Relationship between Serum Ferritin and Outcomes in ß-Thalassemia: A Systematic Literature Review.

Among the difficulties of living with ß-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 ß-thalassemia.

The impact of remission duration on the long-term economic burden of acute myeloid leukemia among patients without hematopoietic stem cell transplant in the United States.

BACKGROUND AND AIMS: Acute myeloid leukemia (AML) prognosis is poor, with sustained remission occurring in <35% of young adults and <15% of older adults. This descriptive study examined the potential benefit of prolonged remission on the economic burden of AML. METHODS: Using the IBM MarketScan Commercial and Medicare Supplemental databases, we identified newly diagnosed patients with AML without hematopoietic stem cell transplantation from January 1, 2012 to December 31, 2018; AML diagnosis was the index date. Patients had 6 months of pre-index eligibility and were followed until the end of continuous eligibility, study data, or death. Active treatment and supportive care cohorts were defined; duration-of-remission subgroups (0 to <3, 3 to <6, 6 to <12, and ≥12 months) were established among active treatment patients with remission. Healthcare service utilization and costs were reported over follow-up and mutually exclusive treatment, remission, and post-relapse periods. RESULTS: This study included 1,558 active treatment and 1,127 supportive care patients who were followed for a median of 232 and 62 days, respectively. Over follow-up, active treatment and supportive care patients incurred mean ± standard deviation all-cause healthcare costs of $55,723 ± $61,994 and $68,596 ± $100,375 per-patient-per-month (PPPM), respectively. Decreasing PPPM costs were observed with increased remission duration (0 to <3 months: $71,823 ± $62,635; 3 to <6 months: $54,262 ± $44,734; 6 to <12 months: $35,287 ± $23,699; and ≥12 months: $15,615 ± $10,560). Although median follow-up varied by up to 5-fold, total costs were largely similar across duration-of-remission subgroups (0 to <3 months: $438,569 ± $332,675; 3 to <6 months: $590,411 ± $598,245; 6 to <12 months: $482,902 ± $369,115; and ≥12 months: $448,867 ± $316,133). CONCLUSIONS: The economic burden of AML is substantial, even among untreated patients. Further, among patients with remission, longer durations in remission are associated with reduced PPPM healthcare costs, suggesting that remission-prolonging treatments could help mitigate healthcare costs.

A Systematic Literature Review of the Relationship between Serum Ferritin and Outcomes in Myelodysplastic Syndromes.

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.

Improved Socio-Emotional and Behavioral Functioning in Students with Autism Following School-Based Smartglasses Intervention: Multi-Stage Feasibility and Controlled Efficacy Study.

Background: Students with Autism Spectrum Disorder (ASD) commonly demonstrate prominent social communication deficits, symptoms of attention-deficit/hyperactivity disorder, and chronic irritability. These challenges hinder academic progress and frequently persist despite educational, behavioral, and medical interventions. An assistive smartglasses technology may aid these individuals, especially if the technology is efficacious in ecologically-valid school settings. This study explored the feasibility and efficacy of Empowered Brain, a computerized smartglasses intervention designed as a socio-emotional behavioral aid for students with ASD. Methods: This two-part six-week study involved four school children with ASD from a public elementary school. The study incorporated an initial three-week feasibility stage followed by a three-week controlled longitudinal efficacy stage. Both stages involved the use of a twice-daily socio-emotional intervention with the smartglasses. Educators completed pre-intervention and post-intervention Aberrant Behavioral Checklist (ABC) ratings at the start of the feasibility stage, and weekly during the efficacy stage. Primary outcome measures were improvements in the ABC subscales of irritability, hyperactivity, and social withdrawal. Results: Students in both feasibility and efficacy stages demonstrated improvements (decreases) in irritability, hyperactivity, and social withdrawal compared to a baseline period and control periods, respectively. Participants in the controlled efficacy stage demonstrated decreased ABC subscale scores of 90% for irritability, 41.6% for hyperactivity, and 45.6% for social withdrawal. An intervention exposure-response improvement in irritability and hyperactivity was found during the efficacy stage. Educators rated the technology as superior or vastly superior compared to other assistive technologies. Conclusion: A substantial number of school children with ASD demonstrate chronic and impairing cognitive and behavioral challenges. This study provides evidence that Empowered Brain, a smartglasses-based socio-emotional aid for autism, is both feasible and efficacious in improving symptoms of social withdrawal, irritability, and hyperactivity in students with autism. The improvement is demonstrated as part of a longitudinal school-based intervention. Further studies involving larger samples and incorporation of randomized controlled trial methodology are underway to further elucidate the impact of this technology.