title: A prospective cohort study of late sequelae of pediatric allogeneic hematopoietic stem cell transplantation.
abstract: As survivors of pediatric allogeneic hematopoietic stem cell transplantations (HSCTs) increase in number, it is increasingly important to evaluate their well-being. We conducted this prospective cohort study to evaluate the cumulative incidence and risk factors for late sequelae of HSCT. Comprehensive surveillance tests were performed annually on every participant, regardless of signs and symptoms, to obtain accurate information on the time-of-onset of each late event to allow hazard function analyses. All participants included in this report had been followed for at least 3 years after HSCT. With a median follow-up of 9 years and a current age of 18.5 years, only 20 of the 155 participants (13%) had no late sequelae; 18 survivors (12%) had 1 chronic health condition, 71 (46%) had 2-4 conditions, and 46 (30%) had 5-9 conditions. Risk factors for increasing number of chronic conditions included young age at the time of HSCT, female sex, high radiation dose, and history of chronic graft-versus-host disease. The cumulative incidence at 10 years for common late events was as follows (ordered by the median time-of-onset): osteonecrosis 13.8%, chronic renal insufficiency 26.8%, hypothyroidism 45.1%, growth hormone deficiency 31.2%, female hypogonadism 57.4%, osteopenia 47.7%, cataracts 43.4%, pulmonary dysfunction 63.2%, and male hypogonadism 20.3%. Coexistence of multiple late sequelae was common in HSCT survivors. Our findings provide a basis for more effective patient counseling, optimal surveillance, and early intervention.
authors.affiliation: From Department of Hematology-Oncology (WL, HA, TS, KG, MOC, GAH, KAK, RCB, RMM, CHP) and Division of Behavioral Medicine (SP), St. Jude Children's Research Hospital, Memphis, Tennessee; and Department of Pediatrics (WL, GAH, KAK, RCB, RMM, CHP) and Division of Endocrinology (SRR), University of Tennessee College of Medicine, Memphis, Tennessee.
authors.forename: Wing
authors.initials: W
authors.lastname: Leung
authors.forename: Hyunah
authors.initials: H
authors.lastname: Ahn
authors.forename: Susan R
authors.initials: SR
authors.lastname: Rose
authors.forename: Sean
authors.initials: S
authors.lastname: Phipps
authors.forename: Teresa
authors.initials: T
authors.lastname: Smith
authors.forename: Kwan
authors.initials: K
authors.lastname: Gan
authors.forename: Madeline
authors.initials: M
authors.lastname: O'Connor
authors.forename: Gregory A
authors.initials: GA
authors.lastname: Hale
authors.forename: Kimberly A
authors.initials: KA
authors.lastname: Kasow
authors.forename: Raymond C
authors.initials: RC
authors.lastname: Barfield
authors.forename: Renee M
authors.initials: RM
authors.lastname: Madden
authors.forename: Ching-Hon
authors.initials: CH
authors.lastname: Pui
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D001851:Bone Diseases, Metabolic / Q000453:epidemiology; D002318:Cardiovascular Diseases / Q000453:epidemiology; D002386:Cataract / Q000453:epidemiology; D002648:Child; D002675:Child, Preschool; D003072:Cognition Disorders / Q000453:epidemiology; D003863:Depression / Q000453:epidemiology; D004700:Endocrine System Diseases / Q000453:epidemiology; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000453:epidemiology; D013006:Growth Hormone / Q000172:deficiency; D034381:Hearing Loss / Q000453:epidemiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D007223:Infant; D008171:Lung Diseases / Q000453:epidemiology; D008297:Male; D009364:Neoplasm Recurrence, Local; D011446:Prospective Studies; D011879:Radiotherapy Dosage; D012008:Recurrence; D051436:Renal Insufficiency, Chronic / Q000453:epidemiology; D012307:Risk Factors; D012640:Seizures / Q000453:epidemiology; D012737:Sex Factors; D017741:Survivors; D014184:Transplantation, Homologous
chemical_list: D013006:Growth Hormone