Supplementary material to "Black and brown carbon over central Amazonia: Long-term aerosol measurements at the ATTO site"

SummaryThe safety, efficacy, and prolonged half-life of recombinant factor IX Fc fusion protein (rFIXFc) were demonstrated in the Phase 3 B-LONG (adults/adolescents ≥12 years) and Kids B-LONG (children <12 years) studies of subjects with haemophilia B (≤2 IU/dl). Here, we report interim, long-term safety and efficacy data from B-YOND, the rFIXFc extension study. Eligible subjects who completed B-LONG or Kids B-LONG could enrol in B-YOND. There were four treatment groups: weekly prophylaxis (20–100 IU/kg every 7 days), individualised prophylaxis (100 IU/kg every 8–16 days), modified prophylaxis (further dosing personalisation to optimise prophylaxis), and episodic (ondemand) treatment. Subjects could change treatment groups at any point. Primary endpoint was inhibitor development. One hundred sixteen subjects enrolled in B-YOND. From the start of the parent studies to the B-YOND interim data cut, median duration of rFIXFc treatment was 39.5 months and 21.9 months among adults/adolescents and children, respectively; 68/93 (73.1 %) adults/adolescents and 9/23 (39.1 %) children had ≥100 cumulative rFIXFc exposure days. No inhibitors were observed. Median annualised bleeding rates (ABRs) were low in all prophylaxis regimens: weekly (≥12 years: 2.3; <6 years: 0.0; 6 to <12 years: 2.7), individualised (≥12 years: 2.3; 6 to <12 years: 2.4), and modified (≥12 years: 2.4). One or two infusions were sufficient to control 97 % (adults/adolescents) and 95 % (children) of bleeding episodes. Interim data from B-YOND are consistent with data from B-LONG and Kids B-LONG, and confirm the longterm safety of rFIXFc, absence of inhibitors, and maintenance of low ABRs with prophylactic dosing every 1 to 2 weeks.Supplementary Material to this article is available online at www.thrombosis-online.com.

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Development and validation of a tool for patient reporting of symptoms and signs of the post-thrombotic syndrome

Thrombosis and Haemostasis ◽

10.1160/th15-04-0318 ◽

2016 ◽

Vol 115 (02) ◽

pp. 361-367 ◽

Cited By ~ 19

Author(s):

Kristin Kornelia Utne ◽

Waleed Ghanima ◽

Siv Foyn ◽

Susan Kahn ◽

Per Morten Sandset ◽

...

Keyword(s):

Reference Method ◽

Patient Reporting ◽

Self Assessment ◽

Post Thrombotic Syndrome ◽

Supplementary Material ◽

Development And Validation ◽

Kappa Agreement ◽

Symptoms And Signs ◽

Patient Self Assessment

SummaryPost-thrombotic syndrome (PTS) is a long-term complication of deepvein thrombosis (DVT). The Villalta scale is the recommended tool for diagnosing PTS, but requires a clinician’s assessment in addition to patient self-assessment. In the present study, we validated a self-administered tool for patient reporting of leg symptoms and signs as a mean to assess PTS. We first validated a form for patient self-reported Villalta (PRV1), then developed and validated a visually assisted form (PRV2). The validity of PRV1 and PRV2 was assessed in patients diagnosed with DVT between 2004 and 2012. Median time from DVT to inclusion was 5.1 and 3.5 years for PRV1 (n=162) and PRV2 (n=94), respectively. Patients were requested to complete the PRV form before a scheduled visit. PTS diagnosed by the original Villalta scale during the visit served as the reference method. PRV1 showed only moderate agreement for diagnosing PTS compared with the original Villalta scale (kappa agreement 0.60, 95 % CI 0.48–0.72), whereas PRV2 showed very good agreement (0.82, 95 % CI 0.71–0.94). In the validation of PRV2, PTS was diagnosed in 54 (57 %) patients according to the original Villalta scale and in 60 (64 %) by PRV2. The sensitivity of PRV2 to detect PTS was 98 % and the specificity was 83 %. We conclude that the visually assisted form for PRV is a valid and sensitive tool for diagnosing PTS. Such a tool could be applied in further clinical studies of PTS, making studies less resource demanding by reducing the need for in-person clinic visits.Supplementary Material to this article is available online at www.thrombosis-online.com.

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