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Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation

Mucopolysaccharidosis type I (MPS I) is an autosomal‐recessive metabolic disorder caused by an enzyme deficiency of lysosomal alpha‐l‐iduronidase (IDUA). Haematopoietic stem cell transplantation (HSCT) is the therapeutic option of choice in MPS I patients younger than 2.5 years, which has a positive...

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Autores principales: Maier, Stefanie, Zivicnjak, Miroslav, Grigull, Lorenz, Hennermann, Julia B., Aries, Charlotte, Maecker‐Kolhoff, Britta, Sauer, Martin, Das, Anibh M., Beier, Rita
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9259397/
https://www.ncbi.nlm.nih.gov/pubmed/35822096
http://dx.doi.org/10.1002/jmd2.12291
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author Maier, Stefanie
Zivicnjak, Miroslav
Grigull, Lorenz
Hennermann, Julia B.
Aries, Charlotte
Maecker‐Kolhoff, Britta
Sauer, Martin
Das, Anibh M.
Beier, Rita
author_facet Maier, Stefanie
Zivicnjak, Miroslav
Grigull, Lorenz
Hennermann, Julia B.
Aries, Charlotte
Maecker‐Kolhoff, Britta
Sauer, Martin
Das, Anibh M.
Beier, Rita
author_sort Maier, Stefanie
collection PubMed
description Mucopolysaccharidosis type I (MPS I) is an autosomal‐recessive metabolic disorder caused by an enzyme deficiency of lysosomal alpha‐l‐iduronidase (IDUA). Haematopoietic stem cell transplantation (HSCT) is the therapeutic option of choice in MPS I patients younger than 2.5 years, which has a positive impact on neurocognitive development. However, impaired growth remains a problem. In this monocentric study, 14 patients with MPS I (mean age 1.72 years, range 0.81–3.08) were monitored according to a standardised follow‐up program after successful allogeneic HSCT. A detailed anthropometric program was carried out to identify growth patterns and to determine predictors of growth in these children. All patients are alive and in outpatient care (mean follow‐up 8.1 years, range 0.1–16.0). Progressively lower standard deviation scores (SDS) were observed for body length (mean SDS −1.61; −4.58 – 3.29), weight (−0.56; −3.19 – 2.95), sitting height (−3.28; −7.37 – 0.26), leg length (−1.64; −3.88 – 1.49) and head circumference (0.91; −2.52 – 6.09). Already at the age of 24 months, significant disproportions were detected being associated with increasing deterioration in growth for age. Younger age at HSCT, lower counts for haemoglobin and platelets, lower potassium, higher donor‐derived chimerism, higher counts for leukocytes and recruitment of a matched unrelated donor (MUD) positively correlated with body length (p ≤ 0.05). In conclusion, this study characterised predictors and aspects of growth patterns in children with MPS I after HSCT, underlining that early HSCT of MUD is essential for slowing body disproportion.
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spelling pubmed-92593972022-07-11 Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation Maier, Stefanie Zivicnjak, Miroslav Grigull, Lorenz Hennermann, Julia B. Aries, Charlotte Maecker‐Kolhoff, Britta Sauer, Martin Das, Anibh M. Beier, Rita JIMD Rep Research Reports Mucopolysaccharidosis type I (MPS I) is an autosomal‐recessive metabolic disorder caused by an enzyme deficiency of lysosomal alpha‐l‐iduronidase (IDUA). Haematopoietic stem cell transplantation (HSCT) is the therapeutic option of choice in MPS I patients younger than 2.5 years, which has a positive impact on neurocognitive development. However, impaired growth remains a problem. In this monocentric study, 14 patients with MPS I (mean age 1.72 years, range 0.81–3.08) were monitored according to a standardised follow‐up program after successful allogeneic HSCT. A detailed anthropometric program was carried out to identify growth patterns and to determine predictors of growth in these children. All patients are alive and in outpatient care (mean follow‐up 8.1 years, range 0.1–16.0). Progressively lower standard deviation scores (SDS) were observed for body length (mean SDS −1.61; −4.58 – 3.29), weight (−0.56; −3.19 – 2.95), sitting height (−3.28; −7.37 – 0.26), leg length (−1.64; −3.88 – 1.49) and head circumference (0.91; −2.52 – 6.09). Already at the age of 24 months, significant disproportions were detected being associated with increasing deterioration in growth for age. Younger age at HSCT, lower counts for haemoglobin and platelets, lower potassium, higher donor‐derived chimerism, higher counts for leukocytes and recruitment of a matched unrelated donor (MUD) positively correlated with body length (p ≤ 0.05). In conclusion, this study characterised predictors and aspects of growth patterns in children with MPS I after HSCT, underlining that early HSCT of MUD is essential for slowing body disproportion. John Wiley & Sons, Inc. 2022-04-26 /pmc/articles/PMC9259397/ /pubmed/35822096 http://dx.doi.org/10.1002/jmd2.12291 Text en © 2022 The Authors. JIMD Reports published by John Wiley & Sons Ltd on behalf of SSIEM. https://creativecommons.org/licenses/by/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
spellingShingle Research Reports
Maier, Stefanie
Zivicnjak, Miroslav
Grigull, Lorenz
Hennermann, Julia B.
Aries, Charlotte
Maecker‐Kolhoff, Britta
Sauer, Martin
Das, Anibh M.
Beier, Rita
Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation
title Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation
title_full Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation
title_fullStr Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation
title_full_unstemmed Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation
title_short Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation
title_sort predictors of growth patterns in children with mucopolysaccharidosis i after haematopoietic stem cell transplantation
topic Research Reports
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9259397/
https://www.ncbi.nlm.nih.gov/pubmed/35822096
http://dx.doi.org/10.1002/jmd2.12291
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