Essentials• rVIII-SingleChain is a novel recombinant factor VIII with covalently bonded heavy and light chains.• Efficacy, safety and pharmacokinetics were studied in pediatric patients with severe hemophilia A.• Across all prophylaxis regimens, the median annualized spontaneous bleeding rate was 0.00.• rVIII-SingleChain showed excellent hemostatic efficacy and a favorable safety profile.Summary. Background: rVIII-SingleChain is a novel B-domain truncated recombinant factor VIII (rFVIII) comprised of covalently bonded FVIII heavy and light chains, demonstrating a high binding affinity to von Willebrand factor. Objectives: This phase III study investigated the safety, efficacy and pharmacokinetics of rVIIISingleChain in previously treated pediatric patients < 12 years of age with severe hemophilia A. Patients/ Methods: Patients could be assigned to prophylaxis or ondemand therapy by the investigator. For patients assigned to prophylaxis, the treatment regimen and dose were based on the bleeding phenotype. For patients receiving on-demand therapy, dosing was guided by World Federation of Hemophilia recommendations. The primary endpoint was treatment success, defined as a rating of 'excellent' or 'good' on the investigator's clinical assessment of hemostatic efficacy for all treated bleeding events. Results: The study enrolled 84 patients (0 to < 6 years, n = 35; ≥ 6 to < 12 years, n = 49); 81 were assigned to prophylaxis and three to an on-demand regimen. Patients accumulated a total of 5239 exposure days (EDs), with 65 participants reaching > 50 EDs. In the 347 bleeds treated and evaluated by the investigator, hemostatic efficacy was rated as excellent or good in 96.3%. The median annualized spontaneous bleeding rate was 0.00 (Q1, Q3: 0.00, 2.20), and the median annualized bleeding rate was 3.69 (Q1, Q3: 0.00, 7.20) across all prophylaxis regimens. No participant developed an inhibitor. Conclusions: rVIII-SingleChain is a novel rFVIII molecule showing excellent hemostatic efficacy and a favorable safety profile in a clinical study in children < 12 years of age with severe hemophilia A.

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BackgroundHeated tobacco products (HTP) generate nicotine-containing aerosol by heating tobacco rather than burning it. The US Food and Drug Administration (FDA) has recently authorised the sale of one HTP brand, iQOS, in the USA. This study examined the awareness, use and risk perceptions of HTP in the USA following FDA authorisation.MethodsA national probability sample of 20 449 US adults completed an online survey between November 2019 and February 2020. In addition to assessing awareness and use of HTP, two ratios were calculated: the ratio of those who experimented with HTP given that they had heard about it (E/H) and the ratio of those who currently used HTP given experimentation (C/E). These ratios for HTP were compared against those for e-cigarettes from a similar national survey in 2012.ResultsOverall, 8.1% of respondents had heard of HTP. Only 0.55% had tried and 0.10% were current users. The rate of experimentation among those who heard about HTP and the rate of current use among experimenters were, however, similar to those for e-cigarettes in 2012: E/H and C/E for HTP were 6.8% and 18.2%, respectively; and 10.7% and 17.8%, respectively for e-cigarettes. The majority of respondents considered HTP either less harmful than (11.6%), or equally harmful as e-cigarettes (42.7%).ConclusionsOnly a small fraction of US population in 2020 have tried any HTP. However, the similarity in early adoption rates following awareness, suggests that future adoption for HTP may be similar to that for e-cigarettes, if HTP are marketed more aggressively.

A high index of clinical suspicion is required to make the diagnosis of perianal tumours while assessing patients presenting with perianal inflammatory conditions. Wide excision of the tumour with abdominoperineal resection is the surgical treatment of choice and can provide good long-term results in patients with localized disease.