Outcomes of allogeneic stem cell transplantation in older patients with acute myeloid leukemia (AML) have been unsatisfactory. At our center, cord blood transplantation (CBT) using FM80TBI has been implemented in older patients or those with comorbidities. We conducted a retrospective single-center study to evaluate transplantation outcomes in patients with AML who underwent CBT following FM80TBI. The FM80TBI regimen consisted of fludarabine (125 or 150 mg/m²), melphalan (80 mg/m²), and total-body irradiation (4 Gy). This study included 58 patients. The median follow-up period was 38 months. The median age at transplant was 67 (52–73) years. At the time of CBT, 39.7
Background and study aims:The efficacy of uncovered self-expandable metal stents (UCSEMS) compared to that of fully covered self-expandable metal stents (FCSEMS) for distal malignant biliary obstruction (dMBO) is controversial. Studies have highlighted the heterogeneity of disease conditions because pancreatic and nonpancreatic cancers exhibit different clinical courses. This is the first study to specifically compare the safety and efficacy of UCSEMS and FCSEMS for dMBO caused by nonpancreatic cancer. Patients and methods:This retrospective multicenter study included patients who underwent UCSEMS or FCSEMS placement for nonpancreatic cancer-induced dMBO at four centers between January 2010 and April 2024. Propensity score matching (PSM) (1:1) of both groups was performed. Technical and clinical success, adverse events, recurrent biliary obstruction (RBO), time to RBO (TRBO), and overall survival (OS) of these groups were compared. Results:After PSM, 24 patients were included in each group. Technical and clinical success rates and TRBO did not differ significantly between the groups (median TRBO: 311 days [UCSEMS] vs 317 days [FCSEMS]; P = 0.42). Similarly, OS was not significantly different (median OS: 432 days [UCSEMS] vs 190 days [FCSEMS]; P = 0.083). Incidence of pancreatitis after endoscopic retrograde cholangiopancreatography in the UCSEMS group was significantly lower than that in the FCSEMS group (4.2% vs. 33%; P = 0.023). Conclusions:UCSEMS may be safer than FCSEMS for managing dMBO caused by nonpancreatic cancer.
Introduction: Moderate-to-severe ulcerative colitis (UC) is commonly treated with oral corticosteroids. However, in cases of oral corticosteroid failure, no clear consensus exists on whether to transition to intravenous corticosteroids (IVCS) or initiate advanced therapies. The aim of this study was to evaluate whether responsiveness to oral corticosteroids is associated with differences in the efficacy of IVCS. Methods: This multicenter cohort study (conducted at 10 facilities) included patients with moderate-to-severe UC who were transitioned to IVCS after failure of oral corticosteroid therapy. The patients were categorized into the partial responder and nonresponder groups based on their response to oral corticosteroids, as measured by improvements in their PRO-2 scores. The primary outcome was clinical remission at day 30, defined as a total PRO-2 score ≤1 with a rectal bleeding subscore of 0. Logistic regression was used to estimate the odds ratio (OR) of achieving clinical remission. Results: A total of 123 patients with UC were included, with 41 and 82 patients in the partial responder and nonresponder groups, respectively. Clinical remission at day 30 was achieved in 41.4% of the partial responders and 18.3% of the nonresponders (multivariable-adjusted OR, 0.35 [95% CI: 0.15–0.83]; p = 0.017). The nonresponder group had a higher risk of requiring advanced therapies within 90 days than the partial responder group (multivariable-adjusted OR, 2.48 [95% CI: 1.09–5.66]; p = 0.030). Conclusions: In patients with UC and oral corticosteroid failure, responsiveness to oral corticosteroids may be associated with differences in IVCS efficacy.
BACKGROUND:Sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) is a serious endothelial injury syndrome following allogeneic hematopoietic stem cell transplantation (allo-HSCT) requiring early diagnosis and treatment. We retrospectively evaluated clinical characteristics, treatments, and outcomes of patients who developed SOS/VOD after allo-HSCT. METHODS:We included all consecutive patients who developed SOS/VOD after allo-HSCT between January 2010 and October 2023. SOS/VOD was diagnosed according to Seattle, Baltimore, or EBMT criteria, and severity was classified using the EBMT criteria. Patients treated with recombinant thrombomodulin (rTM) between January 2010 and August 2019 were compared with those treated with defibrotide between September 2019 and October 2023. RESULTS:Among 755 cases, 48 developed SOS/VOD: 15 treated with rTM and 33 with defibrotide. Median ages were 50 years in rTM group and 55 years in defibrotide group, and median days to onset were 13 and 17, respectively. There was no significant difference in disease stage at transplantation or SOS/VOD severity at diagnosis. At 100 days post-onset, overall survival (OS), non-relapse mortality, and SOS/VOD mortality were 20.0% vs 46.9% (p = 0.17), 66.7% vs 54.5% (p = 0.609), and 46.7% vs 31.2% (p = 0.364) in the rTM and defibrotide groups, respectively. In multivariate analysis adjusted for age and conditioning intensity, defibrotide treatment was associated with improved OS (hazard ratio, 0.41; 95% confidence interval, 0.18-0.92, p = 0.032). Review of most severe cases suggested an association between inflammatory status and the clinical course of SOS/VOD. CONCLUSION:Defibrotide treatment was associated with improved outcome in SOS/VOD. Inflammatory status appeared to be related to disease onset and resolution.
Purpose:Pediatric femoral neck fractures (PFNFs) are rare (≈1% of pediatric fractures), and avascular necrosis (AVN) is a devastating complication without a universally accepted management. High-degree posterior rotational osteotomy (HDPRO) with three-dimensional (3D) navigation is a novel joint-preserving technique with the potential to improve outcomes. Methods:A retrospective case series was conducted with three pediatric patients (2 boys, 1 girl; aged 9-15 years at injury) diagnosed with AVN secondary to PFNF. All underwent HDPRO using Stryker OrthoMap 3D navigation to reposition the viable anteromedial femoral head. Preoperative and postoperative outcomes were assessed using radiographic analysis, functional scoring (Japanese Orthopaedic Association Hip-Disease Evaluation Questionnaire (JHEQ)), and sports participation. Follow-up ranged from 24 to 36 months. Results:All patients showed pain relief, functional improvement, stable femoral head morphology, and revascularization. JHEQ improved to 79-84 points; hip motion normalized without impingement. All patients returned to sports (track, volleyball, cycling). Conclusions:HDPRO with 3D navigation is a promising joint-preserving option for AVN following PFNF. Preliminary results are favorable; however, the small cohort and limited follow-up warrant larger studies. Significance of study:To the best of our knowledge, this is the first report demonstrating that navigation-guided HDPRO reliably relocates the viable anteromedial head to the load-bearing zone. Level of evidence:Level IV.
