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Legg-Perthes disease-like joint involvement and diagnosis delay in Scheie syndrome: a case report
Meltem Alkan Melikoglu1, Hilal Kocabas, Ilhan Sezer
1Department of Physical Medicine and Rehabilitation, School of Medicine, Akdeniz University, Antalya, Turkey. mamelikoglu@gmail.com
Abstract:
Mucopolysaccharidosis (MPS) type I is an inherited disease caused by the absence or malfunctioning of lysosomal enzymes. Three subtypes, based on severity of symptoms, were described, and Scheie syndrome (also called MPS I S) is the mildest form. Although there may be some typical extra-articular manifestations, musculoskeletal involvement may be the only presenting sign in the absence of other symptoms in the patients with less severe forms. The patients with MPS I S, especially in attenuated phenotypes, may be sometimes difficult to recognize for physicians not familiar with the disease. With this case presentation, it is aimed to draw attention to this disease, which could be delayed for the correct diagnosis. An increased awareness of the disease may contribute to more accurate diagnosis, and patients may benefit from early intervention.
Insights
Mucopolysaccharidosis (MPS) type I, specifically the mild Scheie syndrome (MPS I S), can present with only musculoskeletal issues, delaying diagnosis. Increased physician awareness is crucial for early identification and intervention in these subtle cases.
Area of Science:
- Genetics and rare diseases
- Lysosomal storage disorders
Background:
- Mucopolysaccharidosis (MPS) type I is a group of inherited lysosomal storage disorders.
- Scheie syndrome (MPS I S) represents the mildest subtype of MPS I.
Observation:
- Musculoskeletal involvement can be the sole presenting sign in mild MPS I phenotypes.
- Attenuated Scheie syndrome presentations are often challenging for physicians to recognize.
- Delayed diagnosis is common due to the subtle and non-specific initial symptoms.
Findings:
- This case presentation highlights the diagnostic challenges associated with mild MPS I S.
- Emphasizes that musculoskeletal symptoms may be the primary or only indicator.
Implications:
- Increased awareness among healthcare providers is essential for timely diagnosis of MPS I S.
- Early diagnosis facilitates prompt intervention, potentially improving patient outcomes.
- Recognizing subtle musculoskeletal signs can prevent diagnostic delays in rare genetic disorders.
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