PH negative acute lymphoblastic leukemia in adolescents and young adults treated according a MRD adapted BFM ALL IC 2009 protocol: Argentine real-world data on 171 patients.

Intensified pediatric chemotherapy regimens to treat adolescents and young adults (AYA) patients with Philadelphia negative acute lymphoblastic leukemia (ALL) have been associated with better outcomes. The local BFM 2009-based scheme complements the risk stratification assessing the measurable residual disease (MRD) along the induction phase with increasing levels of sensitivity. The present retrospective multicenter analysis included 171 AYA (15-40 years) patients treated accordingly between 2013 and 2019. Ninety-one percent obtained morphological complete remission, 67% a negative (<0.1%) MRD at day 33 (TP1), and 78% a negative (<0.01%) MRD at day 78 (TP2). The overall survival (OS) and the event-free survival (EFS) at 2 years were 62%±4.1 and 55%±4.1, respectively. The OS and EFS were significant better for prednisone responders, who achieved <10% BM blast at day 15, a negative MRD at TP1 or at TP2, and for low-risk patients. Age ≤30 years and WBC <30×109/L, particularly among B-phenotype, were also associated with longer OS. In the multivariable analyses, TP1 MRD positive (OS HR 2.8, 95% CI 1.4-5.7, p=0.004; EFS HR 3.0, 95% CI 1.6-5.7, p=0.001) and at TP2 (OS HR 2.6, 95% CI 1.3-5.3, p=0.012; EFS HR 2.6, 95% CI 1.3-5.1, p=0.006) were independently associated with earlier events. Age >30 years was also associated with a shorter survival (HR 3.1, 95% CI 1.3-7.5, p=0.014). Therefore, those 68 patients ≤30 years with TP1/TP2 negative MRD depicted a longer OS (2 years 85%±4.8). Based on our real-world data, the pediatric-based scheme is feasible in Argentina associated with better outcomes for younger AYA patients who achieved negative MRD at day 33 and 78.

Síndrome Hipereosinofílico / Hypereosinophilic Syndrome

Los síndromes hipereosinofílicos comprenden un grupo heterogéneo de desórdenes caracterizados por una marcada eosinofilia en sangre periférica y/o tisular que resulta en el daño de diferentes órganos blanco. En los últimos 10 años se han producido innumerables avances en los campos de la biología molecular y la inmunología, lo que ha llevado a la identificación de diferentes subtipos de síndromes hipereosinofílicos genéticamente definidos con anomalías moleculares recurrentes y con diferencias a nivel de la epidemiología, patogénesis y pronóstico. La habilidad para distinguir entre estos subtipos de síndromes, combinado con la disponibilidad de nuevas modalidades terapéuticas, incluyendo los inhibidores de tirosina kinasa y anticuerpos monoclonales ha modificado dramáticamente el enfoque diagnóstico y terapéutico y ha permitidorealizar clasificaciones más específicas desde el punto de vista gen ético de estos desórdenes. El propósito de este trabajo es exponer las características clínicas y las diferentes etiologías del síildrome hipereosinofílico y hacer énfasis en los recientesconocimientos acerca de la patogénesis molecular, en las estrategias di agnósticas y en el tratamiento.