Allogeneic hematopoietic stem cell transplantation in congenital hemoglobinopathies with myeloablative conditioning and rabbit anti-thymocyte globulin

BACKGROUND: Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative therapy for β-thalassemia major (TM) and sickle cell disease (SCD) in children. Graft-versus-host disease (GVHD) and treatment-related mortality (TRM) remain significant challenges to improving survival after HSCT. Here, we analyzed the outcome of TM and SCD patients, who received allogeneic HSCT with myeloablative conditioning at our institution. METHODS: Twenty-two patients (15 TM, 7 SCD), with a median age of 9 years (range, 1.6–16.9), underwent allogeneic HSCT using busulfan, cyclophosphamide and rabbit anti-thymocyte globulin-based conditioning. Cells were derived from either the bone marrow (8 patients), or peripheral blood stem cells (14 patients). The majority of patients received HSCT from a matched sibling donor (N=18). GVHD prophylaxis included cyclosporine and short course methotrexate. RESULTS: All patients achieved donor engraftment. Two SCD patients died from TRM-related grade IV gut GVHD (N=1) or severe bronchiolitis obliterans (BO) (N=1). Cumulative incidence of acute and chronic GVHD was 36.4% and 32.7%, respectively. Veno-occlusive disease (VOD) occurred in 8 patients (36.4%), but resolved in all instances. Epstein-Barr virus (EBV)-related post-transplantation lymphoproliferative disease (PTLD) occurred in 1 patient. The overall survival (OS) was 90.9% (TM 100%, SCD 71.4%), with all patients achieving transfusion independence, while 8 achieved complete donor chimerism. CONCLUSION: Busulfan, cyclophosphamide, and ATG-based conditioning for HSCT of TM and SCD patients did not result in graft failure, although modifications may be required to reduce VOD incidence. Further changes to donor type and cell source prioritization are necessary to minimize TRM and morbidity caused by GVHD.

Descending Aorta Blood Flow Characteristics before the Development of Necrotizing Enterocolitis in Preterm Neonates

PURPOSE: To investigate the hemodynamic risk factors for necrotizing enterocolitis (NEC), we analyzed the characteristics of descending aorta (DA) blood flow in preterm neonates, who later developed NEC. METHODS: This was an observational case-control study on 53 preterm neonates at a tertiary referral center. Clinical and echocardiographic data were collected from 23 preterm neonates with NEC (NEC group), and compared with those of 30 preterm neonates without NEC (control group). Echocardiography was done at a median (interquartile range) of 5 (3–9) days after birth and 2 (1–2.5) days before the diagnosis of NEC. RESULTS: Basic clinical characteristics including gestational age, birth weight, Apgar score, breast feeding status, use of umbilical catheters, and mode of invasive ventilator care were similar between the groups. Compared with the control group, the lowest diastolic velocity of DA was significantly decreased, whereas the diastolic reverse flow and the ratio of diastolic reverse to systolic forward flows were significantly increased in the NEC group. In addition, the resistive index (RI) of DA was significantly increased in the NEC group and showed a positive association with the development of NEC. Multivariate logistic regression analysis showed that increasing RI of DA was an independent risk factor for the development of NEC (P=0.008). CONCLUSION: Significant changes in DA flow characteristics including decreased diastolic velocity and increased diastolic reverse flow along with increased peripheral vascular resistance were observed before the development of NEC in preterm neonates. These findings may help clinicians stratify in advance neonates at a risk of developing NEC and may help improve outcomes in these neonates.

The Value of Routine Chest Radiography in Children

To assess the value of routine chest roentgenographic examinations at the time of admission, we analysed chest roentgenogram of 1,283 patients under the age of 15 who were admitted to St. Paul's Hospital from 1975 to 1977. A bonormal radiographic findings were detected in 111out of 1,283 Patients(8.7%). In 108 patients with abnormal findings, no medical or surgical therapy was required except three nowborn infants who had medical therapy as a result of pneumonic infiltration. In view of clinical analysis of 1,283 patients, it is considered that routine chest roentgenographic examinations were not warranted in patients under the age of 15 but justifiable in newborn infants.

Feeding Results of Maeil Dry Milk G-80 in Low Birth Weight Infants

The low birth weight infants have been fed with 15% concentration of Maeil Dry Milk G-80(Table 1) until the date of weight gaining to 2.5kg at the nursery room of our pediatric department, St. Mary's Hospital, Catholic Medical College. Feeding results were obtained as follows. 1. General physical condition was fair. The initial weight loss was average 6.8% of their birth weight and regaining of body weight started on 12th day of age(Table 2). 2. The frequency of stool passage was 1~6 times a day and normal yellowish stools were appeared on 8th day of age(Table 3). 91% of stools was the normal consistency and the other was loose stool. Mean value of stool pH examined on 10th day of age was 5.54(4.9~6.5) 3. Mean daily feeding amount of water, Calorie, protein and fat was 175ml, 131cal,3.4gm and 6.3gm per kg respectively(Table 4).