Desmopressin testing in von Willebrand disease: Lowering the burden
Abstract Background Individuals with von Willebrand disease (VWD) require desmopressin testing because of interindividual response differences. However, testing is burdensome, while not all patients may need extensive testing. Objectives To provide von Willebrand factor (VWF) cutoffs that predict de...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Elsevier
2022-08-01
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| Series: | Research and Practice in Thrombosis and Haemostasis |
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| Online Access: | https://doi.org/10.1002/rth2.12784 |
| _version_ | 1797758069232369664 |
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| author | Jessica M. Heijdra Ferdows Atiq Wala Al Arashi Quincy Kieboom Esmee Wuijster Karina Meijer Marieke J. H. A. Kruip Frank W. G. Leebeek Marjon H. Cnossen in collaboration with the OPTI‐CLOT Study Group |
| author_facet | Jessica M. Heijdra Ferdows Atiq Wala Al Arashi Quincy Kieboom Esmee Wuijster Karina Meijer Marieke J. H. A. Kruip Frank W. G. Leebeek Marjon H. Cnossen in collaboration with the OPTI‐CLOT Study Group |
| author_sort | Jessica M. Heijdra |
| collection | DOAJ |
| description | Abstract Background Individuals with von Willebrand disease (VWD) require desmopressin testing because of interindividual response differences. However, testing is burdensome, while not all patients may need extensive testing. Objectives To provide von Willebrand factor (VWF) cutoffs that predict desmopressin nonresponse and thereby identify individuals who do not need extensive testing in a retrospective cohort. We validated these cutoffs in a prospective cohort. Patients and Methods We included 376 patients (Type 1 VWD with VWF activity [VWF:Act] <0.30 IU/ml: n = 112; with VWF:Act 0.30–0.50 IU/ml: n = 206; Type 2 VWD: n = 58; ages, 5–76 years) from January 2000 to July 2020. We collected VWF:Act and factor VIII activity (FVIII:C) at baseline and several time points after desmopressin (T1–T6). We defined response as VWF:Act and FVIII:C 0.50 IU/ml or greater at T1 and T4. We compared VWF:Act and FVIII:C distribution (historically lowest level, baseline, and T1) between responders and nonresponders and determined cutoffs discriminating between these groups. Results were validated in a group of 30 individuals. Results All individuals with Type 1 VWD and Type 2 VWD, respectively, with baseline VWF:Act 0.34 IU/ml or greater or 0.28 IU/ml or greater were responders. In individuals with T1 VWF:Act ≥0.89 IU/ml (Type 1 VWD) or T1 VWF:Act 1.10 IU/ml or greater (Type 2 VWD), response remained at T4. Conclusion Desmopressin testing is not needed when lowest historical VWF:Act is 0.30 IU/ml or greater. In patients with Type 1 VWD who require testing, measurements after T1 are often not needed. In patients with Type 2 VWD who require testing, we advise performing T1 and T4 measurements. |
| first_indexed | 2024-03-12T18:24:21Z |
| format | Article |
| id | doaj.art-fef425826bc741c78576e14267ed4a79 |
| institution | Directory Open Access Journal |
| issn | 2475-0379 |
| language | English |
| last_indexed | 2024-03-12T18:24:21Z |
| publishDate | 2022-08-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Research and Practice in Thrombosis and Haemostasis |
| spelling | doaj.art-fef425826bc741c78576e14267ed4a792023-08-02T08:36:01ZengElsevierResearch and Practice in Thrombosis and Haemostasis2475-03792022-08-0166n/an/a10.1002/rth2.12784Desmopressin testing in von Willebrand disease: Lowering the burdenJessica M. Heijdra0Ferdows Atiq1Wala Al Arashi2Quincy Kieboom3Esmee Wuijster4Karina Meijer5Marieke J. H. A. Kruip6Frank W. G. Leebeek7Marjon H. Cnossen8in collaboration with the OPTI‐CLOT Study GroupDepartment of Pediatric Hematology Erasmus MC – Sophia Children's Hospital, University Medical Center Rotterdam Rotterdam The NetherlandsDepartment of Hematology Erasmus MC, University Medical Center Rotterdam Rotterdam The NetherlandsDepartment of Pediatric Hematology Erasmus MC – Sophia Children's Hospital, University Medical Center Rotterdam Rotterdam The NetherlandsDepartment of Pediatric Hematology Erasmus MC – Sophia Children's Hospital, University Medical Center Rotterdam Rotterdam The NetherlandsDepartment of Hematology Erasmus MC, University Medical Center Rotterdam Rotterdam The NetherlandsDepartment of Hematology University of Groningen and University Medical Center Groningen Groningen The NetherlandsDepartment of Hematology Erasmus MC, University Medical Center Rotterdam Rotterdam The NetherlandsDepartment of Hematology Erasmus MC, University Medical Center Rotterdam Rotterdam The NetherlandsDepartment of Pediatric Hematology Erasmus MC – Sophia Children's Hospital, University Medical Center Rotterdam Rotterdam The NetherlandsAbstract Background Individuals with von Willebrand disease (VWD) require desmopressin testing because of interindividual response differences. However, testing is burdensome, while not all patients may need extensive testing. Objectives To provide von Willebrand factor (VWF) cutoffs that predict desmopressin nonresponse and thereby identify individuals who do not need extensive testing in a retrospective cohort. We validated these cutoffs in a prospective cohort. Patients and Methods We included 376 patients (Type 1 VWD with VWF activity [VWF:Act] <0.30 IU/ml: n = 112; with VWF:Act 0.30–0.50 IU/ml: n = 206; Type 2 VWD: n = 58; ages, 5–76 years) from January 2000 to July 2020. We collected VWF:Act and factor VIII activity (FVIII:C) at baseline and several time points after desmopressin (T1–T6). We defined response as VWF:Act and FVIII:C 0.50 IU/ml or greater at T1 and T4. We compared VWF:Act and FVIII:C distribution (historically lowest level, baseline, and T1) between responders and nonresponders and determined cutoffs discriminating between these groups. Results were validated in a group of 30 individuals. Results All individuals with Type 1 VWD and Type 2 VWD, respectively, with baseline VWF:Act 0.34 IU/ml or greater or 0.28 IU/ml or greater were responders. In individuals with T1 VWF:Act ≥0.89 IU/ml (Type 1 VWD) or T1 VWF:Act 1.10 IU/ml or greater (Type 2 VWD), response remained at T4. Conclusion Desmopressin testing is not needed when lowest historical VWF:Act is 0.30 IU/ml or greater. In patients with Type 1 VWD who require testing, measurements after T1 are often not needed. In patients with Type 2 VWD who require testing, we advise performing T1 and T4 measurements.https://doi.org/10.1002/rth2.12784desmopressinfactor VIIIhumansvon Willebrand diseasevon Willebrand factor |
| spellingShingle | Jessica M. Heijdra Ferdows Atiq Wala Al Arashi Quincy Kieboom Esmee Wuijster Karina Meijer Marieke J. H. A. Kruip Frank W. G. Leebeek Marjon H. Cnossen in collaboration with the OPTI‐CLOT Study Group Desmopressin testing in von Willebrand disease: Lowering the burden Research and Practice in Thrombosis and Haemostasis desmopressin factor VIII humans von Willebrand disease von Willebrand factor |
| title | Desmopressin testing in von Willebrand disease: Lowering the burden |
| title_full | Desmopressin testing in von Willebrand disease: Lowering the burden |
| title_fullStr | Desmopressin testing in von Willebrand disease: Lowering the burden |
| title_full_unstemmed | Desmopressin testing in von Willebrand disease: Lowering the burden |
| title_short | Desmopressin testing in von Willebrand disease: Lowering the burden |
| title_sort | desmopressin testing in von willebrand disease lowering the burden |
| topic | desmopressin factor VIII humans von Willebrand disease von Willebrand factor |
| url | https://doi.org/10.1002/rth2.12784 |
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