Ivacaftor in a young boy with the rare gating mutation S549R - use of lung clearance index to track progress: a case report

Table 1 Improvement of functional parameters during ivacaftor therapy

	Start	6 weeks	12 weeks	28 weeks	41 weeks
Sweat chloride level [mmol/l]	115	40	30	40	52
FEV1 [l ] (z-score) ^a	1.25 (−1.2)	1.53 (0.5)	1.48 (0.2)	1.63 (0.7)	1.65 (0.5)
LCI (z-score) ^b	14.5 (14.2)	8.3 (2.4)	7.6 (1.1)	8.1 (2.0)	7.8 (1.5)
Weight [kg] (z-score)	21.8 (−1.5)	23.2 (−1.1)	23.7 (−0.9)	23.9 (−0.9)	26 (−0.5)
BMI [kg/m²] (z-score)	14.6 (−0.8)	15.6 (−0.2)	15.7 (−0.1)	15.5 (−0.2)	16.4 (0.2)

Improvement of different functional parameters during 41 weeks of ivacaftor therapy: FEV1 Forced expiratory volume in the first second, LCI lung clearance index. ^aReference population for z-score values of FEV1 are the global multi-ethnic reference equations of the global lung function initiative [16]. ^bReference population for z-score values of the LCI is our intracentric age-matched healthy control population

ISSN: 1471-2466