Deciphering treatment patterns in non-severe/moderate aplastic anemia: an international observational study.

Non-severe aplastic anemia is a rare bone marrow failure disorder characterized by variable degrees and combination of cytopenias, with limited data on management and outcome. We describe a large multicentric series of 259 patients, focusing on clinical and molecular features, treatment, evolution, and survival. The majority required treatment with cyclosporine (CyA) alone (N = 84) or in combination with anti-thymocyte globulin (ATG,44) or eltrombopag (20), eltrombopag alone (10), or others (25) including androgens. Similar outcomes were observed across different strategies, with a 6-month overall response rate of 73% for CyA, 74% for ATG plus CyA, 68% for CyA plus eltrombopag, 87% for eltrombopag, and 79% for others. Notably, 56 patients (39%), mainly receiving CyA plus eltrombopag, achieved a trilineage response (p = 0.02). Progression to myeloid neoplasms was limited (8%) and not related to mutational status. Hemolytic PNH developed in 10% of cases, being predicted by detection of small clones at diagnosis. Survival was negatively impacted by age, male gender, LDH, platelets/erythrocyte transfusion need, and somatic mutations by NGS, and positively by higher neutrophils at diagnosis, PNH clones, and trilineage response at 6 and 12 months. Multivariable analysis confirmed the detrimental role of age and the favorable association with PNH clone and trilineage response at 6 months.

Primary intraventricular hemorrhage outcomes in the CLEAR III trial.

BACKGROUND: Intraventricular hemorrhage occurs due to intracerebral hemorrhage with intraventricular extension or without apparent parenchymal involvement, known as primary intraventricular hemorrhage. AIMS: We evaluated the prognosis of primary intraventricular hemorrhage patients in the CLEAR III trial (Clot Lysis: Evaluating Accelerated Resolution of Intraventricular Hemorrhage). METHODS: In patients with primary intraventricular hemorrhage versus those with secondary intraventricular hemorrhage, we compared intraventricular alteplase response and outcomes including modified Rankin Scale, Barthel Index, National Institutes of Health Stroke Scale (NIHSS), and extended Glasgow Outcome Scale (eGOS) at 30, 180, and 365 days. Outcomes were also compared in primary intraventricular hemorrhage patients who received intraventricular alteplase versus placebo (normal saline) and in matched primary and secondary intraventricular hemorrhage patients using inverse-probability-weighted regression adjustment. RESULTS: Of 500 patients enrolled in CLEAR III, 46 (9.2%) had primary intraventricular hemorrhage. Combining both treatment groups, primary intraventricular hemorrhage patients had larger intraventricular hemorrhage volumes (median: 34.2 mL vs. 20.8 mL, p < 0.01) but similar intraventricular hemorrhage removal (51.0% vs. 59.0%, p = 0.24) compared to secondary intraventricular hemorrhage patients, respectively. Confirming previous studies, primary intraventricular hemorrhage patients achieved better NIHSS, modified Rankin Scale, Barthel Index, and eGOS scores at days 30, 180, and 365, respectively (all p < 0.01), although mortality was similar to secondary intraventricular hemorrhage patients; matching analysis yielded similar results. Primary intraventricular hemorrhage patients who received intraventricular alteplase (n = 19) and saline (n = 27) achieved similar outcomes. CONCLUSIONS: In CLEAR III, primary intraventricular hemorrhage patients who survived achieved better long-term outcomes than surviving secondary intraventricular hemorrhage patients with similar mortality. Outcomes and safety were similar between primary intraventricular hemorrhage patients receiving alteplase and those receiving saline.

School Closure Decisions Made by Local Health Department Officials During the 2009 H1N1 Influenza Outbreak.

OBJECTIVES: The goal of this study was to gain insights into the decision-making processes used by California public health officials during real-time crises. The decision-making processes used by California public health officials during the 2009 H1N1 influenza pandemic were examined by a survey research team from the University of California Berkeley. METHODS: The survey was administered to local public health officials in California. Guidelines published by the Centers for Disease Control and Prevention had recommended school closure, and local public health officials had to decide whether to follow these recommendations. Chi-squared tests were used to make comparisons in the descriptive statistics. RESULTS: The response rate from local public health departments was 79%. A total of 73% of respondents were involved in the decision-making process. Respondents stated whether they used or did not use 15 ethical, logistical, and political preselected criteria. They expressed interest in receiving checklists and additional training in decision-making. CONCLUSIONS: Public health decision-makers do not appear to have a standard process for crisis decision-making and would benefit from having an organized decision-making model. The survey showed that ethical, logistical, and political criteria were considered but were not prioritized in any meaningful way. A new decision-making tool kit for public health decision-makers plus implementation training is warranted.