Rickets-like radiological and biochemical features of neonatal mucolipidosis II (I-cell disease): report of two cases

In this paper, two cases with mucolipidosis type II (I-cell disease) (proven in one presenting newborn and presumed in an elder deceased brother) are presented. These infants showed severe skeletal changes with diffuse periosteal new bone formation in long bones and ribs, marked osteopenia, a...

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Main Authors: Gülcan Türker, Sükrü Hatun, Kaan Gülleroğlu, Filiz Cizmecioğlu, Aye S Gökalp, Turgay Cokun
Format: Article
Language:English
Published: Hacettepe University Institute of Child Health 2005-10-01
Series:The Turkish Journal of Pediatrics
Online Access:https://turkjpediatr.org/article/view/2749
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author Gülcan Türker
Sükrü Hatun
Kaan Gülleroğlu
Filiz Cizmecioğlu
Aye S Gökalp
Turgay Cokun
author_facet Gülcan Türker
Sükrü Hatun
Kaan Gülleroğlu
Filiz Cizmecioğlu
Aye S Gökalp
Turgay Cokun
author_sort Gülcan Türker
collection DOAJ
description In this paper, two cases with mucolipidosis type II (I-cell disease) (proven in one presenting newborn and presumed in an elder deceased brother) are presented. These infants showed severe skeletal changes with diffuse periosteal new bone formation in long bones and ribs, marked osteopenia, and resorption of scapula, clavicula, and mandible. There was also irregular demineralization of metaphyses of long tubular bones, as seen in rickets. The activities of serum alkaline phosphatase and parathyroid hormone were markedly elevated. Phosphorus was decreased. Serum 1,25-dihydroxyvitamin D was slightly elevated, but 25-hydroxyvitamin D and calcium were normal. Dysostosis multiplex resembling rickets and very high alkaline phosphatase activity were due to defective osteoblastic activity, but the mechanism of elevated parathyroid hormone was not clear. We conclude that early skeletal manifestation of mucolipidosis type II is not clearly identified and that differentiation from congenital rickets or congenital hyperparathyroidism could be difficult. It is speculated that hyperparathyroidism in these patients could be related to the calcium-sensing receptor malfunction in the parathyroid gland.
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institution OA Journals
issn 0041-4301
2791-6421
language English
publishDate 2005-10-01
publisher Hacettepe University Institute of Child Health
record_format Article
series The Turkish Journal of Pediatrics
spelling doaj-art-a79a339d2c3f45e6af754400e80e66962025-08-20T02:01:50ZengHacettepe University Institute of Child HealthThe Turkish Journal of Pediatrics0041-43012791-64212005-10-01474Rickets-like radiological and biochemical features of neonatal mucolipidosis II (I-cell disease): report of two casesGülcan Türker0Sükrü HatunKaan GülleroğluFiliz CizmecioğluAye S GökalpTurgay CokunDepartment of Pediatrics, Kocaeli University Faculty of Medicine, Kocaeli, Turkey. In this paper, two cases with mucolipidosis type II (I-cell disease) (proven in one presenting newborn and presumed in an elder deceased brother) are presented. These infants showed severe skeletal changes with diffuse periosteal new bone formation in long bones and ribs, marked osteopenia, and resorption of scapula, clavicula, and mandible. There was also irregular demineralization of metaphyses of long tubular bones, as seen in rickets. The activities of serum alkaline phosphatase and parathyroid hormone were markedly elevated. Phosphorus was decreased. Serum 1,25-dihydroxyvitamin D was slightly elevated, but 25-hydroxyvitamin D and calcium were normal. Dysostosis multiplex resembling rickets and very high alkaline phosphatase activity were due to defective osteoblastic activity, but the mechanism of elevated parathyroid hormone was not clear. We conclude that early skeletal manifestation of mucolipidosis type II is not clearly identified and that differentiation from congenital rickets or congenital hyperparathyroidism could be difficult. It is speculated that hyperparathyroidism in these patients could be related to the calcium-sensing receptor malfunction in the parathyroid gland. https://turkjpediatr.org/article/view/2749
spellingShingle Gülcan Türker
Sükrü Hatun
Kaan Gülleroğlu
Filiz Cizmecioğlu
Aye S Gökalp
Turgay Cokun
Rickets-like radiological and biochemical features of neonatal mucolipidosis II (I-cell disease): report of two cases
The Turkish Journal of Pediatrics
title Rickets-like radiological and biochemical features of neonatal mucolipidosis II (I-cell disease): report of two cases
title_full Rickets-like radiological and biochemical features of neonatal mucolipidosis II (I-cell disease): report of two cases
title_fullStr Rickets-like radiological and biochemical features of neonatal mucolipidosis II (I-cell disease): report of two cases
title_full_unstemmed Rickets-like radiological and biochemical features of neonatal mucolipidosis II (I-cell disease): report of two cases
title_short Rickets-like radiological and biochemical features of neonatal mucolipidosis II (I-cell disease): report of two cases
title_sort rickets like radiological and biochemical features of neonatal mucolipidosis ii i cell disease report of two cases
url https://turkjpediatr.org/article/view/2749
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