المؤلفون: de la Rubia, Javier^1,2 (AUTHOR) delarubia_jav@gva.es, Alonso, Rafael³ (AUTHOR), Clavero, María Esther⁴ (AUTHOR), Askari, Elham⁵ (AUTHOR), García, Alfonso⁶ (AUTHOR), Antón, Cristina⁷ (AUTHOR), Fernández, Margarita⁸ (AUTHOR), Escalante, Fernando⁹ (AUTHOR), García, Ana¹⁰ (AUTHOR), Rios-Tamayo, Rafael¹¹ (AUTHOR), Conesa, Venancio¹² (AUTHOR), Bermúdez, María Arancha¹³ (AUTHOR), Merchán, Beatriz¹⁴ (AUTHOR), Velasco, Alberto E.¹⁵ (AUTHOR), Blanchard, María Jesús¹⁶ (AUTHOR), Sampol, Antonia¹⁷ (AUTHOR), Gainza, Eukene¹⁸ (AUTHOR), Hernández, Prisma Montserrat¹⁹ (AUTHOR), Alegre, Adrián²⁰ (AUTHOR)

المصدر: Cancers. Jun2023, Vol. 15 Issue 11, p2964. 13p.

مصطلحات موضوعية: *THERAPEUTIC use of immunoglobulins, *RESEARCH, *SCIENTIFIC observation, *CONFIDENCE intervals, *IMMUNOGLOBULINS, *OPTIC nerve diseases, *CANCER relapse, *INVESTIGATIONAL drugs, *CELL receptors, *RETROSPECTIVE studies, *ACQUISITION of data, *TREATMENT effectiveness, *COMPARATIVE studies, *MEDICAL records, *DESCRIPTIVE statistics, *MULTIPLE myeloma, *PROGRESSION-free survival, *PATIENT safety, *OVERALL survival, *DISEASE risk factors

مصطلحات جغرافية: SPAIN

مستخلص: Simple Summary: Patients with multiple myeloma (MM) who become refractory to three or more lines of therapy (RRMM patients) have few valid therapeutic alternatives. Among them, drugs directed against the BCMA antigen expressed in plasma cells are very appealing. Belantamab-mafodotin (belamaf) is the first antibody-drug conjugate against BCMA ready for clinical use. In this paper, we report the Spanish experience of belamaf monotherapy in 156 patients with RRMM. The overall response rate was 41.8%, with 39.8% of patients achieving a partial response or better. Median progression-free survival was 3.61 months, but interestingly, it increased to 14.47 months in patients achieving a minimal response or better. Treatment was well tolerated, ocular events being the most reported toxicity (87.9%; grade ≥ 3, 33.7%), but only two patients discontinued treatment due to side effects. Overall, our results confirm the safety and efficacy of belamaf in this poor prognosis subset of patients. Belantamab-mafodotin (belamaf) is a novel antibody-drug conjugate targeting B-cell maturation antigen that showed anti-myeloma activity in patients with relapsed and refractory multiple myeloma (RRMM). We performed an observational, retrospective, and multicenter study aimed to assess the efficacy and safety of single-agent belamaf in 156 Spanish patients with RRMM. The median number of prior therapy lines was 5 (range, 1–10), and 88% of patients were triple-class refractory. Median follow-up was 10.9 months (range, 1–28.6). The overall response rate was 41.8% (≥CR 13.5%, VGPR 9%, PR 17.3%, MR 2%). The median progression-free survival was 3.61 months (95% CI, 2.1–5.1) and 14.47 months (95% CI, 7.91–21.04) in patients achieving at least MR (p < 0.001). Median overall survival in the entire cohort and in patients with MR or better was 11.05 months (95% CI, 8.7–13.3) and 23.35 (NA-NA) months, respectively (p < 0.001). Corneal events (87.9%; grade ≥ 3, 33.7%) were the most commonly adverse events, while thrombocytopenia and infections occurred in 15.4% and 15% of patients, respectively. Two (1.3%) patients discontinued treatment permanently due to ocular toxicity. Belamaf showed a noticeably anti-myeloma activity in this real-life series of patients, particularly among those achieving MR or better. The safety profile was manageable and consistent with prior studies. [ABSTRACT FROM AUTHOR]

المؤلفون: Rosa-Rosa, Juan Manuel, Cuenca, Isabel, Medina, Alejandro, Vázquez, Iria, Sánchez-delaCruz, Andrea, Buenache, Natalia, Sánchez, Ricardo, Jiménez, Cristina, Rosiñol, Laura, Gutiérrez, Norma C., Ruiz-Heredia, Yanira, Barrio, Santiago, Oriol, Albert, Martin-Ramos, Maria-Luisa, Blanchard, María-Jesús, Ayala, Rosa, Ríos-Tamayo, Rafael, Sureda, Anna, Hernández, Miguel-Teodoro, de la Rubia, Javier

المصدر: Cancers; Oct2022, Vol. 14 Issue 20, p5169-N.PAG, 13p

مصطلحات موضوعية: DRUG efficacy, SEQUENCE analysis, GENETIC mutation, PREDICTIVE tests, SINGLE nucleotide polymorphisms, KARYOTYPES, CANCER patients, CHROMOSOME abnormalities, FLUORESCENCE in situ hybridization, GENOMICS, DESCRIPTIVE statistics, MULTIPLE myeloma, TUMOR markers, PROGRESSION-free survival

مستخلص: Simple Summary: Multiple Myeloma (MM) is considered an incurable chronic disease, which prognosis depends on the presence of different genomic alterations. To accomplish a complete molecular diagnosis in a single essay, we have designed and validated a capture-based NGS approach to reliably identify pathogenic mutations (SNVs and indels), genomic alterations (CNVs and chromosomic translocations), and IGH rearrangements. We have observed a good correlation of the results obtained using our capture panel with data obtained by both FISH and WES techniques. In this study, the molecular classification performed using our approach was significantly associated with the stratification and outcome of MM patients. Additionally, this panel has been proven to detect specific IGH rearrangements that could be used as biomarkers in patient follow-ups through minimal residual disease (MRD) assays. In conclusion, we think that MM patients could benefit from the use of this capture-based NGS approach with a more accurate, single-essay molecular diagnosis. Next-generation sequencing (NGS) has greatly improved our ability to detect the genomic aberrations occurring in multiple myeloma (MM); however, its transfer to routine clinical labs and its validation in clinical trials remains to be established. We designed a capture-based NGS targeted panel to identify, in a single assay, known genetic alterations for the prognostic stratification of MM. The NGS panel was designed for the simultaneous study of single nucleotide and copy number variations, insertions and deletions, chromosomal translocations and V(D)J rearrangements. The panel was validated using a cohort of 149 MM patients enrolled in the GEM2012MENOS65 clinical trial. The results showed great global accuracy, with positive and negative predictive values close to 90% when compared with available data from fluorescence in situ hybridization and whole-exome sequencing. While the treatments used in the clinical trial showed high efficacy, patients defined as high-risk by the panel had shorter progression-free survival (p = 0.0015). As expected, the mutational status of TP53 was significant in predicting patient outcomes (p = 0.021). The NGS panel also efficiently detected clonal IGH rearrangements in 81% of patients. In conclusion, molecular karyotyping using a targeted NGS panel can identify relevant prognostic chromosomal abnormalities and translocations for the clinical management of MM patients. [ABSTRACT FROM AUTHOR]

: Copyright of Cancers is the property of MDPI and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)

المؤلفون: Solana-Altabella, Antonio, Megías-Vericat, Juan Eduardo, Ballesta-López, Octavio, Boluda, Blanca, Cano, Isabel, Acuña-Cruz, Evelyn, Rodríguez-Veiga, Rebeca, Torres-Miñana, Laura, Sargas, Claudia, Sanz, Miguel Á., Borrell-García, Carmela, López-Briz, Eduardo, Poveda-Andrés, José Luis, De la Rubia, Javier, Montesinos, Pau, Martínez-Cuadrón, David

المصدر: Cancers; Apr2022, Vol. 14 Issue 8, p1921, 10p

مصطلحات موضوعية: CANCER chemotherapy, RETROSPECTIVE studies, ACQUISITION of data, MEDICAL care costs, MEDICAL care use, CANCER patients, HEALTH insurance reimbursement, MEDICAL records, HOSPITAL care, DESCRIPTIVE statistics, HEMATOPOIETIC stem cell transplantation

مصطلحات جغرافية: SPAIN

مستخلص: Simple Summary: Studies addressing the economic costs and burden of secondary acute myeloid leukemia (sAML) are scarce in the literature. We analyzed this topic in a real-life population of sAML patients between 60–75 years receiving intensive chemotherapy induction. In elderly patients with sAML and intensive regimens, it entails an increase in costs and a longer hospital stay. In these specific patients, almost a third of the time is spent hospitalized after the diagnosis of sAML. There are no studies with this type of population and diagnosis, which gives added value to the results obtained. Pharmacoeconomic studies in patients with AML are being carried out due to the need to evaluate the cost-effectiveness of new oral drugs, therapeutic schemes with higher costs than previous treatments. Background: Information regarding the impact on healthcare systems of secondary acute myeloid leukemia (sAML) is scarce. Methods: A retrospective review of medical charts identified patients aged 60–75 years with sAML between 2010 and 2019. Patient information was collected from diagnosis to death or last follow-up. Outpatient resource use, reimbursement, frequency and duration of hospitalization, and transfusion burden were assessed. Forty-six patients with a median age of 64 years were included. Anthracycline plus cytarabine regimens were the most common induction treatment (39 patients, 85%). The ratio of the total days hospitalized between the total follow-up was 29%, with a sum of 204 hospitalizations (average four/patient; average duration 21 days). The total average reimbursement was EUR 90,008 per patient, with the majority (EUR 77,827) related to hospital admissions (EUR 17,403/hospitalization). Most hospitalizations (163, mean 22 days) occurred in the period before the first allogeneic hematopoietic stem cell transplant (alloHSCT), costing EUR 59,698 per patient and EUR 15,857 per hospitalization. The period after alloHSCT (in only 10 patients) had 41 hospitalizations (mean 21 days), and a mean reimbursement cost of EUR 99,542 per patient and EUR 24,278 per hospitalization. In conclusion, there is a high consumption of economic and healthcare resources in elderly patients with sAML receiving active treatments in Spain. [ABSTRACT FROM AUTHOR]

: Copyright of Cancers is the property of MDPI and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)