1. Coagulopathy in Zellweger spectrum disorders: a role for vitamin K.
- Author
-
Zeynelabidin S, Klouwer FCC, Meijers JCM, Suijker MH, Engelen M, Poll-The BT, and van Ommen CH
- Subjects
- Administration, Intravenous, Administration, Oral, Adolescent, Biomarkers blood, Blood Coagulation Disorders blood, Blood Coagulation Disorders diagnosis, Blood Coagulation Disorders epidemiology, Child, Female, Hemorrhage blood, Hemorrhage diagnosis, Hemorrhage epidemiology, Humans, Incidence, Male, Netherlands epidemiology, Pilot Projects, Proof of Concept Study, Prospective Studies, Protein Precursors blood, Prothrombin, Retrospective Studies, Severity of Illness Index, Treatment Outcome, Vitamin K Deficiency blood, Vitamin K Deficiency diagnosis, Vitamin K Deficiency epidemiology, Young Adult, Zellweger Syndrome blood, Zellweger Syndrome diagnosis, Zellweger Syndrome epidemiology, Blood Coagulation drug effects, Blood Coagulation Disorders drug therapy, Dietary Supplements, Hemorrhage drug therapy, Vitamin K administration & dosage, Vitamin K Deficiency drug therapy, Zellweger Syndrome drug therapy
- Abstract
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.
- Published
- 2018
- Full Text
- View/download PDF