Among the difficulties of living with β-thalassemia, patients frequently require blood transfusions and experience iron overload. As serum ferritin (SF) provides an indication of potential iron overload, we conducted a systematic literature review (SLR) to assess whether SF levels are associated with clinical and economic burden and patient-reported outcomes (PROs). The SLR was conducted on 23 April 2020 and followed by analysis of the literature. Dual-screening was performed at the title, abstract, and full-text levels using predefined inclusion and exclusion criteria. Ten studies identified by the SLR were eligible for inclusion in the analysis. Seven studies were conducted in Europe, and most were prospective or retrospective in design. The patient populations had a median age of 20.7–42.6 years, with a percentage of men of 38–80%. Sparse data were found on the correlation between SF levels and mortality, and hepatic, skeletal, and cardiac complications; however, in general, higher SF levels were associated with worsened outcomes. The bulk of the evidence reported on the significant association between higher SF levels and endocrine dysfunction in its many presentations, including a 14-fold increase in the risk of diabetes for patients with persistently elevated SF levels. No studies reporting data on PROs or economic burden were identified by the SLR. SF levels provide another option for prognostic assessment to predict a range of clinical outcomes in patients with β-thalassemia.
Anemia is the most common form of cytopenia in patients with myelodysplastic syndromes (MDS), who require chronic red blood cell transfusions and may present high serum ferritin (SF) levels as a result of iron overload. To better understand the potential effects of high SF levels, we conducted a systematic literature review (SLR) to identify evidence on the relationship between SF levels and clinical, economic, or humanistic outcomes in adult patients with MDS. Of 267 references identified, 21 were included. No studies assessing SF levels and their relationship with humanistic or economic outcomes were identified. Increased SF levels were an indicator of worse overall survival and other worsened outcomes; however, the association was not consistently significant. SF levels were a significant prognostic factor for relapse incidence of MDS and showed a significant positive correlation with number of blood units transfused but were not associated with progression to acute myeloid leukemia or the time to transformation. Higher SF levels were also an indicator of a lower likelihood of leukemia-free survival, relapse-free survival, and event-free survival. The SLR suggests that SF levels are associated with clinical outcomes in MDS, with higher levels correlated with number of blood units transfused, frequently indicating worse outcomes.
There is considerable demand for special education services for the over half a million students with autism in the United States. While assistive technology may help to augment educational services, their implementation is prevented by a number of practical and attitudinal barriers. These barriers are especially pertinent for the newest and least familiar digital systems, such as computerized smartglasses loaded with specialized software modules, a technology that has already been shown to have an ability to deliver educational interventions through augmented reality. With this in mind, we sought to understand how school educators received and assessed the practicality of a smartglasses-based educational intervention in a single-subject study. The intervention is designed to aid with attention and social educational learning in autism. The intervention was delivered twice a day during a two-week study on a 13-year-old student with autism who attends a mainstream middle school in Massachusetts. Three different school educators delivered the intervention: the student’s general education teacher, special education teacher, and paraprofessional. Educators recorded their attitudes, practicality of the technology, and impact on the student and their classroom through the use of a digital log and a series of in-person interviews. Overall, the school educators experienced a positive view of the smartglasses. The smartglasses intervention was found to be logistically practical to implement, easily usable by educator and student, and was not time consuming to learn or to implement. Educators also identified the experience as being fun for the student, and felt the student demonstrated improvement to verbal and non-verbal skills. There were no adverse effects on the other students or the classroom, and the technology did not result in a distraction. These findings suggest that social skills interventions delivered by smartglasses may be practical, useful, and may lead to improvements in social communication skills. Further research on smartglasses may help to clarify the future role for augmenting special education in students with autism.
Introduction: Acute myeloid leukemia (AML) poses significant economic burden on the healthcare system, particularly during induction therapy and at disease relapse. The burden associated with ongoing post-remission therapy is less clear. In patients diagnosed with AML enrolled in Medicare who received an induction therapy and achieved disease remission, we assessed the healthcare resource utilization (HCRU) and costs associated with each disease period (induction, early/late post-remission, and post-relapse). Methods: A retrospective analysis was conducted using the Surveillance, Epidemiology, and End Results (SEER)-Medicare database, comprising Medicare claims (parts A, B, and D from 2007 to 2016) and the US National Cancer Institute's SEER database (cancer diagnoses from 2007 to 2015). Identified patients had a diagnosis of AML in the SEER registry, were ≥ 65 years at the AML diagnosis date, initiated chemotherapy post-AML diagnosis (i.e. induction), and had an International Classification of Diseases, Ninth/Tenth Revision (ICD-9/10) diagnosis code for AML remission following the start of therapy. Patients were excluded if they had another blood malignancy, had received a prior hematopoietic stem cell transplant, or were enrolled in a clinical trial. Induction was defined as any therapy received from the date of first post-diagnosis chemotherapy initiation (index date) to the end of the cycle during which a patient had an ICD-9/10 code for AML remission. The 6 months prior to the index date was defined as the baseline period. Post-remission therapy was divided into an early post-remission period, which included any therapy initiated within the first 60 days (≤ 60 d) after end of induction, and a late post-remission period, which included therapy initiated more than 60 days (> 60 d) after end of induction. If specific treatment information was available, late post-remission therapy was defined by a treatment switch occurring > 60 d after end of induction. Post-remission therapy ended at the earliest of relapse or end of follow-up (i.e., death, end of eligibility, or end of available data [December 31, 2016]). The post-relapse period was from the date of first AML relapse ICD-9/10 code after remission to the end of follow-up. Baseline patient characteristics, as well as HCRU and costs (adjusted to 2019 US dollars) during the baseline, induction, post-remission, and post-relapse periods, were summarized descriptively. HCRU and costs associated with induction and post-remission therapy periods were assessed during days that were part of a treatment cycle. The average per patient per month (PPPM) HCRU and costs were reported. Duration of response (DoR) from the first remission to the earliest of relapse or death was estimated using Kaplan-Meier analysis. Results: A total of 530 patients were identified. The median age at AML diagnosis was 73 years, 53.6% of patients were male, and 80.6% were white. The median time from index date to the end of follow-up was 13.5 months. Most patients received therapy with hypomethylating agents during the AML treatment. A total of 31.9% of patients who achieved remission did not receive post-remission therapy during follow-up; for these patients, mean (median) time from end of induction to relapse or end of follow-up was 125 (23) days. A total of 63.2% of patients received chemotherapy in the early post-remission period, and 43.0% of all patients went on to receive chemotherapy in the late post-remission period. The median DoR was 5.8 months; a total of 48.9% of patients had relapsed and 80.2% had died by the end of follow-up. The mean PPPM healthcare costs were highest for induction, followed by post-relapse, early post-remission, and late post-remission periods (Table). Costs associated with the inpatient (IP) setting were the greatest contributor to PPPM costs across all periods. IP visits were most common during induction with 92.1% of patients having ≥ 1 IP visit, relative to 53.8% during baseline, 65.7% during early post-remission, 71.5% during late post-remission, and 91.1% post-relapse. Conclusions: The economic burden of relapse is approximately 1.2 and 1.6 times higher than the mean PPPM healthcare costs during early and late post-remission periods, respectively. There exists a large unmet need for therapies that will extend the duration of the post-remission period and reduce the overall economic burden of AML. Disclosures Tabah: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Huggar:Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company; Karyopharm Therapeutics: Current equity holder in publicly-traded company; FibroGen: Current equity holder in publicly-traded company. Huey:Bristol Myers Squibb: Current Employment. Copher:Bristol Myers Squibb: Current Employment. Zhou:BMS: Other: Employee of Analysis Group Inc., which received consulting fees. Zichlin:BMS: Other: Employee of Analysis Group Inc., which received consulting fees. Koenigsberg:BMS: Other: Employee of Analysis Group Inc., which received consulting fees. Brunner:Novartis: Research Funding; Celgene: Membership on an entity's Board of Directors or advisory committees, Research Funding; Takeda: Research Funding; AstraZeneca: Research Funding; Forty-Seven Inc: Membership on an entity's Board of Directors or advisory committees; Jazz Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees.
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