Music education is central to holistic child development; yet, traditional piano instruction in China often emphasizes rote memorization at the expense of meaningful understanding. Elementary school pupils, particularly novices, frequently struggle with rhythm, melody, and music theory. Game-based learning (GBL), which applies game elements to teaching, may address these challenges by enhancing engagement, providing immediate feedback, and fostering long-term learning. This study aimed to examine the effects of a GBL module for piano education on elementary school pupils' music knowledge in Anhui Province, China, compared to traditional instruction. A quasi-experimental, nonequivalent control group pretest-posttest design was used. Participants were allocated nonrandomly to experimental and control groups based on scheduling feasibility and teacher availability. A total of 60 novice piano learners (mean age 8.8 years, SD 1.16 years; 16 boys and 44 girls) from 3 elementary schools were assigned to either the GBL group (n=30) or the control group (n=30). Music knowledge was measured using the standardized Level 1 Basic Music Written Test before and after an 8-week intervention. Nonparametric analyses were applied, including Mann-Whitney U, Wilcoxon signed-rank, and McNemar item-level analyses. Rank-based effect sizes (r) and 95% CIs were reported where applicable. Baseline differences were present, with the control group scoring higher at pretest (median 52, IQR 24-76) than the GBL group (median 28, IQR 16-64; Mann-Whitney U=265.50; r=-0.35; 95% CI -0.39 to -0.32; P=.006). After the intervention, the GBL group significantly outperformed controls (median 100, IQR 88-100 vs median 60, IQR 40-92; Mann-Whitney U=4.0; r=-0.87; 95% CI -0.90 to -0.83; P<.001). Within-group analyses confirmed significant pre-post improvements for both groups (control Z=-3.24; r=-0.59; P=.001; and GBL Z=-4.81; r=-0.88; P<.001). Item-level McNemar tests showed significant improvements (P<.05) in 5 of 25 items for the GBL group. Missing data were negligible (<2%) and handled via listwise deletion after Little's missing completely at random (MCAR) confirmation (P=.08). The GBL module significantly improved pupils' music knowledge, overcoming baseline disparities and producing posttest score gains with consistent mastery. The innovation of the study lies in the systematic integration of gamification with Orff and Dalcroze pedagogy through the Sidek Module Development Model, which distinguishes it from previous music education studies that examine gamification in isolation. By providing a validated, cost-effective, and scalable instructional module, the study contributes empirical evidence to the field of game-based music education and other practical implications for improving piano instruction in resource-constrained elementary school settings.

Cutaneous leishmaniasis (CL) and other skin diseases impose a high disease burden in Ethiopia, yet access to care is poor due to limited and centralized diagnostics and treatment. Decentralizing care could improve this but may necessitate substantial changes to the healthcare system. This study aimed to assess the available resources and the knowledge and skills of healthcare professionals for decentralizing the diagnosis and care of CL and common skin diseases to lower healthcare facilities. A cross-sectional study was conducted in South Ethiopia, from May to July 2023, involving 11 health centers, four primary hospitals, and one general hospital. Infrastructure was assessed on-site. Resources, knowledge, and skills of staff members regarding the treatment of CL and other skin diseases were collected using a newly developed questionnaire, comprising 25 knowledge-based and 14 skill-based questions. Skills were assessed via lesion and pathogen images. Descriptive statistics for the different variables studied and inferential statistics based on (confidence) interval estimation were reported. Most facilities had equipment for diagnosis and localized treatment. Adequate hospitalization space and necessary equipment for systemic treatment of CL were found in 3 out of 4 primary hospitals, but none of the health centers. Consumable and drug shortages were common across all facilities. The median score of BSc laboratory technologists was significantly higher than that of diploma technicians (i.e. 29 vs. 15 out of a maximum of 39, p < 0.001). Clinical staff scores varied significantly across education levels (p = 0.007), with physicians scoring the highest (median 33, IQR 31-36), followed by health officers (median 29, IQR 27-32), BSc nurses (median 28, IQR 16-36), and diploma nurses (median 25, IQR 19-29). Notably, no significant differences in median scores were observed between primary hospitals and health centers for clinical and laboratory staff. Decentralizing the diagnosis and treatment of common skin diseases and localized CL treatment to health centers appears feasible with facility adjustments, continuous training, and reliable supply chains, while referring CL cases requiring systemic treatment to primary hospitals. Strong strategic efforts to maintain staff knowledge and skills and tackle supply shortages are crucial for successful decentralization. Not applicable.

PurposeThe readability of public-facing vaccine-related information is an important aspect of health literacy particularly regarding vaccine uptake. The aims of this study were to analyse the readability of such written literature and to provide recommendations, for improvement.MethodsReadability of vaccine-related information (ntotal = 240) from publicly available sources (n = 20 per category), including PubMed s, Expert Review of Vaccines (ERV) and Cochrane Reviews (CR), paired plain language and scientific abstracts, public health materials, clinical trial summaries and vaccine patient information leaflets, were assessed using the Flesch Reading Ease (FRE), Flesch-Kincaid Grade Level (FKGL), SMOG and Gunning Fog readability metrics using the readability software tool readable.com.ResultsVaccine-related information for all sources had poor readability across all readability metrics with 90.8% and 94.6% not reaching the target FKGL (≤8) (mean 12 ± 3.2 sd) and FRE (≥60) (mean 34 ± 17 sd). Plain language summaries had improved readability, but did not reach reference targets. Scientific abstract and plain language scores for the CR were FRE (mean 25 ± 7.2 sd; median 25) versus (mean 37 ± 8.6 sd; median 36) p < 0.0001), respectively and for ERV FRE the scientific abstract (mean 18 ± 11 sd; median 17) versus the plain language score (mean 26 ± 11 sd; median 28) p = 0.002), respectively, indicating an improvement in readability scores for plain language summaries but again not reaching reference targets.ConclusionThe readability of public-facing vaccination materials is currently not optimum. The readability can be improved through the employment of readability calculators and ensuring, where possible, the use of mono-syllable words and less than fourteen words per sentence. The preparation of public-facing materials with improved readability scores will help aid in the promotion of health literacy and in turn promote vaccination uptake.

Dilated cardiomyopathy (DCM) is often familial and screening of relatives is recommended. However, studies on the yield of screening are scarce in developing countries. The aim of the study was to identify and characterise First-degree relatives of patients with DCM in Tanzania. We recruited first-degree relatives of 57 DCM patients. DCM in the relatives was diagnosed using the 2016 revised definition by the European Society of Cardiology working group on myocardial and pericardial diseases. We screened 120 first-degree relatives. All were asymptomatic (100%) with a median age of 39.0 years (29.5-49.0), slightly over a half (53.3%) were females and 17 (14.1%) were found to have previously unknown DCM. The mean (± SD) indexed left ventricular end-diastolic volume was significantly higher in relatives with DCM (71 ± 11.5 ml) compared to relatives without DCM (50 ± 11.5) (p = 0.001). First-degree relatives of patients with DCM are at risk of developing asymptomatic DCM at a young age.

Single-ventricle physiology is a critical cardiac condition requiring early diagnosis and intervention. The objectives of this study were to report on the management and outcomes of patients diagnosed with single-ventricle physiology in central South Africa. This study was a retrospective, observational analysis of patients presenting with single-ventricle physiology at the Universitas Academic Hospital in central South Africa between November 1997 and June 2021. Patients were referred from the Free State (54%) and Northern Cape (29%) provinces and Lesotho. One hundred and fifty-four patients presented with single-ventricle physiology: 114 received interventions and 40 were not eligible for intervention. Patients presented for the first time at a median age of 34.5 days, with patients from nearby districts presenting within a few days of birth. However, patients from outlying areas presented much later. Eighty-seven patients received systemic-to-pulmonary artery shunting or pulmonary artery banding. Sixty-three patients proceeded to bidirectional Glenn procedures, and 30 patients (26%) had full palliation to Fontan. Twenty-one patients died after stage 1, six after the Glenn procedure and two after the Fontan procedure. Overall, 34 (29.8%) patients were lost to follow up. Patients in our study presented late and follow up of these patients was a challenge. The highest mortality rate occurs during the first stage of palliation. Outcomes from this study are comparable to other sub-Saharan studies.

Clinicopathological factors associated with trichilemmal cysts

BackgroundRobot-assisted pancreatoduodenectomy (RPD) aims to enhance postoperative recovery compared to open pancreatoduodenectomy (OPD). Although recent randomized trials confirmed the short-term safety of RPD, they did not confirm superiority or assess oncological safety. This nationwide observational cohort study compares oncological outcome after RPD versus OPD in patients with resectable pancreatic ductal adenocarcinoma (PDAC) and distal cholangiocarcinoma (DCC) without vascular contact.MethodsAll consecutive patients undergoing RPD and OPD for upfront resectable PDAC and DCC without vascular contact on preoperative imaging in the Netherlands were included. Data were obtained from the Netherlands Cancer Registry (2016–2023). Primary outcomes were overall survival (OS) and R0-resection rate.ResultsOverall, 1675 patients after pancreatoduodenectomy for upfront resectable PDAC and DCC were included (375 RPD; 1300 OPD). Adjusted median OS was 23 months after RPD versus 22 months after OPD, with comparable 5-year survival rate (23.3% versus 22.4%, HR 0.96 [0.82–1.14], P = 0.665). The R0-resection rate was comparable (57.1% versus 59.7%, P = 0.368). RPD was associated with a shorter hospital stay (median 9 versus 11 days, P < 0.001) and comparable in-hospital/30-day (3.1% versus 2.6%, P = 0.618) and 90-day mortality rate (7.7% versus 6.2%, P = 0.276). In patients with PDAC, no differences in receipt (58.2% versus 58.7%, P = 0.900), time to start (median 54 versus 58 days, P = 0.107), or completion of adjuvant chemotherapy (30.4% versus 30.4%, P = 0.999) were observed.ConclusionsIn this nationwide study, oncological outcome including 5-year survival was comparable between patients undergoing RPD and OPD for upfront resectable PDAC and DCC without vascular contact without differences in the use of adjuvant therapy for PDAC.

Monoclonal gammopathy in patients with type I gaucher disease: Data from the Russian gaucher registry

A national lens on follicular lymphoma: Academic care linked to improved survival in 158,000 patients

Challenges in hemoglobinopathy control programs: Insights from Türkiye

Infection risk factors and burden in patients treated with T-cell engaging bispecific antibodies for relapsed B-cell lymphoma and multiple myeloma

Treatment Cessation in Patients with Diabetic Maculopathy under Intravitreal Anti-VEGF Therapy Following a Treat-and-Extend Protocol.

Tricuspid vs Bicuspid Aortic Valve Endocarditis

Background: The role of CREBBP and EP300 mutations in hypermutation and immunotherapy response in gastroesophageal adenocarcinomas is poorly defined and needs further investigation. Methods: We conducted an in silico analysis of 12 publicly available studies (n = 1871; cBioPortal), stratifying samples by CREBBP/EP300 status to assess associations with TMB-High, MSI, co-mutation patterns, and mutation localization. Clinical validation was performed in an independent pan-cancer cohort treated with ICIs (n = 1610) and a gastric cancer cohort with WES data (n = 55). Results: Coding mutations in CREBBP and/or EP300 were significantly associated with TMB-high and MSI-high phenotypes (p < 0.001). All studied samples carrying coding mutations in both CREBBP and EP300 exhibited a TMB-high status. PTVs in functional HAT and bromodomain regions were exclusively associated with TMB-high. Incorporating CREBBP and/or EP300 mutation status improved identification of ultra-hypermutated tumors compared with single-gene biomarkers (p < 0.001). Clinically, these mutations predicted improved overall survival in the pan-cancer cohort (median OS 34 vs. 17 months; HR = 0.68, 95% CI 0.52–0.87, p = 0.0026), as well as in bladder (HR = 0.55, p = 0.0337) and gastrointestinal cancer cohorts (HR = 0.31, p = 0.0021) treated with ICIs. In the gastric cancer validation cohort, all tumors with PTVs demonstrated a partial response to anti-PD-1 therapy. Conclusions: We report CREBBP and EP300 coding mutations as novel potential surrogate biomarkers for hypermutation in gastroesophageal adenocarcinomas and demonstrate their association with favorable immunotherapy outcomes, supporting their potential clinical utility for patient stratification.

Piloting a simplified bio-behavioural survey methodology, the BBS-Lite, among people who inject drugs in Georgia

Introduction Protease inhibitors (PIs) remain an effective antiretroviral therapy (ART) option for people with human immunodeficiency virus (HIV) (PWH), particularly in complex clinical and virological scenarios. However, they are associated with greater metabolic toxicity and drug–drug interactions (DDI) compared with newer ART classes. This study aimed to characterize PWH currently receiving PI-based ART and to explore the reasons for maintaining these regimens.Methods We conducted a cross-sectional, observational study of all PWH on PI-based ART as of 30 June 2024 at the HIV Unit of Hospital Clínic de Barcelona. Demographic, clinical, laboratory, ART history, and genotypic resistance data were extracted from the institutional database and compared with the rest of the cohort.Results Among 6261 PWH on ART, 724 (11.6%) were receiving a regimen including a PI; their use progressively declined over the last two decades (p < 0.001). The most frequent reasons for PI prescription were prior virological failure (36%) and toxicity to previous ART (41%). Compared with other PWH, those on PIs were older (median 54 versus 48 years, p < 0.001), more frequently female patients (19% versus 13%, p < 0.001), and had higher rates of heterosexual (33% versus 21%, p < 0.001) and injection-drug-use transmission (15% versus 7%, p < 0.001). Virological suppression was significantly lower among PWH on PIs (88% versus 96%, p < 0.001). Genotypic resistance testing prior to PI prescription was available for 435 PWH; 74% had at least one major resistance substitution, and 70.4% had substitutions affecting two or more antiretroviral classes. In total, 299 PWH had experienced either virological failure or toxicity to non-nucleoside reverse transcriptase inhibitor (NNRTI)- or integrase strand transfer inhibitor (InSTI)-based regimens prior to initiating a PI-based regimen. Among them, 42 had documented failure of or toxicity to both drug classes.Conclusions Although their use has declined, a substantial number of PWH remain on regimens including a PI. These PWH typically have long-standing infections, prior ART failures, and documented resistance substitutions, supporting the continued use of PIs when other therapeutic options are limited.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40121-025-01229-9.

Hematopoietic stem cell transplantation (HSCT) requires a long hospitalization during which patients have to stay in transplantation wards, mainly in their single isolation rooms. One of the main goals of rehabilitation for HSCT patients is to prevent physical activity deficit by the introduction of an appropriate exercise programme and the monitoring of exercise adherence. The aim of our study was to evaluate the effectiveness of the prescribed exercise programme based on basic locomotor patterns to prevent physical activity deficit. The interim results of the prospective randomized controlled study included data from 20 patients. The study was approved by the Independent Ethics Committee and the Scientific Council of the 1Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation. An experimental group included 11 patients aged 8–16 years old (the median age was 12 years old); a control group included 9 patients aged 5–11 years old (the median age was 8 years old). Patients from the experimental group were preliminarily taught a set of exercises aimed at practicing ontogenically determined standing up which they should further perform during a day in their isolation rooms. The control group patients received recommendations on how to maintain physical activity in their isolation rooms. Their physical activity during a day was then monitored by the researchers. All study subjects underwent an orthoclinostatic test (i.e., transition from lying to standing and back) for the evaluation of the autonomic nervous system function on Day 1 of pre-transplant conditioning therapy and at the time of discharge from the HSCT Department. During the entire stay at the HSCT Department, the patients from both groups were daily assessed using the ADL (activities of daily living) scale. At the time of discharge from the HSCT Department, the experimental group patients showed more adaptive autonomic nervous system responses than the control group patients. Our interim analysis showed that the patients from the experimental group had higher scores of activities of daily living than the control patients. Orthoclinostatic test results confirmed that patients undergoing HSCT for acute leukemia were predisposed to develop maladaptive conditions or even orthostatic hypotension. A set of exercises including those for the training of ontogenetically determined process of standing up was shown to be an effective tool for the prevention of insufficient autonomic nervous system responses. Even a short (30 min) face-to-face session with a physical therapist significantly increased a patient's daily physical activity.

Neuroblastoma (NB) can manifest through neurological symptoms caused by tumor extension into the spinal canal and the resulting epidural compression (EC). Clinical symptoms and management in patients with epidural compression depend on its level and duration, the severity of spinal cord compression, the patient's age and other factors. One of the biggest challenges is the diagnosis and treatment of EC in infants in the first months of life. Our retrospective analysis included 13 patients with NB complicated by spinal cord EC who had been diagnosed at the age of 0–6 months and treated at the D. Rogachev NMRCPHOI over the period from 01.01.2012 to 01.12.2018 (82 months). The study was approved by the Independent Ethics Committee and the Scientific Council of the D. Rogachev NMRCPHOI of the Ministry of Healthcare of the Russian Federation. The diagnosis of NB was based on the international diagnostic criteria. The tumors were staged in accordance with the INSS classification. The patients were stratified into risk groups and treated according to the modified NB-2004 protocol of the German Oncology Group. All the patients underwent diagnostic testing for neurogenic tumors as well as contrast-enhanced magnetic resonance imaging of the spinal cord with the assessment of the level of tumor invasion and the degree of spinal cord compression. The children were evaluated by a neurologist at admission and at follow-up visits. The median age at diagnosis of NB was 3.9 (0.5–6) months. At disease onset, 53.8% of patients had neurological symptoms, with motor deficiencies being the most common ones. In this group of patients, the median time from first neurological symptoms to diagnosis of NB was 1.56 months. Neurological symptoms at disease onset were not present or diagnosed at local healthcare facilities in 46.2% of infants. Extra-organic retroperitoneal primary tumors were found in 61.6% of patients; 30.7% of primary tumors were located in the posterior mediastinum, and 7.7% of primary tumors – in the lesser pelvis. No patients had MYCN-amplified tumors; in 1 case the MYCN gene status was evaluated as Gain; neither 1p nor 11q deletions were detected. The distribution of patients by INSS stages was as follows: stage 2 – 15.3%, stage 3 – 46.1%, stage 4 – 23.3% and stage 4S – 15.3%. The majority of patients (77.7%) were stratified into an observation group, the remaining patients (23.3%) were allocated to a medium risk group in accordance with the NB-2004 protocol. The level of tumor invasion into the spinal cord canal varied. Tumor invasion at the level of the cervicothoracic spine was observed in 15.4% of patients, at the level of the thoracic spine – in 15.4%, at the level of the thoracolumbar spine – in 46.2%, at the level of the lumbar spine – in 15.4%, and at the level of the sacral spine – in 7.7%. Neurosurgical intervention (laminotomy) was performed in 4 cases (30.7%). In one patient, laminotomy was the only treatment option (chemotherapy was not given). In two patients, neurosurgery was performed because of the deterioration of neurological symptoms caused by the start of the first polychemotherapy (PCHT) cycle. Chemotherapy was carried out in 92.3% patients. The patients from the observation group received 1–3 PCHT cycles (the median number of cycles was 2). Only one patient from the observation group did not receive PCHT. This patient underwent 2 surgeries. Currently, 10/13 (77%) patients are alive, 3/13 (33%) patients are dead (2/3 patients died of therapy-related infectious complications, and 1/3 – of acute heart failure in the early postoperative period). The median follow-up was 37.3 months. According to the assessment of late effects, neurological disorders were found in all the analyzed patients (n = 9), and orthopedic disorders were found in 66.6% (6/9) patients. The results of our analysis illustrate both the difficulty of diagnosis and management of EC in patients with NB and the need¬ for uniform testing and treatment standards with established follow-up and rehabilitation strategies for this group of patients.

Acute myeloid leukemia (AML) is the second most common type of leukemia in children and accounts for up to 20 % of all leukemias. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective, and sometimes the only therapeutic option in high-risk patients with AML. Graft-versus-host disease (GVHD) is a major complication of allo-HSCT and the main cause of transplant-related mortality. GVHD prophylaxis in children includes calcineurin inhibitors, either alone or in combination with other immunosuppressants, which can lead to grade II–IV acute GVHD in 40–85 % of cases. Alternatively, GVHD can be prevented with high-dose cyclophosphamide (50 mg/kg/day) administered on days +3, +4 after allo-HSCT, either alone or in combination with other immunosuppressive drugs depending on HLA compatibility of the donor. The aim of this study was to evaluate outcomes after allo-HSCT from an unrelated donor with GVHD prophylaxis with post-transplant cyclophosphamide (PTC) in children in their first and second remission of AML in comparison with a historical control group. We retrospectively analyzed patient outcomes after 53 first-time allo-HSCTs from HLA-matched (n = 40) and partially-matched (8–9/10) (n = 13) unrelated donors performed in pediatric patients (aged 0 to 18 years) in their 1st or 2nd remission of AML at the R. M. Gorbacheva Research Institute for Pediatric Oncology, Hematology and Transplantation from 2008 to 2018. The study was approved by the Independent Ethics Committee and the Scientific Council of the I. P. Pavlov First Saint Petersburg State Medical University of Ministry of Healthcare of the Russian Federation. Our group of interest included 26 patients preventively treated for GVHD with 50 mg/kg of cyclophosphamide on days +3 and +4 in combination with calcineurin inhibitors (cyclosporin A – 2 (7.7 %) patients, tacrolimus – 24 (92.3 %) patients), the mTOR inhibitor sirolimus (5 (19.2 %) patients) or mycophenolate mofetil (21 (80.8 %) patients). The historical control group was made up of 27 patients whose GVHD prophylaxis was based on antithymocyte globulin used in combination with calcineurin inhibitors (tacrolimus – 5 (18.5 %) patients, cyclosporin A – 21 (77.8 %) patients) or the mTOR inhibitor sirolimus (1 (3.7 %) patients) or methotrexate (25 (92.6 %) patients), or mycophenolate mofetil (2 (7.4 %) patients). The groups were matched for diagnosis, age, disease status before allo-HSCT, the matched-to-partially-matched donor ratio, the source of hematopoietic stem cells and conditioning regimen intensity (myeloablative conditioning regimen (MAC) or reduced intensity conditioning regimen (RIC)). The median age at the time of allo-HSCT was 8.6 (0.97–18) years in the PTC group and 6.55 (1.42–17.76) years in the historical control group. In the PTC group, 21 (80.8 %) patients were diagnosed with primary AML and 5 (19.2 %) – with secondary AML, while the historical control group included 22 (81.5 %) and 5 (18.5 %) patients with primary and secondary AML respectively. Disease status at the time of allo-HSCT: 21 (80.8 %) patients treated with PTC were in the 1st complete clinical and hematologic remission (CCHR) and 5 (19.2 %) – in the 2nd CCHR; among the controls, there were 19 (70.4 %) cases of the 1st CCHR and 8 (29.6 %) cases of the 2nd CCHR. In the PTC group, 18 (69.2 %) patients underwent allo-HSCT from 10/10 fully HLA gene-matched donors and 8 (30.8 %) – from 9/10 HLA-matched donors. In the historical control group, 19 (70.4 %) patients had allo-HSCT from 10/10 fully HLA gene-matched donors, 4 (14.8 %) – from 9/10 matched donors, and 1 (3.7 %) – from an 8/10 matched donor. In the PTC group, MAC was used in 14 (53.8 %) patients, RIC – in 12 (46.2 %) patients. In the control group, MAC and RIC were used in 14 (51.9 %) and 13 (48.1 %) patients respectively. In the group treated with PTC, hematopoietic stem cells were derived from the bone marrow in 14 (53.8 %) patients, from the peripheral blood – in 12 (46.2 %) patients. In the historical group, bone marrow was used in 13 (48.1 %) patients and peripheral blood - in 14 patients (51.9 %). The median graft cellularity (CD34+ × 106/kg) in the PTC group was 4.60 (1.7–10.9) × 106/kg, in the historical group – 6.60 (1.0–13.2) × 106/kg. The overall and relapse-free 5-year survival rates were higher in the PTC group than in the historical control group: 83.3 % (95 % confidence interval (CI) 60.9–93.5) vs 59.3 % (95 % CI 38.6–75.0), p = 0.0327 and 76.9 % (95 % CI 55.7–88.9) vs 48.1 % (95 % CI 28.7–65.2), respectively, p = 0.0198. The cumulative incidence of grade II–IV acute GVHD and grade III–IV acute GVHD by day +125 and of moderate and severe chronic GVHD, and the 2-year transplant-related mortality were significantly lower in the PTC group compared to the controls: 15.4 % (95 % CI 4.8–31.5) vs 51.8 % (95 % CI 31,9–68.5), p = 0.004; 7.7 % (95 % CI 1.3–21.7) vs 33.3 (95 % CI 16.8–50.9), p = 0.026; 23.4 % (95 % CI 9.5-41.0) vs 58.6 % (95 % CI 33.8–76.8), p = 0.022; 3.8 % (95 % CI 0.3–16.4) vs 25.9 % (95 % CI 11.5–43.1), p = 0.0232, respectively. GVHD-related mortality was higher in the historical control group than in the PTC group (3.8 % vs 18.5 %, p = 0.192). Thus, PTC-based GVHD prophylaxis was shown to be more effective in managing acute and chronic GVHD compared to antithymocyte globulin, with better overall, relapse-free and GVHD-free relapse-free survival rates and low transplant-related mortality.

Experience in the use of 5-azacytidine, bortezomib and valproic acid for the prevention of leukemia relapses in children after ab-T cell-depleted hematopoietic stem cell transplantation