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Hereditary spastic paraplegia 3A(SPG3A)

MedGen UID:
419393
Concept ID:
C2931355
Disease or Syndrome
Synonyms: FAMILIAL SPASTIC PARAPLEGIA, AUTOSOMAL DOMINANT, 1; Spastic paraplegia 3; SPASTIC PARAPLEGIA 3, AUTOSOMAL DOMINANT; Spastic Paraplegia 3A; Spastic paraplegia 3A, autosomal dominant; SPG3; SPG3A; Strumpell disease
SNOMED CT: Autosomal dominant spastic paraplegia type 3 (782670003); Strumpell disease (782670003)
Modes of inheritance:
Autosomal dominant inheritance
MedGen UID:
141047
Concept ID:
C0443147
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 heterozygotes. In the context of medical genetics, an autosomal dominant disorder is caused when a single copy of the mutant allele is present. Males and females are affected equally, and can both transmit the disorder with a risk of 50% for each child of inheriting the mutant allele.
 
Gene (location): ATL1 (14q22.1)
 
Monarch Initiative: MONDO:0008437
OMIM®: 182600
Orphanet: ORPHA100984

Disease characteristics

Excerpted from the GeneReview: Spastic Paraplegia 3A
Spastic paraplegia 3A (SPG3A; also known as ATL1-HSP) is characterized by progressive bilateral and mostly symmetric spasticity and weakness of the legs. Compared to other forms of autosomal dominant hereditary spastic paraplegia (HSP), in which diminished vibration sense (caused by degeneration of the corticospinal tracts and dorsal columns) and urinary bladder hyperactivity are present in all affected individuals, these findings occur in a minority of individuals with SPG3A. The average age of onset is four years. More than 80% of reported individuals manifest spastic gait before the end of the first decade of life. Most persons with early-onset ATL1-HSP have a "pure" ("uncomplicated") HSP; however, complicated HSP with axonal motor neuropathy and/or distal amyotrophy with lower motor neuron involvement (Silver syndrome phenotype) has been observed. The rate of progression in ATL1-HSP is slow, and wheelchair dependency or need for a walking aid (cane, walker, or wheelchair) is relatively rare. [from GeneReviews]
Authors:
Peter Hedera   view full author information

Additional descriptions

From OMIM
The hereditary spastic paraplegias are a group of clinically and genetically diverse disorders characterized by progressive, usually severe, lower extremity spasticity; see reviews of Fink et al. (1996) and Fink (1997). Zhao et al. (2001) noted that hereditary spastic paraplegia in the families of the SPG3A variety is characterized by early onset (before age 10 and usually before age 5 years). SPG is classified according to both the mode of inheritance (autosomal dominant, autosomal recessive (see 270800), and X-linked (see 303350)) and whether progressive spasticity occurs in isolation ('uncomplicated SPG') or with other neurologic abnormalities ('complicated SPG'), including optic neuropathy, retinopathy, extrapyramidal disturbance, dementia, ataxia, ichthyosis, mental retardation, and deafness. The major neuropathologic feature of autosomal dominant, uncomplicated SPG is axonal degeneration that is maximal in the terminal portions of the longest descending and ascending tracts (crossed and uncrossed corticospinal tracts to the legs and fasciculus gracilis, respectively). Spinocerebellar fibers are involved to a lesser extent. Since the description of 'pure' hereditary spastic paraparesis of late onset by Strumpell (1904), many 'complicated' forms of the disorder have been reported and the question as to whether a 'pure' form exists has been raised off and on. Probably in large part because of their exceptional length, the pyramidal tracts are unusually vulnerable to both acquired and genetic derangement. Although a majority of reported families have displayed recessive inheritance, 10 to 30% of families have a dominant pattern and in fact recessive inheritance of a 'pure' spastic paraplegia may be rare. Genetic Heterogeneity of Autosomal Dominant Spastic Paraplegia In addition to SPG3A, other forms of autosomal dominant spastic paraplegia for which the molecular basis is known include SPG4 (182601), caused by mutation in the SPAST gene (604277) on 2p22; SPG6 (600363), caused by mutation in the NIPA1 gene (608145) on 15q11; SPG8 (603563), caused by mutation in the WASHC5 gene (610657) on 8q24; SPG9A (601162), caused by mutation in the ALDH18A1 gene (138250) on 10q24; SPG10 (604187), caused by mutation in the KIF5A gene (602821) on 12q13; SPG12 (604805), caused by mutation in the RTN2 gene (603183) on 19q13; SPG13 (605280), caused by mutation in the SSPD1 gene (118190) on 2q33; SPG17 (270685), caused by mutation in the BSCL2 gene (606158) on 11q12; SPG18A (620512), caused by mutation in the ERLIN2 gene (611605) on 8p11; SPG30 (610357), caused by mutation in the KIF1A gene (601255) on 2q37; SPG31 (610250), caused by mutation in the REEP1 gene (609139) on 2p11; SPG33 (610244), caused by mutation in the ZFYVE27 gene (610243) on 10q24; SPG42 (612539), caused by mutation in the SLC33A1 gene (603690) on 3q25; SPG72 (615625), caused by mutation in the REEP2 gene (609347) on 5q31; SPG73 (616282), caused by mutation in the CPT1C gene (608846) on 19q13; SPG79A (620221), caused by mutation in the UCHL1 gene (191342) on chromosome 4p13; SPG80 (618418), caused by mutation in the UBAP1 gene (609787) on 9p13; SPG88 (620106), caused by mutation in the KPNA3 gene (601892) on 13q14; SPG90A (620416), caused by mutation in the SPTSSA gene (613540) on 14q13; and SPG91 (620538), caused by mutation in the SPTAN1 gene (182810) on 9q34. Autosomal dominant spastic paraplegia has been mapped to chromosomes 9q (SPG19; 607152), 1p31-p21 (SPG29; 609727), 12q23-q24 (SPG36; 613096), 8p21.1-q13.3 (SPG37; 611945), 4p16-p15 (SPG38; 612335), and 11p14.1-p11.2 (SPG41; 613364).  http://www.omim.org/entry/182600
From MedlinePlus Genetics
In addition to spasticity and weakness, which typically affect both legs equally, people with spastic paraplegia type 3A can also experience progressive muscle wasting (amyotrophy) in the lower limbs, reduced bladder control, an abnormal curvature of the spine (scoliosis), loss of sensation in the feet (peripheral neuropathy), or high arches of the feet (pes cavus). The signs and symptoms of spastic paraplegia type 3A usually appear before the age of 10; the average age of onset is 4 years. In some affected individuals the condition slowly worsens over time, sometimes leading to a need for walking support.

Spastic paraplegia type 3A is one of a group of genetic disorders known as hereditary spastic paraplegias. These disorders are characterized by muscle stiffness (spasticity) and weakness in the lower limbs (paraplegia). Hereditary spastic paraplegias are often divided into two types: pure and complex. The pure types involve only the lower limbs, while the complex types also involve other areas of the body; additional features can include changes in vision, changes in intellectual functioning, difficulty walking, and disturbances in nerve function (neuropathy). Spastic paraplegia type 3A is usually a pure hereditary spastic paraplegia, although a few complex cases have been reported.  https://medlineplus.gov/genetics/condition/spastic-paraplegia-type-3a

Clinical features

From HPO
Urinary incontinence
MedGen UID:
22579
Concept ID:
C0042024
Finding
Loss of the ability to control the urinary bladder leading to involuntary urination.
Urinary urgency
MedGen UID:
39315
Concept ID:
C0085606
Finding
Urge incontinence is the strong, sudden need to urinate.
Urinary bladder sphincter dysfunction
MedGen UID:
334804
Concept ID:
C1843663
Finding
Abnormal function of a sphincter of the urinary bladder.
Pes cavus
MedGen UID:
675590
Concept ID:
C0728829
Congenital Abnormality
An increase in height of the medial longitudinal arch of the foot that does not flatten on weight bearing (i.e., a distinctly hollow form of the sole of the foot when it is bearing weight).
Lower limb muscle weakness
MedGen UID:
324478
Concept ID:
C1836296
Finding
Weakness of the muscles of the legs.
Intellectual disability, mild
MedGen UID:
10044
Concept ID:
C0026106
Mental or Behavioral Dysfunction
Mild intellectual disability is defined as an intelligence quotient (IQ) in the range of 50-69.
Paraplegia
MedGen UID:
45323
Concept ID:
C0030486
Disease or Syndrome
Severe or complete weakness of both lower extremities with sparing of the upper extremities.
Babinski sign
MedGen UID:
19708
Concept ID:
C0034935
Finding
Upturning of the big toe (and sometimes fanning of the other toes) in response to stimulation of the sole of the foot. If the Babinski sign is present it can indicate damage to the corticospinal tract.
Spastic paraplegia
MedGen UID:
20882
Concept ID:
C0037772
Disease or Syndrome
Spasticity and weakness of the leg and hip muscles.
Hyperreflexia
MedGen UID:
57738
Concept ID:
C0151889
Finding
Hyperreflexia is the presence of hyperactive stretch reflexes of the muscles.
Spastic gait
MedGen UID:
115907
Concept ID:
C0231687
Finding
Spasticity is manifested by increased stretch reflex which is intensified with movement velocity. This results in excessive and inappropriate muscle activation which can contribute to muscle hypertonia. Spastic gait is characterized by manifestations such as muscle hypertonia, stiff knee, and circumduction of the leg.
Hypoplasia of the corpus callosum
MedGen UID:
138005
Concept ID:
C0344482
Congenital Abnormality
Underdevelopment of the corpus callosum.
Lower limb spasticity
MedGen UID:
220865
Concept ID:
C1271100
Finding
Spasticity (velocity-dependent increase in tonic stretch reflexes with increased muscle tone and hyperexcitable tendon reflexes) in the muscles of the lower limbs, hips, and pelvis.
Degeneration of the lateral corticospinal tracts
MedGen UID:
375921
Concept ID:
C1846566
Finding
Deterioration of the tissues of the lateral corticospinal tracts.
Impaired vibration sensation in the lower limbs
MedGen UID:
338617
Concept ID:
C1849134
Finding
A decrease in the ability to perceive vibration in the legs.
Motor delay
MedGen UID:
381392
Concept ID:
C1854301
Finding
A type of Developmental delay characterized by a delay in acquiring motor skills.
Scoliosis
MedGen UID:
11348
Concept ID:
C0036439
Disease or Syndrome
The presence of an abnormal lateral curvature of the spine.
Distal lower limb amyotrophy
MedGen UID:
324515
Concept ID:
C1836451
Disease or Syndrome
Muscular atrophy of distal leg muscles.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVHereditary spastic paraplegia 3A
Follow this link to review classifications for Hereditary spastic paraplegia 3A in Orphanet.

Recent clinical studies

Etiology

Hsu SL, Hsueh HW, Chen SY, Chang YY, Tan S, Hong CT, Tsai YS, Yu KW, Wu HM, Liao YC, Soong BW, Hu CJ, Lan MY, Lee YC
Parkinsonism Relat Disord 2021 Jun;87:87-91. Epub 2021 May 11 doi: 10.1016/j.parkreldis.2021.05.004. PMID: 34015694
Servelhere KR, Casseb RF, de Lima FD, Rezende TJR, Ramalho LP, França MC Jr
AJNR Am J Neuroradiol 2021 Mar;42(3):610-615. Epub 2021 Jan 21 doi: 10.3174/ajnr.A7017. PMID: 33478946Free PMC Article
Schiavoni S, Spagnoli C, Rizzi S, Salerno GG, Frattini D, Pisani F, Fusco C
Dev Med Child Neurol 2020 Sep;62(9):1068-1074. Epub 2020 Apr 10 doi: 10.1111/dmcn.14547. PMID: 32277485
Park H, Kang SH, Park S, Kim SY, Seo SH, Lee SJ, Lee JA, Cho SI, Sung JJ, Lee KW, Kim JY, Park SS, Seong MW
J Neurol Sci 2015 Oct 15;357(1-2):167-72. Epub 2015 Jul 17 doi: 10.1016/j.jns.2015.07.024. PMID: 26208798
Ivanova N, Claeys KG, Deconinck T, Litvinenko I, Jordanova A, Auer-Grumbach M, Haberlova J, Löfgren A, Smeyers G, Nelis E, Mercelis R, Plecko B, Priller J, Zámecník J, Ceulemans B, Erichsen AK, Björck E, Nicholson G, Sereda MW, Seeman P, Kremensky I, Mitev V, De Jonghe P
Arch Neurol 2007 May;64(5):706-13. doi: 10.1001/archneur.64.5.706. PMID: 17502470

Diagnosis

Hamamie-Chaar A, Renaud M, Gençpinar P, Bruel AL, Philippe C, Maraval J, Racine C, Hadouiri N, Lambert L, Schmitt E, Banneau G, Hocquel A, Thauvin-Robinet C, Faivre L, Thomas Q
J Neurol 2024 Sep;271(9):6343-6348. Epub 2024 Jul 13 doi: 10.1007/s00415-024-12565-0. PMID: 39003427Free PMC Article
Hsu SL, Hsueh HW, Chen SY, Chang YY, Tan S, Hong CT, Tsai YS, Yu KW, Wu HM, Liao YC, Soong BW, Hu CJ, Lan MY, Lee YC
Parkinsonism Relat Disord 2021 Jun;87:87-91. Epub 2021 May 11 doi: 10.1016/j.parkreldis.2021.05.004. PMID: 34015694
Schiavoni S, Spagnoli C, Rizzi S, Salerno GG, Frattini D, Pisani F, Fusco C
Dev Med Child Neurol 2020 Sep;62(9):1068-1074. Epub 2020 Apr 10 doi: 10.1111/dmcn.14547. PMID: 32277485
Park H, Kang SH, Park S, Kim SY, Seo SH, Lee SJ, Lee JA, Cho SI, Sung JJ, Lee KW, Kim JY, Park SS, Seong MW
J Neurol Sci 2015 Oct 15;357(1-2):167-72. Epub 2015 Jul 17 doi: 10.1016/j.jns.2015.07.024. PMID: 26208798
Yonekawa T, Oya Y, Higuchi Y, Hashiguchi A, Takashima H, Sugai K, Sasaki M
Pediatr Neurol 2014 Nov;51(5):726-9. Epub 2014 Jul 24 doi: 10.1016/j.pediatrneurol.2014.07.027. PMID: 25193411

Prognosis

Zhu PP, Patterson A, Lavoie B, Stadler J, Shoeb M, Patel R, Blackstone C
J Biol Chem 2003 Dec 5;278(49):49063-71. Epub 2003 Sep 23 doi: 10.1074/jbc.M306702200. PMID: 14506257

Clinical prediction guides

Hsu SL, Hsueh HW, Chen SY, Chang YY, Tan S, Hong CT, Tsai YS, Yu KW, Wu HM, Liao YC, Soong BW, Hu CJ, Lan MY, Lee YC
Parkinsonism Relat Disord 2021 Jun;87:87-91. Epub 2021 May 11 doi: 10.1016/j.parkreldis.2021.05.004. PMID: 34015694
Servelhere KR, Casseb RF, de Lima FD, Rezende TJR, Ramalho LP, França MC Jr
AJNR Am J Neuroradiol 2021 Mar;42(3):610-615. Epub 2021 Jan 21 doi: 10.3174/ajnr.A7017. PMID: 33478946Free PMC Article
Kawarai T, Montecchiani C, Miyamoto R, Gaudiello F, Caltagirone C, Izumi Y, Kaji R, Orlacchio A
J Neurol Sci 2017 Sep 15;380:92-97. Epub 2017 Jul 9 doi: 10.1016/j.jns.2017.07.011. PMID: 28870597
Cooper HM, Yang Y, Ylikallio E, Khairullin R, Woldegebriel R, Lin KL, Euro L, Palin E, Wolf A, Trokovic R, Isohanni P, Kaakkola S, Auranen M, Lönnqvist T, Wanrooij S, Tyynismaa H
Hum Mol Genet 2017 Apr 15;26(8):1432-1443. doi: 10.1093/hmg/ddx042. PMID: 28158749Free PMC Article
Zhu PP, Patterson A, Lavoie B, Stadler J, Shoeb M, Patel R, Blackstone C
J Biol Chem 2003 Dec 5;278(49):49063-71. Epub 2003 Sep 23 doi: 10.1074/jbc.M306702200. PMID: 14506257

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