A first-year dornase alfa treatment impact on clinical parameters of patients with cystic fibrosis: the Brazilian cystic fibrosis multicenter study.
OBJECTIVE
To describe the clinical impact of the first year treatment with dornase alfa, according to age groups, in a cohort of Brazilian Cystic Fibrosis (CF) patients.


METHODS
The data on 152 eligible patients, from 16 CF reference centers, that answered the medical questionnaires and performed laboratory tests at baseline (T0), and at six (T2) and 12 (T4) months after dornase alfa initiation, were analyzed. Three age groups were assessed: six to 11, 12 to 13, and >14 years. Pulmonary tests, airway microbiology, emergency room visits, hospitalizations, emergency and routine treatments were evaluated. Student's t-test, chi-square test and analysis of variance were used when appropriated.


RESULTS
Routine treatments were based on respiratory physical therapy, regular exercises, pancreatic enzymes, vitamins, bronchodilators, corticosteroids, and antibiotics. In the six months prior the study (T0 phase), hospitalizations for pulmonary exacerbations occurred in 38.0, 10.0 and 61.4% in the three age groups, respectively. After one year of intervention, there was a significant reduction in the number of emergency room visits in the six to 11 years group. There were no significant changes in forced expiratory volume in one second (VEF(1)), in forced vital capacity (FVC), in oxygen saturation (SpO(2)), and in Tiffenau index for all age groups. A significant improvement in Shwachman-Kulczychi score was observed in the older group. In the last six months of therapy, chronic or intermittent colonization by P. aeruginosa was detected in 75.0, 71.4 and 62.5% of the studied groups, respectively, while S. aureus colonization was identified in 68.6, 66.6 and 41.9% of the cases.


CONCLUSIONS
The treatment with dornase alfa promoted the maintenance of pulmonary function parameters and was associated with a significant reduction of emergency room visits due to pulmonary exacerbations in the six to 11 years age group, with better clinical scores in the >14 age group, one year after the intervention.
Escola Paulista de Medicina, Unifesp, São PauloSP, Brasil.
Tatiana
T
Rozov
Faculdade de Medicina, Universidade Federal do Rio Grande do Sul, Porto AlegreRS, Brasil.
Fernando Antônio A E
FA
Silva
Hospital Especializado Octávio Mangabeira, Secretaria da Saúde da Bahia, SalvadorBA, Brasil.
Maria Angélica
MA
Santana
Hospital das Clínicas, Faculdade de Medicina, Universidade de São Paulo, São PauloSP, Brasil.
Fabíola Villac
FV
Adde
Hospital de Base do Distrito Federal, BrasíliaDF, Brasil.
Rita Heloisa
RH
Mendes
D000293:Adolescent; D001938:Brazil; D002648:Child; D003550:Cystic Fibrosis / Q000188:drug therapy*; D003850:Deoxyribonuclease I / Q000627:therapeutic use*; D006801:Humans; D011446:Prospective Studies; D011994:Recombinant Proteins / Q000627:therapeutic use; D013997:Time Factors
D011994:Recombinant Proteins; D003850:Deoxyribonuclease I; C568813:dornase alfa