Explore the vast range of titles available.

T lymphoblastic leukemia /lymphoma (T-ALL/LBL) is a rare and highly aggressive neoplasm of lymphoblasts. We evaluated 195 T-ALL/LBL adolescent and adult patients who received ALL-type chemotherapy alone (chemo,n = 72) or in combination with autologous hematopoietic stem cell transplantation(auto-HSCT,n = 23) or allogeneic hematopoietic stem cell transplantation(allo-HSCT,n = 100) from January 2006 to September 2020 in three Chinese medical centers. 167 (85.6%) patients achieved overall response (ORR) with 138 complete response (CR) patients (70.8%) and 29 partial response (PR) patients (14.8%). Until October 1, 2023, no difference was found in 5-year overall survival (5-OS) and 5-year progression free survival(5-PFS) between allo-HSCT and auto-HSCT (5-OS 57.9% vs. 36.7%, P = 0.139, 5-year PFS 49.4% vs. 28.6%, P = 0.078) for patients who achieved CR, for patients who achieved PR, allo-HSCT recipients had higher 5-OS compared with chemo alone recipients (5-OS 23.8% vs. 0, P = 0.042). For patients undergoing allo-HSCT, minimal residual disease (MRD) negative population showed better 5-OS survival compared with MRD positive patients (67.8% vs. 19.6%, p = 0.000). There were no significant differences between early T-cell precursor (ETP), NON-ETP patients with or without expression of one or more myeloid-associated or stem cell-associated (M/S+) markers (NON-ETP with M/S+, NON-ETP without M/S+) groups in allo-HSCT population for 5-OS. (62.9% vs. 54.5% vs.48.4%, P > 0.05). Notch mutations were more common in patients with non-relapsed/refractory disease than relapsed/refractory disease (χ² =4.293, P = 0.038). In conclusion, Allo-HSCT could be an effective consolidation therapy not just for patients with CR, but also for those who achieved PR. The prognosis is significantly improved by obtaining MRD negative prior to allogeneic transplantation.

Background The European LeukemiaNet (ELN) 2017 Genetic Risk Stratification System is widely used to categorize adult acute myeloid leukemia (AML) patients into prognostic groups. This study aimed to evaluate the prognostic value of ELN2017 in predicting survival outcomes and to assess the impact of clinical and molecular factors such as age, FLT3 and NPM1 mutations, and allogeneic hematopoietic stem cell transplantation (allo‐HSCT). Methods A cohort of 186 adult AML patients was stratified into ELN2017 risk categories: favorable (19.6%), intermediate (54.6%), and adverse (25.76%). We analyzed overall survival (OS) and relapse‐free survival (RFS), with subgroup analyses based on age, FLT3 and NPM1 mutation status, and allogeneic allo‐HSCT. Results Three‐year OS was 68.09% (favorable), 45.36% (intermediate), and 22.87% (adverse). Five‐year OS declined to 34.47%, 34.99%, and 13.72%, respectively. Corresponding 3‐year RFS rates were 49.96%, 38%, and 12.98%, whereas 5‐year RFS rates were 39.97%, 30.40%, and 6.49%. Patients aged ≥60 years showed poorer survival across all risk groups. Among FLT3‐mutated cases, 5‐year OS was 50% in the favorable group and 41.10% in the intermediate group. Allo‐HSCT significantly improved 3‐year OS in intermediate‐risk patients from 33.80% to 63.00% and in adverse‐risk patients from 10.10% to 46.15%. Conclusions ELN2017 provides strong prognostic discrimination in adult AML despite the introduction of newer ELN guidelines. Patient age, FLT3/NPM1 mutations, and transplantation status further refine risk assessment and should guide individualized treatment strategies. This study aimed to evaluate the prognostic value of ELN2017 in predicting survival outcomes and to assess the impact of clinical and molecular factors such as age, FLT3 and NPM1 mutations, and allogeneic hematopoietic stem cell transplantation (allo‐HSCT).

Purpose Malignant hematological diseases represent the most common pediatric cancer. As they cannot always be cured by chemotherapy alone, leukemia and myelodysplastic syndrome (MDS) are frequent medical indications for hematopoietic stem cell transplantation, yet even this treatment is not capable of preventing relapse for certain. Therefore, molecular markers are used to monitor minimal residual disease (MRD) to be enabled to react early to an impeding relapse. As specific markers are not always available, Wilms’ tumor gene 1 (WT1) has been suggested as a universal marker, but has not yet been established clinically. Methods We determined the level of WT1 gene expression in 130 children, adolescents and young adults with malignant hematological diseases prior to transplantation and evaluated its impact on patients’ outcome. A real-time quantitative RT-PCR was used for this purpose. Results The relationship between a high level of WT1 and the cumulative incidence of relapse, event-free survival and overall survival proved to be highly significant in univariate and multivariate analyses. Forty-eight percent of all patients with high WT1 levels suffered from a relapse, whereas only eight percent showing normal WT1 levels before transplantation relapsed. The most convincing result was found for acute myeloid leukemia (AML) and MDS. Conclusion We conclude that WT1 expression prior to transplantation qualifies as an independent prognostic factor and should be further evaluated for MRD monitoring. It might especially be useful for patients with AML or MDS missing specific markers.

ObjectiveTo investigate the results of real-world application of non-myeloablative autologous HSCT for multiple sclerosis (MS).MethodsBetween July 2003 and October 2019 at a single center (Northwestern University), 414 patients with relapsing remitting MS (RRMS) and 93 patients with newly diagnosed secondary progressive MS (SPMS) underwent non-myeloablative HSCT.ResultsThere was one treatment-related death (0.19%) due to hospital-acquired legionella pneumonia, and one patient developed neutropenic bacteremia (Klebsiella pneumonia) without sepsis. Overall 5-year survival was 98.8%. Post HSCT secondary autoimmune diseases (2nd ADs) were idiopathic thrombocytopenia (ITP) and hypo or hyperthyroidism. ITP was highest with alemtuzumab (14%) and 0 to 2.8% for the non-alemtuzumab regimens. After HSCT, 16 patients developed hypothyroidism (3.5%) and 15 developed hyperthyroidism / Grave’s disease (3.3%). Relapse free survival (RFS) at 5 years for RRMS and SPMS was 80.1% and 98.1%, respectively, while progression free survival (PFS) at 4 years for RRMS and SPMS was 95% versus 66%, respectively. For patients with RRMS, the EDSS significantly improved (p < 0.0001) at each follow-up from a pre-HSCT mean of 3.87 to 2.51, 2.50, 2.41, 2.33, and 2.19 at 1, 2, 3, 4, and 5 years, respectively. For SPMS, the EDSS improved significantly only at 1 year but not thereafter. For SPMS, the mean baseline EDSS of 5.09 changed post-HSCT to 4.85 (p = 0.04), 4.88 (p = 0.2), 4.92 (p = .27), 4.72 (p = 0.07), and 4.2 (p = 0.21) at 1, 2, 3, 4, 5 years, respectively.ConclusionIn patients with RRMS, autologous non-myeloablative HSCT is an effective one-time therapy, while HSCT appears of less benefit for newly diagnosed SPMS.

A descriptive qualitative study was conducted to explore risk perception, self-management challenges, and coping strategies among Chinese hematopoietic stem cell transplantation (HSCT) patients, using the Health Action Process Approach (HAPA) as the guiding framework. Purposive sampling, based on the principle of information power, was employed to recruit 20 HSCT patients from a hospital in south-central China between May and August 2024. The HAPA model informed the development of semi-structured interview questions, and the data were analyzed using a combination of thematic analysis and framework analysis. The findings indicate that both risk perception and self-efficacy play a critical role in motivating patient engagement in self-management. Main barriers to self-management included difficulties in symptom control, lifestyle modification, psychological adjustment, and healthcare-related financial burdens. Effective coping strategies reported by participants involved the use of digital tools, seeking professional medical advice, maintaining self-management journals, and drawing on family and social support networks. In clinical practice, healthcare professionals may capitalize on the heightened risk perception and self-efficacy during the peri-transplant period to promote the transition from risk perception to actionable self-management behaviors. In contrast, during the early post-discharge phase, when both risk perception and self-efficacy tend to decline, tailored support systems and resource allocation are helpful for maintaining the transition from risk perception to self-management action. Graphical Abstract

Acute myeloid leukemia (AML) is the second most common hematologic malignancy in children. The incidence of childhood AML is much lower than acute lymphoblastic leukemia (ALL), which makes childhood AML a rare disease in children. The role of genetic abnormalities in AML classification, management, and prognosis prediction is much more important than before. Disease classifications and risk group classifications, such as the WHO classification, the international consensus classification (ICC), and the European LeukemiaNet (ELN) classification, were revised in 2022. The application of the new information in childhood AML will be upcoming in the next few years. The frequency of each genetic abnormality in adult and childhood AML is different; therefore, in this review, we emphasize well-known genetic subtypes in childhood AML, including core-binding factor AML (CBF AML), KMT2Ar (KMT2A/11q23 rearrangement) AML, normal karyotype AML with somatic mutations, unbalanced cytogenetic abnormalities AML, NUP98 11p15/NUP09 rearrangement AML, and acute promyelocytic leukemia (APL). Current risk group classification, the management algorithm in childhood AML, and novel treatment modalities such as targeted therapy, immune therapy, and chimeric antigen receptor (CAR) T-cell therapy are reviewed. Finally, the indications of hematopoietic stem cell transplantation (HSCT) in AML are discussed.

Seven patients underwent treatment with mesenchymal stem cells (MSCs), together with allogeneic hematopoietic stem cell transplantation (HSCT). MSCs were given to three patients for graft failure and four patients were included in a pilot study. HSCT donors were three human leukocyte antigen (HLA)-identical siblings, three unrelated donors and one cord blood unit. The conditioning was myeloablative in four patients and reduced in three patients. MSC donors were HLA-identical siblings in three cases and haploidentical in four cases. Neutrophil counts >0.5 × 10 9 /l was reached at a median of 12 (range 10–28) days. Platelet counts >30 × 10 9 /l was achieved at a median of 12 (8–36) days. Acute graft-versus-host disease (GVHD) grade 0–I was seen in five patients. Two patients developed grade II, which in one patient evolved into chronic GVHD. One severe combined immunodeficiency (SCID) patient died of aspergillosis, the others are alive and well. One patient, diagnosed with aplastic anemia had graft failure after her first transplantation and severe Henoch–Schönlein Purpura (HSP). After retransplantation of MSCs and HSCs, she recovered from both the HSP and aplasia. Thus, co-transplantation of MSC resulted in fast engraftment of absolute neutrophil count (ANC) and platelets and 100% donor chimerism, even in three patients regrafted for graft failure/rejection.

Among hematopoietic stem cell transplant (HSCT) recipients, morbidity and mortality are largely related to infectious diseases, many of which are vaccine-preventable. The present study aimed to assess the immune response of HSCT patients to the tetanus, diphtheria, and acellular pertussis inactivated vaccine (Tdap). This quasi-experimental study, with individually matched data collection in HSCT patients from January 2018 to December 2020, evaluated sixteen patients for their immune response to each of the Tdap components. The immunization schedule included three doses of Tdap, with a minimum interval of 30 days between each dose, starting at 6 months post-transplantation. Immune competence was measured before vaccination by quantifying CD4+, CD8+, and CD19+ frequencies, as well as serum immunoglobulin levels. After the complete immunization schedule, for diphtheria, we observed a Geometric Mean Concentration (GMC) rise from 0.2509 IU/mL at baseline to 0.7544 IU/mL post-vaccination, p = 0.001. For tetanus, GMC increased from 0.353 to 1.153 IU/mL, p = 0.001. For pertussis, GMC was 31.9 IU/mL before and 99.3 IU/mL after vaccination. Due to the non-normal distribution of pertussis data and a marginal frequentist result (p = 0.077), we used Bayesian analysis assuming an exponential distribution. This model identified a significant increase in antibody levels, with a posterior mean difference (θ) of 58.55 IU/mL and a 95% Credibility Interval (23.9–109.0) that excluded zero, confirming an adequate vaccine response. For the entire Tdap vaccination period, CD4+ cell frequencies remained low, while CD8+, CD19+, and immunoglobulin titers remained within normal range. Immune responses to each of the Tdap vaccine components were not affected (p > 0.05) by any of the clinical, demographic, or immunological variables assessed. The overall Tdap vaccine post-transplant response was considered adequate for diphtheria, tetanus, and pertussis. These findings highlight the immune response to Tdap in transplanted patients and may inform future vaccination guidelines. •This study confirms that the acellular pertussis inactivated vaccine (Tdap) induces an adequate immune response against diphtheria, tetanus, and pertussis.•Detectable pre-vaccination antibody titers for diphtheria, tetanus, and pertussis were present, and these titers increased following Tdap vaccination.•Immune responses to the Tdap vaccine were not affected by any of the assessed variables, including CD8+, CD19+, and serum immunoglobulin.

Recipients after hematopoietic stem cell transplantation (HSCT) or chimeric antigen receptor T-cell (CAR-T) therapy are at increased risk for unfavorable outcomes after SARS-CoV-2 infection. The efficacy of COVID-19 vaccines remains undetermined in this vulnerable population, we therefore conducted a pooled analysis to evaluate the immune response after vaccination. A total of 46 studies were finally included, comprising 4757 HSCT and 174 CAR-T recipients. Our results indicated that HSCT and CAR-T recipients had an attenuated immune response to SARS-CoV-2 vaccination compared with healthy individuals, while time interval between transplant and vaccination, immunosuppressive therapy (IST) and lymphocyte counts at vaccination significantly affected the humoral response in HSCT recipients. In addition, seroconversion was significantly higher in patients with BCMA-based CAR-T than those with CD19-based CAR-T. Thus, an adapted vaccination strategy for HSCT and CAR-T recipients may be required, and further research on the effect of a booster dose of COVID-19 vaccine and the role of cellular response after vaccination is warranted.