title: Radiologic resolution of malignant infantile osteopetrosis skeletal changes following hematopoietic stem cell transplantation.
abstract: BACKGROUND
  Hematopoietic stem cell transplantation (HSCT) is the only known curative treatment of malignant infantile osteopetrosis (MIOP). In this study, short-term serial bone surveys were used to assess radiologic evolution of skeletal changes after HSCT in MIOP.
  
  
  METHODS
  Baseline whole-body bone survey was performed in all patients. HSCT was successful in 14 patients (11 with full chimerism, three with mixed chimerism) in whom follow-up bone surveys were carried out at 6 and 12 months after HSCT.
  
  
  RESULTS
  Normal corticomedullary differentiation was evident in five (P = 0.06) and 12 (P < 0.005) patients at 6 and 12 months, respectively. Abnormal endobone appearance in long bones, present in 11 participants at baseline exam, disappeared in eight (P = 0.008) and all (P = 0.001) patients at 6 and 12 months, respectively. In 6-month follow-up, rachitic changes significantly disappeared (P < 0.01) in long bones; however, they were evident in ribs of 12 patients (P = 0.50). No patient had rickets in ribs or long bones after 12 months.
  
  
  CONCLUSIONS
  We observed considerable resolution of MIOP skeletal changes after HSCT in all patients with either full or mixed chimerism. Rachitic changes in long bones, attenuated corticomedullary differentiation, and endobone appearance were the first to resolve. We propose using single long bone plain x-ray to demonstrate short-term skeletal response to HSCT.
authors.affiliation: Department of Radiology, Shariati hospital, Tehran University of Medical Sciences, Tehran, Iran.
authors.forename: Amir Pejman
authors.initials: AP
authors.lastname: Hashemi Taheri
authors.affiliation: Department of Radiology, Shariati hospital, Tehran University of Medical Sciences, Tehran, Iran.
authors.forename: Amir Reza
authors.initials: AR
authors.lastname: Radmard
authors.affiliation: Department of Radiology, Shariati hospital, Tehran University of Medical Sciences, Tehran, Iran.
authors.forename: Soheil
authors.initials: S
authors.lastname: Kooraki
authors.affiliation: Hematology-Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran.
authors.forename: Maryam
authors.initials: M
authors.lastname: Behfar
authors.affiliation: Department of Radiology, Shariati hospital, Tehran University of Medical Sciences, Tehran, Iran.
authors.forename: Neda
authors.initials: N
authors.lastname: Pak
authors.affiliation: Hematology-Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran.
authors.forename: Amir Ali
authors.initials: AA
authors.lastname: Hamidieh
authors.affiliation: Hematology-Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran.
authors.forename: Ardeshir
authors.initials: A
authors.lastname: Ghavamzadeh
mesh_terms: D001706:Biopsy; D001842:Bone and Bones / Q000000981:diagnostic imaging* / Q000473:pathology; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D010022:Osteopetrosis / Q000151:congenital / Q000000981:diagnostic imaging / Q000473:pathology / Q000628:therapy*; D011446:Prospective Studies; D011859:Radiography; D066027:Transplant Recipients; D018183:Transplantation Chimera; D016896:Treatment Outcome
keywords: hematopoietic stem cell transplantation; malignant infantile osteopetrosis; radiologic resolution; skeletal changes