Metabolic syndrome as a late effect of childhood hematopoietic stem cell transplantation – A thorough statistical evaluation of putative risk factors
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Metabolic syndrome as a late effect of childhood hematopoietic stem cell transplantation – A thorough statistical evaluation of putative risk factors. / Gerbek, Tina; Thomsen, Birthe Lykke; Muhic, Ena; Christiansen, Terkel; Sørensen, Kaspar; Ifversen, Marianne; Kofoed, Klaus; Müller, Klaus.
I: Pediatric Transplantation, Bind 27, Nr. 4, e14530, 2023.Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › fagfællebedømt
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T1 - Metabolic syndrome as a late effect of childhood hematopoietic stem cell transplantation – A thorough statistical evaluation of putative risk factors
AU - Gerbek, Tina
AU - Thomsen, Birthe Lykke
AU - Muhic, Ena
AU - Christiansen, Terkel
AU - Sørensen, Kaspar
AU - Ifversen, Marianne
AU - Kofoed, Klaus
AU - Müller, Klaus
N1 - Publisher Copyright: © 2023 The Authors. Pediatric Transplantation published by Wiley Periodicals LLC.
PY - 2023
Y1 - 2023
N2 - Background: Metabolic syndrome (MetS) is frequent among survivors of childhood hematopoietic stem-cell transplantation (HSCT), but assessment of risk factors is challenged by survivor and participation bias in long-term follow-up studies. Methods: A cohort of 395 pediatric patients transplanted between 1980 and 2018 was investigated. MetS was assessed at follow-up between December 2018 and March 2020. Two composite outcomes ((a) combining MetS and death, (b) combining MetS, death, and nonparticipation) were considered to address the risk of selection bias. Results: Among 234 survivors invited to the follow-up, 96 individuals (median age 27 years) participated. MetS prevalence was 30% among participants. The only significant HSCT risk factor was a variable combining HSCT indication and conditioning with total-body irradiation (TBI) (p =.0011). Compared to acute leukemias (AL) treated with high-grade TBI (8–12 Gy), a lower MetS prevalence was seen for nonmalignant diseases treated with no/low-grade TBI (0–4.5 Gy) (OR = 0.04, 95% confidence interval (CI): 0.00–0.23). Analyses of the composite outcomes indicated overestimation of the effect of high-grade TBI due to selection bias. Scrutiny showed strong residual confounding between HSCT indication and high-grade TBI within AL-patients. The HSCT effect on MetS reflected HSCT effects on high-density-lipoprotein (HDL) and triglycerides. Compared to AL treated with high-grade TBI, nonmalignant diagnoses treated with no/low-grade TBI had higher HDL (+40%, 95% CI: +21% to +62%) and lower triglyceride (−59%, 95% CI: −71% to −42%). Conclusion: The TBI effect on MetS may be overestimated in follow-up studies due to selection bias and confounding. The TBI effect was confined to the potentially modifiable MetS criteria HDL and triglyceride.
AB - Background: Metabolic syndrome (MetS) is frequent among survivors of childhood hematopoietic stem-cell transplantation (HSCT), but assessment of risk factors is challenged by survivor and participation bias in long-term follow-up studies. Methods: A cohort of 395 pediatric patients transplanted between 1980 and 2018 was investigated. MetS was assessed at follow-up between December 2018 and March 2020. Two composite outcomes ((a) combining MetS and death, (b) combining MetS, death, and nonparticipation) were considered to address the risk of selection bias. Results: Among 234 survivors invited to the follow-up, 96 individuals (median age 27 years) participated. MetS prevalence was 30% among participants. The only significant HSCT risk factor was a variable combining HSCT indication and conditioning with total-body irradiation (TBI) (p =.0011). Compared to acute leukemias (AL) treated with high-grade TBI (8–12 Gy), a lower MetS prevalence was seen for nonmalignant diseases treated with no/low-grade TBI (0–4.5 Gy) (OR = 0.04, 95% confidence interval (CI): 0.00–0.23). Analyses of the composite outcomes indicated overestimation of the effect of high-grade TBI due to selection bias. Scrutiny showed strong residual confounding between HSCT indication and high-grade TBI within AL-patients. The HSCT effect on MetS reflected HSCT effects on high-density-lipoprotein (HDL) and triglycerides. Compared to AL treated with high-grade TBI, nonmalignant diagnoses treated with no/low-grade TBI had higher HDL (+40%, 95% CI: +21% to +62%) and lower triglyceride (−59%, 95% CI: −71% to −42%). Conclusion: The TBI effect on MetS may be overestimated in follow-up studies due to selection bias and confounding. The TBI effect was confined to the potentially modifiable MetS criteria HDL and triglyceride.
KW - childhood hematopoietic stem cell transplantation
KW - late effects
KW - metabolic syndrome
U2 - 10.1111/petr.14530
DO - 10.1111/petr.14530
M3 - Journal article
C2 - 37069730
AN - SCOPUS:85153182005
VL - 27
JO - Pediatric Transplantation
JF - Pediatric Transplantation
SN - 1397-3142
IS - 4
M1 - e14530
ER -
ID: 369077899