Coagulopathy in Zellweger spectrum disorders: a role for vitamin K

Autor: Bwee Tien Poll-The, Joost C. M. Meijers, C. Heleen van Ommen, Monique H. Suijker, Femke C. C. Klouwer, Marc Engelen, Sara Zeynelabidin
Přispěvatelé: Graduate School, Paediatric Neurology, Amsterdam Cardiovascular Sciences, Vascular Medicine, Paediatric Infectious Diseases / Rheumatology / Immunology, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam Neuroscience - Cellular & Molecular Mechanisms, ACS - Pulmonary hypertension & thrombosis
Rok vydání: 2017
Předmět:
Male
0301 basic medicine
Vitamin K
Peroxisome biogenesis disorders
Administration
Oral
Pilot Projects
Severity of Illness Index
Gastroenterology
Liver disease
chemistry.chemical_compound
Coagulopathy
Oral administration
Prospective Studies
Child
Zellweger Syndrome
Genetics (clinical)
Netherlands
Factor VII
Incidence
Incidence (epidemiology)
Blood Coagulation Disorders
Treatment Outcome
Cohort
Original Article
Administration
Intravenous
Female
Prothrombin
medicine.medical_specialty
Adolescent
Hemorrhage
Proof of Concept Study
Young Adult
03 medical and health sciences
Zellweger spectrum disorders
Internal medicine
Vitamin K deficiency
Genetics
medicine
Humans
Protein Precursors
Blood Coagulation
Retrospective Studies
business.industry
Retrospective cohort study
medicine.disease
030104 developmental biology
chemistry
Dietary Supplements
Vitamin K Deficiency
business
Biomarkers
Zdroj: Journal of Inherited Metabolic Disease
Journal of inherited metabolic disease, 41(2), 249-255. Springer Netherlands
ISSN: 1573-2665
0141-8955
Popis: Introduction: Zellweger spectrum disorders (ZSDs) are caused by an impairment of peroxisome biogenesis, resulting in multiple metabolic abnormalities. This leads to a range of symptoms, including hepatic dysfunction and coagulopathy. This study evaluated the incidence and severity of coagulopathy and the effect of vitamin K supplementation orally and IV in ZSD. Methods: Data were retrospectively retrieved from the medical records of 30 ZSD patients to study coagulopathy and the effect of vitamin K orally on proteins induced by vitamin K absence (PIVKA-II) levels. Five patients from the cohort with a prolonged prothrombin time, low factor VII, and elevated PIVKA-II levels received 10 mg of vitamin K IV. Laboratory results, including thrombin generation, at baseline and 72 h after vitamin K administration were examined. Results: In the retrospective cohort, four patients (13.3%) experienced intracranial bleedings and 14 (46.7%) reported minor bleeding. No thrombotic events occurred. PIVKA-II levels decreased 38% after start of vitamin K therapy orally. In the five patients with a coagulopathy, despite treatment with oral administration of vitamin K, vitamin K IV caused an additional decrease (23%) of PIVKA-II levels and increased thrombin generation. Conclusion: Bleeding complications frequently occur in ZSD patients due to liver disease and vitamin K deficiency. Vitamin K deficiency is partly corrected by vitamin K supplementation orally, and vitamin K administered IV additionally improves vitamin K status, as shown by further decrease of PIVKA-II and improved thrombin generation.
Databáze: OpenAIRE
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