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Autosomal dominant osteopetrosis

MedGen UID:
1378401
Concept ID:
C4272579
Disease or Syndrome
Synonyms: Autosomal Dominant Osteopetrosis; autosomal dominant osteopetrosis; autosomal dominant osteopetrosis (disease); OPTA; osteopetrosis (disease), autosomal dominant
 
Related genes: LRP5, CLCN7
 
Monarch Initiative: MONDO:0020645
OMIM® Phenotypic series: PS607634

Definition

An autosomal dominant form of osteopetrosis due to mutation(s) in the CLCN7 gene, encoding H(+)/Cl(-) exchange transporter 7. Clinical features include sclerosis involving the spine, the pelvis, and the base of the skull. Complications can include optic nerve compression, dental abscesses, anemia, and bone fragility. One third of individuals who carry a CLCN7 mutation have a normal skeletal phenotype. [from NCI]

Professional guidelines

PubMed

Teti A, Econs MJ
Bone 2017 Sep;102:50-59. Epub 2017 Feb 4 doi: 10.1016/j.bone.2017.02.002. PMID: 28167345
Del Fattore A, Peruzzi B, Rucci N, Recchia I, Cappariello A, Longo M, Fortunati D, Ballanti P, Iacobini M, Luciani M, Devito R, Pinto R, Caniglia M, Lanino E, Messina C, Cesaro S, Letizia C, Bianchini G, Fryssira H, Grabowski P, Shaw N, Bishop N, Hughes D, Kapur RP, Datta HK, Taranta A, Fornari R, Migliaccio S, Teti A
J Med Genet 2006 Apr;43(4):315-25. Epub 2005 Aug 23 doi: 10.1136/jmg.2005.036673. PMID: 16118345Free PMC Article

Recent clinical studies

Etiology

Zhang ZF, Wang D, Wu LD, Dai XS
Chin J Traumatol 2017 Jun;20(3):173-176. Epub 2017 May 10 doi: 10.1016/j.cjtee.2017.02.001. PMID: 28550971Free PMC Article
Teti A, Econs MJ
Bone 2017 Sep;102:50-59. Epub 2017 Feb 4 doi: 10.1016/j.bone.2017.02.002. PMID: 28167345
Stark Z, Savarirayan R
Orphanet J Rare Dis 2009 Feb 20;4:5. doi: 10.1186/1750-1172-4-5. PMID: 19232111Free PMC Article
Vázquez E, López-Arcas JM, Navarro I, Pingarrón L, Cebrián JL
Oral Maxillofac Surg 2009 Jun;13(2):105-8. doi: 10.1007/s10006-009-0151-y. PMID: 19219469
Gwynne Jones DP, Hodgson BF, Hung NA
J Bone Joint Surg Br 2004 Mar;86(2):276-8. doi: 10.1302/0301-620x.86b2.14354. PMID: 15046446

Diagnosis

Rajathi M, Austin RD, Mathew P, Bharathi CS, Srivastava KC
Indian J Dent Res 2010 Oct-Dec;21(4):611-4. doi: 10.4103/0970-9290.74234. PMID: 21187637
Kadono Y, Tanaka S, Nishino J, Nishimura K, Nakamura I, Miyazaki T, Takayanagi H, Nakamura K
Mod Rheumatol 2009;19(6):687-90. Epub 2009 Aug 6 doi: 10.1007/s10165-009-0208-7. PMID: 19657708
Stark Z, Savarirayan R
Orphanet J Rare Dis 2009 Feb 20;4:5. doi: 10.1186/1750-1172-4-5. PMID: 19232111Free PMC Article
de Vernejoul MC
Best Pract Res Clin Rheumatol 2008 Mar;22(1):71-83. doi: 10.1016/j.berh.2007.12.011. PMID: 18328982
Bollerslev J, Mosekilde L
Clin Orthop Relat Res 1993 Sep;(294):45-51. PMID: 8358946

Therapy

Alam I, Hardman SL, Gerard-O'Riley RL, Acton D, Parker RS, Hong JM, Bruzzaniti A, Econs MJ
Calcif Tissue Int 2024 Apr;114(4):419-429. Epub 2024 Feb 1 doi: 10.1007/s00223-023-01180-2. PMID: 38300304
Imel EA, Liu Z, Acton D, Coffman M, Gebregziabher N, Tong Y, Econs MJ
J Bone Miner Res 2019 Aug;34(8):1436-1445. Epub 2019 May 13 doi: 10.1002/jbmr.3715. PMID: 30889272Free PMC Article
Zhang ZF, Wang D, Wu LD, Dai XS
Chin J Traumatol 2017 Jun;20(3):173-176. Epub 2017 May 10 doi: 10.1016/j.cjtee.2017.02.001. PMID: 28550971Free PMC Article
Bollerslev J, Henriksen K, Nielsen MF, Brixen K, Van Hul W
Eur J Endocrinol 2013 Aug;169(2):R39-57. Epub 2013 Jul 13 doi: 10.1530/EJE-13-0136. PMID: 23744590
Al-Aubaidi Z, Pedersen NW
J Pediatr Orthop 2010 Mar;30(2):206-7. doi: 10.1097/BPO.0b013e3181cfcd63. PMID: 20179571

Prognosis

Jodeh W, Katz AJ, Hart M, Warden SJ, Niziolek P, Alam I, Ing S, Polgreen LE, Imel EA, Econs MJ
J Clin Endocrinol Metab 2024 Jun 17;109(7):1726-1732. doi: 10.1210/clinem/dgae040. PMID: 38261998Free PMC Article
Wang Z, Li X, Wang Y, Fu W, Liu Y, Zhang Z, Wang C
Front Endocrinol (Lausanne) 2022;13:819641. Epub 2022 Mar 17 doi: 10.3389/fendo.2022.819641. PMID: 35370969Free PMC Article
Zhang ZF, Wang D, Wu LD, Dai XS
Chin J Traumatol 2017 Jun;20(3):173-176. Epub 2017 May 10 doi: 10.1016/j.cjtee.2017.02.001. PMID: 28550971Free PMC Article
Al-Aubaidi Z, Pedersen NW
J Pediatr Orthop 2010 Mar;30(2):206-7. doi: 10.1097/BPO.0b013e3181cfcd63. PMID: 20179571
Gwynne Jones DP, Hodgson BF, Hung NA
J Bone Joint Surg Br 2004 Mar;86(2):276-8. doi: 10.1302/0301-620x.86b2.14354. PMID: 15046446

Clinical prediction guides

Jodeh W, Katz AJ, Hart M, Warden SJ, Niziolek P, Alam I, Ing S, Polgreen LE, Imel EA, Econs MJ
J Clin Endocrinol Metab 2024 Jun 17;109(7):1726-1732. doi: 10.1210/clinem/dgae040. PMID: 38261998Free PMC Article
Norwood I, Szondi D, Ciocca M, Coudert A, Cohen-Solal M, Rucci N, Teti A, Maurizi A
Bone 2021 Mar;144:115828. Epub 2020 Dec 28 doi: 10.1016/j.bone.2020.115828. PMID: 33359007
Ladd LM, Imel EA, Niziolek PJ, Liu Z, Warden SJ, Liang Y, Econs MJ
Skeletal Radiol 2021 May;50(5):903-913. Epub 2020 Oct 3 doi: 10.1007/s00256-020-03625-3. PMID: 33009917Free PMC Article
Li L, Lv SS, Wang C, Yue H, Zhang ZL
Mol Med Rep 2019 Jun;19(6):5030-5038. Epub 2019 Apr 3 doi: 10.3892/mmr.2019.10123. PMID: 30942407
Butscheidt S, Rolvien T, Kornak U, Schmidt FN, Schinke T, Amling M, Oheim R
Calcif Tissue Int 2018 Jan;102(1):41-52. Epub 2017 Oct 10 doi: 10.1007/s00223-017-0332-x. PMID: 29018903

Recent systematic reviews

Carvalho PHA, Moura LB, Real Gabrielli MF, Cabrini Gabrielli MA, Antonio Pereira Filho V
J Craniomaxillofac Surg 2018 Nov;46(11):1905-1910. Epub 2018 Sep 24 doi: 10.1016/j.jcms.2018.09.004. PMID: 30309794

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