TY - JOUR T1 - Is treatment with hydroxychloroquine effective in surfactant protein C deficiency? JF - Arch Bronconeumol Y1 - 2013 A1 - Rabach, Ingrid A1 - Poli, Furio A1 - Zennaro, Floriana A1 - Germani, Claudio A1 - Ventura, Alessandro A1 - Barbi, Egidio KW - Disease Progression KW - Diseases in Twins KW - Dyspnea KW - Failure to Thrive KW - Humans KW - Hydroxychloroquine KW - Infant, Newborn KW - Male KW - Pulmonary Alveolar Proteinosis KW - Pulmonary Surfactant-Associated Protein C KW - Respiratory Insufficiency KW - Tomography, X-Ray Computed KW - Twins, Monozygotic AB -

We present the case of two twin brothers with surfactant protein C deficiency who were treated with hydroxychloroquine for three years, with apparent success. The exact physiopathology of this disease is not known and there is no specific treatment for it. There is merely news from a few previous descriptions in the literature about the use of hydroxychloroquine for surfactant protein C deficiency with satisfactory results. Two years after the treatment was withdrawn, the twins were evaluated once again: they presented no new infections, growth and general state were normal and chest CT showed a notable additional reduction in the interstitial pneumopathy. These data seem to cast some doubt on the efficacy of hydroxychloroquine, and they suggest that the clinical improvement was simply the natural evolution of the disease.

VL - 49 IS - 5 U1 - http://www.ncbi.nlm.nih.gov/pubmed/23137777?dopt=Abstract ER -