Predictors of mortality in children with cystic fibrosis in India

Abstract

Background There is a lack of studies on cystic fibrosis (CF) outcomes in children from developing countries like India. Identifying risk factors for mortality may help identify the high‐risk group and plan policy management of such patients. We aimed to determine the factors associated with mortality among Indian children with CF. Methods In this retrospective study, we extracted demography, clinical features, laboratory and outcome data from medical records of children with CF. Bivariate and multivariate analysis was performed to identify variables associated with mortality. Results We enrolled 178 children, and there were 32 (18.0%) deaths. Median (IQR) z‐score for body mass index (BMI) at last follow up was −1.5 (−2.7, −0.2) and −2.5 (−4.0, −1.6), p‐value 0.039, in survived and deceased group respectively. Mean (SD) of % predicted of FEV1/FVC and FEV1 25–75 at the time of diagnosis in survived versus diseased group was 94.7 (24.1) versus 81.5 (19.1), p‐value 0.063 and 56.1 (38.9) versus 45.7 (29.9), p‐value 0.347, respectively. Significant factors associated with mortality included history of neonatal complications; hazard ratio (HR): 8.5 (95% confidence interval [CI]: 3.0–23.9, p < 0.001), low Z‐scores for BMI at the time of diagnosis; HR: 7.1 (95% CI: 2.3–22.0, p < 0.001), FEV1/FVC at the time of diagnosis; HR: 5.1 (95% CI: 1.65–15.4, p < 0.004), and FEV1 25–75; HR: 3.6 (95% CI: 1.1–11.8, p = 0.03). Conclusions Factors associated with increased mortality risk included neonatal complications, low BMI, and lower pulmonary function test results. Low BMI and low PFT indices can be improved upon by timely treatment of respiratory infections, better nutrition, early diagnosis, and treatment. A newborn screening program may help in early diagnosis and identification of the neonatal problem of CF.

CFTR mutations and phenotypic correlations in people with cystic fibrosis: a retrospective study from a single centre in south India

“…Eighteen of 32 (56%) deaths were among children younger than two years, including 14 (44%) among infants younger than six months old. Our observation is probably just the tip of the iceberg of CF-related infant and childhood mortality in the Indian subcontinent, 27 and further epidemiologic assessments are needed urgently. Highly pathogenic CFTR variants F508del and c.1029delC were commonly observed in infant and early childhood deaths.…”

Is Cystic Fibrosis Contributing Significantly to Infant Mortality Rate in India?

“…Over the last 80 years, predicted life expectancy of affected individuals has improved from less than one year to the fifth decade of life, largely due to early initiation of aggressive supportive treatment [1]. In India, diagnosis of cystic fibrosis is made at an average age of 4.5 to 6.5 years and is associated with high mortality in childhood [2][3][4].…”