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Morquio syndrome

MedGen UID:
44513
Concept ID:
C0026707
Disease or Syndrome
Synonyms: Eccentrochondrodysplasia; MPS IV; Mucopolysaccharidosis type 4; Mucopolysaccharidosis, Type IV
SNOMED CT: Morquio syndrome (378007); Chondro-osteodystrophy (378007); Morquio-Suarez syndrome (378007); Morquio-Ullrich disease (378007); Osteochondrodystrophia deformans (378007); Atypical chondrodystrophy (378007); Morquio disease (378007); Familial osteochondrodystrophy (378007); Brailsford-Morquio syndrome (378007); Hereditary enchondral dysostosis (378007); Chondrodystrophia tarda (378007); Familial osseous dystrophy (378007); Keratan sulfaturia (378007); Mucopolysaccharidosis, MPS-IV (378007); Morquio-Brailsford disease (378007); Osteochondrodystrophy (378007); Mucopolysaccharidosis type IV (378007)
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
 
Related genes: GLB1, GALNS
 
Monarch Initiative: MONDO:0018938
Orphanet: ORPHA582

Definition

The first signs and symptoms of MPS IV usually become apparent during early childhood. Affected individuals develop various skeletal abnormalities, including short stature, knock knees, and abnormalities of the ribs, chest, spine, hips, and wrists. People with MPS IV often have joints that are loose and very flexible (hypermobile), but they may also have restricted movement in certain joints. A characteristic feature of this condition is underdevelopment (hypoplasia) of a peg-like bone in the neck called the odontoid process. The odontoid process helps stabilize the spinal bones in the neck (cervical vertebrae). Odontoid hypoplasia can lead to misalignment of the cervical vertebrae, which may compress and damage the spinal cord, resulting in paralysis or death.

Mucopolysaccharidosis type IV (MPS IV), also known as Morquio syndrome, is a progressive condition that mainly affects the skeleton. The rate at which symptoms worsen varies among affected individuals.

In people with MPS IV, the clear covering of the eye (cornea) typically becomes cloudy, which can cause vision loss. Some affected individuals have recurrent ear infections and hearing loss. The airway may become narrow in some people with MPS IV, leading to frequent upper respiratory infections and short pauses in breathing during sleep (sleep apnea). Other common features of this condition include mildly "coarse" facial features, thin tooth enamel, multiple cavities, heart valve abnormalities, a mildly enlarged liver (hepatomegaly), and a soft out-pouching around the belly-button (umbilical hernia) or lower abdomen (inguinal hernia). Unlike some other types of mucopolysaccharidosis, MPS IV does not affect intelligence.

The life expectancy of individuals with MPS IV depends on the severity of symptoms. Severely affected individuals may survive only until late childhood or adolescence. Those with milder forms of the disorder usually live into adulthood, although their life expectancy may be reduced. Spinal cord compression and airway obstruction are major causes of death in people with MPS IV. [from MedlinePlus Genetics]

Professional guidelines

PubMed

Sawamoto K, Álvarez González JV, Piechnik M, Otero FJ, Couce ML, Suzuki Y, Tomatsu S
Int J Mol Sci 2020 Feb 23;21(4) doi: 10.3390/ijms21041517. PMID: 32102177Free PMC Article
Charrow J, Alden TD, Breathnach CA, Frawley GP, Hendriksz CJ, Link B, Mackenzie WG, Manara R, Offiah AC, Solano ML, Theroux M
Mol Genet Metab 2015 Jan;114(1):11-8. Epub 2014 Oct 30 doi: 10.1016/j.ymgme.2014.10.010. PMID: 25496828
Hendriksz CJ, Al-Jawad M, Berger KI, Hawley SM, Lawrence R, Mc Ardle C, Summers CG, Wright E, Braunlin E
J Inherit Metab Dis 2013 Mar;36(2):309-22. Epub 2012 Feb 23 doi: 10.1007/s10545-012-9459-0. PMID: 22358740Free PMC Article

Recent clinical studies

Etiology

Sahin MC, Ipek EM, Zinnuroglu M, Borcek AO
Childs Nerv Syst 2023 May;39(5):1323-1328. Epub 2022 Nov 18 doi: 10.1007/s00381-022-05748-5. PMID: 36400977
Andreescu N, Sharma A, Mihailescu A, Zimbru CG, David VL, Horhat R, Kundnani NR, Puiu M, Farcas S
Eur Rev Med Pharmacol Sci 2022 Jul;26(14):5107-5114. doi: 10.26355/eurrev_202207_29298. PMID: 35916808
Charrow J, Alden TD, Breathnach CA, Frawley GP, Hendriksz CJ, Link B, Mackenzie WG, Manara R, Offiah AC, Solano ML, Theroux M
Mol Genet Metab 2015 Jan;114(1):11-8. Epub 2014 Oct 30 doi: 10.1016/j.ymgme.2014.10.010. PMID: 25496828
Theroux MC, Nerker T, Ditro C, Mackenzie WG
Paediatr Anaesth 2012 Sep;22(9):901-7. Epub 2012 Jun 27 doi: 10.1111/j.1460-9592.2012.03904.x. PMID: 22738181
McKay SD, Al-Omari A, Tomlinson LA, Dormans JP
Spine (Phila Pa 1976) 2012 Mar 1;37(5):E269-77. doi: 10.1097/BRS.0b013e31823b3ded. PMID: 22045003

Diagnosis

Padash S, Obaid H, Henderson RDE, Padash Y, Adams SJ, Miller SF, Babyn P
Pediatr Radiol 2023 May;53(5):971-983. Epub 2023 Jan 11 doi: 10.1007/s00247-022-05585-3. PMID: 36627376
Campbell JW
Neurosurg Clin N Am 2022 Jan;33(1S):e1-e10. Epub 2022 Aug 6 doi: 10.1016/j.nec.2022.02.001. PMID: 36115686
Sawamoto K, Álvarez González JV, Piechnik M, Otero FJ, Couce ML, Suzuki Y, Tomatsu S
Int J Mol Sci 2020 Feb 23;21(4) doi: 10.3390/ijms21041517. PMID: 32102177Free PMC Article
Silveira MRMD, Buriti AKL, Martins AM, Gil D, Azevedo MF
Clinics (Sao Paulo) 2018 Dec 3;73:e523. doi: 10.6061/clinics/2018/e523. PMID: 30517303Free PMC Article
Raff ML, Byers PH
Curr Opin Rheumatol 1996 Sep;8(5):459-66. doi: 10.1097/00002281-199609000-00012. PMID: 8941450

Therapy

Padash S, Obaid H, Henderson RDE, Padash Y, Adams SJ, Miller SF, Babyn P
Pediatr Radiol 2023 May;53(5):971-983. Epub 2023 Jan 11 doi: 10.1007/s00247-022-05585-3. PMID: 36627376
Yahanda AT, Chicoine MR
World Neurosurg 2020 Jul;139:151-157. Epub 2020 Apr 17 doi: 10.1016/j.wneu.2020.04.016. PMID: 32305599
Theroux MC, Nerker T, Ditro C, Mackenzie WG
Paediatr Anaesth 2012 Sep;22(9):901-7. Epub 2012 Jun 27 doi: 10.1111/j.1460-9592.2012.03904.x. PMID: 22738181
Valayannopoulos V, Wijburg FA
Rheumatology (Oxford) 2011 Dec;50 Suppl 5:v49-59. doi: 10.1093/rheumatology/ker396. PMID: 22210671
Martell L, Lau K, Mei M, Burnett V, Decker C, Foehr ED
Orphanet J Rare Dis 2011 Dec 16;6:84. doi: 10.1186/1750-1172-6-84. PMID: 22176730Free PMC Article

Prognosis

Averill LW, Kecskemethy HH, Theroux MC, Mackenzie WG, Pizarro C, Bober MB, Ditro CP, Tomatsu S
Pediatr Radiol 2021 Jun;51(7):1202-1213. Epub 2021 Feb 20 doi: 10.1007/s00247-020-04946-0. PMID: 33608742Free PMC Article
Kaissi AA, van Egmond-Fröhlich A, Ryabykh S, Ochirov P, Kenis V, Hofstaetter JG, Grill F, Ganger R, Kircher SG
Medicine (Baltimore) 2016 Dec;95(50):e5505. doi: 10.1097/MD.0000000000005505. PMID: 27977582Free PMC Article
Kubaski F, Tomatsu S, Patel P, Shimada T, Xie L, Yasuda E, Mason R, Mackenzie WG, Theroux M, Bober MB, Oldham HM, Orii T, Shaffer TH
Mol Genet Metab 2015 Aug;115(4):186-92. Epub 2015 Jun 23 doi: 10.1016/j.ymgme.2015.06.007. PMID: 26116954Free PMC Article
Dhawale AA, Thacker MM, Belthur MV, Rogers K, Bober MB, Mackenzie WG
J Pediatr Orthop 2012 Jul-Aug;32(5):534-40. doi: 10.1097/BPO.0b013e318259fe57. PMID: 22706472
Nelson J, Crowhurst J, Carey B, Greed L
Am J Med Genet A 2003 Dec 15;123A(3):310-3. doi: 10.1002/ajmg.a.20314. PMID: 14608657

Clinical prediction guides

Averill LW, Kecskemethy HH, Theroux MC, Mackenzie WG, Pizarro C, Bober MB, Ditro CP, Tomatsu S
Pediatr Radiol 2021 Jun;51(7):1202-1213. Epub 2021 Feb 20 doi: 10.1007/s00247-020-04946-0. PMID: 33608742Free PMC Article
Hughes D, Giugliani R, Guffon N, Jones SA, Mengel KE, Parini R, Matousek R, Hawley SM, Quartel A
Orphanet J Rare Dis 2017 May 23;12(1):98. doi: 10.1186/s13023-017-0634-0. PMID: 28535791Free PMC Article
Kubaski F, Tomatsu S, Patel P, Shimada T, Xie L, Yasuda E, Mason R, Mackenzie WG, Theroux M, Bober MB, Oldham HM, Orii T, Shaffer TH
Mol Genet Metab 2015 Aug;115(4):186-92. Epub 2015 Jun 23 doi: 10.1016/j.ymgme.2015.06.007. PMID: 26116954Free PMC Article
Davison JE, Kearney S, Horton J, Foster K, Peet AC, Hendriksz CJ
J Inherit Metab Dis 2013 Mar;36(2):323-8. Epub 2012 Jan 10 doi: 10.1007/s10545-011-9430-5. PMID: 22231379
De Franceschi L, Roseti L, Desando G, Facchini A, Grigolo B
Osteoarthritis Cartilage 2007 Nov;15(11):1311-7. Epub 2007 Jun 4 doi: 10.1016/j.joca.2007.04.008. PMID: 17548214

Recent systematic reviews

Yahanda AT, Chicoine MR
World Neurosurg 2020 Jul;139:151-157. Epub 2020 Apr 17 doi: 10.1016/j.wneu.2020.04.016. PMID: 32305599

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