Journal of hematology最新文献

Background: Standard management of acute immune thrombocytopenic purpura (ITP) remains debated, with some advocating observation for mild cases, while others recommend pharmacological intervention to accelerate platelet recovery in children with severe thrombocytopenia (TCP) (platelet count < 20,000/mm³) or significant mucosal bleeds. Corticosteroids, particularly prednisolone (PDN), are a widely used first-line treatment due to their rapid immunosuppressive effect. This prospective cohort study evaluated the effectiveness of short-course high-dose PDN (4 mg/kg/day for 7 days) in treating children aged 1 - 12 years with newly diagnosed acute ITP. The study aimed to assess the clinical and hematological profile of these children and the mean time to platelet recovery.

Methods: A total of 61 patients were enrolled, and their response to treatment was monitored at various intervals, including 48 h, 72 h, day 7, 1 month, and 3 months.

Results: Results revealed a rapid platelet recovery in patients receiving high-dose PDN, with 83.6% of patients achieving platelet counts greater than 50,000/mm³ by day 7. Additionally, 91.8% maintained platelet recovery at 1 month. The study also found that the age group 6 - 12 years had a higher risk of persistent ITP (24.5%), highlighting the importance of close monitoring in this demographic. While the treatment was generally well tolerated, some mild side effects like hypertension and glycosuria were observed.

Conclusion: The study suggests that short-course high-dose PDN can be an effective first-line therapy for acute ITP. It promotes faster platelet recovery and reduces hospitalization time with minimal adverse effects.

Background: Vaso-occlusive crisis (VOC), a common clinical manifestation of sickle cell disease (SCD), is mediated by a series of inflammatory responses. Conversely, a high fetal hemoglobin (Hb F) level is a known factor that reduces the severity of clinical presentations associated with SCD. Since the association between cytokine profiles and high Hb F levels in SCD is not well studied, this study aims to investigate the interaction between cytokines and Hb F levels in SCD patients from Odisha, India.

Methods: A total of 276 patients with SCD were recruited for the study, including 180 in steady state and 90 in crisis state. Additionally, 117 individuals with sickle cell trait (SCT) and 128 healthy controls were included for comparison.

Results: The study revealed that the total white blood cell count and interleukin (IL)-6 levels were significantly higher in crisis state SCD patients, while the red cell distribution width, IL-10, and Hb F level were significantly higher in steady state SCD patients. Most importantly, the logistic regression analysis showed that the interaction of Hb F with the cytokines IL-10, IL-1β, and IL-17 provided three times greater protection from crisis in SCD patients.

Conclusion: The significance of these preliminary findings has been discussed in terms of prognostic markers and supplements to increase the efficacy of hydroxyurea used to enhance Hb F production.

Systemic amyloidosis has diverse, often nonspecific, clinical manifestations that overlap or mimic other medical disorders, making amyloidosis a diagnostic challenge. We present a case of a middle-aged female who presented with skin thickening, fatigue, arthritis, and macroglossia, which were initially thought to be due to systemic sclerosis. With no response to immunosuppressive therapies, she was tested for plasma cell dyscrasias. Additional work-up and cardiac biopsy were positive for amyloid light chain (AL) amyloidosis. The diagnosis was delayed by 2 years because the protein electrophoresis ordered at the initial encounter was not accompanied by serum-free light chain testing. This case emphasizes the importance of considering amyloidosis in patients with unexplained systemic symptoms and highlights the role of a comprehensive diagnostic evaluation.

We present the case of a 64-year-old man who suffered a splenic rupture following a fall. A peripheral blood smear showed mononuclear cells with "hairy" cytoplasmic projections. A computed tomography (CT) angiogram revealed splenomegaly with a hematoma and active contrast extravasation. He underwent coil embolization of the splenic artery but subsequently developed worsening abdominal pain and ileus, requiring a splenectomy. Pathological examination of the spleen showed extensive infiltration by hairy cell leukemia (HCL). Bone marrow biopsy revealed hypercellular marrow predominantly infiltrated by HCL cells positive for CD20, CD103, and the BRAF V600E mutation. After the splenectomy, pancytopenia gradually improved. The symptoms of the patient, such as fatigue, weight loss, and night sweats, were also resolved. Circulating HCL cells were significantly reduced, and the "hairy" morphology became smoother. To our knowledge, this morphologic change of the hairy cells after splenectomy has not been reported in the past. Its mechanism is not known. We postulate that splenectomy may induce molecular or immunological changes that alter HCL cell behavior, which warrants further research.

Evans syndrome (ES) is an autoimmune disorder of unknown etiology characterized by autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). In this systematic review, we analyzed the reported cases of ES secondary to coronavirus disease 2019 (COVID-19) infection or COVID-19 vaccination. We examined their clinical presentation, temporality between events, diagnostics tests, and treatment regimens. Our search in four databases from December 2019 to September 2023 yielded 16 case reports that met eligibility criteria for inclusion. COVID-19 and ES symptoms were defined to assess the timeline between infection/vaccination and ES onset. Finally, treatment efficacy was categorized as complete, partial, or no response based on standard hematological criteria. Eleven cases of ES were associated with COVID-19 infection, and five cases of ES were associated with COVID-19 vaccination. All 16 cases presented with anemia, thrombocytopenia, and a positive Coombs test. Four of the five patients from the vaccination subset were found to have an additional autoimmune disease as a comorbidity on presentation. For cases of ES secondary to COVID-19 infection, six patients had concomitant symptoms of COVID-19 and ES on presentation, and four patients had ES symptoms occurring from 5 days to 3 weeks following COVID-19 infection. The remaining case presented a patient with a 3-week history of ES symptoms before a positive COVID-19 test and further ES workup on admission. For the five cases of ES post-COVID-19 vaccination, all five patients presented with ES with a mean presentation time of 9 days following vaccination. Regarding treatment, intravenous immunoglobulin (IVIG) emerged as the primary regimen, administered in 13 out of the 16 cases. Among the infection-related cases, the most frequent treatment outcome was a partial response in both AIHA and ITP, observed in five of the 11 patients. In the vaccination-related cases, a partial response for AIHA and a complete response for ITP were noted in three of the five patients. Overall, while the evidence points to a temporal association especially between COVID-19 vaccination and the onset of ES, larger studies are necessary to strengthen these findings. In terms of management, early initiation of corticosteroids and IVIG appears effective as first-line therapies; however, standardized treatment protocols are needed to help reduce complications associated with COVID-19-related ES.

After ciltacabtagene autoleucel (cilta-cel) in multiple myeloma, 5% of patients can develop parkinsonism, with a high fatality rate. The pathogenesis and optimal therapy of parkinsonism from B-cell maturation antigen chimeric antigen receptor T-cell (CAR T-cell) therapy are unknown. Parkinson's disease occurs from the loss of dopaminergic neurons in the substantia nigra. However, in cilta-cel-associated parkinsonism, dopamine transporter imaging is normal, rendering traditional agents such as carbidopa/levodopa ineffective. Thus, the pathogenesis of cilta-cel-associated parkinsonism and Parkinson's disease is distinct. As CAR T-cell therapy for multiple myeloma is expanding and moving to earlier lines, the need to optimize therapy for parkinsonism, a potentially life-threatening complication, becomes more urgent. This report presents the first documented cases of two patients with immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome and cilta-cel-associated parkinsonism, effectively treated with ruxolitinib.

Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative approach for many hematologic disorders, and donor cell leukemia (DCL) remains a complication rarely observed after HSCT. The number of reported cases of DCL slightly exceeds 100, with acute myeloid leukemia (AML) being the most common type. To date, only a few cases of chronic lymphocytic leukemia (CLL) emerging from donor cells have been described in the literature. Here, we report two cases of CLL of donor origin, which emerged in patients after HSCT for AML. In the reported cases, patients maintained complete remission of AML after HSCT. Both donors were free of CLL before transplantation. Several years after HSCT (9 and 3 years, respectively), lymphocytosis with proven B-cell clonality was detected in recipients prompting a detailed blood analysis to be performed also in donors. CLL population was demonstrated in both cases. The first donor-recipient pair did not meet criteria for CLL treatment and eventually died from causes not related to underlying hematologic malignancy. The second couple received Bruton's tyrosine kinase inhibitors with good disease control. The presented cases raise the question of possible clonal evolution of B-lymphocytes in donor-origin cells. Prospective screening of potential donors for pre-malignant alterations remains a matter of discussion.

Relapsed/refractory diffuse large B-cell lymphomas (R/R DLBCLs) have an extremely poor prognosis, with no established salvage chemotherapy currently available. Polatuzumab, rituximab, and bendamustine combination therapy (Pola-BR) has been approved as a new therapeutic option for R/R DLBCL. Recently, chimeric antigen receptor T-cell therapy and bispecific antibodies have induced long-term remission in many patients with R/R DLBCL. However, allogeneic transplantation remains potentially curative for patients unresponsive to the abovementioned treatments. While allogeneic transplantation can also cause various adverse events, hemorrhagic cystitis is a particularly severe complication that requires effective prevention strategies. Here, we report two cases of severe BK virus-associated hemorrhagic cystitis (BKV-HC) that developed after successive cord blood transplantation with myeloablative conditioning and Pola-BR treatment for early-relapsed DLBCL. Both patients received Pola-BR after undergoing multiple salvage therapies and developed early-onset BKV-HC post-transplant, demonstrating the effects of Pola-BR and myeloablative conditioning. We analyzed the shared characteristics between these two cases to distinguish between the factors that trigger the onset of BKV-HC and those that contribute to its severity. Based on the differences in the clinical course between the two cases, we propose prevention strategies for BKV-HC and identify treatment strategies for Pola-BR in patients with R/R DLBCL undergoing allogeneic transplantation.

Juvenile myelomonocytic leukemia (JMML) is an aggressive pediatric myelodysplastic/myeloproliferative neoplasm characterized by RAS pathway mutations and significant heterogeneity. Minimal residual disease (MRD) monitoring is crucial for evaluating treatment response and predicting relapse risk. MicroRNA (miRNAs), small non-coding RNAs with pivotal roles in gene regulation, have emerged as promising biomarkers for JMML MRD detection. This review explores the mechanistic role of miRNAs in JMML pathogenesis, emphasizing their diagnostic, prognostic, and therapeutic potential. Dysregulated miRNA profiles correlate with distinct JMML subgroups and disease progression, suggesting utility in non-invasive MRD monitoring. Emerging evidence highlights miR-150-5p as a tumor suppressor targeting STAT5b and its therapeutic potential in ameliorating JMML's aberrant signaling pathways. We compare traditional MRD methods, such as flow cytometry and polymerase chain reaction (PCR), with miRNA-based techniques, underscoring the latter's superior sensitivity, specificity, and non-invasiveness. Recent advances in miRNA profiling technologies, including next-generation sequencing and digital PCR, enable precise detection of residual leukemic cells and support personalized treatment approaches. Despite significant progress, challenges persist in standardizing miRNA-based assays and validating their clinical utility. Ethical considerations, including patient privacy and informed consent, remain critical for integrating miRNA diagnostics into routine care. This review provides a comprehensive synthesis of current knowledge on miRNA signatures in JMML, illuminating their transformative potential in MRD monitoring and paving the way for innovative therapeutic strategies.