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Desmopressin responsiveness by age in type 1 von Willebrand disease

BACKGROUND: Patients with type 1 von Willebrand disease (VWD) undergo a desmopressin (DDAVP) responsiveness challenge at diagnosis to assess whether DDAVP reverses their coagulation deficits. Current practice assumes DDAVP responsiveness remains constant over the lifetime. In patients with type 1 VW...

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Autores principales: Goldberg, Nicola, Nisenbaum, Rosane, Song, Hong, Lillicrap, David, Teitel, Jerome, James, Paula, Sholzberg, Michelle
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7443436/
https://www.ncbi.nlm.nih.gov/pubmed/32864555
http://dx.doi.org/10.1002/rth2.12354
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author Goldberg, Nicola
Nisenbaum, Rosane
Song, Hong
Lillicrap, David
Teitel, Jerome
James, Paula
Sholzberg, Michelle
author_facet Goldberg, Nicola
Nisenbaum, Rosane
Song, Hong
Lillicrap, David
Teitel, Jerome
James, Paula
Sholzberg, Michelle
author_sort Goldberg, Nicola
collection PubMed
description BACKGROUND: Patients with type 1 von Willebrand disease (VWD) undergo a desmopressin (DDAVP) responsiveness challenge at diagnosis to assess whether DDAVP reverses their coagulation deficits. Current practice assumes DDAVP responsiveness remains constant over the lifetime. In patients with type 1 VWD, VWF‐related parameters increase with age. This study explores whether DDAVP responsiveness also differs with age in this population. METHODS: We conducted a retrospective chart review of 106 patients enrolled at our center since 1990. Our primary outcome was DDAVP responsiveness at 1 hour after DDAVP challenge. Locally weighted scatterplot smoothing fit and Spearman correlation coefficients were used to study the relationship between age and DDAVP responsiveness. For female participants, we used the Kruskal‐Wallis test to compare absolute and relative changes in DDAVP responsiveness at various ages. RESULTS: We had 79 patients (56 female) with type 1 VWD with at least 1 DDAVP challenge. In women with type 1 VWD, the absolute change in DDAVP responsiveness did not vary significantly with age (VWF:antigen [Ag], −0.08, P = .56; VWF:ristocetin cofactor [RCo], −0.16, P = .26; low‐molecular‐weight component of factor VIII [FVIII:C], −0.01, P = .93), nor did the relative change in DDAVP responsiveness (VWF:Ag, −0.03, P = .86; VWF:RCo, −0.25, P = .09; FVIII:C, −0.14, P = .34). The data plot suggested a relationship. CONCLUSION: In women with type 1 VWD, DDAVP responsiveness may vary over the life cycle. Our exploratory findings are limited by our retrospective data, cross‐sectional design, and small sample. Future studies should investigate the relationship between age and DDAVP responsiveness prospectively to evaluate whether there is clinical utility in rechallenging postpubertal female patients with type 1 VWD.
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spelling pubmed-74434362020-08-28 Desmopressin responsiveness by age in type 1 von Willebrand disease Goldberg, Nicola Nisenbaum, Rosane Song, Hong Lillicrap, David Teitel, Jerome James, Paula Sholzberg, Michelle Res Pract Thromb Haemost Original Articles: Hemostasis BACKGROUND: Patients with type 1 von Willebrand disease (VWD) undergo a desmopressin (DDAVP) responsiveness challenge at diagnosis to assess whether DDAVP reverses their coagulation deficits. Current practice assumes DDAVP responsiveness remains constant over the lifetime. In patients with type 1 VWD, VWF‐related parameters increase with age. This study explores whether DDAVP responsiveness also differs with age in this population. METHODS: We conducted a retrospective chart review of 106 patients enrolled at our center since 1990. Our primary outcome was DDAVP responsiveness at 1 hour after DDAVP challenge. Locally weighted scatterplot smoothing fit and Spearman correlation coefficients were used to study the relationship between age and DDAVP responsiveness. For female participants, we used the Kruskal‐Wallis test to compare absolute and relative changes in DDAVP responsiveness at various ages. RESULTS: We had 79 patients (56 female) with type 1 VWD with at least 1 DDAVP challenge. In women with type 1 VWD, the absolute change in DDAVP responsiveness did not vary significantly with age (VWF:antigen [Ag], −0.08, P = .56; VWF:ristocetin cofactor [RCo], −0.16, P = .26; low‐molecular‐weight component of factor VIII [FVIII:C], −0.01, P = .93), nor did the relative change in DDAVP responsiveness (VWF:Ag, −0.03, P = .86; VWF:RCo, −0.25, P = .09; FVIII:C, −0.14, P = .34). The data plot suggested a relationship. CONCLUSION: In women with type 1 VWD, DDAVP responsiveness may vary over the life cycle. Our exploratory findings are limited by our retrospective data, cross‐sectional design, and small sample. Future studies should investigate the relationship between age and DDAVP responsiveness prospectively to evaluate whether there is clinical utility in rechallenging postpubertal female patients with type 1 VWD. John Wiley and Sons Inc. 2020-05-30 /pmc/articles/PMC7443436/ /pubmed/32864555 http://dx.doi.org/10.1002/rth2.12354 Text en © 2020 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis. This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
spellingShingle Original Articles: Hemostasis
Goldberg, Nicola
Nisenbaum, Rosane
Song, Hong
Lillicrap, David
Teitel, Jerome
James, Paula
Sholzberg, Michelle
Desmopressin responsiveness by age in type 1 von Willebrand disease
title Desmopressin responsiveness by age in type 1 von Willebrand disease
title_full Desmopressin responsiveness by age in type 1 von Willebrand disease
title_fullStr Desmopressin responsiveness by age in type 1 von Willebrand disease
title_full_unstemmed Desmopressin responsiveness by age in type 1 von Willebrand disease
title_short Desmopressin responsiveness by age in type 1 von Willebrand disease
title_sort desmopressin responsiveness by age in type 1 von willebrand disease
topic Original Articles: Hemostasis
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7443436/
https://www.ncbi.nlm.nih.gov/pubmed/32864555
http://dx.doi.org/10.1002/rth2.12354
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