Patient-reported Side Effects Research Articles

Pyridostigmine to Reduce the duration of postoperative Ileus after Colorectal surgery (PyRICo-RCT): randomized clinical trial.

Postoperative ileus, driven by the cholinergic anti-inflammatory pathway, is the most common complication in patients undergoing colorectal surgery. By inhibiting acetylcholinesterase, pyridostigmine can potentially modulate the cholinergic anti-inflammatory pathway and accelerate gastrointestinal recovery. This study aimed to assess the efficacy of pyridostigmine in improving gastrointestinal recovery after colorectal surgery. This double-blinded RCT enrolled adult patients undergoing elective colorectal surgery at two hospitals in South Australia. Patients were randomized to 60 mg oral pyridostigmine or placebo twice daily starting 6 h after surgery until the first passage of stool. The primary outcome was GI-2, a validated composite measure of time to first stool and tolerance of oral diet. Secondary outcomes included incidence of postoperative ileus (defined as GI-2 greater than 4 days), duration of hospital stay, and 30-day complications, evaluated by intention-to-treat univariate analysis. Of 130 patients recruited (mean(s.d.) age 58.4(16.4) years; 73 men, 56%), 65 were allocated to each arm. The median GI-2 was 1 day shorter with pyridostigmine compared with placebo (2 (i.q.r. 1-3) versus 3 (2-4) days; P = 0.015). However, there were no significant differences in postoperative ileus (17.2 versus 21.5%; P = 0.532) or duration of hospital stay (median 5 (i.q.r. 4-8.75) versus 5 (4-7.5) days; P = 0.921). Similarly, there were no significant differences in overall complications, anastomotic leak, cardiac complications, or patient-reported side effects. Pyridostigmine resulted in a quicker return of GI-2 and was well tolerated. Larger multicentre studies are required to determine the optimal dosing and evaluate the impact of pyridostigmine in different surgical settings. Registration number: ACTRN12621000530820 (https://anzctr.org.au).

Side Effects and Adverse Events After Treatment With Teprotumumab for Thyroid Eye Disease: A Retrospective Observational Case Series.

As the use of teprotumumab for thyroid eye disease (TED) becomes more prolific, there remains a scarcity of literature regarding the associated side effects and adverse events of teprotumumab use. The authors present a single-center retrospective, observational case review of TED patients who received at least a single dose of teprotumumab infusion at the oculofacial plastic surgery service between February 2020 and July 2023. The most predominant recollected side effects were fatigue, brittle nails, dry eye symptoms, hair loss, muscle spasms, and dry mouth. Significant adverse events were limited to two cases of a blood clot and a single case of pulmonary embolism. This is the first retrospective study of patient-reported side effects and adverse events experienced by a cohort of teprotumumab users.

Risk Factors for Early Treatment Discontinuation Due to Toxicity Among Patients With Metastatic Castration-resistant Prostate Cancer Receiving Androgen Receptor-targeted Therapy.

Androgen receptor-targeted therapies (ARTs) improve survival outcomes in patients with metastatic castration-resistant prostate cancer (mCRPC); however, a significant portion of patients discontinue treatment for various reasons including treatment-related toxicity. We aim to describe reasons for ART treatment discontinuation and identify predictors associated with increased risk of treatment discontinuation due to toxicity. We performed a single-institution retrospective review of patients with mCRPC receiving ART between 2010 and 2021. Our primary aim was to identify risk factors for treatment discontinuation due to toxicity. Our secondary aim was to describe ART discontinuation patterns among patients with mCRPC. One hundred thirty-three patients with mCRPC started and discontinued ARTs. Fourteen patients (10.5%) discontinued treatment due to toxicity. Common reasons for treatment discontinuation include Prostate Specific Antigen test progression, radiographic progression, toxicity, and death. Significant predictors of treatment discontinuation due to toxicity on bivariate analysis and multivariate analysis included patient-reported falls (odds ratio [OR]: 7.67, CI: [1.31-40.42]; P =0.016), rash (OR: 13.4, CI: [1.35-134.81]; P =0.026), and weakness (OR: 4.16, CI: [1.15-15.0]; P =0.019). Our work presents the first description of ART treatment discontinuation and its causes in the real-world setting, as well as patient-reported side effects. Most patients with mCRPC discontinued treatment due to the progression of disease and a minority of patients discontinued secondary to treatment toxicity. Initial multivariable analysis suggests that patient-reported weakness, falls, and rash were associated with a higher likelihood of treatment discontinuation due to toxicity. Early monitoring of this population can prolong the duration of treatment and prevent unnecessary treatment burden.

Patient-reported Side Effects 1 Year After Radical Prostatectomy or Radiotherapy for Prostate Cancer: A Register-based Nationwide Study

BackgroundData on functional and psychological side effects following curative treatment for prostate cancer are lacking from large, contemporary, unselected, population-based cohorts. ObjectiveTo assess urinary symptoms, bowel disturbances, erectile dysfunction (ED), and quality of life (QoL) 12 mo after robot-assisted radical prostatectomy (RARP) and radiotherapy (RT) using patient-reported outcome measures in the Swedish prostate cancer database. Design, setting, and participantsThis was a nationwide, population-based, cohort study in Sweden of men who underwent primary RARP or RT between January 1, 2018 and December 31, 2020. Outcome measurements and statistical analysisAbsolute proportions and odds ratios (ORs) were calculated using multivariable logistic regression, with adjustment for clinical characteristics. Results and limitationsA total of 2557 men underwent RARP and 1741 received RT. Men who underwent RT were older (69 vs 65 yr) and had more comorbidities at baseline. After RARP, 13% of men experienced incontinence, compared to 6% after RT. The frequency of urinary bother was similar, at 18% after RARP and 18% after RT. Urgency to defecate was reported by 14% of men after RARP and 34% after RT. At 1 yr, 73% of men had ED after RARP, and 77% after RT. High QoL was reported by 85% of men after RARP and 78% of men after RT. On multivariable regression analysis, RT was associated with lower risks of urinary incontinence (OR 0.25, 95% confidence interval [CI] 0.19–0.33), urinary bother (OR 0.79, 95% CI 0.66–0.95), and ED (OR 0.54, 95% CI 0.46–0.65), but higher risk of bowel symptoms (OR 2.86, 95% CI 2.42–3.39). QoL was higher after RARP than after RT (OR 1.34, 95% CI 1.12–1.61). ConclusionsShort-term specific side effects after curative treatment for prostate cancer significantly differed between RARP and RT in this large and unselected cohort. Nevertheless, the risk of urinary bother was lower after RT, while higher QoL was common after RARP. Patient summaryIn our study of patients treated for prostate cancer, urinary bother and overall quality of life are comparable at 1 year after surgical removal of the prostate in comparison to radiotherapy, despite substantial differences in other side effects.

Adalimumab vs placebo as add-on to Standard Therapy for autoimmune Uveitis: Tolerability, Effectiveness and cost-effectiveness—a protocol for a randomised controlled trial (ASTUTE trial)

IntroductionAdalimumab is an effective treatment for autoimmune non-infectious uveitis (ANIU), but it is currently only funded for a minority of patients with ANIU in the UK as it is restricted...

Patient-reported side effects and satisfaction of pre-hospital analgesia with low-dose esketamine: a cross-sectional study

BackgroundAnalgesia is a core intervention in emergency medicine. Pain is subjective, so patient-reported experience with pain and analgesia is essential for healthcare professionals.The aim of this study was to evaluate patient-reported side effects and satisfaction associated with pre-hospital analgesia with low-dose esketamine.MethodsThis is an observational cross-sectional study conducted as part of quality assurance measures of the German Red Cross Emergency Medical Service, Reutlingen, Germany.The survey was administered to all patients who received prehospital esketamine analgesia from paramedics. Addresses were obtained from medical records and mailed 10 days after the event. Patient feedback was anonymous and could not be linked to operational documentation.ResultsA total of 201 patients were contacted, and 119 responses were received via the online questionnaire and postal mail (response rate 59%). The mean age of the patients was 68±13 years, with 64.7% (n=77) being female. The main diagnosis reported was fractures of the extremities in 69.7%. Patients reported initial median pain intensity on a Numeric Rating Scale (NRS) of 10 [8-10]. Pain was unbearable for 96.3% of patients. After administration of analgesia, 95.3% were satisfied or very satisfied. Patients reported no side effects in 78.5%, minor side effects in 10.0%, significant but well tolerable side effects in 11.3%, borderline tolerable side effects in 0.2%, and no unbearable side effects. Borderline tolerable nausea was reported in 2% of patients along with dreams in 0.8%. No nightmares were reported. Further analysis showed that patients older than 80 years reported significantly more side effects (p < 0.001) and were thus less satisfied with the analgesia.ConclusionsBoth patient perception and analgesia with few side effects were important for both safety and satisfaction. In the present study, low-dose esketamine analgesia was associated with low side effects and high patient satisfaction.

Targeting the microenvironment in the treatment of arteriovenous malformations

Extracranial arteriovenous malformations (AVMs) are regarded as rare diseases and are prone to complications such as pain, bleeding, relentless growth, and high volume of shunted blood. Due to the high vascular pressure endothelial cells of AVMs are exposed to mechanical stress. To control symptoms and lesion growth pharmacological treatment strategies are urgently needed in addition to surgery and interventional radiology. AVM cells were isolated from three patients and exposed to cyclic mechanical stretching for 24 h. Thalidomide and bevacizumab, both VEGF inhibitors, were tested for their ability to prevent the formation of circular networks and proliferation of CD31+ endothelial AVM cells. Furthermore, the effect of thalidomide and bevacizumab on stretched endothelial AVM cells was evaluated. In response to mechanical stress, VEGF gene and protein expression increased in patient AVM endothelial cells. Thalidomide and bevacizumab reduced endothelial AVM cell proliferation. Bevacizumab inhibited circular network formation of endothelial AVM cells and lowered VEGF gene and protein expression, even though the cells were exposed to mechanical stress. With promising in vitro results, bevacizumab was used to treat three patients with unresectable AVMs or to prevent regrowth after incomplete resection. Bevacizumab controlled bleeding, pulsation, and pain over the follow up of eight months with no patient-reported side effects. Overall, mechanical stress increases VEGF expression in the microenvironment of AVM cells. The monoclonal VEGF antibody bevacizumab alleviates this effect, prevents circular network formation and proliferation of AVM endothelial cells in vitro. The clinical application of bevacizumab in AVM treatment demonstrates effective symptom control with no side effects.Graphical abstractMechanical stress increases VEGF expression in endothelial AVM cells, possibly causing the VEGF upregulation in the microenvironment of AVM cells. The resulting RAS/RAF/MEK/ERK signaling in leads to progression of fast-flow malformations. The monoclonal VEGF-A antibody bevacizumab alleviates this effect, prevents circular network formation and proliferation of AVM endothelial cells in vitro. Sporadically occurring slow-flow malformations (LMs, VMs) have mutations in TEK or PIK3CA. TEK encodes the endothelial receptor tyrosine kinase TIE2. Sporadic extracranial fast-flow malformations (AVMs) show mutations in KRAS, BRAF and MAP2K1, which encodes the dual specificity mitogen-activated protein kinase MEK1. Combined targeting of the molecular causes of the disease could be key to achieve symptom control and reduce lesion growth. Orange: gain-of-function; Blue, circled with orange: enhanced signaling.

Long-term beneficial effects of mirabegron in pediatric patients with therapy-refractory neurogenic lower urinary tract dysfunction

Neurogenic lower urinary tract dysfunction (NLUTD) in children can cause renal failure and urinary incontinence if not treated sufficiently. Antimuscarinics (AM) and intradetrusor botulinum toxin injections (BoNT-A) with clean intermittent catheterization (CIC) are widely used treatment options for children with NLUTD. However, a considerable number will become refractory to these treatment options. This study aimed to evaluate the efficacy and long-term outcomes of mirabegron in children with NLUTD as an add-on and as a stand-alone treatment. Patients under 18 years of age with NLUTD who were refractory to AM and/or BoNT-A and were treated with mirabegron 50mg were retrospectively studied. Mirabegron was either used as monotherapy or in addition to AM and/or BoNT-A. Video-urodynamic studies (VUDSs) were performed before and after treatment with mirabegron. Changes in video-urodynamic parameters, the need for other NLUTD therapy during follow-up, patient-reported side effects, and urinary incontinence were outcomes of interest. A total of 34 patients with NLUTD were included. All patients were on CIC and the median age was 13.1 years (IQR 15.9-10.3). Median follow-up was 31.4 months (IQR 57.4-11.4). Bladder compliance improved by 89.9%, from 14.9 to 28.3ml/cm H2O (p-value<0.001). Maximum cystometric capacity, end-filling detrusor pressure, volume at first detrusor overactivity, vesicoureteral reflux, and urinary incontinence significantly improved after mirabegron. The add-on therapy group showed more significant improvements in video-urodynamic outcomes compared to the monotherapy group. The median time of requiring other NLUTD therapy was 25.5 months (IQR 39.8-14.8). None of the included patients reported side effects. Mirabegron is an effective treatment for children with therapy-refractory NLUTD with an average efficacy of 2 years after which additional therapy is required. Despite the retrospective character of this study, our results confirm the beneficial effect of mirabegron in children with therapy-refractory NLUTD, in particular when mirabegron is used as add-on therapy in those with low-compliance bladders.

Safety Profile of Methotrexate in Patients with Rheumatoid Arthritis

Background: Methotrexate (MTX) is recommended as first-line therapy in patients with active Rheumatoid Arthritis (RA) as monotherapy or in combination with other disease-modifying anti-rheumatic drugs (DMARDs). Despite being considered a safe drug, some toxicities of MTX are inevitable and patients discontinue further treatment with MTX for the same.&#x0D; Objective: To evaluate the safety profile of Methotrexate in patients with RA.&#x0D; Methodology: An observational cross sectional study was conducted at a tertiary care hospital from 1st January 2020 to 31st December 2020 at rheumatology OPD in department of medicine, KLES, Dr. Prabhakar Kore Hospital, Belagavi. 117 patients diagnosed with RA who had been undergoing methotrexate treatment for at least six months as monotherapy or in combination with other DMARDs were included. Patient demographics, disease and treatment characteristics, side effects, and blood and laboratory markers specific to RA were studied with regards to methotrexate. Continuous variables were presented as mean and standard deviation, while categorical variables were presented as frequency and percentage. Association between categorical variables was done using Fisher exact test and chi square test.&#x0D; Results: The study predominantly consisted of females (85.5%), and the mean age of patients was 47.92±13.398 years. Patient-reported side effects were seen/observed in 47% of patients. Commonly reported minor side effects were fatigue (17.1%), nausea (16.2%), anorexia (11.1%), stomatitis/oral ulcers (10.3%), epigastric burning (8.5%), hair fall (3.4%), and vomiting in 0.9% of patients. Demographic and clinical factors were not associated with side effects. There was no significant association between side effects and type of treatment.&#x0D; Conclusion: Use of methotrexate in combination with other DMARDs is safe and should be encouraged as first line treatment of RA on routine basis. However, periodic blood and laboratory monitoring along with patient follow-ups are essential for early detection of toxicities.

The Incidence and Severity of Patient-Reported Side Effects of Chemotherapy in Routine Clinical Care: A Prospective Observational Study.

Understanding patients' self-reported chemotherapy side effects is significant because it affects patients' quality of life (QOL) and compliance with treatment. Our current knowledge of chemotherapy side effects comes from available literature, whose external validity is questionable. Moreover, there are very few studies available in the literature that focus on various cancers and their associated side effects. A single-center, prospective observational study was conducted at a tertiary care center from July 2019 to July 2021. After deriving the sample size, we interviewed 76 consecutive study patients with gastric, periampullary, colorectal, and breast cancer for six months after chemotherapy initiation with a structured patient-reported outcome tool adapted in Englishand Tamil to record the side effects like diarrhea, vomiting, chest pain, constipation, dyspnea, fatigue, mucositis, and rash. The grading of symptoms was done according to the Common Terminology Criteria for Adverse Eventsversion 5.0. The frequency and prevalence of side effects were calculated as the number of patients who reported the side effect of any grade at least once during the follow-up period. The incidence rate of side effects was calculated in terms of person-time.The association between each side effect and cancer type was calculated using the chi-square test and Fisher's exact test as appropriate. Of the 77 patients in the study, 51.9% were male, 63.6% were between 40 and 60 years of age, 45.5% had stage-3 disease, and 44.2% received neoadjuvant treatment. During the six-month follow-up period, 97.4% of patients experienced at least one side effect. Fatigue was the most common side effect (87%), followed by loss of appetite (71.4%) and diarrhea (49.4%). Approximately 66.7% of patients experienced six or more side effects. There was a statistically significant difference in the frequency of side effects between cancer types. However, age, socioeconomic status, BMI, comorbidity, chemo-intent, and stage of disease did not affect the frequency of side effects. This study highlights the need to integrate patient-reported side effects into routine clinical practice. Identifying these side effects, even if they are mild in intensity, and managing them in a timely manner may improve the patient's emotional state, QOL, and compliance with chemotherapy.

E066 Using social network platforms to evaluate patient perceptions of CBD products in the UK, US, and Canada

Abstract Background/Aims The number of patients using CBD products have become increasingly popular for the treatment of several conditions including rheumatic and musculoskeletal diseases. Cannabidiol (CBD) products often claim numerous benefits to health, however there is limited scientific data relating to why patients take these products, their safety and efficacy since these products are not regulated. Social media provides a valuable platform for patients to discuss their positive/negative medication experiences and seek peer support. The aim of this study was to harnesses the benefits of using social media for research by evaluating patient perceptions of CBD products within the UK, US and Canada. Methods HealthUnlocked is one of the most popular social platforms for health across Europe with over 1.5 million users in 500 communities, covering over 250 health conditions. Over 250,000 HealthUnlocked posts between the 1st of March 2020 to 17th of September 2021 were extracted and anonymized. Posts only from users who consented to share their anonymised data for research were included. CBD specific terms were then used to filter related posts from the UK, US, and Canada for manual thematic analysis following removal of duplicates. Analysis was performed focusing on the following themes: indications of CBD products, potential benefits and limitations including patient reported side-effects associated with CBD, and concomitant treatments with CBD products. Results were then compared between country where possible. Results For this study, 339 CBD-related posts from HealthUnlocked met eligibility criteria. The most common indications for CBD products in the UK were fibromyalgia/chronic pain (29.1%), restless leg syndrome (8.5%) and rheumatoid arthritis (7.5%). In the US, the most common were anxiety/stress (23.0%), cancer (15.9%) and chronic pain (11.1%). Of all posts that discussed benefits of CBD (31.5%), these included helping with pain (47.6%), anxiety (41.1%), and insomnia relief (22.4%). Most common limitations included CBD inefficacy (41.3%) and potential drug-drug interactions (24%) including warfarin, levothyroxine and prednisolone. Patient-reported side effects included drowsiness, diarrhoea, tachycardia and possible hallucinations. Opioid analgesics were the top mentioned concomitant treatment with CBD products (14.3%), followed by antidepressants (12.8%), NSAIDs (11.2%) and corticosteroids (11.2%) across all countries. Conclusion There were differences in the indications of CBD usage between countries. UK patients reported CBD being used more in fibromyalgia/chronic pain and rheumatoid arthritis, whereas US patients used them for anxiety/depression and cancer. CBD inefficacy, drug-drug interactions and side-effects were frequently discussed. Narrative patient experiences from social media can provide new insights about unregulated products such as CBD and provide opportunities to generate new research questions that are important to patients. Disclosure S. Minhas: None. M. Jani: None.

Patient-guided dose reduction of tyrosine kinase inhibitors in chronic myeloid leukaemia (RODEO study): study protocol for a prospective, multicentre, single-arm trial

BackgroundDose reduction of tyrosine kinase inhibitors (TKI) in patients with chronic myeloid leukaemia (CML) with an optimal response to TKIs may support cost-effective medication use by maintaining therapeutic effectiveness while reducing adverse events and medication costs. As the choice for dose reduction depends on patients’ individual needs and preferences, a patient-centred approach is warranted. Therefore, a study to evaluate the effectiveness of patient-guided dose reduction in patients with CML who are in a major or deep molecular response is designed.MethodsThis study is a prospective, multicentre, single-arm study. 147 patients with CML (aged ≥ 18 years) in chronic phase, who are treated with imatinib, bosutinib, dasatinib, nilotinib or ponatinib, and have reached at least major molecular response (defined as having BCR-ABL levels < 0.1% for an uninterrupted period of 6 months) are eligible. Patients will use an online patient decision aid and a shared decision making consultation will be held, after which patients who choose to will receive a personalised, lower TKI dose. Primary outcome is the proportion of patients with intervention failure at 12 months after dose reduction, defined as patients who have restarted their initial dose due to (expected) loss of major molecular response. For this, BCR-ABL1 levels will be analysed from blood samples drawn at baseline, 6 weeks after dose reduction and 3-monthly thereafter. Secondary outcomes include the proportion of patients with intervention failure at 6 and 18 months after dose reduction. Other outcomes include differences before and after dose reduction regarding the number and severity of patient-reported side effects; quality of life; beliefs about medicines; and medication adherence. Patients’ level of decisional conflict and regret after choosing dose reduction will be assessed, as will the decisional process experienced by patients and healthcare providers.DiscussionOutcomes of this trial using a personalised approach will provide clinical and patient-reported data to guide future dose reduction of TKIs in CML. If the strategy appears to be effective, it may be implemented as another valid option to offer next to standard of care to prevent potential unnecessary exposure to higher TKI doses in this selected group of patients.Trial registrationEudraCT number 2021-006581-20.

The use of liquid formulation pentoxifylline and vitamin E in both established and as a prophylaxis for dental extractions “at risk” of osteoradionecrosis

Osteoradionecrosis (ORN) of the jaws remains one of the most debilitating complications of radiotherapy (RT) in patients with head and neck cancer (HNC). Liquid pentoxifylline and vitamin E (PVe) presents an alternative formulation to tablets for patients with dysphagia or enteric feeding. This study aimed to assess the clinical outcomes of using a liquid formulation of PVe for both established ORN and as a prophylaxis to avoid its occurrence after dental extractions. A secondary objective was to determine patient-reported side effects in relation to the liquid formulation of PVe. The clinical records of 111 patients with HNC who were prescribed liquid PVe were reviewed retrospectively (66 with established ORN and 45 as prophylaxis before an invasive dental procedure). In established ORN, 44% healed, and 41% were stable. In the prophylaxis group, 96% of surgical sites healed completely, with 4% (n=2) developing ORN. Most patients (89%) were able to tolerate liquid PVe. Of the 11% (n=12) who could not tolerate this regime, the most commonly reported side effect was gastric irritation (n=5/12), whereas no more than 1 patient reported dizziness, malaise, and bleeding. This retrospective review suggests that liquid PVe is efficacious for both established ORN and as a prophylaxis. Side effects reported were similar to those recognized for the tablet formulation.

Direct-to-Consumer Telemedicine Practices in the Health and Fertility of Men: A Systematic Review of the Literature.

Men are increasingly turning toward online direct-to-consumer (DTC) men's health platforms to fulfill their health needs. Research surrounding these platforms is lacking and the motivations and predictors underlying this online health-seeking behavior is largely unknown. This review scopes the existing literature concerning DTC men's health and identifies factors influencing engagement, as well as health outcomes of this platform. A structured search was performed following PRISMA guidelines. CINAHL via EBSCO, Embase, MEDLINE via Ovid, PsycINFO, PubMed and Web of Science were searched. Peer-reviewed quantitative and qualitative studies with a focus on demographics and characteristics of those using DTC men's health platforms, as well as studies related to patient outcomes using such platforms, were included. Ten of the 3,003 studies identified met the inclusion and exclusion criteria. Four cross-sectional descriptive studies evaluated the motivations behind men's engagement with DTC platforms. Convenience, embarrassment and health motivation were identified as predominant factors associated with DTC platform use. The review identified a lack of qualitative studies, and major limitations were noted in the quantitative studies that impacted the accuracy of findings. Six further quantitative studies explored the quality of care provided by DTC platforms. DTC platforms were found to have a varying level of adherence to established clinical guidelines, but appeared to provide satisfactory patient outcomes with low levels of patient-reported side effects and adverse events. There is a lack of research within the DTC men's health space given the infancy of the field. Important predictors and motivations underlying men's choices in accessing these platforms have been noted across several studies. However, further studies need to be conducted to investigate the psychosocial underpinnings of this behavior. Studies across a wider variety of male health conditions treated by these platforms will also help to provide insights to guide patient-centered care within the DTC landscape.

Patient-reported experiences with side effects of kidney cancer therapies and corresponding information flow

BackgroundTreatment options for metastatic renal cell carcinoma (mRCC) have improved over recent years. Various therapies for metastatic renal cell carcinoma are currently approved for first and successive lines. Having various treatment options makes it important to reflect how patients experience side effects in the real-world setting. So far, data on the side effects of these treatments have only been collected within clinical trials, and have been mostly assessed by the investigator and not as patient-reported outcomes. Our aim was to determine patient-reported experiences of side effects in the real-world setting and to evaluate the doctor-patient communication regarding side effects.Data were collected via an anonymous, voluntary online survey given to members of a support group for RCC; the questionnaire was completed by 104 mRCC patients.Results89.1% of participants were suffering from side effects of any grade. These appeared to be higher for patients treated with tyrosine kinase inhibitors compared to those treated with immune-checkpoint inhibitors (98.4% vs. 68.4%). However, information on side effects is scarce: 4.0% had never heard anything about them while only 18.8% of participants received detailed information on possible side effects. Although 85.6% of participants reported side effects to their physician, 34.6% did not encounter an improvement. Limitations of the study include the design as an online questionnaire and the small sample, consisting only of members of a support group.ConclusionsDifferences can be seen between patient-reported side effects within our survey and those based on clinical trials. A shift towards more patient-reported outcomes is needed. In addition, patients seeking the advice of their physician on side effects are in need of more—or better—information and support.

Lactobacillus ruteri compared with placebo as an adjuvant in quadruple therapy for Helicobacter pylori eradication: A randomized, double-blind, controlled trial

Background and study aimsThe aim of this study was to evaluate the efficacy of probiotic as an adjuvant in quadruple therapy for H. pylori eradication compared with placebo. Patients and methodsThis randomized, double-blind, controlled trial was conducted on 450 patients with confirmed H. pylori infection. We randomly allocated patients (1:1) to receive probiotic (Lactobacillus ruteri, 100 mg) or placebo as an adjuvant in quadruple therapy with bismuth subcitrate 240 mg, pantoprazole 40 mg, amoxicillin 1000 mg and clarithromycin 500 mg all twice daily for 14 days. The primary outcome of the study was the H. pylori eradication rate at eight weeks after the end of treatment using 14C-urea breath test. The secondary endpoint of study was patient-reported side effects of drugs. The study protocol was approved by the Iranian Registry of Clinical Trials (IRCT20080901001155N32). ResultsH. pylori eradication rates were higher in the probiotic group, compared to placebo, but this difference was not statistically significant, according to both intention-to-treat (78.7 % (95 % CI; 71.24–86.16) versus 72 % (95 % CI; 64.9–79.1), respectively) and per-protocol (80.1 % (95 % CI; 73.7 – 86.5) versus 75.2 %(95 % CI; 68.4 – 82), respectively). About 69.7 % of patients in the probiotic group experience side effects compared to 98.6 % in the placebo group that was statistically significant (P-value < 0.001). Headache and all gastrointestinal adverse events except constipation were significantly lower in the probiotic group compared to placebo (P-value < 0.001). ConclusionAdding Lactobacillus ruteri to quadruple therapy did not significantly improve the eradication of H. pylori. However it reduced the frequency of drug-associated side effects.

Study protocol of the BASINEL Study: a pragmatic randomised controlled trial investigating treatment versus no treatment of low-risk basal cell carcinomas in older persons

IntroductionBasal cell carcinomas (BCCs) represent 70% of all skin cancers. These tumours do not metastasise but are locally invasive if left untreated. There is a high incidence of BCC in...

Minimal Patient-Reported Side Effects for a Chemoablative Gel (UGN-102) Used as Frontline Treatment in Adults with Nonmuscle-Invasive Bladder Cancer

Optima II ("OPTimized Instillation of Mitomycin for Bladder Cancer Treatment," clinicaltrials.gov: NCT03558503) was a phase 2b trial evaluating a nonsurgical alternative as a primary treatment for nonmuscle-invasive bladder cancer (NMIBC). Patients received 6 weekly instillations of UGN-102, a mitomycin-containing reverse thermal gel. This is the first study to report on patient-reported side effects of UGN-102. Sixty-three patients enrolled in Optima II from 20 sites. Of these 63 patients, 44 were in the cohort completing a quarterly patient-reported outcome measure assessing side effects. Changes in side effects were evaluated using the Wilcoxon signed-rank test. Associations of 3-month outcomes with demographic and clinical characteristics were examined with regression, controlling for baseline values. Ten of 44 patients (23%) were interviewed after the trial to understand tolerability for future patients making treatment decisions. Transcripts were double-coded using standard methods. In the patient-reported outcome measure cohort (44), 61% were men, 57% aged 65+ years and 89% were non-Hispanic White. UGN-102 did not cause decrements in patient-reported urinary symptoms, bloating/flatulence or malaise at the primary endpoint of 3 months. Sexual function mildly worsened. Future health worries improved. Demographics were not correlated with changes. Clinically, sexual function was correlated with new NMIBC and bloating/flatulence was associated with transurethral resection of bladder tumor within 12 months. In interviews, patients appreciated a nonsurgical alternative, would recommend the gel to other patients and would choose the gel over surgery. A nonsurgical, chemoablative gel (UGN-102) used as a primary treatment for NMIBC offers a more patient-centered therapeutic approach than standard treatments.

Efficacy and safety of isotretinoin in comparison to methotrexate in the patients suffering from moderate-to-severe plaque psoriasis: A prospective cohort study

Background: Psoriasis is a common dermatological disorder with both inflammatory and genetic etiology. In India, the incidence of psoriasis is on the verge of rise. Very less data is available on the efficacy of isotretinoin versus methotrexate in patients suffering from plaque psoriasis. Aims and Objective: In this prospective cohort study, we aimed at comparing the efficacy and safety of systemic isotretinoin with systemic methothotrexate in patients suffering from moderate to severe plaque psoriasis in a tertiary care medical college hospital in eastern India. Materials and Methods: It was a prospective cohort study conducted in the dermatology and pharmacology department of Burdwan Medical College between December 1, 2020, and August 31, 2021, on 60 patients suffering from moderate to severe plaque psoriasis. Patients receiving methotrexate and isotretinoin as systemic therapy of psoriasis from the dermatology outpatient department at the time of the study constituted the methotrexate and isotretinoin group, respectively. Each group had 30 patients. Psoriasis area severity index (PASI) score and dermatology life quality index (DLQI) score were utilized for evaluation of improvement in disease severity and quality of life, respectively. Different laboratory parameters and patients reported side effects were noted for evaluation of safety. Results: Fifty-eight patients completed the study (28 patients from methotrexate group and 30 patients from isotretinoin group). Both drugs were effective in managing psoriasis. 100% patients in the methotrexate group and 89.28% patients in the isotretinoin group had reached the threshold for a minimal response (25% reduction from baseline PASI score after 12 weeks of treatment). The mean percentage reduction in PASI score was 70.23±6.78 and 52.78±7.34 in methotrexate and isotretinoin group respectively at the end of 12 weeks therapy. This difference was statistically significant. Methotrexate was more effective in improving quality of life. The mean percentage reduction in DLQI score were 60.02±5.04 and 28.49±4.84 in methotrexate and isotretinoin group respectively at the end of 12 weeks therapy. This difference was statistically significant. Isotretinoin group showed fewer patient-reported side effects and lower fluctuation of laboratory parameters. Conclusions: Methotrexate is more efficacious than isotretinoin in disease remission and improving quality of life in patients suffering from mild to moderate plaque psoriasis. Isotretinoin is safer than methotrexate.

Abstract P4-10-02: Patient-reported outcomes in EA1131: A randomized phase III trial of platinum vs. capecitabine in patients with residual triple-negative breast cancer after neoadjuvant chemotherapy

Abstract Background: Understanding health-related quality of life (HRQOL), including side effects, is critical to guide supportive care during chemotherapy. The EA1131 trial demonstrated that Platinum (Plat) was unlikely to improve outcomes compared to capecitabine (Cape) in patients with stage II-III triple-negative breast cancer (TNBC) of basal subtype and ≥1 cm residual disease after neoadjuvant chemotherapy (NAC), supporting Cape as the continued standard of care. Patient-reported outcomes (PRO) were administered as a sub-study to understand HRQOL and symptoms from the patient’s perspective. Methods: EA1131 was amended in 9/2017 to add PRO endpoints and all patients enrolled after this amendment were eligible for the PRO sub-study. The Functional Assessment of Cancer Therapy-Breast Cancer Symptom Index (FBSI) and the Functional Assessment of Cancer Therapy/Gynecologic Oncology Group Neurotoxicity Subscale (NtxS; Plat arm only) were administered at baseline (BL), cycle 3 day 1 (C3D1), and following treatment at 6 and 15 months. Due to early trial termination, the PRO sub-study target accrual (n=362) was not reached. It was hypothesized that HRQOL, assessed by the FBSI-Treatment Side Effect (TSE) subscale (range 0-16, higher score = less side effects, better HRQOL), would indicate fewer post-treatment side effects at 6 and 15 months following Plat compared to Cape. The Wilcoxon rank sum test was used to compare FBSI-TSE subscale scores and total FBSI scores (range 0-64, higher score = better overall HRQOL) between arms at BL, C3D1, 6 months and 15 months. Two-sample t-tests were used to compare change in FBSI-TSE subscale scores and total FBSI scores from BL to C3D1 between arms. Change in NtxS scores (range 0-44, higher score = less neurotoxicity) from BL to C3D1 was evaluated with the paired t-test. Analyses were exploratory and p-values &amp;lt;0.05 considered significant without multiple comparisons adjustment. Results: Of 331 patients eligible for the PRO sub-study (156 Plat arm, 175 Cape arm), 296 (89.4%) completed ≥1 PRO. Mean FBSI-TSE subscale scores were better for Cape at BL (Cape 14.5, Plat 13.9, p-value 0.02), for Plat at C3D1 (Cape 13.5, Plat 14.0, p-value 0.04), and did not differ at 6 months (Cape 14.6, Plat 14.7, p-value 0.70) or 15 months (Cape 14.9, Plat 14.5, p-value 0.44). FBSI-TSE subscale scores worsened from BL to C3D1 for Cape but not for Plat (mean change Cape -0.72, mean change Plat 0.15, p-value 0.003). FBSI-TSE subscale change scores from BL to C3D1 exceeded the threshold for clinically meaningful worsening (&amp;gt; 1.5 points) in 27% of patients on Cape and 23% of patients on Plat (p-value 0.51). Mean total FBSI scores did not differ between arms at any time (BL: Cape 50.6, Plat 49.7; C3D1: Cape 48.1, Plat 48.0; 6 months: Cape 49.9, Plat 51.1; 15 months: Cape 53.3, Plat 50.3; all p &amp;gt; 0.05). Mean change in total FBSI scores from BL to C3D1 did not differ between arms (Cape -2.20, Plat -1.83, p = 0.75). Mean (standard deviation) NtxS scores for the Plat arm were 38 (6.3), 36.1 (7.8), 36 (7.1) and 34.5 (7.9) at BL, C3D1, 6 months and 15 months, respectively. Mean NtxS score decreased (indicating worsening neurotoxicity) from BL to C3D1 (p-value 0.006). Conclusions: Despite more frequent severe toxicity by CTCAE criteria for Plat than Cape, patient-reported side effects worsened during treatment with Cape but not Plat. Overall, changes in HRQOL were small for both arms and resolved after therapy. However approximately one-fourth of patients had clinically meaningful worsening side effects on both arms. PRO-assessed neurotoxicity increased in the Plat arm. This PRO sub-study demonstrates that PROs capture toxicities beyond CTCAE criteria and provides novel data about patients’ experience during adjuvant chemotherapy following NAC for TNBC. Citation Format: Karen L Smith, Fengmin Zhao, Ingrid A Mayer, Amye J Tevaarwerk, Sofia F Garcia, Carlos L Arteaga, William F Symmans, Ben H Park, Brian L Burnette, Della F Makower, Margaret Block, Kimberly A Morley, Chirag R Jani, Craig Mescher, Shabana J Dewani, Ursa Brown-Glaberman, Lisa E Flaum, Erica L Mayer, William M Sikov, Eve T Rodler, Angela M DeMichele, Joseph A Sparano, Antonio C Wolff, Kathy D Miller, Lynne I Wagner. Patient-reported outcomes in EA1131: A randomized phase III trial of platinum vs. capecitabine in patients with residual triple-negative breast cancer after neoadjuvant chemotherapy [abstract]. In: Proceedings of the 2021 San Antonio Breast Cancer Symposium; 2021 Dec 7-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2022;82(4 Suppl):Abstract nr P4-10-02.