scholarly journals Effects of the COVID-19 Pandemic on Caregivers of Young Children with Sickle Cell Disease Enrolled in the Engage-HU Trial

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 1891-1891
Author(s):  
Anna M Hood ◽  
Aimee K Hildenbrand ◽  
Joanna Rebitski ◽  
Jasmine Stallworth ◽  
Yolanda Johnson ◽  
...  
Keyword(s):  
Decision Making ◽  
Sickle Cell Disease ◽  
Shared Decision Making ◽  
Sickle Cell ◽  
Research Funding ◽  
The United States ◽  
Data Safety Monitoring Board ◽  
Shared Decision ◽  
Cell Disease ◽  
Grant Funding

Abstract Background: Hydroxyurea (HU) is the primary medication used to prevent the significant medical and neurologic morbidities of pediatric sickle cell disease (SCD; HbSS or HbSB0 thalassemia). Despite the benefits of HU, it remains under-utilized likely due to lack of clinician knowledge/training and negative caregiver perceptions. Thus, we developed the Engage-HU randomized controlled trial (NCT03442114) as a novel approach to address HU utilization barriers. Engage-HU is designed to assess how clinicians can engage caregivers in a shared discussion that considers their values, preferences, and scientific evidence about HU. The COVID-19 pandemic has resulted in significant changes to healthcare delivery for children with SCD, as they are at increased risk of severe illness from COVID-19 infection. Given their risk status, it was recommended that patients with SCD complete telehealth visits when possible. Some families also chose to delay care because they feared their child would get infected at hospitals/healthcare clinics that care for COVID-19 positive patients. Since the lives of all families enrolled in the Engage-HU trial have been affected to some extent, we incorporated measures to capture the impact of the COVID-19 pandemic and the usability of telemedicine implementation and services. Methods: Engage-HU is a randomized control trial comparing two dissemination methods for clinicians to facilitate shared decision-making with caregivers of young children with SCD. Study outcomes include caregiver confidence in decision-making and perceptions of experiencing shared decision-making as well as HU uptake and child health outcomes. Eligible children are 0 to 5 years, candidates for HU, and their caregiver has not decided about HU in the past 3 months. The trial is being conducted at 9 sites in the United States and uses a unidirectional crossover design. The primary endpoints are caregiver decisional uncertainty and caregiver perception of shared decision-making measured using validated tools. Data will be analyzed using the intent-to-treat principle, and all participants will remain in the arm to which they were randomized. A multiple group comparison analysis will be performed to assess significant response variable differences by group randomization. The Engage-HU study aims to recruit 174 caregivers who are considering initiating HU. The trial is being conducted at 9 sites in the United States. Data collection is ongoing, and 160 caregiver-participants have been enrolled to date. Since May 2020, caregiver-participants have completed the COVID-19 Exposure and Family Impact Scales (CEFIS), which contain 2 subscales (exposure to potentially traumatic aspects of the pandemic, impact on families), and the COVID-19 telemedicine use survey during a study visit. Results: Currently, 8 of the 9 sites have collected data from 48 caregivers (93.8% mothers), most of whom (93.8%) identify as African American/Black (see Figure 1). Correlations indicated that older caregivers experienced greater exposure (Mean = 7.0, SD = 4.1, range = 1-19) to potentially traumatic aspects of the pandemic (r = .31, p = .04). Distress related to COVID-19 varied widely across the sample, for both caregivers (Mean = 5.9, SD = 2.9, range = 1-10) and children (Mean = 4.1, SD = 3.4, range = 1-10). Scores on the telemedicine usability survey were generally high, indicating that caregivers are happy with the quality of care delivered via telehealth. However, caregivers (r = .30, p = .09) and children (r = .32, p = .07) experiencing more pandemic-related distress reported less satisfaction with telehealth. Conclusion: Although Engage-HU has resumed research operations, recruitment has not reached pre-pandemic targets, as fewer eligible patients are scheduled for routine care visits at SCD clinics. Our preliminary analyses suggest a significant continued impact of the pandemic on families and general satisfaction with the quality of healthcare delivered via telemedicine. These findings indicate that targeted screenings to identify and intervene for those who demonstrate more COVID-19 pandemic-related distress are needed. Figure 1 Figure 1. Disclosures Quinn: Forma Therapeutics: Consultancy; Aruvant: Research Funding; Novo Nordisk: Consultancy; Emmaus Medical: Research Funding. Yates: Agios Pharmaceuticals: Current Employment. Badawy: Sanofi Genzyme: Consultancy; Vertex Pharmaceuticals Inc: Consultancy; Bluebird Bio Inc: Consultancy. Thompson: bluebird bio, Inc.: Consultancy, Research Funding; Baxalta: Research Funding; Biomarin: Research Funding; Celgene/BMS: Consultancy, Research Funding; CRISPR Therapeutics: Research Funding; Vertex: Research Funding; Editas: Research Funding; Graphite Bio: Research Funding; Novartis: Research Funding; Agios: Consultancy; Beam: Consultancy; Global Blood Therapeutics: Current equity holder in publicly-traded company. Smith-Whitley: Global Blood Therapeutics: Current Employment. King: National Cancer Institute: Research Funding; National Heart, Lung, and Blood Institute: Research Funding; Health Resources and Services Administration: Research Funding; Global Blood Therapeutics: Research Funding. Meier: CVS Caremark: Consultancy; Forma Therapeutic: Membership on an entity's Board of Directors or advisory committees; NovoNordisk: Membership on an entity's Board of Directors or advisory committees; Novartis,: Other: Data Safety Monitoring Board membership; NHLBI: Other: Data Safety Monitoring Board membership; Global Blood Therapeutics: Other: Steering Committee membership, grant funding; CDC,: Other: grant funding; Indiana Department of Health: Other: grant funding . Tubman: Global Blood Therapeutics: Consultancy, Research Funding; Novartis Pharmaceuticals: Honoraria, Research Funding; Forma Pharmaceuticals: Consultancy; Perkin Elmer: Honoraria. Crosby: Forma Therapeutics: Honoraria; PCORI: Research Funding; HRSA: Research Funding; Global Blood Therapeutics Panel: Honoraria; Children's Hospital of Philadelphia: Honoraria; Professional Resource Exchange: Patents & Royalties: $30-$60 every other year; SCDAA: Honoraria; NHLBI: Other: Payment for review of LRP Proposals, Research Funding. OffLabel Disclosure: Hydroxyurea has been FDA approved for the treatment of sickle cell disease for patients ages 2 years and above but NHLBI and ASH Guidelines recommend it be offered to children as young as age 9 months.

Risk Factors for Death in 632 Patients with Sickle Cell Anemia in the United States and United Kingdom

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 3240-3240 ◽  
Author(s):  
Mark T Gladwin ◽  
Robyn J Barst ◽  
J. Simon R. Gibbs ◽  
Marianna Hildesheim ◽  
Vandana Sachdev ◽  
...  
Keyword(s):  
Risk Factors ◽  
Pulmonary Hypertension ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Research Funding ◽  
Proportional Hazards ◽  
The United States ◽  
Right Heart Catheterization ◽  
Heart Catheterization ◽  
Cell Disease

Abstract Abstract 3240 The role of pulmonary hypertension as a common and attributable cause of mortality in patients with sickle cell disease remains controversial. To assess this question and explore risk factors for death in patients with sickle cell disease we evaluated 632 patients in the Walk-PHASST pulmonary hypertension screening cohort, recruited from nine different study sites in the United States and one site in the United Kingdom. Methods: Patient characteristics and their associations with mortality were analyzed with Cox proportional hazards regression analysis. Based on data from three right heart catheterization screenings studies that have recently been published, we defined the presence of pulmonary hypertension for this analysis by a Doppler-echocardiographic measurement of the tricuspid regurgitant jet velocity (TRV) ≥ 3.0 m/s, which has a 67–75% positive predictive value for a mean pulmonary artery pressure ≥ 25 mm Hg by right heart catheterization. This therefore represents a very conservative threshold for a large population screening study. Among subjects with a measurable TRV (n=572), 64 (11.2%) had measurements of ≥ 3.0 m/sec. Among those with measurable NT-proBNP (n=582), 140 (24.1%) had measurements ≥160 pg/mL, a value associated with both pulmonary hypertension and mortality. A total of 39 (7.4%) had both high TRV (≥3.0 m/sec) and high NT-proBNP (≥160 pg/mL). Results: Over a median follow-up time of 29 months, we observed 22 deaths. 50% (N=11) of these patients had a TRV≥ 3.0 m/sec. At 24 months the cumulative survival was 83% for patients with TRV ≥ 3.0 m/sec and 98% for patients with TRV < 3.0 m/sec (p<0.0001). The unadjusted hazard ratios for death were 11.14 (95% CI 4.1–30.1; p<0.0001) for patients with TRV above and below 3.0 m/sec and 4.55 (95% CI 1.8–11.3; p=0.001) for patients with NT-proBNP above and below 160 pg/mL. For patients with both high TRV (≥ 3.0 m/sec) and high NT-proBNP (≥ 160 pg/mL), the unadjusted hazard ratio was 14.86 (95% CI 5.5–39.9; p<0.0001). Overall, an increased risk of death was observed for both age and gender, with males at higher risk relative to females (HR=2.48, 95% CI 1.0–6.1; p=0.05), and patients older than 47 years (HR=2.02, 95% CI 1.1–3.8; p=0.03). Associations with mortality were also observed for chronic transfusions (HR=3.00, 95% CI 1.2–7.8; p=0.02) and a NYHA/WHO class value or III or IV (HR=4.52, 95% CI 1.4–14.3; p=0.01). Other variables associated with mortality in our cohort included a high hemolytic component, aspartate aminotransferase (AST), ferritin, and creatinine. Variables not associated with mortality included current hydroxyurea use, SC disease, self-reported history of painful episodes, and six-minute walk distance. In stepwise multiple proportional hazards regression analysis, the association between TRV and mortality remained significant after adjustment for all other risk factors, including ferritin, AST, creatinine and even NT-proBNP (HR 4.27, 95%CI 1.3–14.1; p=0.04). Conclusions: Using a more conservative cut-off value of TRV ≥ 3.0 m/sec as defining PH in a large screening population of sickle cell disease patients, PH occurs in approximately 10% of unselected screened patients and is associated with the highest unadjusted and adjusted risk for death of any measured variable. Disclosures: Gladwin: Bayer Corp: Consultancy, Research Funding; NIH and NHLBI: Research Funding; Gilead Sciences: Research Funding. Barst:Ventripoint: Stock options Other; Actelion, Eli Lilly, Gilead, Glaxo Smith-Kline (GSK), Medtronics, Bayer, Ikaria, Pfizer, Novartis, VentriPoint: Consultancy, Honoraria. Girgis:NIH/NHLBI: Research Funding, Travel support Other. Rosenzweig:NIH: Research Funding. Badesch:NIH: Research Funding. Lanzkron:NIH: Research Funding.

scholarly journals Intravenous Fluid Boluses Are Commonly Administered to Adults with Sickle Cell Disease and Vaso-Occlusive Pain

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4839-4839 ◽  
Author(s):  
Marcus A. Carden ◽  
Paula Tanabe ◽  
Jeffrey A. Glassberg
Keyword(s):  
United States ◽  
Sickle Cell Disease ◽  
Clinical Outcomes ◽  
Sickle Cell ◽  
Research Funding ◽  
The United States ◽  
Adult Patients ◽  
Fluid Type ◽  
Cell Disease ◽  
Oral Hydration

Background: Sickle cell disease (SCD) affects 100,000 adults and children in the United States (USA). Painful, uncomplicated vaso-occlusive episodes (VOE) are the most common complication of SCD and the #1 reason adults with SCD seek medical attention in the ED. The NHLBI guidelines suggest encouraging oral hydration, along with analgesics, during VOE but to give intravenous (IV) fluids only if the patient cannot drink. However, there exists a clinical equipoise regarding the choice of IV fluids to use during the treatment of VOE in the ED, along with the rate or volume given. Despite lack of guidelines or evidence, the use of large IV volumes of crystalloids given over short periods of time (i.e. bolus) in the ED is common practice and we recently showed normal saline (NS) boluses in particular may be associated with inferior pain control and higher admission rates in pediatric patients with VOE (Carden etal., Am J Hem, 2019). Importantly, investigations into the use and impact these different IV fluids, including IV boluses, and how they may impact clinical outcomes among adult patients with SCD and VOE who present to the ED are lacking. Methods: We conducted a cross-sectional survey of emergency providers at the 2011 annual American College of Emergency Physicians Scientific Assembly, where ED providers from across the United States attend, regarding IV fluid practices during VOE. We specifically used a validated instrument to assess self-reported practices toward patients with SCD regarding IV fluid use, including volumes and rates, during uncomplicated VOE. Basic demographic information was obtained and providers were specifically asked: "Please indicate which type of fluids and rate of administration you give to patients with acute sickle cell pain who are not hypotensive and not severely hypovolemic". Providers responded never, rarely, frequently or always to each fluid type and rate. Results: Of 795 respondents to the survey, 722 indicated they took care of patients with SCD, 669 completely responded to the survey, and of those, 244 respondents only took care of adult patients with SCD. Demographic and experience with SCD patients, as well as preferred fluid type and rate of administration is reported in Table 1. IV fluid use during uncomplicated VOE varied among adult providers, but 83% of providers surveyed used IV fluid crystalloid boluses during VOE. Only 45% of providers recommended oral hydration during VOE among adults. Conclusions: Among adults ED providers who care for patients with SCD in the USA, wide variations in practice utilizing IV fluids are common. Despite no guidelines, IV fluid boluses are commonly given, as was seen in pediatric ED studies, and oral hydration is less commonly recommended among adult ED providers. Further investigation is needed to determine if these practices have an impact on clinical outcomes among euvolemic adult patients with SCD and VOE who present to the ED. Disclosures Carden: GBT: Honoraria; NIH: Research Funding. Tanabe:NIH: Research Funding; AHRQ: Research Funding. Glassberg:ACEP: Research Funding; NHLBI: Research Funding; Pfizer: Research Funding.

scholarly journals COVID-19 Outcomes in Individuals with Sickle Cell Disease and Sickle Cell Trait Compared to Blacks without Sickle Cell Disease or Trait

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 54-56 ◽  
Author(s):  
Ashima Singh ◽  
Amanda M. Brandow ◽  
Julie Panepinto
Keyword(s):  
United States ◽  
Type 1 Diabetes ◽  
Sickle Cell Disease ◽  
Propensity Score ◽  
Sickle Cell ◽  
Research Funding ◽  
Sickle Cell Trait ◽  
The United States ◽  
Cell Disease

Introduction: By August 1, 2020 in the United States, more than 3 million cases of Coronavirus disease 2019 (COVID-19) had been reported with more than 150,000 deaths due to this disease. Growing evidence suggests that individuals with the pre-existing conditions of hypertension, diabetes, cardiovascular disease and obesity are at a higher risk of more serious COVID-19 illness. However, the impact of COVID-19 on individuals with sickle cell disease and sickle cell trait as compared to those without sickle cell disease or trait is not known. The objective of this study was to determine the rate of hospitalization, disease symptoms and deaths due to COVID-19, in patients with sickle cell disease and sickle cell trait compared to Blacks without sickle cell disease or trait. Methods: We leveraged existing electronic health record (EHR) data from multiple sites that contribute data to a research network, TriNetX. TriNetX query platform was used to identify patients with COVID-19 infection based on ICD diagnoses codes or a positive COVID-19 result from a nucleic acid amplification with probe-based detection test, present any time after January 20, 2020 (this is when the first COVID-19 case was detected in the United States) within the patients' EHR data. We report rates of specific COVID-19 related outcomes among individuals with sickle cell disease and trait, calculated as % of patients in cohort with the particular outcome. Our outcomes of interest included COVD-19 related symptoms, hospitalization, and death, which occurred within 2 weeks of COVID diagnosis. We used propensity score matching (greedy nearest-neighbor matching algorithm with a caliper of 0.1 pooled standard deviations) to create balanced cohorts for comparing outcomes between individuals with sickle cell disease or trait and Blacks without sickle cell disease or trait. Risk ratios and risk differences are reported along with 95% confidence intervals. Given multiple outcomes of interest, we considered a more stringent two-sided alpha of less than &lt;0.01, based on a z-test, to determine statistical significance for differences in outcome rates. Results: As of July 15, 2020, there were 122 COVID-19 patients who had sickle cell disease and 172 COVID-19 patients who had sickle cell trait. Our comparator groups included 15,762 Blacks who were diagnosed with COVID-19 but did not have sickle cell trait/disease. COVID-19 patients with sickle cell disease were significantly younger and a higher proportion had asthma, type 1 diabetes and pre-existing liver conditions compared to Blacks without sickle cell trait/disease (Table 1). COVID-19 patients with sickle cell trait were significantly younger, a higher proportion were females, overweight/obese, and a higher proportion had asthma or type 1 diabetes compared to Blacks without sickle cell trait/disease (Table 1). The rate of respective outcomes for the three groups is shown in Figure 1. Propensity score matching yielded a cohort of patients such that there were no significant differences in demographic and clinical characteristics between patients with sickle cell disease/trait compared to Blacks without sickle cell trait/disease. After matching, COVID patients with sickle cell disease remained at a higher risk of hospitalization, pneumonia and pain compared to Blacks without sickle cell trait/disease (Table 2). The case fatality rates were not significantly different between those with sickle cell disease compared to Blacks. There were no significant differences in COVID outcomes between sickle cell trait and Blacks without sickle cell trait/disease, within the matched cohort. Conclusions: These data provide evidence that sickle cell disease imposes additional risk of severe COVID-19 illness and hospitalization, after balancing for age, gender and other preexisting conditions. The death rate between sickle cell disease and Blacks without sickle cell trait/disease was not significantly different. There are no significant differences in COVID-19 outcomes between sickle cell trait and Blacks without sickle cell trait/disease, after balancing for age, gender and other pre-existing conditions. Disclosures Brandow: NIH / NHLBI: Research Funding; Greater Milwaukee Foundation: Research Funding. Panepinto:HRSA: Research Funding; NINDS: Research Funding; NINDS: Research Funding; NHLBI: Research Funding.

scholarly journals Real-World Experience of Patients with Sickle Cell Disease Treated with Voxelotor: A Multicenter, Retrospective Study

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3100-3100
Author(s):  
Biree Andemariam ◽  
Maureen Achebe ◽  
E. Leila Jerome Clay ◽  
Richard A. Drachtman ◽  
Archana Sharma ◽  
...  
Keyword(s):  
Retrospective Study ◽  
Sickle Cell Disease ◽  
Board Of Directors ◽  
Sickle Cell ◽  
Real World ◽  
Research Funding ◽  
The United States ◽  
Post Treatment ◽  
Cell Disease ◽  
Advisory Committees

Abstract Background: Sickle cell disease (SCD) is an inherited systemic disorder in which sickle hemoglobin (HbS) polymerization triggers red blood cell sickling, chronic hemolytic anemia, and recurrent episodes of vaso-occlusion. SCD-related complications lead to acute and chronic life-threatening events, cumulative organ damage, disability, and early mortality. Voxelotor (Oxbryta ®) tablets are approved in the United States for treatment of SCD in adults and adolescents aged ≥12 years, based on the efficacy and safety data from the randomized, placebo-controlled, multicenter HOPE trial. Voxelotor is an oral, once-daily HbS-polymerization inhibitor that has been shown to increase hemoglobin (Hb) levels and reduce markers of hemolysis. The Retrospective Study to Evaluate Outcomes in Patients with Sickle Cell Disease Treated with Oxbryta (RETRO) is designed to collect, aggregate, and characterize real-world, retrospective laboratory and clinical data on adults and adolescents with SCD treated with voxelotor as part of their usual care at multiple clinical centers in the United States. Methods: RETRO is a multicenter, post-marketing, retrospective study of approximately 300 patients (aged ≥12 years) with SCD from 10 US study sites. Independent SCD expertise is provided by a steering committee to inform the design and conduct of the voxelotor registry. Clinical and laboratory data have been collected and aggregated 12 months before initiation of voxelotor treatment and compared with post-treatment data outcomes. Patients with documented SCD (all genotypes) who received voxelotor treatment for ≥2 consecutive weeks were included in this analysis. Only data available from patients' medical records (and other secondary data sources) 1 year before and up to 1 year after the first voxelotor dose were documented in de-identified case report forms via an electronic data capture system. Results: Forty-nine patients whose data were entered at 5 sites at the time of data cutoff (June 25, 2021) were included (mean age [SD]: 34.3 [12.91] years; 57.1% female; 85.7% HbSS and 6.1% HbSβ 0 genotype). Mean (SD) duration of voxelotor treatment was 48.1 (23.0) weeks. The initial prescribed voxelotor dose strengths (n, %) were 500 mg (4, 8.2%), 1000 mg (7, 14.3%), and 1500 mg (38, 77.6%). Rationale for prescription (n, %) included reduction of anemia (36, 73.5%), reduction in frequency of vaso-occlusive crises (23, 46.9%), reduction in pain (34, 69.4%), reduction in the need for blood transfusion (8, 16.3%) and other (5, 10.2%); more than 1 reason may have been selected. In 35 patients with recorded baseline and post-treatment Hb values, the peak observed post-treatment Hb (mean [SD]) was 9.4 (2.44) g/dL, an increase of 1.6 (1.5) g/dL from baseline (7.8 [2.02] g/dL). Fifty percent (11/22) of patients had a clinical response (Hb increase of &gt;1.0 g/dL from baseline) within 12 months of voxelotor treatment. Per-patient peak changes in Hb during the study period showed that 62.9% of patients experienced a response at some time up to 12 months during treatment (Figure). Change in hemolytic markers was also evaluated. In patients with recorded baseline and post-treatment reticulocyte percentage (N=19) and indirect bilirubin (N=24), the mean (SD) absolute post-treatment value was 7.4% (4.65%) for reticulocyte percentage, a decrease of 4.9% (6.63%) compared with baseline (12.4% [8.32%]), and 1.9 (1.66) mg/dL for indirect bilirubin, a decrease of 17.7 (81.83) mg/dL compared with baseline (19.6 [81.82] mg/dL). The most common non-SCD-related treatment-emergent adverse events (AEs) were diarrhea, headache, and rash (Table); 19 (38.8%) patients reported ≥1 AE, and most non-SCD-related AEs were mild in severity. Conclusions: RETRO is the first multicenter, retrospective study to examine the real-world effectiveness of voxelotor and describe the observed changes in laboratory and clinical outcomes after ≥2 weeks of therapy. This study shows that voxelotor treatment was associated with increased Hb levels and decreased hemolytic markers. The safety data are consistent with those from the HOPE trial. Further evaluation is needed, with additional data from all 10 sites, and will be presented later. Funding: This study was supported by Global Blood Therapeutics. Figure 1 Figure 1. Disclosures Achebe: Fulcrum Therapeutics: Consultancy; Pharmacosmos: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees. Clay: GBT: Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria. Nero: Global Blood Therapeutics: Consultancy; Editas Medicine: Consultancy; bluebird bio: Consultancy; Novartis: Consultancy. Osunkwo: Terumo: Consultancy; Global Blood Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Acceleron: Consultancy; Forma Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees; Health and Services Administration: Research Funding; Patient Centered Outcomes Research Instituted: Research Funding; Micella Biopharma: Membership on an entity's Board of Directors or advisory committees; Pfizer: Consultancy; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Chiesi: Consultancy; Cyclerion: Consultancy; Emmaus: Consultancy. Idowu: Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Ironwood: Research Funding; Forma Therapeutics, Inc.: Research Funding; Pfizer: Research Funding. Shah: Novartis: Research Funding, Speakers Bureau; Alexion: Speakers Bureau; Emmaus: Consultancy; GBT: Consultancy, Research Funding, Speakers Bureau; Guidepoint Global: Consultancy; CSL Behring: Consultancy; GLG: Consultancy; Bluebird Bio: Consultancy. Curtis: GBT: Consultancy. Minniti: Bluebird Bio: Other: Endpoint adjudicator ; F. Hoffmann-La Roche Ltd: Consultancy; Chiesi: Consultancy; Novo Nordisk: Consultancy; Forma: Consultancy; Novartis: Consultancy; GBT: Consultancy; CSL Behring: Other: Endpoint adjudicator .

scholarly journals Discordant Beliefs, Perceptions, and Experiences between Patients with Sickle Cell Disease and Their Care Teams: Insights from a Pilot Program to Support Shared Decision-Making

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 15-17
Author(s):  
Kim Smith-Whitley ◽  
Lewis L. Hsu ◽  
Anne Jacobson ◽  
Jeffrey D Carter ◽  
Tamar Sapir
Keyword(s):  
Decision Making ◽  
Sickle Cell Disease ◽  
Collaborative Learning ◽  
Board Of Directors ◽  
Sickle Cell ◽  
Care Team ◽  
Treatment Goals ◽  
Shared Decision ◽  
Cell Disease ◽  
Advisory Committees

Background For patients with sickle cell disease (SCD), shared learning, understanding, and decision-making with their healthcare providers (HCPs) is critical for achieving treatment goals. Using a validated collaborative learning model (Sapir 2017), we evaluated patient and HCP perceptions of shared decision-making (SDM) related to SCD treatment. Methods In June 2020, 113 patients/caregivers of patients with SCD and 30 HCPs participated in 3 live, virtual collaborative learning sessions developed with the Sickle Cell Disease Association of America (Table 1). Before and after the sessions, patients and their HCPs completed tethered surveys to assess alignments and discordances in preferences, experiences, and concerns around SCD treatment and SDM. Results Patients and HCPs held discordant beliefs about the degree of patients' progress toward treatment goals. While HCPs estimated that only 29% of their patients are on track to meet their goals, 85% of patients reported that they felt somewhat (39%) or fully (46%) on track. The magnitude of this discrepancy suggests that patients may not be aware of treatment milestones they are not reaching. Participants differed in their perceptions of how often HCPs engage patients in SDM (Figure 1). Relative to patients' estimates, HCPs were more likely to report that they usually or always: ask about the patient's goals for treatment (41% vs 61%), work with the patient to create a treatment plan (47% vs 55%), explain their own goals for the patient's treatment (47% vs 55%), and explain the pros/cons of each treatment choice (47% vs 55%). The biggest missed opportunity for SDM involved how often HCPs ask patients about stigma related to opioids for pain management (28% vs 35%). The reported lack of communication around stigma may contribute to providers' underestimation of how often their patients encounter stigma. HCPs estimated that 47% of their patients have faced barriers to adequate pain management in the ER due to opioid stigma and/or poor knowledge of pain crises among ER staff. By comparison, 74% of patients reported ever feeling that ER doctors, nurses, or staff questioned their need for pain medication during a pain crisis (20% once; 54% more than once). When asked why patients are not more involved in SDM (Figure 2), HCPs were more likely to select patient factors: feeling too overwhelmed to make treatment decisions (52% HCPs, 14% patients), low health literacy (49% HCPs, 12% patients), and lack of confidence in validity of opinions/concerns (44% HCPs, 10% patients). In contrast, patients were more likely to report that they are completely involved in treatment decisions (40% patients, 4% HCPs) and that they trust their care team to make decisions on their behalf (37% patients, 30% HCPs). Regarding adjusting SCD therapy, HCPs overestimated how many patients were concerned about learning a new routine/schedule (23% HCPs, 7% patients), but underestimated concerns about being able to pay for it (4% HCPs, 9% patients). HCPs correctly estimated concerns about knowing whether treatment will work (33% HCPs, 34% patients), fear of side effects (29% HCPs, 25% patients), and knowing how to manage a pain episode (9% HCPs, 9% patients). Overall, HCPs underestimated how many patients would not worry about switching treatment (1% HCPs, 9% patients). Following the collaborative learning sessions, patients made commitments to talk to their care team about their treatment goals (52%), consider treatment options more closely (50%), take a more active role in decision-making (46%), and notify their care team with concerns (41%). HCPs set goals to increase the types of educational materials they provide (57%), attempt to ease the transition to the adult care setting (50%), engage their patients more frequently in SDM (47%), and train colleagues on how to conduct additional learning sessions (34%). The full data set with 2 additional learning sessions (Aug 2020) will be presented. Conclusions Patients with SCD and their care teams differed in their experiences and beliefs related to SDM. Collaborative education can reveal opportunities for greater understanding and facilitate improved patient-provider communication, leading to greater engagement in SDM around SCD treatment. Study Sponsor Statement The study reported in this abstract was funded by an independent educational grant from bluebird bio. The grantor had no role in the study design, execution, analysis, or reporting. Disclosures Smith-Whitley: Prime: Other: Education material; Novartis: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees; Celgene: Membership on an entity's Board of Directors or advisory committees.

Co-Design of an Electronic Dashboard to Support Coproduction of Care in Pediatric Rheumatic Disease (Preprint)

2021 ◽  
Author(s):  
Alysha Taxter ◽  
Lisa Johnson ◽  
Doreen Tabussi ◽  
Yukiko Kimura ◽  
Brittany Donaldson ◽  
...  
Keyword(s):  
Decision Making ◽  
Shared Decision Making ◽  
Clinical Data ◽  
The United States ◽  
Shared Decision ◽  
Time Data ◽  
Vertical Orientation ◽  
Patient Reported ◽  
Data Elements ◽  
The Right

BACKGROUND Coproduction of care involves patients and families partnering with their clinicians and care teams, with the premise that each brings their own perspective, knowledge, and expertise, as well as their own values, goals, and preferences to the partnership. Dashboards can display meaningful patient and clinical data to assess how a patient is doing and inform shared decision making. Increasing communication between patients and care teams is particularly important for children with chronic conditions, such as juvenile idiopathic arthritis (JIA), which is the most common, chronic rheumatic condition of childhood, and is associated with increased pain, decreased function, and decreased quality of life. OBJECTIVE We aimed to design a dashboard prototype for use in coproducing care for JIA patients. We evaluated the context use and needs of end users, obtained consensus on the necessary dashboard data elements, and constructed display prototypes to inform meaningful discussions for coproduction. METHODS A human-centered design approach involving parents, patients, clinicians, and care team members was used to develop a dashboard to support coproduction of care in four diverse ambulatory pediatric rheumatology clinics across the United States. We engaged a multidisciplinary team (n=18) of patients/parents, clinicians, nurses, and staff during an in-person kick-off meeting, followed by bi-weekly meetings. We also leveraged advisory panels. Teams mapped workflows and patient journeys, created personas, and developed dashboard sketches. Final necessary dashboard components were determined using Delphi consensus voting. Low-tech dashboard testing was completed during clinic visits, and visual display prototypes were iterated using PDSA methodology. Patients and providers were surveyed about their experiences. RESULTS Teams achieved consensus on what data matters most at point-of-care to support JIA patients, families, and clinicians partnering together to make the best possible decisions for better health. Notable themes included: the right data, in the right place, at the right time; data in once for multiple purposes; patient and family self-management components; and opportunity for education and increased transparency. A final set of 11 dashboard data elements were identified which include patient-reported outcomes, clinical data, and medications. Important design considerations include incorporation of real-time data, clearly labeled graphs, and vertical orientation to facilitate review and discussion. Prototype paper testing with 36 patients/families yielded positive feedback about the dashboard’s usefulness during clinic discussions, helped to talk about what mattered most, and informed healthcare decision making. CONCLUSIONS Our study developed a dashboard prototype that displays patient-reported and clinical data over time, along with medications, that can be used during a clinic visit to support meaningful conversations and shared decision making between JIA patients/families and their clinicians and care teams. CLINICALTRIAL N/A

How individual ethical frameworks shape physician trainees’ experiences providing end-of-life care: a qualitative study

2021 ◽  
pp. medethics-2020-106690
Author(s):  
Sarah Rosenwohl-Mack ◽  
Daniel Dohan ◽  
Thea Matthews ◽  
Jason Neil Batten ◽  
Elizabeth Dzeng
Keyword(s):  
Decision Making ◽  
End Of Life ◽  
Shared Decision Making ◽  
End Of Life Care ◽  
The United States ◽  
Ethical Framework ◽  
Shared Decision ◽  
Life Care ◽  
Best Interest ◽  
Ethical Frameworks

ObjectivesThe end of life is an ethically challenging time requiring complex decision-making. This study describes ethical frameworks among physician trainees, explores how these frameworks manifest and relates these frameworks to experiences delivering end-of-life care.DesignWe conducted semistructured in-depth exploratory qualitative interviews with physician trainees about experiences of end-of-life care and moral distress. We analysed the interviews using thematic analysis.SettingAcademic teaching hospitals in the United States and United Kingdom.ParticipantsWe interviewed 30 physician trainees. We purposefully sampled across three domains we expected to be associated with individual ethics (stage of training, gender and national healthcare context) in order to elicit a diversity of ethical and experiential perspectives.ResultsSome trainees subscribed to a best interest ethical framework, characterised by offering recommendations consistent with the patient’s goals and values, presenting only medically appropriate choices and supporting shared decision-making between the patient/family and medical team. Others endorsed an autonomy framework, characterised by presenting all technologically feasible choices, refraining from offering recommendations and prioritising the voice of patient/family as the decision-maker.ConclusionsThis study describes how physician trainees conceptualise their roles as being rooted in an autonomy or best interest framework. Physician trainees have limited clinical experience and decision-making autonomy and may have ethical frameworks that are dynamic and potentially highly influenced by experiences providing end-of-life care. A better understanding of how individual physicians’ ethical frameworks influences the care they give provides opportunities to improve patient communication and advance the role of shared decision-making to ensure goal-aligned end-of-life care.

scholarly journals COVID Symptoms and COVID Anxiety in Sickle Cell Disease

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 16-17
Author(s):  
Wally R Smith ◽  
Benjamin Jaworowski ◽  
Shirley Johnson ◽  
Thokozeni Lipato ◽  
Daniel M Sop
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Research Funding ◽  
Telephone Survey ◽  
Cell Disease ◽  
Weighted Mean ◽  
The Us ◽  
Associated Symptoms ◽  
At Home

Background Even before the US upswing of the current COVID pandemic, the number of sickle cell disease (SCD) patients coming to hospitals and EDs appeared to fall drastically. This happened despite SCD patients having often been heavy utilizers of the ED and hospital for their iconic vaso-occlusive crises (VOC). Though ambulatory SCD clinics quick converted largely to telehealth in order to comply with stay-at-home orders designed to suppress person-to-person transmission, some SCD patients appeared to avoid care, delay care, or refuse doctors' invitations for care. Presumably patients did so out of COVID fears, but this has not been confirmed in the literature. Further, whether these patients had COVID symptoms but stayed at home has not been studied. As part of quality improvement (QI) to conduct COVID surveillance in an adult sickle cell program, we sought to explain and predict SCD health care utilization patterns we were observing, as well as to determine urgent physical and mental health needs of patients who appeared to be avoiding care. Methods Fifteen staff in the Adult Sickle Cell Medical Home at Virginia Commonwealth University, a large urban academic medical center, conducted a telephone survey ("wellness check"was used when we talked to patients) of all known adults with SCD over 19 days in 2020. A staff member confirmed the patient had SCD, asked permission to proceed, then asked about symptoms consistent with COVID-19. At the end of the telephone survey, respondents wer invited to complete an email survey of sickle cell and COVID-19 utilization attitudes (19-33 items, depending on the response pattern, either drawn from the National Health Interview Survey, from the Adult Sickle Cell Quality of Life Measurement quality of care survey, or drafted by the authors), the Sickle Cell Stress Survey-Adult (SCSS-A, a 10-item previously validated survey), and anxiety and depression (PHQ9 of the PRIME-MD). Results Of 622 adults approached by phone call, 353 responded to the following yes/no screening questions regarding the prior 14 days: fever over 100 F 0/353 (0.00%); cough 3/353(0.01%); difficulty breathing 0/353(0.00%); unexplained shortness of breath 2/353(0.01%); sore throat 2/353 (0.01%); unexplained muscle soreness 2/353(0.01%);contact with anyone who tested positive for COVID-19 2/353(0.01%); testing for COVID 19 6/353(0.02%). For QI purposes, we set a threshold of three or more COVID-associated symptoms or the presence of fever as criteria requiring intense telephone or in-person staff monitoring for the following week. Only three patients met criteria. A total of 219/353 had email surveys sent. Of 63 patients (28.8%) who returned email surveys by June 10, 2020, 35.9% had already managed a "pain attack" at home 4 or more times in the prior 12 months, and 45.5% of these said their bad ER experiences were very or somewhat important in that decision. In the prior 14 days, although 30/64 reported a crisis for at least one day, only 4/64 had visited the Emergency Department for pain. On a 0-10 scale, 21/61 patients endorsed "0" for worry that they would be COVID-infected by going for medical care (weighted mean 3.9), but 18/59 endorsed "10" for worry they were more at risk of COVID because of SCD (weighted mean 6.31), and 22/60 endorsed "10" for worry they would fare worse than others if COVID infected (weighted mean 6.97). Many patients forwent "needed" care (16/62) or delayed "needed" care by at least a day (36/61). Eleven patients met criteria for moderately severe to severe depression on the PHQ-9, and 28/63 somewhat or strongly agreed with the statement "death is always on the back of my mind" on the SCSS-A. Conclusions In adolescents and adults with SCD, many were already reticent to come to the ED for pain, but a significant portion reported delays or avoidance of needed care during the early stages of the US COVID pandemic, and few reported using the ED despite over half reporting at least one crisis day in 14. Patients nonetheless reported very few COVID-associated symptoms. Fears of COVID infection/susceptibility may limit visits for needed sickle cell care among adults. Acknowledgements: Mica Ferlis RN, FNP, Caitlin McManus, RN, FNP, Emily Sushko, RN, FNP, Justin West, RN, Kate Osborne, RN, Stefani Vaughan-Sams, Marla Brannon, BS, Nakeiya Williams, BS Disclosures Smith: GlycoMimetics, Inc.: Consultancy; Emmaeus Pharmaceuticals, Inc.: Consultancy; Novartis, Inc.: Consultancy, Other: Investigator, Research Funding; Global Blood Therapeutics, Inc.: Consultancy, Research Funding; Shire, Inc.: Other: Investigator, Research Funding; NHLBI: Research Funding; Patient-Centered Outcomes Research Institute: Other: Investigator, Research Funding; Health Resources and Services Administration: Other: Investigator, Research Funding; Incyte: Other: Investigator; Pfizer: Consultancy; Ironwood: Consultancy; Novo Nordisk: Consultancy; Imara: Research Funding; Shire: Research Funding.

scholarly journals Clinical and Biomarker Predictors for Avascular Necrosis in Sickle Cell Disease

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3091-3091
Author(s):  
Michael Rabaza ◽  
Maria Armila Ruiz ◽  
Liana Posch ◽  
Faiz Ahmed Hussain ◽  
Franklin Njoku ◽  
...  
Keyword(s):  
Risk Factors ◽  
Sickle Cell Disease ◽  
Board Of Directors ◽  
Avascular Necrosis ◽  
Sickle Cell ◽  
Research Funding ◽  
Medical Attention ◽  
Cell Disease ◽  
Advisory Committees ◽  
Early Management

Abstract Introduction Sickle cell disease (SCD) affects 1 in 365 African Americans and approximately 25 million people world-wide. A common skeletal system complication is avascular necrosis (AVN), which can cause substantial pain and a reduced quality of life. While early management of AVN is focused on increasing range of motion with physical therapy and pain relief, there are no clear predictors for who is more likely to develop AVN and earlier institution of these preventive measure could help decrease disease progression. Vascular endothelial growth factor (VEGF) is a biomarker of endothelial injury and may indicate reduced vascular supply to the femoral or humeral head. Here we describe potential risk factors and biologic pathways for AVN in SCD, as understanding these may lead to improvements in future monitoring, early detection, and early intervention practices. Methods We investigated clinical and laboratory risk factors associated with AVN in a cohort of 435 SCD patients from our center. Blood samples, clinical, and laboratory data were collected at the time of enrollment during a clinic visit. Genotyping for alpha thalassemia was performed by PCR and the serum concentration of VEGF was measured by ELISA. AVN status was confirmed by review of the medical record and available imaging. We conducted a cross-sectional analysis comparing categorical and linear variables by AVN status using the chi-square and Kruskal-Wallis test, respectively. The independent association of the clinical and laboratory variables with AVN status was determined by logistic regression analysis. The initial model included variables with a P-value &lt; 0.1 on univariate analysis and the final model was ascertained by stepwise forward and backward selection. Median values and interquartile range (IQR) are provided. Results The median age of the cohort was 32 (IQR, 24 - 43) years, 57% (250/435) were female, and 46% (198/435) were on hydroxyurea. AVN was observed in 34% (149/435) of SCD patients. SCD patients with AVN were older, had more frequent vaso-occlusive crises requiring medical attention, and had a higher body mass index (Table I) (P ≤ 0.002). We measured VEGF in 241 of the SCD patients with serum samples available at the time of enrolment. Serum VEGF concentrations trended higher in SCD patients with versus without AVN (420 vs. 359 pg/mL, respectively; P = 0.078). In the multivariate analysis model, AVN was independently associated with increased number of vaso-occlusive crises (OR 1.1, 95% CI: 1.0 - 1.14; P = 0.02), AST concentration (natural log OR 0.5, 95% CI: 0.2 - 0.9; P = 0.03), VEGF concentration (natural log OR 1.4, 95% CI: 1.0 - 1.9; P = 0.047), and tobacco use (OR 1.9, 95% CI: 0.9 - 3.7; P = 0.078). Discussion In conclusion, we demonstrate a high prevalence of AVN in an adult cohort of SCD patients. The presence of AVN was independently associated with a greater frequency of vaso-occlusive pain episodes, which may demonstrate a shared pathophysiology between AVN and vaso-occlusion that merits further investigation. We demonstrate that serum VEGF concentrations are higher in SCD patients with AVN and may be a clinical tool to identify those at high-risk and for earlier intervention for this complication. Figure 1 Figure 1. Disclosures Gordeuk: Modus Therapeutics: Consultancy; Novartis: Research Funding; Incyte: Research Funding; Emmaus: Consultancy, Research Funding; Global Blood Therapeutics: Consultancy, Research Funding; CSL Behring: Consultancy. Saraf: Pfizer: Research Funding; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding.

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