• Shih Ying Hng, Agnes Suganthi Thinakaran, Chiou Jia Ooi, Kah Peng Eg, Meow Keong Thong, Sok Kun Tae, Saw Huan Goh, Kee Seang Chew, Lay Teng Tan, Mia Tuang Koh, Li Ai Chong, Farah Khalid, Ruey Teng Ng, Anna Marie Nathan, and Jessie Anne de Bruyne.
• Department of Paediatrics, Paediatric Respiratory Unit, University of Malaya, Kuala Lumpur, Malaysia.
• Singapore Med J. 2023 Sep 19.

Introduction: Asian children with cystic fibrosis (CF) managed in Malaysia have significant morbidity with limited access to life-sustaining treatments. We determined the morbidity and treatment cost of CF in a resource-limited country.MethodsThis cross-sectional study included all children diagnosed with CF in our centre. Data on clinical presentation, genetic mutation, serial spirometry results and complications were collected. Out-of-pocket (OOP) and healthcare costs over 1 year were retrieved for patients who were alive. Cohen's d and odds ratio (OR) were used to determine the effect size.ResultsTwenty-four patients were diagnosed with CF. Five patients died at a median (range) age of 18 (0.3-22) years. F508deletion (c. 1521_1523delCTT) was found in 20% of the alleles, while 89% of the variants were detected in nine patients. Body mass index (BMI) Z score was >-1.96 in 70.6% of patients. Two thirds (68%) were colonised with Pseudomonas aeruginosa, and this was associated with lower weight (P = 0.009) and BMI (P = 0.02) Z scores. Only 18% had FEV1 Z scores >-1.96. Early symptom onset (d = 0.74), delayed diagnosis (d = 2.07), a low FEF25-75 Z score (d = 0.82) and a high sweat conductance (d = 1.19) were associated with death. Inpatient cost was mainly from diagnostic tests, while medications contributed to half of the outpatient cost.UnlabelledHealthcare utilisation cost was catastrophic, amounting to 20% of the total income.ConclusionAsian children with CF suffer significant complications such as low weight, low lung function and shortened lifespan. P. aeruginosa colonisation was frequent and associated with poor growth. Healthcare cost to parents was catastrophic.

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