Primera experiencia nacional española de admjnistración domiciliaria de velagluceras en Gaucher.

1. 106. Home enzymatic therapy administration for
Gaucher disease in Spain. First national experience.
*Giner V1
, Barba M2
, Cano H15
, Fernández MA4
, Mora C5
, Dupla6
, Luaña A7
, Solanich X8
, Balanzat J9
, Elisa Luño10
, Perpiñá J11
, Sanz J1
.
1
Rare Diseases Unit. Department of General Internal Medicine. Hospital Mare de Déu dels Lliris. Alcoy (Alicante). 2
National Medical Coordinator. Caregiving Ibérica SL. Madrid. 3
Department of Hematology. Hospital Los
Arcos del Mar Menor. San Javier (Murcia). 4
Department of Hematology. Hospital de Plasencia. Plasencia (Cáceres). 5
Department of Hematology. Hospital Mare de Déu dels Lliris. Alcoy (Alicante). 6
Department of
Hematology. Hospital Joan XXIII. Tarragona. 7
Department of Hematology. Hospital Arnau de Vilanova. Lleida.8
Department of General Internal Medicine. Hospital de Bellvitge. Barcelona. 9
Department of Hematology.
Hospital Can Misses. Ibiza (Baleares). 10
Department of Hematology. Hospital Central Universitario de Asturias. Oviedo. 11
Department of General Internal Medicine. Hospital de la Ribera. Alzira. Spain.
*Presenting and corresponding author: giner_vicgal@gva.es
Internal Medicine
Department
Rare Diseases Unit
Alcoi (Alicante). Spain.
IntroductionIntroduction
In the past two decades there has been a real revolution in rare diseases as the consequence of the obtention of specific
secure and efficacious treatment. It has been specially notorious for lysosomal diseases, and particularly for Gaucher disease
(GD), actually with six different specific drugs.
Once the somatic manifestations of GD has experienced a radical improvement, psicosocial aspects are having and
increasing attetion. Because the majority of treated GD patients has to go each two weeks to the hospital for enzymatic
infusion, the at home administration is emerging as a secure strategy.
To describe the general characteristics of the
first national Spanish program for Home
Enzymatic Therapy Administration (HETA) of
velaglucerase alfa for stable patients with type
1 Gaucher disease (GD).
This project has been supported
by Shire Pharmaceuticals Iberica©
ObjectiveObjective
MethodologyMethodology
Type of study
Restrospective descriptive study about the experience of the initiation of the first Spanish national program for the Home Enzymatic Therapy Administration of velaglucerase alfa for stable patients with type 1 GD.
The evaluation has been made in April 2016, after nearly 2 years of experience).
Inclusion criteria
All Spanish type 1 GD treated with velaglucerase alfa with at least ten previous in-hospital infusions without any incidence.
Adequate home context.
Stable GD.
Home infusion Protocol
1st
: Responsible physicians for each patient invite them to participate in the program.
2nd
: After initial patient’s agreement, the hospital’s representatives sign a specific contract with Care Giving España©
(CG), the direct responsible enterprise for the at home enzyme administration.
3rd
: In-hospital meeting among the patient, responsible hospital physician, hospital pharmacy service responsible and Care Giving España©v
physician and nurse to explain the program and present the CG nurse
to the patient/s.
4th
: After a conversation between the patient/s and the Care Giving©
nurse to stablish wich days and hour for the enzymatic infusion, the planned visits begin.
5th
: For each infusion the Care Giving España©
nurse takes the enzyme from the Hospital Pharmacy to prepare the at home infusion after exhaustive clinical exploration.
6th
: Periodically clinical reports are sent to the responsible physician at the hospital, and in case of any incidence direct contact is available.
7th
: Scheduling the time and day of the infusion can be changed by the patients after contact with the Care Giving España©
nurse.
ResultsResults
ConclusionsConclusions
Figure 1. Regional distribution of GD patients respect
to the total of patients included in the HETA program.
Table1. General characteristics of the participating hospitals. GD: Gaucher disease participating
patients in the HETA Spanish national program. IM: Internal Medicine.. HEM: Haematology. *: Median [interval] .
Graphic 1. Participating physicians by medical
speciality in the general HETA and Gaucher’s HETA.
City Region Hospital Hosp. size
(n beds)
Depart.
Patients (n)
GD Total
Barcelona Cataluña Bellvitge 629 IM 1 7
Lleida Cataluña Arnau de Vilanova 447 HEM 1 3
Tarragona Cataluña Juan XXIII 336 HEM 1 1
Alcoy Valencia Verge dels Lliris 310 IM, HEM 2 6
Alzira Valencia La Ribera 301 IM 2 4
Ibiza Islas Baleares Can Misses 177 HEM 1 1
San Rafael Murcia Los Arcos 329 HEM 1 1
Oviedo Asturias Central de Asturias 1,324 HEM 1 1
Plasencia Extremadura Virgen del Puerto 331 HEM 1 1
TotalTotal 6 of 176 of 17 99 331 [124-1,324]331 [124-1,324] 1313 1111 1111
There was not any secondary effect
related to the infusions.
Doses as well as quality of life scales and
other clinical, radiological and analytical
parameters were all stable.
i. 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].
ii. Elstein D, Abrahamov A, Oz A, Arbel N, Baris H, Zimran A. 13,845 home
therapy infusions with velaglucerase alfa exemplify safety of
velaglucerase alfa and increased compliance to every-other-week
intravenous enzyme replacement therapy for Gaucher disease. Blood
Cells Mol Dis. 2015; 55 (4):415-8.
iii. Zimran A, Hollak CE, Abrahamov A, van Oers MH, Kelly M, Beutler E. Home
treatment with intravenous enzyme replacement therapy for Gaucher
disease: an international collaborative study of 33 patients. Blood. 1993; 82
(4): 1107-9.
General internistsGeneral internists
HaematologistsHaematologistsNephrologistsNephrologists
The initial experience of a HETA national Spanish program for the treatment of GD
is demonstrating that is a good option since any infusion-related secondary effect
has been communicated, and all the patients has been stable in their illness and
has persisted in the program.
As expected, the majority of patients are controlled by and haematologist,
although general internists is also related in the control of FD patients.
It is also noteworthy the high heterogeneity and fragmentation of the assistance as
well as in the characteristics of the participating hospitals.
The small size of the sample is the main limitation for any conclusion, but initial
positive results can help to extend the program to another hospitals and regions.
33 in16in1633 in16in16
11 in 1in 111 in 1in 1
44 in16in1644 in16in16
11 in 1in 111 in 1in 1
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A
B
HaematologistsHaematologists
General internistsGeneral internists
Communicated as a poster
The majority (82%) of the participating hospitals wih GD patients also had FD patients in the program.
Hospitals without GD patients
areCataluña. They have different size as
well as clinical profile (Clinic being a
large general hospital and Fundació
Puigvert being a Nephrological small
hospital).
BibliographyBibliography