Effects of a Comprehensive Patient-centered Health Service in Cystic Fibrosis (VEMSE-CF) (VEMSE-CF)

Effects of a Comprehensive Patient-centered Health Service With a Focus on Psychosocial Services for Patients With Rare Diseases Using Cystic Fibrosis as an Example (Evaluation Eines Ganzheitlichen Patientenzentrierten Versorgungsmodells für Patienten Mit Seltenen Erkrankungen Unter Besonderer Berücksichtigung Der Psychosozialen Versorgung am Beispiel Mukoviszidose)

For the Project VEMSE-CF, a comprehensive patient-centered outpatient health service model was developed offering focused interventions in different areas. These included patient education, as well as nutritional and exercise counselling. Special emphasis was given to the psycho-social services. The implementation was supported by a case manager. The model was implemented in three German CF-Centers. For evaluation, Patient data from 13 additional German CF-Centers offering standard care was used as comparison. In total, 153 patients in the Intervention Group and 163 control patients aged 5-52 years contributed data. The primary endpoint was the number of BMI- and FEV1-measurements over 24 months. Secondary endpoints included mortality, lung transplantation, FEV1, BMI, adherence to therapy, Quality of life, and mental stress.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Behavioral: comprehensive patient-centered outpatient health service with multiple components

• Study Type: Interventional
• Enrollment (Actual): 318
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Germany
  • Bonn, Germany, 53117
    • Mukoviszidose Institut

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• confirmed diagnosis of Cystic Fibrosis - written informed consent
• intervention group: care in one of the 3 CF-centers
• control group: care in one of the 13 control CF-centers

Exclusion Criteria:

• post lung Transplantation
• listed for lung Transplantation and planned inpatient stay for more than 4 weeks

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Health Services Research
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
No Intervention: Control; Standard care
Experimental: Intervention; comprehensive patient-centered outpatient health service with multiple components. These components included the addition of a case-manager, structured interventions to improve patient education and adherence to therapy, psychological counselling, social services, and exercise advice. For each Patient, relevant components were identified and a written Intervention plan was negotiated and signed.	Behavioral: comprehensive patient-centered outpatient health service with multiple components; Individualized multi-component education and counselling program coordinated by a case Manager.; Other Names: Intervention

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of BMI and FEV1 assessments	crude number of assessments (BMI and FEV1) performed during the study period	24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mortality	Number of patients deceased during the study period	24 months
Referrals for Lung Transplantation	number of patients referred for lung Transplantation during the study	24 months
Delta FEV1	Change in FEV1 from baseline to end of study	24 months
Delta BMI	Change in BMI from baseline to end of study	24 months
Outpatient visits	Number of outpatient visits during study period	24 months
Hospitalizations	number of hospitalizations during the study period	24 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Delta Proportion of patients with subnormal FEV1	Change in the Proportion of patients with an FEV1<80% predicted	24 months
Delta Proportion of Patients with well preserved BMI	Change in the Proportion of patients with a BMI equal or greater 22 kg/m2 (females) or 23 kg/m2 (males)	24 months
Adherence to therapy	Performance of planned assessments (four per year; eight during intervention)	24 months
Quality of Life (patients)	Self- or proxy-Report of patients´ Quality of Life: questionnaire - EQ5D (adults) / EQ5D - Y (13-18) / CFQ-R-6-12; three assessments during intervention (begin / after 12 month /after 24 month)	24 months
Quality of Life (parents)	self-report of parents´ Quality of Life; questionnaire - EY5D; three assessments during intervention(begin / after 12 month /after 24 month)	24 months
Emotional stress (Patients)	Self- or proxy-Report of patients´ emotional stress; questionnaire - Hospital Anxiety and Depression Scale (HADS); three assessments during Intervention (begin / after 12 month /after 24 month)	24 months

Collaborators and Investigators

• Sponsor: Mukoviszidose Institut gGmbH
• Collaborators: Hannover Medical School; Goethe University; Federal Ministry of Health, Germany; Wuerzburg University Hospital; University Hospital Ulm; CF-Center Hamburg-Altona; Medical Association of Lower Saxony (Ärztekammer Niedersachsen), Germany
• Investigators: Study Chair: Uta Düesberg, Dr., Mukoviszidose Institut gGmbH; Study Director: Miriam Schlangen, Dr., Mukoviszidose Institut gGmbH; Principal Investigator: Lutz Goldbeck, Prof. Dr., Ulm University Hospital; Principal Investigator: Helge Hebestreit, Prof. Dr., Wuerzburg University Hospital

Publications and helpful links

General Publications

• Kerem E, Conway S, Elborn S, Heijerman H; Consensus Committee. Standards of care for patients with cystic fibrosis: a European consensus. J Cyst Fibros. 2005 Mar;4(1):7-26. doi: 10.1016/j.jcf.2004.12.002. No abstract available.
• Besier T, Born A, Henrich G, Hinz A, Quittner AL, Goldbeck L; TIDES Study Group. Anxiety, depression, and life satisfaction in parents caring for children with cystic fibrosis. Pediatr Pulmonol. 2011 Jul;46(7):672-82. doi: 10.1002/ppul.21423. Epub 2011 Mar 7.
• Goldbeck L, Besier T, Hinz A, Singer S, Quittner AL; TIDES Group. Prevalence of symptoms of anxiety and depression in German patients with cystic fibrosis. Chest. 2010 Oct;138(4):929-36. doi: 10.1378/chest.09-2940. Epub 2010 May 14.
• Ravens-Sieberer U, Otto C, Kriston L, Rothenberger A, Dopfner M, Herpertz-Dahlmann B, Barkmann C, Schon G, Holling H, Schulte-Markwort M, Klasen F; BELLA study group. The longitudinal BELLA study: design, methods and first results on the course of mental health problems. Eur Child Adolesc Psychiatry. 2015 Jun;24(6):651-63. doi: 10.1007/s00787-014-0638-4. Epub 2014 Nov 27.
• Ruf K, Winkler B, Hebestreit A, Gruber W, Hebestreit H. Risks associated with exercise testing and sports participation in cystic fibrosis. J Cyst Fibros. 2010 Sep;9(5):339-45. doi: 10.1016/j.jcf.2010.05.006. Epub 2010 Jul 2.
• Sens B, Stern M. (2012) (Hrsg).Qualitätssicherung Mukoviszidose. Überblick über den Gesundheitszustand der Patienten in Deutschland 2012, Bonn/Hannover
• Graf von der Schulenburg JM, Greiner W, Jost F, Klusen N, Kubin M, Leidl R, Mittendorf T, Rebscher H, Schoeffski O, Vauth C, Volmer T, Wahler S, Wasem J, Weber C; Hanover Consensus Group. German recommendations on health economic evaluation: third and updated version of the Hanover Consensus. Value Health. 2008 Jul-Aug;11(4):539-44. doi: 10.1111/j.1524-4733.2007.00301.x. Epub 2008 Jan 11. No abstract available.

Study record dates

Study Major Dates

• Study Start (Actual): August 1, 2011
• Primary Completion (Actual): February 28, 2016
• Study Completion (Actual): December 31, 2016

Study Registration Dates

• First Submitted: January 23, 2018
• First Submitted That Met QC Criteria: January 30, 2018
• First Posted (Actual): February 6, 2018

Study Record Updates

• Last Update Posted (Actual): February 19, 2018
• Last Update Submitted That Met QC Criteria: February 15, 2018
• Last Verified: February 1, 2018

More Information

Terms related to this study

• Keywords: quality of life; BMI; FEV1; health services; mental stress
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: VEMSE-CF

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No