To evaluate the efficacy of unrelated allogeneic hematopoietic stem cell transplantation (URD-HSCT) in severe aplastic anemia (SAA), this study retrospectively analyzed the clinical data of 206 patients at a single center. The median age was 28 years (range: 5-64 years), and 13.6% had received immunosuppressive therapy prior to transplantation. The median time to neutrophil and platelet engraftment was 12 days (range: 9-22 days / 8-55 days), respectively, with a graft failure rate of 3.4%. The cumulative incidence of grade II-IV acute graft-versus-host disease (aGVHD) was 21.7% (95% CI: 16.6%-27.8%), and that of chronic GVHD was 20.4% (95% CI: 15.4%-26.6%). Survival analysis revealed a 7-year overall survival (OS) rate of 91% for the entire cohort. Notably, the 7-year OS rate was significantly higher in the donor age ≤30 years group compared to the >30 years group (97% vs 85%, P = 0.0028), and in the HLA 10/10 matched group compared to the 9/10 matched group (95% vs 86%, P = 0.023). Multivariate analysis identified donor age >30 years, and HLA 9/10 matching as independent risk factors for OS and graft-versus-host disease-free, relapse-free survival (GRFS). URD-HSCT is an effective treatment for SAA, and selecting an appropriate donor can improve treatment outcomes.

We present a case of aplastic anemia (AA) complicated with gastric cancer that was successfully treated with cyclosporine A (CsA) and romiplostim (ROMI) as a bridging therapy before curative surgery. This patient had untreated gastric cancer at the time of AA diagnosis, and surgery was difficult because of thrombocytopenia. However, CsA and ROMI were effective, resulting in a rapid increase in the platelet count, thereby making surgery for gastric cancer possible. CsA and ROMI as a bridging therapy to curative surgery may therefore represent a feasible treatment option in patients with AA complicated by malignant tumors. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.

Immunosuppressive therapy (IST) plus eltrombopag (EPAG) (IST+EPAG) is the preferred first-line treatment for severe aplastic anemia (SAA) patients over 40 years. However, direct comparison between IST+EPAG and upfront haploidentical hematopoietic stem cell transplantation (HID-HSCT) in this population remains limited. We retrospectively analyzed 123 newly diagnosed SAA patients aged 40-65 who received first-line HID-HSCT (n = 67) or IST+EPAG (n = 56) between 2016 and 2024. Hematological response, survival, and safety were compared. The HID-HSCT group demonstrated superior complete response (CR) rates at 3 months (17.9% vs. 5.4%), 6 months (44.8% vs. 17.9%), and 12 months (64.2% vs. 33.9%) (all P < 0.001). The 3-year overall survival (OS) was comparable between the HID-HSCT and IST+EPAG groups (78.0% vs. 80.3%, P = 0.662), and a landmark analysis conditional on survival to 6 months confirmed comparable subsequent 3-year OS (92.2% vs. 88.9%, P = 0.616). Meanwhile, the 3-year failure-free survival (FFS) was significantly higher in the HID-HSCT group among patients aged 40-49 (82.9% vs. 60.1%, P = 0.045), whereas no difference was observed in those aged 50-65 (P = 0.984). HID-HSCT may offer more durable benefits in selected SAA patients aged 40-49, while its advantage over IST+EPAG is not evident in patients aged 50-65.

Hepatitis-associated aplastic anemia (HAAA) is a rare but life-threatening subtype of acquired aplastic anemia (AA) that predominantly affects children and adolescents, characterized by bone marrow failure developing weeks to months following acute hepatitis. Although HAAA accounts for a small proportion of AA cases, it presents unique diagnostic and therapeutic challenges and remains poorly understood. Most cases are seronegative, with no identifiable viral pathogen, supporting an immune-mediated pathophysiology. Emerging evidence implicates dysregulated cytotoxic T-cell activation, reduced regulatory T-cell function, and a shared immune target within hepatic and hematopoietic tissues. HAAA follows a variable course of hepatitis and pancytopenia, which necessitates a high index of suspicion and thorough evaluation to exclude inherited and secondary causes of bone marrow failure. Management strategies are largely extrapolated from severe AA and include supportive care, immunosuppressive therapy with antithymocyte globulin and cyclosporine, and hematopoietic stem cell transplantation. While outcomes have improved with modern therapy, relapse, treatment failure, and clonal evolution remain challenges. This review synthesizes current knowledge on the epidemiology, pathophysiology, clinical features, diagnostic approach, treatment options, and outcomes of pediatric HAAA, highlighting critical gaps in evidence and the need for prospective studies to guide risk stratification and therapy optimization.

Severe aplastic anemia (SAA) is a T lymphocyte-mediated bone marrow failure disorder. Only porcine ATG (p-ATG) and rabbit ATG (r-ATG) are available in mainland China, with the optimal first-line regimen undetermined. This study compared their pharmacokinetics, efficacy, and safety. Ninety-three SAA patients ineligible for transplantation or lacking a suitable donor (p-ATG, n = 55; r-ATG, n = 38) who were receiving immunosuppressive therapy were prospectively enrolled between 2020 and 2024. The primary endpoint was dynamic changes in lymphocyte subsets. Both ATGs peaked on treatment Day 5; r-ATG cleared significantly faster, with comparable peak active concentrations. The p-ATG group showed a stable early neutrophil increase, whereas the r-ATG group had an initial decline, with comparable recovery at 1 month. The r-ATG group exhibited prolonger lymphopenia, lower CD3[+]/CD4[+] T cells, reduced Treg cells, and higher CD19[+] B-cell proportions. IL-10 was significantly elevated on day 2 after ATG administration. At 6 months, the CRR was 18.87% with p-ATG and 18.92% with r-ATG; ORR was 69.81% with p-ATG and 56.76% with r-ATG. Two-year ORR and overall survival were similar, with no significant differences in infection or serum sickness. P-ATG and r-ATG have distinct pharmacokinetic and immunomodulatory but comparable efficacy and safety profiles, supporting interchangeable use in clinical practice.

Aplastic anemia (AA) is a rare hematological disorder characterized by bone marrow failure and pancytopenia, with viral infections implicated as a significant acquired cause. This scoping review evaluates the pathophysiological mechanisms linking viral infections to AA and assesses current therapeutic strategies for managing virus-associated cases. Following PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines, a scoping review was conducted from April to May 2024 using PubMed, Google Scholar, Web of Science, and ScienceDirect. Eligible studies included those reporting viral infections associated with AA, diagnostic approaches, or therapeutic interventions. Data were extracted on study design, type of virus, diagnostic methods, and treatment strategies. A total of 48 studies were included, identifying parvovirus B19, Epstein-Barr virus, cytomegalovirus, human immunodeficiency virus, hepatitis viruses, human T-cell lymphotropic virus type 1, and SARS-CoV-2 as key pathogens. Rare cases of post-vaccination complications were also reported. Identified mechanisms include direct bone marrow suppression, immune-mediated destruction, and viral reactivation during immunosuppression. Antiviral therapies and hematopoietic stem cell transplantation constitute the primary treatments, and virological monitoring is essential to prevent reactivation. Viral infections play a significant role in AA etiology and progression. Tailored antiviral regimens and proactive virological monitoring are essential for optimizing outcomes. Future research should focus on longitudinal studies to clarify causal relationships and refine therapeutic approaches.

Standard management guidelines generally recommend immunosuppressive therapy (IST) as first-line treatment for older patients with severe aplastic anemia (SAA), whereas the role of upfront haploidentical hematopoietic stem cell transplantation (haplo-HSCT) in this population remains uncertain, particularly in patients with complex comorbidities. We report a 62-year-old woman with SAA, a history of surgically resected stage IA lung adenocarcinoma, and previous left lower extremity deep vein thrombosis treated with venous stent implantation and long-term aspirin therapy. Because no matched sibling or unrelated donor was available, and following individualized counseling regarding IST and transplantation, the patient underwent upfront haplo-HSCT combined with umbilical cord-derived mesenchymal stem cells after a conditioning regimen including fludarabine, busulfan, cyclophosphamide, antithymocyte globulin, and thiotepa. Neutrophil engraftment occurred on post-transplant day +12, and platelet independence was achieved on day +13. Donor chimerism reached 99.6% by day +14 and remained within the complete-chimerism range through the latest available assessment in January 2026. In late December 2025 and January 2026, the patient developed cytomegalovirus infection accompanied by transient trilineage cytopenia; after anti-CMV treatment with maribavir, the infection was controlled and peripheral blood counts subsequently recovered. At the most recent clinical follow-up on August 6, 2026, approximately 17 months after transplantation, no chronic graft-versus-host disease had developed, the previous lung adenocarcinoma remained in remission, and the thrombotic condition remained clinically stable. This case suggests that upfront haplo-HSCT combined with UC-MSCs may represent a feasible individualized treatment option for carefully selected older patients with SAA who lack matched donors, although broader conclusions require larger studies and longer follow-up.