107. Proposal of a tool for the assessment of the quality of Gaucher disease clinical management. *Giner V1, Fernández MA2, Villarrubia J3, Bureo JC4, Fernández JJ5, Núñez R6, Grande M7, Llorente C7, Zoni AC8, Arenas CA9, Vicente D1, Sanz J1.1Rare Diseases Unit. Department of General Internal Medicine. Hospital Mare de Déu dels Lliris. Alcoy. Spain. 2Department of Hematology. Hospital Virgen del Puerto. Plasencia (Cáceres).Spain. 3 Department of Hematology. Hospital Universitario Ramón y Cajal. Madrid. Spain. 4Department of General Internal Medicine. Hospital Infanta Cristina. Badajoz. Spain. 5Department of General Internal Medicine. Hospital do Meixoeiro. Vigo. Spain. 6Unit of Hemophilia. Department of Hematology. Hospital Universitario Virgen del Rocío. Sevilla. Spain. 7Department of Preventive Medicine and Quality of Care. Hospital General Universitario Gregorio Marañón. SERMAS. Madrid. Spain. 8Epidemiology Area. Subdirection for Health Promotion and Prevention. Consejería de Salud de la Comunidad de Madrid. Madrid. Spain. 9 Sociedad Española de Directivos de Salud (SEDISA).*Presenting and corresponding author: giner_vicgal@gva.es

IntroductionIntroduction

MethodologyMethodology

Internal Medicine DepartmentRare Diseases UnitAlcoi (Alicante). Spain.

To avoid unjustified clinical variation in the way we manage our patinets and to assure it is being done in the best way, it is necessary tools for the evaluation of the quality of our work. It is specially noteworthy in rare diseases due to the scarce amount of clear and definite evidence we have.

To create a tool for the evalution of the assistance given to GD patients as the first step to improve our assisstance.

Phase 1Objetive:

Identification and analysis of existing publications about quality in GD management. Methodology:

Review of the published evidence in Medline (PubMed)©, Embase© and Cochrane© databases according to PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) methodology until october 2013.

Phase 2 Objetive:

First proposal of quality criteria for 3 clinical scenarios: Diagnosis, Treatment and Follow-up. Methodology:

Creation of an expert group with seven national experts on GD.Initial proposal for quality criteria after evaluation and adaptation to spanish clinical practice.

As illustrated in Figure 1, the project was developed in four consecutive phases between 2013 and 2016:

Phase 3Objetive:

General consensus for the election of significant quality indicators.Methodology:

Identification of national physicians with expertise in the management of GD. Consensuated evaluation of proposed quality criteria by 31 national treating GD

patients in two Delphi rounds. In each round the panelists evaluated each proposal applying a 9 points Likert scale.

Phase 4Objetive:

Final manuscript for clinical aplication.Methodology:

Creation of a work sheet for each quality indicator according to Sociedad Española de Calidad Asistencial (SECA) (Spanish Society of Assitential Quality) 2012 protocol for the Spanish Public Health System hospitals.

ResultsResults

Phase 1 Phase 2 Phase 3 Phase 4

ScenarioScenario Quality parameterQuality parameter CalculationCalculation ObjectiveObjective

Diagnosis (D)

D1. Anamnesis and physical exploration

GD patients with D1/All GD patients x 100 100%

D2. Basic general analytical study GD patients with D2/All GD patients x 100 100%

D3. Glucocerebrosidase activity GD patients with D3/All GD patients x 100 100%

D4. Genetic study GD patients with D4/All GD patients x 100 100%

D5. Biomarkers GD patients with D5/All GD patients x 100 100%

D6. Bone disease evaluation GD patients with D6/All GD patients x 100 100%

D7. Organomegaly evaluation GD patients with D7/All GD patients x 100 100%

D8. Global severity evaluation GD patients with D8/All GD patients x 100 100%

D9. Exhaustive familial story GD patients with D9/All GD patients x 100 100%

D10. Familial screening GD patients with D10/All GD patients x 100 100%

Treatment (T)

ERT: Enzyme replacement TherapySRT: Substracte reduction Therapy

T1. Specific treatment: ERT GD patients with T1/All GD patients x 100

T1.1 and T1.2 100%, T1.3 50%, T1.4 0%

T2. Specific treatment: SRT GD patients with T2/All GD patients x 100 100%

T3. Therap. objective: Anemia GD patients with T3/All GD patients x 100 100%

T4. Therap. objective: Thrombopenia

GD patients with T4/All GD patients x 100

T4.1, T4.2, T4.3, T4.4, T4.5 100%

T5. Therap. objective: Hepatomegaly

GD patients with T5/All GD patients x 100

T5.1 ≥20 %, T5.2 ≥30 %.

T6. Therap. objective: Splenomegaly

GD patients with T6/All GD patients x 100 T6.1 ≥30 %, T6.2 ≥60 %.

T7. Therap. objective: Bone GD patients with T7/All GD patients x 100

T7.1 ≥20%, T7.2 ≥50%, T7.3 ≥30%.

Follow-up (F)

F1. Continuos and planned follow-up

GD patients with T7/All GD patients x 100 F1.1, F1.2, F1.3 100%

F2. No reached objectives GD patients with T7/All GD patients x 100 100%

ConclusionsConclusions

Figure 1. Methodological phases in the confection of the final document entitled “Guía práctica de indicadores de calidad asistencial en la enfermedad de Gaucher” (“Practical guideline of assistential quality indicators in Gaucher disease”).

This project has been supported by Shire Pharmaceuticals Iberica©

ObjectiveObjective

Although the process of quality is essential to improve health care, the presented is the only reported specific tool to assess quality of actual GD clinical management.

Because of very scarce bibliographic evidence and high level of consensuated decisions, probably a specific document for each Health system is necessary.

The simplicity of the proposed system could facilitate its application as a way to improve and standarize clinical management of GD patients as a continuous improvement process.