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37 results on '"Bernatowska E"'

1. A Multi-center, Open-Label, Single-Arm Trial to Evaluate the Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency (Jan, 10.1007/s10875-021-01181-6, 2022)

Author

Santamaria, M, Neth, O, Douglass, JA, Krivan, G, Kobbe, R, Bernatowska, E, Grigoriadou, S, Bethune, C, Chandra, A, Horneff, G, Borte, M, Sonnenschein, A, Kralickova, P, Ramon, SS, Langguth, DD, Gonzalez-Granado, LI, Alsina L, Querolt, M, Griffin, R, Hames, C, Mondou, E, Price, J, Sanz, A, and Lin, J

Published
2022

2. A Multi-Center, Open-Label, Single-Arm Trial to Evaluate the Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency

Author

Santamaria M, Neth O, Douglass JA, Krivan G, Kobbe R, Bernatowska E, Grigoriadou S, Bethune C, Chandra A, Horneff G, Borte M, Sonnenschein A, Kralickova P, Ramón SS, Langguth D, Gonzalez-Granado LI, Alsina L, Querolt M, Griffin R, Hames C, Mondou E, Price J, Sanz A, and Lin J

Subjects
Primary immunodeficiency, immunoglobulin replacement therapy, GTI1503, subcutaneous, 20% immunoglobulin
Abstract

Purpose The purpose of this phase 3 study was to evaluate the efficacy, pharmacokinetics (PK), and safety of Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) in patients with primary immunodeficiency (PI). Methods Immunoglobulin treatment-experienced subjects with PI received 52 weeks of IGSC 20% given weekly at the same dose as the subject's previous IgG regimen (DAF 1:1); the minimum dose was 100 mg/kg/week. The primary endpoint was serious bacterial infections (SBIs [null vs alternative hypothesis: SBI rate per person per year >= 1 vs < 1]). IgG subclasses and specific pathogen antibody levels were also measured. Results Sixty-one subjects (19 children [ 12-16 years], and 32 adults) were enrolled. The rate of SBIs per person per year was 0.017. The 1-sided 99% upper confidence limit was 0.036 (< 1), and the null hypothesis was rejected. The rate of hospitalization due to infection per person per year was 0.017 (2-sided 95% confidence interval: 0.008-0.033) overall. The mean trough total IgG concentrations were comparable to the previous IgG replacement regimen. The average of the individual mean trough ratios (IGSC 20%:previous regimen) was 1.078 (range: 0.83-1.54). The average steady-state mean trough IgG concentrations were 947.64 and 891.37 mg/dL, respectively. Seven subjects had serious treatment-emergent adverse events (TEAEs); none was drug-related. The rate of all TEAEs, including local infusion site reactions, during 3045 IGSC 20% infusions was 0.135. Most TEAEs were mild or moderate. Conclusions IGSC 20% demonstrated efficacy and good safety and tolerability in subjects with PI.

Published
2022

3. Wiskott-Aldrich Syndrome protein deficiency perturbs the homeostasis of B-cell compartment in humans

Author

Castiello MC, Bosticardo M, Pala F, Catucci M, Chamberlain N, van Zelm MC, Driessen GJ, Pac M, Bernatowska E, Scaramuzza S, Sauer AV, Traggiai E, Meffre E, Villa A, van der Burg M., AIUTI , ALESSANDRO, Castiello, Mc, Bosticardo, M, Pala, F, Catucci, M, Chamberlain, N, van Zelm, Mc, Driessen, Gj, Pac, M, Bernatowska, E, Scaramuzza, S, Aiuti, Alessandro, Sauer, Av, Traggiai, E, Meffre, E, Villa, A, and van der Burg, M.

Published
2014

4. XLA patients with BTK splice-site mutations produce low levels of wild-type BTK transcripts

Author

Noordzij, J.G., Bruin-Versteeg, S. de, Hartwig, N.G., Weemaes, C.M.R., Gerritsen, E.J., Bernatowska, E., Lierde, S. van, Groot, R. de, and Dongen, J.J.M. van

Subjects
immune system diseases, hemic and lymphatic diseases, Pediatric Oncology. Treatment of children with cancer, Kinderoncologie. Behandeling van kinderen met kanker
Abstract

Item does not contain fulltext X-linked agammaglobulinemia is caused by mutations in the BTK gene, which result in a precursor B-cell differentiation arrest in the bone marrow and the absence of or strongly reduced B lymphocytes in blood. We identified a patient with a mild clinical phenotype, low numbers of B lymphocytes, and a splice-site mutation in the BTK gene. The precursor B-cell compartment in the bone marrow of this patient was almost identical to that in healthy children. Using real-time quantitative polymerase chain reaction, we were able to detect low levels of wild-type BTK transcripts in his granulocytes. Therefore, we speculated that wild-type BTK transcripts might be responsible for a milder clinical and immunological phenotype, as has been shown in several other diseases. Consequently, we quantified the expression of wild-type BTK transcripts in granulocytes of eight additional patients with splice-site mutations and compared their phenotypes with 17 patients with other types of BTK mutations. In these eight patients, the presence of low levels of wild-type BTK transcripts did not show a clear correlation with the percentage, absolute number, or immunophenotype of B lymphocytes nor with age or serum immunoglobulin levels at diagnosis. Nevertheless, we postulate that the presence of wild-type BTK transcripts can be one of the many factors that influence the clinical and immunological phenotype in X-linked agammaglobulinemia.

Published
2002

6. Nijmegen Breakage Syndrome. The International Nijmegen Breakage Syndrome Study Group

Author

Hiel, J.A.P., Weemaes, C.M.R., Heuvel, L.P.W.J. van den, Engelen, B.G.M. van, Gabreëls, F.J.M., Smeets, D.F.C.M., Burgt, C.J.A.M. van der, Chrzanowska, K.H., and Bernatowska, E.

Subjects
Breuk-gevoelige plaatsen in chromosomen bij de mens, Immuunstoornissen in relatie tot kinderen met (pre-)maligne aandoeningen, (Fragile) breakage-prone sites in human chromosomes, Ataxia telangiectasia immunologische, genetische en neurologische aspecten, Immune deficiencies in children (pre-)malignant diseases, Ataxia telangiectasia immunological, genetical and neurological aspects
Abstract

Item does not contain fulltext

Published
2000

9. Nijmegen breakage syndrome

Author

Hiel, Ja, Weemaes, Cm, Den Heuvel, Lp, Engelen, Bg, Gabreels, Fj, Smeets, Df, Burgt, I., Chrzanovska, Kh, Bernatowska, E., Krajewska-Walasek, M., Bialecka, M., Abramczuk, D., Gregorek, H., Michalkiewicz, I., Perek, D., Midro, At, Seemanova, E., Belohradsky, Bh, Solder, B., Barbi, G., Wegner, Rd, Sperling, K., Dixon, J., Maraschio, P., Marseglia, Gl, Green, A., Taylor, Am, Kaloustian, Vm, Komatsu, K., Shinya Matsuura, Conley, Me, Concannon, P., Gatti, Ra, and Int Nijmegen Breakage Syndrome Stu

32. Recommendations for prevention of community-acquired pneumonia with bacteremia as the leading form of invasive pneumococcal infections in the population of people over 50 years of age and risk groups above 19 years of age,Rekomendacje dotyczące profilaktyki pozaszpitalnego zapalenia płuc z bakteriemią jako wiodącej postaci inwazyjnych zakazeń pneumokokowych w populacji osób powyzej 50 roku zycia oraz w grupach ryzyka powyzej 19 roku zycia

Author

Albrecht, P., Antczak, A., Hryniewicz, W., Skoczyńska, A., Radzikowski, A., Kędziora-Kornatowska, K., Bernatowska, E., Stompór, T., Tomasz Grodzicki, Gyrczuk, E., Imiela, J., Jędrzejczak, W., and Windak, A.

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