Onasemnogene Abeparvovec Administration via Peripherally Inserted Central Catheter: A Case Report.

Onasemnogene abeparvovec (OA) is the approved intravenous gene therapy for the treatment of spinal muscular atrophy (SMA). A functional copy of the human SMN1 gene was inserted into the target motor neuron cells via a viral vector, AAV9. In clinical trials, OA was infused through a peripheral venous catheter, and no data are available on central catheter use. Recently, we had a case where OA was administered directly into the right atrium via a peripherally inserted central catheter (PICC) instead of a peripheral line, as recommended. The patient was a female child aged 4 months, diagnosed as SMA type I. For practical reasons, a dose of OA according to the weight of the patient (1.1 × 1014 vectorial genomes/kg) was administered via PICC in 1 h, as the product information recommends. The drug was well tolerated, with no hypersensitivity reactions or initial elevation of transaminases or other adverse effects. To our knowledge, this is the first case reported where OA was administered via a central line. This type of administration is not contraindicated, but it is not specifically contemplated or recommended. It is unknown whether central line administration could have any implications for transduction efficiency and immunogenicity. Future studies should clarify these aspects, as each gene therapy has a specific optimal dose recorded that depends on the site and route of administration of the drug, the AAV variant and the transgene.

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.

2023 GEIS Guidelines for gastrointestinal stromal tumors.

Gastrointestinal stromal tumor (GIST) is the most common malignant neoplasm of mesenchymal origin. GIST spans a wide clinical spectrum that ranges from tumors with essentially no metastatic potential to malignant and life-threatening spread diseases. Gain-of-function mutations in KIT or PDGFRA receptor tyrosine kinases are the crucial drivers of most GISTs, responsible for tumor initiation and evolution throughout the entire course of the disease. The introduction of tyrosine kinase inhibitors targeting these receptors has substantially improved the outcomes in this formerly chemoresistant cancer. As of today, five agents hold regulatory approval for the treatment of GIST: imatinib, sunitinib, regorafenib, ripretinib, and avapritinib. This, in turn, represents a success for a rare neoplasm. During the past two decades, GIST has become a paradigmatic model in cancer for multidisciplinary work, given the disease-specific particularities regarding tumor biology and tumor evolution. Herein, we review currently available evidence for the management of GIST. This clinical practice guideline has been developed by a multidisciplinary expert panel (oncologist, pathologist, surgeon, molecular biologist, radiologist, and representative of patients' advocacy groups) from the Spanish Group for Sarcoma Research, and it is conceived to provide, from a critical perspective, the standard approach for diagnosis, treatment, and follow-up.

Genomic landscapes of ovarian clear cell carcinoma from latin countries reveal aberrations linked to survival and progression.

BACKGROUND: Ovarian clear cell carcinomas (OCCCs) are rare, aggressive and chemoresistant tumors. Geographical and ethnic differences in the incidence of OCCC have been reported with a higher incidence in Asiatic countries. There is a paucity of information regarding OCCC in Latin America (LA) and other countries. METHODS: Here, we characterized two cohorts of 33 patients with OCCC from LA (24 from Brazil and 9 from Costa Rica) and a cohort of 27 patients from Spain. Genomic analysis was performed for 26 OCCC using the OncoScan platform. Tumors were classified according to their genomic landscapes into subgroups. Clinical parameters were related to the frequency of genomic aberrations. RESULTS: The median overall survival (OS) was not significantly different between the cohorts. Genomic landscapes were characterized by different homologous recombination deficiency (HRD) levels. No difference in the distribution of genomic landscapes profiles was detected between patients from the different cohorts. OCCCs with MYC-amplified tumors harboring a concomitant loss of a region in chromosome 13q12-q13 that includes the BRCA2 gene had the longest OS. In contrast, patients carrying a high number (> 30) of total copy number (CN) aberrations with no concomitant alterations in MYC and BRCA2 genes presented the shortest OS. Furthermore, amplification of the ASH1L gene was also associated with a shorter OS. Initial-stage OCCCs with early progression were characterized by gains in the JNK1 and MKL1 genes. CONCLUSIONS: Our results provide new data from understudied OCCC populations and reveal new potential markers for OCCCs.

The Prognostic Significance of Tumor-Infiltrating Lymphocytes, PD-L1, BRCA Mutation Status and Tumor Mutational Burden in Early-Stage High-Grade Serous Ovarian Carcinoma-A Study by the Spanish Group for Ovarian Cancer Research (GEICO).

Early stages are under-represented in studies on the molecular and immune features of high-grade serous ovarian carcinoma (HGSOC), and specific studies focused on early-stage HGSOC are required for a better prognostic stratification and to personalize chemotherapy. The aim of this study was to determine the prognostic significance of CD8+ and CD4+ tumor-infiltrating lymphocytes (TILs), tumoral cell PD-L1 expression, BRCA mutational status and tumor mutation burden (TMB) in early-stage HGSOC. A retrospective study was performed on stage I and II HGSOC from the Molecular Reclassification of Early Stages of Ovarian Cancer (RECLAMO) cohort from the Spanish Group of Ovarian Cancer Research (GEICO). Centralized histological typing was performed based on morphological and immunohistochemical features. Intraepithelial (i) and stromal (s) CD8+ and CD4+ T cells and PD-L1 were evaluated on tissue microarrays by immunohistochemistry. BRCA1 and BRCA2 mutation status and TMB were analyzed in tumor DNA using next-generation sequencing. The study included 124 tumors. High iCD8+ (>20 TILs/core), low/intermediate CD4+ (<20 TILs/core) and high CD8+/CD4+ ratio (>35/core) were associated with favorable outcomes. Tumor cell PD-L1 expression (TPS ≥ 1) was present in only 8% of tumors. In total, 11 (16%) and 6 (9%) out of 69 HGSOC tested carried pathogenic or likely pathogenic BRCA1 or BRCA2 mutations, respectively. Median TMB of 40 tumors analyzed was 5.04 mutations/Mb and only 6 tumors had 10 or more mutations/Mb. BRCA status and TMB were not associated with TILs or prognosis. When compared with studies on advanced HGSOC, our results suggested that prognostic variables differed according to stage and that more studies focused on early stages of HGSOC are needed to better stratify these tumors.

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.

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

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. Palabras clave: Esclerosis lateral amiotrófica; Gastrostomía endoscópica percutánea; Interacción fármaco-nutriente; Medicamentos peligrosos; Nutrición enteral; Sondas digestivas (AU)

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 (AU)

Clinical research in ovarian cancer: consensus recommendations from the Gynecologic Cancer InterGroup.

The Gynecologic Cancer InterGroup (GCIG) sixth Ovarian Cancer Conference on Clinical Research was held virtually in October, 2021, following published consensus guidelines. The goal of the consensus meeting was to achieve harmonisation on the design elements of upcoming trials in ovarian cancer, to select important questions for future study, and to identify unmet needs. All 33 GCIG member groups participated in the development, refinement, and adoption of 20 statements within four topic groups on clinical research in ovarian cancer including first line treatment, recurrent disease, disease subgroups, and future trials. Unanimous consensus was obtained for 14 of 20 statements, with greater than 90% concordance in the remaining six statements. The high acceptance rate following active deliberation among the GCIG groups confirmed that a consensus process could be applied in a virtual setting. Together with detailed categorisation of unmet needs, these consensus statements will promote the harmonisation of international clinical research in ovarian cancer.

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.

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.