title: Poor growth, thyroid dysfunction and vitamin D deficiency remain prevalent despite reduced intensity chemotherapy for hematopoietic stem cell transplantation in children and young adults.
abstract: Myeloablative conditioning regimens for hematopoietic stem cell transplant (HSCT) are known to affect endocrine function, but little is known regarding reduced intensity conditioning (RIC) regimens. We retrospectively reviewed 114 children and young adults after single RIC HSCT. The analysis was grouped by age (<2 and ⩾2 years) and diagnosis (hemophagocytic lymphohistiocystosis/X-linked lymphoproliferative syndrome (HLH/XLP), other immune disorders, metabolic/genetic disorders). All groups displayed short stature by mean height-adjusted Z-score (HAZ) before (-1.29) and after HSCT (HAZ -1.38, P=0.47). After HSCT, younger children with HLH/XLP grew better (HAZ -3.41 vs -1.65, P=0.006), whereas older subjects had decline in growth (HAZ -0.8 vs -1.01, P=0.06). Those with steroid therapy beyond standard GVHD prophylaxis were shorter than those without (P 0.04). After HSCT, older subjects with HLH/XLP became thinner with a mean body mass index (BMI) Z-score of 1.20 vs 0.64, P=0.02, and similar to metabolic/genetic disorders (BMI-Z= 0.59 vs -0.99, P<0.001). BMI increased among younger children in these same groups. Thyroid function was abnormal in 24% (18/76). 25-OH vitamin D levels were insufficient in 73% (49/65), with low bone mineral density in 8 of 19 evaluable subjects. Despite RIC, children and young adults still have significant late endocrine effects. Further research is required to compare post-transplant endocrine effects after RIC to those after standard chemotherapy protocols.
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital, Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH, USA.
authors.forename: K C
authors.initials: KC
authors.lastname: Myers
authors.affiliation: Division of Endocrinology, Department of Pediatrics, Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH, USA.
authors.forename: J C
authors.initials: JC
authors.lastname: Howell
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital, Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH, USA.
authors.forename: G
authors.initials: G
authors.lastname: Wallace
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital, Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH, USA.
authors.forename: C
authors.identifier: http://orcid.org/0000-0002-4001-9203
authors.initials: C
authors.lastname: Dandoy
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital, Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH, USA.
authors.forename: J
authors.initials: J
authors.lastname: El-Bietar
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital, Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH, USA.|Division of Biostatistics and Epidemiology, Department of Pediatrics, Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH, USA.
authors.forename: A
authors.initials: A
authors.lastname: Lane
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital, Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH, USA.
authors.forename: S M
authors.initials: SM
authors.lastname: Davies
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital, Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH, USA.
authors.forename: S
authors.initials: S
authors.lastname: Jodele
authors.affiliation: Division of Endocrinology, Department of Pediatrics, Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH, USA.
authors.forename: S R
authors.initials: SR
authors.lastname: Rose
mesh_terms: D000293:Adolescent; D000367:Age Factors; D015992:Body Mass Index; D015519:Bone Density; D002648:Child; D006130:Growth Disorders / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000379:methods; D006801:Humans; D019653:Myeloablative Agonists / Q000008:administration & dosage / Q000494:pharmacology*; D012189:Retrospective Studies; D013959:Thyroid Diseases / Q000209:etiology; D019172:Transplantation Conditioning / Q000009:adverse effects* / Q000379:methods; D014808:Vitamin D Deficiency / Q000209:etiology; D055815:Young Adult
chemical_list: D019653:Myeloablative Agonists