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Stormorken syndrome(STRMK)

MedGen UID:
350028
Concept ID:
C1861451
Disease or Syndrome
Synonyms: STRMK; THROMBOCYTOPATHY, ASPLENIA, AND MIOSIS
SNOMED CT: Stormorken syndrome (711407000); Thrombocytopathy, asplenia and miosis (711407000); Stormorken-Sjaastad-Langslet syndrome (711407000)
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): STIM1 (11p15.4)
 
Monarch Initiative: MONDO:0008497
OMIM®: 185070
Orphanet: ORPHA3204

Definition

Stormorken syndrome is an autosomal dominant disorder characterized by mild bleeding tendency due to platelet dysfunction, thrombocytopenia, anemia, asplenia, tubular aggregate myopathy, congenital miosis, and ichthyosis. Additional features may include headache or recurrent stroke-like episodes (summary by Misceo et al., 2014). [from OMIM]

Additional description

From MedlinePlus Genetics
Stormorken syndrome is a rare condition that affects many body systems. Affected individuals usually have thrombocytopenia, in which there are abnormally low numbers of blood cells called platelets. Platelets are involved in normal blood clotting; a shortage of platelets typically results in easy bruising and abnormal bleeding. In addition, affected individuals often have a muscle disorder, called tubular aggregate myopathy, that leads to muscle weakness. Another feature of Stormorken syndrome is permanent constriction of the pupils of the eyes (miosis), which may be caused by abnormalities in the muscles that control the size of the pupils. Other features include lack of a functioning spleen (asplenia), scaly skin (ichthyosis), headaches, and difficulty with reading and spelling (dyslexia).  https://medlineplus.gov/genetics/condition/stormorken-syndrome

Clinical features

From HPO
Myalgia
MedGen UID:
68541
Concept ID:
C0231528
Sign or Symptom
Pain in muscle.
Hematuria
MedGen UID:
5488
Concept ID:
C0018965
Disease or Syndrome
The presence of blood in the urine. Hematuria may be gross hematuria (visible to the naked eye) or microscopic hematuria (detected by dipstick or microscopic examination of the urine).
Stroke-like episode
MedGen UID:
346558
Concept ID:
C1857287
Finding
No consensus exists on what a stroke-like episode is, but these episodes can be functionally defined as a new neurological deficit, occurring with or without the context of seizures, which last longer than 24 hours.
Short stature
MedGen UID:
87607
Concept ID:
C0349588
Finding
A height below that which is expected according to age and gender norms. Although there is no universally accepted definition of short stature, many refer to "short stature" as height more than 2 standard deviations below the mean for age and gender (or below the 3rd percentile for age and gender dependent norms).
Subarachnoid hemorrhage
MedGen UID:
11625
Concept ID:
C0038525
Disease or Syndrome
Hemorrhage occurring between the arachnoid mater and the pia mater.
Migraine
MedGen UID:
57451
Concept ID:
C0149931
Disease or Syndrome
Migraine is a chronic neurological disorder characterized by episodic attacks of headache and associated symptoms.
Dyslexia
MedGen UID:
96906
Concept ID:
C0476254
Mental or Behavioral Dysfunction
A learning disorder characterized primarily by difficulties in learning to read and spell. Dyslectic children also exhibit a tendency to read words from right to left and to confuse letters such as b and d whose orientation is important for their identification. Children with dyslexia appear to be impaired in phonemic skills (the ability to associate visual symbols with the sounds they represent).
Anemia
MedGen UID:
1526
Concept ID:
C0002871
Disease or Syndrome
A reduction in erythrocytes volume or hemoglobin concentration.
Epistaxis
MedGen UID:
4996
Concept ID:
C0014591
Pathologic Function
Epistaxis, or nosebleed, refers to a hemorrhage localized in the nose.
Howell-Jolly bodies
MedGen UID:
5644
Concept ID:
C0020058
Acquired Abnormality
Howell-Jolly bodies are small, intra-erythrocytic remnants of erythrocyte nuclei. These inclusions are solitary in each erythrocyte and strongly basophilic. These are often confused with overlying platelets, but can be distinguished by the presence of a halo around overlying platelets.
Thrombocytopenia
MedGen UID:
52737
Concept ID:
C0040034
Disease or Syndrome
A reduction in the number of circulating thrombocytes.
Abnormal bleeding
MedGen UID:
264316
Concept ID:
C1458140
Pathologic Function
An abnormal susceptibility to bleeding, often referred to as a bleeding diathesis. A bleeding diathesis may be related to vascular, platelet and coagulation defects.
Myopathy
MedGen UID:
10135
Concept ID:
C0026848
Disease or Syndrome
A disorder of muscle unrelated to impairment of innervation or neuromuscular junction.
Proximal muscle weakness
MedGen UID:
113169
Concept ID:
C0221629
Finding
A lack of strength of the proximal muscles.
Increased muscle fatiguability
MedGen UID:
892934
Concept ID:
C4025573
Finding
An abnormal, increased fatiguability of the musculature.
Hypoplastic spleen
MedGen UID:
410116
Concept ID:
C1970617
Anatomical Abnormality
Underdevelopment of the spleen.
Asplenia
MedGen UID:
1830315
Concept ID:
C5779621
Anatomical Abnormality
Absence (aplasia) of the spleen.
Elevated circulating creatine kinase concentration
MedGen UID:
69128
Concept ID:
C0241005
Finding
An elevation of the level of the enzyme creatine kinase (also known as creatine phosphokinase (CK; EC 2.7.3.2) in the blood. CK levels can be elevated in a number of clinical disorders such as myocardial infarction, rhabdomyolysis, and muscular dystrophy.
Increased circulating lactate dehydrogenase concentration
MedGen UID:
1377250
Concept ID:
C4477095
Finding
An elevated level of the enzyme lactate dehydrogenase in the blood circulation.
Prominent nose
MedGen UID:
98423
Concept ID:
C0426415
Finding
Distance between subnasale and pronasale more than two standard deviations above the mean, or alternatively, an apparently increased anterior protrusion of the nasal tip.
Short philtrum
MedGen UID:
350006
Concept ID:
C1861324
Finding
Distance between nasal base and midline upper lip vermilion border more than 2 SD below the mean. Alternatively, an apparently decreased distance between nasal base and midline upper lip vermilion border.
Ichthyosis
MedGen UID:
7002
Concept ID:
C0020757
Disease or Syndrome
An abnormality of the skin characterized the presence of excessive amounts of dry surface scales on the skin resulting from an abnormality of keratinization.
Bruising susceptibility
MedGen UID:
140849
Concept ID:
C0423798
Finding
An ecchymosis (bruise) refers to the skin discoloration caused by the escape of blood into the tissues from ruptured blood vessels. This term refers to an abnormally increased susceptibility to bruising. The corresponding phenotypic abnormality is generally elicited on medical history as a report of frequent ecchymoses or bruising without adequate trauma.
Miosis
MedGen UID:
6409
Concept ID:
C0026205
Disease or Syndrome
Abnormal (non-physiological) constriction of the pupil.
Deeply set eye
MedGen UID:
473112
Concept ID:
C0423224
Finding
An eye that is more deeply recessed into the plane of the face than is typical.
Hypotelorism
MedGen UID:
96107
Concept ID:
C0424711
Finding
Interpupillary distance less than 2 SD below the mean (alternatively, the appearance of an decreased interpupillary distance or closely spaced eyes).

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVStormorken syndrome
Follow this link to review classifications for Stormorken syndrome in Orphanet.

Professional guidelines

PubMed

Morin G, Biancalana V, Echaniz-Laguna A, Noury JB, Lornage X, Moggio M, Ripolone M, Violano R, Marcorelles P, Maréchal D, Renaud F, Maurage CA, Tard C, Cuisset JM, Laporte J, Böhm J
Hum Mutat 2020 Jan;41(1):17-37. Epub 2019 Sep 15 doi: 10.1002/humu.23899. PMID: 31448844

Recent clinical studies

Etiology

Riva B, Pessolano E, Quaglia E, Cordero-Sanchez C, Bhela IP, Topf A, Serafini M, Cox D, Harris E, Garibaldi M, Barresi R, Pirali T, Genazzani AA
Cell Calcium 2022 Jul;105:102605. Epub 2022 May 18 doi: 10.1016/j.ceca.2022.102605. PMID: 35636153
Nurden AT, Nurden P
J Thromb Haemost 2015 Jun;13 Suppl 1:S2-9. doi: 10.1111/jth.12898. PMID: 26149024

Diagnosis

Riva B, Pessolano E, Quaglia E, Cordero-Sanchez C, Bhela IP, Topf A, Serafini M, Cox D, Harris E, Garibaldi M, Barresi R, Pirali T, Genazzani AA
Cell Calcium 2022 Jul;105:102605. Epub 2022 May 18 doi: 10.1016/j.ceca.2022.102605. PMID: 35636153
Morin G, Biancalana V, Echaniz-Laguna A, Noury JB, Lornage X, Moggio M, Ripolone M, Violano R, Marcorelles P, Maréchal D, Renaud F, Maurage CA, Tard C, Cuisset JM, Laporte J, Böhm J
Hum Mutat 2020 Jan;41(1):17-37. Epub 2019 Sep 15 doi: 10.1002/humu.23899. PMID: 31448844
Li A, Kang X, Edelman F, Waclawik AJ
J Child Neurol 2019 May;34(6):321-324. Epub 2019 Feb 14 doi: 10.1177/0883073819829389. PMID: 30761937
Nurden AT, Nurden P
J Thromb Haemost 2015 Jun;13 Suppl 1:S2-9. doi: 10.1111/jth.12898. PMID: 26149024

Prognosis

Nurden AT, Nurden P
J Thromb Haemost 2015 Jun;13 Suppl 1:S2-9. doi: 10.1111/jth.12898. PMID: 26149024

Clinical prediction guides

Ticci C, Cassandrini D, Rubegni A, Riva B, Vattemi G, Matà S, Ricci G, Baldacci J, Guglielmi V, Di Muzio A, Malandrini A, Tonin P, Siciliano G, Federico A, Genazzani AA, Santorelli FM, Merlini L
Muscle Nerve 2021 Nov;64(5):567-575. Epub 2021 Aug 26 doi: 10.1002/mus.27391. PMID: 34368974
Silva-Rojas R, Treves S, Jacobs H, Kessler P, Messaddeq N, Laporte J, Böhm J
Hum Mol Genet 2019 May 15;28(10):1579-1593. doi: 10.1093/hmg/ddy446. PMID: 30576443
Lacruz RS, Feske S
Ann N Y Acad Sci 2015 Nov;1356(1):45-79. Epub 2015 Oct 15 doi: 10.1111/nyas.12938. PMID: 26469693Free PMC Article
Nurden AT, Nurden P
J Thromb Haemost 2015 Jun;13 Suppl 1:S2-9. doi: 10.1111/jth.12898. PMID: 26149024

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