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Serial cardiovascular magnetic resonance evaluation of children and young adults with SCD who underwent hematopoietic cell transplantation showed mean ECV, representing diffuse myocardial fibrosis, decreased 3.4% from baseline to 12 months posttransplantation. This trial was registered at www.clinicaltrials.govas #NCT04362293.

Evidence of the effectiveness of prophylactic use of tranexamic acid (TXA) in thrombocytopenia is lacking. To determine whether TXA safely reduces bleeding incidence in patients undergoing treatment for hematologic malignancies, a randomized, double-blind clinical trial was conducted from June 2016 through June 2020. Of 3120 screened adults, 356 patients were eligible and enrolled, and 337 patients (mean age, 53.9; 141 [41.8%] women), randomized to 1300 mg TXA orally or 1000 mg TXA through IV (n = 168) vs placebo (n = 169) thrice daily for maximum 30 days. Three hundred thirty patients were activated when their platelet counts fell below 30 000 per µL; 279 (83%) had complete outcome ascertainment. World Health Organization (WHO) grade ≥2 bleeding was observed in the 30 days following activation in 50.3% (73/145) and 54.2% (78/144) of patients in the TXA and placebo groups, with an adjusted odds ratio of 0.83 (95% confidence interval [CI], 0.50-1.34; P = .44). There was no statistically significant difference in the mean number of platelet transfusions (mean difference, 0.1; 95% CI, −1.9 to 2.0), mean days alive without grade ≥2 bleeding (mean difference, 0.8; 95% CI, −0.4 to 2.0), thrombotic events (6/163 [3.7%] TXA, 9/163 [5.5%] placebo), or deaths due to serious bleeding. Most common adverse events were: diarrhea (116/164 [70.7%] TXA and 114/163 [69.9%] placebo); febrile neutropenia (111/164 [67.7%] TXA, 105/163 [64.4%] placebo); fatigue (106/164 [64.6%] TXA, 109/163 [66.9%] placebo); and nausea (104/164 [63.4%] TXA, 97/163 [59.5%] placebo). Among patients with hematologic malignancy undergoing chemotherapy or hematopoietic stem cell transplantation, prophylactic treatment with TXA compared with placebo did not significantly reduce the risk of WHO grade ≥2 bleeding.

Acute megakaryocytic leukemia (AMKL) comprises 4-15% of all acute myeloid leukemia (AML) diagnoses in pediatric patients. Unlike patients with Down syndrome who have a >80% expected survival, the 5-year overall survival (OS) in patients with de novoAMKL ranges between 14-49%. While treatment intensification has improved the outcome of these patients over the last few decades, the role of hematopoietic cell transplantation (HCT) in these patients in complete remission (CR) is often debated. To better define the role of HCT, we evaluated the outcomes of pediatric patients who underwent their first HCT at our institution from October 1990 to January 2021.

Evidence of effectiveness of prophylactic use of tranexamic acid (TXA) in thrombocytopenia is lacking. To determine whether TXA safely reduces bleeding incidence in patients undergoing treatment for hematologic malignancies, a randomized double blind clinical trial was conducted June 2016 through June 2020.

•Subsequent HCT can provide a benefit for a subset of patients who relapse post-HCT•Disease status and interval between HCT episodes are associated with outcome•Haploidentical donors are an acceptable option for second HCT

Introduction

Introduction: Cardiovascular complications are a leading cause of early mortality in patients with sickle cell disease (SCD). Chronic anemia, iron overload and repeated microvascular ischemic insults with reperfusion injury often result in progressive myocardial injury and maladaptive remodeling in SCD patients. Patients with SCD develop diffuse myocardial fibrosis, which can be detected by quantifying the myocardial extracellular volume (ECV) using cardiovascular magnetic resonance (CMR) imaging. Our group and others have previously shown that diffuse myocardial fibrosis is highly prevalent in both children and young adults with SCD, including those who have normal ventricular size and function by echocardiography. Additionally, we have shown that the disease modifying therapies (DMT) such as hydroxyurea or chronic transfusions do not appear to be protective against development of diffuse myocardial fibrosis. In a retrospective study, we found no difference in the myocardial ECV in patients who began DMT early (less than 6 years of age) versus later in life (at least 6 years or after) (Morin CE and Sharma A et al. Blood 2023.). Hematopoietic cell transplantation (HCT) can cure SCD, but myocardial effects of the therapy are unknown. Our goal was to evaluate cardiovascular parameters of function and injury, especially the progression of diffuse myocardial fibrosis, by comparing serial CMR studies performed before and after HCT in patients with SCD.

Given the increase in antimicrobial drug resistance of several pathogens that affect the ear, there is a need for alternative therapy for canine otitis externa (OE). We hypothesised that the efficacy of modified Burow's solution would be non‐inferior to that of standard treatment with Surolan. The treatment success rate of a topical aluminum acetate 2% and betamethasone 0.1% (modified Burow's) otic solution was compared with that of a standard topical otic treatment of polymyxin B sulfate, prednisolone acetate and miconazole (Surolan) suspension in a prospective, open‐label clinical trial. For 10 days, 142 dogs (232 ears) with OE were treated with modified Burow's solution or Surolan. The primary study endpoint was the cure rate at 10 days. Clinical efficacy was analysed as a secondary endpoint but without assessment for non‐inferiority. The 10‐day OE cure rate was 65% and 59% in the Burow's and Surolan groups, respectively. The modified Burow's solution was non‐inferior to Surolan at a non‐inferiority margin of –11.5% and was more clinically effective than Surolan in dogs with OE caused by yeast or bacteria. The results of this study demonstrate that modified Burow's solution is an effective treatment for OE in dogs.