130 results on '"Gaziev, J."'
Search Results
2. Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000–2010
- Author
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Baronciani, D, Angelucci, E, Potschger, U, Gaziev, J, Yesilipek, A, Zecca, M, Orofino, M G, Giardini, C, Al-Ahmari, A, Marktel, S, de la Fuente, J, Ghavamzadeh, A, Hussein, A A, Targhetta, C, Pilo, F, Locatelli, F, Dini, G, Bader, P, and Peters, C
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- 2016
- Full Text
- View/download PDF
3. Hematopoietic SCT for the Black African and non-Black African variants of sickle cell anemia
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Lucarelli, G, Isgrò, A, Sodani, P, Marziali, M, Gaziev, J, Paciaroni, K, Gallucci, C, Cardarelli, L, Ribersani, M, Alfieri, C, De Angelis, G, Armiento, D, Andreani, M, Testi, M, Amato, A, Akinyanju, O O, and Wakama, T T
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- 2014
- Full Text
- View/download PDF
4. Pancreatic beta-Cell Function Before and After Bone Marrow Transplantation for Thalassemia
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Galimberti, M., De Sanctis, V., Lucarelli, G., Polchi, P., Angelucci, E., Baronciani, D., Giardini, C., Erer, B., Gaziev, J., Balducci, R., Vullo, C., Andò, Sebastiano, editor, and Brancati, Carlo, editor
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- 1995
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- View/download PDF
5. COVID-19 infection in adult patients with hematological malignancies: a European Hematology Association Survey (EPICOVIDEHA)
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Pagano, L., Salmanton-Garcia, J., Marchesi, F., Busca, A., Corradini, P., Hoenigl, M., Klimko, N., Koehler, P., Pagliuca, A., Passamonti, F., Verga, L., Visek, B., Ilhan, O., Nadali, G., Weinbergerova, B., Cordoba-Mascunano, R., Marchetti, M., Collins, G. P., Farina, F., Cattaneo, C., Cabirta, A., Gomes-Silva, M., Itri, F., van Doesum, J., Ledoux, M. -P., Cernan, M., Jaksic, O., Duarte, R. F., Magliano, G., Omrani, A. S., Fracchiolla, N. S., Kulasekararaj, A., Valkovic, T., Poulsen, C. B., Machado, M., Glenthoj, A., Stoma, I., Racil, Z., Piukovics, K., Navratil, M., Emarah, Z., Sili, U., Maertens, J., Blennow, O., Bergantim, R., Garcia-Vidal, C., Prezioso, L., Guidetti, A., del Principe, M. I., Popova, M., de Jonge, N., Ormazabal-Velez, I., Fernandez, N., Falces-Romero, I., Cuccaro, A., Meers, S., Buquicchio, C., Antic, D., Al-Khabori, M., Garcia-Sanz, R., Biernat, M. M., Tisi, M. C., Sal, E., Rahimli, L., Colovic, N., Schonlein, M., Calbacho, M., Tascini, C., Miranda-Castillo, C., Khanna, N., Mendez, G. -A., Petzer, V., Novak, J., Besson, C., Dulery, R., Lamure, S., Nucci, M., Zambrotta, G., Zak, P., Seval, G. C., Bonuomo, V., Mayer, J., Lopez-Garcia, A., Sacchi, M. V., Booth, S., Ciceri, F., Oberti, M., Salvini, M., Izuzquiza, M., Nunes-Rodrigues, R., Ammatuna, E., Obr, A., Herbrecht, R., Nunez-Martin-Buitrago, L., Mancini, V., Shwaylia, H., Sciume, M., Essame, J., Nygaard, M., Batinic, J., Gonzaga, Y., Regalado-Artamendi, I., Karlsson, L. K., Shapetska, M., Hanakova, M., El-Ashwah, S., Borbenyi, Z., Colak, G. M., Nordlander, A., Dragonetti, G., Maraglino, A. M. E., Rinaldi, A., De Ramon-Sanchez, C., Cornely, O. A., Finizio, O., Fazzi, R., Sapienza, G., Chauchet, A., Van Praet, J., Prattes, J., Dargenio, M., Rossi, C., Shirinova, A., Malak, S., Tafuri, A., Ommen, H. -B., Bologna, S., Khedr, R. A., Choquet, S., Joly, B., Ceesay, M. M., Philippe, L., Kho, C. S., Desole, M., Tsirigotis, P., Otasevic, V., Borducchi, D. M. M., Antoniadou, A., Gaziev, J., Almaslamani, M. A., Garcia-Pouton, N., Paterno, G., Torres-Lopez, A., Tarantini, G., Mellinghoff, S., Grafe, S., Borschel, N., Passweg, J., Merelli, M., Barac, A., Wolf, D., Shaikh, M. U., Thieblemont, C., Bernard, S., Funke, V. A. M., Daguindau, E., Khostelidi, S., Nucci, F. M., Martin-Gonzalez, J. -A., Landau, M., Soussain, C., Laureana, C., Lacombe, K., Kohn, M., Aliyeva, G., Piedimonte, M., Fouquet, G., Rego, M., Hoell-Neugebauer, B., Cartron, G., Pinto, F., Alburquerque, A. M., Passos, J., Yilmaz, A. F., Redondo-Izal, A. -M., Altuntas, F., Heath, C., Kolditz, M., Schalk, E., Guolo, F., Karthaus, M., Della Pepa, R., Vinh, D., Noel, N., Deau Fischer, B., Drenou, B., Mitra, M. E., Meletiadis, J., Bilgin, Y. M., Jindra, P., Espigado, I., Drgona, L., Serris, A., Di Blasi, R., Ali, N., Pagano L. (ORCID:0000-0001-8287-928X), Dragonetti G., Pagano, L., Salmanton-Garcia, J., Marchesi, F., Busca, A., Corradini, P., Hoenigl, M., Klimko, N., Koehler, P., Pagliuca, A., Passamonti, F., Verga, L., Visek, B., Ilhan, O., Nadali, G., Weinbergerova, B., Cordoba-Mascunano, R., Marchetti, M., Collins, G. P., Farina, F., Cattaneo, C., Cabirta, A., Gomes-Silva, M., Itri, F., van Doesum, J., Ledoux, M. -P., Cernan, M., Jaksic, O., Duarte, R. F., Magliano, G., Omrani, A. S., Fracchiolla, N. S., Kulasekararaj, A., Valkovic, T., Poulsen, C. B., Machado, M., Glenthoj, A., Stoma, I., Racil, Z., Piukovics, K., Navratil, M., Emarah, Z., Sili, U., Maertens, J., Blennow, O., Bergantim, R., Garcia-Vidal, C., Prezioso, L., Guidetti, A., del Principe, M. I., Popova, M., de Jonge, N., Ormazabal-Velez, I., Fernandez, N., Falces-Romero, I., Cuccaro, A., Meers, S., Buquicchio, C., Antic, D., Al-Khabori, M., Garcia-Sanz, R., Biernat, M. M., Tisi, M. C., Sal, E., Rahimli, L., Colovic, N., Schonlein, M., Calbacho, M., Tascini, C., Miranda-Castillo, C., Khanna, N., Mendez, G. -A., Petzer, V., Novak, J., Besson, C., Dulery, R., Lamure, S., Nucci, M., Zambrotta, G., Zak, P., Seval, G. C., Bonuomo, V., Mayer, J., Lopez-Garcia, A., Sacchi, M. V., Booth, S., Ciceri, F., Oberti, M., Salvini, M., Izuzquiza, M., Nunes-Rodrigues, R., Ammatuna, E., Obr, A., Herbrecht, R., Nunez-Martin-Buitrago, L., Mancini, V., Shwaylia, H., Sciume, M., Essame, J., Nygaard, M., Batinic, J., Gonzaga, Y., Regalado-Artamendi, I., Karlsson, L. K., Shapetska, M., Hanakova, M., El-Ashwah, S., Borbenyi, Z., Colak, G. M., Nordlander, A., Dragonetti, G., Maraglino, A. M. E., Rinaldi, A., De Ramon-Sanchez, C., Cornely, O. A., Finizio, O., Fazzi, R., Sapienza, G., Chauchet, A., Van Praet, J., Prattes, J., Dargenio, M., Rossi, C., Shirinova, A., Malak, S., Tafuri, A., Ommen, H. -B., Bologna, S., Khedr, R. A., Choquet, S., Joly, B., Ceesay, M. M., Philippe, L., Kho, C. S., Desole, M., Tsirigotis, P., Otasevic, V., Borducchi, D. M. M., Antoniadou, A., Gaziev, J., Almaslamani, M. A., Garcia-Pouton, N., Paterno, G., Torres-Lopez, A., Tarantini, G., Mellinghoff, S., Grafe, S., Borschel, N., Passweg, J., Merelli, M., Barac, A., Wolf, D., Shaikh, M. U., Thieblemont, C., Bernard, S., Funke, V. A. M., Daguindau, E., Khostelidi, S., Nucci, F. M., Martin-Gonzalez, J. -A., Landau, M., Soussain, C., Laureana, C., Lacombe, K., Kohn, M., Aliyeva, G., Piedimonte, M., Fouquet, G., Rego, M., Hoell-Neugebauer, B., Cartron, G., Pinto, F., Alburquerque, A. M., Passos, J., Yilmaz, A. F., Redondo-Izal, A. -M., Altuntas, F., Heath, C., Kolditz, M., Schalk, E., Guolo, F., Karthaus, M., Della Pepa, R., Vinh, D., Noel, N., Deau Fischer, B., Drenou, B., Mitra, M. E., Meletiadis, J., Bilgin, Y. M., Jindra, P., Espigado, I., Drgona, L., Serris, A., Di Blasi, R., Ali, N., Pagano L. (ORCID:0000-0001-8287-928X), and Dragonetti G.
- Abstract
Background: Patients with hematological malignancies (HM) are at high risk of mortality from SARS-CoV-2 disease 2019 (COVID-19). A better understanding of risk factors for adverse outcomes may improve clinical management in these patients. We therefore studied baseline characteristics of HM patients developing COVID-19 and analyzed predictors of mortality. Methods: The survey was supported by the Scientific Working Group Infection in Hematology of the European Hematology Association (EHA). Eligible for the analysis were adult patients with HM and laboratory-confirmed COVID-19 observed between March and December 2020. Results: The study sample includes 3801 cases, represented by lymphoproliferative (mainly non-Hodgkin lymphoma n = 1084, myeloma n = 684 and chronic lymphoid leukemia n = 474) and myeloproliferative malignancies (mainly acute myeloid leukemia n = 497 and myelodysplastic syndromes n = 279). Severe/critical COVID-19 was observed in 63.8% of patients (n = 2425). Overall, 2778 (73.1%) of the patients were hospitalized, 689 (18.1%) of whom were admitted to intensive care units (ICUs). Overall, 1185 patients (31.2%) died. The primary cause of death was COVID-19 in 688 patients (58.1%), HM in 173 patients (14.6%), and a combination of both COVID-19 and progressing HM in 155 patients (13.1%). Highest mortality was observed in acute myeloid leukemia (199/497, 40%) and myelodysplastic syndromes (118/279, 42.3%). The mortality rate significantly decreased between the first COVID-19 wave (March–May 2020) and the second wave (October–December 2020) (581/1427, 40.7% vs. 439/1773, 24.8%, p value < 0.0001). In the multivariable analysis, age, active malignancy, chronic cardiac disease, liver disease, renal impairment, smoking history, and ICU stay correlated with mortality. Acute myeloid leukemia was a higher mortality risk than lymphoproliferative diseases. Conclusions: This survey confirms that COVID-19 patients with HM are at high risk of lethal complications. Ho
- Published
- 2021
6. Allogeneic cellular gene therapy in hemoglobinopathies—evaluation of hematopoietic SCT in sickle cell anemia
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Lucarelli, G, Gaziev, J, Isgrò, A, Sodani, P, Paciaroni, K, Alfieri, C, De Angelis, G, Marziali, M, Simone, M D, Gallucci, C, Roveda, A, Saltarelli, F, Torelli, F, and Andreani, M
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- 2012
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7. Higher CD3+ and CD34+ cell doses in the graft increase the incidence of acute GVHD in children receiving BMT for thalassemia
- Author
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Gaziev, J, Isgrò, A, Marziali, M, Daniele, N, Gallucci, C, Sodani, P, Simone, M D, Adorno, G, Paciaroni, K, Andreani, M, Lanti, A, Del Proposto, G, Testi, M, De Angelis, G, Roveda, A, Alfieri, C, Saltarelli, F, and Lucarelli, G
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- 2012
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8. Influence of the HLA characteristics of Italian patients on donor search outcome in unrelated hematopoietic stem cell transplantation
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Testi, M., Andreani, M., Locatelli, F., Arcese, W., Troiano, M., Battarra, M., Gaziev, J., and Lucarelli, G.
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- 2014
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9. Second hematopoietic SCT in patients with thalassemia recurrence following rejection of the first graft
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Gaziev, J, Sodani, P, Lucarelli, G, Polchi, P, Marktel, S, Paciaroni, K, Marziali, M, Isgrò, A, Simone, M D, Roveda, A, Montuoro, A, Lanti, A, Alfieri, C, De Angelis, G, Gallucci, C, Ciceri, F, and Roncarolo, M G
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- 2008
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10. Mobilization of PBSCs in heterozygous-for-β thalassemia donor by addition of plerixafor after failure of mobilization with G-CSF alone for (TcR) αβ T lymphocytes depletion in haploidentical transplant in thalassemia patients: R1470
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Sodani, P, Lanti, A., De Simone, M. L., Fiorelli, E., Paciaroni, K, De angelis, G, Alfieri, C, Roveda, A., Isgrò, A., Gallucci, C, Ribersani, M., Cardarelli, L., Marziali, M, Torelli, F., Adorno, G., Chiru, O.m., Gaziev, J, and Lucarelli, G
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- 2013
11. Genotypes and haplotypes in the 3′ untranslated region of the HLA-G gene and their association with clinical outcome of hematopoietic stem cell transplantation for beta-thalassemia
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Sizzano, F., Testi, M., Zito, L., Crocchiolo, R., Troiano, M., Mazzi, B., Turchiano, G., Torchio, M., Pultrone, C., Gregori, S., Chiesa, R., Gaziev, J., Sodani, P., Marktel, S., Amoroso, A., Roncarolo, M. G., Lucarelli, G., Ciceri, F., Andreani, M., and Fleischhauer, K.
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- 2012
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12. Purified T-depleted, CD34+ peripheral blood and bone marrow cell transplantation from haplo-identical mother to child with thalassaemia: P614
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Sodani, P., Gaziev, J., Isgrò, A., Paciaroni, K., Alfieri, C., Marziali, M., Simone, M. d., Roveda, A., De Angelis, G., Gallucci, C., Torelli, F., Isacchi, G., Zinno, F., Landi, F., Adorno, G., Lanti, A., and Lucarelli, G.
- Published
- 2011
13. Intravenous busulfan in young children with thalassaemia undergoing haplo-identical haematopoietic stem cell transplantation from mother: P613
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Sodani, P., Gaziev, J., Isgrò, A., Paciaroni, K., Alfieri, C., Marziali, M., Simone, M. D., Roveda, A., De angelis, G., Gallucci, C., Isacchi, G., Zinno, F., Adorno, G., Lanti, A., and Lucarelli, G.
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- 2011
14. Novel disease-specific behaviour of intravenous busulfan in children with thalassaemia undergoing haematopoietic stem cell transplantation requires a different approach to therapeutic drug monitoring: O395
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Gaziev, J., Nguyen, L., Puozzo, C., Marziali, M., Isgro, A., Sodani, P., Mozzi, A. F., Gravina, P., Simone, M. D., Andreani, M., Formosa, A., Paciaroni, K., De Angelis, G., Alfieri, C., Roveda, A., Testi, M., Bernardini, S., Federici, G., and Lucarelli, G.
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- 2011
15. Distribution of HLA alleles and haplotypes in the Maldivian population
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Testi, M., Lai, S., Orrù, S., Alba, F., Cappai, L., Firdous, N., Gaziev, J., Troiano, M., Andreani, M., and Carcassi, C.
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- 2011
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16. Hematopoietic stem cell transplantation in thalassemia
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Gaziev, J, Sodani, P, and Lucarelli, G
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- 2008
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17. Infections by carbapenem-resistant Klebsiella pneumoniae in SCT recipients: a nationwide retrospective survey from Italy
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Girmenia, C, Rossolini, Gm, Piciocchi, A, Bertaina, A, Pisapia, G, Pastore, D, Sica, S, Severino, A, Cudillo, L, Ciceri, F, Scimè, R, Lombardini, L, Viscoli, C, Rambaldi, A, the Gruppo Italiano Trapianto Midollo Osseo (GITMO), Frigeni, M, Corti, C, Mometto, G, Annaloro, C, Casari, E, Castagna, L, Rossi, G, Cattaneo, C, Russo, D, Cancelli, V, Alessandrino, Ep, Ripamonti, F, Pavan, F, Rovelli, A, Pecoraro, C, Busca, A, Carraro, F, Fagioli, F, Gallo, S, Caravelli, D, De Gobbi, M, Saglio, G, Castellino, C, Mordini, N, Gaidano, G, Nassi, L, Raimondi, R, Vespignani, M, Scattolin, Am, Panizzolo, Is, Cesaro, S, Candoni, A, Patriarca, F, Bacigalupo, A, Raiola, A, Castagnola, E, Lanino, E, Stanzani, M, Bandini, G, Massaccesi, E, Prete, A, Bassi, S, Vallisa, D, Caramatti, C, Aversa, F, Zuffa, E, Guidi, S, Bosi, A, Tintori, V, Iori, Ap, Capria, S, Arcese, W, Dentamaro, T, Fabritiis, Pd, Anaclerico, B, Chierichini, A, Piedimonte, M, Ferrari, A, Marchesi, F, Mengarelli, A, Cerchiara, E, Tirindelli, Mc, Gaziev, J, Majolino, I, Chiusolo, P, Lucarelli, B, Massei, Ms, Carotti, A, Perruccio, K, Caniglia, M, Santarone, S, Bartolomeo, Pd, Mazzotta, S, Galieni, P, Olivieri, A, Rosa, Gd, Risitano, A, Delia, M, Specchia, G, Palazzo, G, Messina, G, Irrera, G, Angelucci, E, Baronciani, D, Vacca, A, Crescimanno, A, Musso, M, Imbriani, A, Milone, G., Girmenia, C, Rossolini, Gm, Piciocchi, A, Bertaina, A, Pisapia, G, Pastore, D, Sica, S, Severino, A, Cudillo, L, Ciceri, Fabio, Scime, R, Lombardini, L, Viscoli, C, Rambaldi, A, and and the Gruppo Italiano Trapianto Midollo Osseo, (GITMO)
- Subjects
Male ,Klebsiella pneumoniae ,Carbapenem resistant Klebsiella pneumoniae ,Drug Resistance ,carbapenem-resistant Klebsiella pneumoniae ,colonization ,sepsis ,Allogeneic Hematopoietic Stem Cell Transplantation ,Hematologic Diseases ,hemic and lymphatic diseases ,polycyclic compounds ,Medicine ,Adolescent ,Adult ,Aged ,Allografts ,Autografts ,Female ,Humans ,Italy ,Klebsiella Infections ,Middle Aged ,Retrospective Studies ,Carbapenems ,Drug Resistance, Bacterial ,Stem Cell Transplantation ,Hematology ,Transplantation ,biology ,Bacterial ,humanities ,surgical procedures, operative ,medicine.medical_specialty ,Retrospective survey ,Internal medicine ,business.industry ,Retrospective cohort study ,Klebsiella infections ,biology.organism_classification ,Settore MED/15 ,infection ,body regions ,Settore MED/15 - MALATTIE DEL SANGUE ,Immunology ,business - Abstract
Infections by carbapenem-resistant Klebsiella pneumoniae (CRKp) represent a challenging problem after SCT. A retrospective survey (January 2010 to July 2013) involving 52 Italian centers was performed to assess the epidemiology and the prognostic factors of CRKp infections in auto- and allo-SCT. Cases of CRKp infection were reported in 53.4% of centers. CRKp infections were documented in 25 auto-SCTs and 87 allo-SCTs, with an incidence of 0.4% (from 0.1% in 2010 to 0.7% in 2013) and 2% (from 0.4% in 2010 to 2.9% in 2013), respectively. A CRKp colonization documented before or after transplant was followed by an infection in 25.8% of auto-SCT and 39.2% of allo-SCT patients. The infection-related mortality rates were 16% and 64.4%, respectively. A pre-transplant CRKp infection (hazard ratio (HR) 0.33, 95% confidence intervals (CIs) 0.15-0.74; P = 0.007) and a not CRKp-targeted first-line treatment (HR 2.67, 95% CI 1.43-4.99; P = 0.002) were independent factors associated with an increased mortality in allo-SCT patients who developed a CRKp infection. Our study shows challenging findings of CRKp infections in SCT patients in Italy particularly after allo-SCT. The detection of carriers and the definition of early therapeutic strategies represent critical aspects of the management of CRKp infections after SCT. OI Aversa, Franco/0000-0002-8871-6817; de Fabritiis, Paolo/0000-0002-1835-0581; TIRINDELLI, MARIA CRISTINA/0000-0003-4645-4465; cudillo, laura/0000-0002-6828-9707
- Published
- 2015
18. Invasive Pulmonary Aspergillosis in a Haematopoietic Stem Cell Transplant Recipient with Sickle Cell Disease: a Successful Treatment
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Paciaroni, K, De Angelis, G, Gallucci, C, Alfieri, C, Ribersani, M, Roveda, A, Isgro, A, Marziali, M, Aloi, Ip, Inserra, A, Gaziev, J, Sodani, P, and Lucarelli, G
- Subjects
surgical procedures, operative ,Settore MED/20 ,Case Report - Abstract
Sickle Cell Anaemia (SCA) is the most common inherited blood disorder and is associated with severe morbidity and decreased survival. Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) is the only curative approach. Nevertheless the decision to perform a bone marrow transplant includes the risk of major complications and transplant-related mortality. Infections represent the leading cause of death in SCA patients undergoing HSCT. Invasive Pulmonary Aspergillosis (IPA) is a devastating opportunistic infection and remains a significant cause of morbidity and mortality in HSCT recipients. Data regarding IPA in the setting of SCA are lacking. In the present report, we describe a patient with SCA, who developed IPA after allogeneic bone marrow transplant. The fungal infection was treated by systemic antifungal therapy in addition to surgery, despite mild chronic graft versus host disease (GVHD) and continuing immunosuppressive therapy. This case shows that IPA occurring in bone marrow recipients with SCA can be successfully treated.
- Published
- 2015
19. New insights into the pharmacokinetics of intravenous busulfan in children with sickle cell anemia undergoing bone marrow transplantation
- Author
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Gaziev, J., Isgro, A., Mozzi, A. F., Petain, A., Nguyen, L., Ialongo, C., Dinallo, V., Sodani, P., Marziali, M., Andreani, M., Testi, M., Paciaroni, K., Gallucci, C., De Angelis, G., Alfieri, C., Ribersani, M., and Lucarelli, G.
- Subjects
Bone marrow transplantation ,Sickle cell anemia ,Clearance ,Intravenous busulfan ,Pharmacokinetics ,Children - Published
- 2015
20. Hemopoietic stem cell transplantation in thalassemia: A report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010
- Author
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Baronciani, D., Angelucci, E., Potschger, U., Gaziev, J., Yesilipek, A., Zecca, M., Orofino, M. G., Giardini, C., Al-Ahmari, A., Marktel, S., De La Fuente, J., Ghavamzadeh, A., Hussein, A. A., Targhetta, C., Pilo, F., Locatelli, Franco, Dini, G., Bader, P., Peters, C., Locatelli F. (ORCID:0000-0002-7976-3654), Baronciani, D., Angelucci, E., Potschger, U., Gaziev, J., Yesilipek, A., Zecca, M., Orofino, M. G., Giardini, C., Al-Ahmari, A., Marktel, S., De La Fuente, J., Ghavamzadeh, A., Hussein, A. A., Targhetta, C., Pilo, F., Locatelli, Franco, Dini, G., Bader, P., Peters, C., and Locatelli F. (ORCID:0000-0002-7976-3654)
- Abstract
Allogeneic hemopoietic stem cell transplantation (HSCT) is the only method currently available to cure transfusion-dependent thalassemia major that has been widely used worldwide. To verify transplantation distribution, demography, activity, policies and outcomes inside the European Group for Blood and Marrow Transplantation (EBMT), we performed a retrospective non-interventional study, extracting data from the EBMT hemoglobinopathy prospective registry database. We included 1493 consecutive patients with thalassemia major transplanted between 1 January 2000 and 31 December 2010. In total, 1359 (91%) transplants were performed on patients <18 years old, 1061 were from a human leukocyte Ag-identical sibling donor. After a median observation time of 2 years, the 2-year overall survival (OS) and event-free survival (EFS; that is, thalassemia-free survival) were 88±1% and 81±1%, respectively. Transplantation from a human leukocyte Ag-identical sibling offered the best results, with OS and EFS of 91±1% and 83±1%, respectively. No significant differences in survival were reported between countries. The threshold age for optimal transplant outcomes was around 14 years, with an OS of 90-96% and an EFS of 83-93% when transplants were performed before this age. Allogeneic HSCT for thalassemia is a curative approach that is employed internationally and produces excellent results.
- Published
- 2016
21. Influence of the HLA characteristics of Italian patients on donor search outcome in unrelated hematopoietic stem cell transplantation
- Author
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Testi, M, Andreani, M, Locatelli, Franco, Arcese, W, Troiano, M, Battarra, M, Gaziev, J, and Lucarelli, G
- Subjects
hematopoietic stem cell transplantation ,human leukocyte antigen ,matched unrelated donor search ,Alleles ,Gene Frequency ,Genetic Loci ,HLA Antigens ,Haplotypes ,Humans ,Italy ,Unrelated Donors ,Donor Selection ,Hematopoietic Stem Cell Transplantation ,Tissue Donors ,Settore MED/15 ,Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA ,Hematopoietic stem cell transplantation ,Human leukocyte antigen ,Matched unrelated donor search - Abstract
The information regarding the probability of finding a matched unrelated donor (MUD) within a relatively short time is crucial for the success of hematopoietic stem cell transplantation (HSCT), particularly in patients with malignancies. In this study, we retrospectively analyzed 315 Italian patients who started a search for a MUD, in order to assess the distribution of human leukocyte antigen (HLA) alleles and haplotypes in this population of patients and to evaluate the probability of finding a donor. Comparing two groups of patients based on whether or not a 10/10 HLA-matched donor was available, we found that patients who had a fully-matched MUD possessed at least one frequent haplotype more often than the others (45.6% vs 14.3%; P = 0.000003). In addition, analysis of data pertaining to the HLA class I alleles distribution showed that, in the first group of patients, less common alleles were under-represented (20.2% vs 40.0%; P = 0.006). Therefore, the presence of less frequent alleles represents a negative factor for the search for a potential compatible donor being successful, whereas the presence of one frequent haplotype represents a positive predictive factor. Antigenic differences between patient and donor observed at C and DQB1 loci, were mostly represented by particular B/C or DRB1/DQB1 allelic associations. Thus, having a particular B or DRB1 allele, linked to multiple C or DQB1 alleles, respectively, might be considered to be associated with a lower probability of a successful search. Taken together, these data may help determine in advance the probability of finding a suitable unrelated donor for an Italian patient.
- Published
- 2014
22. Donor-recipient mismatches for the minor histocompatibilty antigen HA-8 are significantly associated with rejection of haematopoietic stem cell transplantation for thalassemia
- Author
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Andreani, M., Testi, M., Troiano, M., Gaziev, J., Lucarelli, G., Sizzano, F., and Fleischhauer, Katharina
- Subjects
Medizin ,ComputingMethodologies_GENERAL - Abstract
Poster Abstract
- Published
- 2014
23. Late-onset haemorrhagic cystitis in children after haematopoietic stem cell transplantation for thalassaemia and sickle cell anaemia: a prospective evaluation of polyoma (BK) virus infection and treatment with cidofovir (CDV)
- Author
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Gaziev, J, Germani, S, Miano, R, Bove, P, Paba, P, Perno, C, Sodani, P, Marziali, M, Isgro, A, Polchi, P, Paciaroni, K, Gallucci, C, Simone, M, Roveda, A, De Angelis, G, Alfieri, C, and Lucarelli, G
- Subjects
Settore MED/24 - Urologia - Published
- 2010
24. Hematopoietic Stem Cell Transplantation in Thalassemia and Sickle Cell Anemia
- Author
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Lucarelli, G., primary, Isgro, A., additional, Sodani, P., additional, and Gaziev, J., additional
- Published
- 2012
- Full Text
- View/download PDF
25. Allogeneic cellular gene therapy in hemoglobinopathies—evaluation of hematopoietic SCT in sickle cell anemia
- Author
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Lucarelli, G, primary, Gaziev, J, additional, Isgrò, A, additional, Sodani, P, additional, Paciaroni, K, additional, Alfieri, C, additional, De Angelis, G, additional, Marziali, M, additional, Simone, M D, additional, Gallucci, C, additional, Roveda, A, additional, Saltarelli, F, additional, Torelli, F, additional, and Andreani, M, additional
- Published
- 2011
- Full Text
- View/download PDF
26. Higher CD3+ and CD34+ cell doses in the graft increase the incidence of acute GVHD in children receiving BMT for thalassemia
- Author
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Gaziev, J, primary, Isgrò, A, additional, Marziali, M, additional, Daniele, N, additional, Gallucci, C, additional, Sodani, P, additional, Simone, M D, additional, Adorno, G, additional, Paciaroni, K, additional, Andreani, M, additional, Lanti, A, additional, Del Proposto, G, additional, Testi, M, additional, De Angelis, G, additional, Roveda, A, additional, Alfieri, C, additional, Saltarelli, F, additional, and Lucarelli, G, additional
- Published
- 2011
- Full Text
- View/download PDF
27. Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease
- Author
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Andreani, M., primary, Testi, M., additional, Gaziev, J., additional, Condello, R., additional, Bontadini, A., additional, Tazzari, P. L., additional, Ricci, F., additional, De Felice, L., additional, Agostini, F., additional, Fraboni, D., additional, Ferrari, G., additional, Battarra, M., additional, Troiano, M., additional, Sodani, P., additional, and Lucarelli, G., additional
- Published
- 2010
- Full Text
- View/download PDF
28. Higher CD3+ and CD34+ cell doses in the graft increase the incidence of acute GVHD in children receiving BMT for thalassemia.
- Author
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Gaziev, J, Isgrò, A, Marziali, M, Daniele, N, Gallucci, C, Sodani, P, Simone, M D, Adorno, G, Paciaroni, K, Andreani, M, Lanti, A, Del Proposto, G, Testi, M, De Angelis, G, Roveda, A, Alfieri, C, Saltarelli, F, and Lucarelli, G
- Subjects
- *
GRAFT versus host disease , *DISEASE incidence , *DISEASE risk factors , *JUVENILE diseases , *BONE marrow transplantation , *THALASSEMIA treatment - Abstract
We evaluated the incidence of GVHD, risk factors and the impact of graft composition on acute GVHD (aGVHD) in 92 children who underwent BMT for thalassemia following busulfan/cyclophosphamide (BUCY)-based conditioning regimens and GVHD prophylaxis with CSA/short-MTX and methylprednisolone. The incidence of grade 2-4 and 3-4 aGVHD was 35% (95% confidence interval (CI) 25-44) and 9% (95% CI 4-16), respectively. We found that CD3+ and CD34+ cell doses above the median were associated with high incidence of grade 2-4 aGVHD (49 vs 20%, P=0.005 and 46 vs 23%, P=0.021, respectively). In multivariate analysis, high CD3+ (hazard ratio (HR) 4.6; 95% CI 1.4-14.7; P=0.010) and CD34+ (HR 4.3; 95% CI 1.4-12.7; P=0.011) cell doses were associated with grade 2-4 aGVHD. We further examined the effect of CD3+ and CD34+ cell doses on aGVHD using quartile cutoff points and found a minimum threshold for CD3+ (38 × 106/kg) and CD34+ (4 × 106/kg) cells above which the incidence of grade 2-4 aGVHD is significantly increased. This study shows for the first time a positive correlation between the number of CD3+ and CD34+ cells and aGVHD in children receiving sibling BMT, and indicates that using tailored and more intensive post transplant immunosuppression may permit to better control aGVHD. [ABSTRACT FROM AUTHOR]
- Published
- 2012
- Full Text
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29. Bone marrow transplantation in Class 3 thalassaemia patients
- Author
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Emanuele Angelucci, Baronciani, D., Lucarelli, G., Giardini, C., Galimberti, M., Polchi, P., Erer, B., and Gaziev, J.
30. Pancreatic beta-cell function before and after bone marrow transplantation for thalassemia
- Author
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Galimberti, M., Sanctis, V., Lucarelli, G., Polchi, P., Emanuele Angelucci, Baronciani, D., Giardini, C., Erer, B., Gaziev, J., Balducci, R., and Vullo, C.
31. Bone marrow transplantation in Class 1 thalassemia patients
- Author
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Baronciani, D., Galimberti, M., Lucarelli, G., Polchi, P., Emanuele Angelucci, Giardini, C., Giorgi, C., and Gaziev, J.
32. Hemorrhagic cystitis after allogeneic bone marrow transplantation for thalassemia
- Author
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Erer, B., Emanuele Angelucci, Baronciani, D., Tomasucci, M., Giardini, C., and Gaziev, J.
33. Endocrinological study in patient after transplantation for leukemia | STUDIO ENDOCRINO IN PAZIENTI TRAPIANTATI PER TALASSEMIA
- Author
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Galimberti, M., Sanctis, V., Lucarelli, G., Polchi, G., Angelucci, E., Donatella Baronciani, Giardini, C., Durazzi, S. M. T., Erer, B., Gaziev, J., and Vullo, C.
34. COVID-19 infection in adult patients with hematological malignancies:a European Hematology Association Survey (EPICOVIDEHA)
- Author
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Pagano, Livio, Salmanton-García, Jon, Marchesi, Francesco, Busca, Alessandro, Corradini, Paolo, Hoenigl, Martin, Klimko, Nikolai, Koehler, Philipp, Pagliuca, Antonio, Passamonti, Francesco, Verga, Luisa, Víšek, Benjamin, Ilhan, Osman, Nadali, Gianpaolo, Weinbergerová, Barbora, Córdoba-Mascuñano, Raúl, Marchetti, Monia, Collins, Graham P., Farina, Francesca, Cattaneo, Chiara, Cabirta, Alba, Gomes-Silva, Maria, Itri, Federico, van Doesum, Jaap, Ledoux, Marie-Pierre, Čerňan, Martin, Jakšić, Ozren, Duarte, Rafael F., Magliano, Gabriele, Omrani, Ali S., Fracchiolla, Nicola S., Kulasekararaj, Austin, Valković, Toni, Poulsen, Christian Bjørn, Machado, Marina, Glenthøj, Andreas, Stoma, Igor, Ráčil, Zdeněk, Piukovics, Klára, Navrátil, Milan, Emarah, Ziad, Sili, Uluhan, Maertens, Johan, Blennow, Ola, Bergantim, Rui, García-Vidal, Carolina, Prezioso, Lucia, Guidetti, Anna, del Principe, Maria Ilaria, Popova, Marina, de Jonge, Nick, Ormazabal-Vélez, Irati, Fernández, Noemí, Falces-Romero, Iker, Cuccaro, Annarosa, Meers, Stef, Buquicchio, Caterina, Antić, Darko, Al-Khabori, Murtadha, García-Sanz, Ramón, Biernat, Monika M., Tisi, Maria Chiara, Sal, Ertan, Rahimli, Laman, Čolović, Natasa, Schönlein, Martin, Calbacho, Maria, Tascini, Carlo, Miranda-Castillo, Carolina, Khanna, Nina, Méndez, Gustavo-Adolfo, Petzer, Verena, Novák, Jan, Besson, Caroline, Duléry, Rémy, Lamure, Sylvain, Nucci, Marcio, Zambrotta, Giovanni, Žák, Pavel, Seval, Guldane Cengiz, Bonuomo, Valentina, Mayer, Jiří, López-García, Alberto, Sacchi, Maria Vittoria, Booth, Stephen, Ciceri, Fabio, Oberti, Margherita, Salvini, Marco, Izuzquiza, Macarena, Nunes-Rodrigues, Raquel, Ammatuna, Emanuele, Obr, Aleš, Herbrecht, Raoul, Núñez-Martín-Buitrago, Lucía, Mancini, Valentina, Shwaylia, Hawraa, Sciumè, Mariarita, Essame, Jenna, Nygaard, Marietta, Batinić, Josip, Gonzaga, Yung, Regalado-Artamendi, Isabel, Karlsson, Linda Katharina, Shapetska, Maryia, Hanakova, Michaela, El-Ashwah, Shaimaa, Borbényi, Zita, Çolak, Gökçe Melis, Nordlander, Anna, Dragonetti, Giulia, Maraglino, Alessio Maria Edoardo, Rinaldi, Amelia, De Ramón-Sánchez, Cristina, Cornely, Oliver A., Finizio, Olimpia, Fazzi, Rita, Sapienza, Giuseppe, Chauchet, Adrien, Van Praet, Jens, Prattes, Juergen, Dargenio, Michelina, Rossi, Cédric, Shirinova, Ayten, Malak, Sandra, Tafuri, Agostino, Ommen, Hans-Beier, Bologna, Serge, Khedr, Reham Abdelaziz, Choquet, Sylvain, Joly, Bertrand, Ceesay, M. Mansour, Philippe, Laure, Kho, Chi Shan, Desole, Maximilian, Tsirigotis, Panagiotis, Otašević, Vladimir, Borducchi, Davimar M. M., Antoniadou, Anastasia, Gaziev, Javid, Almaslamani, Muna A., García-Poutón, Nicole, Paterno, Giovangiacinto, Torres-López, Andrea, Tarantini, Giuseppe, Mellinghoff, Sibylle, Gräfe, Stefanie, Börschel, Niklas, Passweg, Jakob, Merelli, Maria, Barać, Aleksandra, Wolf, Dominik, Shaikh, Mohammad Usman, Thiéblemont, Catherine, Bernard, Sophie, Funke, Vaneuza Araújo Moreira, Daguindau, Etienne, Khostelidi, Sofya, Nucci, Fabio Moore, Martín-González, Juan-Alberto, Landau, Marianne, Soussain, Carole, Laureana, Cécile, Lacombe, Karine, Kohn, Milena, Aliyeva, Gunay, Piedimonte, Monica, Fouquet, Guillemette, Rêgo, Mayara, Hoell-Neugebauer, Baerbel, Cartron, Guillaume, Pinto, Fernando, Alburquerque, Ana Munhoz, Passos, Juliana, Yilmaz, Asu Fergun, Redondo-Izal, Ana-Margarita, Altuntaş, Fevzi, Heath, Christopher, Kolditz, Martin, Schalk, Enrico, Guolo, Fabio, Karthaus, Meinolf, Della Pepa, Roberta, Vinh, Donald, Noël, Nicolas, Deau Fischer, Bénédicte, Drenou, Bernard, Mitra, Maria Enza, Meletiadis, Joseph, Bilgin, Yavuz M., Jindra, Pavel, Espigado, Ildefonso, Drgoňa, Ľuboš, Serris, Alexandra, Di Blasi, Roberta, Ali, Natasha, EPICOVIDEHA working group, [missing], Pagano, Livio, Salmanton-Garcia, Jon, Marchesi, Francesco, Busca, Alessandro, Corradini, Paolo, Hoenigl, Martin, Klimko, Nikolai, Koehler, Philipp, Pagliuca, Antonio, Passamonti, Francesco, Verga, Luisa, Visek, Benjamin, Ilhan, Osman, Nadali, Gianpaolo, Weinbergerova, Barbora, Cordoba-Mascunano, Raul, Marchetti, Monia, Collins, Graham P., Farina, Francesca, Cattaneo, Chiara, Cabirta, Alba, Gomes-Silva, Maria, Itri, Federico, van Doesum, Jaap, Ledoux, Marie-Pierre, Cernan, Martin, Jaksic, Ozren, Duarte, Rafael F., Magliano, Gabriele, Omrani, Ali S., Fracchiolla, Nicola S., Kulasekararaj, Austin, Valkovic, Toni, Poulsen, Christian Bjorn, Machado, Marina, Glenthoj, Andreas, Stoma, Igor, Racil, Zdenek, Piukovics, Klara, Navratil, Milan, Emarah, Ziad, Sili, Uluhan, Maertens, Johan, Blennow, Ola, Bergantim, Rui, Garcia-Vidal, Carolina, Prezioso, Lucia, Guidetti, Anna, del Principe, Maria Ilaria, Popova, Marina, de Jonge, Nick, Ormazabal-Velez, Irati, Fernandez, Noemi, Falces-Romero, Iker, Cuccaro, Annarosa, Meers, Stef, Buquicchio, Caterina, Antic, Darko, Al-Khabori, Murtadha, Garcia-Sanz, Ramon, Biernat, Monika M., Tisi, Maria Chiara, Sal, Ertan, Rahimli, Laman, Colovic, Natasa, Schonlein, Martin, Calbacho, Maria, Tascini, Carlo, Miranda-Castillo, Carolina, Khanna, Nina, Mendez, Gustavo-Adolfo, Petzer, Verena, Novak, Jan, Besson, Caroline, Dulery, Remy, Lamure, Sylvain, Nucci, Marcio, Zambrotta, Giovanni, Zak, Pavel, Seval, Guldane Cengiz, Bonuomo, Valentina, Mayer, Jiri, Lopez-Garcia, Alberto, Sacchi, Maria Vittoria, Booth, Stephen, Ciceri, Fabio, Oberti, Margherita, Salvini, Marco, Izuzquiza, Macarena, Nunes-Rodrigues, Raquel, Ammatuna, Emanuele, Obr, Ales, Herbrecht, Raoul, Nunez-Martin-Buitrago, Lucia, Mancini, Valentina, Shwaylia, Hawraa, Sciume, Mariarita, Essame, Jenna, Nygaard, Marietta, Batinic, Josip, Gonzaga, Yung, Regalado-Artamendi, Isabel, Karlsson, Linda Katharina, Shapetska, Maryia, Hanakova, Michaela, El-Ashwah, Shaimaa, Borbenyi, Zita, Colak, Gokce Melis, Nordlander, Anna, Dragonetti, Giulia, Maraglino, Alessio Maria Edoardo, Rinaldi, Amelia, De Ramon-Sanchez, Cristina, Cornely, Oliver A., Pagano, L., Salmanton-Garcia, J., Marchesi, F., Busca, A., Corradini, P., Hoenigl, M., Klimko, N., Koehler, P., Pagliuca, A., Passamonti, F., Verga, L., Visek, B., Ilhan, O., Nadali, G., Weinbergerova, B., Cordoba-Mascunano, R., Marchetti, M., Collins, G. P., Farina, F., Cattaneo, C., Cabirta, A., Gomes-Silva, M., Itri, F., van Doesum, J., Ledoux, M. -P., Cernan, M., Jaksic, O., Duarte, R. F., Magliano, G., Omrani, A. S., Fracchiolla, N. S., Kulasekararaj, A., Valkovic, T., Poulsen, C. B., Machado, M., Glenthoj, A., Stoma, I., Racil, Z., Piukovics, K., Navratil, M., Emarah, Z., Sili, U., Maertens, J., Blennow, O., Bergantim, R., Garcia-Vidal, C., Prezioso, L., Guidetti, A., del Principe, M. I., Popova, M., de Jonge, N., Ormazabal-Velez, I., Fernandez, N., Falces-Romero, I., Cuccaro, A., Meers, S., Buquicchio, C., Antic, D., Al-Khabori, M., Garcia-Sanz, R., Biernat, M. M., Tisi, M. C., Sal, E., Rahimli, L., Colovic, N., Schonlein, M., Calbacho, M., Tascini, C., Miranda-Castillo, C., Khanna, N., Mendez, G. -A., Petzer, V., Novak, J., Besson, C., Dulery, R., Lamure, S., Nucci, M., Zambrotta, G., Zak, P., Seval, G. C., Bonuomo, V., Mayer, J., Lopez-Garcia, A., Sacchi, M. V., Booth, S., Ciceri, F., Oberti, M., Salvini, M., Izuzquiza, M., Nunes-Rodrigues, R., Ammatuna, E., Obr, A., Herbrecht, R., Nunez-Martin-Buitrago, L., Mancini, V., Shwaylia, H., Sciume, M., Essame, J., Nygaard, M., Batinic, J., Gonzaga, Y., Regalado-Artamendi, I., Karlsson, L. K., Shapetska, M., Hanakova, M., El-Ashwah, S., Borbenyi, Z., Colak, G. M., Nordlander, A., Dragonetti, G., Maraglino, A. M. E., Rinaldi, A., De Ramon-Sanchez, C., Cornely, O. A., Finizio, O., Fazzi, R., Sapienza, G., Chauchet, A., Van Praet, J., Prattes, J., Dargenio, M., Rossi, C., Shirinova, A., Malak, S., Tafuri, A., Ommen, H. -B., Bologna, S., Khedr, R. A., Choquet, S., Joly, B., Ceesay, M. M., Philippe, L., Kho, C. S., Desole, M., Tsirigotis, P., Otasevic, V., Borducchi, D. M. M., Antoniadou, A., Gaziev, J., Almaslamani, M. A., Garcia-Pouton, N., Paterno, G., Torres-Lopez, A., Tarantini, G., Mellinghoff, S., Grafe, S., Borschel, N., Passweg, J., Merelli, M., Barac, A., Wolf, D., Shaikh, M. U., Thieblemont, C., Bernard, S., Funke, V. A. M., Daguindau, E., Khostelidi, S., Nucci, F. M., Martin-Gonzalez, J. -A., Landau, M., Soussain, C., Laureana, C., Lacombe, K., Kohn, M., Aliyeva, G., Piedimonte, M., Fouquet, G., Rego, M., Hoell-Neugebauer, B., Cartron, G., Pinto, F., Alburquerque, A. M., Passos, J., Yilmaz, A. F., Redondo-Izal, A. -M., Altuntas, F., Heath, C., Kolditz, M., Schalk, E., Guolo, F., Karthaus, M., Della Pepa, R., Vinh, D., Noel, N., Deau Fischer, B., Drenou, B., Mitra, M. E., Meletiadis, J., Bilgin, Y. M., Jindra, P., Espigado, I., Drgona, L., Serris, A., Di Blasi, R., Ali, N., Stem Cell Aging Leukemia and Lymphoma (SALL), Salvy-Córdoba, Nathalie, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Università cattolica del Sacro Cuore = Catholic University of the Sacred Heart [Roma] (Unicatt), University Hospital of Cologne [Cologne], Cologne Excellence Cluster on Cellular Stress Responses in Aging-Associated Diseases (CECAD), University of Cologne, IFO - Istituto Nazionale Tumori Regina Elena [Roma] (IRE), Città della Salute e della Scienza University-Hospital, IRCCS Istituto Nazionale dei Tumori [Milano], University of California [San Diego] (UC San Diego), University of California (UC), Medical University of Graz, Odessa National I.I.Mechnikov University, Faculty of Medicine [Cologne], University Hospital of Cologne [Cologne]-University of Cologne, King's College Hospital (KCH), Universitá degli Studi dell’Insubria = University of Insubria [Varese] (Uninsubria), Dipartimento di Medicina e Chirurgia = School of Medicine and Surgery [Monza], Università degli Studi di Milano-Bicocca = University of Milano-Bicocca (UNIMIB), Faculty of Medicine in Hradec Kralove [Republique Tchèque], Charles University [Prague] (CU), Ankara University School of Medicine [Turkey], Azienda Ospedaliera Universitaria Integrata of Verona, Masaryk University [Brno] (MUNI), Fundación Jiménez Díaz, Fundacion Jimenez Diaz [Madrid] (FJD), Ospedale SS Antonio e Biagio e Cesare Arrigo, Churchill Hospital Oxford Centre for Haematology, IRCCS San Raffaele Scientific Institute [Milan, Italie], ASST Spedali Civili of Brescia, Vall d'Hebron Institute of Oncology [Barcelone] (VHIO), Vall d'Hebron University Hospital [Barcelona], Universitat Autònoma de Barcelona (UAB), Instituto Português de Oncologia de Lisboa Francisco Gentil, Ospedale San Luigi Gonzaga, University Medical Center Groningen [Groningen] (UMCG), Institut de Cancérologie de Strasbourg Europe (ICANS), Palacky University Olomouc, Zagreb School of Medicine [Zagreb, Croatia] (Dubrava University Hospital), University of Zagreb, Hospital Universitario Puerta de Hierro-Majadahonda [Madrid, Spain], ASST Great Metropolitan Niguarda / ASST Grande Ospedale Metropolitano Niguarda [Milan, Italia], Hamad Medical Corporation [Doha, Qatar], Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, University of Rijeka, Croatian Cooperative Group for Hematological Diseases (CROHEM), Zealand University Hospital [Roskilde, Denmark], Hospital General Universitario 'Gregorio Marañón' [Madrid], Department of Clinical Microbiology [Rigshospitalet], Rigshospitalet [Copenhagen], Copenhagen University Hospital-Copenhagen University Hospital, Homieĺ State Medical University (GSMU), Institute of Hematology and Blood Transfusion [Prague, Czech Republic], University of Szeged [Szeged], University Hospital Ostrava, Mansoura University [Egypt], Marmara University [Kadıköy - İstanbul], Catholic University of Leuven - Katholieke Universiteit Leuven (KU Leuven), Karolinska University Hospital [Stockholm], Instituto de Investigação e Inovação em Saúde (I3S), Universidade do Porto = University of Porto, Instituto de Patologia e Imunologia Molecular da Universidade do Porto (IPATIMUP), Hospital de São João [Porto], Faculdade de Medicina da Universidade do Porto (FMUP), Clinic Barcelona Hospital Universitari, Department of Public Health and Cell Biology, University of Rome Tor Vergata, Via Montpellier 1, 00133 Rome, Italy, Pavlov First Saint Petersburg State Medical University [St. Petersburg], Vrije Universiteit Medical Centre (VUMC), Vrije Universiteit Amsterdam [Amsterdam] (VU), Complejo Hospitalario de Navarra, Hospital Universitario Marqués de Valdecilla [Santander], La Paz University Hospital, Azienda Usl Toscana centro [Firenze], AZ Klina, Clinical Center of Serbia (KCS), University of Belgrade [Belgrade], Sultan Qaboos University Hospital, Partenaires INRAE, Hospital Universitario de Salamanca, Servicio de Haematologia, Centro de Investigación del Cáncer-IBMCC (USAL-CSIC), University of Wrocław [Poland] (UWr), San Bortolo Hospital, Universitaetsklinikum Hamburg-Eppendorf = University Medical Center Hamburg-Eppendorf [Hamburg] (UKE), Hospital Universitario 12 de Octubre [Madrid], Azienda Sanitaria Universitaria Friuli Centrale (ASU FC), Universidad Rey Juan Carlos [Madrid] (URJC), University of Basel (Unibas), Innsbruck Medical University = Medizinische Universität Innsbruck (IMU), University Hospital Kralovské Vinohrady, Centre Hospitalier de Versailles André Mignot (CHV), Centre de Recherche Saint-Antoine (CRSA), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU), Institut de Génétique Moléculaire de Montpellier (IGMM), Université de Montpellier (UM)-Centre National de la Recherche Scientifique (CNRS), Département Hématologie biologique [CHRU Montpellier], Pôle Biologie-Pathologie [CHRU Montpellier], Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier)-Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier), Universidade Federal do Estado do Rio de Janeiro (UNIRIO), San Gerardo Hospital of Monza, Oxford NIHR Biomedical Research Centre, IRCCS Ospedale San Raffaele [Milan, Italy], Assi Sette Llaghi Varese, Instituto Português de Oncologia do Porto / Portuguese Oncology Institute of Porto (IPO Porto), University Hospital Olomouc [Czech Republic], ASST Grande Ospedale Metropolitano Niguarda, University Hospital Centre Zagreb, Instituto Nacional do Câncer, Copenhagen University Hospital, Republican Scientific and Practical Center (RSPC) for organ and Tissue Transplantation, Minsk, Republican Scientific and Practical Center (RSPC) for Organ and Transplantation, German Centre for Infection Research (DZIF), Gilead Sciences, Pagano, Livio [0000-0001-8287-928X], Salmanton-García, Jon [0000-0002-6766-8297], Marchesi, Francesco [0000-0001-6353-2272], Busca, Alessandro [0000-0001-5361-5613], Corradini, Paolo [0000-0002-9186-1353], Hoenigl, Martin [0000-0002-1653-2824], Klimko, Nikolay [0000-0001-6095-7531], Koehler, Philipp [0000-0002-7386-7495], Pagliuca, Antonio [0000-0003-2519-0333], Passamonti, Francesco [0000-0001-8068-5289], Verga, Luisa [0000-0003-1142-8435], Víšek, Benjamin [0000-0001-8268-452X], Ilhan, Osman [0000-0003-1665-372X], Weinbergerová, Barbora [0000-0001-6460-2471], Córdoba, Raúl [0000-0002-7654-8836], Marchetti, Monia [0000-0001-7615-0572], Farina, Francesca [0000-0002-5124-6970], Cattaneo, Chiara [0000-0003-0031-3237], Cabirta, Alba [0000-0001-7198-8894], Gomes-Silva, Maria [0000-0002-6993-2450], Itri, Federico [0000-0002-3532-5281], Doesum, Jaap van [0000-0003-0214-3219], Ledoux, Marie-Pierre [0000-0002-3261-3616], Čerňan, Martin [0000-0003-2345-1229], Jakšić, Ozren [0000-0003-4026-285X], Magliano, Gabriel [0000-0002-9129-1530], Omrani, Ali S. [0000-0001-5309-6358], Fracchiolla, Nicola S. [0000-0002-8982-8079], Kulasekararaj, Austin G. [0000-0003-3180-3570], Valković, Toni [0000-0001-6083-8815], Poulsen, Christian Bjørn [0000-0001-9785-1378], Machado, Marina [0000-0002-8370-2248], Glenthøj, Andrea [0000-0003-2082-0738], Stoma, Igor [0000-0003-0483-7329], Ráčil, Zdeněk [0000-0003-3511-4596], Piukovics, Klára [0000-0003-4480-3131], Emarah, Ziad [0000-0003-0622-2598], Sili, Uluhan [0000-0002-9939-9298], Maertens, Johan [0000-0003-4257-5980], Bergantim, Rui [0000-0002-7811-9509], García-Vidal, Carolina [0000-0002-8915-0683], Prezioso, Lucia [0000-0003-1660-4960], Principe, Maria Ilaria del [0000-0002-3958-0669], Popova, Marina [0000-0001-8536-5495], Jonge, Nick de [0000-0002-9901-0887], Ormazabal-Vélez, Irati [0000-0003-1141-5546], Falces-Romero, Iker [0000-0001-5888-7706], Cuccaro, Annarosa [0000-0002-0237-1839], Meers, Stef [0000-0003-1754-2175], Buquicchio, Caterina [0000-0002-3683-5953], Antić, Darko [0000-0002-2608-1342], Al-Khabori, Murtadha [0000-0002-2937-8838], García-Sanz, Ramón [0000-0003-4120-2787], Biernat, Monika [0000-0003-3161-3398], Tisi, Maria Chiara [0000-0001-8231-6700], Sal, Ertan [0000-0003-2761-2675], Rahimli, Laman [0000-0003-2266-445X], Schönlein, Martin [0000-0002-1010-0975], Calbacho, María [0000-0001-8106-4863], Tascini, Carlo [0000-0001-9625-6024], Miranda-Castillo, Carolina [0000-0001-8763-9576], Khanna, Nina [0000-0002-2642-419X], Méndez, Gustavo-Adolfo [0000-0003-0514-7004], Petzer, Verena [0000-0002-9205-1440], Besson, Caroline [0000-0003-4364-7173], Duléry, Rémy [0000-0002-5024-1713], Lamure, Sylvain [0000-0001-5980-305X], Nucci, Marcio [0000-0003-4867-0014], Zambrotta, Giovanni [0000-0002-8612-2994], Žák, Pavel [0000-0003-4465-5343], Cengiz Seval, Guldane [0000-0001-9433-2054], Bonuomo, Valentina [0000-0001-6491-8337], Mayer, Jiří [0000-0003-0567-9887], López-García, Alberto [0000-0002-5354-5261], Sacchi, Maria Vittoria [0000-0001-8133-3357], Booth, Stephen [0000-0003-2687-0234], Ciceri, Fabio [0000-0003-0873-0123], Nunes-Rodrigues, Raquel [0000-0002-8347-4281], Ammatuna, Emanuele [0000-0001-8247-4901], Obr, Aleš [0000-0002-6758-3074], Herbrecht, Raoul [0000-0002-9381-4876], Shwaylia, Hawraa [0000-0002-4098-6092], Sciumè, Mariarita [0000-0001-7958-4966], Essame, Jenna [0000-0003-0926-5577], Batinić, Josip [0000-0001-5595-9911], Gonzaga, Yung [0000-0003-1416-2118], Regalado-Artamendi, Isabel [0000-0002-9673-9015], Karlsson, Linda Katharina [0000-0003-3317-7550], Shapetska, Maryia [0000-0002-1223-9161], El-Ashwah, Shaimaa [0000-0003-2210-1534], Çolak, Gökçe Melis [0000-0002-7662-7454], Dragonetti, Giulia [0000-0003-1775-6333], Rinaldi, Amelia [0000-0002-8211-5076], Ramón, Cristina de [0000-0002-8167-6410], Cornely, Oliver A. [0000-0001-9599-3137], Institut Català de la Salut, [Pagano L] Hematology, Fondazione Policlinico Universitario Agostino Gemelli - IRCCS – Università Cattolica del Sacro Cuore, Rome, Italy. Università Cattolica del Sacro Cuore, Rome, Italy. [Salmanton-García J] Department I of Internal Medicine, Center for Integrated Oncology Aachen Bonn Cologne Duesseldorf, Excellence Center for Medical Mycology (ECMM), University of Cologne, Faculty of Medicine and University Hospital Cologne, Cologne, Germany. Cologne Excellence Cluster On Cellular Stress Responses in Aging Associated Diseases (CECAD), University of Cologne, Faculty of Medicine and University Hospital Cologne, Cologne, Germany. [Marchesi F] Hematology and Stem Cell Transplant Unit, IRCCS Regina Elena National Cancer Institute, Rome, Italy. [Busca A] Stem Cell Transplant Center, AOU Citta’ Della Salute E Della Scienza, Turin, Italy. [Corradini P] University of Milan and Fondazione IRCCS Istituto Nazionale Dei Tumori, Milan, Italy. [Hoenigl M] Division of Infectious Diseases and Global Public Health, Department of Medicine, University of California San Diego, San Diego, CA, USA. Clinical and Translational Fungal Working Group, University of California San Diego, La Jolla, CA, USA. Section of Infectious Diseases and Tropical Medicine, Department of Internal Medicine, Medical University of Graz, Graz, Austria. [Cabirta A, Izuzquiza M] Servei d’Hematologia, Vall d’Hebron Hospital Universitari, Barcelona, Spain. Experimental Hematology, Vall d’Hebron Institute of Oncology (VHIO), Barcelona, Spain. Departament de Medicina, Universitat Autònoma de Barcelona, Bellaterra, Spain, Vall d'Hebron Barcelona Hospital Campus, Hematology, Salmanton-García, Jon, Klimko, Nikolay, Víšek, Benjamin, Weinbergerová, Barbora, Córdoba, Raúl, Doesum, Jaap van, Čerňan, Martin, Jakšić, Ozren, Magliano, Gabriel, Kulasekararaj, Austin G., Valković, Toni, Poulsen, Christian Bjørn, Glenthøj, Andrea, Ráčil, Zdeněk, Piukovics, Klára, García-Vidal, Carolina, Principe, Maria Ilaria del, Jonge, Nick de, Ormazabal-Vélez, Irati, Antić, Darko, García-Sanz, Ramón, Biernat, Monika, Schönlein, Martin, Calbacho, María, Méndez, Gustavo-Adolfo, Duléry, Rémy, Žák, Pavel, Cengiz Seval, Guldane, Mayer, Jiří, López-García, Alberto, Obr, Aleš, Sciumè, Mariarita, Batinić, Josip, Çolak, Gökçe Melis, Ramón, Cristina de, and Universidad de Sevilla. Departamento de Medicina
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[SDV.MHEP.HEM] Life Sciences [q-bio]/Human health and pathology/Hematology ,Male ,Cancer Research ,MESH: Registries ,Epidemiology ,MESH: Hospitalization ,Hematological malignancies ,Otros calificadores::Otros calificadores::/complicaciones [Otros calificadores] ,MESH: Aged, 80 and over ,MESH: Risk Factors ,[SDV.MHEP.MI]Life Sciences [q-bio]/Human health and pathology/Infectious diseases ,Risk Factors ,Malalties - Factors de risc ,Risk of mortality ,Medicine and Health Sciences ,virosis::infecciones por virus ARN::infecciones por Nidovirales::infecciones por Coronaviridae::infecciones por Coronavirus [ENFERMEDADES] ,80 and over ,Medicine ,MESH: COVID-19 ,Registries ,Sang - Malalties - Complicacions ,RC254-282 ,Cause of death ,BIOMEDICINA I ZDRAVSTVO. Kliničke medicinske znanosti. Interna medicina ,MESH: Aged ,Aged, 80 and over ,Hematology ,MESH: Middle Aged ,Mortality rate ,Neoplasms. Tumors. Oncology. Including cancer and carcinogens ,Myeloid leukemia ,Virus Diseases::RNA Virus Infections::Nidovirales Infections::Coronaviridae Infections::Coronavirus Infections [DISEASES] ,[SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology ,Middle Aged ,CANCER ,Europe ,Hospitalization ,Intensive Care Units ,Oncology ,MESH: Young Adult ,Hematologic Neoplasms ,[SDV.MHEP.MI] Life Sciences [q-bio]/Human health and pathology/Infectious diseases ,Female ,BIOMEDICINA I ZDRAVSTVO. Kliničke medicinske znanosti. Infektologija ,Life Sciences & Biomedicine ,Adult ,medicine.medical_specialty ,[SDV.CAN]Life Sciences [q-bio]/Cancer ,COVID-19 ,EHA ,Pandemic ,Aged ,Humans ,SARS-CoV-2 ,Young Adult ,técnicas de investigación::métodos epidemiológicos::estadística como asunto::probabilidad::riesgo::factores de riesgo [TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS] ,[SDV.CAN] Life Sciences [q-bio]/Cancer ,Intensive care ,Internal medicine ,Diseases of the blood and blood-forming organs ,MESH: SARS-CoV-2 ,neoplasias::neoplasias por localización::neoplasias hematológicas [ENFERMEDADES] ,Molecular Biology ,BIOMEDICINE AND HEALTHCARE. Clinical Medical Sciences. Internal Medicine ,pandemic ,hematological malignancies ,epidemiology ,MESH: Humans ,Science & Technology ,business.industry ,Myelodysplastic syndromes ,Research ,MESH: Adult ,Neoplasms::Neoplasms by Site::Hematologic Neoplasms [DISEASES] ,medicine.disease ,BIOMEDICINE AND HEALTHCARE. Clinical Medical Sciences. Infectology ,Settore MED/15 ,MESH: Male ,Settore MED/15 - MALATTIE DEL SANGUE ,[SDV.SPEE] Life Sciences [q-bio]/Santé publique et épidémiologie ,COVID-19 (Malaltia) - Diagnòstic ,MESH: Intensive Care Units ,[SDV.SPEE]Life Sciences [q-bio]/Santé publique et épidémiologie ,Investigative Techniques::Epidemiologic Methods::Statistics as Topic::Probability::Risk::Risk Factors [ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT] ,MESH: Europe ,RC633-647.5 ,business ,MESH: Female ,Other subheadings::Other subheadings::/complications [Other subheadings] ,MESH: Hematologic Neoplasms - Abstract
Background Patients with hematological malignancies (HM) are at high risk of mortality from SARS-CoV-2 disease 2019 (COVID-19). A better understanding of risk factors for adverse outcomes may improve clinical management in these patients. We therefore studied baseline characteristics of HM patients developing COVID-19 and analyzed predictors of mortality. Methods The survey was supported by the Scientific Working Group Infection in Hematology of the European Hematology Association (EHA). Eligible for the analysis were adult patients with HM and laboratory-confirmed COVID-19 observed between March and December 2020. Results The study sample includes 3801 cases, represented by lymphoproliferative (mainly non-Hodgkin lymphoma n = 1084, myeloma n = 684 and chronic lymphoid leukemia n = 474) and myeloproliferative malignancies (mainly acute myeloid leukemia n = 497 and myelodysplastic syndromes n = 279). Severe/critical COVID-19 was observed in 63.8% of patients (n = 2425). Overall, 2778 (73.1%) of the patients were hospitalized, 689 (18.1%) of whom were admitted to intensive care units (ICUs). Overall, 1185 patients (31.2%) died. The primary cause of death was COVID-19 in 688 patients (58.1%), HM in 173 patients (14.6%), and a combination of both COVID-19 and progressing HM in 155 patients (13.1%). Highest mortality was observed in acute myeloid leukemia (199/497, 40%) and myelodysplastic syndromes (118/279, 42.3%). The mortality rate significantly decreased between the first COVID-19 wave (March–May 2020) and the second wave (October–December 2020) (581/1427, 40.7% vs. 439/1773, 24.8%, p value, EPICOVIDEHA has received funds from Optics COMMITTM (COVID-19 Unmet Medical Needs and Associated Research Extension) COVID-19 RFP program by GILEAD Science, United States (Project 2020-8223).
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- 2021
35. Genotypes and haplotypes in the 3′ untranslated region of the HLA-G gene and their association with clinical outcome of hematopoietic stem cell transplantation for beta-thalassemia
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Antonio Amoroso, Benedetta Mazzi, Federico Sizzano, M Torchio, C Pultrone, Fabio Ciceri, Maria Troiano, Robert Chiesa, Sarah Marktel, Katharina Fleischhauer, M. G. Roncarolo, Laura Zito, Javid Gaziev, Roberto Crocchiolo, Silvia Gregori, Guido Lucarelli, Manuela Testi, G Turchiano, Pietro Sodani, Marco Andreani, Sizzano, F, Testi, M, Zito, L, Crocchiolo, R, Troiano, M, Mazzi, B, Turchiano, G, Torchio, M, Pultrone, C, Gregori, S, Chiesa, R, Gaziev, J, Sodani, P, Marktel, S, Amoroso, A, Roncarolo, MARIA GRAZIA, Lucarelli, G, Ciceri, Fabio, Andreani, M, and Fleischhauer, K.
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Adult ,Male ,Linkage disequilibrium ,Adolescent ,Genotype ,medicine.medical_treatment ,Immunology ,Graft vs Host Disease ,Single-nucleotide polymorphism ,Hematopoietic stem cell transplantation ,Human leukocyte antigen ,Biology ,Biochemistry ,Linkage Disequilibrium ,Immune Tolerance ,Genetics ,medicine ,Humans ,Transplantation, Homologous ,Immunology and Allergy ,Child ,3' Untranslated Regions ,Sequence Deletion ,HLA-G Antigens ,Polymorphism, Genetic ,Siblings ,beta-Thalassemia ,Haplotype ,Hematopoietic Stem Cell Transplantation ,Beta thalassemia ,General Medicine ,medicine.disease ,Transplantation ,Mutagenesis, Insertional ,Treatment Outcome ,Haplotypes ,Italy ,Case-Control Studies ,Child, Preschool ,Female - Abstract
Polymorphisms in the 3' untranslated region (3'UTR) of HLA-G, an important player in immunological tolerance, could be involved in post-transcriptional expression control, and their association with different clinical immune-related conditions including autoimmunity and transplantation is of mounting interest. Most studies have focused on a 14 base pair (bp) insertion/deletion (ins/del), while additional single-nucleotide polymorphisms (SNPs) in the HLA-G 3'UTR have been described but not extensively investigated for their clinical relevance. Here we have comparatively studied the association between 3'UTR haplotypes of HLA-G, or the 14 bp ins/del, with clinical outcome of HLA-identical sibling hematopoietic stem cell transplantation (HSCT) in 147 Middle Eastern beta-thalassemia patients. Sequence based typing of 3'UTR HLA-G polymorphisms in the patients and in 102 healthy Italian blood donors showed strong linkage disequilibrium between the 14 bp ins/del and five 3'UTR SNPs, which together could be arranged into eight distinct haplotypes based on expectation-maximization studies, with four predominant haplotypes (UTRs1-4). After HSCT, we found a moderate though not significant association between the presence of UTR-2 in double dose and protection from acute graft versus host disease (hazard ratio (HR) 0.45, 95% confidence intervals (CI): 0.14-1.45; P = 0.18), an effect that was also seen when the corresponding 14 bp ins/ins genotype was considered alone (HR 0.42, 95% CI: 0.16-1.06; P = 0.07). No association was found with rejection or survival. Taken together, our data show that there is no apparent added value of considering entire 3'UTR HLA-G haplotypes for risk prediction after allogeneic HSCT for beta-thalassemia.
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- 2012
36. Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease
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Daniela Fraboni, Andrea Bontadini, Marco Andreani, Pier Luigi Tazzari, F Agostini, Manuela Testi, Francesca Ricci, Lidia De Felice, Pietro Sodani, R. Condello, Guido Lucarelli, Maria Troiano, Mariarosa Battarra, Giuliana Ferrari, Javid Gaziev, Andreani, M, Testi, M, Gaziev, J, Condello, R, Bontadini, A, Tazzari, Pl, Ricci, F, DE FELICE, L, Agostini, F, Fraboni, D, Ferrari, Giuliana, Battarra, M, Troiano, M, Sodani, P, and Lucarelli, G.
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Adult ,Male ,Erythrocytes ,Adolescent ,medicine.medical_treatment ,Hematopoietic stem cell transplantation ,Anemia, Sickle Cell ,Chimerism ,Blood cell ,Young Adult ,Nucleated cell ,medicine ,Humans ,Child ,Editorial and Perspectives ,Bone Marrow Transplantation ,Cell Nucleus ,business.industry ,Graft Survival ,beta-Thalassemia ,Hematology ,Tissue Donors ,Transplantation ,Hemoglobinopathies ,Red blood cell ,Haematopoiesis ,medicine.anatomical_structure ,Child, Preschool ,Immunology ,Original Article ,Female ,Bone marrow ,Stem cell ,business - Abstract
Background. Persistent mixed chimerism represents a state wherein recipient and donor cells stably co-exist after haematopoietic stem cell transplantation. However, since in mostly of the studies reported in literature the engraftment state was observed in the nucleated cells, in this paper we determined the donor origin in the mature erythrocytes of patients with persistent mixed chimerism after transplantation for haemoglobinopathies. Results were compared with the engraftment state observed in singularly picked-up burst-forming unit-erythroid colonies and in the nucleated cells collected from the peripheral blood and from the marrow. Design and Methods. The donor origin of the erythrocytes was determined analyzing differences on the surface antigens of the erythrocytes suspension after incubation with anti-ABO and/or anti-C, -c, -D, -E and -e monoclonal antibodies by a flow cytometer. Short tandem repeats analysis was used to determine the donor origin of nucleated cells and burst-forming unit-erythroid colonies singularly picked up after 14 days incubation. Results. A proportion of donor-derived nucleated cells of 71%, 46%, 15% and 25% was observed at day 1364, 1385, 1314 and 932 respectively, in four transplanted patients affected by haemoglobinopathies. Similar results were also obtained in the erythroid precursors, analyzing the donor/recipient origin of the burst-forming unit-erythroid colonies, while on the contrary, at the same days of observation, a proportion of 100%, 100%, 73% and 90% donor-derived erythrocytes was observed in the four patients with persistent mixed chimerism. Conclusions. Our results showed that mostly of the erythrocytes present in four long-term transplanted patients affected by haemoglobinopathies and characterized by the presence of few donor engrafted nucleated cells were of donor origin. The indication that small proportions of donor engrafted cells might be sufficient to clinical control the disease in patients affected by haemoglobinopathies is relevant, although the biological mechanisms underlying these observations need to be further investigated.
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- 2010
37. Second hematopoietic SCT in patients with thalassemia recurrence following rejection of the first graft
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Paola Polchi, Javid Gaziev, C Alfieri, Maria Grazia Roncarolo, Katia Paciaroni, G De Angelis, Aldo Montuoro, Fabio Ciceri, Alessandro Lanti, Cristiano Gallucci, Antonella Isgrò, G. Lucarelli, Maria Domenica Simone, Pietro Sodani, Andrea Roveda, Sarah Marktel, Marco Marziali, Gaziev, J, Sodani, P, Lucarelli, G, Polchi, P, Marktel, S, Paciaroni, K, Marziali, M, Isgro, A, Simone, Md, Roveda, A, Montuoro, A, Lanti, A, Alfieri, C, De Angelis, G, Gallucci, C, Ciceri, Fabio, and Roncarolo, MARIA GRAZIA
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Adult ,Graft Rejection ,Male ,medicine.medical_specialty ,Time Factors ,Transplantation Conditioning ,Adolescent ,Thalassemia ,ThioTEPA ,Disease-Free Survival ,Recurrence ,Medicine ,Humans ,Prospective Studies ,Child ,Preparative Regimen ,Transplantation ,Thymoglobulin ,business.industry ,Incidence (epidemiology) ,Siblings ,Graft Survival ,Hematology ,medicine.disease ,Surgery ,Survival Rate ,surgical procedures, operative ,medicine.anatomical_structure ,Hemoglobinopathy ,Child, Preschool ,Female ,Bone marrow ,business ,medicine.drug - Abstract
There is a substantial incidence of graft failure in patients with thalassemia after myeloablative conditioning regimens especially in class 3 patients in whom its incidence could be as high as 8-38.5%. Most patients with graft failure have recurrence of thalassemic marrow. Historically, results of second transplants for thalassemia were poor because of a high rejection rate and/or increased TRM. Sixteen patients with thalassemia recurrence following rejection of the first graft and with a median age of 9 years (range, 4-20) were given second transplants using BM (n = 7) or PBSC (n = 9) after preparation with a new treatment protocol. All but two patients received stem cells from the same donor. The median interval between two transplants was 28 months (range, 8-204). The sustained engraftment rate was high (94%) with only one patient having primary graft failure. The probability of overall survival, event-free survival, TRM and graft failure were 79, 79, 16 and 6%, respectively. There were three transplant-related deaths. Thirteen patients are alive with Lansky/Karnofsky score of 100. This intensified treatment protocol was well tolerated with no significant increase in toxicity. The excellent results obtained with this new preparative regimen allow us to recommend it for second transplantation for patients with thalassemia recurrence.
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- 2008
38. Establishment of a Clinical Pharmacist-Led Multiple Myeloma Clinic with Collaborative Prescribing Model at the National Center for Cancer Care and Research in Qatar.
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Ghasoub R, Elazzazy S, Benkhadra M, Kassem N, Cherif H, Gaziev J, Elsabah H, Nasser S, and Hamad A
- Abstract
Background: Multiple Myeloma (MM) is a chronic and incurable hematologic malignancy that is prevalent among the elderly. Interprofessional patient care showed superiority over physician-only care in multiple settings, including MM., Objective: The primary objective of this study was to evaluate the impact of CP-led clinic and CPs interventions on MM patient care., Practice Description: Real-world analysis of ambulatory patients with MM showed that clinical pharmacists (CPs) were central to the optimization of therapy and adherence to treatment schedules and supportive medications., Practice Innovation: The CP-led MM Clinic was established with a collaborative prescribing agreement (CPA) in 2022 at the National Center for Cancer Care and Research (NCCCR) in Qatar and was the first of its kind in the MENA region. This CPA allowed CPs to issue refills for supportive medications and order required laboratory tests., Evaluation Methods: Data collected included the number of CP interventions, refills ordered by CPs, documentation of patient education, and medication reconciliations. The data were retrospectively collected and analyzed comparing ambulatory patients with MM treated before (2021) to those treated after the clinic implementation in 2022., Results: The study population comprised 20 patients. A higher number of CPs interventions were documented post-clinic than pre-clinic (343 vs. 76, P=0.004), with earlier initiation of bisphosphonate post-clinic (25 vs. 206 days, P = 0.008). There were also significant improvements in the introduction of risk appropriate venous thromboembolism (VTE) prophylaxis (43% vs. 6%, P=0.001) as well as vitamin D and calcium supplementation (100% vs. 68%, P=0.02) post-clinic. Twenty-two medication refills for supportive medications and eight pre-chemotherapy laboratory investigations were ordered by CPs., Conclusion: The CP-led clinic provided a timely link to care optimization for ambulatory MM patients. This innovative CPA model implemented in the clinic could potentially be applied to different cancer settings to optimize safe and effective patient care., (Copyright © 2024. Published by Elsevier Inc.)
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- 2024
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39. Strategic priorities for hematopoietic stem cell transplantation in the EMRO region.
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Ahmed SO, El Fakih R, Elhaddad A, Hamidieh AA, Altbakhi A, Chaudhry QU, Bazarbachi A, Adil S, Al-Khabori M, Ben Othman T, Gaziev J, Khalaf M, Alshammeri S, Alotaibi S, Alshahrani M, Bekadja MA, Ibrahim A, Al-Wahadneh AM, Altarshi M, Alsaeed A, Madani A, Abboud M, Abujazar H, Bakr M, Abosoudah I, El Cheikh J, Almasari A, Alfraih F, Baldomero H, Elsolh H, Niederwieser D, Chaudhri N, and Aljurf M
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- Humans, Bone Marrow Transplantation, Transplantation, Homologous, Mediterranean Region, Europe, Hematopoietic Stem Cell Transplantation
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The World Health Organization-designated Eastern Mediterranean region (EMRO) consists of 22 countries in North Africa and Western Asia with a collective population of over 679 million. The area comprises some of the wealthiest countries per capita income and some of the poorest. The population structure is also unique and contrasts with western countries, with a much younger population. The region sits in the heart of the thalassemia belt. Many countries have a significant prevalence of sickle cell disease, and cancer is on the rise in the region. Therefore, the strategic priorities for the growth and development of hematopoietic stem cell transplantation (HSCT) differ from country to country based on resources, healthcare challenges, and prevalent infrastructure. Thirty-one reporting teams to the Eastern Mediterranean Blood and Marrow Transplantation Group have active HSCT programs in 12 countries; allogeneic transplants outnumber autologous transplants, and the proportion of allotransplants for non-malignant conditions is higher in the EMRO region than in Western Europe and North America. The vast majority (99%) of allotransplants are from matched related donors. Matched unrelated donors and other alternate donor transplants are underutilized. The chance of finding a matched related donor for allografts is higher, with a significant chance of finding matched donors among non-sibling related donors. Reasons for relatively lower rates of transplants compared with other countries are multifactorial. Capacity building, development of newer centers, innovative funding, and better utilization of information technology are required to make transplantation as an accessible modality to more patients. Cost-effectiveness and cost-containment, regulation, and ensuring quality will all be priorities in planning HSCT development in the region.
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- 2023
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40. Applying value-based strategies to accelerate access to novel cancer medications: guidance from the Oncology Health Economics Expert Panel in Qatar (Q-OHEP).
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Hamad A, Elazzazy S, Bujassoum S, Rasul K, Gaziev J, Cherif H, Al-Boloshi Z, Hanssens Y, Saleh A, Rasheed HA, Al-Badriyeh D, Babiker A, Hmaidan AA, and Al-Hail M
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- Humans, Qatar, Delivery of Health Care, Consensus, Economics, Medical, Neoplasms drug therapy
- Abstract
Background: In line with global trends, cancer incidence and mortality may have decreased for specific types of cancer in Qatar. However, the cancer-related burden on patients, healthcare systems, and the economy is expected to expand; thus, cancer remains a significant public healthcare issue in Qatar. Qatar's free access to cancer care represents a considerable economic burden. Ensuring the best utilization of financial resources in the healthcare sector is important to provide unified and fair access to cancer care for all patients. Experts from the Qatar Oncology Health Economics Expert Panel (Q-OHEP) aimed to establish a consistent and robust base for evaluating oncology/hematology medications; involve patients' insights to accelerate access to cutting-edge medications; increase the value of cancer care; and reach a consensus for using cost-effective strategies and efficient methodologies in cancer treatment., Methods: The Q-OHEP convened on 30 November 2021 for a 3-hour meeting to discuss cancer management, therapeutics, and health economics in Qatar, focusing on four domains: (1) regulatory, (2) procurement, (3) treatment, and (4) patients. Discussions, guided by a moderator, focused on a list of suggested open-ended questions., Results: Some of the salient recommendations included the development of a formal, fast-track, preliminary approval pathway for drugs needed by patients with severe disease or in critical condition; and encouraging and promoting the conduct of local clinical trials and real-world observational studies using existing registry data. The Q-OHEP also recommended implementing a forecast system using treatment center data based on the supply/demand of formulary oncology drugs to detect treatment patterns, estimate needs, expedite procurement, and prevent shortages/delays. Furthermore, the panel discussed the needs to define value concerning cancer treatment in Qatar, implement value-based models for reimbursement decision-making such as health technology assessment and multiple-criteria decision analysis, and promote patient education and involvement/feedback in developing and implementing cancer management guidelines., Conclusion: Herein, we summarize the first Q-OHEP consensus recommendations, which aim to provide a solid basis for evaluating, registering, and approving new cancer medications to accelerate patient access to novel cancer treatments in Qatar; promote/facilitate the adoption and collection of patient-reported outcomes; and implement value-based cancer care in Qatar., (© 2023. The Author(s).)
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- 2023
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41. Polyomaviruses shedding in stool of patients with hematological disorders: detection analysis and study of the non-coding control region's genetic variability.
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Prezioso C, Ciotti M, Obregon F, Ambroselli D, Rodio DM, Cudillo L, Gaziev J, Mele A, Nardi A, Favalli C, Arcese W, Palamara AT, and Pietropaolo V
- Subjects
- Adult, Child, Female, Humans, Male, Phylogeny, Polyomavirus classification, Polyomavirus genetics, Prevalence, Real-Time Polymerase Chain Reaction, Viral Load, Feces virology, Genetic Variation, Hematologic Neoplasms complications, Polyomavirus isolation & purification, Polyomavirus Infections epidemiology, Polyomavirus Infections virology, Virus Shedding
- Abstract
Fragmented data are available on the human polyomaviruses (HPyVs) prevalence in the gastrointestinal tract. Rearrangements in the non-coding control region (NCCR) of JCPyV and BKPyV have been extensively studied and correlated to clinical outcome; instead, little information is available for KIPyV, WUPyV and MCPyV NCCRs. To get insights into the role of HPyVs in the gastrointestinal tract, we investigated JCPyV, BKPyV, KIPyV, WUPyV and MCPyV distribution among hematological patients in concomitance with gastrointestinal symptoms. In addition, NCCRs and VP1 sequences were examined to characterize the strains circulating among the enrolled patients. DNA was extracted from 62 stool samples and qPCR was carried out to detect and quantify JCPyV, BKPyV, KIPyV, WUPyV and MCPyV genomes. Positive samples were subsequently amplified and sequenced for NCCR and VP1 regions. A phylogenetic tree was constructed aligning the obtained VP1 sequences to a set of reference sequences. qPCR revealed low viral loads for all HPyVs searched. Mono and co-infections were detected. A significant correlation was found between gastrointestinal complications and KIPyV infection. Archetype-like NCCRs were found for JCPyV and BKPyV, and a high degree of NCCRs stability was observed for KIPyV, WUPyV and MCPyV. Analysis of the VP1 sequences revealed a 99% identity with the VP1 reference sequences. The study adds important information on HPyVs prevalence and persistence in the gastrointestinal tract. Gastrointestinal signs were correlated with the presence of KIPyV, although definitive conclusions cannot be drawn. HPyVs NCCRs showed a high degree of sequence stability, suggesting that sequence rearrangements are rare in this anatomical site.
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- 2019
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42. One single bone marrow harvesting from donors under 3 years of age: assessing safety and efficacy of the procedure.
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Paciaroni K, Alfieri C, Isgrò A, De Angelis G, Ribersani M, Marziali M, Dauri M, Sodani P, and Gaziev J
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- Child, Preschool, Female, Humans, Male, Tissue Donors, Bone Marrow Transplantation methods, Tissue and Organ Harvesting methods
- Abstract
To candidate children as bone marrow donors raises two main concerns: donor safety and adequate marrow cell dose. Data in the field are limited and guidelines for child donor care management are lacking. In this context, we herein report the experience collected in our center by comparing very-young donors (defined as age ≤ 3 years) with young donors (defined as age > 3 years) who donated bone marrow (BM) for patients affected by beta-globin disorders.
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- 2019
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43. Radiological findings of Posterior Reversible Encephalopathy Syndrome in transplanted children previous affected by hemoglobinopathy: A neuroimaging retrospective analysis.
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Picchi E, Di Giuliano F, Marziali S, Minosse S, Ferrazzoli V, Da Ros V, Gaziev J, Pistolese CA, Floris R, and Garaci F
- Abstract
To evaluate, by Magnetic Resonance Imaging, if there is a typical pattern or severity of PRES in transplanted children for hemoglobinopathy. Secondary point was to investigate the pattern and severity of PRES in children with thalassemia-THAL and sickle-cell disease-SCD after autologous hematopoietic stem cell transplantation (aHSCT). Finally, we evaluate the presence of atypical PRES presentation and the involved area of central nervous system. Two neuroradiologists analyzed retrospectively MRI of 21 transplanted children for THAL or SCD treated with CI, with neurological symptoms and signs of PRES. The Bartynski and Boardman classification has been used for PRES pattern while McKinney scale for PRES severity. Fisher Exact Probability test or Chi-square test were used to compare the categorical data. In the 21 transplanted children the PRES severity was typically mild (85.7%) without preferring radiological pattern at MRI. The analysis didn't show significant association between PRES pattern or PRES severity and previous hemoglobinopathy (THAL or SCD). No atypical PRES presentation has been found. PRES severity in transplanted children for hemoglobinopathy is typically mild. Notwithstanding children affected by SCD have a damage on the capillary endothelium, after aHSCT our data didn't show a different PRES severity and pattern than THAL children.
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- 2019
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44. Late Effects Screening Guidelines after Hematopoietic Cell Transplantation (HCT) for Hemoglobinopathy: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT.
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Shenoy S, Gaziev J, Angelucci E, King A, Bhatia M, Smith A, Bresters D, Haight AE, Duncan CN, de la Fuente J, Dietz AC, Baker KS, Pulsipher MA, and Walters MC
- Subjects
- Adolescent, Adult, Aged, Aged, 80 and over, Female, Guidelines as Topic, Hemoglobinopathies pathology, Humans, Male, Mass Screening, Middle Aged, Young Adult, Hematopoietic Stem Cell Transplantation methods, Hemoglobinopathies therapy, Transplantation Conditioning methods, Transplantation, Homologous methods
- Abstract
Allogeneic hematopoietic cell transplantation (HCT) can halt organ damage and eliminate symptoms in hemoglobin disorders, including sickle cell disease (SCD) and thalassemia major. Managing the residual manifestations of pre-HCT disease complications and the long-term effects of HCT requires systematic monitoring, follow-up and intervention when indicated. Late complications vary with age and disease status at HCT and with transplant variables such as preparative regimen, donor source and compatibility, and immune reconstitution. An international consensus conference sponsored by the Pediatric Blood and Marrow Transplant Consortium in May 2016 entitled "Late Effects Screening and Recommendations Following HCT for Immune Deficiency and Nonmalignant Hematologic Disorders" focused on follow-up after HCT for hemoglobinopathy. An earlier publication from experts who participated in this session described the pathophysiology and spectrum of complications that HCT recipients experience after HCT for SCD and thalassemia major. This companion publication summarizes the consensus reached by this group of experts about long-term follow-up guidelines after HCT for hemoglobinopathy. In addition, these guidelines might also be included in studies of novel curative therapies such as autologous HCT after hematopoietic progenitor stem cell gene modification., (Copyright © 2018 The American Society for Blood and Marrow Transplantation. All rights reserved.)
- Published
- 2018
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45. Haploidentical HSCT for hemoglobinopathies: improved outcomes with TCRαβ + /CD19 + -depleted grafts.
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Gaziev J, Isgrò A, Sodani P, Paciaroni K, De Angelis G, Marziali M, Ribersani M, Alfieri C, Lanti A, Galluccio T, Adorno G, and Andreani M
- Subjects
- Adolescent, Antigens, CD19, Antigens, CD34, Child, Child, Preschool, Graft Rejection, Graft vs Host Disease etiology, Hematopoietic Stem Cell Transplantation adverse effects, Hemoglobinopathies complications, Hemoglobinopathies mortality, Humans, Male, Receptors, Antigen, T-Cell, alpha-beta, Retrospective Studies, Survival Analysis, Transplantation, Haploidentical adverse effects, Treatment Outcome, Graft Survival immunology, Hematopoietic Stem Cell Transplantation methods, Hemoglobinopathies therapy, Lymphocyte Depletion methods, Transplantation, Haploidentical methods
- Abstract
We examined outcomes of haploidentical hematopoietic cell transplantation (haplo-HCT) using T-cell receptor αβ
+ (TCRαβ+ )/CD19+ -depleted grafts (TCR group, 14 patients) in children with hemoglobinopathies. Patients received a preparative regimen consisting of busulfan, thiotepa, cyclophosphamide, and antithymocyte globulin preceded by fludarabine, hydroxyurea, and azathioprine. The median follow-up among surviving patients was 3.9 years. The 5-year probabilities of overall survival (OS) and disease-free survival (DFS) were 84% and 69%, respectively. The incidence of graft failure was 14%. We compared outcomes to a historical group of 40 patients with hemoglobinopathies who received CD34+ -selected grafts (CD34 group). The median follow-up of surviving patients for the CD34 group was 7.5 years. The 5-year probabilities of OS and DFS were 78% and 39%, respectively. The CD34 group had a significantly higher incidence of graft failure (45%) than the TCR group (14%) ( P = .048). The incidences of grades 2 to 4 acute graft-versus-host disease (GVHD) in the TCR and CD34 groups were 28% and 29%, respectively, and 21% and 10% ( P = .1), respectively, for extensive chronic GVHD. Viral reactivation was common in both groups. The overall incidence of posttransplant lymphoproliferative disorders for the entire group was 16%. Among all patients, 5 developed autoimmune hemolytic anemia or thrombocytopenia, with the overall cumulative incidence of 11%. The 2 groups showed suboptimal CD4+ recovery within the first 6 months of transplantation with no significant difference between groups. These data demonstrate that TCRαβ+ /CD19+ -depleted grafts are associated with a reduced incidence of graft failure, but delayed immune reconstitution and associated morbidity and mortality remain a significant challenge., (© 2018 by The American Society of Hematology.)- Published
- 2018
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46. Posterior Reversible Encephalopathy Syndrome after Hematopoietic Cell Transplantation in Children with Hemoglobinopathies.
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Gaziev J, Marziali S, Paciaroni K, Isgrò A, Di Giuliano F, Rossi G, Marziali M, De Angelis G, Alfieri C, Ribersani M, Andreani M, Palmieri MG, Placidi F, Romigi A, Izzi F, Floris R, and Mercuri NB
- Subjects
- Acute Disease, Adolescent, Anemia, Sickle Cell immunology, Anemia, Sickle Cell mortality, Anemia, Sickle Cell pathology, Calcineurin Inhibitors administration & dosage, Child, Child, Preschool, Cohort Studies, Female, Graft vs Host Disease immunology, Graft vs Host Disease mortality, Graft vs Host Disease pathology, Graft vs Host Disease prevention & control, Humans, Hypertension diagnosis, Hypertension physiopathology, Immunosuppressive Agents administration & dosage, Infant, Male, Posterior Leukoencephalopathy Syndrome chemically induced, Posterior Leukoencephalopathy Syndrome immunology, Posterior Leukoencephalopathy Syndrome mortality, Risk Factors, Seizures chemically induced, Seizures immunology, Seizures mortality, Siblings, Survival Analysis, Transplantation, Homologous, Unrelated Donors, beta-Thalassemia immunology, beta-Thalassemia mortality, beta-Thalassemia pathology, Anemia, Sickle Cell therapy, Calcineurin Inhibitors adverse effects, Hematopoietic Stem Cell Transplantation, Immunosuppressive Agents adverse effects, Posterior Leukoencephalopathy Syndrome therapy, Seizures therapy, beta-Thalassemia therapy
- Abstract
Posterior reversible encephalopathy syndrome (PRES) is a serious adverse event associated with calcineurin inhibitors used for graft-versus-host disease (GVHD) prophylaxis. We compared the incidence of PRES in children with thalassemia (n = 222, 1.4 to 17.8 years old) versus sickle cell disease (SCD; n = 59, 2 to 17 years old) who underwent hematopoietic cell transplantation from HLA-matched siblings or alternative donors and analyzed the risk factors for PRES. Overall, 31 children developed calcineurin inhibitor-related PRES (11%), including 30 patients with seizures and 1 patient without seizures. PRES incidence was significantly higher in SCD patients (22%; 95% confidence interval [CI], 10% to 32%) than in thalassemia patients (8%; 95% CI, 5% to 12%;P = .002). In multivariate analysis, factors associated with PRES were hypertension (hazard ratio [HR], 5.87; 95% CI, 2.57 to 13.43; P = .0001), SCD (HR, 2.49; 95% CI, 1.25 to 4.99; P = .009), and acute GVHD (HR 2.27; 95% CI, 1.06 to 4.85; P= .031). In the entire cohort overall survival (OS) was significantly higher in patients without versus with PRES (90% versus 77%; P = .02). In a subgroup analysis that including matched sibling transplants, OS and disease-free survival (DFS) were similar in thalassemia patients without PRES (92% and 88%, respectively) and with PRES (82% and 73%, respectively), whereas SCD patients with PRES had significantly lower OS (67%) and DFS (67%) than patients without PRES (94% and 94%, respectively; P = .008). Thus, SCD patients had a significantly higher incidence of PRES than thalassemia patients, and hypertension and GVHD were the 2 main risk factors for PRES in patients with hemoglobinopathies. Although PRES did not significantly influence survival in patients with thalassemia, patients with SCD had significantly lower survival after PRES., (Copyright © 2017 The American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.)
- Published
- 2017
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47. Spirometric Evaluation of Pulmonary Function in Nigerian Children underwent Bone Marrow Transplantation for Sickle Cell Anemia.
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Isgro' A, Marziali M, Paciaroni K, De Angelis G, Alfieri C, Ribersani M, Olowoselu FO, Lucarelli G, and Gaziev J
- Abstract
Competing Interests: Competing interests: The authors have declared that no competing interests exist.
- Published
- 2017
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48. Current Results and Future Research Priorities in Late Effects after Hematopoietic Stem Cell Transplantation for Children with Sickle Cell Disease and Thalassemia: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Stem Cell Transplantation.
- Author
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Shenoy S, Angelucci E, Arnold SD, Baker KS, Bhatia M, Bresters D, Dietz AC, De La Fuente J, Duncan C, Gaziev J, King AA, Pulsipher MA, Smith AR, and Walters MC
- Subjects
- Child, Follow-Up Studies, Hematopoietic Stem Cell Transplantation methods, Humans, Risk Factors, Time Factors, Tissue Donors supply & distribution, Transplantation Conditioning adverse effects, Transplantation Conditioning methods, Transplantation, Homologous, Anemia, Sickle Cell therapy, Hematopoietic Stem Cell Transplantation adverse effects, Thalassemia therapy
- Abstract
Sustained donor engraftment after allogeneic hematopoietic cell transplantation (HCT) converts to healthy donor hemoglobin synthesis and halts disease symptoms in patients with sickle cell disease and thalassemia major. A disease-free survival probability that exceeds 90% has been reported when HCT using an HLA-matched sibling donor is performed in young patients with low-risk disease or treatment-related risk factors. Alternate donor HCT and HCT in adults is performed infrequently because of a higher risk profile. Transplant-specific risks include conditioning regimen-related toxicity, graft-versus-host disease, graft rejection with marrow aplasia or disease recurrence, and infections associated with immunosuppression and delayed immune reconstitution. The magnitude of risk depends on patient age, clinical status of the underlying disease (eg, organ injury from vasculopathy and iron overload), donor source, and intensity of the conditioning regimen. These risks are commonly monitored and reported in the short term. Documenting very late outcomes is important, but these data are rarely reported because of challenges imposed by patient drop-out and insufficient resources. This report summarizes long-term follow-up results after HCT for hemoglobin disorders, identifies gaps in knowledge, and discusses opportunities for future investigations. This consensus summary will be followed by a second article detailing comprehensive long-term follow-up recommendations to aid in maintaining health in these individuals and identifying late complication risks that could facilitate interventions to improve outcomes., (Copyright © 2017 The American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.)
- Published
- 2017
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49. Impact of Donor-Specific anti-HLA Antibodies and Donor KIR Characteristics in Haploidentical HSCT for Beta-Thalassemia.
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Andreani M, Testi M, Sodani P, Troiano M, Di Luzio A, Testa G, Falco M, Poggi E, Gaziev J, and Piazza A
- Published
- 2017
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50. Optimal Outcomes in Young Class 3 Patients With Thalassemia Undergoing HLA-Identical Sibling Bone Marrow Transplantation.
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Gaziev J, Isgrò A, Sodani P, Marziali M, Paciaroni K, Gallucci C, De Angelis G, Andreani M, Testi M, Alfieri C, Ribersani M, Galluccio T, Battarra MR, Morrone A, and Lucarelli G
- Subjects
- Adolescent, Age Factors, Bone Marrow Transplantation adverse effects, Child, Child, Preschool, Disease-Free Survival, Drug Therapy, Combination, Female, Graft Rejection epidemiology, Graft Rejection immunology, Graft Survival, Graft vs Host Disease epidemiology, Graft vs Host Disease immunology, Histocompatibility Testing, Humans, Immunosuppressive Agents administration & dosage, Incidence, Kaplan-Meier Estimate, Male, Predictive Value of Tests, Prospective Studies, Risk Factors, Rome epidemiology, Thalassemia diagnosis, Thalassemia genetics, Thalassemia immunology, Time Factors, Transplantation Conditioning, Treatment Outcome, Bone Marrow Transplantation methods, HLA Antigens immunology, Histocompatibility, Living Donors, Siblings, Thalassemia surgery
- Abstract
Background: Bone marrow transplantation (BMT) for class 3 patients with thalassemia is challenging due to high rates of graft rejection and transplant-related mortality. Since the first studies of BMT in the late 1980s, a number of conditioning regimens have been designed to improve outcomes, but with suboptimal results. Here we report the outcome of transplantation in class 3 patients using a modified protocol., Methods: Sixty-three patients between 5 and 16.7 years of age with class 3 thalassemia received HLA-matched sibling BMT following either the original protocol (26 patients) or the modified protocol (37 patients). Both regimens comprised preconditioning cytoreduction with hydroxyurea and azathioprine starting at -45 days pretransplant, and fludarabine from days -16 to -12. Conditioning was performed with busulfan and cyclophosphamide (original protocol) or with busulfan, thiotepa, and cyclophosphamide (modified protocol)., Results: The 2 groups showed similar patient demographics. At day 0, the degree of cytoreduction (lymphopenia, neuthropenia, and thrombocytopenia) achieved by the modified protocol was greater than the original protocol. The incidence of graft failure/rejection was significantly higher in the original group (15%; 95% confidence interval [95% CI], 5-32%) compared with the modified group (0%) (P = 0.014). The respective 5-year thalassemia-free survival rates were 73% (95% CI, 51-86%) and 92% (95% CI, 77-97%) (P = 0.047). Both groups showed similar incidences of grades II to IV acute graft-versus host disease. Modified protocol did not increase nonhematological toxicity or infectious complications., Conclusions: The modified treatment protocol effectively and safely prevented graft failure/rejection and significantly increased thalassemia-free survival of class 3 patients with thalassemia.
- Published
- 2016
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