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Non-consanguineous pediatric myelofibrosis due to MPIG6B mutations in a patient of European ancestry
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  • Leonard Yenwongfai,
  • Ranjana Arora,
  • Alexander Smith,
  • Theodosia Kalfa,
  • Ammar Husami,
  • Vlad Radulescu,
  • Kasiani Myers,
  • Robert Lorsbach
Leonard Yenwongfai
University of Kentucky Medical Center

Corresponding Author:leonardfai@uky.edu

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Ranjana Arora
University of Kentucky College of Medicine
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Alexander Smith
University of Kentucky Medical Center
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Theodosia Kalfa
Cincinnati Children's Hospital Medical Center
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Ammar Husami
Cincinnati Children's Hospital Medical Center
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Vlad Radulescu
University of Kentucky Medical Center
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Kasiani Myers
Cincinnati Children's Hospital Medical Center
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Robert Lorsbach
Cincinnati Children's Hospital Medical Center
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Abstract

Myelofibrosis (MF) in the pediatric setting is uncommon and appears to be pathogenically heterogeneous. MF due to intrinsic bone marrow abnormality (IMF) is distinct from adult-type Primary myelofibrosis (PMF) as they can lack the common genetic markers of clonality. To date, all but two reported patients with pediatric MF and mutated MPIG6B have been Arabic, and all reported cases have had a family history of consanguinity. Here we report the first North American patient of European ancestry with pediatric MF in whom novel compound heterozygous mutations of MPIG6B were identified.