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1. ELTROMBOPAG ADDED TO STANDARD IMMUNOSUPPRESSION IMPROVES RESPONSE RATE IN SEVERE APLASTIC ANEMIA: RESULTS OF THE MULTICENTER PHASE III PROSPECTIVE RANDOMIZED RACE TRIAL

Author

Risitano, AM, Kulasekararaj, A, Iacobelli, S, Terwel, S, Hill, A, Halkes, CJM, Recher, C, Barraco, F, Forcade, E, Llamas, JCV, Drexler, B, Mear, JB, Van Lint, MT, Raymakers, RAP, De Groot, MR, Daguindau, E, Nur, E, Barcellini, W, Russell, NH, Terriou, L, Iori, AP, Sanchez-Ortega, I, Xicoy, B, Jarque, I, Cavenagh, J, De Fontbrune, FS, Marotta, S, Munir, T, Tjon, JML, Tavitian, S, Praire, A, Clement, L, Rabian, F, Smith, AE, Cook, R, Marano, L, Griffin, M, Palmisani, E, Muus, P, Cacace, F, Frieri, C, Passweg, JR, Marsh, JCW, Socie, G, Mufti, GJ, Dufour, C, and de Latour, RP

Published
2021

3. Results of the Ebmt Saawp Phase III Prospective Randomized Multicenter Race Study of Horse Atg and Ciclosporin with or Without Eltrombopag in Naive Saa Patients

Author

de Latour, RP, Marsh, J, Iacobelli, S, Terwel, S, Hill, A, Halkes, CJM, Recher, C, Barraco, F, Forcade, E, Llamas, JCV, Dresler, B, Mear, JB, Van Lint, MT, Raymakers, RAP, De Groot, MR, Daguindau, E, Nur, E, Barcellini, W, Russell, NH, Terriou, L, Iori, AP, Sanchez-Ortega, I, Xicoy, B, Jarque, I, Cavenagh, J, de Fontbrune, FS, Kulasekararaj, A, Marotta, S, Munir, T, Tjon, JML, Tavitian, S, Praire, A, Clement, L, Rabian, F, Smith, AE, Cook, R, Marano, L, Griffin, M, Palmisani, E, Muus, P, Cacace, F, Passweg, JR, Socie, G, Mufti, GJ, Dufour, C, and Risitano, A

Published
2020

4. allogenic hemopoietic stem cell transplant in patients with acute myeloid leukemia prepared with busulfan and fludarabine or thiotepa busulfan and fludarabine a retrospective study

Author

Sora, F, Di Grazia, C, Chiusolo, Patrizia, Raiola, Am, Bregante, S, Mordini, N, Olivieri, A, Iori, Ap, Patriarca, F, Grisariu, S, Terruzzi, E, Rambaldi, A, Sica, Simona, Bruno, B, Angelucci, E, Bacigalupo, A, Chiusolo, P (ORCID:0000-0002-1355-1587), Sica, S (ORCID:0000-0003-2426-3465), Sora, F, Di Grazia, C, Chiusolo, Patrizia, Raiola, Am, Bregante, S, Mordini, N, Olivieri, A, Iori, Ap, Patriarca, F, Grisariu, S, Terruzzi, E, Rambaldi, A, Sica, Simona, Bruno, B, Angelucci, E, Bacigalupo, A, Chiusolo, P (ORCID:0000-0002-1355-1587), and Sica, S (ORCID:0000-0003-2426-3465)

Abstract

his is a multicenter retrospective comparison of 2 myeloablative conditioning regimens in 454 patients with acute myeloid leukemia (AML) in remission: busulfan (4 days) and fludarabine (BUFLU) versus thiotepa, busulfan, and fludarabine (TBF). Eligible for this study were patients allografted between January 2008 and December 2018 in 10 transplant centers, with AML in first or second remission: 201 patients received BUFLU, whereas 253 received TBF. The 2 groups (BUFLU and TBF) were comparable for age (P = .13) and adverse AML risk factors (P = .3). The TBF group had more second remissions and more haploidentical grafts. The donor type included HLA-identical siblings, unrelated donors, and family haploidentical donors. The 5-year cumulative incidence of nonrelapse mortality (NRM) was 19% for BUFLU and 22% for TBF (P = .8), and the 5-year cumulative incidence of relapse was 30% and 15%, respectively (P = .0004). The 5-year actuarial survival was 51% for BUFLU and 68% for TBF (P = .002). In a multivariate Cox analysis, after correcting for confounding factors, the use of TBF reduced the risk of relapse compared with BUFLU (P = .03) and the risk of death (P = .03). In a matched pair analysis of 108 BUFLU patients matched with 108 TBF patients, with the exclusion of haploidentical grafts, TBF reduced the risk of relapse (P = .006) and there was a trend for improved survival (P = .07). Superior survival of patients receiving TBF as compared with BUFLU is due to a reduced risk of relapse, with comparable NRM. The survival advantage is independent of donor type and AML risk factors.

Published
2020

9. Incidence, Risk Factors and Outcome of Pre-engraftment Gram-Negative Bacteremia After Allogeneic and Autologous Hematopoietic Stem Cell Transplantation: An Italian Prospective Multicenter Survey

Author

Girmenia, C, Bertaina, A, Piciocchi, A, Perruccio, K, Algarotti, A, Busca, A, Cattaneo, C, Raiola, Am, Guidi, S, Iori, Ap, Candoni, A, Irrera, G, Milone, G, Marcacci, G, Scimè, R, Musso, M, Cudillo, L, Sica, S, Castagna, L, Corradini, P, Marchesi, F, Pastore, D, Alessandrino, Ep, Annaloro, C, Ciceri, F, Santarone, S, Nassi, L, Farina, C, Viscoli, C, Rossolini, Gm, Bonifazi, F, Rambaldi, A, Capria, S, Mastronuzzi, A, Pagliara, D, Bernaschi, P, Amico, L, Carotti, A, Mencacci, A, Bruno, B, Costa, C, Passi, A, Ravizzola, G, Angelucci, E, Marchese, A, Pecile, P, Ventura, G, Fanin, R, Scarparo, C, Barbaro, A, Leotta, S, Marchese, Ae, Becchimanzi, C, Donnarumma, D, Tringali, S, Baldi, Mt, Scalone, R, Picardi, A, Arcese, W, Fontana, C, Giammarco, S, Spanu, T, Crocchiolo, R, Casari, E, Mussetti, A, Conte, E, Ensoli, F, Miragliotta, G, Marone, P, Arghittu, M, Greco, R, Forcina, A, Chichero, P, Di Bartolomeo, P, Fazii, P, Kroumova, V, Decembrino, N, Zecca, M, Pisapia, G, Palazzo, G, Lanino, E, Faraci, M, Castagnola, E, Bandettini, R, Pastano, R, Sammassimo, S, Passerini, R, Stefani, Pm, Gherlinzoni, F, Rigoli, R, Prezioso, L, Cambò, B, Calderaro, A, Carella, Am, Cascavilla, N, Labonia, Mt, Celeghini, I, Mordini, N, Piana, F, Vacca, A, Sanna, M, Podda, G, Corsetti, Mt, Rocchetti, A, Cilloni, D, De Gobbi, M, Bianco, O, Fagioli, F, Carraro, F, De Intinis, G, Severino, A, Proia, A, Parisi, G, Vallisa, D, Confalonieri, M, Russo, D, Malagola, M, Galieni, P, Falcioni, S, Travaglini, V, Raimondi, R, Borghero, C, Pavan, G, Prete, A, Belotti, T, Ambretti, S, Imola, M, Mianulli, Am, Pedna, Mf, Cesaro, S, Lo Cascio, G, Ferrari, A, Piedimonte, M, Santino, I, Calandrelli, M, Olivieri, A, Orecchioni, F, Mirabile, M, Centurioni, R, Gironacci, L, Caravelli, D, Gallo, S, De Filippi, M, Cupelli, L, Dentamaro, T, Falco, S, Eugenio, Os, Marotta, S, Risitano, A, Lula, D, Musto, P, Pietrantuono, G, Traficante, A, Cerchiara, E, Tirindelli, Mc, Dicuonzo, G, Chierichini, A, Anaclerico, B, and Placanica, P.

Published
2017

10. Polymorphism of the complement receptor 1 gene correlates with hematological response to eculizumab in patients with paroxysmal nocturnal hemoglobinuria

Author

Rondelli T, Peffault de Latour R, Sica M, Peruzzi B, Ricci P, Barcellini W, Iori AP, Boschetti C, Valle V, Frémeaux Bacchi V, De Angioletti M, Socié G, Luzzatto L, Notaro R., RISITANO, ANTONIO MARIA, Rondelli, T, Risitano, ANTONIO MARIA, Peffault de Latour, R, Sica, M, Peruzzi, B, Ricci, P, Barcellini, W, Iori, Ap, Boschetti, C, Valle, V, Frémeaux Bacchi, V, De Angioletti, M, Socié, G, Luzzatto, L, and Notaro, R.

Published
2014

11. Killer Cell Immunoglobulin-Like Receptor Ligand Matching and Outcomes after Unrelated Cord Blood Transplantation in Acute Myeloid Leukemia

Author

Rocha, V, Ruggeri, A, Spellman, S, Wang, T, Sobecks, R, Locatelli, F, Askar, M, Michel, G, Arcese, W, Iori, AP, Purtill, D, Danby, R, Sanz, GF, Gluckman, E, Eapen, M, Netcord, and Ctr Int Blood Marrow Transplant Re

Subjects
Acute leukemia, immune system diseases, Unrelated transplant, otorhinolaryngologic diseases, chemical and pharmacologic phenomena, Cord blood, KIR ligand
Abstract

The effect of killer cell immunoglobulin-like receptor (KIR)-ligand matching on outcomes after unrelated cord blood (CB) transplantation was studied in 461 patients with acute myeloid leukemia, categorizing KIR ligand for HLA-C groups C1 and C2 and Bw4. Donor-recipient HLA matching considered allele-level matching at HLA-A, -B, -C, and -DRB1. Separate analyses were conducted for 6-7/8 HLA-matched and 3-5/8 HLA-matched transplants because HLA matching confounded KIR-ligand matching (ie, KIR-ligand mismatching was less likely with better HLA matching). All patients received single CB unit and myeloablative conditioning. There were no significant differences in nonrelapse mortality (NRM), relapse, and overall mortality by KIR-ligand match status. However, among recipients of 3-5/8 HLA-matched transplants, NRM (HR, 2.26; P =.008) and overall mortality (HR, 1.78; P =.008) but not relapse were higher with KIR-ligand mismatched (host-versus-graft direction) compared with KIR-ligand matched transplants. These data do not support selecting CB units based on KIR-ligand match status for transplants mismatched at 1 or 2 HLA loci. Although transplants mismatched at 3 or more HLA loci are not recommended, avoiding KIR-ligand mismatching in this setting lowers mortality risks. (C) 2016 American Society for Blood and Marrow Transplantation.

Published
2016

12. Infections by carbapenem-resistant Klebsiella pneumoniae in SCT recipients: a nationwide retrospective survey from Italy

Author

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

13. Engraftment kinetics and graft failure after single umbilical cord blood transplantation using a myeloablative conditioning regimen

Author

Ruggeri A, Labopin M, Sormani MP, Sanz G, Sanz J, Volt F, Michel G, Locatelli F, Diaz De Heredia C, O'Brien T, Arcese W, Iori AP, Querol S, Kogler G, Lecchi L, Pouthier F, Garnier F, Navarrete C, Baudoux E, Fernandes J, Kenzey C, Eapen M, Gluckman E, Rocha V, Saccardi R, Eurocord, Cord Blood Committee EBMT, and Netcord

Subjects
surgical procedures, operative
Abstract

Umbilical cord blood transplant recipients are exposed to an increased risk of graft failure, a complication leading to a higher rate of transplant-related mortality. The decision and timing to offer a second transplant after graft failure is challenging. With the aim of addressing this issue, we analyzed engraftment kinetics and outcomes of 1268 patients (73% children) with acute leukemia (64% acute lymphoblastic leukemia, 36% acute myeloid leukemia) in remission who underwent single-unit umbilical cord blood transplantation after a myeloablative conditioning regimen. The median follow-up was 31 months. The overall survival rate at 3 years was 47%; the 100-day cumulative incidence of transplant-related mortality was 16%. Longer time to engraftment was associated with increased transplant-related mortality and shorter overall survival. The cumulative incidence of neutrophil engraftment at day 60 was 86%, while the median time to achieve engraftment was 24 days. Probability density analysis showed that the likelihood of engraftment after umbilical cord blood transplantation increased after day 10, peaked on day 21 and slowly decreased to 21% by day 31. Beyond day 31, the probability of engraftment dropped rapidly, and the residual probability of engrafting after day 42 was 5%. Graft failure was reported in 166 patients, and 66 of them received a second graft (allogeneic, n=45). Rescue actions, such as the search for another graft, should be considered starting after day 21. A diagnosis of graft failure can be established in patients who have not achieved neutrophil recovery by day 42. Moreover, subsequent transplants should not be postponed after day 42.

Published
2014

15. Aplasie midollari acquisite in età pediatrica: Raccomandazioni diagnostico-terapeutiche [Acquired aplastic anaemia in children: Diagnostic-therapeutic recommendations]

Author

Barone, A, Lucarelli, A, Onofrillo, D, Verzegnassi, F, Bonanomi, S, Cesaro, S, Cugno, C, Fioredda, F, Iori, Ap, Ladogana, S, Locasciulli, A, Longoni, D, Lanciotti, M, Macaluso, A, Mandaglio, R, Marra, N, Martire, B, Maruzzi, M, Menna, G, Notarangelo, Ld, Palazzi, G, Pillon, M, Ramenghi, U, Russo, Giovanna, Svahn, J, Timeus, F, Tucci, F, Zecca, M, Farruggia, P, Dufour, C, and Saracco, P.

Published
2014

16. Impact of HLA mismatch direction on outcomes after umbilical cord blood transplantation for hematological malignant disorders: a retrospective Eurocord-EBMT analysis

Author

Cunha, R, Loiseau, P, Ruggeri, A, Sanz, G, Michel, G, Iori, Ap, Paolaiori, A, Socié, G, Arcese, W, Picardi, A, Dias de Heredia, C, Rio, B, Locatelli, F, O'Brien, Ta, Yakoub-Agha, I, Angel Diaz, M, Milpied, N, Bittencourt, H, Pedro Souza, M, Aljurf, M, Charron, D, Boudjedir, K, Labopin, M, Gluckman, E, and Rocha, V

Subjects
Oncology, Male, Cord blood transplantation, Neutrophils, Graft vs Host Disease, Histocompatibility Testing, HLA Antigens, Surveys and Questionnaires, Child, HLA mismatches direction, education.field_of_study, Homozygote, Hematology, Middle Aged, Treatment Outcome, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Cord blood, Child, Preschool, Hematologic Neoplasms, Histocompatibility, Female, Cord Blood Stem Cell Transplantation, Adult, Blood Platelets, medicine.medical_specialty, Adolescent, Population, cord blood transplantation, Human leukocyte antigen, Disease-Free Survival, Young Adult, Internal medicine, medicine, Settore MED/05 - Patologia Clinica, Humans, education, Preschool, malignant diseases, Aged, Retrospective Studies, Malignant diseases, Transplantation, Umbilical Cord Blood Transplantation, business.industry, Infant, Multivariate Analysis, medicine.disease, Settore MED/15, HLA Mismatch, Graft-versus-host disease, Immunology, business, Settore MED/15 - Malattie del Sangue
Abstract

HLA matching is a critical determinant of outcomes for patients who have undergone umbilical cord blood transplantation (UCBT). Data have been published on the importance of donor/recipient HLA mismatch direction on UCBT outcomes. HLA mismatch in the graft-versus-host (GVH) direction is defined as a donor homozygous at an HLA locus, while the recipient shares one HLA Ag with the donor. HLA mismatch in the host-versus-graft (HVG) direction is defined as a recipient homozygous with the donor sharing one HLA Ag. In our study we focused on confirming, using an independent population, whether transplantation outcomes would be different when HLA mismatch direction was considered. We analyzed 1565 patients who received a single-unit UCBT for malignant disease. Median age was 15 years and 72% of patients were transplanted for leukemia. In multivariate analysis, using the 5/6 HLA-matched population as reference, one or two HLA mismatches in the GVH or HVG direction were not associated with non-relapse related mortality and survival. On the basis of our results, there is no evidence to support a change in the current practice for cord blood unit selection.

Published
2014

17. Acquired aplastic anaemia in children: Diagnostic-therapeutic recommendations

Author

Barone, A, Lucarelli, A, Onofrillo, D, Verzegnassi, F, Bonanomi, S, Longoni, D, Cesaro, S, Cugno, C, Zecca, M, Fioredda, F, Svahn, J, Dufour, C, Iori, Ap, Ladogana, S, Maruzzi, M, Locasciulli, A, Lanciotti, M, Macaluso, A, Mandaglio, R, Marra, N, Menna, G, Martire, B, Notarangelo, Ld, Palazzi, G, Pillon, M, Ramenghi, Ugo, Saracco, P, Russo, G, Timeus, F, Tucci, F, and Farruggia, P.

Subjects
Acquired aplastic anemia, Childhood
Published
2014

19. Optimal timing of allogeneic hematopoietic stem cell transplantation in patients with myelodysplastic syndrome

Author

Alessandrino, Ep, Porta, Mg, Malcovati, L, Jackson, Ch, Pascutto, C, Bacigalupo, A, Teresa van Lint, M, Falda, M, Bernardi, M, Onida, F, Guidi, S, Iori, Ap, Cerretti, R, Marenco, P, Pioltelli, P, Angelucci, E, Oneto, R, Ripamonti, F, Rambaldi, A, Bosi, A, Cazzola, M, Gruppo Italiano Trapianto di Midollo Osseo Levis, A, Bandini, G, Casini, M, Rossi, G, Baronciani, D, La Nasa, G, Milone, G, Mordini, N, Van Lint MT, Corradini, P, Milani, R, Morra, E, Lambertenghi Deliliers, G, Ciceri, F, Castagna, L, Narni, F, Selleri, C, Scime, R, Iannitto, E, Musso, M, Locatelli, F, Zecca, M, Martelli, F, Visani, G, Di Bartolomeo, P, Cavanna, L, Papineschi, Federico, Messina, G, Merli, F, Foa, R, Locasciulli, A, Majolino, I, Chiusolo, P, Leone, G, Arcese, W, Carella, Am, Cascavilla, N, Mazza, P, Bruno, B, Boccadoro, M, Fanin, R, Cerno, M, Bortolo, S, and Author information, Raimondi R.

Subjects
Oncology, Adolescent, Adult, Aged, Aged, 80 and over, Child, Cohort Studies, Female, Humans, Male, Markov Chains, Middle Aged, Myelodysplastic Syndromes, Risk, Survival Analysis, Time Factors, Transplantation, Homologous, Hematopoietic Stem Cell Transplantation, Hematology, Homologous, medicine.medical_specialty, Allogeneic transplantation, medicine.medical_treatment, Hematopoietic stem cell transplantation, Natural history of disease, Internal medicine, medicine, 80 and over, Survival analysis, Transplantation, business.industry, Myelodysplastic syndromes, Original Articles, medicine.disease, Settore MED/15, Surgery, allogeneic transplantation, International Prognostic Scoring System, Risk assessment, business
Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) represents the only curative treatment for patients with myelodysplastic syndrome (MDS), but involves non-negligible morbidity and mortality. Registry studies have shown that advanced disease stage at transplantation is associated with inferior overall survival. To define the optimal timing of allogeneic HSCT, we carried out a decision analysis by studying 660 patients who received best supportive care and 449 subjects who underwent transplantation. Risk assessment was based on both the International Prognostic Scoring System (IPSS) and the World Health Organization classification-based Prognostic Scoring System (WPSS). We used a continuous-time multistate Markov model to describe the natural history of disease and evaluate the effect of allogeneic HSCT on survival. This model estimated life expectancy from diagnosis according to treatment policy at different risk stages. Relative to supportive care, estimated life expectancy increased when transplantation was delayed from the initial stages until progression to intermediate-1 IPSS-risk or to intermediate WPSS-risk stage, and then decreased for higher risks. Modeling decision analysis on WPSS versus IPSS allowed better estimation of the optimal timing of transplantation. These observations indicate that allogeneic HSCT offers optimal survival benefits when the procedure is performed before MDS patients progress to advanced disease stages. Am. J. Hematol. 88:581–588, 2013. © 2013 Wiley Periodicals, Inc.

Published
2013

24. Outcome of allogeneic stem cell transplantation following reduced-intensity conditioninig regimen in patients with idiopathic myelofibrosis: the g.I.T.m.o. Experience

Author

Patriarca, Francesca, Bacigalupo, A, Sperotto, A, Isola, Miriam, Bruno, B, van Lint MT, Iori, Ap, Di Bartolomeo, P, Musso, M, Pioltelli, P, Visani, G, Iacopino, P, Fanin, Renato, and Bosi, A.

Subjects
surgical procedures, operative, hemic and lymphatic diseases, MEDITERRANEAN JOURNAL OF HEMATOLOGY AND INFECTIOUS DISEASES
Published
2010

25. Nonpermissive HLA-DPB1 disparity is a significant independent risk factor for mortality after unrelated hematopoietic stem cell transplantation

Author

Crocchiolo, R, Zino, E, Vago, L, Oneto, R, Bruno, B, Pollichieni, S, Sacchi, N, Sormani, Mp, Marcon, J, Lamparelli, T, Fanin, R, Garbarino, L, Miotti, V, Bandini, G, Bosi, A, Ciceri, F, Bacigalupo, A, Fleischhauer, K, Gruppo Italiano Trapianto di Midollo Osseo, Terapia Cellulare, Cellule Staminale Ematopoietiche e., Italian Bone Marrow Donor Registry, Scalari, P, Bontempelli, M, Prinoth, O, Carcassi, C, Marcenò, R, Porfirio, Rombolà, G, Lombardo, Ferrioli, G, Poli, F, Scalamogna, M, Mazzi, B, Rossi, F, Mascaretti, L, Albergoni, Salvaneschi, L, Martinetti, M, Valentini, Renzo, Nesci, S, Papola, F, Scatena, Mariotti, Perrone, Laurenti, Grammatico, P, Mariani, M, Favoino, B, Guizzardi, Pontiero, Leoni, P, Rambaldi, A, Casini, M, Angelucci, E, Baronciani, D, La Nasa, G, Milone, G, Guidi, S, Van Lint MT, Dini, G, Corradini, P, Milani, R, Morra, E, Marenco, P, Lambretenghi Deliliers, G, Onida, F, Marcatti, M, Castagna, L, Pioltelli, P, Selleri, C, Zanesco, L, Scimè, R, Musso, M, Alessandrino, Ep, Locatelli, F, Visani, G, Di Bartolomeo, P, Papineschi, Federico, Favre, C, Iori, Ap, Foà, R, Locasciulli, A, Majolino, I, Majolino, P, Leone, G, Arcese, W, Cerretti, R, Carella, Am, Cascavilla, N, Lauria, F, Mazza, P, Cerno, M, and Benedetti, F.

Published
2009

26. Cyclosporin A response and dependence in children with acquired aplastic anaemia: a multicentre retrospective study with long-term observation follow-up

Author

Saracco, P, Quarello, Paola, Iori, Ap, Zecca, M, Longoni, D, Svahn, J, Varotto, S, Del Vecchio GC, Dufour, C, Ramenghi, Ugo, Bacigalupo, A, Locasciulli, A, and Bone Marrow Failure Study Group of the AIEOP

Subjects
Male, therapy, Adolescent, Anemia, Aplastic, Infant, aplastic anaemia, children, Drug Administration Schedule, Treatment Outcome, Recurrence, Child, Preschool, Granulocyte Colony-Stimulating Factor, Cyclosporine, Disease Progression, Drug Evaluation, Humans, Drug Therapy, Combination, Female, Child, Epidemiologic Methods, Immunosuppressive Agents
Abstract

Immunosuppressive therapy (IST) with antithymocyte globulin and cyclosporin A (CyA) is the standard treatment for children with acquired aplastic anaemia (AAA) lacking a matched donor. Survival rates of more than 80% at 5 years are achieved, but the response is drug-dependent in 15-25% of cases. This study, of 42 consecutive children with AAA treated with IST, assessed the incidence of CyA-dependence, CyA and granulocyte colony-stimulating factor (G-CSF) tapering schedules and the impact of drug accumulation on progression to myelodysplasia/acute myeloid leukaemia (MDS/AML). Overall survival was 83% at 10 years. CyA-dependence without a predictive marker was observed in 18% of responders. Probability of discontinuing CyA was 60.5% at 10 years; a slow CyA tapering schedule was performed in 84% of patients; the cumulative incidence of relapse was 16% at 10 years. Relapse risk was significantly associated with rapid CyA discontinuation: 60% compared to 7.6% in the slow tapering group (P = 0.001). Cumulative incidence of MDS/AML was 8% at 10 years, with a significant correlation with both G-CSF cumulative dose and second IST. This long-term follow-up of children with AAA shows that IST with a slow CyA tapering course is an effective treatment with a low-relapse rate in these cases.

Published
2008

27. WHO classification and WPSS predict posttransplantation outcome in patients with myelodysplastic syndrome : a study from the Gruppo Italiano Trapianto di Midollo Osseo(GITMO)

Author

Alessandrino, Ep, Della Porta MG, Bacigalupo, A, Van Lint MT, Falda, M, Onida, F, Bernardi, M, Iori, Ap, Rambaldi, A, Cerretti, R, Marenco, P, Pioltelli, P, Malcovati, L, Pascutto, C, Oneto, R, Fanin, R, Bosi, A, Collaboratori Levis, A, Bandini, G, Casini, M, Rossi, G, Angelucci, E, Baronciani, D, La Nasa, G, Milone, G, Mordini, N, Guidi, S, Corradini, P, Milani, R, Morra, E, Lambretenghi Deliliers, G, Ciceri, F, Castagna, L, Narni, F, Selleri, C, Scimè, R, Iannitto, E, Musso, M, Locatelli, F, Martelli, F, Visani, G, Di Bartolomeo, P, Cavanna, L, Papineschi, F, Messina, G, Gugliotta, L, Foà, R, Locasciulli, A, Majolino, I, Chiusolo, P, Leone, G, Arcese, W, Carella, Am, Cascavilla, N, Mazza, P, Bruno, Benedetto, Boccadoro, Mario, Cerno, M, and Raimondi, R.

Subjects
Adult, Male, medicine.medical_specialty, Blood transfusion, Adolescent, medicine.medical_treatment, Immunology, World Health Organization, Biochemistry, Gastroenterology, Humans, Myelodysplastic Syndromes, Prognosis, Aged, Recurrence, Survival Rate, Classification, Blood Transfusion, Hematopoietic Stem Cell Transplantation, Middle Aged, Female, Refractory, hemic and lymphatic diseases, Internal medicine, medicine, Survival rate, Acute leukemia, Hematology, business.industry, Cancer, Cell Biology, medicine.disease, Surgery, Transplantation, Who classification, business, Settore MED/15 - Malattie del Sangue
Abstract

We evaluated the impact of World Health Organization (WHO) classification and WHO classification–based Prognostic Scoring System (WPSS) on the outcome of patients with myelodysplastic syndrome (MDS) who underwent allogeneic stem cell transplantation (allo-SCT) between 1990 and 2006. Five-year overall survival (OS) was 80% in refractory anemias, 57% in refractory cytopenias, 51% in refractory anemia with excess blasts 1 (RAEB-1), 28% in RAEB-2, and 25% in acute leukemia from MDS (P = .001). Five-year probability of relapse was 9%, 22%, 24%, 56%, and 53%, respectively (P < .001). Five-year transplant-related mortality (TRM) was 14%, 39%, 38%, 34%, and 44%, respectively (P = .24). In multivariate analysis, WHO classification showed a significant effect on OS (P = .017) and probability of relapse (P = .01); transfusion dependency was associated with a reduced OS (P = .01) and increased TRM (P = .037), whereas WPSS showed a prognostic significance on both OS (P = .001) and probability of relapse (P < .001). In patients without excess blasts, multilineage dysplasia and transfusion dependency affected OS (P = .001 and P = .009, respectively), and were associated with an increased TRM (P = .013 and P = .031, respectively). In these patients, WPSS identified 2 groups with different OS and TRM. These data suggest that WHO classification and WPSS have a relevant prognostic value in posttransplantation outcome of MDS patients.

Published
2008

29. Allogeneic hematopoietic stem cell transplantation in myelofibrosis: the 20-year experience of the Gruppo Italiano Trapianto di Midollo Osseo (GITMO)

Author

Patriarca, F, Bacigalupo, A, Sperotto, A, Isola, M, Soldano, F, Bruno, B, van Lint MT, Iori, Ap, Santarone, S, Porretto, F, Pioltelli, P, Visani, G, Iacopino, P, Fanin, R, Bosi, A, Milone, G, Montanari, M, La Nasa, G, Dessalvi, P, Bonifazi, F., Servida, P, Lambertenghi, G, Corradini, P, Falda, M, Mazza, P, Benedetti, F, Raimondi, R, Selleri, Carmine, Locasciulli, A, Alessandrino, P, Narni, F, and Papineschi, F.

Published
2008

32. WHO classification and WPSS predict posttransplantation outcome in patients with myelodysplastic syndrome: a study from the Gruppo Italiano Trapianto di Midollo Osseo (GITMO)

Author

Alessandrino, Ep, Della Porta MG, Bacigalupo, A, Van Lint MT, Falda, M, Onida, F, Bernardi, M, Iori, Ap, Rambaldi, A, Cerretti, R, Marenco, P, Pioltelli, P, Malcovati, L, Pascutto, C, Oneto, R, Fanin, R, Bosi, A, Gruppo Italiano Trapianto di Midollo Osseo, Levis, A, Bandini, G, Casini, M, Rossi, G, Angelucci, E, Baronciani, D, La Nasa, G, Milone, G, Mordini, N, Guidi, S, Corradini, P, Milani, R, Morra, E, Lambretenghi Deliliers, G, Ciceri, F, Castagna, L, Narni, F, Selleri, C, Scimè, R, Iannitto, E, Musso, M, Locatelli, F, Martelli, F, Visani, G, Di Bartolomeo, P, Cavanna, L, Papineschi, Federico, Messina, G, Gugliotta, L, Foà, R, Locasciulli, A, Majolino, I, Chiusolo, P, Leone, G, Arcese, W, Carella, Am, Cascavilla, N, Mazza, P, Bruno, B, Boccadoro, M, Cerno, M, and Raimondi, R.

Published
2008

42. Outcome of children with all who started a search for a matched donor

Author

Manzitti, C, Valsecchi, Mg, Micalizzi, C, Locatelli, F, Busca, A, Pession, A, Prete, A, Giorgiani, G, Lanino, E, Balduzzi, A, Cesaro, Simone, Iori, Ap, Galimberti, S, Garbarino, L, Mazzolari, E, Rabusin, M, and Dini, G.

Subjects
pediatric, pediatric, allogenic stem cell transplant, donor search, donor search, allogenic stem cell transplant
Published
1999

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