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Infantile-onset pulmonary alveolar proteinosis-hypogammaglobulinemia

MedGen UID:
1810375
Concept ID:
C5680364
Disease or Syndrome
Synonyms: 2'-5'-oligoadenylate synthetase 1-related infantile-onset pulmonary alveolar proteinosis, hypogammaglobulinemia; Infantile-onset pulmonary alveolar proteinosis, hypogammaglobulinemia; infantile-onset pulmonary alveolar proteinosis-hypogammaglobulinemia; OAS1 deficiency; OAS1-related infantile-onset pulmonary alveolar proteinosis, hypogammaglobulinemia; OAS1-related infantile-onset pulmonary alveolar proteinosis-hypogammaglobulinemia
SNOMED CT: OAS1-related infantile-onset pulmonary alveolar proteinosis, hypogammaglobulinemia (1197476009); 2'-5'-oligoadenylate synthetase 1-related infantile-onset pulmonary alveolar proteinosis, hypogammaglobulinemia (1197476009); Infantile-onset pulmonary alveolar proteinosis, hypogammaglobulinemia (1197476009)
 
Monarch Initiative: MONDO:0035529
Orphanet: ORPHA572428

Definition

A rare genetic respiratory disease characterised by infantile onset of pulmonary alveolar proteinosis with hypogammaglobulinaemia. Patients have normal respiratory function at birth, but subsequently develop recurrent, mainly viral, infections and progressive respiratory failure, often leading to death in infancy or early childhood. Additional reported features include leucocytosis and splenomegaly. [from SNOMEDCT_US]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVInfantile-onset pulmonary alveolar proteinosis-hypogammaglobulinemia

Recent clinical studies

Diagnosis

Seidl E, Schramm D, Schön C, Reiter K, Pawlita I, Kappler M, Reu-Hofer S, Hauck F, Albert M, Griese M
Pediatr Pulmonol 2022 Jan;57(1):273-277. Epub 2021 Nov 15 doi: 10.1002/ppul.25728. PMID: 34647697

Prognosis

Seidl E, Schramm D, Schön C, Reiter K, Pawlita I, Kappler M, Reu-Hofer S, Hauck F, Albert M, Griese M
Pediatr Pulmonol 2022 Jan;57(1):273-277. Epub 2021 Nov 15 doi: 10.1002/ppul.25728. PMID: 34647697

Clinical prediction guides

Cho K, Yamada M, Agematsu K, Kanegane H, Miyake N, Ueki M, Akimoto T, Kobayashi N, Ikemoto S, Tanino M, Fujita A, Hayasaka I, Miyamoto S, Tanaka-Kubota M, Nakata K, Shiina M, Ogata K, Minakami H, Matsumoto N, Ariga T
Am J Hum Genet 2018 Mar 1;102(3):480-486. Epub 2018 Feb 15 doi: 10.1016/j.ajhg.2018.01.019. PMID: 29455859Free PMC Article

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