Entry - #209950 - IMMUNODEFICIENCY 27A; IMD27A - OMIM - (MIRROR)
# 209950

IMMUNODEFICIENCY 27A; IMD27A


Alternative titles; symbols

IMMUNODEFICIENCY 27A, MYCOBACTERIOSIS, AUTOSOMAL RECESSIVE
IFNGR1 DEFICIENCY, AUTOSOMAL RECESSIVE


Phenotype-Gene Relationships

Location Phenotype Phenotype
MIM number
Inheritance Phenotype
mapping key
Gene/Locus Gene/Locus
MIM number
6q23.3 Immunodeficiency 27A, mycobacteriosis, AR 209950 AR 3 IFNGR1 107470
Clinical Synopsis
 
Phenotypic Series
 

INHERITANCE
- Autosomal recessive
IMMUNOLOGY
- Increased susceptibility to Mycobacterial infections
- Increased susceptibility to Salmonella infections
- Poor or absent response to gamma-interferon
MISCELLANEOUS
- Onset in early childhood
- May be fatal
- Patients may develop disseminated disease after BCG vaccination
- Patients may respond well to treatment with gamma-interferon
MOLECULAR BASIS
- Caused by mutation in the interferon-gamma receptor 1 gene (IFNGR1, 107470.0001)
Immunodeficiency (select examples) - PS300755 - 137 Entries
Location Phenotype Inheritance Phenotype
mapping key
Phenotype
MIM number
Gene/Locus Gene/Locus
MIM number
1p36.33 Immunodeficiency 38 AR 3 616126 ISG15 147571
1p36.33 ?Immunodeficiency 16 AR 3 615593 TNFRSF4 600315
1p36.23 Immunodeficiency 109 with lymphoproliferation AR 3 620282 TNFRSF9 602250
1p36.22 Immunodeficiency 14A, autosomal dominant AD 3 615513 PIK3CD 602839
1p36.22 Immunodeficiency 14B, autosomal recessive AR 3 619281 PIK3CD 602839
1p35.2 Immunodeficiency 22 AR 3 615758 LCK 153390
1p34.2 Immunodeficiency 24 AR 3 615897 CTPS1 123860
1p22.3 ?Immunodeficiency 37 AR 3 616098 BCL10 603517
1q21.3 Immunodeficiency 42 AR 3 616622 RORC 602943
1q23.3 Immunodeficiency 20 AR 3 615707 FCGR3A 146740
1q24.2 ?Immunodeficiency 25 AR 3 610163 CD247 186780
1q25.3 Immunodeficiency 133 with autoimmunity and autoinflammation AR 3 620565 ARPC5 604227
1q25.3 Immunodeficiency 70 AD 3 618969 IVNS1ABP 609209
1q31.3-q32.1 Immunodeficiency 105, severe combined AR 3 619924 PTPRC 151460
2p16.1 Immunodeficiency 92 AR 3 619652 REL 164910
2p11.2 Immunodeficiency 116 AR 3 608957 CD8A 186910
2q11.2 Immunodeficiency 48 AR 3 269840 ZAP70 176947
2q24.2 Immunodeficiency 95 AR 3 619773 IFIH1 606951
2q32.2 Immunodeficiency 31A, mycobacteriosis, autosomal dominant AD 3 614892 STAT1 600555
2q32.2 Immunodeficiency 31B, mycobacterial and viral infections, autosomal recessive AR 3 613796 STAT1 600555
2q32.2 Immunodeficiency 31C, chronic mucocutaneous candidiasis, autosomal dominant AD 3 614162 STAT1 600555
2q33.2 ?Immunodeficiency 123 with HPV-related verrucosis AR 3 620901 CD28 186760
2q35 Immunodeficiency 124, severe combined AR 3 611291 NHEJ1 611290
3p22.2 Immunodeficiency 68 AR 3 612260 MYD88 602170
3q21.3 Immunodeficiency 21 AD 3 614172 GATA2 137295
3q29 Immunodeficiency 46 AR 3 616740 TFRC 190010
4q24 Immunodeficiency 75 AR 3 619126 TET2 612839
4q35.1 {Immunodeficiency 83, susceptibility to viral infections} AD, AR 3 613002 TLR3 603029
5p15.2 {Immunodeficiency 107, susceptibility to invasive staphylococcus aureus infection} AD 3 619986 OTULIN 615712
5p13.2 Immunodeficiency 104, severe combined AR 3 608971 IL7R 146661
5q11.2 ?Immunodeficiency 94 with autoinflammation and dysmorphic facies AD 3 619750 IL6ST 600694
5q13.1 Immunodeficiency 36 AD 3 616005 PIK3R1 171833
5q31.1 Immunodeficiency 93 and hypertrophic cardiomyopathy AR 3 619705 FNIP1 610594
5q31.1 Immunodeficiency 117, mycobacteriosis, autosomal recessive AR 3 620668 IRF1 147575
5q33.3 Immunodeficiency 29, mycobacteriosis AR 3 614890 IL12B 161561
5q35.1 Immunodeficiency 40 AR 3 616433 DOCK2 603122
5q35.1 Immunodeficiency 81 AR 3 619374 LCP2 601603
6p25.2 Immunodeficiency 57 with autoinflammation AR 3 618108 RIPK1 603453
6p21.33 ?Immunodeficiency 127 AR 3 620977 TNF 191160
6p21.31 Immunodeficiency 87 and autoimmunity AR 3 619573 DEF6 610094
6p21.1 Immunodeficiency 126 AR 3 620931 PTCRA 606817
6q14.1 Immunodeficiency 23 AR 3 615816 PGM3 172100
6q15 Immunodeficiency 60 and autoimmunity AD 3 618394 BACH2 605394
6q23.3 Immunodeficiency 27B, mycobacteriosis, AD AD 3 615978 IFNGR1 107470
6q23.3 Immunodeficiency 27A, mycobacteriosis, AR AR 3 209950 IFNGR1 107470
7p22.2 Immunodeficiency 11A AR 3 615206 CARD11 607210
7p22.2 Immunodeficiency 11B with atopic dermatitis AD 3 617638 CARD11 607210
7q22.1 Immunodeficiency 71 with inflammatory disease and congenital thrombocytopenia AR 3 617718 ARPC1B 604223
7q22.3 Immunodeficiency 97 with autoinflammation AR 3 619802 PIK3CG 601232
8p11.21 Immunodeficiency 15A AD 3 618204 IKBKB 603258
8p11.21 Immunodeficiency 15B AR 3 615592 IKBKB 603258
8q11.21 Immunodeficiency 26, with or without neurologic abnormalities AR 3 615966 PRKDC 600899
8q11.21 Immunodeficiency 54 AR 3 609981 MCM4 602638
9q22.2 Immunodeficiency 82 with systemic inflammation AD 3 619381 SYK 600085
9q34.3 Immunodeficiency 103, susceptibility to fungal infection AR 3 212050 CARD9 607212
10p15.1 Immunodeficiency 41 with lymphoproliferation and autoimmunity AR 3 606367 IL2RA 147730
10p13 Immunodeficiency 80 with or without cardiomyopathy AR 3 619313 MCM10 609357
11p15.5 ?Immunodeficiency 39 AR 3 616345 IRF7 605047
11p15.4 Immunodeficiency 10 AR 3 612783 STIM1 605921
11q12.1 Immunodeficiency 77 AD 3 619223 MPEG1 610390
11q13.3 Immunodeficiency 90 with encephalopathy, functional hyposplenia, and hepatic dysfunction AR 3 613759 FADD 602457
11q13.4 Immunodeficiency 122 AR 3 620869 POLD3 611415
11q23.3 Immunodeficiency 18, SCID variant AR 3 615615 CD3E 186830
11q23.3 Immunodeficiency 18 AR 3 615615 CD3E 186830
11q23.3 Immunodeficiency 19, severe combined AR 3 615617 CD3D 186790
11q23.3 Immunodeficiency 17, CD3 gamma deficient AR 3 615607 CD3G 186740
11q23.3 ?Immunodeficiency 59 and hypoglycemia AR 3 233600 HYOU1 601746
12p13.31 Immunodeficiency 79 AR 3 619238 CD4 186940
12q12 Immunodeficiency 67 AR 3 607676 IRAK4 606883
12q13.13-q13.2 Immunodeficiency 72 with autoinflammation AR 3 618982 NCKAP1L 141180
12q13.3 Immunodeficiency 44 AR 3 616636 STAT2 600556
12q15 ?Immunodeficiency 69, mycobacteriosis AR 3 618963 IFNG 147570
12q24.13 Immunodeficiency 100 with pulmonary alveolar proteinosis and hypogammaglobulinemia AD 3 618042 OAS1 164350
12q24.31 Immunodeficiency 9 AR 3 612782 ORAI1 610277
13q33.1 Immunodeficiency 78 with autoimmunity and developmental delay AR 3 619220 TPP2 190470
14q11.2 Immunodeficiency 7, TCR-alpha/beta deficient AR 3 615387 TRAC 186880
14q11.2 ?Immunodeficiency 108 with autoinflammation AR 3 260570 CEBPE 600749
14q12 Immunodeficiency 115 with autoinflammation AR 3 620632 RNF31 612487
14q12 Immunodeficiency 65, susceptibility to viral infections AR 3 618648 IRF9 147574
14q32.2 Immunodeficiency 49, severe combined AD 3 617237 BCL11B 606558
15q14 Immunodeficiency 64 AR 3 618534 RASGRP1 603962
15q21.1 Immunodeficiency 43 AR 3 241600 B2M 109700
15q21.2 Immunodeficiency 86, mycobacteriosis AR 3 619549 SPPL2A 608238
16p12.1 Immunodeficiency 56 AR 3 615207 IL21R 605383
16p11.2 Immunodeficiency 52 AR 3 617514 LAT 602354
16p11.2 Immunodeficiency 8 AR 3 615401 CORO1A 605000
16q22.1 Immunodeficiency 58 AR 3 618131 CARMIL2 610859
16q22.1 Immunodeficiency 121 with autoinflammation AD 3 620807 PSMB10 176847
16q24.1 Immunodeficiency 32A, mycobacteriosis, autosomal dominant AD 3 614893 IRF8 601565
16q24.1 Immunodeficiency 32B, monocyte and dendritic cell deficiency, autosomal recessive AR 3 226990 IRF8 601565
17q11.2 ?Immunodeficiency 13 AD 3 615518 UNC119 604011
17q12-q21.1 ?Immunodeficiency 84 AD 3 619437 IKZF3 606221
17q21.31 Immunodeficiency 112 AR 3 620449 MAP3K14 604655
17q21.32 ?Immunodeficiency 88 AR 3 619630 TBX21 604895
18q21.32 Immunodeficiency 12 AR 3 615468 MALT1 604860
19p13.3 Hatipoglu immunodeficiency syndrome AR 3 620331 DPP9 608258
19p13.2 Immunodeficiency 35 AR 3 611521 TYK2 176941
19p13.11 Immunodeficiency 76 AR 3 619164 FCHO1 613437
19p13.11 Immunodeficiency 30 AR 3 614891 IL12RB1 601604
19q13.2 ?Immunodeficiency 62 AR 3 618459 ARHGEF1 601855
19q13.32 ?Immunodeficiency 53 AR 3 617585 RELB 604758
19q13.33 Immunodeficiency 96 AR 3 619774 LIG1 126391
19q13.33 ?Immunodeficiency 125 AR 3 620926 FLT3LG 600007
19q13.33 Immunodeficiency 120 AR 3 620836 POLD1 174761
20p11.23 ?Immunodeficiency 101 (varicella zoster virus-specific) AD 3 619872 POLR3F 617455
20p11.21 Immunodeficiency 55 AR 3 617827 GINS1 610608
20q11.23 ?Immunodeficiency 99 with hypogammaglobulinemia and autoimmune cytopenias AR 3 619846 CTNNBL1 611537
20q13.12 T-cell immunodeficiency, recurrent infections, autoimmunity, and cardiac malformations AR 3 614868 STK4 604965
20q13.13 Immunodeficiency 91 and hyperinflammation AR 3 619644 ZNFX1 618931
21q22.11 Immunodeficiency 45 AR 3 616669 IFNAR2 602376
21q22.11 Immunodeficiency 106, susceptibility to viral infections AR 3 619935 IFNAR1 107450
21q22.11 Immunodeficiency 28, mycobacteriosis AR 3 614889 IFNGR2 147569
21q22.3 ?Immunodeficiency 119 AR 3 620825 ICOSLG 605717
21q22.3 Immunodeficiency 114, folate-responsive AR 3 620603 SLC19A1 600424
22q11.1 Immunodeficiency 51 AR 3 613953 IL17RA 605461
22q12.3 ?Immunodeficiency 85 and autoimmunity AD 3 619510 TOM1 604700
22q12.3 Immunodeficiency 63 with lymphoproliferation and autoimmunity AR 3 618495 IL2RB 146710
22q13.1 ?Immunodeficiency 73C with defective neutrophil chemotaxis and hypogammaglobulinemia AR 3 618987 RAC2 602049
22q13.1 Immunodeficiency 73B with defective neutrophil chemotaxis and lymphopenia AD 3 618986 RAC2 602049
22q13.1 Immunodeficiency 73A with defective neutrophil chemotaxix and leukocytosis AD 3 608203 RAC2 602049
22q13.1 ?Immunodeficiency 89 and autoimmunity AR 3 619632 CARD10 607209
22q13.1-q13.2 ?Immunodeficiency 66 AR 3 618847 MKL1 606078
Xp22.2 Immunodeficiency 74, COVID19-related, X-linked XLR 3 301051 TLR7 300365
Xp22.2 Immunodeficiency 98 with autoinflammation, X-linked SMo, XL 3 301078 TLR8 300366
Xp22.12 ?Immunodeficiency 61 XLR 3 300310 SH3KBP1 300374
Xp21.1-p11.4 Immunodeficiency 34, mycobacteriosis, X-linked XLR 3 300645 CYBB 300481
Xp11.23 Wiskott-Aldrich syndrome XLR 3 301000 WAS 300392
Xq12 Immunodeficiency 50 XLR 3 300988 MSN 309845
Xq13.1 Severe combined immunodeficiency, X-linked XLR 3 300400 IL2RG 308380
Xq13.1 Combined immunodeficiency, X-linked, moderate XLR 3 312863 IL2RG 308380
Xq22.1 Agammaglobulinemia, X-linked 1 XLR 3 300755 BTK 300300
Xq24 Immunodeficiency 118, mycobacteriosis XLR 3 301115 MCTS1 300587
Xq25 Lymphoproliferative syndrome, X-linked, 1 XLR 3 308240 SH2D1A 300490
Xq26.1 Immunodeficiency 102 XLR 3 301082 SASH3 300441
Xq26.3 Immunodeficiency, X-linked, with hyper-IgM XLR 3 308230 TNFSF5 300386
Xq28 Immunodeficiency 47 XLR 3 300972 ATP6AP1 300197
Xq28 Immunodeficiency 33 XLR 3 300636 IKBKG 300248

TEXT

A number sign (#) is used with this entry because of evidence that immunodeficiency-27A (IMD27A), an autosomal recessive disorder, is caused by homozygous or compound heterozygous mutation in the IFNGR1 gene (107470) on chromosome 6q23.

Immunodeficiency-27B (IMD27B; 615978), an autosomal dominant disorder, is allelic.


Description

Immunodeficiency-27A (IMD27A) results from autosomal recessive (AR) IFNGR1 deficiency. Patients with complete IFNGR1 deficiency have a severe clinical phenotype characterized by early and often fatal mycobacterial infections. The disorder can thus be categorized as a form of mendelian susceptibility to mycobacterial disease (MSMD). Bacillus Calmette-Guerin (BCG) and environmental mycobacteria are the most frequent pathogens, and infection typically begins before the age of 3 years. Plasma from patients with complete AR IFNGR1 deficiency usually contains large amounts of IFNG (147570), and their cells do not respond to IFNG in vitro. In contrast, cells from patients with partial AR IFNGR1 deficiency, which is caused by a specific mutation in IFNGR1, retain residual responses to high IFNG concentrations. Patients with partial AR IFNGR1 deficiency are susceptible to BCG and environmental mycobacteria, but they have a milder clinical disease and better prognosis than patients with complete AR IFNGR1 deficiency. The clinical features of children with complete AR IFNGR1 deficiency are usually more severe than those in individuals with AD IFNGR1 deficiency (IMD27B), and mycobacterial infection often occurs earlier (mean age of 1.3 years vs 13.4 years), with patients having shorter mean disease-free survival. Salmonellosis is present in about 5% of patients with AR or AD IFNGR1 deficiency, and other infections have been reported in single patients (review by Al-Muhsen and Casanova, 2008).


Clinical Features

Families with multiple cases of disseminated atypical mycobacteriosis, a rare disorder, were reported by Engbaek (1964) and Uchiyama et al. (1981). Uchiyama et al. (1981) reported fatal disseminated atypical mycobacteriosis in 2 young Mexican-American girls. The atypical mycobacterium was of a different serotype in the 2 sisters. One of the sisters died in 1964 and the other in 1977. Studies by the authors suggested a congenital defect in monocyte microbicidal activity. Fischer et al. (1980) observed defective monocyte function in a 12-month-old child with fatal disseminated BCG infection.

Levin et al. (1995) described 6 children with disseminated atypical mycobacterial infection and no recognized form of immunodeficiency. Four, including 2 brothers, came from a village in Malta, and 2 were brothers of Greek Cypriot origin. They presented with fever, weight loss, lymphadenopathy, and hepatosplenomegaly. They had anemia and an acute phase response. A range of different mycobacteria (Mycobacterium fortuitum, M. chelonei, and 4 strains of M. avium intracellulare) were isolated. Treatment with multiple antibiotics failed to eradicate the infection, although treatment with gamma-interferon was associated with improvement. Three of the children had died and the 3 survivors had chronic infection. TNF-alpha (191160) production in response to endotoxin and gamma-interferon was found to be defective in the patients and their parents. T-cell proliferative responses to mycobacterial and recall antigens were reduced in parents of affected children, and gamma-interferon production was diminished in the patients and their parents. Levin et al. (1995) suggested that these patients are phenotypically similar to Lsh/Ity/Bcg susceptible mice (see ANIMAL MODEL).

Toyoda et al. (2004) examined the immunologic abnormality of a patient with recurrent Mycobacterium avium infection. The patient had reduced expression of IL12RB1 and IL12RB2 and a decreased ability to produce IFNG (147570) and to proliferate in response to IL12. However, the patient exhibited no deficiency in IL12-induced tyrosine and serine phosphorylation of STAT4 (600558) in mitogen-activated T cells. EMSA, confocal laser microscopy, and Western blot analysis demonstrated that nuclear translocation of STAT4 in response to IL12 was reduced in the patient compared with healthy control subjects. Pharmacologic treatment indicated that the defect was not due to upregulated STAT4 export from the nucleus. No mutations in IL12RB1, IL12RB2, STAT4, or the IFNG STAT4-binding sequence were identified, and the exact mechanism for the defect could not be determined.


Diagnosis

Fieschi et al. (2001) found that children with complete IFNGR deficiency, unlike patients with other genetic defects predisposing them to mycobacterial diseases, have very high levels of IFNG in their plasma. Fieschi et al. (2001) proposed this measurement as a simple, inexpensive, and accurate diagnostic test for complete IFNGR deficiency. They noted that early identification of such children, who do not respond to exogenous IFNG or antibiotics, may improve management by leading to the consideration of bone marrow transplantation.


Molecular Genetics

Newport et al. (1996) and Jouanguy et al. (1996) demonstrated that mutations in the interferon-gamma-receptor-1 gene (IFNGR1; 107470) conferred autosomal recessive susceptibility to mycobacterium infection.

Al-Muhsen and Casanova (2008) and Cottle (2011) reviewed genetic heterogeneity of susceptibility to mycobacterial disease.


Genotype/Phenotype Correlations

Dorman et al. (2004) compared the clinical features of recessive and dominant IFNGR1 deficiencies using a worldwide cohort of patients. They assessed the patients by medical histories and genetic and immunologic studies. Recessive deficiency, which Dorman et al. (2004) identified in 22 patients, results in complete loss of cellular response to IFNG and absence of surface IFNGR1 expression. Dominant deficiency, which they identified in 38 patients, is typically due to cytoplasmic domain truncations resulting in accumulation of nonfunctional IFNGR1 proteins that may impede the function of molecules encoded by the wildtype allele, thereby leading to diminished but not absent responsiveness to IFNG. Although the clinical phenotypes are related, Dorman et al. (2004) found that patients with the recessive form had an earlier age of onset (3 vs 13 years), more mycobacterial disease episodes (19 vs 8 per 100 person years of observation), more severe mycobacterial disease (involvement of 4 vs 2 organs), shorter mean disease-free intervals (1.6 vs 7.2 years), and lower Kaplan-Meier survival probability. Recessive patients also had more frequent disease from rapidly growing mycobacteria. Patients with a dominant mutation, however, were more likely to have M. avium complex osteomyelitis, and only dominant patients had osteomyelitis without other organ involvement. Dorman et al. (2004) concluded that there is a strong correlation between the IFNGR1 genotype, clinical disease features, and the cellular responsiveness to IFNG. They suggested that subtle defects in IFNG production, signaling, or related pathways may predispose to diseases caused by virulent mycobacteria, including M. tuberculosis.


Animal Model

There is a mouse gene, variously symbolized Lsh, Ity, and Bcg, on murine chromosome 1 which encodes resistance to bacterial and parasitic infections and affects the function of macrophages (Skamene et al., 1982; Brown et al., 1982; Goto et al., 1984; Plant et al., 1982; Swanson and O'Brien, 1983; Nickol and Bonventre, 1985). Bcg is expressed in 2 allelic forms, the dominant resistance allele and the recessive susceptibility allele. The Bcg region on proximal mouse chromosome 1 shows homology of synteny with the telomeric portion of human 2q; a 35-cM fragment around the murine Bcg locus (from Col3a1 (120180) to Col6a3 (120250)), has been conserved between the 2 species, the human region being 2q32-q37.


History

Schurr et al. (1991) studied linkage of genetic markers on distal chromosome 2q with susceptibility to tuberculosis and found a lod score of 2.4. Shaw et al. (1993), however, could not confirm this finding. They performed linkage analysis using a panel of markers from the 2q33-q37 region in 35 multicase families with infection by Mycobacterium leprae, M. tuberculosis, and Leishmania sp. Data from all 3 types of families were pooled to produce a detailed RFLP map of the region. The order of genes in the human was consistent with that determined for the same loci in the mouse. Nonetheless, Shaw et al. (1993) could not demonstrate linkage of infection susceptibility to this region.

Newport et al. (1995) excluded NRAMP (600266) as the site of the mutation causing this disorder, which they referred to as familial disseminated atypical mycobacterial infection, in a Maltese kindred. They typed 8 markers in the region of 2q34-q37 where NRAMP maps.


REFERENCES

  1. Al-Muhsen, S., Casanova, J.-L. The genetic heterogeneity of mendelian susceptibility to mycobacterial diseases. J. Allergy Clin. Immun. 122: 1043-1051, 2008. [PubMed: 19084105, related citations] [Full Text]

  2. Brown, I. N., Glynn, A. A., Plant, J. Inbred mouse strain resistance to Mycobacterium lepraemurium follows the Ity/Lsh pattern. Immunology 47: 149-156, 1982. [PubMed: 6749659, related citations]

  3. Cottle, L. E. Mendelian susceptibility to mycobacterial disease. Clin. Genet. 79: 17-22, 2011. [PubMed: 20718793, related citations] [Full Text]

  4. Dorman, S. E., Picard, C., Lammas, D., Heyne, K., van Dissel, J. T., Baretto, R., Rosenzweig, S. D., Newport, M., Levin, M., Roesler, J., Kumararatne, D., Casanova, J.-L., Holland, S. M. Clinical features of dominant and recessive interferon-gamma receptor 1 deficiencies. Lancet 364: 2113-2121, 2004. [PubMed: 15589309, related citations] [Full Text]

  5. Engbaek, H. C. Three cases in the same family of fatal infection M. avium. Acta Tuberc. Pneumol. Scand. 45: 105-117, 1964. [PubMed: 14215783, related citations]

  6. Fieschi, C., Dupuis, S., Picard, C., Smith, C. I. E., Holland, S. M., Casanova, J.-L. High levels of interferon gamma in the plasma of children with complete interferon gamma receptor deficiency. Pediatrics 107: E48, 2001. Note: Electronic Article. [PubMed: 11335769, related citations] [Full Text]

  7. Fischer, A., Virelizier, J. L., Griscelli, C., Durandy, A., Nezelof, C., Trong, P. H. Defective monocyte functions in a child with fatal disseminated BCG infection. Clin. Immun. Immunopath. 17: 296-306, 1980. [PubMed: 6996876, related citations] [Full Text]

  8. Goto, Y., Nakamura, R. M., Takahashi, H., Tokunaga, T. Genetic control of resistance to Mycobacterium intracellulare infection in mice. Infect. Immun. 46: 135-140, 1984. [PubMed: 6480104, related citations] [Full Text]

  9. Jouanguy, E., Altare, F., Lamhamedi, S., Revy, P., Emile, J.-F., Newport, M., Levin, M., Blanche, S., Seboun, E., Fischer, A., Casanova, J.-L. Interferon-gamma-receptor deficiency in an infant with fatal bacille Calmette-Guerin infection. New Eng. J. Med. 335: 1956-1961, 1996. [PubMed: 8960475, related citations] [Full Text]

  10. Levin, M., Newport, M. J., D'Souza, S., Kalabalikis, P., Brown, I. N., Lenicker, H. M., Agius, P. V., Davies, E. G., Thrasher, A., Klein, N., Blackwell, J. M. Familial disseminated atypical mycobacterial infection in childhood: a human mycobacterial susceptibility gene? Lancet 345: 79-83, 1995. [PubMed: 7815885, related citations] [Full Text]

  11. Newport, M. J., Huxley, C. M., Huston, S., Hawrylowicz, C. M., Oostra, B. A., Williamson, R., Levin, M. A mutation in the interferon-gamma-receptor gene and susceptibility to mycobacterial infection. New Eng. J. Med. 335: 1941-1949, 1996. [PubMed: 8960473, related citations] [Full Text]

  12. Newport, M., Levin, M., Blackwell, J., Shaw, M.-A., Williamson, R., Huxley, C. Evidence for exclusion of a mutation in NRAMP as the cause of familial disseminated atypical mycobacterial infection in a Maltese kindred. J. Med. Genet. 32: 904-906, 1995. [PubMed: 8592339, related citations] [Full Text]

  13. Nickol, A. D., Bonventre, P. F. Visceral leishmaniasis in congenic mice of susceptible and resistant phenotypes: immunosuppression by adherent spleen cells. Infect. Immun. 50: 160-168, 1985. [PubMed: 2931376, related citations] [Full Text]

  14. Plant, J. E., Blackwell, J. M., O'Brien, A. D., Bradley, D. J., Glynn, A. A. Are the Lsh and Ity disease resistance genes at one locus on mouse chromosome 1? Nature 297: 510-511, 1982. [PubMed: 7045676, related citations] [Full Text]

  15. Schurr, E., Radzioch, D., Malo, D., Ros, P., Skamene, E. Molecular genetics of inherited susceptibility to intracellular parasites. Behring Inst. Mitt. Feb.: 1-12, 1991. [PubMed: 1675564, related citations]

  16. Shaw, M. A., Atkinson, S., Dockrell, H., Hussain, R., Lins-Lainson, Z., Shaw, J., Ramos, F., Silveira, F., Mehdi, S. Q., Kaukab, F., Khaliq, S., Chiang, T., Blackwell, J. An RFLP map for 2q33-q37 from multicase mycobacterial and leishmanial disease families: no evidence for an Lsh/Ity/Bcg gene homologue influencing susceptibility to leprosy. Ann. Hum. Genet. 57: 251-271, 1993. [PubMed: 7910002, related citations] [Full Text]

  17. Skamene, E., Gros, P., Forget, A., Kongshavn, P. A. L., St. Charles, C., Taylor, B. A. Genetic regulation of resistance to intracellular pathogens. Nature 297: 506-509, 1982. [PubMed: 7045675, related citations] [Full Text]

  18. Swanson, R. N., O'Brien, A. D. Genetic control of the innate resistance of mice to Salmonella typhimurium: Ity gene is expressed in vivo by 24 hours after infection. J. Immun. 131: 3014-3020, 1983. [PubMed: 6315821, related citations]

  19. Toyoda, H., Ido, M., Hayashi, T., Gabazza, E. C., Suzuki, K., Bu, J., Tanaka, S., Nakano, T., Kamiya, H., Chipeta, J., Kisenge, R. R., Kang, J., Hori, H., Komada, Y. Impairment of IL-12-dependent STAT4 nuclear translocation in a patient with recurrent Mycobacterium avium infection. J. Immun. 172: 3905-3912, 2004. [PubMed: 15004198, related citations] [Full Text]

  20. Uchiyama, N., Greene, G. R., Warren, B. J., Morozume, P. A., Spear, G. S., Galant, S. P. Possible monocyte killing defect in familial atypical mycobacteriosis. J. Pediat. 98: 785-788, 1981. [PubMed: 7229761, related citations] [Full Text]


Matthew B. Gross - updated : 9/8/2014
Paul J. Converse - updated : 10/2/2012
Paul J. Converse - updated : 2/27/2006
Paul J. Converse - updated : 11/8/2005
Natalie E. Krasikov - updated : 2/9/2004
Paul J. Converse - updated : 12/18/2003
Victor A. McKusick - updated : 12/13/2002
Paul J. Converse - updated : 8/8/2001
Victor A. McKusick - updated : 12/30/1998
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joanna : 12/20/2005
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terry : 6/11/1999
carol : 1/13/1999
terry : 12/30/1998
carol : 6/30/1998
alopez : 5/28/1998
terry : 5/26/1998
terry : 5/26/1998
terry : 5/26/1998
joanna : 8/12/1997
joanna : 1/9/1997
jamie : 1/7/1997
jamie : 1/7/1997
terry : 1/6/1997
jamie : 12/6/1996
terry : 12/3/1996
mark : 1/30/1996
terry : 1/24/1996
mark : 7/18/1995
carol : 2/27/1995
jason : 7/1/1994
pfoster : 4/1/1994
warfield : 3/31/1994
mimadm : 2/19/1994

# 209950

IMMUNODEFICIENCY 27A; IMD27A


Alternative titles; symbols

IMMUNODEFICIENCY 27A, MYCOBACTERIOSIS, AUTOSOMAL RECESSIVE
IFNGR1 DEFICIENCY, AUTOSOMAL RECESSIVE


SNOMEDCT: 718230004;   ORPHA: 319569, 99898;   DO: 0111955;  


Phenotype-Gene Relationships

Location Phenotype Phenotype
MIM number
Inheritance Phenotype
mapping key
Gene/Locus Gene/Locus
MIM number
6q23.3 Immunodeficiency 27A, mycobacteriosis, AR 209950 Autosomal recessive 3 IFNGR1 107470

TEXT

A number sign (#) is used with this entry because of evidence that immunodeficiency-27A (IMD27A), an autosomal recessive disorder, is caused by homozygous or compound heterozygous mutation in the IFNGR1 gene (107470) on chromosome 6q23.

Immunodeficiency-27B (IMD27B; 615978), an autosomal dominant disorder, is allelic.


Description

Immunodeficiency-27A (IMD27A) results from autosomal recessive (AR) IFNGR1 deficiency. Patients with complete IFNGR1 deficiency have a severe clinical phenotype characterized by early and often fatal mycobacterial infections. The disorder can thus be categorized as a form of mendelian susceptibility to mycobacterial disease (MSMD). Bacillus Calmette-Guerin (BCG) and environmental mycobacteria are the most frequent pathogens, and infection typically begins before the age of 3 years. Plasma from patients with complete AR IFNGR1 deficiency usually contains large amounts of IFNG (147570), and their cells do not respond to IFNG in vitro. In contrast, cells from patients with partial AR IFNGR1 deficiency, which is caused by a specific mutation in IFNGR1, retain residual responses to high IFNG concentrations. Patients with partial AR IFNGR1 deficiency are susceptible to BCG and environmental mycobacteria, but they have a milder clinical disease and better prognosis than patients with complete AR IFNGR1 deficiency. The clinical features of children with complete AR IFNGR1 deficiency are usually more severe than those in individuals with AD IFNGR1 deficiency (IMD27B), and mycobacterial infection often occurs earlier (mean age of 1.3 years vs 13.4 years), with patients having shorter mean disease-free survival. Salmonellosis is present in about 5% of patients with AR or AD IFNGR1 deficiency, and other infections have been reported in single patients (review by Al-Muhsen and Casanova, 2008).


Clinical Features

Families with multiple cases of disseminated atypical mycobacteriosis, a rare disorder, were reported by Engbaek (1964) and Uchiyama et al. (1981). Uchiyama et al. (1981) reported fatal disseminated atypical mycobacteriosis in 2 young Mexican-American girls. The atypical mycobacterium was of a different serotype in the 2 sisters. One of the sisters died in 1964 and the other in 1977. Studies by the authors suggested a congenital defect in monocyte microbicidal activity. Fischer et al. (1980) observed defective monocyte function in a 12-month-old child with fatal disseminated BCG infection.

Levin et al. (1995) described 6 children with disseminated atypical mycobacterial infection and no recognized form of immunodeficiency. Four, including 2 brothers, came from a village in Malta, and 2 were brothers of Greek Cypriot origin. They presented with fever, weight loss, lymphadenopathy, and hepatosplenomegaly. They had anemia and an acute phase response. A range of different mycobacteria (Mycobacterium fortuitum, M. chelonei, and 4 strains of M. avium intracellulare) were isolated. Treatment with multiple antibiotics failed to eradicate the infection, although treatment with gamma-interferon was associated with improvement. Three of the children had died and the 3 survivors had chronic infection. TNF-alpha (191160) production in response to endotoxin and gamma-interferon was found to be defective in the patients and their parents. T-cell proliferative responses to mycobacterial and recall antigens were reduced in parents of affected children, and gamma-interferon production was diminished in the patients and their parents. Levin et al. (1995) suggested that these patients are phenotypically similar to Lsh/Ity/Bcg susceptible mice (see ANIMAL MODEL).

Toyoda et al. (2004) examined the immunologic abnormality of a patient with recurrent Mycobacterium avium infection. The patient had reduced expression of IL12RB1 and IL12RB2 and a decreased ability to produce IFNG (147570) and to proliferate in response to IL12. However, the patient exhibited no deficiency in IL12-induced tyrosine and serine phosphorylation of STAT4 (600558) in mitogen-activated T cells. EMSA, confocal laser microscopy, and Western blot analysis demonstrated that nuclear translocation of STAT4 in response to IL12 was reduced in the patient compared with healthy control subjects. Pharmacologic treatment indicated that the defect was not due to upregulated STAT4 export from the nucleus. No mutations in IL12RB1, IL12RB2, STAT4, or the IFNG STAT4-binding sequence were identified, and the exact mechanism for the defect could not be determined.


Diagnosis

Fieschi et al. (2001) found that children with complete IFNGR deficiency, unlike patients with other genetic defects predisposing them to mycobacterial diseases, have very high levels of IFNG in their plasma. Fieschi et al. (2001) proposed this measurement as a simple, inexpensive, and accurate diagnostic test for complete IFNGR deficiency. They noted that early identification of such children, who do not respond to exogenous IFNG or antibiotics, may improve management by leading to the consideration of bone marrow transplantation.


Molecular Genetics

Newport et al. (1996) and Jouanguy et al. (1996) demonstrated that mutations in the interferon-gamma-receptor-1 gene (IFNGR1; 107470) conferred autosomal recessive susceptibility to mycobacterium infection.

Al-Muhsen and Casanova (2008) and Cottle (2011) reviewed genetic heterogeneity of susceptibility to mycobacterial disease.


Genotype/Phenotype Correlations

Dorman et al. (2004) compared the clinical features of recessive and dominant IFNGR1 deficiencies using a worldwide cohort of patients. They assessed the patients by medical histories and genetic and immunologic studies. Recessive deficiency, which Dorman et al. (2004) identified in 22 patients, results in complete loss of cellular response to IFNG and absence of surface IFNGR1 expression. Dominant deficiency, which they identified in 38 patients, is typically due to cytoplasmic domain truncations resulting in accumulation of nonfunctional IFNGR1 proteins that may impede the function of molecules encoded by the wildtype allele, thereby leading to diminished but not absent responsiveness to IFNG. Although the clinical phenotypes are related, Dorman et al. (2004) found that patients with the recessive form had an earlier age of onset (3 vs 13 years), more mycobacterial disease episodes (19 vs 8 per 100 person years of observation), more severe mycobacterial disease (involvement of 4 vs 2 organs), shorter mean disease-free intervals (1.6 vs 7.2 years), and lower Kaplan-Meier survival probability. Recessive patients also had more frequent disease from rapidly growing mycobacteria. Patients with a dominant mutation, however, were more likely to have M. avium complex osteomyelitis, and only dominant patients had osteomyelitis without other organ involvement. Dorman et al. (2004) concluded that there is a strong correlation between the IFNGR1 genotype, clinical disease features, and the cellular responsiveness to IFNG. They suggested that subtle defects in IFNG production, signaling, or related pathways may predispose to diseases caused by virulent mycobacteria, including M. tuberculosis.


Animal Model

There is a mouse gene, variously symbolized Lsh, Ity, and Bcg, on murine chromosome 1 which encodes resistance to bacterial and parasitic infections and affects the function of macrophages (Skamene et al., 1982; Brown et al., 1982; Goto et al., 1984; Plant et al., 1982; Swanson and O'Brien, 1983; Nickol and Bonventre, 1985). Bcg is expressed in 2 allelic forms, the dominant resistance allele and the recessive susceptibility allele. The Bcg region on proximal mouse chromosome 1 shows homology of synteny with the telomeric portion of human 2q; a 35-cM fragment around the murine Bcg locus (from Col3a1 (120180) to Col6a3 (120250)), has been conserved between the 2 species, the human region being 2q32-q37.


History

Schurr et al. (1991) studied linkage of genetic markers on distal chromosome 2q with susceptibility to tuberculosis and found a lod score of 2.4. Shaw et al. (1993), however, could not confirm this finding. They performed linkage analysis using a panel of markers from the 2q33-q37 region in 35 multicase families with infection by Mycobacterium leprae, M. tuberculosis, and Leishmania sp. Data from all 3 types of families were pooled to produce a detailed RFLP map of the region. The order of genes in the human was consistent with that determined for the same loci in the mouse. Nonetheless, Shaw et al. (1993) could not demonstrate linkage of infection susceptibility to this region.

Newport et al. (1995) excluded NRAMP (600266) as the site of the mutation causing this disorder, which they referred to as familial disseminated atypical mycobacterial infection, in a Maltese kindred. They typed 8 markers in the region of 2q34-q37 where NRAMP maps.


REFERENCES

  1. Al-Muhsen, S., Casanova, J.-L. The genetic heterogeneity of mendelian susceptibility to mycobacterial diseases. J. Allergy Clin. Immun. 122: 1043-1051, 2008. [PubMed: 19084105] [Full Text: https://doi.org/10.1016/j.jaci.2008.10.037]

  2. Brown, I. N., Glynn, A. A., Plant, J. Inbred mouse strain resistance to Mycobacterium lepraemurium follows the Ity/Lsh pattern. Immunology 47: 149-156, 1982. [PubMed: 6749659]

  3. Cottle, L. E. Mendelian susceptibility to mycobacterial disease. Clin. Genet. 79: 17-22, 2011. [PubMed: 20718793] [Full Text: https://doi.org/10.1111/j.1399-0004.2010.01510.x]

  4. Dorman, S. E., Picard, C., Lammas, D., Heyne, K., van Dissel, J. T., Baretto, R., Rosenzweig, S. D., Newport, M., Levin, M., Roesler, J., Kumararatne, D., Casanova, J.-L., Holland, S. M. Clinical features of dominant and recessive interferon-gamma receptor 1 deficiencies. Lancet 364: 2113-2121, 2004. [PubMed: 15589309] [Full Text: https://doi.org/10.1016/S0140-6736(04)17552-1]

  5. Engbaek, H. C. Three cases in the same family of fatal infection M. avium. Acta Tuberc. Pneumol. Scand. 45: 105-117, 1964. [PubMed: 14215783]

  6. Fieschi, C., Dupuis, S., Picard, C., Smith, C. I. E., Holland, S. M., Casanova, J.-L. High levels of interferon gamma in the plasma of children with complete interferon gamma receptor deficiency. Pediatrics 107: E48, 2001. Note: Electronic Article. [PubMed: 11335769] [Full Text: https://doi.org/10.1542/peds.107.4.e48]

  7. Fischer, A., Virelizier, J. L., Griscelli, C., Durandy, A., Nezelof, C., Trong, P. H. Defective monocyte functions in a child with fatal disseminated BCG infection. Clin. Immun. Immunopath. 17: 296-306, 1980. [PubMed: 6996876] [Full Text: https://doi.org/10.1016/0090-1229(80)90099-9]

  8. Goto, Y., Nakamura, R. M., Takahashi, H., Tokunaga, T. Genetic control of resistance to Mycobacterium intracellulare infection in mice. Infect. Immun. 46: 135-140, 1984. [PubMed: 6480104] [Full Text: https://doi.org/10.1128/iai.46.1.135-140.1984]

  9. Jouanguy, E., Altare, F., Lamhamedi, S., Revy, P., Emile, J.-F., Newport, M., Levin, M., Blanche, S., Seboun, E., Fischer, A., Casanova, J.-L. Interferon-gamma-receptor deficiency in an infant with fatal bacille Calmette-Guerin infection. New Eng. J. Med. 335: 1956-1961, 1996. [PubMed: 8960475] [Full Text: https://doi.org/10.1056/NEJM199612263352604]

  10. Levin, M., Newport, M. J., D'Souza, S., Kalabalikis, P., Brown, I. N., Lenicker, H. M., Agius, P. V., Davies, E. G., Thrasher, A., Klein, N., Blackwell, J. M. Familial disseminated atypical mycobacterial infection in childhood: a human mycobacterial susceptibility gene? Lancet 345: 79-83, 1995. [PubMed: 7815885] [Full Text: https://doi.org/10.1016/s0140-6736(95)90059-4]

  11. Newport, M. J., Huxley, C. M., Huston, S., Hawrylowicz, C. M., Oostra, B. A., Williamson, R., Levin, M. A mutation in the interferon-gamma-receptor gene and susceptibility to mycobacterial infection. New Eng. J. Med. 335: 1941-1949, 1996. [PubMed: 8960473] [Full Text: https://doi.org/10.1056/NEJM199612263352602]

  12. Newport, M., Levin, M., Blackwell, J., Shaw, M.-A., Williamson, R., Huxley, C. Evidence for exclusion of a mutation in NRAMP as the cause of familial disseminated atypical mycobacterial infection in a Maltese kindred. J. Med. Genet. 32: 904-906, 1995. [PubMed: 8592339] [Full Text: https://doi.org/10.1136/jmg.32.11.904]

  13. Nickol, A. D., Bonventre, P. F. Visceral leishmaniasis in congenic mice of susceptible and resistant phenotypes: immunosuppression by adherent spleen cells. Infect. Immun. 50: 160-168, 1985. [PubMed: 2931376] [Full Text: https://doi.org/10.1128/iai.50.1.160-168.1985]

  14. Plant, J. E., Blackwell, J. M., O'Brien, A. D., Bradley, D. J., Glynn, A. A. Are the Lsh and Ity disease resistance genes at one locus on mouse chromosome 1? Nature 297: 510-511, 1982. [PubMed: 7045676] [Full Text: https://doi.org/10.1038/297510a0]

  15. Schurr, E., Radzioch, D., Malo, D., Ros, P., Skamene, E. Molecular genetics of inherited susceptibility to intracellular parasites. Behring Inst. Mitt. Feb.: 1-12, 1991. [PubMed: 1675564]

  16. Shaw, M. A., Atkinson, S., Dockrell, H., Hussain, R., Lins-Lainson, Z., Shaw, J., Ramos, F., Silveira, F., Mehdi, S. Q., Kaukab, F., Khaliq, S., Chiang, T., Blackwell, J. An RFLP map for 2q33-q37 from multicase mycobacterial and leishmanial disease families: no evidence for an Lsh/Ity/Bcg gene homologue influencing susceptibility to leprosy. Ann. Hum. Genet. 57: 251-271, 1993. [PubMed: 7910002] [Full Text: https://doi.org/10.1111/j.1469-1809.1993.tb00899.x]

  17. Skamene, E., Gros, P., Forget, A., Kongshavn, P. A. L., St. Charles, C., Taylor, B. A. Genetic regulation of resistance to intracellular pathogens. Nature 297: 506-509, 1982. [PubMed: 7045675] [Full Text: https://doi.org/10.1038/297506a0]

  18. Swanson, R. N., O'Brien, A. D. Genetic control of the innate resistance of mice to Salmonella typhimurium: Ity gene is expressed in vivo by 24 hours after infection. J. Immun. 131: 3014-3020, 1983. [PubMed: 6315821]

  19. Toyoda, H., Ido, M., Hayashi, T., Gabazza, E. C., Suzuki, K., Bu, J., Tanaka, S., Nakano, T., Kamiya, H., Chipeta, J., Kisenge, R. R., Kang, J., Hori, H., Komada, Y. Impairment of IL-12-dependent STAT4 nuclear translocation in a patient with recurrent Mycobacterium avium infection. J. Immun. 172: 3905-3912, 2004. [PubMed: 15004198] [Full Text: https://doi.org/10.4049/jimmunol.172.6.3905]

  20. Uchiyama, N., Greene, G. R., Warren, B. J., Morozume, P. A., Spear, G. S., Galant, S. P. Possible monocyte killing defect in familial atypical mycobacteriosis. J. Pediat. 98: 785-788, 1981. [PubMed: 7229761] [Full Text: https://doi.org/10.1016/s0022-3476(81)80848-7]


Contributors:
Matthew B. Gross - updated : 9/8/2014
Paul J. Converse - updated : 10/2/2012
Paul J. Converse - updated : 2/27/2006
Paul J. Converse - updated : 11/8/2005
Natalie E. Krasikov - updated : 2/9/2004
Paul J. Converse - updated : 12/18/2003
Victor A. McKusick - updated : 12/13/2002
Paul J. Converse - updated : 8/8/2001
Victor A. McKusick - updated : 12/30/1998
Victor A. McKusick - updated : 5/26/1998

Creation Date:
Victor A. McKusick : 6/3/1986

Edit History:
carol : 07/25/2023
carol : 08/03/2020
alopez : 07/31/2020
ckniffin : 07/28/2020
alopez : 08/07/2015
carol : 7/22/2015
mgross : 12/9/2014
mgross : 9/8/2014
mgross : 10/8/2012
terry : 10/2/2012
alopez : 3/13/2009
mgross : 3/19/2008
mgross : 3/22/2007
mgross : 2/5/2007
mgross : 6/22/2006
terry : 2/27/2006
joanna : 12/20/2005
mgross : 11/8/2005
alopez : 7/5/2005
alopez : 6/15/2005
alopez : 6/15/2005
terry : 6/3/2005
ckniffin : 5/4/2005
carol : 2/9/2004
mgross : 12/18/2003
tkritzer : 12/18/2002
tkritzer : 12/16/2002
terry : 12/13/2002
mgross : 2/20/2002
mgross : 8/8/2001
terry : 6/11/1999
carol : 1/13/1999
terry : 12/30/1998
carol : 6/30/1998
alopez : 5/28/1998
terry : 5/26/1998
terry : 5/26/1998
terry : 5/26/1998
joanna : 8/12/1997
joanna : 1/9/1997
jamie : 1/7/1997
jamie : 1/7/1997
terry : 1/6/1997
jamie : 12/6/1996
terry : 12/3/1996
mark : 1/30/1996
terry : 1/24/1996
mark : 7/18/1995
carol : 2/27/1995
jason : 7/1/1994
pfoster : 4/1/1994
warfield : 3/31/1994
mimadm : 2/19/1994