Successful Bone Marrow Transplantation With Intensive Post-transplant Intrathecal Chemotherapy for CNS Relapsed AML in 2 Infants.

BACKGROUND: Infant acute myeloid leukemia is a rare but aggressive form of leukemia. OBSERVATION: We report 2 children who presented with hyperleukocytosis, subsequently diagnosed with infant acute myeloid leukemia, and both developed isolated central nervous system relapse while on chemotherapy. Both infants underwent successful bone marrow transplantation with myeloablative conditioning (thiotepa, busulfan, and cyclophosphamide) without radiation, followed by 12 empiric post-transplant lumbar punctures with intrathecal cytarabine. Both patients tolerated these therapies well, and are without infections, chronic graft-versus-host disease, or any post-transplant sequelae. CONCLUSION: Nonradiation-based conditioning followed by empiric central nervous system-directed intrathecal chemotherapy may be considered for high-risk infants with leukemia.

Alloimmune Neutropenia in a Neonate: Case Report and Review of Literature.

Neonatal alloimmune neutropenia, variably referred to in the literature as NAIN, FNAIN or NIN, is a disorder of neutrophil destruction in newborns similar to better-known conditions such as hemolytic disease of the newborn and neonatal alloimmune thrombocytopenia (FNAIT). Infants affected by this self-limiting condition can present asymptomatically or have a wide range of symptoms, from skin manifestations and mucositis to severe infections such as sepsis and pneumonia. In our case, we report an otherwise asymptomatic term infant born with severe neutropenia to a mother affected by COVID-19 in the 3rd trimester. However, it is unclear if COVID-19 contributed to our patients' neutropenia. Diagnostic testing eventually revealed the presence of anti-neutrophil antibodies, confirming the diagnosis of alloimmune neutropenia. The infant was conservatively managed with early discharge prior to resolution of neutropenia and close post-discharge follow up.

Elevation of tricuspid regurgitant jet velocity, a marker for pulmonary hypertension in children with sickle cell disease.

BACKGROUND: Pulmonary hypertension (PHTN) is a potentially life-threatening complication, detected by echocardiographic evidence of elevated tricuspid regurgitant velocity (TRV). This condition has been described in adults with sickle cell disease (SCD) and other hemolytic disorders; however, there is little information on the occurrence of this condition in pediatric patients. METHODS: Records for pediatric SCD patients were retrospectively reviewed to determine clinical characteristics and co-morbidities of patients with elevated TRV on echocardiograms obtained under steady state conditions as an outpatient. Correlation of TRV > or =2.5 m/sec with age, sex, type of SCD, number of outpatient echocardiograms per patient, episodes of vasoocclusive crisis (VOC) and acute chest syndrome (ACS), mean hemoglobin and reticulocyte count, asthma, obstructive sleep apnea, cerebrovascular disease (CVD), and hydroxyurea therapy was determined. RESULTS: Of 224 SCD patients, 44 had outpatient echocardiographic measurement of TRV. Patients (11 of 44) (26.2%) with TRV > or =2.5 m/sec were compared to 31 patients without elevated TRV. Significant differences were noted for percent with HbSS disease (P = 0.041), CVD (P = 0.021), hemoglobin (P = 0.003), % reticulocytes (P = 0.037), and number of echocardiograms performed (P < 0.001). No significant differences were observed for gender, age, asthma, or frequency of VOC and ACS. CONCLUSIONS: Elevated TRV, a surrogate marker for PHTN, occurs in children with SCD and is associated with low hemoglobin, elevated reticulocyte count, and cerebral vasculopathy. Appropriate screening by echocardiography can lead to detection and treatment that may reduce TRV and potentially reverse the disease process, prevent the increased morbidity and mortality associated with PHTN.