{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,6,23]],"date-time":"2025-06-23T13:30:42Z","timestamp":1750685442574,"version":"3.40.5"},"reference-count":35,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2022,4,26]],"date-time":"2022-04-26T00:00:00Z","timestamp":1650931200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100005624","name":"Medizinischen Hochschule Hannover","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100005624","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["JIMD Reports"],"published-print":{"date-parts":[[2022,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Mucopolysaccharidosis type I (MPS I) is an autosomal\u2010recessive metabolic disorder caused by an enzyme deficiency of lysosomal alpha\u2010<jats:sc>l<\/jats:sc>\u2010iduronidase (IDUA). Haematopoietic stem cell transplantation (HSCT) is the therapeutic option of choice in MPS I patients younger than 2.5\u2009years, 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\u2009years, range 0.81\u20133.08) were monitored according to a standardised follow\u2010up 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\u2010up 8.1\u2009years, range 0.1\u201316.0). Progressively lower standard deviation scores (SDS) were observed for body length (mean SDS \u22121.61; \u22124.58 \u2013 3.29), weight (\u22120.56; \u22123.19 \u2013 2.95), sitting height (\u22123.28; \u22127.37 \u2013 0.26), leg length (\u22121.64; \u22123.88 \u2013 1.49) and head circumference (0.91; \u22122.52 \u2013 6.09). Already at the age of 24\u2009months, 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\u2010derived chimerism, higher counts for leukocytes and recruitment of a matched unrelated donor (MUD) positively correlated with body length (<jats:italic>p<\/jats:italic>\u2009\u2264\u20090.05). 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.<\/jats:p>","DOI":"10.1002\/jmd2.12291","type":"journal-article","created":{"date-parts":[[2022,4,26]],"date-time":"2022-04-26T09:27:36Z","timestamp":1650965256000},"page":"371-378","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation"],"prefix":"10.1002","volume":"63","author":[{"given":"Stefanie","family":"Maier","sequence":"first","affiliation":[{"name":"Department of Paediatric Haematology and Oncology Hannover Medical School  Hannover Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Miroslav","family":"Zivicnjak","sequence":"additional","affiliation":[{"name":"Department of Paediatric Kidney Liver and Metabolic Diseases at Hannover Medical School  Hannover Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lorenz","family":"Grigull","sequence":"additional","affiliation":[{"name":"Rare Disease Centre, Bonn University Medical Centre  Bonn Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Julia B.","family":"Hennermann","sequence":"additional","affiliation":[{"name":"Villa Metabolica, Department of Paediatric and Adolescent Medicine University Medical Centre Mainz  Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Charlotte","family":"Aries","sequence":"additional","affiliation":[{"name":"Department of Paediatrics Hamburg\u2010Eppendorf University Medical Centre  Hamburg Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Britta","family":"Maecker\u2010Kolhoff","sequence":"additional","affiliation":[{"name":"Department of Paediatric Haematology and Oncology Hannover Medical School  Hannover Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Martin","family":"Sauer","sequence":"additional","affiliation":[{"name":"Department of Paediatric Haematology and Oncology Hannover Medical School  Hannover Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anibh M.","family":"Das","sequence":"additional","affiliation":[{"name":"Department of Paediatric Kidney Liver and Metabolic Diseases at Hannover Medical School  Hannover Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7568-8749","authenticated-orcid":false,"given":"Rita","family":"Beier","sequence":"additional","affiliation":[{"name":"Department of Paediatric Haematology and Oncology Hannover Medical School  Hannover Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2022,4,26]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/cge.13583"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1023\/A:1012463605992"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/ker393"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2021.03.002"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-6-55"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-11-608075"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2015.02.011"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2019.02.012"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1302\/0301-620X.76B6.7983131"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbadis.2011.07.013"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.2353\/ajpath.2008.070564"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2106596"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2016.01.009"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1704786"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2008.328"},{"volume-title":"Practical Human Biology","year":"1981","author":"Weiner J","key":"e_1_2_9_17_1"},{"key":"e_1_2_9_18_1","first-page":"321","article-title":"Gender\u2010specific growth patterns for stature, sitting height and limbs length in Croatian children and youth (3 to 18\u2009years of age)","volume":"27","author":"\u017divi\u010dnjak M","year":"2003","journal-title":"Coll Antropol"},{"volume-title":"Referenzperzentile f\u00fcr anthropometrische Ma\u00dfzahlen und Blutdruck aus der Studie zur Gesundheit von Kindern und Jugendlichen in Deutschland (KiGGS)","year":"2013","author":"Robert\u2010Koch\u2010Institut","key":"e_1_2_9_19_1"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12519-014-0517-6"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1038\/nature05817"},{"key":"e_1_2_9_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.61378"},{"key":"e_1_2_9_23_1","doi-asserted-by":"publisher","DOI":"10.1097\/MD.0000000000006814"},{"key":"e_1_2_9_24_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0583-7"},{"key":"e_1_2_9_25_1","doi-asserted-by":"publisher","DOI":"10.1136\/adc.76.2.92"},{"key":"e_1_2_9_26_1","doi-asserted-by":"publisher","DOI":"10.1097\/01241398-200401000-00019"},{"key":"e_1_2_9_27_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1705569"},{"key":"e_1_2_9_28_1","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2008.20"},{"key":"e_1_2_9_29_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2013.11.013"},{"key":"e_1_2_9_30_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13052-019-0691-1"},{"key":"e_1_2_9_31_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1704853"},{"key":"e_1_2_9_32_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1704481"},{"key":"e_1_2_9_33_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01644-w"},{"key":"e_1_2_9_34_1","first-page":"1","article-title":"Correlation between respiratory function and spine and thorax deformity in children with mild scoliosis","volume":"96","author":"Szopa A","year":"2017","journal-title":"Med (United States)"},{"key":"e_1_2_9_35_1","first-page":"1896","article-title":"St\u00f6rungen des S\u00e4uren\u2010Basen\u2010Haushalts. Diagnostik und klinisches Bild","volume":"102","author":"Kosch M","year":"2005","journal-title":"Deutsches \u00c4rzteblatt"},{"key":"e_1_2_9_36_1","doi-asserted-by":"publisher","DOI":"10.2214\/ajr.157.4.1909834"}],"container-title":["JIMD Reports"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jmd2.12291","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/jmd2.12291","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jmd2.12291","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,22]],"date-time":"2023-08-22T04:50:17Z","timestamp":1692679817000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/jmd2.12291"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,4,26]]},"references-count":35,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2022,7]]}},"alternative-id":["10.1002\/jmd2.12291"],"URL":"https:\/\/doi.org\/10.1002\/jmd2.12291","archive":["Portico"],"relation":{},"ISSN":["2192-8312","2192-8312"],"issn-type":[{"type":"print","value":"2192-8312"},{"type":"electronic","value":"2192-8312"}],"subject":[],"published":{"date-parts":[[2022,4,26]]},"assertion":[{"value":"2021-07-01","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-04-11","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-04-26","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}