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Introduction: Payment for oncology care is increasingly moving from fee-for-service to value-based payment (VBP). VBPs are agreements in which providers are held accountable for total cost of care (TCOC) through risk-sharing arrangements with payers that tie reimbursement levels to TCOC benchmarks. Oncology biosimilars may play an important role in managing financial risk in the VBPs like Medicare's Oncology Care Model (OCM), but there has been limited research in this area. The objective of this study is to estimate the impact of biosimilar adoption on TCOC and oncology provider financial performance under the terms of the Medicare OCM. Methods: We conducted a population-based simulation study using the Medicare Limited Data Set (LDS) and the methodology of Medicare's OCM. The primary outcome was the simulated average change in TCOC per 6-month episode of care attributable to use of biosimilars as an alternative to reference products. The study population consisted of episodes of care in 2020 and using the reference product or corresponding biosimilar for bevacizumab, rituximab, trastuzumab, epoetin alfa, filgrastim, or pegfilgrastim. TCOC was calculated for each episode of care with use of reference products only and compared with TCOC with corresponding biosimilars. The simulation calculated TCOC outcomes in cohorts of 100 episodes sampled from the Medicare LDS study population using a Monte Carlo simulation with 10,000 iterations. Results: Among the total of 8281 6-month oncology care episodes identified in the study period (initiating January 2020 to July 2020) in Medicare claims, 1586 (19.2%) episodes met OCM and study criteria and were included. Applying the simulation methods to these observed episodes, biosimilar substitution reduced mean TCOC per episode by $1193 (95% CI $583–1840). The cost reduction from biosimilars represented 2.4% of the average TCOC benchmark and led to a 15% reduction in the risk of providers needing to pay recoupments to Medicare for exceeding TCOC benchmarks. Conclusions: On the basis of our simulation study using observed Medicare claims and OCM criteria, we found that biosimilar substitution for reference products can significantly lower episode TCOC and improve provider financial performance under the terms of the largest value-based payment model implemented to date. [ABSTRACT FROM AUTHOR]

Abstract Background Patients with preoperative anemia have a higher risk of requiring blood transfusion after major orthopedic surgery due to increased blood loss and closer transfusion thresholds. Various patient blood management (PBM) policies aim to reduce transfusion rates. This observational study aimed to investigate blood loss and evaluate the effectiveness of an adjusted surgical PBM protocol in patients with anemic chronic disease (ACD) undergoing elective total knee arthroplasty (TKA). Methods A consecutive cohort of patients underwent elective unilateral TKA with an adjusted PBM protocol. The protocol consisted of epoetin (EPO) alfa therapy prescribed by the surgeon, routine administration of tranexamic acid (TXA), and standardized postoperative pharmacologic prophylaxis for thromboembolism. The performance of this PBM protocol was analyzed in patients with a baseline hemoglobin level of less than 12 g/dl. Hemoglobin levels were controlled at admission, on postoperative day (POD) 1, and on POD 7 ± 1. A bleeding index (BI-7) was used as an estimate of blood loss up until POD 7. Multiple linear regression was used to assess whether there were any differences in BI-7 between ACD– and ACD + patients. Results A total of 751 patients with complete hemoglobin monitoring were included in the study. Of these patients, 68 (9.1%) had a baseline hemoglobin concentration of less than 12 g/dl (ACD group). In this group, 28 patients (41.2%) received preoperative EPO therapy. The mean adjusted BI-7 for the study population was 3.0 (95% CI, 2.9 to 3.0) g/dl in the ACD– group and 2.3 (95% CI, 2.0–2.6) g/dl in the ACD + group. The difference in BI-7 was statistically significant (difference, 0.6 [95% CI: 0.3 to 0.9] g/dl, p

Background: Patients with preoperative anemia have a higher risk of requiring blood transfusion after major orthopedic surgery due to increased blood loss and closer transfusion thresholds. Various patient blood management (PBM) policies aim to reduce transfusion rates. This observational study aimed to investigate blood loss and evaluate the effectiveness of an adjusted surgical PBM protocol in patients with anemic chronic disease (ACD) undergoing elective total knee arthroplasty (TKA). Methods: A consecutive cohort of patients underwent elective unilateral TKA with an adjusted PBM protocol. The protocol consisted of epoetin (EPO) alfa therapy prescribed by the surgeon, routine administration of tranexamic acid (TXA), and standardized postoperative pharmacologic prophylaxis for thromboembolism. The performance of this PBM protocol was analyzed in patients with a baseline hemoglobin level of less than 12 g/dl. Hemoglobin levels were controlled at admission, on postoperative day (POD) 1, and on POD 7 ± 1. A bleeding index (BI-7) was used as an estimate of blood loss up until POD 7. Multiple linear regression was used to assess whether there were any differences in BI-7 between ACD– and ACD + patients. Results: A total of 751 patients with complete hemoglobin monitoring were included in the study. Of these patients, 68 (9.1%) had a baseline hemoglobin concentration of less than 12 g/dl (ACD group). In this group, 28 patients (41.2%) received preoperative EPO therapy. The mean adjusted BI-7 for the study population was 3.0 (95% CI, 2.9 to 3.0) g/dl in the ACD– group and 2.3 (95% CI, 2.0–2.6) g/dl in the ACD + group. The difference in BI-7 was statistically significant (difference, 0.6 [95% CI: 0.3 to 0.9] g/dl, p < 0.001). No major complications occurred in the ACD + group, whereas there were three complications in the ACD– group (p = 1.00). Conclusions: ACD patients undergoing TKA did not have an increased risk of bleeding or bleeding complications with the use of the adjusted PBM protocol. None of ACD patients required transfusion. ACD patients undergoing TKA experienced significantly less blood loss than patients with no preoperative anemia with the use of the adjusted PBM. We now consider the use of EPO in ACD patients with no nutritional deficiencies undergoing TKA at a baseline hemoglobin level of 11 g/dl. However, this should be validated in larger cohorts with a higher prevalence of ACD patients. [ABSTRACT FROM AUTHOR]

Objective: This study was undertaken to evaluate the long-term clinical efficacy of epoetin alfa and darbepoetin alfa in patients with myelodysplastic syndrome (MDS) in a real-life setting. Materials and Methods: A total of 204 patients with low-risk or intermediate-1-risk MDS who received epoetin alfa or darbepoetin alfa were included. Hemoglobin levels and transfusion needs were recorded before treatment and at 12 months, 24 months, 36 months, and 48 months of treatment. Results: At the 36-month (p=0.025) and 48-month (p=0.022) visits, epoetin alfa yielded significantly higher hemoglobin levels compared to darbepoetin alfa. Transfusion needs were also significantly lower with epoetin alfa compared to darbepoetin alfa at 24 months (p=0.012) and in the low-risk group compared to the intermediate-risk group at 24 months (p=0.018), 36 months (p=0.025), and 48 months (p

BackgroundRecent advances in genomics have enabled the successful identification of a number of rare pathogenic mutations. Uncovering these mutations is essential as the first step towards devising a cure for the often debilitating and life-limiting diseases arising from them. For many of these mutations targeted agents do not yet exist. Here, we describe the case of a patient who has a novel pathogenic mutation in the erythropoietin (EPO) gene, which is essential for normal erythropoiesis, and who presented with a profound hypoplastic anemia.MethodsThe patient aged 5 months, was started on recombinant erythropoietin, at a standard dose of 500 units (50 U/kg) and subsequently 800 units three time weekly and her blood counts were monitored over 4 years.FindingsA prompt response to the recombinant erythropoietin was found with an increase in hemoglobin levels to 12.8 g/dL and increase in red cell count to 4.89×106/uL. The patient became transfusion independent. The therapy enabled the patient to maintain a hemoglobin level in the normal range without any adverse effects and with no requirement for further blood transfusions.ConclusionsPatient-customized therapies can be highly effective in the treatment of rare genetic disorders and for many of these disorders effective treatment may already exist in the clinical domain, as described for the patient in this report.FundingThis work was supported by the New York Stem Cell Foundation (V.G.S.), a gift from the Lodish Family to Boston Children's Hospital (V.G.S.), and National Institutes of Health Grants R01 DK103794 and R01 HL146500 (V.G.S.).

Background and purposePerinatal stroke is a common cause of life-long neurobehavioral compromise. Mesenchymal stromal cells (MSCs) and EPO (erythropoietin) have each demonstrated short-term benefit with delayed administration after stroke, and combination therapy may provide the most benefit. The purpose of this study is to determine the long-term histological and functional efficacy of enhanced, intranasal stem cell therapy (MSC preexposed to EPO) compared with standard MSC or multidose systemic EPO.MethodsTransient middle cerebral artery occlusion or sham surgery was performed in postnatal day (P) 10 Sprague-Dawley rats, who were treated with single-dose intranasal MSC, MSC preexposed to EPO (MSC/EPO), multidose systemic EPO (EPO3; 1000 u/kg per dose×3 every 72 hours), or cell-conditioned media on P13 (day 3 [P13-P19] for EPO), or on P17 (day 7 [P17-P23] for EPO). At 2 months of age, animals underwent novel object recognition, cylinder rearing, and open field testing to assess recognition memory, sensorimotor function, and anxiety in adulthood.ResultsMSC, MSC/EPO, and EPO3 improved brain volume when administered at 3 or 7 days after middle cerebral artery occlusion. MSC/EPO also enhanced long-term recognition memory with either day 3 or day 7 treatment, but EPO3 had the most long-term benefit, improving recognition memory and exploratory behavior and reducing anxiety.ConclusionsThese data suggest that single-dose MSC/EPO and multidose systemic EPO improve long-term neurobehavioral outcomes even when administration is delayed, although EPO was the most effective treatment overall. It is possible that EPO represents a final common pathway for improved long-term repair, although the specific mechanisms remain to be determined.

Summary: Background: Pegmolesatide, a synthetic peptide-based erythropoietin (EPO) receptor agonist, is being evaluated as an alternative to epoetin alfa for treating anemia of chronic kidney disease (CKD) in Chinese dialysis patients. There is a critical need for a long-acting, cost-effective erythropoiesis-stimulating agent that does not produce EPO antibodies. Methods: A randomized, open-label, active-comparator, non-inferiority phase three trial was conducted at 43 dialysis centers in China between May 17th, 2019, and March 28th, 2022. Eligible patients aged 18–70 years were randomly assigned (2:1) to receive pegmolesatide once every four weeks or epoetin alfa one to three times per week, with doses adjusted to maintain a hemoglobin level between 10.0 and 12.0 g/dL. The primary efficacy endpoint was the mean change in hemoglobin level from baseline to the efficacy evaluation period in the per-protocol set (PPS) population. Non-inferiority of pegmolesatide to epoetin alfa was established if the lower limit of the two-sided 95% confidence interval for the between-group difference was ≥ −1.0 g/dL. Safety assessment included adverse events and potential anaphylaxis reactions. This trial is registered at ClinicalTrials.gov, NCT03902691. Findings: Three hundreds and seventy-two patients were randomly assigned to the pegmolesatide group (248 patients) or the epoetin alfa group (124 patients). A total of 347 patients (233 in the pegmolesatide group and 114 in the epoetin alfa group) were included in the PPS population. In the PPS, the mean change (standard deviation, SD) in hemoglobin level from baseline to the efficacy evaluation period was 0.07 (0.92) g/dL in the pegmolesatide group and −0.22 (0.97) g/dL in the epoetin alfa group. The between-group difference was 0.29 g/dL (95% confidence interval: 0.11–0.47), verifying non-inferiority of pegmolesatide to epoetin alfa. Adverse events occurred in 231 (94%) participants in the pegmolesatide group and in 110 (89%) in the epoetin alfa group. Hypertension was the most common treatment-related adverse event. No fatal cases of anaphylaxis or hypotension were reported. Interpretation: Monthly subcutaneously injection of pegmolesatide was as effective and safe as conventional epoetin alfa administrated one to three times a week in treating anemia in Chinese dialysis patients. Funding: The study was supported by Hansoh Medical Development Group.