bims-conane Biomed News
on Congenital anemias
Issue of 2025–08–24
eight papers selected by
João Conrado Khouri dos Santos, Universidade de São Paulo



  1. Clin Biochem. 2025 Aug 14. pii: S0009-9120(25)00119-5. [Epub ahead of print] 110990
       BACKGROUND: Capillary electrophoresis is a widely used method for hemoglobin (Hb) fraction separation and relative quantitation, where pre-defined Hb peaks (typically HbA and HbA2) act as reference points to "anchor" the electropherogram and define migration zones. In cases lacking HbA or involving variants with migration patterns similar to HbA or HbA2, mis-anchoring can occur-leading to incorrect zoning of Hb variants. This presents a diagnostic challenge, where follow-up investigations, often including molecular testing, are required to establish an accurate diagnosis.
    CASE REPORT: We report a case of a 43-year-old Thai female who underwent hemoglobinopathy investigation for microcytic anemia. Capillary electrophoresis showed peaks in the HbA (70.8%), HbA2 (24.4%), and HbC (2.9%) zones, as well as two small peaks in the Z11 (0.9%) and HbD (1.0%) zones. Gel electrophoresis at acid pH showed a band in the HbA position and one slightly anodal to the HbF position and at alkaline pH showed a band in the HbC/E position and another slightly anodal to the HbA position. HBB sequencing identified heterozygosity for the pathogenic HbE and clinically benign Hb Hope variants. HBA PCR detected a single alpha globin gene deletion (αα/α-3.7), consistent with alpha thalassemia silent carrier. Reinterpretation of the electropherogram showed that Hb Hope and HbE mis-anchored as HbA and HbA2, respectively, due to their similar migration deltas.
    CONCLUSION: This is the first documented case of compound heterozygosity for Hb Hope and HbE characterized by capillary electrophoresis. It highlights how beta chain variants with similar migration spacing to HbA and HbA2 can mis-anchor, emphasizing the need for molecular testing when results are unclear. Definitive testing helps avoid diagnostic misclassification and ensure accurate interpretation in complex hemoglobinopathy cases.
    Keywords:  Alpha Thalassemia; Hb E; Hb Hope; Hemoglobin Variant; Hemoglobinopathy
    DOI:  https://doi.org/10.1016/j.clinbiochem.2025.110990
  2. Cureus. 2025 Jul;17(7): e88234
      Hemoglobinopathy has a diverse clinical presentation and complications, and is severe among individuals with the homozygous form. It is the most common cause of chronic anemia among affected individuals. Hemoglobinopathy is an inherited blood disorder arising from mutations in globin genes and is broadly categorized into those involving structural changes that produce abnormal hemoglobin variants or defects in globin chain production. This review aims to evaluate the risk factors and outcomes of pulmonary hypertension among individuals with hemoglobinopathies. A search was conducted on PubMed and Google Scholar databases from inception to April 30, 2025. In total, 1,825 articles were synthesized, of which 13 were included in the final qualitative analysis and data extraction. We included English-language original articles published in peer-reviewed journals that reported the risk factors and outcomes of pulmonary hypertension in patients of any age and gender diagnosed with any type of hemoglobinopathy. This review synthesized 13 articles from 10 countries. A total of 2,873 individuals were diagnosed with hemoglobinopathy (1,031 (36%) with sickle cell disease and 1,842 (64%) with β-thalassemia), and 472 were diagnosed with pulmonary hypertension. Among those with pulmonary hypertension, 289 (61%) had sickle cell disease, while 183 (39%) had β-thalassemia. Older age (>40 years), a history of splenectomy, a hemoglobin level of <8 g/dL, frequent blood transfusions, frequent hospitalization for vaso-occlusive crisis, and β-thalassemia were associated with pulmonary hypertension. Some laboratory parameters (serum creatinine, reticulocyte, albumin, nucleated red blood cells, globulin, cell-free hemoglobin, N-terminal pro-B-type natriuretic peptide, high-sensitivity C-reactive protein, soluble vascular cell adhesion molecule, platelet, lactate dehydrogenase) were associated with pulmonary hypertension. Overall, the mortality rate was 27 (10%), with respiratory failure, sudden death, and cor pulmonale as the common causes of mortality. Early recognition and risk stratification for pulmonary hypertension must become integral components of hemoglobinopathy care, particularly in adult patients and those with high-risk profiles. Establishment of a standardized treatment guideline and optimizing the use of disease-modifying therapies, such as hydroxyurea and iron chelators, and exploring novel pharmacologic strategies (endothelin receptor antagonist, phosphodiesterase type 5 inhibitors), may hold promise for altering the trajectory of pulmonary hypertension in this vulnerable group. Our findings confirm that pulmonary hypertension is not only a prevalent complication but also a serious prognostic marker associated with increased morbidity and mortality in this population.
    Keywords:  hemoglobinopathy; outcome; pulmonary hypertension; risk factor; sickle cell disease (scd); thalassemia
    DOI:  https://doi.org/10.7759/cureus.88234
  3. Ann Hematol. 2025 Aug 19.
      Sirolimus is an effective treatment for acquired pure red cell aplasia (aPRCA), but the detail mechanism of sirolimus on red cell differentiation is incompletely understood. The aim of the study was to investigate whether sirolimus can induce the erythroid differentiation of K562 cells and investigate the potential regulation mechanism. Benzidine staining and flow cytometry were performed to determine the benzidine-/CD71-positive K562 cells with or without sirolimus treatment. The expression level of α-/ γ-globin in each group was determined by real-time qPCR, respectively. siRNA-targeted assays were performed to investigate the role of protein tyrosine phosphatase 1 (SHP1) in sirolimus-induced erythroid differentiation of K562 cells. The proportion of benzidine-staining/CD71-positive cells, and α- / γ-globin mRNA expression were increased in K562 cells treated with sirolimus. Sirolimus markedly inhibited mTOR and p-mTOR expression while upregulated SHP1 expression in K562 cells. Knockdown of SHP1 decrease α-/γ-globin mRNA level, CD71 protein expression levels and the proportion of benzidine-positive cells in the K562 cells. Additionally, knockdown of SHP1 can reverse the sirolimus-induced erythroid differentiation of K562 cells. Sirolimus treatment can promote the erythroid differentiation of K562 cells via upregulating of SHP1.
    Keywords:  Erythroid differentiation; K562 cell; SHP1; Sirolimus
    DOI:  https://doi.org/10.1007/s00277-025-06518-4
  4. Annu Rev Genet. 2025 Aug 19.
      Studies of globin gene clusters have established many paradigms of gene regulation. This review focuses on the α- and β-globin gene clusters of humans and mice, summarizing important insights from high-throughput biochemical assays and directed genetic dissections and emphasizing similarities across the types of gene clusters and between species. The overall arrangements and architectures are similar, with each gene cluster being localized within a topologically constrained unit of chromatin containing a multicomponent enhancer (i.e., a locus control region) and other regulatory elements bound by a similar set of transcription factors and coactivators. Differential expression of the globin genes within each cluster during ontogeny is associated with changes in contacts with the locus control region and involves the action of gene-specific repressors. Detailed study of the fetal β-like HBG1 and HBG2 globin genes has revealed a remarkable diversity of regulatory pathways that provide candidates for therapeutic approaches to reactivate these genes for β-hemoglobinopathies.
    DOI:  https://doi.org/10.1146/annurev-genet-020325-095743
  5. Sci Rep. 2025 Aug 18. 15(1): 30192
      Individuals with glucose-6-phosphate dehydrogenase (G6PD) deficiency hold a significant risk of severe hemolytic crises under oxidative stress. Currently, the definitive and curative treatment for the disorder has not been developed. Among over 200 G6PD variants, G6PD Viangchan (c.871 G > A) is the most prevalent and has been extensively studied in Southeast Asia. This study assessed the effectiveness of prime editing for correcting the G6PD Viangchan mutation in an established mutant HEK293T cell line and G6PD-deficient induced pluripotent stem cells (iPSCs). Using optimized modalities, prime editing achieved a high correction efficiency of over 25% in the HEK293T cells. In iPSCs, this gene editing tool yielded satisfactory correction outcomes, with approximately 5% corrected alleles. Our findings indicate that prime editing provides high precision, producing minimal by-products below baseline and showing undetectable off-target effects. Overall, prime editing has the potential to correct the G6PD Viangchan mutation, providing a valuable approach for future therapeutic strategies and the generation of isogenic cell lines to promote extensive studies in drug discovery and the pathogenesis of the G6PD variant.
    Keywords:  CRISPR-Cas9; G6PD; IPSCs; Prime editing; Sequencing; Viangchan mutation
    DOI:  https://doi.org/10.1038/s41598-025-15463-1
  6. Front Med (Lausanne). 2025 ;12 1608870
       Objective: To investigate the difference of black blood (DB) and bright blood (BB) T2* techniques at 1.5 T and 3 T in the assessment of myocardial iron load in patients with thalassemia (TM).
    Methods: As a retrospective study. CMRtools software was used to measure myocardial T2* in 359 patients with moderate (60 g/L < Hemoglobin<90 g/L) or severe (Hemoglobin<60 g/L) thalassemia. A truncation method was used to remove signal values that deviated from the fitted curve. T2* (DBx-T2*, BBx-T2*) containing all (eight echoes) signals (DB8-T2*, BB8-T2*) and the optimal signal (coefficient of determination R 2 > 0.95) were recorded. The difference, correlation and consistency of T2* measured by different methods were compared.
    Results: There was no significant difference (p > 0.05) in myocardial T2* measured by different methods (1.5 T, 3 T), and all of them were highly positively correlated (p < 0.05, rs > 0.9). Bland-Altman analysis showed that (1.5 T) DB8-T2* and DBx-T2*, DBx-T2* and BBx-T2* had good consistency (p > 0.05). (3 T) DB8-T2* and DBx-T2* had good consistency (p > 0.05). There were proportional differences in T2* values measured by the other methods (p < 0.05), and none of them could be considered to have good consistency.
    Conclusion: DB CMR T2* and BB CMR T2* can be interchangeable in the assessment of myocardial iron load in TM patients. However, DB CMR T2* is more stable and reliable.
    Keywords:  bright blood; cardiac magnetic resonance; dark blood; myocardial T2*; thalassemia
    DOI:  https://doi.org/10.3389/fmed.2025.1608870