Abstract Pediatric ischemic stroke still represents a burden, and more than half of the survivors will experience cognitive or motor disabilities. The objective of this study was to investigate the ...role of thrombophilia in a cohort of children with arterial ischemic stroke. The records of infants and children with clinically and radiologically confirmed stroke were reviewed. Patients with venous or perinatal stroke were not included. Thirty-three patients were diagnosed with arterial ischemic stroke. The male/female ratio was 1.75:1. The median age was 4 years. The most frequent clinical manifestations were hemiparesis (54.5%) and seizures (33.3%). Genetic thrombophilia testing was available on 24 patients. Nine of the 24 patients (37.5%) were heterozygous for factor V Leiden. None of the patients carried the factor II G20210A variant. Ten patients (41.7%) were heterozygous and 3 (12.5%) were homozygous for methylenetetrahydrofolate reductase (MTHFR) C677T variant. Fifteen patients (62.5%) had one or more genetic polymorphism. Factor V Leiden was significantly associated with arterial ischemic stroke ( P < 0.001). Stroke recurred in 2 children with multiple risk factors and MTHFR C677T mutation. Factor V Leiden is one of the major genetic risk factors for pediatric arterial ischemic stroke in Lebanon. MTHFR C677T was prevalent among patients with recurrent stroke.
Abstract Background Optimizing conditioning and post-transplant intervention may reduce non-relapse mortality and relapse, improving survival after allogeneic hematopoietic cell transplantation. ...Materials and Methods We used a risk-adapted intensity of busulfan at 130mg/m2 /day for either 2, 3 or 4 days, with a fixed dose of fludarabine (30mg/m2 /day for 5 days), and thymoglobulin (2.5mg/kg/day for 2 days). Our algorithm was based on age, co-morbidity(ies) and disease risk. Results Fifty-three patients with hematological malignancies, median age, 37 (16-65) years, received an allograft from HLA identical siblings. Post-transplant therapy was initiated between days 30 and 60 after allo-HCT. Twenty-five of 26 patients who were planned for post allo-HCT therapy received it (10 with myeloid malignancies received 5-azacytidine, 5 with FLT-3 ITD acute myeloid leukemia received sorafenib, 4 with Ph+ALL or CML in blast crisis received dasatinib, or dasatinib followed by imatinib and 5 with ALL received intrathecal cytarabine). The remaining 27 patients (51%) did not receive post-transplant therapy because of lack of approval by third-party payers. After a median follow-up of 13 (2-57) months, 1-year NRM was 2% and cumulative incidences of grade 2 to 4 acute GVHD and all grades chronic GVHD were 23% and 9%, respectively. The 2-year overall survival (95% vs 61%; p=0.04) and progression-free survival (81% vs 53%; p=0.05) were significantly better for patients in the post-transplant therapy group. Conclusion This risk-adapted combined approach of selecting conditioning intensity and integrating post-transplant therapies results in lower NRM and encouraging improvement in survival. Our findings warrant confirmation in a large prospective multicenter trial.
