Addressing gaps in care for patients with rare cancers and blood disorders: The impact of a collaborative digital education initiative.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e23005-e23005
Author(s):  
Wendy Turell ◽  
Tariqa Ackbarali ◽  
Elizabeth L. del Nido ◽  
Kathryn Pucci ◽  
Randi O'Hara ◽  
...  

e23005 Background: Clinicians are uniquely challenged to manage patients with rare cancers and blood disorders due to a lack of knowledge of diagnosis and management guidelines for these conditions. These conditions comprise a larger proportion of diagnosed cancers in minority populations, among whom a worse 5-year survival is realized in adults versus adolescents and children. Patients often experience delayed diagnoses, have limited treatment options, access to specialists, and limited clinical trials. Education surrounding rare cancers and blood disorders, diagnostic criteria, approved agents, and emerging novel agents is essential to improve treatment outcomes for patients with these rare malignancies. Methods: A 2-part CE and American Board of Internal Medicine (ABIM) Maintenance of Certification activity was launched live-online in October and November, 2020, and remains on-demand through November, 2021 at OMedLive.com, comprising 9 hours of virtual education. This first annual activity was launched in partnership with National Organization for Rare Disorders (NORD), and featured key opinion leaders with extensive expertise in rare cancers and blood disorders. Session components included case presentations, downloadable resources, live polling, and audience real-time Q&A. Knowledge and competence questions were administered pre- and immediate post-activity. Patient and clinical practice impact questions were also asked at 2-month follow-up. Data from these questions were analyzed to determine engagement, remaining gaps, and the unique challenges of managing rare cancers and blood disorders. Results: 996 clinicians have participated in the activity to date. All 28 CE test questions reflected improvements in knowledge and competence regarding symptom recognition, diagnosis, clinical trial data, guidelines, treatment options, and adverse event management. At 2-mos. follow up, 71% reported improvements in clinical practice and 67% reported improvements in patient impact (n = 66). Almost half (48%) indicated diagnosis as their most significant challenge, with other noted barriers including: limited access to molecular testing (29%) and limited knowledge of tests to order (17%). The top 3 reported treatment challenges were: a lack of clinical evidence (24%), limited treatment options (22%), and high treatment cost (16%). Updated and expanded data will be shared. Conclusions: Following the targeted collaborative educational initiative, clinicians demonstrated clear knowledge and competency improvements in critical areas such as recognition, diagnosis, and management, and reported significant improvements in patient outcomes and clinical practice. The results of the two-month impact survey demonstrated a need for further tailored education on diagnostic testing and clinical evidence on treatment options for rare malignancies.

BJS Open ◽  
2021 ◽  
Vol 5 (Supplement_1) ◽  
Author(s):  
Chan Hee Koh ◽  
Danyal Z Khan ◽  
Ronneil Digpal ◽  
Hugo Layard Horsfall ◽  
Hani J Marcus ◽  
...  

Abstract Introduction The clinical practice and research in the diagnosis and management of Cushing’s disease remains heterogeneous and challenging to this day. We sought to establish the characteristics of Cushing’s disease, and the trends in diagnosis, management and reporting in this field. Methods Searches of PubMed and Embase were conducted. Study protocol was registered a-priori. Random-effects analyses were conducted to establish numerical estimates. Results Our screening returned 159 papers. The average age of adult patients with Cushing’s disease was 39.3, and 13.6 for children. The male:female ratio was 1:3. 8% of patients had undergone previous transsphenoidal resection. The ratio of macroadenomas: microadenomas:imaging-undetectable adenomas was 18:53:29. The most commonly reported preoperative biochemical investigations were serum cortisol (average 26.4µg/dL) and ACTH (77.5pg/dL). Postoperative cortisol was most frequently used to define remission (74.8%), most commonly with threshold of 5µg/dL (44.8%). Average remission rates were 77.8% with recurrence rate of 13.9%. Median follow-up was 38 months. Majority of papers reported age (81.9%) and sex (79.4%). Only 56.6% reported whether their patients had previous pituitary surgery. 45.3% reported whether their adenomas were macroadenoma, microadenoma or undetectable. Only 24.1% reported preoperative cortisol, and this did not improve over time. 60.4% reported numerical thresholds for cortisol in defining remission, and this improved significantly over time (p = 0.004). Visual inspection of bubbleplots showed increasing preference for threshold of 5µg/dL. 70.4% reported the length of follow up. Conclusion We quantified the characteristics of Cushing’s disease, and analysed the trends in investigation and reporting. This review may help to inform future efforts in forming guidelines for research and clinical practice.


2021 ◽  
Vol 8 (1) ◽  
pp. e000840
Author(s):  
Lianne Parkin ◽  
Sheila Williams ◽  
David Barson ◽  
Katrina Sharples ◽  
Simon Horsburgh ◽  
...  

BackgroundCardiovascular comorbidity is common among patients with chronic obstructive pulmonary disease (COPD) and there is concern that long-acting bronchodilators (long-acting muscarinic antagonists (LAMAs) and long-acting beta2 agonists (LABAs)) may further increase the risk of acute coronary events. Information about the impact of treatment intensification on acute coronary syndrome (ACS) risk in real-world settings is limited. We undertook a nationwide nested case–control study to estimate the risk of ACS in users of both a LAMA and a LABA relative to users of a LAMA.MethodsWe used routinely collected national health and pharmaceutical dispensing data to establish a cohort of patients aged >45 years who initiated long-acting bronchodilator therapy for COPD between 1 February 2006 and 30 December 2013. Fatal and non-fatal ACS events during follow-up were identified using hospital discharge and mortality records. For each case we used risk set sampling to randomly select up to 10 controls, matched by date of birth, sex, date of cohort entry (first LAMA and/or LABA dispensing), and COPD severity.ResultsFrom the cohort (n=83 417), we identified 5399 ACS cases during 281 292 person-years of follow-up. Compared with current use of LAMA therapy, current use of LAMA and LABA dual therapy was associated with a higher risk of ACS (OR 1.28 (95% CI 1.13 to 1.44)). The OR in an analysis restricted to fatal cases was 1.46 (95% CI 1.12 to 1.91).ConclusionIn real-world clinical practice, use of two versus one long-acting bronchodilator by people with COPD is associated with a higher risk of ACS.


2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 733.2-734
Author(s):  
I. Flouri ◽  
N. Kougkas ◽  
N. Avgustidis ◽  
A. Repa ◽  
A. Eskitzis ◽  
...  

Background:Long-term observational studies of patients under biologic disease-modifying anti-rheumatic drug (bDMARD) therapies in routine clinical practice can provide us with important data regarding patients with comorbidities, who are usually excluded from randomized controlled studies.Objectives:To study the impact of comorbidities in the outcome (response and persistence to therapy) of patients with spondyloarthritis (SpA) receiving bDMARDs in real-world clinical practice.Methods:Prospective study of all patients who start a bDMARD in a tertiary centre University Hospital after their consent. All patient comorbidities [among a list of approximately 100 pre-specified major comorbidities] are registered by treating physicians at baseline and during follow-up.Comorbidities were studied as total Comorbidities Count (CC) and rheumatic disease comorbidity index (RDCI). Statistical analyses were performed using logistic and Cox regression models, adjusting for the potential confounding of age, sex, disease duration, diagnosis (axial vs. peripheral SpA), number of previous conventional synthetic and biologic DMARDs, year of therapy start, and co-administered methotrexate and corticosteroids (yes/no). Analyses of response to therapy also included baseline BASDAI or ASDAS indices as confounding variables.Results:A total of 603 biologic treatments (1st: 298, 2nd: 157, ≥3rd: 148) were analyzed. Half (51%) of the patients were female, 413 patients had axial SpA (AxSpA) and 190 peripheral SpA (perSpA). At baseline, median (IQR) age: 48 (38-57) years, disease duration: 11 (4-19) years, CC: 2 (1-4) and RDCI: 1 (0-2). Both comorbidity indices were significantly higher in perSpA compared to AxSpA (p<0.001).At 6 months of therapy, 31% of patients with AxSpA achieved BASDAI50 and 39% had ASDAS-ESR < 2.1. Higher CC was an independent predictor of insufficient response according to BASDAI50 [OR (95%) = 0.70 (0.52-0.94), p=0.019] and higher RDCI was predicting failure to achieve ASDAS-ESR < 2.1 [OR (95%) = 0.59 (0.37-0.94), p=0.027]. Other independent predictors of non-response were age, longer disease duration and (for ASDAS-ESR<2.1) higher baseline disease activity.During 1405 patient-years of follow-up, 349 (58%) treatments were discontinued. The adjusted hazard ratio for bDMARD discontinuation within the first 2 years of treatment due to insufficient response was doubled in patients with CC ≥2 versus those with CC ≤1 [HR = 2.27 (1.14-4.53), p=0.020] or with RDCI ≥1 (vs. RDCI = 0) [HR = 2.23 (1.22-4.07), p=0.009]. Comorbidities’ indices were not significant predictors of treatment discontinuations due to adverse events.Conclusion:The presence of comorbidities in patients with SpA is an independent predictor for insufficient 6-month response to bDMARDs and resultant treatment discontinuation due to failure.Acknowledgements:This research is co-financed by Greece and the European Union (European Social Fund- ESF) through the Operational Programme «Human Resources Development, Education and Lifelong Learning» in the context of the project “Reinforcement of Postdoctoral Researchers - 2nd Cycle” (MIS-5033021), implemented by the State Scholarships Foundation (ΙΚΥ).Disclosure of Interests:None declared


2021 ◽  
Vol 39 (28_suppl) ◽  
pp. 14-14
Author(s):  
Charu Aggarwal ◽  
Melina Elpi Marmarelis ◽  
Wei-Ting Hwang ◽  
Dylan G. Scholes ◽  
Aditi Puri Singh ◽  
...  

14 Background: Current NCCN guidelines recommend comprehensive molecular profiling for all newly diagnosed patients with metastatic non-squamous NSCLC to enable the delivery of personalized medicine. We have previously demonstrated that incorporation of plasma based next-generation gene sequencing (NGS) improves detection of clinically actionable mutations in patients with advanced NSCLC (Aggarwal et al, JAMA Oncology, 2018). To increase rates of comprehensive molecular testing at our institution, we adapted our clinical practice to include concurrent use of plasma (P) and tissue (T) based NGS upon initial diagnosis. P NGS testing was performed using a commercial 74 gene assay. We analyzed the impact of this practice change on guideline concordant molecular testing at our institution. Methods: A retrospective cohort study of patients with newly diagnosed metastatic non-squamous NSCLC following the implementation of this practice change in 12/2018 was performed. Tiers of NCCN guideline concordant testing were defined, Tier 1: complete EGFR, ALK, BRAF, ROS1, MET, RET, NTRK testing, Tier 2: included above, but with incomplete NTRK testing, Tier 3: > 2 genes tested, Tier 4: single gene testing, Tier 5: no testing. Proportion of patients with comprehensive molecular testing by modality (T NGS vs. T+P NGS) were compared using one-sided Fisher’s exact test. Results: Between 01/2019, and 12/2019, 170 patients with newly diagnosed metastatic non-Sq NSCLC were treated at our institution. Overall, 98.2% (167/170) patients underwent molecular testing, Tier 1: n = 100 (59%), Tier 2: n = 39 (23%), Tier 3/4: n = 28 (16.5%), Tier 5: n = 3 (2%). Amongst these patients, 43.1% (72/167) were tested with T NGS alone, 8% (15/167) with P NGS alone, and 47.9% (80/167) with T+P NGS. A higher proportion of patients underwent comprehensive molecular testing (Tiers 1+2) using T+P NGS: 95.7% (79/80) compared to T alone: 62.5% (45/72), p < 0.0005. Prior to the initiation of first line treatment, 72.4% (123/170) patients underwent molecular testing, Tier 1: n = 73 (59%), Tier 2: n = 27 (22%) and Tier 3/4: n = 23 (18%). Amongst these, 39% (48/123) were tested with T NGS alone, 7% (9/123) with P NGS alone and 53.6% (66/123) with T+P NGS. A higher proportion of patients underwent comprehensive molecular testing (Tiers 1+2) using T+P NGS, 100% (66/66) compared to 52% (25/48) with T NGS alone (p < 0.0005). Conclusions: Incorporation of concurrent T+P NGS testing in treatment naïve metastatic non-Sq NSCLC significantly increased the proportion of patients undergoing guideline concordant molecular testing, including prior to initiation of first-line therapy at our institution. Concurrent T+P NGS should be adopted into institutional pathways and routine clinical practice.


2018 ◽  
Vol 4 (4) ◽  
pp. 190-195 ◽  
Author(s):  
Owain Michael Leng ◽  
Charlotte Rothwell ◽  
Annamarie Buckton ◽  
Catherine Elmer ◽  
Jan Illing ◽  
...  

BackgroundThe patient safety agenda has propelled the rise of simulation education, but relatively few evaluations of simulation-based educational interventions have focused on patient outcomes.ObjectiveTo evaluate the impact of an in situ, high-fidelity simulation teaching intervention on the management of community-acquired pneumonia in the ambulatory care unit of a district general hospital.MethodsThis study used a mixed-methods approach to evaluate the impact of a programme of 10 in situ high-fidelity simulation education sessions delivered to a total of 10 junior doctors, nine nurses and seven healthcare assistants. Participants were tasked with managing a manikin simulating a patient with pneumonia in real time in a working clinical area. Subsequent structured debrief emphasised key themes from the national guidelines on pneumonia management. The intervention was evaluated through an immediate feedback form, follow-up semistructured interviews by independent qualitative researchers that underwent content analysis and triangulation with audit data on compliance with national pneumonia guidelines before and after the simulation intervention.ResultsThe in situ simulation intervention was valued by participants both in immediate written feedback and in follow-up semistructured interviews. In these interviews, 17 of 18 participants were able to identify a self-reported change in practice following the simulation intervention. Furthermore, most participants reported observing a change in the clinical practice of their colleagues following the training. Collected audit data did not show a statistically significant change in compliance with the guidelines for the management of pneumonia.ConclusionThis study found evidence of a change in both self-reported and observed clinical practice following a simulation intervention, supporting expert opinion that simulation education can impact clinician behaviours and patient outcomes in complex clinical scenarios. Furthermore, this feasibility study provides a transferrable method to evaluate the real-world impact of simulation education that merits further investigation through an appropriately powered study.


2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e18228-e18228
Author(s):  
Dazhi Liu ◽  
Thu Oanh Dang ◽  
Stephen Harnicar ◽  
Katherine Kargus ◽  
Lauren A Evans ◽  
...  

e18228 Background: Early phase clinical trials have broadened treatment options for patients with cancer. Expert management of these new therapies is essential to positive patient outcomes. At Memorial Sloan Kettering Cancer Center, the Developmental Therapeutic Center (DTC) satisfies this need. Oncology clinical pharmacists collaborate with other healthcare professionals to maximize the benefits of drug therapy and minimize toxicities. The purpose of this project is to describe the interventions from a clinical pharmacist assigned to the DTC. Methods: A clinical pharmacist joined DTC to serve adult patients with cancer undergoing clinical trials. The clinical pharmacist acted as a liaison between pharmacy team and medical team, and sees patients during their trial eligibility screening and follow-up visits. The interventions were documented by the clinical pharmacist in patients’ medical charts and email communications. All interventions during 1 month were retrospectively collected and categorized into supportive care optimization, protocol violation prevention, and operational. Results: The oncology clinical pharmacist was involved in 115 patient visits for trial eligibility screening or protocol follow-up. A total of 769 interventions were addressed including supportive care optimization (40.2%), protocol violation prevention (24.7%), and operational (35.1%). Conclusions: The oncology clinical pharmacist is actively engaged in many aspects of cancer care at the early phase trial clinic. Our results demonstrate the vital role of an oncology clinical pharmacist. The impact of these categorized intervention areas would require a formal outcome and cost-saving analysis. [Table: see text]


2018 ◽  
Vol 36 (18_suppl) ◽  
pp. LBA2553-LBA2553 ◽  
Author(s):  
Apostolia Maria Tsimberidou ◽  
David S. Hong ◽  
Jennifer J. Wheler ◽  
Gerald Steven Falchook ◽  
Aung Naing ◽  
...  

LBA2553 Background: We evaluated the impact of pathway targeted and long-term follow-up of patients (pts) with refractory cancers referred to phase I trials. Methods: Pts referred to our program (2007-2013) had CLIA molecular testing. Pts treated with matched targeted therapy (MTT) vs. non-matched therapy (NMT) were analyzed. Results: Of 3,743 pts who had testing, 1,307 had ≥1 alteration and received therapy (MTT 711, NMT 596): med. age 57 yrs, range 16-86; 39% men; med. no. of prior therapies 4, range 0-16. The most common tumors were gastrointestinal 24.2%, gynecologic 19.4%, breast 13.5%, melanoma 11.9%, and lung 8.7%. Targeting MEK/RAF and RET pathways correlated with higher rates of CR/PR/SD≥6 months (mos), PFS and OS compared to others (all P < .001) (Table). Plateau was noted in OS (start, 38 mos): 74 of 711 (10.4%) in the MTT (max 10.7+ yrs) vs. 24 of 596 (4%) in the NMT (max 6 yrs) group were alive (p < .0001). In the MTT group, factors predicting longer PFS were non-PI3K pathway MTT (p < .001), no liver metastases (p < .001), PS < 2 (p = .006), normal LDH (p < .001) and albumin (p = .01) levels, and non-single agent therapy (p = .02). Factors predicting longer OS were non-PI3K pathway MTT (p < .001), no liver metastases (p < .001), PS < 2 (p < .001), normal LDH (p < .001) and albumin (p = .001) levels, and normal PLT counts (p = .03). Conclusions: Outcomes were superior in pts matched to RET and MEK/RAF inhibitors. Factors predicting longer PFS and OS were identified. In the MTT group, 10.4% of patients had OS ≥ 38 mos, the plateau starting point. Clinical trial information: NCT00851032. [Table: see text]


2019 ◽  
Vol 37 (7_suppl) ◽  
pp. 114-114
Author(s):  
Ahmed I. Ghanem ◽  
Remonda M Khalil ◽  
Gehan Abd Elatti Khedr ◽  
Amy Tang ◽  
Amr A. Elsaid ◽  
...  

114 Background: Life expectancy is very essential in deciding treatment options in men with prostate cancer (PCa); however, the impact of comorbidities on outcomes is not well-established. We investigated the influence of Charlson Comorbidity Index (CCI) on survival endpoints in men with localized PCa who were treated with prostate radiotherapy (RT). Methods: Men with intermediate and high risk PCa who were treated with definitive RT between 1/2007 and 12/2012 were included. Groups were created according to their baseline CCI score at diagnosis into no, mild and severe comorbidity (CCI 0, 1 or 2+). The groups were then compared based on patients’ characteristics and prognostic factors. Kaplan-Meier curves and Uni/multivariate analyses (MVA) were used to examine the impact of CCI groups on overall (OS), disease specific (DSS), and biochemical relapse free (BRFS) survival. Results: 257 patients were identified after excluding low risk, metastatic cases and those with inadequate follow up. Median follow-up was 92 months (range: 2-135) and median age was 73 years (range: 48-85). 53% of the cases were black and 67% were of intermediate risk. Median RT dose was 76 Gy and 47% received androgen deprivation therapy. CCI groups 0, 1 and 2+ encompassed 76 (30%), 54 (21%) and 127 (49%) patients, respectively. Groups were generally well-balanced. 10 and 15 years OS was significantly different across CCI groups (76% & 53%, 46% & 31% and 55% & 14%, for CCI-0, 1 and 2+ respectively; p < 0.001). CCI-0 had better DSS than CCI-2+ ( p = 0.03) with no difference for CCI-0 vs 1 ( p = 0.1). BRFS was non-different among CCI groups ( p = 0.99). On MVA, increased CCI was deterministic for OS ( p < 0.001) after adjusting for age, Gleason’s score and T-stage. For DSS, only age and T3 vs T1/2 were independently prognostic ( p < 0.001); whereas CCI-1 vs 0 was only marginal ( p = 0.05). Conclusions: Higher CCI was a significant predictor of shorter OS in intermediate and high-risk PCa. Baseline comorbidities should be taken into consideration during patient counselling for treatment options and in designing prospective trials for men with localized prostate cancer.


2018 ◽  
Vol 56 (8) ◽  
pp. 93-96 ◽  

Before a medicine can be marketed in the UK, marketing authorisation approval is needed from the European Medicines Agency (EMA) or the Medicines and Healthcare products Regulatory Agency (MHRA). However, the time it takes to appraise a medicine is considered by some to delay access to new treatments for people with serious or life-threatening conditions who have no other treatment options. Also, the standard regulatory process may be less suitable for medicines for rare conditions in which it is difficult to gather a large amount of clinical trial data. Here we look at a range of new regulatory and access pathways that have been developed to respond to these challenges and consider some of their potential pitfalls. In a future article we will review the impact that the UK’s departure from the European Union (EU) will have on licensing processes.


BJGP Open ◽  
2019 ◽  
Vol 3 (3) ◽  
pp. bjgpopen19X101659 ◽  
Author(s):  
Jan Lecouturier ◽  
Jason Scott ◽  
Nikki Rousseau ◽  
Gerard Stansby ◽  
Andrew Sims ◽  
...  

BackgroundPatients diagnosed with peripheral arterial disease (PAD) are at an increased risk of coronary heart disease, stroke, heart attack, and PAD progression. If diagnosed early, cardiovascular risk factors can be treated and the risk of other cardiovascular diseases can be reduced. There are clear guidelines on PAD diagnosis and management, but little is known about the issues faced in primary care with regards adherence to these, and about the impact of these issues on patients.AimTo identify the issues for primary care health professionals (HPs) and patients in PAD diagnosis and management, and to explore the impact of these on HPs and PAD patients.Design & settingQualitative study conducted in a primary care setting in the North East of England. Data was collected between December 2014 and July 2017.MethodSemi-structured interviews and focus groups were conducted with PAD register patients (n = 17), practice nurses ([PNs], n = 17), district nurses (DNs], n = 20), tissue viability nurses (n = 21), and GPs (n = 21).ResultsHPs’ attitudes to PAD, difficulty accessing tests, and patient delays impacted upon diagnosis. Some HPs had a reactive approach to PAD identification. Patients lacked understanding about PAD and some reported a delay consulting their GP after the onset of PAD symptoms. After diagnosis, few were attending for regular GP follow-up.ConclusionPatient education about PAD symptoms and risks, and questioning about exercise tolerance, could address the problem of under-reporting. Annual reviews could provide an opportunity to probe for PAD symptoms and highlight those requiring further investigation. Improved information when PAD is diagnosed and, considering the propensity for patients to tolerate worsening symptoms, the introduction of annual follow-up (at minimum) is warranted.


Sign in / Sign up

Export Citation Format

Share Document