Massively Parallel Sequencing
- Use to confirm a diagnosis of FMF in a symptomatic individual and to guide appropriate treatment options (response to colchicine differs for some pathogenic variants).
- May also be useful as a diagnostic or carrier test for individuals with a family history of FMF.
If a familial sequence variant has been previously identified, targeted sequencing for that variant may be appropriate; refer to the Laboratory Test Directory for additional information.
Familial Mediterranean fever (FMF) is a genetic condition characterized by recurrent but short-lived attacks of fever, abdominal pain, joint pain, and/or skin rashes. Symptoms and frequency of these attacks are highly variable. Renal amyloidosis is a common complication in untreated individuals and may be the only manifestation in some patients.
Disease Overview
Common symptoms of FMF include:
- Recurrent fever
- Erysipelas-like erythema
- Acute attacks of abdominal pain with board-like rigidity of the abdominal muscles, rebound tenderness, abdominal distension, and loss of peristaltic sounds
- Acute attacks of arthritis, often with synovial effusion
- Acute attacks of pleuritis
- Type AA amyloidosis, leading to nephrotic syndrome and end-stage renal disease if untreated
- Increased erythrocyte sedimentation rate (ESR)
- Leukocytosis
- Elevated serum fibrinogen concentration
Treatment with colchicine can prevent inflammatory attacks and deposition of amyloid in affected individuals, although specific treatment recommendations vary based on the disease-causing variant(s) and clinical history.
Genetics
Gene
MEFV (NM_000243)
Incidence
FMF is common among ethnic groups in the Mediterranean region, including individuals with Ashkenazi Jewish, Armenian, Turkish, Arab, North African Jewish, and Iraqi Jewish ancestry. Incidences of 1 in 400 to 1 in 1000 have been reported in these regions.
Inheritance
Autosomal recessive, although some heterozygous individuals may have symptoms
Genotype/Phenotype Correlations
- The most common severe variant in MEFV is p.Met694Val, which is almost always correlated with a severe disease course in homozygotes and a higher risk for amyloidosis compared with other variants.
- Most heterozygotes do not have symptoms, but autosomal dominant transmission of a milder phenotype with reduced penetrance has been reported in some families with severe MEFV variants such as p.Met694del and p.Ile692del.
- Milder variants such as p.Val726Ala have been associated with milder disease course or incomplete penetrance, with some homozygous or compound heterozygous individuals remaining asymptomatic.
- Pathogenic variants are gain of function; loss of function variants are not a known disease mechanism.
Test Interpretation
Clinical Sensitivity
Analytic Sensitivity
For massively parallel sequencing:
Variant Class | Analytic Sensitivity (PPA) Estimatea (%) | Analytic Sensitivity (PPA) 95% Credibility Regiona (%) |
---|---|---|
SNVs |
>99 |
96.9-99.4 |
Deletions 1-10 bp |
93.8 |
84.3-98.2 |
Deletions 11-44 bp |
>99 |
87.8-100 |
Insertions 1-10 bp |
94.8 |
86.8-98.5 |
Insertions 11-23 bp |
>99 |
62.1-100 |
aThe gene included on this test is a subset of a larger methods-based validation from which the PPA values are derived. bp, base pairs; PPA, positive percent agreement; SNVs, single nucleotide variants |
Results
Variant(s) Detected | Clinical Significance |
---|---|
Two pathogenic variants detected in the MEFV gene |
Consistent with a diagnosis of FMF |
No pathogenic variants detected in the MEFV gene |
Decreases the likelihood of, but does not exclude, a diagnosis of FMF |
One pathogenic variant detected in the MEFV gene |
At least a carrier of FMF Some individuals with only one detected variant may have clinical features. Medical management should rely on clinical findings. |
One or more variant(s) of unknown clinical significance detected in the MEFV gene |
Inconclusive; based on currently available information, it is unknown whether the variant is disease-associated or benign. Medical management should rely on clinical findings. Surveillance of the literature for new information concerning the uncertain variant is recommended. |
Limitations
- A negative result does not exclude a diagnosis of FMF.
- Diagnostic errors can occur due to rare sequence variations.
- Interpretation of this test result may be impacted if this patient has had an allogeneic stem cell transplantation.
- The following will not be evaluated:
- Variants outside the coding regions and intron-exon boundaries of targeted gene
- Variants in the mitochondrial genome
- Regulatory region and deep intronic variants
- Large deletions/duplications (large deletions/duplications have not been reported as causative variants for FMF)
- Noncoding transcripts
- The following may not be detected:
- Deletions/duplications/insertions of any size by massively parallel sequencing
- Some variants due to technical limitations in the presence of pseudogenes, repetitive, or homologous regions
- Low-level somatic variants
References
-
11464238
Touitou I. The spectrum of familial Mediterranean fever (FMF) mutations. Eur J Hum Genet. 2001;9(7):473-483.
-
19790133
Ben-Chetrit E, Touitou I. Familial Mediterranean fever in the world. Arthritis Rheum. 2009;61(10):1447-1453.
-
GeneReviews - Familial Mediterranean Fever
Shohat M. Familial Mediterranean fever. In: Adam MP, Ardinger HH, Pagon RA, et al, eds. GeneReviews. University of Washington, Seattle. Last update Dec 2016; accessed Oct 2021.
19479870
Booty MG, Chae JJ, Masters SL, et al. Familial Mediterranean fever with a single MEFV mutation: where is the second hit? Arthritis Rheum. 2009;60(6):1851-1861.