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Immunodeficiency 67(IPD; IKAK4D; IMD67)

MedGen UID:
375137
Concept ID:
C1843256
Disease or Syndrome
Synonyms: Immunodeficiency due to interleukin-1 receptor-associated kinase-4 deficiency; Interleukin receptor-associated kinase deficiency; IRAK4 deficiency
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
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 individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
 
Gene (location): IRAK4 (12q12)
 
Monarch Initiative: MONDO:0011888
OMIM®: 607676
Orphanet: ORPHA70592

Definition

Immunodeficiency-67 (IMD67) is an autosomal recessive primary immunodeficiency characterized by recurrent severe systemic and invasive bacterial infections beginning in infancy or early childhood. The most common organisms implicated are Streptococcus pneumoniae and Staphylococcus aureus; Pseudomonas and atypical Mycobacteria may also be observed. IMD67 is life-threatening in infancy and early childhood. The first invasive infection typically occurs before 2 years of age, with meningitis representing up to 41% of the bacterial infections. The mortality rate in early childhood is high, with most deaths occurring before 8 years of age. Affected individuals have an impaired inflammatory response to infection, including lack of fever and neutropenia, although erythrocyte sedimentation rate (ESR) and C-reactive protein may be elevated. General immunologic workup tends to be normal, with normal levels of B cells, T cells, and NK cells. However, more detailed studies indicate impaired cytokine response to lipopolysaccharide (LPS) and IL1B (147720) stimulation; response to TNFA (191160) is usually normal. Patients have good antibody responses to most vaccinations, with the notable exception of pneumococcal vaccination. Viral, fungal, and parasitic infections are not generally observed. Early detection is critical in early childhood because prophylactic treatment with IVIg or certain antibiotics is effective; the disorder tends to improve naturally around adolescence. At the molecular level, the disorder results from impaired function of selective Toll receptor (see TLR4, 603030)/IL1R (see IL1R1, 147810) signaling pathways that ultimately activate NFKB (164011) to produce cytokines (summary by Ku et al., 2007; Picard et al., 2010; Grazioli et al., 2016). See also IMD68 (612260), caused by mutation in the MYD88 gene (602170), which shows a similar phenotype to IMD67. As the MYD88 and IRAK4 genes interact in the same intracellular signaling pathway, the clinical and cellular features are almost indistinguishable (summary by Picard et al., 2010). [from OMIM]

Additional description

From MedlinePlus Genetics
IRAK-4 deficiency is an inherited disorder of the immune system (primary immunodeficiency). This immunodeficiency leads to recurrent infections by a subset of bacteria known as pyogenic bacteria but not by other infectious agents. (Infection with pyogenic bacteria causes the production of pus.) The most common infections in IRAK-4 deficiency are caused by the Streptococcus pneumoniae, Staphylococcus aureus, and Pseudomonas aeruginosa bacteria. Most people with this condition have their first bacterial infection before age 2, and the infections can be life-threatening in infancy and childhood. Infections become less frequent with age.

Most people with IRAK-4 deficiency have invasive bacterial infections, which can involve the blood (septicemia), the membrane covering the brain and spinal cord (meningitis), or the joints (leading to inflammation and arthritis). Invasive infections can also cause areas of tissue breakdown and pus production (abscesses) on internal organs. In addition, affected individuals can have localized infections of the upper respiratory tract, skin, or eyes. Although fever is a common reaction to bacterial infections, many people with IRAK-4 deficiency do not at first develop a high fever in response to these infections, even if the infection is severe.  https://medlineplus.gov/genetics/condition/irak-4-deficiency

Clinical features

From HPO
Liver abscess
MedGen UID:
6124
Concept ID:
C0023885
Disease or Syndrome
A circumscribed area of pus or necrotic debris in the liver.
Infective arthritis
MedGen UID:
13918
Concept ID:
C0003869
Disease or Syndrome
The inflammation of one or more joints caused by any infectious pathogen within the joint space. Symptoms include pain, stiffness, and decreased range of motion in the affected joint.
Immunodeficiency
MedGen UID:
7034
Concept ID:
C0021051
Disease or Syndrome
Failure of the immune system to protect the body adequately from infection, due to the absence or insufficiency of some component process or substance.
Meningitis
MedGen UID:
6298
Concept ID:
C0025289
Disease or Syndrome
Inflammation of the meninges.
Increased circulating IgE concentration
MedGen UID:
116018
Concept ID:
C0236175
Finding
An abnormally increased overall level of immunoglobulin E in blood.
Abnormal natural killer cell count
MedGen UID:
866689
Concept ID:
C4021036
Finding
Any deviation from the normal overall count of natural killer (NK) cells in the circulation or a deviation from the normal distribution of NK cell subtypes.
Abnormal T cell count
MedGen UID:
866762
Concept ID:
C4021113
Finding
A deviation from the normal count of T cells.
Abnormal B cell count
MedGen UID:
866853
Concept ID:
C4021208
Finding
A deviation from the normal count of B cells, i.e., the cells that are formed in the bone marrow, migrate to the peripheral lymphatic system, and mature into plasma cells or memory cells.
Recurrent staphylococcal infections
MedGen UID:
870417
Concept ID:
C4024862
Finding
Increased susceptibility to staphylococcal infections, as manifested by recurrent episodes of staphylococcal infections.
Recurrent streptococcal infections
MedGen UID:
1694122
Concept ID:
C5139076
Finding
Increased susceptibility to streptococcal infections, as manifested by recurrent episodes of streptococcal infections.
Transient neutropenia
MedGen UID:
1692023
Concept ID:
C5139431
Finding
A transient reduction in the number of neutrophils in the peripheral blood. Transient neutropenia is most commonly associated with viral infections, but other causes include drugs and autoimmunity.
Complete or near-complete absence of specific antibody response to unconjugated pneumococcus vaccine
MedGen UID:
1704477
Concept ID:
C5139463
Finding
The inability to synthesize postvaccination antibodies against a pneumococcus antigen, as measured by antibody titer determination following vaccination.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVImmunodeficiency 67
Follow this link to review classifications for Immunodeficiency 67 in Orphanet.

Professional guidelines

PubMed

Schwab C, Gabrysch A, Olbrich P, Patiño V, Warnatz K, Wolff D, Hoshino A, Kobayashi M, Imai K, Takagi M, Dybedal I, Haddock JA, Sansom DM, Lucena JM, Seidl M, Schmitt-Graeff A, Reiser V, Emmerich F, Frede N, Bulashevska A, Salzer U, Schubert D, Hayakawa S, Okada S, Kanariou M, Kucuk ZY, Chapdelaine H, Petruzelkova L, Sumnik Z, Sediva A, Slatter M, Arkwright PD, Cant A, Lorenz HM, Giese T, Lougaris V, Plebani A, Price C, Sullivan KE, Moutschen M, Litzman J, Freiberger T, van de Veerdonk FL, Recher M, Albert MH, Hauck F, Seneviratne S, Pachlopnik Schmid J, Kolios A, Unglik G, Klemann C, Speckmann C, Ehl S, Leichtner A, Blumberg R, Franke A, Snapper S, Zeissig S, Cunningham-Rundles C, Giulino-Roth L, Elemento O, Dückers G, Niehues T, Fronkova E, Kanderová V, Platt CD, Chou J, Chatila TA, Geha R, McDermott E, Bunn S, Kurzai M, Schulz A, Alsina L, Casals F, Deyà-Martinez A, Hambleton S, Kanegane H, Taskén K, Neth O, Grimbacher B
J Allergy Clin Immunol 2018 Dec;142(6):1932-1946. Epub 2018 May 4 doi: 10.1016/j.jaci.2018.02.055. PMID: 29729943Free PMC Article
Hurst JR, Verma N, Lowe D, Baxendale HE, Jolles S, Kelleher P, Longhurst HJ, Patel SY, Renzoni EA, Sander CR, Avery GR, Babar JL, Buckland MS, Burns S, Egner W, Gompels MM, Gordins P, Haddock JA, Hart SP, Hayman GR, Herriot R, Hoyles RK, Huissoon AP, Jacob J, Nicholson AG, Rassl DM, Sargur RB, Savic S, Seneviratne SL, Sheaff M, Vaitla PM, Walters GI, Whitehouse JL, Wright PA, Condliffe AM
J Allergy Clin Immunol Pract 2017 Jul-Aug;5(4):938-945. Epub 2017 Mar 25 doi: 10.1016/j.jaip.2017.01.021. PMID: 28351785
Galiè N, Humbert M, Vachiery JL, Gibbs S, Lang I, Torbicki A, Simonneau G, Peacock A, Vonk Noordegraaf A, Beghetti M, Ghofrani A, Gomez Sanchez MA, Hansmann G, Klepetko W, Lancellotti P, Matucci M, McDonagh T, Pierard LA, Trindade PT, Zompatori M, Hoeper M; ESC Scientific Document Group
Eur Heart J 2016 Jan 1;37(1):67-119. Epub 2015 Aug 29 doi: 10.1093/eurheartj/ehv317. PMID: 26320113

Recent clinical studies

Etiology

Turkova A, Chappell E, Chalermpantmetagul S, Negra MD, Volokha A, Primak N, Solokha S, Rozenberg V, Kiselyova G, Yastrebova E, Miloenko M, Bashakatova N, Kanjanavanit S, Calvert J, Rojo P, Ansone S, Jourdain G, Malyuta R, Goodall R, Judd A, Thorne C
Int J Tuberc Lung Dis 2016 Nov;20(11):1448-1456. doi: 10.5588/ijtld.16.0067. PMID: 27776584

Diagnosis

Turkova A, Chappell E, Chalermpantmetagul S, Negra MD, Volokha A, Primak N, Solokha S, Rozenberg V, Kiselyova G, Yastrebova E, Miloenko M, Bashakatova N, Kanjanavanit S, Calvert J, Rojo P, Ansone S, Jourdain G, Malyuta R, Goodall R, Judd A, Thorne C
Int J Tuberc Lung Dis 2016 Nov;20(11):1448-1456. doi: 10.5588/ijtld.16.0067. PMID: 27776584

Therapy

Turkova A, Chappell E, Chalermpantmetagul S, Negra MD, Volokha A, Primak N, Solokha S, Rozenberg V, Kiselyova G, Yastrebova E, Miloenko M, Bashakatova N, Kanjanavanit S, Calvert J, Rojo P, Ansone S, Jourdain G, Malyuta R, Goodall R, Judd A, Thorne C
Int J Tuberc Lung Dis 2016 Nov;20(11):1448-1456. doi: 10.5588/ijtld.16.0067. PMID: 27776584

Prognosis

Turkova A, Chappell E, Chalermpantmetagul S, Negra MD, Volokha A, Primak N, Solokha S, Rozenberg V, Kiselyova G, Yastrebova E, Miloenko M, Bashakatova N, Kanjanavanit S, Calvert J, Rojo P, Ansone S, Jourdain G, Malyuta R, Goodall R, Judd A, Thorne C
Int J Tuberc Lung Dis 2016 Nov;20(11):1448-1456. doi: 10.5588/ijtld.16.0067. PMID: 27776584

Clinical prediction guides

Turkova A, Chappell E, Chalermpantmetagul S, Negra MD, Volokha A, Primak N, Solokha S, Rozenberg V, Kiselyova G, Yastrebova E, Miloenko M, Bashakatova N, Kanjanavanit S, Calvert J, Rojo P, Ansone S, Jourdain G, Malyuta R, Goodall R, Judd A, Thorne C
Int J Tuberc Lung Dis 2016 Nov;20(11):1448-1456. doi: 10.5588/ijtld.16.0067. PMID: 27776584

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