المؤلفون: Salas, María Queralt, Eikema, Diderik Jan, Koster, Linda, Maertens, Johan, Passweg, Jakob, Finke, Jürgen, Broers, Annoek E.C., Koc, Yener, Kröger, Nicolaus, Ozkurt, Zubeyde Nur, Pascual-Cascon, María Jesús, Platzbecker, Uwe, Van Gorkom, Gwendolyn, Schroeder, Thomas, López-Lorenzo, José Luis, Martino, Massimo, Chiusolo, Patrizia, Kaufmann, Martin, Onida, Francesco, Gurnari, Carmelo, Scheid, Christof, Drozd-Sokolowska, Joanna, Raj, Kavita, Robin, Marie, McLornan, Donal P.

المصدر: Salas , M Q , Eikema , D J , Koster , L , Maertens , J , Passweg , J , Finke , J , Broers , A E C , Koc , Y , Kröger , N , Ozkurt , Z N , Pascual-Cascon , M J , Platzbecker , U , Van Gorkom , G , Schroeder , T , López-Lorenzo , J L , Martino , M , Chiusolo , P , Kaufmann , M , Onida , F , Gurnari , C , Scheid , C , Drozd-Sokolowska , J , Raj , K ....

الوصف: We retrospectively compared outcomes of 404 MDS patients undergoing 1st matched sibling donor allo-HCT receiving either PTCy-based (n = 66) or other "conventional prophylaxis" (n = 338; mostly calcineurin inhibitor + methotrexate or MMF). Baseline characteristics were balanced, except for higher use of myeloablative regimens in the PTCy group (52.3% vs. 38.2%, p = 0.047). Incidences of neutrophil (Day +28: 89% vs. 97%, p = 0.011) and platelet (Day +100: 89% vs. 97%, p < 0.001) engraftment were lower for PTCy-based. Day +100 cumulative incidences of grade II-IV and III-IV aGVHD, and 5-year CI of extensive cGVHD were 32%, 18% and 18% for PTCy-based and 25% (p = 0.3), 13% (p = 0.4) and 31% (p = 0.09) for the conventional cohort. Five-year OS (51% vs. 52%, p = 0.6) and GRFS (33% vs. 25%, p = 0.6) were similar between groups. Patients receiving PTCy had a trend to a lower cumulative incidence of relapse (20% vs. 33%, p = 0.06), not confirmed on multivariable analysis (p = 0.3). Although higher NRM rates were observed in patients receiving PTCy (32% vs. 21%, p = 0.02) on univariate analysis, this was not confirmed on multivariate analysis (HR 1.46, p = 0.18), and there was no resultant effect on OS (HR 1.20, p = 0.5). Based on these data, PTCy prophylaxis appears to be an attractive option for patients with MDS undergoing MSD allo-HCT.

العلاقة: https://cris.maastrichtuniversity.nl/en/publications/9f4c7e71-aa0c-496c-8de5-5cbf3d695eb6Test

الإتاحة: https://doi.org/10.1038/s41409-023-02159-1Test
https://cris.maastrichtuniversity.nl/en/publications/9f4c7e71-aa0c-496c-8de5-5cbf3d695eb6Test

المؤلفون: Pascual Izquierdo, Cristina, Mingot Castellano, María Eva, Kerguelen Fuentes, Ana E., García-Arroba Peinado, José, Cid, Joan, Moraima Jimenez, Maria, Valcarcel, David, Gómez Seguí, Inés, de la Rubia, Javier, Martin, Paz, Goterris, Rosa, Hernández, Luis, Tallón, Inmaculada, Varea, Sara, Fernández, Marta, García Muñoz, Nadia, Vara, Míriam, Fernández Zarzoso, Miguel, García Candel, Faustino, Paciello, María Liz, García García, Irene, Zalba, Saioa, Campuzano, Verónica, Gala, José María, Vidán Estévez, Julia, Moreno Jiménez, Gemma, López Lorenzo, José Luis, González Arias, Elena, Freiría, Carmen, Solé, María, Ávila Idrovo, Laura Francisca, Hernández Castellet, José Carlos, Cruz, Naylen, Lavilla, Esperanza, Pérez Montaña, Albert, Atucha, Jon Ander, Moreno Beltrán, María Esperanza, Moreno Macías, Juán Ramón, Salinas, Ramón, del Rio Garma, Julio

المصدر: Blood Advances. Vol. 6, nº 24, December 2022, pp. 6219 - 6227

مصطلحات موضوعية: caplacizumab, prednisone, rituximab

الوصف: Immune thrombotic thrombocytopenic purpura (iTTP) is a thrombotic microangiopathy caused by anti-ADAMTS13 antibodies. Caplacizumab is approved for adults with an acute episode of iTTP in conjunction with plasma exchange (PEX) and immunosuppression. The objective of this study was to analyze and compare the safety and efficacy of caplacizumab vs the standard of care and assess the effect of the concomitant use of rituximab. A retrospective study from the Spanish TTP Registry of patients treated with caplacizumab vs those who did not receive it was conducted. A total of 155 patients with iTTP (77 caplacizumab, 78 no caplacizumab) were included. Patients initially treated with caplacizumab had fewer exacerbations (4.5% vs 20.5%; P < .05) and less refractoriness (4.5% vs 14.1%; P < .05) than those who were not treated. Time to clinical response was shorter when caplacizumab was used as initial treatment vs caplacizumab used after refractoriness or exacerbation. The multivariate analysis showed that its use in the first 3 days after PEX was associated with a lower number of PEX (odds ratio, 7.5; CI, 2.3-12.7; P < .05) and days of hospitalization (odds ratio, 11.2; CI, 5.6-16.9; P < .001) compared with standard therapy. There was no difference in time to clinical remission in patients treated with caplacizumab compared with the use of rituximab. No severe adverse event was described in the caplacizumab group. In summary, caplacizumab reduced exacerbations and refractoriness compared with standard of care regimens. When administered within the first 3 days after PEX, it also provided a faster clinical response, reducing hospitalization time and the need for PEX. ; 9 páginas

وصف الملف: application/pdf

العلاقة: http://hdl.handle.net/10498/29789Test

الإتاحة: https://doi.org/10.1182/bloodadvances.2022008028Test
http://hdl.handle.net/10498/29789Test

المؤلفون: Bailén, Rebeca, Alenda, Raquel, Herruzo-Delgado, Beatriz, Acosta-Fleitas, Cynthia, Vallés, Ana, Esquirol, Albert, Fonseca, Marta, Solán, Laura, Sánchez-Vadillo, Irene, Bautista, Guiomar, Bento, Leyre, López-Godino, Oriana, Pérez-Martínez, Ariadna, Torrent Catarineu, Anna, Zanabili, Joud, Calbacho, Maria, Moreno, Miguel Ángel, Pascual-Cascón, María Jesús, Guerra-Domínguez, Luisa, Chinea, Anabelle, Garcia Cadenas, Irene, López-Corral, Lucía, Boix, Francisco, López Lorenzo, José Luis, Humala, Karem, Duarte, Rafael, Sampol, Antonia, Heras, Inmaculada, Vicario, José Luis, Balas, Antonio, Oarbeascoa, Gillen, Fernández-Caldas, Paula, Anguita, Javier, Kwon, Mi, Universitat Autònoma de Barcelona

مصطلحات موضوعية: DSA kinetics, Desensitization strategies, Donor-specific antibodies, Graft failure, Haplo-hsct

الوصف: Donor-specific antibodies (DSAs) are IgG allo-antibodies against mismatched donor HLA molecules and can cause graft failure (GF) in the setting of haploidentical hematopoietic stem cell transplantation (haplo-HSCT). Our aim was to report the experience of the Spanish Group of Hematopoietic Transplant (GETH-TC) in DSA-positive patients who had undergone haplo-HSCT. We conducted a survey of patients who underwent haplo-HSCT in GETH-TC centers between 2012 and 2021. Data were collected on the DSA assay used, monitoring strategy, complement fixation, criteria for desensitization, desensitization strategies and transplant outcomes. Fifteen centers from the GETH-TC responded to the survey. During the study period, 1,454 patients underwent haplo-HSCT. Seventy of the transplants were performed in 69 DSA-positive patients, all of whom lacked a suitable alternative donor; 61 (88%) patients were female (90% with prior pregnancies). All patients received post-transplant cyclophosphamide-based graft-versus-host disease prophylaxis. Regarding baseline DSA intensity, 46 (67%) patients presented mean fluorescence intensity (MFI) >5,000, including 21 (30%) with MFI >10,000 and three (4%) with MFI >20,000. Six patients did not receive desensitization treatment, four of them with MFI <5,000. Of 63 patients receiving desensitization treatment, 48 (76%) were tested after desensitization therapy, and a reduction in intensity was confirmed in 45 (71%). Three patients (5%) experienced an increase in MFI after desensitization, two of whom experienced primary GF. Cumulative incidence of neutrophil engraftment at day 28 was 74% in a median of 18 days (IQR, 15─20); six patients died before engraftment due to toxicity or infection and eight patients had primary GF despite desensitization in seven of them. After a median follow-up of 30 months, two-year overall and event-free survival were 46.5% and 39%, respectively. The two-year cumulative incidence of relapse was 16% and non-relapse mortality (NRM) was 43%. Infection was the ...

وصف الملف: application/pdf

العلاقة: Frontiers in immunology; Vol. 14 (may 2023); https://ddd.uab.cat/record/279352Test; urn:10.3389/fimmu.2023.1165759; urn:oai:ddd.uab.cat:279352; urn:pmcid:PMC10250708; urn:pmc-uid:10250708; urn:pmid:37304258; urn:oai:pubmedcentral.nih.gov:10250708

الإتاحة: https://ddd.uab.cat/record/279352Test

المؤلفون: Sofía, Álvarez-Galeano, Salazar-Hidalgo, Milton, Benavente, Celina, López-Lorenzo, José Luis, Anguita, Eduardo

المصدر: HemaSphere ; volume 7, issue S3, page e36901ed ; ISSN 2572-9241

الإتاحة: https://doi.org/10.1097/01.hs9.0000974116.36901.edTest

المؤلفون: Long‐Boyle, Janel R., Kohn, Donald B., Shah, Ami J., Spencer, Sueli Marques, Sevilla, Julian, Booth, Claire, López Lorenzo, José Luis, Nicoletti, Eileen, Shah, Arpita, Reatz, Meredith, Matos, Joana, Schwartz, Jonathan D.

المصدر: Pediatric Blood & Cancer ; volume 71, issue 1 ; ISSN 1545-5009 1545-5017

الوصف: Background The incidence of secondary malignancies associated with busulfan exposure is considered low, but has been poorly characterized. Because this alkylating agent is increasingly utilized as conditioning prior to gene therapy in nonmalignant hematologic and related disorders, more precise characterization of busulfan's potential contribution to subsequent malignant risk is warranted. Procedure We conducted a literature‐based assessment of busulfan and subsequent late effects, with emphasis on secondary malignancies, identifying publications via PubMed searches, and selecting those reporting at least 3 years of follow‐up. Results We identified eight pediatric and 13 adult publications describing long‐term follow‐up in 570 pediatric and 2076 adult hematopoietic cell transplant (HCT) recipients. Secondary malignancies were reported in 0.5% of pediatric HCT recipients, with no cases of myelodysplastic syndrome (MDS) or acute myelocytic leukemia (AML). Fatal secondary malignancies were reported in 0.8% of 1887 evaluable adult HCT recipients, and an overall incidence of secondary malignancies of 4.8% was reported in a subset of 389 evaluable adult patients. We also reviewed long‐term results from eight publications evaluating lentiviral‐ and human promotor‐based HSC‐targeted gene therapy in 215 patients with nonmalignant conditions, in which busulfan/treosulfan monotherapy or busulfan/fludarabine was the only conditioning. Two malignancies were reported in patients with sickle cell disease (SCD), one of which was potentially busulfan‐related. No additional malignancies were reported in 173 patients with follow‐up of 5–12 years. Conclusion The incidence of busulfan‐related secondary malignancies is low, and likely to be substantially less than 1% in pediatric transplant recipients, especially those receiving busulfan monotherapy for nonmalignant conditions other than SCD.

الإتاحة: https://doi.org/10.1002/pbc.30738Test

المؤلفون: Bernal, Teresa, Moreno, Ainhoa Fernández, de LaIglesia, Almudena, Benavente, Celina, García‐Noblejas, Ana, Belmonte, Daniel García, Riaza, Rosalía, Salamero, Olga, Foncillas, Maria Angeles, Roldán, Alicia, Concepción, Víctor Noriega, González, Laura Llorente, Bergua Burgués, Juan Miguel, Lorente de Uña, Soraya, Rodríguez‐Macías, Gabriela, de la Fuente Burguera, Adolfo, García Pérez, Maria José, López‐Lorenzo, Jose Luis, Martínez, Pilar, Aláez, Concepción, Callejas, Marta, Martínez‐Chamorro, Carmen, Roca, José Rifón, Barciela, Lourdes Amador, Mena Durán, Armando V., Gómez Correcha, Karoll, Lavilla Rubira, Esperanza, Amigo, María Luz, Vall‐llovera, Ferran, Garrido, Ana, García‐Fortes, María, de Miguel Llorente, Dunia, Leonardo, Anastasia Aules, Cervero, Carlos, Jordá, Rosa Coll, Pérez‐Encinas, Manuel M., Zarzuela, Marta Polo, Figuera, Angela, Rad, Guillermo, Martínez‐Cuadrón, David, Montesinos, Pau

المساهمون: Jazz Pharmaceuticals

المصدر: Cancer Medicine ; volume 12, issue 14, page 14892-14901 ; ISSN 2045-7634 2045-7634

الوصف: Background CPX‐351 is approved for the treatment of therapy related acute myeloid leukemia (t‐AML) and AML with myelodysplastic related changes (MRC‐AML). The benefits of this treatment over standard chemotherapy has not been addressed in well matched cohorts of real‐life patients. Methods Retrospective analysis of AML patients treated with CPX‐351 as per routine practice. A propensity score matching (PSM) was used to compare their main outcomes with those observed in a matched cohort among 765 historical patients receiving intensive chemotherapy (IC), all of them reported to the PETHEMA epidemiologic registry. Results Median age of 79 patients treated with CPX‐351 was 67 years old (interquartile range 62–71), 53 were MRC‐AML. The complete remission (CR) rate or CR without recovery (CRi) after 1 or 2 cycles of CPX‐351 was 52%, 60‐days mortality 18%, measurable residual disease <0.1% in 54% (12 out of 22) of them. Stem cell transplant (SCT) was performed in 27 patients (34%), median OS was 10.3 months, and 3‐year relapse incidence was 50%. Using PSM, we obtained two comparable cohorts treated with CPX‐351 ( n = 52) or IC ( n = 99), without significant differences in CR/CRi (60% vs. 54%) and median OS (10.3 months vs. 9.1 months), although more patients were bridged to SCT in the CPX‐351 group (35% vs. 12%). The results were confirmed when only 3 + 7 patients were included in the historical cohort. In multivariable analyses, SCT was associated with better OS (HR 0.33 95% CI: 0.18–0.59), p < 0.001. Conclusion Larger post‐authorization studies may provide evidence of the clinical benefits of CPX‐351 for AML in the real‐life setting.

الإتاحة: https://doi.org/10.1002/cam4.6120Test

المؤلفون: Suárez, Edwin Uriel, Piris, Miguel Ángel, Rodríguez-Pinilla, Socorro María, García, Juan F, López-Lorenzo, José Luis, Cornago-Navascués, Javier, Salgado-Sánchez, Rocío, Castaño-Bonilla, Tamara, Mata-Serna, Raquel, Alonso-Domínguez, Juan M, Llamas, Pilar

المصدر: Ann Hematol ; ISSN:1432-0584 ; Volume:103 ; Issue:5

العلاقة: https://doi.org/10.1007/s00277-024-05647-6Test; https://pubmed.ncbi.nlm.nih.gov/38308706Test

الإتاحة: https://doi.org/10.1007/s00277-024-05647-6Test
https://pubmed.ncbi.nlm.nih.gov/38308706Test

المؤلفون: Lázaro-García, Alberto, Láinez-González, Daniel, González-Rodríguez, Marta, Cano Alsua, Santiago, Suárez M, Edwin Uriel, Solán-Blanco, Laura, Cornago-Navascués, Javier, López-Lorenzo, José Luis, Llamas-Sillero, Pilar, Alonso-Domínguez, Juan Manuel

المصدر: Journal of Pain and Symptom Management ; ISSN 0885-3924

مصطلحات موضوعية: Anesthesiology and Pain Medicine, Neurology (clinical), General Nursing

الإتاحة: https://doi.org/10.1016/j.jpainsymman.2024.02.567Test
https://api.elsevier.com/content/article/PII:S0885392424006493?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S0885392424006493?httpAccept=text/plainTest

المؤلفون: Pascual Izquierdo, Cristina, Mingot Castellano, María Eva, Kerguelen Fuentes, Ana E., García Arroba Peinado, José, Cid Vidal, Joan, Jimenez, Maria Moraima, Valcarcel, David, Gómez Seguí, Inés, Rubia, Javier de la, Martin, Paz, Goterris, Rosa, Hernández, Luis, Tallón, Inmaculada, Varea, Sara, Fernández, Marta, García Muñoz, Nadia, Vara, Míriam, Fernández Zarzoso, Miguel, García Candel, Faustino, Paciello, María Liz, García García, Irene, Zalba, Saioa, Campuzano, Verónica, Gala, José María, Vidán Estévez, Julia, Moreno Jiménez, Gemma, López Lorenzo, José Luis, González Arias, Elena, Freiría, Carmen, Solé, María, Ávila Idrovo, Laura Francisca, Hernández Castellet, José Carlos, Cruz, Naylen, Lavilla, Esperanza, Pérez Montaña, Albert, Atucha, Jon Ander, Moreno Beltrán, María Esperanza, Moreno Macías, Juán Ramón, Salinas, Ramón, Rio Garma, Julio del, Spanish Apheresis Group (GEA), Spanish Thrombotic Thrombocytopenic Purpura Registry (REPTT)

المصدر: Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL))

مصطلحات موضوعية: Rituximab, Trombosi, Assaigs clínics, Thrombosis, Clinical trials

الوصف: Immune thrombotic thrombocytopenic purpura (iTTP) is a thrombotic microangiopathy caused by anti-ADAMTS13 antibodies. Caplacizumab is approved for adults with an acute episode of iTTP in conjunction with plasma exchange (PEX) and immunosuppression. The objective of this study was to analyze and compare the safety and efficacy of caplacizumab vs the standard of care and assess the effect of the concomitant use of rituximab. A retrospective study from the Spanish TTP Registry of patients treated with caplacizumab vs those who did not receive it was conducted. A total of 155 patients with iTTP (77 caplacizumab, 78 no caplacizumab) were included. Patients initially treated with caplacizumab had fewer exacerbations (4.5% vs 20.5%; P < .05) and less refractoriness (4.5% vs 14.1%; P < .05) than those who were not treated. Time to clinical response was shorter when caplacizumab was used as initial treatment vs caplacizumab used after refractoriness or exacerbation. The multivariate analysis showed that its use in the first 3 days after PEX was associated with a lower number of PEX (odds ratio, 7.5; CI, 2.3-12.7; P < .05) and days of hospitalization (odds ratio, 11.2; CI, 5.6-16.9; P < .001) compared with standard therapy. There was no difference in time to clinical remission in patients treated with caplacizumab compared with the use of rituximab. No severe adverse event was described in the caplacizumab group. In summary, caplacizumab reduced exacerbations and refractoriness compared with standard of care regimens. When administered within the first 3 days after PEX, it also provided a faster clinical response, reducing hospitalization time and the need for PEX.

وصف الملف: 9 p.; application/pdf

العلاقة: Reproducció del document publicat a: https://doi.org/10.1182/bloodadvances.2022008028Test; Blood Advances, 2022, vol. 6, num. 24, p. 6219-6227; https://doi.org/10.1182/bloodadvances.2022008028Test; http://hdl.handle.net/2445/196688Test

الإتاحة: https://doi.org/10.1182/bloodadvances.2022008028Test
http://hdl.handle.net/2445/196688Test

المؤلفون: Schwartz, Jonathan D., Barcellini, Wilma, Grace, Rachel F., Bianchi, Paola, Zanella, Alberto, López Lorenzo, José Luis, Sevilla, Julián, Shah, Ami J., Glader, Bertil, Nicoletti, Eileen, Navarro Ordoñez, Susana, Segovia, José Carlos

المصدر: American Journal of Hematology ; volume 97, issue 3 ; ISSN 0361-8609 1096-8652

الإتاحة: https://doi.org/10.1002/ajh.26458Test