Qualitative evaluation of the Home Therapy administration of enzymatic treatment for patients with...
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Introduction Introduction Although the disposable literature is quiet scarce, home enzyme administration for lysosomal diseases has demonstrated to improve not only patient’s quality of life but also for their families. However, in Spain the administration of enzymes for lysosomal diseases use to be at the hospital, although in the majority of regions hospital-depending at-home programs for the administration of drugs are an increasing strategy to improve the quality of the assistance. To analyze the opinion of all the participants in a national Program of Home Enzymatic Therapy Administration (HETA) of velaglucerase alpha and agalsidase alpha at home, a program characterized by the possibility of an individualized flexible enzyme adminitsration time. Bibliography Bibliography Objective Objective Methodology Methodology HETA Protocol See poster #108. Inclusion criteria Patients treated with agalsidase alpha and velaglucerase alpha included in the HETA program in the Comunidad Valenciana, a Spanish region in the southeast coast. Opinion survey In July 2015-April 2016 four patients with type 1 Gaucher disease (GD) and six with Fabry disease (FD) in the hospitals of Alzira and Alcoy (Comunidad Valenciana, Spain) have received enzymatic treatment at home by an specifically trained team independent of the hospital (Caregiving España © nurses, CGn). One year after the beginning a qualitative survey was answered by patients , hospital pharmacists ( Hp ) , hospital nurses (Hn) and CG nurses ( CGn ) . A 0 to 10 scale (0 as the worst opinion. 10 as the best opinion) was applied to express the personal opinion about global evaluation as well as for comfort, security, confidence, and flexibility. Results Results i. Hughes DA, Milligan A, Mehta A. Home therapy for lysosomal storage disorders. Br J Nutr. 2007; 16 (22):1386-9. ii. Elstein D, Burrow TA, Charrow J, Giraldo P, Mehta A Pastores GM, Lee HM, Mellgard B. Zimran A. Home infusion of intravenous velaglucerase alfa: Experience from pooled clinical studies in 104 patients with type 1 Gaucher disease. Mol Genet Metab. 2016 Aug 23. pii: S1096-7192(16)30194-9. doi: 10.1016/j.ymgme.2016.08.005. [Epub ahead of print]. iii.Smid BE, Hoogendijk SL, Wijburg FA, Hollak CE, Linthorst GE. A revised home treatment algorithm for Fabry disease: influence of antibody formation. Mol Genet Metab. 2013;108 (2): 132-7. iv. Guest JF Concolino D, Di Vito R, Feliciani C, Parini R, Zampetti A. Modelling the resource implications of managing adults with Fabry disease in Italy. Eur J Clin Invest. 2 011; 41 (7): 710-8. The HETA program has been very well considered by all the participants, especially patients, although also professionals. Some doubts exist about security, mainly from CG nursery. Opinions among hospital professionals are very heterogeneous although with a general very positive evaluation. Limitations of the study is the small number of participating patients as well as the use of a no validated scale for evaluation. Actually an extension of the present study is being done including all the 30 included patients in Spain using a validated scale to compare with the actually applied. General characteristics of the sample Sample: Four type 1 Gaucher disease patients and six Fabry disease patients representing 30 and 40% patients for each disease included in the Spanish national HETA program. Observational period: July 2015-April 2016. 135 home enzyme administrations: 88 agalsidase-α. 47 velaglucerase-α. Distance from home to the hospital (Median and range) was 6.5 (0.3-24) Km, with a temporal distance of 22 (10-45) minutes. Full therapeutical calendar and in-hospital ambulatory controls compliance were observed. 100% persistence in the program by the patients. No side effects were reported as a whole nor infusion-related. All the disease parameters were stable. Survey results by participating collectives (0: The worst opinion, 10: the best opinion). Conclusions Conclusions The HETA program is supported by Shire Pharmaceuticals Iberica © . The actual study, however is independt of this compnay.. Communicated as a poster Internal Medicine Department Rare Diseases Unit Alcoi (Alicante). Spain.
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Transcript of Qualitative evaluation of the Home Therapy administration of enzymatic treatment for patients with...
IntroductionIntroductionAlthough the disposable literature is quiet scarce, home enzyme administration for lysosomal diseases has demonstrated to improve not only patient’s quality of life but also for their families. However, in Spain the administration of enzymes for lysosomal diseases use to be at the hospital, although in the majority of regions hospital-depending at-home programs for the administration of drugs are an increasing strategy to improve the quality of the assistance.
To analyze the opinion of all the participants in a national Program of Home Enzymatic Therapy Administration (HETA) of velaglucerase alpha and agalsidase alpha at home, a program characterized by the possibility of an individualized flexible enzyme adminitsration time.
BibliographyBibliography
ObjectiveObjective
MethodologyMethodology
HETA ProtocolSee poster #108.
Inclusion criteriaPatients treated with agalsidase alpha and velaglucerase alpha included in the HETA program in the Comunidad Valenciana, a Spanish region in the southeast coast.
Opinion surveyIn July 2015-April 2016 four patients with type 1 Gaucher disease (GD) and six with Fabry disease (FD) in the hospitals of Alzira and Alcoy (Comunidad Valenciana, Spain) have received enzymatic treatment at home by an specifically trained team independent of the hospital (Caregiving España© nurses, CGn).
One year after the beginning a qualitative survey was answered by patients, hospital pharmacists (Hp), hospital nurses (Hn) and CG nurses (CGn). A 0 to 10 scale (0 as the worst opinion. 10 as the best opinion) was applied to express the personal opinion about global evaluation as well as for comfort, security, confidence, and flexibility.
ResultsResults
i. Hughes DA, Milligan A, Mehta A. Home therapy for lysosomal storage disorders. Br J Nutr. 2007; 16 (22):1386-9.
ii. Elstein D, Burrow TA, Charrow J, Giraldo P, Mehta A Pastores GM, Lee HM, Mellgard B. Zimran A. Home infusion of intravenous velaglucerase alfa: Experience from pooled clinical studies in 104 patients with type 1 Gaucher disease. Mol Genet Metab. 2016 Aug 23. pii: S1096-7192(16)30194-9. doi: 10.1016/j.ymgme.2016.08.005. [Epub ahead of print].
iii. Smid BE, Hoogendijk SL, Wijburg FA, Hollak CE, Linthorst GE. A revised home treatment algorithm for Fabry disease: influence of antibody formation. Mol Genet Metab. 2013;108 (2): 132-7.
iv. Guest JF Concolino D, Di Vito R, Feliciani C, Parini R, Zampetti A. Modelling the resource implications of managing adults with Fabry disease in Italy. Eur J Clin Invest. 2 011; 41 (7): 710-8.
The HETA program has been very well considered by all the participants, especially patients, although also professionals.
Some doubts exist about security, mainly from CG nursery.
Opinions among hospital professionals are very heterogeneous although with a general very positive evaluation.
Limitations of the study is the small number of participating patients as well as the use of a no validated scale for evaluation.
Actually an extension of the present study is being done including all the 30 included patients in Spain using a validated scale to compare with the actually applied.
General characteristics of the sample
Sample: Four type 1 Gaucher disease patients and six Fabry disease patients representing 30 and 40% patients for each disease included in the Spanish national HETA program.
Observational period: July 2015-April 2016.
135 home enzyme administrations:
88 agalsidase-α.47 velaglucerase-α.
Distance from home to the hospital (Median and range) was 6.5 (0.3-24) Km, with a temporal distance of 22 (10-45) minutes.
Full therapeutical calendar and in-hospital ambulatory controls compliance were observed.
100% persistence in the program by the patients.
No side effects were reported as a whole nor infusion-related.
All the disease parameters were stable.
Survey results by participating collectives (0: The worst opinion, 10: the best opinion).
ConclusionsConclusions
The HETA program is supported by Shire Pharmaceuticals Iberica©.The actual study, however is independt of this compnay.. Communicated as a poster
Internal Medicine DepartmentRare Diseases UnitAlcoi (Alicante). Spain.
