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. 1Rare Diseases Unit. Department of General Internal Medicine. Hospital Mare de Déu dels Lliris. Alcoy (Alicante). 2National Medical Coordinator. Caregiving Ibérica SL. Madrid. 3Department of Hematology. Hospital Los Arcos del Mar Menor. San Javier (Murcia). 4Department of Hematology. Hospital de Plasencia. Plasencia (Cáceres). 5Department of Hematology. Hospital Mare de Déu dels Lliris. Alcoy (Alicante). 6Department of Hematology. Hospital Joan XXIII. Tarragona. 7Department of Hematology. Hospital Arnau de Vilanova. Lleida.8Department of General Internal Medicine. Hospital de Bellvitge. Barcelona. 9Department of Hematology. Hospital Can Misses. Ibiza (Baleares). 10Department 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 DepartmentRare Diseases UnitAlcoi (Alicante). Spain.

IntroductionIntroductionIn 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

MethodologyMethodologyType 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 Protocol1st: 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©vphysician 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 TotalBarcelona Cataluña Bellvitge 629 IM 1 7

Lleida Cataluña Arnau de Vilanova 447 HEM 1 3

Tarragona Cataluña Juan XXIII 336 HEM 1 1Alcoy Valencia Verge dels Lliris 310 IM, HEM 2 6Alzira 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 17 6 of 17 99 331 [124-331 [124-1,324]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.

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44 in16in16

1 1 in in 11

1 1 in in 11

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A

BHaematologistsHaematologists

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