COVID-19's impact on care practice for alpha-1-antitrypsin deficiency patients.

BACKGROUND: Patients with alpha-1 antitrypsin deficiency (AATD), commonly categorized as a rare disease, have been affected by the changes in healthcare management brought about by COVID-19. This study's aim was to identify the changes that have taken place in AATD patient care as a result of the COVID-19 pandemic in Spain and to propose experts' recommendations aimed at ensuring humanized and quality care for people with AATD in the post-pandemic situation. METHODS: A qualitative descriptive case study with a holistic single-case design was conducted, using focus groups with experts in AATD clinical management, including 15 health professionals with ties to the Spanish health system (12 pneumologists and 2 hospital pharmacists from 11 different hospitals in Spain) and 1 patient representative. RESULTS: COVID-19 has had a major impact on numerous aspects of AATD clinical patient management in Spain, including diagnostic, treatment, and follow-up phases. The experts concluded that there is a need to strengthen coordination between Primary Care and Hospital Care and improve the coordination processes across all the organizations and actors involved in the healthcare system. Regarding telemedicine and telecare, experts have concluded that it is necessary to promote this methodology and to develop protocols and training programs. Experts have recommended developing personalized and precision medicine, and patient participation in decision-making, promoting self-care and patient autonomy to optimize their healthcare and improve their quality of life. The possibility of monitoring and treating AATD patients from home has also been proposed by experts. Another result of the study was the recommendation of the need to ensure that plasma donations are made on a regular basis by a sufficient number of healthy individuals. CONCLUSION: The study advances knowledge by highlighting the challenges faced by health professionals and changes in AATD patient management in the context of the COVID-19 pandemic. It also proposes experts' recommendations aimed at ensuring humanized and quality care for people with AATD in the post-pandemic situation. This work could serve as a reference study for physicians on their daily clinical practice with AATD patients and may also provide guidance on the changes to be put in place for the post-pandemic situation.

Comparison of the Cockcroft-Gault, MDRD and CKD-EPI equations for estimating ganciclovir clearance.

AIMS: Accurately estimating kidney function is essential for the safe administration of renally cleared drugs such as ganciclovir. Current practice recommends adjusting renally eliminated drugs according to the Cockcroft-Gault equation. There are no data on the utility of the Modification of Diet in Renal Disease (MDRD) and Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equations in ganciclovir dosing. To evaluate which renal function equation best predicts ganciclovir clearance. METHODS: The performance of the Cockcroft-Gault equation, isotope dilution mass spectrometry (IDMS)-traceable 4-variable MDRD study (MDRD4-IDMS) equation and CKD-EPI equation in determining ganciclovir clearance were assessed retrospectively in patients treated with ganciclovir from 2004-2015. The MDRD4-IDMS and CKD-EPI equations adjusted to individual body surface area (MDRD4-IDMS·BSA and CKD-EPI·BSA, respectively) were also evaluated. Patients with intravenous ganciclovir peak and trough concentrations in their medical records were included in the study. Ganciclovir clearance was calculated from serum concentrations using a one-compartment model. The five equations were compared based on their predictive ability, the coefficient of determination, through a linear regression analysis. The results were validated in a group of patients. RESULTS: One hundred patients were included in the final analysis. Seventy-four patients were analysed in the learning group and 26 in the validation group. The coefficient of determination was 0.281 for Cockcroft-Gault, 0.301 for CKD-EPI·BSA, 0.308 for MDRD4-IDMS·BSA, 0.324 for MDRD4-IDMS and 0.360 for CKD-EPI. Subgroup analysis also showed that CKD-EPI is a better predictor of ganciclovir clearance. Analysis of the validation group confirmed these results. CONCLUSIONS: The CKD-EPI equation correlates better with ganciclovir clearance than the Cockcroft-Gault and MDRD4-IDMS equations, even the clinical difference between the equations is scarce.

Selection of initiatives to improve the management of patients with hereditary angioedema by the hospital pharmacy using the nominal group technique.

OBJECTIVE: To identify and promote hospital pharmacy initiatives to improve the management of patients with hereditary angioedema (HAE) within the Spanish healthcare system. METHOD: A panel of experts comprising hospital pharmacists, an allergist and a nurse/member of the Spanish Hereditary Angioedema Association (Asociación Española de Angioedema Familiar) highlighted initiatives to improve care for patients with HAE after identifying, evaluating and prioritising them. Prioritisation was assessed based on the impact on patient care and the feasibility of their implementation on a scale of 1-5. RESULTS: Seven key areas of activity for the role of hospital pharmacists in the management of patients with HAE were identified: evaluation and selection of medicines; hospital pharmacy dispensation and telepharmacy; pharmacotherapy follow-up and telemedicine; coordination with other healthcare teams involved in the care of patients with HAE; patient health education and training; research on HAE; and continuous education and training of hospital pharmacy service personnel. Ten initiatives with a mean impact score of 5 and a mean feasibility score of ≥4.1 were considered as high-priority initiatives. Half of the initiatives belong to the area concerning patient education and training (50%), followed by care coordination initiatives (30%) and continuous education and training (20%). CONCLUSIONS: Ten high-priority initiatives for the management of patients with HAE were identified by a panel of experts. The implementation of such initiatives by the hospital pharmacy service should enhance the management of patients with HAE in the Spanish healthcare system.

Comparative effectiveness of two lipid emulsions in preventing retinopathy of prematurity in preterm infants requiring parenteral nutrition.

OBJECTIVES: The main aim was to compare the effects of 2 parenteral lipid emulsions on retinopathy of prematurity (ROP) incidence, severity, and need for treatment. Secondary aim was to compare the effect on weight gain in the first 6 weeks of life. METHODS: Single-center, observational, retrospective study analyzing preterm infants with a gestational age (GA) <31 weeks and a birth weight <1251 g born between April 2015 and December 2018. The infants' medical records were reviewed to collect clinical data. Parenteral nutrition (PN) details were obtained from the hospital pharmacy database. RESULTS: In total, 180 patients were included: 90 received ClinOleic® and 90 received SMOFlipid®. No significant differences were observed for the incidence of ROP (40% in ClinOleic® group and 41% in SMOFlipid® group, p=.88) or ROP requiring treatment (4% and 10%, respectively, p=.152). Weekly weight gain was similar in the 2 groups. CONCLUSIONS: This study showed no difference between the 2 groups regarding ROP, ROP requiring treatment, or weekly weight gain in the first 6 weeks of life.

Comparative effectiveness of two lipid emulsions in preventing retinopathy of prematurity in preterm infants requiring parenteral nutrition. / [Artículo traducido] Efectividad comparativa de 2 emulsiones lipídicas en la prevención de la retinopatía del prematuro en neonatos pretérmino que requieren nutrición parenteral.

OBJECTIVES: The main aim was to compare the effects of two parenteral lipid emulsions on retinopathy of prematurity (ROP) incidence, severity, and need for treatment. Secondary aim was to compare the effect on weight gain in the first 6 weeks of life. METHODS: Single-center, observational, retrospective study analyzing preterm infants with a gestational age < 31 weeks and a birth weight < 1,251 g, born between April 2015 and December 2018. The infants' medical records were reviewed to collect clinical data. Parenteral nutrition details were obtained from the hospital pharmacy database. RESULTS: In total, 180 patients were included: 90 received ClinOleic® and 90 received SMOFlipid®. No significant differences were observed for the incidence of ROP (40% in ClinOleic® group and 41% in SMOFlipid® group, p=0.88) or ROP requiring treatment (4% and 10% respectively, p=0.152). Weekly weight gain was similar in the two groups. CONCLUSIONS: This study showed no difference between the two groups regarding ROP, ROP requiring treatment or weekly weight gain in the first 6 weeks of life.

Long-term effect of tocilizumab on mortality, readmissions, persistent symptoms and lung function in SARS-CoV-2 patients 1 year after hospital discharge: A matched cohort study.

BACKGROUND: Tocilizumab is presumed to be an effective and safe treatment for severe SARS-Cov-2, but its usefulness has not been investigated yet for long-term outcomes. This study aimed to evaluate the influence of tocilizumab on mortality in patients with SARS-CoV-2 throughout the year following discharge. METHODS: A retrospective observational analysis was performed on electronic medical records of patients with SARS-CoV2 who were discharged from our hospital after surviving the first wave in March-April 2020. Logistic regression was used to analyse the effect of tocilizumab on mortality, as the main outcome, and propensity-score analysis to further validate their effect. Secondary outcomes were readmissions, persistent symptoms and lung function evolution. Patients were selected by matching their individual propensity for receiving therapy with tocilizumab, conditional on their demographic and clinical variables. RESULTS: A total of 405 patients were included in the mortality study (33.6 % were treated with tocilizumab) and 390 were included in the assessment of persistent symptoms. After propensity-score analysis, no association between tocilizumab use and 1-year overall mortality was found (HR= 2.05, 95 % CI: 0.21-19.98). No differences regarding persistent symptoms (OR= 1.01 95 %CI 0.57-1.79), nor lung function parameters (forced vital capacity: coefficient -0.16 95 %CI -0.45 to 0.14) were found throughout the year follow-up between control and tocilizumab group. CONCLUSIONS: The administration of tocilizumab in patients with SARS-CoV-2 did not show any effect on long-term mortality. Identically, no association were found regarding readmissions, persistent symptoms or lung function evolution and tocilizumab administration in our cohort of patients after 1 year follow-up.

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.

[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.

Recommendations for the Implementation of the Self-Administration of Alpha-1 Antitrypsin.

Purpose: Administration of exogenous alpha-1 antitrypsin (AAT) is the only specific therapy for the management of pulmonary morbidity in patients with AAT deficiency. It requires weekly or biweekly intravenous infusions, which may impact patient independence and quality of life. Self-administration of AAT therapy is an alternative to reduce the burden for patients who require AAT therapy. We presented herein experts' recommendations for the implementation of a program for the self-administration of AAT. Methods: This project was conducted using a modified nominal group technique and was undertaken in two online meetings involving the participation of 25 experts: specialists in pulmonology (n=17), nurses (n=5) and hospital pharmacists (n=3). Results: The following issues were discussed, and several recommendations were agreed upon on the following topics: a) patient profile and clinical evaluation, establishing selection criteria that should include clinical as well as social criteria; b) role of health care professionals, suggested roles for specialists in pulmonology, nurses, and hospital pharmacists; c) training by the nurse, including recommendations before initiating the training and the content of the training sessions; and d) logistic issues and follow-up, adherence, and patient support. Conclusion: We expect this proposal to increase awareness of this therapeutic alternative and facilitate the implementation of self-administration programs, thus contributing to optimizing the patient experience with AAT therapy. Further research on the outcomes of these programs, especially from the patient perspective, will also help to improve their design and implementation.

Effect of tocilizumab versus standard of care in adults hospitalized with moderate-severe COVID-19 pneumonia.

Introduction and objectives: Tocilizumab is an interleukin-6 receptor-blocking agent proposed for the treatment of severe COVID-19; however, limited data are available on their efficacy. The aim of this study was to assess the effect of tocilizumab on the outcomes of patients with COVID-19 pneumonia by using propensity-score-matching (PSM) analysis. Methods: A retrospective observational analysis of hospitalized COVID-19 adult patients admitted to the Vall d'Hebron Hospital was performed between March and April 2020. We used the logistic regression to analyze the effect of tocilizumab on mortality, as main outcome, and PSM analysis to further validate their effect. Secondary outcomes were length-of-stay (LOS) and intensive-care-unit (ICU) stay. Same outcomes were also assessed for early tocilizumab administration, within 72 h after admission. Patients were selected by matching their individual propensity for receiving therapy with tocilizumab, conditional on their demographic and clinical variables. Results: A total of 544 COVID-19 patients were included, 197 (36.2%) were treated with tocilizumab of whom 147 were treated within the first 72 h after admission; and 347 were included in the control group. After PSM analyses, the results showed no association between tocilizumab use and overall mortality (OR = 1.03, 95%CI: 0.63-1.68). However, shorter ICU-stay in the tocilizumab group was found compared to the control group (Coefficient -4.27 95%CI: -6.63 to -1.92). Similar results were found in the early tocilizumab cohort. Conclusions: The administration of tocilizumab in patients with moderate to severe COVID-19 did not reduce the risk of mortality in our cohort of patients, regardless of the time of administration.

Introducción y objetivos: El tocilizumab es un agente bloqueador del receptor de la interleucina 6 propuesto para el tratamiento de la COVID-19 grave; sin embargo, se dispone de datos limitados sobre su eficacia. El objetivo de este estudio fue evaluar el efecto de tocilizumab en los resultados de los pacientes con neumonía por COVID-19 mediante un análisis de emparejamiento por propensity-score-matching (PSM, «puntuación de propensión¼). Métodos: Se realizó un análisis observacional retrospectivo de los pacientes adultos con COVID-19 ingresados en el Hospital Vall d'Hebron entre marzo y abril de 2020. Se utilizó la regresión logística para analizar el efecto de tocilizumab en la mortalidad, como resultado principal, y el análisis PSM para validar aún más su efecto. Los resultados secundarios fueron la duración de la estancia y la estancia en la unidad de cuidados intensivos (UCI). También se evaluaron los mismos resultados para la administración temprana de tocilizumab, dentro de las 72 h posteriores al ingreso. Los pacientes se seleccionaron mediante el emparejamiento de su propensión individual a recibir tratamiento con tocilizumab, condicionado a sus variables demográficas y clínicas. Resultados: Se incluyeron 544 pacientes de COVID-19, 197 (36,2%) fueron tratados con tocilizumab, de los cuales 147 fueron tratados dentro de las primeras 72 h tras el ingreso; y 347 fueron incluidos en el grupo control. Tras los análisis PSM, los resultados no mostraron ninguna asociación entre el uso de tocilizumab y la mortalidad global (OR = 1,03; IC del 95%: 0,63-1,68). Sin embargo, se encontró una menor estancia en la UCI en el grupo de tocilizumab en comparación con el grupo de control (coeficiente −4,27; IC del 95%: −6,63 − −1,92). Se encontraron resultados similares en la cohorte de tocilizumab temprano. Conclusiones: La administración de tocilizumab en pacientes con COVID-19 moderada a grave no redujo el riesgo de mortalidad en nuestra cohorte de pacientes, independientemente del momento de la administración.

Effect of tocilizumab versus standard of care in adults hospitalized with moderate-severe COVID-19 pneumonia.

INTRODUCTION AND OBJECTIVES: Tocilizumab is an interleukin-6 receptor-blocking agent proposed for the treatment of severe COVID-19; however, limited data are available on their efficacy. The aim of this study was to assess the effect of tocilizumab on the outcomes of patients with COVID-19 pneumonia by using propensity-score-matching (PSM) analysis. METHODS: A retrospective observational analysis of hospitalized COVID-19 adult patients admitted to the Vall d'Hebron Hospital was performed between March and April 2020. We used the logistic regression to analyze the effect of tocilizumab on mortality, as main outcome, and PSM analysis to further validate their effect. Secondary outcomes were length-of-stay (LOS) and intensive-care-unit (ICU) stay. Same outcomes were also assessed for early tocilizumab administration, within 72h after admission. Patients were selected by matching their individual propensity for receiving therapy with tocilizumab, conditional on their demographic and clinical variables. RESULTS: A total of 544 COVID-19 patients were included, 197 (36.2%) were treated with tocilizumab of whom 147 were treated within the first 72h after admission; and 347 were included in the control group. After PSM analyses, the results showed no association between tocilizumab use and overall mortality (OR=1.03, 95%CI: 0.63-1.68). However, shorter ICU-stay in the tocilizumab group was found compared to the control group (Coefficient -4.27 95%CI: -6.63 to -1.92). Similar results were found in the early tocilizumab cohort. CONCLUSIONS: The administration of tocilizumab in patients with moderate to severe COVID-19 did not reduce the risk of mortality in our cohort of patients, regardless of the time of administration.

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.

Association between postnatal weight gain and need for treatment in retinopathy of prematurity.

OBJECTIVE: To study the association between gestational age (GA) and weight at birth and the development of retinopathy of prematurity (ROP), and in particular the link between postnatal weight gain during the first 6 weeks and need for ROP treatment. MATERIAL AND METHODS: Retrospective observational study of premature infants who underwent ophthalmoscopy at Hospital Universitari Vall d'Hebron in Barcelona, Spain, between June 2017 and December 2018. We collected data on obstetric and birth characteristics, comorbidities, GA and weight at birth, and weekly weight for the first 6 weeks. RESULTS: Ninety patients with a mean ± SD GA of 26.87 ± 1.90 weeks and a mean birth weight of 884.29 ± 227.40 g were studied. The mean weight at 6 weeks was 1656.89 ± 478.51 g, which corresponds to a gain of 776.17 ± 298.12 g. Thirty-seven patients (41.1%) were diagnosed with ROP and nine (10%) needed treatment. Significant predictors of the need for treatment in patients with ROP were GA (p = .018) and weight at 6 weeks (p = .021). Birth weight was not significant (p = .361). CONCLUSIONS: GA and weight gain during the first 6 weeks of life are significantly associated with the need for treatment in infants with ROP. Sex and birth weight were not significant predictors. Postnatal weight gain at 6 weeks is predictive of the need for ROP treatment.

The influence of parenteral protein intake on electrolyte disturbances in premature infants.

INTRODUCTION: Aggressive parenteral nutrition with delivery of high amino acid and energy doses is used to improve growth and neurodevelopmental outcomes in very low birth weight (VLBW) preterm infants. Recent findings, however, suggest that this approach may cause electrolyte imbalances. The aim of our study was to compare the prevalence of hypercalcaemia, hypophosphataemia, and hypokalaemia in 2 groups of preterm infants that received parenteral nutrition with different amounts of amino acids and to analyse perinatal and nutritional variables associated with the development of electrolyte imbalances. METHODS: We conducted a retrospective observational study comparing 2 groups of preterm infants born before 33 weeks' gestation with birth weights of less than 1500 g managed with parenteral nutrition. One of the groups received less than 3 g/kg/day of amino acids and the other received 3 g/kg//day of amino acids or more. We analysed the prevalence of electrolyte imbalances and possible associations with aggressive parenteral nutrition, adjusting for potential confounders. RESULTS: We studied 114 infants: 60 given less than 3 g/kg/day of amino acids (low-intake group) and 54 given at least 3 g/kg/day (high-intake group). The prevalence of electrolyte imbalances was similar in both groups. The prevalence of hypercalcaemia was 1.67% in the low-intake group and 1.85% in the high-intake group (P > .99), the prevalence of severe hypophosphataemia 11.7% vs 9.3%, and the prevalence of hypokalaemia 15.0% vs 11.1% (P > .99). A calcium to phosphorus ratio greater than 1.05 had a protective effect against hypophosphataemia (P = .007). CONCLUSIONS: We did not find an association between hypercalcaemia, hypophosphataemia, and hypokalaemia and the amino acid dose delivered by PN in the high-intake group of preterm infants.

Long-term effectiveness of cinacalcet in non-dialysis patients with chronic kidney disease and secondary hyperparathyroidism.

BACKGROUND: Secondary hyperparathyroidism (SHPT) is a common complication of chronic kidney disease (CKD). Cinacalcet use is controversial in non-dialysis patients. METHODS: This retrospective observational study recruited patients receiving cinacalcet (off-label use) in 2010 and 2011. Patients were followed for three years from the beginning of treatment using an intention-to-treat approach. RESULTS: Forty-one patients were studied: 14 CKD stage 3 (34.1%), 21 CKD stage 4 (51.2%), and 6 CKD stage 5 (14.6%). Median baseline parathyroid hormone (PTH) was 396 (101-1,300) pg/mL. Upon cinacalcet treatment (22 ± 12 months), PTH levels decreased by ≥ 30% in 73.2% of patients (P < 0.001; 95% confidence interval [CI], 59-87%), with a mean time for response of 18.7 months (95% CI, 15.4-22.1). Sixteen patients were followed for 36 months and treated for 32 ± 9 months. Mean reduction in their PTH levels was 50.1% (P < 0.001; 95% CI, 33.8-66.4%) at 36 months, with 62.5% of patients (P < 0.001; 95% CI, 35.9-89.1%) presenting reductions of ≥ 30%. Serum calcium levels decreased from 9.95 ± 0.62 mg/dL to 9.21 ± 0.83 and 9.12 ± 0.78 mg/dL at 12 and 36 months, respectively (P < 0.001). Serum phosphorus levels increased from 3.59 ± 0.43 to 3.82 ± 0.84 at 12 months (P = 0.180), remaining so at 36 months (P = 0.324). At 12 and 36 months, 2 (12.5%) patients experienced hypocalcemia. Meanwhile, 1 (6.3%) and 4 (25.0%) patients reported hyperphosphatemia at 12 and 36 months, respectively. CONCLUSION: Cinacalcet remained effective for at least 36 months in non-dialysis patients with SHPT. Electrolytic disturbances were managed with concurrent use of vitamin D and its analogs or phosphate binders.

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)

[Pharmacokinetics of alpha1-antitrypsin replacement therapy in severe congenital emphysema]. / Farmacocinética de la alfa-1-antitripsina utilizada en el tratamiento sustitutivo del enfisema congénito grave.

OBJECTIVE: Alpha1-antitrypsin (AAT) deficiency is a codominant autosomal genetic disorder that predisposes a patient to chronic obstructive pulmonary disease and emphysema. Specific treatment is systemic, consisting of intravenous infusion of AAT. The protocol currently recommended by the Spanish Registry is infusion of 180 mg/kg every 21 days. The objective of this study was to assess the pharmacokinetic behavior of AAT and estimate the level of protection, defined as the percentage of time that the AAT plasma concentration was above the assumed protective threshold of 50 mg/dL with the usual protocol and with other alternative ones. MATERIAL AND METHODS: Plasma concentrations at 4 times were analyzed for 9 patients to profile the pharmacokinetics of AAT. The data were fitted to a single compartment open model with the WinNonlin software package. The duration of protection was estimated by simulating the evolution of AAT plasma activity over time according to the model constructed based on data recorded in the study. RESULTS: Five men and 4 women (mean weight, 69 kg; range, 59-84 kg) were given a mean AAT dose of 12.06 g (range, 11-15 g). The mean (SD) volume infused was 516.67 (88.17) mL. The half-life of AAT was 8.7 days and the volume of distribution was 127.6 mL/kg. The currently recommended treatment protocol (180 mg/kg every 21 days) gave a level of protection of 67% (considering 60 mg/dL to be protective threshold) or 76% (for a threshold of 50 mg/dL). Protection values for the alternative protocol of 120 mg/kg every 14 days were 82% and 100%, respectively. For the alternative protocol of 60 mg/kg every 7 days, protection was 100% for both thresholds. CONCLUSIONS: Profiling the pharmacokinetic behavior of AAT has enabled the coverage time to be assessed for several treatment protocols. The regimen of 120 mg/kg every 14 days had the most appropriate profile.

Effectiveness of Cinacalcet in Patients with Chronic Kidney Disease and Secondary Hyperparathyroidism Not Receiving Dialysis.

BACKGROUND: Secondary hyperparathyroidism (SHPT) is a common complication in chronic kidney disease (CKD) patients. Cinacalcet could be a therapeutic option although its use is controversial in patients not receiving dialysis. Thus, the aim of this study is to assess the effectiveness and safety of cinacalcet in patients with CKD and SHPT without renal replacement treatment (RRT) and without renal transplantation (RT). METHODS: A retrospective observational study was conducted. Patients were included if they had collected cinacalcet, under off-label use, during 2010 and 2011. Patients selected were followed from the beginning of cinacalcet therapy for one year of treatment. RESULTS: A total of 37 patients were included with CKD stage 3 (38%), 4 (51%) and 5 (11%). Baseline mean PTH value was 400.86 ± 168.60 mg/dl. At 12 months, a 67% of patients achieved at least a 30% reduction in their PTH value (p<0.001; CI 49.7-83.6), and the overall mean reduction of PTH values was 38% (p< 0.001; IC -49.1, -27.5). A 28% of the patients achieved KDOQI PTH goals (p = 0.003, CI 12%-50%). At 12 months, mean serum calcium values decreased by 6% and mean serum phosphorus values increased by 13%. A 19% of patients experienced hypocalcemia episodes while an increase of 24% in hyperphosphatemia episodes was observed. A 25% of patients finished cinacalcet before a year of treatment. Main withdrawal reasons were: gastrointestinal and other discomfort (8%), hypocalcaemia (8%), non-compliance (3%), interactions (3%) and excess of efficacy (3%). CONCLUSIONS: Cinacalcet was effective in patients with CKD and SHPT not receiving dialysis. Electrolytic imbalances could be managed with administration of vitamin D and analogues or phosphate binders.

Influencia del aporte proteico parenteral en las alteraciones electrolíticas en recién nacidos prematuros / The influence of parenteral protein intake on electrolyte disturbances in premature infants

Introducción: La nutrición parenteral agresiva con aportes energéticos y proteicos altos se utiliza para mejorar el crecimiento y el neurodesarrollo en recién nacidos prematuros de muy bajo peso. No obstante, hallazgos recientes sugieren que su uso puede ocasionar alteraciones electrolíticas. El objetivo del estudio era comparar la prevalencia de hipercalcemia, hipofosfatemia e hipopotasemia en dos grupos de recién nacidos prematuros que recibieron nutrición parenteral con distintos aportes de aminoácidos y analizar variables perinatales y nutricionales asociadas a la ocurrencia de alteraciones electrolíticas. Métodos: Estudio retrospectivo observacional, con comparación de dos grupos de recién nacidos prematuros con peso < 1.500 g y edad gestacional < 33 semanas, que recibían nutrición parenteral. Uno de los grupos recibió < 3 g/kg/d de aminoácidos, mientras que el otro recibió ≥ 3 g/kg/d. Se analizó la prevalencia de distintas alteraciones electrolíticas y su asociación con la nutrición parenteral agresiva, con ajustes para posibles factores de confusión. Resultados: El análisis incluyó 114 recién nacidos: 60 que recibieron < 3 g/kg/d de aminoácidos (bajo aporte) y 54 que recibieron ≥ 3 g/kg/d (alto aporte). La prevalencia de alteraciones electrolíticas fue similar en ambos grupos. La prevalencia de hipercalcemia fue de 1,67% en el grupo de bajo aporte y 1,85% en el grupo de alto aporte (p > 0,99). Los respectivos valores para las otras alteraciones fueron 11,7 vs. 9,3% en el caso de la hipofosfatemia grave y 15,0 vs. 11,1% en el caso de la hipopotasemia (p > 0,99). Se observó que una relación calcio:fósforo superior a 1,05 mostraba un efecto protector frente a la hipofosfatemia (p = 0,007). Conclusiones: No se observó asociación entre la hipercalcemia, hipofosfatemia o la hipopotasemia y el aporte de aminoácidos mediante nutrición parenteral en la población de recién nacidos prematuros con altos aportes de aminoácidos. (AU)

Introduction: Aggressive parenteral nutrition with delivery of high amino acid and energy doses is used to improve growth and neurodevelopmental outcomes in very low birth weight (VLBW) preterm infants. Recent findings, however, suggest that this approach may cause electrolyte imbalances. The aim of our study was to compare the prevalence of hypercalcaemia, hypophosphataemia, and hypokalaemia in 2 groups of preterm infants that received parenteral nutrition with different amounts of amino acids and to analyse perinatal and nutritional variables associated with the development of electrolyte imbalances. Methods: We conducted a retrospective observational study comparing 2 groups of preterm infants born before 33 weeks’ gestation with birth weights of less than 1,500 g managed with parenteral nutrition. One of the groups received less than 3 g/kg/day of amino acids and the other received 3 g/kg/day of amino acids or more. We analysed the prevalence of electrolyte imbalances and possible associations with aggressive parenteral nutrition, adjusting for potential confounders. Results: We studied 114 infants: 60 given less than 3 g/kg/day of amino acids (low-intake group) and 54 given at least 3 g/kg/day (high-intake group). The prevalence of electrolyte imbalances was similar in both groups. The prevalence of hypercalcaemia was 1.67% in the low-intake group and 1.85% in the high-intake group (p > .99), the prevalence of severe hypophosphataemia 11.7 vs. 9.3%, and the prevalence of hypokalaemia 15.0 vs. 11.1% (p > .99). A calcium to phosphorus ratio greater than 1.05 had a protective effect against hypophosphataemia (p = .007). Conclusions: We did not find any association between hypercalcemia, hypophosphatemia, and hypokalemia and amino acid intake by PN in the population of premature infants with quite high amino acid intake values. (AU)