Hypoplastic myelodysplastic syndrome (MDS) often overlaps clinically with aplastic anemia and frequently involves clonal hematopoiesis. Thrombopoietin receptor agonists (TPO-RAs), including eltrombopag, are increasingly used to treat cytopenias in hypoplastic marrow failure syndromes; however, their long-term effects on clonal dynamics and marrow fibrosis remain incompletely defined. We report a case of ASXL1- and TET2-mutated hypoplastic MDS, initially managed as aplastic anemia, in which eltrombopag therapy was associated with a biphasic clinical course. Hematologic parameters initially improved with reduced transfusion requirements; however, prolonged exposure was followed by progressive marrow fibrosis (MF-2 to MF-3), worsening cytopenias, and expansion of a minor paroxysmal nocturnal hemoglobinuria (PNH) clone. Serum lactate dehydrogenase levels increased in parallel with fibrosis progression. After eltrombopag discontinuation, the patient achieved transfusion independence within 3 months and substantial regression of marrow fibrosis, accompanied by recovery of marrow cellularity exceeding the pretreatment baseline. Longitudinal molecular analysis demonstrated modest fluctuations in variant allele frequencies without evidence of overt unidirectional clonal progression. The temporal association between fibrosis progression and hematologic deterioration, together with sustained improvement after drug withdrawal, suggests that eltrombopag may have contributed to treatment-associated microenvironmental remodeling that constrained hematopoiesis after an initial stimulatory phase. This case suggests that eltrombopag-associated marrow fibrosis may be reversible even in the presence of clonal hematopoiesis, and that clinical worsening during TPO-RA therapy does not necessarily indicate irreversible clonal evolution. Careful reassessment of marrow morphology, clonal dynamics, and treatment-related niche perturbation may be warranted in patients with hypoplastic MDS who deteriorate during TPO-RA therapy.

Allogeneic hematopoietic stem cell transplantation (HCT) is a curative treatment for acquired aplastic anemia (AA). More than 1,000 cases of transplants for AA are performed annually in China, and transplant protocols have been continuously refined over recent years. To characterize contemporary transplant practices in China and provide useful insights for global professionals, this study analyzed the 2024 Chinese National Registry data. In 2024, 1,919 transplants were reported among 1,893 patients by 158 centers. Haploidentical donor (HID) was the leading donor source (57.22%). Matched sibling donor (MSD) and unrelated donor (URD) were used in 19.23% and 17.87% of cases, respectively. The G-CSF/ATG-based platform (84.42%) was the predominant transplant modality, followed by the ATG/ATLG+PTCY platform (14.17%). Low-dose busulfan was frequently used in conditioning regimens for both platforms. Due to the dominant use of HID and peripheral blood stem cell grafts, novel graft-versus-host disease (GVHD) prophylactic strategies have been explored. Based on a representative contemporary cohort of 226 patients, we report a favorable 1-year survival rate of 92.0% and a GVHD-/failure-free survival rate of 77.8%, which are comparable to reports from the EBMT and CIBMTR. Moreover, modern HCT practice supports broader consideration of transplantation for older patients, especially those aged 40-50 years. In conclusion, our report offers a comprehensive overview of the current HCT landscape in China and serves as a valuable resource for global practice.

Allogeneic hematopoietic cell transplantations (allo-HCT) is a procedure with long history and well-documented efficacy in treating various malignant and non-malignant diseases of the hematopoietic system. While there is a potential for late complications even over 10 years following allo-HCT, the data on longer observations are limited due to loss in follow-up, personnel and organizational changes in transplant centers, and focusing on new promising therapies instead of HCT. The study aimed to evaluate 15- and 20-year overall and progression-free survival of allo-HCT recipients in our center. During the inclusion period from 1999 to 2014, we identified 269 allo-HCT recipients. Survival analyses were performed using Kaplan-Meier curves, the Cox proportional hazards model, and the log-rank test. First, we evaluated 15- and 20-year survival rates for the entire cohort of allo-HCT recipients from matched related donor (MRD) and matched unrelated donor (MUD). Then we performed similar analyses for allo-HCT recipients due to acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myeloid leukemia (CML), myelodysplastic neoplasms (MDS), non-Hodgkin lymphomas (NHL), and severe aplastic anemia (SAA). It was confirmed that survival benefit continues also during long-term observation. Notably, the plateau effect of PFS was often observed over 20 years following allo-HCT, implying the ability to cure in a relatively large group of patients with otherwise incurable diseases.

Chronic hepatitis B virus (HBV) infection is associated with both hepatic and extrahepatic complications. While liver-related outcomes are well characterized, the burden, demographic patterns, and survival associated with extrahepatic manifestations (EHMs) remain incompletely defined. This study aimed to evaluate the spectrum, incidence, and survival impact of EHMs in a large, geographically diverse cohort of patients with chronic HBV infection. We conducted a retrospective cohort study using the TriNetX Global Research Network, including 2,32,820 patients aged 0 to 90 years with chronic HBV infection. Demographic variables included age, sex, race, ethnicity, and geographic region. EHMs assessed comprised renal (glomerulonephritis), vasculitic (polyarteritis nodosa), musculoskeletal (arthralgia), hematologic (aplastic anemia), immunologic (cryoglobulinemia), and neurologic (polyneuropathy) conditions. Incidence proportions and incidence rates per person-day were calculated and stratified by demographic characteristics. Survival probabilities were estimated for each manifestation. The mean age of the cohort was 60 ± 14 years, and 56.9% were male. Racial distribution included Asian (34.4%), White (15.4%), Black or African American (12.1%), and unknown race (34.4%). Most patients were non-Hispanic (53.6%), with 44.1% reporting unknown ethnicity. Patients were represented across all US regions and internationally. Glomerulonephritis occurred in 1.26% of patients, with higher incidence in older adults, males, and Native Hawaiian/Other Pacific Islander and Hispanic/Latino populations. Polyarteritis nodosa was rare (0.04%) and predominated in older individuals, American Indian/Alaska Native, and Hispanic/Latino patients. Arthralgia was the most frequent EHM (13.76%), increasing with age and more common among females and Black or African American individuals. Cryoglobulinemia (0.10%) and aplastic anemia (0.12%) were uncommon. Polyneuropathy affected 3.27% of patients, with higher prevalence among older adults and males. Chronic HBV infection is associated with a wide spectrum of extrahepatic manifestations, with substantial variation by demographic factors. These findings underscore the need for comprehensive multisystem surveillance and targeted risk stratification in patients with chronic HBV infection.

Acute Splenic Sequestration Crisis (ASSC) and Transient Aplastic Crisis (TAC) are severe, but distinct, hematologic complications of sickle cell disease (SCD). ASSC typically presents with acute splenomegaly and anemia due to red blood cell (RBC) pooling, while TAC, often secondary to Parvovirus B19 infection, results in a sudden halt in erythropoiesis and reticulocytopenia. The concurrence of these two conditions is rare, particularly in adults and non-Hb-SS genotypes. We describe a 36-year-old woman with Hb-SC and Hb-G Philadelphia variant SCD who presented with non-specific symptoms who was found to have marked splenomegaly and laboratory findings of severe anemia (hemoglobin 3.8 g/dL), thrombocytopenia, and reticulocytopenia. These combined findings raised concern for ASSC with suspected TAC, especially in light of her positive Parvovirus B19 IgM antibodies. We describe the importance of having a structured and precise diagnostic work-up to elucidate differences between the two conditions in the absence of a bone marrow biopsy, and the rarity of concurrent ASSC with presumed TAC in a patient with Hb-SC SCD. Our case highlights that early recognition of overlapping sickle cell complications despite a bone marrow biopsy is critical as it led to timely diagnosis, optimized management strategies, and reduced morbidity.

Fanconi anemia (FA) is a rare inherited disorder of bone marrow failure that presents with progressive pancytopenia, multiple congenital abnormalities, and an increased predisposition to various malignancies. We describe a 19-year-old male who reported generalized weakness, shortness of breath on exertion, and a dry cough persisting for 1 month. He had undergone three blood transfusions within the preceding 3 months. Clinical examination revealed marked pallor without evidence of jaundice, cyanosis, clubbing, or lymphadenopathy. Laboratory investigations showed pancytopenia, while bone marrow biopsy demonstrated normocellular marrow with trilineage hematopoiesis and mild hemophagocytosis. Radiological assessment identified bilateral ectopic kidneys, and serum ferritin levels were significantly elevated (2753.93 ng/mL). These findings raised a strong clinical suspicion of FA, which was subsequently confirmed through comprehensive genetic testing. This case underscores the importance of considering FA in young adults with unexplained pancytopenia and congenital anomalies, highlighting the need for an integrated, multidisciplinary diagnostic and management approach.

Hematopoietic stem-cell transplantation (HSCT) has evolved from a salvage procedure for otherwise-fatal leukemia into a curative modality spanning nearly every hematological malignancy and several non-malignant disorders. The contemporary landscape has been reshaped by three converging forces: the refinement of disease-specific risk stratification (European LeukemiaNet [ELN] 2022 for acute myeloid leukemia, Molecular International Prognostic Scoring System [IPSS-M] for myelodysplastic syndromes, Mutation-Enhanced International Prognostic Scoring System [MIPSS70+ v2.0], and Myelofibrosis Transplant Scoring System [MTSS] for myelofibrosis); the integration of measurable residual disease (MRD) into dynamic, response-adapted transplant decisions; and the approval of immune effector cell therapies that have displaced transplantation from several long-standing indications while creating new ones (bridge-to-transplant, post-CAR-T consolidation). In parallel, post-transplant cyclophosphamide (PTCy) has largely equalized outcomes across matched sibling, matched unrelated, and mismatched alternative donors, and novel agents (ruxolitinib, belumosudil, axatilimab) have materially reduced graft-versus-host disease (GVHD) morbidity. This narrative review synthesizes current indications for autologous (auto-HSCT) and allogeneic (allo-HSCT) transplantations in adults, and flags areas of persistent controversy where randomized data are still maturing. Across all indications, the clinician's question is shifting from "transplant or not?" towards "which donor, which conditioning, which bridge, and which post-transplant maintenance?", each tailored to disease biology, MRD trajectory, and patient fitness.

Pancytopenia is a significant hematological complication in patients with systemic lupus erythematosus (SLE), with an estimated prevalence of 10-40%. It is defined as a simultaneous decrease in red blood cells, white blood cells, and platelets. Although isolated cytopenias are more common, pancytopenia may indicate severe disease activity or secondary complications, such as bone marrow suppression or hemophagocytic syndromes. This review aims to summarize and synthesize current knowledge on the etiology, pathophysiology, diagnostic approach, and treatment strategies for pancytopenia in SLE, as well as its differentiation from other causes of bone marrow failure. The pathogenesis of pancytopenia in SLE is multifactorial and includes drug-induced bone marrow suppression, hypersplenism, myelofibrosis, macrophage activation syndrome (MAS), and autoimmune bone marrow failure. The diagnostic evaluation includes hematologic assessment, bone marrow examination, and exclusion of alternative diagnoses, such as aplastic anemia and paroxysmal nocturnal hemoglobinuria. Therapeutic management depends on the underlying cause. We also summarize published case reports comparing treatment approaches and clinical outcomes. The role of rituximab (RTX) in the management of pancytopenia and isolated cytopenias associated with SLE is discussed in detail. Particular attention is given to its mechanisms of action, safety profile and mixed clinical outcomes, documented in multicenter retrospective cohort studies, meta-analyses, and case series. Early recognition of pancytopenia remains a clinical challenge and requires well-structured diagnostic and therapeutic strategies. A thorough understanding of its underlying mechanisms and clinical manifestations is essential to avoid delays in treatment and prevent complications.