IntroductionFrequent infusions and bleeds can impact on the health‐related quality of life (HRQoL) of paediatric haemophilia B patients. rIX‐FP (IDELVION®) is a fusion protein linking recombinant factor IX with recombinant albumin, and is associated with low bleeding rates with a weekly regimen, which could improve HRQoL.AimsTo measure the effect of rIX‐FP prophylaxis on the HRQoL of paediatric patients and treatment satisfaction in their caregivers using the Haemo‐QoL and Hemo‐SATP questionnaires, respectively.MethodsAt baseline and end‐of‐study (EOS), patients 4‐11 years old participating in the PROLONG‐9FP program answered the Haemo‐QoL questionnaire and gave information on their socio‐demographic data and physical activity. Caregivers completed the Hemo‐SatP. Minimal important differences (MID) (|Cohen's d| > 0.5) between baseline and EOS and the number of responders (patients with meaningful subject‐level improvements over time) at EOS were calculated.ResultsTwenty patients (age group I: 4‐7 years old [n = 12]; age group II: 8‐12 years old [n = 8]) completed the Haemo‐QoL questionnaire at baseline. MIDs were found in age group I representing improvement for “physical health” (d = −0.547) domain; 60% of patients were responders for “physical health.” In age group II, MIDs were seen in most domains; 71.4% patients were responders in “total score.” In caregivers, improvements were seen for most domains of the Hemo‐SatP with a small effect size. Fewer patients missed school when treated with rIX‐FP and 94.1% patients maintained their physical activity level.ConclusionProphylaxis with rIX‐FP led to substantial improvements in HRQoL in paediatric patients and treatment satisfaction in caregivers.

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A previously healthy 36-year-old woman presented at the emergency department with gradual-onset confusion, ataxia, and aphasia. Her vital signs were normal. On physical examination, she reacted to painful stimuli but was nonverbal and unable to follow commands. Initial laboratory results revealed no abnormalities. Computed tomograms of the head and results of a lumbar puncture were nondiagnostic. During hospitalization, the patient decompensated and needed emergency intubation and vasopressor support. An electrocardiogram (ECG) was obtained ( Fig. 1). An echocardiogram revealed an acute reduction of left ventricular ejection fraction (range, 0.35-0.40) and anterior wall-motion abnormalities. Notable laboratory results included troponin I, 17.1 ng/mL; normal thyroid values; and negative toxicology screening. Which of the following diagnoses explains the ECG?A) Supraventricular tachycardia with aberrant conduction B) Bidirectional ventricular tachycardia C) Drug toxicity D) Accelerated junctional rhythm

ST-elevation myocardial infarction (STEMI) is a clinical diagnosis based on a compatible history and characteristic electrocardiographic changes. In the current era, STEMI is treated emergently with angiography, leading to percutaneous coronary intervention. However, false-positive electrocardiograms (ECGs) occur, resulting in unnecessary emergent catheterizations. We hypothesized that the Vectraplex cardiac electrical biomarker (CEB) would increase the specificity for the diagnosis of STEMI. We studied 50 patients who were identified by standard of care (clinical history, physical exam, and 12-lead ECG) as suspected to have STEMI and tested the sensitivity and specificity of the Vectraplex ECG system. Using the final clinical diagnosis (based on history, ECGs, troponin values, and angiographic findings) as the gold standard, we found the CEB value to be quite dynamic, with a reasonable sensitivity and a good positive predictive value but generally poor specificity and negative predictive value. It offered only a 20% improvement compared to 50-50 performance on receiver operator curves.