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Specific granule deficiency

MedGen UID:
140766
Concept ID:
C0398593
Disease or Syndrome
Synonyms: Lactoferrin-Deficient Neutrophils; Neutrophil Lactoferrin Deficiency; Specific Granule Deficiency
SNOMED CT: Specific granule deficiency (234431006); SGD - Specific granule deficiency (234431006); Neutrophil lactoferrin deficiency (234587000)
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).
 
Monarch Initiative: MONDO:0009506
OMIM®: 245480
OMIM® Phenotypic series: PS245480
Orphanet: ORPHA169142

Definition

Specific granule deficiency-1 (SGD1) is an immunologic disorder characterized by onset of recurrent bacterial infections in infancy or early childhood. Both autosomal recessive and autosomal dominant inheritance have been reported. Affected individuals usually develop skin infections due to Staphylococcus aureus which may progress to more invasive infections. Otitis media and other upper respiratory infections are frequently observed. Neutrophils from patients with the disorder display atypical hyposegmented or bilobed nuclei and show decreased or absent expression of secondary and tertiary granule proteins due to a defect in myeloid maturation. Neutrophils may also demonstrate defects in chemotaxis or bactericidal activity; some patients show neutropenia and lack of eosinophils. Treatment consists mainly of prophylactic antibiotics, although at least 1 patient has been cured with hematopoietic stem cell transplantation (summary by Gombart et al., 2001 and Leszcynska et al., 2020). Genetic Heterogeneity of Specific Granule Deficiency See also SGD2 (617475), caused by mutation in the SMARCD2 gene (601736) on chromosome 17q23. [from OMIM]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVSpecific granule deficiency
Follow this link to review classifications for Specific granule deficiency in Orphanet.

Professional guidelines

PubMed

Uzel G, Holland SM
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Recent clinical studies

Etiology

Ye Y, Carlsson G, Karlsson-Sjöberg JM, Borregaard N, Modéer TU, Andersson ML, Pütsep KL
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Diagnosis

Kihtir Z, Çelik K, Tayfun Küpesiz F, Küpesiz OA, Kocacik Uygun DF, Arayici S, Ongun H, Acarbulut İ, Sağlam C, Ceylaner G, Bingöl A
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Therapy

Yucel E, Karakus IS, Krolo A, Kiykim A, Heredia RJ, Tamay Z, Cipe FE, Karakoc-Aydiner E, Ozen A, Karaman S, Boztug K, Baris S
J Clin Immunol 2021 Jan;41(1):59-65. Epub 2020 Oct 6 doi: 10.1007/s10875-020-00878-4. PMID: 33025377
Taverniti V, Koirala R, Dalla Via A, Gargari G, Leonardis E, Arioli S, Guglielmetti S
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Prognosis

Yucel E, Karakus IS, Krolo A, Kiykim A, Heredia RJ, Tamay Z, Cipe FE, Karakoc-Aydiner E, Ozen A, Karaman S, Boztug K, Baris S
J Clin Immunol 2021 Jan;41(1):59-65. Epub 2020 Oct 6 doi: 10.1007/s10875-020-00878-4. PMID: 33025377
Skoczylas MM, Walat A, Kordek A, Loniewska B, Rudnicki J, Maleszka R, Torbé A
Ann Parasitol 2014;60(3):179-89. PMID: 25281815
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J Clin Immunol 2014 Oct;34(7):780-3. Epub 2014 Aug 1 doi: 10.1007/s10875-014-0082-8. PMID: 25081842
Gombart AF, Shiohara M, Kwok SH, Agematsu K, Komiyama A, Koeffler HP
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Clinical prediction guides

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J Clin Immunol 2022 Oct;42(7):1434-1450. Epub 2022 Jun 20 doi: 10.1007/s10875-022-01304-7. PMID: 35726044
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Front Immunol 2018;9:588. Epub 2018 Mar 29 doi: 10.3389/fimmu.2018.00588. PMID: 29651288Free PMC Article
Wada T, Akagi T
Crit Rev Immunol 2016;36(4):349-358. doi: 10.1615/CritRevImmunol.2017019385. PMID: 28322138
Shiohara M, Gombart AF, Sekiguchi Y, Hidaka E, Ito S, Yamazaki T, Koeffler HP, Komiyama A
J Leukoc Biol 2004 Feb;75(2):190-7. Epub 2003 Oct 23 doi: 10.1189/jlb.0203063. PMID: 14576362
Lekstrom-Himes JA, Dorman SE, Kopar P, Holland SM, Gallin JI
J Exp Med 1999 Jun 7;189(11):1847-52. doi: 10.1084/jem.189.11.1847. PMID: 10359588Free PMC Article

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