Helmut Ellemunter - Academia.edu (original) (raw)

Papers by Helmut Ellemunter

Research paper thumbnail of Arm muscle area for the longitudinal assessment of nutritional status in paediatric patients with cystic fibrosis - A single centre experience

Journal of Cystic Fibrosis, 2021

Background: In cystic fibrosis, adequate nutrition contributes to good long-term prognosis. A bod... more Background: In cystic fibrosis, adequate nutrition contributes to good long-term prognosis. A body mass index (BMI) at or above the 50th percentile for age and sex in all children has been recommended. As researchers have described a depletion of fat-free mass despite normal BMI, longitudinal studies using more sensitive nutritional parameters are warranted. We evaluated anthropometric measurements in an attempt to identify early indicators of deteriorating nutritional status in our paediatric cohort. Methods: We analysed datasets from children and adolescents between 2 and 17.9 years with at least two entries for triceps skinfold thickness and upper arm circumference in our patient database between January 1995 and December 2018. Arm muscle area (AMA) was calculated, and all values were expressed as z-scores from CDC growth charts. Results: A total of 4,862 encounters from 161 paediatric patients (78 girls) were available, representing a median number of 28 visits during a median follow-up of 8.1 years per patient. Linear mixed effects models revealed relatively stable courses for weight, height, BMI and skinfold thickness up to adulthood. AMA was the only parameter which declined slightly (r =-0.036), particularly in boys. Kaplan-Meieranalyses showed that AMA was the earliest parameter to decrease below-1 z-score between 6 and 18 years. Conclusions: The present data suggest that compared with weight or BMI, AMA could serve as an earlier indicator of a deteriorating nutritional status. The benefit of assessing skinfold thickness and arm circumference routinely and calculating AMA from these measurements should be evaluated in large, prospective, multi-centre studies.

Research paper thumbnail of REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report

Clinical Case Reports, 2022

This study summarizes efficacy of ivacaftor treatment in 2 infants in a real‐world setting. A dis... more This study summarizes efficacy of ivacaftor treatment in 2 infants in a real‐world setting. A distinct decline of sweat chloride and lung clearance index plus increase in fecal elastase was seen. The results underline the early and sustainable effect and give cause for discussing whether a reduction in standard cystic fibrosis therapy is possible.

Research paper thumbnail of Lung clearance index to detect the efficacy of Aztreonam lysine inhalation in patients with cystic fibrosis and near normal spirometry – A single-centre feasibility study

PLOS ONE, 2019

Comparing the efficacy of inhaled antibiotics can be difficult in small groups of patients with c... more Comparing the efficacy of inhaled antibiotics can be difficult in small groups of patients with cystic fibrosis and mild lung disease. In a feasibility study we compared Aztreonam lysine for inhalation solution (AZLI; Cayston ®) to standard inhaled antibiotic therapy in patients with cystic fibrosis and near normal spirometry. To detect treatment responses we used both lung clearance index (LCI) and forced expiratory volume in one second (FEV 1). At baseline, median FEV 1 was 87% pred. and median LCI was 8.6 (upper limit of normal: 7.0). After 4 weeks, LCI improved by-0.36 after AZLI and deteriorated by +0.12 after tobramycin treatment (p = 0.039). No significant differences between treatments (p = 0.195) were observed using FEV 1. These results suggest that lung clearance index can be used to detect treatment induced changes in subjects with mild lung disease.

Research paper thumbnail of Care in an ISO Certified Centre for Patients With Cystic Fibrosis and Their Families

Die Mukoviszidose (CF) als Störung exokriner Drüsen betrifft verschiedene Organsysteme. Hauptmani... more Die Mukoviszidose (CF) als Störung exokriner Drüsen betrifft verschiedene Organsysteme. Hauptmanifestationen sind die chronisch obstruktive Lungenerkrankung und die Pankreasinsuffizienz. Bei vielen Patienten kommen diverse andere Probleme hinzu, beispielsweise Nasenpolypen, Hepatopathie, CF-Diabetes, Unterernährung, Infertilität (bei den meisten Männern) oder Osteoporose. Während vor 60 Jahren nach der Erstbeschreibung der Erkrankung die meisten BeZusammenfassung !

Research paper thumbnail of Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations

Journal of Cystic Fibrosis

Research paper thumbnail of Faecal calprotectin concentrations in young children with cystic fibrosis

Journal of Cystic Fibrosis, 2018

Research paper thumbnail of P271 Malignant diseases in patients treated at the CF Centre Innsbruck

Journal of Cystic Fibrosis

Research paper thumbnail of P399 High utilisation of an ongoing group education programme for parents as part of routine care

Journal of Cystic Fibrosis

Research paper thumbnail of P452 Predicting psychological well-being and symptoms in adults living with cystic fibrosis: the role of self-compassion and psychological resilience

Journal of Cystic Fibrosis

Research paper thumbnail of S2-Konsensus-Leitlinie „Diagnose der Mukoviszidose“

Research paper thumbnail of Long-term improvement of lung clearance index in patients with mild cystic fibrosis lung disease: Does hypertonic saline play a role?

Journal of Cystic Fibrosis, 2015

To assess whether long-term inhalation with hypertonic saline is able to halt the progression of ... more To assess whether long-term inhalation with hypertonic saline is able to halt the progression of mild CF lung disease, we analysed longitudinal data of lung clearance index (LCI) and spirometry. A total of 34 patients with mild lung disease (FEV 1 ≥ 70% of predicted) had at least one LCI result before and ≥ 2 LCI measurements after start of hypertonic saline (HS) therapy. After a mean follow-up of 39.7 (SD 7.4) months after starting HS, LCI improved significantly from 7.89 (SD 1.35) at baseline to 6.96 (SD 1.03), and 19/34 patients had a normal LCI value at the last measurement. No decrease in mean FEV 1 was observed. Thus, ventilation inhomogeneity can improve in patients with mild lung disease.

Research paper thumbnail of WS09.4 The role of resilience in quality of life of adult patients with cystic fibrosis (CF)

Journal of Cystic Fibrosis, 2015

Objectives Although several psychological studies on coping and quality of life suggested high le... more Objectives Although several psychological studies on coping and quality of life suggested high levels of resilience in CF patients, empirical data on resilience in CF are barely existent. Thus, the purpose of the study was to investigate resilience in adult CF patients and its impact on quality of life. Methods 57 adult CF patients (18–59 years) participated in the study at their routine medical check-up. In addition to their regular psychological assessment including the Cystic Fibrosis Questionnaire – Revised (CFQ-R), the Resilience Scale (RS) was administered. The constructs were also correlated with physical parameters (age, gender and lung function). Results Patients reported high levels of resilience and overall good quality of life in all domains. Patient's resilience was significantly higher compared to the large healthy reference group (t[2086] = 4.7510, p=0.0001) and was independent of gender (t[55] = 0.826, p=0.412), age (r = 0.076, p=0.573) and lung function (FEV1%, r=0.165, p=0.220). Significant associations between resilience and CFQ-R domains could be confirmed. Conclusion Our study is one of the first attempts to explicitely test resilience in CF patients and could demonstrate high levels of resilience in these patient group and positive correlations with quality of life. These results suggest that resilience could be seen as an important factor on quality of life and coping with CF. Therefore fostering resilience early in patient's life should be encouraged.

Research paper thumbnail of 288 Resilience, intolerance of uncertainty, and CF patients’ quality of life

Journal of Cystic Fibrosis, 2015

Objectives In several large epidemiological studies CF patients report equal levels of anxiety an... more Objectives In several large epidemiological studies CF patients report equal levels of anxiety and depression compared to healthy controls. They seem to cope particularly well considering their uncertainty and restrictions in living. (1) "Resilience" (the "bouncing back from adversity") and (2) "Intolerance of uncertainty" (IU, a vulnerability factor) have been investigated in their predictive power on CF quality of life. Research on resilience and IU in CF is hardly existent yet. Methods Adult patients treated at the CF centre in Innsbruck (n = 57) were assessed at their routine medical check-up. Resilience (assessed with the Resilience Scale – RS; Wagnild & Young, 1993) and IU (assessed with the Intolerance of Uncertainty Scale – IUS; Freeston et al., 1994) were explored as predictors on CF quality of life (assessed with the CFQ-R) in multiple regression analyses. Results CF patients had comparably excellent medical status and reported significantly higher levels of resilience compared to healthy controls, but equal levels of IU. The constructs explained up to 54% of variance in CFQ-R subscales. None of the IUS subscales retained their predictive power when resilience was included in the regression model and lung function was controlled for. Of the two RS subscales "personal competence" had strong predictive power whereas "acceptance" with its purported beneficial effect had a substantial number of negative associations with QoL. Conclusions Data support the notion that the majority of CF patients in our sample rather successfully regulate than suppress negative emotions. Perceived personal coping competence has been confirmed as a major focus for intervention.

Research paper thumbnail of 311 Do quality of life responses signal severe loss of lung function?

Journal of Cystic Fibrosis, 2013

Objective: To investigate an alternative method of intravenous (IV) antibiotic desensitisation in... more Objective: To investigate an alternative method of intravenous (IV) antibiotic desensitisation in patients with cystic fibrosis (CF). The use of IV antibiotics is a central part of the management of pulmonary infections in CF. Allergic reactions to antibiotics are more common in patients with CF in part due to repeated exposure to high doses [1]. Desensitisation, an established procedure in CF allows use of these antibiotics in all but severe allergic reaction through induction of temporary immune tolerance. Desensitisation involves a 7 step administration of incremental doses of antibiotic. Doses are made by our pharmacy department, require 24 hours notice for production and the service is costly. Method: We developed a protocol to use a volumetric infusion device (VID) to administer established 7 step desensitisation regimens. Desensitisations using the VID were prepared and administered by a clinical nurse specialist. Results: Over 5 months 9 patients received IV antibiotic desensitisation using a VID. This was successful in all but 1 patient. The flow rate for Step 7 of the original desensitisation regimen was reduced as this flow rate could not be administered in patients with TIVADs. The use of a VID allowed greater flexibility in the timing and frequency of desensitisation. There was no delay in treatment for patients requiring desensitisation when the VID was used. Conclusion: A 7 step IV antibiotic desensitization programme using a VID is well tolerated, safe and cost effective. The use of a VID can reduce delay in patients with drug allergies receiving IV antibiotics.

Research paper thumbnail of Malignancies in patients with cystic fibrosis: a case series

Journal of Medical Case Reports

Background Previous reports have shown an increased number of colorectal cancers in patients with... more Background Previous reports have shown an increased number of colorectal cancers in patients with cystic fibrosis. We assessed the database of our cystic fibrosis center to identify patients with all kinds of cancer retrospectively. All patients visiting the Cystic Fibrosis Centre Innsbruck between 1995 and 2019 were included. Case presentation Among 229 patients with cystic fibrosis treated at the Cystic Fibrosis Centre in Innsbruck between 1995 and 2019, 11 subjects were diagnosed with a malignant disease. The median age at diagnosis was 25.2 years (mean 24.3 years). There were four gynecological malignancies (cervical intraepithelial neoplasia and cervical cancer), two hematological malignancies (acute lymphocytic leukemia), one gastrointestinal malignancy (peritoneal mesothelioma), and four malignancies from other origins (malignant melanoma, neuroblastoma, adrenocortical carcinoma, and thyroid cancer). One malignancy occurred after lung transplantation. There was a strong prepo...

Research paper thumbnail of Cystic Fibrosis Newborn Screening in Austria Using PAP and the Numeric Product of PAP and IRT Concentrations as Second-Tier Parameters

Diagnostics

In Austria, newborns have been screened for cystic fibrosis (CF) by analyzing immunoreactive tryp... more In Austria, newborns have been screened for cystic fibrosis (CF) by analyzing immunoreactive trypsinogen (IRT) from dried blood spots (DBS)s for nearly 20 years. Recently, pancreatitis-associated protein (PAP) analysis was introduced as a second-tier test with the aim of reducing recalls for second DBS cards while keeping sensitivity high. For 28 months, when IRT was elevated (65–130 ng/mL), PAP was measured from the first DBS (n = 198,927) with a two-step cut-off applied. For the last 12 months of the observation period (n = 85,421), an additional IRT×PAP cut-off was introduced. If PAP or IRT×PAP were above cut-off, a second card was analyzed for IRT and in case of elevated values identified as screen-positive. Above 130 ng/mL IRT in the first DBS, newborns were classified as screen-positive. IRT analysis of first DBS resulted in 1961 (1%) tests for PAP. In the first 16 months, 26 of 93 screen-positive were confirmed to have CF. Two false-negatives have been reported (sensitivity =...

Research paper thumbnail of Reference Values Matter: Fewer Patients With Malnutrition Using American Compared to More Recent German Growth Charts

Journal of Pediatric Gastroenterology & Nutrition

Research paper thumbnail of Antiviral Drugs from the Nucleoside Analog Family Block Volume-Activated Chloride Channels

Research paper thumbnail of Amikacin containing self-emulsifying delivery systems via pulmonary administration for treatment of bacterial infections of cystic fibrosis patients

Nanomedicine (London, England), Apr 28, 2018

The aim of the study was to develop self-emulsifying delivery systems (SEDDS) exhibiting improved... more The aim of the study was to develop self-emulsifying delivery systems (SEDDS) exhibiting improved permeation rate for pulmonary delivery of amikacin for treatment of cystic fibrosis (CF) patients. Solubility of amikacin in lipids was improved by hydrophobic ion pairing with sodium myristyl sulfate. The complex was loaded into SEDDS. Drug-release studies were performed and the permeation properties of SEDDS through human CF mucus were examined. A total of 10% complex could be loaded into SEDDS. SEDDS exhibited sustained release. Up to twofold more amounts of amikacin permeated through the CF mucus compared with reference. The developed SEDDS with amikacin may be a promising tool for the treatment of certain bacterial infections of CF patients.

Research paper thumbnail of Faecal Calprotectin in Cystic Fibrosis and ITʼS Relation to Disease Parameters – a Longitudinal Analysis Over 12 Years

Journal of Pediatric Gastroenterology and Nutrition, 2017

Faecal calprotectin (FC) is a marker of inflammation in the intestinal tract. We assessed FC leve... more Faecal calprotectin (FC) is a marker of inflammation in the intestinal tract. We assessed FC levels longitudinally in patients with cystic fibrosis (CF) and evaluated the relation between FC results and relevant markers of disease. Calprotectin was measured in faecal samples starting in 2003 and values were stored in the centre's patient database. In this retrospective analysis we searched for associations of FC concentrations with disease severity and progression. Linear mixed effects models were used to model the logarithm of FC levels. A total of 171 patients (0-61 years) had 2434 FC measurements between 2003 and 2015, with a total observation period of 1686 patient years. Median (IQR) FC concentrations were 60.9 (75.9) μg/g and 61% of the samples showed elevated FC concentrations (>50 μg/g). Despite some statistically significant effects, there was no clinically relevant association between FC and sex, age, FEV1 z-score, or BMI z-score. Pancreatic insufficiency (i.e. faecal elastase < 100 μg/g stool) was associated with considerably higher FC values compared to normal pancreatic function (median FC 68 vs. 29 μg/g, p < 0.0001). F508del homozygous subjects showed a trend to higher FC values than heterozygous patients (median 71 vs. 62 μg/g, p = 0.173). In addition, a significant association with increasing serum CRP concentrations (p < 0.0001) was observed. FC was elevated in two thirds of stool specimens. Increased FC was more common in patients with pancreatic insufficiency. Whether increased faecal calprotectin reflects intestinal inflammation in patients with cystic fibrosis remains to be determined.

Research paper thumbnail of Arm muscle area for the longitudinal assessment of nutritional status in paediatric patients with cystic fibrosis - A single centre experience

Journal of Cystic Fibrosis, 2021

Background: In cystic fibrosis, adequate nutrition contributes to good long-term prognosis. A bod... more Background: In cystic fibrosis, adequate nutrition contributes to good long-term prognosis. A body mass index (BMI) at or above the 50th percentile for age and sex in all children has been recommended. As researchers have described a depletion of fat-free mass despite normal BMI, longitudinal studies using more sensitive nutritional parameters are warranted. We evaluated anthropometric measurements in an attempt to identify early indicators of deteriorating nutritional status in our paediatric cohort. Methods: We analysed datasets from children and adolescents between 2 and 17.9 years with at least two entries for triceps skinfold thickness and upper arm circumference in our patient database between January 1995 and December 2018. Arm muscle area (AMA) was calculated, and all values were expressed as z-scores from CDC growth charts. Results: A total of 4,862 encounters from 161 paediatric patients (78 girls) were available, representing a median number of 28 visits during a median follow-up of 8.1 years per patient. Linear mixed effects models revealed relatively stable courses for weight, height, BMI and skinfold thickness up to adulthood. AMA was the only parameter which declined slightly (r =-0.036), particularly in boys. Kaplan-Meieranalyses showed that AMA was the earliest parameter to decrease below-1 z-score between 6 and 18 years. Conclusions: The present data suggest that compared with weight or BMI, AMA could serve as an earlier indicator of a deteriorating nutritional status. The benefit of assessing skinfold thickness and arm circumference routinely and calculating AMA from these measurements should be evaluated in large, prospective, multi-centre studies.

Research paper thumbnail of REAL‐world clinical effectiveness of ivacaftor therapy in the first 24 months in two infants with cystic fibrosis and different gating mutations—A case report

Clinical Case Reports, 2022

This study summarizes efficacy of ivacaftor treatment in 2 infants in a real‐world setting. A dis... more This study summarizes efficacy of ivacaftor treatment in 2 infants in a real‐world setting. A distinct decline of sweat chloride and lung clearance index plus increase in fecal elastase was seen. The results underline the early and sustainable effect and give cause for discussing whether a reduction in standard cystic fibrosis therapy is possible.

Research paper thumbnail of Lung clearance index to detect the efficacy of Aztreonam lysine inhalation in patients with cystic fibrosis and near normal spirometry – A single-centre feasibility study

PLOS ONE, 2019

Comparing the efficacy of inhaled antibiotics can be difficult in small groups of patients with c... more Comparing the efficacy of inhaled antibiotics can be difficult in small groups of patients with cystic fibrosis and mild lung disease. In a feasibility study we compared Aztreonam lysine for inhalation solution (AZLI; Cayston ®) to standard inhaled antibiotic therapy in patients with cystic fibrosis and near normal spirometry. To detect treatment responses we used both lung clearance index (LCI) and forced expiratory volume in one second (FEV 1). At baseline, median FEV 1 was 87% pred. and median LCI was 8.6 (upper limit of normal: 7.0). After 4 weeks, LCI improved by-0.36 after AZLI and deteriorated by +0.12 after tobramycin treatment (p = 0.039). No significant differences between treatments (p = 0.195) were observed using FEV 1. These results suggest that lung clearance index can be used to detect treatment induced changes in subjects with mild lung disease.

Research paper thumbnail of Care in an ISO Certified Centre for Patients With Cystic Fibrosis and Their Families

Die Mukoviszidose (CF) als Störung exokriner Drüsen betrifft verschiedene Organsysteme. Hauptmani... more Die Mukoviszidose (CF) als Störung exokriner Drüsen betrifft verschiedene Organsysteme. Hauptmanifestationen sind die chronisch obstruktive Lungenerkrankung und die Pankreasinsuffizienz. Bei vielen Patienten kommen diverse andere Probleme hinzu, beispielsweise Nasenpolypen, Hepatopathie, CF-Diabetes, Unterernährung, Infertilität (bei den meisten Männern) oder Osteoporose. Während vor 60 Jahren nach der Erstbeschreibung der Erkrankung die meisten BeZusammenfassung !

Research paper thumbnail of Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations

Journal of Cystic Fibrosis

Research paper thumbnail of Faecal calprotectin concentrations in young children with cystic fibrosis

Journal of Cystic Fibrosis, 2018

Research paper thumbnail of P271 Malignant diseases in patients treated at the CF Centre Innsbruck

Journal of Cystic Fibrosis

Research paper thumbnail of P399 High utilisation of an ongoing group education programme for parents as part of routine care

Journal of Cystic Fibrosis

Research paper thumbnail of P452 Predicting psychological well-being and symptoms in adults living with cystic fibrosis: the role of self-compassion and psychological resilience

Journal of Cystic Fibrosis

Research paper thumbnail of S2-Konsensus-Leitlinie „Diagnose der Mukoviszidose“

Research paper thumbnail of Long-term improvement of lung clearance index in patients with mild cystic fibrosis lung disease: Does hypertonic saline play a role?

Journal of Cystic Fibrosis, 2015

To assess whether long-term inhalation with hypertonic saline is able to halt the progression of ... more To assess whether long-term inhalation with hypertonic saline is able to halt the progression of mild CF lung disease, we analysed longitudinal data of lung clearance index (LCI) and spirometry. A total of 34 patients with mild lung disease (FEV 1 ≥ 70% of predicted) had at least one LCI result before and ≥ 2 LCI measurements after start of hypertonic saline (HS) therapy. After a mean follow-up of 39.7 (SD 7.4) months after starting HS, LCI improved significantly from 7.89 (SD 1.35) at baseline to 6.96 (SD 1.03), and 19/34 patients had a normal LCI value at the last measurement. No decrease in mean FEV 1 was observed. Thus, ventilation inhomogeneity can improve in patients with mild lung disease.

Research paper thumbnail of WS09.4 The role of resilience in quality of life of adult patients with cystic fibrosis (CF)

Journal of Cystic Fibrosis, 2015

Objectives Although several psychological studies on coping and quality of life suggested high le... more Objectives Although several psychological studies on coping and quality of life suggested high levels of resilience in CF patients, empirical data on resilience in CF are barely existent. Thus, the purpose of the study was to investigate resilience in adult CF patients and its impact on quality of life. Methods 57 adult CF patients (18–59 years) participated in the study at their routine medical check-up. In addition to their regular psychological assessment including the Cystic Fibrosis Questionnaire – Revised (CFQ-R), the Resilience Scale (RS) was administered. The constructs were also correlated with physical parameters (age, gender and lung function). Results Patients reported high levels of resilience and overall good quality of life in all domains. Patient's resilience was significantly higher compared to the large healthy reference group (t[2086] = 4.7510, p=0.0001) and was independent of gender (t[55] = 0.826, p=0.412), age (r = 0.076, p=0.573) and lung function (FEV1%, r=0.165, p=0.220). Significant associations between resilience and CFQ-R domains could be confirmed. Conclusion Our study is one of the first attempts to explicitely test resilience in CF patients and could demonstrate high levels of resilience in these patient group and positive correlations with quality of life. These results suggest that resilience could be seen as an important factor on quality of life and coping with CF. Therefore fostering resilience early in patient's life should be encouraged.

Research paper thumbnail of 288 Resilience, intolerance of uncertainty, and CF patients’ quality of life

Journal of Cystic Fibrosis, 2015

Objectives In several large epidemiological studies CF patients report equal levels of anxiety an... more Objectives In several large epidemiological studies CF patients report equal levels of anxiety and depression compared to healthy controls. They seem to cope particularly well considering their uncertainty and restrictions in living. (1) "Resilience" (the "bouncing back from adversity") and (2) "Intolerance of uncertainty" (IU, a vulnerability factor) have been investigated in their predictive power on CF quality of life. Research on resilience and IU in CF is hardly existent yet. Methods Adult patients treated at the CF centre in Innsbruck (n = 57) were assessed at their routine medical check-up. Resilience (assessed with the Resilience Scale – RS; Wagnild & Young, 1993) and IU (assessed with the Intolerance of Uncertainty Scale – IUS; Freeston et al., 1994) were explored as predictors on CF quality of life (assessed with the CFQ-R) in multiple regression analyses. Results CF patients had comparably excellent medical status and reported significantly higher levels of resilience compared to healthy controls, but equal levels of IU. The constructs explained up to 54% of variance in CFQ-R subscales. None of the IUS subscales retained their predictive power when resilience was included in the regression model and lung function was controlled for. Of the two RS subscales "personal competence" had strong predictive power whereas "acceptance" with its purported beneficial effect had a substantial number of negative associations with QoL. Conclusions Data support the notion that the majority of CF patients in our sample rather successfully regulate than suppress negative emotions. Perceived personal coping competence has been confirmed as a major focus for intervention.

Research paper thumbnail of 311 Do quality of life responses signal severe loss of lung function?

Journal of Cystic Fibrosis, 2013

Objective: To investigate an alternative method of intravenous (IV) antibiotic desensitisation in... more Objective: To investigate an alternative method of intravenous (IV) antibiotic desensitisation in patients with cystic fibrosis (CF). The use of IV antibiotics is a central part of the management of pulmonary infections in CF. Allergic reactions to antibiotics are more common in patients with CF in part due to repeated exposure to high doses [1]. Desensitisation, an established procedure in CF allows use of these antibiotics in all but severe allergic reaction through induction of temporary immune tolerance. Desensitisation involves a 7 step administration of incremental doses of antibiotic. Doses are made by our pharmacy department, require 24 hours notice for production and the service is costly. Method: We developed a protocol to use a volumetric infusion device (VID) to administer established 7 step desensitisation regimens. Desensitisations using the VID were prepared and administered by a clinical nurse specialist. Results: Over 5 months 9 patients received IV antibiotic desensitisation using a VID. This was successful in all but 1 patient. The flow rate for Step 7 of the original desensitisation regimen was reduced as this flow rate could not be administered in patients with TIVADs. The use of a VID allowed greater flexibility in the timing and frequency of desensitisation. There was no delay in treatment for patients requiring desensitisation when the VID was used. Conclusion: A 7 step IV antibiotic desensitization programme using a VID is well tolerated, safe and cost effective. The use of a VID can reduce delay in patients with drug allergies receiving IV antibiotics.

Research paper thumbnail of Malignancies in patients with cystic fibrosis: a case series

Journal of Medical Case Reports

Background Previous reports have shown an increased number of colorectal cancers in patients with... more Background Previous reports have shown an increased number of colorectal cancers in patients with cystic fibrosis. We assessed the database of our cystic fibrosis center to identify patients with all kinds of cancer retrospectively. All patients visiting the Cystic Fibrosis Centre Innsbruck between 1995 and 2019 were included. Case presentation Among 229 patients with cystic fibrosis treated at the Cystic Fibrosis Centre in Innsbruck between 1995 and 2019, 11 subjects were diagnosed with a malignant disease. The median age at diagnosis was 25.2 years (mean 24.3 years). There were four gynecological malignancies (cervical intraepithelial neoplasia and cervical cancer), two hematological malignancies (acute lymphocytic leukemia), one gastrointestinal malignancy (peritoneal mesothelioma), and four malignancies from other origins (malignant melanoma, neuroblastoma, adrenocortical carcinoma, and thyroid cancer). One malignancy occurred after lung transplantation. There was a strong prepo...

Research paper thumbnail of Cystic Fibrosis Newborn Screening in Austria Using PAP and the Numeric Product of PAP and IRT Concentrations as Second-Tier Parameters

Diagnostics

In Austria, newborns have been screened for cystic fibrosis (CF) by analyzing immunoreactive tryp... more In Austria, newborns have been screened for cystic fibrosis (CF) by analyzing immunoreactive trypsinogen (IRT) from dried blood spots (DBS)s for nearly 20 years. Recently, pancreatitis-associated protein (PAP) analysis was introduced as a second-tier test with the aim of reducing recalls for second DBS cards while keeping sensitivity high. For 28 months, when IRT was elevated (65–130 ng/mL), PAP was measured from the first DBS (n = 198,927) with a two-step cut-off applied. For the last 12 months of the observation period (n = 85,421), an additional IRT×PAP cut-off was introduced. If PAP or IRT×PAP were above cut-off, a second card was analyzed for IRT and in case of elevated values identified as screen-positive. Above 130 ng/mL IRT in the first DBS, newborns were classified as screen-positive. IRT analysis of first DBS resulted in 1961 (1%) tests for PAP. In the first 16 months, 26 of 93 screen-positive were confirmed to have CF. Two false-negatives have been reported (sensitivity =...

Research paper thumbnail of Reference Values Matter: Fewer Patients With Malnutrition Using American Compared to More Recent German Growth Charts

Journal of Pediatric Gastroenterology & Nutrition

Research paper thumbnail of Antiviral Drugs from the Nucleoside Analog Family Block Volume-Activated Chloride Channels

Research paper thumbnail of Amikacin containing self-emulsifying delivery systems via pulmonary administration for treatment of bacterial infections of cystic fibrosis patients

Nanomedicine (London, England), Apr 28, 2018

The aim of the study was to develop self-emulsifying delivery systems (SEDDS) exhibiting improved... more The aim of the study was to develop self-emulsifying delivery systems (SEDDS) exhibiting improved permeation rate for pulmonary delivery of amikacin for treatment of cystic fibrosis (CF) patients. Solubility of amikacin in lipids was improved by hydrophobic ion pairing with sodium myristyl sulfate. The complex was loaded into SEDDS. Drug-release studies were performed and the permeation properties of SEDDS through human CF mucus were examined. A total of 10% complex could be loaded into SEDDS. SEDDS exhibited sustained release. Up to twofold more amounts of amikacin permeated through the CF mucus compared with reference. The developed SEDDS with amikacin may be a promising tool for the treatment of certain bacterial infections of CF patients.

Research paper thumbnail of Faecal Calprotectin in Cystic Fibrosis and ITʼS Relation to Disease Parameters – a Longitudinal Analysis Over 12 Years

Journal of Pediatric Gastroenterology and Nutrition, 2017

Faecal calprotectin (FC) is a marker of inflammation in the intestinal tract. We assessed FC leve... more Faecal calprotectin (FC) is a marker of inflammation in the intestinal tract. We assessed FC levels longitudinally in patients with cystic fibrosis (CF) and evaluated the relation between FC results and relevant markers of disease. Calprotectin was measured in faecal samples starting in 2003 and values were stored in the centre's patient database. In this retrospective analysis we searched for associations of FC concentrations with disease severity and progression. Linear mixed effects models were used to model the logarithm of FC levels. A total of 171 patients (0-61 years) had 2434 FC measurements between 2003 and 2015, with a total observation period of 1686 patient years. Median (IQR) FC concentrations were 60.9 (75.9) μg/g and 61% of the samples showed elevated FC concentrations (>50 μg/g). Despite some statistically significant effects, there was no clinically relevant association between FC and sex, age, FEV1 z-score, or BMI z-score. Pancreatic insufficiency (i.e. faecal elastase < 100 μg/g stool) was associated with considerably higher FC values compared to normal pancreatic function (median FC 68 vs. 29 μg/g, p < 0.0001). F508del homozygous subjects showed a trend to higher FC values than heterozygous patients (median 71 vs. 62 μg/g, p = 0.173). In addition, a significant association with increasing serum CRP concentrations (p < 0.0001) was observed. FC was elevated in two thirds of stool specimens. Increased FC was more common in patients with pancreatic insufficiency. Whether increased faecal calprotectin reflects intestinal inflammation in patients with cystic fibrosis remains to be determined.