonstrated significant functional improvement post-operatively, compared to the patients’ preoperative function. While there are many post-operative studies showing abnormalities of gait in patients who have had ankle arthrodesis, there are very limited published data in ankle fusion which compare post-operative gait to the patients’ own preoperative function. Moreover, there are no published correlation of those gait changes with patient reported outcomes. Methods: Twenty-six consecutive ankle arthrodesis patients were prospectively studied, with pre- and post-operative three- dimensional gait analysis. Kinematic data were collected at 100 Hz with a twelve-camera digital Vicon motion capture system, and two OR6-5 AMTI force plates. Patients walked barefoot at a self-selected speed over a 10-meter walkway. A minimum 20 gait cycles were used for averaging and statistical analysis for temporal-spatial and kinematic parameters and a minimum of five force plate readings for kinetic parameters. Gait parameters were collected for both operated and unaffected limbs (i.e. control). Demographic data were compiled. Prospectively collected patient-reported outcomes included SF-36, VAS, AOFAS scores, which were repeated annually postoperatively. Results: Mean age was 56 (19 – 80) years, BMI 29.7 (18.6 - 45.6), and follow-up 21.9 (12 - 69) months. There were multiple objective parameters of gait which showed statistically significant functional improvements compared to preoperative performance. Temporal-spatial: Improvements were detected in walking speed, step length and cadence; single and double limb support time. Kinematic parameters: Increase in maximum plantarflexion, decrease in maximum dorsiflexion, but total sagittal range of motion was not diminished, and slightly increased (mean 1.8 degrees). The preservation of total sagittal motion after arthrodesis suggested either precise postoperative compensation by the hindfoot, or that most of the preoperative motion was already occurring in the surrounding hindfoot joints. Kinetic parameters: Improvement in ankle moment and hip power were detected. Patient reported outcomes (table) Conclusion: Ankle arthrodesis improves gait in end-stage arthritis, as demonstrated by statistically significant improvements in multiple, objective parameters. Moreover, the increase in sagittal plane motion of the hindfoot, presumed in the literature to be compensatory to the arthrodesis, was shown to be present pre-operatively, as well, suggesting it is also an effect of tibiotalar stiffness due to end-stage arthritis. Improvements in gait corresponded to clinical improvements as well, as demonstrated by improvements in patient reported outcomes in this population. Biomechanical improvements corresponded to clinical outcomes.

scholarly journals Effect of autologous adipose-derived mesenchymal stem cell therapy in the treatment of acromioclavicular joint osteoarthritis

2019 ◽  
Vol 12 (2) ◽  
pp. e227865 ◽  
Author(s):  
Julien Freitag ◽  
James Wickham ◽  
Kiran Shah ◽  
Abi Tenen
Keyword(s):  
Stem Cell ◽  
Mesenchymal Stem Cell ◽  
Upper Limb ◽  
Rating Scale ◽  
Functional Improvement ◽  
Ac Joint ◽  
Mesenchymal Stem Cell Therapy ◽  
Knee Oa ◽  
Patient Reported ◽  
Small Joint

The aim of this case report is to evaluate the efficacy of mesenchymal stem cell (MSC) therapy in the treatment of small joint osteoarthritis (OA). Acromio-clavicular (AC) joint OA is an under-diagnosed and yet frequent source of shoulder pain. MSCs have shown evidence of benefit in the treatment of knee OA. This is the first report to describe the use of MSC therapy in OA of the upper limb. A 43-year-old patient presents with painful AC joint OA and undergoes MSC therapy. The patient reported pain and functional improvement as assessed by the Disability of Arm, Shoulder and Hand Score and Numeric Pain Rating Scale. Imaging at 12 months showed structural improvement with reduction in subchondral oedema, synovitis and subchondral cysts. This case is the first to show the benefit of MSC therapy in the treatment of small joint arthropathy and also of the upper limb.Trial registration number: Australian New Zealand Clinical Trials Registry (ACTRN12617000638336).

scholarly journals AB0587 ASSESSING THE CHARACTERISTICS AND MANAGEMENT OF KNEE OSTEOARTHRITIS PATIENTS WITH HYPERTENSION BY CLINICIAN SPECIALITY

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 1330.2-1331
Author(s):  
D. Baldock ◽  
E. Baynton ◽  
C. F. Ng
Keyword(s):  
Knee Osteoarthritis ◽  
Sports Medicine ◽  
Primary Care Physicians ◽  
Bone Erosion ◽  
Holistic Treatment ◽  
Knee Oa ◽  
Patient Demographics ◽  
Medical Chart Review ◽  
Complex Patients ◽  
Minimum Number

Background:Though the pathogenesis of knee osteoarthritis (OA) is complex, patients with OA frequently have other comorbidities, including hypertension, which eludes to other considerations needed when deciding appropriate treatment management.Objectives:This study aims to examine the profiles of knee OA patients with hypertension vs. those without any comorbidities, and to elucidate key differences between these patient groups as potential areas of consideration.Methods:A multi-center, online medical chart review study of patients with OA was conducted between May – July 2020 among US rheumatologists (rheums), orthopedic surgeons (orthos), primary care physicians with a focus in sports medicine (SM PCPs), and pain specialists. Physicians recruited were screened for duration of practice in their specialty (3-50 years) and caseload (>=35 knee OA patients personally managed, at least 10 being moderate-severe). Patient charts were recorded for the next 5 eligible patients seen during the screening period. Respondents abstracted patient demographics and treatments used. Descriptive statistics were used to analyse the data.Results:260 physicians were recruited and collectively reported 796 knee OA patients; 559 were reported to experience hypertension whilst 237 were reported as not experiencing any comorbidities.Reported hypertension patients were significantly older (mean 67 vs 59 years old, respectively; p≤0.01) and weighed more (mean 82kg vs 77kg, respectively; p≤0.01) than patients without comorbidities; they were also significantly more likely to be previous smokers compared to those without comorbidities (23% vs 8%, respectively; p≤0.01). With regards to current knee OA severity, both orthos and SM PCPs reported a significantly higher proportion of hypertension patients that were deemed ‘severe’ (physician opinion) vs patients without comorbidities (orthos: 50% vs 32%, respectively; SM PCPs: 42% vs 23%, respectively; p≤0.01).Rheums and pain specialists reported greater mild opioid usage amongst hypertension patients compared to those without comorbidities (rheums: 28% vs 10%, respectively (p≤0.05); pain specialists: 40% vs 9%, respectively; (p≤0.01)); orthos and SM PCPs stated significantly greater use of corticosteroid injections amongst their reported hypertension patients vs those without comorbidities (orthos: 60% vs 41%, respectively; SM PCPs: 40% vs 19%, respectively; p≤0.01). Hypertension patients reported by orthos and SM PCPs are more likely to be considered for total knee replacement (TKR) surgery compared to those without comorbidities (orthos: 59% vs 32%, respectively; SM PCPs: 37% vs 19%, respectively; p≤0.01). Conversely, hypertension patients reported by rheums are less likely to be considered for TKR vs those without comorbidities (41% vs 18%, respectively; p≤0.05).Reported hypertension patients had a significantly higher mean Visual Analogue Scale for Pain (VAS) score than patients without comorbidities (6.6 vs 5.9, respectively; p≤0.01). A significantly higher proportion of patients with hypertension demonstrate radiographic evidence of bone erosion compared to those without comorbidities (69% vs 56%, respectively; p≤0.01).Conclusion:From the sample surveyed, knee OA patients with hypertension may require a more specific and holistic treatment approach that takes into account their CV status and managing physician specialty. Further investigation using comparator cohort is warranted.References:[1]Ipsos Osteoarthritis Therapy Monitor (May – July 2020, 260 specialists reporting on 769 knee OA patients seen in consultation, data collected online. Participating physicians were primary treaters and saw a minimum number of 35 knee OA patients). Data © Ipsos 2021, all rights reserved.[2]Ipsos Osteoarthritis Therapy Monitor (May – July 2020, 260 specialists reporting on 769 knee OA patients seen in consultation, data collected online. Participating physicians were primary treaters and saw a minimum number of 35 knee OA patients). Data © Ipsos 2021, all rights reserved.Disclosure of Interests:None declared.

scholarly journals Healthcare Provider Perspectives on Bipolar I Disorder Screening and the Rapid Mood Screener (RMS), a Pragmatic, New Tool

CNS Spectrums ◽  
2021 ◽  
Vol 26 (2) ◽  
pp. 181-183
Author(s):  
Michael E. Thase ◽  
Stephen M. Stahl ◽  
Roger S. McIntyre ◽  
Tina Matthews-Hayes ◽  
Mehul Patel ◽  
...  
Keyword(s):  
Depressive Symptoms ◽  
Nurse Practitioners ◽  
Primary Care Physicians ◽  
Screening Tool ◽  
Positive Impact ◽  
Screening Tools ◽  
Predictive Values ◽  
Bipolar I Disorder ◽  
Provider Perspectives ◽  
Patient Reported

AbstractIntroductionAlthough mania is the hallmark symptom of bipolar I disorder (BD-I), most patients initially present for treatment with depressive symptoms. Misdiagnosis of BD-I as major depressive disorder (MDD) is common, potentially resulting in poor outcomes and inappropriate antidepressant monotherapy treatment. Screening patients with depressive symptoms is a practical strategy to help healthcare providers (HCPs) identify when additional assessment for BD-I is warranted. The new 6-item Rapid Mood Screener (RMS) is a pragmatic patient-reported BD-I screening tool that relies on easily understood terminology to screen for manic symptoms and other BD-I features in <2 minutes. The RMS was validated in an observational study in patients with clinically confirmed BD-I (n=67) or MDD (n=72). When 4 or more items were endorsed (“yes”), the sensitivity of the RMS for identifying patients with BP-I was 0.88 and specificity was 0.80; positive and negative predictive values were 0.80 and 0.88, respectively. To more thoroughly understand screening tool use among HCPs, a 10-minute survey was conducted.MethodsA nationwide sample of HCPs (N=200) was selected using multiple HCP panels; HCPs were asked to describe their opinions/current use of screening tools, assess the RMS, and evaluate the RMS versus the widely recognized Mood Disorder Questionnaire (MDQ). Results were reported by grouped specialties (primary care physicians, general nurse practitioners [NPs]/physician assistants [PAs], psychiatrists, and psychiatric NPs/PAs). Included HCPs were in practice <30 years, spent at least 75% of their time in clinical practice, saw at least 10 patients with depression per month, and diagnosed MDD or BD in at least 1 patient per month. Findings were reported using descriptive statistics; statistical significance was reported at the 95% confidence interval.ResultsAmong HCPs, 82% used a tool to screen for MDD, while 32% used a tool for BD. Screening tool attributes considered to be of the greatest value included sensitivity (68%), easy to answer questions (66%), specificity (65%), confidence in results (64%), and practicality (62%). Of HCPs familiar with screening tools, 70% thought the RMS was at least somewhat better than other screening tools. Most HCPs were aware of the MDQ (85%), but only 29% reported current use. Most HCPs (81%) preferred the RMS to the MDQ, and the RMS significantly outperformed the MDQ across valued attributes; 76% reported that they were likely to use the RMS to screen new patients with depressive symptoms. A total of 84% said the RMS would have a positive impact on their practice, with 46% saying they would screen more patients for bipolar disorder.DiscussionThe RMS was viewed positively by HCPs who participated in a brief survey. A large percentage of respondents preferred the RMS over the MDQ and indicated that they would use it in their practice. Collectively, responses indicated that the RMS is likely to have a positive impact on screening behavior.FundingAbbVie Inc.

scholarly journals AB0953 CANNABINOIDS: FRIEND OR FOE OR A BYSTANDER?

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1774.2-1774
Author(s):  
N. Jain ◽  
N. Reddy ◽  
A. Moorthy
Keyword(s):  
Chronic Pain ◽  
Complementary Medicine ◽  
Health Care Professionals ◽  
Primary Care Physicians ◽  
Therapeutic Potential ◽  
Current Therapy ◽  
Common Diagnosis ◽  
Patient Reported ◽  
Ongoing Pain ◽  
Primary Physicians

Background:Cannabinoids has recently gained popularity for use in chronic pain. There is a lot of inquisitiveness among our patients wherein health care professionals are asked about its efficacy, side effects and sometimes even ask for a prescription! As there is paucity of data and research about its use in rheumatology, patient reported outcome(PROM) can guide ahead in expanding our knowledge and experience.Objectives:To study usage of cannabinoids by rheumatology patientsTo study awareness among primary physicians regarding Cannabinoid usage in rheumatology.Methods:Cross sectional survey with two arms. Arm 1 Information from patients attending tertiary rheumatology clinic,including perception regarding the use of Cannabinoids.Arm 2 consisted of collecting data via web-based survey with20-question from 100 GPs of Leicestershire. Questions on demographics, perspectives on and knowledge of cannabinoid use. Statistical analysis SPSS software.Results:Arm1 Total 102 rheumatology patients with 60%were females and 45% secondary education. 48% were unemployed. 75% Caucasians, 18% Asians. RA most common diagnosis followed by OA and FMS. 40 % depression and anxiety in addition to Rheumatic disease. 94% reported ongoing pain with 6-8 on a VAS scale. 79% were satisfied with their current therapy. 65% had heard about complementary medicine and 15% reported using cannabinoids.Most common form Cannabinoids oil 60% followed by smoking 20%. 56% reported using >3 months and majority 72% use daily. Median age 55 years. 88% users Caucasians. Mean disease duration 6.25 years among users indicates chronicity of disease has a direct proportion in usage. All users had ongoing pain of 7 on VAS. 87% believed it helps them managing pain effectively with a pain free state. On an average spends between 50-100 pounds per week. More than half believe cannabinoids should be available as a prescription drug in NHS and 30% interested to know more about it.In Arm 2 consisting of Primary care physicians, response rate 50%. Average clinical experience 5 years. Only 20% heard about usage of complementary medicine by rheumatology patient. Most replied that 10% of their patients use Cannabinoids for pain management. Most did not believe use of cannabinoids benefited the patients. Only 4% recommend its usage. 25% think it should be available as prescription. 40% experienced patients asking about cannabinoids during appointment. 88% of respondents did not know much about cannabinoid usage in rheumatology and have never prescribed it in their practice.Conclusion:Cannabinoids widely used by the rheumatology patients with PROM favouring its efficacy for control of chronic pain. Preclinical data suggest that cannabinoids might have a therapeutic potential RA1, OA, FMS2. Clinical data regarding cannabinoid treatment for rheumatic diseases are scarce, therefore, recommendations concerning cannabinoid treatment cannot be made. All patients who reported using it suffered from moderate to severe chronic pain. Thus main indication of usage was pain rather than recreational purpose. Although a small survey it clearly highlights lack of knowledge among primary physicians. These results emphasise the need for further research regarding the benefits and risks of cannabinoids in rheumatology.References:[1]RichardsonD. etal Characterisation ofthe cannabinoid receptor system in synovial tissue andfluid in patients with OA and RA Arthritis Res.Ther. 10, R43 (2008).[2]Walitt, B etal Cannabinoids for fibromyalgia. Cochrane DatabaseSyst. Rev. 7, CD011694 (2016).Disclosure of Interests:Nibha Jain: None declared, Neelima Reddy: None declared, Arumugam Moorthy Speakers bureau: Abbvie, Novartis,UCB,MSD

scholarly journals In vitro responses to platelet-rich-plasma are associated with variable clinical outcomes in patients with knee osteoarthritis

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Habib Zahir ◽  
Bijan Dehghani ◽  
Xiaoning Yuan ◽  
Yurii Chinenov ◽  
Christine Kim ◽  
...  
Keyword(s):  
Knee Osteoarthritis ◽  
Clinical Outcomes ◽  
Inflammatory Responses ◽  
Platelet Rich Plasma ◽  
Autologous Blood ◽  
Limited Information ◽  
Knee Oa ◽  
Patient Reported ◽  
Treatment Comparisons

AbstractAutologous blood-derived products such as platelet-rich plasma (PRP) are widely used to treat musculoskeletal conditions, including knee osteoarthritis (OA). However, the clinical outcomes after PRP administration are often variable, and there is limited information about the specific characteristics of PRP that impact bioactivity and clinical responses. In this study, we aimed to develop an integrative workflow to evaluate responses to PRP in vitro, and to assess if the in vitro responses to PRP are associated with the PRP composition and clinical outcomes in patients with knee OA. To do this, we used a coculture system of macrophages and fibroblasts paired with transcriptomic analyses to comprehensively characterize the modulation of inflammatory responses by PRP in vitro. Relying on patient-reported outcomes and achievement of minimal clinically important differences in OA patients receiving PRP injections, we identified responders and non-responders to the treatment. Comparisons of PRP from these patient groups allowed us to identify differences in the composition and in vitro activity of PRP. We believe that our integrative workflow may enable the development of targeted approaches that rely on PRP and other orthobiologics to treat musculoskeletal pathologies.

scholarly journals Safety and efficacy of low-dose PI3K inhibitor taselisib in adult patients with CLOVES and Klippel–Trenaunay syndrome (KTS): the TOTEM trial, a phase 1/2 multicenter, open-label, single-arm study

2021 ◽  
Author(s):  
M. Luu ◽  
P. Vabres ◽  
H. Devilliers ◽  
R. Loffroy ◽  
A. Phan ◽  
...  
Keyword(s):  
Low Dose ◽  
Phase 1 ◽  
Pi3k Inhibitor ◽  
Functional Improvement ◽  
Open Label ◽  
Tissue Volume ◽  
Pathogenic Variants ◽  
Patient Reported ◽  
Affected Tissue ◽  
Phosphoinositide 3 Kinase

ABSTRACT Purpose PIK3CA pathogenic variants in the PIK3CA-related overgrowth spectrum (PROS) activate phosphoinositide 3-kinase signaling, providing a rationale for targeted therapy, but no drug has proven efficacy and safety in this population. Our aim was to establish the six-month tolerability and efficacy of low-dose taselisib, a selective class I PI3K inhibitor, in PROS patients. Methods Patients over 16 years with PROS and PIK3CA pathogenic variants were included in a phase IB/IIA multicenter, open-label single-arm trial (six patients at 1 mg/day of taselisib, then 24 at 2 mg/day). The primary outcome was the occurrence of dose limiting toxicity (DLT). Efficacy outcomes were the relative changes after treatment of (1) tissue volume at affected and unaffected sites, both clinically and on imaging; (2) cutaneous vascular outcomes when relevant; (3) biologic parameters; (4) quality of life; and (5) patient-reported outcomes. Results Among 19 enrolled patients, 2 experienced a DLT (enteritis and pachymeningitis) leading to early trial termination (17 treated, 10 completed the study). No serious adverse reaction occurred in the 1 mg cohort (n = 6). No significant reduction in affected tissue volume was observed (mean −4.2%; p = 0.81; SD 14.01). Thirteen (76.4%) participants reported clinical improvement (pain reduction, chronic bleeding resolution, functional improvement). Conclusion Despite functional improvement, the safety profile of low-dose taselisib precludes its long-term use.

scholarly journals Linguistic validation of the simplified Chinese version of the US National Cancer Institute’s patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE™)

BMC Cancer ◽  
2020 ◽  
Vol 20 (1) ◽  
Author(s):  
Cheng KKF ◽  
S. A. Mitchell ◽  
N. Chan ◽  
E. Ang ◽  
W. Tam ◽  
...  
Keyword(s):  
Adverse Events ◽  
Cancer Patients ◽  
Patient Reported Outcomes ◽  
A Priori ◽  
Cognitive Interviews ◽  
Simplified Chinese ◽  
Patient Reported ◽  
The Us ◽  
The Common ◽  
Response Choices

Abstract Background The aim of this study was to translate and linguistically validate the U.S. National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE™) into Simplified Chinese for use in Singapore. Methods All 124 items of the English source PRO-CTCAE item library were translated into Simplified Chinese using internationally established translation procedures. Two rounds of cognitive interviews were conducted with 96 cancer patients undergoing adjuvant treatment to determine if the translations adequately captured the PRO-CTCAE source concepts, and to evaluate comprehension, clarity and ease of judgement. Interview probes addressed the 78 PRO-CTCAE symptom terms (e.g. fatigue), as well as the attributes (e.g. severity), response choices, and phrasing of ‘at its worst’. Items that met the a priori threshold of ≥20% of participants with comprehension difficulties were considered for rephrasing and retesting. Items where < 20% of the sample experienced comprehension difficulties were also considered for rephrasing if better phrasing options were available. Results A majority of PRO-CTCAE-Simplified Chinese items were well comprehended by participants in Round 1. One item posed difficulties in ≥20% and was revised. Two items presented difficulties in < 20% but were revised as there were preferred alternative phrasings. Twenty-four items presented difficulties in < 10% of respondents. Of these, eleven items were revised to an alternative preferred phrasing, four items were revised to include synonyms. Revised items were tested in Round 2 and demonstrated satisfactory comprehension. Conclusions PRO-CTCAE-Simplified Chinese has been successfully developed and linguistically validated in a sample of cancer patients residing in Singapore.

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