Coagulopathy in Zellweger spectrum disorders: a role for vitamin K. Issue 2 (14th November 2017)

• Record Type: Journal Article
• Authors: Zeynelabidin, Sara; Klouwer, Femke C. C.; Meijers, Joost C. M.; Suijker, Monique H.; Engelen, Marc; Poll‐The, Bwee Tien; van Ommen, C. Heleen
• Is Part Of: Journal of inherited metabolic disease. Volume 41:Issue 2(2018)
• Page Start: 249
• Page End: 255
• Publication Date: 2017-11-14
• Subjects: Metabolism, Inborn errors of -- Periodicals; Metabolism -- Disorders -- Periodicals; 616.39042
• DOI: 10.1007/s10545-017-0113-8
• Languages: English
• ISSNs: 0141-8955

Abstract

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.