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Assessing prophylactic use and clinical outcomes in hemophilia A patients treated with rVIII-SingleChain and other common rFVIII products in Germany.

ABSTRACT: Objective: To evaluate real-world outcomes with rVIII-SingleChain and other commonly used recombinant FVIII (rFVIII) products.

Methods: Hemophilia treatment centers in Germany (n = 21) contributed patient chart data. Inclusion criteria were prophylactic treatment with one of five rFVIII products for ?8 weeks.

Results: Male patients (n = 225) were included: rVIII-SingleChain (n = 40), rFVIIIFc (n = 47), octocog alfa (rFVIII; n = 58), octocog alfa (BAY 81-8973; n = 40), or moroctocog alfa (n = 40). In patients with severe disease (n = 76), 66.6%, 70.0%, 20.0%, 7.7%, and 27.3% were dosed ?2×/week, respectively. Irrespective of dosing frequency, mean annualized bleed rates (ABRs)/annualized spontaneous bleed rates (AsBRs) were 0.3/0.1, 0.8/0.4, 1.1/0.5, 1.5/0.8, and 1.4/0.6, and mean FVIII consumption (IU/kg/week) was 83.2, 97.2, 92.5, 104.0, and 102.1, respectively. Results for all patients were similar. Of the patients on prophylaxis with prior therapy and after switching to rVIII-SingleChain (n = 21), mean ABR/AsBRs were 0.7/0.3 and 0.2/0.0, respectively. After switching to rVIII-SingleChain, mean FVIII consumption reduced (109.4 vs 74.5 IU/kg/week), and percentage of patients dosed ?2×/week increased (0% to 71.4%).

Conclusions: rVIII-SingleChain prophylaxis provides excellent bleeding protection, with potentially lowest factor consumption among the products assessed. Patients who switched to rVIII-SingleChain prophylaxis reduced dosing frequency and consumption compared with prior treatment, with similar or potentially lower bleeding rates.

SUBMITTER: Olivieri M

PROVIDER: S-EPMC7079114 | biostudies-literature | 2020 Apr

REPOSITORIES: biostudies-literature

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Assessing prophylactic use and clinical outcomes in hemophilia A patients treated with rVIII-SingleChain and other common rFVIII products in Germany.

Olivieri Martin M Sommerer Patrick P Maro Geraldine G Yan Songkai S

European journal of haematology 20200121 4

<h4>Objective</h4>To evaluate real-world outcomes with rVIII-SingleChain and other commonly used recombinant FVIII (rFVIII) products.<h4>Methods</h4>Hemophilia treatment centers in Germany (n = 21) contributed patient chart data. Inclusion criteria were prophylactic treatment with one of five rFVIII products for ≥8 weeks.<h4>Results</h4>Male patients (n = 225) were included: rVIII-SingleChain (n = 40), rFVIIIFc (n = 47), octocog alfa (rFVIII; n = 58), octocog alfa (BAY 81-8973; n = 40), or moroc ...[more]

PMID: 31883398

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Project description:In neurological patients, a lack of insight into their impairments can lead to possibly dangerous situations and non-compliance in rehabilitation therapy with worse rehabilitation outcomes as a result. This so called anosognosia is a multifaceted syndrome that can occur after brain damage affecting different neurological or cognitive functions. To our knowledge no study has investigated anosognosia for apraxia of common tool-use (CTU) so far. CTU-apraxia is a disorder frequently occurring after stroke that affects the use of familiar objects. Here, we introduce a new questionnaire to diagnose anosognosia for CTU-apraxia, the Visual Analogue Test assessing Anosognosia for Naturalistic Action Tasks (VATA-NAT). This assessment is adapted from a series of VATA-questionnaires that evaluate insight into motor (VATA-M) or language (VATA-L) impairment and take known challenges such as aphasia into account. Fifty one subacute stroke patients with left (LBD) or right (RBD) brain damage were investigated including patients with and without CTU-apraxia. Patients were assessed with the VATA-L, -M and -NAT before and after applying a diagnostics session for each function. Interrater reliability, composite reliability as well as convergent and divergent validity were evaluated for the VATA-NAT. Seven percent of the LBD patients with CTU-apraxia demonstrated anosognosia. After tool-use diagnostics this number increased to 20 percent. For the VATA-NAT, psychometric data revealed high interrater-reliability (τ ≥ 0.828), composite reliability (CR ≥ 0.809) and convergent validity (τ = -0.626). When assessing patients with severe aphasia, the possible influence of language comprehension difficulties needs to be taken into account for interpretation. Overall, close monitoring of anosognosia over the course of rehabilitation is recommended. With the VATA-NAT we hereby provide a novel assessment for anosognosia in patients with CTU-apraxia. For diagnosing anosognosia we recommend to combine this new tool with the existing VATA-M and -L subtests, particularly in patients who demonstrate severe functional deficits.

Dataset Information

Assessing prophylactic use and clinical outcomes in hemophilia A patients treated with rVIII-SingleChain and other common rFVIII products in Germany.

Publications

Assessing prophylactic use and clinical outcomes in hemophilia A patients treated with rVIII-SingleChain and other common rFVIII products in Germany.

Similar Datasets

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