Citations

Abstract Aims Congenital heart disease (CHD) presents lifelong challenges that require effective self-management skills, particularly for youth transitioning to adulthood. This systematic review evaluates the impact of self-management interventions, grounded in self-efficacy, on both health and patient-reported outcomes in youths with CHD. Methods and results Adhering to Preferred Reporting Items for Systematic Review and Meta-Analysis guidelines, we conducted a systematic review and registered the protocol in PROSPERO. We included interventional studies targeting youth aged 15–24 years with CHD that employed self-management interventions based on self-efficacy. Comprehensive searches were performed in Ovid MEDLINE, Ovid EMBASE, CINAHL, the Cochrane Central Register of Controlled Trials, the Cochrane Database of Systematic Reviews, and Scopus from January 1973 to March 2024, with an update to include studies published until December 2024. Two reviewers independently screened titles, abstracts, and full texts, resolving disagreements by consensus. Data extraction followed Template for Intervention Description and Replication guidelines, and risk of bias was assessed using the Cochrane Risk of Bias in Non-Randomized Studies of Interventions and Risk of Bias 2 tools. Out of 54 potential studies, 4 met the eligibility criteria, representing 150 patients aged 16–19 years. The studies reported improvements in condition-related knowledge, self-management skills, and social support, although data on clinical health outcomes were limited. A narrative synthesis of the selected interventions was performed. Conclusion Self-management interventions using self-efficacy frameworks show promise for improving patient-reported outcomes among youth with CHD. However, there is a scarcity of data on their impact on health outcomes. Further research is needed to develop and implement interventions tailored to this population, potentially improving both self-management and clinical outcomes. Registration PROSPERO: CRD42024499008

Abstract Background Patients with inflammatory bowel disease (IBD) often experience symptoms refractory to available treatments, prompting the use of alternative therapies like cannabis. Previous studies have shown cannabis users require higher levels of sedation for procedures. The anti-inflammatory effects of cannabis have been studied with mixed conclusions. We aimed to investigate if patients with IBD who reported cannabis use required more resources for endoscopic procedures and were more likely to have endoscopic inflammation. Methods This is a retrospective case–control study of adult patients with IBD between November 2018 and November 2022 at a tertiary academic medical center undergoing endoscopic evaluation of acute complaints related to IBD. Cases were matched for age, sex, and body mass index. Results There were 124 patients with IBD with 62 patients reporting cannabis use and 62 patients without reported cannabis use. There was a significant difference in endoscopy duration (P < .001) and endoscopic inflammation (P = .044) between groups. There was no significant difference in recovery room length of stay (P = .15), IBD treatment during time of endoscopy (P = .84), stricture (P = .53), propofol dose administered when adjusted for procedure duration (P = .082), or endoscopic duration between cannabis users with and without endoscopic inflammation (P = .194). Conclusions IBD cannabis users had longer endoscopic durations and were more likely to exhibit inflammation on endoscopic evaluation compared to cannabis non-users. Our study underscores the importance of medication reconciliation for more accurate resource allocation. Additionally, federal expansion of cannabis research is needed for randomized control trials to fulfill the presently unmet need for data on patient outcomes.

Abstract Aims Systemic ventricular (SV) strain imaging improves early detection of systolic dysfunction in patients with congenital heart disease and biventricular circulation. However, its role for risk stratification in the Fontan population has not been systematically studied. We hypothesized that among Fontan patients with normal SV ejection fraction (Echo_EF) at baseline, those with reduced SV longitudinal strain (Echo_LS) would have a higher risk of progressive SV systolic dysfunction, greater neurohormonal activation, and cardiovascular events (heart failure hospitalization/death/transplant) during follow-up. Methods and results We studied adults with Fontan palliation and normal SV systolic function (Echo_EF >50%) at baseline who underwent follow-up echocardiogram >36 months after the baseline echocardiogram. Echo_LS was assessed from the apical 4-chamber window using speckle tracking echocardiography. Patients were divided into reduced vs. normal Echo_LS groups, using −18% as the cut-off point. Echo_LS, Echo_EF, and NT-proBNP were assessed at baseline and follow-up encounters. Of 272 patients (age, 28 ± 8 years), 98 (36%) and 174 (64%) had reduced vs. normal Echo_LS. The reduced_Echo_LS group had progressive SV systolic dysfunction [worse temporal decline in Echo_EF (relative_Δ_Echo_EF −12.8% vs. −2.1%, P < 0.001)], and higher prevalence of new-onset SV systolic dysfunction [30% (29/98) vs. 8% (13/174), P < 0.001]. The reduced Echo_LS group also had greater neurohormonal activation [relative Δ_ NT-proBNP 36 (95%CI, 27–43) % vs. 17 (95%CI, 11–23) %, P < 0.001], and higher risk of cardiovascular events (adjusted hazard ratio 1.84, P = 0.009). Conclusion Echo_LS improves risk stratification in Fontan patients with normal Echo_EF and may provide an opportunity for early interventions to improve outcomes.

Abstract Overall response (OR) that combines complete (CR) and partial responses (PR) is the conventional end point for acute graft-versus-host disease (GVHD) trials. Because PR includes heterogeneous clinical presentations, reclassifying PR could produce a better end point. Patients in the primary treatment cohort from the Japanese Society for Transplantation and Cellular Therapy (JSTCT) were randomly divided into training and validation sets. In the training set, a classification and regression tree algorithm generated day 28 refined response (RR) criteria based on symptoms at treatment and day 28. We then evaluated RR for primary and second-line treatments, using the area under the receiver operating characteristic curve (AUC) and negative predictive value (NPV) for 6-month nonrelapse mortality as performance measures. RR considered patients with grade 0/1 at day 28 without additional treatment as responders. RR for primary treatment produced higher AUCs than OR with small improvement of NPVs in both validation sets: JSTCT (AUC, 0.73 vs 0.69 [P < .001]; NPV, 92.0% vs 89.6% [P < .001]) and the Mount Sinai Acute GVHD International Consortium (MAGIC; AUC, 0.71 vs 0.68 [P = .032]; NPV, 90.9% vs 89.8% [P = .009]). RR for second-line treatment produced similar AUCs but much higher NPVs than OR in both validation sets of JSTCT (AUC, 0.64 vs 0.63 [P = .775]; NPV, 74.5% vs 66.0% [P < .001]) and MAGIC (AUC, 0.67 vs 0.64 [P = .105]; NPV, 86.8% vs 76.1% [P = .004]). Classifying persistent but mild skin symptoms as responses and residual lower gastrointestinal GVHD as nonresponses were major drivers in improving the prognostic performance of RR. Our externally validated day 28 RR would serve as a better end point than conventional criteria in future first- and second-line treatment trials.

Abstract We conducted a post hoc analysis of data from Blood and Marrow Transplant Clinical Trials Network 1506 (MORPHO), a randomized trial of gilteritinib vs placebo as posttransplantation maintenance for patients with FLT3-ITD–mutated acute myeloid leukemia (AML) undergoing allogeneic hematopoietic cell transplantation (HCT), focusing the interactions between conditioning regimen intensity, measurable residual disease (MRD), and NPM1 comutation status reported from diagnosis. Comparing FLT3-ITD MRD before and after conditioning, there was no difference between myeloablative conditioning (MAC) and reduced-intensity conditioning (RIC) in eradication or reduction of FLT3-ITD MRD. For participants who were FLT3-ITD MRD negative before HCT, there was no difference in the cumulative incidence of relapse during follow-up between those receiving MAC vs RIC. NPM1 comutation was associated with the largest magnitude of relapse-free survival benefit from post-HCT gilteritinib, and in these participants, post-HCT gilteritinib in the setting of RIC appeared to be as effective as MAC at preventing relapse. MAC appeared superior to RIC in preventing relapse only in participants who were NPM1 wild type at diagnosis and FLT3-ITD MRD positive before HCT. Our findings suggest that only a subset of patients with FLT3-ITD AML undergoing HCT may benefit from MAC and that, similar to AML therapy before HCT, the intensity of the HCT regimen should be adapted according to the molecular features of the disease. This trial was registered at www.clinicaltrials.gov as #NCT02997202.

Background: Secondary shoulder deformities are the most frequent sequelae of upper trunk-type brachial plexus birth injury (BPBI). Coracoid process dysplasia was recently described as being associated with glenoid retroversion and was recognized to block successful humeral head relocation. There are little to no reports on coracoid process deformity causing anterior shoulder pain. Our hypothesis is that coracoid process impingement causes anterior shoulder pain in BPBI, which is relieved by coracoid excision. This study presents the short-term results of this new technique in BPBI patients. Methods: A retrospective review identified BPBI patients who underwent coracoid excision from 2000 to 2022. Indications for this procedure include BPBI patients with anterior shoulder pain limiting the range of motion, localized tenderness over a prominent coracoid process, a dysplastic coracoid process seen on imaging, and persistent pain despite activity modification, medication, and therapy. Patient characteristics, preoperative shoulder pain score, range of motion, degree of glenohumeral subluxation, patient-reported outcomes measurement information system (PROMIS) scores for pain interference, and physical and upper extremity function were collected from chart review. Postoperative pain scores, range of motion, and PROMIS scores were collected at an average of 1 year after excision. Results: Five upper trunk BPBI patients, ages 10 to 25 years old, with anterior shoulder pain underwent coracoid excision. All had statistically significant pain relief from 6.5–8.5/10 before to 0–3/10 after the procedure. Forward flexion and external rotation improved by an average of 18 degrees. The pain interference score decreased by 25%. There was a 33% improvement in physical function scores and a 31% increase in upper extremity function scores. No complications or recurrence were observed at 7.5 months to 1 year and 7 months after surgery. Conclusions: Coracoid excision is a simple procedure that can relieve localized shoulder pain caused by coracoid impingement in adolescent/adult BPBI. Continued follow-up of all patients will be undertaken to determine long-term recurrence rates and complications. Level of Evidence: Level IV.

Background/Objectives: Spinoplastic surgery is an emerging multidisciplinary field developed to address and reduce the complication of pseudoarthrosis following complex spinal reconstructions. While the number of spinal fusion procedures continues to rise every year, fusion failure rates remain as high as 40%. Although pseudoarthrosis may not always manifest clinically, it remains a leading cause of persistent pain and need for subsequent revision surgeries. The multidisciplinary collaboration between spine and plastic surgeons in spinoplastic surgery has therefore emerged as a proactive strategy aimed at preventing complications, particularly in patients identified as high-risk for pseudoarthrosis. As the patient population expands and spinoplastic surgery continues to evolve, refining patient selection criteria becomes essential for achieving optimal surgical outcomes. This review aims to provide a comprehensive overview of recent advancements in spinoplastic surgery, highlighting current indications, surgical techniques, recent case reports, and strategies for identifying suitable candidates. Methods: We performed a narrative review of English language literature through April 2025. Spinoplastic case reports and case series published within the last 20 years were included in the review. Results: Indications for use of a spinoplastic approach clustered into prior fusion failure, extensive oncologic resection, severe spinal deformity, procedures requiring extensive spinal involvement, and/or patients at risk for impaired bone healing. Succesful radiographic union and improvement of symptoms were widely reported across all 9 case reports/series. Conclusions: Although evidence is presently limited, spinoplastic surgery appears to achieve high bone graft fusion rates with acceptable morbidity and functional improvement in a carefully selected group of high-risk spinal reconstruction patients. Still, larger prospective studies are warranted to refine patient selection and validate functional benefit.

Segmental bone loss reconstruction using frozen bone allografts has a great advantage over amputation. Unfortunately, frozen allografts are avascular, and their use often results in infection, non-union, or fracture. We have attempted to overcome these complications by revascularizing the bone using gene therapy. Segmental tibial bone transplants were performed in 16 Yucatan minipigs. An arteriovenous (AV) bundle was placed intramedullary inside the allografts, which were transfected with growth factors vascular endothelial growth factor (VEGF) and platelet-derived growth factor (PDGF) in 8 out of the 16 pigs. Survival time was set at 20 weeks. Bone remodeling and biomechanical properties were tested using histology, compression testing, bone mineralization, and gait analysis. In the growth factor group, five out of eight pigs developed cutaneous vascular tumors, causing excessive bleeding, which led to the exclusion of one pig. Bone remodeling was significantly more present in the VEGF group compared to the control group. This correlated with the amount of pressure the pig allowed on the operated leg for 10 weeks, which was also significantly more (p = 0.029) than the control. Both bone mineralization (p = 0.156) of the area inside the cortex and the amount of stress the allograft could endure before breaking (p = 0.078) seemed to be no different for the VEGF group compared to its healthy contralateral side, which was the control group. We have shown that growth factor-induced frozen allograft bone revascularization using an intramedullary AV bundle results in significantly more bone remodeling and better biomechanical bone properties. However, we encountered benign vascular cutaneous tumors in five out of eight pigs, making the clinical application of this technique impossible at this time.