Ten-year follow up of hydroxychloroquine treatment for ABCA3 deficiency

Pediatric Pulmonology

Volumn 49, Issue 3, 2014, Pages 299-301

  Williamson, Michael ^a   Wallis, Colin ^a  

^a GREAT ORMOND STREET HOSPITAL   (United Kingdom)

Author keywords

ABCA3; hydroxychloroquine; interstitial lung disease; surfactant protein deficiency

Indexed keywords

ABCA3; HYDROXYCHLOROQUINE; INTERSTITIAL LUNG DISEASE; SURFACTANT PROTEIN DEFICIENCY;

ADOLESCENT; ATP-BINDING CASSETTE TRANSPORTERS; CHILD; CHILD, PRESCHOOL; FEMALE; HUMANS; HYDROXYCHLOROQUINE; IMMUNOSUPPRESSIVE AGENTS; LONGITUDINAL STUDIES; LUNG DISEASES, INTERSTITIAL; TREATMENT OUTCOME;

EID: 84894228217     PISSN: 87556863     EISSN: 10990496     Source Type: Journal    
DOI: 10.1002/ppul.22811     Document Type: Article

References (10)

1. Yamanoa G, Funahashib H, Kawanamic O, Zhaoa LX, Bana N, Uchidad Y, Morohoshie T, Ogawaf J, Shiodab S, Inagaki N,. ABCA3 is a lamellar body membrane protein in human lung alveolar type II cells. FEBS Lett 2001; 508: 221-225.
2. Shulenin S, Nogee LM, Annilo T, Wert SE, Whitsett JA, Dean M,. ABCA3 gene mutations in newborns with fatal surfactant deficiency. N Eng J Med 2004; 350: 1296-1303.
3. Bullard JE, Wert SE, Whitsett JA, Dean M, Nogee LM,. ABCA3 mutations associated with pediatric interstitial lung disease. Am J Respir Crit Care Med 2005; 172: 1026-1031.
4. Dinwiddie R,. Treatment of interstitial lung disease in children. Paediatr Respir Rev 2004; 5: 108-115.
5. Weichert N, Kaltenborn E, Hector A, Woischnik M, Schams A, Holzinger A, Kern S, Griese M,. Some ABCA3 mutations elevate ER stress and initiate apoptosis of lung epithelial cells. Respir. Res 2011; 12: 4.
6. Matsumara Y, Ban N, Inagaki N,. Aberrant catalytic cycle and impaired lipid transport into intracellular vesicles in ABCA3 mutants associated with nonfatal pediatric interstitial lung disease. Am J Physiol Lung Cell Mol Physiol 2008; 295: L698-L707.
7. Yokota T, Matsumura Y, Ban N, Matsubayashi T, Inagaki N,. Heterozygous ABCA3 mutation associated with non-fatal evolution of respiratory distress. Eur J Pediatr 2008; 167: 691-693.
8. Lombard-Platlet S, Bertolino P, Deng H, Gerlier D, Raboudin-Combe C,. Inhibition by chloroquine of the class II major histocompatibility complex-restricted presentation of endogenous antigens varies according to the cellular origin of the antigen-presenting cells, the nature of the T-cell epitope, and the responding T cell. Immunology 1993; 80: 566-573.
9. Goldman FD, Gilman AL, Hollenback C, Kato RM, Premack BA, Rawlings DJ,. Hydroxychloroquine inhibits calcium signals in T cells: a new mechanism to explain its immunomodulatory properties. Blood 2000; 95: 3460-3466.
10. The Royal College of Ophthalmologists-Hydroxychloroquine and ocular toxicity: recommendations on screening. October 2009.