6-month approval of Kaftrio (R) in Germany-First experiences from real life of people with CF

Thomassen, J. C., Meyer, M., Hagmeyer, L., Striegel, A. K., Metz, F., Koerner, R., Rietschel, E. and van Koningsbruggen-Rietschel, S. . 6-month approval of Kaftrio (R) in Germany-First experiences from real life of people with CF. Mon.schr. Kinderheilkd.. NEW YORK: SPRINGER. ISSN 1433-0474

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DOI:10.1007/s00112-021-01397-1

Abstract

Background By advancement of the cystic fibrosis transmembrane conductance regulator (CFTR) modulators therapy and the approval of Kaftrio (R), for the majority of people with CF a further promising therapeutic option for the causal treatment of CF in addition to the established symptomatic therapy is now available. Objective The aim of this investigation was to analyze the first effects of Kaftrio (R) therapy on the clinical phenotype of people with CF 6 months after approval as well as to put the results into the context of new challenges and questions for future treatment of CF patients. Material and methods Clinical data (ppFEV(1), BMI, HbA(1c), sweat chloride, liver enzymes) from 56 CF patients were collected before and after receiving Kaftrio (R) for more than 4 weeks. The analysis of data was anonymized and retrospective. The paired t-test was used to compare the changes of the different parameters, a p-value Results Treatment with Kaftrio (R) was associated with an increase in ppFEV(1) (+11.3% predicted), an increased body mass index (+0.89 kg/m(2)), a decreased sweat chloride concentration (-58.8 mmol/l) and an increase of liver enzymes. Discussion Our retrospective data collection confirms the positive effect of Kaftrio (R) therapy on the clinical phenotype of people with CF as described in previous clinical trials. The improvement of lung function and nutritional status as well as the resulting increased life expectancy, will lead to the need for a re-evaluation of previous treatment standards and the future focus should also be on maintaining the quality of life through early diagnosis and efficient treatment of possible concomitant diseases.

Item Type:

Journal Article

Creators:

Creators	Email	ORCID	ORCID Put Code
Thomassen, J. C.	UNSPECIFIED	UNSPECIFIED	UNSPECIFIED
Meyer, M.	UNSPECIFIED	UNSPECIFIED	UNSPECIFIED
Hagmeyer, L.	UNSPECIFIED	UNSPECIFIED	UNSPECIFIED
Striegel, A. K.	UNSPECIFIED	UNSPECIFIED	UNSPECIFIED
Metz, F.	UNSPECIFIED	UNSPECIFIED	UNSPECIFIED
Koerner, R.	UNSPECIFIED	UNSPECIFIED	UNSPECIFIED
Rietschel, E.	UNSPECIFIED	UNSPECIFIED	UNSPECIFIED
van Koningsbruggen-Rietschel, S.	UNSPECIFIED	UNSPECIFIED	UNSPECIFIED