High-grade dysplastic spondylolisthesis (HGDS) is a rare pediatric spinal deformity characterized by severe lumbosacral dysplasia and sagittal malalignment. The optimal surgical strategy for achieving deformity correction while minimizing neurological risk remains controversial. To evaluate radiographic and clinical outcomes of sacral dome osteotomy combined with complete L5 reduction and single-level posterior L5–S1 fusion in pediatric patients with HGDS, and to explore factors associated with residual postoperative sagittal imbalance. A multicenter retrospective case-series study. Thirty-one patients (30 females, 1 male; mean age 9.66 ± 2.27 years) with L5 high-grade dysplastic spondylolisthesis who underwent sacral dome osteotomy, complete L5 reduction, and posterior L5–S1 single-level fusion between 2008 and 2023. The primary outcome was residual sagittal imbalance at the latest follow-up, defined as sagittal vertical axis (SVA) > 5.0 cm or pelvic tilt to sacral slope ratio (PT/SS) > 1. These criteria represented global and regional malalignment, respectively. Clinical and radiographic data were retrospectively reviewed. Patients were categorized according to the presence of residual sagittal imbalance at the final follow-up. Univariable and multivariable logistic regression analyses were performed in an exploratory manner to examine associations between selected clinical and radiographic variables and residual sagittal imbalance. Receiver operating characteristic (ROC) curve analysis was conducted to assess the discriminatory ability of preoperative sagittal vertical axis within this cohort. Mean follow-up was 3.05 ± 2.01 years. Slip percentage improved from 61.0 ± 11.0 preoperatively to 13.0 ± 10.0 postoperatively (p < 0.001) and remained stable at 9.0 ± 12.0 at the latest follow-up. Lumbosacral and global sagittal alignment parameters demonstrated significant correction that was largely maintained over time. Seven patients (22.6

BackgroundAntigen escape and PD-L1/PD-1 axis-mediated immunosuppression in the tumor microenvironment (TME) are the predominant drivers of treatment failure in adult patients with relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL). CD19-targeted CAR-T monotherapy has limited ability to overcome these two core resistance mechanisms, and achieving long-term durable remission remains a critical unmet clinical need in adults with high-risk, chemorefractory B-ALL.MethodsThis is a single-center, retrospective case report of an adult patient with early-relapsed, chemorefractory B-ALL. Two independent CAR-T products were manufactured from the patient’s autologous peripheral blood mononuclear cells (PBMCs): (1) CD19 CAR-T cells containing an anti-CD19 single-chain variable fragment (scFv), a CD28 transmembrane domain, a 4-1BB co-stimulatory domain, and a CD3ζ signaling domain; (2) PD-L1-armored CD22 CAR-T cells containing an anti-CD22 scFv, a CD8 transmembrane domain, a 4-1BB co-stimulatory domain, a CD3ζ signaling domain, and a membrane-tethered anti-PD-L1 scFv for spatially restricted immune checkpoint modulation. The two CAR-T products were co-infused at doses of 5.0×105 cells/kg (CD19 CAR-T) and 3.1×105 cells/kg (PD-L1-armored CD22 CAR-T), respectively. We evaluated the feasibility, anti-leukemic efficacy, and long-term safety profile of this regimen as a bridging strategy to allogeneic hematopoietic stem cell transplantation (allo-HSCT). This treatment was administered under an institutional compassionate use program approved by the Ethics Committee of the Second Hospital of Hebei Medical University (approval number: 2017-R207), with written informed consent obtained from the patient prior to all treatment procedures. Comprehensive diagnostic workup for B-ALL was performed using 8-color multiparameter flow cytometry (MFC), conventional G-banding cytogenetic analysis, and multiplex leukemia fusion gene screening. Serial lumbar punctures with triple intrathecal chemotherapy (dexamethasone 5 mg + methotrexate 10 mg + cytarabine 30 mg) were performed throughout the treatment course; no abnormalities were detected in cerebrospinal fluid (CSF) routine, biochemistry, or flow cytometry assays, and no evidence of central nervous system (CNS) leukemia involvement was observed at any time point. This study is a retrospective observational analysis of a single clinical case, not a prospective interventional clinical trial, and thus was exempt from clinical trial registration requirements in accordance with institutional and national regulatory guidelines for retrospective observational studies.ResultsThe patient achieved minimal residual disease (MRD)-negative complete remission (CR) on day 14 post-infusion, as confirmed by 8-color MFC (detection sensitivity: 0.01%) and next-generation sequencing (NGS) of immunoglobulin heavy chain (IGH) gene rearrangements (limit of detection [LOD]: 10−6). Peak in vivo expansion of CAR-T cells was observed on day 10 post-infusion, with CD19 CAR-T cells accounting for 43.25% and CD22 CAR-T cells accounting for 14.58% of circulating CD3+ T lymphocytes. Only grade 1 cytokine release syndrome (CRS), per the American Society for Transplantation and Cellular Therapy (ASTCT) consensus criteria, occurred and resolved completely with supportive care; no immune effector cell-associated neurotoxicity syndrome (ICANS) was observed. Following consolidative allo-HSCT, rapid hematopoietic reconstitution was achieved, with neutrophil engraftment on day +12 and platelet engraftment on day +14, consistent with the median engraftment timeline for haploidentical HSCT at our institution. Complete donor chimerism (99.86%) was confirmed by short tandem repeat (STR) analysis on day +30 post-transplantation. Notably, the patient maintained MRD-negative sustained remission for 7 consecutive years, with no occurrence of acute or chronic graft-versus-host disease (GVHD) or late treatment-related adverse events. Complete immune reconstitution was achieved by 24 months post-transplantation, with sustained functional immune recovery and a Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) total score of 158/172 at the 7-year follow-up.ConclusionsThis case report details the clinical course of an adult patient with early-relapsed, chemorefractory B-ALL who achieved 7-year MRD-negative sustained remission after treatment with PD-L1-armored CD19/CD22 dual-targeted CAR-T cell co-infusion followed by consolidative allo-HSCT. Our preliminary clinical observation demonstrates that this integrated regimen may mitigate antigen escape and immunosuppressive TME-mediated drug resistance, and effectively function as a bridging strategy to allo-HSCT in high-risk patient populations. The 7-year event-free survival (EFS) and sustained disease control observed in this case provide valuable clinical insights for the structural optimization of armored CAR-T constructs and the design of future prospective clinical trials for R/R B-ALL.

[This corrects the article DOI: 10.3389/fimmu.2025.1675328.].

Atlantoaxial instability (AAI) is a common but potentially severe complication in pediatric patients with Down syndrome, while its surgical characteristics and outcomes remain understudied compared with non-Down syndrome populations. To compare the clinical presentation, radiological features, surgical strategies, and postoperative outcomes of AAI between pediatric patients with Down syndrome and matched non-Down syndrome controls. A retrospective case-match study was conducted, including 15 patients with Down syndrome along with AAI who underwent surgical atlantoaxial arthrodesis between 2009 and 2022. Each case was matched with two non-Down syndrome controls by age, sex, and AAI severity. The patients were divided into two groups: the Down syndrome group (group DS) and the control group (group C). Data included clinical presentation, radiographic parameters [atlantodental interval (ADI) and space available for the spinal cord (SAC)], surgical approach, complications, and fusion rates were compared between the two groups. Patients with Down syndrome exhibited a higher incidence of neurological symptoms (12/15, 80%) compared with controls (5/30, 16.7%) (P < 0.05). Os odontoideum was more common in patients with Down syndrome (10/15, 66.7%), while rotatory dislocation was more common in patients with non-Down syndrome (9/30, 30%); nine (60%) in group DS and one (2.9%) in group C had a high-signal area on MRI. Preoperative ADI was larger for group DS compared with group C (9.0 vs. 7.4 mm; P < 0.01). The ADI and SAC were significantly corrected and were comparable at the last follow-up. Preoperative Japanese Orthopaedic Association scores were significantly smaller in group DS compared with group C (13.3 vs. 16.5; P < 0.01). Neurological symptoms were significantly improved in all patients at the last follow-up. All included patients underwent posterior atlantoaxial screw-rod fixation and fusion. Only two patients suffered superficial wound infection at the iliac bone area, and another patient in the neck (20%), and no complications occurred in group C. Solid fusion was shown in all patients by the time of the last follow-up. Pediatric patients with AAI often have os odontoideum and hypoplasia of the dental process. Posterior atlantoaxial screw-rod fixation can result in good fusion and neurological function recovery.

ObjectiveThis study aimed to evaluate the influence of pre-transplant spleen volume on the prognosis of patients with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).MethodsWe evaluated 58 patients diagnosed with AML and who have undergone first allogeneic stem cell transplant at the Department of Hematology, Second Hospital of Hebei Medical University from 2017 to 2022. All patients were consecutively evaluated. Patients with AML evolving from myeloproliferative neoplasms (MPN) or secondary AML were excluded. Only de novo AML patients were included. The median follow-up time was 24 months, and the patients were categorized into non-enlarged spleen volume (NLSV) and large spleen volume (LSV) groups based on the spleen volume ranges of 120 normal individuals. A retrospective analysis was performed to evaluate the impact of spleen volume on post-allo-HSCT outcomes, including the incidence of infection, graft-versus-host disease (GVHD), relapse, overall survival (OS), and non-relapse mortality (NRM). Log-rank test was employed to compare survival curves, and a multivariable Cox regression model was utilized to assess spleen volume as a prognostic factor for long-term survival.ResultsAccording to the survival curve, the LSV group exhibited lower OS compared to the NLSV group (P=0.034), along with higher cumulative NRM (P=0.049) and lower relapse-free survival (P=0.023). No significant differences were observed in disease relapse, granulocyte engraftment, CMV infection, or GVHD incidence. The multivariable regression model confirmed spleen volume as a significant risk factor for OS, with cytomegalovirus (CMV) infection also influencing OS and NRM.ConclusionPre-transplant splenomegaly is independently associated with poor prognosis in AML patients.

To analyze the imaging findings of hemimetameric shift (HMMS) in children and propose a new classification system. The clinical data and imaging studies of 85 HMMS patients aged 2 to 14 years were retrospectively reviewed. HMMS was classified as follows: type 1, two hemivertebrae (HV); type 2, three HV; type 3, four or more HV; and type 4, two or more HV and multiple spinal and/or costal anomalies. Inter- and intraobserver reliability of the classification system were evaluated using Fleiss’ kappa coefficient. Imaging features of HMMS were analyzed using whole-spine standing radiographs, computed tomography and magnetic resonance imaging. HMMS was classified as type 1 in 54 patients, type 2 in 16, type 3 in eight, and type 4 in seven. The overall kappa value for the reliability study was 0.796, indicating that the classification system is simple and consistent. The cranial and caudal compensatory curves, coronal balance, thoracic kyphosis, and lumbar lordosis did not significantly differ between type 1 patients and patients with types 2–4 HMMS. Thoracolumbar kyphosis was significantly greater in patients with types 2–4. Discordant HMMS was found in 27 patients (31.8

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

Background:The optimal timing of ruxolitinib initiation for acute graft-versus-host disease (aGVHD) prophylaxis after myeloablative allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains undefined, particularly within anti-thymocyte globulin (ATG)-based platforms. This study compared immediate versus delayed short-course ruxolitinib initiation using a concurrent two-strategy design. Methods:This single-center, retrospective, concurrent two-strategy cohort study enrolled 39 consecutive patients aged 15-65 years with hematologic malignancies who received standard "Beijing Protocol" backbone plus a 14-day short-course ruxolitinib. Patients were non-randomly assigned to immediate (day +1; n = 19) or delayed (at confirmed neutrophil engraftment; median day +13; n = 20) initiation. Haploidentical transplantation and ATG use were perfectly collinear in this cohort, complicating attribution of outcome differences. Co-primary endpoints were 100-day cumulative incidence of grade II-IV aGVHD and 2-year overall survival (OS). A 1:1 propensity score matching (PSM) analysis (12 pairs) served as a sensitivity analysis. Given the limited sample size, multivariable models were constructed as exploratory analyses and should be interpreted with caution. Results:Delayed initiation was associated with lower 100-day grade II-IV aGVHD (10.0% vs. 42.1%, P = 0.031), lower cytomegalovirus (CMV) reactivation (35.0% vs. 73.7%, P = 0.025), reduced grade ≥3 hematologic toxicity (35.0% vs. 68.4%, P = 0.043), and superior 2-year OS rate (90.0% vs. 57.9%, P = 0.012). PSM confirmed these findings (aGVHD: 8.3% vs. 41.7%, P = 0.039; OS: 91.7% vs. 58.3%, P = 0.028). In the exploratory haploidentical subgroup (n = 26), grade II-IV aGVHD was 11.8% vs. 55.6% (P = 0.028) and OS was 94.1% vs. 55.6% (P = 0.006). In exploratory multivariable models, immediate initiation was associated with higher aGVHD risk [adjusted subdistribution hazard ratio (sHR) 2.31, 95% CI 1.15-4.64, P = 0.019] and higher NRM (adjusted sHR 4.65, 95% CI 1.47-14.72, P = 0.009). Relapse risk was not significantly different (adjusted sHR 2.17, 95% CI 0.85-5.56, P = 0.095). Pre-transplant measurable residual disease (MRD) positivity was associated with inferior OS (adjusted hazard ratio [HR] 7.81, 95% CI 1.12-54.32, P = 0.038). Median follow-up was 42.1 months. Interpretation of the CMV reactivation difference is limited by the absence of donor and recipient CMV serostatus data. Conclusion:Delaying short-course ruxolitinib until neutrophil engraftment was associated with reduced aGVHD and NRM and improved survival after myeloablative allo-HSCT. No statistically significant difference in relapse risk was observed, although a clinically meaningful difference cannot be excluded. These findings warrant confirmation in a multicenter randomized trial with adequate sample size, stratified by donor type and pre-transplant MRD.

This article systematically reviews the management of key complications in hematopoietic stem cell transplantation (HSCT), including infections, graft-versus-host disease (GVHD), and hepatic sinusoidal obstruction syndrome (VOD/SOS). It highlights the importance of optimizing conditioning regimens to reduce infection risk and discusses the role of novel antiviral agents like letermovir in transforming infection control. For GVHD, the pathogenesis involving effector and regulatory T-cell imbalances is analyzed, together with prevention strategies such as post-transplant cyclophosphamide with antithymocyte globulin and TCRαβ/CD19 depletion. Ruxolitinib is emphasized for steroid-refractory GVHD, and gut microbiota modulation is noted as a promising intervention. For VOD/SOS, early biomarker detection and defibrotide treatment are critical. The review also explores the impact of immune reconstitution on infection control, GVHD development, and relapse, and examines how emerging approaches, including single-cell sequencing, microbiome analysis, and artificial intelligence, can be applied in building whole-course risk management models. Future directions include developing intelligent platforms and personalized strategies to enhance long-term patient outcomes.

This study aimed to evaluate synergistic effects and molecular mechanisms of the histone deacetylase inhibitor chidamide combined with the BCL-2 inhibitor venetoclax in diffuse large B-cell lymphoma (DLBCL) cell lines. Human DLBCL cell lines (U2932, SUDHL-4) were cultured in vitro and treated with chidamide and venetoclax. Cell proliferation inhibition rates were measured using the CCK-8 assay, and IC50 values were calculated. Cells were also treated with a 5:1 combination of chidamide and venetoclax, along with p53 or p21 siRNA. Cell viability, HDAC activity, apoptosis, cell cycle, the amount of p53 and p21 proteins, and BCL-2-BIM binding were analyzed via CCK-8, enzyme activity assays, the Caspase 3/7 Activity Apoptosis Assay Kit, flow cytometry, RT-qPCR, western blot, and co-IP. The binding of p53 and p21 was verified by dual-luciferase reporter assay and chromatin immunoprecipitation. Chidamide and venetoclax exhibited dose- and time-dependent anti-proliferative effects in U2932 and SUDHL-4 cells, with IC50 values of 3.54 μM (chidamide) and 0.67 μM (venetoclax) in the SUDHL-4 cell line and 5.6 μM (chidamide) and 0.91 μM (venetoclax) in the U2932 cell line. Combination treatment significantly enhanced HDAC inhibition, histone H3/H4 acetylation, and p53 expression, leading to increased cell apoptosis. p53 knockdown partially reversed these effects and increased BCL-2/BIM complex formation. The combination also upregulated p53 expression to increase p21 expression, inducing G1/S phase arrest, which was partially reversed by p21 knockdown. To conclude, the chidamide-venetoclax combination synergistically activates the p53-p21 signaling pathway, leading to cell cycle arrest and apoptosis, representing a potential therapeutic strategy for DLBCL.

Introduction:This multicenter, open-label Phase Ib/II study evaluated TDI01 in patients with moderate or severe chronic graft-versus-host disease (cGVHD) after 1-5 lines prior therapies. Our previous analysis in 57 evaluable patients showed 24-week best overall response rates (BORRs) of 67.9% (200 mg), 86.2% (400 mg), and 77.2% (total) (EBMT 2025). Here, we presented the updated results with extended follow-up, focusing on response durability, long-term safety, overall survival, pharmacokinetics (PK), and exposure–response (E–R) analysis.Methods:After a safety lead-in, patients with moderate or severe cGVHD who had failed 1–5 lines prior systemic therapies were sequentially assigned to receive TDI01 200 mg once daily (QD) (n = 30) or 400 mg QD (n = 30) until experiencing GVHD progression or unacceptable toxicity (NCT06169722). Data were collected on overall response rate (ORR), duration of response (DOR), failure-free survival (FFS), overall survival (OS), PK, E–R, and long-term safety, and are reported here.Results:As of July 18, 2025, 60 patients were evaluable for safety, PK and E–R, and 57 for efficacy. Median follow-up was 48.1 (95% CI, 48.0, 48.3) weeks and 72% (41/57) of evaluable patients had been previously treated with ruxolitinib. At week 24, ORR was 50.0% in 200 mg cohort and 55.2% in 400 mg cohort. Over the entire treatment course, the overall BORR reached 75.0% in 200 mg cohort and 86.2% in 400 mg cohort. Several subgroups (e.g., severe cGVHD and heavily pretreated patients) showed similarly high best ORR (BORR), which was 80.0%, 80.6%, 86.2%, and 80.5%, respectively, in patients with severe cGVHD, with ≥4 organs involved, with ≥3 prior lines of therapy, and those with prior ruxolitinib treatment. The median DOR was not reached in either cohort, with a lower 95% CI of 23.1 months in 200 mg cohort and 31.0 months in 400 mg cohort. At 9 months, FFS rates were 53.6% and 77.7% in 200 mg and 400 mg cohorts, respectively, while OS rates at 12 months were 88.1% and 92.5%.Organ-specific analyses demonstrated BORRs of 52.6% (10/19) in liver, 50.0% (4/8) in esophagus, 50.0% (4/8) in upper gastrointestinal (GI) tract, 47.6% (10/21) in joints/fascia, 46.3% (19/41) in mouth, 38.1% (16/42) in skin, 35.6% (16/45) in eyes, 18.4% (7/38) in lungs, and 0% (0/1) in lower GI tract. On the modified Lee Symptom Scale, 29.8% (17/57) of the patients had ≥7-point improvement.All patients had ≥1 TEAE and TRAEs occurred in 96.7% of patients. Grade ≥3 TEAEs and TRAEs were reported in 51.7% and 38.3% of patients, respectively. SAEs occurred in 31.7%, including 10% that were considered drug-related. One death occurred in the 400 mg cohort was deemed unrelated to the study drug. The most commonly reported TRAEs (≥20%) were elevations of total bilirubin (81.7%), unconjugated bilirubin (60.0%) and conjugated bilirubin (48.3%), upper-respiratory infections (25.0%), headache (23.3%), diarrhea (20.0%), and cough (20.0%). Grade ≥3 hyperbilirubinaemia occurred in 16.7% of patients, but no grade ≥ 3 ALT and/or AST elevations were observed. Treatment discontinuation due to TEAEs occurred in 6.7% of patients. TDI01 was well tolerated without dose-limiting toxicities in both dosage cohorts.PK analysis in all 60 patients showed TDI01 was rapidly absorbed with median Tmax of 4.1-4.5 hours after single doses. Plasma exposure (Cmax and AUC) increased dose-proportionally. Steady-state was achieved by day 15 with moderate accumulation (RAAUC0-t: 1.6-1.9). E–R analysis demonstrated significant correlation between TDI01 exposure parameters (Cmax,ss, AUCss, Ctrough) and 24-week BORR (p < 0.05), with 400 mg approaching maximum efficacy. A positive trend between exposure and FFS was also observed. Notably, no correlation existed between TDI01 exposure and grade ≥3 bilirubin elevations.Conclusion:TDI01 demonstrated durable responses in moderate-to-severe cGVHD. The 400 mg cohort showed a higher ORR, a longer DOR, and a favorable FFS/OS than that of 200 mg cohort. Safety was manageable with primarily reversible hyperbilirubinemia. Our data supported 400 mg QD as the recommended dose of phase III randomized controlled trial which is planned.

Neurofibromatosis type 1 (NF-1) scoliosis can be difficult to treat without early detection. Correcting deformities while considering long-term growth in early-onset scoliosis (EOS) treatment is important. This study was performed to establish the safety and effectiveness of halo gravity traction (HGT) with traditional growing rods (TGRs) in NF-1 EOS. We retrospectively reviewed a cohort of 15 children (7 boys and 8 girls; mean age, 5.61 years) diagnosed with NF-1 EOS from October 2016 to March 2021. All patients underwent HGT before growing rod implantation. The growing rods were lengthened every 9–12 months, with a follow-up of 2–7 years. Cobb angle, thoracic kyphosis (TK), trunk shift (TS), sagittal vertebral axis and T1–S1 height were measured before operation, after traction, after operation and at last follow-up. Complications were also recorded. Fifteen patients with NF-1 EOS were treated with an average traction weight of 10.00 kg. After 29.20 days of HGT, the Cobb angle improved from 99.10° to 62.60°, TK from 79.33° to 55.04°, TS from 31.05 to 17.71 mm, sagittal vertebral axis from 42.07 to 25.63 mm and T1–S1 height from 27.50 to 29.70 cm ( P < 0.05 for all). Postoperatively, compared with post-traction, the Cobb angle was 52.40° ( P = 0.002) and TK was 44.54° ( P = 0.004). No complications occurred during traction. Growing rod dislocation occurred in one patient and growing rod breakage in one patient. HGT combined with TGRs was well-tolerated and effective for treating severe NF-1 EOS. It significantly corrected the Cobb angle and TK, restored trunk balance, and increased spinal height with few complications.

BackgroundNeurofibromatosis type 1 (NF1) is an autosomal dominant genetic disorder affecting multiple systems. However, arterial stenosis is a rare manifestation in patients with NF1. Since the symptoms of arterial stenosis caused by NF1 are often atypical and have a high under-diagnosis rate, this can lead to serious complications such as hypertension, ischemic stroke, or even death. The aim of our research is to analyse the clinical characteristics of arterial stenosis in pediatric patients with NF1 and to summarise its diagnosis, treatment and prognosis.MethodsWe conducted a retrospective review of data from patients with NF1 treated at Beijing Children's Hospital from 2016 to 2020. Patients diagnosed with arterial stenosis, identified through clinical symptoms, physical examination, arterial ultrasonography, or imaging studies, were included in this study. These patients received symptomatic drug and/or surgical treatments and were followed up regularly. We summarized demographic characteristics, sites of arterial stenosis, clinical manifestations, and treatment outcomes.ResultAmong the 258 patients with NF1 treated at our hospital, 12 (4.7%) had arterial stenosis, comprising 9 males and 3 females with a median age of 7 years (range: 1-14 years). Renal artery stenosis was diagnosed in 7 patients (58.3%), while internal carotid artery (ICA) stenosis was diagnosed in 5 patients (41.7%). The predominant symptoms of renal artery and ICA stenosis were renal hypertension and convulsions, respectively. Antihypertensive drugs were effective in 5 patients with renal hypertension; 2 patients required balloon dilatation of the renal artery due to inadequate response to medication. Oral antiepileptic treatment was effective in 3 patients with ICA stenosis, and encephaloduroarteriosynangiosis was effective in the remaining 2 cases. The follow-up period ranged from 2 to 6 years, with a median duration of 3 years. No deterioration or mortality was observed during the follow-up period.ConclusionsArterial stenosis was present in approximately 4.7% of patients with NF1, predominantly affecting the renal artery and ICA. Renal hypertension and convulsions were the primary symptoms of renal artery and ICA stenosis, respectively. Early diagnosis and intervention can substantially improve the prognosis of these patients.

STUDY DESIGN/SETTING:This retrospective study analyzed bracing outcomes in patients with adolescent idiopathic scoliosis (AIS), focusing on curve pattern changes and brace efficacy. OBJECTIVE:To analyze the effectiveness of the Chêneau brace across different curve patterns and to evaluate the tendencies in curve evolution during treatment. BACKGROUND:AIS presents diverse curve patterns, each responding differently to bracing. Understanding these variations is crucial for optimizing treatment strategies. PATIENTS AND METHODS:The study included 177 patients with AIS treated with Chêneau orthoses, categorized based on curve patterns as per the main curve and modified Lenke (mLenke) classifications. We compared patients according to curve patterns and assessed changes in curve magnitude and pattern before and after treatment. RESULTS:Over an average follow-up of 28.1 ± 10.7 months, the primary curve magnitude decreased from 28.8 ± 6.6° to 25.9 ± 10.5°. Significant reductions were observed in mLenke V and VI patients ( P < 0.05). Patients with main lumbar curves showed better initial in-brace correction and curve control compared with those with main thoracic curves ( P < 0.05). In single-curve patterns, binary logistic regression indicated that mLenke V patients demonstrated higher rates of curve control compared with mLenke I patients ( P < 0.05). No significant differences were found in double-curve patterns between mLenke III and VI ( P > 0.05). At the final follow-up, thoracolumbar/lumbar curves improved significantly in mLenke III and VI patients ( P < 0.05), whereas thoracic curves did not ( P > 0.05). Furthermore, at the last follow-up, the proportions of mLenke I, II, and IV increased, whereas mLenke III, V, and VI decreased. CONCLUSIONS:Bracing outcomes were more favorable in patients with main lumbar curves than those with main thoracic curves. However, no significant differences were found in patients with double-curve patterns. Thoracic curves exhibited a higher progression risk compared with thoracolumbar/lumbar curves within the same curve pattern. During bracing, a tendency for primary curves to shift proximally was noted. LEVEL OF EVIDENCE:Level III.

OBJECTIVE The objective of this study was to investigate whether extending fusion to L4 is imperative in the surgical treatment of pediatric L5-S1 spondylolisthesis. METHODS This retrospective analysis encompassed 68 pediatric cases of dysplastic L5-S1 spondylolisthesis who underwent posterior lumbar interbody fusion surgery at two hospitals. Patients were categorized into two groups based on the upper instrumented vertebra (group L4 and group L5). Data were collected from medical records and radiological images obtained preoperatively and at last follow-up. Radiographic parameters including slip percentage (SP), lumbar angle (SDSG-LSA), pelvic tilt (PT), Dubousset's lumbosacral angle (Dub-LSA), sacral slope (SS), and severity index (SI) were measured. Surgery-related data and complication data were also collected. The incidence rates of complications were compared, including those of neurological deficit, adjacent-segment instability (ASI), and other complications. ASI was defined as progression of slippage > 3 mm or posterior opening > 5(degrees) in the adjacent segment. Clinical outcomes were assessed with the numeric rating scale (NRS) and the Oswestry Disability Index (ODI) scores. The follow-up period for all patients lasted a minimum of 2 years. RESULTS Among all 68 patients, group L4 consisted of 15 patients and group L5 comprised 53 patients. The patients included in both groups had comparable baseline demographic characteristics and radiographic parameters. Postoperative SP and SDSG-LSA were significantly lower in group L5 (p < 0.05). No other postoperative radiographic differences were observed between groups. One patient in group L4 and 3 patients in group L5 experienced transient neurological deficits (p > 0.05). There were 13 cases of ASI in group L5 compared with none in group L4 (24.5% vs 0%, p > 0.05). Of the 13 patients with ASI, 4 underwent revision surgery due to L4-5 level instability and clinical symptoms. The remaining individuals exhibited no symptoms, and regular annual follow-up assessments are being conducted for all patients. The NRS and ODI scores at final follow-up did not exhibit any significant differences between the two groups. CONCLUSIONS Fusion to L5 could achieve comparable satisfactory results to fixation to L4, albeit with increased likelihood of ASI. Extension of fusion to L4 may not be necessary for most patients with pediatric L5-S1 spondylolisthesis.

Purpose: This study was performed to compare the radiographic results of robot-assisted and traditional methods of treating lower extremity deformities (LEDs). Methods: From January 2019 to February 2022, 55 patients with LEDs were treated by temporary hemiepiphysiodesis with eight-plates. They were divided into a robot group and a freehand group. The fluoroscopy time and operation time were recorded. The accuracy of screw placement was measured after the operation using the following parameters: coronal entering point (CEP), sagittal entering point (SEP), and angle between the screw and epiphyseal plate (ASEP). The limb length discrepancy (LLD) and femorotibial angle (FTA) were measured before the operation, after the operation, and at the last follow-up. Patients were followed up for 12 to 24 months, and the radiographic results of the 2 groups were compared. Results: Among the 55 patients with LEDs, 36 had LLD and 19 had angular deformities. Seventy-six screws were placed in the robot group and 85 in the freehand group. There was no difference in the CEP between the 2 groups ( P >0.05). The robot group had a better SEP (2.96±1.60 vs. 6.47±2.80 mm) and ASEP (3.46°±1.58° vs. 6.92°±3.92°) than the freehand group ( P <0.001). At the last follow-up, there was no difference in the LLD or FTA improvement between the two groups ( P >0.05). The incidence of complications was significantly lower in the robot group than in the freehand group (0/27 vs. 5/28, P <0.05). Conclusion: Robot-assisted temporary hemiepiphysiodesis with eight-plates is a safe and effective method for treating LEDs in children. Robotic placement of screws is superior to freehand placement with respect to the entering position and direction. Although the correction effect for LLD and angular deformity is similar, screw dislocation is less common when using robot assistance. Levels of Evidence: Level—III. Retrospective comparative study.

AbstractNewly diagnosed patients with high-risk acute graft-versus-host disease (aGVHD) often experience poor clinical outcomes and low complete remission rates. Ruxolitinib with corticosteroids showed promising efficacy in improving response and failure free survival in our phase I study. This study (ClinicalTrials.gov: NCT04061876) sought to evaluate the safety and effectiveness of combining ruxolitinib (RUX, 5 mg/day) with corticosteroids (1 mg/kg/day methylprednisolone, RUX/steroids combined group) versus using methylprednisolone alone (2 mg/kg/day, steroids-only group). Newly diagnosed patients with intermediate- or high-risk aGVHD were included, with risk levels classified by either the Minnesota aGVHD Risk Score or biomarker assessment. Patients were randomized in a ratio of 1:1 into 2 groups: 99 patients received RUX combined with methylprednisolone, while the other 99 received methylprednisolone alone as the initial treatment. The RUX/steroids group showed a significantly higher overall response rate (ORR) on day 28 (92.9%) compared to the steroids-only group (70.7%, Odds Ratio [OR] = 5.8; 95% Confidence Interval [CI], 2.4–14.0; P < 0.001). Similarly, the ORR on day 56 was higher in the RUX/steroids group (85.9% vs. 46.5%; OR = 7.07; 95% CI, 3.36–15.75; P < 0.001). Additionally, the 18-month failure-free survival was significantly better in the RUX/steroids group (57.2%) compared to the steroids-only group (33.3%; Hazard Ratio = 0.46; 95% CI, 0.31–0.68; P < 0.001). Adverse events (AEs) frequencies were comparable between both groups, with the exception of fewer grade 4 AEs in the RUX/steroids group (26.3% vs. 50.5% P = 0.005). To our knowledge, this study is the first prospective, randomized controlled trial to demonstrate that adding ruxolitinib to the standard methylprednisolone regimen provides an effective and safe first-line treatment for newly diagnosed high-risk acute GVHD.

Abstract Objectives The application of a growing rod technique can retain the growth and development potential of the spine and thorax while controlling the progression of scoliosis deformity. Theoretically, convex side short fusion combined with a concave side single growing rod technique can significantly reduce the asymmetric growth of the spine in the vertex region in most patients. However, the final clinical outcome of various techniques is yet to be clearly determined and compared between studies. Therefore, we compared the efficacy of these two growing rod techniques in treating early onset scoliosis. Methods In a retrospective study of 152 EOS patients seen between 2013.1 and 2019.12, 36 cases of EOS patients were selected for inclusion. Among the 36 cases, 11 cases were treated with convex side short fusion combined with a concave side single growing rod technique, group (A) The remaining 25 cases were treated with traditional bilateral growing rod technique, group (B) Age, gender, etiology, follow-up time, Cobb angle of main curve, T1-S1 height, coronal trunk shift, sagittal vertical axis (SVA), Cobb angle of thoracic kyphosis at last follow-up, and Cobb angle at proximal junction kyphosis of the first and last post-operation follow-up were recorded. In addition, internal fixation related complications, infection, nervous system complications were recorded as well. Results There was no statistically significant difference between group A and group B in preoperative age, Cobb angle of main curve, coronal trunk shift, T1-S1 height, SVA, Cobb angle of thoracic kyphosis (p > 0.05). However, at the last follow-up (Group A, mean 4.4 ± 1.01 years; Group B, mean 3.6 ± 0.01 years) the Cobb angle of the main curve was less and T1-S1 height greater in group A compared with group B (p < 0.05). There was no statistically significant difference between group A and group B in the correction rate of the Cobb angle of the main curve or the growth rate of T1-S1 height (p > 0.05). There was no statistically significant difference in the coronal imbalance ratio, thoracic kyphosis abnormality ratio, or the occurrence PJK ratio between group A and group B at the last follow-up (p > 0.05), but the sagittal imbalance ratio and internal fixation abnormality ratio were higher in group A than in the group B (p < 0.05). Conclusions During the treatment of EOS, both the convex side short fusion combined with concave side single growing rod technique and traditional bilateral growing rod technique can correct the Cobb angle of main curve with no significant hindering of the spinal growth observed. The traditional bilateral growing rod technique has advantages in control of the sagittal balance of the spine, and the complications associated with internal fixation were lower.

Purpose: Pediatric pelvic fractures are uncommon. This study aimed to investigate the clinical characteristics of pediatric pelvic fractures requiring hospitalization and analyze their correlation with associated injuries and complications. Methods: Data from 315 pediatric pelvic fracture patients admitted to our hospital from January 2006 to December 2021 were retrospectively analyzed. Sex, age, modified Torode–Zieg classification, abbreviated injury scale score, injury severity score, mortality, and concomitant injuries were analyzed. Results: Of the 285 (90.5%) cases of combined injuries, most injuries occurred in the abdomen (64.8%) and lower extremities (47.6%), followed by the chest (45.4%) and head (34.6%). A total of 78 patients (24.8%) were transferred to the intensive care unit. In total, 94 patients (29.8%) had complications during hospitalization. There were differences based on injury mechanism ( p = 0.001), with the highest complication rate in the fall injury group (32 cases (46.4%)). Approximately 51.4% of patients received surgical treatment for problems that were not related to pelvic fractures. Among these, 30.2% necessitated surgical intervention on the lower limbs. Abdominal surgery was necessary in 19.0% of patients. Conclusions: Children who have pelvic fractures frequently require hospitalization due to the presence of severe injuries in other areas of their bodies. IIIB pelvic fractures frequently occur in conjunction with more severe abdominal injuries; therefore, the prompt management of cavity and organ injuries is of particular importance. Blood transfusion and injury severity score were associated risk factors for intensive care unit admission.