epoetin alfa (Panpotin / Erisa / PDA10)

Feasibility and safety of pegunigalsidase alfa infusion during hemodialysis: first case report in a Fabry patient with kidney failure.

Riccio Eleonora E, Gambardella Jessica J, Cuomo Vincenzo V, Iaccarino Guido G et al.

Mexican version of the Autism Behavior Checklist: validity and reliability.

Albores-Gallo Lilia L, Varela-Orozco Karen L KL, Roldán-Ceballos Ofelia O, List-Hilton Claudia C et al.

The Autism Behavior Checklist (ABC) evaluates symptoms in individuals with Autism Spectrum Disorders (ASD) within a school setting. The purpose of this study was to evaluate the validity of the ABC Mexican version. Participants were children (n = 133, aged 2-17). All parents were interviewed with the autism diagnostic interview-revised (ADI-R) to confirm an ASD diagnosis and then answered the ABC checklist. The sample for the ABC test-retest analysis consisted of 19 parents with unaffected typically developing children between 2 and 17 years old. Children and adolescents (n = 133) with a mean age of 6.9 years (standard deviation [SD] 3.7), 83.5% were males. The ABC total mean score was 69.3 (SD 25). The Internal consistency through the Cronbach Alfa coefficient was α = 0.83 < p = 0.001 for the 57 ABC items. The 10-day test-retest reliability showed a Pearson correlation coefficient of r = 0.98, p < 0.001. The Spearman correlation coefficients between the ABC subscales and the ADI-R ranged from (rs = 0.494) to (rs = 0.816). Criterion validity with a cutoff of 30 resulted in a sensitivity of 87% and a specificity of 37%. The best Kappa coefficient was 0.285 between the ABC and the ADI-R. The Mexican ABC has good psychometric properties. Further studies should investigate its value in educational settings.

Weekly Metronomic Venetoclax and Decitabine/Cedazuridine in Acute Myeloid Leukemia Patient Declining Blood Transfusion.

Aldapt Mahmood M, Badar Talha T, Saliba Antoine N AN, Foran James J et al.

Acute myeloid leukemia (AML) treatment typically involves intensive chemotherapy associated with prolonged cytopenias and frequent transfusion requirements. Patients who decline blood products, such as Jehovah's Witnesses (JW), present a therapeutic challenge, as transfusion support is integral to standard care. Emerging low-intensity regimens combining venetoclax (VEN) with hypomethylating agents offer a potential alternative with reduced myelosuppression. A 77-year-old male JW with AML with myelodysplastic syndrome-related changes and mutations in DDX41, ASXL1, and PPM1D presented with pancytopenia. Due to refusal of transfusions, he was treated with a metronomic, all-oral regimen of VEN 400 mg weekly and decitabine/cedazuridine (DEC-C) 35/100 mg weekly, supported by erythropoiesis-stimulating agent (darbepoetin alfa) and thrombopoietin agonist (romiplostim) as needed. After 4 weeks, bone marrow blasts decreased from 20-25% to 10%, with clearance of ASXL1 and PPM1D mutations and reduction of DDX41 variant allele frequency (VAF) to 2.9% from 9.7%. At 26 weeks, marrow blasts further decreased to 5%, DDX41 VAF remained low (2.6%), and counts normalized (ANC 1.73 × 103/µL, hemoglobin 13.1 g/dL, PLT 256 × 103/µL) without any transfusions. This case demonstrates that a metronomic, all-oral VEN and DEC-C regimen can achieve hematologic improvement and molecular response in AML while maintaining transfusion independence. It highlights a feasible and safe therapeutic option for patients declining blood products, achieving disease control with minimal toxicity.

Updated oncolytic viruses for the treatment of bladder cancer: a systematic review.

Khatami Fatemeh F, Mohammadi Abdolreza A, Azadmanesh Kayhan K, Deyhimfar Roham R et al.

Bladder cancer (BCa) is the most prevalent malignancy of the urinary tract and is often diagnosed at a late stage. Recurrence remains a major challenge for current treatment strategies. Over the past decade, the antitumor potential of oncolytic viruses (OVs) has been increasingly recognized, with several studies evaluating their safety and efficacy in BCa treatment. In this systematic review, we present clinical trials and animal studies to comprehensively evaluate the safety and therapeutic efficiency of various OVs in BCa. After receiving the PROSPERO registration code, four electronic databases PubMed, Web of Science, Scopus, and Embase, were systematically searched to identify studies evaluating the safety and treatment potential of OVs in BCa. Two reviewers independently conducted the screening process, and data from clinical trials and animal studies were systematically extracted into Excel for subsequent synthesis and reporting. All steps of this systematic review adhered to the guidelines outlined in the Cochrane Handbook for Systematic Reviews of Interventions, upholding high standards of methodological rigor and transparency. The initial search strategy yielded 1,061 records. After removing duplicates, 809 articles were screened for relevance. Following the exclusion of 761 studies, 48 were deemed eligible for data extraction, comprising 17 clinical trials and 29 animal studies. Various oncolytic viruses were investigated, either as monotherapies or in combination with other agents, including Vaccinia virus, Adenovirus, Oncolytic adenovirus ONYX, Coxsackievirus A21 (CVA21), oncolytic CVA21 (V937), attenuated measles virus (MV-NIS virus), recombinant adenovirus (rAd), and a serotype 5 adenovirus engineered to express GM-CSF. All studies reported their most effective doses administered within safe parameters. The common virus was the non-replicative recombinant adenovirus serotype 5 (Ad5), which encodes human interferon alfa-2b (IFNα2b) a cytokine with anti-tumor properties. It is administered via intravesical instillation and has received FDA approval.