title: Is treatment with hydroxychloroquine effective in surfactant protein C deficiency?
abstract: 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 Two years after the treatment was withdrawn, the twins were evaluated once again: they presented no new infections, growth and general state were normal and chest CT showed a notable additional reduction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.
authors.affiliation: Institute for Maternal and Child Health - IRCCS «Burlo Garofolo», University of Trieste, Trieste, Italia. ingridrabach@gmail.com
authors.forename: Ingrid
authors.initials: I
authors.lastname: Rabach
authors.forename: Furio
authors.initials: F
authors.lastname: Poli
authors.forename: Floriana
authors.initials: F
authors.lastname: Zennaro
authors.forename: Claudio
authors.initials: C
authors.lastname: Germani
authors.forename: Alessandro
authors.initials: A
authors.lastname: Ventura
authors.forename: Egidio
authors.initials: E
authors.lastname: Barbi
mesh_terms: D018450:Disease Progression; D004200:Diseases in Twins / Q000188:drug therapy* / Q000235:genetics; D004417:Dyspnea / Q000209:etiology; D005183:Failure to Thrive / Q000209:etiology; D006801:Humans; D006886:Hydroxychloroquine / Q000627:therapeutic use*; D007231:Infant, Newborn; D008297:Male; D011649:Pulmonary Alveolar Proteinosis / Q000150:complications / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000235:genetics; D037721:Pulmonary Surfactant-Associated Protein C / Q000172:deficiency*; D012131:Respiratory Insufficiency / Q000209:etiology; D014057:Tomography, X-Ray Computed; D014430:Twins, Monozygotic
chemical_list: D037721:Pulmonary Surfactant-Associated Protein C; D006886:Hydroxychloroquine