We prospectively studied clofarabine-fludarabine-busulfan (CloFluBu)-conditioning in allogeneic hematopoietic cell therapy (HCT) for lymphoid and myeloid malignancies and hypothesized that CloFluBu... Show moreWe prospectively studied clofarabine-fludarabine-busulfan (CloFluBu)-conditioning in allogeneic hematopoietic cell therapy (HCT) for lymphoid and myeloid malignancies and hypothesized that CloFluBu provides a less toxic alternative to conventional conditioning regimens, with adequate antileukemic activity. All patients receiving their first HCT, from 2011-2019, were included and received CloFluBu. The primary endpoint was event-free survival (EFS). Secondary endpoints were overall survival (OS), graft-versus-host disease (GvHD)-relapse-free survival (GRFS), treatment-related mortality (TRM), cumulative incidence of relapse (CIR), acute and chronic GvHD (aGvHD and cGvHD), and veno-occlusive disease (VOD). Cox proportional hazard and Fine and Gray competing-risk models were used for data analysis. One hundred fifty-five children were included: 60 acute lymphoid leukemia (ALL), 69 acute myeloid leukemia (AML), and 26 other malignancies (mostly MDS-EB). The median age was 9.7 (0.5 to 18.6) years. Estimated 2-year EFS was 72.0% +/- 6.0 in ALL patients, and 62.4% +/- 6.0 in AML patients. TRM in the whole cohort was 11.0% +/- 2.6, incidence of aGvHD 3 to 4 at 6 months was 12.3% +/- 2.7, extensive cGvHD at 2 years was 6.4% +/- 2.1. Minimal residual disease-positivity prior to HCT was associated with higher CIR, both in ALL and AML. CloFluBu showed limited toxicity and encouraging EFS. CloFluBu is a potentially less toxic alternative to conventional conditioning regimens. Randomized prospective studies are needed. Show less
Objective. To determine the incidence, management, complications, and outcome in neonates with thrombotic events. Study Design. We performed a retrospective study of all neonates with thrombotic... Show moreObjective. To determine the incidence, management, complications, and outcome in neonates with thrombotic events. Study Design. We performed a retrospective study of all neonates with thrombotic events admitted to our neonatal intensive care unit from January 2004 to July 2010. Results. Thrombotic events were identified in 32 of 4734 neonates (0.7%). Seven neonates were managed expectantly and 25 neonates received anticoagulant treatment. Complete resolution of the clot within 3 months of age was found in 68% (17/25) of the treated and in 86% (6/7) of the nontreated neonates. Major complications due to anticoagulant therapy occurred in 3/25 cases (12%) and included severe hemorrhage (n = 2) and abscess at the injection site (n = 1). Conclusion. Complete or partial clot resolution in neonatal thrombosis occurred in both the treated group and nontreated group. Randomized controlled trials are warranted to determine the optimal management in neonatal thrombosis. Show less
Thromboembolic events in preterm neonates are increasingly being diagnosed due to the increasing use of umbilical catheters and central venous catheters. Whether thromboembolic events should be... Show moreThromboembolic events in preterm neonates are increasingly being diagnosed due to the increasing use of umbilical catheters and central venous catheters. Whether thromboembolic events should be treated routinely with low-molecular-weight heparin (LMWH) is controversial and the optimal management is still not clear due to the lack of randomized controlled trials. Most importantly, knowledge about the safety of treatment with LMWH in neonates with thromboembolic events is very limited. We present a case of severe hemorrhage in a preterm neonate after LMWH treatment and summarize the scarce data reported in the literature. Copyright (C) 2010 S. Karger AG, Basel Show less
