A preliminary study of roxadustat in the treatment of aplastic anemia patients with inadequate erythroid responses.

Some aplastic anemia(AA) patients only have partial hematological responses after immunosuppressive therapy. Failure to achieve complete normalization of blood counts, particularly hemoglobin, will reduce their quality of life. This open-label pilot study was conducted to evaluate the efficacy and safety of roxadustat in this setting. A total of 14 patients with AA who had inadequate erythroid response after immunosuppressive therapy were included in the study. The primary efficacy endpoint was hemoglobin response at week 8 after roxadustat treatment. The median duration of roxadustat therapy was 14 (4-30) weeks, with 12 patients receiving roxadustat for ≥ 8 weeks. At week 8, nine patients (9/14, 64.3%) had their hemoglobin rising for at least 15 g/L, with two patients (2/14, 14.3%) achieving normal hemoglobin levels. By the last follow-up, hemoglobin responses were observed in 10 patients (10/14, 71.4%), with 4 patients(4/14, 28.6%) having normal hemoglobin levels. Roxadustat was tapered or discontinued in four responded patients; one relapsed after 12 weeks of tapering, and three maintained their response. Four patients (4/14, 28.6%) experienced mild adverse effects during therapy. Roxadustat is safe and well tolerated by patients with AA. Treatment with the hypoxia-inducible factor prolyl hydroxylase inhibitor improves hemoglobin levels in AA patients with inadequate erythroid responses.

Prognostic Impact of Early Administration of ß-Blockers in Critically Ill Patients with Acute Myocardial Infarction.

In critically ill patients with acute myocardial infarction (AMI), the relationship between the early administration of ß-blockers and the risks of in-hospital and long-term mortality remains controversial. Furthermore, there are conflicting evidences for the efficacy of the early administration of intravenous followed by oral ß-blockers in AMI. We conducted a retrospective analysis of critically ill patients with AMI who received the early administration of ß-blockers within 24 hours of admission. The data were extracted from the Medical Information Mart for Intensive Care IV database. We enrolled 2467 critically ill patients with AMI in the study, with 1355 patients who received the early administration of ß-blockers and 1112 patients who were non-users. Kaplan-Meier survival analysis and Cox proportional hazards models showed that the early administration of ß-blockers was associated with a lower risk of in-hospital mortality (adjusted hazard ratio [aHR] 0.52; 95% confidence interval [95%CI] 0.42-0.64), 1-year mortality (aHR 0.54, 95%CI 0.47-0.63), and 5-year mortality (aHR 0.60, 95%CI 0.52-0.69). Furthermore, the early administration of both oral ß-blockers and intravenous ß-blockers followed by oral ß-blockers may reduce the mortality risk, compared with non-users. The risks of in-hospital and long-term mortality were significantly decreased in patients who underwent revascularization with the early administration of ß-blockers. We found that the early administration of ß-blockers could lower the risks of in-hospital and long-term mortality. Furthermore, the early administration of both oral ß-blockers and intravenous ß-blockers followed by oral ß-blockers may reduce the mortality risk, compared with non-users. Notably, patients who underwent revascularization with the early administration of ß-blockers showed the lowest risks of in-hospital and long-term mortality.

Prognostic value of red blood cell distribution width combined with chloride in predicting short- and long-term mortality in critically ill patients with congestive heart failure: Findings from the MIMIC-IV database.

Background: Hypochloremia and red blood cell distribution width (RDW) play important roles in congestive heart failure (CHF) pathophysiology, and they were associated with the prognosis of CHF. However, the prognostic value of chloride combined with RDW in patients with CHF remains unknown. Methods: We retrospectively analyzed critically ill patients with CHF. The database was derived from the Medical Information Mart for Intensive Care IV v2.0 (MIMIC-IV-v2.0) database. Results: In the final analysis, 5376 critically ill patients with CHF were included, and 2428 patients (45.2 %) experienced 5-year mortality. The restricted cubic spline model revealed a positive correlation between RDW and 5-year mortality, whereas chloride showed a U-shaped correlation with 5-year mortality. The median values of RDW and chloride were used to classify patients into four groups: high chloride/low RDW, low chloride/low RDW, high chloride/high RDW, and low chloride/high RDW. We observed the prognostic value of RDW combined with chloride in the Cox proportional hazard model, in predicting 5-year mortality, in-hospital mortality and 1-year mortality. Furthermore, we discovered that patients with chronic kidney disease (CKD) had a higher 5-year mortality risk than patients without CKD. Conclusion: We found the translational potential role of chloride combined with RDW in prioritizing patients at high risk for short- and long-term mortality in a cohort of critically ill patients with CHF. Prospective multicenter investigations are warranted to validate our results.

The elevation of red blood cell distribution width is an independent prognostic factor for juvenile myelomonocytic leukemia.

Juvenile myelomonocytic leukemia (JMML) is a disorder characterized by the simultaneous presence of myeloproliferative and myelodysplastic features, primarily affecting infants and young children. Due to the heterogeneous genetic background among patients, the current clinical and laboratory prognostic features are insufficient for accurately predicting outcomes. Thus, there is a pressing need to identify novel prognostic indicators. Red cell distribution width (RDW) is a critical parameter reflecting the variability in erythrocyte size. Recent studies have emphasized that elevated RDW serves as a valuable predictive marker for unfavorable outcomes across various diseases. However, the prognostic role of RDW in JMML remains unclear. Patients with JMML from our single-center cohort between January 2008 and December 2019 were included. Overall, 77 patients were eligible. Multivariate Cox proportional hazard models showed that patients with red cell distribution width coefficient of variation (RDW-CV) >17.35% at diagnosis were susceptible to much worse overall survival rate (hazard ratio [HR] = 5.22, confidence interval [CI] = 1.50-18.21, P = .010). Besides, the combination of RDW elevation and protein phosphatase non-receptor type 11 (PTPN11) mutation was likely to predict a subgroup with the worst outcomes in our cohort. RDW is an independent prognostic variable in JMML subjects. RDW may be regarded as an inexpensive biomarker to predict the clinical outcome in patients with JMML.

Relationship between stress hyperglycemia ratio and allcause mortality in critically ill patients: Results from the MIMIC-IV database.

Background: Stress hyperglycemia ratio (SHR) was developed to reduce the impact of long-term chronic glycemic factors on stress hyperglycemia levels, which have been linked to clinical adverse events. However, the relationship between SHR and the short- and long-term prognoses of intensive care unit (ICU) patients remains unclear. Methods: We retrospectively analyzed 3,887 ICU patients (cohort 1) whose initial fasting blood glucose and hemoglobin A1c data within 24 hours of admission were available and 3,636 ICU patients (cohort 2) who were followed-up for 1-year using the Medical Information Mart for Intensive Care IV v2.0 database. Patients were divided into two groups based on the optimal cutoff value of SHR, which was determined using the receiver operating characteristic (ROC) curve. Results: There were 176 ICU deaths in cohort 1 and 378 patients experienced all-cause mortality during 1 year of follow-up in cohort 2. The results of logistic regression revealed that SHR was associated with ICU death (odds ratio 2.92 [95% confidence interval 2.14-3.97] P < 0.001), and non-diabetic patients rather than diabetic patients showed an increased risk of ICU death. As per the Cox proportional hazards model, the high SHR group experienced a higher incidence of 1-year all-cause mortality (hazard ratio 1.55 [95% confidence interval 1.26-1.90] P < 0.001). Moreover, SHR had an incremental effect on various illness scores in predicting ICU all-cause mortality. Conclusion: SHR is linked to ICU death and 1-year all-cause mortality in critically ill patients, and it has an incremental predictive value in different illness scores. Moreover, we found that non-diabetic patients, rather than diabetic patients, showed an increased risk of all-cause mortality.