Hematopoietic cell transplantation for a child with OSTM1 osteopetrosis

Blood Adv. 2016 Dec 30;1(4):279-281. doi: 10.1182/bloodadvances.2016002345. eCollection 2017 Jan 10.

Authors

Kathleen M Overholt^{1

2}, Melissa J Rose², Sarita Joshi², Gail E Herman³, Rajinder Bajwa², Rolla Abu-Arja², Hemalatha G Rangarajan², Edwin M Horwitz^{1

2

4

5}

Affiliations

¹ Center for Childhood Cancer and Blood Diseases, The Research Institute.
² Division of Pediatric Hematology/Oncology/BMT, and.
³ Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, OH; and.
⁴ Department of Pediatrics and.
⁵ Department of Medicine, Ohio State University, Columbus, OH.

Abstract

HCT prior to onset of neurologic symptoms in children with OSTM1 osteopetrosis does not halt neurologic progression.