ABSTRACT
BACKGROUND: Different prophylactic and episodic clotting factor treatments are used in the management of hemophilia. A summarize of the evidence is needed inform decision-making. OBJECTIVE: To compare the effects of factor replacement therapies in patients with hemophilia. METHODS: We performed a systematic search in PubMed, Central Cochrane Library, and Scopus. We included randomized controlled trials (RCTs) published up to December 2020, which compared different factor replacement therapies in patients with hemophilia. Random-effects meta-analyses were performed whenever possible. The certainty of the evidence was assessed using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) methodology. The study protocol was registered in PROSPERO (CRD42021225857). RESULTS: Nine RCTs were included in this review, of which six compared episodic with prophylactic treatment, all of them performed in patients with hemophilia A. Pooled results showed that, compared to the episodic treatment group, the annualized bleeding rate was lower in the low-dose prophylactic group (ratio of means [RM]: 0.27, 95% CI: 0.17 to 0.43), intermediate-dose prophylactic group (RM: 0.15, 95% CI: 0.07 to 0.36), and high-dose prophylactic group (RM: 0.07, 95% CI: 0.04 to 0.13). With significant difference between these subgroups (p = 0.003, I2 = 82.9%). In addition, compared to the episodic treatment group, the annualized joint bleeding rate was lower in the low-dose prophylactic group (RM: 0.17, 95% CI: 0.06 to 0.43), intermediate-dose prophylactic group (RM of 0.14, 95% CI: 0.07 to 0.27), and high-dose prophylactic group (RM of 0.08, 95% CI: 0.04 to 0.16). Without significant subgroup differences. The certainty of the evidence was very low for all outcomes according to GRADE methodology. The other studies compared different types of clotting factor concentrates (CFCs), assessed pharmacokinetic prophylaxis, or compared different frequencies of medication administration. CONCLUSIONS: Our results suggest that prophylactic treatment (at either low, intermediate, or high doses) is superior to episodic treatment for bleeding prevention. In patients with hemophilia A, the bleeding rate seems to have a dose-response effect. However, no study compared different doses of prophylactic treatment, and all results had a very low certainty of the evidence. Thus, future studies are needed to confirm these results and inform decision making.
Subject(s)
Blood Coagulation Factors/therapeutic use , Hemophilia A/drug therapy , Pre-Exposure Prophylaxis/methods , Blood Coagulation Factors/pharmacology , Factor IX , Factor VIII , Female , Hemarthrosis/drug therapy , Hemophilia B/drug therapy , Hemorrhage/drug therapy , Humans , Male , Randomized Controlled Trials as TopicABSTRACT
Introducción: El presente artículo resume la guía de práctica clínica (GPC) para diagnóstico y tratamiento de hemofilia en el Seguro Social del Perú (EsSalud). Objetivo: Proveer recomendaciones clínicas basadas en evidencia para el diagnóstico y tratamiento de la hemofilia en EsSalud. Material y Métodos: Se conformó un grupo elaborador de la guía (GEG) que incluyó médicos especialistas y metodólogos, el cual formuló preguntas clínicas. Se realizaron búsquedas sistemáticas de revisiones sistemáticas y cuando fue considerado pertinente estudios primarios en PubMed durante el 2020 y 2021. Se seleccionó la evidencia para responder cada una de las preguntas clínicasformuladas. Se evaluó la certeza de la evidencia usando la metodología Grading of Recommendations Assessment, Development, and Evaluation (GRADE). En reuniones de trabajo periódicas, el GEG usó la metodología GRADE para revisar la evidencia y formular las recomendaciones. La GPC fue revisada por expertos externos previa a su aprobación. Resultados: La GPC abordó 09 preguntas clínicas de diagnóstico y tratamiento. En base a dichas preguntas se formularon 05 recomendaciones (01 fuerte y 04 condicionales), 51 puntos de buena práctica clínica, y 02 flujogramas. Conclusión: Se emitieron recomendaciones basadas en evidencia para el diagnóstico y tratamiento de personas con hemofilia.
Introduction: This article summarizes the clinical practice guideline (CPG) for diagnosis and treatment of hemophilia in the Social Security of Peru (EsSalud). Objective: To provide evidence-based clinical recommendations for the diagnosis and treatment of hemophilia in EsSalud. Material and Methods: A guideline development group (GDG) was formed, including medical specialists and methodologists, which formulated clinical questions. Systematic searches of systematic reviews and -when considered relevant- primary studies were performed in PubMed during 2020 and 2021. Evidence was selected to answer each of the clinical questions formulated. The certainty of the evidence was evaluated using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) methodology. In periodic working meetings, the GEG used the GRADE methodology to review the evidence and formulate recommendations. The CPG was reviewed by external experts prior to approval. Results: The CPG addressed 09 clinical diagnostic and treatment questions. Based on these questions, 05 recommendations (01 strong and 04 conditional), 51 points of good clinical practice, and 02 flowcharts were formulated. Conclusion: Evidence-based recommendations were issued for the diagnosis and treatment of persons with hemophilia.
ABSTRACT
Se reportan dos casos de aplasia medular jsevera adquirida, evaluados en el servicio de Pediatría que respondieron satisfactoriamente a la terapia con dosis altas de metilprednisolona, administrado en bolos intravenosos, iniciándose con 25 mg/kg/día disminuyéndolo en un período de cinco semanas. El tratamiento fue bien tolerado por ambos pacientes y no presentaron efectos adversos significativos. En nuestro medio que no contamos con la posibilidad del transplante de médula ósea y de la globulina antilinfocítica, debe considerarse la metilprednisolona como una alternativa terapéutica en los casos de aplasia medular severa.
