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Long-term Follow-up of Homozygote Protein C Deficiency After Multimodal Therapy

Monagle, Kate BBmed; Ignjatovic, Vera BSc, PhD; Hardikar, Winita MBBS; Newall, Fiona PhD, MN, B.Sci(Nsg), RN; Monagle, Paul MBBS, MD, MSc(HRM)

Journal of Pediatric Hematology/Oncology: October 2014 - Volume 36 - Issue 7 - p e452–e455
doi: 10.1097/MPH.0000000000000032
Online Articles: Clinical and Laboratory Observations
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Homozygous protein C deficiency is an extremely rare condition presenting in the neonatal period with purpura fulminans, with very high rates of morbidity and mortality. Optimal treatment for this condition is highly complex, poorly understood, and often limited by cost and product supply. We report a child who presented 2 days after birth with purpura fulminans and severe prenatal eye damage, but no cerebral lesions. He was treated with novel multimodal therapy culminating in liver transplant at 3 years of age. The patient is now 12 years of age, well, with blindness as his only long-term deficit.

Departments of *Paediatrics

§Nursing, The University of Melbourne

Murdoch Children’s Research Institute

Departments of Gastroenterology

Clinical Haematology, Royal Children’s Hospital, Melbourne, Australia

The authors declare no conflict of interest.

Reprints: Paul Monagle, MBBS, MD, MSc(HRM), Department of Clinical Haematology, Royal Children’s Hospital Flemington Road, Parkville, Vic. 3052, Australia (e-mail: paul.monagle@rch.org.au).

Received May 27, 2013

Accepted August 22, 2013

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