PURPOSE:The Children's Oncology Group (COG) protocol AALL0434 evaluated the safety and efficacy of multi-agent chemotherapy with Capizzi-based methotrexate/pegaspargase (C-MTX) in patients with newly diagnosed pediatric T-cell lymphoblastic lymphoma (T-LL) and gained preliminary data using nelarabine in high-risk patients. PATIENTS AND METHODS:The trial enrolled 299 patients, age 1-31 years. High-risk (HR) patients had ≥ 1% minimal detectable disease (MDD) in the bone marrow at diagnosis or received prior steroid treatment. Induction failure was defined as failure to achieve a partial response (PR) by the end of the 4-week induction. All patients received the augmented Berlin-Frankfurt-Muenster (ABFM) C-MTX regimen. HR patients were randomly assigned to receive or not receive 6 5-day courses of nelarabine incorporated into ABFM. Patients with induction failure were nonrandomly assigned to ABFM C-MTX plus nelarabine. No patients received prophylactic cranial radiation; however, patients with CNS3 disease (CSF WBC ≥ 5/μL with blasts or cranial nerve palsies, brain/eye involvement, or hypothalamic syndrome) were ineligible. RESULTS:At end-induction, 98.8% of evaluable participants had at least a PR. The 4-year event-free survival (EFS) and overall survival (OS) were 84.7% ± 2.3% and 89.0% ± 2.0%. The 4-year disease-free survival (DFS) from end-induction was 85.9% ± 2.6%. There was no difference in DFS observed between the HR and standard-risk groups (P = .29) or by treatment regimen (P = .55). Disease stage, tumor response, and MDD at diagnosis did not demonstrate thresholds that resulted in differences in EFS. Nelarabine did not show an advantage for HR patients. CNS relapse occurred in only 4 patients. CONCLUSION:COG AALL0434 produced excellent outcomes in one of the largest trials ever conducted for patients with newly diagnosed T-LL. The COG ABFM regimen with C-MTX provided excellent EFS and OS without cranial radiation.
Truncus arteriosus most often has absence of a patent ductus arteriosus unless there is concomitant aortic arch interruption. To date, there are no reports of fetal echocardiography visualizing a patent ductus arteriosus in truncus arteriosus without arch interruption. Here, we present the case of a fetus prenatally diagnosed with truncus arteriosus type 1 with a patent ductus arteriosus noted on fetal echocardiography. Visualizing this in-utero allowed for proper surgical planning and ligation during truncus arteriosus repair and excellent outcome.
To compare the clinical outcomes of posterior spinal fusion (PSF) and traditional growing rod (TGR) surgery for neurofibromatosis type 1-associated dystrophic scoliosis (NF1-DS) in children aged 8–11 years. The aim is also to identify the factors that influence surgical selection and spinal growth. Patients with NF1-DS and major thoracic curves involving at least five vertebral levels were enrolled and divided into PSF and TGR groups. Demographic, radiographic and surgical data were analyzed for both a 1:1 propensity score-matched cohort (n = 26) and the full cohort (n = 39). Logistic regression was used to identify factors influencing surgical selection, and Spearman correlation was used to analyze spinal growth predictors. PSF achieved greater initial curve correction than TGR (61.0
Importance:Platelet transfusions are critical interventions for neonates and children who are at risk of or who are experiencing bleeding. Objective:To describe the epidemiology of platelet transfusions and associations of blood donor and platelet characteristics with posttransfusion platelet increments, transfusion burden, and clinical outcomes in neonates and children. Design, Setting, and Participants:This cohort study included patients younger than 18 years of age (with birth weights higher than 2500 g) between April 1, 2019, and June 30, 2023, from the Recipient Epidemiology and Donor Evaluation Study-IV-Pediatric Vein-to-Vein multicenter, retrospective, donor-component-recipient-linked database. Neonates were considered individuals less than 28 days of age; otherwise, participants were considered older children. Exposure:Platelet transfusion. Main Outcome and Measures:The main outcomes were posttransfusion platelet increments and subsequent transfusion burden associated with platelet processing and donor characteristics. Results:Of 249 340 inpatient encounters, platelet transfusion was reported in 8874 (3.6%) patients (4934 of 131 592 encounters [3.7%] male; median [IQR] age 2.5 [0.6-11.2] years). Platelet transfusion was lowest among children younger than 1 year of age (2.6%) and highest among children 1 to less than 6 years of age (4.7%; P < .001). The median (IQR) dose was 14.9 mL/kg for neonates and 9.6 mL/kg for older children. After excluding patients with bleeding, most transfusions in neonates (67.8%) and older children (81.0%) were performed at pretransfusion platelet counts greater than 25 × 103/µL and greater than 10 × 103/µL, respectively. Median (IQR) pretransfusion platelet counts in neonates (34 × 103/µL [20-54 × 103/µL]) were significantly higher than in older children (22 × 103/µL [11-40 × 103/µL]; P < .001). Pathogen reduction (PR) (adjusted odds ratio [AOR], 0.82 [95% CI, 0.73-0.92]), use of platelet additive solution (PAS) (AOR, 0.32 [95% CI, 0.27-0.37]), platelet storage duration longer than 3 days (AORs ranged from 0.67 [95% CI, 0.58-0.76] to 0.82 [95% CI, 0.76-0.88]), male sex (AOR, 0.92 [95% CI, 0.86-0.98]), and donor age 40 years or older (AOR, 0.79 [95% CI, 0.72-0.86]) were associated with lower platelet increments (all P < .001). Use of PR platelets (adjusted rate ratio [ARR], 1.05 [95% CI, 1.02-1.07), use of PAS (ARR, 1.44 [95% CI, 1.40-1.47]), storage duration longer than 3 days (ARR, 1.11 [95% CI, 1.09-1.13] for 4 to <5 days and ARR, 1.28 [95% CI, 1.26-1.30] for ≥5 days), and donor age of 40 years or older (ARR, 1.15 [95% CI, 1.13-1.17] for 40 to <60 years and ARR, 1.10 [95% CI, 1.08-1.12] for ≥60 years) on the first transfusion were associated with a significantly higher rate of receiving a subsequent transfusion. These factors were not associated with hospital length of stay or mortality. Conclusions and Relevance:In this multicenter, donor-product-recipient linked cohort study, after excluding patients with bleeding, most transfusions among neonates and older children were at high pretransfusion platelet counts. Use of PAS, PR, longer storage duration, male donors, and donor age older than 40 years were independently associated with lower posttransfusion platelet increments. Use of PAS, PR, platelet storage longer than 3 days, and donor age 40 years or older were associated with a significantly higher overall platelet transfusion burden but were not associated with hospital length of stay or mortality. These results have important implications for transfusion practices for platelets among neonates and children and need validation in well-designed prospective studies.
Complex venous outflow reconstruction in living donor liver transplantation (LDLT) is technically demanding, particularly in resource-limited settings lacking consistent access to synthetic or cryopreserved grafts. We retrospectively analyzed 45 consecutive LDLTs performed during the initiation of a national program. Venous anatomy was evaluated using preoperative CT volumetry and intraoperative findings. Reconstruction strategies included direct anastomosis, unification venoplasty, PTFE grafts, and autologous conduits (falciform ligament, umbilical vein). Outcomes were compared between patients with (n = 17) and without (n = 28) venoplasty. Additional venous reconstruction was required in 37.8% of cases. In 6.7%, anatomically indicated veins could not be reconstructed due to lack of suitable conduits. No early venous thrombosis occurred, and all autologous conduits remained patent during follow-up. Small-for-size physiology developed in 11.1% of recipients, resolved conservatively, and was not associated with unreconstructed major veins. Major morbidity (Clavien–Dindo ≥ IIIb) occurred in 42.2%. The 90-day mortality rate was 11.1%, and 3-year survival was 82.2%, without significant differences between groups. In a newly established program within a resource-limited setting, predominantly autologous venoplasty was feasible and provided satisfactory early and mid-term outcomes.
