Eficacia y seguridad de la profilaxis con emicizumab en la hemofilia A: estudio de 13 pacientes / Efficacy and safety of prophylaxis with emicizumab in hemophilia A: A study of 13 patients

Introducción: Emicizumab es el primer tratamiento no sustitutivo para profilaxis en hemofilia A grave. Objetivos: Describir los resultados de nuestros pacientes en profilaxis con emicizumab, según la práctica clínica habitual. Material y métodos: Seguimiento de 13 pacientes desde el inicio de la profilaxis, registro de hemorragias, cirugías, reacciones adversas y necesidad o no de tratamiento factorial. Se midieron los niveles plasmáticos en las visitas de seguimiento; la técnica ha sido coagulativa en una etapa, modificada mediante dilución 1:20. Resultados: La mediana de niveles plasmáticos fue de 52,2mg [30,7-71,9]. La profilaxis resultó segura y eficaz; solamente se contabilizó una hemorragia espontánea a lo largo del tiempo y no precisó tratamiento. No hubo episodios tromboembólicos ni reacciones graves de hipersensibilidad, anafilaxia o anafilactoides. La incidencia de reacciones en el lugar de la inyección fue del 8%. El abordaje perioperatorio en las intervenciones menores se llevó a cabo sin tratamiento factorial coadyuvante, en 2 cirugías mayores se precisó una dosis de concentrado de FVIII plasmático en el paciente con hemofilia A sin inhibidor y FVII en el paciente con inhibidor, y fue suficiente para parar la hemorragia. Conclusión: Este estudio demostró que la farmacocinética de emicizumab y su vida media aseguran niveles óptimos con tratamiento profiláctico a las dosis establecidas en la ficha técnica.(AU)

Introduction: Emicizumab is the first non-replacement therapy for prophylaxis in severe hemophilia A. Aims: The principal aim of this study is to describe the results of our patients in prophylaxis with emicizumab, according to the usual clinical practice. Material and methods: Follow-up of 13 patients from the start of prophylaxis, recording of bleeding, surgeries, adverse reactions and the need or not for factor therapy. Plasma levels were measured at follow-up visits, the technique was coagulative in one stage, modified by 1:20 dilution. Results: Median plasma levels were 52.2mg [30.7–71.9]. Prophylaxis was safe and effective; only one spontaneous haemorrhage was recorded over time and no treatment was required. There were no thromboembolic events or serious hypersensitivity, anaphylaxis or anaphylactoid reactions. The incidence of injection site reactions was 8%. Perioperative management in minor interventions was carried out without adjuvant factorial therapy, in 2 major surgeries a dose of plasmatic FVIII concentrate was required in the patient with hemophilia A without inhibitor and FVII in the patient with inhibitor, and it was sufficient to stop the bleeding. Conclusion: This study demonstrated emicizumab pharmacokinetics and its half life ensure optimal levels with prophylaxis treatment at doses established in the technical data sheet.(AU)

Efficacy and safety of prophylaxis with emicizumab in hemophilia A: A study of 13 patients. / Eficacia y seguridad de la profilaxis con emicizumab en la hemofilia A: estudio de 13 pacientes.

INTRODUCTION: Emicizumab is the first non-replacement therapy for prophylaxis in severe hemophilia A. AIMS: The principal aim of this study is to describe the results of our patients in prophylaxis with emicizumab, according to the usual clinical practice. MATERIAL AND METHODS: Follow-up of 13 patients from the start of prophylaxis, recording of bleeding, surgeries, adverse reactions and the need or not for factor therapy. Plasma levels were measured at follow-up visits, the technique was coagulative in one stage, modified by 1:20 dilution. RESULTS: Median plasma levels were 52.2mg [30.7-71.9]. Prophylaxis was safe and effective; only one spontaneous haemorrhage was recorded over time and no treatment was required. There were no thromboembolic events or serious hypersensitivity, anaphylaxis or anaphylactoid reactions. The incidence of injection site reactions was 8%. Perioperative management in minor interventions was carried out without adjuvant factorial therapy, in 2 major surgeries a dose of plasmatic FVIII concentrate was required in the patient with hemophilia A without inhibitor and FVII in the patient with inhibitor, and it was sufficient to stop the bleeding. CONCLUSION: This study demonstrated emicizumab pharmacokinetics and its half life ensure optimal levels with prophylaxis treatment at doses established in the technical data sheet.

Efficacy of Human Papillomavirus Vaccination 4 Years After Conization for High-Grade Cervical Neoplasia.

OBJECTIVE: The aim of the study was to estimate human papillomavirus (HPV) vaccination efficacy in reducing recurrence risk within 4 years after conization for high-grade cervical neoplasia. MATERIALS AND METHODS: From January 2012 to June 2015, we performed a longitudinal, observational study (case-series study) on patients diagnosed with cervical intraepithelial neoplasia 2-3 neoplasia. Efficacy was estimated by a 95% CI of the relative risk, relative risk reduction, attributable risk, and number needed to treat. Parametric and nonparametric tests were used as appropriate to compare 160 vaccinated with 171 nonvaccinated patients. To estimate the hazard ratio of the vaccinated status, patients were subjected to multivariable analyses based on the Cox proportional hazard model. To compare recurrence-free survival, a Kaplan-Meier model and a log-rank test were applied. RESULTS: The overall recurrence was 9.4% in the nonvaccinated and 2.5% in the vaccinated group (p = .009). Vaccination was associated with a significant decrease in the relative risk (73.5%, 95% CI = 21.8%-90.9%) with a mean number needed to treat of 14 patients per relapse prevented. Although positive conization margins were related to the highest recurrence risk, not being vaccinated independently increased this risk 3.5-fold in a 4-year follow-up (p = .025). Cumulative recurrence-free rates differed significantly between both groups (log-rank test: p = .009). CONCLUSIONS: Our study corroborates the benefits of HPV vaccination, recommends a closer and longer follow-up in nonvaccinated women, and offers a 4-year prognosis for patients undergoing conization for high-grade cervical lesions.

Evaluación de la neutropenia secundariaa micofenolato de mofetilo asociado a valganciclovir en pacientes trasplantados hepáticos / Evaluation of neutropenia secondary to mycophenolate mofetil associated with valganciclovir in liver transplant patients

Objetivo: Los fármacos inmunosupresores son necesarios para evitar oreducir el riesgo de rechazo de órganos trasplantados. La inmunosupresión generada puede dar lugar a que estos pacientes necesiten recibirantibióticos y antivíricos con los inmunosupresores para evitar el riesgo deinfecciones. Esto ha generado un incremento de neutropenia en pacientestratados conjuntamente con micofenolato de mofetilo y valganciclovir. Elobjetivo de este estudio es estimar el riesgo de neutropenia atribuible altratamiento concomitante de micofenolato de mofetilo y valganciclovir enpacientes trasplantados hepáticos.Método: Estudio de cohorte retrospectiva. Se incluyeron pacientesreceptores de hígado entre 2012 y 2017 tratados con micofenolato demofetilo o con la combinación de micofenolato de mofetilo y valganciclovir, con al menos 100 días de seguimiento postrasplante. Se excluyeronmenores de 16 años y pacientes fallecidos durante el seguimiento. Elanálisis de regresión logística binaria se utilizó para determinar la asociación del riesgo de neutropenia con el sexo, edad, diabetes, creatininabasal y al alta, y tratamiento concomitante de micofenolato de mofetilo yvalganciclovir. El riesgo relativo y los IC 95% se calcularon mediante loscoeficientes de regresión logística. (AU)

Objective: Immunosuppressive drugs are necessary to avoid or reducethe risk of rejection of transplanted organs. The immunosuppression generated may result in these patients needing antibiotics and antivirals to beprescribed to them in conjunction with their immunosuppressants to avoidthe risk of infection. This has generated an increase in neutropenia inpatients treated with mycophenolate mofetil in combination with valganciclovir. The purpose of this study is to estimate the risk of neutropeniaattributable to combination treatment of mycophenolate mofetil with valganciclovir in patients with a transplanted liver.Method: This is a retrospective cohort study. It included patients whoreceived a liver transplant between 2012 and 2017 and who were treated with mycophenolate mofetil or with a combination of mycophenolate mofetil and valganciclovir. Minimum follow-up was 100 days posttransplantation. Children under 16 years of age and patients who diedduring follow-up were excluded. Binary logistic regression analysis wasused to determine the association of neutropenia with sex, age, diabetes,creatinine at baseline and at discharge, and concomitant treatment ofmycophenolate mofetil with valganciclovir. Relative risk and 95% CI werecalculated using logistic regression coefficients. (AU)

Evaluation of neutropenia secondary to mycophenolate mofetil associated with valganciclovir in liver transplant patients.

OBJECTIVE: Immunosuppressive drugs are necessary to avoid or reduce the risk of rejection of transplanted organs. The immunosuppression generated may result in these patients needing  antibiotics and antivirals to be prescribed to them in conjunction with their immunosuppressants to avoid the risk of infection. This has generated an increase in neutropenia in patients treated with mycophenolate mofetil in combination with valganciclovir. The purpose of this study is to estimate the risk of neutropenia attributable to combination treatment of mycophenolate mofetil with valganciclovir in patients with a transplanted liver. METHOD: This is a retrospective cohort study. It included patients who received a liver transplant between 2012 and 2017 and who were treated with mycophenolate mofetil or with a combination of mycophenolate mofetil and valganciclovir. Minimum follow-up was 100 days posttransplantation. Children under 16 years of age and patients who died during follow-up were excluded. Binary logistic regression analysis was used to determine the association of neutropenia with sex, age, diabetes, creatinine at baseline and at discharge, and concomitant treatment of mycophenolate mofetil with valganciclovir. Relative risk and 95% CI were calculated using logistic regression coefficients. RESULTS: 144 patients were analyzed, 87 were treated with mycophenolate mofetil and 57 received mycophenolate mofetil and valganciclovir together. An overall risk of neutropenia of 37% [95% CI (29- 45)] was observed. The risk was significantly higher in patients who received the combination of mycophenolate mofetil and valganciclovir (56%) than in those treated with mycophenolate mofetil alone (24%), p = 0.001. Binarylogistic-regression analysis revealed that concomitant use of mycophenolate mofetil with valganciclovir was associated with an increased risk of neutropenia: Relative risk = 4.97, 95% CI [2.25-11.00]. CONCLUSIONS: Our study shows that concomitant use of mycophenolate mofetil and valganciclovir increases the risk of neutropenia in patients with a transplanted liver.

Objetivo: Los fármacos inmunosupresores son necesarios para evitar o reducir el riesgo de rechazo de órganos trasplantados. La inmunosupresión generada puede dar lugar a que estos pacientes necesiten recibir antibióticos y antivíricos con los inmunosupresores para evitar el riesgo de infecciones. Esto ha generado un incremento de neutropenia en pacientes tratados conjuntamente con micofenolato de mofetilo y valganciclovir. El objetivo de este estudio es estimar el riesgo de neutropenia atribuible al tratamiento concomitante de micofenolato de ofetilo y valganciclovir en pacientes trasplantados hepáticos.Método: Estudio de cohorte retrospectiva. Se incluyeron pacientes receptores de hígado entre 2012 y 2017 tratados con micofenolato de mofetilo o con la combinación de micofenolato de mofetilo y valganciclovir, con al menos 100 días de seguimiento postrasplante. Se excluyeron menores de 16 años y pacientes fallecidos durante el seguimiento. El análisis de regresión logística binaria se utilizó para determinar la asociación del riesgo de neutropenia con el sexo, edad, diabetes, creatinina basal y al alta, y tratamiento concomitante de micofenolato de mofetilo y valganciclovir. El riesgo relativo y los IC 95% se calcularon mediante los coeficientes de regresión logística.Resultados: Un total de 144 pacientes fueron analizados, 87 se trataron con micofenolato de mofetilo y 57 recibieron conjuntamente micofenolato de mofetilo y valganciclovir, observándose un riesgo de neutropenia del 37%, IC 95% [29-45]. Este riesgo fue significativamente mayor en pacientes que recibieron la combinación de micofenolato de mofetilo y valganciclovir (56%) respecto a los tratados solo con micofenolato de mofetilo (24%), p = 0,001. El análisis de regresión logística binaria reveló que el uso concomitante de micofenolato de mofetilo y valganciclovir se asociaba a un mayor riesgo de neutropenia: riesgo relativo = 4,97, IC 95% [2,25-11,00].Conclusiones: Nuestro estudio demuestra que el uso concomitante de micofenolato de mofetilo y valganciclovir aumenta el riesgo de neutropenia en pacientes trasplantados hepáticos.

Cidofovir intravesical, uso en cistitis hemorrágica por poliomavirus BK tras un trasplante de progenitores hematopoyéticos: off-label / Cidofovir intravesical, use in BK poliomavirus associated haemorrhagic cystitis after a transplantation of hematopoyetic progenitors: Off-label

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