Presencia de buenas prácticas de humanización en la atención a los pacientes con enfermedades raras en los servicios de farmacia / Presence of good humanization practices in the healthcare of patients with rare diseases in Pharmacy Services

Objetivo analizar la presencia de buenas prácticas de humanización en la atención a los pacientes con enfermedades raras en los servicios de farmacia hospitalaria para identificar las fortalezas y las áreas prevalentes de mejora para una atención más humanizada. Métodos se elaboró un cuestionario online empleando Google Form® estructurado en 2 partes: la primera recogía datos identificativos y la segunda incluía las preguntas relacionadas con el cumplimiento de los 61 estándares del Manual de buenas prácticas de humanización en la atención a pacientes con enfermedades raras en los servicios de farmacia hospitalaria. El acceso al cuestionario se envió por correo electrónico a los jefes de servicio de farmacia hospitalaria de 18 hospitales. El periodo de estudio fue de octubre 2021 a octubre 2022. Las variables analizadas fueron el número de criterios cumplidos, el cumplimiento total (porcentaje de criterios cumplidos) tanto por línea estratégica como por tipo o nivel de estándar (básico, básico de obligado cumplimiento, avanzado o excelente), de forma global y agrupados por comunidades autónomas. Resultados el estudio incluyó 18 servicios de farmacia hospitalaria. La media global de estándares cumplidos fue de 31,1 (IC 95%: 24,8–37,6) y el cumplimiento total medio del 52,1% (IC 95%: 44,4–59,7). La línea 1, Cultura de humanización, tuvo un cumplimiento medio del 46,5% (IC 95%: 35,3–57,7), la línea 2, Empoderamiento del paciente, del 47,4% (IC 95%: 37,1–57,8), la línea 3, Cuidado del profesional, del 49,7% (IC 95%: 39,8–59,1), la línea 4, Espacios físicos y confort del 55,6% (IC 95%: 46,3–64,8) y la línea 5, Organización de la atención, del 63,8% (IC 95%: 55,8–71,9). Conclusión el cumplimiento medio de los estándares está entre 40 y 60%, lo que indica que la humanización está presente en los servicios de farmacia hospitalaria, pero existe un amplio margen de mejora (AU)

Objective To analyze the presence of Good Humanization Practices in the care of patients with rare diseases in Hospital Pharmacy Services and to identify the strengths and prevalent areas for improvement in the humanization of healthcare. Methods Online questionnaire structured in two parts was developed using Google Form®. The first one was designed to collect identifying data and the second one included questions related to compliance with the 61 standards of the Manual of Good Humanization Practices in the healthcare of patients with rare diseases in Hospital Pharmacy Services. Access to the questionnaire was sent by email to the Heads of the Hospital Pharmacy Service of 18 hospitals. The study period was from October 2021 to October 2022. The analyzed variables were the number of criteria that were considered met, total compliance (percentage of criteria met), by strategic line and by type or level of standard, globally and grouped by regions of Spain. Results 18 Hospital Pharmacy Services were included. The overall mean of standards met was 31.1 (95% CI: 24.8–37.6) and mean total compliance was 52.1% (95% CI: 44.4–59.7). The mean compliance by strategic line was line 1 Humanization culture: 46.5% (95% CI: 35.3–57.7), line 2 Patient empowerment: 47.4% (95% CI: 37.1– 57.8), line 3 Professional care: 49.7% (95% CI: 39.8–59.1), line 4 Physical spaces and comfort: 55.6% (95% CI: 46.3–64.8) and line 5 Organization of healthcare: 63.8% (95% CI: 55.8–71.9). Conclusion The average compliance with the standards is between 40 and 60%, which indicates that humanization is present in the Hospital Pharmacy Services, but there is a wide margin for improvement. The main strength in the humanization of Hospital Pharmacy Services is a patient-centered care organization, and the area with the greatest room for improvement is the culture of humanization (AU)

Presence of good humanization practices in the healthcare of patients with rare diseases in Pharmacy Services. / Presencia de buenas prácticas de humanización en la atención a los pacientes con enfermedades raras en los servicios de farmacia.

OBJECTIVE: To analyze the presence of Good Humanization Practices in the care of patients with rare diseases in Hospital Pharmacy Services and to identify the strengths and prevalent areas for improvement in the humanization of healthcare. METHODS: Online questionnaire structured in two parts was developed using Google Form®. The first one was designed to collect identifying data and the second one included questions related to compliance with the 61 standards of the Manual of Good Humanization Practices in the healthcare of patients with rare diseases in Hospital Pharmacy Services. Access to the questionnaire was sent by email to the Heads of the Hospital Pharmacy Service of 18 hospitals. The study period was from October 2021 to October 2022. The analyzed variables were the number of criteria that were considered met, total compliance (percentage of criteria met), by strategic line and by type or level of standard, globally and grouped by regions of Spain. RESULTS: 18 Hospital Pharmacy Services were included. The overall mean of standards met was 31.1 (95% CI: 24.8-37.6) and mean total compliance was 52.1% (95% CI: 44.4-59.7). The mean compliance by strategic line was line 1 Humanization culture: 46.5% (95% CI: 35.3-57.7), line 2 Patient empowerment: 47.4% (95% CI: 37.1- 57.8), line 3 Professional care: 49.7% (95% CI: 39.8-59.1), line 4 Physical spaces and comfort: 55.6% (95% CI: 46.3-64.8) and line 5 Organization of healthcare: 63.8% (95% CI: 55.8-71.9). CONCLUSION: The average compliance with the standards is between 40 and 60%, which indicates that humanization is present in the Hospital Pharmacy Services, but there is a wide margin for improvement. The main strength in the humanization of Hospital Pharmacy Services is a patient-centered care organization, and the area with the greatest room for improvement is the culture of humanization.

Omics-Based Approaches for the Characterization of Pompe Disease Metabolic Phenotypes.

Lysosomal storage disorders (LSDs) constitute a large group of rare, multisystemic, inherited disorders of metabolism, characterized by defects in lysosomal enzymes, accessory proteins, membrane transporters or trafficking proteins. Pompe disease (PD) is produced by mutations in the acid alpha-glucosidase (GAA) lysosomal enzyme. This enzymatic deficiency leads to the aberrant accumulation of glycogen in the lysosome. The onset of symptoms, including a variety of neurological and multiple-organ pathologies, can range from birth to adulthood, and disease severity can vary between individuals. Although very significant advances related to the development of new treatments, and also to the improvement of newborn screening programs and tools for a more accurate diagnosis and follow-up of patients, have occurred over recent years, there exists an unmet need for further understanding the molecular mechanisms underlying the progression of the disease. Also, the reason why currently available treatments lose effectiveness over time in some patients is not completely understood. In this scenario, characterization of the metabolic phenotype is a valuable approach to gain insights into the global impact of lysosomal dysfunction, and its potential correlation with clinical progression and response to therapies. These approaches represent a discovery tool for investigating disease-induced modifications in the complete metabolic profile, including large numbers of metabolites that are simultaneously analyzed, enabling the identification of novel potential biomarkers associated with these conditions. This review aims to highlight the most relevant findings of recently published omics-based studies with a particular focus on describing the clinical potential of the specific metabolic phenotypes associated to different subgroups of PD patients.

Consenso de recomendaciones para la mejora de la coordinación asistencial inter e intra-centros en el abordaje de la hemofilia / Consensus recommendations for the improvement of inter- and intra-centre care coordination in the management of hemophilia

Objetivo: definir las recomendaciones consensuadas para mejorar la coordinación asistencial entre Farmacia Hospitalaria, Hematología y Enfermería, inter e intra-centros, en la atención a los pacientes con hemofilia. Método: se identificaron y valoraron las recomendaciones para la mejora de la coordinación asistencial en el abordaje de los pacientes con hemofilia, por parte de un panel multidisciplinar de profesionales con experiencia en este campo (Farmacia Hospitalaria, Hematología y Enfermería) y apoyado en la evidencia científica. La valoración de las recomendaciones identificadas se realizó por metodología de consenso Rand/UCLA (Delphi-adaptado) con base en su adecuación y, posteriormente, a su necesidad. En ambos casos, se empleó la escala ordinal de Likert. Los datos se analizaron estadísticamente a través de diferentes métricas. Resultados: se identificaron 53 recomendaciones para la mejora de la coordinación asistencial entre Farmacia Hospitalaria, Hematología y Enfermería en el manejo del paciente con hemofilia, agrupadas en 8 ámbitos de actuación: i) Unidades de Hemofilia, centros de referencia y abordaje multidisciplinar; ii) papel de Hematología, Farmacia Hospitalaria y Enfermería en el recorrido asistencial de los pacientes con hemofilia; iii) telefarmacia y telemedicina; iv) monitorización farmacocinética; v) transición al régimen de paciente adulto; vi) educación sanitaria al paciente; vii) cirugía, urgencias e ingreso hospitalario; y viii) evaluación de los resultados. Todas las recomendaciones fueron valoradas por el panel de expertos externos como adecuadas y necesarias. Conclusiones: el recorrido asistencial del paciente con hemofilia es complejo y depende de diversas variables. Además, requiere la implicación de distintos profesionales sanitarios que deben actuar de manera coordinada e integrada en todas las etapas de la vida del paciente, de manera adaptada a sus necesidades individuales. ... (AU)

Objective: Define consensus recommendations to improve care coordination between Hospital Pharmacy, Haematology and Nursing, inter- and intra-center, in the care of haemophilia patients.Method: Recommendations for the improvement of care coordination in the management of haemophilia patients were identified and assessed by a multidisciplinary panel of professionals with experience in this field (Hospital Pharmacy, Haematology and Nursing) and supported by scientific evidence. The identified recommendations were assessed by Rand/UCLA consensus methodology (Delphi-adapted) based on their appropriateness and, subsequently, on their necessity. In both cases, it was used ordinal Likert scale. Data were statistically analysed through different metrics. Results: Fifty-three recommendations for the improvement of care coordination between Hospital Pharmacy, Haematology and Nursing in the management of haemophilia patients were identified, grouped into eight areas of action: i) Haemophilia units, reference centers and multidisciplinary care; ii) Role of Haematology, Hospital Pharmacy and Nursing in the patient journey of haemophilia patients; iii) Telepharmacy and telemedicine; iv) Pharmacokinetic monitoring; v) Transition to adult patient regimen; vi) Patient health education; vii) Surgery, emergency room and hospital admission; and viii) Outcome evaluation. All recommendations were assessed as appropriate and necessary by the external expert panel. Conclusions: Haemophilia patient journey is complex and depends on different variables. It also requires the involvement of different healthcare professionals who must act in a coordinated and integrated manner at all stages of the patient's life, adapted to their individual needs. ... (AU)

[Translated article] Consensus recommendations for the improvement of inter- and intra-centre care coordination in the management of hemophilia.

OBJECTIVE: Define consensus recommendations to improve care coordination between Hospital Pharmacy, Hematology and Nursing, inter- and intra-center, in the care of hemophilia patients. METHOD: Recommendations for the improvement of care coordination in the management of hemophilia patients were identified and assessed by a multidisciplinary panel of professionals with experience in this field (Hospital Pharmacy, Hematology and Nursing) and supported by scientific evidence. The identified recommendations were assessed by Rand/UCLA consensus methodology (Delphi-adapted) based on their appropriateness and, subsequently, on their necessity. In both cases, it was used ordinal Likert scale. Data were statistically analyzed through different metrics. RESULTS: Fifty-three recommendations for the improvement of care coordination between Hospital Pharmacy, Hematology and Nursing in the management of hemophilia patients were identified, grouped into eight areas of action: i) Hemophilia units, reference centers and multidisciplinary care; ii) Role of Hematology, Hospital Pharmacy and Nursing in the patient journey of hemophilia patients; iii) Telepharmacy and telemedicine; iv) Pharmacokinetic monitoring; v) Transition to adult patient regimen; vi) Patient health education; vii) Surgery, emergency room and hospital admission; and viii) Outcome evaluation. All recommendations were assessed as appropriate and necessary by the external expert panel. CONCLUSIONS: Hemophilia patient journey is complex and depends on different variables. It also requires the involvement of different healthcare professionals who must act in a coordinated and integrated manner at all stages of the patient's life, adapted to their individual needs. On this matter, the identified and agreed recommendations may improve continuity and quality of care, as they facilitate the integration and coordination of the professionals involved in the management of this pathology, especially Hospital Pharmacy, Hematology and Nursing.

Consensus recommendations for the improvement of inter- and intra-centre care coordination in the management of hemophilia. / Consenso de recomendaciones para la mejora de la coordinación asistencial inter e intra-centros en el abordaje de la hemofilia.

OBJECTIVE: Define consensus recommendations to improve care coordination between Hospital Pharmacy, Haematology and Nursing, inter- and intra-center, in the care of haemophilia patients. METHOD: Recommendations for the improvement of care coordination in the management of haemophilia patients were identified and assessed by a multidisciplinary panel of professionals with experience in this field (Hospital Pharmacy, Haematology and Nursing) and supported by scientific evidence. The identified recommendations were assessed by Rand/UCLA consensus methodology (Delphi-adapted) based on their appropriateness and, subsequently, on their necessity. In both cases, it was used ordinal Likert scale. Data were statistically analysed through different metrics. RESULTS: Fifty-three recommendations for the improvement of care coordination between Hospital Pharmacy, Haematology and Nursing in the management of haemophilia patients were identified, grouped into eight areas of action: i) Haemophilia units, reference centers and multidisciplinary care; ii) Role of Haematology, Hospital Pharmacy and Nursing in the patient journey of haemophilia patients; iii) Telepharmacy and telemedicine; iv) Pharmacokinetic monitoring; v) Transition to adult patient regimen; vi) Patient health education; vii) Surgery, emergency room and hospital admission; and viii) Outcome evaluation. All recommendations were assessed as appropriate and necessary by the external expert panel. CONCLUSIONS: Haemophilia patient journey is complex and depends on different variables. It also requires the involvement of different healthcare professionals who must act in a coordinated and integrated manner at all stages of the patient's life, adapted to their individual needs. On this matter, the identified and agreed recommendations may improve continuity and quality of care, as they facilitate the integration and coordination of the professionals involved in the management of this pathology, especially Hospital Pharmacy, Haematology and Nursing.

Gestión farmacoterapéutica de los medicamentos huérfanos en los Servicios de Farmacia / Orphan drugs pharmacotherapeutic management in Hospital Pharmacy

El tratamiento adecuado de las enfermedades raras y el uso racional de los medicamentos huérfanos constituye actualmente uno de los retos más importantes para el Sistema Sanitario, y un continuo desafío para los Servicios de Farmacia Hospitalaria. El reducido número de medicamentos huérfanos comercializados, la escasa evidencia sobre su relación beneficio-riesgo y su elevado precio conllevan una elevada incertidumbre en la gestión de estos fármacos. Desde la Farmacia Hospitalaria se pretende trabajar para reducir el grado de incertidumbre asociado a la gestión farmacoterapeutica de las enfermedades raras. Para ello, es preciso trabajar en el conjunto de las etapas que constituyen el proceso farmacoterapéutico y que faciliten, entre otras, la disponibilidad de tratamientos, evitar inequidades, y conseguir los mejores resultados en salud, además de contribuir a la sostenibilidad del sistema. La aportación del farmacéutico de hospital ha sido clave en múltiples etapas del proceso como la evaluación, selección y planificación de medicamentos huérfanos, la adquisición y la gestión farmacoeconómica de medicamentos huérfanos, la elaboración de tratamientos, la dispensación de medicamentos, el seguimiento y optimación de los tratamientos con la aplicación de la farmacocinética clínica o más recientemente el abordaje de prácticas de humanización en la atención al paciente con enfermedades raras. (AU)

Currently the adequate management of rare diseases and the rational use of orphan drugs constitute one of the most important challenges for the health system, furthermore these constitute a continuous challenge for Hospital Pharmacy Services. Rare diseases and orphan drugs management have high level of uncertainty due to small number of orphan drugs available, limited evidence on their benefit-risk ratio, and orphan drugs high price. The Hospital Pharmacy works to reduce the degree of uncertainty associated with the rare diseases pharmacotherapeutic management. To reach this goal is necessary to work on all the stages that constitute the pharmacotherapeutic process and that facilitate treatments availability, avoid inequalities, and achieve the best health results, and finally to contribute to health system sustainability . The work of the hospital pharmacist has been key in multiple stages of pharmacotherapeutic process such as evaluation, selection and planning of orphan drugs, acquisition and pharmacoeconomic management of orphan drugs, preparation of treatments, dispensing of medicines, monitoring and optimization of treatments by application of clinical pharmacokinetics or, more recently, approach humanization healthcare practices in patients with rare diseases. (AU)

Hospital pharmacy initiatives for improving the management of patients with congenital coagulopathies.

OBJECTIVE: To identify and promote initiatives aimed at improving the management by hospital pharmacists of patients with congenital  coagulopathies in the Spanish healthcare context. METHOD: A series of initiatives to improve the care of patients with congenital coagulopathies were identified, evaluated, and prioritized by a panel of hospital pharmacists. Prioritization was based on an assessment of each  initiative's impact and feasibility on a scale of 1 to 5. Once initiatives were  prioritized, those assigned the highest priority were grouped into three action  areas. RESULTS: Seven areas of activity were identified in which the role of hospital  pharmacists is key for the management of patients with congenital coagulopathies: coordination with the healthcare team; drug  evaluation and selection; dispensing; patient information and education;  pharmacotherapeutic follow-up; research and innovation in the field of  congenital coagulopathies; and capacity-building and training of hospital  pharmacists. Fifteen initiatives were considered a priority, with an average  impact score ≥ 3.8 and a feasibility score ≥ 3.2. A total of, 29.4% of the  prioritized initiatives corresponded to healthcare, 23.5% to patient information  and education, 11.8% to drug evaluation and selection, 11.8% to  phar macotherapeutic monitoring, 11.8% to cross-sectional initiatives, 5.9% to dispensing and 5.9% to research and innovation in the field of congenital coagulopathies: In contrast, initiatives related to capacity-building and training were not prioritized. CONCLUSIONS: Three main action areas were proposed based on the initiatives  identified as high priority for the management of patients with congenital  coagulopathies by a panel of 16 hospital pharmacists. Action areas revolved  around specific activities that hospital pharmacy departments can undertake to  contribute to improving the healthcare situation in Spain.

OBJETIVO: Identificar e impulsar iniciativas orientadas a la mejora del manejo  de los pacientes con coagulopatías congénitas por parte de farmacia hospitalaria en el contexto sanitario español.Método: Se identificaron, evaluaron y priorizaron, por parte de un panel de  farmacéuticos especialistas en farmacia hospitalaria, iniciativas para la mejora  de la atención a los pacientes con coagulopatías congénitas. La priorización se  realizó en base a la valoración de su impacto y factibilidad en una escala del 1  al 5. Una vez obtenida la priorización de las iniciativas, las de mayor  puntuación se agruparon en tres grandes líneas de actuación. RESULTADOS: Se identificaron siete áreas de actividad en las que el papel de  los farmacéuticos especialistas en farmacia hospitalaria resulta clave para el  manejo del paciente con coagulopatías congénitas: coordinación con el equipo  asistencial de pacientes con coagulopatías congénitas; evaluación y selección  de medicamentos; dispensación; información y formación al paciente;  seguimiento farmacoterapéutico; investigación e innovación en estas  patologías; formación y capacitación continuada del farmacéutico especialista  en farmacia hospitalaria. Se consideraron prioritarias 15 iniciativas por tener  una puntuación media de impacto ≥ 3,8 y factibilidad ≥ 3,2. Así, el 29,4% de  las iniciativas priorizadas pertenecen al ámbito asistencial, el 23,5% a  información y formación al paciente, el 11,8% a evaluación y selección de  medicamentos, el 11,8% al seguimiento farmacoterapéutico, el 11,8% a  iniciativas transversales, el 5,9% a dispensación y el 5,9% a investigación e  innovación en el campo de las coagulopatías congénitas, mientras que las  iniciativas referentes a la formación y capacitación a profesionales no  resultaron priorizadas. CONCLUSIONES: Se han propuesto tres grandes líneas de actuación basadas en  las iniciativas identificadas como altamente prioritarias por un panel de 16  expertos farmacéuticos especialistas en farmacia hospitalaria para el manejo  de pacientes con coagulopatías congénitas. Estas iniciativas se basan en  acciones concretas y pueden llevarse a cabo desde los servicios de farmacia  hospitalaria, por lo que se cree que podrán llegar a tener un impacto real en el  contexto sanitario español.

A multi-criteria decision analysis on the value of nintedanib for interstitial lung diseases.

OBJECTIVES: Our aim was to assess the value of nintedanib for non-idiopathic progressive fibrosing interstitial lung disease (non-IPF PF-ILD) and systemic sclerosis-associated ILD (SSc-ILD) in the Spanish context, using a multi-criteria decision analysis (MCDA). METHODS: Following an adaptation of the Evidence and Value: Impact on DEcision Making (EVIDEM) MCDA methodology, the estimated value of nintedanib was obtained by means of an additive linear model that combined individual weights (100-points distribution) of criteria with the individual scoring of nintedanib in each criterion for every indication, assigned by a multidisciplinary committee of twelve clinicians, patients, pharmacists, and decision-makers. To assess the reproducibility, an alternative weighting method was applied, as well as a re-test of weights and scores at a different moment of time. RESULTS: The experts committee recognized nintedanib as an intervention with a positive value contribution in comparison to placebo for the treatment of non-IPF PF-ILD (0.50 ± 0.16, on a scale from -1 to 1) and SSc-ILD (0.40 ± 0.12), diseases which were considered as very severe and with high unmet needs. The drug was perceived as a treatment that provides an added therapeutic benefit for patients (0.06-0.07), given its proven clinical efficacy (0.05-0.06), slight improvements in patient-reported outcomes (0.01-0.02), and similar safety profile than placebo (-0.04-0.00), which will likely be positioned as a recommended therapy in the next clinical practice guidelines updates. CONCLUSIONS: Under this increasingly used methodology, nintedanib has shown to provide a positive value estimate for non-IPF PF-ILD and SSc-ILD when compared to placebo in Spain.

Tall man lettering application in medication information systems as a quality and safety strategy in hospital organization.

WHAT IS KNOWN AND OBJECTIVE: The technique of highlighted capital letters, Tall Man lettering, is a tool that allows differentiating the names of similar drugs in a way that contributes to reduce medication errors related to the drug identification. The objective was to implement and monitor the application protocol of the Tall Man lettering tool in drug information systems in the healthcare environment to improve the quality of care and patient safety in the pharmacy service and the medical institution. METHODS: Scope: Tertiary general hospital with 1000 beds in which a Tall Man lettering application protocol was approved in the pharmacy service information systems. DESIGN: Retrospective observational study. PERIOD: 2019-2021. POPULATION: Information systems and databases used in the pharmacy service. Working group: Made up of people in charge of information areas and systems. IMPLEMENTATION: Five phases were defined: organization of the working group, selection of drug names and information systems, establishment of actions, their execution, and results monitoring. VARIABLES: Number of pharmaceutical specialties, names of active ingredient and brands incorporated in the information systems. RESULTS AND DISCUSSION: The application of Tall Man lettering was authorized in 13/13 information systems, six have been fully implemented and seven are in the database update phase. Of the implanted systems, a total of 210 drug names have been modified (168/210 active ingredient and 42/210 brand names), corresponding to 659 pharmaceutical specialties. WHAT IS NEW AND CONCLUSION: The application of Tall Man lettering in hospital information systems is a tool for improving the quality of the pharmacy service and guarantees the safety of medicines in the hospital.

Iniciativas para la mejora del manejode los pacientes con coagulopatías congénitas por parte de farmacia hospitalaria / Hospital pharmacy initiatives for improving the management of patients with congenital coagulopathies

Objetivo: Identificar e impulsar iniciativas orientadas a la mejora delmanejo de los pacientes con coagulopatías congénitas por parte de farmaciahospitalaria en el contexto sanitario español.Método: Se identificaron, evaluaron y priorizaron, por parte de un panelde farmacéuticos especialistas en farmacia hospitalaria, iniciativas para lamejora de la atención a los pacientes con coagulopatías congénitas. Lapriorización se realizó en base a la valoración de su impacto y factibilidaden una escala del 1 al 5. Una vez obtenida la priorización de las iniciativas,las de mayor puntuación se agruparon en tres grandes líneas de actuación.Resultados: Se identificaron siete áreas de actividad en las que el papelde los farmacéuticos especialistas en farmacia hospitalaria resulta clavepara el manejo del paciente con coagulopatías congénitas: coordinacióncon el equipo asistencial de pacientes con coagulopatías congénitas;evaluación y selección de medicamentos; dispensación; información yformación al paciente; seguimiento farmacoterapéutico; investigacióne innovación en estas patologías; formación y capacitación continuadadel farmacéutico especialista en farmacia hospitalaria. Se consideraronprioritarias 15 iniciativas por tener una puntuación media de impacto≥ 3,8 y factibilidad ≥ 3,2. Así, el 29,4% de las iniciativas priorizadas pertenecen al ámbito asistencial, el 23,5% a información y formación alpaciente, el 11,8% a evaluación y selección de medicamentos, el 11,8%al seguimiento farmacoterapéutico, el 11,8% a iniciativas transversales, el5,9% a dispensación y el 5,9% a investigación e innovación en el campode las coagulopatías congénitas, mientras que las iniciativas referentesa la formación y capacitación a profesionales no resultaron priorizadas.

Objective: To identify and promote initiatives aimed at improving themanagement by hospital pharmacists of patients with congenital coagulopathiesin the Spanish healthcare context.Method: A series of initiatives to improve the care of patients with congenitalcoagulopathies were identified, evaluated, and prioritized by apanel of hospital pharmacists. Prioritization was based on an assessmentof each initiative’s impact and feasibility on a scale of 1 to 5. Once initiativeswere prioritized, those assigned the highest priority were groupedinto three action areas.Results: Seven areas of activity were identified in which the role ofhospital pharmacists is key for the management of patients with congenitalcoagulopathies: coordination with the healthcare team; drug evaluationand selection; dispensing; patient information and education; pharmacotherapeuticfollow-up; research and innovation in the field of congenitalcoagulopathies; and capacity-building and training of hospital pharmacists.Fifteen initiatives were considered a priority, with an average impactscore ≥ 3.8 and a feasibility score ≥ 3.2. A total of, 29.4% of the prioritizedinitiatives corresponded to healthcare, 23.5% to patient informationand education, 11.8% to drug evaluation and selection, 11.8% to pharmacotherapeutic monitoring, 11.8% to cross-sectional initiatives, 5.9% todispensing and 5.9% to research and innovation in the field of congenitalcoagulopathies: In contrast, initiatives related to capacity-building andtraining were not prioritized.Conclusions: Three main action areas were proposed based onthe initiatives identified as high priority for the management of patientswith congenital coagulopathies by a panel of 16 hospital pharmacists.Action areas revolved around specific activities that hospital pharmacydepartments can undertake to contribute to improving the healthcare situationin Spain.

[Handling of drugs for administration by percutaneous endoscopic gastrostomy in patients with amyotrophic lateral sclerosis and enteral nutrition]. / Manipulación de fármacos para su administración por gastrostomía endoscópica percutánea en pacientes con esclerosis lateral amiotrófica y nutrición enteral.

Introduction: Introduction: amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease. Its symptoms include dysphagia that may make it necessary to place a percutaneous endoscopic gastrostomy (PEG) for feeding. The administration of drugs by PEG can obstruct it, decrease the effectiveness of treatment, and increase the risk of toxicity by altering the original pharmaceutical form. Objective: to describe and analyze the degree of adequacy of the prescription of drugs administered by PEG in patients with ALS and with enteral nutrition (EN). Material and methods: the prescription of pharmacological treatment for patients with ALS who were admitted for placement of a PEG was reviewed. The degree of adequacy of the prescribed drugs was analyzed according to criteria of loss of efficacy, toxicity, risk for handler, and compatibility with EN by consulting the available scientific evidence. Results: the medical prescriptions of the treatments of 34 patients were reviewed, with a total of 307 medications (median of 9 drugs per patient, range 2-17). Via PEG 267 oral medications (median 8 per patient, range 2-15) were prescribed; 81.65 % were solid forms, and the pharmaceutical form was modified in 43 %, due to the risk of catheter occlusion, toxicity or loss of efficacy, affecting 97 % of the patients. Conclusions: patients with ALS and PEG are at risk of presenting safety problems and loss of treatment efficacy related to alteration of the original pharmaceutical form and the interaction with EN.

Introducción: Introducción: la esclerosis lateral amiotrófica (ELA) es una enfermedad neurodegenerativa. Entre sus síntomas destaca la disfagia, que hace necesaria la colocación de una gastrostomía endoscópica percutánea (PEG) para alimentarse. La administración de fármacos por la PEG puede obstruirla, disminuir la eficacia del tratamiento y aumentar el riesgo de toxicidad, al alterar la forma farmacéutica original. Objetivo: describir y analizar el grado de adecuación de la prescripción de fármacos administrados por PEG en pacientes con ELA y con nutrición enteral (NE). Material y métodos: se revisó la prescripción del tratamiento farmacológico de los pacientes con ELA que ingresaban para la colocación de una PEG. Se analizó el grado de adecuación de los fármacos prescritos según los criterios de pérdida de eficacia, toxicidad, riesgo para el manipulador y compatibilidad con la NE, consultando la evidencia científica disponible. Resultados: se revisaron las prescripciones médicas de los tratamientos de 34 pacientes, con un total de 307 medicamentos (mediana de 9 fármacos por paciente; rango, 2-17). Se pautaron por la PEG 267 medicamentos de administración oral (mediana de 8 por paciente; rango, 2-15). El 81,65 % fueron formas sólidas y se modificó la forma farmacéutica en el 43 % por riesgo de oclusión de la sonda, toxicidad o pérdida de eficacia, afectando al 97 % de los pacientes. Conclusiones: los pacientes con ELA y con PEG tienen riesgo de presentar problemas de seguridad y de pérdida de eficacia del tratamiento relacionados con la alteración de la forma farmacéutica original y de la interacción con la NE.

Pharmacotherapeutic management of advanced therapy drugs.

Advanced therapy drugs have emerged in recent years as new pharmacotherapeutic strategies. In this context, hospital pharmacy services have had to adapt to the new challenges posed by the  inclusion of advanced therapies in their roster of services against the  background of the complex pharmacotherapeutic process patients typically go through.All the activities carried out in the hospital pharmacy services must abide by  the rules established in the Spanish legislation and ensure both the quality of  the different drugs they manage and the safety of every single patient.Advanced therapy drugs are associated certain peculiarities, including the need  to select and evaluate potential candidates to receive them; recourse to  financing mechanisms based on risk sharing; and their extreme fragility, which  means that the personnel in charge of handling them must be properly trained  to maintain their viability and that special storage conditions, involving  temperatures below 180 ºC in the case of chimeric antigen receptor T cell  therapies, must be maintained. In addition, use of advanced therapy  medications in the clinical setting has made it necessary for scientific societies  to produce consensus documents recognizing the pivotal role of hospital  pharmacists as indispensable members of the multidisciplinary healthcare team  and ensuring the same traceability, conservation, custody and  pharmacotherapeutical monitoring standards imposed on other drugs to  provide for adequate pharmaceutical care. Scientific societies have also  highlighted the importance of intensifying clinical research, an essential  requirement for the safe incorporation of new therapeutic targets. The present  document is intended to describe the challenges pharmacists may face when  using advanced therapy drugs at the different stages or processes in the  patient's clinical journey.

Los medicamentos de terapia avanzada han emergido en los últimos años  como nuevas estrategias farmacoterapéuticas. En este contexto, los servicios de farmacia hospitalaria nos hemos tenido que adaptar al nuevo reto que ha supuesto su inclusión en nuestra cartera de servicios dentro del  complejo proceso farmacoterapéutico en el que están inmersos los pacientes. Todas las actividades que se desarrollan en los servicios de farmacia hospitalaria cumplen con una base legal establecida en nuestra  legislación y garantizan la calidad y seguridad tanto de los pacientes atendidos  como de todos y cada uno de los medicamentos que se gestionan. Los  medicamentos de terapia avanzada tienen unas características especiales a  considerar que van desde las fases iniciales de selección y evaluación de los  pacientes candidatos y su modelo de financiación, basado en riesgo  compartido, hasta una fragilidad en su manipulación que requiere de una  adecuada y adaptada formación del personal implicado en la logística para  mantener su viabilidad, al necesitar unas condiciones de conservación, en  ocasiones, a temperaturas de menos 180 ºC, en el caso de las células T con  receptores quiméricos de antígenos. Además, la utilización clínica de los  medicamentos de terapia avanzada ha necesitado de documentos de consenso  de las sociedades científicas que pongan en valor el posicionamiento del  farmacéutico hospitalario, como miembro indispensable dentro del equipo  multidisciplinar asistencial, y que garanticen, como en cualquier otro  medicamento, la trazabilidad, la correcta conservación y custodia y el  seguimiento farmacoterapéutico asociado a una adecuada atención  farmacéutica de nuestros pacientes, sin olvidar la importancia de la creciente  investigación clínica, necesaria e imprescindible para una incorporación segura  de nuevas dianas terapéuticas. Por todo ello, consideramos necesario el  presente documento, en donde se ponen de manifiesto los retos o necesidades, desde el punto de vista farmacéutico, en cada una de las etapas o procesos a  considerar en la utilización de los medicamentos de terapia avanzada dentro de nuestro amplio arsenal terapéutico.

Healthcare Resource Utilization among Patients between 60-75 Years with Secondary Acute Myeloid Leukemia Receiving Intensive Chemotherapy Induction: A Spanish Retrospective Observational Study.

BACKGROUND: Information regarding the impact on healthcare systems of secondary acute myeloid leukemia (sAML) is scarce. METHODS: A retrospective review of medical charts identified patients aged 60-75 years with sAML between 2010 and 2019. Patient information was collected from diagnosis to death or last follow-up. Outpatient resource use, reimbursement, frequency and duration of hospitalization, and transfusion burden were assessed. Forty-six patients with a median age of 64 years were included. Anthracycline plus cytarabine regimens were the most common induction treatment (39 patients, 85%). The ratio of the total days hospitalized between the total follow-up was 29%, with a sum of 204 hospitalizations (average four/patient; average duration 21 days). The total average reimbursement was EUR 90,008 per patient, with the majority (EUR 77,827) related to hospital admissions (EUR 17,403/hospitalization). Most hospitalizations (163, mean 22 days) occurred in the period before the first allogeneic hematopoietic stem cell transplant (alloHSCT), costing EUR 59,698 per patient and EUR 15,857 per hospitalization. The period after alloHSCT (in only 10 patients) had 41 hospitalizations (mean 21 days), and a mean reimbursement cost of EUR 99,542 per patient and EUR 24,278 per hospitalization. In conclusion, there is a high consumption of economic and healthcare resources in elderly patients with sAML receiving active treatments in Spain.

Gestión farmacoterapéutica de los medicamentosde terapias avanzadas / Pharmacotherapeutic management of advanced therapy medicinal products

Los medicamentos de terapia avanzada han emergido en los últimosaños como nuevas estrategias farmacoterapéuticas. En este contexto, los serviciosde farmacia hospitalaria nos hemos tenido que adaptar al nuevo retoque ha supuesto su inclusión en nuestra cartera de servicios dentro del complejoproceso farmacoterapéutico en el que están inmersos los pacientes.Todas las actividades que se desarrollan en los servicios de farmaciahospitalaria cumplen con una base legal establecida en nuestra legislacióny garantizan la calidad y seguridad tanto de los pacientes atendidos comode todos y cada uno de los medicamentos que se gestionan.Los medicamentos de terapia avanzada tienen unas característicasespeciales a considerar que van desde las fases iniciales de seleccióny evaluación de los pacientes candidatos y su modelo de financiación,basado en riesgo compartido, hasta una fragilidad en su manipulación querequiere de una adecuada y adaptada formación del personal implicadoen la logística para mantener su viabilidad, al necesitar unas condicionesde conservación, en ocasiones, a temperaturas de menos 180 ºC, en elcaso de las células T con receptores quiméricos de antígenos.Además, la utilización clínica de los medicamentos de terapia avanzadaha necesitado de documentos de consenso de las sociedades científicas utiliquepongan en valor el posicionamiento del farmacéutico hospitalario, comomiembro indispensable dentro del equipo multidisciplinar asistencial, y quegaranticen, como en cualquier otro medicamento, la trazabilidad, la correctaconservación y custodia y el seguimiento farmacoterapéutico asociado auna adecuada atención farmacéutica de nuestros pacientes, sin olvidar laimportancia de la creciente investigación clínica, necesaria e imprescindiblepara una incorporación segura de nuevas dianas terapéuticas.

Advanced therapy medicinal products have emerged in recentyears as new pharmacotherapeutic strategies. In this context, hospitalpharmacy services have had to adapt to the new challenges posed bythe inclusion of advanced therapies in their roster of services againstthe background of the complex pharmacotherapeutic process patientstypically go through.All the activities carried out in the hospital pharmacy services mustabide by the rules established in the Spanish legislation and ensure boththe quality of the different drugs they manage and the safety of every singlepatient.Advanced therapy medicinal products are associated certain peculiarities,including the need to select and evaluate potential candidates toreceive them; recourse to financing mechanisms based on risk sharing; andtheir extreme fragility, which means that the personnel in charge of handlingthem must be properly trained to maintain their viability and that specialstorage conditions, involving temperatures below 180 ºC in the case ofchimeric antigen receptor T cell therapies, must be maintained.In addition, use of advanced therapy medicinal products in the clinicalsetting has made it necessary for scientific societies to produce consensus documents recognizing the pivotal role of hospital pharmacists as indispensablemembers of the multidisciplinary healthcare team and ensuringthe same traceability, conservation, custody and pharmacotherapeuticalmonitoring standards imposed on other drugs to provide for adequate pharmaceuticalcare. Scientific societies have also highlighted the importance ofintensifying clinical research, an essential requirement for the safe incorporationof new therapeutic targets.The present document is intended to describe the challenges pharmacistsmay face when using advanced therapy medicinal products at thedifferent stages or processes in the patient’s clinical journey.

High incidence of pulmonary thromboembolism in hospitalized SARS-CoV-2 infected patients despite thrombo-prophylaxis.

BACKGROUND: SARS-CoV-2 infected patients present thrombotic complications caused by direct endothelial cells injury of the microvessels. Pulmonary thromboembolism (PE) has been reported by Computed Tomography pulmonary angiogram (CTPA) in patients with COVID-19 pneumonia with high D-dimer levels. OBJECTIVES: We present the characteristics of SARS-CoV-2 infected patients diagnosed of PE by CTPA in our hospital. We also present the comparison of these findings with non-infected patients with PE data. METHODS: Retrospective observational cohort study that included patients over 18 years of age hospitalised consecutively between 26th February and 20th May 2020 in an European Hospital with SARS-CoV2 virus infection, and with suspected infection at beginning of admission but with negative PCR, who were studied with CTPA for suspicion of VTE, during their hospitalization. RESULTS: During the study period, 52 CTPA were performed in our hospital, sixteen in SARS-CoV-2 infected patients, with 4 cases (33%) of PE in the infected group, and 11 (44%) in the non-infected group. No significant differences in age (p = 0.43) and sex (p = 0.31) were found between the two groups, infected and non-infected patients. In the infected group, the patients who had PE had a much lower median age (47.8 years) than those without PE (73.3 years). No differences between infected and non-infected patients were detected in the diagnosis of PE with CTPA, 28.6% versus 27.8% (p = 1.00). Overall patient mortality was 1.9%; one patient died (6.3%) in the infected group, and none in the non-infected group (p = 0.31). CONCLUSION: A considerable incidence of PE diagnosed by CTPA in SARS-CoV-2 infected patients has been observed, despite thrombo-prophylaxis.

Observational study to evaluate discontinuation of monotherapy with cobicistat-boosted darunavir in patients with human immunodeficiency virus.

To evaluate the reasons for changing to monotherapy with protease inhibitors, together with the proportion and reasons for the interruption to treatment, in patients who have been treated at some point with cobicistat-boosted darunavir (DRV/c). Outpatients in a tertiary hospital. Observational retrospective study to evaluate monotherapy with DRV/c (800 mg/150 mg) in adult patients with human immunodeficiency virus infection, from December 2014 to July 2022. Demographic variables, viral load, cluster of differentiation 4 lymphocyte lymphocyte count, and antiretroviral therapy were assessed. 42 patients were included. 36% of the patients were undergoing monotherapy at the time of the analysis. The main reason for discontinuation was poor adherence. At time of analysis, 80% of the patients in monotherapy had an undetectable viral load. Antiretroviral therapy recommendations advise against exposing the patient to functional monotherapy with a single drug due to the high risk of virological failure and the onset of resistance to a single drug. Following the analysis of the results, DRV/c in monotherapy is not an effective strategy in the medium and long term due to factors such as lack of adherence or virological failure, although it can be maintained in specific circumstances. Therefore, patients undergoing monotherapy require close monitoring.

A multicriteria decision analysis (MCDA) applied to three long-term prophylactic treatments for hereditary angioedema in Spain.

Introduction: Hereditary angioedema (HAE) is a rare genetic disease that impairs quality of life and could be life-threatening. The aim of this study was to apply a multicriteria decision analysis to assess the value of three long-term prophylactic (LTP) therapies for HAE in Spain. Methods: A multidisciplinary committee of 10 experts assessed the value of lanadelumab (subcutaneous use), C1-inhibitor (C1-INH; intravenous), and danazol (orally), using placebo as comparator. We followed the EVIDEM methodology that considers a set of 13 quantitative criteria. The overall estimated value of each intervention was obtained combining the weighting of each criterion with the scoring of each intervention in each criterion. We used two alternative weighting methods: hierarchical point allocation (HPA) and direct rating scale (DRS). A reevaluation of weightings and scores was performed. Results: Lanadelumab obtained higher mean scores than C1-INH and danazol in all criteria, except for the cost of the intervention and clinical practice guidelines. Under the HPA method, the estimated values were 0.51 (95% confidence interval [CI]: 0.44-0.58) for lanadelumab, 0.47 (95%CI: 0.41-0.53) for C1-INH, and 0.31 (95%CI: 0.24-0.39) for danazol. Similar results were obtained with the DRS method: 0.51 (95%CI: 0.42-0.60), 0.47 (95%CI: 0.40-0.54), and 0.27 (95%CI: 0.18-0.37), respectively. The comparative cost of the intervention was the only criterion that contributed negatively to the values of lanadelumab and C1-INH. For danazol, four criteria contributed negatively, mainly comparative safety. Conclusion: Lanadelumab was assessed as a high-value intervention, better than C1-INH and substantially better than danazol for LTP treatment of HAE.

Design and Rationale for a Real-World Prospective, Multicenter Registry of Myocardial Revascularization Failure and Secondary Revascularization: The REVASEC Study.

AIM: To investigate key aspects of the problem of myocardial revascularization failure (MRF) and repeat or secondary myocardial revascularization (SR) in contemporary practice. METHODS: The registry of secondary revascularization (REVASEC) is an investigator-initiated, multicenter, prospective registry enhanced with data monitoring and independent event adjudication (ClinicalTrials.govNCT03349385). It includes patients with prior revascularization referred to coronary angiography for suspected MRF with broad inclusion criteria. The main objectives are to describe the characteristics of patients with prior revascularization referred for repeat angiography, to describe and the rate and mechanisms of MRF (stent or graft failure, coronary artery disease progression or residual coronary artery disease); to evaluate the management including medical treatment and SR of these patients; and to assess the prognosis according to the outlined causative mechanisms. The registry has one year follow up for the primary endpoint (Patient-oriented composite endpoint including all-cause death, any myocardial infarction or any new unplanned revascularization according to subsets of MRF), but extended follow-up will be carried out up to 5 years. CONCLUSION: The REVASEC Registry will provide updated data on the characteristics, patterns of treatment, and 1-year outcomes of patients with MRF and SR in contemporary clinical practice.

Effectiveness of Caplacizumab Nanobody in Acquired Thrombotic Thrombocytopenic Purpura Refractory to Conventional Treatment.

Acquired thrombocytopenic thrombotic purpura (aTTP) is an autoantibody-mediated disease against the enzyme A Disintegrin and Metalloprotease domain with ThromboSpondin-1 type motif 13, which until now has been treated with plasma exchange (PEX) and corticosteroids. A 29-year-old female patient, who presented with aTTP in the context of pregnancy, has developed multiple relapses after treatment with PEX, corticosteroids, and rituximab. Recently, caplacizumab, a nanobody against von Willebrand factor, has been approved for the treatment of aTTP. In our patient, caplacizumab achieved better disease control, with a lower platelet count restoration time, days of PEX and hospitalization duration, as compared to standard therapy, reproducing the results of clinical trials. Caplacizumab represents a significant advance in the treatment of aTTP, especially in cases of recurrent relapses.