When They Mutate by Dr. Apurva Mishra & Prof. R. K. Pandey - HTML preview

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MANIFESTATIONS OF GENETIC DISEASES

 

1.Adiposity, hyperthermia, oligomenorrhea and parotid swelling syndrome :

 

This is also known as AHOP or AOP syndrome. It is caused by a hereditary alteration of some part of the diencephalon. It is thought to be transmitted as sex linked dominant character.  Clinical features :

 

The clinical findings are usually limited to females. It  consists of adiposity, hyperthermia, oligomenorrhea, parotid swelling and psychic disturbances. The parotid swelling usually begins at the time of puberty and is bilateral. Occasionally the submandibular glands are also involved.

 

2.AARSKOG SYNDROME

(Faciodigitogenital Syndrome)

 

Aarskog–Scott syndrome is a rare disease inherited as  autosomal dominant or X-linked and characterized by short stature, facial abnormalities, skeletal and genital anomalies. The Aarskog– Scott syndrome (AAS) is also known as the Aarskog syndrome, faciodigitogenital syndrome, shawl scrotum syndrome and faciogenital dysplasia.

 

Clinical Features:

 

The Aarskog–Scott syndrome is a disorder with short  stature, hypertelorism, downslanting palpebral fissures, anteverted nostrils, joint laxity, shawl scrotum, and mental retardation. The physical phenotype varies with age and postpuberal males may have only minor remnant manifestations of the prepuberal phenotype.

 

Growth mild to moderate short stature evident by 1–3 years of age

  • delayed adolescent growth spurt
  • Performance slight (dull normal) to moderate mental deficiency
  • hyperactivity and attention deficit
  • social performance usually good
  • Face, rounded face
  • widow's peak hairline
  • wide-set eyes (hypertelorism)
  • droopy eyelids (blepharoptosis)
  • downslanting eye slits (palpebral fissures)
  • small nose with nostrils tipped forward (anteverted)
  • underdeveloped mid-portion of the face (maxilla)
  • wide groove above the upper lip (broad philtrum)
  • crease below the lower lip
  • delayed eruption of teeth
  • top portion (upper helix) of the ear folded over slightly
  • Hands and feet
  • small, broad hands and feet
  • short fingers and toes (brachydactyly)
  • in-curving of the 5th finger (clinodactyly)
  • mild interdigital webbing, between fingers as well as toes
  • single transverse "simian crease" in palm
  • broad thumbs and big toes
  • Neck, short neck
  • webbing of sides of the neck
  • Chest, mild pectus excavatum (sunken chest)
  • Abdomen, protruding navel
  • inguinal hernias
  • shawl scrotum
  • undescended testicles

 

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3) Adrenogenital syndrome :

  • It occurs due to hyperplasia or tumors of the renal cortex.
  • Pseudohermaphroditism, sexual precocity and virilizem in  women or ferminization in men.
  • Premature eruption of teeth if the disease begins in the early life.

 

4) Aglossia adactylia syndrome :

(Hypoglossia hypodactylia syndrome)

 

The etiology for the condition is unknown. There is no  evidence that heredity plays a role in its genesis. There is no sex predilection. There is absence of tongue associated with failure of development of digits. The tongue in most cases is completely absent, or may be present as a small lobule in posterior part of the mouth. But the speech is not severely impaired. The sublingual muscular ridges may be markedly enlarged. Sublingual and submandibular glands may be hypertrophic. The mandible is usually small and poorly developed. Lower incisors have been noted to be missing. Extremities are severely affected, from peromelia to absence of single digit. Both hands and feet are usually involved. Syndactyly has also been reported. Intelligence in these patients is not impaired.

 

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5) Aldrich’s syndrome :

(‘Wiskot Aldrich’ syndrome)

 

Wiskott–Aldrich syndrome was linked in 1994[6] to  mutations in a gene on the short arm of the X chromosome, which was termed Wiskott-Aldrich syndrome protein gene (WASp). The disease X-linked thrombocytopenia was later discovered to be also due to WASp mutations, but different ones from those that cause full-blown Wiskott–Aldrich syndrome

 

  • Inheritance: X-linked recessive
  • It is characterized by thrombocytopenic purpura,eczema and increased succesptibilty to infection.
  • Spontaneous bleeding from gingival and palatal petechiae can be seen.

 

Classification:

Jin et al. (2004) employ a numerical grading of severity:

0.5: intermittent thrombocytopenia

1.0: thrombocytopenia and small platelets (microthrombocytopenia)

2.0: microthrombocytopenia plus normally responsive eczema  or occasional upper respiratory tract infections

2.5: microthrombocytopenia plus therapy-responsive but severe  eczema or airway infections requiring antibiotics

3.0: microthrombocytopenia plus both eczema and airway  infections requiring antibiotics

4.0: microthrombocytopenia plus eczema continuously  requiring therapy and/or severe or life-threatening infections

5.0: microthrombocytopenia plus autoimmune disease or malignancy

 

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6) Amelo-onycho-hypohydrotic syndrome :

 

A rare disorder characterized primarily by tooth and nail abnormalities and reduced sweating ability Clincal features:

  • Late tooth eruption
  • Premature tooth eruption
  • Unusual tooth shape
  • Everted lower lip
  • Decreased sweating
  • Dental staining
  • Enamel anomaly
  • Anodontia
  • Oligodontia
  • Dry skin
  • Fine hair
  • Thin tooth enamel
  • Thickened skin under nails
  • Thin toenails
  • Thin fingernails
  • Hyperconvex nailsSeborrheic dermatitis will be present.
  • In oral features severe hypoplastic hypocalcified enamel seen.

 

7) Antley-Bixler Syndrome:

(Trapezoidocephaly-synostosis syndrome)

 

It is a rare, very severe autosomal recessive congenital disorder  characterized by malformations and deformities affecting the majority of the skeleton and other areas of the body.

 

The disorder is suggested to be genetically heterogenous as two distinct gene mutation (FGFR2 & POR gene) are responsible for phenotypic expression of this syndrome. 

 

General manifestations:

a) Coronal and lambdoid craniosynostosis

b) Brachycephaly

c) Proptosis

d) Choanal stenosis/atresia

e) Maxillary hypoplasia

f) Humeroraadial synoostosis

g) Camptodactyly (fused interphalangeal joints  in the fingers)

h) Multiple contractures

i) Cardiac malformations

j) Nasal/anal atresia

 

8) Albright’s Syndrome :

(Polyostotic fibrous dysplasia, cutaneous pigmentation and endocrine disorders, Brown spot syndrome).

 

Etiology of the syndrome is obscure. Heredity does not appear to be a factor in the syndrome. The syndrome consists of a classic triad of polyostotic fibrous dysplasia, pigmentation of skin and rarely mucous membrane and endocrine dysfunction.

 

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Oral manifestations –

Mandible is more commonly involved. It is enlarged,  expanded and distorted. Pigmentation of the lips and buccal mucosa is rarely seen.

 

Radiographic appearance :

 

Medullary portions of bone are rarefied and present irregular trabeculations. Radiographically the condition presents as a unilocular, multilocular or ground-glass or peau de orange appearance.

 

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9. Anderson Syndrome:

 (Familial Osteodysplasia)

 

It is inherited as an autosomal recessive trait. It is  characterized by craniofacial and skeletal anomalies, presence of hyperuricemia and diastolic hypertension.

 

Clincal features :

 

1. Mandibular prognathism.

2. Hypoplastic.

3. Maxilla and resultant malocclusion.

4. The mandible is with wide angle, increased body length, and reduced ramus and body height.

5. Spine abnormalities

6. Collar bone abnormalities

7. Pelvic abnormalities

8. Thigh bone abnormalities

9. Foot abnormalities

10. Skull abnormalities

11. High uric acid level in blood

12. Diastolic hypertension

13. Recurring fractures

14. Abnormal tooth positioning

15. Flat nose

16. Flat cheek bones

17. Kyphosis

18. Large nose

19. Pointy chin

20. Dental decay

21. Curved fifth finger

22. High blood pressure

23. Thick eyebrows

 

10) Angioosteohypertrophy Syndrome :

 (Klippel-Trenaunay-Weber syndrome, Hemangiectatic hypertrophy)

 

 The syndrome consists of unilateral nevus flammeus of the skin arranged segmentally, varices and hypertrophy of the skeletal and soft tissue developing at about the same time. It is transmitted as either an autosomal dominant or recessive trait.

 

Oral manifestations :

 

The soft and hard palate are most frequently involved.  Angiomatosis of the tongue and pharynx were also reported. There is asymmetry of face because of bony hypertrophy; resulting in malocclusion and premature eruption of teeth on the involved side.

 

11) Ankyloglossum Superius Syndrome :

It consists of congenital ankylosis of tongue to the hard palate and anomalies of hands and feet such as hypoplasia or aplasia of digits and syndactyly.

 

Oral manifestations :

 

The tongue is usually attached to the hard palate, but may  also be attached to upper alveolar ridge. The tongue is attached usually in the anterior part. The tongue functions like mobility and extrusion beyond the teeth are hindered. The upper central portion of lip is usually hypoplastic and mandible has been described as hypoplastic. Cleft lip and palate has also been recorded.

 

12) Aortic arch syndrome :

(Subclavian artery occlusive syndrome, subclavian steal syndrome, vetebral –basilar artery occlusive syndrome, pulseless diesease)

 

 Aortic arch syndrome problems are most often associated with trauma, blood clots, or malformations that develop before birth. The arteries' defects result in abnormal blood flow to the head, neck, or arms. In children, there are multiple types of aortic arch syndromes, including:

 

  • Congenital absence of a branch of the aorta
  • Isolation of the subclavian arteries
  • Vascular rings

 

Symptoms vary according to the affected artery, but may include:

 

  • Neurological changes such as:
  • Dizziness
  • Blurred vision
  • Weakness
  • Blood pressure changes
  • Breathing problems
  • Numbness of an arm
  • Reduced pulse
  • Transient ischemic attacks
  • General features include dizziness, headache, visual disturbance and anginal pain.
  • In oral features there is tropic ulceration and pain while  chewing. It results due to deficient blood supply to the muscles of mastication.

 

13) Apert’s Syndrome (Acrocephalosyndactyly) :

It is classified as a branchial arch syndrome, affecting thefirst branchial (or pharyngeal) arch, the precursor of the maxilla andmandible.

 

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Apert syndrome is a developmental malformation characterized by–

1) Craniosynostosis

2) A cone shaped calvarium

3) Midface hypoplasia

4) Pharyngeal attenuation

5) Ocular manifestations

6) Syndactyly of the hands and feet

7) Mutations of either Ser252Trp or Pro253Arg in fibroblast  growth factor receptor 2 (FGFR2) are responsible for nearly all known cases of Apert syndrome.

8) Common dental features of acrocephalosyndactyly are a higharched palate, pseudomandibular prognathism (appearing as mandibular prognathism), a narrow palate, and crowding of the teeth.

 

Clinical features :

a) Face :

Face is usually asymmetric. The middle third of the face is underdeveloped and flat, producing a relative prognathism. The nose is usually described as parrot’s beak. Hypertelorism, exophthalmos and strabismus are often reported.

 

b) Skull :

The cranium is usually oxycephalic in appearance. The apex of the cranium is located near to or anterior to the bregma. Anterior fontanelle is open in numerous patients.

 

14) Ascher’s Syndrome:

(Laffer-Ascher Syndrome)

 

The etiology is unknown. However hormonal dysfunction,  trauma and heredity are suggested as etiological agents. It is characterized by repeated episodes of lip and eyelid edema and occasionally euthyroid goiter. The syndrome generally occurs within the first 20 years of life

 

Oral manifestations :

 

There is a horizontal line running between the inner and  outer parts of upper lip. Very rarely lower lip is also enlarged. The enlargement of lip may exist from childhood. The ‘extra lip’ or the tissue is usually seen when the patient smiles or during talking.

 

Microscopic examination of this excessive tissue usually consists of loose areolar tissue and hyperplastic mucous glands.

 

15) Ataxia Telangiectasia Syndrome:

 Also referred to as Louis–Bar syndrome is a rare, neurodegenerative, inherited disease causing severe disability. Ataxia refers to poor coordination and telangiectasia to small dilated blood vessels, both of which are hallmarks of the disease. Mutations in the ATM gene cause ataxia-telangiectasia. The ATM gene provides instructions for making a protein that helps control cell division and is involved in DNA repair.

 

It is a neurocutaneous disorder transmitted as an autosomal recessive trait.

 

Clinical features:

 

a) Face:

Face is relaxed, dull or sad. Shoulders are drooped and the head is tilted to one side. There are mild athetoid movements around the shoulders.

 

b) Oral manifestations:

Telangiectasia of the hard and soft palate has been  reported. There is drooling of saliva and speech is slow, slurred and interrupted. Eye movements are slow and halted during lateral and upward gaze.

 

Warning Signs of a Swallowing Problem

  • Choking or coughing when eating or drinking
  • Poor weight gain (during ages of expected growth) or weight loss at any age.
  • Excessive drooling
  • Mealtimes longer than 40 – 45 minutes, on a regular basis
  • Foods or drinks previously enjoyed are now refused or difficult
  • Chewing problems
  • Increase in the frequency or duration of breathing or respiratory
  • problems
  • Increase in lung infections

 

16) Baby Bottle Syndrome :

 

Bottle mouth syndrome; also called as Nursing bottle caries. This syndrome has been attributed to prolonged use of:

1. Nursing bottle containing milk or milk formula, fruit juice or sweet water.

2. Breastfeeding or

3. Sugar or honey, sweetened pacifiers.

 

Clinical features :

 

It presents as a widespread carious destruction of deciduous  teeth, most commonly the maxillary incisors, followed by first molars and then the cuspids. But the mandibular incisors are spared, which distinguishes this disease from rampant caries.

 

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17) Beckwith-Wiedemann Syndrome :

Five common features used to define BWS are: macroglossia, macrosomia (birth weight and length greater than the 90th percentile), midline abdominal wall defects (omphalocele/ exomphalos, umbilical hernia, diastasis recti), ear creases or ear pits, and neonatal hypoglycemia (low blood sugar after birth).

 

Oral features:

1. Macroglossia

2. Malocclusion,

3. Anterior open bite

4. Widely spaced teeth likely due to macroglossia

5. Speech problems due to macroglossia

 

Most children with BWS do not have all of these five features. In addition, some children with BWS have other findings including: nevus flammeus, prominent occiput, midfacehypoplasia, hemihypertrophy, genitourinary anomalies (enlarged kidneys), cardiac anomalies, musculoskeletal abnormalities, and hearing loss. Also, some premature newborns with BWS do not have macroglossia until closer to their anticipated delivery date.

 

18) Beal syndrome:

(Congenital contractual arachnodactyly)

 

It is a rare congenital connective tissue disorder. It is  caused by a mutation in FBN2 gene on chromosome 5q23. Contractures of varying degrees at birth, mainly involving the large joints, are present in all affected children. Elbows, knees and fingers are most commonly involved. The contractures may be mild and tend to reduce in severity.

 

Manifestations:

a. Cranial deformity

b. Bowed long bones

c. Muscle hypoplasia

d. Mitral valve prolapse

e. Arthrogyposis

f. Arachnodactyly

g. Kyphoscoliosis

h. Abnormal helix of ear (crumpled ear)

 

19) Behcet’s Syndrome:

 

Behcet’s disease is a systemic vasculitis characterized by recurrent oral and genital ulcers, and ocular inflammation, and which may involve the joints, skin, central nervous system and gastrointestinal tract. It is most common in those of Mediterranean and Eastern origin, although it also affects Caucasians. Genetic susceptibility :

 

The genetic locus most widely studied in Behcet’s disease is the human leukocyte antigen (HLA) complex on chromosome.

 

Clinical features :

 

1. Recurrent aphthous ulceration –

  • Oral ulcers
  • Genital ulcers

 

2. Skin disease –

  • Erythema nodosum
  • Superficial thrombophlebitis
  • Papulopustular lesions and acneiform nodules
  • Pathergy is the name give to non-specific  hyperreactivity of the skin following minor trauma, which is specific to Behcet’s disease.

 

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20) Blepharonasal Facial Syndrome:

 

It is inherited as an autosomal dominant trait. It is characterized by joint disorders, craniofacial anomalies and mental retardation. Individual presents with microcephaly, and amtomongoloid slant of palpebral fissures, hypoplastic maxial, protruding lip and mild malocclusion as a result of mid facial hypoplasia.

 

21) Bloch-Sulzberger Syndrome:

 

Incontinentia pigmenti also known as "Bloch–Siemens syndrome, "Bloch–Sulzbergerdisease, "Bloch–Sulzberger syndrome "melanoblastosis cutis," and "naevus pigmentosus systematicus". It is a genetic disorder that affects the skin, hair, teeth, nails, and central nervous system. It is named due to its microscopic appearance.

 

 It is transmitted as a sex-linked dominant trait, and it is lethal in males. More than 95% of reported cases occur in females. The etiology is not known. It is postulated that viral or infectious causes during pregnancy are predisposing factors.

 

a) Oral manifestations :

Oral changes are limited to the teeth. They include delayed  teeth eruption, pegged or conical shaped crowns of teeth, missing  teeth, malformed teeth and additional cusps. Both deciduous and permanent teeth are affected.

 

b) Skin and skin appendages :

After 2–3 days of birth, linear or grouped vesicles  containing a honey colored serum appear on extremities. By the end of the first month the vesicles may disappear, recur or be replaced by irregularly distributed violaceous papules and inflammatory lesions. Pigmented macules, brownish-gray in color and arranged in a reticulated pattern or in streaks, whorls or patches over trunk and extremities are seen at birth. This pigmentation begins to fade within a few years. It is heavy melanin pigmentation of the epithelium, dropping down into clusters of chromatophores in the upper dermis (incontinence, which gives the disease its name and is considered the hallmark of the syndrome). There is generalized baldness. Rarely finger nails are dystrophic and the breasts asymmetric. During the vesicular stage, microscopic examination reveals dense intraepithelial (or occasionally subepithelial) vesicles containing eosinophils.

 

22) Bogorad’s Syndrome

(Crocodile tears syndrome, Gustatory lacrimation)

 

In this condition the patient exhibits profuse lacrimation  when food is eaten; particularly hot and spicy food. It generally follows facial paralysis; either of Bell’s palsy type or the result of herpes zoster, head injury or intracranial operative trauma.

 

Facial nerve has several components – motor, sensory and autonomic – parasympathetic component. The autonomic parasympathetic fi bers arise in superior salivatory nucleus provide secretory and vasodilatory function to submandibular sublingual glands, via chorda tympani and to lacrimal gland the nerve of Wrisberg. If a lesion occurs proximal to geniculate ganglion, then during regeneration, fibers destined to submandibular and sublingual can get interchanged with those destined for lacrimal gland. Thus when gustatory stimuli a