Expert Review of Hematology最新文献

Background: Studies showed that lncRNA HCP5 was associated with a variety of autoimmune diseases. This study evaluated the diagnostic potential of lncRNA HCP5 for immune thrombocytopenia (ITP) and its prognostic value in predicting disease progression, offering clinical application insights.

Research design and methods: This study analyzed 40 ITP patients and 40 controls. qRT-PCR measured lncRNA HCP5 expression, while flow cytometry quantified Th17/Treg percentages. Pearson correlation assessed HCP5-clinical feature relationships. ROC analysis determined diagnostic potential, and Kaplan-Meier/Cox regression evaluated prognostic significance.

Results: ITP patients showed decreased platelet counts, Treg percentages, and lncRNA HCP5 levels, but increased Th17%s versus controls. LncRNA HCP5 showed positive correlation with platelets/Tregs but negative with Th17 cells, and was associated with ITP bleeding severity. With a cutoff of 0.825, lncRNA HCP5 had an AUC of 0.979 for ITP diagnosis, sensitivity of 0.900, and specificity of 0.925. Kaplan-Meier analysis showed increased 1-year recurrence with low HCP5 expression, and Cox regression confirmed it as a poor prognostic factor.

Conclusions: LncRNA HCP5 expression correlated significantly with Treg cell percentage, Th17 cell percentage, and the degree of bleeding in ITP patients. LncRNA HCP5 has high diagnostic and prognostic value for ITP.

Introduction: Cytopenia is one of the most common adverse events after BCMA chimeric antigen receptor (CAR) T cell therapy in the treatment of relapsed multiple myeloma (MM). The term Immune Effector Cell Associated Hematotoxicity (ICAHT) was coined to describe the unique hematological toxicities following novel CAR T cell therapies. The management of prolonged ICAHT ( > 30 days) is quite challenging, and patients have high incidences of infections, require prolonged transfusion support and have an increased non-relapse mortality. Stem cell boost (SCB) leads to prompt and durable count recovery and can minimize long-term complications while enabling therapeutic options in the post CAR T-cell therapy relapse setting.

Areas covered: Herein we review current data on ICAHT, determine how SCB can lead to improved outcomes, and offer a view on future applications of SCB. The database 'pubmed' was searched for the terms 'CAR-T,' 'ICAHT', and 'Stem Cell', and results as well as selected citations were used for the present study.

Expert opinion: SCB for prolonged ICAHT improves morbidity and potentially mortality. Future use of SCB will depend on the long-term outcomes of CAR-T cell therapy in earlier treatment lines. For patients with high likelihood of ICAHT, prophylactive stem cell collection should be considered.

Background: Artificial Intelligence (AI) is increasingly vital in transfusion medicine for enhancing service quality and efficiency. However, bibliometric studies in this area are scarce. This analysis maps current and emerging research trends.

Research design and methods: Publications from 1 January 2000 to 31 August 2025, were retrieved from the Web of Science Core Collection. VOSviewer, CiteSpace, and Excel were used to visualize contributions and trends across authors, institutions, journals, and countries.

Results: Among 159 publications, the U.S.A. China, and India led in output. The University of Colorado was the top institution, while Transfusion had the highest citations. Axel Hofmann was the most cited author. Keywords such as 'machine learning' and 'deep learning' highlight the rapid adoption of advanced AI technologies.

Conclusions: This study outlines current trends and emerging frontiers, offering valuable insights and guidance for future AI applications in transfusion medicine.

Background: Hemophilia A is an inherited bleeding disorder caused by a deficiency of clotting factor VIII (FVIII), leading to joint bleeding and arthropathy. While prophylactic FVIII therapy reduces bleeding, evidence suggests maintaining higher FVIII levels (FL) may better protect joint health, particularly in physically active individuals and those with joint damage. However, data on optimal FLs required to prevent joint deterioration and complications remains limited.

Research design and methods: This study utilized the Sheffield Elicitation Framework (SHELF) methodology to elicit expert opinions on optimal FLs for patient-centric and clinical outcomes. Five European hemophilia experts participated in virtual workshops, providing probability-based estimates of FLs required to prevent bleed-related hospitalizations, orthopedic procedures, target joint incidence, and support physical activity without additional infusions or joint damage.

Results: Experts consistently recommended higher FLs for individuals with joint damage than for those without. Optimal average FLs ranged from 24% to 51%, exceeding traditionally recommended prophylactic trough levels (3-5%). Considerable uncertainty was noted around FLs for physical activity, reflecting the complexity of individualized care.

Conclusions: Standard prophylaxis regimens may not provide sufficient protection for all patients, particularly those with joint damage. A personalized treatment approach, targeting higher FLs when necessary, may be critical for optimizing outcomes.

Background: This systematic review and network meta-analysis(NMA) comprehensively evaluated the efficacy and safety of non-first-line drugs in the treatment of adult patients with immune thrombocytopenia (ITP).

Researchdesign and methods: PubMed, Web of Science, Embase, and the Cochrane Library were systematically searched. Randomized controlled trials (RCTs) investigating Count data were extracted in the form of event occurrences/non-occurrences. NMA was carried out via R.

Results: 29 RCTs were encompassed. In contrast to placebo, the avatrombopag 20 mg group demonstrated the highest PR (RR = 12.23, 95% CrI: 5.48-33.72). The combination of eltrombopag and danazol exhibited the lowest incidence of bleeding events (RR = 0.31, 95% CrI: 0.16-0.57), while the avatrombopag 5 mg group had the lowest incidence of SAEs (RR = 0.44, 95% CrI: 0.22-0.84). The comprehensive evaluation suggested that romiplostim, initiated at a dose of 1 μg/kg within a dose-adjustment regimen, may confer one of the most favorable benefit - risk profiles, with a relatively high PR (SUCRA = 75.1%) and a low incidence of bleeding events (SUCRA = 54.8%).

Conclusions: When initiating therapy with romiplostim at a dose of 1 μg/kg and subsequently titrating the dosage according to the patient's platelet response, romiplostim may represent one of the most effective therapeutic options.

Registration: The study protocol for this systematic review was registered in the International Prospective Registry of Systematic Reviews (PROSPERO) database (http://www.crd.york.ac.uk/prospero/), and it was allocated the PROSPERO identification number.

Introduction: Fragmentation across operations, data systems, governance, and regulation leaves many blood supply networks ill-equipped to provide timely, equitable, and crisis-resilient transfusion support. Public health emergencies, such as COVID-19 and natural disasters, have exposed the human and economic costs of these structural flaws, and how variability in practice about who can see and share data still impedes coordination even when the overall blood inventory is adequate.

Areas covered: This Critical Perspective examines blood supply coordination challenges in high-income countries, focusing on governance structures, operational isolation, regulatory inconsistencies, and data system incompatibilities. We analyze evidence from crisis events including pandemics, natural disasters, and mass casualty incidents to illustrate coordination failures and successful response models. The review synthesizes peer-reviewed literature identified through PubMed searches (January 2010 - September 2025), supplemented by regulatory documents, industry reports, and government policy analyses from blood regulatory agencies in the United States, United Kingdom, Canada, and other high-income countries.

Expert opinion: Effective solutions require coordinated interventions across multiple domains rather than isolated or localized improvements. Priority areas include governance structures that enable cross-institutional collaboration, interoperable data systems with standardized sharing protocols, regulatory frameworks that incentivize coordination, and value-based reimbursement models that reward system-wide performance.

Introduction: Chronic myeloid leukemia (CML) is a myeloproliferative disorder, usually diagnosed in its chronic phase (CP), often requiring life-long therapy. Despite the effectiveness of targeted therapy with tyrosine kinase inhibitors (TKIs), resistance or intolerance may occur, requiring a switch. The probability of achieving guideline-recommended cytogenetic and molecular responses declines from first (1 L) to second (2 L) and subsequent lines. About half of the patients receiving third line TKI treatment exhibit resistance or intolerance to prior TKIs. Post-2 L, patients experience non-durable responses, persistent adverse events, and a decreased quality of life.

Areas covered: This narrative review is based on targeted literature search in Medline via PubMed and expert clinical input from Indian hematologists to cover unmet needs of patients with CP-CML post-2 L of TKI therapy. We present an overview of the available clinical data, outline the challenges associated with treatment resistance and intolerance, identify gaps in patient management, and discuss personalized treatment approaches that could bridge these gaps and improve patient outcomes post-2 L.

Expert opinion: Management of CP-CML beyond 2 L remains a significant clinical challenge due to resistance, intolerance, and suboptimal long-term responses with currently available TKIs. Integrating newer agents like asciminib or ponatinib, can help overcome resistance and improve patient outcomes.

Background: Inherited bone marrow failure syndromes (IBMFS) often present with overlapping features and may be misdiagnosed as idiopathic aplastic anemia (iAA). Genetic testing is critical for accurate diagnosis, especially in consanguineous populations.

Research design and methods: We retrospectively analyzed 41 pediatric patients who underwent genetic evaluation for suspected bone marrow failure. Clinical features, diagnostic classifications, and genetic findings were reviewed to assess diagnostic yield and impact.

Results: The cohort included 21 males and 20 females (median age: 8 years). Pancytopenia was the most common presentation (27/41; 65%), half (20/41; 49%) were products of consanguineous marriage. iAA was the initial diagnosis in 56% (23/41). Genetic testing identified pathogenic/likely pathogenic (P/LP) variants in 14 patients (34%), enabling a molecular diagnosis. An additional 13 patients (32%) had variants of uncertain significance, one of which was later reclassified as LP, confirming Noonan syndrome. Genetic findings prompted diagnostic revisions, including Fanconi anemia, Congenital Amegakaryocytic Thrombocytopenia, Shwachman-Diamond syndrome, and Diamond-Blackfan anemia. Commonly affected genes included MPL, FANCA, followed by DANJC21.

Conclusions: In this Pakistani cohort, genetic testing clarified IBMFS diagnoses in 34% of cases, matching global yields. It enhanced diagnostic precision, informed management, and supported family counseling, though high VUS rates underscore the need for ongoing reclassification and multidisciplinary care.

Background: The diagnostic and prognostic value of the systemic immune inflammation (SII) index for immune thrombocytopenia (ITP) remains to be determined.

Research design and methods: A retrospective analysis was conducted on 120 ITP children admitted to The Affiliated Children's Hospital of Xiangya School of Medicine, Central South University (Hunan Children's Hospital) between January 2017 and June 2022. A control group comprised 79 healthy children undergoing outpatient physical examinations during the same period. Based on recurrence within 2 years post-treatment, ITP children were categorized into recurrence and non-recurrence groups. Multivariate Cox regression analysis was conducted, and receiver operating characteristic curves and Kaplan-Meier curves were plotted.

Results: ITP children exhibited significantly reduced SII. The SII index assisted in predicting ITP occurrence and recurrence within 2 years post-treatment. PLT count and SII were independent protective factors against disease recurrence within 2 years post-treatment in ITP patients. A decrease in SII increased the risk of disease recurrence within 2 years.

Conclusions: ITP children exhibit reduced SII index. The SII index can assist in the diagnosis of ITP and demonstrates strong predictive value for disease recurrence risk within 2 years post-treatment in ITP children.