Safety and efficacy of cryoprecipitate-poor plasma as a replacement fluid for therapeutic plasma exchange in thrombotic thrombocytopenic purpura: a single center retrospective evaluation.

INTRODUCTION: Thrombotic thrombocytopenic purpura (TTP) is a thrombotic microangiopathy caused by decreased activity of ADAMTS13, resulting in reduced clearance of ultralarge von Willebrand factor (VWF) multimers. Treatment of TTP is therapeutic plasma exchange (TPE) with replacement with fresh frozen plasma (FFP). Cryoprecipitate-poor plasma (CPP) is a plasma product with lower concentrations of large VWF multimers, and similar amounts of ADAMTS13. CPP is regarded as at least as efficacious as FFP in TTP but evidence of additional benefits has not been demonstrated. Furthermore, there are limited data on the frequency of adverse events associated with CPP. MATERIAL AND METHODS: In our center, the choice between CPP and FFP is performed before the 1st TPE session at the physicians' discretion. Here, we retrospectively evaluated the efficacy and safety of CPP based on the number of sessions, volume of plasma exposure, frequency of exacerbations/relapses, and adverse events. RESULTS: Fourteen patients with newly diagnosed TTP were included in this analysis. The proportion of CPP:FFP use was 5:9. There were no significant differences in age, gender, initial hemoglobin, platelet count, LDH, or etiology of TTP between groups. We observed a trend toward a higher number of TPE sessions and higher plasma exposure in CPP, compared to FFP-treated patients. Acute exacerbations were more frequent among patients treated with CPP (OR 26.6; 95%CI 1.01-703.51; P = 0.03). Mild allergic reactions were the most common treatment-related adverse event in both groups. DISCUSSION: Our data suggest that CPP should not be used as 1st line treatment for newly diagnosed TTP patients.

Diagnosis and treatment of congenital hemophilia with inhibitors: A Latin American perspective / Diagnóstico y tratamiento de la hemofilia congénita con inhibidores: Una perspectiva latinomericana

The Committee of Latin America on the Therapeutics of Inhibitor Groups (CLOTTING) is composed of a number of hemophilia specialists from Latin America. The group aims to encourage the adoption of a good standard of care for Latin American patients with hemophilia. The occurrence of inhibitors in patients with hemophilia poses clinical challenges, and it is estimated that between 1000 and 3 000 patients in Latin America are affected by hemophilia with inhibitors. There is an urgent need to establish a regional consensus and clinical guidelines for the diagnosis and treatment of these patients. We present an extensive review based on best current clinical practice and published literature, as seen from a Latin American perspective, taking into account the variable nature of hemophilia care available in the various countries in this Region.

El Comité Latinoamericano sobre la Terapéutica de Personas con Inhibidores (CLOTTING) está compuesto por un grupo de especialistas en hemofilia de Latinoamérica. El objetivo del grupo es promover la adopción de un estándar de tratamiento óptimo para los pacientes con hemofilia en Latinoamérica. La prevalencia de inhibidores en pacientes con hemofilia en Latinoamérica determina desafíos clínicos y se estima que de 1000 a 3000 pacientes en esta región están afectados con hemofilia e inhibidores. Existe una necesidad urgente de establecer un consenso regional y guías clínicas para el diagnóstico y tratamiento de estos pacientes. Nosotros presentamos una revisión exhaustiva basada en las mejores prácticas clínicas vigentes y en los datos publicados en la literatura, con una perspectiva latinoamericana, tomando en cuenta la variabilidad existente de los tratamientos de la hemofilia disponibles en los diferentes países de esta Región.