Peter van Mourik

145 Personalized treatment of CF using rectal organoids Table 1: Patient characteristics and treatment regimes Treatment (duration) CFTR-genotypeMedian age in years at baseline (IQR) Median ppFEV 1 at baseline (IQR) Median SCC in mmol/L at baseline (IQR) Genistein plus Curcumin (8 weeks) S1251N (p.Ser1251Asn) / F508del (p.Phe508del) n=12 a S1251N (p.Ser1251Asn) / R117H (p.Arg117His) n=1 a 15.0 (10.0 – 33.0) 75.5 (64.0 – 93.8) 80.0 (65.5 – 91.0) Ivacaftor (4-8 weeks) S1251N (p.Ser1251Asn) / F508del (p.Phe508del) n=12 a S1251N (p.Ser1251Asn) / R117H (p.Arg117His) n=1 a S1251N (p.Ser1251Asn) / A455E (p.Ala455Glu) n=1 S1251N (p.Ser1251Asn) / 1717-1G>A (c.1585-1G>A) n=1 G1249R (p.Gly1249Arg) / F508del (p.Phe508del) n=2 G461R (p.Gly461Arg) / F508del (p.Phe508del) n=2 S945L (p.Ser945Leu) / F508del (p.Phe508del) n=1 R334W (p.Arg334Trp) / R764X (p.Arg764X) n=1 R553X (p.Arg553X) / 4375-3T>A (c.4243-3T>A) n=1 16.5 (11.3 – 35.8) 73.0 (59.5 – 94.5) 77.0 (64.0 – 94.0) Lumacaftor plus Ivacaftor (4 weeks) R347P (p.Arg347Pro) / F508del (p.Phe508del) n=1 W1282X (p.Trp1282X) / F508del (p.Phe508del) n=1 35.0 30.0 97.0 a Patients were treated with both genistein/curcumin and ivacaftor. CFTR, Cystic Fibrosis Transmembrane conductance Regulator; IQR, Inter Quartile Range; ppFEV 1 , percentage of predicted forced expiratory volume in one second; SCC, Sweat Chloride Concentration 7